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Core Outcome Measures in Effectiveness Trials
www.comet-initiative.org
Acknowledgements• COMET Management Group:
Doug Altman, Jane Blazeby, Mike Clarke, Paula Williamson
• COMET project coordinator: Elizabeth Gargon
• Funding: MRC, FP7
• Collaborators: Peter Tugwell, Maarten Boers, Caroline Terwee, Holger Schunemann, Michael Rose, Sunita Vohra, Roberto D’Amico, Lorenzo Moja
Health care research is untidy
• It needs to be tidied up if it is to achieve its aim of helping practitioners and patients to improve health care and health
• This needs initiatives such as The Cochrane Collaboration for the preparation and maintenance of systematic reviews and COMET for core outcome sets
4
Systematic review of evidence on selective outcome reporting
• Studies reporting positive or significant results are more likely to be published
• Outcomes that are statistically significant are more likely to be fully reported
• 40–62% of publications had at least one primary outcome changed, newly introduced or omitted compared to protocol
[Dwan et al, PLoS ONE 2008]
5
Interviews with trialists (n=59)
• Discussion of outcomes listed in their protocol but not their trial report
• 29% trials displayed outcome reporting bias• All trialists mentioned either - outcomes not measured - outcomes measured but not analysed - Lack of clarity about importance or feasibility of
measurement for outcomes chosen[Smyth et al, BMJ 2010]
Core outcome set
• An agreed standardised set of outcomes that should be measured and reported, as a minimum, in all clinical trials in specific areas of health or health care
• Consider both benefits and harms
• The minimum (other outcomes can be collected)
• Focus is on trials of effectiveness
• “What” to measure, then “How”
Core outcome set
Scope of the COS • “The specific area of health or healthcare that the COS is to apply
to, in terms of health condition, population and types of interventions needs to be determined.”
• All stages or severity of a specific health condition or focussed on a particular disease category
- e.g. in colorectal cancer, a COS might be developed for all patients or it may focus on patients with metastatic disease
• All treatment types or for a particular intervention - e.g. in morbid obesity, a COS may be created to use in trials of all
interventions or just bariatric surgery alone
Advantages of core outcome sets
• Increases consistency across trials• Maximise potential for trial to
contribute to systematic reviews of these key outcomes
• Much more likely to measure appropriate outcomes
• Major reduction in selective reporting
9
Improvements over time (Kirkham et al, Trials 2013)
Studies reporting full RA COS (%)
WHO/ILARRA COS
EMAguideline
FDAguideline
100
80
60
40
20
1985 1990 1995 2000 2005 2010
0
Mean number of clinical outcomes
6.0
6.5
7.0
0.0
drug studies
non-drug studies
The COMET Initiative • To raise awareness of current problems with
outcomes in clinical trials• To encourage COS development and uptake• To provide resources to allow practitioners to
develop COS, e.g. COMET database
• An international network of trialists, systematic reviewers, health service users, practitioners, editors, funders, policy makers, regulators
COMET Initiative
• ‘What’ to measure
• ‘How’ to measure (validity, reliability, feasibility) - COSMIN
- PROMIS- PROQOLID- TREAT-NMD ROM
Website
Background
COMET Database• A publically accessible internet-based
resource to collate the knowledge base for core outcome set development
• To include planned and ongoing work, as well as published core outcome set development
• 200+ published, 50+ ongoing
Search results
ScopeIdentifying existing knowledgeStakeholder involvementConsensus methodsAchieving global consensusRegular review, feedback, updatingImplementation Clear presentation
Stakeholder involvement
• Health care practitioners • Patients, carers, representatives • Regulators• Industry representatives • Researchers • Stage of involvement may vary by group
Patient and public involvement
• Impact of involvement– Rheumatology (OMERACT): fatigue – Chronic pain (IMMPACT): expansion of
previously proposed core outcome domains
– Multiple sclerosis: fatigue, continence– Paediatric asthma
Comparison of studies SINHA ET AL
REDDELL ET AL BUSSE ET AL
5-11 years 12+ years
TOP 6 PARENTS +/- CLINICIANS ESSENTIAL OPTIONAL ESSENTIAL OPTIONAL ESSENTIAL OPTIONAL
Symptoms
√ √ √
Exacerbations
√ √ √ √
QoL
√ √ √ √
Death
√ √
Normal activities
√ √
Exercise ability
√
Reliever use
√ √
Lung function
√ √ √
Tx side effects
√
Healthcare utilisation
√ √ √
Biomarkers
√
Hyper-responsiveness
√
Professor Hywel Williams, Chair of the NIHR HTA Commissioning Board: ‘Patients and professionals making decisions about health care need access to reliable evidence. The new COMET database will help researchers across the NIHR family and beyond when choosing the outcomes to include in the studies that will establish this evidence base'.
• Since the COMET website and database were launched in August 2011:– 3800+ searches have been undertaken– 12000+ individuals visited – 110+ countries visiting the site
Impact
