An expedited process for new drugs to be speedily approved by the Food and Drug Administration and introduced to the market has come under scrutiny this week for its potential to put the public at risk.
Two studies published this week in the medical journal The BMJ concluded that the FDA's expedited development and review programs — which were originally put in place in the wake of the HIV crisis to get medicines to patients quickly — are potentially being applied too often with insufficient efficacy evidence, allowing new pharmaceuticals to win approval before they have been fully vetted for safety and effectiveness.
The expedited pathways can shorten a drug's approval time from multiple years of clinical trials to as short as six months, according to the new analysis. One study focused on the overuse of the expedited options for innovative drugs, while the other focused on problems with drugs being quickly approved for "supplemental" uses not included in their original approvals.
Aaron Kesselheim of Harvard Medical School and Brigham and Women's Hospital, who led both studies, found that the use of these processes has increased over the past decade while the quality of the material supporting drug companies' medical claims has grown weaker.
"We undertook this research because we noticed that the number of expedited approvals in recent years had risen and we wanted to see whether it was an anomaly or a trend," Kesselheim explained. "We found a very clear trend."
The most problematic aspect, he added, was that speedy options were originally meant for innovative, first-in-class products, but are now being used mainly for drugs that are "less likely to be innovative." The study found that of 774 drugs approved between 1999 and 2014, only a third of them were considered first-in-class or groundbreaking.
"I do worry that these expedited programs, some of which were created in response to the AIDS crisis of the 1980s, are now being applied in cases that they don't belong, which wastes resources and potentially exposes patients to drugs that will be later found to be unsafe or not work as well as they were predicted to," Kesselheim said.
The FDA told VICE News that the four expedited programs — Fast Track, Accelerated Approval, Breakthrough Therapy, and Priority Review — were all put in place to "speed the availability of new drugs to treat patients with serious conditions," and insisted that the processes did not change the standards for approval.
"All drugs approved through expedited programs must meet the same statutory standards for safety and effectiveness and have a positive risk benefit profile as drugs approved in a traditional review program," the agency said in a statement.
But the research led by Kesselheim has raised questions among critics of the FDA who already suspect that the FDA is eager to please pharmaceutical companies.
Michael Carome, a physician and director of the consumer advocacy organization Public Citizen's Health Research Group, traces the FDA's increasing willingness to approve drugs to a series of congressional reauthorizations of the Prescription Drug User Fee Act, a bill that requires reauthorization every five years and mandates that pharmaceutical companies pay fees as part of the FDA's approval process.
The negotiations behind the reauthorizations allow the companies to ask for changes to policies and processes, he said, including expedited drug approval. Carome thinks that the FDA has become so reliant on pharmaceutical fees that it typically accepts these adjustments. The companies pour resources into lobbying Congress, and the bills are typically reauthorized by a bipartisan majority.
"In our view, too often decisions have been made to have a product approved in the interest of the industry rather than public or patient health," Carome remarked.
He described the FDA as the "most important public health entity in the United States," noting that it regulates about a third of the economy, including medical devices, pharmaceuticals, supplements, food, and tobacco products.
"It's important to have a regulator that's independent and acting in the interest of consumer health, and doesn't let itself be swayed by industry interests, which is too often the case," Carome said.
Adriane Fugh-Berman, a pharmacology professor at Georgetown University and director of PharmedOut, a pharmaceutical watchdog group, said that the studies were important because they lend credence to her belief that "the FDA has abdicated its responsibility to protect the public from ineffective or unsafe drugs."
She added that Kesselheim's findings that less than a third of supplemental approvals were supported by clinical studies was "appalling."
"It bears noting that pharmaceutical companies have been abusing expedited drug approval programs to get mediocre drugs approved for some time," she said. "The FDA has become a sieve for drug approval."
Follow Colleen Curry on Twitter: @colleencurry