PRO121 THE USE OF REAL-WORLD DATA IN HEALTH TECHNOLOGY ASSESSMENT OF MEDICATIONS FOR RARE DISEASES

Abstract

Given the scarcity of data to characterize the burden of rare diseases and the efficacy and safety of their treatment, health-services researchers and decision-makers are advocating for the consideration of real-world data (RWD) – data collected outside of randomized controlled trials – in health technology assessment (HTA). However, how frequently these are being used and their impact is unclear. This study characterizes the use of RWD in the HTA of medications for rare diseases appraised by the National Institute for Health and Care Excellent (NICE). Orphanet was used to identify rare diseases (prevalence of 10 to 50 per 100,000). Those with NICE technology appraisals for medications published between 2010-2019 were selected for data extraction and analysis. The percentage of appraisals including RWD was assessed, and whether submissions received favorable recommendations was tabulated. RWD were classified according to type; and whether they provided support for clinical effectiveness, cost effectiveness, safety and tolerability. Trends over time were examined. Twenty-two of 205 rare diseases (10.7%) had published appraisals in NICE, corresponding to 74 individual appraisals. Overall, 26 (35.1%) submissions included RWD; they were included in submissions for 39.7% of recommended products and 9.1% of those not recommended. Almost all (96.2%) published appraisals using RWD had a favorable recommendation. Most frequently (75.9%) RWD were derived from observational studies (for non-comparative evidence of clinical effectiveness), with a few submissions presenting preference or quality-of-life data (which contributed to cost-effectiveness evidence). Over time, the inclusion of RWD increased, with 18 (69.2%) submissions including RWD between 2015-2019 compared to eight (30.8%) between 2010-2014. The incorporation of RWD in NICE HTA submissions for medications for rare diseases is increasing; and among published appraisals where RWD were included, the majority had a favorable recommendation. How RWD are being incorporated in submissions in other HTA jurisdictions remains unclear.