GAP President John Dwyer was quoted in the Washington Post on the CMS decision.
The Centers for Medicare and Medicaid Services said it would cover a class of Alzheimer’s drugs that includes Leqembi if the medicines get traditional approval by drug regulators. (Eisai/Reuters)
Medicare officials outlined plans Thursday to broadly cover a new class of Alzheimer’s drugs amid an intense lobbying campaign by patient advocates and drugmakers to ensure access to the first medications shown to slow cognitive decline from the disease.
In a statement, the Centers for Medicare and Medicaid Services, which runs the federal health program for seniors and people with disabilities, said the drugs must have traditional approval from the Food and Drug Administration — none of the medications has that now. And physicians must participate in registries designed to collect information on how the drugs work in the real world, as opposed to the tightly controlled environment of clinical trials. The plan was foreshadowed in previous agency remarks.
The approach disappointed some advocates who said it did not go far enough in guaranteeing that patients with early-stage disease — the group most likely to benefit — could access the medications. Some doctors, they said, might choose not to participate in registries. Other experts praised the plan, saying registries would provide much-needed information on how the therapies work.
The first drug in the class likely to receive traditional approval from the FDA is Leqembi, made by the pharmaceutical companies Eisai and Biogen. The medication, which the FDA approved on an accelerated basis in January, may get traditional approval as soon as July. A similar drug by Eli Lilly may be fully cleared by the end of the year.
For drugs cleared by the FDA on an accelerated basis, Medicare will stick with its policy of covering the medications only for patients enrolled in federally approved clinical trials. That is in sharp contrast to its coverage plan for medications that receive traditional approval.
Accelerated approvals are based on a biomarker or other sign suggesting the medication may help patients. Such approvals typically occur more quickly than traditional approvals, which require clinical evidence of a benefit.
The medications target amyloid plaque in the brain, a signature characteristic of Alzheimer’s, and are designed for people with mild cognitive impairment or early-stage dementia caused by the disease. Some doctors have greeted the drugs as a welcome advance after years of failure, but others say they raise serious safety and effectiveness concerns and are not ready for widespread use.
The Alzheimer’s Association criticized the CMS plan, saying in a statement that requiring a “registry as a condition of coverage is an unnecessary barrier.” While registries can be important tools to gather information about a drug,the association acknowledged, “we remain concerned that the requirement of clinicians to register and enter data will create unnecessary hurdles.”
Other experts said they don’t expect that patient registries will hinder the accessibility of the drug and support the approach from CMS.
The collection of information “will be critical for further analyses to be done, both by CMS to make sure that their beneficiaries are continuing to benefit from the drug in a meaningful way … and then also to collect safety information,” said Reshma Ramachandran, assistant professor of medicine at Yale School of Medicine.
Diana Zuckerman, president of the National Center for Health Research, a nonprofit think tank, agreed, saying such data will show “who is benefiting, how much and whether certain patients will be at such high risk — perhaps those taking bloodthinners, for example — that it is not worth the risk.”
But John Dwyer, president of the Global Alzheimer’s Platform Foundation, which works to make Alzheimer’s trials more efficient, said CMS should not place conditions on drugs that have been approved by the FDA. He described the agency’s approach as “half a loaf” for patients.
The news from CMS removed lingering uncertainty over how broadly Medicare would cover anti-amyloid drugs, a key focus of investors assessing how profitable they could be, with Wall Street analysts expecting that Leqembi will receive full approval. Biogen’s shares were up more than 3 percent in early trading Thursday.
“We believe broader CMS reimbursement will be a tailwind for Eisai and Biogen,” Myles Minter, a biotech analyst at William Blair, wrote in a research note Thursday, adding that Medicare’s approach could benefit other companiessuch as Eli Lilly and Prothena that are developing Alzheimer’s drugs targeting amyloid.
Clinical trials have shown that the drug, which is administered twice a month intravenously and priced at $26,500 a year, can modestly slow progression of early Alzheimer’s.
