Prurisol: What is a Proof of Concept Trial? (Seel

Prurisol: What is a Proof of Concept Trial? (Seel can explain more)

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