Discovery Performance Unit on Neuronal Targets, Respiratory Disorder Therapeutic Area, GlaxoSmithKline ltd., King of Prussia, PA 19123, USA.
Curr Opin Pharmacol. 2011 Oct;11(5):557-62. doi: 10.1016/j.coph.2011.06.010. Epub 2011 Jul 21.
The discovery of novel drugs is a complex and highly regulated process organized around a critical moment, that is, when the novel compound is tested in humans. This process encompasses a series of clinical studies, identified as Phase I and Phase II, whose composite outcome should deliver the data needed for an informed decision about progressing or not the compound in full development (Phase III). Over the last 10 years the global delivery of novel treatments from the pharmaceutical industry has plunged to the level of the '70ies in spite of a 10-fold larger investment, the differential mostly due to failures in Phase III. There is the need to improve the decision making at the early clinical stage by using innovation and the high-profile achievements of basic science generated in academic and biomedical labs. A specific attention should be paid to applied biotechnologies, in particular nanotechnology and biomedical devices not only for drug deliver but also for biomarker detection. This path, also supported by regulatory agencies, is calling for an important change of perspective about how drug discovery is made, which we believe should start from the full implementation of the paradigm of Translational Medicine.
新药的发现是一个复杂而高度规范的过程，其核心是新型化合物在人体中的测试。这一过程包括一系列临床研究，分为 I 期和 II 期，综合结果应提供关于是否继续全面开发该化合物（III 期）所需的数据。尽管投资增加了 10 倍，但过去 10 年来，制药行业向全球提供的新型治疗方法却降至 70 年代的水平，这主要是由于 III 期临床试验失败所致。有必要通过创新和学术及生物医学实验室中产生的基础科学的突出成就，在早期临床阶段改进决策。应特别关注应用生物技术，特别是纳米技术和生物医学设备，不仅用于药物输送，还用于生物标志物检测。这条道路也得到了监管机构的支持，呼吁对药物发现的方式进行重要的观念转变，我们认为，这应该从全面实施转化医学的范例开始。
