FDA Proposes Policies for Pediatric Studies

Study sponsors will be subject to a new requirement for pediatric investigations beginning August 18, with new Food and Drug Administration draft guidance offering transitional information. 

The Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act (PREA) set in motion a paradigm shift to incentivize companies to run pediatric studies for drugs intended for adult populations. The new policy clarifications come due to amendments feeding into this goal.

FDA Reauthorization Act of 2017 (FDARA) section 504 amended section 505B of the FD&C Act to require any original new drug application or biologics license application to conduct pediatric investigations of certain molecularly targeted cancer drugs. 

FDA says FDARA “created a mechanism to require evaluation of certain novel medicines that may have the potential to address an unmet medical need in the pediatric population.” It adds “timely investigation in children” aims “to accelerate the early pediatric evaluation of these products” to “facilitate the development of appropriate new therapies for pediatric patients.”

From when an initial pediatric study plan (iPSP) will be required prior to, on or after Aug. 18 to when an iPSP can be abbreviated to seek a waiver, the draft guidance posted on Tuesday addresses ten industry questions using a Q&A format on the submission of NDAs and BLAs.

Any original NDA or BLA submitted on or after the specified date must contain reports of molecularly targeted pediatric cancer investigations unless FDA grants a deferral or waiver. 

A deferral or waiver can be granted only if the drug is intended for the treatment of an adult cancer type and is directed at a molecular target that has been determined to be substantially relevant to the growth or progression of a pediatric cancer type. FDA keeps a list of the later. 

The requirement on the submission of iPSPs will be applicable even if the adult cancer indication does not occur in the pediatric population and the drug was previously an orphan designation. FDA, however, says it “recommends all sponsors developing such drugs submit an iPSP anyway because development timelines can be unpredictable and pediatric investigations will be required, unless waived, if the original application is submitted on or after” Aug. 18.

To be on the safe side, FDA recommends submitting an iPSP even for marketing applications likely to be submitted prior to Aug. 18 because “depending on the ultimate submission date of the marketing application, molecularly targeted pediatric cancer investigations may be required, and early discussion with the agency prior to application submission may facilitate the development of studies to ensure” compliance with the relevant requirements under PREA.