Improving development of therapies: A collaborative approach with the Food and Drug Administration (FDA), industry and academia

Monday, April 28, 2025

9:15am – 10:45am HST

Location: Convention Center: 316 A

CE: 1.5

Selected By: ASPN

Chair(s)

Katherine Dell, MD (she/her/hers)

Professor of Pediatrics and Director of Clinical and Translational Research
Case Western Reserve University School of Medicine
Cleveland, Ohio, United States
MK

Mona Khurana, MD (she/her/hers)

Clinical Team Leader
FDA
Rockville, Maryland, United States

Disclosure(s):

Katherine Dell, MD: I do not have any relevant financial / non-financial relationships with any proprietary interests.

Mona Khurana, MD: I do not have any relevant financial / non-financial relationships with any proprietary interests.

Most medicines available in the U.S. are not approved for children, resulting in off-label use. Inherent issues with off-label medicine use include clinician reluctance to prescribe these products and the potential for adverse drug reactions. Since the early 2000s, drug development for children has improved because of the passage of important legislation in the U.S. However, on average, there remains an 8-to-9-year lag between the time of medicinal approval in adults to approval in pediatric populations. This session is intended to explore the myths and realities about pediatric therapeutics development from bench to bedside. Examples of successes and failures in the development of therapies for children will be reviewed. The session will also examine potential collaborations to optimize the development of therapies for children. Specifically, cross-functional roles in the pediatric therapeutics development “ecosystem” will be discussed, to enhance future collaborations to decrease the delay in medicinal approvals for children.

Learning Objectives:

Review the regulatory framework for medicine development in children and explain how this regulatory framework is applied to pediatric clinical research.
Compare and contrast industry-sponsored pediatric drug development programs to examine factors that can be applied to pediatric clinical research
Characterize the important role that academic pediatricians play in therapeutics development and discuss the utility of regulatory-grade research in academic pediatrics

Presentations:

9:15am – 9:45am HST

FDA: Regulatory Definitions and Perspectives
Speaker: Shamir Tuchman, MD, MPH (he/him/his) – U.S. Food and Drug Administration (FDA)
9:45am – 10:15am HST

Pediatric Drug Development Successes and Failures: The Industry Perspective
Speaker: Deepa H. Chand, MD, MHSA (she/her/hers) – University of Illinois College of Medicine
10:15am – 10:45am HST

The Role of Academia in Drug Development
Speaker: Bradley A. Warady, MD – Children's Mercy