— Independent appraisal committee votes that evidence is adequate to demonstrate siponimod offers superior effectiveness for patients with active SPMS, but not inactive SPMS, compared to best supportive care —

— Policy roundtable discussion highlights siponimod’s evidence limitations, its similarity to fingolimod, and its high price, suggesting that siponimod does not merit a unique role in therapy —

BOSTON, June 20, 2019 – The Institute for Clinical and Economic Review (ICER) today released a Final Evidence Report and Report-at-a-Glance 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 just for patients with SPMS (both active and non-active), which was the population studied in the phase III trial.

ICER’s report was reviewed at the May 2019 public meeting of the Midwest Comparative Effectiveness Public Advisory Council (Midwest CEPAC), one of ICER’s three independent evidence appraisal committees. During the meeting, the majority of the panel voted that the evidence demonstrated siponimod to be clinically superior to best supportive care for patients with active SPMS, though some panel members noted that they still had concerns about trial methodology. Panel members unanimously found that the evidence was insufficient to demonstrate that siponimod is superior to best supportive care for patients with non-active SPMS.

“There is a large unmet need for effective treatments for progression in SPMS,” said David Rind, MD, ICER’s Chief Medical Officer. “Like other disease modifying therapies (DMTs), including the related drug fingolimod, siponimod has demonstrated the ability to reduce relapses in patients with relapsing forms of MS, but siponimod has not been proven to affect the disability progression independent of relapses that is the devastating hallmark of SPMS. Additionally, it is unfortunate that Novartis chose a price that is so far out of line with siponimod’s benefits to patients with active SPMS, particularly with it entering a crowded field of DMTs.”

Economic analyses assessing long-term cost-effectiveness found that, when compared to best supportive care 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.

Key Policy Recommendations

Following the voting session, a policy roundtable of experts – including clinicians, patient advocates, and manufacturer and payer representatives – convened to discuss the implications of the evidence for policy and practice. Key recommendations stemming from the roundtable discussion include:

  • To provide fair value to patients and the health system, the manufacturer should lower the price of siponimod so it aligns with the added value it brings to patients.
  • Evidence and clinical testimony suggested that siponimod does not have a unique role in therapy for any phenotype of MS, including active SPMS. Given its similarities to fingolimod, siponimod should be considered amongst a group of highly effective disease modifying therapies (DMTs) for relapsing forms of MS, including fingolimod, alemtuzumab, natalizumab, and ocrelizumab.
  • Payers may wish to specifically consider granting preferential formulary status to fingolimod when its generic formulation comes to the market.
  • Patient organizations should view their longer-term mission in support of patients to include active engagement with manufacturers to demand reasonable value-based pricing of the therapies that patients and their families helped bring to the market.

ICER’s detailed set of policy recommendations, including considerations for establishing prior authorization criteria, is available in the Final Evidence Report.

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.