Regulatory considerations for early clinical development of drugs for diabetes, obesity, and cardiometabolic disorders

Abstract

Advancing therapeutic discoveries through early clinical studies benefits from a working knowledge of regulatory history, practices, provisions, procedures, and controversies. This chapter aims to supplement readily available published and web-based resources that describe regulatory expectations and requirements. Though the Food and Drug Administration is emphasized, the importance of the European Medicines Agency (EMA) and other major authorities is acknowledged. Some relevant differences between EMA and the Food and Drug Administration (FDA) are discussed. With emphasis on early studies, selected regulatory considerations are presented for developing diabetes, anti-obesity, and lipid-lowering therapies toward existing therapeutic indications. Novel therapeutic indications that have reasonable prospects for approval are also described. Interpretations and preferences expressed reflect the author’s experience as an endocrinologist and an FDA representative, educator, and reviewer responsible for approval of the first statin and insulin analog, metformin, and other metabolic products.