OBJECTIVES: In the past 15 years there have been 17 treatments in multiple sclerosis (MS) assessed by the National Institute for Health and Care Excellence (NICE), across different types of MS. NICE recommendations can often result in different reimbursed populations to the label population. We investigated these differences and why there was variation in NICE recommendations in MS.

METHODS: A targeted review of completed NICE technology appraisals in MS (Jan 2007-June 2021) was conducted. Data on European Medicines Agency (EMA) marketing authorisation, clinical trial populations, NICE recommendations, sub-group analyses and lines of therapy were extracted.

RESULTS: 13 NICE appraisals were identified, which assessed 17 treatments (including ocrelizumab twice). These included 12 STAs and 1 MTA. For 9/17 (53%) of the treatments, there was a difference between the label population and the NICE-recommended population. Of these, 4 had highly active disease (HAD) and/or rapidly evolving severe (RES) subgroup exclusions versus label, 3 had a broader MS label but were restricted to relapsing-remitting MS only, 1 was restricted to patients only when another therapy was contraindicated, and 1 treatment was restricted to the RES subgroup and use in second-line. In one instance, the company proposed a restriction but this was rejected by NICE. In 3 appraisals, clinical experts stated there are limitations to the MS categorisations used in NICE appraisals, and that these can be difficult to apply in clinical practice.

CONCLUSIONS: Variation exists between label populations and NICE-recommended populations in prior MS appraisals. Clinical input during these appraisals suggests that these variations may impact clinical decision-making. Further research is required to understand to what extent NICE-optimised recommendations are relevant in clinical practice, and if changes to the way NICE assesses MS drugs are required to reflect the complexity of the UK MS treatment pathway and disease presentation.