Methodological aspects of superiority, equivalence, and non-inferiority trials
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CE - REVIEW
Methodological aspects of superiority, equivalence, and non‑inferiority trials Roumeliotis Stefanos1,2 · D.’Arrigo Graziella2 · Tripepi Giovanni2 Received: 27 April 2020 / Accepted: 15 July 2020 © Società Italiana di Medicina Interna (SIMI) 2020
Abstract Depending on the scientific hypothesis to be addressed, randomized-controlled trials (RCT) are accordingly designed. RCTs that aim to determine whether a novel, experimental therapeutic intervention (either a drug or a treatment) is superior to a placebo or control intervention, are called superiority trials. Less common are the non-inferiority RCTs, designed to assess whether a new intervention is not unacceptably worse than an already existing reference intervention. An equivalence RCT is designed to investigate whether a novel treatment is equivalently effective to another, already existing, control intervention. In equivalence and non-inferiority RCTs, the efficacy of the reference intervention (active comparator) is already established, and therefore, an untreated control group would not be ethical. In this review, using a series of examples derived from equivalence and non-inferiority/superiority RCTs, we describe the main differences and methodological aspects among these three different types of RCTs. Keywords Equivalence · Non-inferiority · Randomized-controlled trial · Superiority
Introduction Depending on the scientific question to be answered, randomized-controlled trials (RCTs) are accordingly designed. Most researchers and clinicians are familiar with RCTs designed to determine whether a novel, experimental therapeutic intervention (either a drug or a generic treatment) is superior to a placebo or control intervention, known as superiority trials. Less common are the non-inferiority trials, that seek to assess whether a new intervention is not unacceptably worse than an already existing reference intervention [1]. An equivalence RCT is designed to investigate whether a novel intervention is equivalent to another, already existing, control intervention. In equivalence and non-inferiority
* Roumeliotis Stefanos [email protected] 1
Division of Nephrology and Hypertension, 1st Department of Internal Medicine, School of Medicine, AHEPA Hospital, Aristotle University of Thessaloniki, St. Kyriakidi 1, 54636 Thessaloniki, Greece
Institute of Clinical Physiology (IFC‑CNR), Clinical Epidemiology and Physiopathology of Renal Diseases and Hypertension, Ospedali Riuniti, via Vallone Petrara, Reggio Calabria, Italy
2
RCTs, the occurrence of a specific outcome in patients of the experimental arm is compared to that observed in patients of the active comparator arm. In fact, if there is a well-established treatment for a given clinical condition, it would be unethical to interrupt that drug to test the efficacy of a new therapy versus a placebo thus exposing patients allocated to the control arm to an unacceptable risk. Thus, a face to face comparison of new and previous therapies, using the established treatment as an active
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