Eisai warned that mandatory data collection by U.S. health authorities could limit patients’ access to the Japanese drugmaker’s new Alzheimer’s treatment.
The decision by the Centers for Medicare and Medicaid Services to limit financial support for Leqembi to Alzheimer’s patients whose physicians participate in the health agency’s database could have “unintended consequences,” the company said.
The database, also known as a registry, will collect evidence about how the drug works in practice and make it available to researchers conducting studies on Leqembi and similar drugs, CMS said.
It marks the first time that Medicare, the U.S. government’s health plan for people over 65, has enforced such a data collection requirement for a drug that regulators deem safe and effective.
Eisai said it was concerned that the registry might hinder coverage of some populations who do not want to participate and raised questions about fairness. The company said it would wait to see details of the registration before finalizing a spending plan to support the drug’s launch.
“There is a difference between gathering real-world evidence to learn more (about a drug) and forcing reimbursement based on participation in real-world data collection,” Eisai US CEO Ivan Cheung said in an interview.
“Some people don’t want to participate for some reason. It’s a free country, right?”
Leqembi, jointly developed by Eisai and US biotech company Biogen, is expected to be the first Alzheimer’s drug capable of delaying disease progression to receive traditional approval from the US Food and Drug Administration.
This month, an outside panel of FDA experts recommended approval of the drug, a decision that typically prompts Medicare to cover the treatment for all patients over the age of 65. Reimbursement from the government health plan is critical to improving patients’ access to Leqembi, which costs $26,500 a year for a disease that primarily affects older adults.
But CMS is taking a cautious approach to a new generation of Alzheimer’s drugs that slow the progression of the disease by reducing the buildup of sticky amyloid plaques in the brain.
Last year, the agency imposed tighter restrictions on Biogen’s Aduhelm and other amyloid-reducing drugs, which received the green light in a fast-track process known as accelerated approval.
The decision limited reimbursement for patients enrolled in clinical trials of the drug and caused Biogen to abandon the launch of Aduhelm. At the time CMS cited the drug’s “potential harms,” ranging from headaches to brain bleeds, as well as concerns about its effectiveness in patients.
Eisai, which received accelerated approval for Leqembi in January, said not many patients are currently taking the drug because the requirement ties reimbursement to participation in clinical trials.
Some researchers have warned that the drugs would pose a huge financial burden on Medicare, study Published in JAMA Internal Medicine, estimates that Leqembi could cost the program more than $5 billion a year. They questioned whether Leqembi’s efficacy — it reduced the rate of cognitive decline in early-stage Alzheimer’s patients by 27 percent compared with a placebo in a late-stage trial — justified the high price and risk of side effects.
But patient advocacy groups have strongly criticized CMS’ decision to limit reimbursement for the drug because the FDA has given the green light under both its accelerated approval program and its traditional process.
John Dwyer, chairman of the Global Alzheimer’s Platform Foundation, said requiring registration for a drug to be approved would be “unprecedented and unacceptable” for doctors and patients.
The CMS decision could limit access for some Alzheimer’s patients in rural areas or those without electronic data entry from a health care provider, he said.
Rival Eli Lilly, which is developing a similar Alzheimer’s drug, also criticized CMS for tying reimbursement to registration.
Wall Street analysts say the registration requirement may delay Leqembi’s adoption in the short term because it requires physicians to sign up for a “CMS-facilitated portal,” but it’s not a big challenge in the long run.
“We believe ongoing registration requirements (combined with previously anticipated infrastructure buildout/prescriber education) may slow initial uptake of the category. Our long-term expectations for the market remain unchanged,” said JPMorgan, which forecasts new Sales of anti-amyloid drugs could reach $25 billion.
In a statement, CMS said it is committed to helping people access innovative treatments in a timely manner that can meaningfully improve Alzheimer’s disease care and outcomes.