FDA-Regulated Research Track

2019 AER Conference

E04 - Assessing the Prospect of Direct Benefit in Early-Phase Pediatric Studies

Speaker(s)

• 

  Melanie Bhatnagar, MD
  

  Medical Officer, Food & Drug Administration
  
• 

  Donna L. Snyder, MD
  

  Senior Pediatric Ethicist and Team Leader, Food and Drug Administration
  
• 

  Albert J. Allen, MD, PhD
  

  Senior Medical Fellow, Pediatric Capabilities, Global Medical Policy Strategy and Operations, Lilly Research Labs, Eli Lilly and Company
  

FDA regulations permit IRBs to approve pediatric research that presents more than a low level of risk if participation in the research holds out the prospect of direct benefit for the individual subjects. Given that there is no regulatory definition for prospect of direct benefit, how should it be defined? How should IRBs consider the prospect of direct benefit in the context of early-phase clinical trials involving pediatric subjects? During this session, speakers and attendees will:

Learning Objectives:

• Discuss IRB considerations for assessing prospect of direct benefit in pediatric studies
• Examine how to assess prospect of direct benefit in the setting of early-phase clinical trials
• Use case examples to explore concepts such as the appropriate use of non-human data and the applicability of biomarkers to reflect clinical benefit