Agreeing what outcomes to measure Importance of core outcome sets - - PowerPoint PPT Presentation

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 credible 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
• r 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

• utcome 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

• utcomes

Limits the usefulness of research

• Use of different outcomes is like comparing

apples and bananas

• Impossible to combine study findings = waste
• f 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

• utcome 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 40 20 WHO/ILAR RA COS 1985 1990 1995 2000 2005 2010 drug studies 2015

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

• utcomes 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 every day regarding their child. It’s important that they’re included… so that the outcomes are relevant to their child’s condition” (Parent

• f child with rare disease)

“Only the patient can say which of the symptoms have the greatest impact on their lives”. (Patient 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

Rapid Review

• utcomes

reported in previous PKU and MCAD studies Delphi survey 1 Families, healthcare providers and decision- makers Delphi survey 2/3 Families, healthcare providers and decision- makers Consensus meeting Core

• utcome

set

Rapid Review

• utcomes

reported in previous PKU and MCAD studies Delphi survey 1 Families, healthcare providers and decision- makers Delphi survey 2/3 Families, healthcare providers and decision- makers Consensus meeting Core

• utcome

set Family Advisory Forum to ensure project incorporates their perspectives

Rapid Review

• utcomes

reported in previous PKU and MCAD studies Delphi survey 1 Families, healthcare providers and decision- makers Delphi survey 2/3 Families, healthcare providers and decision- makers Consensus meeting Core

• utcome

set 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

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

Participation – contribute data to COS studies Involvement – input to design and running of COS studies

Identified “real, wide-ranging problems that we currently do not assess” “Clear guidance for what was acceptable and relevant“ “Enabled the patient voice to be heard throughout the COS development process” “Part of the writing team”

Both patient participation and involvement helped to keep “the values of patients high on the agenda”

“ Patient involvement was an integral and transformative step “

What input are patients having in COS?

Participation Involvement

63/264 (24%) COS published up to end of 2014 had patient participants 171/189 (91%) ongoing COS plan to include patient participants Rates of patient involvement not known Participation and involvement often blurred

(Gargon JCE 86 2017, 140-152)

What can patients and families do?

Search the COMET database for COS studies: Encourage use of COS (if available) Encourage development

• f COS if none available

www.comet-initiative.org

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
• utcomes 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.