For observers of pharmaceutical regulation and the Food and Drug Administration, these are uncertain times. Events in late 2016 raised concerns that the FDA's evidentiary standards were being weakened, compromising the agency's ability to adequately perform its regulatory and public health responsibilities. Two developments most directly contributed to these fears—the approval of eteplirsen, a treatment for Duchenne muscular dystrophy, against the recommendations of both FDA staff and an advisory committee and the December 2016 signing of the 21st Century Cures Act, (...) which encouraged greater use by the FDA of “real-world” evidence not obtained through randomized controlled trials. The arrival of the Trump administration—with its deregulatory, industry-friendly approach—has only amplified concerns over the future of the FDA. It is too early to know whether the recent developments are truly harbingers of an FDA less likely to prevent unsafe or ineffective products from reaching the market. But elements in the two events—the role of patient narratives in deliberations regarding eteplirsen and the enthusiasm for real-world evidence in the 21st Century Cures Act—raise critical issues for the future of evidence in the FDA's work. The rigorous, inclusive approach under way to consider issues related to real-world evidence provides a model for a similarly needed inquiry regarding public participation in FDA decision-making. (shrink)

Ethics advisory groups, in various forms, have existed for at least 50 years in the United States and other countries. In science and biomedicine, four principal types of committees can be distinguished: policy-making and/or advisory committees, health professional association committees, health care ethics committees, and research ethics committees. Overall, these bodies have been of use to governments, policy makers, health care professionals, and the public in considering ‘what is ethical’ and ‘what is unethical’ in areas such as research involving humans (...) or animals or environmental questions such as whether to use genetically modified crops. This paper concentrates on a particular and... (shrink)

Comparative assessments of national bioethics commissions in the United States commonly look at the differences among these groups over their forty‐year history. A particular focus has been differences in the membership, mission, methods, and reports of the President's Council on Bioethics, which was active from 2001 until 2009, compared to those of its predecessors and the recent Presidential Commission for the Study of Bioethical Issues, active from 2009 until 2016. The differences are real, but disproportionate attention to them can obscure (...) the substantial similarities in commissions’ structure and function throughout the history of expert bioethics advice to government. As the Trump administration considers what role, if any, a bioethics commission will play in its work, it would be well served to consider how choices regarding the design of such a group and the topics it examines can best facilitate the unique contributions it can make to the government and to the country. (shrink)