Utilization of Model‐Informed Drug Development in Early Clinical Development: Case Study of a Hb Modulator for Treatment of Sickle Cell Disease

Model-informed drug development (MIDD) has an important role to play at various stages of drug development. In this case study, the application of MIDD in the early clinical development of a small-molecule inhibitor of sickle hemoglobin (Hb) polymerization is highlighted. Starting from the setting of quantitative criteria, to using a combination of PKPD and semi-mechanistic RBC modeling, and key biomarker analysis, based on data from a healthy participant first-in-human (FIH) study and historica