No choice of outcomes about us without us Patient and public involvement in core outcome set development. Paula Williamson

Transcription

1 No choice of outcomes about us without us Patient and public involvement in core outcome set development Paula Williamson

2 Core Outcome Measures in Effectiveness Trials

3 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

4 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

5 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

6 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.

7 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

8 Results improvements over time Studies reporting full RA COS (%) 100 Mean number of clinical outcomes drug studies non-drug studies WHO/ILAR RA COS EMA guideline FDA guideline

9 Scope Identifying existing knowledge Stakeholder involvement Consensus methods Achieving global consensus Regular review, feedback, updating Trials 2012; 13: 132 Implementation Clear presentation

10 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

11 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

12 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

13 Website

14 PLoS ONE 2014; 9(6): e99111

15 Year of publication

16 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

17 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

18 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

19 Plain language resources resources/plainlanguagesummary

20

21 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

22 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

23 COMET V Wednesday 20th and Thursday 21st May 2015 University of Calgary Alberta, Canada

24