Department of Health Policy, London School of Economics and Political Science, London, United Kingdom.
Department of Health Services Research and Policy, London School of Hygiene and Tropical Medicine, London, United Kingdom.
JAMA. 2020 Mar 3;323(9):844-853. doi: 10.1001/jama.2020.1166.
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.
To estimate the research and development investment required to bring a new therapeutic agent to market, using publicly available data.
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.
Conduct of preclinical and clinical studies of new therapeutic agents.
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.
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.
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.
开发一种新药的平均成本一直是争论的主题，最近的估计范围从 3.14 亿美元到 28 亿美元。
使用公开数据估算将新治疗药物推向市场所需的研发投资。
分析了美国食品和药物管理局 (FDA) 在 2009 年至 2018 年间批准的新治疗药物的数据，以估算将一种新药物推向市场所需的研发支出。数据来自美国证券交易委员会、Drugs@FDA 数据库和 ClinicalTrials.gov，以及临床研究成功率的已发表数据。
新治疗药物的临床前和临床研究的开展。
FDA 批准的新治疗药物的中位数和平均值研发支出，按每年 10.5%的实际资本成本率（投资者的要求回报率）进行资本化，并进行了自举置信区间。所有金额均以 2018 年美元报告。
在研究期间，FDA 批准了 355 种新药物和生物制剂。对于 47 家不同公司开发的 63 种（18%）产品，可获得研发支出数据。在考虑到失败试验的成本后，将一种新药推向市场的资本化研发投资中位数估计为 9.853 亿美元（95%CI，6.836 亿美元至 12.289 亿美元），而在基础案例分析中，平均投资估计为 13.359 亿美元（95%CI，10.425 亿美元至 16.375 亿美元）。按治疗领域（≥5 种药物）的中位数估计，从神经系统药物的 7.659 亿美元（95%CI，3.230 亿美元至 14.735 亿美元）到抗肿瘤和免疫调节药物的 27.716 亿美元（95%CI，20.518 亿美元至 53.662 亿美元）不等。数据主要可用于较小的公司、孤儿药、某些治疗领域的产品、同类首创药物、获得加速批准的治疗药物以及 2014 年至 2018 年批准的产品。使用不同的临床试验成功率、临床前支出和资本成本估算进行敏感性分析，结果会有所不同。
本研究根据公开数据估算了新治疗药物的研发成本。与以往研究的差异可能反映了分析产品的范围、公共领域数据的有限可用性以及成本计算中基本假设的差异。
