According to a story from Boston Health News, The US Food and Drug Administration’s Accelerated Approval program is being used to approve a steadily growing number of drugs in recent years. The program is a way to get promising drugs through the approval process faster, but could this come at the cost of less stringent regulatory standards?
About Accelerated Approval
The Accelerated Approval program is one of several that can help move a drug through the FDA approval process more quickly. However, they all vary somewhat in precisely what they do. In the case of Accelerated Approval, the program helps speed up the development of therapies that show the potential to fulfill a currently unmet medical need based on data derived from an endpoint that predicts drug efficacy.
Ostensibly, programs such as this are helpful for rare disease patients that have run out of treatment options and need to have new options urgently. For some patients, it is likely that Accelerated Approval has helped save their lives. With that said, the primary beneficiaries of these program are drug developers, not rare disease patients. After all, a therapy that gains Accelerated Approval can get on the market and start raking in money that much faster.
Unfortunately, the reduced standard of evidence and data that Accelerated Approval allows does have its consequences. Take, for example, the story of the drug Avastin. This drug is still on the market, and was first approved for the treating colon cancer and lung cancer. For a period of three years, under Accelerated Approval, Avastin was also used for breast cancer. Then, despite the protests of patients who had used it, the FDA withdrew this indication. More evidence revealed that the drug didn’t work that well against breast cancer and its side effects, which could be lethal, were not worth the risk.
Whining About Regulations Never Goes Out of Style
So there are clear risks when using this program for a drug, but many rare disease patients who have few to no options are more willing to accept said risks. In the last three years, about seven drugs per year are approved with the program. The FDA rarely receives criticism for these speed-up style programs, especially since politicians like to poke fun at its normal approval process, which is portrayed as an example of inefficient government bureaucracy.
In truth, this portrayal is up for debate. Take another drug that was approved as a treatment for Duchenne muscular dystrophy named eteplirsen. This drug received Accelerated Approval from the FDA because it increased the level of dystrophin in patients, a protein that is deficient in people affected by the disease. However, most scientists agreed that the increase was too small to do anything.
Ultimately, Accelerated Approval is limited in usefulness if its data standards are so low that drugs that produce practically no benefit are able to earn it.