Pediatric Research: Products Studied under Two Related Laws, but Improved Tracking Needed by FDA
Highlights
In 2007, Congress reauthorized two laws, the Pediatric Research Equity Act (PREA) and the Best Pharmaceuticals for Children Act (BPCA). PREA requires that sponsors conduct pediatric studies for certain products unless the Department of Health and Human Services' (HHS) Food and Drug Administration (FDA) grants a waiver or deferral. Sponsors submit studies to FDA in applications for review. BPCA is voluntary for sponsors. The FDA Amendments Act of 2007 required that GAO describe the effect of these laws since the 2007 reauthorization. GAO (1) examined how many and what types of products have been studied; (2) described the number and type of labeling changes and FDA's review periods; and (3) described challenges identified by stakeholders to conducting studies. GAO examined data on the studies from the 2007 reauthorization through June 2010, reviewed statutory requirements, and interviewed stakeholders and agency officials.
Recommendations
Recommendations for Executive Action
| Agency Affected | Recommendation | Status |
|---|---|---|
| Food and Drug Administration | The Commissioner of FDA should move expeditiously to track applications upon their submission and throughout its review process and maintain aggregate data, including the total number of applications that are subject to PREA and whether those applications include pediatric studies. |
In its comments on GAO's report, FDA stated that in May 2011 the agency made an improvement to DARRTS that was not in place during the time of GAO's review. FDA added a code to DARRTS that indicates whether or not a particular filed application triggers PREA. The agency also noted that RMS-BLA already had a code in place to track applications filed under PREA. Since GAO issued its report, FDA has taken steps to implement this recommendation. FDA has made improvements to its systems for tracking applications, including those subject to PREA. FDA uses the Document Archiving, Reporting, and Regulatory Tracking System (DARRTS) for tracking drug applications and the Regulatory Management System for Biologics Licensing Application (RMS-BLA) for tracking biological product applications. FDA modified both systems to allow it to track information as applications are filed with the agency. For example, it can be used to track new applications that trigger PREA. As part of the filing checklist for all applications, FDA staff has revised its filing checklist so that all applications that are subject to PREA are identified prior to filing. In addition, FDA staff are directed to schedule a meeting with the Pediatric Review Committee for all applications that are subject to PREA. According to FDA, the agency also tracks information regarding the granting of waivers and deferrals through documentation from the Pediatric Review committee minutes and verifies the information in all approval letters. This is a supplementary mechanism to ensure that all applications subject to PREA are identified and tracked. FDA also tracks information related to the completion of deferred pediatric studies under PREA on an ongoing basis in order to post all information required under PREA. FDA publishes reports containing this information on its website. The procedures FDA has put in place should allow it to better track applications that are subject to PREA and maintain aggregate data as called for by GAO's recommendation.
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