1. Responding to the Patient’s Voice: the importance of Patient Reported Outcomes Dr. Kirstie Haywood Senior Research Fellow RCN Research Institute, School of Health and Social Sciences, University of Warwick. k.l.haywood@warwick.ac.uk

2. Questions … Are we measuring the right thing? Are we measuring patient important outcomes in clinical trials? Are we measuring in the right way? How well do current methods of assessment capture what is important to the patient? Are patient reported outcomes, patient important outcomes?

3. Presentation structure Responding to the Patient’s Voice Patient Important Outcomes Patient Reported Outcomes Patient Reported Outcome Measures (PROMs) Selecting PROMs Current status Future challenges Patient Involvement Patient Partnership

4. The Patient’s Voice Patient Involvement Patient-centred care Shared decision-making - what information do patients need? Evaluation - which outcomes really matter to patients? Patient experience and expertise

5. Measuring health We can only be sure of a change in health if we measure …. Historically –Physiological or lab-based assessment –Clinician-based assessment Relevance to patients? –discrepancy between patients and providers Patient Important Outcomes –evidence that they will ‘live longer and feel better’

6. Patient Important Outcomes Mortality Survival Disease-specific Hospital Perioperative Morbidity Illness Injuries Hospitalisation Incapacitation Carer- reported Care-giver / Family burden Patient Reported Outcomes Fatigue Pain Emotional well-being Work status ‘Live Longer’ ‘Feel better’

7. Patient-Reported Outcomes (PRO): definition “Any report coming directly from patients, without interpretation by physicians or others, about how they function or feel in relation to a health condition or its therapy” (Patrick et al., 2007) PRO is the concept to be measured Range from symptoms through to health status and quality of life

8. PRO: measurement PRO measures (or instruments) A systematic, structured assessment of the patient’s perspective Include self completed or interviewer administered questionnaires Patient involvement should be central to development and evaluation

9. Why are PROMs important? A direct, patient-derived, assessment of health status or treatment effect –valid and reliable –unfiltered –surrogate measures often not reflect patient’s view Some treatment effects are known only to the patient Communicate the patient perspective –value of treatment to other stakeholders

10. PROMs in Clinical Trials When the primary goal of treatment is to improve how a patient feels …. … patient perspective is imperative Litmus test –If the measured end-points were the only things that changed, would the patients be willing to accept the treatment? (Guyatt et al, 2007)

11. Selecting PROMs I: What does the PROM measure? Review PROM content –Appropriateness to research question –What does it appear to measure? –What do the developers suggest it measures? –How was it developed? –Patient involvement? –Are patient important outcomes measured?

12. Selecting PROMs II: Does the PROM work as intended? Measurement properties –Credibility of PROM results –Reliability –Validity –Responsiveness –Interpretation Poor quality = misleading results

13. Selecting PROMs III: How easy is it to use the PROM? Practical properties –Acceptability respondent burden –Feasibility

14. Selecting PROMs IV: What type of PROM? Generic –profile; utility Specific –condition or population –domain Individualised

15. Selecting PROMs V: Guidance Reviews of PROMs Professional recommendations –US Drug Regulators: FDA report (Feb 2006) www.fda.gov/cder/guidance/5460dft.pdf –European Drug Regulators: EMEA report (Jan 2006) Expert consensus –OMERACT - Outcome Measures in Rheumatology www.omeract.org

16. PRO in Clinical Research: current status? Myalgic Encephalopathy/Chronic Fatigue Syndrome Diabetes Clinical Trials OMERACT

17. PROMs in ME/Chronic Fatigue PRIME 1: Patient experience - http://www.prime-cfs.org/http://www.prime-cfs.org/ –fatigue, social well-being, physical disability, general well-being PRIME 2: PRO assessed in published studies –Most often assessed: emotional well-being; fatigue; sleep dysfunction; physical function Conclusion: –PIO infrequently assessed –Poor quality PROMs; often non-reproducible –Standardisation and guidance essential Haywood et al, 2007

18. PROMs in Diabetes Clinical Trials Objective: to systematically determine the extent to which ongoing and future RCTs in DM will ascertain patient-important outcomes Conclusion: –Only 18% included PIO as primary outcomes –46% reported PIO as primary or secondary outcomes –Trials often powered to assess surrogate / clinician- driven measures –Call for OMERACT-type guidance Gandhi et al, 2008; Montori et al, 2007

19. OMERACT – Rheumatoid Arthritis 8 core endpoints (Goldsmith et al, 1995) Pain Patient global assessment Physical disability Swollen joints Tender joints Acute phase reactants Physician global assessment Radiograph of joints (> 1 year) Patient perspective (Carr et al, 2002) Fatigue General well-being (HRQL)

20. Are we measuring the “right thing” ….? Patient important outcomes Patient reported outcomes ….. in the “right way”? Well-developed PROMs Patient involvement

21. Quote from J. Tukey “It is often much worse to have a good measurement of the wrong thing – especially when, as is so often the case, the wrong thing will in fact be used as an indicator of the right thing – than to have poor measurement of the right thing.” (cited by Donald Patrick, 2007)

22. Key challenges … Standardisation, consensus and guidance –Core outcomes –Appropriate PROMs How do we ensure appropriate ‘active’ patient involvement? –Consultation –Collaboration –User-led Do we move towards a model of partnership?

23. Outcomes in Clinical Research – whose responsibility? Joint responsibility If we fail … ‘we will have left the heart out of all health-care research: the patient’ (Sloan et al, 2007)