OBJECTIVES: NICE is developing a new approach to health technology assessment (HTA) based on evaluations of technologies across a disease pathway. Building on a core disease model and incorporating real-world-evidence (RWE), pathway evaluations will assess the technologies’ impact on treatment pathways and where necessary, treatment hierarchy or sequencing may be recommended. Using renal cell carcinoma (RCC) as an example, this study describes the use of RWE in an RCC pathway appraisal and the challenges encountered. Specifically, it aims to share insight on how RWE may contribute to decision making in this space and lessons learned.

METHODS: A review of key commentary papers or policy statements on the use of RWE in HTA published in the past 3 years, and semi-structured interviews with key stakeholders involved in the pilot were undertaken. Thematic analysis of qualitative data were conducted.

RESULTS: RWE’s main use cases identified ranged from treatment pathways to model parameters such as quality of life, and monitoring. Using RWE to inform pathway evaluations is broadly supported by stakeholders but there are challenges. These included: potential changes in the types, level, and quality of RWE needed and in focus of scrutiny; practicality issues such as data sources, completeness, accessibility and transparency; robustness of evidence and rigor of methods such as confounding and generalisability; complexity in the evidence to which this approach could pragmatically accommodate; and engagement and communication with stakeholders because there may be a lack of understanding of what types of evidence may influence decision making and why.

CONCLUSIONS: Pathway evaluations are a shift from NICE’s current approach to technology appraisals. This project identifies opportunities for expanded use case of RWE alongside challenges about and beyond the evidence itself. These could be disease or decision making context specific and may vary across settings and need to be thoroughly explored.