Proof of concept is an experiment and the experimental design is critical - pharmacology!
The definition of science from Sir Karl Popper comes down to what can be experimentally tested and falsified.
Furthermore the new areas opening up (see below) clearly indicates that much of our preconceptions are WRONG. This opens up major opportunities.
The key issue is to understand the disease process, to find the main molecular lever(s) which can change it and to design drugs which can reach their site of action for the appropriate time without causing unacceptable side effects AND to have powerful experimental tests preclinically, clinically (phase I, II and III) which define drug activity – and measure the same thing !
So why aren’t we more efficient with the vast amount of new knowledge ? Three reasons:
- First human beings evolved to run, massively increasing metabolism and the brain, while having a long lifespan – this isn’t normally taken into account by pharma.
- Second, the exponential increase in knowledge is increased by an exceptional number of variables which we don’t control (sorry systems biology prophets..) Hence, the experimental design is critical.
- Third, we are now realising that there are many new areas of knowledge where we aren’t experts – and where new (expensive) mistakes can be made.
A solution? Paul Chapman (GM Takeda pharma)
Competitive advantage in industry comes from making better judgements based on all available data and using it to create better molecules; everything else can and should be shared.
How to optimise this knowledge for drug discovery and development ? How to exploit consortia ?
Our solutions are to use experts from many different fields to give open recommendations on the state of a new area and to clearly indicate what we don’t know and where the variables are.
Create consortia with multidisciplinary experts for serious diseases.
This has enabled a major breakthrough in ALS.