Institutions, Markets, Technologies Institute for Advanced Studies Lucca, Piazza S. Ponziano 6, 55100 Lucca, Italy.
Nat Rev Drug Discov. 2011 Jun;10(6):428-38. doi: 10.1038/nrd3405.
Advances in the understanding of the molecular basis of diseases have expanded the number of plausible therapeutic targets for the development of innovative agents in recent decades. However, although investment in pharmaceutical research and development (R&D) has increased substantially in this time, the lack of a corresponding increase in the output in terms of new drugs being approved indicates that therapeutic innovation has become more challenging. Here, using a large database that contains information on R&D projects for more than 28,000 compounds investigated since 1990, we examine the decline of R&D productivity in pharmaceuticals in the past two decades and its determinants. We show that this decline is associated with an increasing concentration of R&D investments in areas in which the risk of failure is high, which correspond to unmet therapeutic needs and unexploited biological mechanisms. We also investigate the potential variations in productivity with regard to the regional location of companies and find that although companies based in the United States and Europe differ in the composition of their R&D portfolios, there is no evidence of any productivity gap.
近年来，对疾病分子基础的认识不断深入，为开发创新药物提供了更多可能的治疗靶点。然而，尽管在此期间医药研发（R&D）的投资大幅增加，但新药批准数量却没有相应增加，这表明治疗创新变得更加具有挑战性。在这里，我们使用一个大型数据库，其中包含自 1990 年以来对 28000 多种化合物进行的 R&D 项目的信息，研究了过去二十年医药 R&D 生产力的下降及其决定因素。我们表明，这种下降与 R&D 投资在高失败风险领域的集中有关，这些领域对应着未满足的治疗需求和未开发的生物学机制。我们还研究了公司地域位置对生产力的潜在影响，发现尽管美国和欧洲的公司在其 R&D 组合的构成上存在差异，但没有证据表明存在任何生产力差距。
