By Cathy Kelly
There is no fee to participate, and entries to the registry must be made at baseline and then every six months for up to two years.
The Centers for Medicare and Medicaid Services is sticking with its plan to provide broader Medicare coverage for Eisai Co., Ltd. and Biogen, Inc.’s Leqembi for Alzheimer’s – now that it has obtained a traditional approval – only when patients are enrolled in a registry.
The move is groundbreaking in that it is the first time Medicare coverage for a traditionally approved drug will be conditioned on further evidence generation. It remains to be seen how much the registry as envisioned by CMS will deter access to the drug. (Also see “Medicare’s Alzheimer’s Drug Registry: Ease Of Use May Lead To Data Quality Tradeoffs” – Pink Sheet, 25 Jun, 2023.)
The registry requirement was established in a national coverage determination for the class of amyloid-targeting monoclonal antibodies for Alzheimer’s disease that was issued by CMS in April 2022. (Also see “Medicare Alzheimer’s Decision Varies Evidence Mandate For Accelerated vs. Traditional Approvals” – Pink Sheet, 7 Apr, 2022.) Under the NCD, coverage of Leqembi was previously restricted to use in a clinical trial because it had an accelerated approval. In effect, that has basically meant non coverage in Medicare.
CMS released a statement with a link to the CMS-facilitated registry portal for anti-amyloid drugs for Alzheimer’s disease on 6 July following the US Food and Drug Administration’s announcement about full approval. (See box for story on the expanded warnings in the label.)
“With FDA’s decision, CMS will cover this medication broadly while continuing to gather data that will help us understand how the drug works,” the agency said. “To receive Medicare coverage, people will need to:
- “Be enrolled in Medicare,
- “Be diagnosed with mild cognitive impairment or mild Alzheimer’s disease dementia, with documented evidence of beta-amyloid plaque on the brain, and
- “Have a physician who participates in a qualifying registry with an appropriate clinical team and follow-up care.”
Clinicians participating in the registry “will only need to complete a short, easy-to-use data submission,” CMS said. There is no fee to participate, and entries to the registry must be made at baseline and then every six months for up to two years.
Physicians may participate in more than one registry for the treatments but are not required to. “Other registries may become available in the coming months and will be posted on the CMS website,” the agency noted.
Details On Data To Be Submitted
The agency provided additional information on the data points that physicians would need to enter and said that “researchers will have access to the information to conduct studies intended to further the knowledge of how these drugs can help people with Medicare by answering the research questions outlined in the national coverage determination.”
Physicians are asked to record their contact information, the patient diagnosis, date of diagnosis, what kind of test was performed to confirm the presence of amyloid plaque on the brain (amyloid PET scan, cerebral spinal fluid test or “other” amyloid test), results of the Montreal Cognitive Assessment (MoCA) cognitive test or other cognitive test that was administered, such as the Mini-Mental State Exam, and results of the Functional Activities Questionnaire
It also requests physicians to indicate whether they performed a Clinical Dementia Rating, while noting the information is optional. If it was performed, clinicians are asked to provide scores for memory, orientation, judgment and problem solving, community affairs, home and hobbies, personal care, the CDR sum of boxes and global CDR.
Additionally required is information on whether a patient is on anticoagulants or antiplatelets and which monoclonal antibody for Alzheimer’s was used, which at this point would be either Leqembi or Biogen’s Aduhelm (aducanumab). Finally, physicians must indicate whether there is evidence of significant amyloid related imaging abnormalities with edema or ARIA with hemorrhage.
In information for providers, the agency provides a description of the type of study it would like to see on the drug. Its primary objectives are:
- To assess whether anti-amyloid mAbs meaningfully improve health outcomes (i.e., slow the decline of cognition and function) for patients in broad community practice.
- Whether there has been statistically significant stability in daily living functional activities over 24 months.
- Whether benefits and harms such as brain hemorrhage and edema associated with use of the drug varies according to characteristics of patients, providers, and clinical setting. Harms will be defined primarily as the occurrence of ARIA-E and ARIA-H, which will be evaluated at 6, 12, 18 and 24 months.
- How clinical benefits and harms change over time.
CMS said Medicare will cover 80% of the cost of Leqembi with patients responsible for a standard 20% coinsurance share of the drug’s cost after patients meet their Medicare Part B deductible. Patients’ shares of Leqembi’s costs may differ if they have Medicare supplemental coverage or other secondary insurance or are enrolled in a Medicare Advantage plan.
The agency did not address coverage for MRIs, amyloid PET scans and other monitoring required or recommended in Leqembi’s label.
Patients Advocates Remain Skeptical About Registry
Initial statements on the Leqembi approval from Alzheimer’s disease patient advocacy groups reiterate their position that Medicare coverage for a traditional approval should be unencumbered by any evidence collection.
“While this decision is unquestionably welcome news for people living with disease, attention now turns to the … CMS to fulfill their public commitment to provide broad coverage for the treatment,” UsAgainstAlzheimer’s VP and senior advisor for health equity Stephanie Monroe said.
“The scientists at FDA are in the best position to determine whether a drug is safe and effective. Now that FDA has made their decision, it is not appropriate for CMS to second-guess that decision. We urge them to keep their word and provide coverage for Leqembi for all populations, including those in the black and brown community disproportionally impacted by Alzheimer’s.”
Similarly, “we now look to … CMS to immediately pay for this safe and effective therapy for Medicare patients living with Alzheimer’s disease just like they do for any other drug. We expect nothing less than coverage to label,” Alzheimer’s Global Platform Foundation president John Dwyer said.
Although a number of the data points requested for the portal are consistent with the FDA label, the fact that CMS is requiring five assessments over 24 months is an “unprecedented burden on physicians,” Dwyer told the Pink Sheet.
Furthermore, there will be “no patient history, recording of comorbidities, race, gender, or ethnicity in this registry so the value of this data is minimal and not well designed to elucidate the questions for which CMS claims it wants to gather more data to answer,” he added.
This article was originally published in Pink Sheet Citeline Regulatory