Food and Drug Administration Takes First-Ever Enforcement Action To Ensure Clinical Trial Transparency, Following NYU Technology Law & Policy Clinic’s Work with Universities Allied for Essential Medicines (UAEM)
This post is part of a series exploring the Clinic's work during the 2020-21 year
On April 28, 2021, the Food and Drug Administration (FDA) sent a Notice of Noncompliance to Acceleron Pharma, Inc. after the company failed to submit legally required clinical trial results to the public ClinicalTrials.gov database. This marks the first time the agency has ever taken such action against a trial sponsor. In July of 2020, the FDA had requested that the manufacturer promptly submit Phase 2 clinical trial results for their experimental kidney cancer treatments, which the company failed to do.
FDA and the National Institutes of Health (NIH) share responsibility for enforcing the Food and Drug Administration Amendments Act (FDAAA) of 2007, the federal statute that requires trial sponsors to submit clinical trial results and other data to the ClinicalTrials.gov website. NIH then publishes the data for the benefit of patients, doctors, scientists, and the public at large. Over a decade has passed since FDAAA’s enactment, yet hundreds of trial sponsors continue to withhold results and other important data from the public because FDA and NIH have—until now—undertaken no meaningful enforcement.
FDA’s landmark step—sending its first-ever Notice of Noncompliance—follows ongoing efforts from Universities Allied for Essential Medicines (UAEM) in partnership with the NYU Technology Law and Policy (TLP) Clinic to obtain relevant documents from the FDA and NIH through the Freedom of Information Act (FOIA). On March 10, 2021, the NYU TLP Clinic filed FOIA requests on behalf of UAEM with both FDA and NIH. UAEM’s requests seek documentation of whether and how the agencies plan to begin meaningful enforcement. These requests build on UAEM’s joint research report with TranspariMed in 2019, which reviewed the compliance of leading American universities in reporting their clinical trial results and found that a majority were noncompliant. Following publication of the 2019 report, the total number of fully compliant academic medical research institutions in the United States, among the top 40, increased from 13 in March 2019 to 17 in February 2021, as documented in UAEM’s new report, published in May 2021. Over the same period, these institutions’ percentage of unreported clinical trials decreased from 31% to 7%.
Through its FOIA requests, UAEM has demanded further information about FDA’s and NIH’s enforcement actions to date, including any pre-notice and non-compliance communications between the agencies and trial sponsors. In its ongoing FOIA investigation, UAEM has already gathered evidence that over an eight-year period, the FDA sent only a few dozen “pre-notice” letters to non-compliant trial sponsors. FDA’s Notice of Noncompliance is the first time that more meaningful enforcement action has been taken by either FDA or NIH, and it may signal that FDA is finally starting to take clinical trial transparency enforcement seriously.
UAEM and the NYU TLP Clinic will continue to work with both agencies on these FOIA requests.
In addition, Celine Rohr (’22) and Joe Rabinovitsj (’21), the TLP student attorneys on this project, together with Dr. Reshma Ramachandran, a UAEM board member, published an op-ed in The Baltimore Sun arguing that clinical trial transparency enforcement should be a priority for whoever becomes the next FDA commissioner.
TLP Clinic Deputy Director Christopher Morten supervised this project.