Masterclass
Core outcome sets for research and clinical practice

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Highlights

  • A COS is a consensus-based set of outcomes for research or clinical practice.

  • Consensus should be achieved by multiple stakeholders on domains and instruments.

  • A longstanding COS for low back pain was recently updated and is ready for use.

  • COSs for chronic pain (in general) and osteoarthritis are also available.

  • Little research is done on COSs for other areas relevant to physical therapy.

Abstract

Background

This masterclass introduces the topic of core outcome sets, describing rationale and methods for developing them, and providing some examples that are relevant for clinical research and practice.

Method

A core outcome set is a minimum consensus-based set of outcomes that should be measured and reported in all clinical trials for a specific health condition and/or intervention. Issues surrounding outcome assessment, such as selective reporting and inconsistency across studies, can be addressed by the development of a core set. As suggested by key initiatives in this field (i.e. OMERACT and COMET), the development requires achieving consensus on: (1) core outcome domains and (2) core outcome measurement instruments. Different methods can be used to reach consensus, including: literature systematic reviews to inform the process, qualitative research with clinicians and patients, group discussions (e.g. nominal group technique), and structured surveys (e.g. Delphi technique). Various stakeholders should be involved in the process, with particular attention to patients.

Results and conclusions

Several COSs have been developed for musculoskeletal conditions including a longstanding one for low back pain, IMMPACT recommendations on outcomes for chronic pain, and OMERACT COSs for hip, knee and hand osteoarthritis. There is a lack of COSs for neurological, geriatric, cardio-respiratory and pediatric conditions, therefore, future research could determine the value of developing COSs for these conditions.

Section snippets

Background

The efficacy or effectiveness of health interventions is ordinarily assessed in randomized clinical trials which compare the outcome of the health intervention under study with a control group such as placebo treatment (for efficacy trials), or alternatives such as usual care or no treatment (for effectiveness trials).1 Since outcomes are supposed to reflect beneficial and adverse effects of the interventions, they need to be appropriate and assessed with validated instruments to make the

What is a core outcome set?

A COS is an agreed, standardized and minimum set of outcomes that should be measured and reported in all clinical trials for a specific health condition.13 This core set should be consensually agreed to by all the relevant stakeholders (e.g., health care professionals, researchers, policy makers, people who fund health services and research, industry representatives, patients, and the public).14 A COS does not mandate which outcome(s) should be designated as primary outcome(s) in a trial, and

How to develop a core outcome set?

The first step in the development of a COS is to establish its scope, i.e. to determine which population, interventions, study design and/or settings, the COS should apply to.13, 20 Regarding the scope, there are COSs that have been developed to apply to the effectiveness of all interventions for a quite generic health condition, such as the Initiative on Methods, Measurement, and Pain Assessment in Clinical Trials (IMMPACT) which focuses on all interventions for all chronic pain conditions.21

Core outcome sets for musculoskeletal conditions

Musculoskeletal disorders are among the most burdensome health conditions, with LBP recognized to be the highest ranked cause of disability worldwide.56 Various health care professionals are routinely involved in managing patients with musculoskeletal conditions and, for example, this is the area in which there are more publications on physical therapy interventions.57 An early COS in this field was proposed by Deyo et al.58 for LBP and included the following five health domains: ‘pain

Core outcome sets for other conditions and directions for future research

While the majority of initiatives around outcomes standardization relevant for rehabilitation is in the musculoskeletal health area, there are also initial attempts to develop COSs in other areas. For example, in the stroke rehabilitation field, while there is already a COS for acute stroke management,74 an international collaboration named as the Stroke Recovery and Rehabilitation Roundtable was recently formed with the goal of standardizing of research and, specifically, also outcomes

Conclusion

A COS can facilitate outcomes consistency across studies and consistent reporting in clinical research. Evidence synthesis on the effectiveness of various health interventions could benefit from a wide and structured implementation of COSs in clinical trials. A COS is represented by a set of recommendations regarding core outcome domains and measurement instruments to be measured and reported, thereby not mandating which outcome(s) should be designated as primary outcome in a clinical trial.

Conflicts of interest

The authors declare no conflicts of interest.

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