Prioritisation and design of clinical trials

Anna Heath, M.G.M. (Myriam) Hunink*, Eline Krijkamp, P. (Petros) Pechlivanoglou

*Corresponding author for this work

Research output: Contribution to journalArticleAcademicpeer-review

8 Citations (Scopus)
39 Downloads (Pure)


Clinical trials require participation of numerous patients, enormous research resources and substantial public funding. Time-consuming trials lead to delayed implementation of beneficial interventions and to reduced benefit to patients. This manuscript discusses two methods for the allocation of research resources and reviews a framework for prioritisation and design of clinical trials. The traditional error-driven approach of clinical trial design controls for type I and II errors. However, controlling for those statistical errors has limited relevance to policy makers. Therefore, this error-driven approach can be inefficient, waste research resources and lead to research with limited impact on daily practice. The novel value-driven approach assesses the currently available evidence and focuses on designing clinical trials that directly inform policy and treatment decisions. Estimating the net value of collecting further information, prior to undertaking a trial, informs a decision maker whether a clinical or health policy decision can be made with current information or if collection of extra evidence is justified. Additionally, estimating the net value of new information guides study design, data collection choices, and sample size estimation. The value-driven approach ensures the efficient use of research resources, reduces unnecessary burden to trial participants, and accelerates implementation of beneficial healthcare interventions.

Original languageEnglish
Pages (from-to)1111-1121
Number of pages11
JournalEuropean Journal of Epidemiology
Issue number11
Publication statusPublished - Nov 2021

Bibliographical note

Funding Information:
Dr Hunink receives Royalties from Cambridge University Press for a textbook on Medical Decision Making, reimbursement of expenses from the European Society of Radiology (ESR) for work on the ESR guidelines for imaging referrals, reimbursement of expenses from the European Institute for Biomedical Imaging Research (EIBIR) for membership of the Scientific Advisory Board, and research funding from the American Diabetes Association, the Netherlands Organization for Health Research and Development, and the German Innovation Fund. Anna Heath was funded through an Innovative Clinical Trials Multi-year Grant from the Canadian Institutes of Health Research (funding reference number MYG-151207; 2017—2020). Eline Krijkamp is supported by the Society for Medical Decision Making (SMDM) fellowship through a grant by the Gordon and Betty Moore Foundation (GBMF7853).

Publisher Copyright:
© 2021, The Author(s).


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