The U.S. Food and Drug Administration (FDA) is sometimes accused of being sluggish and slow to approved promising new medications. It’s often guilty as charged but the FDA is doing better in some respects. The federal drug agency has an accelerated approval program meant to allow patients early access to promising drugs before they would otherwise be approved. The program was created more than 30 years ago to grant patients with AIDS early access to drugs for HIV. According to a recent analysis of the program:
It allows the FDA to grant early approval to drugs that show promising initial results for treating debilitating or fatal diseases. In exchange, drug companies are expected to do rigorous testing and produce better evidence before gaining full approval.
Nowadays more than four-in-five drugs granted accelerated approval are oncology drugs (85%). How well is it working? That depends. The program to grant patients early access to promising new drugs does just that: it is used a lot. Since it began in 1992, 290 drugs have been approved through the accelerated program. That works out to more than nine a year, on average, or nearly one a month. By any measure that accelerated access to new drugs.
A new study aimed to identify the benefits derived from early access. In other words, did the drugs that Americans were granted early access to go on to become lifesavers? In terms of cancer drugs critics say the data is mixed at best.
In a new study, researchers found that most cancer drugs granted accelerated approval do not demonstrate such benefits within five years.
“Five years after the initial accelerated approval, you should have a definitive answer,” said Dr. Ezekiel Emanuel, a cancer specialist and bioethicist at the University of Pennsylvania who was not involved in the research. “Thousands of people are getting those drugs. That seems a mistake if we don’t know whether they work or not.”
Skeptics point out there is a trade-off. Patients get access to drugs earlier, but some of those drugs don’t work as well as hoped. The FDA can remove the ones that fail to show the benefits that the agency used to grant accelerated access, but the FDA sometimes decides minor endpoint improvements are good enough.
The new study found that between 2013 and 2017, there were 46 cancer drugs granted accelerated approval. Of those, 63% were converted to regular approval even though only 43% demonstrated a clinical benefit in confirmatory trials.
A physician from MD Anderson Cancer Center added some context to the study findings:
Drugs that got accelerated approval may be the only option for patients with rare or advanced cancers, said Dr. Jennifer Litton of MD Anderson Cancer Center in Houston, who was not involved in the study.
It’s important for doctors to carefully explain the evidence, Litton said.
“It might be shrinking of tumor. It might be how long the tumor stays stable,” Litton said. “You can provide the data you have, but you shouldn’t overpromise.”
The FDA recently received more power to withdraw accelerated approval drugs that fail to meet benchmarks. The FDA can also set conditions to accelerated approval, like requiring confirmatory trials be underway before granting conditional approval. These all sound like steps in the right direction. The agency should be free to work with drug makers to get drugs on the market faster but have the authority (and the will) to withdraw the ones that fail to show benefit or require more testing when evidence seems lacking after a period of time. Overall, the program appears to be working.