Using real-world data (RWD) in health technology assessment (HTA) practice: A comparative study of 5 HTA agencies

Objectives: Reimbursement decisions are conventionally based on evidence from randomised controlled trials (RCTs) which often have high internal validity but low external validity. Real-world data (RWD) may provide complimentary evidence for relative effectiveness assessments (REAs) and cost-effectiveness assessments (CEAs). This study examines whether RWD is incorporated in Health Technology Assessment (HTA) of melanoma drugs by European HTA agencies, differences in RWD use between agencies and across time. Methods: HTA reports published between 01.01.2011 and 31.12.2016 were retrieved from websites of agencies representing 5 jurisdictions: England (NICE), Scotland (SMC), France (HAS), Germany (IQWiG) and the Netherlands (ZIN). A standardized data-extraction form was used to extract information on RWD inclusion for both REAs and CEAs. A panel of senior HTA assessors representing the 5 agencies was consulted to check the robustness of data extracted and interpretation. Results: Fifty-two reports were retrieved. All 52 reports contained REAs; CEAs were present in 25. RWD was included in 28 of 52 REAs (54%); mainly to estimate melanoma prevalence. RWD was included in 22 of 25 (88%) of CEAs; mainly to extrapolate long-term effectiveness and/or identify drug-related costs drugs. Differences emerged between agencies regarding RWD use in REAs; ZIN and IQWiG cited RWD for evidence on prevalence whereas NICE, SMC and HAS additionally cited RWD use for drug effectiveness. No visible trend for RWD use in REAs and CEAs over time was observed. Conclusions: In general, RWD inclusion was higher in CEAs than REAs. It was mostly used to estimate melanoma prevalence in REAs or to predict long-term effectiveness in CEAs. Differences emerged between agencies' use of RWD. However, no visible trends for RWD use over time were observed. Future research should explore the use of RWD in HTA of drugs in other disease indications and in conditional reimbursement schemes