IIn a clash between two American health giants, the Centers for Medicare and Medicaid Services (CMS) and the Food and Drug Administration (FDA) had opposed Aduhelm, a controversial drug against Alzheimer’s disease. Medicare took the upper hand Thursday when it released its final decision to limit coverage for the FDA-approved drug.
To recap: The FDA gave the green light to Aduhelm (aducanumab) in June 2021. It was the first drug approved to treat Alzheimer’s disease in 18 years. The announcement came amid a whirlwind of controversy, marked by the resignation of several FDA advisers and by Congress investigating the FDA approval process, which heavily involved Biogen, the drug’s maker. In January 2022, Medicare announced its intention to cover the cost of the drug only for people enrolled in randomized trials testing the effectiveness of Aduhelm. On Thursday, three days before its decision was made public, Medicare announced its final decision to restrict coverage to people participating in clinical trials.
As federally funded physician-scientists with expertise in measuring the value of health care who treat patients with Alzheimer’s dementia, we believe that Medicare made the right decision based on the benefits very uncertain of Aduhelm and known wrongdoings. The move was met with intense opposition from special interest groups and lobbyists, who ultimately failed to convince Medicare to back down. Meanwhile, Biogen threatened to sue Medicare, and the Wall Street Journal’s editorial board accused Medicare of becoming an “Alzheimer’s death committee” for the government’s “rationing” of care.
Rationing, however, means restricting care due to cost. In our view, Medicare’s decision is justified regardless of the costs.
Historically, Medicare coverage decisions have required health services to be “safe and effective.” Aduhelm fails on both counts. Of those treated with the drug, 41% developed brain swelling or bleeding, 2% experienced serious adverse events, such as irreversible vision loss due to brain damage, and long-term drug toxicities remain unknown. The death of at least one patient who experienced brain swelling after receiving Aduhelm is under investigation.
Although some of Aduhelm’s disadvantages are well known, its advantages are far from clear. Several previous clinical trials show that antibodies targeting amyloid plaque, the drug’s mechanism of action, failed to improve cognition. Two trials using higher doses of Aduhelm showed a possible benefit in one trial but not the other. These two trials were not representative of Medicare beneficiaries, especially with respect to race or ethnicity since the vast majority of participants were white. Additionally, Biogen only noticed the positive result after the company announced that the trials had no chance of showing the drug had any benefit.
Retrospective analyzes increase the risk of false positive results, especially when two similar trials show divergent results, as is the case for Aduhelm. False-positive results are more than theoretical risks: Retrospective analyzes previously offered false hope for solanezumab, another drug that targets amyloid plaque.
Given its troubling safety profile, uncertain benefits, and unrepresentative trial population, Aduhelm merits a racially and ethnically representative confirmatory trial, which should have been completed prior to FDA approval. , not after it became widely used.
Although Medicare cannot consider cost when determining coverage, the American public certainly can and should, especially for unproven treatments with obvious harms. Medicare is already expected to exhaust its trust fund by 2026, jeopardizing its ability to cover even basic, life-saving therapies such as blood pressure medications. Meanwhile, beneficiaries’ health expenditures are rising rapidly, putting pressure on household spending on necessities such as food or rent.
Against this backdrop, the staggering $56,000 annual price Biogen had initially set for Aduhelm raised concerns that offering an unproven drug to millions of Alzheimer’s patients could hasten the company’s bankruptcy. Medicare. Invariably, the costs would be passed on to beneficiaries and taxpayers who fund Medicare, which has already begun to happen. In November 2021, Medicare preemptively increased beneficiary premiums by nearly 15%, citing Aduhelm’s budget impact. Biogen’s subsequent reduction in the drug’s annual price to $28,200 does not resolve Aduhelm’s potential financial strain, and whether premiums would remain heavily increased was highly dependent on Medicare’s final coverage decision.
To quantify the precise financial impact of Aduhelm had Medicare approved it for widespread use, we and several colleagues identified between 1.1 million and 5.7 million U.S. Medicare beneficiaries with mild cognitive impairment or dementia. mild who would be eligible to receive the drug. As we wrote in the JAMA Health Forum, if only a quarter of them received it, Medicare would have to pay an additional $7 billion to $37 billion each year, even after taking into account Biogen’s price cut. Ancillary care, such as MRI scans needed to monitor swelling or bleeding in the brain, would account for almost 20% of total Aduhelm costs. Annual out-of-pocket costs for an individual patient could reach $6,800, or 26% of the median annual income of Medicare beneficiaries.
Opponents of Medicare’s decision to cover the cost of Aduhelm only for people participating in clinical trials argue that rebuking the FDA sets a chilling precedent for future drug development. But this isn’t the first time Medicare has refused to cover an FDA-approved therapy, and the possible effects on innovation, if any, must be weighed against Aduhelm’s unproven benefits and harms. known.
Critics also say Medicare will undermine equity by excluding racial and ethnic minority groups who cannot access Aduhelm at academic centers that run trials. Yet it is the widespread use of Aduhelm that would harm fairness. If Medicare had backtracked and decided to cover the cost of Aduhelm for all, its 60 million beneficiaries — especially minority people who bear the brunt of out-of-pocket costs — would have been burdened with higher premiums needed to fund the widespread use of Aduhelm. .
We believe that Medicare wisely decided not to cover Aduhelm outside of randomized trials until more is known about its safety and effectiveness among various populations. Despite intense pressure from lobbyists and special interest groups, he stopped an unproven drug from threatening the financial stability of Medicare and millions of American homes.
John N. Mafi is a general internist and associate professor of medicine at UCLA. Catherine Sarkisian is a geriatrician and professor of medicine at UCLA. Both care for patients with Alzheimer’s dementia and receive grants from Arnold Ventures and the National Institutes of Health to study harmful, low-value care in older Americans. The opinions expressed here are their own and do not necessarily reflect the views of UCLA or the NIH.