OBJECTIVES: Randomized controlled trials (RCTs) are the gold standard evidence for reimbursement decisions by health technology assessment (HTA) bodies. However, the inclusion and acceptance of RWE to support HTA submissions has increased. This study assesses how RWE is used to support HTA submissions over a 3-month period across 4 HTA agencies.

METHODS: HTA submissions published between 8^th March-8^th June 2023 were retrieved from 5 HTA bodies: NICE (England), SMC (Scotland), HAS (France) and IQWiG and G-BA (Germany). Data regarding use of RWE data sources were extracted, along with appraisal decisions.

RESULTS: In total, 90 HTA appraisals were identified. RWE was not utilized in any of the German HTA submissions, with only RCT and single arm trial data being utilized for clinical and economic assessment. Of the 20 submissions to NICE, RWE was utilized in 3 to support clinical effectiveness, with 2 of these also utilizing RWE to support economic model inputs. Of the 15 SMC submissions, RWE was utilized to support clinical effectiveness and economic assessment in 2 submissions, both of which presented single arm trial data as primary evidence. Of the 25 HAS submissions, RWE was used to support clinical effectiveness in 6 appraisals, of which three appraisals presented RCT data and 3 presented single-arm trial data as primary evidence.

CONCLUSIONS: In this 3-month period the inclusion of RWE differed amongst European HTA agencies, with no submissions to German HTA utilising RWE. In cases where RWE was utilised, data was used to support clinical and economic evidence in addition to primary evidence from RCT and single arm trial data.