Value-Based Clinical Trials: Selecting Recruitment Rates and Trial Lengths in Different Regulatory Contexts

• Published in: Management science Vol. 69; no. 6; pp. 3516 - 3535
• Main Authors: Alban, Andres, Chick, Stephen E., Forster, Martin
• Format: Journal Article
• Language: English
• Published: Linthicum INFORMS 01-06-2023; Institute for Operations Research and the Management Sciences
• Subjects: Asymptotic methods; Bayesian statistics; Clinical research; Clinical trials; cost-effectiveness; Health care; health economics; health technology assessment; Management science; Medical technology; Patients; Recruitment; Technology adoption; Technology assessment; value of information
• ISSN: 0025-1909; 1526-5501
• DOI: 10.1287/mnsc.2022.4540

Health systems are placing increasing emphasis on improving the design and operation of clinical trials with the aim of making the health technology adoption process more
value-based
. We present a model of a value-based, two-armed clinical trial in which both the recruitment rate and trial length are optimized. The model is value-based because it balances the cost of the trial with the expected benefit it generates for patients, valued by the relative health benefits and costs of the technologies. We consider a wide range of regulatory and practical contexts that address how patient health is valued (discount rate, time horizon, pragmatic trials). We present comparative statics and asymptotic analysis together with a retrospective application to a recent health technology assessment and an extension for adaptive trials. Results challenge traditional perceptions concerning the efficiency, length, and knowledge that may be gained from clinical research for trial managers or funders charged with delivering value efficiently: we highlight trade-offs between trial costs and population health benefits influenced by trial outcomes and the importance of optimizing both recruitment rate and trial duration rather than sample size alone.
This paper was accepted by Stefan Scholtes, health.
Funding:
Alban and Chick acknowledge the support of the European Union through the Marie Skłodowska-Curie Actions European Sepsis Academy Initial Training Network (MSCA-ESA-ITN) project [Grant 676129]. Forster acknowledges funding from the Research Infrastructure Support Fund of the Department of Economics and Related Studies, University of York.
Supplemental Material:
The online companion and data are available at
https://doi.org/10.1287/mnsc.2022.4540
.