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Specification of Estimands for Complex Disease Processes Using Multistate Models and Utility Functions.

Alexandra Bühler1, Richard J Cook1, Jerald F Lawless1

  • 1Department of Statistics and Actuarial Science, University of Waterloo, Waterloo, Ontario, Canada.

Statistics in Medicine
|October 14, 2025
PubMed
Summary
This summary is machine-generated.

This study highlights multistate models and utilities for analyzing complex diseases in clinical trials. These methods offer interpretable ways to assess treatment effects on various disease aspects, improving trial design for complex conditions.

Keywords:
clinical trialsestimandsinfinitesimal jackknifemultistate processesrankingutilities

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Area of Science:

  • Clinical Trials Methodology
  • Biostatistics
  • Complex Disease Modeling

Background:

  • Complex diseases involve multiple semi-competing events and recurrent episodes, complicating clinical trial endpoint definition.
  • Traditional composite endpoints may not fully capture the nuances of complex disease progression.
  • Recent innovations like the win ratio offer ranking-based approaches to disease course analysis.

Purpose of the Study:

  • To demonstrate the utility of multistate models for analyzing complex disease courses in clinical trials.
  • To emphasize the interpretability and simplicity of using utilities to synthesize treatment effect evidence.
  • To provide a robust statistical framework for primary analyses in clinical trials involving complex disease outcomes.

Main Methods:

  • Application of multistate models to represent complex disease pathways.
  • Development and use of utility functions to synthesize treatment effects across different disease aspects.
  • Implementation of robust variance estimation using the infinitesimal jackknife for statistical validity.

Main Results:

  • Multistate models provide a flexible framework for defining target estimands in complex diseases.
  • Utilities offer a simple and interpretable method to combine evidence on various treatment effects.
  • The infinitesimal jackknife ensures reliable variance estimation for primary trial analyses.

Conclusions:

  • Multistate models and utilities are valuable tools for clinical trials in complex diseases.
  • These methods enhance the ability to assess treatment effects on diverse disease outcomes, such as bleeding in cancer patients.
  • The proposed approach supports robust and interpretable primary analyses in challenging clinical trial settings.