比利时鲁汶大学Jeroen Luyten研究小组对2009-2018年将新药推向市场所需的研发投资进行了估算。2020年3月3日出版的《美国医学会杂志》发表了这项成果。

开发新药的平均成本一直是争议话题，最近估计在3.14亿至28亿美元之间。

为了使用公开数据来估算将新治疗药物推向市场所需的研发投资，研究组分析了2009年至2018年美国食品药品监督管理局（FDA）批准的新药数据，以此估算将新药推向市场所需的研发支出。

在研究期内，FDA批准了355种新药和生物制剂。47家不同公司开发的63种（18％）产品的研发支出数据可使用。把试验失败的费用也考虑在内，将新药推向市场的研发投资中位数估计为9.853亿美元，基础案例分析中的中位投资估计为13.36亿美元。

按治疗领域（每种领域至少5种药物）来分，投资中位数最低的为神经系统药物，为7.659亿美元；最高的为抗肿瘤药和免疫调节剂，为27.72亿美元。数据主要为较小公司、孤儿药、特定治疗领域的产品、一级药物、获得加速批准的治疗剂以及2014至2018年间批准的产品数据。

使用不同的临床试验成功率、临床前支出和资本成本估算值进行敏感性分析时，结果会有所不同。

附：英文原文

Title: Estimated Research and Development Investment Needed to Bring a New Medicine to Market, 2009-2018

Author: Olivier J. Wouters, Martin McKee, Jeroen Luyten

Issue&Volume: 2020/03/03

Abstract: Importance  The mean cost of developing a new drug has been the subject of debate, with recent estimates ranging from $314 million to $2.8 billion.Objective  To estimate the research and development investment required to bring a new therapeutic agent to market, using publicly available data.Design and Setting  Data were analyzed on new therapeutic agents approved by the US Food and Drug Administration (FDA) between 2009 and 2018 to estimate the research and development expenditure required to bring a new medicine to market. Data were accessed from the US Securities and Exchange Commission, Drugs@FDA database, and ClinicalTrials.gov, alongside published data on clinical trial success rates.Exposures  Conduct of preclinical and clinical studies of new therapeutic agents.Main Outcomes and Measures  Median and mean research and development spending on new therapeutic agents approved by the FDA, capitalized at a real cost of capital rate (the required rate of return for an investor) of 10.5% per year, with bootstrapped CIs. All amounts were reported in 2018 US dollars.Results  The FDA approved 355 new drugs and biologics over the study period. Research and development expenditures were available for 63 (18%) products, developed by 47 different companies. After accounting for the costs of failed trials, the median capitalized research and development investment to bring a new drug to market was estimated at $985.3 million (95% CI, $683.6 million-$1228.9 million), and the mean investment was estimated at $1335.9 million (95% CI, $1042.5 million-$1637.5 million) in the base case analysis. Median estimates by therapeutic area (for areas with ≥5 drugs) ranged from $765.9 million (95% CI, $323.0 million-$1473.5 million) for nervous system agents to $2771.6 million (95% CI, $2051.8 million-$5366.2 million) for antineoplastic and immunomodulating agents. Data were mainly accessible for smaller firms, orphan drugs, products in certain therapeutic areas, first-in-class drugs, therapeutic agents that received accelerated approval, and products approved between 2014 and 2018. Results varied in sensitivity analyses using different estimates of clinical trial success rates, preclinical expenditures, and cost of capital.Conclusions and Relevance  This study provides an estimate of research and development costs for new therapeutic agents based on publicly available data. Differences from previous studies may reflect the spectrum of products analyzed, the restricted availability of data in the public domain, and differences in underlying assumptions in the cost calculations.

DOI: 10.1001/jama.2020.1166

Source: https://jamanetwork.com/journals/jama/article-abstract/2762311