Development of anti-cancer medicines in the current era

Abstract

The successful development of anticancer medicines is arduous and highly technical. Although clear to some, many sponsors are not aware of regulatory mandates and processes leading to delays and missed opportunities. Drug development begins with molecular discovery and product synthesis leading to preclinical models and ultimately human trials. In addition, “Chemistry, Manufacturing, and Controls” is highly regulated and critical to quality control in providing available medicines for clinical trials. Recent advances in the design and execution of phase 1 studies have improved efficiency and reduced cost despite an increase in trial complexity. Dose expansion cohorts provide “proof of concept”, but interpretability of these small studies can be challenging. This is further complicated by the frequent lack of a “control arm” and the need for “dose optimization”. A diversity plan is important to contextualize the efficacy and safety across various populations. Networks and central processes can improve clinical trial speed and efficiency and reduce cost. This review summarizes and synthesizes best practices in bringing anti-cancer medicines to the clinic while integrating recent guidance's from the US Food and Drug Administration.