The CMS CED Policy Limits Treatment for Early Alzheimer's Disease: What It Is and How It Works

A Patient Care interview^© with Sue Peschin, MHS, president and CEO of the Alliance for Aging Research in Washington, DC.

Patient Care. Would you set the stage for a deeper look at barriers to treatment access with some background on the medications approved by the US FDA for treating early Alzheimer’s disease?

Sue Peschin, MHS: The Alliance for Aging Research is dedicated to changing the narrative to achieve healthy aging and equitable access to care. We’ve been involved in this issue because for more than two years because the Medicare program has barred equitable coverage for an entire class of FDA-approved treatments for early Alzheimer’s. There are two treatments currently available—one called Leqembi (lecanemab; Eisai) and a second, newer FDA-approved drug in the same class, called Kisunla (donanemab; Lilly).

The Centers for Medicare & Medicaid Services (CMS), which is the federal agency that runs the Medicare program, restricts access to these life-changing treatments by requiring beneficiaries and their physicians participate in coverage with evidence development (CED). Under a CED coverage policy, beneficiaries must enroll in clinical studies to get coverage. This is inequitable because these studies are primarily run in large medical centers in wealthier urban areas. That’s because CMS imposes strict eligibility criteria on the types of doctors and hospitals that can qualify to collect data and run the studies. They do this as a way to cut costs in the Medicare program.

Are there estimates of the size of the population eligible for the drugs and of that group, what proportion is actually receiving treatment?

It’s estimated that more than 1 million Americans have early Alzheimer’s. We’ve been told that the Medicare claims data to-date shows there are about only 10 000 patients currently receiving treatment—but, CMS has not offered any public reporting on this.

Time matters for Alzheimer’s patient and their loved ones. Each day that CMS leaves this misguided policy in place, nearly 2000 individuals aged 65 or older may transition from mild dementia due to Alzheimer's to a more advanced stage of the disease where they are no longer eligible to receive the new FDA-approved treatments.

Delaying progression in early disease represents real time—time where cognition, personality, and the ability to care for oneself remain largely intact. If this value is not reflected in Medicare's assessment of what should be considered "reasonable and necessary," that is a deficiency in the way CMS' leadership is looking at this drug.

Patient Care: The drugs are approved, meaning they have satisfied the FDA’s scientific and clinical efficacy requirements. Cost is a significant barrier, but there are more fundamental health care system forces at work that you have referred to as rationing barriers. Would you talk about those?

Peschin: CMS is a payer, not a scientist who determines efficacy. CED has become an efficient way for CMS to ration items and services estimated to create a higher cost burden in Medicare Part B, which covers treatments administered in clinics and doctor's offices.

Furthermore, once CMS places a treatment in CED it's extraordinarily difficult to reverse the decision. An August 2022 review in The American Journal of Managed Care identified that between 2005-2022, CMS denied coverage under CED for 27 different therapies. Since 2005, only five of the denials have been retired by the agency, and several of the current 22 CEDs "studies" have been ongoing for more than 15 years.

CMS has applied CED rationing to procedures for severe hearing loss (cochlear implants), less-invasive heart valve replacement, and stem cell transplant for certain cancers and sickle cell disease.

Patient Care: How does the CMS CED for the early Alzheimer’s medications differ from other drugs or devices and procedures?

Peschin: The agency's April 7, 2022, Alzheimer's coverage decision was the first time CMS applied CED to an on-label use of an FDA-approved drug. There are strong signals from CMS that it won't be the last, as gene and stem cell therapies for cancer and rare diseases continue to progress through clinical development.

In Medicare’s final coverage policy, the CED requirements differ based on the FDA approval process of the Alzheimer’s treatment. If a therapy is approved through the FDA’s “accelerated approval” pathway — designed to give patients earlier access to medicines that are likely to be clinically beneficial, though not fully proven — Medicare will require Alzheimer’s beneficiaries to enroll in randomized control clinical trials. Such studies can take years to complete, and patients risk receiving a placebo rather than the actual treatment.

For drugs approved through the traditional FDA pathway — drugs that are fully proven to provide clinical benefits, such as slowing cognitive and functional decline — Medicare will then require Alzheimer’s patients to enroll in a data registry. While these mandated registries may sound good on paper, they have historically exacerbated health disparities in practice.

