The Centers for Medicare and Medicaid Services (CMS) last month released its plan, or lack thereof, for patients to receive coverage for the new Alzheimer’s drug, Aduhelm, along with the entire class of monoclonal antibody (mAB) therapies that target amyloid for the disease. This decision was met with an uproar in the healthcare community. Much of that uproar, from doctors and patients but also the media and the general public, continued to focus on the initial $56,000 price tag that Biogen, the manufacturer, had announced the previous July, despite the fact that the company has since announced they would halve the price.
There are, of course, important reasons to discuss the cost of drugs, especially for the aging. But in this instance, the intensity of the focus on cost has distracted attention from a larger concern raised by the proposed announced CMS plan for Aduhelm: what will it mean long-term for access to medications approved by the U.S. Food and Drug Administration (FDA), and in the immediate future, for people living with Alzheimer’s disease and their families?
In June 2021, the FDA approved Aduhelm under the FDA’s Accelerated Approval Program. The Accelerated Approval pathway is used frequently in oncology and has been used for other life-threatening conditions such as multiple sclerosis and rare diseases, where patients and physicians have run out of, or lack entirely, options to treat a fatal illness. This process provides interim approval to drugs that meet a surrogate endpoint, a measure that is believed to predict likely clinical benefit but is not a direct measure of clinical benefit. Under FDA requirements, sponsors conduct post-marketing studies that then describe and verify the clinical benefit of the drug.
An even cursory read of the CMS announcement about its action on Aduhelm reveals an unsettling path forward that potentially moves beyond existing CMS precedents and essentially challenges the validity of FDA drug approval. The proposed National Coverage Determination (NCD) process requires beneficiaries to participate in randomized controlled clinical trials for mAB therapies that have already been approved by the FDA as safe and effective-. As a result, only individuals participating in these clinical trials would have their costs covered for FDA-approved drugs in this class. Others, for all practical purposes, would be left without coverage, and would either have to pay completely out-of-pocket for the treatment, or lose out entirely. Since clinical trials typically only include a few thousand participants, millions of Alzheimer’s patients who might, in conjunction with their physician, make a medically informed decision to seek treatment with mABs are having that choice stripped away from them.
It’s also important to note that a randomized controlled trial is commonly understood to mean a study that includes a control or placebo arm. Requiring patients to roll the dice on actually getting treatment as a predicate to having Medicare cover their (possible) care when FDA has already approved a treatment which is available is a violation of basic medical ethics. It is also medically unethical for CMS to charge patients receiving a placebo a co-payment; to require those patients to undergo placebo IV administration in higher-risk hospital outpatient settings during an ongoing pandemic; to further be put at risk of potential infection from the placebo IV administration; and to pay out-of-pocket for transportation to, and parking at, the hospital. It is unfathomable for CMS to require coinsurance payments from beneficiaries in exchange for receiving a placebo, especially when the treatment is to slow the progression of a debilitating, fatal disease like AD.
The Alliance, along with our Alzheimer’s Disease Policy Task Force partners, have submitted a public comment in support of coverage for Food and Drug Administration (FDA)-approved uses of monoclonal antibody therapies targeting amyloid for the treatment of Alzheimer’s disease for Medicare beneficiaries nationwide. CMS has never before used this approach to restrict Medicare coverage for prescriptions drugs. This would be the first time, and we believe it will not be the last. In the Alliance’s experience and view, this coverage tool is all about saving Medicare costs, full stop. Medicare beneficiaries with Alzheimer’s have paid into the program for decades, with the expectation that the program would help pay for their medical care as they age. It is time for CMS to meet that expectation by allowing people living with Alzheimer’s disease to decide whether using FDA-approved drugs is appropriate for their care.
