The Development of a Drug: A Pharmaceutical Drug Development Perspective

Clinical investigation of New Molecular Entities (NME) in oncology is changing. Drivers of this transformation are advances in pharmacological platforms, such as antibody technology, changes in the regulatory framework to accelerate approval of new treatments, and rapid scientific discovery. As a result of this transformation the established drug development process is being modified and continues to adapt. Today significant resources are being moved towards early clinical development and NME have to show early promise of therapeutic activity. The ideal NME targets specific pathways, for which diagnostic tools can be developed to select or enrich patients for the treatment with NME. This chapter reviews the critical steps enabling the early phase clinical development from the perspective of a pharmaceutical drug developer. The required steps include non-clinical pharmacokinetic (PK) studies, pharmacokinetic/pharmacodynamic (PK/PD) models, pharmacology and toxicology studies, and biomarker development plans.