In an unprecedented enforcement action, the Food and Drug Administration (FDA) yesterday informed a clinical trial sponsor that it had violated the law requiring that results from human studies of medical treatments and devices be posted to the federal repository ClinicalTrials.gov. The action was leveled against the Cambridge, Massachusetts–based drug company Acceleron Pharma for its failure to provide data from a completed trial of its experimental drug to treat kidney cancer.
Such data must generally be deposited within 1 year of a trial’s end, but Acceleron’s results are nearly 3 years past due. Acting FDA Commissioner Janet Woodcock said in a written statement about the 27 April notification that the agency takes its enforcement role “very seriously … for the benefit of clinical trial participants and public health.”
Acceleron ceased the drug’s development in 2017, following the trial’s disappointing results, according to a company spokesperson. The results were published in a scientific journal in 2019 but not everyone can access such publications so the law mandates the basic results be added to the public website. FDA gave the company 30 more days to post the results or face financial penalties; the spokesperson says it will comply.
Since 2007, the U.S. government has required companies, universities, federal agencies, and nonprofits that sponsor clinical trials to report their results, whether positive, negative, or inconclusive, so that doctors, patients, and researchers can learn about the safety and efficacy of new drugs or devices. Congress created the reporting law after pharma companies suppressed data revealing lucrative drugs to be unsafe or ineffective.
In 2017, FDA and the National Institutes of Health, which oversees the law for the researchers it funds, released a “final rule” to clarify the requirements and the penalties for ignoring them. When NIH Director Francis Collins announced that rule, he warned trial sponsors that if they violate the reporting law they would be listed on a ClinicalTrials.gov “wall of shame”—Acceleron is now its first entry. Since the rule took full effect in January 2018, some large universities and companies have improved their reporting performance, but for thousands of trials, sponsors still ignore the law, investigations in Science and elsewhere have found.
FDA can collect more than $10,000 per day in penalties when companies break the law; the total penalties could have amounted to more than $19 billion since 2018, according to a tracking site at the University of Oxford. FDA has yet to collect a single dollar. NIH can also withhold violators’ grant funds but has never done so. Instead, both agencies have encouraged voluntary compliance—until now.
As vice president, Joe Biden pledged in 2016 to enforce the clinical trials law after learning it was being ignored. Wednesday’s action is the first sign that his administration would reverse years of passive acceptance that the law is widely ignored.
It remains to be seen whether FDA will crack down on the many other trials with overdue results. The agency did not immediately respond to a request for information on its future enforcement plans.
Deborah Zarin, a physician at the Multi-Regional Clinical Trials Center of Brigham and Women’s Hospital and Harvard University who headed ClinicalTrials.gov between 2005 and 2018, applauds FDA’s action. But she says it was unclear why the agency chose Acceleron out of thousands of other violators. “It will be important for FDA and NIH to follow this up with a program of systematic monitoring and enforcement,” Zarin says. “Human nature is such that without a clear risk of sanctions for noncompliance, institutions and investigators are simply not going to report results when it is inconvenient or seems contrary to their interests.”