clarity osoba

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clarity osoba
PROMs to inform patient care:
What are we doing well?
What could we do better?
Professor Madeleine King
Cancer Australia Chair in Cancer Quality of Life
Director, Quality of Life Office
University of Sydney
Overview
•
•
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Standards and standardisation
Clinical trials
Clinical care
Improvement and innovation
Education
Standards and standardisation
•
•
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Terminology
Measurment tools
Metrics
Standards for instrument development and
PRO reporting
“Quality of life”: LACK OF STANDARD DEFINITION
"Quality of life is a vague and ethereal entity, something that
many people talk about, but which nobody clearly knows what to
do about.“
Campbell et al, 1976
“The idea has become a kind of umbrella under which are placed
many different indexes dealing with whatever the user wants to
focus on.”
Feinstein, 1987
“Concepts of health often lack clarity... The term ‘quality of life’
also has many meanings.”
Patrick & Erickson, 1993
Terminology – a source of confusion
“Health-related QOL (HRQOL) refers to the extent
to which one’s usual or expected physical,
emotional, and social well-being are affected by a
medical condition or its treatment.”
Broad umbrella: ‘Quality
of Life’
‘Health-related quality of life (HRQOL)’
Symptoms
Side-effects
Diseasespecific
HRQOL
Generic
HRQOL
QOL
Well
being
‘health-related - HRQOL’
Cella, D. (1995).
Measuring quality of life in
palliative care. Seminars in
Oncology, 22, 73-81.
‘Patient-reported outcomes PRO’
‘A measurement based on a report that comes directly from the patient about
the status of a patient’s health condition without amendment or
interpretation of the patient’s response by a clinician or anyone else.’
DEFINITIONS
“HRQOL is a multidimensional construct
encompassing perceptions of both positive and
negative aspects of dimensions, such as physical,
emotional, social, and cognitive functions, as well
as the negative aspects of somatic discomfort and
other symptoms produced by a disease or its
treatment.”
Revicki, D.A., Osoba, D., Fairclough, D., Barofsky, I., Berzon, R., Leidy, N.K.,
Rothman, M. (2000). Recommendations on health-related quality of life
research to support labeling and promotional claims in the United States.
Quality of Life Research, 9(8), 887-900.
DEFINITIONS
FDA Guidance (2009)
Emerging term: Patient-reported
Experiences (PREs)
Process of care
• Expectations, experience,
perceptions, satisfaction
with health services, care
providers, information
UK NHS: “The way people experience health
services is an important component of the
quality of care”
TOOLS – a plethora!
H E L P !!!
Too
many!
Tools – Core Outcome sets
Core Outcome Sets
• the minimum that should be measured and reported in all
clinical trials of a specific condition
• also suitable for use in clinical audit and other research
• Facilitate comparison and combination of results
• does not imply that outcomes in a particular trial should be
restricted to those in the relevant core outcome set
• COMET encourages researchers to continue to explore
other outcomes as well.
• COMET aims to collate and stimulate relevant resources,
both applied and methodological, to facilitate exchange of
ideas and information, and to foster methodological
research in this area.
E.g. OMERACT –Rheumatology
Developing a Core Domains Set
Innovation: Standardised metrics
PROMIS
www.nihpromis.org
PROs – what’s
in the toolkit?
There is no “best”
tool choose the right
tool for the job
Dynamic field –
new tools and
measurement
innovations
NIH investment in PROMIS®
Innovation:
PRO-CTCAE
• NIH-backed PROM R&D
• Patient-reported outcomes
version of the NCI’s common
toxicity criteria - standard
lexicon for adverse event
reporting, the CTCAE
• in development under a
contract from the NCI
• PI: Ethan Basch
• Web-based platform
• https://wiki.nci.nih.gov/displa
y/PROCTCAE/
– 100 million dollars (US) over 10 years
– Grants led by many of the USA’s leaders in PROM
development over past 30 years
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14
What is PROMIS®?
• Patient Reported Outcomes Measurement
Information System
• System of generic PROMS that are highly reliable,
valid, flexible, precise, and responsive
• Includes measures for wide range of domains &
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subdomains of physical, mental, and social
functioning and symptoms
• For specific domains, short-forms (fixed) and
computer-adaptive test (CAT, “dynamic”)
• Designed for electronic administration
Innovation: Computer Adaptive Testing
(CAT)
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– Short-forms can be pencil-&-paper
What makes PROMIS® better than
anything else?
