— Concerns about long-term safety influenced majority of independent appraisal committee to vote that evidence is not currently adequate to demonstrate a net health benefit of mavacamten added to background therapy —

— Using estimates from modeling of short-term benefits, mavacamten does show an overall benefit but would have to be priced below $15,000 per year to reach common thresholds for cost-effectiveness —

— Recommendations on appropriate insurance coverage support an evidence-based requirement for patients to be adequately treated with background therapy prior to consideration of mavacamten, but highlight reasons that formal step therapy through disopyramide, septal ablation, or myectomy would not be reasonable —

BOSTON, November 16, 2021 – The Institute for Clinical and Economic Review (ICER) today released a Final Evidence Report assessing the comparative clinical effectiveness and value of mavacamten (MyoKardia and Bristol-Myers Squibb) for the treatment of hypertrophic cardiomyopathy (HCM).

ICER’s report on these therapies was reviewed at the October 2021 public meeting of the CTAF (CTAF), one of ICER’s three independent evidence appraisal committees.

“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 lower rate of side effects than seen with some 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. Additional safety data are needed to resolve these issues.”

Voting on Clinical Effectiveness and Contextual Considerations

  • A majority of panelists found that the evidence is not adequate to demonstrate a net health benefit of mavacamten added to background therapy when compared to background therapy alone.
  • A majority of panelists found that the evidence is not adequate to demonstrate a net health benefit of mavacamten when compared to disopyramide.  

During their deliberations, panel members also weighed the therapies’ other potential benefits, disadvantages, and contextual considerations. For both treatments, voting highlighted the following as particularly important for payers and other policymakers to note:

  • The magnitude of the lifetime impact on individuals living with HCM as an important contextual consideration for any effective therapy for HCM;
  • The effect of mavacamten on patients’ ability to achieve major life goals related to education, work, or family life;
  • The effect of mavacamten on caregivers’ quality of life and/or ability to achieve major life goals related to education, work, or family life
  • Opportunities to improve access to treatment with an oral therapy that does not require access to a center with expertise in myectomy or septal ablation; and
  • Availability of a treatment with different timing and types of risks and benefits reflecting that, while mavacamten does not improve symptoms as much as septal procedures, it also does not carry an immediate short-term risk of death or the need for recovery from a procedure.

Voting on Long-Term Value for Money

Consistent with ICER’s process, the CTAF did not vote on long-term value for money because the manufacturer has not yet announced a price for mavacamten.

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 HBPBs are price ranges 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.

Key Policy Recommendations for Fair Access:

ICER’s independent assessment of value informs the critical decisions that stakeholders across the US health system need to make around pricing and coverage. Following the voting session, a policy roundtable of experts — including clinical experts, patient advocates, and representatives from US payers— convened to discuss the pricing implications and recommendations to ensure fair access. Key recommendations stemming from the roundtable discussion include:

  • Payers should use the FDA label as the guide to coverage policy and engage clinical experts and diverse patient representatives in considering how to address coverage issues for which there is limited or no evidence at the current time.
  • It is reasonable for payers to require an attempt to manage symptomatic HOCM with beta blockers and calcium channel blockers before approving mavacamten.
  • It is unreasonable for insurers to require either myectomy or septal ablation prior to approval of mavacamten.
  • Unless patients have better access to disopyramide, it seems unreasonable to consider requiring a trial of disopyramide prior to coverage of mavacamten. Further clinical evidence on the clinical benefits of disopyramide is also required to strengthen any consideration of this step therapy option.

ICER’s detailed set of policy recommendations, including comprehensive considerations for establishing evidence-based prior authorization criteria, is available in the Final Evidence Report and in the standalone Policy Recommendations document.

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.