Agreeing what outcomes to measure Importance of core outcome sets (COS) and the crucial roles that patients have in the development and use of COS Bridget Young, Professor of Psychology, University of Liverpool, UK
“Clinical trials are only as c redible as their outcomes” Tugwell P, Boers M. OMERACT conference on outcome measures in rheumatoid arthritis clinical trials: introduction. J Rheumatol 1993; 20 :528 – 30
What are health outcomes? • Changes in health that result from treatments or interventions • Indicators of how well a treatment works • Many different health outcomes … – even within one condition there may be 100s
Objectives 1) the importance of early interaction between patients and researchers regarding what outcomes to measure 2) how to make sure patient input to developing core outcome sets (COS) is meaningful and that the COS produced are fit for purpose. 3) how we need the input of patients, not only in determining what outcomes to include in a COS, but to also ensure that COS are used once they have been developed
Health research outcomes: need a tidy
24 trials of treatments for rheumatoid arthritis Lack of common outcomes
Limits the usefulness of research • Use of different outcomes is like comparing apples and bananas • Impossible to combine study findings = waste of research resources
Rare diseases • Problem even greater in rare diseases - few patients so inconsistency in outcomes has a major impact • On positive note, rare disease patient registries can help in harmonizing outcomes
Core outcome sets as a solution Agreed list of outcomes that should be used in all clinical trials for a specific condition or area of health Core outcomes sets (COS) = minimum; freedom to measure additional outcomes Focus on what to measure ( how to measure each outcome is the next step)
Core outcome set for rheumatoid arthritis Tender joints Swollen Joints Pain Physician Global Assessment Patient Global Assessment Physical Disability Acute Phase Reactants Boers M, 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.
Advantage 1 Increases consistency - all trials using COS contribute to evidence base E.g. data can be pooled in systematic reviews But are COS used once developed?
Improvements over time (Kirkham et al, BMJ 2017 ) Studies reporting full rheumatoid arthritis COS (%) 100 80 60 drug studies 40 20 0 1985 1990 1995 2000 2005 2010 2015 WHO/ILAR RA COS BMJ 2017;357:j2262
Advantage 2 - prevents selective reporting of outcomes
Advantage 3 – Outcomes of relevance to patients/families Traditionally clinicians and researchers have decided what outcomes to measure in trials But it is patients/families whose lives are directly affected - their perspective is crucial to know which outcomes really matter
Outcomes of relevance “Parents… make vital decisions “Only the patient every day regarding their child. It’s can say which of the important that they’re included … symptoms have the so that the outcomes are relevant greatest impact on to their child’s condition” (Parent their lives” . (Patient of child with rare disease) with rare disease) ‘You have to start with an understanding of the impact of the disease on the people who have it, and what they value most in terms of alleviation before you set up a measurement and go forward with truly patient focused drug development’ U.S. Food and Drug Administration (FDA)
24 trials of treatments for rheumatoid arthritis None looked at whether the treatments improved patient quality of life
Whose input is needed to agree COS? COS a vehicle for patient input – Biggane et al
Consensus process
Delphi Delphi Rapid survey 1 survey 2/3 Review outcomes Core Consensus Families, Families, reported in outcome healthcare healthcare previous meeting set providers providers PKU and and and MCAD decision- decision- studies makers makers
Delphi Delphi Rapid survey 1 survey 2/3 Review outcomes Core Consensus Families, Families, reported in outcome healthcare healthcare previous meeting set providers providers PKU and and and MCAD decision- decision- studies makers makers Family Advisory Forum to ensure project incorporates their perspectives
Delphi Delphi Rapid survey 1 survey 2/3 Review outcomes Core Consensus Families, Families, reported in outcome healthcare healthcare previous meeting set providers providers PKU and and and MCAD decision- decision- studies makers makers Family Advisory Forum to ensure project incorporates their perspectives COS as a “ crucial step … to launch RCTs with meaningful outcomes”
Core Outcome Measures in Effectiveness Trials www.comet-initiative.org Twitter: @COMETinitiative Email: info@comet-initiative.org Database + Awareness raising + Advice, resources + Meetings, training + Development of methods
Patient participation versus involvement Participation – contribute data Involvement – input to design to COS studies and running of COS studies Identified “real, wide -ranging problems “Clear guidance for what was that we currently do not assess” acceptable and relevant“ “Enabled the patient voice to be heard “ Part of the writing team ” throughout the COS development process” Both patient participation and involvement helped to keep “the values of patients high on the agenda” Haywood et al Public Involvement in Developing a Core Outcome Set for Cardiac Arrest Clinical Trials: COSCA initiative - http://www.oxfordahsn.org/wp-content/uploads/2017/01/K-Haywood-Outcomes-that-matter-slides.pdf
“ Patient involvement was an integral and transformative step “
What input are patients having in COS? Participation Involvement 63/264 (24%) COS published up to Rates of patient end of 2014 had patient involvement not known participants Participation and 171/189 (91%) ongoing COS plan to involvement often blurred include patient participants (Gargon JCE 86 2017, 140-152)
What can patients and families do? Search the COMET database for COS studies: www.comet-initiative.org Encourage use of COS (if available) Encourage development of COS if none available
What can patients/families do? Participate in COS studies Get involved in design and running of COS studies
COMET People and Patient Participation Involvement and Engagement (PoPPIE) Working Group Supporting patient participation and involvement in COS
Resources on web-site • COMET and core outcome sets (plain language summary) • Delphi study (plain language summary) • Webinar about COMET and COS (hosted by EURORDIS) • Video presentation from COMET IV- Patient and public involvement in a COS (MoMENT study).
Further reading (for the very interested) • 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. • 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.
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