It won’t be easy to get the first fully approved drug for Alzheimer’s disease

IIt’s a decision that millions of people with Alzheimer’s disease and their families have been waiting for — the first fully approved drug that treats the disease, rather than its symptoms. On July 6, the US Food and Drug Administration (FDA) announced Grant full consent Licanimab, or liquimbe, to treat Alzheimer’s disease in people in the early, mild stages of the neurodegenerative condition.

Doctors can already prescribe lecanemab, which is made by Eisai Inc. and Biogen, since it’s been available under fast-track approval from the Food and Drug Administration since January, but that conditional approval has limited access. The Centers for Medicare and Medicaid Services (CMS), which administers Medicare, I decided in response to the expedited approval that it will not cover the $26,500 annual cost of lecanemab unless people are part of a clinical trial that continues to collect safety and efficacy data on the drug. CMS said it would only reimburse the drug outside of the studies if the drug received full approval from the Food and Drug Administration, which it has now, and people Register in the registry It requires physicians to continue to report safety and efficacy data on their patients who use it.

The registration requirement makes consent a bittersweet victory, say some patient advocates. Jim Taylor, co-founder of Alzheimer’s soundsa patient advocacy group, whose wife, Jerry, was diagnosed with the disease in 2012. “But we can’t even know whether or not people with the disease will have access to it.”

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the Alzheimer’s Association She remained adamant that while it supports the need for a better understanding of how lecanemab works in real patients, “the traditional approval of the type of data that Eisai gave Leqembi should never require any kind of registration at all,” says Maria Carrillo, the association’s chief scientific officer. “We feel it is essential in all treatments to learn more about safety and efficacy. But is it required for coverage? It shouldn’t be.”

Within hours of the FDA’s decision, CMS issued a connection For doctors to register on its registration portal, keeping its predecessor formulations that it would be “easy to use” and “where possible, drop-down menus will be available”. In response to questions from TIME magazine about how common such records are, and whether other drug coverage is associated with similar conditions, a CMS spokesperson cited radiation drugs to treat cancer and devices such as heart valves and stents. In defending the registry, the spokesperson said that covering treatments under registration requirements “can accelerate beneficiaries’ early access to innovative technology while ensuring that systemic patient safeguards, including ensuring technology is made available to clinically appropriate patients, are in place to reduce inherent risks.” in new technologies.

Based on the previous data, the types of information the CMS will require in its log will be data that doctors already collect, including reports of side effects such as encephalitis and results of cognitive tests to verify a patient’s diagnosis of mild Alzheimer’s disease. CMS also contains files Requests Additional data documenting any meaningful improvement in patients taking the drug, any side effects, and how these benefits and risks change over time with continued use.

More pessimistic experts say CMS’ decision to seek registration may have been motivated by the more practical goal of limiting payments for the first effective treatment for Alzheimer’s disease. While another drug, aducanumab, was approved before lecanemab and also treats the disease, its results have been less consistent, and both doctors and patients have been less willing to use it. “Usually when a CMS delays things…it’s because of cost concerns,” says Thomas Phillipson, professor emeritus of economics at the University of Chicago who previously worked on CMS. “The question is, are they staring at the overall costs or are they just staring at the drug costs?”

in paper Published by the University of Chicago, Philipson calculated that the health system could save anywhere from $300 billion to $1.8 trillion overall if more people with Alzheimer’s disease were treated with drugs like lecanemab that could delay the progression of their disease. “There is a general misconception, common in CMS, that more spending on medicines necessarily leads to higher costs for Medicare which is often not true,” says Phillipson. “Often, when you have new drugs, total healthcare spending goes down because the new drug offsets other spending.”

In any case, doctors will need to be on the registry in order for their patients to receive Medicare reimbursement for the drug, and in the coming months, it will become clear whether or not registration becomes an obstacle to access. “I have concerns that if [CMS] make it very difficult for practitioners who aren’t in large academic medical centers — in private practice or those who help disadvantaged communities — then you’ll further restrict access,” says Dr. Hospital. For professionals like her, in large medical centers, joining may not be To the registry is a huge lift.In fact, many groups like hers have already joined the Alzheimer’s Association ZentIt is a portal for recording patients’ experiences with drugs such as likanimab so that other clinicians can learn from them and better understand how people respond to the drug. It’s not clear if the CMS gateway will be a duplicate of AlzNet or if there are ways to simplify data collection between the two systems; Carillo says the Alzheimer’s Society plans to discuss this issue with CMS at an upcoming meeting.

Addressing coverage, and thus access, is critical to better understanding the true impact that disease-modifying drugs such as lecanemab could have on Alzheimer’s disease. “I see this drug primarily as a long-sought catalyst for transformational change,” says Dr. Alvaro Pascual Leon, MD, professor of neurology at Harvard Medical School and medical director of Hebrew SeniorLife, a not-for-profit health facility in New England. “Dementia care right now isn’t what it should be. It’s reactive — we wait until people are significantly disabled because we don’t do any screening earlier,” he says. The presence of effective drugs like lecanemab could spur more clinicians to begin testing their patients for the first signs of Alzheimer’s dementia, and direct them not only to drug therapies but also to lifestyle changes, which studies have shown can, in some people, reduce of advancing cognition. increased by up to 40% – more than the 27% recorded with lecanemap.

says Dr. Charles Bernick, a neurologist at the Cleveland Clinic Lou Ruvo Center for Brain Health in Nevada. “But now, with effective treatment, there is.”

It is important to set expectations, because although lecanemab can slow down the ongoing decline of cognitive function, it cannot improve people’s memory. Essentially, this steady decline pushes back to buy patients more time during which they can remain independent and able to take care of themselves. Taking that time is critical for patients like John Domick, a retired attorney in Aurora, Ohio, who was diagnosed with Alzheimer’s disease in 2019. His doctor has been honest about his outlook, telling him and his wife, Anne, that he’ll have about eight years before it starts showing up. More severe symptoms of the condition, including problems with speech, physical clumsiness, more serious memory loss, and finally the inability to swallow. For about a year they’ve focused on lifestyle activities that can slow Alzheimer’s — reading, exercising, solving puzzles, and trying to stay social, despite the COVID-19 lockdown. “We sat in our driveway and talked to our neighbors,” says Ann. In 2020, Domeck joined the lecanemab trial, and he and Ann didn’t know if he was taking the drug or a placebo twice a month for 18 months. After the study ended, he began receiving the drug for sure in July 2022, in the open part of the trial, which will continue for another two years or so. “I’ve noticed a difference,” says Anne of her husband’s short-term memory improvement since last year. At a beach gathering with her relatives two weeks ago, she said John remembered the drinks served during the family’s previous Christmas celebration. “It shocked me,” she says. “The short-term things he didn’t remember before, he does now.” Slowing down the disease has allowed them to continue traveling and, for John, to continue playing golf.

Ann and John Domecq go on a trip to Alaska in 2021, timed between injections while studying; Ann and John during John’s injection at the Cleveland Clinic in January 2023

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Domic family

John is now testing a more convenient way to deliver licanimab, through autoinjections that Ann gives him in the abdomen once a week at home rather than intravenous injections twice a month at the Cleveland Clinic, where he was part of the study. Eisai says the company plans to report the results of the self-injection by the end of the year.

John never hesitated to volunteer, and says, “Participating in the trial was something we both looked forward to.” With likanimab’s approval, he and Ann begin to appreciate what this decision could mean for Alzheimer’s patients. “I don’t think we understood what that would result in,” Ann says. “But the more we participate in it, the greater the impact we have. It’s amazing.”

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