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Research, Methods, Statistics

Economic Evaluation of Cost and Time Required for a Platform Trial vs Conventional Trials

Educational Objective
To identify the key insights or developments described in this article

1 Credit CME

JAMA Network Open

Published Online: July 12, 2022

Original Investigation

Article Information and Disclosures

Jay J. H. Park, PhD^1,2;

Behnam Sharif, PhD³;

Ofir Harari, PhD³;

Louis Dron, MSc³;

Anna Heath, PhD^4,5,6;

Maureen Meade, MD, MSc^2,7;

Ryan Zarychanski, MD, MSc^8,9;

Raymond Lee, MSc³;

Gabriel Tremblay, DBA³;

Edward J. Mills, PhD²;

Yannis Jemiai, PhD³;

Cyrus Mehta, PhD^3,10;

J. Kyle Wathen, PhD³

Author Affiliations

¹Experimental Medicine, Department of Medicine, University of British Columbia, Vancouver, British Columbia, Canada
²Department of Health Research Methods, Evidence, and Impact, McMaster University Health Sciences Centre, Hamilton, Ontario, Canada
³Cytel, Inc, Waltham, Massachusetts
⁴Child Health Evaluative Sciences, The Hospital for Sick Children, Toronto, Ontario, Canada
⁵Dalla Lana School of Public Health, University of Toronto, Toronto, Ontario, Canada
⁶Department of Statistical Science, University College London, London, United Kingdom
⁷Interdepartmental Division of Critical Care, Hamilton Health Sciences, Critical Care, Hamilton, Ontario, Canada
⁸Department of Internal Medicine, Section of Critical Care, University of Manitoba, Winnipeg, Manitoba, Canada
⁹Department of Internal Medicine, Section of Hematology/Medical Oncology, University of Manitoba, Winnipeg, Manitoba, Canada
¹⁰Department of Biostatistics, Harvard T.H. Chan School of Public Health, Cambridge, Massachusetts

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Key Points

Question What are the cost and time requirements of conducting a single platform trial vs conventional clinical trials?

Findings In this economic evaluation, according to the opinions of platform trial experts elicited and scenarios constructed from the longest ongoing platform trial, conducting a series of 2-group trials for evaluation of 10 interventions showed median (IQR) increases in total trial costs by 57.5% (43.1%-69.9%) and cumulative trial duration by 311.9% (282.0%-349.1%) compared with conducting a single platform trial.

Meaning These findings suggest that despite having larger initial setup requirements, consolidating clinical evaluation of multiple interventions into a single platform trial can drastically reduce cost and efforts.

Abstract

Importance Platform trial design allows the introduction of new interventions after the trial is initiated and offers efficiencies to clinical research. However, limited guidance exists on the economic resources required to establish and maintain platform trials.

Objective To compare cost (US dollars) and time requirements of conducting a platform trial vs a series of conventional (nonplatform) trials using a real-life example.

Design, Setting, and Participants For this economic evaluation, an online survey was administered to a group of international experts (146 participants) with publication records of platform trials to elicit their opinions on cost and time to set up and conduct platform, multigroup, and 2-group trials. Using the reported entry dates of 10 interventions into Systemic Therapy in Advancing Metastatic Prostate Cancer: Evaluation of Drug Efficacy, the longest ongoing platform trial, 3 scenarios were designed involving a single platform trial (scenario 1), 1 multigroup followed by 5 2-group trials (scenario 2), and a series of 10 2-group trials (scenario 3). All scenarios started with 5 interventions, then 5 more interventions were either added to the platform or evaluated independently. Simulations with the survey results as inputs were used to compare the platform vs conventional trial designs. Data were analyzed from July to September 2021.

Exposure Platform trial design.

Main Outcomes and Measures Total trial setup and conduct cost and cumulative duration.

Results Although setup time and cost requirements of a single trial were highest for the platform trial, cumulative requirements of setting up a series of multiple trials in scenarios 2 and 3 were larger. Compared with the platform trial, there was a median (IQR) increase of 216.7% (202.2%-242.5%) in cumulative setup costs for scenario 2 and 391.1% (365.3%-437.9%) for scenario 3. In terms of total cost, there was a median (IQR) increase of 17.4% (12.1%-22.5%) for scenario 2 and 57.5% (43.1%-69.9%) for scenario 3. There was a median (IQR) increase in cumulative trial duration of 171.1% (158.3%-184.3%) for scenario 2 and 311.9% (282.0%-349.1%) for scenario 3. Cost and time reductions in the platform trial were observed in both the initial and subsequently evaluated interventions.

Conclusions and Relevance Although setting up platform trials can take longer and be costly, the findings of this study suggest that having a single infrastructure can improve efficiencies with respect to costs and efforts.

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Article Information

Accepted for Publication: April 21, 2022.

Published: July 12, 2022. doi:10.1001/jamanetworkopen.2022.21140

Open Access: This is an open access article distributed under the terms of the CC-BY License. © 2022 Park JJH et al. JAMA Network Open.

Corresponding Author: Jay J. H. Park, PhD, McMaster University Health Sciences Centre, Department of Health Research Methods, Evidence, and Impact (HEI), 1280 Main St W, Hamilton, ON L8S 4L8 Canada (parkj136@mcmaster.ca).

Author Contributions: Dr Park had full access to all of the data in the study and takes responsibility for the integrity of the data and the accuracy of the data analysis.

Concept and design: Park, Sharif, Mills, Jemiai, Wathen.

Acquisition, analysis, or interpretation of data: Park, Sharif, Harari, Dron, Heath, Meade, Zarychanski, Lee, Tremblay, Mehta, Wathen.

Drafting of the manuscript: Park, Sharif, Harari, Dron, Mills, Wathen.

Critical revision of the manuscript for important intellectual content: Park, Sharif, Heath, Meade, Zarychanski, Lee, Tremblay, Jemiai, Mehta, Wathen.

Statistical analysis: Park, Sharif, Harari, Lee, Mills, Wathen.

Obtained funding: Park.

Administrative, technical, or material support: Park, Sharif, Dron, Zarychanski, Lee, Jemiai.

Supervision: Park, Sharif, Jemiai.

Conflict of Interest Disclosures: Dr Heath reported receiving grants from Canada Research Chair in Statistical Trial Design during the conduct of the study. Dr Zarychanski reported receiving grants from Canadian Institutes of Health Research, LifeArc, Research Manitoba, CancerCare Manitoba Foundation, and Victoria General Hospital Foundation outside the submitted work. No other disclosures were reported.

AMA CME Accreditation Information

Credit Designation Statement: The American Medical Association designates this Journal-based CME activity activity for a maximum of 1.00  AMA PRA Category 1 Credit(s)™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

Successful completion of this CME activity, which includes participation in the evaluation component, enables the participant to earn up to:

1.00 Medical Knowledge MOC points in the American Board of Internal Medicine's (ABIM) Maintenance of Certification (MOC) program;;
1.00 Self-Assessment points in the American Board of Otolaryngology – Head and Neck Surgery’s (ABOHNS) Continuing Certification program;
1.00 MOC points in the American Board of Pediatrics’ (ABP) Maintenance of Certification (MOC) program;
1.00 Lifelong Learning points in the American Board of Pathology’s (ABPath) Continuing Certification program; and
1.00 credit toward the CME of the American Board of Surgery’s Continuous Certification program

It is the CME activity provider's responsibility to submit participant completion information to ACCME for the purpose of granting MOC credit.