ICER Publishes Evidence Report on Mavacamten for Hypertrophic Cardiomyopathy

— Evidence is rated as promising but inconclusive to determine whether mavacamten provides a net health benefit for patients over the long term; using patient outcomes from short-term studies, treatment would achieve common thresholds for cost-effectiveness if priced lower than $15,000 per year —

— At October 22 virtual meeting, ICER's independent appraisal committee will review the evidence, hear further testimony from stakeholders, and deliberate over mavacamten's comparative clinical effectiveness, other potential benefits, and long-term value for money —

BOSTON, October 7, 2021 - The Institute for Clinical and Economic Review (ICER) today posted its revised Evidence Report assessing the comparative clinical effectiveness and value of mavacamten (MyoKardia and Bristol-Myers Squibb) for the treatment of hypertrophic cardiomyopathy (HCM).

"Hypertrophic cardiomyopathy is a genetic disorder of the heart muscle that can cause shortness of breath and chest pain, and also arrhythmias that can result in strokes or even sudden death," said David Rind, MD, ICER's Chief Medical Officer. "The evidence suggests that mavacamten may deliver important health benefits for patients with a low rate of side effects seen with other medications for HCM, but clinical experts differ in their opinions about the long-term clinical implications of mavacamten reducing left ventricular ejection fraction in some patients. This balance between potential benefits and harms will be a key component of the deliberations during our public meeting."

This Evidence Report will be reviewed at a virtual public meeting of the California Technology Assessment Forum (CTAF) on October 22, 2021. The CTAF 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

ICER rated the evidence promising but inconclusive ("P/I") to determine if mavacamten added to first-line therapy provides a net health benefit over first-line therapy care alone or the addition of disopyramide. The clinical trial was well designed with concordant outcomes; however, some clinical experts are concerned that the reduction in left ventricular ejection fraction (LVEF) seen in some patients may lead to long-term harm rather than benefit.

ICER did not provide a formal evidence rating comparing mavacamten to septal reduction therapies (i.e., alcohol septal ablation and myectomy) given the likelihood that the different timing of risks and benefits of these options would lead to diverse individual patient preferences. Septal reduction procedures can rapidly improve symptoms in many patients, but can also sometimes result in death.

Key Cost-Effectiveness Findings

Mavacamten is not yet approved by the FDA, and its manufacturers have not yet announced what the treatment's US price would be if approved. Despite uncertainties about the longer-term risks of reduction in LVEF for some patients, modeling the impact of short-term clinical benefits over a longer time period produces a health-benefit price benchmark (HBPB) for mavacamten between $12,000-$15,000 per year, significantly lower than the $75,000 annual price that some industry analysts have forecasted.

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.

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.

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