Pediatric Clinical Development

Organizers: Freda Cooner (Amgen), Yeh-Fong Chen (FDA)
Chair: Freda Cooner (Amgen)
Vice Chair: Miaomiao Ge (Boehringer-Ingelheim)

Speakers:
Mark Rothmann (FDA)
Barbara Wendelberger (Berry Consultants)
Forrest Williamson (Lilly)

Abstracts:

Title: Bayesian Analysis in Pediatric Settings
Speaker: Mark Rothmann (FDA)

Typically, the effects of medical products are studied in children after marketing approval in adults. Despite success in adult studies, clinical trials in children may fail to demonstrate efficacy. This may be due to differences between adults and children in physiology, disease pathology, exposure to the product, or tolerability of the product. This could also be due to an undersized pediatric study. Undersized studies can produce inconclusive results with little incentive to further study the medical product in children. Different but related questions can be addressed together. The question of whether a drug works in children is not same as whether it works in adults, but it’s not completely separate, either. Decisions on the effectiveness and safety of medical products in children can consider relevant adult data through Bayesian approaches.

Title: Leveraging Bayesian modeling in pediatric clinical trial design
Speaker: Barbara Wendelberger (Berry Consultants)

Clinical trials conducted in pediatrics differ from those conducted in adults due to a combination of biological, medical, and ethical factors. These factors impact the trial design process in both obvious and nuanced ways. In this talk, I will first focus on the differences between pediatric and adult trials. Next, I will discuss how we can leverage Bayesian modeling to address issues that arise in the pediatric landscape to design efficient pediatric clinical trials. Finally, I will provide examples of statistical strategies that I have employed during the design of several pediatric clinical trials.

Title: Closing the Gap – Accelerating Pediatric Drug Development
Speaker: Forrest Williamson (Lilly)

The traditional drug development paradigm focuses on studying novel therapies in adults first, from Phase I through Phase III. Pediatric studies often initiate only after a therapy has demonstrated safety and efficacy in adults. This can cause lengthy lag times between approval in adults and understanding dose, safety, or efficacy in pediatrics. This is enhanced by the fact that pediatric patients and their caregivers have less motivation to participate in clinical trials if the therapy is already on the market, making recruiting more difficult and adding to the time to complete pediatric studies. There is a need to balance the risk of including pediatric patients in trials earlier, versus the time a therapy is approved and on the market without information on pediatrics. This talk focuses on opportunities to include pediatric patients earlier in clinical programs, such as the inclusion of adolescents with adults.