Sertkaya Aylin, Wong Hui-Hsing, Jessup Amber, Beleche Trinidad
Eastern Research Group, Inc., Lexington, MA, USA
Office of Science and Data Policy, Office of the Assistant Secretary for Planning and Evaluation, US Department of Health and Human Services, Washington, DC, USA.
Clin Trials. 2016 Apr;13(2):117-26. doi: 10.1177/1740774515625964. Epub 2016 Feb 8.
The increasing cost of clinical research has significant implications for public health, as it affects drug companies' willingness to undertake clinical trials, which in turn limits patient access to novel treatments. Thus, gaining a better understanding of the key cost drivers of clinical research in the United States is important.
The study which is based on a report prepared by Eastern Research Group, Inc., for the US Department of Health and Human Services, examined different factors, such as therapeutic area, patient recruitment, administrative staff, and clinical procedure expenditures, and their contribution to pharmaceutical clinical trial costs in the United States by clinical trial phase.
The study used aggregate data from three proprietary databases on clinical trial costs provided by Medidata Solutions. We evaluated per-study costs across therapeutic areas by aggregating detailed (per patient and per site) cost information. We also compared average expenditures on cost drivers with the use of weighted mean and standard deviation statistics.
Therapeutic area was an important determinant of clinical trial costs by phase. The average cost of a Phase 1 study conducted at a US site ranged from US$1.4 million (pain and anesthesia) to US$6.6 million (immunomodulation), including estimated site overhead and monitoring costs of the sponsoring organization. A Phase 2 study cost from US$7.0 million (cardiovascular) to US$19.6 million (hematology), whereas a Phase 3 study cost ranged from US$11.5 million (dermatology) to US$52.9 (pain and anesthesia) on average. Across all study phases and excluding estimated site overhead costs and costs for sponsors to monitor the study, the top three cost drivers of clinical trial expenditures were clinical procedure costs (15%-22% of total), administrative staff costs (11%-29% of total), and site monitoring costs (9%-14% of total).
The data were from 2004 through 2012 and were not adjusted for inflation. Additionally, the databases used represented a convenience, that is, non-probability, sample and did not allow for statistically valid estimates of cost drivers. Finally, the data were from trials funded by the global pharmaceutical and biotechnology industry only. Hence, our study findings are limited to that segment.
Therapeutic area being studied as well as number and types of clinical procedures involved were the key drivers of direct costs in Phase 1 through Phase 3 studies. Research shows that strategies exist for reducing the price tag of some of these major direct cost components. Therefore, to increase clinical trial efficiency and reduce costs, gaining a better understanding of the key direct cost drivers is an important step.
临床研究成本的不断增加对公共卫生具有重大影响，因为它影响了制药公司开展临床试验的意愿，进而限制了患者获得新疗法的机会。因此，更好地了解美国临床研究的关键成本驱动因素非常重要。
该研究基于东部研究集团公司为美国卫生与公众服务部编写的一份报告，考察了不同因素，如治疗领域、患者招募、行政人员以及临床程序支出，及其在不同临床试验阶段对美国药物临床试验成本的贡献。
该研究使用了来自Medidata Solutions提供的三个关于临床试验成本的专有数据库的汇总数据。我们通过汇总详细的（按患者和按站点）成本信息来评估各治疗领域每项研究的成本。我们还使用加权均值和标准差统计数据比较了成本驱动因素的平均支出。
治疗领域是不同阶段临床试验成本的重要决定因素。在美国站点进行的1期研究的平均成本从140万美元（疼痛与麻醉）到660万美元（免疫调节）不等，包括申办组织的估计站点间接费用和监测成本。2期研究成本从700万美元（心血管）到1960万美元（血液学），而3期研究成本平均从1150万美元（皮肤病学）到5290万美元（疼痛与麻醉）。在所有研究阶段，排除估计的站点间接费用和申办方监测研究的成本，临床试验支出的三大成本驱动因素是临床程序成本（占总成本的15%-22%）、行政人员成本（占总成本的11%-29%）和站点监测成本（占总成本的9%-14%）。
数据来自2004年至2叭2年，未进行通货膨胀调整。此外，所使用的数据库只是一个便利样本，即非概率样本，无法对成本驱动因素进行具有统计学有效性的估计。最后，数据仅来自全球制药和生物技术行业资助的试验。因此，我们的研究结果仅限于该领域。
所研究的治疗领域以及所涉及的临床程序的数量和类型是1期至3期研究直接成本的关键驱动因素。研究表明，存在降低其中一些主要直接成本组成部分价格标签的策略。因此，为了提高临床试验效率并降低成本，更好地了解关键直接成本驱动因素是重要的一步。
