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In a highly unusual decision for a therapy approved by FDA, the Centers for Medicare and Medicaid Services (CMS) took action to limit prescribing and use of Biogen’s new and controversial Alzheimer’s treatment Aduhelm (aducanumab).1 The CMS National Coverage Determination (NCD) did this by applying its Coverage with Evidence Development (CED) requirement to the drug, utilizing a process that has been directed primarily in recent years to a limited number of approved medical products, mainly medical devices and diagnostics.2 If the proposal is finalized, only patients able to enroll in postapproval trials would have access to the drug.
The aim of the NCD process is to gather further evidence to demonstrate the drug’s potential for improving the health of patients suffering mild cognitive impairment due to Alzheimer’s disease. The CMS policy will authorize access to the drug only to those individuals able to enroll in a clinical trial, most taking place at academic research institutes that CMS will identify and list on its website. Under the accelerated approval granted Aduhelm by FDA’s Center for Drug Evaluation and Research (CDER) in June 2021, Biogen is required to conduct at least one Phase IV trial to demonstrate that the drug’s ability to reduce amyloid in the brain has a broader benefit for patients’ cognitive ability, and that drug’s serious side effects are limited. Biogen objected loudly to the CMS proposal, noting that it may take years to design and launch postapproval studies, and that such trials will enroll only a limited number of patients. CMS also indicates that its CED requirement will apply to additional monoclonal antibodies targeting amyloid beta in Alzheimer’s disease.
Aduhelm came to market last year amidst heightened controversy over its approval despite limited evidence of efficacy. The debate turned to outrage when Biogen set the price for the new treatment at $56,000 a year. At that level, Medicare spending on the drug was projected to far exceed any other drug if even just a small portion of some 6 million U.S. Alzheimer’s patients sought treatment. In December 2021, after a year of dismal uptake of the drug, Biogen cut its launch price in half to $28,200 a year in an effort to boost prescribing — and avoid a negative NCD from CMS. However, even that reduced cost continued to promise hefty outlays for Medicare. Many leading health centers and physicians said they still would not prescribe the drug due to its limited evidence of efficacy, known side effects and continued too-high cost. The Institute for Clinical and Economic Review (ICER) calculated that Aduhelm would be cost-effective if priced at $3000 to $8000 a year.
In making national coverage decisions, CMS is supposed to evaluate whether a medical product or process is “reasonable and necessary” and not bring cost into the equation. But the fact that a very high number of Medicare patients are potential users of the new drug made that difficult.Before Biogen cut its price, CMS had announced a whopping 15% boost in premiums for the Medicare Part B program, which administers injectables such as Aduhelm. CMS had explained that about half of the premium increase was designed to protect the program against high spending on the new Alzheimer’s therapy. Following Biogen’s cost reduction, Health & Human Services (HHS) secretary Xavier Becerra said CMS would reassess the scope of the premium hike,3 although the uncertain financial health of the Medicare Trust Fund may deter any significant readjustment in rates.
CMS emphasizes that its CED decision arises from extensive review over the last six months of all relevant published evidence and feedback from stakeholders. The agency opted for the CED process to gain further evidence on whether this new therapy provides any improvement or relief from the effects of Alzheimer’s disease. CMS will accept further public comments on this proposal for 30 days and announce a final coverage decision by April 11, 2022. Alzheimer’s disease groups have called loudly for Medicare coverage of Aduhelm as the only available treatment for the widespread debilitating condition.
It will be interesting to see how the CMS proposal affects other biopharma companies that are testing their own Alzheimer’s therapies. Eli Lilly is conducting clinical trials that directly compare its donanemab to Aduhelm, while Eisai and Roche are moving forward with additional treatments. These and other sponsors now will have to calculate the impact of bringing new drugs to market that would be subject to CED requirements.
Meanwhile, the scientific and medical community continues to criticize the Aduhelm approval. The European Medicines Agency (EMA) recently recommended against approving the drug, and objections have been raised in Canada to a pending review. CMS has navigated its process to greatly limit Aduhelm prescribing and its outlays for the drug, but the debate is far from over.