The Centers for Medicare & Medicaid Services (CMS) should act quickly on determining coverage for the newly-approved Alzheimer's disease therapy aducanumab, developed by Biogen and Eisai, experts say.
The drug, an infusion that aims to break down amyloid plaques that are thought to worsen the disease, was approved by the Food and Drug Administration on Monday, but the therapy's path through the development pipeline has been fraught with controversy.
Aducanumab is the first treatment for Alzheimer's approved in two decades, and while patients and advocates pushed for approval, some providers and clinical experts were skeptical about the drug's trial results.
In a blog post for Health Affairs, health policy experts at Harvard Medical School, West Health Policy Center and Killelea Consulting said CMS should begin the process for a national coverage determination for aducanumab now to avoid potential confusion over coverage in the future.
The drug meets all of the conditions for an NCD, according to the post:
- It is estimated to cost as much as $70,000 annually, though based on its trial results should instead be priced at $2,500 to $3,800 per year to be "cost effective."
- Other controversial therapies have been met with varied local coverage decisions.
- Scientists, and the FDA's advisory board, have been concerned about coverage for the drug.
In the case of CAR-T, insurer UnitedHealthcare requested that CMS issue a national coverage decision to avoid the potential for variance in local decisions. The complexity of the therapy and the potential for negative side effects for Medicare patients were cited as key reasons the review was necessary.
"CMS should preemptively generate an internal request for an NCD regarding aducanumab to best protect Medicare beneficiaries from receiving a therapy where the risks likely outweigh the benefits," the authors wrote.
"Given the public attention to aducanumab and the proportion of Medicare beneficiaries who could be eligible for treatment, a clear, evidence-based policy would protect beneficiaries and taxpayers and avoid differential policies across the nation when aducanumab launches," they wrote. "CMS has a clear path to limit aducanumab usage to only the most appropriate cases, and, should aducanumab be approved, it should not hesitate to use this authority."
What payer industry experts are saying
David Steinberg, director of pharmacy insights at Scripta Insights, told Fierce Healthcare that the drug's likely hefty price tag will be an immediate concern for self-insured employers.
"Self-insured employer groups will look at their PBMs for guidance," he said. "It doesn't take much looking to see that this new drug is not all it's cracked up to be. In November, the FDA’s independent advisory committee voted overwhelmingly against recommending approval, saying data failed to demonstrate persuasively that aducanumab slowed cognitive decline. Additionally, three advisory committee members later wrote a point-by-point critique of the evidence."
In an article published last fall in JAMA Health Forum, clinical and pharmacy experts at Humana echoed the Health Affairs blog post, saying CMS should act quickly on a coverage decision but should consider a "coverage with evidence development" requirement, which could force Biogen to conduct further study into the drug's efficacy before approval.
The FDA issued a similar requirement in its approval of aducanumab. If the drug doesn't show significant results in phase 4 trials, it could be pulled from the market.
CMS initially intended to include a CED requirement in its coverage decision for CAR-T, but removed the requirement following significant industry pushback.
"Despite the tension between clinical effectiveness and payment, one thing is clear: Should aducanumab gain approval, our health care system will face both an opportunity and a challenge to ensure that the therapy is evaluated and used in a manner that safeguards value for patients, families, and the Medicare program," the Humana clinicians said.
The Humana team wrote that the drug is likely to balloon healthcare costs by billions of dollars each year. For example, based on a modest market of 5 million eligible people, uptake of 50% and a $40,000 annual price tag, aducanumab would add about $100 billion in direct costs per year.
On the high end, assuming the 18 million people with Alzheimer's disease and/or mild cognitive impairment are eligible for the drugs, annual costs could exceed $390 billion per year, or close to a third of all Medicare spending in 2019, according to the Humana article.
Analysts at Bernstein said Monday that payers who do experience sticker shock and choose to block aducanumab in Medicare Advantage may push people to switch to traditional Medicare instead to secure coverage. Many beneficiaries already do this when diagnosed with Alzheimer's disease, the analysts said, and insurers have little incentive to try to reverse this trend.
The vast majority of the market (96%) for this drug is expected to be in the Medicare population, the analysts said.