Unlocking Access to Alzheimer’s Treatment: The Controversy Surrounding Mandatory Data Collection

Eisai, a Japanese drugmaker, has expressed concerns about the potential limitations on patient access to their new Alzheimer’s treatment due to mandatory data collection by US health authorities. The Centers for Medicare and Medicaid Services (CMS) has decided to restrict financial support for Eisai’s drug, Leqembi, to Alzheimer’s patients whose doctors participate in a health agency database. This decision by CMS has the potential for unintended consequences.

The Purpose and Impact of the Registry

CMS’s registry, also known as a database, aims to collect evidence on the real-world effectiveness of Leqembi and similar drugs. It will make this evidence available for researchers to conduct studies. This move marks the first time that Medicare, the US government health program for individuals over the age of 65, has made this level of data collection mandatory for a drug that regulators already deem safe and effective.

Concerns and Potential Consequences

Eisai raises concerns about the potential consequences of the registry on coverage for certain groups of people who may choose not to participate. The company questions the fairness of linking reimbursement obligations to participation in data collection. Eisai plans to wait for further details about the registry before finalizing their spending plans to support the drug’s launch.

Ivan Cheung, the US Chief Executive Officer of Eisai, emphasizes the distinction between gathering real-world evidence for understanding the drug’s effectiveness and mandatory participation in data collection for reimbursement purposes. He acknowledges that there are individuals who may choose not to participate, citing the freedom of choice in a democratic country.

The Development and Approval of Leqembi

Leqembi, jointly developed by Eisai and US biotech company Biogen, is set to become the first FDA-approved Alzheimer’s drug that can slow the progression of the disease. In a recent panel recommendation, external experts advised the FDA to approve the drug, a decision that typically triggers Medicare coverage for all patients over the age of 65. Reimbursement from the government health system plays a critical role in increasing patient access to Leqembi, which costs $26,500 per year and primarily targets the elderly.

CMS’s Cautious Approach to Alzheimer’s Drugs

CMS has taken a cautious approach to the new generation of Alzheimer’s drugs that aim to slow disease progression by reducing the buildup of sticky amyloid plaques in the brain. In the case of Biogen’s Aduhelm and other amyloid-depleting drugs approved under an expedited process called accelerated approval, CMS imposed stricter restrictions last year. Reimbursement was limited to patients enrolled in clinical trials, resulting in Biogen discontinuing the Aduhelm launch. CMS cited concerns about the drug’s harmful potential, including side effects ranging from headaches to bleeding in the brain, as well as doubts about its effectiveness.

Eisai, having received accelerated approval for Leqembi in January, reports limited patient intake due to the reimbursement requirement tied to clinical trial participation. While some researchers argue that these drugs will impose a significant financial burden on Medicare, patient advocacy groups strongly criticize CMS’s decisions to limit reimbursement for drugs approved through expedited and traditional processes.

The Financial Burden and Ethical Concerns

A study published in JAMA Internal Medicine estimates that Leqembi could cost the Medicare system over $5 billion annually, raising questions about the drug’s price and the risk of side effects versus its efficacy. Critics argue that requiring participation in a registry to access an approved drug sets an unprecedented and unacceptable precedent for doctors and patients. They argue that this requirement may limit access for Alzheimer’s patients in rural areas or those lacking healthcare providers equipped for electronic data entry.

Industry Insights and Long-Term Expectations

Industry experts predict that the registry requirement may cause initial delays in the adoption of Leqembi but anticipate no significant long-term challenges. JPMorgan believes the new class of anti-amyloid drugs, including Leqembi, could generate peak sales of $25 billion. They reference the ongoing registry requirement, prescriber education, and infrastructure construction as factors contributing to the potential initial slowdown in category uptake.

Commitment to Innovative Treatments and Care

CMS has emphasized its commitment to facilitating timely access to innovative treatments that significantly improve care and outcomes for Alzheimer’s disease. The registry aims to provide comprehensive data that can guide future research and decision-making processes.

Additional Piece: Exploring the Future of Alzheimer’s Treatment and Healthcare Systems

While the controversy surrounding the mandatory data collection for Alzheimer’s treatment raises valid concerns about patient access and the balance between real-world evidence and reimbursement requirements, it also opens up discussions about the future of healthcare systems and the challenges they face in accommodating innovative therapies.

Alzheimer’s disease, affecting millions of individuals globally, continues to pose significant challenges for patients, families, and healthcare providers. The development of drugs like Leqembi and Aduhelm offers hope for slowing disease progression and improving patients’ quality of life. However, the high price tags and potential side effects necessitate a careful evaluation of their cost-effectiveness and equitable patient access.

As technology advances and more data becomes available, healthcare systems must grapple with the practical implementation of these new treatment options. The CMS registry represents an effort to collect valuable information on the real-world effectiveness and safety of drugs like Leqembi and Aduhelm. This data will inform future research and healthcare policy decisions, laying the groundwork for improved care and outcomes.

Nevertheless, the challenges of balancing access, affordability, and quality care persist. The high costs associated with innovative treatments place strains on public health systems like Medicare, requiring policymakers to carefully consider the allocation of resources. Additionally, issues of equity and fairness arise when reimbursement requirements tie access to participation in registries or clinical trials, potentially excluding certain patient groups.

