Medicare will only offer Biogen’s Alzheimer’s disease drug Aduhelm to beneficiaries enrolled in clinical trials, a decision that limits the product’s use and affects experimental neurodegenerative disease drugs that are still in development. That’s because this coverage decision extends to antibody drugs in the same class, and agency coverage decisions are also a guide for payers as they weigh whether to reimburse the Biogen drug and other Alzheimer’s drugs that may follow.
Explaining the final coverage decision announced late Thursday, officials from the Centers for Medicare and Medicaid Services said the decision reflected Aduhelm’s available data and the need to find more evidence about the drug’s benefits and risks.
“This treatment has the potential to offer hope; however, there is currently insufficient evidence of improved health outcomes to be reasonable and necessary for those on Medicare, a key CMS in determining state coverage considerations,” said CMS chief medical officer and director of the Lee Fleisher Center for Clinical Standards and Quality in the agency’s announcement.
Aduhelm’s final CMS decision concludes a coverage determination process at FDA accelerated approval of Biogen drug last June. Accelerated Approval is a pathway designed to bring drugs to market faster that address a major unmet medical need. This pathway requires less evidence than standard drug reviews. The accelerated approval decision was based on a surrogate endpoint, which suggests that the drug may be effective even if patient benefit is unclear.
For Aduhelm, the surrogate endpoint was a reduction in beta amyloid. Aduhelm is an antibody drug designed to destroy amyloid plaques. While amyloid accumulation is a hallmark of Alzheimer’s disease, it remains unclear whether reducing these plaques will help patients, and Biogen’s own clinical data provide a mixed picture.Independent Analysis of 2019 Phase III Interim Data Conclusions that clinical trials are unlikely to be successfulBut Biogen conducted a new analysis that it said was successful in one of two pivotal studies. On this basis, the company seeks FDA approval.
The final CMS determination covers Aduhelm and other amyloid-targeting antibody drugs, so-called evidence development coverage. This CMS provision allows Medicare to cover drugs in clinical trials designed to generate additional data that could support broader coverage. The coverage of evidence development has been applied before; examples include stem cell transplantation for blood disorders and some medical devices such as cochlear implants.
this CMS decision It largely aligns with the proposal the agency released in January. then, The agency says it will only offer Aduhelm to patients in CMS-approved randomized controlled clinical trials. The final decision slightly expanded the trial requirements to include comparative studies with a range of study designs. These data can be collected through routine clinical practice, also known as registries.
CMS’ decision rescinds some of the requests the agency originally made in January. For example, the criteria for excluding certain patient groups, such as those with Down syndrome, are no longer included in the final coverage determination. CMS said it removed the exclusion criteria based on public comment. The agency also removed a rule that required approved clinical trials to be conducted in hospital outpatient facilities. The proposed requirements are based on security considerations. Serious side effects of Aduhelm include brain swelling and bleeding. The removal of this requirement, also based on public comment, will allow clinical studies to provide data on patient outcomes from a broader community setting, CMS said.
The decision to limit Aduhelm’s coverage to clinical trials is the latest in a series of setbacks for the drug and Cambridge, Mass.-based Biogen. Market acceptance for Aduhelm, initially priced at $56,000 a year, has been slow. Despite the Alzheimer’s epidemic and patients’ need for new treatments, the drug has fallen far short of blockbuster expectations, with sales of just $3 million in 2021, according to Biogen’s financial statements. Biogen slashed the price of the drug in half on Jan. 1the company said the decision will make the drug more accessible to more people.
in a statement Posted after the CMS announcement, Biogen said the coverage determination effectively denied Aduhelm to all Medicare beneficiaries. The company urged the agency to reconsider the decision after more clinical data on such drugs became available. Eisai, Biogen’s partner in developing Alzheimer’s drugs, is following Aduhelm’s accelerated approval pathway for another antibody drug, lecanemab. Phase 3 data for the drug is expected to be released this fall.
CMS refutes suggestion that its coverage decisions hinder the development of innovative Alzheimer’s treatments. The agency said it decided to foster innovation by clarifying the criteria for Medicare coverage. Under the law, CMS covers medical products and services that are deemed “reasonable and necessary.” CMS said its final decision outlines what evidence such Alzheimer’s drugs need to meet this criterion.
“Medical innovation must include demonstrating evidence of health outcomes to patients, taking into account potential harms,” the agency said in a statement. Fact Sheet and more details about the decision.
Photo: Scott Eisen/Bloomberg via Getty Images
