Alzheimer’s Drugs Face Uncommon Market Access Challenges
Approximately 6.9 million Americans aged 65 and older have been diagnosed with Alzheimer’s disease (AD). By 2060, prevalence could reach 13.8 million if no therapies are approved to prevent or cure AD.
For decades, Alzheimer’s treatment focused on symptom management, with stagnant progress in experimental breakthroughs. That changed recently with the development of beta-amyloid targeting therapies. Although these disease-modifying drugs are not a cure for AD, they do remove beta-amyloid buildup in the brain, a promising yet still debated approach to slowing cognitive decline.
In June 2021, Biogen’s Aduhelm became the first of these therapies to hit market through the FDA’s accelerated approval pathway. Aduhelm received heavy pushback from the get-go, due to its high price tag and irregularities in the approval process. The drug was later discontinued in January 2024. The Alzheimer’s space pushed forward with Eisai’s Leqembi receiving the first traditional FDA approval as an anti-amyloid treatment in July 2023, after initially gaining accelerated approval seven months prior. Eli Lilly’s Kisunla followed in July 2024 with traditional approval.
The Alzheimer’s market is forecasted to grow from $7.7 billion in 2023 to $12.3 billion in 2032, driven by research advancement, an increasing aging population with higher disease prevalence, and more investments in drug development. Despite the recent progress, significant barriers persist, including regulatory approvals, coverage not equating to utilization, and a burdensome CMS policy that continues to impede access to Alzheimer’s therapies.
Regulatory Requirements and Approval Concerns
Alzheimer’s treatments face especially strict regulatory requirements. As AD is a complex disease, long-term trials are required to demonstrate efficacy and safety. However, drugs can receive accelerated approval through surrogate endpoints to expedite patient access to treatment.
Aduhelm’s accelerated approval was based on its reduction of beta-amyloid plaque, but many critics argued that the evidence was inconclusive, and raised concerns about the drug’s clinical value and safety. Known as the “Aduhelm controversy,” this event led to a congressional inquiry and raised questions on the validity of the accelerated pathway for Alzheimer’s treatments.
As a result, many access experts predict that AD pipeline drugs may face delayed approvals or skepticism regarding their efficacy data.
Promising Medicare Coverage
Unlike the many therapeutic areas shifting from medical benefit to pharmacy benefit coverage, AD is experiencing the opposite transition, as these drugs require intravenous administration in clinical settings. According to MMIT’s December 2024 Analytics PAR coverage data for Alzheimer’s, Medicare coverage for pharmacy benefit products focuses on the utilization of symptom-reducing generics to mitigate cost.
Coverage data also reveals that Medicare trumps all other channels in covering Leqembi and Kisunla under the medical benefit, at 81% and 37%, respectively. This is promising, given that Alzheimer’s primarily affects the older population. Historical coverage data shows that Leqembi saw increased coverage from 26% of overall lives at six months post-launch, to 52% at 10 months post-launch.
Comparatively, Kisunla had 37% coverage of overall lives at six months post-launch. While Kisunla’s coverage may expand throughout 2025 as its market share increases, it remains to be seen if Kisunla coverage will exceed Leqembi coverage. Patients may terminate Kisunla treatment once brain amyloid plaques are significantly reduced, which could result in a shorter duration of treatment and lower costs.
CED Policy Results in Utilization Disparity
While Medicare coverage is arguably high for Leqembi, the number of patients actually receiving treatment may be small. Private and public payers alike have been rationing access to these therapies. This is largely due to the fact that Medicare requires coverage with evidence development (CED) for all FDA approved early-Alzheimer’s therapies. As of late 2024, CED is not required for any other FDA-approved drugs with on-label use.
Under the CED policy, to receive coverage for traditionally approved AD drugs like Kisunla, Medicare beneficiaries must enroll in a data registry. Benefits include a standard data collection method for future studies, while drawbacks include limited treatment access, worsened health disparities, and workflow burden.
CED requirements for drugs approved via the accelerated pathway are even more rigorous, which may hinder drugs in the pipeline. In order to receive Medicare coverage for accelerated approved drugs, Alzheimer’s patients must enroll in clinical trials. There are extensive regulations on which facilities and physicians qualify to run these studies. In addition to the fact that many beneficiaries are unable to access clinical study sites, these trials span several years, and some patients could potentially receive placebo treatments.
While the CED policy was initially intended to accelerate access to medical devices, its expansion to other therapeutic areas like Alzheimer’s has in fact impeded patient access to necessary therapies. The Alliance for Aging Research reported that in more than two decades of implementation, the CED policy has largely missed the mark in either broadening timely access or generating meaningful evidence. As a result, any novel, high-cost Alzheimer’s drug faces not only coverage challenges, but uncommonly restrictive patient access challenges.
Market Access Strategies for Manufacturers
To secure fair prices and improve patient access, there are a few strategies manufacturers of AD therapies can consider.
First, given that the two beta-amyloid drugs, Leqembi and Kisunla, have been shown to reduce cognitive decline, payers may see the benefit in value-based contracting. Slowed disease progression or reduced hospitalizations means improved quality of life and reduced costs for this patient population. Manufacturers should educate payers on the long-term value and cost-effectiveness of these treatments as well as others in the pipeline, as this data may support negotiations for fairer prices and reimbursements.
In addition to offering patient assistance programs, manufacturers should also consider including travel reimbursements for patients who will be participating in clinical trials. Manufacturers can also collaborate with HCPs so that both providers and patients understand how to effectively navigate insurance for these new therapies. Manufacturers of other high-cost drugs for diseases like hemophilia can also learn from Alzheimer’s market access challenges by applying these strategies.
In the near future, new breakthroughs in the Alzheimer’s space are expected. Disease-modifying drugs, including tau protein targeting therapies, are in development, and could join beta-amyloid targeting treatments in the successful reduction of cognitive decline. Existing treatments, like Leqmebi, are receiving label expansions for maintenance dosing, easing the patient burden.
Despite access challenges exacerbated by the CED policy, manufacturers that collaborate with insurers and physicians in providing patient support can potentially broaden access to their therapies. Increased awareness of the long-term value of these drugs will ultimately help increase prescriber and payer confidence.
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