OBJECTIVES: RWE is gaining momentum where obtaining evidence from randomized controlled trials is challenging or sparse. Typically, RWE is used to support RCT findings to increase precision and enhance decision-making process for HTA bodies and payers. Thus, these bodies are increasingly accepting RWE in decision making. The objective of this research was to identify HTA guidance updates on RWE usage and analyzing trends in reimbursement decisions involving RWE since start of 2023.

METHODS: Hand searching of HTA websites (UK-NICE, Canada-CADTH, France-HAS, Germany-IQWiG, Belgian-KCE) and grey literature was undertaken to retrieve latest guidance and publications on RWE usage in HTA decision making during 2023.

RESULTS: The search identified a guidance document from CADTH and HAS position paper. CADTH provided guidance on reporting of RWE (12 sections covering, research question & study design; setting & context; data specifications, access, cleaning method linkages; data sources, data dictionary and variables; participants; exposure definitions & comparators; outcomes; bias confounding and effect modifiers or sub-group effects; statistical methods; study findings; interpretation and generalizability; and limitations) and recommendations on submission preparation to both regulatory and HTA agencies. CADTH launched this guidance for faster patient access to newer technologies while resolving issues of uncertainty. HAS paper suggested use of RWE as external control arm in absence of RCTs and conducting pragmatic trials (trials within a cohort, registry-based RCT, contactless trial or direct-to-patient trial). RWE usage in HTA submissions showed upward trend with 12/38 NICE and 19/33 CADTH submissions referring to RWE while appraising technologies. Most of these (9/12, NICE submissions; 18/19 CADTH submissions) received positive outcome from agencies.

CONCLUSIONS: RWE usage in HTA submissions is increasing with availability of recently published guidance to generating best-practices for planning, conducting, and reporting of RWE. Early engagement and further collaboration between HTA agencies and industry is warranted.