KENNETH E. THORPE: Why is Medicare rationing Alzheimer’s drugs?

The FDA recently approved exciting new treatments that can slow the progression of Alzheimer’s disease, a scourge that claims more American lives than breast cancer and prostate cancer combined.

One such medicine, Leqembi, reduced early-stage Alzheimer’s patients’ cognitive decline by 27% in clinical trials — a breakthrough that offers hope to millions of Americans with degenerative dementia. Other treatments designed along the same lines are also in the pipeline.

The question, though, is whether Medicare will allow access to these and other game-changing medications through seniors’ prescription drug coverage. For Leqembi, the answer as of July is yes — but it’s complicated. And the complications should be ringing alarm bells for seniors nationwide.

Medicare initially balked at providing coverage for Leqembi and similar Alzheimer’s treatments. The Centers for Medicare & Medicaid Services, which administers the Medicare program, imposed unprecedented restrictions on Leqembi and other monoclonal antibody therapies by applying a tool known as “coverage with evidence development.” Medicare would cover such drugs only for those participating in clinical studies carrying out further assessments of their effectiveness.

In a nutshell, Medicare officials decided unilaterally that FDA approval wasn’t good enough. Without the expertise or even necessarily the authority to do so, CMS demanded additional testing for monoclonal antibody treatments for Alzheimer’s before covering them without requiring patients to join a study.

The first FDA approvals for these treatments came under the FDA’s “accelerated approval” pathway, which is designed to get critical medicines to patients as soon as safely possible. Under FDA’s traditional approval process, the agency requires demonstration of a treatment’s clinical benefit — a measured improvement in such areas as survival rates or quality of patient life. Under accelerated approval, FDA gives the go-ahead on the basis of measured “surrogate endpoints” — outcomes that predict a clinical benefit that has yet to be shown.

Leqembi received accelerated approval in January this year based on its ability to clear the brain of plaques associated with Alzheimer’s disease. In theory, this should have allowed hundreds of thousands of Medicare patients access to a medicine that could substantially slow the progress of their Alzheimer’s.

It didn’t. CMS effectively refused to cover the medicine by requiring participation in agency-approved clinical trials or an evidence-gathering data registry as a condition of coverage — neither of which existed at the time. This meant that anyone seeking this novel therapy would need to forgo Medicare coverage and face an annual estimated cash price of $26,500 — effectively reserving this breakthrough treatment for the wealthy.

The CMS actions suggest that the agency was working to avoid having the government pay for the medication. But doing so violated a core Medicare tenet of equal access to FDA-approved medications, setting a dangerous precedent.

Leqembi went on to gain traditional approval in July. CMS now says it will provide coverage as long as a patient’s prescribing clinician participates in an easily accessible national registry. That’s a big improvement — but it still leaves troubling precedents in place and restricts coverage to people willing to share their data with a registry.

The FDA has long served as the standard of drug approval globally. Agencies such as CMS, which serves as an insurer and payment administrator, lack the expertise and some have argued the legal authority to so condition Medicare coverage on additional clinical studies or evidence development requirements.

Worse, delaying or denying access to medications like Leqembi is incredibly short-sighted. The Alzheimer’s Association estimates that Alzheimer’s and other dementias will cost the United States more than $345 billion in 2023, with Medicare and Medicaid covering much of those costs. Without adequate treatments, such expenditures will balloon to nearly $1 trillion annually by 2050.

These figures are certainly understated, as they do not encompass the productivity nor tax revenue losses from those exiting the work force prematurely, nor the uncompensated care provided by loved ones — an estimated loss in economic value north of $339 billion in 2022 alone.

Of course, all this discussion of cost distracts from what really matters: the quality of life of more than 6 million patients suffering from this cruel disease. Leqembi is designed to treat patients in the early stages of Alzheimer’s. Yet every day during the six months that separated its accelerated and traditional FDA approvals, more than 2,000 people with Alzheimer’s progressed past the point at which Leqembi could have slowed their cognitive decline. These individuals and their families have lost out permanently, as will those who can benefit from future breakthrough Alzheimer’s therapies.

President Biden pledged to reduce the “heartbreaking human toll” of Alzheimer’s. We are on the cusp of doing so — provided officials at CMS stop trying to ration treatments.