Administrator Chiquita Brooks-LaSure
The Centers for Medicare and Medicaid Services
The Hubert H. Humphrey Building
200 Independence Ave., S.W.
Washington, DC 20001
May 3, 2022
Dear Administrator Brooks-LaSure:
We are writing to express our deep gratitude for protecting patients across the country with the recently finalized National Coverage Determination (NCD) for aducanumab and other similar monoclonal antibodies directed against amyloid confirmation for the treatment of Alzheimer’s disease.
We applaud your leadership in making this thoughtful coverage decision that will provide the necessary evidence of whether these therapies are truly safe and clinically effective for patients. We are hopeful that such a decision will move manufacturers to prove clinical benefit ahead of FDA approval. In addition, we are grateful for the standard this sets to encourage manufacturers to continue meaningful innovation in this area and overseeing agencies to rely on the clinical endpoints rather than real-world evidence or surrogate endpoints.
As you know, many patient and provider organizations raised significant concern over the reliance on alternative endpoints and assumed clinical benefit in the FDA’s original review of and Aduhelm™ (aducanumab) for the treatment of Alzheimer’s Disease. We are grateful for the consideration you clearly took when reviewing our comments. As a collective we care deeply about patients and their access to therapies that provide meaningful clinical benefit.
We fully agree with the statement in the NCD memo that this stronger definition will encourage innovation while protecting patients: “We believe by defining the criteria by which a forthcoming therapy for Alzheimer’s disease could be covered would actually facilitate innovation. The CMS final decision gives the public a clear definition of where the goal line is for what constitutes success in a trial (and thus what meets the standard for “reasonable and necessary” required by law). Medical innovation must include clinical trials that demonstrate benefit to patients. Conversely, covering a drug that has not been shown to be effective may incentivize production, marketing and sales of similarly ineffective drugs, at the cost of hard research to find ones that do provide a clinical benefit to patients.” Requiring manufacturers to produce evidence proving that a therapy is clinically beneficial and safe in truly representative trials will provide reassurance to clinicians and patients when prescribing or accessing the treatment. This decision is incredibly important in ensuring that patients are not prescribed therapies that are not only ineffective, but also unsafe under the assumption that surrogate endpoints are predictive of clinical benefit.
Furthermore, CMS’ decision to recognize “the disappointing lack of inclusion of underserved populations in past trials” is important to increasing the effectiveness of treatments to all patients. We are grateful that CMS is finalizing a study requirement that ensures future studies are reflective of the diversity of patients that represent the national population diagnosed with Alzheimer’s Disease.
This landmark decision sets an important precedent that our federal agencies care about patient safety. Thank you again for making this important decision to protect patients and providers around the country.
Doctors for America
National Center for Health Research
Our Bodies Ourselves
Salud y Fármacos
Treatment Action Group
USA Patient Network