Our thesis is a simple one: although a
drug can fail in an individual patient for many reasons, appropriately sized and conducted
drug-development programs often fail because of insensitive, uninformative end points, and/or poor a priori regimen decisions. The difficulty in successfully developing
antimicrobial agents at present is often exacerbated by company decision-makers who are either uninformed or disregard the difference between empirical-based (ie, akin to playing pin-the-tail on the donkey) and quantitative model-based development plans. Frequently, the focus is on Gantt charts (project event schedules) and the on-time submission of a New
Drug Application to a regulatory body, such as the US Food and Drug Administration. Such misplaced focus has led and will continue to lead to a number of problems, including program failure or, even worse, regulatory approval of an inappropriate dosing regimen with associated negative safety and efficacy sequelae. We believe that the goal of
drug development is not a New
Drug Application submitted on time but, rather, an approved, differentiated, safe, and effective new medicine. Here, we focus on the pharmacokinetic-pharmacodynamic data needed to guide dosing regimen decisions for patients with hospital-acquired
bacterial pneumonia or
ventilator-associated
bacterial pneumonia. Early consideration of these data in development programs will reduce risk not only to sponsors but also, most importantly, to the patients enrolled in the clinical trials.