The Ann Hayes Consultancy Buntingford, Hertfordshire, United Kingdom.
Pharmacol Res Perspect. 2015 Aug;3(4):e00163. doi: 10.1002/prp2.163. Epub 2015 Jul 24.
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to promote the publication of clinical data, but many clinical results, particularly negative results, remain unpublished, are published in obscure places or are published after a considerable delay.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.
近年来,药物发现的效率并没有显著提高,一个关键问题是由于最初的概念验证临床研究中的疗效失败而导致大量淘汰。这反映了一个事实,即临床前的“靶标验证”研究并不总是能转化为人类,而最终的靶标验证必须存在于人类中。鉴于这些事实,很容易看出这些首次临床验证研究对整体靶标验证的重要性。有各种举措来促进临床数据的发表,但许多临床结果,特别是负面结果,仍然没有发表,发表在不为人知的地方,或者发表的时间相当滞后。这就产生了一种可能性,即大量的资金可能会花在那些分子靶标实际上已经被否定的化合物上,但这些数据并不公开。《药理学、研究与展望》表示,它将欢迎发表所有与靶标验证相关的数据,特别是负面数据(临床和临床前),并且它将以一种相对快速和容易实现的形式来发表。