Clinical Testing of a New Drug

ABSTRACT

Developing a new drug is a high-risk and high-reward enterprise. The high risk is reflected by the low success rate of turning a new molecular entity (NME) into an approved drug even after the NME has successfully met the preclinical testing requirements. In this chapter, we offer a high-level summary of the clinical testing which is composed of four distinct phases under a traditional development plan. We discuss deviations from the traditional development plan and new regulatory approval pathways. In particular, we highlight the speed with which COVID-19 vaccines were developed and authorized for emergency use through extensive public–private partnerships and the adoption of a high-risk business model. We offer some examples of recent advances in clinical trial designs and conclude the chapter with a short summary of the salient points covered in the chapter.