The discovery of “pioneer medicines” (i.e. those acting via novel molecular targets) has proven to be an immensely complex, long term, expensive and high risk endeavour. Despite formidable investments by the pharmaceutical industry and public/ charitable funders, over the past few decades in both infrastructure and technology, the success rates have remained low. There are several reasons for this: inadequate understanding of human disease and of the mode of action of existing drugs, poor clinical biomarkers and preclinical models, and an overwhelmingly large choice of potential drug targets. Furthermore, many academic groups and private organisations continue to work “in parallel and in secret” on a relatively narrow sub-set of targets, most of which are destined for failure in subsequent clinical studies. This duplication of effort and hence wastage is perpetuated by the many clinical studies which are not published (or published after a delay, or in insufficient detail). Several groups therefore waste limited resources on such “failed” targets. This poses an ethical dilemma, because current practice is resulting in patients being exposed to molecules for such targets.
During my presentation, I will discuss ways in which we can pool resources to share risk, reduce duplication, improve translation, minimise patient harm and help industry discover new medicines for society.