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Empowering Clinical Development With Disease Progression Modeling: Recommendations From the Clinical Trials
Lindsay S Kehoe1, Shu Chin Ma2, Jiang Liu3
1The Clinical Trials Transformation Initiative, Duke Clinical Research Institute, Durham, North Carolina, USA.
Clinical and Translational Science
|August 5, 2026
Summary
Disease progression modeling (DPM) offers efficient, patient-centric drug development. This article provides CTTI
Area of Science:
- Quantitative medicine
- Clinical trial methodology
- Regulatory science
Background:
- Evaluating medical product benefit-risk requires integrated evidence across development stages.
- Real-world data and technological advances promote efficient, patient-centric, and sustainable quantitative paradigms.
- Disease progression modeling (DPM) is underutilized despite regulatory recognition of model-informed drug development.
Purpose of the Study:
- To provide evidence-based, cross-sector recommendations for adopting and implementing disease progression modeling (DPM) in medical product development.
- To define DPM, summarize its value in clinical development, and outline CTTI's collaborative process for generating recommendations.
- To present strategic questions demonstrating DPM's utility in addressing key development decisions.
Main Methods:
- Collaborative, cross-sector development of nine DPM recommendations by the Clinical Trials Transformation Initiative (CTTI).
- Creation of a practical considerations framework to guide DPM implementation.
- Illustrative examples of strategic development questions addressed by DPM.
Main Results:
- Nine evidence-based recommendations for the use of DPM in medical product development.
- A framework for practical considerations in DPM implementation.
- Demonstrated application of DPM in answering critical questions regarding indication, population, endpoints, and dose selection.
Conclusions:
- CTTI recommendations and a shared vocabulary can lower barriers to DPM implementation.
- Strategic use of DPM supports fit-for-purpose model application, enhancing clinical development efficiency.
- DPM adoption promotes more patient-focused and sustainable medical product development.
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