— There are significant uncertainties regarding long term efficacy of tolebrutinib; ICER estimates that treatment would achieve common thresholds of cost-effectiveness if priced between $3,250 and $5,900 per year —
BOSTON, May 29, 2025 – The Institute for Clinical and Economic Review (ICER) today posted its revised Evidence Report assessing the comparative clinical effectiveness and value of tolebrutinib (Sanofi) for the treatment of secondary progressive multiple sclerosis (SPMS).
“There is a large unmet need for effective treatments for progression in non-relapsing SPMS, particularly with the non-active type,” said ICER’s Senior Vice President of Research, Foluso Agboola, MBBS, MPH. “Clinical trial evidence suggests that tolebrutinib slows disease progression in patients with non-relapsing SPMS; however, there are still uncertainties about the balance of its overall efficacy and adverse events. Longer-term data will help define the overall net health benefit of tolebrutinib.”
This Evidence Report will be reviewed at a virtual public meeting of the CTAF on June 13, 2025. 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
Available data demonstrate that tolebrutinib slows disease progression in non-relapsing SPMS. However, there was a lack of improvement in some additional outcomes, such as brain atrophy and the nine-hole peg test, a measure of upper extremity function. This inconsistency in the direction of outcomes is an area of uncertainty. Additionally, there is a small but non-trivial risk of severe liver toxicity with tolebrutininb; though this risk may be mitigated by weekly monitoring of liver function tests, such intensive monitoring may not be a realistic expectation in clinical practice.
Given the above uncertainties, ICER rated the current evidence on tolebrutinib as promising but inconclusive (“P/I”) for individuals with non-relapsing SPMS.
Key Cost-Effectiveness Findings
Tolebrutinib has not yet been approved by the FDA for SPMS, and the manufacturers have not yet announced a US price for the therapy if approved.
ICER has calculated a health benefit price benchmark (HBPB) to be between $3,250 and $5,900 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 conducts evidence-based reviews of health care interventions, including prescription drugs, other treatments, and diagnostic tests. In collaboration with patients, clinical experts, and other key stakeholders, ICER analyzes the available evidence on the benefits and risks of these interventions to measure their value and suggest fair prices. ICER also regularly reports on the barriers to care for patients and recommends solutions to ensure fair access to prescription drugs. For more information about ICER, please visit www.icer.org.