By Rachel Cohrs
January 14, 2022
Medicare has outlined how it plans to cover the controversial, pricey new Alzheimer’s treatment Aduhelm, but the plan is missing a crucial detail: how much money Medicare patients who receive the drug would actually pay.
The draft coverage policy, announced Tuesday, would severely restrict the number of seniors who can access the drug at all, as Medicare plans to only cover the drug for patients participating in rigorous, agency-approved clinical trials. But Medicare remained mum on the potential cost of the drug for patients enrolled in the clinical trials.
Normally, patients do not pay to participate in clinical trials, as trial sponsors pay the cost of medication and related services. But these wouldn’t be normal clinical trials.
Thorny issues remain that likely won’t be resolved for months, and “now taxpayers are involved in financing the trials instead of the drugmakers themselves,” said Sean Dickson, director of health policy at the West Health Policy Center.
The cost of the treatment could also be relevant to trial sponsors’ ability to recruit clinical trial participants, as Medicare is also requiring an unprecedented level of diversity in trials to better reflect the Alzheimer’s patient population. Black and Hispanic people are significantly more likely to develop Alzheimer’s, but are more often left out of clinical trials.
Under Medicare, seniors usually pay 20% of the price of a drug administered in a physician’s office — though most seniors have additional insurance to help them cover that cost. Biogen, the company that makes Aduhelm, slashed the drug’s price from $56,000 to $28,200 per year in December.
Medicare officials provided little detail about potential clinical trial designs and didn’t mention anything in the draft policy about whether patients would be required to pay to participate in the trials, or whether patients would have to be kept in the dark about whether they were receiving treatment.
The gold standard for randomized controlled trials requires that both patients and providers are unaware of whether they are providing or receiving the drug or a placebo.
That begs the question: Is there a possibility that patients would be receiving a placebo and still paying for it?
In this highly unusual case, Medicare has more flexibility than usual to change how patients pay for drugs because Congress allows Medicare to adjust payments if it’s necessary to preserve the validity of clinical trials.
Normally, it would be illegal for drug makers to directly help Medicare patients pay their out-of-pocket costs. This provision could allow for some flexibility when sponsors submit clinical trial designs after a final coverage decision, which is expected by April 11.
“That’s a solvable problem… I don’t think that’s the major stumbling block,” said Joseph Ross, a professor of public health and medicine at Yale University and an expert in Food and Drug Administration regulatory science.
The idea that any patients would be receiving a placebo at all when an FDA-approved treatment is available is riling Biogen and the Alzheimer’s Association. The scientific community considers it unethical in many cases for patients to get a placebo after a treatment has demonstrated a benefit.
“We believe that the idea that someone would get a placebo in a trial, that would be a duplication of the FDA’s role, and would be, again, flatly wrong,” said Alzheimer’s Association CEO Harry Johns. “It’s hardly imaginable as something, again, that this administration in particular would pursue.”
Biogen also called the possibility that some patients could receive placebos “particularly concerning.”
Despite its complaints, however, Biogen is also setting up a confirmatory trial — one that was required by the FDA to prove whether the drug actually helps slow patients’ cognitive decline — that will be placebo-controlled.
Steven Joffe, the interim chair of the department of medical ethics & health policy at the University of Pennsylvania, said it’s perfectly ethical to run a placebo-controlled trial because Aduhelm is not proven to have clinical benefits for patients.
“This is a first-in-class approval, and it would be no ethical problem to conduct a randomized trial with a placebo-controlled arm,” Joffe said.
The argument that it would be unethical to withhold an FDA-approved medication from a patient didn’t sway Ross either, who said the line of argument is “a misuse of the field of ethics.”
Medicare may choose to approve clinical trials that compare treatment on the drug to the standard of care, though there aren’t alternative disease-altering therapies available for Alzheimer’s disease, said Sean Tunis, a principal at Rubix Health who helped develop the Coverage with Evidence Development process during his tenure at the Centers for Medicare & Medicaid Services. In that case, it would be possible trial participants would know whether they were receiving Aduhelm.
Tamara Syrek Jensen, director of the Coverage and Analysis Group at CMS, told stakeholders on Thursday that there may be trials that meet the criteria for approval that are not double-blinded and placebo-controlled.
Medicare will issue guidance surrounding payment rates, including required beneficiary cost-sharing, upon the publication of the final coverage decision, a CMS spokesperson said. The agency is accepting public comments on the draft proposal for 30 days.
“The mechanics of this is going to be one of the most fascinating aspects of this whole situation,” Joffe said.