Two new draft guidances released by the FDA last week share the agency’s recommendations on designing effective pediatric trials and dealing with various issues inherent to developing pediatric therapies.

The guidances replace the 2005 draft guidance “How to Comply With the Pediatric Research Equity Act” and cover requirements of the Pediatric Research Equity Act (PREA) and the Best Pharmaceuticals for Children Act (BPCA).

One guidance, “Pediatric Drug Development Under the Pediatric Research Equity Act and the Best Pharmaceuticals for Children Act: Scientific Considerations,” deals with the clinical, scientific and ethical issues in developing pediatric products and points out that important clinical data is often missing when sponsors file for a pediatric indication on an adult product.

Primarily, the guidance explains, data gaps can lead to inappropriate dosing, an increased risk of adverse events and even undermining of clinician confidence in a newly approved therapy. The lack of pediatric labeling also may result in denying pediatric patients therapeutic advances “due to the lack of information regarding use,” the guidance states.

The second document, “Pediatric Drug Development: Regulatory Considerations — Complying with PREA and Qualifying for Pediatric Exclusivity Under the BPCA,” advises sponsors on how to design effective pediatric studies that qualify for pediatric exclusivity.

This document covers everything in the 2005 document (the pediatric assessment, pediatric plan, waivers and deferrals, compliance issues, and pediatric exclusivity provisions) but also offers additional guidance on meeting the statutory requirements of adverse event reporting, pediatric study plans, deferral extensions and noncompliance with requirements under PREA.

The FDA is taking public comments on both drafts through Aug. 17.

Read the draft scientific guidance here: https://bit.ly/3ooIv1s.

Read the draft regulatory guidance here: https://bit.ly/43fBizT.