Final CMS Aducanumab Decision Limits Coverage to Clinical Trials

Kerry Dooley Young

April 07, 2022

Updated April 8, 2022 // Editor's note: This story has been revised with additional comments from the Alzheimer's Association

In a final decision on the funding of the controversial Alzheimer's drug aducanumab (Aduhelm), federal officials largely stuck to their draft plan to limit Medicare payment to patients enrolled in clinical trials.

On Thursday, the Centers for Medicare & Medicaid Services (CMS) released its national coverage determination (NCD) for funding aducanumab and other anticipated similar drugs that target beta-amyloid in the brain, which is considered a hallmark of Alzheimer's disease (AD).

In a press release, the CMS said its decision is a two-part policy pegged to the strength of currently available evidence for anti-amyloid agents.

As reported by Medscape Medical News, last year the FDA approved aducanumab using the accelerated approval pathway, a decision that sparked a great deal of controversy and led to the resignation of three members of the FDA's advisory panel on the drug.

In its final decision, CMS set far less restrictive conditions for potential coverage of future cases where there’s enough evidence for the FDA to grant traditional, or full, approval to anti-amyloid drugs. In such cases, coverage would be pegged to participation in CMS-approved studies, such as a data collection through routine clinical practice or registries.

"Any new drugs in this class that receive FDA traditional approval may be available in additional care settings that people with Medicare can use, such as an outpatient department or an infusion center," CMS said.

In addition, the agency also offered more flexibility for coverage of aducanumab and similar amyloid-targeting antibody drugs under accelerated approvals, for which CMS will require participation in clinical trials.

Commenting on the decision, Howard Fillit, MD, co-founder and chief science officer of the Alzheimer’s Drug Discovery Foundation (ADDF), applauded this stipulation.

"With this decision, Medicare will now provide coverage for patients enrolled in any FDA- or NIH-approved trial. This is an important change that will expand access for more patients to access the drug in additional settings," said Fillit.

"Although this is an improvement, it would have been preferable for CMS to include patient registries, which collect real-world evidence that more closely reflects the actual population living with Alzheimer's, compared to the limited populations that enroll in clinical trials. This approach would also have extended coverage of Aduhelm to more patients currently living with Alzheimer's disease," he added.

He also welcomed the flexibility CMS outlined for future coverage of drugs with more compelling data.

"If you've seen one anti-amyloid antibody, then you've seen one anti-amyloid antibody. Drugs in the same class are not necessarily equal and each should be evaluated and made available to patients on its own merits," Fillit said.

In a statement, Biogen disagreed with CMS' limit on aducanumab's coverage, and protested the two-tiered approach created for amyloid-targeting antibody drugs.

"These coverage restrictions, including the distinction between accelerated approval and traditional approval, have never been applied to FDA-approved medicines for other disease areas," Biogen said.

Biogen and other critics of the CMS decision contend it denies patients access to a medication that might slow AD-related cognitive decline, while supporters question whether using beta-amyloid clearance as an endpoint translates to clinical benefit.

In a statement from the Alzheimer's Association, the organization said it is "deeply disappointed" that the CMS essentially ignored the needs of patients with AD — and that the decision to tightly restrict coverage of FDA-approved treatments in this drug class under the accelerated approval pathway "is wrong."

"CMS has created unnecessary barriers" for these individuals, Harry Johns, chief executive officer of the Alzheimer's Association, said in the press release.

"People living with MCI, Alzheimer's disease, and other dementia deserve the same access to therapies given to those living with other conditions like cancer, heart disease, and HIV/AIDS," added Alzheimer's Association president Joanne Pike, DrPH.

"Drugs that treat people in the early stages of Alzheimer's could mean more time for individuals to actively participate in daily life, have sustained independence, and hold on to memories longer," she said.

The organization added it is also concerned about "the immediate impact" the CMS' decision may have on research.

"Years of increased research funding has led to more progress and innovation than ever before, but today's decision may halt this progress as developers question if there is a pathway forward to coverage," said Maria C. Carrillo, PhD, chief science officer for the Alzheimer's Association.

CMS officials said the agency tried to make the process leading to today's decision transparent and evidence-based. It noted it gathered more than 10,000 stakeholder comments and considered more than 250 peer-reviewed documents. 

"This final National Coverage Determination reflects CMS' commitment to provide the American public with a transparent, trusted, evidence-based decision — without regard to cost — that is made only after a thorough analysis of public feedback," CMS Administrator Chiquita Brooks-LaSure said in a statement.

"Through this decision, we are creating a pathway for people with Medicare to quickly access drugs the FDA determines have shown a clinical benefit and encourages manufacturers and trial administrators to ensure that the clinical trials recruit racially diverse participants," she added.

CMS posted a fact sheet on the decision along with the decision memorandum.

Kerry Dooley Young is a freelance journalist based in Miami Beach, Florida. She is the core topic leader on patient safety issues for the Association of Health Care Journalists. Young earlier covered health policy and the federal budget for Congressional Quarterly/CQ Roll Call and the pharmaceutical industry and the Food and Drug Administration for Bloomberg. Follow her on Twitter at @kdooleyyoung.

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