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:
• Bioavailability/Pharmacokinetics (PK)
• Pharmacodynamics (PD)
• Duration of action
• Relationship between Dose and PD
• Patient acceptability
• Commercial viability in the indication at potential time of launch
Proof of concept (POC) may be defined as the earliest point in the drug development process at which the weight of evidence suggests that it is “reasonably likely” that the key attributes for success are present and the key causes of failure are absent. POC is multidimensional but is focused on attributes that, if not addressed, represent a threat to the success of the project in crucial areas such as safety, efficacy, pharmaceutics, and commercial and regulatory issues. The appropriate weight of evidence is assessed through the use of mathematical models and by evaluating the consequences of advancing a candidate drug that is not safe, effective, or commercially viable, vs. failing to advance a candidate that possesses these attributes. Tools for POC include biomarkers, targeted populations, pharmacokinetic (PK)/pharmacodynamic (PD) modeling, simulation,...