FDA RELEASES GUIDANCE FOR COMPLYING WITH PEDIATRIC RESEARCH EQUITY ACT

The FDA recently released a draft guidance on how to comply with the Pediatric Research Equity Act, which was signed into law Dec. 3, 2003, and requires new drug applications (NDAs) and biologics licensing applications (BLAs) to contain a pediatric assessment unless a waiver or deferral has been approved by the FDA.

The guidance, published in the Sept. 7 Federal Register, covers the pediatric assessment, the pediatric plan, waivers and deferrals, compliance issues and pediatric exclusivity provisions.

A pediatric assessment should be submitted with the application for the NDA or BLA, or new indication or dosage, etc. Failure to submit a pediatric assessment could result in the product being misbranded, the guidance states. The assessment should include data gathered from pediatric studies using appropriate formulations for each age group in order to: assess safety and effectiveness for the claimed indications; and support dosing and administration for each pediatric subpopulation for safety and efficacy.

The agency noted that data submitted in the pediatric assessment will depend on the nature of the application, the underlying disease or condition being treated and what is known about the product in pediatric populations.

Moreover, applicants are not required to conduct separate safety and effectiveness studies in pediatric patients in every case. The guidance also clarified that a pediatric plan is a statement of intent that outlines the pediatric studies and addresses the development of an age-appropriate formulation. The FDA stressed that early consultation and discussions are particularly important for products intended to treat life-threatening or severely debilitating illnesses.

To view the draft guidance, "How to Comply with the Pediatric Research Equity Act," go to http://www.fda.gov/cber/gdlns/pedreseq.pdf (http://www.fda.gov/cber/gdlns/pedreseq.pdf).