DIA 2024 Global Annual Meeting: Full Schedule

8:00am PDT

#100: Opening Plenary and Keynote Address

Component Type: Forum

Join our opening plenary session, Innovation: The Possibilities, The Boundaries: Charting New Horizons, led by Tom and Emily Whitehead, who will give the incredible journey of Emily Whitehead, the pioneering pediatric patient who defied the odds and became the first in the world to undergo CAR T-cell therapy. Tom and Emily will share their invaluable insights into the transformative power of CAR-T therapy, offering a unique perspective that promises to captivate and inspire. Dean Kamen will discuss the Future of Regenerative Therapies, How Public-Private Partnerships Can Impact Advancement. Following your keynotes, we will have a panel discussion. Attendees can expect a lively and engaging discussion, with a diverse range of perspectives and insights from panelists who are leaders and at the forefront of our industry; including panelists from the Gates Foundation, regulatory agencies, and the patient perspective.

Chair

Marwan Fathallah, MBA, MSc

Speaker

Developing Cell Therapies for Cancer Patients
Carsten Linnermann, PhD

Keynote Speaker: Insights into the Transformative Power of CAR-T Therapy
Tom Whitehead

Keynote Speaker: Insights into the Transformative Power of CAR-T Therapy
Emily Whitehead

Keynote Speaker: Future of Regenerative Therapies - How Public-Private Partnerships Can Impact Advancement
Dean Kamen

Plenary Panel: Innovation - The Possibilities, the Boundaries - Charting New Horizons
David Mukanga, PhD, MPH

Panelist: Innovation
Dean Kamen

Panelist: EMA Perspective
Emer Cooke, MBA, MSc

Panelist: FDA Perspective
Peter W. Marks, MD, PhD

Panelist: Patient Perspective
Stacy Hurt, MBA, MHA

Speakers

Marwan Fathallah

President & Chief Executive Officer, DIA, United States

Prior to joining DIA in 2023, Fathallah was the Operating Officer and EVP at Ortho Clinical Diagnostics, accountable for Operations, Post Market Product Science and Engineering, Regulatory, Quality, Medical, Clinical, Scientific Affairs, and Process Excellence. Before joining Ortho... Read More →

Dean Kamen

Founder, DEKA Research & Development Corp., United States

Serial inventor and the founder of the ARMI-Advance Regeneratve Manufacturing Institute

Peter Marks

Director, Center for Biologics Evaluation and Research, FDA, United States

Peter Marks received his graduate degree in cell and molecular biology and his medical degree at New York University and completed Internal Medicine residency and Hematology/Medical Oncology training at Brigham and Women’s Hospital in Boston. He has worked in academic settings teaching... Read More →

Emily Whitehead

Co-founder, Emily Whitehead Foundation, United States

Emily Whitehead was diagnosed with acute lymphoblastic leukemia (ALL) in May 2010 when she was five years old. Typically, children diagnosed with this type of leukemia have an 85% to 90% chance of being cured; however, she relapsed twice. The leukemia became resistant to treatment... Read More →

Tom Whitehead

Co-founder and President, Emily Whitehead Foundation, United States

Emily’s parents, Tom and Kari, were not willing to give up. They heard about an experimental treatment, called CAR T-cell therapy, that was available as a Phase I clinical trial at Children’s Hospital of Philadelphia (CHOP). In April 2012, they enrolled Emily in the clinical trial... Read More →

Stacy Hurt, MBA, MHA

Chief Patient Officer, Parexel , United States

Stacy Hurt, MHA, MBA is an award-winning 25 year pharma executive who bridges the industry/consumer gap as a stage IV cancer survivor and 24/7 caregiver to her son with a rare disease. Her unique professional and personal experience position her as an esteemed patient advocate as... Read More →

Emer Cooke

Chair, ICMRA; Executive Director, European Medicines Agency, Netherlands

Ms Cooke is Executive Director of the EMA and Chair of ICMRA. Starting her mandate as ED in June 2020 amid a public health crisis of unprecedented scale she announced “My number one priority will be to drive forward EMA’s response to the pandemic and the work already ongoing to... Read More →

Carsten Linnermann

Chief Executive Officer, Neogene Therapeutics, AstraZeneca Group, Netherlands

Co-founder and CEO of Neogene Therapeutics, AstraZeneca Group will speak about his experience in developing cell therapies for cancer patients and the importance of collaboration to transform innovative science into new medicines for patients.

Monday June 17, 2024 8:00am - 10:00am PDT
Ballroom San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

00: Plenary, Forum | 09: Regulatory, Forum | 06: TranslationalDelivery Tech-PrecMedicine, Forum

Featured Topics Artificial Intelligence (AI), Cell and Gene Therapy, Patient Focused, Regulator Perspectives
Feature Topics Artificial Intelligence (AI),Cell and Gene Therapy,Patient Focused,Regulator Perspectives
Tags Forum

11:00am PDT

#105: The Intersection of Science, Ethics, and Participant Collaboration in Pediatric Rare Disease Product Development

Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-519-L04-P; CME 1.00; RN 1.00

Experts in pediatric rare disease product development, pediatric ethics and patients/advocates discuss designing studies to best benefit the patient, and how patients/caregivers assess risks and benefits in early phase trials.

Learning Objectives

Discuss the ethical and scientific considerations for designing studies for rare diseases that impact children, including the level of evidence needed to initiate early phase trials; Discuss how patients and caregivers weigh the risks of early-phase studies against the benefits of a potential treatment for their rare disease, especially when there are limited to no other options.

Chair

Donna Snyder, MD

Speaker

Ethical Considerations for Including Children in Rare Disease Trials
Donna Snyder, MD

Update from ORPURM/DRDMG
Mehul Desai, MD

Patient Perspective
Mark Dant

Patient Perspective
Ryan Dant

Speakers

Donna Snyder

Executive Physician, WCG, United States

Donna Snyder, MD, MBE is the Executive Physician of WCG where she contributes subject matter expertise and operational guidance across WCG to ensure that research will be conducted to the highest ethical standards. Prior to WCG, Dr. Snyder served as the leader and Senior Pediatric... Read More →

Mark Dant

Volunteer Executive Director, The Ryan Foundation, The Ryan Foundation for Rare Disease Research, United States

Mark is the Founder and Volunteer Executive Director of the Ryan Foundation and is the former Board Chair of the of the EveryLife Foundation for Rare Diseases. Mark and his family founded the Ryan Foundation in 1992 shortly after their only child Ryan was diagnosed with MPS I. Since... Read More →

Ryan Dant

Patient Advocate, The Ryan Foundation for Rare Disease Research, United States

Ryan Dant is a long-time patient advocate diagnosed at the age of three with Mucopolysaccharidosis type 1 (MPS 1), a rare lysosomal storage disorder. When doctor’s predicted Ryan’s life to be cut short due to the disease, Ryan’s parents, Mark and Jeanne, spearheaded the funding... Read More →

Mehul Desai

Medical Reviewer, ORDPURM/DRDMG, OND, CDER, FDA, United States

Mehul Desai M.D. is a medical officer in the Division of Rare Diseases and Medical Genetics (DRDMG), Office of New Drugs (OND), Center for Drug Evaluation Research (CDER), with the US Food and Drug Administration (FDA). Dr. Desai has clinical development experience in both the pharmaceutical... Read More →

Monday June 17, 2024 11:00am - 12:00pm PDT
Room 17AB San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

05: Patient-Focused Drug Dev, Session | 09: Regulatory, Session | 02: ClinTrials -ClinOps, Session

Format Session
Level Intermediate
Featured Topics Ethics, Rare Disease, Special Populations
Level Intermediate
Feature Topics Ethics,Rare Disease,Special Populations
Credit Type ACPE, CME, RN
Tags Session

11:00am PDT

#110: Modernizing CDER’s New Drugs Review Program (NDRP): A Progress Update

Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-524-L04-P; CME 1.00; RN 1.00

In 2017, CDER began modernizing the New Drugs Regulatory Program. This session shares progress updates and offers dialogue to enhance the program's key areas.

Learning Objectives

Describe the impetus for modernizing CDER’s Drug Regulatory Program (NDRP); Identify the NDRP strategic objectives and their associated initiatives; Discuss the progress and outcomes of two NDRP workstreams: Advisory Committees and Investigational New Drugs

Chair

Yoni Tyberg, MHA

Speaker

Panelist
Kevin Bugin, PhD, MS, RAC

Panelist
Meghana Chalasani, MHA

Panelist
Michael Monteleone, MS, RAC

Panelist
Andrew Emmett, MPH

Speakers

Kevin Bugin

Deputy Director of Operations, Office of New Drugs, CDER, FDA, United States

Dr. Kevin Bugin is the Deputy Director of Operations in the Office of New Drugs (OND) in FDA’s Center for Drug Evaluation and Research (CDER). In addition, he leads the CDER Center for Clinical Trial Innovation (C3TI) which aims to promote the implementation of innovative clinical... Read More →

Meghana Chalasani

Associate Director for Clinical Trial Innovation, Office of New Drugs, CDER, FDA, United States

Meghana Chalasani is the Associate Director for Clinical Trial Innovation in the Office of New Drugs (OND) in U.S. FDA’s Center for Drug Evaluation and Research (CDER). She is the program manager for the CDER Center for Clinical Trial Innovation and also co-leads the New Drugs Regulatory... Read More →

Andrew Emmett

FDA Liaison | US Regulatory Policy and Global Intelligence, Pfizer Inc, United States

Andrew Emmett is Pfizer's FDA Liaison and Executive Director for U.S. Regulatory Policy & Global Intelligence. In his role, Andrew develops and advocates for Pfizer policy positions to enhance the regulatory environment for biomedical innovation. He leads key initiatives on real-world... Read More →

Michael Monteleone

Associate Director for Labeling, OND, CDER, FDA, United States

Mr. Michael Monteleone is currently an Associate Director for Labeling in the Office of New Drugs at CDER, FDA. Michael has also held positions in Regulatory Affairs and Project Management throughout his 15 year tenure at the FDA.

Yoni Tyberg

Associate Director of Special Programs, Office of New Drugs, CDER, FDA, United States

Yoni Tyberg is the Associate Director of Special Program Staff (SPS) at the Office of New Drugs within CDER. In this role, Yoni supports the strategic planning, project development and execution of the New Drug Regulatory Program Modernization efforts across OND and CDER. He had also... Read More →

Monday June 17, 2024 11:00am - 12:00pm PDT
Room 6F San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

09: Regulatory, Forum

Format Forum
Level Intermediate
Featured Topics Regulator Perspectives
Level Intermediate
Feature Topics Regulator Perspectives
Credit Type ACPE, CME, RN
Tags Forum

11:00am PDT

#111: China Town Hall

Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-525-L04-P; CME 1.00; RN 1.00

In this forum, speakers from the National Medical Products Administration (NMPA) and the Center for Drug Evaluation (CDE) will present and discuss the latest information on regulation, regulatory science, and drug review and approval. Speakers will also discuss recent development in clinical research in China. Attendees will have an opportunity to discuss the issues related to drug regulation with NMPA officials.

Learning Objectives

Summarize the latest information on the National Medical Products Administration’s activities in drug regulation; Describe the recent update on the progress in drug review, approval and clinical research in China; Identify challenges in drug development and regulation in China.

Chair

Ling Su, PhD

Speaker

Opening Remarks and Introductions
Lili Cao, MS

Initiatives to Bring More New Drugs to Patients, and Faster
Zhimin Yang, MD

Recent Developments in Regulatory Science and Regulation
Jiangyong Yu

Advancing Clinical Research in China: A Clinical Oncologist’s Perspective
Shun Lu

Speakers

Ling Su

Research Fellow, Shenyang Pharmaceutical University, Yeehong Business School, China

Dr. Ling Su is a Research Fellow in Shengyang Pharmaceutical University Yeehong Business School and a Venture Partner in Lilly Asia Ventures (LAV). He has 30 years of experience in drug regulatory and development. He had worked in various R&D management positions in the pharma industry... Read More →

Lili Cao

Deputy Director, China Center for Food and Drug International Exchange, National Medical Products, China

Lili Cao is the Deputy Director-General, China Center for Food & Drug Int’l Exchange (CCFDIE), National Medical Products Administration (NMPA). She received her bachelor degree at Nankai University, and master degree at London School of Economics and Political Science, majoring... Read More →

Shun Lu

Director, Center for Clinical Medicine of Lung Cancer, Shanghai Chest Hospital,, Shanghai Jiaotong University, China

Shun Lu, MD, PhD, is a Director of the Oncology Department and Professor at Shanghai Chest Hospital. Dr. Lu serves on the International Affairs Committee of the American Society of Clinical Oncology, and Multidisciplinary Cancer Management Courses Working Group. He is also a member... Read More →

Zhimin Yang

Deputy Director, Center for Drug Evaluation, National Medical Products Administration (NMPA), China

Dr. Yang is theDeputy Director-General ,Center for Drug Evaluation of the National Medical Products Administration. Graduated from Peking University Medical Center, majoring in clinical medicine. She used to be a known pediatrician working at Peking University People's Hospital. In... Read More →

Jiangyong Yu

Director, Department of Drug Registration, National Medical Products Administration (NMPA), China

Dr. Yu Jiangyong, Doctor of Science, Chief Pharmacist. Currently, he is the consultant and director of division of TCMs and Ethno-Medicines, Department of Drug Registration (Department of TCMs and Ethno-Medicines Regulation) of China's NMPA.

Monday June 17, 2024 11:00am - 12:00pm PDT
Room 6D San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

09: Regulatory, Forum

Format Forum
Level Intermediate
Featured Topics Regulator Perspectives
Level Intermediate
Feature Topics Regulator Perspectives
Credit Type ACPE, CME, RN
Tags Forum

2:15pm PDT

#139: Comparative Perspectives on Regulating AI in Drug Development: US Versus EU

Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-539-L04-P; CME 1.00; RN 1.00

Explore US and EU AI drug regulation with experts from regulator, industry, and academic perspectives. Learn about principles, data privacy, transparency, and real-world examples. Gain insights into vital regulatory nuances.

Learning Objectives

Describe the regulatory frameworks in the US and EU; Discuss the practical necessity of regulation in various AI-driven drug development scenarios.

Chair

Jayne Ware, MPH, MSc

Speaker

EMA Update
Steffen Thirstrup, MD, PhD

FDA Update
Tala Fakhouri, PhD, MPH

Industry Perspective
Rose Purcell, JD, MA

Speakers

Jayne Ware

Senior Director, Global Regulatory Policy, Merck Sharp & Dohme LLC , United States

Jayne Ware is a Director for Global Regulatory Policy at Merck. She joined Merck in 2012 after 15 years at the US FDA. Jayne is responsible for coordinating regulatory policy intelligence and advocacy activities for key topic areas at Merck such as digital health issues, safety/pharmacovigilance... Read More →

Tala Fakhouri

Associate Director for Policy Analysis, Office of Medical Policy, CDER, FDA, United States

Rose Purcell

Director, Global Regulatory Policy and Innovation, Takeda, United States

Steffen Thirstrup

Chief Medical Officer, European Medicines Agency, Netherlands

Steffen Thirstrup is a medical doctor and board certified specialist in clinical pharmacology and therapeutics. He holds a PhD in pharmacology and has a long background in clinical internal medicine. He is an adjunct professor at the Faculty of Health Sciences, University of Copenhagen... Read More →

Monday June 17, 2024 2:15pm - 3:15pm PDT
Room 6C San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

09: Regulatory, Session

Format Session
Level Intermediate
Featured Topics Artificial Intelligence (AI), Regulator Perspectives
Level Intermediate
Feature Topics Artificial Intelligence (AI),Regulator Perspectives
Credit Type ACPE, CME, RN
Tags Session

2:15pm PDT

#140: The State of Real-World Evidence for Regulatory Decision-Making: Views from FDA, EMA, and PMDA

Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-540-L04-P; CME 1.00; RN 1.00

This session will be a panel discussion with senior leaders from FDA, EMA, and PMDA who are closely involved with real-world evidence (RWE) initiatives within their respective agencies. An open Q&A with leaders in RWE from FDA, EMA, PMDA, will be included.

Learning Objectives

Describe current perspectives of regulators from several agencies on the use of real-world evidence (RWE) for regulatory decision-making; Discuss from examples of successful use of RWE in regulatory decision-making and how they can be applied; Identify current initiatives and programs regarding RWE in each regulatory agency.

Chair

Charles Lee, MBA, MS

Speaker

FDA Update
Motiur Rahman, PhD, MPharm, MS

EMA Update
Sabine Haubenreisser, PhD, MSc

PMDA Update
Yuki Ando, PhD

Speakers

Charles Lee

Executive Regulatory Science Director, AstraZeneca, United States

Charles is currently Executive Regulatory Science Director at Astrazeneca. He oversees Global Regulatory science and strategy for therapeutic products in the renal, cardiovascular, diabetes, and NASH disease areas. Prior to this role, Charles was a Product Development Team Leader... Read More →

Yuki Ando

Principal Senior Scientist for Biostatistics, Pharmaceuticals and Medical Devices Agency (PMDA), Japan

Dr. Yuki Ando is a Principal Senior Scientist for Biostatistics of the Pharmaceuticals and Medical Devices Agency (PMDA), Japan. She has over 20 years' experience as Biostatistics Reviewer, and currently she is responsible for the biostatistics review and consultation in the new drug... Read More →

Sabine Haubenreisser

Principal Scientific Administrator, Stakeholders and Communication Division, European Medicines Agency, Netherlands

Sabine Haubenreisser is a pharmacologist with special interest in scientific cooperation and regulatory convergence to the benefit of public health, through engagement with external stakeholders, the European network of experts and international partners and regulators. She has held... Read More →

Motiur Rahman

Senior Epidemiologist, Real World Evidence Analytics, OMP, CDER, FDA, United States

Motiur Rahman is a Senior Epidemiologist at Real-World Evidence (RWE) Analytics in the Office of Medical Policy, Center for Drug Evaluation and Research (CDER), US Food and Drug Administration (FDA). His responsibilities include developing guidance, improving internal Agency processes... Read More →

Monday June 17, 2024 2:15pm - 3:15pm PDT
Room 6F San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

09: Regulatory, Forum

Format Forum
Level Intermediate
Featured Topics Regulator Perspectives, Real-World Data (RWD)- Real-World Evidence (RWE)
Level Intermediate
Feature Topics Regulator Perspectives,Real-World Data (RWD)- Real-World Evidence (RWE)
Credit Type ACPE, CME, RN
Tags Forum

2:15pm PDT

#137: Options to Consider When Balancing Risk, Timelines, Cost, and Patient Centricity While Meeting Project Optimus Guidelines

Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-537-L04-P; CME 1.00; PDU 1.00 PMI 2166YE3LVS; RN 1.00

Review principles of FDA's Project Optimus and discuss the impact on oncology clinical development strategies. Scenario analysis with risk management is needed to balance project cost, timelines, patient centricity and health equity approaches.

Learning Objectives

Describe the essential points of FDA's Project Optimus Guidance and how it may impact drug development; Discuss how project managers and oncology development teams gain insight into options to consider in order to balance cost, timelines, and risk during dose finding/dose optimization in new molecular entities and new indications.

