Methodologic considerations in randomized clinical trials in reproductive medicine

Christos Venetis; Thomas d'Hooghe; Kurt T Barnhart; Patrick M M Bossuyt; Ben Willem J Mol

doi:10.1016/j.fertnstert.2020.04.038

Methodologic considerations in randomized clinical trials in reproductive medicine

Fertil Steril. 2020 Jun;113(6):1107-1112. doi: 10.1016/j.fertnstert.2020.04.038.

Authors

Christos Venetis¹, Thomas d'Hooghe², Kurt T Barnhart³, Patrick M M Bossuyt⁴, Ben Willem J Mol⁵

Affiliations

¹ Centre for Big Data Research in Health, University of New South Wales Medicine, New South Wales, Australia; School of Women's and Children's Health, University of New South Wales Medicine, New South Wales, Australia; IVF Australia, Sydney, New South Wales, Australia.
² Global Medical Affairs Fertility, Research and Development, Merck Healthcare KGaA, Darmstadt, Germany; Reproductive Medicine Research Group, Department of Development and Regeneration, Organ Systems, Group Biomedical Sciences, KU Leuven (University of Leuven), Leuven, Belgium; Department of Obstetrics, Gynecology and Reproductive Sciences, Yale School of Medicine, New Haven, Connecticut.
³ Department of Obstetrics and Gynecology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania.
⁴ Department of Clinical Epidemiology, Biostatistics and Bioinformatics, Amsterdam University Medical Centers, Amsterdam, The Netherlands.
⁵ Department of Obstetrics and Gynaecology, Monash University, Clayton, Victoria, Australia. Electronic address: ben.mol@monash.edu.

PMID: 32482246
DOI: 10.1016/j.fertnstert.2020.04.038

Abstract

Randomized controlled trials (RCTs) are the cornerstone of evidence-based medicine. In this series in Fertility and Sterility, several aspects of RCTs are discussed, with contributions on multicenter RCTs, different international settings, and integrity of RCTs. The present contribution deals with methodologic issues. We discuss different types of RCTs based on null hypothesis (superiority vs. noninferiority vs. equivalence) as well as frequentist versus Bayesian interpretation. We also discuss the use of RCTs in the era of personalized medicine and RCTs to address diagnostic and prognostic questions. Finally, we address the use of big data compared with the use of RCTs.

Keywords: Randomized clinical trial; bayesian analysis; big data; frequentist approach; methodology; personalized medicine.

Publication types

Review

MeSH terms

Big Data
Data Accuracy
Data Mining
Evidence-Based Medicine
Humans
Randomized Controlled Trials as Topic*
Reproductive Medicine*
Research Design