CMS Requires Registry to Access FDA-approved Alzheimer’s Treatments:

Implications for Pharmaceuticals Seeking to Gain Medicare Access

Situation

The Centers for Medicare & Medicaid Services (CMS) announced on June 22nd a detailed plan regarding its coverage of new Alzheimer’s treatments, following the full approval of Eisai’s Leqembi by the FDA. In the National Coverage Determination (NCD) the CMS outlined that Medicare would cover the drug when a prescriber or hospital participates in a nationwide, CMS-facilitated registry to track real-world efficacy outcomes [1]. Specifically, to get coverage, patients will need to be 1) enrolled in Medicare, 2) diagnosed with mild cognitive impairment of mild dementia and 3) have a physician participating in a registry with an appropriate clinical team and follow up care. [1,2]

Background

The backdrop for this decision dates to the summer of 2021, when the FDA approved the Alzheimer’s drug Aduhelm (developed by Biogen and Eisai) based on a surrogate endpoint that showed patients had significantly decreased levels of amyloid plaques, a biomarker that has been the central focus of most Alzheimer’s research [3] . Despite 10 out of 11 FDA advisory panel members voting against the drug’s approval, Aduhelm was approved under the accelerated approval pathway [3,4]. However, the link of the biomarker to patient clinical outcomes was not well-established, and the continued safety concerns put the benefit-risk profile of the drug into question [5]. As a result, in April 2022 the CMS issued a controversial Medicare coverage proposal which restricted Aduhelm and any future monoclonal antibodies directed against amyloid to only patients who are enrolled in qualifying clinical trials [6].

Insights on CMS Registry

CMS Requires Registry to Access FDA-approved Alzheimer’s Treatments: Article by Steven Lin, Windrose Consulting Group

The new registry approach by CMS intends to make Alzheimer’s drugs more widely available for Medicare beneficiaries. The information collected via an “easy-to-use” portal will help the CMS to answer the following questions outlined in National Coverage Determination (NCD) [1]:

1. Does the drug meaningfully improve health outcomes (i.e., slow the decline of cognition and function) for patients in broad community practice?

2. Do benefits and harms, such as brain hemorrhage and edema, associated with the use of the drug, depend on the characteristics of patients, treating clinicians, and setting?

3. How do the benefits and harms change over time?

Clinicians will be required to submit to the registry information on patients’ demographics, clinical diagnosis, any adverse events, whether the patient is taking an anticoagulation or antiplatelet drugs, results of PET scan, cerebrospinal fluid test, or other amyloid test, and results of cognitive function tests [1]. To support collection of data, this October, CMS has abolished their NCD on coverage of PET scans, previously restricted to 1 scan per person enrolled in a clinical trial, and now look to regional Medicare administrative contractors (MACs) to determine their own local coverage decisions. [7]

Windrose’s Take

For manufacturers seeking reimbursement from Medicare, a comprehensive P&MA strategy that encompasses plans for CMS compliance and collection of registry data is vital. Facilitating CMS data collection requirements is not only a regulatory obligation but also a gateway to access the very large Medicare market for Alzheimer’s disease.

Registry data will help secure Medicare access and can also yield influence in coverage negotiations with commercial plans. Moreover, the availability of robust registry data can enhance market acceptance, garnering the trust of payers, healthcare providers, and patients alike. However, the process of collecting and reporting data can place a substantial administrative burden on providers, even if sponsored by the CMS, which demands additional time and resources for data entry and management. While CMS advertises the easy-to-use portal and streamlined process, since April 2022, only 3 studies have been determined to meet requirements for coverage [8] which speaks to the administrative challenges. Securing early physician engagement and patient support will be crucial to streamline time-to-access post-FDA approval.

The NCD for Alzheimer’s represents a shift in the Medicare access landscape for high budget impact therapies which have unclear patient relevant benefit. Continuous registry data collection could be an essential part of the post-marketing strategy for Medicare Part B access in the future. Manufacturers should explore collaborative opportunities with providers and organizations involved in data collection to streamline the process post-FDA approval and demonstrate a commitment to real-world evidence generation ahead of Medicare negotiation.