Chair

Nancy Linda Styple

Speaker

Patient Update
Julia Maues, MA

Industry Update
Gary Wilkinson, PhD

Project Management Considerations for Project Optimus
Nancy Linda Styple

Speakers

Nancy Styple

Project Management Leader, Bayer AG, United States

Nancy has been excited to work in Program Management for over 20 years and is a strong advocate for the work that Project Managers and Project Leaders deliver along the value chain in drug development. Nancy starting as a bench research scientist in biotech and has experience in multiple... Read More →

Julia Maues

Patient Advocate, Patient-Centered Dosing Initiative, United States

Julia Maues spearheads the Patient-Centered Dosing Initiative (PCDI), challenging conventional cancer drug dosing practices and prioritizing patient well-being alongside efficacy. She is co-founder of GRASP (Guiding Researchers and Advocates to Scientific Partnerships), an organization... Read More →

Gary Wilkinson

Director of Clinical Pharmacology, Ryvu Therapeutics, Poland

Monday June 17, 2024 2:15pm - 3:15pm PDT
Room 1AB San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

09: Regulatory, Session | 05: Patient-Focused Drug Dev, Session | 07: ProjectManagement-StrategicPlanning, Session

Format Session
Level Intermediate
Featured Topics Risk Management
Level Intermediate
Feature Topics Risk Management
Credit Type ACPE, CME, PMI, RN
Tags Session

2:15pm PDT

#136: In Vitro Diagnostic/Companion Diagnostics Developments in the US and Impact on Global Programs

Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-536-L04-P; CME 1.00; RN 1.00

This session will present on the shifting regulatory landscape on diagnostics, including laboratory developed tests. Discussions will focus on a new proposed rule, the FDA’s oncology diagnostics pilot program, and global development implications.

Learning Objectives

Explain recent developments in the In Vitro Diagnostic/Companion Diagnostics (IVD/CDx) regulatory landscape in the US; Discuss regulatory implications of the proposed rule on LDTs; Evaluate the impact of FDA IVD initiatives on global drug development programs

Chair

Stephanie Choi, PhD

Speaker

Insights into FDA's Proposed Rule and LDT Initiatives
Cortez McBerry, PhD

New and Anticipated Trends in IVD and LDT Regulation
Laura DiAngelo, MPH

Impact of Changing IVD/LDT Landscape on Global Drug Development Programs
Megan Doyle, JD, MPH

Speakers

Stephanie Choi

Director, US Global Regulatory and Scientific Policy (GRASP), EMD Serono, United States

Stephanie Choi, Ph.D. is currently a Director in Global Regulatory and Scientific Policy at EMD Serono. Her current interests relate to regulatory policy in oncology, CDx, and RWE. Prior to EMD Serono, she worked as a regulatory strategist at Jazz Pharmaceuticals supporting early... Read More →

Laura DiAngelo

Research Director, AgencyIQ by POLITICO, United States

Laura DiAngelo is the Director of Regulatory Intelligence for POLITICO's AgencyIQ. She focuses primarily on regulatory intelligence issues impacting the medical device, diagnostics and digital health industries.

Megan Doyle

Associate Vice President, Assistant General Counsel - Diagnostics, Eli Lilly & Company, United States

Megan Doyle is an attorney and policy professional with 15 years of experience in the regulation of drugs, medical devices, combination products, and in vitro diagnostics. She received her law degree from Georgetown University Law Center and her master’s degree in public health... Read More →

Cortez McBerry

Partner, NDA Partners, United States

Monday June 17, 2024 2:15pm - 3:15pm PDT
Room 15AB San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

09: Regulatory, Session | 06: TranslationalDelivery Tech-PrecMedicine, Session

Format Session
Level Intermediate
Level Intermediate
Credit Type ACPE, CME, RN
Tags Session

3:30pm PDT

#153: Designing your Regulatory Intelligence Framework: Powered by Artificial Intelligence and Critical Thinking

Component Type: Session
Level: Advanced
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-552-L04-P; CME 1.00; RN 1.00

Regulatory intelligence is the key to formulating regulatory strategy. We will share the application of AI and critical thinking in developing science and risk-based strategies for the development and lifecycle management of medicinal product.

Learning Objectives

Apply skills learned in this session to set up regulatory intelligence frameworks for their team and/or company; Develop critical thinking skills that are imperative to the interpretation and proper utilization of intelligence data; Identify how to setup and manage tools and processes for surveilling and monitoring regulatory changes.

Chair

Ana Sengupta, MBA, MS, RAC

Speaker

Designing Regulatory Intelligence Framework: Powered by Critical Thinking
Ana Sengupta, MBA, MS, RAC

Designing Regulatory Intelligence Framework: Powered by Artificial Intelligence
Susan Carino, MBA, MS, PMP, RAC

Designing Regulatory Intelligence Framework: Powered by Artificial Intelligence
Aditi Khurana, MBA, PMP, RAC

Speakers

Ana Sengupta

Associate Director Regulatory Affairs CMC, Mirum Pharmaceuticals, Inc., United States

Ana Sengupta is the Associate Director of Regulatory Affairs at Mirum Pharmaceuticals, Inc. She has more than 24 years of pharmaceutical industry experience and 14 years of direct experience in regulatory affairs of drugs and biologics. Before working at Mirum Pharmaceuticals, she... Read More →

Susan Carino

Director, Program Management, Mammoth Biosciences, United States

Susan Carino is a Director of Program Management with Mammoth Biosciences. She has over 30 years of industry experience in R&D across numerous therapeutic areas. She has led diverse cross-functional program teams engaged in complex product development of pharmaceuticals, biologics... Read More →

Aditi Khurana

Director, Project Management, Summit Therapeutics, Inc., United States

Aditi Khurana, is the Director of Project Management at Asher Biotherapeutics. She is a Project Management Professional (PMP) and is certified in Global Regulatory Affairs (RAC). She holds a Bachelors in Neuroscience from University of California, Los Angeles (UCLA) and a Masters... Read More →

Monday June 17, 2024 3:30pm - 4:30pm PDT
Room 15AB San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

09: Regulatory, Session

Format Session
Level Advanced
Featured Topics Artificial Intelligence (AI)
Level Advanced
Feature Topics Artificial Intelligence (AI)
Credit Type ACPE, CME, RN
Tags Session

3:30pm PDT

#154: Impact of Accelerated Pathways on Patients in Five Countries/Regions

Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-553-L04-P; CME 1.00; RN 1.00

Several global regulatory authorities have similar expedited pathways in which a regulatory approval can be granted that would fulfill an unmet medical need based on preliminary/early data from clinical studies. This session will use a recent Oncology case study comparing the different outcomes resulting from using these five different systems aiming to accelerate development and speed up availability of new treatments to patients. This will be followed by a panel with senior regulators and a patient representative to discuss the regulatory perspective and experience with accelerated approvals. The objective is to identify potential opportunities that could help to achieve more collaboration and equity of access for patients across multiple countries/regions.

Learning Objectives

Recognize the differences of accelerated pathways in US, EU, Canada, Japan and Australia; Discuss the impact of these pathways on patient access and treatment outcomes; Identify opportunities to increase global collaboration and international patient equity.

Chair

Sunita Zalani, PhD

Speaker

EMA Update
Francesco Pignatti, MD

Health Canada Update
Isabelle Rousse, MSc

PMDA Update
Yoko Aoi, PhD

FDA Update
R. Angelo De Claro, MD

Industry Update
Kathleen Winson, MPH, MS

Speakers

Yoko Aoi

Deputy Review Director, Office of New Drug V, Pharmaceuticals and Medical Devices Agency (PMDA), Japan

Dr. Yoko Aoi is a deputy review director of Office of New Drug V responsible for review and scientific advice on oncology drugs. After joining PMDA, Dr. Aoi was involved in review and scientific advice on new drugs for rheumatoid arthritis, asthma, COPD etc. (2011-2016) and planning... Read More →

R. Angelo De Claro

Associate Director for Global Clinical Sciences, Oncology Center of Excellence, FDA, United States

Dr. de Claro is the Division Director for Division of Hematologic Malignancies I with Office of Oncologic Diseases. He provides leadership and scientific direction to staff engaged in the review and evaluation of applications for investigational new drugs and drug approvals. Dr. de... Read More →

Francesco Pignatti

Scientific Adviser for Oncology, European Medicines Agency, Netherlands

Francesco Pignatti graduated as Medical Doctor at the University of Rome La Sapienza, Italy. In 1995 he became Research Fellow at the EORTC Data Center in Brussels, Belgium In 1997 he obtained a Master of Science degree in Biostatistics from the University of Limburg, Belgium. In... Read More →

Isabelle Rousse

Associate Director, Bureau of Metabolism, Oncology, Reproductive Sciences, PDD, Health Canada, Canada

Isabelle Rousse joined Health Canada in 2006. She holds a Bachelor of Science in Biomedical Sciences and a Master of Science in Physiology from University of Montreal. At Health Canada, she has been a Scientific Evaluator in the Marketed Pharmaceuticals Bureau (MPB) within the Marketed... Read More →

Kathleen Winson

Executive Group Director, Product Development Regulatory, Genentech, A Member of the Roche Group, United States

Kathleen Winson is an Executive Group Director in Regulatory at Genentech/Roche, where she oversees regulatory strategy for the Oncology Lung and GU programs. She has been involved in clinical research for over 24 years accumulating a broad breadth of experience in pharmaceutical... Read More →

Sunita Zalani

Vice President, Regulatory Affairs, Oncology, Merck & Co., Inc., United States

Sunita Zalani, PhD is the Vice President and Therapeutic Area Head for Oncology and In-vitro diagnostics in Global Regulatory Affairs and Clinical Safety at Merck. In her current role, she has responsibilities for formulation and execution of global regulatory strategy for the oncology... Read More →

Monday June 17, 2024 3:30pm - 4:30pm PDT
Room 11AB San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

09: Regulatory, Session

Format Session
Level Intermediate
Featured Topics Patient Focused, Patient Focused
Level Intermediate
Feature Topics Patient Focused,Patient Focused
Credit Type ACPE, CME, RN
Tags Session

3:30pm PDT

#155: Health Canada Town Hall

Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-554-L04-P; CME 1.00; RN 1.00

In this session, leaders from Health Canada will provide an update on regulatory priorities for Canada. The audience will be invited to submit questions of general interest.

Learning Objectives

Describe Health Canada’s regulatory initiatives and strategic priorities; Identify opportunities for engagement with the Canadian regulator.

Chair

Alysha Croker, PhD

Speaker

Panelist
Sophie Sommerer

Speakers

Alysha Croker

Director, Centre for Policy, Pediatrics and International Collaboration, BRDD, Health Canada, Canada

Dr. Alysha Croker is the Director of the Centre for Policy, Pediatrics and International Collaboration, Health Products and Food Branch, Heath Canada. In this position, Dr. Croker is responsible for developing ways to increase access to safe and effective health products for pediatric... Read More →

Sophie Sommerer

Director General, Health Canada, Canada

Sophie Sommerer is the Director General of Health Canada’s Biologic and Radiopharmaceutical Drugs Directorate. She is responsible for regulating biologic drugs (including vaccines), radiopharmaceuticals, blood, cells, tissues and organs, and products for Assisted Human Reproduction... Read More →

Monday June 17, 2024 3:30pm - 4:30pm PDT
Room 6D San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

09: Regulatory, Forum

Format Session
Level Intermediate
Featured Topics Regulator Perspectives
Level Intermediate
Feature Topics Regulator Perspectives
Credit Type ACPE, CME, RN
Tags Forum

3:30pm PDT

#146: Past, Present, and Future: How Industry and FDA are Handling the Evolving Clinical Trial Diversity Regulatory Landscape

Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-545-L04-P; CME 1.00; RN 1.00

This forum will include discussion among multiple sponsors sharing their first-hand, cross-therapeutic area experience on notable trends and challenges they have faced in preparing these early diversity plan submissions. Panelists will also address how they plan to adapt to more expansive categories enumerated within diversity action plan guidance and share reflections on recently released FDA guidances on this topic (i.e. diversity action plans, post market approaches to obtain data on underrepresented populations, collection of race and ethnicity data). Additionally, panelists will discuss what policy deliverables and clarifications they would like to see reflected in the final guidances. The panel will also include FDA’s perspective and reflections from 2 years of diversity plan submissions as well as any considerations as we look to the future.

Learning Objectives

Describe best practices and regulatory feedback on diversity plan submissions.; Compare the perspectives of both FDA and sponsors about the current landscape of trial diversity-related guidances, including the latest diversity action plan guidance.; Discuss how by using an interactive audience feature where we seek others’ experience compiling and feedback from the FDA on diversity plan submissions.

Chair

Dylan Bechtle, MS

Speaker

Industry Update
Andrea Doyle

Industry Update
Del Smith, PhD, MS

Industry Update
Stacy Holdsworth, PharmD

FDA Update
Tamy Kim, PharmD

Speakers

Dylan Bechtle

Associate Director, Regulatory Policy Lead- US, Johnson & Johnson, United States

Dylan is an Associate Director in Johnson and Johnson Innovative Medicine's Global Regulatory Policy and Intelligence (GRPI) group. Dylan leads J&J’s regulatory policy efforts around clinical trial diversity. He is interested in the intersection of novel clinical methodologies (e.g... Read More →

Andrea Doyle

Head, Global Regulatory Affairs, Neurology, Sanofi, United States

Stacy Holdsworth

Associate Vice President, US Regulatory Policy & Strategy, Eli Lilly and Company, United States

Stacy Holdsworth is the Associate Vice President for U.S. Regulatory Policy & Strategy at Eli Lilly and Company. Her primary responsibilities are to lead a team of regulator professionals in the development and implementation of an external regulatory policy agenda that encourages... Read More →

Tamy Kim

Director, Regulatory Affairs/ Regulatory Policy, Oncology Center of Excellence, FDA, United States

Tamy Kim is the Director for Regulatory Affairs and Policy at FDA's Oncology Center for Excellence and the Associate Director for Regulatory Affairs in the Office of Oncologic Diseases (OOD). Dr. Kim is responsible for developing policies and procedures affecting the review of products... Read More →

Del Smith

Co-Founder and Chief Executive Officer, Acclinate Inc., United States

Del is the co-founder and CEO of Acclinate, a trusted digital health company focused on greater health equity through inclusive research. He has built a diverse team and AI/ML platform to help the pharmaceutical and healthcare industry access and engage communities of color so that... Read More →

Monday June 17, 2024 3:30pm - 4:30pm PDT
Room 6A San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

09: Regulatory, Forum | 02: ClinTrials -ClinOps, Forum

Format Forum
Level Intermediate
Featured Topics Diversity Equity and Inclusion (DEI), Regulator Perspectives
Level Intermediate
Feature Topics Diversity Equity and Inclusion (DEI),Regulator Perspectives
Credit Type ACPE, CME, RN
Tags Forum

8:00am PDT

#209: Leveraging New Meeting Opportunities in PDUFA VII: Experiences with Type D and INTERACT

Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-564-L04-P; CME 1.00; RN 1.00

This forum will address experience to date with new meeting types included in PDUFA VII, including Type D and INTERACT. Forum participants will evaluate successes, opportunities for improvement, and discuss how learnings can be shared.

Learning Objectives

Evaluate experiences to date with Type D and INTERACT meetings included in PDUFA VII; Identify opportunities to improve implementation of new meeting types to promote innovation and optimize FDA-Sponsor Interactions.

Chair

Victoria A. Dohnal, RAC

Speaker

Industry Update
Victoria A. Dohnal, RAC

FDA Update
Christy L. Cottrell

FDA Update
Adriane Fisher, MBA, MPH

Industry Update
Alex May, MS

Speakers

Christy Cottrell

Chief, Project Management Staff, ORO, DRO-OD, CDER, FDA, United States

Christy Cottrell is a Chief Project Manager in the FDA's Office of New Drugs, Division of Regulatory Operations for Oncologic Diseases. She co-leads a team of Regulatory Project Managers who support the Division of Oncology 1 which handles the review of drugs and biologics for reproductive... Read More →

Victoria Dohnal

Director, Global Regulatory Policy and Intelligence, Johnson & Johnson Innovative Medicine, United States

Victoria Dohnal is currently Director, Global Regulatory Policy and Intelligence at Johnson and Johnson (J&J) where she is responsible for driving US regulatory intelligence and policy work related to rare diseases, patient focused drug development, cell and gene therapies, and PDUFA... Read More →

Adriane Fisher

Regulatory Project Manager, OTP, CBER, FDA, United States

Adriane Fisher joined the FDA as a Regulatory Project Manager in September 2018, where she has provided review management throughout the product life cycle, from pre-submission meetings to post-marketing submissions. As an RPM, Adriane also acts as the liaison between FDA and industry... Read More →

Alex May

North America Lead - Regulatory Science, Policy, & Intelligence, District of Columbia, United States

Alex May leads regulatory policy, science, & intelligence for North America and global regulatory policy efforts on plasma at CSL Behring. He serves as co-chair on BIO's Meetings Task Force and NORD's Regulatory Affairs Working Group. Previously, Alex was a Director of Science & Regulatory... Read More →

Tuesday June 18, 2024 8:00am - 9:00am PDT
Room 11AB San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

09: Regulatory, Forum

Format Forum
Level Intermediate
Level Intermediate
Credit Type ACPE, CME, RN
Tags Forum

8:00am PDT

#210: Digital Biomarkers as Clinical Endpoints: The Road to Regulatory Acceptability

Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-565-L04-P; CME 1.00; RN 1.00

This forum will review the current regulatory paradigms for the use of digital biomarkers as endpoints in clinical trials in the US and EU. A panel of experts will discuss validation pathways and compare regulatory approaches between regions.

Learning Objectives

Describe the V3 process (verification, analytical validation, clinical validation) for validating digital health technologies as fit-for-purpose in a clinical trial; Identify validation pathways in the US and EU; Discuss outstanding challenges to the use of digital health technologies for remote data capture in clinical trials.

Chair

Lina AlJuburi, PharmD, MSc

Speaker

Enhancing Clinical Research in Europe: Navigating the Regulatory Landscape
Max Wegner, PharmD, RPh

Panelist
Anindita Saha

Industry Perspective
Steve Berman, MPH

Industry Update
Gul Erdemli, MD, PhD

Speakers

Lina AlJuburi

Head, Global Regulatory Science and Policy - North America, Sanofi, United States

A background in pharmacy and an interest in drug development and regulation led me to the US FDA where I spent several years within CDER, Office of New Drugs. By far the hardest decision, in 2012, I transitioned to the private sector in the field of Regulatory Science and Policy... Read More →

Steve Berman

Deputy Vice President, Science and Regulatory Affairs, BIO, United States

Steve Berman serves as Deputy VP of Science and Regulatory Strategy for the Biotechnology Innovation Organization (BIO). Steve joined BIO after serving as a Director, Translational Regulatory Affairs, at AstraZeneca. Steve joined AZ after eight years at FDA, working in both CDER and... Read More →

Gul Erdemli

Global Program Regulatory Director, Novartis Pharmaceuticals Corporation, United States

Gül Erdemli is a Global Program Regulatory Director at Novartis Regulatory Affairs Early Development. She is a drug development leader with more than 15 years industry experience from target identification to clinical development. She has an MD degree from Hacettepe University, Medical... Read More →

Anindita Saha

Associate Director Strategic Initiatives, Digital Health Center of Excellence, FDA, United States

Anindita (Annie) Saha is the Associate Director for Strategic Initiatives for the Digital Health Center of Excellence (DHCoE) at the Food and Drug Administration (FDA). She is leading strategic initiatives in moving care into the home, Artificial Intelligence, and international collaborations... Read More →

Max Wegner

Head Regulatory Affairs, Bayer AG, Germany

After studying pharmacy at the University of Bonn, Max Wegner commenced his PhD in pharmacology at Bayer and started in Regulatory Affairs as a Dossier Manager in 1996 in Wuppertal. Since then, Max has been employed in various functions within Product Development at Bayer and also... Read More →

Tuesday June 18, 2024 8:00am - 9:00am PDT
Room 6B San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

09: Regulatory, Forum

Format Forum
Level Intermediate
Featured Topics Digital Technology
Level Intermediate
Feature Topics Digital Technology
Credit Type ACPE, CME, RN
Tags Forum

8:00am PDT

#211: PMDA Town Hall

Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-566-L04-P; CME 1.00; RN 1.00

PMDA strives and implements various supports to promote regulatory sciences and accelerate product developments based on “4Fs” concept of priorities. In this session, PMDA will share the latest information on its policies and initiatives.