Looking forward, stakeholders must engage in constructive dialogue to address these challenges effectively. This includes exploring alternative reimbursement models, such as value-based pricing, that incentivize the development of innovative treatments while ensuring affordable access for those in need.

Furthermore, a focus on preventive strategies, early detection, and holistic care models can help alleviate the burden of Alzheimer’s disease on both patients and healthcare systems. Investing in research and support for caregivers, expanding education and awareness programs, and fostering collaboration between stakeholders are vital components of a comprehensive approach to addressing Alzheimer’s disease.

Summary:

Eisai, a Japanese drugmaker, has expressed concerns about the potential limitations on patient access to their new Alzheimer’s treatment due to mandatory data collection by US health authorities. CMS’s decision to link financial support for Eisai’s drug, Leqembi, to participation in a health agency database raises questions about unintended consequences and fairness. The registry aims to collect real-world evidence on drug effectiveness but may hinder coverage for certain patient groups. The development of drugs like Leqembi and Aduhelm brings hope for Alzheimer’s patients, but their high costs and potential side effects require careful evaluations. Balancing access, affordability, and quality care remains a challenge for healthcare systems, and stakeholders must engage in constructive dialogue to address these concerns. The future of Alzheimer’s treatment lies in innovative reimbursement models, preventive strategies, early detection, and holistic care approaches.

Eisai warned that mandatory data collection by US health authorities could limit patients’ access to the Japanese drugmaker’s new Alzheimer’s treatment.

The company said a decision by the Centers for Medicare and Medicaid Services to limit Leqembi’s financial support to Alzheimer’s patients whose doctors participate in a health agency database could have “unintended consequences.”

CMS said the database, also known as the registry, will collect evidence on how the drug it works in practice and makes it available to researchers to conduct studies on Leqembi and similar drugs.

It marks the first time Medicare — the US government Health regime for people over the age of 65 – made that data collection requirement mandatory for a drug deemed safe and effective by regulators.

Eisai said he feared the registry could hamper coverage for some groups of people who didn’t want to participate, and raised questions about fairness. The company said it will wait to see registry details before finalizing spending plans to support the drug’s launch.

“There is a distinction between gathering real-world evidence to understand more [about the drug] against the reimbursement obligation based on participation in that real-world data collection,” said Ivan Cheung, US chief executive officer of Eisai, in an interview.

“There are people who just don’t want to participate for whatever reason. It’s a free country, right?

Leqembi, which was jointly developed by Eisai and US biotech firm Biogen, is set to become the first Alzheimer’s drug that can slow the progression of the disease to gain mainstream approval from the US Food and Drug Administration.

This month, a panel of experts outside the FDA recommended approval of the drug, a decision that typically would prompt Medicare to cover the treatment for all patients over age 65. Reimbursement from the government health system is critical to increasing patient access to Leqembi, which costs $26,500 a year and targets a disease that primarily affects the elderly.

But CMS has taken a cautious approach to the new generation of Alzheimer’s drugs, which slow disease progression by reducing the buildup of sticky amyloid plaques in the brain.

Last year the agency imposed even tighter restrictions on Biogen’s Aduhelm and other amyloid-depleting drugs that have been given the green light under a fast-track process called accelerated approval.

That decision limited reimbursement to patients enrolled in a clinical trial of the drug and caused Biogen to discontinue the Aduhelm launch. At the time, CMS cited the drug’s “harmful potential,” ranging from headaches to bleeding in the brain, as well as concerns about the drug’s effectiveness for patients.

Eisai, who received accelerated approval for Leqembi in January, said he doesn’t have many patients taking the drug at the moment because of this requirement that links reimbursement to participation in a clinical trial.

Some researchers have warned that the drugs will place a large financial burden on Medicare, with a study published in JAMA Internal Medicine estimating that Leqembi could cost the scheme more than $5 billion annually. They wonder if Leqembi’s efficacy — it slowed the rate of cognitive decline in early-stage Alzheimer’s patients by 27 percent compared to placebo in a late-stage study — justifies the high price and risk of side effects.

But patient advocacy groups have strongly criticized CMS’s decisions to limit reimbursement for drugs given FDA greenlights under both its expedited approval process and the traditional process.

John Dwyer, president of the Global Alzheimer’s Platform Foundation, said requiring participation in a registry to access an approved drug was “unprecedented and unacceptable” for doctors and patients.

He said CMS’s decision could limit access for some Alzheimer’s patients in rural areas or those without a healthcare provider equipped to provide electronic data entry.

Rival drugmaker Eli Lilly, which is developing a similar Alzheimer’s drug, also criticized CMS for linking the reimbursement to a registry.

Wall Street analysts said the registry requirement could delay Leqembi adoption in the short term, as it would require doctors to sign up for a “CMS-facilitated portal,” but that it hasn’t been a huge challenge in the long run.

“We believe the ongoing registry requirement (combined with the previously envisaged prescriber education/infrastructure construction) may slow initial uptake for the category. Our long-term expectations for the market remain unchanged,” said JPMorgan, which expects the new class of anti-amyloid drug could generate peak sales of $25 billion.

CMS said in a statement that it is committed to helping people gain timely access to innovative treatments that significantly improve care and outcomes for Alzheimer’s disease.