The Agency Keeping Alzheimer’s Drugs from Patients

Editor’s note: This article was first published in the Wall Street Journal on May 5, 2023.

Another Alzheimer’s drug has yielded promising results, demonstrating a 35% decline in early-disease progression in a trial of 1,736 patients after 18 months. Donanemab, made by Eli Lilly, represents the strongest showing against Alzheimer’s to date. This success follows the Food and Drug Administration’s accelerated approval in January of lecanemab—a similar treatment from Biogen and Eisai—which showed a 27% decline in a trial of 1,795 patients after 18 months.

Yet the Centers for Medicare and Medicaid Services is denying seniors and their families access to these treatments, and rebuffing innovators who have produced the biggest breakthroughs in Alzheimer’s in two decades. Such regulatory overreach must stop.

If the FDA deems a treatment safe and effective, historically CMS has almost always determined it to be worthy of reimbursement under the “reasonable and necessary” standard—a nebulous term that gives the agency maximum leeway. The FDA and CMS have followed this path in granting broad access to innovative treatments for cancer, heart disease and HIV/AIDS.

But CMS has inexplicably bucked precedent with Alzheimer’s, questioning the FDA’s judgment on safety and efficacy and restricting coverage of any new medicines to CMS-approved clinical trials. Guess how many such trials are currently enrolling Alzheimer’s patients? None.

The policy CMS is wielding, known as the “Coverage With Evidence Development,” or CED, is an innovation killer. It was created during President George W. Bush’s administration to speed access to medical devices. The idea was to have CMS cover medical technologies on which research data was still being collected. In practice CED has become an interminable slog for companies, which often have to follow more than 30 complex steps to establish an evidence development program. This entails identifying study sponsors and investigators, establishing protocols, securing CMS approvals and funding, establishing databases, enrolling patients, and running clinical trials.

Since 2005, 27 medical devices or procedures have been subjected to CED, and only four have had their evidence development programs retired. One took 12 years to do so. Others are still required to generate clinical data today.

Positron emission tomography, or PET scans, used to diagnose certain cancers and neurological disorders, fell under CED 18 years ago. CMS loosened the policy in 2013 for cancers but continues to restrict coverage for neurological disorders, including Alzheimer’s, even though hospitals perform more than two million PET scans a year.

By subjecting Alzheimer’s treatments to CED, CMS has expanded the policy beyond devices and procedures. This move calls into question the FDA’s scientific judgment and will restrict, rather than expand, patients’ access to these treatments. There is no indication CMS will reverse course but bipartisan pressure on the agency is growing.

Democratic and Republican lawmakers hammered CMS Administrator Chiquita Brooks-LaSure on the policy before the House Energy and Commerce Committee’s Subcommittee on Health last week. The same day, 26 attorneys general from red and blue states sent Ms. Brooks-LaSure and Health and Human Services Secretary Xavier Becerra a letter urging them to expand access. Both groups highlighted the administration’s conflicting policy on the matter, noting that the Department of Veterans Affairs granted coverage for Eisai and Biogen’s treatment in February.

Alzheimer’s is a very risky bet for innovators. In the past quarter-century, more than 150 clinical trials have ended in failure. Despite billions of dollars of private investment, only six treatments have achieved FDA approval. The recent momentous advances are milestones toward treatments that can slow the disease and perhaps one day reverse it. Innovation must be rewarded—not suppressed.

In many ways, we are today with Alzheimer’s where we were with cancer 40 years ago. When President Richard Nixon signed the National Cancer Act of 1971, the five-year survival rate was below 50%. After decades of investment, supported by policies rewarding incremental innovation, the rate is approaching 70%. It is nearly 100% for breast, prostate and other cancers caught in the early stages. Similar progress is possible for Alzheimer’s—and the first step is getting FDA-approved treatments out of CED.

The Alzheimer’s Association estimates that, every day, 2,000 people transition from mild to moderate dementia, putting them out of reach for these breakthroughs. How many will miss out by the time CMS acts?

Mr. Grogan is a visiting senior fellow at the USC Schaeffer Center and served as domestic policy adviser to President Trump, 2019-20. He consults for the healthcare industry, including companies working to develop treatments for Alzheimer’s disease.

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