Washington, D.C. (February 9, 2022) – More than 50 patient advocacy groups urged Health and Human Services Secretary Xavier Becerra to direct the Centers for Medicare and Medicaid Services (CMS) to revise its draft National Coverage Determination (NCD) to ensure patients with serious and life-threatening conditions have access to novel treatments now and in the future. The letter was also submitted as part of CMS’ public comments on the draft decision.  

The letter was signed by leading patient advocacy organizations representing millions of patients and caregivers living with different acute, chronic, common, and rare diseases, including cancer, HIV/AIDS, and muscular dystrophy. 

The patient advocacy groups warned that the CMS-proposed National Coverage Determination would have implications beyond Alzheimer’s disease. This proposed CMS coverage plan raises concerns, the groups wrote, because it “sets a precedent that could have far-ranging and damaging impacts on approval and access to new therapeutics across all disease areas.”

The full text of the letter is below.

Comment Letter from Patient Advocacy Groups

Centers for Medicare and Medicaid Services’ Proposed Decision Memo: Monoclonal Antibodies Directed Against Amyloid for the Treatment of Alzheimer’s Disease  

February 9, 2022

Dear Secretary Becerra:

We, the undersigned, write to express our concern that the recent proposed National Coverage Determination (NCD) on anti-amyloid monoclonal antibodies (mAbs) sets a precedent that could have far-ranging and damaging impacts on approval and access to new therapeutics across all disease areas. The draft NCD creates the potential for a new paradigm in which U.S. Food and Drug Administration (FDA) approval is not sufficient for access. It suggests that drug approvals, whether based on Accelerated Approval or on traditional approval to market, will be second-guessed by the Centers for Medicare and Medicaid Services (CMS) and made subject to a new set of requirements that may preclude patients from having access to an FDA-approved product for which they qualify.

The draft NCD sends a signal that the CMS can effectively set aside the FDA’s approval of a new therapy and require a duplicative system for its own re-evaluation of the evidence upon which an approval is based.  This proposed approach disregards the primacy of the FDA’s jurisdiction as therapy regulator, its critical role in overseeing trial design (endpoint selection, methodology, target product profile, and biomarker selection, to name a few areas), and its benefit/risk determination based on significant scientific expertise in the relevant disease state. The unfortunate effect of the decision will be to prevent access for patients who meet the label criteria, especially those from racially, ethnically, or geographically diverse populations that have disproportionate challenges to accessing clinical trials. It creates a system that will thwart the very goal for diversity espoused by the agency. 

We urge you to consider the implications of this draft decision. The NCD raises a number of questions that stand unanswered, including:  

1. Does this determination create a paradigm where FDA approval is not sufficient for patient access? While CMS states that, “Generally, an intervention is not reasonable and necessary if its risks outweigh its benefits,”[1] based on the proposed NCD, we are left unclear whether the converse remains the standard. Specifically, when the benefits outweigh the risks, as determined by the FDA, should this not be tantamount to a finding that the intervention is reasonable and necessary?
    
2. What is the role of Coverage with Evidence development? As stated by CMS, is it a tool that can be a bridge for patient access while additional evidence is generated?  Further CMS’s own guidance provides, “CED will not duplicate or replace the FDA’s authority in assuring the safety, efficacy, and security of drugs, biological products, and devices.”[2]  How does this decision abide by and advance these principles for a CED? 

The significant urgency, unmet need, and public health impact of our communities is matched by the potential for scientific discoveries that will transform health outcomes for millions of Americans. We must protect and preserve the processes enabling this progress, while also working towards collaborative solutions to address identified gaps in evidence. 

On behalf of millions of patients and caregivers living with different acute, chronic, common, and rare diseases, we ask you exercise the overarching jurisdictional authority inherent in your office as Secretary of Health and Human Services (HHS) to:

• Take steps to address the sweeping, negative impacts of this draft decision;
• Guide CMS and FDA to work in collaboration, not contradiction; and
• Direct CMS to issue a substantially revised NCD that:
  • Respects and relies upon FDA’s review authorities; and
  • Assures coverage for beneficiaries who meet the FDA label requirements coupled with a robust post-market evidence generation strategy from various sources including the sponsors’ post-market commitments that will deliver real-time, longitudinal data on the safety and efficacy profile across diverse populations for each drug in this class of disease-modifying therapeutics as these may come to market.

 

We are anxious to work with you toward these important and urgent goals. 

Sincerely,

Adult Polyglucosan Body Disease Research Foundation
Advocacy & Awareness for Immune Disorders Association (AAIDA)
Alliance for Aging Research
Alpha-1 Foundation
ALS Association
American Behcet’s Disease Association
Amyloidosis Research Consortium
Angelman Syndrome Foundation
Avery’s Hope
The Balm in Gilead, Inc.
Batten Disease Support and Research Association
BrightFocus Foundation
Cancer Support Community
Caregiver Action Network
Caring Ambassadors Program, Inc.
Color of Crohn’s and Chronic Illness
Congenital Adrenal Hyperplasia Research, Education & Support Foundation (DBA: CARES Foundation)
Cure GM1 Foundation
Cure Mito Foundation
Cure Sanfilippo Foundation
Cure SMA
Dusty Joy Foundation
EveryLife Foundation for Rare Diseases
Fabry Support and Information Group
Friends of Cancer Research
The Global Foundation of Peroxisomal Disorders
Global Liver Institute
Haystack Project
HealthyWomen
Histiocytosis Association
HIV + Hepatitis Policy Institute
HypoPara Support & Advocacy, Inc.
International Pemphigus Pemphigoid Foundation
Little Hercules Foundation
LUNGevity Foundation
Marilyn’s Legacy
MarylandRARE
MEPAN Foundation
Mission: Cure
National Fabry Disease Foundation
National PKU Alliance
NTM Info & Research
One Rare
The Oral Cancer Foundation
Parent Project Muscular Dystrophy
Partnership to Fight Chronic Disease
Pompe Alliance
Powerful Patient, Inc.
Sarcoidosis of Long Island
Sudden Arrhythmia Death Syndromes (SADS) Foundation
SynGAP Research Fund (SRF) 501(c)(3)
Texas Rare Alliance
Undiagnosed Diseases Network Foundation (UNDF)
UsAgainstAlzheimer’s
Voices of Alzheimer’s

 

[1] Centers for Medicare and Medicaid Services. Proposed Decision Memo: Monoclonal Antibodies Directed Against Amyloid for the Treatment of Alzheimer’s Disease. January 11, 2022.

[2] https://www.cms.gov/medicare-coverage-database/view/medicare-coverage-document.aspx?MCDId=27

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UsAgainstAlzheimer’s exists to conquer Alzheimer’s disease. We take on the toughest problems; bring all of “Us” together to break down barriers; advocate for research that will speed treatments to market; and drive changes that matter most to people living with the disease. We will not rest until brain-span equals lifespan - for everyone.