Learning Objectives

Recognize latest information on the Pharmaceuticals and Medical Devices Agency’s activity and Japanese regulations in medical products area; Describe how to develop medical products in Japan; Discuss issues on medical products regulation with senior PMDA officials.

Chair

Daisuke Tanaka, PhD

Speaker

PMDA Update
Daisuke Koga, MSc, RPh

Industry Update
Atsushi Tsukamoto, PhD, MSc

Panelist
Yoshiaki Uyama, PhD, RPh

Speakers

Daisuke Tanaka

Office Director, Office of International Programs, Pharmaceuticals and Medical Devices Agency (PMDA), Japan

Dr. Tanaka joined the Ministry of Health, Labour and Welfare (MHLW) in April 2001 and was involved in orphan drug designation and new drug approval as well as amendment of the Pharmaceutical Affairs Law. Subsequently, he worked in several organisations including European Medicines... Read More →

Daisuke Koga

Director, Office of International Regulatory Affairs, Ministry of Health, Labour and Welfare (MHLW), Japan

Mr. Daisuke Koga is Director, Office of International Regulatory Affairs, Pharmaceutical Safety Bureau, Ministry of Health, Labour and Welfare (MHLW) of Japan. He started his career in 1996 at the Ministry of Health and Welfare of Japan. He worked in the areas of drugs, medical devices... Read More →

Atsushi Tsukamoto

Head is Therapeutic Area Strategies, Daiichi Sankyo, United States

Atsushi is the Corporate Officer and Head of Therapeutic Area Strategy in Daiichi Sankyo, to manage multiple therapeutic area strategy. Prior to that, he was VP of New Drug Regulatory Affairs (RA) at Daiichi Sankyo. He has been global project manager for nearly 20 years (in JPN... Read More →

Yoshiaki Uyama

Associate Executive Director, Pharmaceuticals and Medical Devices Agency (PMDA), Japan

Dr Yoshiaki Uyama is currently Associate Executive Director, Pharmaceuticals & Medical Devices Agency (PMDA) of Japan. He is responsible for regulatory science researches in the agency. He has many experiences in new drug evaluation and real world data utilization, including the ICH... Read More →

Cynthia Verst

President, R&D Solutions D&DI; 2023 President, IQVIA, United States

Dr. Verst is president, Design and Delivery Innovation, Research & Development Solutions at IQVIA. She is responsible for driving innovation and transformation throughout the trial lifecycle. Having previously served as president of both clinical operations and real world and late... Read More →

Tuesday June 18, 2024 8:00am - 9:00am PDT
Room 15AB San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

09: Regulatory, Session

Format Session
Level Intermediate
Featured Topics Regulator Perspectives
Level Intermediate
Feature Topics Regulator Perspectives
Credit Type ACPE, CME, RN
Tags Session

10:00am PDT

#228: Access Consortium and Project Orbis: Experiences from Industry and Regulatory Authorities and Recommendations for Improvement

Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-579-L04-P; CME 1.00; RN 1.00

Results from international industry experience surveys on the Access Consortium and Project Orbis followed by a panel discussion, involving health authorities, to discuss recommendations and next steps to improve and promote these schemes.

Learning Objectives

Recognize the benefits and challenges of international collaborative initiatives from both the industry and regulatory authority perspectives; Discuss the recommendations to improve these schemes; Describe how to influence your own organization to adopt or increase usage of these schemes.

Chair

Sarah Powell, RAC

Speaker

Results of Industry Survey of Orbis Projects
Sarah Powell, RAC

Results of Industry Survey on Access Program
Tse Siang Kang, PhD

Speakers

Sarah Powell

President, Powell Regulatory Services, United States

Ms. Powell is the President of Powell Regulatory Services. Sarah has 30+ years of experience in pharmaceutical and related regulated industries (Clinical, Quality Control, Regulatory Affairs and Regulatory Operations). In the past 23 years as a consultant, she has assisted clients... Read More →

Tse Siang Kang

Head of Regulatory Affairs, North, East, West Asia, Global Regulatory, Pfizer Pte Ltd, Singapore

Dr. Tse Siang Kang is the Head of Regulatory Sciences in North, East and West Asia cluster, Pfizer Global Regulatory Sciences. In addition to his role in managing a diverse Asia team in Pfizer. Dr Kang also has a particular interest in publishing emerging Regulatory Sciences topics... Read More →

Tuesday June 18, 2024 10:00am - 11:00am PDT
Room 16AB San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

09: Regulatory, Session

Format Session
Level Intermediate
Featured Topics Regulator Perspectives, Regulatory Collaboration
Level Intermediate
Feature Topics Regulator Perspectives,Regulatory Collaboration
Credit Type ACPE, CME, RN
Tags Session

11:00am PDT

#231 CH: Quality in Focus: Diethylene and Ethylene Glycol Challenges

Component Type: Workshop
Level: Intermediate

Participants will delve into the US FDA's regulatory guidance, exploring the testing protocols for high-risk drug components and explore the essential requirements and recommended practices to ensure product safety and compliance.

Learning Objectives

Recognize key factors contributing to diethylene glycol (DEG) and ethylene glycol (EG) contamination; Examine the US FDA's regulatory guidance, delving into the testing procedures for high-risk drug components contaminated with DEG and EG; Explore requirements and recommended practices for manufacturers, repackagers, preparers, and distributors.

Chair

Chaitanya Koduri

Speakers

Chaitanya Koduri

Director, International Government and Regulatory Engagement, US Pharmacopeia, United States

Tuesday June 18, 2024 11:00am - 11:30am PDT
Content Hub Sails Pav San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

15: Content Hubs, Workshop | 09: Regulatory, Workshop

Level Intermediate
Level Intermediate
Tags Workshop

11:00am PDT

#233 RT: Roundtable Discussion: The State of Real-World Evidence for Regulatory Decision-Making - Views from FDA, EMA, and PMDA

Component Type: Session

Join the Real-World Evidence Community for a follow up round table discussion tied to session: The State of Real-World Evidence for Regulatory Decision-Making - Views from FDA, EMA, and PMDA (Monday, June 17 | 2:15pm - 3:15 PM PDT). Space is limited.

Chair

Jingyu (Julia) Luan, PhD

Speakers

Jingyu (Julia) Luan

Executive Regulatory Science Director, BioPharmaceuticals R&D, AstraZeneca, United States

Dr. Jingyu (Julia) Luan is a Senior Director of Global Regulatory Affairs in AstraZeneca, leading global drug development and regulatory strategies as well as innovative regulatory initiatives, eg, RWE. Prior to AstraZeneca, she worked at FDA for 13 years and held positions with increasing... Read More →

Tuesday June 18, 2024 11:00am - 12:00pm PDT
Ballroom 20 BC/ Zone 2 San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

16: Community Rounds, Session | 09: Regulatory, Session

Tags Session

1:15pm PDT

#248: The Evolving Role of Artificial Intelligence in the Medicinal Product Lifecycle

Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-586-L04-P; CME 1.00; RN 1.00

This session will provide perspectives from stakeholders on successes and limitations from practical examples and discuss principles and best practices for maximizing the potential while managing the risks moving forward.

Learning Objectives

Describe the current state of the use of artificial intelligence (AI) in the medicinal product lifecycle including its opportunities and challenges; Discuss principles and best practices to maximize potential of AI while managing risks.

Chair

Hilmar Hamann, PhD

Speaker

EMA Update
Lorenzo De Angelis, PhD

Industry Update
Carlyn Crisostomo, MBA

Industry Update
Alexandra (Zan) Cha, PhD, MA

Industry Update
Timothe Menard, PharmD, MSc

Speakers

Lorenzo De Angelis

Platform Architect, European Medicines Agency, Netherlands

Lorenzo De Angelis studied Physics in Sapienza University of Rome, where he graduated cum laude in July 2014. He then moved to the Netherlands, where he obtained his physics PhD in December 2018, with a thesis entitled "The Singular Optics of Random Light". Afterwards, he joined the... Read More →

Alexandra (Zan) Cha

Principal, Chief Technology Office, Booz Allen Hamilton, United States

Zan brings 20+ years of experience in health AI / analytics, and IT /product / ops management, applied to medical devices, pharmaceuticals, supply chain, clinical trials, insurance tech, chronic disease management, and population health & wellness. She is currently focused on advancing... Read More →

Carlyn Crisostomo

Vice President, Head of R&D AI Strategy and Execution (RAISE), Amgen, United States

Hilmar Hamann

Head of Information Management, European Medicines Agency, Netherlands

Dr. Hilmar Hamann is the Head of Information Management at the European Medicines Agency where he focuses on transforming technology capabilities for the Network of Regulatory Agencies in the EU and its stakeholders to become an all-digital, efficient and data-driven Network of the... Read More →

Timothe Menard

Global Head, Quality Excellence Digital | Bioethics Coach (Data Ethics), F. Hoffmann-La Roche Ltd, Switzerland

Started in drug safety at Merck KGaA, joined Roche as a GCP/PV auditor. Transitioned to analytics; now heads Quality Excellence Digital. Focuses on digital & analytics for core quality deliverables. Co-founded the Inter coMPany quALity Analytics consortium to drive data/quality policy... Read More →

Tuesday June 18, 2024 1:15pm - 2:15pm PDT
Room 6F San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

03: Data-Tech, Session | 09: Regulatory, Session

Format Session
Level Intermediate
Featured Topics Artificial Intelligence (AI), Regulator Perspectives
Level Intermediate
Feature Topics Artificial Intelligence (AI),Regulator Perspectives
Credit Type ACPE, CME, RN
Tags Session

1:15pm PDT

#253: Platform Technology Designation: Opportunities and Challenges for Implementation

Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-591-L04-P; CME 1.00; RN 1.00

This forum will review the key components of the new FDA Platform Technologies Designation program. A panel of experts will provide their perspectives on the proposal and discuss opportunities and challenges related to its implementation.

Learning Objectives

Describe the key attributes of the proposed Platform Technology Designation Program as described by the anticipated FDA draft guidance; Discuss stakeholder perspectives about the new designation program; Describe anticipated opportunities and challenges related to its implementation.

Chair

Andrea Doyle

Speaker

Panelist
Rachael Anatol

Panelist
Evdokia Korakianiti, PhD, MSc

Panelist
Monica Veldman

Speakers

Rachael Anatol

Deputy Super Office Director, Office of Therapeutic Products, CBER, FDA, United States

Andrea Doyle

Head, Global Regulatory Affairs, Neurology, Sanofi, United States

Evdokia Korakianiti

Head of Quality and Safety of Medicines, European Medicines Agency, Netherlands

Evdokia has joined the Agency in 2002. Since 2020, she is leading the Quality and Safety Department, which ensures that patients take medicine that are safe, are of the best possible quality and are manufactured in compliance with EU and international standards. She leads the Agency’s... Read More →

Monica Veldman

Director, Global Regulatory Policy, Alliance for Regenerative Medicine, United States

Tuesday June 18, 2024 1:15pm - 2:15pm PDT
Room 6B San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

09: Regulatory, Forum

Format Forum
Level Intermediate
Featured Topics Digital Technology
Level Intermediate
Feature Topics Digital Technology
Credit Type ACPE, CME, RN
Tags Forum

1:15pm PDT

#254: FDA Rare Disease Town Hall

Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-593-L04-P; CME 1.00; RN 1.00

Hear from senior FDA leadership about challenges and opportunities in reviewing drugs for rare diseases. Representatives from key Agency Offices/Centers will provide updates on key trends and initiatives, then will discuss hot topic questions.

Learning Objectives

Discuss recent trends in the development and FDA review of orphan drugs; Explain new and ongoing FDA policies and initiatives to facilitate the development of drugs for rare diseases; Identify opportunities to further address the challenges associated with rare disease medical product development.

Chair

James Valentine, JD, MHS

Speaker

Panelist
Kerry Jo Lee, MD

Panelist
Peter W. Marks, MD, PhD

Speakers

James Valentine

Director, Hyman, Phelps & McNamara, PC, United States

James Valentine, JD, MHS is an attorney at Hyman, Phelps & McNamara where he assists medical product industry and patient advocacy organization clients in a wide range of regulatory matters, including new drug and biologic development and approval issues. Mr. Valentine has been central... Read More →

Kerry Jo Lee

Assoc. Dir. for Rare Diseases; Program Manager, Advancing Rare Disease Cures Pro, FDA, United States

Dr. Kerry Jo Lee is a graduate of Princeton University and of the New York University School of Medicine with an honors degree conferred in microbiology. She completed residency in pediatrics at the Children’s Hospital of Los Angeles and a post-doctoral clinical fellowship in Pediatric... Read More →

Peter Marks

Director, Center for Biologics Evaluation and Research, FDA, United States

Tuesday June 18, 2024 1:15pm - 2:15pm PDT
Room 6A San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

09: Regulatory, Forum

Format Session
Level Intermediate
Featured Topics Rare Disease, Special Populations
Level Intermediate
Feature Topics Rare Disease,Special Populations
Credit Type ACPE, CME, RN
Tags Forum

1:15pm PDT

#245: The European Clinical Trials Environment Under the Accelerating Clinical Trials (ACT EU) Initiative: Two Years On

Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-583-L04-P; CME 1.00; RN 1.00

Accelerating Clinical Trials in the EU (ACT EU) and the successful launch of the clinical trial regulation have modernized the clinical trial environment. This session is focused on how Europe is embracing the change and will also present other international initiatives offering opportunities for collaboration.

Learning Objectives

Describe how ACT EU, together with other EU initiatives, is providing a platform to optimize clinical research and development, and fostering innovation through joint collaborations between member states, ethics committees, and stakeholders, bringing tangible benefits for patients, sponsors, and regulators; Discuss other international clinical trial initiatives and potential areas of collaboration.

Chair

Ana Zanoletty Perez

Speaker

Academic Update
Sara Bristol Calvert, PharmD

Patient Advocate Perspective
Deborah Collyar

WHO Update
Marion Laumonier, MSc

Speakers

Kevin Bugin

Deputy Director of Operations, Office of New Drugs, CDER, FDA, United States

Sara Calvert

Director of Projects, Clinical Trials Transformation Initiative (CTTI), United States

Dr. Sara Bristol Calvert is currently the Director of Projects at the Clinical Trials Transformation Initiative (CTTI). Prior to this role, she was a CTTI Senior Project Manager which included leading projects in Diversity in Clinical Trials, Single IRB, Registry Trials, HABP/VABP... Read More →

Deborah Collyar

President, Patient Advocates In Research (PAIR), United States

Deborah Collyar is founder and president of Patient Advocates in Research (PAIR), “where research meets reality.” Her leadership in patient engagement and advocacy started in the 1990s after her first cancer diagnosis. She applies business leadership, IT, communication and strategic... Read More →

Marion Laumonier

Technical Officer, Regulatory Convergence and Networks, World Health Organization (WHO), Switzerland

Marion Laumonier is currently Technical Officer in the Regulatory Convergence and Networks team at the WHO in Geneva, Switzerland. Marion Laumonier joined the WHO in 2021 and has over 20 years of experience gained in different settings, pharmaceutical industries and non-profit organizations... Read More →

Ana Zanoletty Perez

Head of Clinical Trials Transformation workstream, European Medicines Agency, Netherlands

Ana qualified as a pharmacist and following various roles managing clinical trials for the biopharmaceutical industry joined EMA in 2006. At EMA she spent her initial years coordinating initial marketing authorisation procedures, and later transitioned into the management of post-authorisation... Read More →

Tuesday June 18, 2024 1:15pm - 2:15pm PDT
Room 5AB San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

09: Regulatory, Session | 02: ClinTrials -ClinOps, Session

Format Session
Level Intermediate
Featured Topics Ethics, Regulator Perspectives
Level Intermediate
Feature Topics Ethics,Regulator Perspectives
Credit Type ACPE, CME, RN
Tags Session

1:15pm PDT

#255: International Harmonization to Support Pharmaceutical Quality and Lifecycle Management

Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Application UAN: 0286-0000-24-594-L04-P; CME 1.00; RN 1.00

Since establishment in 1990, the International Council for Harmonisation (ICH) has expanded its global footprint to harmonize requirements for the development of medicines and to increase patient access to medicines. Importantly, ICH’s quality portfolio serves to support continual improvement and innovation in biopharmaceutical manufacturing. In 2018, ICH published its quality mission to “develop a harmonised pharmaceutical quality system applicable across the lifecycle of the product emphasizing an integrated approach to quality risk management and science” driving the development of several new guidelines addressing topics on lifecycle management, quality risk management, continuous manufacturing, and analytical procedure development and validation. With ICH’s expansion, it has prioritized the adequate implementation and adherence of guidelines through its work to promote training and periodically conduct a survey to assess guideline implementation by ICH Members and Observers. This session will provide global regulatory and industry perspective regarding progress made on the ICH quality mission and explore future opportunities to achieve greater harmonization and encourage the global community to embrace the science and risk-based approaches. Case studies will be presented highlighting the objectives and work to facilitate a globally aligned approach to implementation of ICH guidelines.

Learning Objectives

Describe objectives of international harmonization through ICH to promote innovation and facilitate more efficient lifecycle and post-approval change management; Discuss status of key ICH Guidelines such as Q13, Q12 and Q14 and their implementation globally; Evaluate case studies and identify opportunities to advance global harmonization.

Chair

Amanda Marie Roache, MS

Speaker

Lifecycle Strategies at the Interface Between ICH Q12 and ICH Q14
Connie Langer, MSc

ICH Q14: Analytical Procedure Development and Lifecycle Management
Nina S. Cauchon, PhD

FDA Update
Sau L Lee, PhD

Update from Japan on Q12
Satomi Yagi

Update from EMA
Anabela Marcal, PharmD

Speakers

Amanda Roache

Senior Director, Science and Regulatory Advocacy, Pharmaceutical Research and Manufacturers of America (PhRMA), United States

Amanda Roache, MPP, is a Senior Director in PhRMA’s Science and Regulatory Advocacy Department. She leads PhRMA’s work under the International Council for Harmonisation (ICH) as well as key science and regulatory activities related to international regulatory policy, advocacy... Read More →

Nina Cauchon

Director Regulatory Affairs CMC, Amgen, United States

Nina S. Cauchon, PhD, leads external engagement activities within RA-CMC. She has experience leading both early phase & commercial programs, including small molecules and biologics. Her areas of interest are regulatory challenges for innovative modalities and emerging technologies... Read More →

Connie Langer

Senior Director, Pfizer Inc, United States

Connie Langer is a Senior Director at Pfizer who leads a team that monitors and advises on regulatory changes and impacts. She collaborates with experts to communicate Pfizer's feedback and position to health authorities on regulations and guidance documents. She also has experience... Read More →

Satomi Yagi

Reviewer, Office of New Drug III, Pharmaceuticals and Medical Devices Agency (PMDA)

Sau Lee

Deputy Super Office Director, OPQ, CDER, FDA, United States

Dr. Lee is currently Deputy Super Office Director for Operations who oversees research, quality surveillance, policy, quality assurance, and administrative operation functions in CDER’s Office of Pharmaceutical Quality. Dr. Lee has been with the FDA since 2005, serving as a regulatory... Read More →

Anabela Marcal

EMA Liaison Official to the US FDA, European Medicines Agency, Netherlands

Anabela holds a degree in pharmacy and a professional certification in hospital pharmacy. Anabela had joined the EMA in 1999. During her career at the Agency she held a number of roles in various areas, including Head of Committees and Inspections and Head of Clinical Trials. Currently... Read More →

Tuesday June 18, 2024 1:15pm - 2:15pm PDT
Room 6C San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

09: Regulatory, Forum | 10: RegCMC-Product Quality, Forum

Format Forum
Level Intermediate
Featured Topics ICH
Level Intermediate
Feature Topics ICH
Credit Type ACPE, CME, RN
Tags Forum

2:15pm PDT

#258 RT: Roundtable Discussion: Supporting Regulatory Convergence and Reliance Through a Pharmaceutical Quality Knowledge Management Capability

Component Type: Session

Join the Regulatory Affairs Community for a follow up round table discussion tied to session: Supporting Regulatory Convergence and Reliance Through a Pharmaceutical Quality Knowledge Management Capability (Tuesday, June 18 | 8:00am - 9:00AM PDT). Space is limited.

