Validation is a crucial step in the drug-discovery process and has been proposed as the major reason for later failure of drug discovery projects. Most drugs are inhibitors that block the action of a particular target protein. But the only way to be completely certain that a protein is instrumental in a given disease is to test the idea in humans. Obviously such clinical trials cannot be used for initial drug discovery, which means that a potential target must undergo a validation process — its role in disease must be clearly defined before drugs are sought that act against it, patient populations susceptible to intervention using this target must be identified, and simple experiments carried out to confirm regulation of the target (or its downstream effects) have the expected affect in a cellular system.
We believe the link between Drug Discovery and the Clinical setting to be critical for improving success rates for translating fundamental scientific discoveries into new treatments for patients. To that end it is vital that clinical observations are used to direct and focus drug discovery objectives at an early stage. These include (i) information on genetic backgrounds thought to be susceptible to specific intervention using s mall molecules, (ii) opportunities for co-dosing a potential treatment with emerging therapies and (iii) early evaluation of drug candidates in well annotated tissue banks. We anticipate all such activities will reduce attrition rates in phase 2 and phase 3 clinical trials.