Transcript Responding to the Patient’s Voice: the importance of

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.
[email protected]
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 Voice
Patient Important Outcomes
Patient Reported Outcomes
Patient Reported Outcome Measures (PROMs)
Selecting PROMs
Current status
Future challenges
Patient Involvement
Patient Partnership
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
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
Morbidity
Survival
Disease-specific
Hospital
Perioperative
Illness
Injuries
Hospitalisation
Incapacitation
‘Live Longer’
Carerreported
Care-giver /
Family burden
Patient Reported
Outcomes
Fatigue
Pain
Emotional well-being
Work status
‘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 / cliniciandriven 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 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)
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)