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biostatistics, public health, and health policy. Those experts agreed to prepare both this letter report, which focuses on question 1 of the charge, by July 2010 and a final report that addresses all the questions in the charge by 2011.

For the present letter report, the committee held one meeting, which included an open session in which it heard from representatives of FDA and representatives of the Agency for Healthcare Research and Quality (AHRQ) and the National Institutes of Health (NIH), which funded this report with FDA. The committee provided an opportunity for other stakeholders to present their perspectives and concerns at the meeting. The committee conducted searches of the literature on the ethics of clinical trials and informed consent relevant to postmarketing clinical trials. This letter report does not, however, present a comprehensive literature review of the subject.

Given the short period available for preparing this letter report, the committee focused on identifying a conceptual framework to guide its analysis of the ethics of the design and conduct of postmarketing safety research required by FDA, including key issues that need to be taken into account in assessing ethics and informed consent in randomized controlled trials. In developing this framework, and in its explication in this letter report, the committee relied on the extensive body of codes, regulations and guidance on the ethics of research involving human participants, much of which is built around a commitment to several basic moral principles, including beneficence, respect for persons and their autonomy, and justice. The committee did not enumerate all the ways in which the issues raised in this letter report can affect the ethics of a study, did not detail how the various issues should be weighed against one another, and did not explore in depth issues related to the ethical and scientific justifications of randomized controlled trials. A more detailed analysis of those issues and their implications and effects will be included in the committee’s final report.

The committee’s conceptual framework consists of four classes of considerations, as shown in Box 2. In accordance with the framework, the remainder of this letter report is organized in four major sections: the public health context of drug safety, regulatory science and public accountability, design considerations, and additional ethical obligations to research participants.


The ethics of any postmarketing study required by FDA, including randomized controlled trials, should be assessed in the context of FDA’s mission to promote and protect public health. The safety of the US drug supply contributes to the nation’s health, and FDA is the agency responsible for ensuring this safety. As stated by the FDA commissioner and deputy commissioner, “to be healthy, people need access to … innovative, safe, and effective medical products” and “FDA’s job is to support this access and, in doing so, to promote health, prevent illness, and prolong life” (Hamburg and Sharfstein, 2009). With specific reference to drugs, FDA’s job includes (FDA, 2010a)

  • “Protecting the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products”

  • “Advancing the public health by helping to speed product innovations”

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