Target selection for FDA-approved medicines.

ABSTRACT

The biopharmaceutical industry translates fundamental understanding of disease into new medicines. As part of a comprehensive analysis of FDA-approved new molecular entities (NMEs), we assessed the mechanistic basis of drug efficacy, with emphasis on target selection. Three target families capture almost half of all NMEs and the leading ten families capture more than three-quarters of NME approvals. Target families were related to their clinical application and identify dynamic trends in targeting over time. These data suggest increasing attention toward novel target families, which presumably reflects increased understanding of disease etiology. We also suggest the need to balance the ongoing emphasis on target-based drug discovery with phenotypic approaches to drug discovery.