Is the bar to FDA Approval too low? These experts think so

For 25 years, the United States Food and Drug Administration (FDA) has offered drugs that treat serious diseases and fill unmet medical needs an abbreviated route to market. The program has received praise from drug developers and patients, who, if anything, would like the FDA to move faster still. But a paper by regulatory experts has now questioned the merits of the program.

Is the bar to FDA Approval too low? These experts think so

For 25 years, the United States Food and Drug Administration (FDA) has offered drugs that treat serious diseases and fill unmet medical needs an abbreviated route to market. The program has received praise from drug developers and patients, who, if anything, would like the FDA to move faster still. But a paper by regulatory experts has now questioned the merits of the program.

Writing in the Journal of the American Medical Association, the academics focus in on the 22 drugs that won accelerated approvals in 24 indications from 2009 to 2013. Most of these drugs came to market on the strength of data from clinical trials that lacked control arms. This cut the time it took for the drugs to reach the full patient population but left questions about their safety and efficacy unanswered at the time of approval.
The FDA asked the companies behind the drugs to run 38 postapproval studies to fill these gaps. This, in the eyes of the paper’s authors, is where the accelerated approval process is going awry.

As of April 2017, one-third of the postapproval programs were yet to deliver data confirming the clinical benefits of the tested drugs, despite having five or more years to do so. These products are on the market on the basis of the slender, provisional data that won them accelerated approvals.
To the authors of the study, this is a worry. However, it is unlikely important groups including drug developers and patients would welcome a more cautious approach by the FDA. The row about Sarepta Therapeutics’ Duchenne muscular dystrophy drug and proliferation of right-to-try laws show many patients will accept risks to get new treatments.

These trends, rather than academic criticism, appear more likely to influence the FDA. Under the leadership of Scott Gottlieb and ultimate oversight of President Donald Trump, the FDA is set to try to get drugs to market faster, not slow the process down and increase data requirements.

Quality is not an act. It is a habit
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