WHAT'S THE REALITY OF REAL-WORLD EVIDENCE IN HEALTH TECHNOLOGY APPRAISAL?
Author(s)
Sarri G, Lambrelli D, Gulea C, GomezEspinosa E, Iheanacho I
Evidera, London, UK
OBJECTIVES: How real-world evidence (RWE) can contribute to assessment of product value is increasingly recognised. However, there is lack of formal guidance on using such data in health technology appraisal (HTA) for reimbursement. Against this background, we conducted two systematic literature reviews (SLRs) to explore expert opinion on using RWE in HTA (SLR 1) and on what forms of RWE could meet HTA requirements (SLR 2). METHODS: Following PRISMA guidelines, MEDLINE and Embase were systematically searched for English-language publications indexed between 2012 and 2017 to conduct the two SLRs, with supplementary searches of HTA-body websites without time limits. RESULTS: Limited HTA guidance was found, and this focused on analytical methods for comparative individual-patient data from RWE. Of 200 references screened in SLR1, 20 were considered relevant. Most of these commented that, to date, RWE’s role in HTA has been to help address gaps in randomised-controlled-trial (RCT) evidence or to validate trial-based results (e.g., from single-arm studies in conditions with poor prognosis). Of 300 references screened for SLR2, 40 were relevant. These publications focused on statistical techniques to control for selection bias and adjust for confounder effects in RWE (e.g., adjusted survival curves, propensity modelling, inverse probability-of-censoring weighted correction, and machine-learning techniques). Additionally, a few studies suggested RWE could offer advantages in network meta-analysis, through enabling connection of networks and increasing sample size and generalizability of results. Experts’ main concerns about using RWE related to selection of the most appropriate data sources, and the validity of exchangeability assumptions where RCT data and RWE are combined. CONCLUSIONS: While RCTs remain the cornerstone in assessing comparative efficacy of products in HTA, RWE is increasingly acknowledged as a valuable information source. Further decision and methodological frameworks are needed to offer clear guidance on when and how RWE should be incorporated into HTA submissions.
Conference/Value in Health Info
2017-11, ISPOR Europe 2017, Glasgow, Scotland
Value in Health, Vol. 20, No. 9 (October 2017)
Code
PRM235
Topic
Study Approaches
Disease
Multiple Diseases