In a final guidance document, the US Food and Drug Administration provides sponsors with a detailed framework for how to prepare and submit pediatric study plans. The final guidance supplants a draft guidance published in 2016.
The new pediatric study plan guidance follows on from regulations and laws promulgated in the late 1990s and early 2000s that addressed the dearth of pediatric data in drug development. These rules aimed to increase pediatric studies for exclusive or patent-protected drugs, and to address a prior lack of data supporting pediatric use and dosing of drugs for claimed indications.
The Pediatric Research Equity Act (PREA), passed in 2003 and reauthorized in 2012, requires sponsors making applications for drugs or biologics subject to the act to submit an initial pediatric study plan (iPSP) early in the course of drug development. This early dialog, according to the guidance, “is intended to result in a more efficient pediatric drug development program.”
Sponsors must submit iPSPs if they plan to submit a marketing application of supplement for a new active ingredient, new indication, new dosage form or regimen, or a new route of administration; an exception is made for indications that have received orphan designations.
Starting 18 August 2020, sponsors planning applications for new active ingredients subject to the molecularly targeted cancer drug provision of PREA must also submit iPSPs, whether or not the indication has orphan status. Biosimilars not determined to be interchangeable with their referents are considered new active ingredients for this purpose…