Modeling and simulations are powerful tools that can be leveraged to inform clinical trial design, support regulatory decision making, and accelerate the process of bringing treatments to patients. Despite these benefits, the use of modeling and simulations is still not widely accepted among sponsors, regulators, and researchers. In particular, there is growing interest across the clinical trials ecosystem in advancing the use of disease progression modeling in order to improve clinical trial quality and efficiency. Disease progression modeling leverages data from many different sources — including real-world data and registry data — enabling better diversity in trials by recognizing and incorporating data from different populations and combining various types of models to better inform decision making throughout the medical product development lifecycle.

CTTI’s work in this area will engage a variety of experts in the development of a framework for the use of DPM in clinical trial decision making, recommendations, and other resources.

Using Disease Progression Modeling to Advance Trial Design and Decision Making

As part of this work, CTTI will: 

• Describe disease progression modeling and its current contexts of use 
• Identify and catalog examples of disease progression modeling that point to areas where it could be valuable in advancing decision making 
• Develop and disseminate recommendations that address disease progression modeling integration best practices.

By promoting the increased recognition, value, and consistent use of disease progression modeling, CTTI’s work aims to improve trial and clinical development efficiency. CTTI’s work in this area will also help to advance the broader application of modeling and simulation for trial design and regulatory decision making. 

CTTI will utilize a variety of iterative evidence gathering strategies, including a scoping review assessing the landscape of disease progression modeling applications and an expert meeting discussing the barriers to disease progression modeling application and avenues for advancement. The insights generated by these strategies will be used to develop a variety of recommendations and resources to help sponsors, regulators, and researchers better apply disease progression modeling in their clinical trials. 

The Using Disease Progression Modeling to Advance Trial Design and Decision Making project is expected to yield the following: 

• A considerations framework to inform decisions about implementing disease progression modeling. 
• A set of recommendations that highlight the unique benefits of using disease progression modeling in medical product development and how to incorporate it. 
• A review paper detailing relevant applications of disease progression modeling in clinical trials. 

Organization affiliations are listed as active affiliations during the project.

*Indicates former project manager, team leader, or team member.