Medicare, the federal health insurance program that covers Americans over 65, is facing an impossible dilemma: Should it cover a new and expensive medication for Alzheimer\u2019s disease, which afflicts 6 million Americans and for which there is no existing treatment, even though the drug might not actually work?<\/p>\n
It is an enormous question. Alzheimer\u2019s patients and other families with members who endure mild cognitive impairment that may progress to Alzheimer\u2019s have been waiting decades for an effective treatment. For them, even a few more months of life with improved cognition, one more birthday party or a grandchild\u2019s graduation, is the priority.<\/p>\n
But the evidence on whether Biogen\u2019s treatment, called aducanumab, is effective is, at best, mixed; the FDA approved it this week over the objections of its own advisory committee. And with a preliminary announced price of nearly $60,000 annually per patient, covering the treatment could cost upward of $100 billion a year, mostly to Medicare, which would almost double the program\u2019s drug spending. Patients themselves could be on the hook for thousands of dollars in out-of-pocket costs.<\/p>\n
What Medicare does about aducanumab will have major ramifications not only for the millions of patients who could potentially be eligible for the drug, but for the future of US health care writ large.<\/p>\n
The dilemma results from a feature of the American health care system: Unlike in other countries, the federal government has little room to negotiate what Medicare will pay for treatments. <\/p>\n
Independent analysts think the drug is worth more like $8,000, but Medicare has no authority to charge a lower price. Instead, the federal program is likely in effect obligated to cover the new drug now that it has FDA approval. The tools it has to make a determination about whether or not to cover aducanumab and for whom are fraught with legal and ethical risk. <\/p>\n
The government now finds itself trying to figure out how to satisfy patients who desperately need help, even though scientists think this particular treatment lacks strong evidence for its effectiveness and policy experts warn it is setting up a budgetary nightmare for Medicare in the future.<\/p>\n
\u201cEvery conversation we\u2019re going to have for the next few years about health care access is going to be about this drug, whether implicitly or explicitly,\u201d Rachel Sachs, a law professor at Washington University in St. Louis who studies drug pricing, told me this week.<\/p>\n
The troubled path to aducanumab\u2019s approval<\/p>\n
Alzheimer\u2019s is a terrible disease that robs people of their agency during the final years of their lives and robs families of the loved ones they once knew. The emotional and financial costs are severe. And as the number of Americans over 65 grows, those costs are only expected to increase.<\/p>\n
In recent history, the decades-long search for an effective treatment or cure has been driven by what\u2019s known as the amyloid hypothesis, which holds that plaque in the brain found in Alzheimer\u2019s patients is at least in part responsible for the disease and removing that plaque could help relieve the symptoms.<\/p>\n
Aducanumab, accordingly, targets the amyloid plaque. Clinical trials of the drug started in 2015 but were halted in March 2019 because it did not appear it would meet the threshold for clinical effectiveness established at the start of the trials. It appeared, in other words, as though the drug didn\u2019t work. <\/p>\n
Normally, that would be the end of the story. But an unexpected twist came a few months later when Biogen revealed that, after additional data analysis with the FDA, some patients in one trial had actually seen \u201cbetter but ultimately mixed results,\u201d as the authors of a Health Affairs post on the controversy put it. Biogen announced it would push ahead with seeking FDA approval in October 2019, with the FDA\u2019s apparent support. <\/p>\n
Then, in November 2020, Biogen and aducanumab faced what looked like the ultimate setback: The FDA\u2019s advisory committee on neurological therapies voted the data did not demonstrate the drug was clinically effective. The vote was all but unanimous, with zero in favor, 10 nays, and one uncertain. They raised concerns about potential side effects, such as brain swelling in patients who were given high doses. <\/p>\n
But, in defiance of its own advisory committee\u2019s recommendation, the FDA granted aducanumab its approval on Monday. The news was welcomed by Alzheimer\u2019s patient groups but roundly criticized by experts in drug development.<\/p>\n
\u201cThe FDA … has failed in its responsibility to protect patients and families from unproven treatments with known harms,\u201d the Institute for Clinical and Economic Review (ICER), an independent non-government group that gauges the value of new drugs, said in a blistering statement.<\/p>\n
And the agency not only approved the drug over the advice of its scientific advisers, but it put effectively no restrictions on which patients with cognitive impairment should be given the drug, a decision that further stunned experts, as STAT reported.<\/p>\n
\u201cFor the FDA to approve it and with a very broad indication, I was shocked,\u201d Stacie Dusetzina, who studies drug costs at Vanderbilt University, told me. \u201cI really expected them to say no, based on the body of evidence.\u201d<\/p>\n
Medicare almost always covers FDA-approved drugs<\/p>\n
Now that aducanumab is approved by the FDA, the issue of coverage falls largely to Medicare; because of the age of the patient population most affected by Alzheimer\u2019s, the federal program is likely to bear the brunt of the drug\u2019s costs. <\/p>\n
In practice, if the FDA approves a drug, Medicare will pay for it. Aducanumab would be covered through Medicare Part B, which covers outpatient care, because it is an infusion treatment administered directly by doctors. To be covered by Part B, medical care must be \u201creasonable and necessary\u201d \u2014 a vague standard that has, for medications, historically been mostly synonymous with FDA approval.<\/p>\n
Because the drug is covered by Part B, doctors will even have a financial incentive to prescribe it. For prescription drugs, the program pays physicians the average price plus 6 percent, a policy that both Presidents Obama and Trump proposed changing but nevertheless remains in place. Determining which patients would benefit from the drug requires expensive scans, and practices will be able to bill Medicare for those, too. <\/p>\n