Responding to the Patient’s Voice: the importance of Patient Reported Outcomes Dr. Kirstie Haywood Senior Research Fellow RCN Research Institute, School.

Responding to the Patient’s Voice: the importance of

Patient Reported Outcomes

Dr. Kirstie HaywoodSenior Research Fellow

RCN Research Institute, School of Health and Social Sciences, University of Warwick.

k.l.haywood@warwick.ac.uk

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?

Presentation structure

Responding to the Patient’s VoicePatient Important OutcomesPatient Reported Outcomes

Patient Reported Outcome Measures (PROMs)Selecting PROMsCurrent status

Future challenges Patient Involvement Patient Partnership

The Patient’s Voice

Patient InvolvementPatient Involvement

Patient-centred carePatient-centred care

Shared decision-makingShared decision-making- what information do patients need?- what information do patients need?

Evaluation - which outcomes really matter to patients?- which outcomes really matter to patients?

Patient experience and expertise

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’

Patient Important Outcomes

Mortality MorbidityCarer-

reportedPatient Reported

Outcomes

SurvivalDisease-specific

HospitalPerioperative

IllnessInjuries

HospitalisationIncapacitation

Care-giver /Family burden

FatiguePain

Emotional well-beingWork status

‘Live Longer’

‘Feel better’

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

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

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

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)

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?

Selecting PROMs II: Does the PROM work as intended?

• Measurement properties– Credibility of PROM results

– Reliability – Validity– Responsiveness– Interpretation

• Poor quality = misleading results

Selecting PROMs III: How easy is it to use the PROM?

• Practical properties

– Acceptability• respondent burden

– Feasibility

Selecting PROMs IV: What type of PROM?

• Generic– profile; utility

• Specific– condition or population– domain

• Individualised

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

PRO in Clinical Research: current status?

• Myalgic Encephalopathy/Chronic Fatigue Syndrome

• Diabetes Clinical Trials

• OMERACT

PROMs in ME/Chronic Fatigue

PRIME 1: Patient experience - 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

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

OMERACT – Rheumatoid Arthritis

• 8 core endpoints (Goldsmith et al, 1995)

Pain Patient global assessmentPhysical disabilitySwollen jointsTender jointsAcute phase reactantsPhysician global assessmentRadiograph of joints (> 1 year)

• Patient perspective (Carr et al, 2002)

FatigueGeneral well-being (HRQL)

Are we measuring the “right thing” ….?

• Patient important outcomes• Patient reported outcomes

….. in the “right way”?

• Well-developed PROMs• Patient involvement

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)

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?

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)