Presentation (CTI)

OBJECTIVES: Evidence generation for health technology assessment (HTA) in BCG-unresponsive non-muscle-invasive bladder cancer (NMIBC) is challenging because pivotal studies are frequently single-arm trials. This project aimed to establish a real-world comparator cohort from NHS England registry data to support indirect comparative effectiveness analyses and future HTA submissions for novel bladder-preserving therapies.
METHODS: A Data Access Request Service (DARS) agreement was established with NHS England to access pseudonymised data within the Secure Data Environment (SDE). Patients with NMIBC were identified from the National Disease Registration Service (NDRS) Cancer Consolidated Data Set using ICD-10 diagnostic codes and tumour stage information. Prior Bacillus Calmette-Guérin (BCG) exposure and subsequent radical cystectomy were proxied using Hospital Episode Statistics (HES) OPCS-4 procedure codes and validated treatment event algorithms. Aggregate outputs for use in indirect treatment comparisons and HTA submissions, including baseline characteristics, treatment pathways, Kaplan-Meier survival curves, and risk tables, were generated within the SDE with small-number suppression applied.
RESULTS: Tumour- and patient-level data were adequate for identifying NMIBC patients although missing tumour staging identifiers required a large proportion of the sample to be excluded. BCG treatment could not be differentiated from other intravesical treatments (mitomycin-C) using HES OPCS-4 codes alone; however, cross-referencing against distinct treatment schedules was reliable for avoiding the false positive identifiers. The analysis enabled the identification of clinically relevant cohorts of patients with BCG-treated NMIBC and generation of aggregate evidence suitable for comparative effectiveness research. The analysis addressed a recognised evidence gap in BCG-unresponsive NMIBC by providing nationally representative UK real-world comparator data for decision makers and clinicians.
CONCLUSIONS: This NHS England registry-based framework demonstrates the feasibility of generating robust real-world comparator evidence for rare oncology populations using privacy-preserving aggregate outputs. The methodology provides an important evidence source for indirect treatment comparisons, HTA evaluations, and future clinical research in BCG-unresponsive NMIBC.