OBJECTIVES: For health technology assessment (HTA) bodies that make reimbursement decisions, real-world evidence (RWE) is becoming more important in understanding lifelong diseases like multiple sclerosis (MS), as randomized controlled trials provide limited insights in the short term. RWE includes data from sources such as registries, observational studies and electronic health records. This analysis aims to assess the value of RWE in recent HTA appraisals for MS.

METHODS: Published HTA appraisals between 2018 and 2022 in MS were evaluated across four leading HTA bodies: the National Institute for Health and Care Excellence (NICE), the Scottish Medicines Consortium (SMC), the Canadian Agency for Drugs and Technologies in Health (CADTH) and the Pharmaceutical Benefits Advisory Committee (PBAC). RWE and reimbursement data were extracted from these appraisals.

RESULTS: 25 HTA appraisals were relevant to review criteria, and RWE data were presented in 18 of these appraisals (72%). RWE data were taken from registries and observational studies. Of these 18 appraisals, 16 (88.8%) were accepted by HTA bodies, including seven appraisals from NICE, five from SMC and four from CADTH. In these 16 accepted appraisals, RWE was used in 81.2% of submissions in economic models as a data source majorly for natural history of disease (including transition probabilities for health states). It was also used for deriving health state utilities (from EDSS scores) and for mortality rates. RWE was also presented as clinical evidence in 37.5% of these submissions, reflecting on natural history of MS.

CONCLUSIONS: For multiple sclerosis appraisals, HTA bodies have encouraged and accepted RWE as supportive data for economic models and clinical evidence for the natural history of the disease, which has been poorly understood otherwise. As managing MS is associated with high costs, using RWE data can help patient assessment in clinical practice, and can potentially accelerate patient access to care.