In July 2014, the FDA released a comprehensive report and strategic plan addressing the complex issues in developing drugs and biological products for rare diseases and accelerating the development of therapies for pediatric rare diseases. This report included a detailed summary of the background and legislative actions leading to the development of the FDA report and strategic plan, as well as a summary of a 3-day workshop that was held by the FDA in January 2014 to address issues in clinical trial design for rare diseases, and ways to encourage and accelerate the development of new therapies for pediatric rare diseases.
Multiple FDA workgroups were established to develop the strategic plan, entitled “Strategic Plan: Accelerating the Development of Therapies for Pediatric Rare Diseases.” There are four primary objectives with supporting strategies separated into two categories, either drugs/biologics or devices. The objectives include (in order of the product development process, not hierarchical order of importance): 1) Enhance foundational and translational science for pediatric rare diseases; 2) Strengthen communication, collaboration, and partnering for PRD within and outside FDA; 3) Advance the use of regulatory science to aid in clinical trial design and performance for pediatric rare diseases; and 4) Enhance FDA’s review process for PRD products. Under each of these objectives, common themes among strategies included the development of additional guidance documents by the FDA and additional training for both pediatric clinical investigators and FDA reviewers for rare and pediatric rare diseases.