Evidence-Based Approach in Pediatric Drug Development: Progress and Lessons Learned — Professional Development Continuing Education Course
ASA, Biopharmaceutical Section
Evidence-based medicine is required for adults and nothing less so for the pediatric population. Pediatric trials are unique for several reasons: small number of patients, limited physiologic data, and ethical complexity increase the difficulty and costs of pediatric trials. Extrapolation of adult or other pediatric data has facilitated the conduct of pediatric product development trials, subsequent marketing approval, and labeling. This approach reduces the number of children that need to be enrolled and the type of clinical trials that need to be conducted for pediatric product marketing approval.
During the first part of the short course, we will introduce the general scientific framework of extrapolation and provide overview of available design and analysis approaches. We’ll elaborate on the use of Bayesian hierarchical model (BHM) and discuss the practicality of the underlying assumptions associated with it. Finally, we’ll discuss extensions of BHM to handle possible deviations from underlying assumptions and implementation using the R.
Later, we will focus on the regulatory aspects and case studies. A real-life case study will be included to illustrate the practical implementation and regulatory hurdles. The aim of the short course is to enable participants to apply the extrapolation techniques themselves in the real-life trial.
Instructor(s): Satrajit Roychoudhury, Pfizer Inc; Margaret Gamalo , Pfizer Inc.; Robert ‘Skip’ Nelson, Johnson & Johnson