OBJECTIVES: Real world evidence (RWE) is increasingly used in regulatory and reimbursement decision making. It is important to understand how RWE has historically been used to identify learnings for future uses of RWE in these contexts. The objective of this study was to systematically investigate how RWE has been used by manufacturers to inform National Institute for Health and Care Excellence (NICE) technology appraisals (TAs) for multiple myeloma (MM) medicines and to describe the impact of RWE on reimbursement decisions made by NICE.

METHODS: A systematic literature review (SLR) of all NICE TAs for MM medicines was conducted. Information on the RWE studies (methods and results), the use of these studies in the TAs and the impact of each study on NICE decisions were extracted from TA materials published on the NICE website. Screening and data extraction were undertaken using pre-defined templates and set eligibility criteria. All analyses were descriptive.

RESULTS: Interim analyses found 34 TAs of MM medicines, published on the NICE website from October 2007 to May 2023, which were identified and screened. Of these, 20 (58.8%) were excluded (terminated TA, superseded by updated guidance or were multiple technology appraisals). The 14 (41.2%) remaining TAs contained at least one document that was accessible for screening and review in the TA supporting evidence section on the NICE website. Of these TAs, 11 (78.6%) met all the inclusion criteria for data extraction. These TAs were published by NICE between 2007 and 2023 and each reported results from at least one RWE study.

CONCLUSIONS: Over half of TAs for MM medicines submitted to NICE were terminated. This SLR of TAs published by NICE up until May 2023 found that the majority of completed TAs utilized results from at least one RWE study in their evidence submission.