FDA Publishes First Pediatric Drug Study Non-Compliance Letters and Responses

The FDA began publishing the first non-compliance letters and responses from drug sponsors who have failed to submit required pediatric assessments by the final due date. Pediatric assessments, required under Section 505B of the Food, Drug, and Cosmetics Act (FDC Act) (21 U.S.C. sec. 355c) are intended to determine whether the safety and effectiveness of a drug are substantially similar in adult and pediatric patients.

Pediatric Studies

Pediatric research is necessary for drugs that are used in children because children can react differently to drugs than adults; there may be different safety concerns or dosage requirements for children. The Pediatric Research Equity Act (PREA) (P.L. 107-109) describes the circumstances in which drug companies must test their products for use in children. Pediatric studies can be waived, for example, in drugs that treat conditions that do not exist in children. Depending on circumstances, the FDA has allowed sponsors to defer pediatric studies, but was without recourse if sponsors missed deadlines.

PREA Non-Compliance

The Food and Drug Administration Safety and Innovation Act (FDASIA) (P.L. 112-144) reauthorized PREA and gave the FDA additional authority regarding pediatric studies. At a sponsor’s request, the FDA can grant extensions for deferred pediatric studies if there is good cause for delay, such as difficulty recruiting patients for the study despite diligent attempts. Conversely, the FDA can send a non-compliance letter to sponsors who fail to seek or obtain a deferral extension, fail to submit deferred pediatric studies by the final agreed due date, or fail to request approval for a required pediatric formulation. Section 505B(d)(1) requires the FDA to post PREA non-compliance letters and responses online.

The FDA intends to post PREA non-compliance letters and responses only after it has determined that the non-compliance letter was proper. If a sponsor has a pending deferral extension request or waiver, letters will not be posted until the pending matter is resolved. The FDA will continue to update its online postings as sponsors fulfill or are otherwise released from pediatric study obligations. Of the first five letters and responses posted, three are to Genzyme Corporation, and the others are to Amedra Pharmaceuticals and The Medicines Company. Each sponsor has filed a response, which is posted on the FDA’s website. The responses generally discuss each sponsor’s intention to complete the required pediatric studies as soon as possible.