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Beyond traditional endpoints: A multi-state modelling approach to integrating morbidity and mortality in cancer treatments evaluation - European Journal of Cancer

As cancer treatments continue to evolve, understanding and integrating novel strategies is essential. This article co-authored by EMA colleagues contributes to this ongoing conversation by highlighting areas where further refinement is possible, integrating patient preferences, mortality, and morbidity data. Click on 'more' for more information.

Date:  18/09/2025

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Summary of a Recent Paper on a Strategy for Integrating Morbidity and Mortality in Cancer Treatments Evaluation

We are pleased to share our recent article, now published online: Beyond traditional endpoints: A multi-state modelling approach to integrating morbidity and mortality in cancer treatments evaluation. The study examines possible approaches to evaluate cancer treatment, with a focus on integrating different outcomes to better describe the patient experience.

 Key messages from the paper include:

  1. Limitation of Traditional Endpoints: Conventional analyses of endpoints such as overall survival and toxicity, using cumulative probabilities separately, may not fully capture the patient experience, particularly morbidity and quality of life aspects.
  2. Integration of Morbidity and Mortality, and Preferences: The paper advocates for using multi-state models to provide an additional unified analysis of transitions between different health states (e.g., healthy, morbid, deceased), using preferences to weigh the importance of different states, giving a detailed understanding of treatment impact over time.
  3. Informed Decision-Making: By capturing the full spectrum of patient experience, this modelling approach can add to the information that may be used for clinical and policy decisions, balancing benefits and risks more effectively.

What readers can expect to find:

  • An illustration of multi-state modelling techniques and how the model estimates transition probabilities between health states such as progression, morbidity, and death over time, providing a dynamic view of patient trajectories.
  • A discussion on integrating quality-of-life measures within the multi-state framework to better reflect patient experience.
  • A discussion of how this approach, combined with patient preference data, can refine evaluation strategies for new treatments, moving beyond traditional survival endpoints to include morbidity and quality of life outcomes.

Significance of the study:
As cancer treatments continue to evolve, understanding and integrating novel strategies is essential. Our paper contributes to this ongoing conversation by highlighting areas where further refinement is possible, integrating patient preferences, mortality, and morbidity data. We hope this article will serve as a useful resource for patients, researchers, clinicians, and others interested in the evaluation of cancer treatments, and inspire further research and discussion in this area.