Expedited drug approvals have slowed this year as the Food and Drug Administration’s controversial accelerated path comes under renewed scrutiny from Congress, government watchdogs and some regulators. leader of the agency itself.
With less than a month left in the year, the FDA’s drug center has granted 10 expedited approvals — fewer than in each of the past five years, when program usage hit an all-time high.
The program allows drugs to be marketed in the United States based on promising early results, before they are proven to benefit patients.
Scholars have long complained that this practice has led to an overabundance of expensive, unproven drugs, especially for cancer. But last year’s accelerated approval of a controversial Alzheimer’s drug sparked a new wave of criticism, including investigations into the FDA’s decision by federal inspectors and Congress. .
A recent FDA hearing that revoked the approval of an unproven preterm birth drug further underscored the program’s shortcomings. The FDA has been trying to remove Makena from the market for more than two years, because a follow-up study found it to be of no benefit to infants or mothers. The manufacturer refused to cooperate.
Amid the latest controversy, FDA leaders have taken unprecedented steps to show a tougher stance toward expedited approvals: pushing drugmakers to scrap the growing list unproven uses of the drug and asked Congress to grant new powers to accelerate elimination as companies falter.
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Dr. Reshma Ramachandran, a Yale University researcher who advocates for congressional reforms, believes that scrutiny of the recent accelerated approval process “certainly gave them pause and follow suit. another way.” However, Ramachandran and other critics say it’s too early to tell whether the FDA’s recent actions reflect a long-term policy change.
An FDA spokesman said the agency’s stance on expedited approval “remains consistent and the agency is committed to ensuring program integrity.” She added that the FDA is using “everyone of our authorities” to ensure drug manufacturers quickly conduct validation studies after approval.
Launched in 1992, the accelerated approval program is credited with accelerating the availability of early breakthroughs for HIV and cancer. About 300 drugs have been approved that way, nearly half of which have been approved in the past five years.
But researchers and government watchdogs have noted problems with FDA oversight, including delays in rapidly phasing out drugs whose studies confirm failure or lack thereof. omission. Federal inspectors recently found that 40% of expedited approvals have incomplete validation studies.
The picture is starting to change. In less than two years, the FDA has successfully called for drugmakers to scrap more than 20 approvals for accelerated drugs — mainly cancer therapies — that have shown no benefit. That’s more than half of the withdrawals in the program’s history.
The FDA’s director of oncology, Dr. Rick Pazdur, has called the effort to regain approval his “jihad”.
Several drug manufacturers have recently refused FDA requests to voluntarily withdraw their drugs. That raises the specter of lengthy and more difficult public hearings to force dismissal, a process Pazdur calls a “nightmare.”
The ability to require drug manufacturers to conduct research before approval is part of legislation that congressional lawmakers hope to attach to a major spending bill by the end of the year. If passed, it would be the largest overhaul in the 30-year history of the expedited approval process.
“We need more opportunity to start validation trials before rapid approval,” FDA Commissioner Robert Califf said at a recent cancer conference. “Once approved, it is very difficult to hold back marketers.”
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FDA regulators have tested this approach. Increasingly, the agency is telling drug manufacturers that it must conduct validation studies before approving it.
Mike Sherman, CEO of startup Chimerix, told investors on a recent call discussing the company’s plans for the upcoming cancer: “We know the FDA has raised the bar. high standards for quick approval and that’s why we’re looking for more clarity on their stance.” medicine.
A higher standard could cause trouble for smaller drugmakers, who often rely on expedited approvals to gain a foothold in the market before raising more capital.
To be sure, FDA approval trends are influenced by a variety of forces. The agency’s total of new drugs has fallen markedly this year at about 30, compared with 50 last year. And even the recent peak in accelerator usage — 45 approvals in 2020 — reflects some trends.
Drugmakers are actively pursuing a new type of “immunotherapy” that helps the body recognize and attack cancer. The FDA has granted more than 80 uses for these drugs since 2015, pushing the number of approvals up rapidly every year.
In addition, disruptions due to the COVID-19 pandemic have forced some drugmakers to cut their research plans short, requiring FDA approval for fast approval based on partially completed studies, rather than results. full results.
“There has been a lot of reinterpretation of the FDA rules to ensure that those companies can proceed,” said Sam Kay, a consultant at Clarivate, which advises pharmaceutical companies. otherwise they will lose billions of dollars.”
It’s a reminder of how flexible the FDA is.
Ramachandran noted that many of the reforms awaiting Congressional approval “are things the FDA can do right now.”
“I think having Congress do that will give them the cover and the blessing to get on with what they want to do,” she said.
