Quantitative Medicine Consultant Independent Consultant Quantitative Medicine Lansdale, Pennsylvania
The aim for a model-informed drug development approach is to provide a quantitative framework for prediction and extrapolation based on insights generated from quantitative models of compound, mechanism, and disease to ultimately improve quality, efficiency and cost effectiveness of decision making. These decisions can be in areas such as, but not limited to, clinical trial design, dose and schedule selection and characterization of risk/benefit. Strategic utilization of biomarkers to provide relevant insights in compound, mechanism and disease is important to reduce translational risk and increase robustness of the totality of evidence and as such can be particularly useful for the development of novel therapies for rare diseases. This talk will highlight the opportunities for model-informed approaches and provide strategies to reduce translational risk for biomarkers, combined with some illustrative examples.
Learning Objectives:
Upon completion, participants will gain understanding about model-informed drug development opportunities in context of rare diseases
Upon completion, participants will become familiar with assessing translational risk of biomarkers supporting the development of novel therapeutics for rare diseases
Upon completion, participants will learn from illustrative case studies how to maximize quantitative biomarker strategies in the development of therapeutics for rare diseases
