Biogen’s Aduhelm treatment for Alzheimer’s disease.
Medicare officials recently finalized a plan to deny almost every patient with early-stage Alzheimer’s disease access to a new, FDA-approved drug that could slow the progress of their disease.
This decision is devastating for California, which is home to more Alzheimer’s patients than any other state. Additionally, the Medicare decision will increase the racial inequities already surrounding Alzheimer’s. Worse still, this rationale could be used to deny access to patients with equally debilitating diseases such as Parkinson’s, multiple sclerosis, epilepsy, and others.
The determination is an explicit rebuke to the federal Food and Drug Administration, the agency responsible for assessing the safety and efficacy of potential new treatments. Last summer, the FDA approved the monoclonal antibody treatment “aducanumab,” marketed as Aduhelm, under the agency’s accelerated approval pathway.
This well-established approval process is available for treatments with exceptional promise, especially those involving new biological avenues or targeting diseases with no or few current treatment options. Indeed, aducanumab is the first new Alzheimer’s treatment the FDA has approved in nearly 20 years.
The accelerated approval pathway allows scientists to evaluate a drug’s efficacy by monitoring changes in so-called “surrogate endpoints.” For aducanumab, that surrogate endpoint is shrinkage of buildups of amyloid-beta protein plaque in the brain.
Scientists strongly believe shrinking those buildups, as aducanumab does, will slow the progression of Alzheimer’s.
Alzheimer’s is a perfect example of why the FDA’s accelerated approval pathway exists. It’s a slow-moving illness, and gauging drugs’ effectiveness solely by monitoring clinical effects in patients would take decades. By measuring amyloid-beta plaques instead of Alzheimer’s symptoms themselves, scientists can gather information in a shorter period of time.
So, it came as a surprise when Medicare officials announced that it would not fully cover aducanumab partly because it was given accelerated, rather than “traditional,” FDA approval. The accelerated approval program has been responsible for the development of more than 250 drugs, including many lifesaving cancer treatments.
Even more concerning, the decision applies not just to aducanumab. The agency has preemptively decided Medicare won’t cover any similar monoclonal antibody-based Alzheimer’s treatment that receive accelerated FDA approval. Only patients enrolled in additional clinical trials will be eligible for coverage.
It’s hard to overstate the impact this decision will have on the development of new treatments for Alzheimer’s. If pharmaceutical companies don’t have confidence that Medicare will cover their FDA-approved products, they will have little reason to invest in future research and development efforts.
The Alzheimer’s population among those 65 or older is projected to reach 840,000 in California by 2025 The disparate racial impact of this drug determination is staggering. It will hit Hispanic Californians, who make up nearly 40% of our state’s population, especially hard.
Hispanic-identifying Americans are 1.5 times as likely as white Americans to develop Alzheimer’s or another form of dementia in their lifetime. A recent report co-authored by the California Department of Health concluded that the number of Hispanic California patients with Alzheimer’s will triple over the next two decades.
The restriction of Medicare coverage to those enrolled in clinical trials is especially galling given the dismal record of our healthcare system in including Hispanics. One analysis of over 100 past clinical trials for Alzheimer’s drugs found that the median percentage of trial participants of Hispanic origin was just 5.6%, less than one-third of the 18.5% of Americans who identify as such. Hispanics and their allies have a right to be skeptical about promises that future trials will be more equitable.
Supporters of this decision often openly admit what Medicare itself denies: that the real purpose of this determination is to save the government money by limiting access to treatment. Restrictions on coverage in the period between FDA accelerated approval and final approval could save billions of dollars a year — by denying treatment to millions whom the FDA has concluded may benefit.
You can be sure, however, that those affluent Californians who can afford to pay cash for aducanumab will access it freely.
Medicare officials may claim that their policy is “final,” but it’s never too late to do what’s right for patients and their families. Federal officials must vacate this terrible decision.
Scott Suckow is the executive director of Liver Coalition of San Diego. He also serves as the chairperson of Patient Advocates United in San Diego County, a group that works collaboratively with policymakers, industry leaders, providers, and consumers to ensure access to care and treatment for San Diegans with chronic health conditions.