A Review of Survival Analysis Methods Used in NICE Technology Appraisals of Cancer Treatments: Consistency, Limitations, and Areas for Improvement

摘要

Objectives. In June 2011, the National Institute for Health and Care Excellence (NICE) Decision Support Unit published a Technical Support Document (TSD) providing recommendations on survival analysis for NICE technology appraisals (TAs). Survival analysis outputs are influential inputs into economic models estimating the cost-effectiveness of new cancer treatments. Hence, it is important that systematic and justifiable model selection approaches are used. This study investigates the extent to which the TSD recommendations have been followed since its publication. Methods. We reviewed NICE cancer TAs completed between July 2011 and July 2017. Information on survival analyses undertaken and associated critiques for overall survival (OS) and progression-free survival were extracted from the company submissions, Evidence Review Group (ERG) reports, and final appraisal determination documents. Results. Information was extracted from 58 TAs. Only 4 (7%) followed all TSD recommendations for OS outcomes. The vast majority (91%) compared a range of common parametric models and assessed their fit to the data (86%). Only a minority of TAs included an assessment of the shape of the hazard function (38%) or proportional hazards assumption (40%). Validation of the extrapolated portion of the survival function using external data was attempted in a minority of TAs (40%). Extrapolated survival functions were frequently criticized by ERGs (71%). Conclusions. Survival analysis within NICE TAs remains suboptimal, despite publication of the TSD. Model selection is not undertaken in a systematic way, resulting in inconsistencies between TAs. More attention needs to be given to assessing hazard functions and validation of extrapolated survival functions. Novel methods not described in the TSD have been used, particularly in the context of immuno-oncology, suggesting that an updated TSD may be of value.