BrightFocus Statement on Draft Plan for Alzheimer’s Treatments

The following comments were submitted today to the U.S. Centers for Medicare & Medicaid Services by BrightFocus President and CEO Stacy Pagos Haller:

February 10, 2022

Chiquita Brooks-LaSure
Administrator
U.S. Centers for Medicare & Medicaid Services
7500 Security Boulevard
Baltimore MD 21244

Re: CAG000460N, Monoclonal Antibodies Directed Against Amyloid for the Treatment of Alzheimer’s Disease

Dear Administrator Brooks-LaSure:

On behalf of the BrightFocus Foundation, I am expressing our concern that the proposed National Coverage Determination (NCD) will greatly hamper ongoing and future Alzheimer’s research, creating a chilling effect delaying and disincentivizing badly-needed scientific innovation to better treat and ultimately prevent this currently terminal and progressive disease.

For context, BrightFocus is a premier private funder of research on diseases of mind and sight, currently leading a scientific portfolio of 260 projects around the world, a $60 million investment.

The draft NCD’s Coverage with Evidence Development (CED) declines coverage for this class of Alzheimer’s treatments outside of a clinical trial. BrightFocus recommends it not be applicable to potential therapies still under development and yet to be submitted to the Food and Drug Administration for approval for clinical use. The CED would prejudge research findings yet to be completed and shared with scientific and regulatory leaders.

Applying this CED to an entire class of monoclonal antibody treatments, both the one currently available and those still in development, would put the brakes on Alzheimer’s research by adding an additional layer of expensive, multi-year clinical trials, one whose design structure would drive down participation, add time, and perpetuate lingering, systemic inequities in our health care system. The draft plan would broadly and severely restrict access to the latest available treatments for patients and families desperately looking for any form of promising respite and relief.

The draft CED would require that these trials exclusively occur at major medical centers, meaning those facing economic and logistical barriers to transportation would be less likely to participate. Experience has shown that the absence of a diverse array of community-based trial sites further exacerbates the already glaring under-representation of racial and ethnic minorities in Alzheimer’s trials, as well as the inadequate infrastructure to change that. With Black Americans twice as likely and Latinx 1.5 times as likely as whites to get the disease, we must be fully inclusive in trial recruitment to ensure treatments are their most effective. We are supportive of similar concerns from the intellectual disabilities community that this population would be less likely to be included in these trials.

Moreover, an additional time-consuming clinical trial for all upcoming Alzheimer’s drugs in this class would further strain and add more competition for volunteers across the broader field of dementia research, which already struggles with trial recruitment. These challenges consistently put promising science at risk of withering on the vine; we need federal policies which encourage, not deter, people becoming a vital part of clinical research.

Our understanding is that under this proposed CED, volunteers would need to cover co-pays, plus the cost of any additional PET imaging beyond the initial one, all the while knowing that there is a 50 percent chance they will get the placebo in a trial of an already FDA-approved treatment. In our opinion, this would suppress interest in volunteering, putting the trial at risk for delays, or even cancellation, due to lack of public engagement.

We are concerned that as Alzheimer’s trials become longer and more costly, and with corporate investment at risk of eroding, the generative iteration of scientific discovery would be at risk. In science, progress begets more progress, as we are seeing with the early-mid stages of development for potential new monoclonal antibodies for the disease, where vascular side effects leading to ARIA can potentially be avoided by designing antibodies that target the correct amyloid-beta structure. We have seen across other disease states that first in class may not always end up best in class.

In conclusion, the proposed CED would create a chilling effect on Alzheimer’s research at the very time that our world’s older population is increasing so rapidly. It would add expensive, time-consuming, unnecessary barriers and further skew the findings of clinical trials in favor of those in certain socio-economic and geographic categories.

Persons living with Alzheimer’s are in a race against time, a race they will continue to lose unless we as a nation do everything we can to best support and accelerate the pace of scientific progress.

Thank you for your consideration of our views.

Sincerely,

Stacy Pagos Haller
President and CEO