Introduction

Patients are angry that a once-promising drug in development has disappeared without a trace. Companies wait before developing a generic drug because they do not know how many others are in the pipeline. Physicians express frustration at company statements misrepresenting the data about a medication. In each of these scenarios, key information that could resolve the issue may have been transmitted to the U.S. Food and Drug Administration (FDA), but manufacturers’ efforts to maintain confidentiality protections and the FDA’s current disclosure rules supporting those claims keep the information from the public eye. Can the FDA take better approaches to transparency that can simultaneously support the public health and not undermine appropriate recognition of trade secrets?

This issue of the Journal of Law, Medicine & Ethics is devoted to the Blueprint for Transparency at the U.S. Food and Drug Administration. Developed by a team of researchers from Harvard Medical School/Brigham and Women’s Hospital, Yale, and Johns Hopkins, and supported by funding from the Laura & John Arnold Foundation, the Blueprint sets forward 18 specific recommendations for agency action on transparency. These ideas support greater disclosure about product applications, failed and withdrawn products, and scientific studies. To encourage discussion on these ideas, the editors invited commentaries from a range of experts in medicine, law, public policy, and regulatory science.

The first commentary is by Robert Califf, who was FDA Commissioner from February 2016 to January 2017. In his commentary, among other points, Dr. Califf tempers his support for greater transparency with caution about some of the difficulties in implementing several of the recommendations. He concludes with “unabashed enthusiasm” about FDA correcting misinformation in the marketplace and calling for FDA to provide more rapid guidance on its thinking about key scientific topics.

The second commentary is by Daniel Carpenter from the Government Department at Harvard University. Professor Carpenter argues that the Blueprint should have tackled transparency issues related to the influence of regulated industry — including disclosure of all potential avenues of this influence in the guidance and rulemaking process. He also notes that countering misleading information in the market, while important, is a fraught and politicized task.

The third commentary is by Amy Kapczynski and Jeanie Kim from Yale Law School. They discuss the practical limitations of the Freedom of Information Act as a route to obtain information from FDA. They also provide an analysis of FDA’s legal authority to disclose information proactively.

The fourth commentary is by Nathan Cortez from the Dedman School of Law of Southern Methodist University. He calls on FDA to lose its traditional reticence to question industry disclosures under securities laws, by establishing guidelines for identifying and correcting misleading information.

The fifth commentary is by Peter Doshi at the University of Maryland School of Pharmacy and Tom Jefferson at the Centre for Evidence Based Medicine of the University of Oxford. They discuss publication and other biases that, in their view, would be ameliorated by release of certain types of clinical trial data by FDA. They do not support the Blueprint’s idea of having an independent organization or group review proposals for scientific merit before releasing data.

The final commentary is by Michael Carome and Sammy Almashat at Public Citizen. They emphasize the benefits of disclosing information about product applications that are not approved, or are withdrawn or abandoned. These include more rapid abandonment of unfruitful lines of research and clinical practice, to the benefit of patients, as well as the more efficient pursuit of more fruitful avenues of exploration, also to the benefit of patients.

What emerges from this collection of perspectives is a clear sense that transparency supports the FDA’s mission as a public health agency and that it should be seen as the norm, with exceptions carved out to account for trade secrecy protections, rather than the other way around. There is general recognition that a strong foundation in transparency can contribute to enhanced patient outcomes, efficient market dynamics, and a greater public trust in the extremely important work of the FDA.