Comment: Drug companies nearing breakthrough for Alzheimer’s

By Lisa Jarvis / Bloomberg Opinion

New data for Eli Lilly’s Alzheimer’s treatment, donanemab, is a big win for patients and their families after decades of disappointments. Perhaps as important, it will help overcome the field’s next challenge: getting insurers to pay for the drugs.

Lilly’s experimental drug works by clearing away the amyloid plaques known to coat the brains of people with Alzheimer’s. Monthly infusions slowed the relentless cognitive decline in Alzheimer’s by 35 percent during the 18-month study of people in the early stages of the disease.

In the past year, the field has surmounted a daunting hurdle: proving, after decades of failures, that clearing away amyloid can translate into clinical benefits. Both Lilly and Eisai have shown their drugs can at least modestly slow the loss of memory and daily functioning that so profoundly affects people with Alzheimer’s and their families.

Translating those benefits into the real world means convincing insurers, the final arbiters of treatment options, of their value.

The patient population in Alzheimer’s is huge. Some 6 million people in the U.S. have the disease, with about 2 million of those in the early stages, where they might benefit from these new treatments. So far, insurers have balked at paying for Alzheimer’s drugs, putting them out of reach for most.

In January, the Food and Drug Administration granted Eisai’s Leqembi conditional approval based on its ability to lower levels of amyloid plaque in the brains of people with Alzheimer’s. But the Centers for Medicare & Medicaid Services has said that it will only pay for Leqembi and other similar amyloid-busting drugs after they’ve received full FDA approval based on data showing they actually improve people’s lives.

The CMS decision severely limits the market for these drugs; Leqembi, for example, costs $26,500 a year, a price few seniors can afford to pay out of pocket.

In recent congressional testimony, CMS Administrator Chiquita Brooks-LaSure reiterated her stance that “when FDA fully approves drugs for Alzheimer’s disease, CMS will cover it more broadly.” Eisai has since come out with data showing the drug does indeed offer cognitive benefits, and is expected to gain full approval for Leqembi in July. Lilly on Wednesday said it is rushing its new data to the FDA and anticipates full approval for donanemab by the end of the year.

The companies got another boost last month when the U.S. Veterans Health Administration added Leqembi to the list of drugs it will cover.

The shifting winds should open access by insurers, but companies still face other barriers to getting their drugs to a wide swath of patients.

For Lilly and Eisai, the looming question is how neurologists will approach prescribing these drugs. Lilly’s data, which came in a press release not a peer-reviewed publication, suggests its drug is more effective than Leqembi at preserving cognitive function. But those benefits also seem to come with more frequent serious side effects. Nearly a quarter of patients taking donanemab in Lilly’s trial experienced brain swelling that is common to these drugs, and about a third had small bleeds in the brain. Those side effects, which are also seen with Leqembi, are manageable for most people, but were directly linked to two deaths in Lilly’s trial; a risk that will require careful conversations between physicians, patients and caregivers.

There is also a question of how quickly the U.S. health-care system can meet demand for the drugs. That’s because of the onerous process of diagnosing and administering these treatments, a situation that’s already prompted neurologists in the United Kingdom to warn that they don’t have the infrastructure required to serve the many people who will want access to new drugs.

Currently, patients need an expensive PET scan or spinal tap that shows amyloid build up in their brain before they can be prescribed Leqembi. Although many groups are working on blood tests for diagnosing Alzheimer’s disease, which could significantly ease the diagnostic burden, they are not expected to be accurate enough to replace brain scans or spinal taps in the near term. And both drugs are given intravenously – Leqembi once every two weeks and donanemab once a month – which could create another challenge for the health-care system given the sheer numbers of people who might want to try the treatments.

Lilly’s data is unquestionably good news for people with Alzheimer’s disease and their families. Let’s hope the other barriers to access don’t prevent these breakthroughs from making a difference in the lives of patients.

Lisa Jarvis is a Bloomberg Opinion columnist covering biotech, health care and the pharmaceutical industry. Previously, she was executive editor of Chemical & Engineering News.