ABSTRACT:
The development of new drugs is expensive and time-consuming, with later-stage clinical trials leading to high expenses. Herein, we examine the cases of recent late-stage drug failures to determine which drugs failed due to a lack of scientific innovation as well as those we feel might have avoided failure had different strategies been pursued. From this analysis, the important considerations for effective drug development become clear. These include an understanding of the pathophysiologic mechanism of the disease in question, its molecular targets, and their pharmacological activity in terms of safety and efficacy, as well as its pharmacokinetics and optimal dosage. Early stage studies should be planned to provide answers to important questions, but with an emphasis on knowledge gathering during the earlier stages of development rather than Phase 3 being used for validation purposes.