Transcript ARC glucocorticoids in RA study

Taking patients’ and clinicians’
questions about the effects of
treatment seriously.
The James Lind Alliance
Tackling treatment uncertainties together
Rheumatoid arthritis –
What have we found and
what are we trying to do
about it?
Enid Quest
John Kirwan
University of Bristol
Academic Rheumatology Unit
Academic Rheumatology Unit
Bristol Royal Infirmary
Bristol BS2 8HW
United Kingdom
Researchers and patients
can be friends!
Enid Quest
Patient Research Partner
Academic Rheumatology Unit
Bristol Royal Infirmary, Bristol BS2 8HW, UK
Researchers and patients
can be friends!
Enid Quest
John Kirwan
Patient Research Partner
Professor of Rheumatic
Diseases
Academic Rheumatology Unit
Bristol Royal Infirmary, Bristol BS2 8HW, UK
Agenda for our presentation
• Rheumatoid arthritis (John & Enid)
• How international standardised
outcomes were devised (John)
• How patients got into the act (Enid)
• Differences between patients and
professionals (John)
• What we are trying to do about it in
Bristol (Enid & John)
What is
rheumatoid arthritis?
Symptoms of rheumatoid
arthritis
 anxiety
 depression
 helplessness
Severity of symptoms
 Pain
 Stiffness and ‘gelling’ after inactivity
 Unpredictable ‘flares’ of inflammation
Symptoms of RA
 Fatigue
 Disability
 Psychological effects
Weeks
Signs of rheumatoid arthritis
in the joints
Swelling
 effusions
 soft tissue swelling
Warmth
Joint deformity
Reduced range of movement
Bony destruction
Crepitus
2003
Swollen finger
joints in early RA
Joint damage in RA
of the hands
2004
2005
Which joints
are affected?
Who gets RA?
 Men
 Women
 Children
 Young
 Prime of
 Elderly


()

life 

So…
 Rheumatoid arthritis (RA) involves
 Inflammation inside joints
 Destruction of joints
 It can also affect other parts of the
body in some people
 About 1 person in 100 will get RA
 RA costs the country £1,200,000,000
per year
 Severe RA shortens life
Rheumatoid arthritis
my experience
• Onset
– Early symptoms
• Diagnosis
– Relief only rheumatoid arthritis
– Soon replaced with the realisation that
it was more serious
Rheumatoid arthritis
my experience
•Living with rheumatoid arthritis
– Anxiety
– Loss of independence soon
replaced the pain and stiffness
– Finding the right medication
– Coping with fatigue
What did we used to do?
The operation was a success…
... but the pain remained.
What did we used to do?
•Trials used a variety of outomes
which were considered by a particular
investigator to be important
•Different investigators
rated different outcomes as
important
•Doctors do not make
‘standard’ decisions about
treatment response
Questioning the paradigm that
doctors make ‘standard’ decisions
in rheumatoid arthritis….
….Helped in founding OMERACT
(Outcome Measures in Rheumatology
Clinical trials)
OMERACT
• Working conference of clinicians and
methodologists
• Formed 14 years ago, meets 2-yearly
• Seeks to identify outcomes, instruments
and methods of asessing benefit in
rheumatology
• Standardised a ‘core set’ of outcome
measures for rheumatoid arthritis clinical
trials
• The core set has be adopted by the
regulatory agencies in USA and Europe
OMERACT 5, May 2000
• Tried to define ‘minimum clinically
important difference’ for changes in
outcomes
• Realised this depends on who defines
‘important’!
• Resolved to include patients directly
as participants in future work on
defining the outcomes of arthritis
Patients at OMERACT 6
OMERACT 6
• The OMERACT meeting of 2002
– included 9 patients as full delegates to
the meeting and
– had a Patient Perspective Workshop as a
major part of the programme….
Patients at OMERACT 6
OMERACT 6
• During the this conference it became
evident that there was a miss-match
between the views of the researchers
and the patients
• Patients soon became involved in a
series of discussions and were able to
attend and contribute in all sessions
Patients at OMERACT 6
OMERACT 6
• One stumbling block was the use of
technical and medical terminology
• A glossary has been produced by
patients to enable them to be
confident in their participation
Patients at OMERACT 6
OMERACT 6
• The openness and encouragement of
the professionals at this first
conference has led the way for
involvement at other international
conferences
• Many patients now feel able to make
presentations and contribute on many
different topics.
