DIA 2024 Global Annual Meeting

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

Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-542-L04-P; CME 1.00; RN 1.00

This session will explore special topics in causal inference. Presenters will describe trial design and analysis issues in drug development, related to both randomized trials and real-world data (RWD), that may benefit from statistical methodology to allow for better and more accurate generation of evidence with respect to drug treatment effects.

Learning Objectives

Describe various statistical approaches to handle complex design and analysis issues in drug development. Explain use case examples for illustration for causal inference methods.

Chair

Pallavi Mishra-Kalyani, PhD, MS

Speaker

Causal Inference in Clinical Drug Development: Industry Update
Antara Majumdar, PhD

Methodological Challenges and Potential Solutions When Conducting External Control Studies Intended for Causal Inference
Shia Kent, PhD

Regulatory Considerations and Case Studies of Externally Controlled Trials
Pallavi Mishra-Kalyani, PhD, MS

Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Application UAN: 0286-0000-24-547-L04-P; CME 1.00; RN 1.00

This session delves into the latest guidance on real-world data (RWD) and real-world evidence (RWE), offers insights from case studies in which such data were critical for regulatory decisions, and discusses how to prepare the next generation of medical writers for this complex landscape.

Learning Objectives

Analyze the FDA’s current stance and guidance on real-world data (RWD) and real-world evidence (RWE) in regulatory submissions; Compare real-world applications of RWD and RWE, showcased through several case studies; Outline educational strategies for equipping future regulatory and medical writers with the skills and understanding needed to effectively incorporate RWD and RWE into documentation.

Chair

Jeanette Towles, MA

Speaker

Navigating the Regulatory Landscape: Real-World Data and Real-World Evidence in Regulatory Documents
Clarence L Young, MD

Navigating the Regulatory Landscape: Real-World Data and Real-World Evidence in Regulatory Documents
Tina Schlecht, PharmD, MBA

Component Type: Session
Level: Advanced
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-568-L04-P; CME 1.00; RN 1.00

Advanced cancers pose a dilemma between evidentiary requirements and speedy access to innovative agents. We present the results of a survey with regulators and discuss factors that influence acceptability of real-world data and possible optimal submission strategies.

Learning Objectives

Describe the challenges of drug development with evidence using external controls; Discuss regulators’ attitudes about real-world evidence and opportunities for addressing the challenges; Identify strategies using real-world evidence to maximize impact on the regulatory decision.

Chair

Francesco Pignatti, MD

Speaker

Real-World Data to Support Benefit-Risk Assessment: Regulatory Perspective
Douwe Postmus, PhD, MSc

External Comparators Using Real-World Data: Results of a Simulation Study
Gerd Rippin, DrSc

The Role and Impact of Real-World Data in Cancer Drug Applications
Catherine C. Lerro, PhD, MPH

Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-573-L04-P; CME 1.00; RN 1.00

The focus of the panel is to understand how to use RWD in clinical trials so that it can be relied upon as core evidence for regulatory decisions, including external control arms (ECAs), hybrid or pragmatic clinical trials, and RWD powered covariate adjustment. For RWD to be used in a clinical trial, the processes to prepare RWD require the same scale and rigor as global multicenter trials, where evidence is generated in a harmonized, coordinated, and systematic way across geographies, sites, and data sources. Even with access to relevant RWD, the messy nature of the data results in data processing delays, low-quality data, and ultimately a lack of trust in analytical results by the regulator. This panel will address the biggest challenges for accepting RWD as core evidence, which include ensuring data relevance, quality, reliability, and scalable data validation, as well as strategies to scale up RWD preparation from ad hoc projects to enterprise-level machinery for pharma.

Learning Objectives

Explore strategies to address the inherent challenges of real-world data (RWD) preparation at scale for external control arms (ECAs), including data scalability and quality; Describe how to transition from ad hoc ECA projects to an enterprise-level approach that ensures data quality, processing efficiency, and trustworthiness in analytical results for pharma clinical development.

Chair

Mayur Saxena, PhD

Speaker

Industry Update
Victoria Chia, PhD, MPH

Industry Update
Hongwei Wang

Industry Update
Jingyu (Julia) Luan, PhD

Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-585-L04-P; CME 1.00; RN 1.00

Data linkage is a methodology to capture data from patients before, during, and after a clinical trial. The panel will discuss the challenges of conducting randomized clinical trials (RCTs) linked with real-world data (RWD), and will emphasize the data linkage methodologies and current needs.

Learning Objectives

Describe the methodology of data linkage, a relatively new method to collect individual patient data before, during, and after a clinical trial outside the randomized clinical trial (RCT) setting; Discuss challenges for different stakeholders involved in utilization of real-world evidence (RWE) and more specifically data linkage, including industry, regulators, and payers/HTA.

Chair

Aaron Kamauu, MD, MPH, MS

Speaker

Panelist
Francesco Pignatti, MD

Panelist
Kajsa Kvist

Panelist
Brian Bradbury, DrSc, MA

Panelist
Akshay Vashist

Component Type: Session
Level: Basic
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-692-L04-P; CME 1.00; RN 1.00

Social media is an important new source of real-world data (RWD) for patient-focused drug development. This session will present a review of considerations from an industry and regulators perspectives when listening to social media from patients.

