title: New era for drug discovery and development in renal disease.
abstract: Drug discovery and development is a lengthy and expensive process. Testing new agents in humans at an early stage could reduce the time and costs involved in identifying drugs that are likely to succeed in clinical studies. New guidance has outlined the concept of exploratory clinical trials, which provide important information on a drug's distribution as well as its physiological and pharmacological effects in humans. This strategy reduces the need for preclinical testing by limiting the dose and duration of exposure to a new drug in humans to below those required by the traditional testing of investigational new drugs. Exploratory, first-in-man studies should provide insights into human physiology and pharmacology, identify therapeutic targets relevant to disease and increase our knowledge of a drug's characteristics. Implementation of a new drug also requires the development of useful biomarkers of disease and of the drug's efficacy, as well as sensitive molecular imaging techniques. In this Review, we outline the benefits of exploratory clinical trials, especially in academia, and provide an overview of the experimental tools necessary for rational drug discovery and development.
authors: United Centers for Advanced Research and Translational Medicine, Tohoku University Graduate School of Medicine, 2-1 Seiryo-Machi, Aoba-ku, Sendai 980-8575, Japan.
authors: Toshio
authors: T
authors: Miyata
authors: Katsushi
authors: K
authors: Kikuchi
authors: Hideyasu
authors: H
authors: Kiyomoto
authors: Charles
authors: C
authors: van Ypersele de Strihou
mesh_terms: D002626:Chemistry, Pharmaceutical; D002986:Clinical Trials as Topic; D015195:Drug Design; D004341:Drug Evaluation; D004353:Drug Evaluation, Preclinical; D015507:Drugs, Investigational; D006801:Humans; D007674:Kidney Diseases / Q000188:drug therapy*; D010597:Pharmacogenetics
chemical_list: D015507:Drugs, Investigational