— Given the risk of side effects and the uncertainty of benefit, ICER assigns a draft rating of “insufficient” to the strength of evidence on whether aducanumab provides a net health benefit for patients —

— Despite significant uncertainty, data from both pivotal trials were merged and used for cost-effectiveness modeling; results showed estimates of small overall health gains commensurate with value-based prices at traditional cost-effectiveness thresholds between $2,500-$8,300 per year; report also provides scenarios with more conservative and optimistic assumptions about the treatment’s efficacy and side effects —

— Results are preliminary and subject to change based on public comment, which ICER is accepting through June 2, 2021 —

BOSTON, May 5, 2021 – The Institute for Clinical and Economic Review (ICER) today released a Draft Evidence Report assessing the comparative clinical effectiveness and value of aducanumab (Biogen) for the treatment of Alzheimer’s disease. An FDA decision on aducanumab is expected in June. This preliminary draft marks the midpoint of ICER’s iterative process of assessing the evidence on aducanumab, and these findings should not be interpreted to be ICER’s final conclusions.

“Nearly everyone has been touched by Alzheimer’s disease through its effects on family members, friends, colleagues, or themselves,” said David Rind, MD, ICER’s Chief Medical Officer. “It is a condition that people dread like no other, and the first therapy that effectively cures or stops the progression of the disease will warrant a very high price in the US health system. However, the clinical trial history and evidence regarding aducanumab are complex, and we believe that at the current time the evidence remains insufficient to determine whether the drug provides an overall health benefit. This is an area of tremendous unmet need, but this treatment has important side effects, and its wide use would have important ramifications for patients and health care budgets, making it all the more important that we know whether a therapy like aducanumab is, or is not, effective.”

Although ICER’s preliminary judgment is that substantial uncertainty remains regarding the health benefits of aducanumab, a base-case cost-effectiveness analysis was performed using the combined results from the two contradictory phase III randomized trials. Results showed estimates of small overall health gains commensurate with value-based prices at traditional cost-effectiveness thresholds between $2,500-$8,300 per year. The report also includes draft calculations for multiple additional scenarios based on differing assumptions about the data on effectiveness of treatment. If the evidence is judged as suggesting no health benefit for aducanumab, then its value-based price would be $0. In the optimistic scenario, which only considers the favorable pivotal trial and disregards the negative trial, the health gains for patients could support an annual price for aducanumab between $11,100-$23,100.

ICER also calculated what a fair price would be for a hypothetical chronic maintenance therapy that halted the progression of dementia but did not cure Alzheimer’s disease. Although the budget impact would be considerable for Medicare and other US payers due to the large potential patient population, such a treatment could be priced between $50,000-$70,000 per year and still meet commonly cited thresholds for long-term cost-effectiveness.

Submit a Public Comment

The Draft Evidence Report and Draft Voting Questions are now open to public comment until 5 PM ET on June 2, 2021. All stakeholders are invited to submit formal comments by email to publiccomments@icer.org. Guidelines for submitting public comments, including formatting specifications, are available on ICER’s website. ICER’s Manufacturer Engagement Guide and Patient Participation Guide provide additional detail on what types of information may be most informative to the report.

ICER will review all comments and incorporate any necessary changes in the Evidence Report and Revised Voting Questions that will be posted on or about June 30, 2021. All comments and ICER’s response to comments will be posted publicly along with the Evidence Report.

Register for the Virtual Public Meeting

Following the public comment period, the revised Evidence Report will be the subject of a virtual public meeting of the California Technology Assessment Forum (CTAF) on July 15, 2021. During the meeting, the independent council will vote on key questions raised in the report. Registration for the virtual public meeting is now open.

Register to Make an Oral Comment 

During the virtual public meeting, there will be a limited amount of time available for interested stakeholders to make an oral comment on the report. Requests to submit oral comments must be emailed to publiccomments@icer.org by 5 PM ET on June 2, 2021. Individuals who wish to deliver oral comments must separately register to attend the meeting. For more information about registering for oral comment, please visit our website.

About ICER

The Institute for Clinical and Economic Review (ICER) is an independent non-profit research institute that produces reports analyzing the evidence on the effectiveness and value of drugs and other medical services. ICER’s reports include evidence-based calculations of prices for new drugs that accurately reflect the degree of improvement expected in long-term patient outcomes, while also highlighting price levels that might contribute to unaffordable short-term cost growth for the overall health care system.

ICER’s reports incorporate extensive input from all stakeholders and are the subject of public hearings through three core programs: the California Technology Assessment Forum (CTAF), the Midwest Comparative Effectiveness Public Advisory Council (Midwest CEPAC), and the New England Comparative Effectiveness Public Advisory Council (New England CEPAC). These independent panels review ICER’s reports at public meetings to deliberate on the evidence and develop recommendations for how patients, clinicians, insurers, and policymakers can improve the quality and value of health care. For more information about ICER, please visit ICER’s website.