TY - JOUR
T1 - Estimated costs of pivotal trials for U.S. Food and Drug Administration–approved cancer drugs, 2015–2017
AU - Hsiue, Emily Han Chung
AU - Moore, Thomas J.
AU - Alexander, G. Caleb
N1 - Publisher Copyright:
© The Author(s) 2020.
PY - 2020/4/1
Y1 - 2020/4/1
N2 - Background: Pivotal clinical trials provide critical evidence to regulators regarding a product’s suitability for marketing approval. The objectives of this study are (1) to characterize select features of trials for oncology products approved by the U.S. Food and Drug Administration between 2015 and 2017; and (2) to quantify the costs of these trials and how such costs varied based on trial characteristics. Methods: We identified novel oncology therapeutic drugs, and their respective pivotal trials, approved between 2015 and 2017 using annual summary reports from the Food and Drug Administration. Cost estimates for each pivotal trial were calculated using IQVIA’s CostPro, a clinical trial cost estimating tool based on executed contracts between pharmaceutical manufacturers and contract research organizations. Measures of drug and trial characteristics included trial design, end point, patient enrollment, and regulatory pathway. We also performed sensitivity analyses that varied assumptions regarding how efficiently each trial was conducted. Results: A total of 39 pivotal clinical trials provided the basis for Food and Drug Administration approval of 30 new oncology drugs from 2015 to 2017. Among these trials, primary end points were objective response rate in 20 (51.3%), progression-free survival in 13 (33.3%), and overall survival in 6 (15.4%). Twenty trials (51.3%) were single-arm studies. The median estimated cost of oncology pivotal trials was $31.7 million (interquartile range = $17.0–$60.4 million). Trials with objective response rate as primary end point had a median estimate of $17.7 million (interquartile range = $11.9–$27.1 million), compared with trials examining progression-free survival ($42.3 million, interquartile range = $34.6–$101.2 million) or overall survival ($79.4 million, interquartile range = $56.9–$97.7 million) (p < 0.001). Estimated costs for single-arm trials ($17.7 million, interquartile range = $11.9–$23.7 million) were less than for trials with a placebo-controlled ($56.7 million, interquartile range = $40.9–$103.9 million) or active control arm ($67.6 million, IQR = $35.5–$93.5 million) (p < 0.001). Conclusions: Relative to the estimated costs of drug development, the costs of these oncology pivotal trials were modest, with trials that produced more valuable scientific information costing more than their counterparts.
AB - Background: Pivotal clinical trials provide critical evidence to regulators regarding a product’s suitability for marketing approval. The objectives of this study are (1) to characterize select features of trials for oncology products approved by the U.S. Food and Drug Administration between 2015 and 2017; and (2) to quantify the costs of these trials and how such costs varied based on trial characteristics. Methods: We identified novel oncology therapeutic drugs, and their respective pivotal trials, approved between 2015 and 2017 using annual summary reports from the Food and Drug Administration. Cost estimates for each pivotal trial were calculated using IQVIA’s CostPro, a clinical trial cost estimating tool based on executed contracts between pharmaceutical manufacturers and contract research organizations. Measures of drug and trial characteristics included trial design, end point, patient enrollment, and regulatory pathway. We also performed sensitivity analyses that varied assumptions regarding how efficiently each trial was conducted. Results: A total of 39 pivotal clinical trials provided the basis for Food and Drug Administration approval of 30 new oncology drugs from 2015 to 2017. Among these trials, primary end points were objective response rate in 20 (51.3%), progression-free survival in 13 (33.3%), and overall survival in 6 (15.4%). Twenty trials (51.3%) were single-arm studies. The median estimated cost of oncology pivotal trials was $31.7 million (interquartile range = $17.0–$60.4 million). Trials with objective response rate as primary end point had a median estimate of $17.7 million (interquartile range = $11.9–$27.1 million), compared with trials examining progression-free survival ($42.3 million, interquartile range = $34.6–$101.2 million) or overall survival ($79.4 million, interquartile range = $56.9–$97.7 million) (p < 0.001). Estimated costs for single-arm trials ($17.7 million, interquartile range = $11.9–$23.7 million) were less than for trials with a placebo-controlled ($56.7 million, interquartile range = $40.9–$103.9 million) or active control arm ($67.6 million, IQR = $35.5–$93.5 million) (p < 0.001). Conclusions: Relative to the estimated costs of drug development, the costs of these oncology pivotal trials were modest, with trials that produced more valuable scientific information costing more than their counterparts.
KW - Cost
KW - clinical trial
KW - oncology
UR - http://www.scopus.com/inward/record.url?scp=85081602896&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85081602896&partnerID=8YFLogxK
U2 - 10.1177/1740774520907609
DO - 10.1177/1740774520907609
M3 - Article
C2 - 32114790
AN - SCOPUS:85081602896
SN - 1740-7745
VL - 17
SP - 119
EP - 125
JO - Clinical Trials
JF - Clinical Trials
IS - 2
ER -