TL14 - Evaluating Early Overall Survival Information in Oncology/Hematology

Overall survival (OS) is both a safety and an efficacy endpoint. When other endpoints have been used for approval, the FDA has still required submission of OS data at the time of approval and/or as a post-marketing requirement after approval. When OS is not the primary endpoint, it has often been analyzed in a descriptive manner without formal statistical power calculations or Type I error control. In this setting, particularly in diseases with long natural histories, the number of events may be small at the time of analysis of the primary endpoint and there can be significant uncertainty regarding the estimates.

This round table discussion will focus on the following questions:
• What amount of OS information is feasible and useful for benefit-risk evaluation in diseases with a long natural history?
• When pre-specifying analyses for OS, when it is not the primary endpoint and there may or may not be planned formal statistical testing, what analyses would be useful to rule out harm?
• What should be prespecified regarding maturity of the OS data to adequately inform a benefit-risk assessment for regulatory evaluation?
• What safety and efficacy analyses of OS should be conducted post-hoc, especially if there were no pre-specified analyses to assess for harm?
• What would be considered "immature" data such that the observed HR could be considered an unreliable estimate?

OS

overall survival

time-to-event

oncology

hematology 

Oncology

ASA Biopharmaceutical Section Regulatory-Industry Statistics Workshop 2023