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Extrapolating Survival from Randomized Trials Using External Data: A Review of Methods.

Published version
Peer-reviewed

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Authors

Jackson, Christopher  ORCID logo  https://orcid.org/0000-0002-6656-8913
Stevens, John 
Ren, Shijie 
Latimer, Nick 
Bojke, Laura 

Abstract

This article describes methods used to estimate parameters governing long-term survival, or times to other events, for health economic models. Specifically, the focus is on methods that combine shorter-term individual-level survival data from randomized trials with longer-term external data, thus using the longer-term data to aid extrapolation of the short-term data. This requires assumptions about how trends in survival for each treatment arm will continue after the follow-up period of the trial. Furthermore, using external data requires assumptions about how survival differs between the populations represented by the trial and external data. Study reports from a national health technology assessment program in the United Kingdom were searched, and the findings were combined with "pearl-growing" searches of the academic literature. We categorized the methods that have been used according to the assumptions they made about how the hazards of death vary between the external and internal data and through time, and we discuss the appropriateness of the assumptions in different circumstances. Modeling choices, parameter estimation, and characterization of uncertainty are discussed, and some suggestions for future research priorities in this area are given.

Description

Keywords

detailed methodology, internal medicine, multiparameter evidence synthesis, survival analysis, technology assessment, Cost-Benefit Analysis, Data Interpretation, Statistical, Humans, Models, Economic, Models, Statistical, Quality-Adjusted Life Years, Randomized Controlled Trials as Topic, Survival Analysis, Uncertainty, United Kingdom

Journal Title

Med Decis Making

Conference Name

Journal ISSN

0272-989X
1552-681X

Volume Title

37

Publisher

SAGE Publications
Sponsorship
MRC (unknown)