OBJECTIVES: Like other health technology assessment (HTA) bodies, NICE is seeing an increasing number of technologies being appraised in the same disease area, creating complex pathways with multiple sequential decisions and multi-comparator decision spaces. HTA bodies need to adapt their methods to meet the challenges of providing robust, useful, and timely guidance with ever increasing demand. NICE is developing refined appraisal processes that are proportionate to different needs. Pathways is one approach, built around a core model that spans a disease pathway and multiple technologies.

METHODS: NICE’s pathways pilot explores the development of a core model to appraise treatments in the systemic treatment pathway for renal cell carcinoma. The pilot was informed by a targeted review of the literature, a focus group, and engagement sessions with key stakeholders. The pilot process is being monitored and refined.

RESULTS: The review identified that 43 percent of HTA guidance produced by NICE covers only ten disease areas. Engagement sessions established a range of procedural, methodological, and operational challenges for disease-specific reference models, including the complexity of analysis, the reliance on partition survival modeling in cancer, and the availability of evidence. However, it was clear that this is an evolving area of interest for HTA bodies. The use of a single model promises to increase transparency and reduce structural uncertainties, but concerns exist that these models may accentuate challenges common to all economic evaluations. The pathways pilot explores whether technological developments and real-world evidence can address these challenges whilst producing robust guidance.

CONCLUSIONS: The pathways pilot can pave the way for dynamic changes in HTA. It can create efficiencies by incorporating novel technologies into disease-specific models, with economic and clinical data updated as required. Using a pilot to address the challenges allows solutions to be found, and future directions to be considered collaboratively.