Chair

Demetra Macheras, MBA

Speakers

Demetra Macheras

Director, Regulatory Policy and Intelligence - Regulatory Affairs, AbbVie, United States

Demetra Macheras is currently Director, Regulatory Policy and Intelligence at AbbVie. She assesses and coordinates comments on proposed regulatory policies for the U.S., Canada, ICH, and WHO regulatory environments and ensures appropriate AbbVie personnel are aware of emerging and... Read More →

Tuesday June 18, 2024 2:15pm - 3:15pm PDT
Ballroom 20 BC/ Zone 2 San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

16: Community Rounds, Session | 09: Regulatory, Session | 10: RegCMC-Product Quality, Session

Tags Session

3:15pm PDT

#264: Enhancing Adoption of Innovative Approaches in Clinical Trials

Component Type: Forum
Level: Basic
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-596-L04-P; CME 1.00; RN 1.00

This forum will explore the U.S. FDA Center for Drug Evaluation and Research's portfolio of clinical trial innovation activities, including its newly established CDER Center for Clinical Trial Innovation (C3TI). C3TI serves as a central hub supporting innovative approaches to clinical trials aimed at improving the efficiency of drug development. Speakers from the FDA will elaborate on C3TI and its role in supporting innovative clinical trial designs. The FDA presenters will be joined by industry and patient speakers for a panel discussion on opportunities to enhance implementation and adoption of innovative approaches in clinical trials, aiming to ensure patient-centric trial design, improve the efficiency of clinical trials, increase the participation of diverse populations, and, in turn, accelerate the development of safe and effective new drugs.

Learning Objectives

Discuss key challenges or barriers to implementation and adoption of innovative approaches in clinical trial design, conduct, and execution; Discuss strategies to enhance implementation and adoption of innovative approaches in clinical trial design, conduct, and execution.

Chair

Meghana Chalasani, MHA

Speaker

Panelist
Kevin Bugin, PhD, MS, RAC

Panelist
Ella Balasa

Panelist
Michelle Rohrer, PhD

Panelist
Allison Cuff Shimooka, MBA

Speakers

Meghana Chalasani

Associate Director for Clinical Trial Innovation, Office of New Drugs, CDER, FDA, United States

Kevin Bugin

Deputy Director of Operations, Office of New Drugs, CDER, FDA, United States

Michelle Rohrer

Senior Vice President, Global Head of Product Development Regulatory and Policy, Genentech Roche, United States

Michelle Rohrer, PhD is Global Head of Product Development Regulatory and Policy. Michelle has worked at the company for 27 years, starting as a post-doctoral fellow. She has worked as a clinical scientist, team leader, and regulatory strategist. In 2013, Michelle was named by the... Read More →

Ella Balasa

Patient Advocate and Engagement Consultant, Balasa Consulting, United States

Ella Balasa is a patient advocate, consultant, and a person living with cystic fibrosis. She has committed her time to empowering patients and advancing healthcare strategies. She speaks publicly about the value of patient perspective and has a passion for distilling clinical information... Read More →

Allison Cuff Shimooka

Chief Operating Officer, TransCelerate Biopharma Inc, United States

Allison is Chief Operating Officer of TransCelerate BioPharma Inc. In this role, Allison is responsible for shaping and delivering on TransCelerate’s strategic vision: to advance collaboration in driving efficient, effective and high-quality delivery of new medicines through the... Read More →

Tuesday June 18, 2024 3:15pm - 4:15pm PDT
Room 6A San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

05: Patient-Focused Drug Dev, Forum | 09: Regulatory, Forum | 02: ClinTrials -ClinOps, Forum

Format Forum
Level Basic
Featured Topics Regulator Perspectives
Level Basic
Feature Topics Regulator Perspectives
Credit Type ACPE, CME, RN
Tags Forum

3:15pm PDT

#272: This is Your Pilot Speaking: The Journey from Regulatory Pilot Program Innovation to Routine Practice

Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Application UAN: 0286-0000-24-604-L04-P; CME 1.00; RN 1.00

This panel will explore the blue skies of global regulatory pilot programs, including initiatives on real-world evidence (RWE), model-informed drug development (MIDD), innovative trials, and endpoints. Panelists will educate the audience on new opportunities and best practices for future pilot success.

Learning Objectives

Critique new opportunities to participate in regulatory pilot programs at FDA and globally; Outline key lessons learned from pilot program case studies and experiences; Identify best practices to increase the likelihood of success for future pilot programs.

Chair

Andrew Emmett, MPH

Speaker

Moderator
Danielle Friend Economo, PhD

PDUFA VII Rare Disease Endpoint Advancement Program
Mary Jo Salerno, MPH, MS

Learnings from FDA PDUFA VII Advancing Real-World Evidence Program
Yueqin Zhao, PhD

Industry Update
Donna Boyce, MS, RAC

Industry Update
Rasika Kalamegham, PhD

Speakers

Danielle Friend Economo

Senior Director, US Head Regulatory Policy and Intelligence, Johnson & Johnson Innovative Medicine, United States

Dr. Danielle Friend received her Ph.D. in neuroscience in 2013 from the University of Utah. After completing her graduate work, Danielle conducted postdoctoral research at the National Institutes of Health. In 2016, Danielle was selected as a Science and Technology Policy Fellow with... Read More →

Andrew Emmett

FDA Liaison | US Regulatory Policy and Global Intelligence, Pfizer Inc, United States

Donna Boyce

Head and Senior Vice President of Global Regulatory Sciences, Pfizer Inc, United States

Donna Boyce is Senior Vice President of Global Regulatory Sciences and a member of the Pfizer Research and Development (PRD) Leadership Team. She joined Pfizer in May 2013 as the Vice President, Global Regulatory Affairs, Vaccine. In her current role, Donna is responsible for the... Read More →

Rasika Kalamegham

Head, US Regulatory Policy, Genentech, A Member of the Roche Group, United States

Rasika Kalamegham, Ph.D, is Head of U.S. Regulatory Policy at Genentech. She oversees all FDA related policy work related to the pipeline and serves as the principal representative to Congressional and advocacy relations for regulatory policy. She plays an active role in many trade... Read More →

Mary Jo Salerno

Science Policy Analyst, ORDPURM, OND, CDER, FDA, United States

Mary Jo Salerno currently works as a science policy analyst on the Rare Diseases Team in FDA CDER's Office of New Drugs. Mary Jo has broad FDA regulatory, policy, and program development and management experience, including previous positions in FDA Europe Office, CDER Office of Biostatistics... Read More →

Yueqin Zhao

Lead Mathematical Statistician, OB, OTS, CDER, FDA, United States

Yueqin Zhao is a lead mathematical statistician in Office of Biostatistics, Center for Drug Evaluation and Research, FDA. Her research interests include benefit-risk assessment, signal detection, and statistical methods in observational studies.

Tuesday June 18, 2024 3:15pm - 4:15pm PDT
Room 6F San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

09: Regulatory, Session

Format Session
Level Intermediate
Featured Topics Real-World Data (RWD)- Real-World Evidence (RWE)
Level Intermediate
Feature Topics Real-World Data (RWD)- Real-World Evidence (RWE)
Credit Type ACPE, CME, RN
Tags Session

3:15pm PDT

#273: How to Provide Necessary Medicinal Products to Children?

Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-605-L04-P; CME 1.00; RN 1.00

In this session, speakers share the efficacy of exsiting pediatric regulation and discuss future collaboration to promote pediatric drug development worldwide

Learning Objectives

Describe the pediatric regulation of Japan and ASEAN; Discuss collaboration to promote pediatric drug development in these countries.

Chair

Junko Sato, PhD

Speaker

PMDA Update
Michiyo Sakiyama, MD

Industry Upate
Robert "Skip" Nelson, MD, PhD

FDA Update
Gerold Wharton

Speakers

Junko Sato

Associate Executive Director, Pharmaceuticals and Medical Devices Agency (PMDA), Japan

Dr. Sato joined the Pharmaceuticals and Medical Devices Agency (PMDA) in 1998, and she is currently the Associate Executive Director. She has work experiences in new drug review for 11 yrs, risk management for 3 yrs, and international area for 11 yrs. She also worked in U.S. FDA as... Read More →

Robert Nelson

Senior Director, Pediatric Drug Development (CHILD), Johnson & Johnson, United States

Robert “Skip” Nelson, MD, MDiv, PhD. is currently Senior Director, Pediatric Product Development in the Child Health Innovation Leadership Department (CHILD) at Johnson & Johnson. Previously, he was the Deputy Director and Senior Pediatric Ethicist in the Office of Pediatric Therapeutics... Read More →

Michiyo Sakiyama

Associate Senior Scientist for Clinical Medicine, Office of Vaccines and Blood, Pharmaceuticals and Medical Devices Agency (PMDA), Japan

Michiyo Sakiyama, MD is an Associate Senior Scientist for Clinical Medicine of the office of Vaccines and Blood Products at the Pharmaceuticals and Medical Devices Agency (PMDA). She is a pediatrician and currently involved in reviewing vaccines against infectious diseases, blood... Read More →

Gerold Wharton

Program Analyst, Office of Clinical Policy and Programs, OC, FDA, United States

Tuesday June 18, 2024 3:15pm - 4:15pm PDT
Room 17AB San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

09: Regulatory, Session

Format Session
Level Intermediate
Featured Topics Special Populations
Level Intermediate
Feature Topics Special Populations
Credit Type ACPE, CME, RN
Tags Session

3:15pm PDT

#267: Evolution of Electronic Product Information in LATAM: Challenges and Opportunities to Improve the Value of Healthcare

Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-599-L04-P; CME 1.00; RN 1.00

This session provides a comprehensive overview of the status/evolution of the concept of electronic product information (ePI) in LATAM from multiple perspectives, including industry, international regulatory bodies, and various stakeholders involving patients and healthcare professionals (HCPs). It also reviews some of the relevant initiatives undertaken in Low to Medium income countries and their benefits and potential.

Learning Objectives

Identify the impact of electronic product information (ePI) hybrid pilots and regulatory framework evolution in LATAM countries; Discuss relevant initiatives undertaken in Low to medium income countries; Examine industry motivations and challenges in implementing ePI pilots; Describe the evolving landscape (efforts of HA's based on international standard for ePI, regulatory advancements made to ensure an optimal experience for patients/ HCPs, implications of these challenges on healthcare.

Chair

Leonardo Semprun Semprun, RPh

Speaker

Electonic Product Information Regulatory Evolution in Brazil
Nélio Cézar de Aquino, SR, MSc

Electronic Product Information in Low to Middle Income Countries
Rutendo Kuwana, RPh

Industry Outlook in Brazil: Exploring eLabeling Perspectives
Rosana M. Mastellaro, PharmD, RPh

Considerations for Implementation and Adoption of Digital Labels
Maria Cristina Mota Pina, MBA

Speakers

Leonardo Semprun

Global Regulatory Policy Lead-LatAm, MSD, Panama

Leonardo Semprún is currently Senior Director, Global Regulatory Policy at MSD. In this role, Leonardo is responsible to define and execute a regional regulatory policy plan that addresses current and future needs, while also advocating for and anticipating regulatory change with... Read More →

Nélio Cézar de Aquino

General Manager of Medicines, ANVISA, Brazil

Nélio Cézar de Aquino is the General Manager of Medicines at the Brazilian Health Regulatory Agency (Anvisa). He has held various positions at Anvisa, including General Manager of Information Technology, Ports, Airports, Borders, and Customs Warehouses, Public Quality Control Laboratories... Read More →

Rutendo Kuwana

Team Lead, Incidents and Substandard/Falsified Medical Products, World Health Organization (WHO), Switzerland

Rutendo is leading the Incidents and Substandard/Falsified (ISF) medical products team at the World Health Organization (WHO) since June 2021. The ISF team is part of the Regulation and Safety (REG) Unit of the Regulation and Prequalification (RPQ) department that is within the Access... Read More →

Rosana Mastellaro

Director, Technical Regulatory Affairs and Innovation, Sindusfarma, Brazil

Pharmacist, She is currently Director of Technical Regulatory Affairs and Inovation at Sindusfarma. She has worked for 17 years in the pharmaceutical industry. Specialist in Project Management. She acts in defense of the pharmaceutical industrial sector and coordinates regulatory... Read More →

Maria Cristina Mota Pina

Head, Regulatory Policy for Emerging Markets -Japan and Australia, AbbVie, United States

Cristina is currently Director at AbbVie where she coordinates regulatory policy and intelligence activities for the Emerging Markets. Cristina worked for Boehringer Ingelheim for 13 years in different roles including Quality, Validation and Regulatory Affairs. Cristina attended Universidad... Read More →

Tuesday June 18, 2024 3:15pm - 4:15pm PDT
Room 11AB San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

09: Regulatory, Session | 03: Data-Tech, Session

Format Session
Level Intermediate
Featured Topics Regulator Perspectives
Level Intermediate
Feature Topics Regulator Perspectives
Credit Type ACPE, CME, RN
Tags Session

3:15pm PDT

#274: Regulatory Policy Roundtable: Pharmaceutical Quality, Generics, Innovative Medicines

Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-606-L04-P; CME 1.00; RN 1.00

Industry leaders will discuss how regulatory and industrial policy can support delivering quality-assured medicines to patients faster, how to assure pharmaceutical quality for innovative and generic medicines especially in light of new analytical technologies, and how to evaluate potential threats to quality in the context of benefit-risk and patient access.

Learning Objectives

Define quality management maturity (QMM) and how the government is proposing to use it to improve the quality of medicines and resiliency of the supply chain; Identify policy solutions to quality challenges like nitrosamines, PFAS, and related impurities/chemicals; Understand the role of third parties like USP in defining quality standards; Comprehend policy ideas that enable earlier patient access to medicines without compromising quality

Chair

Aaron Josephson, MS

Speaker

How to Integrate Quality Management Maturity into Regulatory Assessment and Oversight
Aaron Josephson, MS

Panelist
Katherine Wilson, JD, MA

Quality Policy Priorities of the Canadian Generic Industry
Kristin Willemsen, MS

Panelist
Brett Howard, JD, PhD

Speakers

Aaron Josephson

Senior Director, Global Regulatory Policy, Teva Pharmaceuticals, United States

Aaron develops and advocates for policies that enable more efficient and predictable regulatory systems aligned with Teva’s development programs, business objectives, and patient needs. Prior to joining Teva, Aaron worked at FDA for more than a decade in positions at CDER, CBER... Read More →

Brett Howard

Senior Director, US Regulatory Policy, USP, United States

Brett Howard is a Senior Director of US Regulatory Policy at the US Pharmacopeia, where he manages USP's regulatory efforts concerning pharmaceutical quality, covering both large and small molecules. Prior to joining USP, he spent the previous decade working for chemical and pharmaceutical... Read More →

Kristin Willemsen

Vice President, Scientific and Regulatory Affairs, Canadian Generic Pharmaceutical Association, Canada

Kristin Willemsen is the Vice President of Scientific & Regulatory Affairs for CGPA. She works with CGPA members advocating for the implementation of regulatory, policy and guidance changes to increase access to safe, effective, and affordable generic medicines for Canadians. A key... Read More →

Katherine Wilson

Head, Global Regulatory Policy and Intelligence, Viatris, United States

Katherine (Katie) Wilson, leads Global Regulatory Policy + Intelligence at Viatris, a global healthcare company committed to empowering people worldwide to live healthier at every stage of life. In her current role, Katie provides regulatory support to drive global policy initiatives... Read More →

Tuesday June 18, 2024 3:15pm - 4:15pm PDT
Room 6C San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

09: Regulatory, Forum | 10: RegCMC-Product Quality, Forum

Format Forum
Level Intermediate
Featured Topics Regulator Perspectives
Level Intermediate
Feature Topics Regulator Perspectives
Credit Type ACPE, CME, RN
Tags Forum

4:30pm PDT

#285: Remote Regulatory Assessments: Examining Another Tool in the FDA Regulatory Toolbox

Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-617-L04-P; CME 1.00; RN 1.00

This session will describe the evolution of remote regulatory assessments (RRA’s), FDA’s authority to conduct an RRA in lieu of an inspection, the process for conducting an RRA, and recent RRAs conducted as part of the FY23 BIMO program

Learning Objectives

Describe what a remote regulatory assessments is and FDA’s authority to conduct a remote regulatory assessment; Describe the steps taken by the agency and site before, during, and after a remote regulatory assessment is conducted; Discuss FY23 metrics including recently conducted remote regulatory assessments.

Chair

Jan Hewett, BSN, JD

Speaker

FDA Update
Kavita C. Dada, PharmD

Industry Perspective on Remote Assessments
James Riddle

Health Canada Update
Jennifer Evans

Speakers

Kavita Dada

Associate Director for Regulatory Operations, OSI, OC, CDER, FDA, United States

Jennifer Evans

Compliance and Enforcement Specialist, Health Canada, Canada

Jennifer Evans obtained her degree in Biochemistry from the University of Victoria, in British Columbia, Canada. She worked in the pharmaceutical and biotechnology industries in research, clinical and quality roles for 14 years prior to joining Health Canada in 2012 as a compliance... Read More →

Jan Hewett

Regulatory Counsel for Policy, OSI, CDER, FDA, United States

Jan Hewett joined the U.S. Food and Drug Administration's Center for Drug Evaluation and Research -Office of Compliance- Office of Scientific Investigations office as Regulatory Counsel (Policy) in February 2015. Prior to joining OSI, Jan served as the Director of the Yale University... Read More →

James Riddle

Senior Vice President, Global Review Operations, Advarra, United States

JJames Riddle is the Senior Vice President of Global Review Operations at Advarra, the world’s largest provider of ethics, safety, and compliance oversight reviews services. With 25+ years’ experience providing support to the clinical research community, Riddle has operational... Read More →

Tuesday June 18, 2024 4:30pm - 5:30pm PDT
Room 16AB San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

09: Regulatory, Session

Format Session
Level Intermediate
Featured Topics Regulator Perspectives
Level Intermediate
Feature Topics Regulator Perspectives
Credit Type ACPE, CME, RN
Tags Session

4:30pm PDT

#286: Recent Evolution of the Accelerated Approval Pathway: Impacts on Use and Implementation

Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-618-L04-P; CME 1.00; RN 1.00

Thought leaders will be brought together in a fireside chat to discuss impacts observed due to recent changes to Accelerated Approval as well as current trends in how the pathway is being used and implemented.

