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Facebook’s “move fast and break things” motto doesn’t really work when it comes to healthcare (just ask Elizabeth Holmes). That’s especially true when it comes to getting new drugs to the market, which takes an average of 10 years.
However, the FDA created an accelerated approval pathway in 1992—during the height of the AIDS epidemic—to get treatments approved faster for the deadly disease caused by HIV. The pathway is intended only “to allow for earlier approval of drugs that treat serious conditions, and fill an unmet medical need,” according to the FDA.
In the decades since, drugs including Merck’s blockbuster cancer treatment Keytruda (which made Merck $20.9 billion in 2022) and Biogen’s controversial Alzheimer’s drug Aduhelm have received accelerated approval.
How the process works
Drugmakers can apply for accelerated approval while a drug is still in clinical trials. The FDA then works with the drugmaker to determine the “surrogate endpoint” for the trial. A surrogate endpoint is a measure that shows whether the drug is likely to be clinically beneficial for patients. If the drug meets that endpoint, it gets FDA approval and can go on the market.
The major difference from the standard approval process is that the drugmaker isn’t required to prove the drug is clinically beneficial, just that it’s likely to be.
After the drug is approved and on the market, the drugmaker must do another trial, called a confirmatory trial, to confirm that the drug is actually beneficial to patients. The FDA can now require drugmakers to complete the confirmatory trial “within a specified time period after the date of approval,” as of the end of 2022.
If the drug fails the confirmatory trial, the FDA can revoke its approval. By the end of 2021, a total of 32 accelerated approval drugs had been withdrawn (12% of drugs approved under the pathway), either voluntarily or by the FDA.
More accelerated approvals than ever
In the early days of accelerated approvals, only a few drugs were approved each year using the pathway. In 2012, Congress codified the pathway into law, and the number of drugs approved “boomed,” according to a 2022 NPR investigation.
“It’s hard to understand what the motivation might be for why more drugs came to that pathway, except it’s an easy way to market,” said Gregg Gonsalves, a professor of epidemiology at Yale School of Public Health who has expertise in health policy and the HIV/AIDS epidemic.
In 2021, 28% of FDA-approved drugs went through this expedited process. From the pathway’s creation through the end of 2022, a total of 290 drugs have received accelerated approval.