Abstract
To assess the application of progression-free survival (PFS) as surrogate endpoint for overall survival (OS) within cost-effectiveness models appraised by National institute for Health and Care Excellence (NICE) given the consistent clinical evidence supporting correlation between PFS and OS in advanced breast cancer. Using published NICE reports, 14 unique technology appraisals (TAs) in advanced breast cancer were identified from January 2012 to May 2021. Full-text screening of the committee papers and TA guidance was conducted by a single investigator and the abstracted data were validated by a second investigator. Information on drug of interest, specific indication, model structure, source of PFS and OS, approach for long-term OS projection, and committee commentaries was extracted. Of the 14 final appraisals, only three used PFS to predict long-term OS beyond trial period. Although this approach has generally been accepted by the committee, given the complex and difficult-to-predict relationship between PFS and OS, the committee supported a partial surrogacy between the two endpoints—that is, an improved PFS is likely to be partially (27.5%–100%) translated into an OS gain. Approximately half of the remaining 11 appraisals acknowledged the OS data from trial were immature and projected long-term OS based on direct extrapolation of trial data using parametric fittings and informed by external data in some cases. Both approaches did not preclude the validity of long-term OS estimates as a key consideration by the committee. In the absence of mature trial data on OS, to derive an estimate of long-term OS within the economic analysis surrogate measure provides an alternative to directly projecting OS from trial data in advanced breast cancer; however, it has not been commonly used in the existing models appraised by NICE. Strong evidence is warranted to facilitate the acceptance of this approach.
Sign-in/Register to access full text options 
Talk to us
Join us for a 30 min session where you can share your feedback and ask us any queries you have
Schedule a callDisclaimer: All third-party content on this website/platform is and will remain the property of their respective owners and is provided on "as is" basis without any warranties, express or implied. Use of third-party content does not indicate any affiliation, sponsorship with or endorsement by them. Any references to third-party content is to identify the corresponding services and shall be considered fair use under The CopyrightLaw.