Learning Objectives

Summarize the work done by group of experts from companies’ members; Discuss the challenges and opportunities of exploiting real-world data (RWD) from social media listening for R&D purposes and regulatory submissions; Propose best practices.

Chair

Thierry H Escudier, MSc

Speaker

Industry Update
Sarah Lyons

Academic Update
Philipp Cimiano, PhD

Industry Update
Vishakha Sharma, PhD

Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-603-L04-P; CME 1.00; RN 1.00

This session will bring regulators and industry quality experts together to explore how quality management systems can be developed to meet the challenges real-world evidence presents to QA activities and regulatory inspection expectations.

Learning Objectives

Evaluate quality challenges associated with the use of real-world evidence as part of regulatory studies; Discuss how to apply proportionate risk-based quality assurance activities which meet regulatory expectations.

Chair

Andrew Gray, PhD

Speaker

FDA Update
L'Oreal Walker

FDA Update
Lee H. Pai-Scherf, MD

Industry Perspective
Abi Seifert, MBA

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

Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-613-L04-P; CME 1.00; RN 1.00

A rapidly changing landscape has led to an increased use of real-world evidence (RWE) in regulatory decision making, as a complement to randomized clinical trials (RCTs), impacting how medicines are developed and assessed. The session will present where we are and discuss the exciting future ahead.

Learning Objectives

Describe how real-world data (RWD) is being leveraged to generate real-world evidence (RWE) in support of regulatory assessment and decision-making processes in Europe; Discuss the progress of DARWIN EU, and a high-level overview of the use of RWE in recent applications in EU and see how the generation of meaningful evidence can support safer and more effective treatments for patients.

Chair

Steffen Thirstrup, MD, PhD

Speaker

EMA Update
Sabine Haubenreisser, PhD, MSc

Industry Update
Laura T. Pizzi, PharmD, MPH

Patient Update
Dave deBronkart

Industry Update
Brian Bradbury, DrSc, MA

Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-611-L04-P; CME 1.00; RN 1.00

In this forum, speakers from regulatory, industry, and research organizations will discuss methodological considerations and evaluate various innovative use cases in different settings that integrate real-world data (RWD) in clinical designs.

Learning Objectives

Discuss global regulatory guidance, frameworks, and best practices for real-world data (RWD) in innovative hybrid study designs for regulatory purposes; Describe innovative approaches and statistical methods for integrating RWD and clinical trial data for clinical decision-making; Appraise use cases where RWD have been used in hybrid study designs to support clinical and regulatory development.

Chair

Rachele Hendricks-Sturrup, DrSc, MA, MSc

Speaker

Panelist
Mehmet Burcu, PhD, MS

Panelist
Christina Mack, PhD, MPH

Panelist
Catherine C. Lerro, PhD, MPH

Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-621-L04-P; CME 1.00; RN 1.00

This session will provide an overview of today’s biosimilars landscape, explore trends in uptake, describe innovative programs from industry disruptors, and report perspectives from key stakeholders through their primary market research.

Learning Objectives

Summarize results of current payer and provider research on barriers to biosimilar coverage and adoption, with a focus on product interchangeability; Describe the challenges and solutions of real-world data (RWD) and real-world evidence (RWE) in decision-making; Identify a framework for operational readiness in biosimilar management.

Chair

Charlie Dragovich, RPh

Speaker

Industry Update
Chara Reid, PharmD, RPh

Industry Update
Catherine Lockhart, PharmD, PhD, MS

Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-624-L04-P; CME 1.00; RN 1.00

This panel will discuss different types of data models that exist and their strengths and weaknesses in relation to real-world data (RWD) and interoperability.

Learning Objectives

Recognize the different types of data models that exist and their strengths and weaknesses in relation to real-world data (RWD) and interoperability; Discuss the appropriate data models for primary and secondary data and how clinical data managers can potentially help RWD and trial data to meet the evidentiary requirements for regulatory decision-making.

Chair

Jesper Kjær, MS

Speaker

Panelist
Davera Gabriel, RN

Panelist
Jonathan Chainey

Panelist
Stacy Tegan

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

Component Type: Session
Level: Basic
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-677-L04-P; CME 1.00; RN 1.00

Session will focus on considerations when using real-world data for post-marketing safety studies, with use cases provided.

Learning Objectives

Identify specific study design and analytic challenges related to the safety of medical products, with a focus on rheumatology and ophthalmology; Describe key elements to consider when identifying real-world data sources to generate real-world evidence.

Chair

Alicia Gilsenan, PhD, MS, RPh, FISPE

Speaker

FDA RWE Program and Considerations in Assessing Validity of Safety Studies using RWD
Marie Bradley, PhD, MPH, MPharm

Value of Real-World Data/Real-World Evidence to Assess Post-marketing Safety: Use Case- Ophthalmology
Angelika Manthripragada, PhD, MPH