HHS on Friday finalized a rule that expands and clarifies what clinical-trial data legally needs to be made public. The goal is to help scientists, doctors and patients learn more about ongoing research and potentially ease access to experimental treatments.
A 2007 law required certain universities and manufacturers to report their results. But some exemptions and lax enforcement caused few to follow through. A Duke University study in 2015 found that only 13.4% of trials reported summary results within the mandated 12-month time-frame following the end of a trial.
The final rule clarifies that most interventional trials involving drug, biological and device products need to be registered on ClinicalTrials.gov, and summary results for the studies need to be submitted within a year of a trial's completion. Clinicaltrials.gov is run by the National Institutes of Health. The website offers clinical trial results and information on registered trials. It was established so patients could see whether they were eligible for experimental treatments.
The final rule, which will go into effect Jan. 18, 2017, also requires applicable trial results to be submitted even if the product being tested is not yet approved by the Food and Drug Administration.
The Biotechnology Innovation Organization, which represents biotech firms, and the Advanced Medical Technology Association, which represents devicemakers, both said they are still reviewing the rule and aren't ready to comment. Pharmaceutical Research and Manufacturers of America, which represents drugmakers, said the organization supports "responsible" clinical trial data sharing.
"While we are still reviewing the new rule issued today, we note that biopharmaceutical companies are committed to enhancing public health through responsible sharing of clinical trial data in a manner that is consistent with the safeguarding the privacy of patients; respecting the integrity of national regulatory systems; and maintaining incentives for investment in biomedical research," PhRMA said in a statement.
Officials at the FDA and the NIH on Friday said the rule will uphold researchers' “end of the bargain” in releasing information with the goal of expanding opportunities for patients. In a statement, FDA Commissioner Dr. Robert Califf pledged the FDA's resources in enforcing the clarified requirements.
“When people participate in clinical trials, they are volunteering to create generalizable knowledge to help others in the future, and we want their participation honored by ensuring that the existence of trials and their results are available to all patients and their healthcare providers, as well as researchers,” Califf said.
The rule requires that applicable clinical trials be registered no later than 21 days after the first human subject is enrolled. The rule also requires that information about adverse events be submitted and mandates reporting on race and ethnicity information in results.
HHS will provide a web-based checklist tool by which trial administrators can evaluate whether their trial is subject to reporting requirements, so researchers will have a harder time claiming ignorance or confusion. Following a period of little-to-no enforcement from the FDA, the rule offers potential legal consequences for non-compliance, including civil or criminal actions, fines and consequences for a non-compliant organization's grant funding.
A non-compliant clinical trial will be flagged on ClinicalTrials.gov, is subject to civil monetary penalties up to $10,000 a day, and can have its current or future federal funding pulled.
A larger body of clinical trial information could help emerging technology like IBM Watson Health, which is working with the Mayo Clinic to help match patients to trials. It also pulls from clinical trial data and studies to help physicians with treatment plans.
The rule is a step in the right direction to ensure doctors have access to accurate information about trials, whether it's good or bad, said Suzan Shinazy, founder of Medical Errors Transparency Plan, a patient advocacy group looking for ways to decrease medical error. Shinazy was the leading author of a letter sent earlier this year urging the NIH to improve clinical trial data reporting, co-signed by leaders of Consumers Union, the National Physicians Alliance and a number of state-based patient advocacy groups.
"I am happy with the NIH and HHS for giving attention to transparency and increasing the public access to current trials and final summary data of other trials," Shinazy said. "It seems they are paying attention to the people."