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Impact of Limited Sample Size and Follow-up on Partitioned Survival and Multistate Modeling-Based Health Economic
Jaclyn M Beca1,2,3, Kelvin K W Chan1,2,4, David M J Naimark1,4
1Institute of Health Policy, Management and Evaluation, University of Toronto, Toronto, Canada.
Summary
Partitioned survival models (PSM) and multistate modeling (MSM) are used for economic models in oncology. This simulation study found that while both methods can be accurate, limited data increases errors, with PSMs showing larger inaccuracies, especially with short follow-up.
Area of Science:
- Health economics and outcomes research
- Mathematical modeling in oncology
- Survival analysis and time-to-event data extrapolation
Background:
- Economic models frequently require extrapolation of clinical time-to-event data for multiple events.
- Partitioned survival models (PSM) and multistate modeling (MSM) are two time-dependent approaches used in oncology.
- The performance of these models with limited data is not well understood.
Purpose of the Study:
- To assess the performance of PSM and MSM in estimating mean survival.
- To evaluate model performance across varying sample sizes and degrees of censoring.
- To compare the accuracy of PSM and MSM under data limitations.
Main Methods:
- Generated disease trajectories for hypothetical advanced cancer populations.
- Simulated trial cohorts with varying sample sizes and follow-up durations.
- Estimated PSM and MSM, incorporating general population mortality (GPM), and compared mean survival estimates to true values.
Main Results:
- Both PSM and MSM accurately estimated mean survival with near complete follow-up.
- Smaller sample sizes and shorter follow-up times increased error for both models, particularly for distant endpoints.
- MSMs were less often estimable with limited data but produced smaller errors when estimable compared to PSMs.
Conclusions:
- Caution is advised when using PSM or MSM with very limited data, especially PSMs due to potentially large errors.
- When estimable and selected based on statistical criteria, MSMs performed similarly to or better than PSMs in estimating mean survival with limited data.
- Further research is needed to improve the estimation of MSM and similar state-transition models with limited data.
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