U.S. News: FDA Program Is Set for Makeover

"A federal program that has sped hundreds of new drugs to cancer patients and others is facing the biggest makeover of its three-decade history as Congress considers ways to avoid approval of drugs that don't work.

Congress is poised to amend the Food and Drug Administration's accelerated-approval program to address complaints that it has sometimes led to the use of costly, ineffective drugs. Proposals in the House and Senate would give the FDA more authority to make sure companies conduct the large follow-up studies needed to confirm that a fast-tracked drug works, and to pull from the market any therapy that doesn't.

The changes, if enacted, would be among the most significant to the accelerated-approval program since its creation in 1992, according to industry officials and people who study the FDA.

The House passed a bill on Wednesday that would make the changes. The legislation has to pass by September to avoid funding gaps for the FDA.

The activity comes after the FDA's accelerated approval of an Alzheimer's drug from Biogen Inc., Aduhelm, drew criticism from many doctors and researchers and some of the agency's own advisers, who questioned whether the therapy helped.

Citing the doubts, many hospitals refused to give Aduhelm while several health insurers, including Medicare, restricted use. Biogen said last month it would effectively stop marketing the drug.

"Patients want chances that are legitimate. They don't want to pay tens of thousands of dollars for something that isn't a very promising treatment," said Aaron Kesselheim, a professor of medicine at Harvard Medical School, who resigned from an FDA advisory committee over the agency's Aduhelm decision.

The industry's big trade groups, the Pharmaceutical Research and Manufacturers of America and the Biotechnology Innovation Organization, praised the pathway for speeding access to lifesaving medicines. PhRMA said it supports the program's "usage in its existing form."

"Accelerated approval has really proved itself as a very effective tool," said Janet Woodcock, the FDA's principal deputy commissioner. "People pick out the failures, but when we did accelerated approval, we said there would be more uncertainty. If accelerated approval had the same success rate as regular approval, we wouldn't be doing anything special at all."

Under the program, the agency clears drugs in as little as five months, or sometimes in as little as a few weeks, versus the 10 months a traditional form of approval can take.

To assess an experimental drug more quickly, the FDA doesn't require the drug to show a clinical benefit, such as preventing heart attacks or prolonging the life of a cancer patient, in a clinical trial. Such trials can take years.

Instead, the agency will look for a sign, such as reduced cholesterol or arrested tumor growth, that indicates the drug could have a clinical benefit.

The FDA now approves more drugs through the accelerated-approval program and other fast tracks than through the traditional route." [1]

1. U.S. News: FDA Program Is Set for Makeover
Whyte, Liz Essley. 
Wall Street Journal, Eastern edition; New York, N.Y. [New York, N.Y]. 11 June 2022: A.3.