OBJECTIVES: Real-world data (RWD) has become an important source of evidence to support payer decision-making during health technology assessment (HTA). RWD is increasingly reviewed by HTA bodies as supporting evidence for effectiveness and safety of interventions. The aim is to identify and describe the extent published RWD evidence is being reviewed as part of systematic literature reviews (SLRs) for HTA.

METHODS: A retrospective descriptive analysis was performed using IQVIA’s HTA Accelerator (HTAA). All HTA reports published between January 2013 and May 2022 from six HTA bodies (NICE, HAS, SMC, G-BA, IQWiG and EUnetHTA) were reviewed for inclusion of RWD in SLRs.

RESULTS: Overall 450 HTA reports were included for the analysis using RWE keywords (e.g. observational, cohort and retrospective etc.) in type of evidence evaluated field in HTAA. Of 450, 13.5% (61) HTA reports included published RWD studies in submitted SLRs across four HTA bodies, with highest number for NICE 64% (39) followed by HAS 21% (13), EUnetHTA 11% (7) and IQWiG 3% (2). In these submissions, 48% (29) were original, 25% (15) were resubmissions and 20% (12) were indication extensions. Overall, 40% of identified HTA reports represents oncology indications followed by endocrine/metabolic disorders, infectious diseases, and immune system (16%, 10% and 7%, respectively). Over the last 5 years, an increase in RWD use in SLRs for HTA submissions was observed, from 6.5% in 2017 to 22% in 2021. Around 77% (47/61) received positive recommendations with top two being HAS with 100% (13/13) and NICE with 87% (34/39) acceptance.

CONCLUSIONS: Our analysis illustrates an increasing trend in reviewing published RWD evidence for HTA. As HTA bodies show an increasing appreciation and acceptable of RWD evidence, a review of published RWD will increasingly be an integral part of SLRs in future HTA submissions. An assessment of its impact on the final recommendation deserves further attention.