• The promise of PROMIS
– Do away with the floor and ceiling effects of the legacy
instruments
– Do away with the “Tower of Babel” in PROMS – the
inability to compare and interpret across scales and
domainsto edit Master title style
Click
– facilitate comparison and combination of results from
multiple studies
– Simplify administration via computer-based
administration, scoring, and reporting
– Reduce response burden … ONLY CATS
– Improve measurement precision … ONLY CATS
How does it work?
• By forming “banks” of items, then calibrating
them
– Calibration puts all items on a common metric
– Within a domain, results are comparable for long-forms,
short-forms and CATs, regardless of which items are
Click
assessedto edit Master title style
Example: Physical Function spectrum
Scientific standards in PRO measurement
• Questionnaires as ‘instruments’ to measure
subjective phenomena
– Psychometrics
0
• Measurement properties
50
10
0
Physical Functioning
•
Are you able to run five miles?
•
Are you able to run or jog for two miles?
•
Are you able to walk a block on flat ground?
•
Are you able to walk from one room to another?
•
Are you able to stand without losing your balance for 1 minute?
•
Are you able to get in and out of bed?
Examples of short-forms that cover different
parts of the spectrum
Depression
Short Form
– Validity, reliability, sensitivity
– Interpretability
• Ongoing refinement and evolution
Explanations and benchmarks set for PROs
by FDA
C
no
depression
Depression
Short Form
Depression
Short Form
B
A
mild
depression
moderate
depression
severe
extreme
depressio depression
n
http://www.fda.gov/downloads/Drugs/
GuidanceComplianceRegulatoryInformation/Guidances/
UCM193282.pdf
Depression Item Bank
Item
1
Item
2
Item
3
Item
4
Item
5
Item
6
Item
7
Item
8
Item
9
Item
n
EORTC module development
http://groups.eortc.be/qol/manuals
Clinical trials
• Phase 1 – focus groups with patients to
identify range of issues that matter to
patients across stages and treatments
• Phase 2 – item generation
• Phase 3 – validation and psychometrics
• Phase 4 – international field testing
Whole process typically takes several
years
Evidence about candidate therapies
JAMA 2013
1.
2.
3.
4.
5.
the PRO be identified as a primary or secondary outcome in the abstract
a description of the PRO hypothesis and relevant domains (if a
multidimensional PRO tool has been used) be provided
evidence of PRO instrument validity and reliability be provided or cited
the statistical approaches for dealing with missing data be explicitly stated
PRO-specific limitations of study findings and generalizability of results to
other populations and clinical practice be discussed.
• Primary management
+/- Adjuvant Therapy
• Metastatic disease
• Survivorship
• End of life
Morbidity &
toxicity for cure
Extending survival,
symptom control,
toxicity trade-off
Why include PRO endpoints?
• Choosing therapy
–
–
–
–
Primary outcome
Support primary outcome (often a survival endpoint)
Counterbalance primary outcome
Differentiate when equivalent on primary outcome
• Enriching understanding
– Benefits or risks
– Prognosis
– Under-evaluated populations
• Improving methods
– Prognostic determinant (stratification)
– Measurement advances
Au et al., 2010
http://promotion.gimema.it
How well are we doing ?
