新药研发成本的系统性综述

目的:系统性地梳理和分析国内外新药研发成本的实证研究。方法:计算机检索CNKI,WanFang,VIP,PubMed,EconLit等数据库,搜集国内外公开发表的有关新药研发成本的研究,检索时限为建库至2018年7月。根据纳入与排除标准独立筛选文献、提取信息,从研究方法、研究类群及数据来源、新药研发时长及成功率、新药研发成本等对纳入文献进行统计分析。结果:最终纳入文献15篇,新药研发成本从1979年的1.8亿美元上升到2016年的26.5亿美元,相差约15倍。而同一时期的研究由于研究样本、数据来源与研究设计的不同,得出的新药研发成本也有较大差异。新药研发临床阶段的总成功率为10.7%～30.2%,完成一个新药的临床试验平均需要58个月(约5年)。结论:尽管关于新药研发成本的研究已开展了近40年,但仍然没有一项研究被视为"金标准",在研究方法的合理性以及研究结果的可重复性和可对比性方面还有待提升。

Systematic review of new drug research and development (R&D) cost

Objective: To sort out and analyze the empirical research on the cost of research and development of new drugs in China and abroad systematically. Methods: Databases including CNKI, WanFan, VIP, PubMed, and EconLit, et al were searched. The research on the cost of research and development of new drugs published in China and abroad from the databases establishment to July 2018 were enrolled. According to the inclusion and exclusion criteria, the literature was selected independently and the information was extracted. Statistical analysis was carried out from the aspects of research method, research group, data source, research time and success rate of new drug development. Results: Finally, 15 articles were included. The cost of research and development of new drugs increased from 0.18 billion US dollars in 1979 to 2.65 billion US dollars in 2016, with a difference of about 15 times. In the same period, the costs of different studies on research and development of new drugs were also quite different due to the differences in research samples, data sources and research design. The total success rate of new drug development in the clinical phase was between 10.7%~30.2%. It took an average of 58 months(about 5 years) to complete a clinical trial of a new drug. Conclusion: Although the research on the cost of research and development of new drugs has been underway for nearly 40 years, no single study has been considered a "gold standard", and the rationality of the research method and the reproducibility and comparability of the research results remain to be improved.