Source/Disclosures
Disclosures: Shepard reports no relevant financial disclosures. Bruckner is the founder of TranspariMED.
November 25, 2020
4 min read
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Is the FDA’s guidance on FDAAA 810 adequate to ensure compliance?

Source/Disclosures
Disclosures: Shepard reports no relevant financial disclosures. Bruckner is the founder of TranspariMED.
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POINT

Yes.

I agree that there is a need for enforcement and compliance, which historically has not happened. However, I don’t think rigid enforcement ultimately helps patients.

Dale Shepard, MD, PhD
Dale Shepard

When we think about access to data from previous trials and how we interpret those trials to move forward, it’s about studies that have been peer-reviewed and published. The researchers who put together the trial can think about the results and present them in a thoughtful way that provides guidance on what went well and what did not. If there are trial data related to safety or efficacy, it is the responsibility of the investigators to ensure that these data are published. Merely adding data to a database doesn’t relieve the investigators of that responsibility.

When considering how we can do better with trial data to help patients, the focus shouldn’t be on putting information in databases as the endpoint. It makes more sense to work toward better publication practices, for instance, because that’s where researchers and clinicians can learn whether someone has done a similar trial and what those findings are. It’s not just looking at raw data that exist in a database. Numbers on tables just don’t help patients. Many trials already have data available on ClinicalTrials.gov. I’m interested in how those data has been used to this point to improve patient safety or to develop better therapies for our patients. This is the true measure of value.

ClinicalTrials.gov was originally designed to provide access and information to physicians and their patients about what trials are available. It is a tremendous resource for this purpose. Unfortunately, about 5% of patients participate in trials, so the more we can do to boost access, the better. Clinical trial participation leads to medical advances. As a clinician, the focus on trial awareness through ClinicalTrials.gov rather than its use as a repository of trial data is more appropriate. Neither I nor my colleagues would typically go to ClinicalTrials.gov to look for data to guide management of our patients. Unfortunately, there are many failed trials, and we’re not likely to benefit from looking through tables of data from those studies. That’s not as productive as review of publications or data presented at medical conferences.

The current system is perfectly fine. We shouldn’t do more to make it more rigorous. Without context, the data from ClinicalTrials.gov are just numbers. There is some information about the protocol, for example, but usually not enough to be as meaningful as one would find in a publication.

Although I completely understand the need for transparency for research data that may impact patient safety and lead to better outcomes, a push toward more rigid enforcement of mandatory data dumps is not likely the solution. This should not be about our right to see numbers or how much money the FDA could collect in penalties. This is about how we can provide the best care for our patients.

Dale Shepard, MD, PhD, is director of Cleveland Clinic Taussig Center Cancer Institute phase 1 and sarcoma programs. He can be reached at 9500 Euclid Ave. #R35, Cleveland, OH 44195; email: shepard@ccf.org.

COUNTER

No.

During the 1980s, American doctors inadvertently killed over 100,000 patients because harms of the drug lorcainide had gone unrecognized. The outcomes of a small academic trial could have provided an early warning sign, but they remained invisible because journal editors did not consider the trial’s results interesting enough to merit publication.

Till Bruckner, PhD
Till Bruckner

After a string of similar scandals involving numerous patient deaths, the FDA Amendments Act sailed through Congress with overwhelming bipartisan support and, in 2007, was signed into law by a Republican president.

The Act contains a commonsensical provision: companies and universities must make the outcomes of certain drug and medical device trials public within 12 months, regardless of whether they — or journal editors — like their results.

Thirteen years on, over 2,200 clinical trials subject to the law are still missing results on ClinicalTrials.gov. The outcomes of some of those trials have been published in journals, but outcomes of many others remain completely unknown, leaving vast gaps in the medical evidence base. To date, the FDA has turned a blind eye to those violations, forgoing over $12 billion in potential revenue.

In 2018, the FDA ran a public consultation on the issue. Patients, doctors and health experts unanimously urged the FDA to impose fines. Out of all respondents to the consultation, only PhRMA and the medical device lobby AdvaMed opposed sanctions on law-breaking trial sponsors.

This August, the FDA finally presented its plan for enforcing the law. In a nutshell, if the FDA happens to “discover” that a trial is missing results through its Bioresearch Monitoring Program (BIMO), it will send violators a series of letters before it — maybe, possibly, eventually — imposes a fine.

This is absurd. ClinicalTrials.gov already clearly shows which trials are missing results. The whole BIMO-and-letters charade seems to have the sole aim of perpetuating impunity for companies and universities that continue to break the law. Unsurprisingly, Karla Soares-Weiser, PhD, editor-in-chief of the Cochrane Library, called the FDA’s nonplan “disappointing.”

Americans through their representatives in Congress have made it crystal clear that they expect the results of these 2,200 clinical trials to be made public. The FDA should stop playing bureaucratic games and start fully enforcing the law — now. Trial participants, doctors and patients have waited 13 years for these potentially life-saving trial results. They should not have to wait a single day longer.

References:

FDAAA TrialsTracker. Available at: fdaaa.trialstracker.net. Accessed Nov. 4, 2020.

TranspariMED. “Patient hopes dashed as FDA fails to tackle hidden clinical trial results.” Available at: www.transparimed.org/single-post/2020/08/18/FDA-FDAAA-transparency-fines. Accessed Nov. 4, 2020.

TranspariMED. “Pharma and medical device lobbies stonewall on transparency as doctors and patients call for fines on companies hiding clinical trial results.” Available at: www.transparimed.org/single-post/2018/12/07/Pharma-and-medical-device-lobbies-stonewall-on-transparency-as-doctors-and-patients-call-for-fines-on-companies-hiding-clinical-trial-results. Accessed Nov. 4, 2020.

Till Bruckner, PhD, is the founder of TranspariMED, a campaign that works to end evidence distortion in medicine, and a research fellow at the Berlin Institute of Health. He can be reached at tillbruckner@gmail.com.