Presentation (CTI)

OBJECTIVES: Real-world evidence (RWE) plays a well-established role in economic evaluation of health technologies, particularly in oncology, where it addresses gaps in clinical trials, such as limited follow-up or underrepresented subgroups. Its importance has grown among health technology assessment bodies, exemplified by the National Institute for Health and Care Excellence (NICE) with the publication of a comprehensive RWE framework in 2022 and its update in 2024. This review evaluates the use of RWE in breast cancer drug submissions to NICE
METHODS: A hand search of the NICE appraisals database targeted breast cancer submissions published between June 2020 and June 2024. RWE use was reviewed across four mutually exclusive categories: cost-effectiveness model (CEM) inputs, comparative analysis, model validation, and background/supportive evidence.
RESULTS: Out of 15 appraisals identified, 12 incorporating keywords related to RWE were included in the extraction. RWE was utilized for CEM inputs in three submissions, which included informing transition probabilities, and utility values. RWE was used in three submissions to support comparative analysis, and in four submissions for external validation of survival analysis. Six submissions referenced RWE to provide background context or enhance the discussion. Key critiques from the committee centred around the limited alignment and comparability between RWE and clinical trial populations, raising concerns about the generalizability of findings derived from RWE.
CONCLUSIONS: The use of RWE in NICE breast cancer appraisals varied, primarily supporting background evidence and validating of survival outcomes. However, critiques highlighted misalignment with clinical trial populations, limiting generalizability, and raised methodological concerns. Enhancing the quality of RWE generation and adopting robust analytical methodologies could significantly improve its applicability and impact in oncology modeling.