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Second Opinion

Redefining “Confidential”

Why greater transparency at the FDA will speed development of drugs and devices.

illustration of giant person opening a door for a smaller person

Illustration by Andre DaLoba

If there is a science-based agency that people most love to hate, it is the U.S. Food and Drug Administration. When I served as principal deputy commissioner, I faced questions and skepticism from patient advocates as well as pharmaceutical companies, from consumer activists, food producers and medical device manufacturers. 

Once, on a family trip to Hawaii, we traveled to a remote mountaintop to visit a farm and restaurant. Without cell phone service, there was no way for the office to reach me. Looking out at the spectacular vista, I exhaled deeply and, finally, felt my body begin to relax. I smiled as another guest asked the owner if his farm might be heaven on earth. Then the owner replied, “It would be if it weren’t for the damn FDA inspectors.”

Sometimes the concerns I heard about the FDA were on target, and I tried to push the agency to reverse course. Most often, however, the complaints were based on misunderstandings. When I left the federal government in 2011, it was a therapeutic experience for me to write an essay for the Journal of the American Medical Association entitled, “FDA: A Misunderstood Agency.”

It does not take Sherlock Holmes to figure out one major cause of the problem: The FDA does not do a particularly good job explaining itself. Much of the work of the agency—and many of the reasons for the actions it takes—is never disclosed to the public. Of course, some of this secrecy is necessary to protect trade secrets. However, there is a lot of other information and analysis that is now classified as “confidential,” but the agency can change the classification without Congress having to pass a new law. For example, the FDA does not generally disclose why it declines to approve drugs or devices. And it does not provide access to important datasets created to assess important questions of disease history or product safety and effectiveness. The agency has within its authority the ability to take these two steps. 

Improving transparency by redefining “confidential” is, therefore, an important strategy for the agency in order for it to engage at a more thoughtful level with patients, physicians, companies and researchers.

Recently, I worked with a team of experts from Johns Hopkins, Harvard and Yale in proposing a Blueprint for Transparency at the FDA. We made 18 recommendations in five focus areas. 

These included calling for disclosure of when applications are filed, when and why studies are placed on hold, which expedited procedures are used, and why the FDA declines to approve medical products. We also called for the FDA to disclose what is in the pipeline for generic drugs and how the agency thinks about bioequivalence for specific products. Of greatest relevance to researchers, we recommended that the agency disclose key datasets created in the regulatory process and assure access to patient level data from major clinical trials, with appropriate protections for patient privacy. 

The basic argument we made is that in the process of doing its work as a regulator, the FDA can also release information and analysis that will advance the scientific enterprise of developing safe and effective medical products. The Blueprint is available online at jhsph.edu/blueprintFDA.

In putting together the Blueprint, we received input from pharmaceutical companies, attorneys, academic experts, clinical researchers and advocates. We also received technical assistance from the FDA, which, despite the change in presidential administration, is poised to take some great steps forward.

Now that I am working at Johns Hopkins, I can see that opening up the FDA will help the clinical and research communities provide better care and make discoveries more rapidly. I also think transparency is a path for greater appreciation of an agency that plays a vital role in all of our lives.?

Joshua M. Sharfstein is associate dean for Public Health Practice and Training at the Johns Hopkins Bloomberg School of Public Health and a faculty member in pediatrics at the Johns Hopkins School of Medicine.