Learning Objectives

Identify the changes to Accelerated Approval resulting from the passage of FDORA; Describe the benefits and challenges encountered by regulators, industry, and patient organizations when it comes to the use of Accelerated Approval; Discuss the impacts of the changes to the Accelerated Approval pathway and how it is currently being implemented.

Chair

Danielle Friend Economo, PhD

Speaker

Industry Update
Alexis Miller, JD

FDA Update
Peter W. Marks, MD, PhD

Academic Update
Edward Neilan, MD, PhD

Speakers

Danielle Friend Economo

Senior Director, US Head Regulatory Policy and Intelligence, Johnson & Johnson Innovative Medicine, United States

Peter Marks

Director, Center for Biologics Evaluation and Research, FDA, United States

Alexis Miller

Acting Global Lead, Global Regulatory Policy and Intelligence, Merck & Co., Inc., United States

Alexis Reisin Miller leads Global Regulatory Policy at Merck & Co., Inc., helping to shape the regulatory and policy environments on topics like evolution in regulatory science and policy frameworks, upholding expedited pathways, leveraging RWE and other tech opportunities, embedding... Read More →

Edward Neilan

Chief Medical and Scientific Officer, National Organization for Rare Disorders (NORD), United States

Dr. Ed Neilan is the Chief Medical and Scientific Officer at the National Organization for Rare Diseases (NORD). He is a medical geneticist with experience in both academic medicine and the pharmaceutical industry. His expertise spans laboratory research and medical practice, as well... Read More →

Tuesday June 18, 2024 4:30pm - 5:30pm PDT
Room 6A San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

09: Regulatory, Forum

Format Forum
Level Intermediate
Level Intermediate
Credit Type ACPE, CME, RN
Tags Forum

4:30pm PDT

#287: ANVISA Town Hall

Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-619-L04-P; CME 1.00; RN 1.00

In this forum, leaders from ANVISA will provide an update on regulatory priorities for Brazil, and inform about local trends, regulatory convergence, and collaboration initiatives. The discussion will also invite questions of general interest from the audience.

Learning Objectives

Describe ANVISA’s regulatory initiatives and strategic priorities; Discuss regulatory convergence of Brazilian regulations for medicines and medical devices to international guidelines and standards; Identify trends and opportunities for engagement with the Brazilian regulator.

Chair

Cammilla Horta Gomes, MA, MPharm

Speaker

Panelist
Antonio Barra Torres, MD

Panelist
Patricia Oliveira Pereira Tagliari, LLM, MPH

Panelist
Bianca Zimon, LLM

Speakers

Antonio Barra Torres

President Director, ANVISA, Brazil

Mr. Barra, President-Director of Anvisa, graduated in Medicine from Foundation Souza Marques in 1986 and had his residency in Vascular and Endovascular surgery done at Marcílio Dias Naval Hospital (HNMD) in Rio de Janeiro. MBA in Health Services Administration at Federal University... Read More →

Cammilla Horta Gomes

LATAM Regulatory Policy Lead, Roche, Brazil

Specialist in Global Health and Health Regulation, with a Master’s Degree in Development and International Cooperation. Broad experience in regulatory policy, both as regulator in ANVISA and industry, leading or contributing to bilateral and multilateral negotiations, harmonization... Read More →

Patricia Oliveira Pereira Tagliari

Associate Director, ANVISA, Brazil

Patricia Oliveira Pereira Tagliari holds a master’s degree in Public Health (Global Health and Health Diplomacy) from the National School of Public Health (2014), specializations in Health Regulation and Surveillance (2010) and Health Law (2007), both from Fiocruz, and a degree... Read More →

Bianca Zimon

Health Regulation Specialist, ANVISA, Brazil

Advisor to the International Affairs Office at the Brazilian Health Regulatory Agency (Anvisa). Specialist in Health Regulation and Constitutional Law, with extensive experience in health regulation and enforcement, as well as in bilateral and multilateral negotiations, international... Read More →

Tuesday June 18, 2024 4:30pm - 5:30pm PDT
Room 14AB San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

09: Regulatory, Forum

Format Forum
Level Intermediate
Featured Topics Regulator Perspectives
Level Intermediate
Feature Topics Regulator Perspectives
Credit Type ACPE, CME, RN
Tags Forum

8:00am PDT

#302: Have a Safe Trip: Clinical and Patient Alignment in Clinical Trials with Psychedelics

Component Type: Session
Level: Basic
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-623-L04-P; CME 1.00; RN 1.00

This discussion will provide the setting and background of clinical trials of psychedelics and issues to consider. The session will provide details with a clinical trial participant providing example feedback and insight from that perspective.

Learning Objectives

Discuss the conflicted background of psychedelic clinical trials; Discuss reasonable expectations for clinical trial progression; Identify one participant’s perspective from two clinical trials; compare and contrast the participant perspective with standard clinical outcomes.

Chair

Currien MacDonald, MD

Speaker

Panelist
Sara Reed, MA

Speakers

Currien MacDonald

Medical Chair Director, WCG, United States

Dr. Currien MacDonald has lead the medical chairs at WCG for the past 7 years. Prior to that, Dr. MacDonald served for four years as the medical director for Aspire IRB, another WCG company. After graduating top of his class from the University of Minnesota Medical School, Dr. MacDonald... Read More →

Sara Reed

Video Production, Consultant, United States

Sara Reed holds a MFA in Documentary Media from Northwestern University. With over a decade of freelance video production experience spanning the U.S., Canada, Central/South America, and Africa, she’s contributed to projects for renowned clients such as Discovery, HBO, NASA, and... Read More →

Wednesday June 19, 2024 8:00am - 9:00am PDT
Room 6A San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

05: Patient-Focused Drug Dev, Session | 09: Regulatory, Session | 02: ClinTrials -ClinOps, Session

Format Session
Level Basic
Featured Topics Ethics
Level Basic
Feature Topics Ethics
Credit Type ACPE, CME, RN
Tags Session

8:00am PDT

#309: FDA Oncology Center of Excellence: Are Sponsors Taking Full Advantage of OCE Regulatory Policy “Projects?”

Component Type: Session
Level: Advanced
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-630-L04-P; CME 1.00; RN 1.00

The FDA Oncology Center of Excellence "Projects" address gaps in oncology drug development and present opportunities as well as challenges for industry. Learn from FDA and industry experts the value of these initiatives and how and when to join in.

Learning Objectives

Recognize high-value FDA OCE priorities and projects that impact industry; Identify how and when to engage with these FDA initiatives to produce optimal results for industry, patients, and other stakeholders; Describe how to advance opportunities for harmonization of these high-value OCE Projects with global Health Authorities.

Chair

Sabrina Girty, JD

Speaker

FDA Overview and Perspective on OCE Projects
Tamy Kim, PharmD

From Policy to Practice: Maximizing Collaboration to Support Oncology Innovation
Mark Stewart, PhD

Industry Case Study: Implementing OCE Projects in Drug Development
Vandana Pathak, MS, RAC

Speakers

Sabrina Girty

Executive Director, Global Regulatory Affairs, Oncology, Merck & Co., Inc., United States

Sabrina Girty is an Executive Director in Global Regulatory Affairs, Oncology at Merck & Co. Inc and leads a team of global regulatory strategy professionals across the esophageal, gastric, colorectal and head & neck cancer programs. At Merck, her team secured global marketing authorizations... Read More →

Tamy Kim

Director, Regulatory Affairs/ Regulatory Policy, Oncology Center of Excellence, FDA, United States

Vandana Pathak

Director Global Regulatory Affairs, Amgen, United States

Vandana Pathak, MS, RAC is a Director, Global Regulatory Affairs - Oncology at Amgen Inc. She leads global regulatory teams to drive global regulatory strategy and provides regulatory leadership to cross-functional teams to develop and execute global regulatory product strategies... Read More →

Mark Stewart

Vice President, Science Policy, Friends of Cancer Research, United States

Mark Stewart is vice president of science policy at Friends of Cancer Research (Friends), an advocacy organization based in Washington, DC that drives collaboration among partners of every healthcare sector and creates and implements policies ensuring patients receive the best treatments... Read More →

Wednesday June 19, 2024 8:00am - 9:00am PDT
Room 6F San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

09: Regulatory, Session

Format Session
Level Advanced
Featured Topics Regulator Perspectives
Level Advanced
Feature Topics Regulator Perspectives
Credit Type ACPE, CME, RN
Tags Session

8:00am PDT

#310: WHO Town Hall: Safeguarding Public Health - WHO's Vision for Global Regulatory Excellence

Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-631-L04-P; CME 1.00; RN 1.00

This session aims to delve into WHO's pivotal role in the evolving landscape of global regulatory excellence. The discussion will revolve around the transformative impact of WHO-listed Authorities on medical product regulation, emphasizing regulatory reliance as a holistic approach to enhance efficiency and accessibility. Additionally, the session will address the pressing issue of substandard/falsified medical products and their potential threats to global health.

Learning Objectives

Describe the significance of WHO-listed Authorities in shaping the future of medical product regulation; Examine the concept of regulatory reliance as a key strategy to enhance access and efficiency in global regulatory practices; Discuss the critical role of regulatory excellence in safeguarding public health against the threats posed by substandard/falsified medical products.

Chair

Samvel Azatyan, MD, PhD

Speaker

Elevating Regulatory Reliance: The Strategic Impact of WHO-Listed Authorities in Transforming Global Regulatory Landscapes
Hiiti Sillo, MSc, RPh

Securing Supply Chains: A Global Imperative
Rutendo Kuwana, RPh

EMA Update
Emer Cooke, MBA, MSc

Industry Update
Janis Bernat, MSc

Moderator for Panel Discussion and Q&A
Marion Laumonier, MSc

Speakers

Samvel Azatyan

Team Lead, Regulatory Convergence and Networks (RCN/REG), World Health Organization (WHO), Switzerland

Dr Samvel Azatyan is a Paediatrician with a PhD in clinical pharmacology and medical products regulation. Over his more than 24 years at WHO, he has had a wide range of leadership roles associated with the regulation of medical products, including supporting regulatory collaboration... Read More →

Janis Bernat

Director, Scientific and Regulatory Affairs, IFPMA, Switzerland

Janis leads cross-functional activities in regulatory science and international health policy for IFPMA, while partnering with policy experts and stakeholders to strengthen the pharmaceutical regulatory environment. She is responsible for guiding the organization’s regulatory team... Read More →

Emer Cooke

Chair, ICMRA; Executive Director, European Medicines Agency, Netherlands

Rutendo Kuwana

Team Lead, Incidents and Substandard/Falsified Medical Products, World Health Organization (WHO), Switzerland

Marion Laumonier

Technical Officer, Regulatory Convergence and Networks, World Health Organization (WHO), Switzerland

Hiiti Sillo

Unit Head, Regulation and Safety, Department of Regulation and Prequalification, World Health Organization (WHO), Switzerland

Hiiti Sillo is the Unit Head, Regulation and Safety within the WHO department of Regulation and Prequalification. Until August 2021, he was the Team Lead, Regulatory Systems Strengthening (RSS), Regulation and Safety Unit. Before joining WHO in January 2018, he was the Director General... Read More →

Wednesday June 19, 2024 8:00am - 9:00am PDT
Room 1AB San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

09: Regulatory, Forum

Format Forum
Level Intermediate
Featured Topics Regulatory Reliance
Level Intermediate
Feature Topics Regulatory Reliance
Credit Type ACPE, CME, RN
Tags Forum

8:00am PDT

#311: Australia, Canada, Singapore, Switzerland, and United Kingdom Consortium (Access): Re-Imagining Regulatory Collaboration

Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-632-L04-P; CME 1.00; RN 1.00

Established in 2007, the Access Consortium brings together like-minded regulatory agencies to promote regulatory convergence and foster synergy to address scientific and regulatory issues. In this session, leaders from the Access Consortium will provide an update on achievements using work-sharing initiatives for timely medicines approvals for our populations, enabling industry to bring new, innovative products through Access pathways.

Learning Objectives

Discuss pathways and opportunities for industry to bring innovative products through Access; Describe new tools and methodologies to optimize regulatory collaboration under the Access Consortium to support access to medicines.

Chair

Pamela Aung-Thin

Speaker

Panelist
June Raine, MD, MSc, FRCP

Panelist
Anthony Lawler, MD, MBA

Panelist
Mimi May Ling Choong, DrMed, MD

Panelist
Jörg Schläpfer, PhD

Panelist
Eveline Trachsel

Speakers

Pamela Aung-Thin

Assistant Deputy Minister of the Health Products and Food Branch, Health Canada, Canada

Pamela Aung Thin became the Assistant Deputy Minister of the Health Products and Food Branch (HPFB) on March 27, 2023.

Mimi May Ling Choong

Chief Executive Officer, Health Sciences Authority (HSA), Singapore

Dr Choong was appointed as the Chief Executive Officer of the Health Sciences Authority (HSA) of Singapore on 1 July 2014. She oversees HSA’s wide-ranging public health responsibilities, comprising health products regulation, the national blood service, the national analytical and... Read More →

Anthony Lawler

Deputy Secretary, Health Products Regulation Group, Therapeutic Goods Administration, Australia

Professor Tony Lawler is the Head of the Australian Therapeutic Goods Administration, which works to safeguard and enhance the health of all Australians through effective, timely and risk-proportionate regulation of therapeutic goods, and the control of drug imports, exports and production... Read More →

June Raine

Chief Executive, Medicines and Healthcare Products Regulatory Agency (MHRA), United Kingdom

Dr June Raine DBE is CEO of the Medicines and Healthcare products Regulatory Agency. She trained in medicine in Oxford after completing a master’s degree by research in Pharmacology. Her interest in drug safety led to a career in medicines regulation which has spanned a number of... Read More →

Jörg Schläpfer

Head of Management Services and International Affairs, Swissmedic, Switzerland

Dr. Jörg Schläpfer, PhD is the Head of Management Services & International Affairs and Member of the Management Board of Swissmedic. He studied Veterinary Medicine at the University of Berne, completing his studies in 1991. After obtaining the title of Doctor of Veterinary Medicine... Read More →

Eveline Trachsel

Head of Medicinal Product Authorisation and Vigilance, Swissmedic, Switzerland

Wednesday June 19, 2024 8:00am - 9:00am PDT
Room 6B San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

09: Regulatory, Forum

Level Intermediate
Featured Topics Regulatory Collaboration, Regulator Perspectives
Level Intermediate
Feature Topics Regulatory Collaboration,Regulator Perspectives
Credit Type ACPE, CME, RN
Tags Forum

8:00am PDT

#307: Partnering with Regulatory Authorities to Unlock the Value in Digital Health Products

Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-628-L04-P; CME 1.00; RN 1.00

Discussion of partnering with regulators to get novel digital health products through the approval processes. Learn insights from industry and regulators for creative, strategic, and flexible thinking on regulation of novel technologies.

Learning Objectives

Discuss the challenges of introducing novel digital technologies to FDA; Describe tactics to build better partnerships with regulators.

Chair

Rebecca Joy James, MBA, RAC

Speaker

Panelist
Anindita Saha

Panelist
James Wabby, MHS

Panelist
Adam Cargill, MS

Panelist
Wes Gerbig, PhD, MS

Speakers

Rebecca James

Director - Advisory, KPMG, LLC, United States

Rebecca is a Director in the Life Sciences consulting group at KPMG based out of Chicago, IL. She has over 14 years of experience in both consulting and industry roles with companies from pharmaceutical, medical device, and nutritional industries with an emphasis in Regulatory Affairs... Read More →

Adam Cargill

Director, QARACA, MediView XR, Inc., United States

Adam Cargill, MS RAC, is the Vice President of Quality, Regulatory, and Clinical for MediView XR. In his role he is accountable for the oversight of all regulatory, clinical, and quality functions. Responsibilities include overseeing the company’s global regulatory, clinical, and... Read More →

Wes Gerbig

Director, Global Regulatory Affairs, Drug Delivery and Digital Health, Eli Lilly and Company, United States

Anindita Saha

Associate Director Strategic Initiatives, Digital Health Center of Excellence, FDA, United States

James Wabby

Global Head, Regulatory Affairs, Emerging Technologies and Combination Products, AbbVie, United States

James Wabby is the Global Head, Regulatory Affairs - (CoE) Emerging Technologies, Devices, and Combination Products at AbbVie in Irvine, California. He has 22 years of experience in increasing quality compliance and regulatory affairs responsibilities within the GxP regulated environment... Read More →

Wednesday June 19, 2024 8:00am - 9:00am PDT
Room 2 San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

09: Regulatory, Session | 06: TranslationalDelivery Tech-PrecMedicine, Session

Format Session
Level Intermediate
Featured Topics Digital Technology
Level Intermediate
Feature Topics Digital Technology
Credit Type ACPE, CME, RN
Tags Session

10:00am PDT

#326: Bringing Transformational Treatments to Patients: Regulatory Convergence and Reliance on Cell and Gene Therapy Products

Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-644-L04-P; CME 1.00; RN 1.00

In this session, we will discuss international efforts to promote regulatory convergence and expand the use of reliance to bring these highly complex therapies to market earlier, particularly in less developed countries.

Learning Objectives

Describe how international harmonization, collaboration, and reliance principles can be effectively applied to the regulation of cell and gene therapies; Discuss the key considerations and regulatory challenges associated with bringing complex, innovative medicines such as cell and gene therapy products to less experienced, emerging markets.

