A Review of Real-World Evidence in Non-Oncology Submissions Made to Four Health Technology Assessment (HTA) Agencies

OBJECTIVES: Health Technology Assessment (HTA) agencies are showing increased interest in real-world evidence (RWE) in support of pivotal trial data with HTA identified evidence gaps within reimbursement submissions. Our objective was to discover how RWE was appraised in non-oncology submissions by select HTA agencies.

METHODS: Reimbursement review reports of non-oncology submissions from Canadian Agency for Drugs and Technologies in Health (CADTH), Institut national d'excellence en santé et services sociaux (INESSS), National Institute for Health and Care Excellence (NICE), Pharmaceutical Benefits Advisory Committee (PBAC) for 2021 and 2022 were reviewed to extract data.

RESULTS: Overall, 11/72 (15%) of submissions to CADTH, 34/141 (24%) of submissions to INESSS, 19/71 of submissions (27%) to NICE, and 23/188 (12%) of submissions to PBAC included RWE in their evidence. Critique of the RWE evidence was provided in 9/11 (82%) of CADTH submissions; 1/34 (3%) by INESSS; 8/19 (42%) by NICE Evidence Review Group; 11/23 (48%) of PBAC submissions. The chief critiques for RWE were non-comparative design, limited follow-up, low sample sizes, and selection bias. Overall, for all submissions that included RWE, 9/11 submissions (82%) for CADTH, 25/34 submissions (74%) for INESSS, 18/19 submissions (95%) for NICE, and 11/23 submissions (48%) for PBAC received a positive reimbursement recommendation. Of these positive recommendations, clinical benefit from the RWE was acknowledged in 7/9 (78%) submissions for CADTH, 15/25 (60%) submissions for INESSS, 12/18 submissions (75%) for NICE, 8/11 submissions (73%) for PBAC. Of the submissions with RWE, 82% of submissions to CADTH, 32% to INESSS, 53% to NICE, and 78% to PBAC were for rare disease indications.

CONCLUSIONS: This review showed that the inclusion of RWE in support of reimbursement submissions is low overall, with the exception of rare disease submissions. RWE inclusion appears to support the clinical benefit assessment of the reviewed therapies.