How to Avoid Surprises in Small-Molecule Drug Development Programs

Over the years, the pharmaceutical industry has made steady progress in improving the safety of drugs in development. Consequently, in 2015, only 14 percent of Phase III studies failed due to safety (compared to 55 percent for efficacy), down from 21 percent in 2013. In other words, “efforts to remove unsafe agents earlier in the development cycle are paying off.” Much of this success in small molecules relates to careful and methodical work done well before the Investigational New Drug (IND) application.

Preparing a pre-clinical program to support an IND application takes time and is resource intensive, which is why it is often tempting for companies to “rush to the finish line” and skip certain steps. There is ample value in the long run, however, in taking the necessary time to follow best practices in exploratory toxicology testing. An investment at the right stage will avoid unpleasant surprises that can result in otherwise avoidable costs and delays often amounting to millions of dollars and months – even years – added to the development timeline.

The following paper provides practical advice on how to usher a small molecule through preliminary toxicology testing and derive information required to design IND-enabling studies. Indeed, with a rigorous exploratory toxicity program, sponsors can mitigate the risk of unexpected challenges and optimize the Good Laboratory Practice (GLP) study plan.