Keywords: pediatric, extrapolation, borrowing
The Pediatric Research Equity Act (PREA) and the Best Pharmaceuticals for Children Act aim to increase the safety and efficacy information on the use of drugs in pediatric populations. While these regulations have encouraged substantial investments in pediatric drug research, many barriers to development remain. In particular, many of the traditional approaches to clinical trial design used in adult populations are not applicable due to recruitment issues or ethical reasons in the pediatric setting. Given these challenges and the differences across therapeutic areas, the approaches currently used in pediatric drug development represent a continuum, in terms acquiring new evidence of efficacy and safety. On one end of this continuum, certain therapeutic areas have begun the practice of entirely relying on extrapolation of the adult efficacy data to pediatric patients, without collecting any additional efficacy data. This approach is generally used after showing similarity in the exposure-response relationships in the two populations. On the other end of the spectrum, other therapeutic areas continue to perform full pediatric development programs, including adequate and well-controlled trials within the pediatric population, powered for efficacy and safety evaluations. This round table will discuss the settings in which each approach is appropriate and the challenges each brings. In addition, we will discuss innovative designs and statistical methods that could fall along this continuum, e.g., Bayesian methods that leverage adult data or information across a drug class, and the applicability and appropriate context of such approaches.