"The Food and Drug Administration is pressing pause on drug-company testing of experimental medicines more often, a side effect of the industry's move into promising but less-proven technologies.
The agency, which must sign off before companies can begin testing an experimental drug in people, has long used its authority to place holds on studies due to safety concerns.
As biotechs pursue more cutting-edge cell and gene therapies, the FDA has been issuing more suspensions than it had, according to a Wall Street Journal review of FDA data on clinical holds, some of which was obtained through a Freedom of Information Act request.
The agency halted clinical trials for experimental drugs an average of 664 times each year from 2017 to 2021, up from 557 each of the previous five years, according to the review of agency data. Through mid-December last year, the FDA had placed 747 of the holds.
"The barometer has changed from go as fast as you can to let's be cautious," because the technologies are newer, said Jefferies Financial Group analyst Michael Yee.
New technologies promise breakthroughs against challenging diseases such as hemophilia and muscular dystrophy, but their development presents risks. Testing of gene therapies, which replace defective genes, has been shadowed by the deaths of some young study subjects and other safety scares. The new technologies also require more time and effort from regulators to keep up with complicated advances.
Among the closely watched drugs whose studies were temporarily stopped in recent months were a sickle-cell gene therapy from Bluebird Bio Inc., after a teenage subject developed anemia. Testing of a Vertex Pharmaceuticals Inc. diabetes cell therapy was halted after the FDA said it needed more information on doses. Both trials have restarted.
At issue is the hundreds of new studies that companies sponsor each year to evaluate whether a drug candidate works safely. The FDA reviews study results when deciding whether to approve a drug.
Under federal regulations, it can halt clinical trials for reasons including to protect study volunteers from unreasonable risk of illness or injury or because a study's design is deficient.
Some holds have triggered shareholder lawsuits alleging that companies withheld information about a hold or delayed sharing information about a hold. Some investors have criticized certain companies for misrepresenting why the FDA requested a hold, after the reasons emerged in public documents.
An FDA task force on transparency called on the agency, in 2010, to disclose holds and their removals, but its recommendations haven't been implemented.
"For every trial, there are people who are watching with bated breath," said Joshua Sharfstein, who led the 2010 effort while FDA deputy commissioner and who is now a professor and vice dean at Johns Hopkins School of Public Health. "It helps patients, providers, the companies, investors, everyone to know the truth."
Both the FDA division that approves new drugs and the division that approves new biological products, such as vaccines, have been issuing more study holds in recent years, according to the Journal analysis of agency data.
The increase in holds by the Center for Biologics Evaluation and Research, the FDA division that reviews big-molecule drugs, is likely due to more new clinical trials for cell and gene therapies, an agency spokeswoman said.
She said trends in holds from the FDA's small-molecule drugs division would be harder to identify without a review of individual decisions.
"The FDA continues to work with medical product developers and researchers to facilitate the development of new potential treatments," the agency said. "This includes ensuring that study participants will not be subjected to unreasonable risks in early clinical testing and that study designs address their objectives."
The uptick in pauses might also be in part due to an increase in the number of new trials overall in recent years, analysts said.
"The big bull market led to an unprecedented expansion of the sector in terms of the number of programs and companies," said Brian Skorney, an industry analyst with Robert W. Baird & Co. Many of the companies had trouble hiring seasoned teams, he said. "So you have a dynamic of a lot of programs being managed by relatively inexperienced groups."
The FDA saw a 43% increase in the number of requests for permission to conduct clinical trials using experimental drugs from 2014 to 2021, according to the analysis of agency data. It saw a 66% increase in clinical holds during that time." [1]
1. U.S. News: Pharmaceutical Testing Faces a Pause
Whyte, Liz Essley. Wall Street Journal, Eastern edition; New York, N.Y. [New York, N.Y]. 11 Jan 2023: A.6.
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