Abstract

Real-world evidence (RWE) is increasingly used to support value and ensure timely access to new technologies. A systematic literature review (SLR) was undertaken to assess the use of RWE in key areas of selected oncology indications across six different HTA agencies. Single technology assessments (STAs) evaluating pharmacological interventions published by NICE were searched in accordance with Preferred Reporting Item for Systematic Reviews and Meta-Analyses (PRISMA) guidelines to identify appraisals in oncology published between January 2016 and November 2020. RWE use within the company submission was assessed in key areas: target population, comparative effectiveness, economic model parameters, healthcare resource utilization (HCRU), and early access programs. Reception of RWE by HTA agencies was extracted, along with commitments to collect data post-reimbursement. 20 STA case studies where RWE was used extensively were identified and compared across five additional HTA agencies: SMC (Scotland), CADTH (Canada), INESSS (Quebec), HAS (France) and IQWiG (Germany). Among 284 NICE STAs screened, 145 STAs in oncology were identified. 121 (83.4%) included any RWE within the company submission across key areas. RWE was mostly submitted for economic model parameters (68% of appraisals), and HCRU (57% of appraisals). Of 80 RWE references identified in 20 STA case studies from NICE, 67 were identified in the respective CADTH submissions, 46 in IQWiG, 37 in INESSS, 37 in HAS, and 33 in SMC. RWE acceptance rate was highest within NICE (90%), followed by HAS (88%), SMC (85%), INESSS (73%), IQWiG (68%) and CADTH (67%). A mandate or recommendation to collect data post-reimbursement was issued by NICE in 9 of 20 STA case studies, 10 from HAS, 6 from CADTH, 3 from INESSS, and none from IQWiG or SMC. RWE use in HTA oncology appraisals was well-received by respective committees within the UK, Canada, France and Germany, potentially allowing improved access to innovative treatments.

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