This guidance is intended to assist industry developing drug products to comply with the pediatric study requirements under the Pediatric Research Equity Act (PREA), and to describe the process for qualifying for pediatric exclusivity and the protections that pediatric exclusivity offers under the Best Pharmaceuticals for Children Act (BPCA). In 2010, the Biologics Price Competition and Innovation Act of 2009 extended provisions of the BPCA to biological products.
Note that section 505B(b) of the Federal Food, Drug, and Cosmetic (FD&C) Act on already marketed drugs and section 409I of the Public Health Service (PHS) Act are only briefly addressed in this guidance. Future guidance may address these issues in greater detail. Furthermore, this guidance only briefly addresses the Food and Drug Administration Reauthorization Act of 2017’s (FDARA’s) amendments to section 505B of the FD&C Act relating to requirements that sponsors of certain adult oncology drugs with molecular targets that are determined to be substantially relevant to the growth or progression of a pediatric cancer submit reports on molecularly targeted pediatric cancer investigations.
The scientific aspects of a pediatric program (e.g., considerations regarding data in pediatric subjects, timing of pediatric studies) are addressed in the draft guidance for industry Pediatric Drug Development Under the Pediatric Research Equity Act and the Best Pharmaceuticals for Children Act: Scientific Considerations (May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This guidance, along with the draft guidance for industry Pediatric Drug Development Under the Pediatric Research Equity Act and the Best Pharmaceuticals for Children Act: Scientific Considerations, revises and replaces the draft guidance for industry How to Comply With the Pediatric Research Equity Act. In addition to addressing the PREA topics covered in the earlier draft guidance (i.e., the pediatric assessment, pediatric plan, waivers and deferrals, compliance issues, and pediatric exclusivity provisions), this guidance addresses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.
In general, FDA’s guidance documents do not establish legally enforceable responsibilities. Instead, guidances describe the Agency’s current thinking on a topic and should be viewed only as recommendations, unless specific regulatory or statutory requirements are cited. The use of the word should in Agency guidances means that something is suggested or recommended, but not required.
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