Sotatercept added to background therapy appears to improve clinical outcomes, but there are uncertainties on long term efficacy and safety; sotatercept would achieve common thresholds for cost-effectiveness if priced between $18,700 -$36,200 per year  —

    BOSTON, November 14, 2023 – The Institute for Clinical and Economic Review (ICER) today posted its revised Evidence Report assessing the comparative clinical effectiveness and value of sotatercept (Merck & Co) for pulmonary arterial hypertension (PAH). 

    “Pulmonary arterial hypertension is a progressive disease that can lead to debilitating shortness of breath, fatigue, and lightheadedness as the heart struggles to pump blood through the lungs,” said ICER’s Chief Medical Officer, David Rind, MD. “Current drug treatments are primarily combinations of vasodilators, and these can be very burdensome and cause significant side effects. Sotatercept has a novel mechanism of action and it is administered subcutaneously every few weeks, which reduces burdens. Current evidence suggests that sotatercept has fewer side effects and improves short-term outcomes, but we have some uncertainties about long-term efficacy and safety.”

    This Evidence Report will be reviewed at a virtual public meeting of the Midwest CEPAC (Midwest CEPAC) on December 1, 2023. 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

    Based on the currently available data, sotatercept added to background therapy appears to improve clinical outcomes for patients with PAH. It has a less burdensome delivery system than many other current PAH treatments.

    However, uncertainty remains about sotatercept’s efficacy in sicker populations, and in those with connective tissue disease, and about the durability of effect. In the absence of longer-term data, we necessarily have uncertainties about sotatercept’s effects on mortality and concerns about as-yet-undetected adverse effects.  Therefore, we have moderate certainty of a small to substantial net health benefit, with a high certainty of at least a small net health benefit, corresponding to an ICER Evidence Rating of B+.

    Key Cost-Effectiveness Findings

    Sotatercept has not yet been approved by the FDA, and the manufacturer has not announced a US price if approved. ICER has calculated a health-benefit price benchmark (HBPB) for sotatercept to be between $18,700 -$36,200 per year.

    ICER’s HBPB is a price range suggesting the highest US price a manufacturer should charge for a treatment, net of all rebates and discounts, 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.