Chair

Ginny Beakes-Read, BSN, JD

Speaker

FDA Update
Julia Tierney, JD

WHO Update
Samvel Azatyan, MD, PhD

PMDA Update
Yasuhiro Kishioka, PhD

Industry Update
Maria Antonieta Tony Roman, MPharm

EMA Update
Anabela Marcal, PharmD

Speakers

Samvel Azatyan

Team Lead, Regulatory Convergence and Networks (RCN/REG), World Health Organization (WHO), Switzerland

Ginny Beakes-Read

Head, Global Regulatory Policy and Intelligence, Johnson & Johnson Innovative Medicine, United States

Ms. Beakes-Read is VP, Global Regulatory Policy and Intelligence at Johnson & Johnson. She leads the policy group, which works to shape the regulatory environment to support innovative drug development and patient access to new therapies. Before that, Ginny was at Amgen leading the... Read More →

Yasuhiro Kishioka

Reviewer Director, Office of Cellular and Tissue-based Products, Pharmaceuticals and Medical Devices Agency (PMDA), Japan

Dr. Kishioka is a Review Director of the Office of Cellular and Tissue-based Products, PMDA. In this role, he is responsible for the assessment of gene therapy products and the quality of biotechnological/biological products. Since joining PMDA in 2008, Dr. Kishioka has been involved... Read More →

Anabela Marcal

EMA Liaison Official to the US FDA, European Medicines Agency, Netherlands

Maria Antonieta Roman

Head Regulatory Policy Emerging Markets LATAM, Novartis, Mexico

Degree in Pharmacy, Master of Science (pharmacy) UNAM; Diploma in Clinical development and regulatory affairs, Universidad Anahuac; diploma in public health by the Swiss School of Public Health. 32 years of experience in various areas in the Pharmaceutical industry like R&D, quality... Read More →

Julia Tierney

Deputy Center Director for Strategy, Policy, and Legislation, CBER, FDA, United States

JULIE TIERNEY is the Deputy Center Director for Strategy, Policy and Legislation for the Food and Drug Administration’s (FDA’s) Center for Biologics Evaluation and Research (CBER). Ms. Tierney has held several senior roles in the Office of the Commissioner and CBER during her... Read More →

Wednesday June 19, 2024 10:00am - 11:00am PDT
Room 6C San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

05: Patient-Focused Drug Dev, Session | 09: Regulatory, Session

Format Session
Level Intermediate
Featured Topics Cell and Gene Therapy, Regulatory Reliance, Regulatory Collaboration
Level Intermediate
Feature Topics Cell and Gene Therapy,Regulatory Reliance,Regulatory Collaboration
Credit Type ACPE, CME, RN
Tags Session

10:00am PDT

#328: Pioneering New Frontiers: Advanced Drug Delivery Technologies and Cell/Gene Therapies in Combination Products

Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Application UAN: 0286-0000-24-646-L04-P; CME 1.00; RN 1.00

Advanced Therapies and combined advanced therapies emerging as innovative medical products due to their contribution to advancing medical care and are thus expected to have major impact in the coming years. Future technologies are most appealing to patients with ongoing medical conditions that require consistent treatment with daily injections or weekly procedures and unmet medical needs. Overall, the successful development of combination products will require great collaboration within the industry to overcome regulatory, clinical, and technical challenges. When developing an advanced therapy product, there are many things to be considered – relationships between tissue, biologic, and device development as well as early establishment of regulatory and clinical strategies, understanding user needs, determining product requirements, as well as device manufacturing variation. With the dawn of a new millennium and the rapidly aging baby-boomer population, the pressure for companies to create safer and more effective treatments is growing. Advanced therapies are one of the solutions that have emerged to meet the more rigorous demands of patients. Drug-enhanced devices, including drug eluting stents and antimicrobial-coated catheters, have already proven themselves on the market. Pre-filled syringes and transdermal patches, which are novel drug delivery systems, have also been widely used treatments. However, advanced therapies and combined advanced therapies are innovative products and could provide solutions as well as cures for previously untreatable conditions, which can truly change the face of the industry. Speakers will be FDA and industry leaders and FDA illustrating hot topics and example case studies in a panel discussion for us to navigate the future regulatory framework for unmet medical needs and next generation of medical products.

Learning Objectives

Analyze latest regulatory expectations and challenges; Identify potential future innovation platforms; Acknowledge potential and future global convergence/harmonization initiatives for combination products; Describe the regulatory challenges and opportunities ahead.

Chair

James Wabby, MHS

Speaker

FDA Update
Andrea Gray, PhD

Panelist
Rob Schulz

Panelist
William Daunch, PhD

Panelist
Michael Lehmicke, MSc

Speakers

James Wabby

Global Head, Regulatory Affairs, Emerging Technologies and Combination Products, AbbVie, United States

William Daunch

Chief Technology Officer, Focal Medical, Inc., United States

Professionally trained polymer scientist + analytical chemist. Began career as an industrial chemist/materials scientist in Belgium (ICI). Moved to Scotland and applied skills to helping early stage company (Aortech) develop the first fully synthetic heart valve (now commercialized... Read More →

Andrea Gray

Biomedical Engineer Advisor, CBER, FDA , United States

Andrea Gray is a Biomedical Engineer Advisor in the Regulatory Operations Branch of the Division of Regulatory Operations and Programs (DROP) in the Office of Regulatory Operations (ORO), Center for Biologics Evaluation and Research (CBER). She specializes in regulatory review of... Read More →

Michael Lehmicke

Senior Vice President, Science and Industry Affairs, Alliance for Regenerative Medicine, United States

Michael has over 20 years of R&D experience in biomaterials, medical devices and regenerative medicine. He has led product development teams for class II devices, human cell and tissue-based products, and drug/device combination products. He is a creator and an inventor with multiple... Read More →

Rob Schulz

President and COO, Suttons Creek, Inc., United States

Wednesday June 19, 2024 10:00am - 11:00am PDT
Room 6A San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

06: TranslationalDelivery Tech-PrecMedicine, Forum | 09: Regulatory, Forum | 14: DIAmond, Forum

Format Forum
Level Intermediate
Featured Topics Cell and Gene Therapy
Level Intermediate
Feature Topics Cell and Gene Therapy
Credit Type ACPE, CME, RN
Tags Forum

10:00am PDT

#325: A Paradigm Shift in Global Regulatory Reviews: Has the New Normal Arrived?

Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-643-L04-P; CME 1.00; RN 1.00

Global expert presentations and in-depth panel discussion on the regulatory agilities, regulatory reliance, platform approaches, and other regulatory innovations that are pivotal during public health emergencies and necessary for future pandemic preparedness.

Learning Objectives

Discuss regulatory agilities, reliance and platform approaches which significantly decreased review times internationally for COVID vaccines. Discuss additional CEPI case studies that highlight regulatory innovations to address future public health emergencies, and the inherent complexities of sustaining these innovations from a health authority perspective.

Chair

Helen Bayes Hartman, PhD

Speaker

Regulatory Reliance and Platform Approaches: Has the New Normal Arrived??
Donna Boyce, MS, RAC

Preparedness: Challenging the Traditional Regulatory Paradigms to Address Public Health Emergencies
Debra A Yeskey

FDA Update
David C. Kaslow, MD

Moderator
Khushboo Sharma, MBA

Speakers

Helen Hartman

Director, Global Regulatory Sciences, Pfizer Inc, United States

Helen has over 15 year of drug development experience as a global regulatory strategist and regulatory intelligence/policy professional across multiple therapeutic areas.

Donna Boyce

Head and Senior Vice President of Global Regulatory Sciences, Pfizer Inc, United States

David Kaslow

Director, Vaccines Research and Review (OVRR), CBER, FDA, United States

David is director of the Office of Vaccines Research and Review, CBER, US FDA, where he oversees regulation of vaccines, allergenics, live biotherapeutics and bacteriophage. His 35+ years of experience in use-inspired research, product development, and product introduction include... Read More →

Khushboo Sharma

Chief Regulatory Innovation Officer, Accumulus Synergy, United States

Khushboo Sharma is the Chief Regulatory Innovation Officer for Accumulus. Sharma joined Accumulus Synergy in 2022 from BIO, where she focused on major regulatory policy initiatives to build the appropriate regulatory framework. Prior to BIO, Sharma spent 11 years in various roles... Read More →

Debra Yeskey

Head of Regulatory Policy and Intelligence, CEPI, United States

Wednesday June 19, 2024 10:00am - 11:00am PDT
Room 11AB San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

09: Regulatory, Forum

Format Forum
Level Intermediate
Level Intermediate
Credit Type ACPE, CME, RN
Tags Forum

11:00am PDT

#329 RT: Roundtable Discussion: The Evolving Role of Artificial Intelligence in the Medicinal Product Lifecycle

Component Type: Session

Join the Regulatory Affairs Community for a follow up round table discussion tied to session: The Evolving Role of Artificial Intelligence in the Medicinal Product Lifecycle (Tuesday, June 18 | 1:15PM - 2:15 PM PDT). Space is limited.

Chair

Brooke Casselberry, MS, RAC

Speakers

Brooke Casselberry

Vice President, Advisory and Delivery, Epista Life Sciences, United States

Brooke is known for her pivotal roles in collaborating with Sponsor Companies, Health Authorities, and Technology Developers. She has maintained focus on leveraging cutting-edge technologies as a catalyst for regulatory advancements, optimization, and collaboration for global go-to-market... Read More →

Wednesday June 19, 2024 11:00am - 12:00pm PDT
Ballroom 20 A/ Zone 1 San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

16: Community Rounds, Session | 03: Data-Tech, Session | 09: Regulatory, Session

Tags Session

11:00am PDT

#330 RT: Roundtable Discussion: Securing the Chain - US and EU Legislative Reforms and Regulatory Actions for Drug Shortage Mitigation

Component Type: Session

Join the Meaningful Change Working Group Community for a follow up round table discussion tied to session: Securing the Chain - US and EU Legislative Reforms and Regulatory Actions for Drug Shortage Mitigation (Tuesday, June 18 | 3:15 PM - 4:15 PM PDT). Space is limited.

Chair

Chaitanya Koduri

Speakers

Chaitanya Koduri

Director, International Government and Regulatory Engagement, US Pharmacopeia, United States

Wednesday June 19, 2024 11:00am - 12:00pm PDT
Ballroom 20 BC/ Zone 2 San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

16: Community Rounds, Session | 10: RegCMC-Product Quality, Session | 09: Regulatory, Session

Tags Session

1:15pm PDT

#345: Regulatory Considerations in Trial Design for Cell and Gene Therapies

Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-648-L04-P; CME 1.00; RN 1.00

Cell and gene therapies have enormous potential to address unmet medical need. The application of validated clinical trials design tools along with biomarkers to assess safety and endpoints can help address regulatory uncertainty.

Learning Objectives

Discuss regulatory considerations for cell and gene therapies including the use of data modeling in clinical trial design and the application of biomarkers both for safety assessment and measures of outcome; Discuss how to plan for meeting regulatory requirements and be able to incorporate these plans into your company’s drug development plans.

Chair

Kanwaljit Singh, MD, MBA, MPH

Speaker

Regulatory Considerations for Development of Pediatric Cell and Gene Therapies: FDA Update
Najat Bouchkouj, MD

Regulatory Consideration for Pediatric Cell and Gene Therapies - Industry Perspective
Paulla Dennis

Path from Bench-to-Bedside for a Cell and Gene Therapy Drug Product
Stephanie Cherqui, PhD

Speakers

Najat Bouchkouj

Associate Director for Pediatrics (Acting), Office of Clinical Evaluation, FDA, United States

Dr. Najat Bouchkouj is the Acting Associate Director for Pediatrics in the Office of Clinical Evaluation, OTP, CBER, FDA. Dr. Bouchkouj joined the FDA in 2016. She earned her medical degree from Damascus University, Syria and completed a residency in Pediatrics at the State University... Read More →

Stephanie Cherqui

Director, UC San Diego Gene Therapy Initiative, University of California San Diego, United States

Stephanie Cherqui is Professor in the Department of Pediatrics, Division of Genetics at the University of California San Diego. She is also the Director of the UC San Diego Gene Therapy Initiative. Her laboratory primarily centers on two research areas: the development of hematopoietic... Read More →

Paulla Dennis

Senior Director, Rare Diseases, Advanced Therapies, and Pediatrics, Fortrea, United States

• Paulla Dennis, Senior Director, is a leading member of the Rare Disease, Advanced Therapies, and Paediatric Team (RAPT) with over 20 years in the clinical research industry. Paulla has expertise in global phase I through IV pediatric and rare disease operational strategy and planning... Read More →

Kanwaljit Singh

Executive Director, International Neonatal Consortium, Critical Path Institute, United States

Wednesday June 19, 2024 1:15pm - 2:15pm PDT
Room 5AB San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

06: TranslationalDelivery Tech-PrecMedicine, Session | 09: Regulatory, Session | 02: ClinTrials -ClinOps, Session

Format Session
Level Intermediate
Featured Topics Cell and Gene Therapy
Level Intermediate
Feature Topics Cell and Gene Therapy
Credit Type ACPE, CME, RN
Tags Session

1:15pm PDT

#352: Applying Machine Learning and Artificial intelligence for Predicting Product Profile Approvability (PoPPA)

Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Application UAN: 0286-0000-24-655-L04-P; CME 1.00; RN 1.00

This will be an interactive panel discussion with experts sharing examples how AI/ML can be used to create solutions for better regulatory decision-making and predicting regulatory success. Risks and benefits of using this approach will be discussed.

Panelists will include world-renowned experts on drug development, drug regulations, decision science and data analytics.

Learning Objectives

Evaluate how AI/ML can be used to help with regulatory decision making; Analyze scenarios using case studies by world’s leading expert on drug development, cloud computing, and predicting regulatory success; Compare risks and benefits for using this evolving technology.

Chair

Romi Singh, PhD

Speaker

Panelist
Kilian Weiss

Panelist
Subha Madhavan, PhD, FACMI

Panelist
Lily Li, JD

Speakers

Romi Singh

Head, Regional Regulatory Sciences, Asia, Pfizer Inc, United States

Dr. Rominder (Romi) Singh has extensive scientific and management experience at top biopharmaceutical companies--Pfizer, Amgen, Merck and GlaxoWellcome. His experience spans across drug development—drug discovery to commercialization. He is recognized as an expert on international... Read More →

Lily Li

Founder and President, Metaverse Law, United States

Lily Li is the founder and president of Metaverse Law, a firm that focuses exclusively on privacy, AI, and cybersecurity law. She advises mid-sized, public, and multinational clients on a broad range of AI and data protection matters, such as the CCPA, GDPR, NIST AI Framework, Biden... Read More →

Subha Madhavan

Vice President and Head of AI/ML, Global Biometrics and Data Management, Pfizer Inc, United States

Subha Madhavan heads up AI/ML, Quantitative and Digital Sciences within Global Product Development at Pfizer. Prior to joining Pfizer, she was Head of Data Science and AI within Oncology R&D Strategy at AstraZeneca Pharmaceuticals. She has initiated and successfully directed several... Read More →

Kilian Weiss

General Manager, Veeva Link, Veeva, Germany

As general manager of Veeva Link, Kilian is responsible for the strategic direction, growth, and customer success of Veeva’s global Link Platform. He is also responsible for Veeva’s European Data business and commercial strategy. Before joining Veeva, Kilian founded Mederi, a... Read More →

Wednesday June 19, 2024 1:15pm - 2:15pm PDT
Room 6F San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

09: Regulatory, Forum

Format Forum
Level Intermediate
Featured Topics Artificial Intelligence (AI)
Level Intermediate
Feature Topics Artificial Intelligence (AI)
Credit Type ACPE, CME, RN
Tags Forum

1:15pm PDT

#353: ICH M15 Model Informed Drug Development: Steps Toward Harmonized Guidance

Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-656-L04-P; CME 1.00; RN 1.00

Model Informed Drug Development (MIDD) can enhance the efficiency of drug development and regulatory decision-making. The ICH M15 guideline aims to enable the further integration and consistent use of MIDD. This session will cover the draft guideline in greater detail.

Learning Objectives

Discuss the draft guideline and what may be new to drug developers and the regulatory community, including the concept of a risk-based assessment, the value of multidisciplinary team alignment on MIDD strategy, and early alignment with regulators; Identify current approaches, best practices, and future opportunities to enhance development and decision-making.

Chair

Erin Greene, JD

Speaker

Industry Perspective
Malidi Ahamadi

Regulatory Perspective
Hao Zhu, PhD

Industry Perspective
Mark C. Peterson, PhD

Speakers

Erin Greene

US Lead, Global Regulatory Policy and Innovation, Takeda, United States

Experienced leader in Global Regulatory Policy and Intelligence with a demonstrated history of working in the pharmaceutical and healthcare industries. Graduated from American University, Washington College of Law with an emphasis on FDA regulation and health law and policy.

Malidi Ahamadi

US Head, Modeling and Simulation, Sanofi, United States

Mark Peterson

Senior Director, Modeling and Simulation, Vertex Pharmaceuticals, United States

Dr. Mark C. Peterson works at Vertex Pharmaceuticals as a Senior Director in Clinical & Quantitative Pharmacology. Prior to that, he was at Pfizer (9 years), Biogen (2 years) and Amgen (8 years). Dr. Peterson received a Bachelor of Sciences in Pharmacy (1992) and a Doctorate in Pharmaceutical... Read More →

Hao Zhu

Division Director, Division of Pharmacometrics, OCP, OTS, CDER, FDA, United States

Wednesday June 19, 2024 1:15pm - 2:15pm PDT
Room 16AB San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

09: Regulatory, Session

Format Session
Level Intermediate
Featured Topics ICH, Risk Management
Level Intermediate
Feature Topics ICH,Risk Management
Credit Type ACPE, CME, RN
Tags Session

1:15pm PDT

#354: ICMRA Post-Pandemic: Regulators Looking into the Future

Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-657-L04-P; CME 1.00; RN 1.00

ICMRA regulators will share their views on the opportunities and challenges brought by some of the main innovative concepts and technologies they are facing, including artificial intelligence, evolving clinical trials, and real-world evidence in the authorization of advanced therapy medical products (ATMPs).

Learning Objectives

Identify how regulators are working on maximizing the opportunities offered by artificial intelligence while minimizing the risks; Discuss new concepts for enhanced clinical trials (e.g.: platform CTs, decentralized CTs); Describe opportunities and challenges brought by the use of real-world evidence data for authorization of advanced therapy medical products.

Chair

Emer Cooke, MBA, MSc

Speaker

Panelist
Anthony Lawler, MD, MBA

Panelist
Antonio Barra Torres, MD

Panelist
Lorraine Nolan, PhD

Panelist
Daisuke Koga, MSc, RPh

Panelist
Robert M. Califf, MD

Speakers

Emer Cooke

Chair, ICMRA; Executive Director, European Medicines Agency, Netherlands

Anthony Lawler

Deputy Secretary, Health Products Regulation Group, Therapeutic Goods Administration, Australia

Antonio Barra Torres

President Director, ANVISA, Brazil

Lorraine Nolan

Chief Executive, Health Products Regulatory Authority , Ireland

Dr Nolan joined the HPRA in 2001 and since that time has held a number of different roles at senior level within the organisation across a range of areas including inspection and authorisation of human medicines, medical devices, cosmetics and controlled substances. Prior to assuming... Read More →

Daisuke Koga

Director, Office of International Regulatory Affairs, Ministry of Health, Labour and Welfare (MHLW), Japan

Robert Califf

Commissioner, FDA, United States

Dr. Robert M. Califf was confirmed as the 25th Commissioner of Food and Drugs. He also served in 2016 as the 22nd Commissioner, and immediately prior to that as the FDA’s Deputy Commissioner for Medical Products and Tobacco. He has spent a good portion of his career affiliated with... Read More →

Wednesday June 19, 2024 1:15pm - 2:15pm PDT
Room 6A San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

09: Regulatory, Session

Format Session
Level Intermediate
Featured Topics Decentralized Clinical Trials (DCTs), Real-World Data (RWD)- Real-World Evidence (RWE)
Level Intermediate
Feature Topics Decentralized Clinical Trials (DCTs),Real-World Data (RWD)- Real-World Evidence (RWE)
Credit Type ACPE, CME, RN
Tags Session

1:15pm PDT

#349: Study Design for Cell and Gene Therapy Trials: Regulatory Overview, Challenges, and Updates

Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-652-L04-P; CME 1.00; RN 1.00

This forum provides a general view and challenges of the study design in cell and gene therapy trials. A panel of experts interpret FDA guidance, share their own experience for study design, and discuss the trends in this area.

Learning Objectives

Describe regulatory principles of the trial design for cell and gene therapy products; Identify specific design elements such as selection of study population; Discuss the selection of control, safety monitoring; Discuss challenges and new trends.