Patient Care: Primary care clinicians today are responsible for nearly all diagnoses of early dementia – how do these policies impact their ability to provide care?

CED interferes in the doctor-patient relationship, full stop. It’s not only rationing, but it’s paternalistic to older people and their families and the ability for them to have benefit-risk conversations with their docs and make decisions about treatment.

Your organization, the Alliance for Aging Research recently conducted a poll of American voters on the restrictive CMS policies – would you highlight the findings?

Peschin: The poll, commissioned by the Alliance for Aging Research, UsAgainstAlzheimer’s, and the Partnership to Fight Chronic Disease (PFCD), shows voters – both Democrat and Republican – disagree with the CMS’s restrictive policies:

• 63% of voters said that they, a family member or friend has Alzheimer’s disease.
• 92% of voters agree that Medicare should provide all beneficiaries access to FDA-approved Alzheimer’s drugs, just as they do to FDA-approved drugs for all other diseases.
• 89% of voters agree that Medicare should immediately cover the costs of these FDA-approved Alzheimer’s drugs.
• 78% of voters agree that the government should not require patients to participate in medical research studies on any FDA-approved drug in order to get access to that drug.
• 90% of voters agree that Medicare should cover all FDA-approved drugs and therapies for Alzheimer’s disease and allow patients and their doctors to make decisions based on risks, benefits, and individual health needs.
• 78% of voters agree that if Medicare does not act, Congress should step in to require that Medicare cover FDA-approved drugs and therapies that can slow the progression of early Alzheimer’s disease.
• 58% of voters said they would be more likely to vote for a candidate for elected office if that candidate supported requiring Medicare to cover FDA-approved drugs and therapies that can slow the progression of early Alzheimer’s disease. Just 3% of voters would be less likely to vote for them.

The American people get that Medicare is a payer, and it’s their job to cover and pay for FDA-approved medications, not to dictate clinical care. Voters know that doctors and families with Alzheimer’s are more than capable of making treatment decisions based on risks, benefits, and individual health needs—and they’ll vote for candidates who promise to make sure that happens. They don’t like what Medicare has done to restrict access to Alzheimer’s treatments, and they want Congress and the President to step in if the agency doesn’t change its ways.

Patient Care: What are some of the key policy changes Alzheimer’s advocates are currently fighting for to improve access to medication?

Peschin: We need CMS to end it’s CED policy on this class of medications. We also need Medicare to pay more for newly approved Alzheimer’s blood tests. Right now agency is only willing to pay $17 when advisory unanimously recommended $130.

Patient Care: Looking to the future, what gives you the most hope for advancing access to Alzheimer’s treatment?

The science is more exciting than ever, with multiple targets and more companies investing in finding even better treatments.

Most families contending with Alzheimer’s know that, just like most cancer drugs do not cure cancer, [lecanemab] and [donanemab] do not cure early Alzheimer’s disease. However, like many cancer drugs, clinical trials of these Alzheimer’s therapies have shown effectiveness in delaying disease progression. A recently published study that modeled long-term scenarios found that starting the treatments during the early symptomatic stages of Alzheimer’s disease could delay severe dementia by four to seven months using conservative estimates, and potentially up to 2 to 4 years. This is particularly important for people living with early disease, as cognition, personality and ability to care for oneself slowly decline with each passing day.

The risk of side effects related to [lecanemab] is low, especially compared with almost any cancer drug. Yet, because Alzheimer’s is a deadly disease primarily affecting older adults, it is more likely to be underdiagnosed and undertreated.

Patient Care: Is there anything you’d like to add that we haven’t discussed?

Peschin: We need to keep in mind what’s at stake here. It’s Medicare beneficiaries navigating early Alzheimer’s who will really pay the price, as treatments to delay progression of their disease are finally available but still out of reach due to Medicare’s CED policy.

It is time for members of Congress to closely examine CMS’ abuse of CED and oppose any efforts to expand or codify it. This is a case of CMS overreaching on the FDA’s biomedical agency expertise and authority, at the expense of patient access. This must stop. If we don’t do something today, we will pay a much bigger societal price tomorrow.