A Proof of Concept study is a trial to demonstrate clinical efficacy with a small number of strictly selected patients.

The objectives of the Proof of Concept Study:

• Validation of the relevance of novel therapeutic targets and in vivo preclinical models to man. 
• Define potential biological markets for clinical efficacy or toxicity. 
• Provide earlier evidence of the potential to show clinical efficacy. 
• Rapidly eliminate potential failures from the pipeline. 
• Provide an assessment of the commercial potential of NCE development candidates.

It is essential to have well defined criteria for decision-making at all stages of the R&D process. This is particularly true for those that will result in a NO GO decision.

Potential GO/NO GO Decision criteria:

• Safety 
• Tolerability 
• Bioavailability/Pharmacokinetics (PK) 
• Biopharmaceutics 
• Pharmacodynamics (PD) 
•     Duration of action 
•     Relationship between Dose and PD 
• Efficacy 
• Patient acceptability 
• Commercial viability in the indication at potential time of launch