The Centers for Medicare & Medicaid Services (CMS) released new details Thursday about how it plans to collect patient data as a condition of coverage for a new class of Alzheimer’s drugs, once the medications get full approval from the FDA.
CMS detailed a plan earlier this month to cover the costly drugs for patients enrolled in Medicare Part B who meet the criteria for coverage. That criteria includes patients' physicians and clinical teams participating in registries to collect evidence about how the drugs work in the real world.
The coverage plan will be likely put to the test with a potential approval for Biogen and Eisai's Leqembi slated for this summer.
A year ago, CMS issued a narrow and controversial Medicare coverage proposal for Biogen’s Alzheimer’s disease drug Aduhelm. The agency said drugs approved under the FDA's accelerated pathway will only be covered when administered in eligible clinical trials.
The new CMS registry approach is intended to make the drugs more widely available once they gain full FDA approvals.
The nationwide registry for clinicians, which will be available on CMS' website, will be free to use, according to the fact sheet issued Thursday.
Clinicians will submit information to this registry when furnishing this drug to people with Medicare. The “Monoclonal Antibodies Directed Against Amyloid for the Treatment of Alzheimer’s Disease Registry” will be available nationwide on CMS’s website if the FDA gives any of these drugs traditional approval, the agency said.
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"In facilitating the development of this registry, CMS is carefully balancing the need to collect information while keeping the registry as easy to use as possible," agency officials said.
CMS said this week it is also working with multiple organizations preparing to open their own registries. Once more registries are available, they will also be listed on the site and clinicians will be able to choose which registry to participate in, the agency said.
To receive reimbursement under the criteria for coverage, doctors will be required to submit demographic information on the provider and patient, the patient's diagnosis and whether the patient is taking any anticoagulation or antiplatelet drugs.
Doctors also will need to submit results of the patient's amyloid positron emission tomography (PET) scan, cerebrospinal fluid (CSF) test, or other amyloid test as well as any specific anti-amyloid monoclonal antibody being administered. CMS is requesting doctors also submit the results of tests of cognition and overall function that were used to diagnose and treat the individual with mild cognitive impairment or mild Alzheimer’s disease dementia.
CMS also wants to collect data on adverse events such as brain swelling or hemorrhages.
In the fact sheet, CMS said the data submission portal was designed with an easy-to-use format. Whenever possible, drop-down menus will be available. Clinicians furnishing this drug will have already gathered this information as part of routine clinical assessment and follow-up care for patients with mild cognitive impairment or mild Alzheimer’s disease dementia who are being evaluated for or treated with these medications, the agency said. The required elements may already be available to the clinician from the patient’s medical record.
Industry trade group PhRMA has pushed back on CMS' approach to covering the new class of Alzheimer's drugs. The organization believes the plan will “severely restrict” patient access and that the registry requirement will enact barriers to “potentially life-changing” treatments, a spokesperson said in an emailed statement to Fierce Pharma.
CMS maintains there is a “strong precedent” for using registries for newly approved treatments. In its own statement, the agency celebrated the approach as a way to "ensure availability" of the drugs.
But PhRMA argues that not all patients are able or willing to participate in a registry. Plus, the plan could force patients to travel to providers who are participating in the registry program, the trade group says.
Further, PhRMA flagged concerns that the registry might not be available when the FDA approval lands.
Patient advocacy groups also have expressed concerns with CMS' approach. Earlier this month, the Alzheimer's Association called the registries "an unnecessary barrier" and creates extra red tape for patients to get access to the drug.
Broader CMS reimbursement will be a tailwind for Eisai and Biogen but also Eli Lilly and earlier-stage players like Prothena among others in the anti-amyloid space given over 5 million Medicare beneficiaries suffer from Alzheimer's disease, noted William Blair analysts in a note issued earlier this month.
One outstanding question had been whether the details of a proposed registry would be restrictive to physicians who wanted to prescribe Leqembi and potentially donanemab.
"We view today’s CMS update as an incremental positive given the seemingly modest data requirements and anticipated minimal burden on physicians, who would likely have nearly all (if not all) the requested data readily available from patient records," William Blair analysts wrote in a note issued Thursday.
Biogen does not anticipate any additional cost on the part of the sponsor to support registry operation, the analysts noted. "The goal of the registry is to collect real-world outcome and risk/benefit data, and the requirement is unsurprising in our view given ongoing debate on overall clinical benefit and safety," they wrote.