Opinion | FDA Has Neglected Clinical Trial Transparency — Plus $45 Billion in Fines

The FDA recently requested $7.2 billion for its 2024 budget — an increase of $372 million from 2023. However, missing from that request is urgently-needed support for FDA enforcement of clinical trial transparency, despite thousands (and counting) of trials not reporting results as required by law.

When patients enroll in clinical trials, they reasonably assume that the research results will be shared with the public, physicians, and fellow patients. In 2019, however, our organization, Universities Allied for Essential Medicines (UAEM), found that the top U.S. universities were failing to report their results to a public database on time — and flagrantly violating federal law in the process. Unfortunately, this problem is not a new one, and neither is the FDA’s lack of urgency in solving it.

After we published our report, several universities responded by immediately submitting their overdue trial results. This suggested that the problem was not the difficulty of complying with the law, but the fact that it was rarely enforced. Two years later, we found that while university compliance with the law improved following advocacy efforts, trial sponsors were still broadly failing to prioritize access to research results, including for trials concerning FDA-approved therapies. Our findings raised serious concerns. For physicians and patients seeking to make informed decisions about the safety and value of expensive new therapies, access to results is critical.

Today, over 5,000 clinical trials are in violation of federal reporting requirements created under the FDA Amendments Act of 2007 (FDAAA), which requires timely results reporting to the ClinicalTrials.Gov database. Under FDAAA, FDA and NIH share the responsibility to ensure that sponsors of clinical trials report their results in a timely manner.

How is this system supposed to work? First, the FDA sends warning letters called “pre-notices” to sponsors suspected of violating FDAAA. If the sponsor fails to address this violation within 30 days, the FDA can initiate an investigation confirming the violation and send a notice of noncompliance. Should the violation persist after 30 days, FDA can then levy fines of $10,000 or more each day, and can even pursue criminal prosecution of the sponsor. To date, the FDA could have imposed over $45 billion in fines. In spite of the thousands of trials violating the law, the FDA has not yet collected any fines.

Through an ongoing Freedom of Information Act (FOIA) investigation, we found that the FDA has taken minimal action to enforce the law even in terms of preliminary steps. As of April 2021, FDA had sent only 57 pre-notices. Not much has changed since then. In January, Rep. Frank Pallone (D-N.J.) sent a letter to the FDA and NIH on behalf of the House Energy and Commerce Committee, criticizing agency inaction and demanding details about the enforcement of clinical trials reporting requirements. Responding to requests for comment on the letter, the FDA revealed that between 2007 and 2023, the agency sent only 92 pre-notices and four notices of noncompliance, even as noncompliant trials numbered in the thousands. In spite of public criticism, the FDA appears unwilling to take a more aggressive enforcement strategy. In the same statement, FDA emphasized that it prefers to encourage voluntary compliance with the law, although it left the door open to further action.

With the aim of urging that action, UAEM recently filed a formal citizen petition to the FDA, with support from Columbia Law School’s Science, Health, and Information Clinic. Our petition asks the FDA to take specific steps that will close the gap of thousands of trial results that remain unavailable to patients, clinicians, researchers, and the public.

First, we demand that the agency issue more pre-notices to ensure that trial sponsors know they have violated the law. There is evidence suggesting that increased enforcement by the FDA would lead to increased compliance. After the FDA issued its 57 preliminary notices of noncompliance, all but five of the recipients reported their missing information to ClinicalTrials.gov. If the FDA were to expand its issuance of pre-notices, there would likely be widespread improvements in compliance rates.

Second, we recommend that the FDA prioritize enforcement for products that pose greater risk to human life, are intended for use in treating life-threatening conditions, or are unapproved products of high importance without therapeutic alternatives. If the FDA has limited resources for enforcement, it makes little sense to deploy resources at random. By focusing on the trials for which access to information is most critical for public health, the FDA can best fulfill the goals of the law.

Third, we ask the FDA to create a public dashboard of pre-notices. The FDA already publicizes its notices of noncompliance, but because the agency issues so few of those notices, it remains difficult for public watchdogs like UAEM to assess the extent and success of agency enforcement. By publicizing pre-notices, the FDA can increase transparency and accountability.

By increasing and optimizing its enforcement of the law, the FDA can grow access to potentially lifesaving information for patients, physicians, and researchers. For too long, UAEM and other advocates have had to push sponsors on a case-by-case basis to report trial results. It’s time for the FDA to act.

Megan Curtin is a fourth-year student at the University of California, Berkeley. She is a member of the UAEM North American Coordinating Committee and the UAEM Transparency team. Navya Dasari is a third-year student at New York University School of Law, where she is a Furman Public Policy Scholar and Institute for International Law and Justice Scholar. She is a member of UAEM’s North American Coordinating Committee and the team lead for UAEM’s Transparency Campaign. Justin Mendoza, MPH, is the executive director of UAEM North America.