CMS is coming under increasing pressure to modify its proposed national coverage decision for anti-amyloid mAbs, including Biogen’s Aduhelm. Congressional attention could be reflected in legislation coupled with reauthorization of FDA medical product user fees.
In separate letters released Wednesday, Republican members of the House of Representatives and patient advocacy groups suggested that in proposing to restrict coverage of Aduhelm aducanumab-avwa from Biogen Inc. (NASDAQ:BIIB), CMS usurped FDA’s authority.
The letters urged the two agencies to collaborate more closely on approval and coverage decisions.
Fifty-five patient advocacy groups asserted that CMS’s proposal “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.”
Former FDA Commissioner Scott Gottlieb made similar points in an interview with The BioCentury Show, arguing that the proposed coverage decision set a precedent for CMS to deny coverage to drugs because they have received accelerated approval and that it blurred lines of authority between the Medicare agency and FDA.
Some former CMS officials, however, have applauded the proposed coverage decision, arguing that it offsets an accelerated approval decision that they believe should not have been granted. Many members of the medical community, including those in the American Geriatrics Society, feel the same.
The patient advocates pointed to confusion over the difference between the FDA’s approval and CMS’s coverage standards. “When the benefits outweigh the risks, as determined by the FDA,” they asked, “should this not be tantamount to a finding that the intervention is reasonable and necessary?”
The advocates’ letter, requested that HHS Secretary Xavier Becerra, “guide CMS and FDA to work in collaboration, not contradiction.” It also requested that he direct CMS to issue a revised NCD that “respects and relies upon FDA’s review authorities” and provides coverage for Medicare beneficiaries who meet the requirements in the labels for Aduhelm and future approved Alzheimer’s mAbs.
Organizations signing the letter include the ALS Association, EveryLife Foundation for Rare Diseases, Friends of Cancer Research, and UsAgainstAlzheimer’s. These groups and most of the others that joined them on the letter receive funding from the biopharmaceutical industry and other sources.
The congressional letter, signed by 72 Republicans — including Reps. Cathy McMorris Rodgers (Wash.), Kevin Brady (Texas), ranking Republicans on the Energy and Commerce and Ways and Means committees, respectively — also highlighted confusion over the two agencies’ standards. “Patient advocates, manufacturers, and other stakeholders have expressed growing confusion about the meaning of the respective FDA ‘safe and effective’ definition and the CMS ‘reasonable and necessary’ definition and how CMS has unilaterally added unpredictable, subjective criteria into the definition of ‘reasonable and necessary.’”
The lawmakers suggested CMS may have overstepped its role. “Preventing potentially millions of seniors suffering from a deadly disease from accessing an FDA-approved treatment without a reasonable policy and explanation will send mixed signals about the respective agencies’ roles and sow greater confusion.”
The letter poses questions to CMS about its consultation with FDA, a topic that could be included in user fee reauthorization legislation that must be enacted this fiscal year to avoid catastrophic disruption of medical product review and oversight. “Did CMS have the opportunity — and did the Agency avail itself of that opportunity — to work directly with FDA and evaluate all relevant data, not just data that is ‘publicly available’?” The letter also asks of CMS “itself of the expertise of the FDA, which Congress has determined should make decisions regarding whether prescription drugs should be made available to the American public based on their safety and efficacy?”
Republicans also criticized CMS for excluding many patients with intellectual and developmental disabilities, especially those with Down syndrome, from receiving Aduhelm in the trials the agency has proposed to cover. The letter notes research indicating that “there appears to be a correlation between the additional 21st chromosome present in people with Down Syndrome and the chromosome’s gene that makes amyloid precursor proteins and can cause a build-up of the beta-amyloid plaques common amongst those with AD.”
Speaking at a Feb. 3 hearing on user fee reauthorization, Rodgers highlighted the exclusion of Down syndrome patients from the proposed NCD. She also called for closer communication and collaboration between FDA and CMS.
In addition to attacking the proposed Alzheimer’s mAb coverage decision, the congressional letter criticized the Biden administration’s decision to repeal a rule that would have required Medicare to cover devices that received breakthrough designation. In contrast to drugs, which CMS usually covers for FDA-approved indications, device coverage is more often restricted or denied. The rule, finalized in the Trump administration, would have given device manufacturers a blank check for up to four years.
At least two democratic members of Congress have come out in support of CMS’s decision. Reps. Carolyn Maloney (D-N.Y.) and Frank Pallone (D-N.J.) issued a statement shortly after the draft NCD was released saying they had sent a letter to CMS Administrator Chiquita Brooks-LaSure supporting the proposed NCD. Pallone, who chairs the Energy & Commerce Committee, and Maloney, who chairs Oversight & Reform, have been investigating the Aduhelm approval, including potential inappropriate interactions between Biogen and FDA.