The COVID-19 pandemic has reinforced the importance of the Food and Drug Administration (FDA) in instilling trust in authorized and approved therapies and vaccines. Recent, repeated missteps from AstraZeneca surrounding its characterization of COVID-19 vaccine trial results, as well as possible safety concerns, have led to a clamoring call for increased FDA scrutiny of and transparency around AstraZeneca’s clinical trial data.
President Biden has yet to nominate a new FDA commissioner to take the helm of this critical agency. Whoever steps into this role should commit to enforcing clinical trial transparency. Doing so is simple, requires no congressional action and will meaningfully protect patients.
Clinical trials are the primary means of testing whether a particular drug, vaccine or other medical intervention is safe and effective. After decades of drug companies hiding trial data that led to the approval of blockbuster drugs with significant, even deadly problems, Congress passed the Food and Drug Administration Amendments Act (FDAAA) in 2007. Under this Act, drug and device manufacturers, universities, government laboratories, and all other institutions that run clinical trials are required to disclose protocols and results from the vast majority of late-stage trials of all FDA-approved health products via the ClinicalTrials.gov website.
Clinical trial transparency allows watchdogs, providers and the public to confirm that drugs meet the FDA’s benchmarks for safety and efficacy, scrutinize data and determine optimal patient treatment and boost innovation — a benefit keenly felt during the pandemic.
Yet enforcement of FDAAA was woefully lax during both the Obama and Trump administrations. The National Institutes of Health (NIH) and FDA have joint legal authority to enforce FDAAA — including by issuing notices of noncompliance, imposing fines and withholding grant money. But NIH has all but abdicated its enforcement and monitoring power. In federal court, NIH said it has no “obligation” to issue Notices of Noncompliance unless “FDA first makes a determination of noncompliance.” While NIH should also be enforcing FDAAA, its abdication makes it doubly important that the FDA step up.
Until this April, when FDA sent its first ever Notice of Noncompliance to a clinical trial sponsor, FDA had only sent a few dozen pre-notice letters warning sponsors of possible noncompliance, initially through a pilot program from 2013-2014. It is worth noting that each of the 14 noncompliant sponsors contacted in this pilot program became fully compliant and reported all missing data after receiving the FDA’s notice. But left unchecked, hundreds more drug companies, universities, and other trial sponsors have opportunistically reported positive trial results and delayed or withheld others. Today, about a third of applicable clinical trials on ClinicalTrials.gov are missing results.
FDA can also fine sponsors who fail to report applicable clinical trials to ClinicalTrials.gov — over $12,000 per trial, per day. That adds up to a staggering $19 billion in fines FDA could have levied since FDAAA’s enactment in 2007. But FDA has never issued such fines, a decision the agency historically justified by arguing that encouraging “voluntary compliance” would be cheaper than issuing fines. While zero enforcement may be less expensive for the FDA, it costs patients much more in the form of ineffective and possibly dangerous treatments. It also violates Congress’ express enforcement mandate: FDAAA.
The FDA could close the clinical trial transparency enforcement gap at low cost simply by notifying trial sponsors that they’re noncompliant. In fact, Universities Allied for Essential Medicines (UAEM), a nonprofit organization of student volunteers, has been doing just this, stepping up where the FDA, a regulatory juggernaut with a multibillion-dollar budget, has fallen short: drawing public attention to noncompliant institutions to pressure them into compliance.
In 2019, UAEM co-authored a report identifying widespread noncompliance among top universities, sparking nationwide press coverage focused on the most egregiously noncompliant. As a result, 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. Over the same period, these institutions’ percentage of unreported clinical trials decreased from 31% to 7%.
While such efforts to publicly name noncompliant sponsors have been fairly effective, nonprofit organizations like UAEM cannot shoulder the entire enforcement burden alone. Only through timely action by the FDA, enforcing Congress’ mandate, can 100% compliance be achieved.
FDA’s breadth of resources, power and insight will allow it to publicly identify industry sponsors, who have been beyond the scope of UAEM’s reports and bring them into compliance. FDA’s ability to enforce transparency in real time will also close the lingering compliance gap among universities and research institutions.
In early March, we filed Freedom of Information Act requests with the FDA to understand how and why the agency has shirked its responsibility to enforce clinical trial transparency. Just weeks later, the agency sent its first Notice of Noncompliance to a company that had failed to submit legally required clinical trial results for its experimental kidney cancer treatments, threatening to levy fines should the company fail to comply within 30 days. We applaud the agency for taking this long overdue step and hope there will be more, unprompted by public prodding from organizations like ours.
A new FDA commissioner can commit to such a course and rack up an easy win: By ensuring public access to clinical trial data, the incoming commissioner can regain public trust in FDA-approved drugs, vaccines and health technologies, and keep Americans safer.
Joe Rabinovitsj (email@example.com) and Celine Rohr (firstname.lastname@example.org) are students at New York University School of Law. Reshma Ramachandran (email@example.com) is a physician, board member of UAEM, and fellow in the National Clinician Scholars Program at Yale University.