Gotham Dzintars, Martin Manuel, Barber Melissa J, Kazounis Emil, Batts Charlotte, Scourse Rosalind, Elder Greg, Nyang'wa Bern-Thomas
MSF Access Campaign, Geneva, Switzerland.
Yale Collaboration for Regulatory Rigor, Integrity, and Transparency (CRRIT), New Haven, United States of America.
PLOS Glob Public Health. 2025 Apr 23;5(4):e0003759. doi: 10.1371/journal.pgph.0003759. eCollection 2025.
Clinical trials are considered to be the largest contributor to pharmaceutical development costs. However, public disclosure of the costs of individual clinical trials is rare. Médecins Sans Frontières (MSF) sponsored a phase 2b-3 randomised controlled trial (TB-PRACTECAL), which identified a new treatment regimen for drug-resistant TB. We aimed to analyse the costs of undertaking a pivotal clinical trial conducted in relatively low-resource health settings and to demonstrate the feasibility of reporting clinical trial costs. TB-PRACTECAL trial costs were analysed using MSF accounting documents. Costs were broken down by cost category, year, and trial site. Total costs for TB-PRACTECAL were €33.9 million and the average cost per patient was €61,460. Twenty-six percent of total costs represented central activities (e.g. trial planning, trial management) and 72% represented trial site activities, with 2% uncategorizable. Within trial site costs, personnel costs were the largest cost (43%) followed by external diagnostic services (11%), medicines (9%), and other medical consumables (7%). Cost variation across trial sites was driven by different varying levels of pre-existing trial infrastructure. A review of previous studies yielded a wide range of cost estimates for clinical trials (ranging US$7-221 million/trial for pharmaceutical phase 2 and 3 trials). Nearly all previous estimates derive from industry reporting that is neither standardized nor auditable; to our knowledge, this is the first published comprehensive analysis of direct expenditures of a specific clinical trial including detailed cost breakdowns. The €34 million cost of TB-PRACTECAL included investments in developing clinical trial infrastructure, the complexity of managing six sites across three health systems, and medical expenditures that are not typical of standard clinical trials. Greater transparency in drug development costs can inform medicine pricing negotiations and is a key element in the design and implementation of more equitable systems of biomedical research and development.
临床试验被认为是制药研发成本的最大组成部分。然而,个别临床试验成本的公开披露却很少见。无国界医生组织(MSF)赞助了一项2b-3期随机对照试验(TB-PRACTECAL),该试验确定了一种耐多药结核病的新治疗方案。我们旨在分析在资源相对匮乏的卫生环境中进行关键临床试验的成本,并证明报告临床试验成本的可行性。使用无国界医生组织的会计文件分析了TB-PRACTECAL试验的成本。成本按成本类别、年份和试验地点进行了细分。TB-PRACTECAL的总成本为3390万欧元,每位患者的平均成本为61460欧元。总成本的26%为核心活动(如试验规划、试验管理),72%为试验地点活动,2%无法归类。在试验地点成本中,人员成本是最大的成本(43%),其次是外部诊断服务(11%)、药品(9%)和其他医疗耗材(7%)。试验地点之间的成本差异是由不同的现有试验基础设施水平驱动的。对先前研究的回顾得出了临床试验成本的广泛估计(制药2期和3期试验的成本估计为每项试验700万-2.21亿美元)。几乎所有先前的估计都来自行业报告,这些报告既不规范也不可审计;据我们所知,这是首次发表的对特定临床试验直接支出的全面分析,包括详细的成本细分。TB-PRACTECAL的3400万欧元成本包括对开发临床试验基础设施的投资、管理三个卫生系统中六个地点的复杂性以及非标准临床试验典型的医疗支出。提高药物研发成本的透明度可以为药品定价谈判提供信息,并且是设计和实施更公平的生物医学研发系统的关键要素。