The Centers for Medicare and Medicaid Services (CMS) will not cover Biogen Inc.’s new approved drug for Alzheimer’s disease except in cases where the patient is enrolled in a controlled randomized trial or a government study.
The April 7 decision came nearly one year after the Food and Drug Administration (FDA) approved Aduhelm under its “accelerated approval pathway.” The FDA gave the drug the green light despite objections from 10 of the 11 members on its advisory panel who said Aduhelm had failed to demonstrate efficacy.
The approval was wrought with controversy because it could open the door for Medicare to cover a drug with questionable efficacy and one that came with a significant price tag. A minimum estimate was $29 billion annually or about $56,000 per patient.
Biogen announced in October that one of the trials did demonstrate those receiving high doses of the drug had fared better than those receiving placebo. The other trial found no meaningful benefit.
Drug Makers Respond
The requirement for enrollment in an approved randomized controlled trial doesn’t only apply to the Biogen drug, but to all beta amyloid-directed monoclonal antibodies, indicating a general concern over this approach as an effective treatment of Alzheimer’s.
Pharmaceutical trade groups have attacked the decision as a precedent against general coverage of therapies approved by the FDA under the “accelerated approval pathway.”
The president and CEO of the Biotechnology Innovation Organization, Michelle McMurry-Heath issued a statement, echoing industry concerns.
“With this decision, CMS is not just saying it has no confidence in Alzheimer’s drugs approved under the FDA’s accelerated approval pathway,” she said. “It also is undermining confidence in FDA’s traditional drug approval process more broadly.”
A Flawed Approval Model?
Experts disagree as to whether the CMS decision represents a sea change in how it determines eligibility for reimbursement of FDA-approved drugs, as well as whether the entire approval process is predicated on an inherently flawed model.
Jeffrey A. Singer, M.D., a senior fellow at Cato Institute, believes the decision was correct but that FDA approval is flawed.
“I think CMS made the right decision, but this is probably an isolated decision, not a policy shift,” said Singer. “The controversy over the FDA’s approval of Aduhelm is another example of why the FDA should not be basing the approval process on its determination of a drug’s efficacy. That falls under the realm of real‐world clinical research. Efficacy requirements add years to the approval process.”
Michael F. Cannon, Cato’s director of health policy studies, views the issue from a constitutional perspective.
“The amount that the federal government should be paying for Aduhelm is the same as it should be paying for all types of medical care: $0.00,” said Cannon. “If Congress nevertheless decides that it will violate individual rights and the Constitution by robbing Peter to purchase medical care for Paul, it should do so to the minimum extent possible,” said Cannon.
“Refusing to purchase medical care that doesn’t even work seems like a reasonable limit to impose on what was already bad behavior. Unfortunately, this has not been Medicare’s historical practice,” said Cannon. “Medicare pays for tons of medical care that does not work, which serves only to injure Peter and enrich Paul’s health care providers, without benefiting Paul at all.”
Penny Wise, Dollar Foolish?
Edward Hudgins, founder of Human Achievement Alliance and author of the 2019 policy brief, “A Modern System for Approving the Cures of the Future,” says opening the gate on new treatments could demonstrate unknown benefits.
“No doubt the costs of the treatment played a part in the CMS decision to only cover patients in clinical trials. Medicare costs are always strained, and policymakers have an incentive to keep costs down in the short term and ignore the long-term implications of this policy. This points to a fundamental problem with government medical insurance,” said Hudgins.
The current government insurance system is focused on “sick care,” rather than “health care,” says Hudgins.
“While it has shown results only in limited circumstances so far, Aduhelm is the first treatment in almost two decades to treat Alzheimer’s,” said Hudgins. “It would be better for this treatment to be tested as widely as possible because if it does in fact prove effective for a wider category of sufferers, it would reduce one of the worst scourges afflicting the elderly and reduce health care costs as well.”
Who Pays for New Drugs?
Hudgins says one way to get around the cost concerns of testing innovative drugs would be a reform proposal known as “Free to Choose Medicine.”
“The sponsor could offer Aduhelm on such a track to all who desired it. Results would be logged in a database which would supplement clinical trials and allow researchers more quickly to evaluate the treatment’s efficacy,” said Hudgins. “Further, if the treatment proves successful, over time it would be refined and the market for the treatment would expand, bringing down costs.
The decision to participate in efficacy trials should be left to patients, not regulators, says Singer.
“I think patients should be allowed to purchase and try drugs that may have not been approved by the FDA but are approved by other credible third-party certifiers,” said Singer. “With such a serious and fatal disease as Alzheimer’s, many patients don’t have the time to wait for the FDA to give them permission to save themselves from this terrible fate.”
“While rational people value the FDA’s opinion on these matters, the decision is a personal one and an autonomous adult should not require permission from the government when they want to try to save their life,” he said. “If the patients themselves are paying for the drug, they and their doctors will perform much better due diligence before committing their own money to what might be a dry hole.”
Kevin Stone (firstname.lastname@example.org) writes from Arlington, Texas.