TY - JOUR
T1 - Globalization of clinical trials
T2 - Variation in estimated regional costs of pivotal trials, 2015–2016
AU - Qiao, Yao
AU - Alexander, G. Caleb
AU - Moore, Thomas J.
N1 - Funding Information:
The authors thank Hanzhe Zang BA of the Johns Hopkins Bloomberg School of Public Health and Dora Lin MHS of the Carle Illinois College of Medicine for their assistance in using the CostPro software tool to calculate estimated costs for each trial. They were compensated for this work. The author(s) disclosed receipt of the following financial support for the research, authorship, and/or publication of this article: This project was supported in part by a grant from the Laura and John Arnold Foundation and in part by a Center of Excellence in Regulatory Science and Innovation (CERSI) grant to Johns Hopkins University from the US Food & Drug Administration (U01FD005942-01). Its contents are solely the responsibility of the authors and do not necessarily represent the official views of the HHS or FDA. The funding sources had no role in the design and conduct of the study, analysis, or interpretation of the data and preparation or final approval of the manuscript prior to publication. The findings and conclusions of this study are those of the authors and do not necessarily reflect the views of the US Food and Drug Administration.
Funding Information:
The author(s) disclosed receipt of the following financial support for the research, authorship, and/or publication of this article: This project was supported in part by a grant from the Laura and John Arnold Foundation and in part by a Center of Excellence in Regulatory Science and Innovation (CERSI) grant to Johns Hopkins University from the US Food & Drug Administration (U01FD005942-01). Its contents are solely the responsibility of the authors and do not necessarily represent the official views of the HHS or FDA. The funding sources had no role in the design and conduct of the study, analysis, or interpretation of the data and preparation or final approval of the manuscript prior to publication. The findings and conclusions of this study are those of the authors and do not necessarily reflect the views of the US Food and Drug Administration.
Publisher Copyright:
© The Author(s) 2019.
PY - 2019/6/1
Y1 - 2019/6/1
N2 - Background/Aims: Despite the increasing globalization of clinical trials, little is known regarding how the trial site costs vary around the world. We quantified the geographical distribution and regional cost differences for the clinical trials that established the benefits for new therapeutic drugs approved by the US Food and Drug Administration in 2015 and 2016. Methods: We included all pivotal clinical trials for 59 new molecular entities approved by the US Food and Drug Administration in 2015 and 2016 that included at least one site in North America. We derived cost estimates from IQVIA’s CostPro, a global clinical trial cost-estimating tool used by pharmaceutical sponsors. We assessed the patient and site allocation of these trials across eight geographic regions. To quantify the region-specific cost differences, we conducted a within-trial comparison by expressing the estimated regional costs associated with the sites in each global region as a percent of the same costs in North America. We also estimated the percentage breakdown of regional cost components (pass-through, site management, regulatory, and study conduct costs) for each trial and for all endpoints reported the median and interquartile range. Results: Overall, 127 pivotal clinical trials enrolled 91,415 patients from 13,264 sites. Most patients (60.3%) and sites (57.3%) were outside North America. A median of 66% of the total estimated trial costs (interquartile range: 60%–72%) were spent on regional tasks, with the largest share (53.3%) going directly to trial sites and the remainder going to other regional trial management tasks. Differences were greatest in four lower cost regions: Africa, with an estimated regional cost per site of 49% of North America (interquartile range: 44%–56%), Central Europe 50% (interquartile range: 41%–63%), Middle East 53% (interquartile range: 42%–64%) and Latin America 59% (interquartile range: 50%–70%). Overall, 90 (71%) of the 127 pivotal trials had a total of 3160 sites in these lower cost regions. In contrast, savings were more limited in Western Europe, Oceania, and Asia, where estimated regional costs were 78% of North America (interquartile range: 67%–89%). One-quarter of the trials with sites in Asia and Oceana did not achieve cost savings in those regions relative to North America. Conclusion: Among this sample of pivotal trials for recently approved US Food and Drug Administration products, most patients and sites enrolled were outside of North America, with selection of regional sites having a significant impact on total trial costs.
AB - Background/Aims: Despite the increasing globalization of clinical trials, little is known regarding how the trial site costs vary around the world. We quantified the geographical distribution and regional cost differences for the clinical trials that established the benefits for new therapeutic drugs approved by the US Food and Drug Administration in 2015 and 2016. Methods: We included all pivotal clinical trials for 59 new molecular entities approved by the US Food and Drug Administration in 2015 and 2016 that included at least one site in North America. We derived cost estimates from IQVIA’s CostPro, a global clinical trial cost-estimating tool used by pharmaceutical sponsors. We assessed the patient and site allocation of these trials across eight geographic regions. To quantify the region-specific cost differences, we conducted a within-trial comparison by expressing the estimated regional costs associated with the sites in each global region as a percent of the same costs in North America. We also estimated the percentage breakdown of regional cost components (pass-through, site management, regulatory, and study conduct costs) for each trial and for all endpoints reported the median and interquartile range. Results: Overall, 127 pivotal clinical trials enrolled 91,415 patients from 13,264 sites. Most patients (60.3%) and sites (57.3%) were outside North America. A median of 66% of the total estimated trial costs (interquartile range: 60%–72%) were spent on regional tasks, with the largest share (53.3%) going directly to trial sites and the remainder going to other regional trial management tasks. Differences were greatest in four lower cost regions: Africa, with an estimated regional cost per site of 49% of North America (interquartile range: 44%–56%), Central Europe 50% (interquartile range: 41%–63%), Middle East 53% (interquartile range: 42%–64%) and Latin America 59% (interquartile range: 50%–70%). Overall, 90 (71%) of the 127 pivotal trials had a total of 3160 sites in these lower cost regions. In contrast, savings were more limited in Western Europe, Oceania, and Asia, where estimated regional costs were 78% of North America (interquartile range: 67%–89%). One-quarter of the trials with sites in Asia and Oceana did not achieve cost savings in those regions relative to North America. Conclusion: Among this sample of pivotal trials for recently approved US Food and Drug Administration products, most patients and sites enrolled were outside of North America, with selection of regional sites having a significant impact on total trial costs.
KW - Clinical trials
KW - cost savings
KW - globalization of clinical trials
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U2 - 10.1177/1740774519839391
DO - 10.1177/1740774519839391
M3 - Article
C2 - 30922113
AN - SCOPUS:85066510768
SN - 1740-7745
VL - 16
SP - 329
EP - 333
JO - Clinical Trials
JF - Clinical Trials
IS - 3
ER -