— Midwest CEPAC to deliberate on the treatment’s clinical effectiveness and other potential benefits during its May 23 public meeting —

BOSTON, May 2, 2019 – The Institute for Clinical and Economic Review (ICER) today released an Evidence Report assessing the comparative clinical effectiveness and value of siponimod (Mayzent™, Novartis) for the treatment of secondary progressive multiple sclerosis (SPMS).

Siponimod was recently approved by the FDA for the treatment of relapsing forms of multiple sclerosis (MS), including active SPMS. However, ICER’s assessment focuses on the clinical and cost-effectiveness of siponimod for all patients with SPMS (both active and non-active), which was the population studied in the phase III trial.

“Progression occurring independent of episodes of relapse is the hallmark of progressive MS, and while a number of therapies for MS have efficacy in reducing relapses, few have shown benefit in slowing this independent progression,” said David Rind, MD, ICER’s Chief Medical Officer. “While the FDA did not approve siponimod for non-active SPMS, we felt it remained important to review the data assessing siponimod’s potential benefit in all SPMS, including in those patients for whom disease mainly involves progression rather than relapses.”

This Evidence Report will be reviewed at an upcoming public meeting of the Midwest Comparative Effectiveness Public Advisory Council (Midwest CEPAC), in Rosemont, IL on May 23, 2019. This two-topic meeting will also cover ICER’s review of esketamine for treatment-resistant depression.

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.  ICER previously reviewed disease-modifying therapies for relapsing-remitting and primary progressive forms of MS in 2017.

Key Findings

In March 2019, the FDA approved siponimod “to treat adults with relapsing forms of multiple sclerosis (MS), to include clinically isolated syndrome, relapsing-remitting disease, and active secondary progressive disease.” The FDA also explained that “Active SPMS is one of the relapsing forms of MS, and drugs approved for the treatment of relapsing forms of MS can be used to treat active SPMS.” This latter statement, in particular, clarified that many therapies currently approved for relapsing forms of MS are approved for active SPMS.

Given that much of the interest in siponimod has been due to its potential efficacy in both active and non-active SPMS, the ICER report focuses on its effectiveness in all patients with SPMS. Although the degree to which siponimod delays progression independent of its effect on relapse activity remains uncertain, evidence provides high certainty that siponimod provides at least a small net benefit in patients with active SPMS when compared to best supportive care. Evidence provides moderate certainty that siponimod has comparable or potentially better net benefit versus best supportive care in patients with non-active SPMS, with a small possibility of overall net harm.

Economic analyses assessing long-term cost-effectiveness found that, when compared to best supportive care, siponimod at its current price far exceeds commonly cited thresholds of $50,000-$150,000 per quality-adjusted life year (QALY) gained, as well as the alternative thresholds of $50,000-$150,000 per life year gained (LYG). For the overall SPMS trial population, siponimod is estimated to cost $1.15 million per QALY and $3.76 million per LYG. For the active SPMS sub-population, siponimod is estimated to cost $433,000 per QALY and $1.57 million per LYG. ICER did not model the cost-effectiveness of siponimod for the treatment of relapsing-remitting MS.

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.