Alzheimer's drug approval unlikely to ease tension over coverage

An ongoing fight among Medicare officials, Alzheimer's patient groups and lawmakers over coverage of a new Alzheimer's drug is not likely to abate, despite it getting the green light from federal health officials.

The Food and Drug Administration on Thursday granted full approval to the drug, called Leqembi, based on clinical trial data showing it slowed cognitive decline by about 27 percent compared to a placebo. 

Leqembi, developed by Eisai and Biogen, is the first drug approved that can slow the course of Alzheimer's disease. FDA gave it fast-track approval in January, allowing manufacturers to launch the treatment based on preliminary evidence.

FDA's full approval also triggered expanded Medicare coverage for patients, but with a major caveat.

Specifically, the Centers for Medicare and Medicaid Services will require patients and clinicians to participate in a registry, which will collect evidence about how the drug works in the real world. 

Medicare's requirement for a registry applies to all so-called anti-amyloid drugs "that may slow the progression of Alzheimer’s disease." No other FDA-approved medication has that type of requirement.

Patient advocacy and industry groups have been lobbying intensely for no coverage limits and have the support of bipartisan lawmakers. They were incensed by the decision and claim it will be a barrier to widespread treatment access. 

Leqembi is just the second drug in its class, and there are more on the horizon. Advocates want Medicare to change its policy before any others get approved.

"I can't see how they would justify letting a policy stay in place that's based on such an outdated read of the evidence, based on what we have today," said Robert Egge, chief public policy officer and executive vice president of public affairs at the Alzheimer's Association.

In a statement Thursday,  Alzheimer's Association leaders said they felt CMS listened to them by designing a "low touch" registry to be as easy to use as possible. 

But Egge said he was disappointed the agency did not start a formal process to reconsider the requirement altogether.

"This reconsideration is extremely important and long overdue. We will continue to work with the administration and Congress to ensure the initiation of this process,” Egge said in a statement.

Congress has also been pushing for broad Medicare coverage.

House Energy and Commerce Chair Cathy McMorris Rodgers (R-Wash.) said in a statement on Wednesday, ahead of FDA's decision, that CMS should already be covering Alzheimer's treatments that have been cleared under the FDA's accelerated approval process. 

Once the agency grants traditional approval, "there will be no excuses for CMS to effectively deny coverage to Americans in need," McMorris Rodgers said. 

Rep. Anna Eshoo (D-Calif.), the ranking Democrat on the Energy and Commerce health subcommittee, on Thursday said it was "essential for Medicare to provide the broad coverage it has promised for Alzheimer’s treatments that receive traditional approval. I will be watching closely to make sure that promise is delivered. Families and patients deserve access to the life-changing drug without unnecessary burdens on patients or doctors.”

The concern is not merely hypothetical. 

Eli Lilly in May released initial results from a clinical trial of its new Alzheimer's antibody treatment that were comparable to Leqembi, and the FDA could approve it by the end of the year. 

Jason Karlawish, a co-director of the University of Pennsylvania’s Penn Memory Center, said he thinks the U.S. health system is unprepared for these new drugs. Fighting over access, he said, misses the point.

"There's been this rhetoric around the need for wide access to the drug that kind of simply misses the fact that the issue is rational access to a drug," Karlawish said. "It's got benefits, it's got risks. It's complicated and it's landing in a health care system that's not ready for it."

But others said a registry requirement doesn't make sense, especially for future drugs. 

Dennis Selkoe, co-director of the Center for Neurologic Diseases at Brigham and Women's Hospital in Boston, who has consulted with Eisai, said having a registry requirement for only Alzheimer's drugs could send a message to patients that regulators don't think the drugs work.

"You ask yourself ... what was happening here? Why is this so sensitive that it needs a registry of all patients? Because the implication could be, we don't think this stuff really works. We're worried that it might not be useful. And of course, that will give pause to patients," Selkoe said. 

"Many of us assume that Medicare cannot say what they really are concerned about, which is the total cost of an extensive therapeutic for arguably the most common brain disease in humanity," Selkoe said.

Leqembi is expensive; it costs $26,500 a year, and experts said widespread uptake could significantly increase Medicare premiums. Leqembi is an infusion drug administered by a physician, so it is covered under Medicare Part B.

About 6.7 million people in the United States are diagnosed with Alzheimer’s, and that number is expected to rise as the population ages. There about 1.5 million people estimated to be in the early stages of Alzheimer's. 

Drugmaker Eisai has said it expects only about 100,000 patients will be using the drug in its first three years on the market.

"Even if that relatively conservative number turns out to be correct, that's still $2.7 billion in annual spending for this drug. If the take up rate is higher, the numbers obviously are going to be greater," said Tricia Neuman, senior vice president of the health research group KFF and executive director of its Medicare policy program.

If just 5 percent of people with Alzheimer's disease take the drug, the cost would be closer to $9 billion in additional spending annually for Medicare— roughly equal to spending on the top three Part B drugs combined in 2021, Neuman said.

Some Medicare patients could be also responsible for more than $5,000 per year in out-of-pocket copay costs for the drug.  

There are also serious safety concerns with Leqembi and Eli Lilly's drug, called donanemab.

If Medicare requires reporting of the side effects, that can help inform the development and trials of other drugs in the future.

FDA's approval of Leqembi came with a black box warning on the drug’s label, stating that in rare cases it can cause “serious and life-threatening events” and that there have been cases of brain bleeding, “some of which have been fatal."

Three people died during the primary trial, and about 13 percent of all participants who took Leqembi showed potentially serious brain bleeding and swelling. 

"I think given the controversies, given the costs, given the side effects, it's important that we keep collecting data, and I think it's reasonable to require [a registry]," said University of Kansas Medical Center neurologist Jeffrey Burns.

"It will on some level exacerbate access issues, but ... I don't think the registry will be necessary forever. But right now, I think it's reasonable," Burns said.