Work

Cancer patient survival can be accurately parameterized, improving trial precision and revealing time-dependent therapeutic effects

Year: 2021

Type: other

Abstract: Individual participant data (IPD) from oncology clinical trials represent an invaluable source of information for identifying factors that influence trial success and failure, improving trial design and interpretation, and comparing pre-clinical studies to clinical outcomes. However, the IPD used to generate published survival curves are not generally available. We imputed survival IPD from ~500 arms of Phase 3 oncology trials (representing ~220,000 events) and found that they are well fit by a two-parameter Weibull distribution. This finding supports the use of parametric statistics to increase trial precision with small patient cohorts typical of Phase 1 and 2 trials. We also show that frequent violations of the proportional hazards assumption, particularly in Phase 3 trials of immune checkpoint inhibitors, arise from time-dependent therapeutic effects. Trial duration therefore has an underappreciated impact on the likelihood of success. All imputed IPD and analysis are available as supplementary materials and via the website https://cancertrials.io./

DOI: 10.7490/f1000research.1118881.1

Authors: Deborah Plana, Geoffrey Fell, Brian M. Alexander, Adam C. Palmer, Peter K. Sorger

Institutions: University of North Carolina at Chapel Hill, Harvard University

Language: English

Topic: Statistical Methods in Clinical Trials

Subfield: Statistics and Probability

Field: Mathematics

Domain: Physical Sciences

SDG: Good health and well-being

Open Access status: gold

OpenAlex ID: W4416509616

Cites:

Cited by:

Related to: