On Tuesday (11 Jan), the CMS released a proposed National Coverage Determination decision memorandum: stating it would cover FDA-approved monoclonal antibodies that target amyloid for the treatment of Alzheimer’s disease solely through coverage with evidence development – meaning it will cover the drug solely for people enrolled in qualifying clinical trials.
Aduhelm is currently the only therapy in this category: but the CMS’s decision is set to apply to similar therapies coming through the pipeline.
The proposal is open to public comment for 30 days, with a final decision to follow by April 11.
“This proposed National Coverage Determination is the result of robust evidence analysis conducted through a thorough review process that found while there may be the potential for promise with this treatment [Aduhelm], there is also the potential for harm to patients. This harm may range from headaches, dizziness, and falls, to other potentially serious complications such as brain bleeds,” said Dr. Lee Fleisher, CMS Chief Medical Officer and Director of the Center for Clinical Standards and Quality.
“We believe that any appropriate assessment of patient health outcomes must weigh both harm and benefit before arriving at a final decision. Therefore, based on the public comments submitted previously and evidence CMS reviewed, the potential for harm, and important questions that remain, we have determined that coverage with evidence development through clinical trials is the right decision for Medicare patients, clinicians, and caregivers, and we look forward to receiving feedback on the proposal.”
Aduhelm was approved by the FDA in June: although the decision was not without controversy with questions over its efficacy. In December, the European Medicines Agency said it would not approve the drug in the bloc over safety and efficacy concerns.
Approval in the US was based on data from clinical trials showing the effect of Aduhelm on reducing amyloid beta plaques in the brain: with the accumulation of such plaques a feature of Alzheimer’s disease.
The EMA, however, said that the link between reducing amyloid beta in the brain and clinical improvement had not been established.
Biogen: Each day matters to Alzheimer’s patients
Responding to the announcement on Tuesday, Biogen says it will make a formal response to the NCD consultation, noting the proposed coverage plan ‘denies nearly all Medicare beneficiaries from accessing Aduhelm’.
“It is imperative to change this draft decision to be aligned with reimbursement for other therapies for progressive diseases, where patients have immediate and equal access to medicines approved by the FDA,” it said in its statement on Tuesday.
“The FDA’s accelerated approval was supported by clinical data showing that Aduhelm impacted the underlying pathology of Alzheimer’s disease. This includes a robust reduction in pathological hallmarks of Alzheimer’s disease, specifically for both amyloid plaques and neurofibrillary tangles in the brain.
“It is urgent to act. Thousands of patients progress each day from mild to moderate disease stages, where treatment may potentially no longer be an option. For these patients, each day matters. If a final NCD, expected to be issued in April, continues to require a randomized controlled trial as outlined in this draft, it would likely take in excess of a year to begin enrolling patients, further delaying reimbursement for Medicare beneficiaries. It is also particularly concerning that this draft implies that some Medicare beneficiaries will receive a placebo instead of a treatment they are seeking.”
“We at Biogen urge CMS to align Medicare coverage for the class of amyloid-directed therapies consistent with the criteria used in the respective clinical trials and guided by expert recommendations for appropriate use.”
Precedent for other drugs?
FDA approval for Aduhelm in June was met with hope that it would invigorate investment and R&D in the field: with several significant drugs in the pipeline. The hurdles since then, however, have shown the path forward may not be as easy as hoped: particularly if longer clinical trials are required to convince regulators and insurers.
Jayne Hornung, the Chief Clinical Officer at market data firm MMIT (Managed Markets Insight & Technology), is a clinical pharmacist working on research on drug, indication and policy data. She notes that the proposed policy for Aduhelm – the first Alzheimer’s drug approved since 2003 – could have a large impact on other drugs in the pipeline.
“Medicare almost always pays for FDA-approved drugs, at least for the medical conditions designated on their label in the process of treating a disease,” she said.
“But with Aduhelm, Medicare officials have decided to severely limit the coverage to people who have mild forms of cognitive impairment or mild dementia, and who have amyloid plaques. This patient population is much narrower than the one the FDA approved the drug to treat. If the preliminary decision is finalized later this year, it would significantly limit the number of patients who would gain access to Aduhelm.
“The proposed coverage decision is not specific to Aduhelm, either. It appears to apply to all monoclonal antibodies directed against amyloid for the treatment of Alzheimer’s disease. And since private insurers often follow Medicare’s lead, we’re likely to see private payers limiting coverage of monoclonal antibodies treating amyloid plaque to clinical trials or not covering the products at all, considering them investigational and not proven therapies.
“This could have ripple effects in the pipeline products being developed by Lilly, Eisai and Roche, who all have monoclonal antibodies in Phase III clinical trials. We may also see clinical trials going longer to prove the specific requirements in Section C put forth in the proposed decision by CMS.”