The announcement from CMS also shows that the agency has been working to lay the groundwork for a patient registry, so that the process could be ready as soon as FDA makes a decision on full approval, analysts at Cowen wrote. Laura Chico, an analyst for Wedbush, wrote Thursday that CMS’s position is “not terribly surprising” but that the patient registry could complicate the logistics for Leqembi’s launch.
Medicare said its approach would expand the number of patients eligible to receive fully approved drugs while allowing the collection of critical information that would be made available to researchers. The agencysaid doctors and their teams would be able to submit evidence through a CMS-facilitated portal and that it would also work with others in the private sector that are creating their own registries.
The data requirements of the agency will be simple and straightforward, according to a CMS official who spokeon the condition of anonymity before the plan was made public. Doctors and their staffs won’t be paid for the additional work of filing the information, the official said.
The new Alzheimer’s drugs have been at the center of a high-stakes struggle over how to handle the emergence of disease-modifying therapies that studies show provide some benefit in slowing progression of the disease. Critics say those benefits are insignificant and pale in the face of safety concerns such as potentially fatal bleeding in the brain. They also warn that widespread use of the drugs could imperil Medicare’s finances.
Alzheimer’s advocates counter that the drugs are the first glimmer of hope in treating a disease that robs patients of their memories and identities and devastates families and their finances. About 6.7 million people in the United States are diagnosed with Alzheimer’s, and the number is expected to rise sharply as the population ages, with the costs of care skyrocketing.
The new drug class is made up of monoclonal antibodies, which are proteins produced in a lab that can bind to certain molecules. Outside FDA experts — the agency’s Peripheral and Central Nervous System Advisory Committee — are expected to meet June 9 to discuss whether Leqembi should receive traditional approval. The FDA’s deadline for deciding that issue is July 6.
A similar drug made by Eli Lilly may get traditional approval from the FDA by the end of this year or early next year. A third medication, Aduhelm, received accelerated approval in June 2021 but failed in the marketplace amid controversy over contradictory data and resistance by physicians.
Groups such as the Alzheimer’s Association have pushed Medicare to cover the new drugs — including those cleared only on an expedited basis — saying that the FDA should be the final arbiter of safety and efficacy of drugs. They also note that cancer drugs cleared on an accelerated basis are routinely covered by Medicare.
But critics of the medications say the FDA has been far too quick to approve the new therapies and that Medicare is providing an important brake.
On Capitol Hill, lawmakers in both parties during the past year have pressed Medicare to consider broad coverage for the Alzheimer’s drugs.
In an April hearing, Rep. Anna G. Eshoo (Calif.) — the ranking Democrat on the House Energy and Commerce health subcommittee — pressed CMS Administrator Chiquita Brooks-LaSure on how a registry would be structured to allow for broader access to Leqembi. Eshoo quizzed the CMS chief on whether patients and physicians will be aware of the potential for broader coverage if an Alzheimer’s drug gets full FDA approval this summer, andsheexpressed concern the agency wasn’t ready.
“If doctors don’t know, patients don’t know, and Medicare doesn’t really seem to know what this registry entails, how are Medicare patients going to get the drug potentially beginning in July?” Eshoo asked. “That’s really the $64,000 question in my mind.”
Brooks-LaSure told Congress that her agency intended to publish details about the upcoming registry. But there were few in the statement issued Thursday.
The CMS statement said that physicians and their staffs would be able to submit data on the drugs through a CMS-facilitated portal and that several private organizations are preparing to open their own registries. The agency did not explain exactly how the two would work together and said more information would be released as they come online.
Registries have been used before to collect information about a drug or medical device, including for a transcatheter aortic valve replacement, CMS said.
CMS officials have said they base their coverage decisions on whether a drug is “reasonable and necessary” for treating a disease — and that the answer determines whether Medicare pays for the expensive drug.