New Drugs Development

Abstract

Phase I, II, and III clinical studies govern the progression of a new agent, or regimen, towards its incorporation into clinical practice. The characteristics of most recent anticancer agents, i.e., molecularly targeted drugs and immune therapies, as compared to cytotoxic chemotherapy, are driving an evolution of the format of such studies, with an increasing overlap of classical study phases and transformation of end-points. This parallels an ongoing evolution of the methodology of clinical research in the era of precision medicine, driven by molecular biology, and personalized medicine, driven by the attempt to individually tailor the patient-physician shared clinical decision-making process. Clinical studies in small populations, as determined by disease partitioning of precision medicine or inherently found in rare cancers, are in need of steps forward in terms of research methodology. Meanwhile, artificial intelligence tools are currently entering clinical medicine, with the potential of emulating clinical skills, but also with the ability of machine learning to treat big data, from omics-based to real-world clinical data. At the moment, how artificial intelligence tools might interact with the classical instruments of clinical research is unclear. In any case, the whole body of evidence provided throughout the development of a new agent is used for its risk/benefit assessment by regulatory bodies governing new medicine approval and, in European systems, for cost/effectiveness health technology assessment by reimbursement bodies. Following this, there is always room for studying further the positioning of any agent, or regimen, in the clinic, hopefully with the academia as a driver. Even when it may be pathologically “agnostic” for regulatory purposes, new anticancer drug development should always be seen in the context of the advancement of cancer treatment as a whole under a multidisciplinary perspective.