The Role of Clinical Medicine

Abstract

This chapter outlines the interface between specific, therapeutic-area focussed, drug repurposing objectives to decisions, and various approaches, surrounding the subsequent configuration and conduct of clinical trials. Ideally, these clinical trials will provide definitive proof of concept, safety, tolerability and efficacy in patient studies that lead to regulatory approval. We have never come across a how-to guide that helps with establishing and running a major drug repurposing clinical trial programme, so we have had to learn by experience alone. We therefore attempt here to offer suggestions about how such objectives can be achieved and share details of ways of solving some of the various expected and unexpected topics that are often encountered and necessitate solving in order to maintain forward momentum. To provide this practical experience we turn to, and share, what we have learned during our decade-long, global drug repurposing programme, now involving >20 repurposed drugs, and which focuses on neuroprotective clinical trials of 1–2 years duration in patients with Parkinson's disease. We concentrate on ensuring the maintenance of high quality in all aspects of our repurposing initiative as this helps it grow much faster with the active help of clinicians, non-clinical academics, patients, pharmaceutical companies and governments.