Optimizing the science of drug development: Opportunities for better candidate selection and accelerated evaluation in humans

This report is a distillation of two tandem conferences, the first entitled "AAPS, ACCP, ASCPT, FDA Symposium on Clinical Pharmacology: Optimizing the Science of Drug Development," held in September, 1998 in Arlington, Virgin ia, USA, and the second entitled "5(th) EUFEPS Conference on Optimizing Dru g Development: Fast Tracking into Human," held in December, 1998 in Wiesbad en, Germany. The collective aims of these conferences were: To identify critical issues which currently limit drug candidate selection and the early phases of human drug development. To explore those modern scientific and technological innovations which coul d further improve preclinical and clinical development. To assess the impact of using modern approaches of clinical pharmacology in the early stages of drug development on the time, cost, quality, and regul atory decisions associated with this process. To assess the implications of the new standards for the definition of evide nce of efficacy in the US as they relate to International Conference on Har monisation (ICH) and US regulatory guidances, with special emphasis on the characteristics of confirmatory evidence. To generate recommendations for the design and analysis of early phase, sta te-of the-art clinical studies in healthy volunteers and patients that allo w bridging of information from nonclinical to late-phase clinical studies d esigned to demonstrate safety and efficacy. To prepare a combined summary of the two conferences to facilitate communic ation of these new ideas for optimizing drug candidate selection and early- phase clinical development, as well as to provide a rationale for the devel opment of future regulatory guidances.