OBJECTIVES:

Increasingly real-world evidence (RWE) is also appraised to support the value of new health technologies that ensures timely patient access. RWE is considered as evidence gathered outside of RCTs and derived from data obtained from non-randomized trials, observational studies, or databases amongst others. This analysis aims to assess the value of RWE in recent HTA appraisals for melanoma drugs in England, France, Canada, Australia, Germany, Scotland, and the Netherlands.

METHODS:

All publicly available appraisal reports from the NICE,UK; HAS, France; CADTH/pCODR(Canada), PBAC, Australia; IQWiG, Germany; SMC, Scotland; ZIN, Netherlands; between 1st January 2011 and 30^th June, 2022 for melanoma drugs were identified, from which RWE and reimbursement outcomes were extracted.

RESULTS:

Eighty melanoma health technologies appraisal reports were assessed, of which RWE was submitted in 27 reports (33.75%); specifically, 11/15 of NICE appraisals, 2/10 of PBAC, 5/15 of pCODR, 0/5 of HAS, 0/15 of IQWIG, 9/17 of SMC, and 0/3 of ZIN. RWE from registry data (19 studies), observational studies (6 studies), retrospective analyses (1 study) , or databases (4 studies), was submitted to identify treatment patterns and patient characteristics, and as supportive evidence for the economic evaluation, such as long-term survival data extrapolation and validation of economic model inputs. Where clinical efficacy data was submitted on single-arm study, RWE was submitted to reduce uncertainty. NICE, PBAC, SMC, and PBAC has cited RWE use for drug effectiveness while the ZIN and IQWiG have cited RWE for evidence on prevalence. The melanoma technologies assessed were nivolumab, dabrafenib/trametinib, encorafenib /binimetinib, pembrolizumab, talimogene laherparepvec, ipilimumab, dabrafenib, cobimetinib/vemurafenib, vemurafenib, amongst others.

CONCLUSIONS:

RWE adds value to HTA appraisals particularly by supporting economic evaluation such as long-term survival extrapolation assumptions, to identify treatment patterns and patient characteristics. This allows expedited access of technologies in areas of high unmet clinical need through managed access schemes.