No choice of outcomes about us without us Patient and public involvement in core outcome set development Paula Williamson
Core Outcome Measures in Effectiveness Trials www.comet-initiative.org
DMARD trials for Rheumatoid Arthritis Study Year Tender Joints Swollen Joints Pain Patient Global Physician Global Function Acute Phase Reactant Radiological Damage ERC 1960 CCC 1973 Huskisson 1976 Woodland 1981 Palmer 1982 Ward 1983 Williams 1983 Skosey 1988 Morgan 1990 Willkins 1992 Pinheiro 1993 Rozman 1994 Farr 1995 Willkins 1995 Dougados 1999 Cohen 2001 Kalden 2001 Kremer 2002 Bao 2003 Mariette 2004 Dougados 2005 Hetland 2006 Karanikolas 2006 Capell 2007 Ogrendik 2007
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
Scope of a COS Health condition, population and types of intervention e.g. in colorectal cancer, a COS might be developed for all patients or it may focus on patients with metastatic disease e.g. in morbid obesity, a COS may be created to use in trials of all interventions or just bariatric surgery alone Research or practice setting
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.
DMARD trials for Rheumatoid Arthritis Study Year Tender Joints Swollen Joints Pain Patient Global Physician Global Function Acute Phase Reactant Radiological Damage ERC 1960 CCC 1973 Huskisson 1976 Woodland 1981 Palmer 1982 Ward 1983 Williams 1983 Skosey 1988 Morgan 1990 Willkins 1992 Pinheiro 1993 Rozman 1994 Farr 1995 Willkins 1995 Dougados 1999 Cohen 2001 Kalden 2001 Kremer 2002 Bao 2003 Mariette 2004 Dougados 2005 Hetland 2006 Karanikolas 2006 Capell 2007 Ogrendik 2007
Results improvements over time Studies reporting full RA COS (%) 100 Mean number of clinical outcomes 80 7.0 60 40 drug studies 6.5 20 non-drug studies 6.0 0 0.0 1985 1990 1995 2000 2005 2010 WHO/ILAR RA COS EMA guideline FDA guideline
Scope Identifying existing knowledge Stakeholder involvement Consensus methods Achieving global consensus Regular review, feedback, updating Trials 2012; 13: 132 Implementation Clear presentation
Impact of patient and public involvement Rheumatology (OMERACT): fatigue Chronic pain (IMMPACT): expansion of previously proposed core outcome domains Paediatric asthma: normal activities, exercise ability Multiple sclerosis: fatigue, continence
Paediatric asthma 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
COMET Initiative To raise awareness of current problems with outcomes in clinical trials To encourage COS development and uptake To promote patient involvement in COS development To provide resources to facilitate this To encourage evidence-based COS development
Website www.comet-initiative.org
PLoS ONE 2014; 9(6): e99111
Year of publication
Patients as COS participants Published COS (n=198) 16% involved either patients, carers, patient support group representatives, service users Represented 4% to 100% of all participants Ongoing COS (n=49) 90% involve either patients, carers, patient support group representatives, service users No longer whether to involve, but rather what is the nature of involvement
Patient involvement Review previous evidence - Interviews and surveys to obtain patients perspectives (48 in COMET database) - Meta-synthesis (Hoppe, COMET IV) - PROs (Macefield, Trials 2014) Interviews, focus groups, survey Delphi Consensus meeting
COMET PoPPIE Working Group People and Public Participation, Involvement and Engagement Inspired by Heather Bagley, COMET PPI Coordinator Membership COMET website presence EUPATI webinar Videos parent involved with COS project, head of patient organisation
Plain language resources www.comet-initiative.org/ resources/plainlanguagesummary
Research agenda how to? Involve patients as research partners in design of COS studies Identify and meet information needs of patients as both research partners and participants Identify appropriate consensus methods for the patient group Generate appropriate questions for patients taking part in COS study
Research agenda how to? Access and engage patients in COS studies (Facebook, newsletters) Ensure hard to reach communities are involved Bring different stakeholder groups views together Evaluate the stakeholder experience of taking part
COMET V Wednesday 20th and Thursday 21st May 2015 University of Calgary Alberta, Canada
www.comet-initiative.org Twitter: @COMETinitiative