The Department of Health and Human Services (HHS) issued its final rule on submitting registration and summary results to clinicaltrials.gov. In a nutshell, the new guidelines define which clinical trials must be registered and when, how results should be reported (and when) and potential penalties for non-compliance.
The rule seeks to clarify the Food and Drug Administration Amendments Act of 2007, which left significant room for interpretation. For example, the law said sponsors must report results within one year of a trial’s completion. However, if they file a certificate of delay, they can wait to file results until 30 days after FDA approval.
“Companies filed their certificate and waited for FDA approval before disclosing,” said Jennifer Miller, assistant professor in the Division of Medical Ethics and Department of Population Health at NYU School of Medicine and principal investigator for the Good Pharma Scorecard at Bioethics International. “The challenge was some sponsors thought they didn’t have to file a certificate of delay if it was the first time it was approved by the FDA. Others thought you had to file a certificate whether it was a new drug or a new use of an old drug.”
Under the regulation, sponsors must register a trial within 21 days of enrolling the first patient and must include recruitment, contact and design information, phase of the trial and eligibility criteria. They must also submit detailed results, such as patient demographics and condition, as well as adverse events. The penalties for non-compliance max out at $10,000 a day.
Enhancing access to clinical trial information means more useful data for patients and scientists. People who have exhausted their treatment options may have more access to experimental therapies. Scientists can build on trial success and avoid failures.
“More access to all trial results information means more patients, patient groups, networks like ours, consortia, academia, and industry will all be able to leverage it to advance our understanding of health and disease,” said Marni Hall, senior vice president of Research and Development, Informatics and Policy at PatientsLikeMe via email. “We work with many forward-thinking pharmaceutical companies and we’re finding they are embracing transparency and a greater ability to derive new meaning from it.”
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
However, there is still concern over how these guidelines will function in the real world. Both Hall and Miller are pleased that compliance is baked into the rule but are concerned about enforcement. In particular, does it do enough to take the ambiguity out of results reporting?
“I think the final rule was trying to address that, but we have yet to see,” said Miller. “It’s the interpretation. I hope there’s a harmonization now, and that everyone gets on the same page and we understand exactly when the results are due.”
The question may revolve around who is actually interpreting the rules: the trial sponsors or their lawyers. “Educating law firms should be part of the discussion,” said Miller.
Overall, the rule is getting good reviews for its effort to increase transparency and respond to systemic flaws.
“It reflects that we have this learning healthcare system, which is what we want,” said Miller. “We want laws that learn, that take feedback and are able to understand what is working and what is not working and be amended in order to work better.”
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