The Food and Drug Administration (FDA) made a significant move on Thursday by authorizing Leqembi, a groundbreaking treatment for Alzheimer’s disease. This pivotal decision is expected to enhance accessibility to this expensive medication for senior citizens in America. With the increasing prevalence of dementia-related disorders, the approval of Leqembi represents a crucial step towards addressing the healthcare needs of the aging population.

In conjunction with the FDA’s approval, Medicare has committed to begin covering Leqembi, albeit under certain conditions. This coverage will commence on the same day the FDA greenlights the antibody treatment. Developed by the Japanese pharmaceutical company Eisai and its partner, Biogen from Cambridge, Massachusetts, Leqembi is positioned to become a vital component of Alzheimer’s care for many older Americans.

Leqembi holds the distinction of being the first antibody treatment for Alzheimer’s to receive full FDA approval. It also stands out as the first drug of its kind to potentially receive comprehensive coverage from Medicare, which is crucial for many patients seeking effective therapies.

Medicare’s decision to cover Leqembi is monumental for assisting older adults diagnosed with early-stage Alzheimer’s disease in affording this treatment. Given that the average income for Medicare beneficiaries hovers around $30,000, the hefty annual price tag of $26,500 set by Eisai could be insurmountable without insurance coverage.

It is important to note that Leqembi is not a cure for Alzheimer’s. The treatment has been shown to slow cognitive decline associated with early Alzheimer’s disease by 27% over an 18-month period during clinical trials conducted by Eisai. This antibody treatment is administered via intravenous infusion twice a month and specifically targets a protein known as amyloid, which has been linked to the progression of Alzheimer’s disease.

Joanna Pike, president of the Alzheimer’s Association, a leading advocacy group for individuals living with the disease, emphasized that while Leqembi is not a definitive cure, it can significantly help patients in the early stages maintain their independence, engage in daily activities, and spend valuable time with their families. She stated, “This provides individuals with more months of recognizing their loved ones and engaging in life.” This sentiment underscores the emotional and social benefits that such treatments can bring to patients and their families.

However, it is crucial to acknowledge the serious risks associated with this treatment, including potential brain swelling and bleeding. There have been reports of three participants in Eisai’s study who died, raising questions about whether Leqembi played a role in these fatalities, a matter that remains under investigation by FDA scientists.

Alzheimer’s disease is recognized as the most common cause of dementia among older adults and is the sixth leading cause of death in the United States, according to FDA data. This highlights the urgent need for effective treatments and ongoing research in this critical area of healthcare.

Dr. David Knopman, a neurologist specializing in Alzheimer’s disease at the Mayo Clinic in Minnesota, remarked that Leqembi clearly demonstrated a benefit to patients in Eisai’s trial, although he cautioned that the efficacy of the treatment is modest. He believes that well-informed patients should have the autonomy to decide whether to pursue Leqembi after carefully weighing the potential benefits and risks, as well as the logistical challenges of receiving the treatment.

Medicare has announced its intention to impose specific conditions regarding coverage for Leqembi. Patients enrolled in Medicare who receive a diagnosis of early Alzheimer’s disease will be required to seek treatment from a healthcare provider participating in a registry system designed to collect real-world data on the drug’s efficacy and safety.

This registry system has sparked controversy, as both the Alzheimer’s Association and some members of Congress express concerns that this requirement may create unnecessary barriers to accessing treatment. There are worries that the number of healthcare providers participating in such registries may be limited, potentially forcing individuals from rural areas and underserved communities to travel significant distances to find a suitable provider.

To address these concerns, the Centers for Medicare and Medicaid Services (CMS) has committed to establishing a national portal that will facilitate the submission of necessary data by healthcare providers administering Leqembi. This user-friendly portal is expected to be available as soon as the FDA approves the treatment.

California Representatives Anna Eshoo and Nanette Barragan have raised concerns in a letter to CMS regarding the potential difficulty patients might face in finding a participating physician within the system. Alzheimer’s disease is typically diagnosed through advanced imaging techniques such as PET scans to detect amyloid proteins associated with the condition or, in some cases, through spinal taps. Currently, Medicare only covers one PET scan per lifetime for dementia diagnoses, and it remains unclear if this policy will be revised to accommodate the new treatment.

There are also worries about the adequacy of specialist physicians available to administer the infusions if Leqembi experiences widespread adoption and patient demand surges. Some studies predict that wait times for antibody therapies like Leqembi could extend from months to even years in the coming decade, depending on demand levels.