OMERACT 6
OMERACT 6 – April 2002
Patient Perspective Workshop
Research Agenda
Three areas for exploration and development
• Novel outcomes and approaches to assessment
• Terminology and current knowledge
• The role of the patient
OMERACT 6
OMERACT 6 – April 2002
Patient Perspective Workshop
• An international patient panel has now
been formed
• Around 20 patients from Europe,
America, Canada and Australia.
• The panel attended the 7th
conference in California in 2004, and
are preparing for the OMERACT 8 in
Malta in 2006.
OMERACT 7 May 2004
– Patient participation increased to 20
– Funding underwritten to ensure
patient participation
– Patient input into three topic areas
– Patient orientation built into
programme
– Clinical support available if needed
OMERACT 7 May 2004
• Patients have been involved in a
number of research projects and
discussion groups on a variety of
subjects
–
–
–
–
–
Fatigue
Low disease activity state
Being an effective consumer
Drug safety workshops
The question of well-being
OMERACT 7 May 2004
• Training sessions have been held in
order to help patients develop skills
and the necessary confidence to
participate with professionals on a
level playing field.
Patient Perspective Workshop
Objectives
• Review the recent progress in the
area
• Identify requirements for further
development of valid instruments
• Delineate a research agenda which
can attain these requirements
• Enthuse participants to undertake
the appropriate research
Research Agenda
Main Topics
•
•
•
•
•
Fatigue
Well being
Real time assessment of symptoms
Incorporating patient priorities
Outcomes in early and late disease
Patients and
professionals view
things differently…
Reviews of the available evidence
made it clear that:
Patients and professionals view
the outcome of rheumatoid
arthritis differently.
Many of the consequences of
arthritis which patients find
important to them are not
measured at all, or are not
included in the ‘core set’.
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Relative Importance Score
Patients and professionals view the
outcome of rheumatoid arthritis
differently
80
70
60
50
40
30
20
10
Non-arthritic controls
Patients with rheumatoid arthritis
Health professionals
0
Health Assessment Questionnaire activities
Patients and
professionals view
things differently…
…this makes a
difference to our work!
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Relative score
Patients and professionals view the
outcome of rheumatoid arthritis
differently
Importance of outcomes reported by women
with established rheumatoid arthritis
Patricia Minnock, Barry Bresnihan (in preparation)
Patient Involvement in Bristol
• Patient Advisory Group.
– Meets every three months
– Advised on the refurbishment of the
Rheumatology Centre
– Produced a booklet for new patients, and
a yearly newsletter
– Corresponds with other agencies
regarding issues that affect patients.
Patient Involvement in Bristol
• Volunteers in clinic.
– Meeting other patients in clinic gives the
opportunity to discuss issues and offer
support and encouragement.
• Involvement in Research.
– Patients help to make grant applications,
suggest topics for research and are
often the subject of research.
Patient Involvement in Bristol
• Patient Teaching Partners
– Patients speak to groups of students to
help them understand the consequences
of being diagnosed with Rheumatoid
Arthritis and answer questions about the
affect on daily living
• International Involvement
– Several patients serve on an
international patient panel and have
made presentations at conferences
around the world
Patient Involvement in Bristol
Our involvement as patients and
our dialogue with staff has
brought about benefits for the
patients, the service and the
staff.
Patients and Professionals as Research
Partners:
Challenges, Practicalities and Benefits
Pamela Richards1, Maarten de Wit2, John Kirwan3, Enid Quest1,
Rod Hughes4, Turid Heiberg5, Sarah Hewlett3
1Bristol
University Academic Rheumatology, UK, 2Dutch Arthritis Patients League, The
Netherlands, 3University of Bristol Academic Rheumatology, UK, 4St Peter’s Hospital, Chertsey,
UK, 5Centre Education & Skill Training, Ulleval University Hospital, Norway
Background
Most researchers do not have personal experience of
arthritis
Patients’ experiences can complement researchers’
analytical skills
Effective patient involvement requires new ways of
working
Patients and Professionals as Research
Partners:
Challenges, Practicalities and Benefits
Methods:
The authors have been collaborating in research
Some are patient research partners or are on research
steering committees
Patient Research Partners have taken an active role in
OMERACT and EULAR
We reviewed our collective experience of partnership
in practice
Results - Challenges and barriers encountered:
Challenges: Enabling and empowering partner
participation
Barriers:
Terminology, training, expense
Establishing boundaries
Patient and clinician or colleagues?
Avoiding tokenism
Understanding patients’ barriers
No real contribution encouraged
Anxiety about ability to contribute
Results - Challenges and barriers encountered:
Challenges: Enabling and empowering partner
participation
Barriers:
Terminology, training, expense
Establishing boundaries
Patient and clinician or colleagues?
