— Given current data, AMX0035 is comparable or better to standard of care; AMX0035 would achieve common thresholds for cost-effectiveness if priced between $9,100 – $30,600 per year —
— Evidence indicates oral edaravone is comparable or incrementally better to standard of care; treatment would achieve common thresholds for cost-effectiveness if priced between $1,400-$3,200 per year —
— At the August 19 virtual public meeting, ICER’s independent appraisal committee will review the evidence, hear further testimony from stakeholders, and deliberate over these treatments’ comparative clinical effectiveness, other potential benefits, and long-term value for money —
BOSTON, August 4, 2022 – The Institute for Clinical and Economic Review (ICER) today posted its revised Evidence Report assessing the comparative clinical effectiveness and value of AMX0035 (Amylyx Pharmaceuticals, Inc.) and oral edaravone (Mitsubishi Tanabe Pharma Development America, Inc.) for the treatment of amyotrophic lateral sclerosis (ALS).
“There is tremendous need for new therapies in ALS,” said David Rind, MD ICER’s Chief Medical Officer. “Current treatment of this devastating disease is largely focused on supportive care. AMX0035 appears to extend life and modestly slow disease progression, but we expect vigorous debate at the public meeting on the strength of evidence, which hinges largely on the findings from a single trial, particularly given the negative vote by the FDA Advisory Committee. We also anticipate a policy discussion on whether, should this drug receive FDA approval, the degree of uncertainty should be reflected in a price scaled closer to the cost of production pending completion of trials that can confirm the magnitude of the benefit for patients. In this report we also evaluated the evidence on an oral formulation of edaravone. The oral version is much less burdensome to patients than the intravenous formulation, which is likely to lead to increased use, but the price of edaravone appears too high to align reasonably with the benefits it provides.”
This Evidence Report will be reviewed at a virtual public meeting of the Midwest CEPAC (Midwest CEPAC) on August 19, 2022. The Midwest CEPAC is one of ICER’s three independent evidence appraisal committees comprising medical evidence experts, practicing clinicians, methodologists, and leaders in patient engagement and advocacy.
A draft version of this report was previously open for a four-week public comment period. The updated Evidence Report and voting questions reflect changes made based on comments received from patient groups, clinicians, drug manufacturers, and other stakeholders. Detailed responses to public comments can be found here.
Key Clinical Findings
The evidence suggests that AMX0035 provides a small to substantial benefit in ALS, but there remains substantial uncertainty about this estimate. We therefore rate AMX0035 added to standard of care as comparable or better compared to standard of care alone (“C++”).
Oral edaravone is much less burdensome than the intravenous formulation. The evidence on its effectiveness is limited to a subpopulation of patients with ALS but the drug is labeled broadly for all patients with ALS. For patients who meet the narrowly defined criteria of the clinical trials, we rate oral edaravone added to standard of care to provide a comparable or incremental added net health benefit compared to standard of care alone (“C+”). However, for patients who do not meet these criteria, we rate the evidence to be insufficient (“I”).
Key Cost-Effectiveness Findings
AMX0035 is not yet approved by the FDA, and its manufacturer has not yet announced what the treatment’s US price will be if approved. ICER’s health-benefit price benchmark (HBPB) range for AMX0035 is between $9,100 – $30,600 per year. If priced similarly to IV edaravone at approximately $169,000 per year, the incremental cost effectiveness of AMX0035 based on the clinical data available to date would far exceed typical cost-effectiveness thresholds.
The incremental cost effectiveness of oral edaravone at an annual price of $171,000 per year far exceeds typical cost-effectiveness thresholds across multiple analyses. ICER’s health-benefit price benchmark range for oral edaravone is between $1,400 – $3,200 per year.
ICER’s HBPB is a price range suggesting the highest US price a manufacturer should charge for a treatment, based on the amount of improvement in overall health patients receive from that treatment, when a higher price would cause disproportionately greater losses in health among other patients in the health system due to rising overall costs of health care and health insurance. In short, it is the top price range at which a health system can reward innovation and better health for patients without doing more harm than good.
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.