Drugs

Suggestions for Pediatric Drug Guidance

Pharmaceutical Research and Manufacturers of America (PhRMA) says an FDA draft guidance on scientific considerations for pediatric drug development under the Pediatric Research Equity Act (PREA) and the Best Pharmaceuticals for Children Act (BPCA) “offers valuable direction on various important scientific considerations for pediatric drug development programs, including considerations regarding the appropriate design and timing of pediatric studies and the use of pediatric extrapolation.” The association’s 7/17 comment letter identifies several areas PhRMA says would benefit from further discussion, confirmation, clarification, and revision:

recognize the success of the two laws in generating pediatric labeling;
address outstanding issues in FDA implementation of the FDA Reauthorization Act amendments to PREA about molecularly targeted pediatric cancer investigations;
describe FDA’s role in fostering global alignment on pediatric studies;
expand the guidance content on pharmacokinetic studies and formulation development;
clarify the section on safety information for pediatric studies;
expressly recognize that the optimal timing of pediatric studies will vary case-by-case; and
provide additional detail on how FDA expects applicants to address the challenges associated with drug development for the neonatal population.

The Biotechnology Innovation Organization gives several general comments:

guidances that are expected to be read in conjunction would benefit from a common structure to allow quick cross-referencing;
FDA should explain how it will align with other health authorities on pediatric plans;
consider the chilling effect that can occur if the use of BPCA is limited to only studies that are above and beyond those required by PREA;
a policy to not issue written requests based on PREA studies alone is a major change that is not justified based on data included in the guidance;
an observation that PREA has resulted in more labeling changes than BPCA does not support a change in position that FDA will only offer written requests for additional studies beyond those required by PREA;
the agency should more broadly allow a flexible approach; and
the document is sometimes repetitive and confusing to the reader.

AbbVie says it “shares FDA’s goal of ensuring adequate testing of drug and biological products, and is fully supportive of the agency’s goal of evidence-based pediatric pharmacotherapy and the spirit and intent of PREA and BLCA.” It says it generally expects that FDA’s approach to the scientific considerations associated with pediatric medical product development will be aligned with the principles in an International Council for Harmonization E11 guideline and addendum where it is reasonable to do so. It recommends that FDA insert appropriate references to ICH E11 throughout the draft guidance once it is finalized.

Boehringer Ingelheim submitted technical edits.

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