Guiding Clinical Development in Rare Diseases and Oncology: Insights from Quantitative Systems Pharmacology

抄録

Application of Model Informed Drug Development (MIDD) provides a robust quantitative framework for making key decisions during all stages of the drug development process. For rare diseases, sparse clinical data and the utility of new treatment modalities such as gene therapies might limit the ability to apply standard approaches of exposure-response analysis, efficacious dose determination, and preclinical-to-clinical translation. Similar challenges are also faced in oncology with alternative trial designs in place of large clinical trials and the need to optimize combination therapies. These necessitate the development of a Quantitative Systems Pharmacology (QSP) models that reflect the underlying mechanisms of disease pathophysiology and can integrate disparate sources of data for model parameterization and calibration. In this presentation, I will highlight a few case studies showcasing the utility and impact of QSP modeling.