Core Outcome Measures in Effectiveness Trials www.comet-initiative.org Twitter: @COMETinitiative Email: info@comet-initiative.org

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. COMET definition One solution is this idea of a core outcome set. It’s by no means a new idea, a core outcome set is essentially 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.

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 (third bullet) Much more likely to measure appropriate outcomes because of the need to involve multiple, different stakeholders to determine what should be core.

ILAR/WHO Core Outcome Set for RA (7 outcomes) Tender joints Swollen Joints Pain Physician Global Assessment Patient Global Assessment Physical Disability Acute Phase Reactants Boers M, Tugwell P, Felson DT, et al. World health organization and international league of associations for rheumatology core endpoints for symptom modifying antirheumatic drugs in rheumatoid arthritis clinical trials. J Rheumatol 1994;21 (suppl 41):86-9.

Improvements over time (Kirkham et al, BMJ 2017) Studies reporting full RA COS (%) 100 80 drug studies 60 40 20 1985 1990 1995 2000 2005 2010 2015 WHO/ILAR RA COS BMJ 2017;357:j2262

COMET Initiative To raise awareness of current problems with outcomes in clinical trials To encourage COS development and uptake To promote patient and public involvement in COS development To provide resources to facilitate this To avoid unnecessary duplication of effort To encourage evidence-based COS development So what is the COMET Initiative all about? COMET was launched at a meeting in Liverpool in 2010, attended by individuals from many different stakeholder groups. Subsequent meetings and activity have shown that COMET is now truly an international network.

www.comet-initiative.org

Systematic review of COS >300 published COS for trials >180 ongoing COS studies

Promotion and collaboration Trialists – SPIRIT guidelines Trial funders - NIHR, ARUK, AMRC, HRB Ireland, Horizon 2020 Industry – EFPIA Regulators – EMA, FDA Systematic reviewers – Cochrane Guideline developers – NICE, CMTP, GIN Journal editors – CROWN Patients and the public – PoPPIE

Promotion and collaboration Trial funders – NIHR HTA guidance: ‘Where established Core Outcomes exist they should be included amongst the list of outcomes unless there is good reason to do otherwise. Please see The COMET Initiative website at www.comet-initiative.org to identify whether Core Outcomes have been established.'

COMET People and Patient Participation Involvement and Engagement (PoPPIE) Working Group To lead and oversee the public participation, involvement and engagement work of the COMET Initiative, as set out in the COMET Public Involvement Strategy The COMET PoPPIE Working Group was set up to lead and oversee the public participation, involvement and engagement work of the COMET Initiative, as set out in the COMET Public Involvement Strategy. On November 4th 2015 the COMET Initiative hosted its inaugural PoPPIE Working Group in Manchester. In this initial meeting the Working Group focussed discussions around the COMET Public Involvement Strategy and the challenges of patient participation in core outcome set studies. Discussions also focussed on the importance of patient and public involvement in developing and overseeing core outcome set studies and the wider activities of patient and public engagement in relation to the work of the COMET Initiative.

Acknowledgements COMET Management Group: Doug Altman, Jane Blazeby, Mike Clarke, Paula Williamson, Sean Tunis COMET project coordinator: Elizabeth Gargon International collaborators and ambassadors

Reading list Clarke, M. (2007). "Standardising outcomes for clinical trials and systematic reviews." Trials 8: 39. Williamson, P., D. Altman, et al. (2012). "Driving up the quality and relevance of research through the use of agreed core outcomes." J Health Serv Res Policy 17(1): 1-2. Gargon, E., B. Gurung, et al. (2014). "Choosing important health outcomes for comparative effectiveness research: a systematic review." PLoS ONE 9(6): e99111. Gorst, S. L., E. Gargon, et al. (2016). "Choosing Important Health Outcomes for Comparative Effectiveness Research: An Updated Review and User Survey." PLoS ONE 11(1): e0146444. Gorst, S. L., E. Gargon, et al. (2016). "Choosing Important Health Outcomes for Comparative Effectiveness Research: An Updated Review and Identification of Gaps." PLoS ONE 11(12): e0168403. Sinha, I., R. L. Smyth, et al. (2011). "Using the delphi technique to determine which outcomes to measure in clinical trials: recommendations for the future based on a systematic review of existing studies." PLoS Med 8(1): e1000393. Kirkham, J. J., M. Boers, et al. (2013). "Outcome measures in rheumatoid arthritis randomised trials over the last 50 years." Trials 14(1): 324. Young, B. and H. Bagley (2016). "Including patients in core outcome set development: issues to consider based on three workshops with around 100 international delegates." Research Involvement and Engagement 2(1): 1-13. Prinsen, C. A., S. Vohra, et al. (2016). "How to select outcome measurement instruments for outcomes included in a "Core Outcome Set" - a practical guideline." Trials 17(1): 449. Kirkham, J. J., S. Gorst, et al. (2016). "Core Outcome Set-STAndards for Reporting: The COS-STAR Statement." PLoS Med 13(10): e1002148. Williamson, P. R., D. Altman, et al. (2017). “The COMET Handbook: version 1.0“. Trials 18(Suppl 3): 280.

Twitter: @COMETinitiative www.comet-initiative.org info@comet-initiative.org Twitter: @COMETinitiative