Decision Follows Robust Stakeholder Input and Creates Pathway for Enhanced Access and Coverage of Drugs that Receive Traditional FDA Approval
Today, the Centers for Medicare & Medicaid Services (CMS) released a national policy for coverage of aducanumab (brand name Aduhelm™) and any future monoclonal antibodies directed against amyloid approved by the FDA with an indication for use in treating Alzheimer’s disease. From the onset, CMS ran a transparent, evidence-based process that incorporated more than 10,000 stakeholder comments and more than 250 peer-reviewed documents into the determination.
As finalized in this two-part National Coverage Determination (NCD), Medicare will cover monoclonal antibodies that target amyloid (or plaque) for the treatment of Alzheimer’s disease that receive traditional approval from the Food and Drug Administration (FDA) under coverage with evidence development (CED). CMS, as a part of this decision, will provide enhanced access and coverage for people with Medicare participating in CMS-approved studies, such as a data collection through routine clinical practice or registries. Registry data may be used to assess whether outcomes seen in carefully controlled clinical trials (e.g., FDA trials) are reproduced in the real-world and in a broader range of patients. Any new drugs in this class that receive FDA traditional approval may be available in additional care settings that people with Medicare can use, such as an outpatient department or an infusion center. Secondly, for drugs that FDA has not determined to have shown a clinical benefit (or that receive an accelerated FDA approval), Medicare will cover in the case of FDA or National Institutes of Health (NIH) approved trials. Under this NCD, CMS will support the FDA by covering the drug and any related services (including, in some cases, PET scans if required by trial protocol) for people with Medicare who are participating in these trials.
This decision is specific to individuals who have a clinical diagnosis of mild cognitive impairment (MCI) due to Alzheimer’s Disease or mild dementia with a confirmed presence of plaque on the brain. This is consistent with use outlined on the FDA label. Should the FDA update the label, CMS would reevaluate the coverage policy accordingly.
“Alzheimer’s disease is a highly destructive illness that affects millions of Americans and their families. CMS has a responsibility to ensure that people with Medicare have equitable and appropriate access to therapies that are reasonable and necessary for use in the Medicare population,” said CMS Administrator Chiquita Brooks-LaSure. “This final National Coverage Determination reflects CMS’ commitment to provide the American public with a transparent, trusted, evidence-based decision – without regard to cost - that is made only after a thorough analysis of public feedback. Through this decision, we are creating a pathway for people with Medicare to quickly access drugs the FDA determines have shown a clinical benefit and encourages manufacturers and trial administrators to ensure that the clinical trials recruit racially diverse participants.”
Over 6 million older Americans are believed to have Alzheimer’s Disease, and this prevalence is expected to rise to 14 million by 2060 barring effective interventions. Effective treatments are needed, and because of the early but promising evidence and the immense burden of this devastating disease on the Medicare population, CMS is finalizing Medicare coverage under CED to support rigorous studies approved by the FDA and NIH to help answer whether this class of drugs improves health outcomes for patients.
The FDA has an important role in making sure that what is on the market is safe and effective for all individuals. Currently, Aduhelm™ (aducanumab) is the only monoclonal antibody directed against amyloid that has been granted approval by the FDA through its accelerated program for the treatment of Alzheimer’s disease based on evidence that it may reduce plaque on the brain. FDA and the NIH have approved clinical trials for Aduhelm, which are ongoing, to continue studying the drug and determine whether there is a reasonable assurance of a clinical benefit. Under this NCD, CMS will support the FDA and NIH by covering the drug and any related services (including PET scans in some cases if required by trial protocol) for people with Medicare who are participating in these randomized controlled trials.
Importantly, if the FDA determines a clinical benefit for drugs within this class through its traditional approval program, CMS, as a part of this decision, will provide enhanced access and coverage for people with Medicare participating in CMS-approved studies, such as a study where an intervention is tested in a real care setting. Any new drugs in this class that receive FDA traditional approval will be available in a variety of care settings.
While drugs in this class that receive FDA traditional approval will have broader coverage in the Medicare program, CMS believes important questions still need to be answered to support people with Medicare, caregivers, and their referring and treating physicians to make informed, appropriate decisions about use of any drug in this particular class. People with Medicare, if they are enrolled in an CMS-approved study, will have access to the drug with a multidisciplinary team of dementia experts that have the expertise to provide optimal medical management. Under CED for drugs in this class that receive FDA traditional approval, CMS-approved studies could include a large registry-based study with a comparator, such as the results of the randomized control trials (RCTs) used for FDA approval. CED is the appropriate Medicare coverage pathway until CMS knows that specific monoclonal antibodies for the treatment of Alzheimer’s Disease demonstrate health outcomes while minimizing harm to individuals.
“Science, evidence, and stakeholder input led our team of career civil servants and clinicians through this national coverage determination process. There is the potential for promise with this treatment; however, there is not currently enough evidence of demonstrating improved health outcomes to say that it is reasonable and necessary for people with Medicare, which is a key consideration for CMS when making national coverage determinations,” said Dr. Lee Fleisher, CMS Chief Medical Officer and Director of the Center for Clinical Standards and Quality. “In arriving at this final decision, we looked at the very unique circumstances around this class of treatments and made a decision that weighed the potential for patient benefit against the significance of serious unknown factors that could lead to harm. If a drug in this class shows evidence of clinical benefit through the traditional FDA approval process, then CMS will provide broad access and ensure the results from the rigorous trials are generalizable for people with Medicare participating in a CMS-approved study, such as a registry. Lastly, we structured this decision to provide flexibility and assurance that CMS can respond quickly to providing coverage for any new drugs in this class when a clinical benefit is determined.”
CMS followed a long-standing process established by Congress in making NCDs, including a determination whether a medical item or service is reasonable and necessary for the diagnosis of and/or treatment of an illness or injury. CMS develops NCDs following this process, which uses all relevant published evidence and feedback received from stakeholders with multiple opportunities for public input.
CMS reviewed over 10,000 comments after proposing an initial NCD approach earlier this year. In addition, during the 2021 national coverage analysis public comment period, CMS held two national listening sessions, and reviewed public comments and more than 250 relevant peer-reviewed documents to help inform the initial proposed coverage determination. In response to the feedback the Agency heard, this final NCD creates a pathway for drugs in this class to be covered and for people with Medicare to quickly access these drugs upon determination of a clinical benefit through FDA traditional approval.
CMS will be monitoring future developments in this class of treatments and is committed to continuing to explore ways to improve care for people with Alzheimer’s disease.
For a fact sheet on Medicare coverage policy for monoclonal antibodies directed against amyloid for the treatment of Alzheimer’s disease, visit https://www.cms.gov/newsroom/fact-sheets/medicare-coverage-policy-monoclonal-antibodies-directed-against-amyloid-treatment-alzheimers-disease.
To read the final NCD CED decision memorandum, visit https://www.cms.gov/medicare-coverage-database/view/ncacal-decision-memo.aspx?proposed=N&ncaid=305.