The approach has disappointed some advocates who said it didn’t go far enough in ensuring that patients with early-stage disease, the group most likely to benefit, could access the drugs. Some doctors, they said, may choose not to participate in the registries. Other experts have praised the plan, saying the registries would provide much-needed insight into how the therapies work.
The first drug in the class likely to receive mainstream FDA approval is Leqembi, manufactured by pharmaceutical companies Eisai and Biogen. The drug, which the FDA approved on an accelerated basis in January, could gain mainstream approval as early as July. A similar drug from Eli Lilly could be completely phased out by the end of the year.
For drugs approved by the FDA on an accelerated basis, Medicare will comply with its drug coverage policy only for patients in federally approved clinical trials. This is in stark contrast to its coverage plan for drugs that receive regular or traditional approval.
Accelerated approvals are based on a biomarker or other sign that suggests the drug may help patients. Such approvals typically occur more quickly than traditional approvals, which require proof of actual clinical benefit.
The drugs target amyloid plaque in the brain, a hallmark feature of Alzheimer’s disease, and are designed for people with mild cognitive impairment or early-stage dementia caused by the disease. Some doctors have hailed the drugs as a welcome advance after years of failure, but others say they raise serious safety and efficacy issues and aren’t ready for widespread use.
The Alzheimer’s Association has 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 for gathering information about a drug, the association acknowledged, we remain concerned that the requirement for doctors to register and enter data creates unnecessary hurdles.
Other experts said they didn’t expect patient registries to hinder drug accessibility and support CMS’s approach.
Gathering the information will be critical for further analyzes to be done, both by CMS to make sure their beneficiaries continue to benefit from the drug in a meaningful way, and also to gather safety information, said Reshma Ramachandran, assistant professor of medicine at the Yale School of Medicine.
Diana Zuckerman, president of the National Center for Health Research, a nonprofit think tank, agrees, saying the data will show who will benefit, how much, and whether some patients will be at that high risk, perhaps those taking blood thinners. , for example that it is not worth the risk.
But John Dwyer, president of the Global Alzheimers Platform Foundation, which works to streamline Alzheimer’s drug trials, 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 of bread for patients.
The news from CMS has removed lingering uncertainty about how broadly Medicare will cover anti-amyloid drugs, a key focus of investors assessing how profitable they could be, with Wall Street analysts expecting Leqembi to receive full approval. Shares of Biogens rose more than 3% in early trading on Thursday.
We believe larger CMS reimbursement will benefit Eisai and Biogen, Myles Minter, a biotech analyst at William Blair, wrote in a research note Thursday, adding that Medicares’ approach could benefit other companies. such as Eli Lilly and Prothena who are developing Alzheimer’s drugs targeting amyloid.
Clinical trials have shown that the drug, which is given intravenously twice a month and is priced at $26,500 a year, can modestly slow the progression of early Alzheimer’s.
CMS’s announcement also shows that the agency has been working to lay the groundwork for a patient registry so that the process can be ready as soon as the FDA makes a decision on full approval, Cowen’s analysts wrote. Wedbush analyst Laura Chico wrote Thursday that CMS’s stance isn’t particularly surprising, but that the patient registry could complicate the logistics for Leqembis’ launch.
The Medicares coverage decision will apply to both the original program and private Medicare Advantage plans. Youth insurers often follow Medicare’s lead in deciding what to pay.
Medicare said its approach would expand the number of patients eligible to receive fully approved drugs while allowing for the collection of critical information that would be made available to researchers.
The agency’s data requirements will be simple and straightforward, according to a CMS official who spoke on condition of anonymity before the plan was made public. Doctors and their staff will not be paid for the extra work of filing information, the official said.
New Alzheimer’s drugs have been at the center of a high-stakes fight over how to manage the emergence of disease-modifying therapies that studies show provide some benefits in slowing the progression of the disease. Critics have said those benefits are insignificant and pale in comparison to safety concerns like potentially fatal bleeding into the brain. They also warn that widespread use of the drugs could endanger Medicares finances.
Alzheimer’s advocates argue that the drugs are the first glimmer of hope in treating a disease that robs patients of their memories and identities and wreaks havoc on families and their finances. An estimated 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 costs of care skyrocketing.
The new class of drugs consists of monoclonal antibodies, which are lab-made proteins that can bind to certain molecules. Outside of FDA experts, the agencies’ Central and Peripheral Nervous System Drugs Advisory Committee is expected to meet on June 9 to discuss whether Leqembi should receive traditional approval. The FDA deadline to decide this matter is July 6.
A similar drug made by Eli Lilly could get mainstream FDA approval later this year or early next year. A third drug, Aduhelm, received accelerated approval in June 2021 but failed to market due to controversy over conflicting data and resistance from doctors.
Groups like the Alzheimers Association have pushed Medicare to cover new drugs, including those approved only on an accelerated basis, saying the FDA should be the final arbiter of the drugs’ safety and efficacy. They also note that cancer drugs licensed on an accelerated basis are regularly covered by Medicare.
But drug critics say the FDA has been too quick to approve new therapies and that Medicare is providing a major drag.
On Capitol Hill, lawmakers from both parties have lobbied Medicare over the past year to consider broad coverage for Alzheimer’s drugs.
In an April hearing, Congresswoman Anna G. Eshoo (California), the Democratic representative on the House Commerce and Energy Health Subcommittee, pressed CMS Administrator Chiquita Brooks-LaSure on how a registry to allow wider access to Leqembi. Eshoo asked the head of CMS whether patients and doctors will be aware of the potential for broader coverage if an Alzheimer’s drug gets full FDA approval this summer, and She expressed concern about the agency was not 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 starting in July? Eshoo asked. That’s really the $64,000 question on my mind.
Brooks-LaSure told Congress his agency intended to publish details of the upcoming registry. But there were few of them in the statement released on Thursday.
The CMS statement said doctors and their staff will be able to submit drug data through a portal facilitated by CMS, and that several private organizations are preparing to open their own registries. The agency didn’t explain exactly how the two would work together and said more information would be released as soon as it was online.
The registries have previously been used to gather information about a drug or medical device, including for transcatheter aortic valve replacement, CMS said.
CMS officials said they base their coverage decisions on whether a drug is reasonable and necessary to treat a disease, and that the answer determines whether Medicare pays for the expensive drug.
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