Avoiding tokenism
Understanding patients’ barriers
No real contribution encouraged
Anxiety about ability to contribute
Results - Practicalities:
We have developed a
philosophy of patient
partner involvement based
on FIRST principles
Results - Practicalities:
We have developed a
philosophy of patient
partner involvement based
on FIRST principles
Results - Practicalities:
We have developed a
philosophy of patient
partner involvement based
on FIRST principles
Results - Practicalities:
We have developed a
philosophy of patient
partner involvement based
on FIRST principles
Results - Practicalities:
We have developed a
philosophy of patient
partner involvement based
on FIRST principles
Results - Practicalities:
We have developed a
philosophy of patient
partner involvement based
on FIRST principles
Results - Practicalities:
We have developed a
philosophy of patient
partner involvement based
on FIRST principles
For further details see…
In Press
Conclusions
Professionals and Patient Research Partners
bring different skills, values and experiences
Partnership requires effort and commitment
Providing training, support & encouragement
empowers partners
Patients’ experience-based knowledge is a
resource to be utilised, not wasted
Conclusions
Professionals and Patient Research Partners
bring different skills, values and experiences
Partnership requires effort and commitment
Providing training, support & encouragement
empowers partners
Patients’ experience-based knowledge is a
resource to be utilised, not wasted
Conclusions
Professionals and Patient Research Partners
bring different skills, values and experiences
Partnership requires effort and commitment
Providing training, support & encouragement
empowers partners
Patients’ experience-based knowledge is a
resource to be utilised, not wasted
Conclusions
Professionals and Patient Research Partners
bring different skills, values and experiences
Partnership requires effort and commitment
Providing training, support & encouragement
empowers partners
Patients’ experience-based knowledge is a
resource to be utilised, not wasted
University of Bristol
Academic Rheumatology Unit
Spare
How do patients want us to include their
perspective in outcome assessment?
Three steps to success…
•Measure outcomes of interest
to patients
•Incorporate patients’ values into
instruments used to measure
outcome
•Incorporate patients into the
research process
OMERACT 6
OMERACT 6
Patient Perspective
Workshop
Did it work?
OMERACT 6 – April 2002
Patient Perspective Workshop
Did it make a difference?
OMERACT 6
These articulate volunteers provided
important input on missing dimensions of
RA outcome measurement.
The OMERACT patients believed the
current core sets of RA criteria do not
capture or give adequate weight to a
global sense of well being, fatigue, or
disturbed sleep.
Saag K. OMERACT 6 Brings New Perspectives to Rheumatology
Measurement Research. (Editorial) J Rheumatol 2003; 30: 639-641
How do patients want us to include
their perspective in outcome
assessment?
Three steps to success…
• Measure outcomes of interest to
patients
• Incorporate patients’ values into
instruments used to measure outcome
• Incorporate patients into the
research process
How can patients be
partners in research?
•
•
•
•
•
•
•
•
Identify relevant research questions
Identify relevant outcomes
Define clinically important changes
Patient’s perspective built in to outcomes
Design and undertake research
Interpret results
Disseminate results
Help turn research into service
developments
Patient and professional views in arthritis
 47% discordance of >0.25 HAQ: 15 patients rated
higher than Drs, 9 rated lower Van den Ende 1995
 35% discordance (AIMS vs 4 point ACR). Patients
and Drs rated higher and lower equally Berkanovic et
al, 1995
 Poor association between change as perceived by
the patient and change as calculated by repeated
questionnaires:
Correlation of r= 0.18
Fischer et al, 1995
Correlation of r= 0.28
Hewlett, 2000
Patient and professional views in arthritis
 Overall health - Drs rated higher; Pain - Drs
rated lower; Willing to take risk - Drs rated lower
Suarez-Almazor et al, 2001
 Physical health - 42% discordance; Psychological
health - 47% discordance Kwoh et al, 1992
 In osteoarthritis GPs rated lower for disability,
depression & anxiety (Kappas = 0.11 to 0.24)
Memel et al, 2000
Patient and professional views in arthritis
Some overall conclusions
 Doctors cannot easily identify important
psychological outcomes
 Professionals and patients agree only
moderately on values for disability
 Patients place value on emotions & mental
state, doctors value function & role
Patients and
professionals view
things differently…
…this makes a
difference to our work!
OMERACT
• Did not start the notion of seeing
outcomes from the patient perspective
but…
• Has been the main stimulus to
developments in this area over the last
four years