New FDA Draft guidance: Pediatric Drug Development: Regulatory Considerations — Complying With the Pediatric Research Equity Act and Qualifying for Pediatric Exclusivity Under the Best Pharmaceuticals for Children Act

 The US government passed the Pediatric Research Equity Act (PREA) in 2003. A number of prior regulations and FDA reauthorizations supported that law.  This new draft guidance is specifically focused on:

37. The scientific aspects of a pediatric program (e.g., considerations regarding data in pediatric Pediatric Drug Development Under the Pediatric Research Equity Act and the Best Pharmaceuticals for Children Act: Scientific Considerations (May 2023).  

    This guidance, along with the draft guidance for industry Pediatric Drug Development Under the Pediatric Research Equity Act and the Best Pharmaceuticals for Children Act: Scientific Considerations, revises and replaces the draft guidance for industry How to Comply With the Pediatric Research Equity Act.  In addition to addressing the PREA topics covered in the earlier draft guidance (i.e., the pediatric assessment, pediatric plan, waivers and deferrals, compliance issues, and pediatric exclusivity provisions), this guidance addresses statutory changes relating to adverse event reporting, pediatric study plans (PSPs), deferral extensions, and noncompliance.

UPDATE on 18May23 - Note that there was a second draft guidance released that covers Scientific Considerations including: 1) Formulation Development, 2) Nonclinical Information, 3) Clinical Pharmacology, 4) Safety Information, and a major section on Pediatric Extrapolation.

This guidance goes into when a pediatric version of a drug is required to be developed and when it is not (e.g., adult only diseases, orphan products).  For testing of oncology drugs in pediatric populations, upon sponsor request, the FDA may waive the requirement to submit pediatric assessments or reports if certain criteria are met 1)extremely small patient group, 2) likelihood of the drug being unsafe or ineffective or 3) the drug is unlikely to have benefit greater than existing therapies and is unlikely to be used in a substantial number of pediatric patients. 

The FDA may also issue Written Requests (WR) to sponsors "requesting submission of a study or studies intended to provide meaningful health benefits in the pediatric population".  Interestingly, "Completion of studies described in a WR is voluntary." Other details around content of the WR and its implications e.g., potential 6-month extension of exclusivity, are also included in the draft. 

While there is not much here for my bioanalytical colleagues, those working on drug development strategy will find this interesting

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