BackgroundThe U.S. Food and Drug Administration (FDA) traditionally has kept confidential significant amounts of information relevant to the approval or non-approval of specific drugs, devices, and biologics and about the regulatory status of such medical products in FDA’s pipeline.ObjectiveTo develop practical recommendations for FDA to improve its transparency to the public that FDA could implement by rulemaking or other regulatory processes without further congressional authorization. These recommendations would build on the work of FDA’s Transparency Task Force in 2010.MethodsIn 2016-2017, we convened a team of academic faculty from Harvard Medical School, Brigham and Women’s Hospital, Yale Medical School, Yale Law School, and Johns Hopkins Bloomberg School of Public Health to develop recommendations through an iterative process of reviewing FDA’s practices, considering the legal and policy constraints on FDA in expanding transparency, and obtaining insights from independent observers of FDA.ResultsThe team developed 18 specific recommendations for improving FDA’s transparency to the public. FDA could adopt all these recommendations without further congressional action.FundingThe development of the Blueprint for Transparency at the U.S. Food and Drug Administration was funded by the Laura and John Arnold Foundation.
The US Food and Drug Administration (FDA) has been the standard of drug regulation worldwide for decades. In 1962, in response to the thalidomide tragedy, Congress directed the FDA to evaluate the effectiveness of drugs based on "adequate and well-controlled investigations, including clinical investigations" conducted by qualified experts.Backed by this congressional enactment, the FDA crafted groundbreaking administrative regulations (eg, the requirements of a prespecified protocol, placebo or active controls, the phasing of clinical investigations, and informed consent from patients) that have guided the global clinical trial enterprise ever since. 1 The FDA's regulations revolutionized the evaluation of drugs in the United States and abroad.However, the European Medicines Agency (EMA), the counterpart to the FDA in the European Union, is poised to leapfrog ahead of the FDA in an equally important drug regulatory space of transparency. In October 2016, the EMA published on its website the clinical study reports for 2 drugs (carfilzomib and lesinurad) it recently approved. In so doing, the EMA VIEWPOINT
Sharing of final research data from clinical research is an essential part of the scientific method. The U.S. National Institutes of Health require some grant applications to include plans for sharing final research data, which it defines as the factual materials necessary to document, support, and validate research findings. In the U.S., however, the Privacy Rule adopted under the Health Insurance Portability and Accountability Act impedes the sharing of final research data. In most situations, final research data may be shared only where all information that could possibly be used to identify the subject has been deleted, or where the subject has given authorization for specific research, or an Institutional Review Board has granted a waiver.
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