This review investigates trends in NICE single technology appraisals (STAs) in urothelial cancer since the introduction of the new Cancer Drugs Fund (CDF) in July 2016, to better understand decision making. A targeted review was conducted of NICE STAs in urothelial cancer published on the NICE website. All STAs with a Technology Appraisal Guidance (TAG) or Final Appraisal Determination published since the introduction of the new CDF in July 2016 were included. A total of five STAs were identified, assessing three immunotherapies for untreated and treated metastatic urothelial cancer. All of the appraised therapies are indicated for other oncology conditions and have Managed Access Agreements in place. The clinical evidence packages at the time of NICE appraisal varied from Phase II trial data only (3) to Phase II and III data (2). In order to derive comparative efficacy data due to lack of Phase III data, a simulated treatment comparison approach was taken in three STAs. The economic assessments for all therapies were based on end-of-life criteria, allowing for a higher cost-effectiveness threshold. The recommendations varied: baseline commissioning (1), not recommended (1), and CDF – due to plausible potential of the therapy to be cost-effective pending further data collection (3). For CDF recommended therapies, the uncertainties were around the lack of direct comparative data, immaturity of survival data, methods for indirect treatment comparisons (ITC), and utility values upon disease progression. Three indications were recommended for interim funding on the new CDF. The drivers for a CDF recommendation include non-comparative evidence, immature survival data, and uncertainty around the robustness of ITCs. Further research should assess whether data collection through the CDF can sufficiently reduce these uncertainties for decision making purposes, and allow new therapies to be granted a final positive recommendation through NICE.
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