Tomas Philipson, who served as an advisor to the FDA commissioner and CMS administrator during the second Bush administration, expressed that the registry may pose an unnecessary obstacle and that Medicare should consider eliminating it. However, he does not believe this requirement will create an insurmountable barrier for patients seeking access to Leqembi.

If demand for Leqembi is substantial, physicians may be incentivized to participate in the registry, and pharmaceutical companies will likely support this endeavor, according to Philipson, who specializes in healthcare economics at the University of Chicago. The level of demand for Leqembi remains uncertain, as some families may be hesitant due to the potential for serious side effects, while others may feel the benefits outweigh the associated risks.

The pricing of Leqembi and the associated benefit-risk profile have sparked considerable debate. Even with Medicare coverage, patients could still face out-of-pocket expenses amounting to $6,600 annually for Leqembi, according to a study published in the journal JAMA Internal Medicine. The overall cost of the drug could reach up to $5 billion annually for Medicare, depending on the number of individuals receiving the infusions, as estimated in the same study.

Senator Bernie Sanders, chair of the Senate Health Committee, has labeled Leqembi’s pricing as “unconscionable” and recently urged Health and Human Services Secretary Xavier Becerra to take action to reduce the medication’s cost. Sanders highlighted that the out-of-pocket expenses for Leqembi would consume a significant portion of many seniors’ annual income and noted that the high price may lead to increased premiums for all Medicare beneficiaries.

Eisai maintains that its annual list price of $26,500 for Leqembi is lower than its estimated total treatment cost of $37,600 for each patient. The Institute for Clinical and Economic Review, a nonprofit organization that evaluates healthcare costs, estimated that the drug should ideally be priced between $8,900 and $21,500 annually to be considered reasonably accessible.

Philipson warned that delaying Medicare coverage for Leqembi could result in increased healthcare expenditures as patients with mild Alzheimer’s disease, who could manage their condition at home, progress to a more severe stage requiring costly nursing home care. His research indicates that postponing Medicare coverage for Alzheimer’s antibody therapies by just one year could lead to an additional $6.8 billion in overall spending, with projections suggesting a total increase of $248 billion in healthcare costs by 2040.

The full FDA approval announced on Thursday follows a unanimous vote from a panel of six external advisors in June, supporting the drug’s medical benefits for patients. This panel was notably small, as some members recused themselves due to conflicts of interest.

The American Academy of Neurology communicated in a February letter to CMS that there is a consensus among its specialists regarding the robust design of Eisai’s clinical trial and the results being “clinically and statistically significant.”

Conversely, some nonprofit organizations, including Public Citizen, a consumer advocacy group, have strongly opposed the FDA’s approval of Leqembi. A representative from Public Citizen advised the advisory panel that the evidence supporting the drug’s benefits does not sufficiently outweigh the significant risks of brain swelling and bleeding.

Additionally, representatives from the National Center for Health Research and Doctors for America, two other nonprofit organizations, expressed concerns to the panel regarding the lack of adequate representation of Black patients in Eisai’s clinical trial, despite their higher risk for Alzheimer’s disease.

Leqembi has been technically available for the U.S. market since January, when the FDA granted the treatment approval through an expedited pathway designed to facilitate quicker access to medications for patients suffering from serious conditions.

However, at that time, Medicare declined to provide coverage for Leqembi, citing the need for further evidence demonstrating that the costly treatment offered meaningful medical benefits that outweighed its risks.

This cautious approach by Medicare stems from the FDA’s contentious 2021 approval of a different Alzheimer’s antibody treatment, Aduhelm, also produced by Eisai and Biogen. The FDA’s advisory committee previously refrained from endorsing Aduhelm, due to insufficient data supporting a medical benefit for patients. Following the agency’s decision to approve the drug, three advisory members resigned in protest.

Dr. Knopman was among those who resigned, but he noted that the data for Leqembi is distinct, as Eisai conducted a transparent trial demonstrating that the antibody provides a modest medical benefit for patients. Additionally, a congressional investigation revealed that the FDA’s approval process for Aduhelm was fraught with irregularities, further complicating public trust in the agency.

In his letter to Secretary Becerra, Sanders emphasized that the FDA holds a special responsibility to restore public confidence following its controversial relationship with Biogen during the review of Aduhelm, stressing the importance of rigorous evaluation in the approval of Alzheimer’s treatments.