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Abstract
Objectives Little is routinely disclosed about the costs of the pivotal clinical trials that provide the key scientific evidence of the treatment benefits of new therapeutic agents. We expand our earlier research to examine why the estimated costs may vary 100-fold.
Design A cross-sectional study of the estimated costs of the pivotal clinical trials supporting the approval of 101 new therapeutic agents approved by the US Food and Drug Administration from 2015 to 2017.
Methods We licensed a software tool used by the pharmaceutical industry to estimate the likely costs of clinical trials to be conducted by contract research organisations. For each trial we collected 52 study characteristics. Linear regression was used to assess the most important factors affecting costs.
Primary and secondary outcome measures The mean and 95% CI of 225 pivotal clinical trials using varying assumptions. We also assessed median estimated costs per patient, per clinic visit and per drug.
Results Measured as pivotal trials cost per approved drug, the 101 new molecular entities had an estimated median cost of US$48 million (IQR US$20 million–US$102 million). The 225 individual clinical trials had a median estimate of US$19 million (IQR US$12 million–US$33 million) per trial and US$41 413 (IQR, US$29 894–US$75 047) per patient. The largest single factor driving cost was the number of patients required to establish the treatment effects and varied from 4 patients to 8442. Next was the number of trial clinic visits, which ranged from 2 to 166. Our statistical model showed trial costs rose exponentially with these two variables (R2=0.696, F=257.9, p<0.01).
Conclusions The estimated costs are modest for measuring the benefits of new therapeutic agents but rise exponentially as more patients and clinic visits are required to establish a drug effect.
- clinical pharmacology
- epidemiology
- medical law
- public health
- clinical trials
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Footnotes
Contributors Conception and design: TJM, GA, GCA. Data acquisition and analysis: JH, TJM, GCA. Data interpretation: TJM, GCA, GA. Drafting of the manuscript: TJM. Critical revision of the manuscript for important intellectual content: GCA, GA, JH. Final approval of the version to be submitted: TJM, GCA, JH, GA. TJM takes full responsibility for the integrity of the data and analyses.
Funding This project was supported in part by a grant from the Laura and John Arnold Foundation and in part by grant U01 FD005942 (JH and GCA) from the Johns Hopkins Bloomberg School of Public Health, Center of Excellence in Regulatory Science and Innovation, a collaborative research initiative with the US Food and Drug Administration. The funding sources had no role in the design, conduct or reporting of this study.
Disclaimer This article is based in part on data obtained under license from the following IQVIA information service: CRO CostPro Clinical Trial Optimization Solutions. All Rights Reserved. The statements, findings, conclusions, views and opinions contained and expressed herein are not necessarily those of IQVIA or any of its affiliated or subsidiary entities.
IQVIA had no role whatever in the design, analysis, results and preparation of or review of manuscript content (except for statements about IQVIA). GCA role was negotiating with IQVIA to obtain and pay for a license for Johns Hopkins University to provide access to the data, which had been previously limited to internal use at life science companies.
Competing interests GCA is past Chair of FDA’s Peripheral and Central Nervous System Advisory Committee; serves as a paid advisor to IQVIA; is a paid consultant and holds equity in Monument Analytics, a healthcare consultancy whose clients include the life sciences industry as well as plaintiffs in opioid litigation and is a member of OptumRx’s National P&T Committee. This arrangement has been reviewed and approved by Johns Hopkins University in accordance with its conflict of interest policies.
Patient and public involvement Patients and/or the public were not involved in the design, or conduct, or reporting, or dissemination plans of this research.
Patient consent for publication Not required.
Ethics approval Declared exempt from review by the Johns Hopkins School of Public Health Institutional Review Board because the source data did not contain identifiable human data.
Provenance and peer review Not commissioned; externally peer reviewed.
Data availability statement No data are available. The cost estimates for this study were obtained under license and are not available for sharing.