DIA 2024 Global Annual Meeting

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

Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-531-L04-P; CME 1.00; RN 1.00

The ICH E11A guidance advances use of pediatric extrapolation in three areas: (1) role of exposure-response; (2) statistical implications of using external data; and (3) extrapolation of safety. After an overview, these three areas will be explored.

Learning Objectives

Define pediatric extrapolation and three components of extrapolation concept; Describe role of exposure-response in supporting data extrapolation from reference to target pediatric population; Examine the impact of extrapolation on false positive rate of a pediatric clinical trial; Outline impact of pediatric extrapolation on generation of safety data in the target pediatric population.

Chair

Robert "Skip" Nelson, MD, PhD

Speaker

FDA Update on ICH E11A EWG on Pediatric Extrapolation
Lynne Yao, MD

Industry Update
Forrest Williamson, PhD, MS

Industry Update on ICH E11A EWG on Pediatric Extrapolation
Brenda Cirincione, PhD, MS

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

Component Type: Workshop
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-600-L04-P; CME 1.00; RN 1.00

This workshop will assist attendees with ways to properly accommodate medically complex and/or rare disease patients through providing accessibility tools and solutions. Soft skills and “person first” language will be discussed.

Learning Objectives

Identify how to expand the definition of diversity; Describe how to have conversations with patients about disabilities; Explore solutions and recommendations for no/low-cost actions you can take to make your facility as accessible as possible for a variety of disabilities; Define resources for support and training for site staff.

Chair

Stacy Hurt, MBA, MHA

Speaker

Facilitator
Carlos Orantes, MBA

Facilitator
Cecile Gonzalez-Cerimele

Facilitator
Mindy Cameron

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

How to Provide Necessary Medicinal Products to Children - An Industry Perspective
Robert "Skip" Nelson, MD, PhD

FDA Update
Gerold Wharton

Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-610-L04-P; CME 1.00; RN 1.00

Pregnant individuals are underrepresented in research. This session provides an overview of how the evolving regulatory framework, pharmaco-epidemiology, and clinical trials can advance evidence generation and contribute to pregnancy labeling.

Learning Objectives

Discuss important global advances in the collection of data in drugs and biological products used during pregnancy; Identify the evolving regulatory framework, and the value of premarket and postmarketing safety data that can lead to improved labeling information for drugs and biological products used in pregnancy; Describe stakeholder efforts to include pregnant individuals in clinical trials.

Chair

Martine Dehlinger-Kremer, PhD

Speaker

Assessment of Drugs Used During Pregnancy: Current Status
Martine Dehlinger-Kremer, PhD

Safety of Drugs Used During Pregnancy: Regulatory Considerations and Updates
Lynne Yao, MD

Post-Marketing Approval Safety Studies: The Role of Pregnancy Registries
Christina Chambers, PhD, MPH

Evaluation of Drug Use During Pregnancy: Post-Marketing Surveillance and Beyond - Time for a Paradigm Shift
Laura Shaughnessy, PhD

Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-634-L04-P; CME 1.00; RN 1.00

There is a critical need for innovation in women’s health, particularly maternal health. What will it take to improve care in this area? How can innovative regulatory, reimbursement and access policies encourage novel approaches to women’s health?

Learning Objectives

Identify areas of women’s health which face barriers preventing innovation; Describe disparities in women’s/maternal health and existing regulatory, payment and access policies intended to improve outcomes; Evaluate the types of policy changes needed to encourage innovation

Chair

Nancy Bradish Myers, Esq, JD

Speaker

Panelist
Lynne Yao, MD

Panelist
Tara Croft, MBA

Panelist
Sandy Milligan, JD, MD

Component Type: Workshop
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-658-L04-P; CME 1.00; RN 1.00

Recruiting pediatric patients is a challenge and one of the solutions is to carefully design the study in an hybrid way to borrow adult data for pediatric study using innovative statistical techniques. The workshop discusses the recruitment challenges involved and then attendees will play a role of providing their thoughts on pediatric recruitment challenges. The workshop explains how well a study can be designed in an innovative way with appropriately utilizing the statistical techniques with case study to overcome recruitment challenges.

Learning Objectives

Discuss how to borrow historical adult data for pediatric study where recruiting pediatric patients is a huge challenging task.

Chair

Rajesh Kumar Gajendran, SR, MSc

Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-670-L04-P; CME 1.00; RN 1.00

Influencers are community connectors that can be leveraged to expand patient recruitment, particularly for hard-to-engage populations. We will discuss how influencers can engage patient audiences for clinical trial participation.

Learning Objectives

Discuss the power of influencer marketing to improve clinical trial participation, including among diverse populations; Describe how to build an effective influencer campaign — matching the right influencer to your objectives; Develop effective digital assets; Identify how to maximize exposure among a captivated audience.

Chair

Sarah McKeown-Cannon

Speaker

Using Influencers to Recruit Hard-to-Engage Populations
Lara Lane

Using Influencers to Recruit Hard-to-Engage Populations
Jane Elizabeth Myles, MSc

Using Influencers to Recruit Hard-to-Engage Populations
Dezbee McDaniel