The Impact of Using England Real-World Evidence to Establish the NICE Severity Modifier in Metastatic Urothelial Carcinoma (MUC)
Author(s)
Will Perkin, MSc1, Craig Knott, PhD2, Maximiliaan Alexander Nievaart, PharmD1, Simran Gill, MSc1, Tinevimbo Shiri, PhD1.
1Johnson & Johnson, High Wycombe, United Kingdom, 2HDI, Cambridge, United Kingdom.
1Johnson & Johnson, High Wycombe, United Kingdom, 2HDI, Cambridge, United Kingdom.
OBJECTIVES: Retrospectively identify and follow-up patients diagnosed with metastatic urothelial cancer (mUC) in England to provide insight into real-world practice and quantify unmet need. This includes describing the baseline characteristics and distribution of patients diagnosed with mUC in England. Use overall survival (OS) to establish expected total remaining life years and model long-term extrapolations to determine the QALY shortfall relative to the general population to inform the National Institute for Health and Care Excellence (NICE) severity modifier.
METHODS: A retrospective analysis was conducted using routine patient data from the National Disease Registration Service (NDRS) which collects data from all patients diagnosed with cancer in England from January 2016 to December 2021, followed up until March 2023. The analysis included patient demographics, treatment regimens and OS.
RESULTS: Of the 10,787 patients diagnosed, 3,942 (36.5%) received systemic anti-cancer treatment (SACT). The median age was 70 years for patients on treatment with 72% of the patients being male. The median overall survival was 5.4 months from diagnosis, with significant attrition rates observed between treatment lines - only 24% of patients received a second line treatment. Long term modelling simulates the remaining QALYs for patients in second line mUC and predicts remaining QALYs of only 0.45 compared to 8.63 in the general population, giving a proportional QALY shortfall of 95.04%.
CONCLUSIONS: The findings underscore the critical need for improved treatment pathways and newer therapies for mUC patients in the UK due to poor treatment outcomes and short overall survival estimates. The proportional QALY shortfall indicates a NICE severity modifier of x1.7 should be used for decision making for patients treated in the second line, highlighting a critical need for advances in innovation to better treat mUC.
METHODS: A retrospective analysis was conducted using routine patient data from the National Disease Registration Service (NDRS) which collects data from all patients diagnosed with cancer in England from January 2016 to December 2021, followed up until March 2023. The analysis included patient demographics, treatment regimens and OS.
RESULTS: Of the 10,787 patients diagnosed, 3,942 (36.5%) received systemic anti-cancer treatment (SACT). The median age was 70 years for patients on treatment with 72% of the patients being male. The median overall survival was 5.4 months from diagnosis, with significant attrition rates observed between treatment lines - only 24% of patients received a second line treatment. Long term modelling simulates the remaining QALYs for patients in second line mUC and predicts remaining QALYs of only 0.45 compared to 8.63 in the general population, giving a proportional QALY shortfall of 95.04%.
CONCLUSIONS: The findings underscore the critical need for improved treatment pathways and newer therapies for mUC patients in the UK due to poor treatment outcomes and short overall survival estimates. The proportional QALY shortfall indicates a NICE severity modifier of x1.7 should be used for decision making for patients treated in the second line, highlighting a critical need for advances in innovation to better treat mUC.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HTA320
Topic
Clinical Outcomes, Economic Evaluation, Health Technology Assessment
Topic Subcategory
Decision & Deliberative Processes, Systems & Structure
Disease
Oncology