CMS decision not to cover Alzheimer’s treatment a bad one

NCBIO stands in solidarity with patient advocates and industry leaders in opposing the Centers for Medicare and Medicaid Services’ draft proposal to restrict coverage for current and future Alzheimer's drugs targeting beta amyloid, a known hallmark of the disease. 

Restricting coverage is a bad idea for so many reasons: 

• CMS is competing directly with the FDA as the arbiter of what medicines will be available to Americans. 
• CMS doesn’t want to cover any new drug in this class unless the patients getting the drugs are enrolled in clinical trials approved by CMS. This means that CMS has already made coverage decisions for drugs that don’t exist yet.  

• This CMS decision would make these drugs available only to patients who can travel to the site of a clinical trial, who have the time and resources needed to participate in a clinical trial and who meet the requirements of the trial. Additionally, patients in the trial’s control group get a placebo, not the real medicine, further limiting access. 
• CMS is increasing the uncertainty and risk for biotech companies and investors, which will have a chilling effect on investment. A 2021 analysis shows that an investment of $5.2 billion is needed for a company to have a 50/50 chance of getting a new advanced therapy to market. 
• Increasing the risk associated with developing new therapies will further increase their ultimate cost to the patient.    

The Back Story 

On Jan. 11, 2022, the Centers for Medicare & Medicaid Services issued a proposed decision memo in its National Coverage Analysis for Monoclonal Antibodies Directed against Amyloid for the Treatment of Alzheimer’s Disease (Proposed National Coverage Determination). 

To date, only one drug in this class has been approved by the FDA: Biogen’s Aduhelm, the first treatment for Alzheimer’s that actually targets the disease instead of just treating symptoms. The FDA granted accelerated approval for Aduhelm, but CMS only wants to cover the drug and future drugs in the same class for Medicare patients participating in government approved randomized clinical trials. 

Bad for patients 

CMS’s proposal to limit coverage only to patients enrolled in government-approved trials means that the vast majority of individuals suffering from Alzheimer’s will have no meaningful access to an approved medicine simply because of where they live and where they go for care. CMS is imposing new and stringent clinical trial criteria which strongly suggest that the number of clinical trials approved will be extremely limited. 

Alzheimer’s is a degenerative medical condition; each year an AD patient goes without treatment means the potential for permanent and progressive loss of cognitive ability. Patients with AD cannot afford to wait a year or more for CMS-approved clinical trials to be available. Even when approved trials are initiated, patients will still face the risk of being placed on a placebo, an untenable situation for a patient when the FDA has determined the drug’s safety and efficacy. 

Undermining the FDA 

The current proposal undermines FDA’s Accelerated Approval pathway. This pathway enables therapies for serious, life limiting conditions to be approved and available to patients more quickly and relies on scientifically validated surrogate endpoints. Accelerated approvals require a commitment from the manufacturer to conduct a confirmatory Phase 4 study. 

Chilling innovation 

The risk of CED restrictions would make it untenable for developers of novel therapies to pursue the FDA Accelerated Approval pathway. Developing a new drug requires billions of dollars in research and investment. The investment required is exponentially higher for difficult to treat medical conditions like Alzheimer’s.  

To promote research in difficult-to-treat conditions like Alzheimer’s, manufacturers and investors need certainty that CMS will cover drugs for on-label use once the FDA has determined the therapy to be safe and effective. CMS’s proposed national coverage determination threatens to chill research and investment in new drugs targeting challenging diseases.  It will also reduce the appeal of FDA Accelerated Approval, a key mechanism that patients rely on to get faster access to desperately needed new therapies. 

A new study from Vital Transformation found that the CMS decision could add three or more years to the process of approving new Alzheimer’s therapies for coverage. This delay would have a profound impact on the financial viability of developing and commercializing those treatments successfully. According to the VT model, a three-year delay has the potential to reduce the number of therapies that will provide a positive return-on-investment by more than 80%. 

• Of Alzheimer’s programs currently in development – if the proposed NCD was in place at the time of program initiation — 93% of investments would have had negative return on investment and therefore would not likely have been made. 
• Furthermore, the research found that many existing clinical development programs would likely be halted. This is not only true for amyloid-targeting therapies, but all Alzheimer’s treatments, as neurological disorders often use the same endpoint threshold when applying for CMS program participation. 
• The CMS national coverage decision, if implemented, reduces an estimated 39 treatments with a net positive ROI to just 3, with an assumed three-year delay. With a four-year delay, only one therapy with a positive ROI emerges in the VT model. 

CMS should be encouraging development of new and innovative treatments for conditions like Alzheimer’s, not adopting restrictions on coverage that threaten to chill the development of an entire class of therapies for a degenerative condition that has no viable treatment option. Ultimately, we believe any national coverage policy should help ensure consistent patient access for vulnerable patients – not introduce inappropriate barriers to care. NCBIO joins the Council of State Bioscience Associations in asking CMS to not implement this draft proposal.