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Biogen Alzheimer’s Drug Blow Called Distinct From Other Research

April 26, 2022, 9:31 PM

Medicare’s decision to limit coverage for Biogen‘s Alzheimer’s drug won’t necessarily hinder the development of several similar treatments currently in clinical trials, an FDA official said Tuesday.

Patrizia Cavazzoni, director of the FDA’s Center for Drug Evaluation and Research, told senators that the current pipeline for Alzheimer’s drug research is “very robust,” with “a lot of drugs in the pipeline for Alzheimer’s disease, including drugs in the same class” as Biogen’s Aduhelm.

The remarks, in response to questions at a Senate committee hearing on must-pass user fee legislation, are some of the latest efforts by the FDA to quell concerns among lawmakers and manufacturers that Medicare’s recent decision to limit coverage of Alzheimer’s treatments will ward off investment in new drugs for unmet medical needs. Those drugmakers often seek FDA approval on an accelerated basis.

“It would be speculation” to predict “whether the decisions might impact future development,” but “what I can tell you is that the pipeline is very, very healthy,” Cavazzoni said during the Senate Health, Education, Labor, and Pensions Committee hearing. The drug center director was joined by Peter Marks, director of the Center for Biologics Evaluation and Research, as well as Jeffrey Shuren, the head of the FDA’s Center for Devices and Radiological Health.

The Centers for Medicare & Medicaid Services restricted coverage for Biogen’s Aduhelm and other treatments like it to patients in CMS-approved clinical trials, effectively preventing most Alzheimer’s patients from receiving it. Biogen, other manufacturers, and Alzheimer’s patient groups pushed back on the decision, arguing that it would unfairly restrict access for patients who have a difficult time getting into clinical trials, including rural and low-income groups.

CMS leaders said it wasn’t reasonable and necessary to cover Aduhelm and other drugs that target amyloid plaques—sheets of abnormal protein in Alzheimer’s patients’ brains—because there wasn’t enough evidence to show that lowered amyloid levels are an adequate predictor of a slowed progression of Alzheimer’s disease. But the agency in its decision said it would provide a pathway to expanded coverage for FDA-approved treatments or those yet to be approved that have shown a clinical benefit.

Sen. Susan Collins (R-Maine) said at the hearing that the CMS was acting outside its authority by making judgments on a product’s safety. Collins asked Cavazzoni whether “CMS is getting outside of its lane and imposing additional restrictions will affect the number of pipeline products that may be reviewed through this pathway and may be of benefit to those suffering from Alzheimer’s.”

Cavazzoni defended the pace of ongoing developments in the Alzheimer’s treatment space and said the FDA is “very encouraged by the advances that are taking place in the field.”

In addition to Biogen, other pharmaceutical companies like Eli Lilly and Roche are carrying out clinical trials on their own amyloid-targeting treatments. Manufacturers can challenge the CMS coverage determination only by supporting an administrative appeal or lawsuit filed by individual Medicare beneficiaries who argue they have unreasonably been denied access to an Alzheimer’s treatment.

Lee Fleisher, chief medical officer for the CMS, previously said the Aduhelm decision shouldn’t be viewed as setting a new standard for how Medicare will cover future therapies that receive FDA accelerated approval. He argued that this is a unique situation in which the agency “made a decision that weighed the potential for patient benefit against the significance of serious unknown factors that could lead to harm.”

Innovations to Come

CMS’s Aduhelm decision also provides the possibility for expanded coverage should treatments coming down the pipeline show a more clear promise of clinical benefit for Alzheimer’s patients.

Mark McClellan, who has previously served as the FDA commissioner and CMS administrator, said in a webinar hosted by The Hill Tuesday that “there are three other drugs that are in pivotal FDA clinical trials now that are expected to have results in the coming weeks and months” that could eventually demonstrate “benefit in terms of slowing the progression of loss of cognitive function in Alzheimer’s disease.”

“The first HIV drugs or the first cancer drugs are not nearly as good as the second, third or fourth generation,” he said. “Often it takes more than one treatment or a combination of drugs and other interventions to really get a disease under control.”

User Fees

The FDA and lawmakers defend the accelerated approval pathway and also seek changes that could improve surrogate endpoints used to predict clinical benefit and push companies to complete required post-approval studies.

In its proposed user fee agreement with the prescription drug industry, the FDA has committed to devoting a portion of drug industry funding over the next five years to early consultation between review teams and sponsors to develop new surrogate endpoints that can better predict a treatment’s clinical benefit. The FDA also committed to creating a pilot program to look at novel endpoints for rare diseases.

Separate bills that could be added as policy riders to the user fee legislation—which must be passed before the current one expires Sept. 30—would expand the FDA’s authority to push companies with products that received accelerated approvals to complete studies confirming a clinical benefit. Nearly half of the accelerated approved drugs currently on the market haven’t had a confirmatory trial done.

House Energy and Commerce Committee Chairman Frank Pallone Jr. (D-N.J.) introduced a bill (H.R. 6963) in March that would require drugmakers and the FDA to agree on how to conduct postmarket studies before the agency could grant an accelerated approval. A second bill (H.R. 6996), by committee ranking member Rep. Cathy McMorris Rodgers (R-Wash.) would make the FDA responsible for seeking changes to a company’s postmarket study.

To contact the reporter on this story: Celine Castronuovo at ccastronuovo@bloombergindustry.com

To contact the editors responsible for this story: Fawn Johnson at fjohnson@bloombergindustry.com; Karl Hardy at khardy@bloomberglaw.com