Blog written by Sue Peschin, President & CEO, Alliance for Aging Research

Cute child and doctor talking in clinics

In mid-January of this year, the Centers for Medicare and Medicaid Services (CMS) released a proposed National Coverage Determination (NCD) which would only cover anti-amyloid monoclonal antibodies under a Coverage with Evidence Development (CED), requiring beneficiaries to be enrolled in CMS clinical trials to have the treatment covered. On April 7, CMS released their final NCD which confirmed their proposed CED approach.

Monoclonal antibodies that target amyloid, a protein that builds up into plaques which develop first in the areas of the brain that control memory and other cognitive functions, are a promising class of drugs to treat Alzheimer’s. Yet, the CMS determination threatens to severely limit access to the only FDA-approved disease-modifying treatment for Alzheimer’s (Aduhelm), as well as anti-amyloid monoclonal antibodies currently in the treatment pipeline, leaving patients, their families, and caregivers with limited options and little hope.

While this NCD decision clearly has direct and near-term impacts on the Alzheimer’s community, it also establishes a dangerous precedent for future breakthrough medical innovations for other conditions. A deeper dive into the coverage determination also reveals significant concerns for other patient communities, particularly those who currently have no treatments on the market.

Citing Discriminatory ICER Reports as a Source

In recent years, CMS has begun citing data from reports conducted by the Institute for Clinical and Economic Review (ICER) in its materials. ICER and the cost effectiveness framework that it employs – known as the Quality-Adjusted Life-Year (QALY) – discriminate against America’s most vulnerable populations. Evaluating the price of a treatment against one year of “perfect health” disadvantages patient groups such as the disabled, seniors, and those living with chronic diseases as these populations are unlikely to ever reach what ICER’s QALY methodology would consider the threshold of “perfect health.”

Importantly, the Patient Protection and Affordable Care Act disallows the HHS Secretary from using QALY as “a threshold to determine coverage, [or] reimbursement” (i.e., CMS cannot use a cost-effectiveness assessment in its coverage decision-making for patients with AD). Despite this clear parameter, CMS conceded in the final NCD memo that it reviewed ICER’s May 2021 report “Aducanumab for Alzheimer’s Disease: Effectiveness and Value.” Citing ICER publications is also in direct opposition to CMS’ efforts to advance health equity – as these reports discriminate against communities of color and other vulnerable patient populations and increase already significant barriers to treatment access. This inclusion of ICER reports negatively impacts the Alzheimer’s community while also setting a dangerous precedent for other patient populations.

The Threat to Accelerated Approval

Not only does the CMS coverage determination bring about alarming implications for other patient groups, it also threatens an important mechanism established by the FDA to approve urgently needed treatments, such as treatments for Alzheimer’s, on an accelerated basis. The FDA Accelerated Approval Program, used previously to approve treatments for diseases like AIDS and cancer, was instituted by the agency in 1992 to allow for the expedited approval of drugs for very dire disease states where patients and physicians have run out of, or lack entirely, options to treat a fatal illness. With 6.2 million people currently living with Alzheimer’s in the U.S. and cases of dementia expected to rise to over 150 million worldwide by 2050, Aduhelm and other breakthrough Alzheimer’s treatments certainly fit the approval criteria for this accelerated pathway.

However, with this new coverage determination, CMS is calling into question a treatment approved by the FDA using accelerated approval, in turn threatening the use of the pathway in the future for other critically needed cures. In February, the Alliance joined with more than 50 patient advocacy groups to warn HHS Secretary Xavier Becerra that this NCD would have implications beyond Alzheimer’s, stating that it “sets a precedent that could have far-ranging and damaging impacts on approval and access to new therapeutics across all disease areas.”

This decision to undermine the accelerated approval pathway threatens to cut innovation off at the knees, leaving patients and their loved ones without a chance for better health outcomes. We cannot allow this to happen.

Discriminatory Clinical Trials

The clinical trials and registries CMS has required under CED have historically failed at enrolling minority and rural populations, resulting in prolonged coverage restrictions that last for a decade or more. Though CMS often points to its transcatheter aortic valve replacement (TAVR) CED as a success, mandatory registry participation and strict coverage requirements have instead created unnecessary hurdles for smaller and rural hospitals, eliminating access to the less-invasive alternative to open heart surgery for the patients they serve. In effect, it has exacerbated disparities for minority and rural populations—and what’s worse, CMS has cited that the “evidence is insufficient for minority populations” as justification to drag it out further.

A similar scenario is likely to play out for Medicare beneficiaries with Alzheimer’s disease. With Black Americans 2-3 times more likely to be diagnosed with Alzheimer’s and Latino Americans 1.5 times more likely, limiting access for these groups to clinical trials would only worsen existing health inequities.

CMS will also be able to use this decision as a precedent to restrict access to future FDA-approved drugs for other serious and life-threatening conditions, including cancer, ALS, and rare diseases.

And therein lies the rub. Although the term “evidence development” is in the name, CMS uses this type of coverage policy to ration treatments and services that are expected to have large budget impacts. CMS officials deny this, insisting they do not “consider the cost of the treatment” when determining coverage. Yet, on the same day the FDA approved Aduhelm, former HHS assistant secretary Dr. Richard Frank urged Medicare to proactively restrict access to it by initiating an NCD, asserting that “NCDs are the most powerful coverage tool that Medicare has and have generally been reserved for Medicare services that are costly.”

To pile on, Medicare announced that it will apply a unique reimbursement metric to the new Alzheimer’s treatment that will likely result in doctors losing money each time they prescribe it. It was implemented right before the coverage policy was finalized. And Medicare beneficiaries are still waiting on HHS Secretary Becerra to lower beneficiaries’ 2022 Medicare Part B premiums and deductibles. What has gone unacknowledged is the effect of this grandstanding on people with Alzheimer’s and their families. Imagine if the cost of a new cancer treatment was called out to blame for insurance premium increases.

The process of developing and delivering innovative treatments to vulnerable patient groups is an essential one – and one that is currently under threat both by CMS’ coverage determination for an entire class of Alzheimer’s treatments and organizations like ICER that discriminate against those groups. In the face of actions that threaten access to treatment and care, we must raise our voices for patients across the country in opposition to discriminatory policies and decisions that undermine treatment access and future innovations.

Sue Peschin, MHS is the President and CEO of the Alliance for Aging Research, a nonprofit organization dedicated to accelerating the pace of scientific discoveries and their application to vastly improve the universal human experience of aging and health.