Scientific process, pharmacology and drug discovery

Scientific method in drug discovery has centered on generating a hypothesis (target identification) and hypothesis testing (target validation). Traditionally, both processes were performed using animal data, with the basic pharmacologist being pivotal. Many therapeutic areas that rely on human data to validate targets as animal models are seen as poorly predictive. Failures of molecules in Phase III for poor efficacy raise questions about target identification and validation. The clinical pharmacologist, working with the basic pharmacologist can play a major role in aiding target identification and by developing trial designs using small patient populations, mitigating the need for full Phase III studies to test the hypothesis. Technologies such as genomics, non-invasive imaging and proteomics are in the forefront of improving target identification and in some cases in providing paradigms for target validation in man.

► Hypothesis generation and validation in drug discovery is changing.
► Human data is becoming essential in identifying and validating novel targets.
► The roles of the basic and clinical pharmacologist are becoming more blurred.
► Technological innovation is an essential enabler for future success.