FDA Provides Detailed Guidance on Developing Drugs for Children
(Thursday, May 25, 2023)
Two new FDA Guidance Documents released last week describe in great details the various regulatory and scientific considerations for developing drugs and biologics for children. Two laws passed more than 20 years ago for pediatric drug developments mandate that all new drug approval applications either contain pediatric data or the plan to generate such data in a timely fashion post-market approval. However, pediatric indications are still treated as secondary priorities by most developers for various practical, logistical, and financial reasons. The Guidance Docs try to address these issues.
From a regulatory point of view the Best Pharmaceuticals for Children Act (BPCA) of 2002 and the Pediatric Research Equity Act (PREA) of 2003 together created several requirements for developing drugs for children. The main difference between BPCA and PREA is that the requirements under BPCA for pediatric studies are optional but those under PREA are mandatory. BPCA addresses various regulatory paths to developing pediatric indications and provides incentives for doing that, while PREA discusses the necessity of conducting research in pediatric populations. The key discussions in BPCA are regarding approval for products intended for life threatening and severely debilitating diseases where pediatric studies could delay marketing application submission. PREA on the other hand established specific timelines for the pediatric studies but allowed alternate sources of data such as non-clinical studies to support pediatric indications.
The first guidance document discusses regulatory issues such as the timing and criteria for the full or partial waiver requests, FDA meetings to discuss pediatric study designs, and pediatric exclusivity requirements. The second guidance discusses scientific issues such as considerations for pediatric formulation development, non-clinical and clinical study designs, modeling for pediatric extrapolation, and other considerations such as post-market, real-world data to support pediatric application.
The guidance documents emphasize that pediatric study discussions should initiate at the End-of-Phase 2 stage of development. The guidance also clarifies that FDA will consider deferred or delayed pediatric study plans for serious and life-threatening disease for adults, cancer, and diseases that rarely affect children. Orphan drugs are exempted from these requirements unless the target indication is intended for pediatric population. In essence the two guidance documents emphasize that developing drugs for pediatric populations should not be deprioritized over adult populations.
Dr. Mukesh Kumar
Founder & CEO, FDAMap
Linkedin: Mukesh Kumar, PhD, RAC