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BOSTON, July 15, 2025 – The Institute for Clinical and Economic Review (ICER) today released a Final Evidence Report assessing the comparative clinical effectiveness and value of for the treatment of tolebrutinib (Sanofi) for the treatment of secondary progressive multiple sclerosis (SPMS).
ICER’s report on this therapy was the subject of the June 2025 public meeting of the CTAF, one of ICER’s three independent evidence appraisal committees.
Downloads: Final Evidence Report | Report-at-a-Glance | Policy Recommendations
“There is a large unmet need for effective treatments 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, as reflected in the voting results of the independent appraisal committee at ICER’s public meeting. Longer-term data will help define the overall net health benefit of tolebrutinib.”
ICER’s Virtual Public Meeting: Voting Results on Clinical Effectiveness and Contextual Considerations
ICER assessed, and the independent appraisal committee voted on the evidence for the net health benefit of tolebrutinib in adults with non-relapsing secondary progressive multiple sclerosis:
- A majority of the panelists (12-1) found that current evidence is not adequate to demonstrate a net health benefit of tolebrutinib when compared to best supportive care (defined as pharmacological and non-pharmacological treatments to alleviate the symptoms of multiple sclerosis).
Panel members also weighed potential benefits and disadvantages beyond the direct health effects and weighed special ethical priorities. Voting highlighted the following as particularly important for payers and other policymakers to note:
- There is substantial unmet need despite currently available treatments.
- Multiple sclerosis is of substantial relevance for people from a racial/ethnic group that has not been equitably served by the healthcare system.
ICER’s Virtual Public Meeting: Voting Results on Long-Term Value for Money
Tolebrutinib has not yet been approved by the FDA, and the manufacturer has not announced a US price if approved.
Consistent with ICER’s process, because there is no firm estimate yet of a potential launch price for the treatment, the panel did not take a vote on the treatment’s long-term value for money.
ICER has calculated a health benefit price benchmark (HBPB) to be between $3,250 and $5,900 per year.
Key Policy Recommendations
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, patients and patient advocates, the manufacturer, 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:
- Trial inclusion criteria are a reasonable starting point for developing coverage policies for tolebrutinib to identify patients with SPMS. Payers should engage clinical experts and patient representatives in considering how to address coverage requests for which there is limited or no evidence at the current time.
- Manufacturers have a responsibility to release clinical trial data in a timely manner such that all relevant data is available to payers, clinicians, and patients prior to the time of FDA approval to facilitate timely coverage and treatment decisions.
- Clinical specialty societies should endeavor to facilitate the education of general neurologists about SPMS diagnosis and treatment, including education to help clinicians make more prompt diagnoses of SPMS, improve standard of care treatment for SPMS, and understand the efficacy and safety of new therapies such as tolebrutinib.
ICER’s detailed set of policy recommendations is available in the Final Evidence Report and in the standalone Policy Recommendations document.
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