A group of medical and healthcare policy experts raised significant doubts about the potential benefits and high cost of Biogen's new Alzheimer's drug Aduhelm, reiterating at a Thursday meeting many of the criticisms that have made the treatment's recent approval extremely controversial.
Convened by the Institute of Clinical and Economic Review, an influential nonprofit, the meeting featured yet another rejection of the clinical trial evidence marshaled by Biogen to win Food and Drug Administration clearance of Aduhelm. Experts voted unanimously that the available data did not demonstrate a net health benefit for treatment over supportive care, citing the contradictory results from Biogen's main two studies as well as the drug's known side effects.
"Given the data, such as it is, and given the risks, from the standpoint of do no harm it makes one feel uneasy," said Sarah Kremen, a director of the neurobehavior program at Cedars-Sinai Medical Center and Aduhelm trial investigator who was invited by ICER to review the drug at the meeting. Kremen was not one of the 15 panel members who voted.
The vote against Aduhelm by ICER's panel was somewhat expected after the group, which frequently clashes with drugmakers over the value and cost of new therapies, concluded in a report updated last month that Biogen's evidence was "insufficient."
But the rebuke adds to a growing number of outside voices that have expressed substantial skepticism about whether the FDA's approval of Aduhelm was justified by the data. Three members of an FDA advisory panel that similarly criticized Biogen's application last November resigned soon after the regulator's decision. And in the weeks since the drug's launch, a number of insurers have indicated they will not cover treatment. Two major medical centers, the Cleveland Clinic and Mount Sinai, said this week they won't administer the drug.
Panel members also wrestled with the drug's price, which Biogen set to an average of $56,000 per year, and its potential impact on the healthcare system without clear evidence it actually helps people's lives.
"We're talking about taking little bits of money from the average American through the healthcare system and transferring it to the shareholders of Biogen," said Annette Langer-Gould, a neurologist with Kaiser Permanente in California, likening paying for the drug to a "reverse Robin Hood."
Amid similar criticism from lawmakers and patients groups, Biogen has indicated it would work with insurers to address pricing concerns if the company's assumptions about how many patients are likely to receive the drug prove incorrect.
An estimated 6 million Americans are living with Alzheimer's and, should many of them eventually receive Aduhelm, experts predict tens of billions of dollars could be added to Medicare's drug budget. Biogen's estimate of the patient population who would benefit from Aduhelm is much lower at between 1 million and 2 million.
At the meeting, Biogen executive Chris Leibman said the company expects uptake will be slow, citing past examples of new drugs for hepatitis C, cancer and autoimmune diseases where adoption five years after approval remained a fraction of the overall patient population.
"This will be a challenge to go anywhere near the numbers described," said Leibman. "We do not believe that there is going to be a large number of patients."
The Alzheimer's Association, which supported Aduhelm's approval, expects about 500,000 patients will receive Aduhelm, according to ICER President Steve Pearson. At $56,000 per year, the total outlay on Aduhelm would reach $28 billion — a number similar to what the Kaiser Family Foundation has estimated.
Uptake will depend heavily on insurance coverage, which for most Alzheimer's patients would be through Medicare. Currently, reimbursement is made through 12 local Medicare contractors, but the federal program has started the process for considering a single national policy.
Until that process completes — the Center for Medicare and Medicaid Services estimates nine months — access is likely to be restricted, said former FDA and CMS head Mark McClellan, at the meeting.
"Right now, and I think for a while, the access is going to be pretty limited, at least until that nine month period is over," he said.
"The numbers that we are going to see are probably going to driven by ... people who can afford more access, or have more access based on where they live and how their particular healthcare provider is set up. But it will probably be pretty limited for the coming months," he added.
In addition to the drug costs, insurers would also need to cover the MRI testing and other brain scans used to diagnose Alzheimer's patients and determine whether they would be good candidates for Aduhelm.
Leslie Fish, a vice president of clinical pharmacy at IPD Analytics, estimated the total cost could approach $100,000. She also questioned how doctors and insurers would determine whether to continue treatment.
"If you have people that really are not the appropriate patients on it, it is going to be really hard to stop them from getting it," said Fish. "A huge huge issue is going to be how we can assess whether it is working or not."
Biogen only tested the drug in patients with mild dementia who were positive for the brain plaques that Aduhelm targets. Last week, the FDA revised its originally broad labeling for the drug to instruct doctors to only treat people in the early stages of the disease.