Key role of publication of clinical data for target validation

Abstract

Drug discovery productivity has failed to improve markedly in recent years and a key issue is high attrition due to efficacy failures in initial proof-of concept clinical studies. This reflects the fact that preclinical “target validation” studies do not always translate into man, where ultimate target validation must reside. Given these facts, it is easy to see the importance of results from those first clinical validation studies for overall target validation. There are various initiatives to promote the publication of clinical data, but many clinical results, particularly negative results, remain unpublished, are published in obscure places or are published after a considerable delay. This gives rise to the potential that vast sums of money could be spent on compounds for which the molecular target has essentially been invalidated, but the data are not publicly available. Pharmacology, Research and Perspectives has indicated that it will welcome publication of all data pertinent to target validation, particularly negative data (clinical and preclinical), and it will do so in a form that should be relatively rapid and easy to achieve.