Use of core outcome sets was low in clinical trials published in major medical journals

Karen Matvienko‐Sikar ,Jane M Blazeby,Karen Hughes,Pamela Jacobsen,Valerie Smith,Katie Mellor,Susanna Dodd,Kerry Avery,Fiona Quirke,Declan Devane,Jan Kottner,Christopher P Millward,Smitaa Patel,Jamie J Kirkham,Aoife M Egan ,Ian J Saldanha ,Sarah L Gorst ,Caroline B Terwee ,Amber Young ,Paula Williamson

doi:10.1016/j.jclinepi.2021.10.012

Abstract

ObjectivesTo examine current practices in late-phase trials published in major medical journals and examine trialists’ views about core outcome set (COS) use. Study Design and SettingA sequential multi-methods study was conducted. We examined late-phase trials published between October 2019 and March 2020 in JAMA, NEJM, The Lancet, BMJ, and Annals of Internal Medicine. The COMET database was searched for COS potentially relevant to trials not reporting using a COS; overlap of trial and COS outcomes was examined. An online survey examined awareness of, and decisions to search for and use a COS. ResultsNinety-five trials were examined; 93 (98%) did not report using a COS. Relevant COS were identified for 31 trials (33%). Core outcomes were measured in 9 (23%) studies; all trials measured at least one core outcome. Thirty-one trialists (33%) completed our survey. The most common barrier to COS use was trialist's own outcome preferences and choice (68%). The most common perceived facilitator was awareness and knowledge about COS (90%). ConclusionCOS use in this cohort of trials was low, even when relevant COS were available. Increased use of COS in clinical trials can improve evaluation of intervention effects and evidence synthesis and reduce research waste.

Highlights

Appropriate choice and reporting of outcomes in clinical trials are essential for evaluating and synthesizing evidence about intervention effects and informing clinical practice [1,2]
We reviewed five major medical journals to identify late phase trials: Journal of the American Medical Association (JAMA), New England Journal of Medicine, The Lancet, BMJ, and Annals of Internal Medicine
If we identified potentially relevant core outcome set (COS) for trials, trialists were asked whether they thought the identified COS was a good fit for their trial

Summary

Introduction

Appropriate choice and reporting of outcomes in clinical trials are essential for evaluating and synthesizing evidence about intervention effects and informing clinical practice [1,2]. A core outcome set (COS) is a standardized set of outcomes, agreed upon by stakeholders, that should be the minimum outcomes measured, and reported in all trials in particular health areas [3]. COS are not necessarily the only outcomes to be measured, other outcomes can be used, they are instead the minimum that should be included [3]. Using COS in clinical trials can improve intervention evaluation and evidence syntheses [3]. COS use can minimize issues in trials, including selective outcome reporting [4], outcome heterogeneity [5,6,7], and research waste [8], while enhancing research transparency [3,9]. Integrating stakeholder views in COS development ensures inclusion of outcomes of clinical importance and relevance to stakeholders [10], including patients, which increases the likelihood that COS will be used in trials [ 8 , 10 , 11 ]

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