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NIH warns drug and device companies to post missing trial data - STAT

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WASHINGTON — Hundreds of drug companies, medical device manufacturers, and universities owe the public a decade’s worth of missing data from clinical trials, federal officials warned last week.

New rules issued last week in the wake of a federal court ruling in February instructed clinical trial sponsors to submit missing data for trials conducted between 2007 and 2017 “as soon as possible.” For years, many trials conducted during that span have largely been exempted from reporting their data to ClinicalTrials.gov, a public database, meaning a decade of data about approved drugs and medical devices has never been made public.

The court’s ruling, and the federal government’s decision not to appeal it and instead to urge trial sponsors to submit the missing information, represent a major win for transparency advocates, who for years have fought to recover the decadelong gap in publicly available clinical trial data. 

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“This is a case about the public’s right of access to clinical trial results,” said Christopher Morten, a supervising attorney at New York University’s Technology Law and Policy Clinic, who represented the plaintiffs. “Now, with the court’s decision, those trial results are very clearly required, and the trial sponsors have a legal obligation to report them.” 

It’s unclear, however, how many trial sponsors the federal government warned about noncompliance, and whether or when they would follow the new rules. In a July 30 letter that the Department of Justice shared with Morten, the National Institutes of Health warned trial sponsors to upload data “as soon as possible,” but it doesn’t specify a timeline.   

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While Morten said he was “cautiously optimistic” that trial sponsors would comply with the new rules, he stressed that the Food and Drug Administration and the NIH have never levied a monetary penalty or withheld grant money from noncompliant trial sponsors — options they could exercise if they chose to. 

The court ruling, and the resulting change in federal policy, come after years of reporting that has detailed how federal research agencies routinely fail to enforce their own rules regarding clinical trial transparency — which advocates say is critical for the public’s understanding of a given medicines’s safety and efficacy. 

The initial lawsuit was brought by Peter Lurie, formerly an associate FDA commissioner who worked on transparency issues, and Charles Seife, a New York University journalism professor who has written for Science and other outlets.

In legal documents, Seife singled out specific drugs for which trial sponsors have failed to submit data, hampering his own investigations into their safety and effectiveness. In one case, he detailed a clinical trial for omadacycline, a broad-spectrum antibiotic the FDA approved as the brand drug Nuzyra in 2018. The trial, scheduled for completion in 2010, at one point claimed to have enrolled 790 patients but only ever enrolled 143, Seife wrote, and was never completed. Yet because of the trial’s scheduled completion date in 2010, he wrote, the drug’s manufacturer, Paratek, was not subject to disclosure requirements, potentially denying the public vital knowledge about whether the trial supported the FDA’s decision to approve the antibiotic. 

“This Basic Results data bears directly upon the safety and efficacy of omadacycline, a drug that has been approved by the FDA and is currently being used by patients and doctors,” he wrote. “Because this Basic Results data is missing from ClinicalTrials.gov, I cannot complete my investigation of … the safety and efficacy of omadacycline.”

A STAT investigation in 2015 revealed widespread inconsistencies in how top research universities report clinical trial data. The NIH said then it was waiting for forthcoming  regulations that would give it “a firmer basis for taking enforcement actions” against institutions that fail to report. Those regulations were eventually published in 2017. 

Subsequent reporting in Science magazine has revealed that despite the transparency requirements, the FDA has still never imposed a fine on a clinical trial’s sponsor, and the NIH has never publicly named or withheld grant funding.

The FDA and NIH — and their parent agency, the Department of Health and Human Services — did not respond to STAT’s request for comment. 

Despite the court ruling and the recent NIH warning to trail sponsors, Seife said he expected little to change unless the agencies began enforcing rules they have long ignored.

Do I think I’ll be able to get my hands on those data?” he wrote in an email. No, probably not. The FDA hasn’t shown any interest in enforcing compliance with trial registry reporting requirements; I doubt that the court order will suddenly change their attitude. And without some penalty for noncompliance, I don’t think that the companies in question will decide to cough up the old data just because it’s the right thing to do.

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