Tufts Center for the Study of Drug Development, Tufts University, Boston, Massachusetts, USA.
Clin Pharmacol Ther. 2010 Mar;87(3):272-7. doi: 10.1038/clpt.2009.295. Epub 2010 Feb 3.
This study utilizes both public and private data sources to estimate clinical phase transition and clinical approval probabilities for drugs in the development pipelines of the 50 largest pharmaceutical firms (by sales). The study examined the development histories of these investigational compounds from the time point at which they first entered clinical testing (1993-2004) through June 2009. The clinical approval success rate in the United States was 16% for self-originated drugs (originating from the pharmaceutical company itself) during both the 1993-1998 and the 1999-2004 subperiods. For all compounds (including licensed-in and licensed-out drugs in addition to self-originated drugs), the clinical approval success rate for the entire study period was 19%. The estimated clinical approval success rates and phase transition probabilities differed significantly by therapeutic class. The estimated clinical approval success rate for self-originated compounds over the entire study period was 32% for large molecules and 13% for small molecules. The estimated transition probabilities were also higher for all clinical phases with respect to large molecules.
本研究利用公共和私人数据源，估算了 50 家最大制药公司（按销售额计）研发管道中药物的临床阶段转变和临床批准概率。该研究考察了这些研究化合物从首次进入临床测试（1993-2004 年）到 2009 年 6 月的发展历史。在 1993-1998 年和 1999-2004 年两个子期间，自行研发药物（源自制药公司自身）在美国的临床批准成功率为 16%。对于所有化合物（包括许可引进和许可输出药物以及自行研发药物），整个研究期间的临床批准成功率为 19%。不同治疗类别之间的估计临床批准成功率和阶段转变概率差异显著。整个研究期间，自行研发化合物的大型分子的临床批准成功率估计为 32%，小分子为 13%。对于所有临床阶段，大型分子的估计转变概率也较高。
