Research Project 1 will develop in silico models that predict PD progression and inform clinical trial design

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SUMMARY Disease progression modeling uses mathematical functions and data-driven algorithms to quantitatively describe and predict the time course of progressive disease. In 2004, the United States Food and Drug Administration (FDA) launched its Critical Path Initiative, which aims to modernize drug development. In this initiative, disease progression modeling was promoted as a key opportunity for the advancement of novel therapeutics. Since that time, disease progression modeling has been widely adopted by the pharmaceutical industry and regulatory agencies. However, the application of disease progression modeling for PD is at a nascent stage. Direct evidence demonstrating that disease progression modeling and clinical trial simulations will improve PD research, care, and outcomes is limited. For example, the common notion that the identification of specific types of patients (e.g., those at high-risk for specific progression rates) will lead to clinical trials being conducted with greater efficiency cannot be assumed. This lack of evidence strongly supports the need for more research on modeling and simulation tools in relation to PD research, care and outcomes. The expected outcomes of the efforts, networks, and platforms developed from this research project are modeling and simulation tools, and rapidly published studies to form the evidence base supporting the use of such tools in PD research.