Payers should be keeping a close eye on developments in the drug pipeline for Alzheimer's disease therapies—and how the Centers for Medicare & Medicaid Services (CMS) responds to those developments, according to a new report from Optum.
Optum released its latest quarterly Drug Pipeline Report, which highlights products coming through the pipeline that payers should be watching. In this edition, the report includes two monoclonal antibody drugs that target beta-amyloid plaques to treat Alzheimer's: lecanemab and donanemab.
This class of drugs has been controversial since the first therapy, Aduhelm, secured accelerated Food and Drug Administration approval in the summer of 2021. Clinical experts had expressed concern about the drug's efficacy and the high price tag.
As a result, CMS issued a restrictive national coverage decision that limits coverage to patients in clinical trials in the absence of full FDA approval. Patient advocates have pushed the agency for greater access.
Optum's report notes that lecanemab, developed by Biogen and Eisai, secured accelerated approval from the FDA in January, while Eli Lilly's donanemab was rejected. Biogen and Eisai have already filed for traditional FDA approval, and Eli Lilly expects to have the data necessary to follow suit in the second quarter of this year, according to the report.
Just how successful the data are for either drug will be critical to watch for insurers making decisions about coverage, Arash Sadeghi, a clinical pharmacist at OptumRx, told Fierce Healthcare.
"What CMS ends up doing in terms of revisiting that policy based on some of this phase 3 trial data is really going to shape how successful these drugs are," he said.
While CMS said it is awaiting those additional data, or a full FDA approval, to take another look at the coverage decision, patient advocates have argued that it should adjust more quickly to allow people with Alzheimer's to access the drug sooner.
In addition to the emerging therapies for Alzheimer's , the report highlights two more drugs of interest in the early part of this year: Roctavian, a gene therapy for hemophilia, and fezolinetant, which treats moderate to severe vasomotor symptoms associated with menopause.
An FDA decision on Roctavian is expected March 31, though it could slip into the second quarter should an additional phase 3 analysis be added to the application, according to the report. Like other gene therapies, if approved Roctavian could carry a hefty, multimillion-dollar price tag.
Sadeghi said payers can prepare now for these pricey therapies to come to market by examining their member populations and identifying the likely areas of greatest need. Using Roctavian as an example, he said that some 30,000 people in the U.S. have hemophilia, but they won't all immediately need to transition to a gene therapy.
The goal is to avoid "payers getting caught off guard" by mounting costs, though the price of these therapies isn't necessarily a shock as more come to market, he said.
The final drug in the report, fezolinetant, would enter a market where it could see significant demand, as between 60% and 80% of women experience vasomotor symptoms either during or after menopause. The front-line treatment for these symptoms is hormone therapy, which may not work as an option for patients with risk of breast cancer, heart disease and stroke.
The drug, produced by Astellas Pharma, could also face future competition from another therapy in the pipeline, developed by Bayer, according to the report.
While fezolinetant could prove an appealing option for some patients, there is a lack of data comparing its efficacy head-to-head with established treatments, many of which are offered as generics, the report said.