Chair

Yao-Yao Zhu, MD, PhD

Speaker

Panelist
Katherine A. High, MD

Panelist
Vaishali Popat, MD, MPH

Clinical Considerations for Oncology Cell and Gene Therapy Trials
Ke Liu, MD, PhD

Speakers

Yao-Yao Zhu

Regulatory Affairs Director, AstraZeneca, United States

Yao-Yao Zhu, MD, PhD, has over 15 years of regulatory experience. She is currently a Regulatory Affairs Director for cardiovascular, renal, and metabolic diseases at AstraZeneca (AZ). Prior to AZ, she was a head of Global Regulatory Affairs at Biosion for antibody products in oncology... Read More →

Katherine High

Co-Founder and Former President, Sparks Therapeutics; Visiting Professor, Rockefeller University, United States

Dr. Katherine High is the former President at Spark Therapeutics, a biotech company that she co-founded in 2013. Under Dr. High’s leadership, Spark received FDA approval of the first AAV gene therapy product in the US, a treatment for a rare form of congenital blindness. Dr. High... Read More →

Ke Liu

Chief Development Officer, Marengo Therapeutics, United States

As Chief Development Officer of Marengo Therapeutics, Ke Liu joins Marengo with over 20 years of experience in the field of oncology, immuno-oncology, and cell and gene therapy, most recently serving as Senior Vice President and led the key regulatory activities at Sana Biotechnology... Read More →

Vaishali Popat

Branch Chief, Office of Clinical Evaluation,Office of Therapeutic Products, CBER, FDA, United States

Dr. Vaishali Popat is an Associate Director for Biomedical Informatics in the Office of New Drugs, Immediate Office. Dr. Popat is the OND lead on biomedical informatics issues in areas such as safety analytics, staff training on the use of new review tools to make the evaluation of... Read More →

Wednesday June 19, 2024 1:15pm - 2:15pm PDT
Room 15AB San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

09: Regulatory, Forum | 02: ClinTrials -ClinOps, Forum | 04: MedAffairs-SciComm, Forum

Format Forum
Level Intermediate
Featured Topics Regulator Perspectives, Cell and Gene Therapy
Level Intermediate
Feature Topics Regulator Perspectives,Cell and Gene Therapy
Credit Type ACPE, CME, RN
Tags Forum

1:15pm PDT

#347: Artificial Intelligence in the Drug Development Lifecycle: Opportunities and Challenges in Regulatory Operations

Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-650-L04-P; CME 1.00; RN 1.00

This panel will discuss opportunities and challenges of leveraging artificial intelligence (AI) in the drug development lifecycle, especially in regulatory operations. The panel will include representation from regulatory, and industry.

Learning Objectives

Identify the impact of artificial intelligence (AI) in the drug development lifecycle from health authorities' and, industry's perspectives; Recognize opportunities for the regulators and industry to leverage rapid advancement of AI to the drug development lifecycle; Discuss challenges to implement changes from policy, technology, standard, data security in regulatory operations.

Chair

Ethan Chen, MBA, MS, PMP

Speaker

The Evolving Role of Artificial Intelligence in the Medicinal Product Lifecycle
Hilmar Hamann, PhD

Panelist
Tala Fakhouri, PhD, MPH

Panelist
Nicole Mahoney, PhD

Speakers

Ethan Chen

Director, Division of Data Management Services and Solutions, OBI, OSP, CDER, FDA, United States

Ethan Chen provides overall leadership to CDER in streamlining electronic and traditional submissions and delivering solutions to enable rapid adoption of emerging electronic data standards. Since joining the FDA in 2012, Mr. Chen has led several critical initiatives as the CDER Informatics... Read More →

Tala Fakhouri

Associate Director for Policy Analysis, Office of Medical Policy, CDER, FDA, United States

Hilmar Hamann

Head of Information Management, European Medicines Agency, Netherlands

Nicole Mahoney

Executive Director US Regulatory Policy & Intelligence, Novartis Pharmaceuticals Corporation, United States

Nicole Mahoney is an Executive Director for Regulatory Policy at Novartis, and global regulatory policy lead for data and digital technologies, including real-world evidence and artificial intelligence. Prior to Novartis, she led Regulatory Policy at Flatiron Health, helping support... Read More →

Wednesday June 19, 2024 1:15pm - 2:15pm PDT
Room 6B San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

09: Regulatory, Forum | 03: Data-Tech, Forum

Format Forum
Level Intermediate
Featured Topics Artificial Intelligence (AI), Regulator Perspectives
Level Intermediate
Feature Topics Artificial Intelligence (AI),Regulator Perspectives
Credit Type ACPE, CME, RN
Tags Forum

1:15pm PDT

#350: ICH Efforts to Incorporate Patient’s Perspective to Enhance Quality, Relevance, Safety and Efficacy of Drug Development

Component Type: Session
Level: Basic
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-653-L04-P; CME 1.00; RN 1.00

Discussion of ICH work to harmonize recommendations for inclusion of patient’s perspective in drug development. Patient group representatives will also highlight key considerations for clinical research and development of international standards.

Learning Objectives

Discuss ICH’s portfolio for inclusion of patient’s perspective in drug development and outline specific case examples; Describe the patient perspective in clinical research and identify key considerations that may be relevant for the international community in developing standards; Evaluate linkages between ICH’s mission and key interests of patient, health care professional, and life science communities.

Chair

Amanda Marie Roache, MS

Speaker

Moderator
Theresa Mullin, PhD

ICH E22 Patient Preference Studies
Brett Hauber, PhD, MA

ICH Patient Engagement
Francesco Pignatti, MD

Patient Perspective on Engagement to Inform Drug Development
Nicholas Brooke, MBA

Speakers

Nicholas Brooke

Executive Director, Patient Focused Medicines Development (PFMD), PFMD, Belgium

Nicholas Brooke is the Founder and Executive Director of The Synergist, an incubator and managing organization to multi-stakeholder platforms that brings key players together with the express aim of solving significant societal problems through collective actions. The Synergist also... Read More →

Brett Hauber

Senior Director, Patient Preference Elicitation, Pfizer Inc, United States

Theresa Mullin

Associate Center Director - Strategic Initiatives, CDER, FDA, United States

Dr. Mullin is Associate Center Director for Strategic Initiatives. She leads various efforts including Patient-Focused Drug Development and CDER’s International Program. She leads the FDA delegation to ICH and currently chairs the ICH Management Committee. She led FDA negotiations... Read More →

Francesco Pignatti

Scientific Adviser for Oncology, European Medicines Agency, Netherlands

Amanda Roache

Senior Director, Science and Regulatory Advocacy, Pharmaceutical Research and Manufacturers of America (PhRMA), United States

Wednesday June 19, 2024 1:15pm - 2:15pm PDT
Room 11AB San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

09: Regulatory, Session | 05: Patient-Focused Drug Dev, Session

Format Session
Level Basic
Featured Topics Patient Focused, ICH
Level Basic
Feature Topics Patient Focused,ICH
Credit Type ACPE, CME, RN
Tags Session

2:15pm PDT

#357 RT: Roundtable Discussion: Leveraging Cloud-Based Technologies to Accelerate Reliance and Convergence - An Industry and Regulatory Agency Experience

Component Type: Session

Join the Digital Acceleration Community for a follow up round table discussion tied to session: Leveraging Cloud-Based Technologies to Accelerate Reliance and Convergence - An Industry and Regulatory Agency Experience (Wednesday, June 19 | 10:00AM - 11:00AM PDT). Space is limited.

Chair

Cesar Vinces

Speakers

Cesar Vinces

EU Interim and International Policy Lead, Accumulus Synergy, United States

Cesar serves as Interim EU & International Policy Lead at Accumulus Synergy within Regulatory Innovation. He engages with industry experts, national regulators, and global organizations to foster policy changes and develop strategies that enhance regulatory frameworks, promoting industry... Read More →

Wednesday June 19, 2024 2:15pm - 3:15pm PDT
Ballroom 20 BC/ Zone 2 San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

16: Community Rounds, Session | 09: Regulatory, Session | 03: Data-Tech, Session

Tags Session

3:15pm PDT

#366: The Intersection of Patient-Experience Data and Benefit-Risk Analysis

Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-666-L04-P; CME 1.00; RN 1.00

This session will explore how patient experience data (PED) can be used to inform benefit-risk analysis. The panel will include discussion on regulators’ views and incorporation of PED and successful case examples.

Learning Objectives

Describe the role patient experience data (PED) plays in benefit-risk analysis; Identify how to develop PED in a way that can inform regulatory decision-making; Discuss if and how new diversity and inclusion regulations will impact the development of PED and regulators’ interpretation of benefit-risk.

Chair

Allison Martin, MS
Victoria DiBiaso, BSN, MPH, RN

Speaker

The Intersection of Patient-Experience Data and Benefit-Risk Analysis: FDA Update
Michelle Campbell, PhD

The Intersection of Patient-Experience Data and Benefit-Risk Analysis: EMA Update
Francesco Pignatti, MD

The Intersection of Patient-Experience Data and Benefit-Risk Analysis: Industry Update
Jill Yersak

The Intersection of Patient-Experience Data and Benefit-Risk Analysis: patient Update
Hollie Schmidt, MS

Speakers

Allison Martin

Director, Regulatory Science and Policy, North America, Sanofi, United States

As a Director on Sanofi’s Regulatory Science and Policy North America team, Allison is responsible for advancing regulatory science and strategically engaging in regulatory policy efforts. In this role, Ms. Martin collaborates with internal and external stakeholders to identify... Read More →

Michelle Campbell

Associate Director, Stakeholder Engagement and Clinical Outcomes, ON, OND, CDER, FDA, United States

Michelle Campbell is the Associate Director for Stakeholder Engagement and Clinical Outcomes in the Office of Neuroscience, Office of New Drugs (OND), Center for Drug Evaluation and Research (CDER), U.S. Food and Drug Administration (FDA). Dr. Campbell joined FDA in 2014 and was previously... Read More →

Victoria DiBiaso

Global Head, Patient Informed Development and Health Value Translation, Sanofi, France

Vicky has 25+ years of clinical research experience. She holds a Master of Public Health, is a nurse by training. Her work has been published in The Wall Street Journal, she has been recognized as one of the Top 20 Industry Innovators, through integrating patient communities into... Read More →

Francesco Pignatti

Scientific Adviser for Oncology, European Medicines Agency, Netherlands

Hollie Schmidt

Vice President of Scientific Operations, Accelerated Cure Project, United States

Hollie Schmidt is the VP of Scientific Operations at the Accelerated Cure Project, a research-focused patient advocacy organization for MS. She has planned, developed, and directed numerous initiatives aimed at creating research resources and bridging different stakeholder communities... Read More →

Jill Yersak

Head, US ALS Patient Advocacy and Engagement, Amylyx Pharmaceuticals, United States

Wednesday June 19, 2024 3:15pm - 4:15pm PDT
Room 5AB San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

05: Patient-Focused Drug Dev, Session | 09: Regulatory, Session

Format Session
Level Intermediate
Featured Topics Diversity Equity and Inclusion (DEI), Patient Focused, Risk Management
Level Intermediate
Feature Topics Diversity Equity and Inclusion (DEI),Patient Focused,Risk Management
Credit Type ACPE, CME, RN
Tags Session

3:15pm PDT

#365: Embedding Diversity and Inclusion into Global Clinical Research: Where Have we Been and Where are we Going?

Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-665-L04-P; CME 1.00; RN 1.00

Adequate enrollment of underrepresented groups continues to be a challenge in a complex, global regulatory and research ecosystem. Review and assess evolving expectations and processes in the US, Canada, and other regulatory systems, what has been implemented, what adjustments have been made, what has worked, and what hurdles or differences remain.

Learning Objectives

Describe the background and underlying requirements to FDA’s and other health authorities’ diversity and inclusion policies and guidance; Discuss how different stakeholders have implemented and reacted to implementation of new and evolving policies; Identify areas where views or expectations diverge; Discuss and describe what has worked, what has not; Identify how this area might change in future.

Chair

Alexis Miller, JD

Speaker

FDA Update
Karen Hicks, DrMed

Health Canada Update
Alysha Croker, PhD

UK Update
Naho Yamazaki, PhD

Patient Update
Loriana Hernandez-Aldama

Speakers

Alexis Miller

Acting Global Lead, Global Regulatory Policy and Intelligence, Merck & Co., Inc., United States

Alysha Croker

Director, Centre for Policy, Pediatrics and International Collaboration, BRDD, Health Canada, Canada

Loriana Hernandez-Aldama

Patient Advocate|2x Survivor| Founder, ArmorUp for Life, Armorup For Life, United States

Loriana is a 2x cancer survivor, international motivational speaker, award-winning journalist, podcast host, and author of "Becoming the Story: The Power of Prehab." Bold, transparent, and honest, Loriana has emerged as a game-changer in healthcare, advocating globally for transformative... Read More →

Karen Hicks

Deputy Director, Office of Medical Policy, CDER, FDA, United States

Dr. Hicks is the Deputy Director of the Office of Medical Policy in the Center for Drug Evaluation and Research (CDER) at the United States Food and Drug Administration (FDA). She oversees regulation and policy development for CDER and participates in cross-cutting agency initiatives... Read More →

Naho Yamazaki

Deputy Director, Policy and Partnerships, Health Research Authority, United Kingdom

Naho is Deputy Director, Policy and Partnerships at the Health Research Authority (HRA), where she is leading on a range of policy and engagement initiatives for the HRA to make it easy to do research that people can trust. This includes pushing for change to increase diversity and... Read More →

Wednesday June 19, 2024 3:15pm - 4:15pm PDT
Room 6F San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

09: Regulatory, Forum

Format Forum
Level Intermediate
Featured Topics Diversity Equity and Inclusion (DEI)
Level Intermediate
Feature Topics Diversity Equity and Inclusion (DEI)
Credit Type ACPE, CME, RN
Tags Forum

3:15pm PDT

#367: EMA Town Hall

Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-667-L04-P; CME 1.00; RN 1.00

The European Forum will explain our various platforms and ways to engage to support innovation, illustrated on an example of an Advanced Therapy Medicinal Product (ATMP).

Learning Objectives

Describe the assessment procedure and the role of CHMP and rappoteur; Describe platforms to support developers; Discuss a successful submission of an ATMP.

Chair

Steffen Thirstrup, MD, PhD

Speaker

Panelist
Sabine Haubenreisser, PhD, MSc

Panelist
Anabela Marcal, PharmD

Panelist
Evdokia Korakianiti, PhD, MSc

Speakers

Sabine Haubenreisser

Principal Scientific Administrator, Stakeholders and Communication Division, European Medicines Agency, Netherlands

Evdokia Korakianiti

Head of Quality and Safety of Medicines, European Medicines Agency, Netherlands

Anabela Marcal

EMA Liaison Official to the US FDA, European Medicines Agency, Netherlands

Steffen Thirstrup

Chief Medical Officer, European Medicines Agency, Netherlands

Wednesday June 19, 2024 3:15pm - 4:15pm PDT
Room 6A San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

09: Regulatory, Forum

Format Forum
Level Intermediate
Featured Topics Regulator Perspectives
Level Intermediate
Feature Topics Regulator Perspectives
Credit Type ACPE, CME, RN
Tags Forum

3:15pm PDT

#363: Implementing Changes To Drug-Device Combination Products Globally

Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-663-L04-P; CME 1.00; RN 1.00

Industry and regulators will collaborate to discuss current challenges and future opportunities to streamline post approval changes to combination products.

Learning Objectives

Describe current regulatory frameworks for lifecycle management changes to combination products; Compare differences in expectations for submissions of post-approval changes globally; Discuss the utilization of harmonized guideline, ICH Q12, for management of post approval changes to combination products globally.

Chair

Chelsea O'Connell, MS, RAC

Speaker

FDA CBER Perspective on Postmarket Changes to CBER-Led Combination Products
Andrea Gray, PhD

Industry Perspective on the Latest ICH Q12 Training Module 8
Andrew Chang, PhD

Industry Perspective on Postmarket Changes to Combination Products: Global
Amy Wise, MBA

PMDA Update: Postmarket Changes to Combination Products in Japan
Keisuke Tanaka, PhD

Industry Perspective on Postmarket Changes to Combination Products: US
Lori-Anne Boville

Speakers

Chelsea O'Connell

Director, Global Regulatory and R&D Policy, Amgen, United States

Chelsea O’Connell, MS, RAC, joined Amgen in 2011 and is currently a Global Regulatory and R&D Policy Director, where she advances strategic regulatory policy goals by engaging with regulatory authorities, industry, professional associations, and other stakeholders across various... Read More →

Andrew Chang

Vice President, Quality and Regulatory Compliance, Novo Nordisk, United States

Dr. Andrew Chang is a multifaceted quality and CMC leader with 28 years well-rounded medical product regulatory and industry experiences. He is a board director for CASSS-Sharing Science Solutions and PDA, respectively. Andrew has served as a member of the planning committee for DIA... Read More →

Amy Wise

Associate Director, RA Device and Combination Products, AbbVie, United States

Amy Wise is an Associate Director Regulatory Affairs Device and Combination Products at AbbVie. Amy has been in the pharmaceutical industry for 25 years with 15 years in regulatory affairs, 5 years in quality and 5 years in pharmaceutical sciences. She has successfully provided the... Read More →

Andrea Gray

Biomedical Engineer Advisor, CBER, FDA , United States

Keisuke Tanaka

Reviewer, Pharmaceuticals and Medical Devices Agency (PMDA), Japan

Dr. Tanaka is Reviewer of the Office of Cellular and Tissue-based Products, PMDA. He received his Ph, D. from Osaka University in Bioorganic chemistry with emphasis in nucleic acid therapy. Since joining PMDA in 2021, he has worked in the Office of Cellular and Tissue-based Products... Read More →

Lori-Anne Boville

Group Director, Pharma Technical Regulatory, Device and Combination Products, Genentech, A Member of the Roche Group, United States

She is a Regulatory Affairs Expert in Biologics products, Medical Devices & Combination Products at Genentech. Lori has over 20 years of Biopharmaceutical product development, analytical testing, commercial quality, regulatory affairs and medical device experience. She has successfully... Read More →

Wednesday June 19, 2024 3:15pm - 4:15pm PDT
Room 1AB San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

09: Regulatory, Session | 06: TranslationalDelivery Tech-PrecMedicine, Session

Format Session
Level Intermediate
Featured Topics ICH, Regulator Perspectives
Level Intermediate
Feature Topics ICH,Regulator Perspectives
Credit Type ACPE, CME, RN
Tags Session

4:30pm PDT

#374: Updated Status of Multiregional Clinical Trials Based on ICH E17 Guideline: 5 Years After the Implementation

Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-673-L04-P; CME 1.00; RN 1.00

The forum will consist of regulators and industry experts who were the authors of the ICH E17 guidelines. As regulators and sponsors, they will provide first-hand narrative on their experiences in implementing the ICH E17 guidelines.

Learning Objectives

Discuss the status of adoption of ICH E17 guidelines on conduct of multiregional clinical trials (MRCT) by the regulators and industry. • Identify reasons behind slow uptake of the ICH E17 guidelines. • Discuss points for increasing usage of E17 for conduct of MRCT and simultaneous global development.

Chair

Yoshiaki Uyama, PhD, RPh

Speaker

PMDA Perspective
Yoko Aoi, PhD

Pharmaceutical Industry Perspective
Romi Singh, PhD

FDA Update
Douglas Pratt

Taiwan Perspective
I-Chun Lai, MD, MS

China Perspective
Yangfeng Wu, MD, PhD

Speakers

Yoshiaki Uyama

Associate Executive Director, Pharmaceuticals and Medical Devices Agency (PMDA), Japan

Yoko Aoi

Deputy Review Director, Office of New Drug V, Pharmaceuticals and Medical Devices Agency (PMDA), Japan

I-Chun Lai

Director, Consultation Division, Center for Drug Evaluation, Taiwan

Dr. I-Chun Lai graduated with her medical degree from Taipei Medical University in Taiwan in 2001 and worked as a physician until 2007, when she joined the Center for Drug Evaluation (CDE) in Taiwan. In 2013, she received her Master of Science from the Institute of Health Policy and... Read More →

Douglas Pratt

Supervisory Medical Officer, OVRR, CBER, FDA , United States

Romi Singh

Head, Regional Regulatory Sciences, Asia, Pfizer Inc, United States

Yangfeng Wu

Executive Director, Peking University Clinical Research Institute, China

Professor Wu is the Executive Director of Peking University Clinical Research Institute. He has been principal investigators of tens of research projects sponsored by national and international founding agencies including China Ministry of Science and Technology, China Natural Science... Read More →

Wednesday June 19, 2024 4:30pm - 5:30pm PDT
Room 5AB San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

02: ClinTrials -ClinOps, Forum | 09: Regulatory, Forum

Format Forum
Level Intermediate
Featured Topics ICH
Level Intermediate
Feature Topics ICH
Credit Type ACPE, CME, RN
Tags Forum

4:30pm PDT

#375.1: Middle East Emerging Opportunities

Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-694-L04-P; CME 1.00; RN 1.00

In this forum, regulatory authorities from the Middle East region will share updates, success stories, lessons learned and plans for the future.

