OBJECTIVES

: Primary-progressive multiple sclerosis (PPMS) is a chronic neurodegenerative disease of the central nervous system. In the UK, ocrelizumab is the only approved disease-modifying treatment. Ocrelizumab is available on the English NHS via a simple patient access scheme, following NICE guidance published in June 2019. This analysis aimed to identify cost and efficacy profiles that an emerging hypothetical treatment in PPMS would require to be considered cost-effective versus best supportive care (BSC) from an English NHS perspective.

METHODS

: A cohort-based Markov model was constructed using a lifetime horizon to estimate costs and health outcomes for PPMS patients treated with BSC and a hypothetical emerging treatment. The model structure was based on the ocrelizumab NICE submission, with health states representing different levels of disability, measured by EDSS, and with treatment influencing outcomes by slowing confirmed disability progression (CDP).

Clinical data, along with cost and utility inputs, were obtained from the literature, HTA appraisals and relevant UK databases. Two-way sensitivity analysis was used to explore cost and efficacy (defined as the hazard ratio for CDP) profiles for the emerging treatment and one-way sensitivity analysis was used to identify key drivers of cost-effectiveness.

RESULTS

: For an emerging treatment with HRs of 0.8, 0.6, and 0.4, the maximum annual treatment cost at a willingness-to-pay threshold of £30,000 was found to be £2,486, £5,161 and £7,934, respectively. The key drivers of cost-effectiveness were annual treatment cost, treatment efficacy, and health state utility values.

CONCLUSIONS

: Using established PPMS modelling approaches a range of cost-effective profiles were identified for a hypothetical emerging treatment. The analysis suggests that a commercial arrangement would likely be required for an emerging treatment to be considered cost-effective from an English NHS perspective. Drivers of cost-effectiveness in this PPMS analysis were found to be consistent with those detailed in the RRMS literature.