CMS’s Devastating Blow to Seniors

Read Incubate Advisory Council member Gaurav Gupta's op-ed on the CMS's decision to cover Alzheimer's drugs.


By Gaurav Gupta

America’s biotech companies have spent a staggering $42.5 billion developing treatments for Alzheimer’s since 1995.

But they have little to show for their efforts. Despite conducting nearly 1,100 clinical trials enrolling 184,000 patients over that period, almost all those experimental drugs failed. The FDA approved just six treatments, a success rate of just 0.5%.

Thankfully, those long odds haven’t forced biotech companies to abandon the field — at least not yet. Firms have continued to invest money into Alzheimer’s research, even though these projects are statistically doomed to fail, because they know that if an experimental drug does prove effective, the returns — both for investors and society as a whole — would be enormous.

Worryingly, a recent proposal by the federal agency that oversees Medicare could fundamentally alter that risk calculus — and cause investment in Alzheimer’s research to plummet.

Earlier this month, the federal Centers for Medicare and Medicaid Services (CMS) issued a draft “national coverage determination” that, if finalized, would severely restrict how Medicare covers an entire class of promising Alzheimer’s treatments, including Aduhelm, an antibody drug targeting a protein called amyloid in the brain. The FDA granted accelerated approval to the new drug in June 2021, based on clinical trial data showing that it reduces amyloid plaques in Alzheimer’s patients.

The FDA has taken similar steps before. In fact, the nation’s top scientific and regulatory body created its accelerated approval pathway all the way back in 1992 to address “unmet needs for serious or life-threatening diseases.” This pathway allows regulators to approve medications whose clinical benefit has yet to be demonstrated directly in patients, so long as those drugs meet so-called surrogate endpoints that are predictive of clinical benefits.

CMS oversees insurance coverage for over 100 million Americans, including millions of Alzheimer’s patients. And by law, Medicare Part B must cover medicines that are “reasonable and necessary” to treat a patient, and Part B cannot decline to cover an FDA-approved drug because of its cost.

Yet CMS is proposing to deny coverage for Aduhelm and any future treatments targeting amyloid plaque, outside of a small subset of patients participating in clinical trials, because “health outcomes” have not been ascertained.

CMS’s claim is technically accurate — Aduhelm hasn’t yet been proven to stall the progression of Alzheimer’s.

But the claim is also tautological. The purpose of the accelerated approval process is to grant patients earlier access to medications that, based on initial data, are reasonably likely to yield positive health benefits.

CMS’s second-guessing is a shocking overreach by an administrative agency primarily staffed with bureaucrats. It seeks to elbow CMS into a fundamentally scientific, medical, and regulatory question that falls squarely within the purview of FDA — and in so doing, to overrule FDA’s own approval processes.

Particularly audacious is that the proposed CMS determination covers not just Aduhelm but the entire class of Alzheimer’s disease therapies that target amyloid protein. This sweeping decree effectively imposes a moratorium on scientific research and clinical trials targeting amyloid, a protein which is indisputably the core biological hallmark of Alzheimer’s disease. There is no justification for anyone at CMS to be engaging in an appraisal of such far-reaching magnitude.

The proposed CMS coverage decision is also noteworthy for its absence of basic scientific rigor. At no point does CMS state which specific outcomes would constitute a “clinically meaningful difference in decline in cognition and function.” This provides drug researchers — and the FDA itself, which CMS is presuming to instruct — with no guidance on how to proceed with the development of Alzheimer’s therapies to CMS’s satisfaction.

That will have a chilling effect on Alzheimer’s research. If biotech companies suddenly have to worry not just about inventing effective treatments — which was already extraordinarily difficult — but also whether CMS officials will cover drugs that the FDA has approved, many firms will decide the risk is simply too large to bear.

Biotech companies cannot operate or raise funding in an erratic regulatory environment. The prospect of CMS transforming itself into an alternative FDA would signal the beginning of the end for our current golden age of medical innovation.

Gaurav Gupta, a physician, is the founder of the biotechnology investment firm Ascendant BioCapital.

See article online HERE.