Learning Objectives

Identify the regulatory authorities' representatives from the Middle East region; Discuss new developments and opportunities in the region.

Wednesday June 19, 2024 4:30pm - 5:30pm PDT
Room 6C San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

09: Regulatory, Forum

Level Intermediate
Level Intermediate
Credit Type ACPE, CME, RN
Tags Forum

4:30pm PDT

#375: Sustainability of Rare Disease Drug Development

Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-674-L04-P; CME 1.00; RN 1.00

Panelists will reflect on the past forty years of the orphan drug act including advancements and impediments. The panel will discuss what is needed in the future to continue to advance rare disease drug development to treat as many patients as possible.

Learning Objectives

Discuss the outcomes, challenges, and opportunities in the next forty years of the Orphan Drug Act; Identify policy advancements that need to be made to continue innovative rare disease drug development.

Chair

Darcy Frear, PhD

Speaker

Panelist
Katherine Donigan, PhD

Panelist
Patroula Smpokou, MD

Panelist
Karin Hoelzer, DVM, PhD

Panelist
Julia Tierney, JD

Speakers

Darcy Frear

Associate Director, Bridgebio, United States

Dr. Darcy Frear is currently the associate director for Regulatory Policy and Intelligence at BridgeBio. Prior to joining BridgeBio in 2022, Darcy began her regulatory career at Genentech supporting program management in oncology and US regulatory policy. Subsequently, she joined... Read More →

Katherine Donigan

Senior Director, Science and Regulatory Policy, Sarepta, United States

Dr. Katherine Donigan is currently a Senior Director of Science and Regulatory Policy at Sarepta Therapeutics. Prior to joining Sarepta, Dr. Donigan was a Senior Director of Science and Regulatory at Biotechnology Innovation Organization (BIO) and spent 7 years in regulatory policy... Read More →

Karin Hoelzer

Senior Director, Policy and Regulatory Affairs, NORD, United States

Dr. Karin Hoelzer directs Policy and Regulatory Affairs for the National Organization for Rare Disorders (NORD®). She works closely with pharmaceutical and biotech partners to ensure public policy supports innovative approaches and new treatments, and adequately incorporates patient... Read More →

Patroula Smpokou

Lead Physician, Division of Rare Diseases/Medical Genetics, OND, CDER, FDA, United States

Patroula Smpokou, MD, is a board-certified pediatric clinical geneticist working in the Division of Rare Diseases and Medical Genetics at CDER. Her work involves the clinical review and regulation of products intended for rare genetic diseases. Prior to joining FDA, she was a practicing... Read More →

Julia Tierney

Deputy Center Director for Strategy, Policy, and Legislation, CBER, FDA, United States

Wednesday June 19, 2024 4:30pm - 5:30pm PDT
Room 6F San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

09: Regulatory, Forum

Format Forum
Level Intermediate
Featured Topics Rare Disease
Level Intermediate
Feature Topics Rare Disease
Credit Type ACPE, CME, RN
Tags Forum

4:30pm PDT

#376: Challenges in Quality Control for Cell and Gene Therapy Development: Regulatory Perspectives and Best Practices

Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-675-L04-P; CME 1.00; RN 1.00

This session will describe key challenges in quality assessment for cell and gene therapies, focusing on challenges related to raw materials; discuss regulatory perspectives; and present best practices to control and evaluate raw materials.

Learning Objectives

Identify the key current challenges in quality control for cell and gene therapy development; Explain global regulators’ perspectives on quality control, including raw material selection, control, and qualification; Discuss best practices, including standards and reference materials, to support the development and manufacture of cell and gene therapies.

Chair

Fouad Atouf, PhD

Speaker

Qualification and Selection of Raw Materials During Early Stage of Development
Mo Heidaran, PhD

Perspective on the Selection of Raw Materials for Cell Therapy Manufacturing
Haritha Vallabhaneni, PhD

Cell and Gene Therapy Products: Global Regulatory Perspective
Michael Day, PhD

Speakers

Fouad Atouf

Senior Vice President, Global Biologics, United States Pharmacopeia (USP), United States

Dr. Atouf has over 20 years’ experience in research, product development, characterization and quality assessment of biologics including cell and gene therapies (CGT). At USP, he heads the Biologics program and oversees standards development, stakeholder engagement and external... Read More →

Michael Day

Senior Director, Regulatory Science, VCLS, United States

As a Senior Director, Regulatory Science and Head of Cell and Gene Therapies at VCLS, Michael (Mike) is responsible for providing both regulatory strategy and CMC consulting services to clients for global product development ranging from small molecule drugs to biologics and advanced... Read More →

Mo Heidaran

Chief Regulatory Scientist, Cellx Inc., Consulting, United States

Dr. Mo Heidaran is currently Chief Regulatory Scientist at Cellx Inc. He previously worked as Head of Translational and Regulatory Strategy at GCTx (GC Therapeutics). He is a recognized expert in the development of cell and gene therapies with more than 9 years of experience at the... Read More →

Haritha Vallabhaneni

Biological Reviewer, Office of Cellular Therapy and Human Tissue CMC, CBER, FDA, United States

Dr. Vallabhaneni is a CMC Reviewer in the Division of Cell Therapy 1, CBER, FDA. She is involved in CMC review of regulatory applications involving cellular therapy products. She has extensive research experience in stem cells previously working on multiple research projects investigating... Read More →

Wednesday June 19, 2024 4:30pm - 5:30pm PDT
Room 6E San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

09: Regulatory, Session | 06: TranslationalDelivery Tech-PrecMedicine, Session | 10: RegCMC-Product Quality, Session

Format Session
Level Intermediate
Featured Topics Cell and Gene Therapy
Level Intermediate
Feature Topics Cell and Gene Therapy
Credit Type ACPE, CME, RN
Tags Session

8:00am PDT

#405: The FDA's Commitment to Patient-Centric Research: Key Themes from Decentralized Trial Draft Guidance

Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-681-L04-P; CME 1.00; RN 1.00

This interactive forum will explore the key themes and industry feedback from the decentralized trials alliance members’ response to the FDA draft guidance on decentralized clinical trials.

Learning Objectives

Examine how the FDA draft guidance reflects a commitment to patient-centric decentralized clinical trials; Identify key themes and industry feedback provided in the alliance's response to the draft guidance; Discuss the next steps needed to enable adoption of decentralized trials under the draft framework.

Chair

Rasika Kalamegham, PhD

Speaker

Collaboration Perspective
Jane Elizabeth Myles, MSc

FDA Perspective
M. Khair ElZarrad, PhD, MPH

Industry Perspective
Dylan Bechtle, MS

Speakers

Dylan Bechtle

Associate Director, Regulatory Policy Lead- US, Johnson & Johnson, United States

M. Khair ElZarrad

Director, Office of Medical Policy, CDER, FDA, United States

Dr. ElZarrad is the Director of the Office of Medical Policy (OMP) at FDA’s Center for Drug Evaluation and Research (CDER), where he leads the development, coordination, and implementation of medical policy programs and strategic initiatives. Dr. ElZarrad currently leads multiple... Read More →

Rasika Kalamegham

Head, US Regulatory Policy, Genentech, A Member of the Roche Group, United States

Jane Myles

Program Director, Decentralized Trials and Research Alliance (DTRA), United States

Jane has over 25 years of experience improving clinical trials and patient experiences. She has focused on driving innovation in trial design and execution to accelerate getting medicines to patients. Jane transitioned from molecule focus to portfolio focus about 14 years ago, first... Read More →

Thursday June 20, 2024 8:00am - 9:00am PDT
Room 6A San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

05: Patient-Focused Drug Dev, Forum | 09: Regulatory, Forum

Format Forum
Level Intermediate
Featured Topics Decentralized Clinical Trials (DCTs)
Level Intermediate
Feature Topics Decentralized Clinical Trials (DCTs)
Credit Type ACPE, CME, RN
Tags Forum

8:00am PDT

#406: More Nuanced Clinical Outcomes from Person-Generated Health Data: The New Frontier

Component Type: Forum
Level: Basic
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-682-L04-P; CME 1.00; RN 1.00

Person-generated data offers nuanced effectiveness measures for clinical and regulatory decision-making for new medical products, like the wearable-derived digital clinical outcome assessment for Duchenne muscular dystrophy. What's next?

Learning Objectives

Describe practical applications of person-generated health data, and key business and regulatory perspectives on opportunities and challenges; Explain the value of this type of information to patients, clinicians, and colleagues to help inform decision-making.

Chair

Nancy A Dreyer, PhD, MPH, FISPE

Speaker

Panelist
Christina Mack, PhD, MPH

Panelist
Paul Strijbos, PhD

Panelist
Jeffrey Nahum Siegel, DrMed, MD

Panelist
Martha Azer, PharmD, RPh

Speakers

Nancy Dreyer

Founder, Dreyer Strategies LLC, United States

Nancy Dreyer is Chief Scientific Advisor to Picnic Health and Adjunct Professor of Epidemiology at the University of North Carolina. Recently retired from full-time employment as Chief Scientific Officer at IQVIA Real World Solutions, she maintains an active career consulting, publishing... Read More →

Christina Mack

Chief Scientific Officer, Real-World Solutions, IQVIA, United States

Christina Mack is Chief Scientific Officer of IQVIA real world solutions. An epidemiologist and engineer by training, she is responsible for driving scientific and technical innovation that directly impacts patient health. Recognized as a 2023 PharmaVoice100 Honoree, she’s committed... Read More →

Paul Strijbos

Senior Principal Neurotech Innovation Leader, F. Hoffmann-La Roche Ltd, Switzerland

Paul is a PhD neuroscientist with over 25 years of clinical development experience across the product lifecycle. He works as Neurotechnology Innovation Leader in the late stage Neuroscience organization of F. Hoffmann-La Roche in Basel and focusses on the development, validation and... Read More →

Martha Azer

Associate Director, Global Regulatory Policy & Intelligence, Johnson & Johnson, United States

Martha Azer is an Associate Director of Global Regulatory Policy and Intelligence at Johnson & Johnson. She leads the US policy work on Digital Health and Artificial Intelligence & Machine Learning as well as Precision Medicine to help shape the regulatory policy landscape to provide... Read More →

Jeffrey Siegel

Director, Office of Drug Evaluation Sciences, OND, CDER, FDA, United States

Jeffrey Siegel is the Director of the Office of Drug Evaluation Sciences (ODES) in the Office of New Drugs (OND), CDER, FDA. ODES oversees Clinical Outcome Assessments, Biomarker Qualification, Research and Bioinformatics in OND. Dr Siegel has over 20 years of experience in research... Read More →

Thursday June 20, 2024 8:00am - 9:00am PDT
Room 6B San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

05: Patient-Focused Drug Dev, Forum | 09: Regulatory, Forum

Format Forum
Level Basic
Featured Topics Patient Focused
Level Basic
Feature Topics Patient Focused
Credit Type ACPE, CME, RN
Tags Forum

9:15am PDT

#413: What is the Value of Reference Agency Assessment Reports in Enabling Reliance and What do Relying Agencies Require?

Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-688-L04-P; CME 1.00; RN 1.00

Challenges remain in the implementation of reliance. This session will bring together stakeholders to discuss the utilisation of assessment reports, and recommend ways to ensure effective and efficient systems in the future.

Learning Objectives

Recognize the value of assessment reports in enabling reliance; Describe current use of public and non-public information for enabling regulatory reviews; Discuss how documentation and information sharing could improve to ensure reliance becomes successful.

Chair

Magda Bujar, PhD, MSc

Speaker

WHO Update
Samvel Azatyan, MD, PhD

Reference Agency Viewpoint
Emer Cooke, MBA, MSc

Relying Agency Viewpoint
Patricia Oliveira Pereira Tagliari, LLM, MPH

Industry Viewpoint
Susanne Ausborn, PhD

Speakers

Magda Bujar

Senior Manager, Regulatory Programme and Strategic Partnerships, Centre for Innovation in Regulatory Science (CIRS), United Kingdom

Dr Magda Bujar is Senior Manager, Regulatory Programme and Strategic Partnerships and has over 8 years’ experience working in Regulatory Policy and Science. She has co-authored a number of publications and has presented and chaired at major scientific meetings including those of... Read More →

Susanne Ausborn

Global Head International Regulatory Policy, Roche, Switzerland

Susanne Ausborn has more than 20 years of experience in technical regulatory affairs and regulatory policy, being currently Global Head International Regulatory Policy in Roche. M.Sci. in Analytical Chemistry and PhD in Biophysical Chemistry, Susanne is a strong advocate for global... Read More →

Samvel Azatyan

Team Lead, Regulatory Convergence and Networks (RCN/REG), World Health Organization (WHO), Switzerland

Emer Cooke

Chair, ICMRA; Executive Director, European Medicines Agency, Netherlands

Patricia Oliveira Pereira Tagliari

Associate Director, ANVISA, Brazil

Thursday June 20, 2024 9:15am - 10:15am PDT
Room 6C San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

09: Regulatory, Session

Format Session
Level Intermediate
Featured Topics Regulatory Reliance
Level Intermediate
Feature Topics Regulatory Reliance
Credit Type ACPE, CME, RN
Tags Session

9:15am PDT

#414: Regulatory Affairs of the Future: Is Now

Component Type: Forum
Level: Basic
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-689-L04-P; CME 1.00; RN 1.00

Global regulatory affairs is a critical function at the key interface between the innovation from our pipelines and patients. The world around us is changing very rapidly leading to implications and opportunities for regulatory affairs. We must evolve. But how?

Learning Objectives

Identify what global changes are leading to an evolved regulatory affairs organization; Discuss what skillset global regulatory affairs professionals of the future will need.

Chair

Lina AlJuburi, PharmD, MSc

Speaker

FDA Update
M. Khair ElZarrad, PhD, MPH

Academic Update
Maria Florez, MA

Industry Update
Nicole Mahoney, PhD

Speakers

Lina AlJuburi

Head, Global Regulatory Science and Policy - North America, Sanofi, United States

M. Khair ElZarrad

Director, Office of Medical Policy, CDER, FDA, United States

Maria Florez

Senior Consultant, Tufts Center For the Study of Drug Development, Tufts Univers, United States

Maria Florez studies the practices, strategies, attitudes, and incentives driving innovation in the pharmaceutical R&D process, as well as the impact of digital transformation in clinical research. Maria has been a researcher, consultant, and advisor in the healthcare sector for over... Read More →

Nicole Mahoney

Executive Director US Regulatory Policy & Intelligence, Novartis Pharmaceuticals Corporation, United States

Thursday June 20, 2024 9:15am - 10:15am PDT
Room 6B San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

09: Regulatory, Forum

Format Forum
Level Basic
Featured Topics Student Programming
Level Basic
Feature Topics Student Programming
Credit Type ACPE, CME, RN
Tags Forum

9:15am PDT

#415: EMA-FDA Question Time

Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-690-L04-P; CME 1.00; RN 1.00

In this interactive forum experts from EMA and FDA will address questions from the audience and share their experiences of collaboration in specific areas as well as on how both Agencies collaborate in addressing current regulatory, scientific, and communication challenges.

Learning Objectives

Identify an overall understanding of the regulatory and scientific collaboration between EMA and FDA; Describe experience and explore specific areas of collaboration between the two Agencies; Examine how EMA and FDA are addressing regulatory and scientific challenges in new areas.

Chair

Anabela Marcal, PharmD
Katherine Tyner, PhD

Speaker

Panelist
Kerry Jo Lee, MD

Panelist
Adam C Fisher, PhD

Panelist
R. Angelo De Claro, MD

Panelist
Evdokia Korakianiti, PhD, MSc

Panelist
Steffen Thirstrup, MD, PhD

Panelist
Francesco Pignatti, MD

Speakers

Anabela Marcal

EMA Liaison Official to the US FDA, European Medicines Agency, Netherlands

Katherine Tyner

FDA Liaison to the EMA, Office of Global Policy and Strategy, OC, FDA, United States

Dr. Katherine Tyner is the FDA liaison to the EMA. Dr. Tyner joined FDA in 2007 and has held numerous positions within CDER to advance the quality, safety, and efficacy of complex drug products. In her most recent role as the Associate Director for Science in the Office of Pharmaceutical... Read More →

R. Angelo De Claro

Associate Director for Global Clinical Sciences, Oncology Center of Excellence, FDA, United States

Kerry Jo Lee

Assoc. Dir. for Rare Diseases; Program Manager, Advancing Rare Disease Cures Pro, FDA, United States

Adam Fisher

Director, Enterprise Project Staff, OQA, OPQ, CDER, FDA, United States

Adam Fisher, PhD., is the Director of Enterprise Project Staff in the Office of Pharmaceutical Quality and lead of the Framework for Regulatory Advanced Manufacturing Evaluation (FRAME) Initiative at the U.S. FDA. He has been a primary and secondary reviewer of generic drug applications... Read More →

Francesco Pignatti

Scientific Adviser for Oncology, European Medicines Agency, Netherlands

Steffen Thirstrup

Chief Medical Officer, European Medicines Agency, Netherlands

Evdokia Korakianiti

Head of Quality and Safety of Medicines, European Medicines Agency, Netherlands

Thursday June 20, 2024 9:15am - 10:15am PDT
Room 6A San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

09: Regulatory, Forum | 14: DIAmond, Forum

Format Forum
Level Intermediate
Featured Topics Regulator Perspectives, Regulatory Collaboration
Level Intermediate
Feature Topics Regulator Perspectives,Regulatory Collaboration
Credit Type ACPE, CME, RN
Tags Forum

10:45am PDT

#416: FDA Town Hall

Component Type: Forum
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-24-691-L04-P; CME 1.25; RN 1.25

The FDA Town Hall is the forum to hear from FDA leadership about Center priorities. The audience will be invited to submit questions of general interest.

Learning Objectives

Discuss FDA Center priorities.

Chair

Kim Quaintance-Lunn

Speaker

Panelist
Kevin Bugin, PhD, MS, RAC

Panelist
Julia Tierney, JD

Panelist
Anindita Saha

Panelist
Tamy Kim, PharmD

Speakers

Kim Quaintance-Lunn

Vice President, Regulatory Affairs Americas, Bayer AG, United States

Kim Quaintance-Lunn is the Vice President, Regulatory Affairs Americas and a Distinguished Science Fellow at Bayer, a Director at Large for the FDA Alumni Association, and Chair of DIA's Americas Regional Advisory Council. At Bayer, Kim serves in a leadership capacity for US Regulatory... Read More →

09: Regulatory, Forum | 14: DIAmond, Forum

Format Forum
Level Intermediate
Featured Topics Regulator Perspectives
Level Intermediate
Feature Topics Regulator Perspectives
Credit Type ACPE, CME, RN
Tags Forum