Fast-track drug approval process marked for limited changes
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A week after the maker of a controversial Alzheimer's drug announced it would largely stop marketing it, Congress is readying legislation that tinkers with the pathway used by the FDA to approve the drug, but avoids making large-scale changes.
Why it matters: Aduhelm's approval created heightened scrutiny around whether the FDA's "accelerated approval" process is being used appropriately. But as the drug fades largely into the background, so are the calls for stringent reforms.
Be smart: Accelerated approval lets drugmakers sell their products based on preliminary evidence and finish clinical trials once they're already on the market.
- 14 of the 50 new drugs the FDA approved last year went through the accelerated pathway, including Takeda Pharmaceuticals' Livtencity for post-transplant infections.
- But the process has drawn concern because of the way it could put big-ticket drugs in the public's hands that may wind up being ineffective or unsafe.
- The Aduhelm approval came amid doubts that the criteria used for its approval proved the drug was effective, and the FDA's own advisory board had recommended against its approval. Manufacturer Biogen said last week it's cutting back on marketing the drug as part of efforts to refocus its business.
Driving the news: House Energy and Commerce leaders are proposing fixes as part of a broader legislative package to renew FDA programs that will be marked up today.
- They would strengthen the FDA's power to ensure there are clear conditions for completing confirmatory trials and gives regulators the authority to require studies to be underway before approval is granted.
- The bipartisan legislation also clarifies conditions under which the FDA could withdraw an accelerated approval and adds labeling requirements for drugs that go through the process.
Between the lines: Democrats and Republicans were divided over how far to go, with Democrats in favor of putting more of the onus on manufacturers to justify keeping the approvals and Republicans wanting to expand the use of the pathway, per Cowen analysts Eric Assaraf and Rick Weissenstein.
- The House package focuses on areas where there's consensus and approaches that have been endorsed by the Biden administration, the analysts said.
- The big drug industry lobby PhRMA says the process should be left as is, adding just six of 165 products that received accelerated approval in the last decade have been withdrawn, and that another nine sponsors pulled their applications.
The big picture: While experts and policymakers say the pathway can deliver breakthrough therapies faster, there are lingering questions about whether the program could be misused.
- As with drug pricing, lawmakers appear set on making changes the pharmaceutical industry can tolerate.
Editor's note: This story has been corrected to reflect that Regeneron's Evkeeza was granted FDA's priority review but not accelerated approval.