Success Online
The PROMOTION Registry
• a registry of all cancer Randomized Controlled Trials (RCTs)
with a Patient-Reported Outcome (PRO) endpoint
published since 2004
• contains nearly 700 PRO-RCTs conducted across a wide
range of cancer specialties that have enrolled, overall, more
than a quarter of a million of patients
• GOAL: to address research questions about the use and the
added value of PROs assessment in cancer clinical trials
• Several collaborations are now ongoing with a number of
Universities and research groups dedicated to quality of life
and PRO research in oncology
• Reviews/publications so far:
ISOQOL Taskforce for Best Practice in
PROs in Clinical Trials
• Co-Chairs: Melanie Calvert (UK), Michael Brundage
(Canada) and Madeleine King (Aus)
• Developing an evidence- and consensus-based
PROtocol Checklist
• Hypothesis: more complete PRO components of
protocols results in higher quality PRO evidence, and
more complete reporting in papers
– Brain, prostate, gyne
– H&N underway with Univ Sydney QOL Office
Checklist for PROtocols
• Importance of the protocol
– Procedures for good conduct of the trial
– success or failure of a trial may depend on how well the
protocol was designed and written
– outlines procedures for good conduct of the trial
– all relevant requirements so the study can be implemented
uniformly by all sites and staff
– detailed and clearly worded
• These general points apply just as much to
PRO assessment as to any other aspect of a
Chan et al., 2013
trial
QOL Office
PROtocol
Checklist
Available on QOL
Office Website
pocog.org.au/qoloffice
Innovation
Clinical care
• Translation of clinical trial evidence into
policy and care
• Direct evidence
• Initiatives and evaluation
Magnitude of Clinical Benefit Scale
• Standardised scoring of clinical benefit in studies of new
agents/approaches in the management of cancers
• graded 5, 4, 3, 2, 1, where grades 5 and 4 represent a high
level of proven clinical benefit
• Preliminary grading based on survival outcomes - hazard
ratio for DFS/OS and median survival gain as well as late
survival advantage and is reported on a 4 point scale.
• Preliminary scores can be upgraded by 1 point when the
experimental arm demonstrates improved QoL or delayed
deterioration in QoL using a validated scale or substantial
reduction in grade 3 or 4 toxicity.
• A score of 5 can only be achieved when optimal survival
outcomes are further enhanced by data indicating reduced
toxicity or improved quality of life.
Translation of evidence from clinical
trials into policy and care
• Same challenges for PROs as for any other
endpoints
– Behaviour change
• Additional challenges
– Many questionnaires/scales
• Cochrane PRO Methods Group
– The quality of the PRO evidence and reporting
– Interpretation in terms of clinical importance /
minimally important difference (MID)
Direct evidence: effect of PRO
assessment in clinical practice
• 5 systematic reviews to date
– Valderas et al. (2008)
– Frost et al. (2007; cancer-specific)
– Marshall et al. (2006)
– Espallargues et al. (2000)
– Greenhalgh et al. (1999)
Example: PROMs in Clinical Care
Screening for psychosocial problems
Step 1: Routine assessment of patient wellbeing
Patient completes online survey
in waiting room
Acceptable strategy?
• 60% of 229 patients had
no previous contact with
computers
• 89% happy to complete at
each visit
• 96% happy for oncologist
to get summary of results
• 99% usually have time to
do while waiting for doctor
Newell et al, 1997; Boyes et al, 2002
PRO assessment in clinical practice
What the evidence says:
Improves:
– communication
– awareness
Equivocal:
– patient management
– satisfaction
– PROs / HRQoL
+
+
+/+/+/-
Does NOT increase consultation time
Routine Screening Model
Step 2: Real time feedback to
clinical team
• Summary report placed in
medical file includes:
• patient outcomes
• score interpretation
• recommended
interventions (triage)
• Adapted patient version
Routine Screening Model
Step 3: Tailored psychosocial
care
Clinical team:
• reviews the summary report
• offers patients psychosocial care
matched to type and level of need
Other in-clinic initiatives
• Lots of examples emerging
– UK NHS – QA & quality improvement
– NSW Integrated Care
– Cancer Institute NSW
• Challenges
–
–
–
–
–
Patient experiences v outcomes
Which PREMs and PROMs? When?
How to integrate with eHealth records?
How to get staff on board?
What will the data be used for?
• Benchmarking? Managing individual patients?
• If managing individual patients, are measure error v signal?
– Evaluation - collecting baseline data as comparator
Routine Screening Model
Step 4: Benchmarking
• Annual benchmarking
report comparing patient
outcomes from each
centre with all other
treatment centres
combined
• Helps to identify strengths
and weaknesses in
service delivery
Education
http://www.isoqol.org/research/onlin
e-education/archived-webinars
ISOQOL Annual Conferences
QOL Office Support / Resources
Assistance with
questionnaire selection
& protocol development
QOL Protocol Checklist
2-day w’shop each May/June
QLQ v FACT
content
comparison
spreadsheets
for each
cancer site
Statistical
analysis
position paper
FAQs
Various QOL
topics
Searchable database of 350+
Patient Reported Outcome
Measures (PROMs)
CoMiDa form
CRF to record PRO form
completion rates &
reasons for missing data.
QOL Data management & QA
guidelines in development
Contact details
• [email protected][email protected]

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