OBJECTIVE: This analysis aimed to assess the value of real-world evidence (RWE) in recent health technology assessment (HTA) appraisals for melanoma therapies in England, France, Canada and Australia. 

METHOD: All publicly available final HTA assessment reports from England  , France, Canada, and Australia between 1st January 2011 and 30th September 2019 for melanoma therapies were identified, from which RWE data, assessment commentary and reimbursement outcomes were extracted.

RESULTS: Eleven melanoma therapies (cobimetinib plus vemurafenib, dabrafenib, dabrafenib plus trametinib, encorafenib plus binimetinib, ipilimumab, nivolumab, nivolumab plus ipilimumab, pembrolizumab, talimogene laherparepvec, trametinib, and trametinib plus dabrafenib) were assessed in 43 assessments in total, of which RWE was submitted as follows: 2 of 14 assessments by the National Institute for Health and Care Excellence (NICE), 1 of 6 assessments by Haute Autorité de Santé (HAS), 1 of 12 assessments by the Canadian Agency for Drugs and Technologies in Health (CADTH), and 1 of 11 assessments by the Pharmaceutical Benefits Advisory Committee (PBAC) appraisals.
 
RWE submitted spanned retrospective and prospective observational studies, as well as medical chart reviews. The focus of these was both efficacy and safety. RWE was considered where prospective randomized controlled trial (RCT) data was not available at the time of decision making, and to determine which treatments were being used in clinical practice. In most cases, RWE was accepted and contributed to HTA decision making. In their assessment of ipilimumab, however, NICE specified that they did not consider that RWE studies provided additional relevant reliable data. 

CONCLUSION: Where used, RWE has – for the most part – been accepted by HTA bodies in their assessment of melanoma therapies in England, France, Canada, and Australia, and has contributed to their decision making.  Pharmaceutical companies are increasingly using RWE to provide effectiveness or safety data in HTA submissions or reimbursement negotiations, where RCT data is not available at the time of decision making, and to determine which treatments are being used in clinical practice.

Assessing the value of real-world evidence in melanoma in health technology assessment appraisals

DIA Europe 2020 

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