- Biogen would need to drastically lower the price of its new Alzheimer's drug for treatment to be cost effective, according to a revised report from the Institute for Clinical and Economic Review, which affirmed the influential nonprofit group's earlier conclusion that the company's clinical trial evidence is insufficient to prove a health benefit.
- Biogen priced Aduhelm, as the drug is now called, at an average list cost of $56,000 a year. Assuming a level of efficacy suggested by blending results from the company's two Phase 3 trials, one of which failed, ICER calculated treatment would be cost effective only at prices between about $3,000 and $8,400, a reduction of 85% to 95%.
- ICER plans to convene a group of stakeholders, including Biogen, the Alzheimer's Association and a former Food and Drug Administration commissioner, to further discuss the company's high price, which is expected to balloon Medicare's budget for drugs administered by doctors.
ICER's previous draft assessment of Aduhelm was published in May, before the FDA controversially cleared the closely watched drug and Biogen picked a price higher than even some on Wall Street had expected.
The revised report is little changed, but reiterates the group's skepticism of the data marshaled by Biogen and FDA in support of Aduhelm.
"After months of delving into the data, and working with patient groups, clinical experts, and the manufacturer to gain their perspectives, our judgment remains that the evidence on aducanumab is insufficient to be able to demonstrate that patients get benefits that would outweigh the risks and harms of this treatment," said David Rind, ICER's chief medical officer, in a Wednesday statement. Aducanumab is the non-branded name for Aduhelm.
In its report, ICER acknowledged that the conflicting trial results for Aduhelm could be due, as Biogen and FDA primarily argue, to differences in exposure to what's thought to be the most effective drug dose. But the group thinks other explanations are "equally or more likely," including the possibility that the positive findings from one trial are due to chance.
Even if that's not the case, ICER doesn't view the modest benefit shown in that study as worth the $56,000 a year that Aduhelm will cost on average. The group's suggested range of $3,000 to $8,400 is based on different cost thresholds for valuing what is known as a "quality-adjusted life year," or QALY.
Drugmakers and some patient groups have vigorously disputed ICER's approach to assessing cost-effectiveness in the past. In comments on the draft Aduhelm report, Biogen as well as Eli Lilly and Roche, both of which are developing other Alzheimer's treatments, criticized some of ICER's decisions in calculating Aduhelm's benefits and the impact of the disease.
The Alzheimer's Association, a leading patient advocacy group and a vocal advocate for Aduhelm, also raised numerous objections to ICER's conclusions, suggesting the drug could provide "years of positive benefits" and comparing it to AZT, the first therapy approved for HIV.
The cost-effectiveness estimate calculated by ICER in the revised report is slightly higher than in the draft report from May, a change mainly due to the FDA requiring fewer brain MRIs alongside treatment than Biogen did in clinical testing.
The group also added some discussion of the broad label granted by FDA for Aduhelm, which does not limit use to only those patients whose disease is in early stages. The trial excluded patients with more moderate or severe Alzheimer's, as they are not expected to benefit from a treatment targeted at the disease's characteristic brain plaques.
For those patients, ICER thinks it's "reasonably likely" that Aduhelm's side effects outweigh any potential benefit.