Proof of concept

I was asked yesterday what is a proof of concept, so we are going to get a bit "techie" today. A proof of concept study is a trial to demonstrate clinical efficacy with a small number of strictly selected patients. It basically shows early evidence of the potential clinical efficacy of a new drug. It is an important concept for all biotech initiatives.

The objectives of the Proof of Concept Study are (a) to validate the relevance of novel therapeutic targets and in vivo preclinical models, (b) define potential biological markets for clinical efficacy, and (c) provide an assessment of the commercial potential of “new chemical entities” (NCE).

Proof of concept may be demonstrated sometimes through experiments performed at the cellular level and on animals, resulting in data that can reasonably be extrapolated to realistic predictions of significance and human trial results. Investors feel safer if proof of concept has been established, meaning the biotech start-up has demonstrated, at least in preliminary experiments that the idea will work.

When undergoing a proof of concept study, it is essential to have well defined criteria for decision-making according to its results. Among the things you would be looking at in a proof of concept study you may find:

• 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