Presentation (CTI)

OBJECTIVES: Multiple sclerosis (MS) is a complex neurodegenerative disease, with a range of clinical outcomes used to assess the effectiveness of its treatments. Consequently, outcome selection varies across health technology assessment (HTA) submissions, introducing uncertainty when comparing treatment effects and potentially affecting reimbursement decisions. This study audited recent National Institute for Health and Care Excellence (NICE) appraisals to examine trends in outcome selection and their impact on decision-making.
METHODS: A targeted review of NICE technology appraisals for MS published between 2020 and 2025 was conducted. Clinical effectiveness data and supporting evidence sources were recorded, as well as their role in committee decision-making. Outcomes were categorised as relapse/disease activity, disability progression, radiological outcomes, patient-reported outcomes, safety outcomes, or treatment persistence. Patterns in outcome use and their relationship with appraisal recommendations were assessed descriptively.
RESULTS: Eight appraisals were identified, covering relapsing-remitting (n=6), secondary progressive (n=1), and active relapsing MS populations (n=1). All submissions were supported by randomised controlled trials (RCTs): six were based on two RCTs, one on a single RCT, and one combined head-to-head and single-arm evidence. In all submission, indirect comparisons were used to estimate relative effectiveness. Annualised relapse rate (ARR) was the most common primary endpoint (6/8). Disability progression was the primary endpoint in one appraisal, while patient-reported gastrointestinal tolerability was the primary endpoint in another. External Assessment Group critiques were identified in 4/8 appraisals, primarily relating to limitations in outcome measurement and capture, endpoint selection, and uncertainty in comparative effectiveness. However, committees generally considered the evidence to be sufficient for decision-making.
CONCLUSIONS: MS HTA submissions demonstrate consistent reliance on ARR and disability progression despite heterogeneity in trial endpoints and evidence sources. Greater consistency in clinically meaningful, patient-relevant outcomes, alongside stronger comparative evidence, may reduce uncertainty in future appraisals.