SlideShare a Scribd company logo
What Are Patient Preferences,
How Do You Measure Patient
Preferences, and
How Do I Use Them?
Deborah A Marshall, PhD
Professor and Arthur J.E. Child Chair in
Rheumatology Research
Cumming School of Medicine
OARSI, Toronto, May 3, 2019
2
At the end of this session participants will be able to:
1. Differentiate between patient reported outcome and
experience measures (PROMs and PREMs) and patient
preferences
2. Identify and describe good research practices to develop,
design, and execute a patient preferences survey
3. Describe how patient preferences can be measured and
how to interpret the results from patient preferences
studies
4. Identify ways in which patient preferences can be applied in
clinical practice for patients with osteoarthritis
Learning Objectives
What are Patient Reported Outcome Measures
(PROMs) and Patient Reported Experience
Measures (PREMs)?
PROMs PREMs
Umbrella term that includes
outcome data reported directly by
the patient
Encompasses range of interactions that
patients have with health care system - care
from doctors, nurses, and staff in hospitals,
health care facilities
Includes global impressions,
functional status, well-being,
symptoms, health-related quality of
life, satisfaction
ACR core set of 3 PROMs: physical
function, pain, global assessment
of disease activity
Communication with doctors and nurses; Pain
management; Timeliness of assistance;
Explanation of medications administered;
Different from patient satisfaction (which
relates to meeting expectations)
Example:
Health Assessment Questionnaire
(HAQ)
Example: Hospital Consumer Assessment of
Healthcare Providers and Systems Survey
(HCAHPS)
- Van Tuyl et al. Rheum Dis Clin N Am 2016; Felson et al. Arth Rheum 1993; Felson et al. Arth Rheum 1995 ; Manery et al. N Engl J Med 2013
Beyond PROMS and PREMS –
Preferences Consider Choices and Trade-Offs
• Experimental survey methods that ask respondents
to express the relative desirability or acceptability of
features that differ amongst alternatives …which
reflects their underlying utility for that alternative
Attributes Treatment A Treatment B
Functional Improvement 20% improvement 40% improvement
Side Effects Mild Moderate
Mode of Administration Oral Injection
Which Treatment would you
choose? □ □
- Medical Device Innovation Consortium Framework for Patient-Centered Benefit-Risk Assessment, 2015
Arthritis is a ‘preference sensitive’ condition
Treatments are preference sensitive with a key issue of
compliance and adherence to therapy.
5
Patient Preference Methods
Medical Device Innovation Consortium Catalogue of Methods
Source: MDIC PCBR Framework Report Release Event, May 13, 2015.
Available at: http://mdic.org/pcbr-framework-report-release/
Group Method
Structured-
weighting
• Simple direct weighting
• Ranking exercises
• Swing weighting
• Point allocation
• Analytic hierarchy process
• Outranking methods
Health-state
utility
• Time tradeoff
• Standard gamble
Stated-
preference
• Direct-assessment questions
• Threshold technique
• Discrete-choice experiments
• Best-worst scaling exercises
Revealed-
preference
• Patient-preference trials
• Direct questions in clinical trials
Preference Methods
• For uni-dimensional decisions
(i.e., consider one attribute or outcome at a time)
– Standard Gamble
– Time tradeoff
– Contingent valuation
• For multi-dimensional decisions
(i.e., consider multiple attributes or outcomes
simultaneously)
– ConjointAnalysis
– Best-Worst Scaling (BWS)
– Discrete-Choice Experiments (DCE)*
– Analytic Hierarchy Process (AHP)
7
– Respondents choose amongst a set of alternatives
– Each alternative is a profile defined by attributes
– (e.g., efficacy, tolerability, mode of administration, cost, etc…)
– Each attribute can take on different levels
– For example, if efficacy attribute is defined as a response rate, then
levels could be
– 60 out of 100 (60%)
– 75 out of 100 (75%)
– 85 out of 100 (85%)
Direct Preference Elicitation with Discrete-
Choice Experiment (DCE)
– Profiles are combined into sets in each choice task
– Alternative choice formats can include
• two or more active alternatives (forced choice),
• opt-out or status quo (neither or none)
• Each respondents completes a series of choice tasks
• Each choice task has a different set of profiles determined by an
experimental design
• The key to a DCE is that one alternative is chosen in each
choice task
Direct Preference Elicitation with Discrete-
Choice Experiment (DCE)
Anatomy of a DCE Choice Task
Attributes
Prefer 1 Prefer 2
X Prefer 1 Prefer 2
X
Outcome Medicine 1 Medicine 2
Response Rate
90 out of 100
(90%)
50 out of 100
(50%)
Adverse Event Rate
10 out of 100
(10%)
1 out of 100
(1%)
Which medicine would you
prefer?
Prefer 1 Prefer 2
Attributes
Benefit
Risk
Anatomy of a DCE Choice Task
Attribute Levels
Prefer 1 Prefer 2
X Prefer 1 Prefer 2
X
Outcome Medicine 1 Medicine 2
Response Rate
90 out of 100
(90%)
50 out of 100
(50%)
Adverse Event Rate
10 out of 100
(10%)
1 out of 100
(1%)
Which medicine would you
prefer?
Prefer 1 Prefer 2
Attribute
Levels
Anatomy of a DCE Choice Task
Profile
Prefer 1 Prefer 2
X Prefer 1 Prefer 2
X
Outcome Medicine 1 Medicine 2
Response Rate
90 out of 100
(90%)
50 out of 100
(50%)
Adverse Event Rate
10 out of 100
(10%)
1 out of 100
(1%)
Which medicine would you
prefer?
Prefer 1 Prefer 2
Profile
Attributes
Benefit
Risk
Anatomy of a DCE Choice Task
Choice
Prefer 1 Prefer 2
X Prefer 1 Prefer 2
X
Outcome Medicine 1 Medicine 2
Response Rate
90 out of 100
(90%)
50 out of 100
(50%)
Adverse Event Rate
10 out of 100
(10%)
1 out of 100
(1%)
Which medicine would you
prefer?
Prefer 1 Prefer 2
X
Choice
Anatomy of a DCE Choice Task
Series of Choice Tasks
Outcome Medicine 1 Medicine 2
Response Rate
90 out of 100
(90%)
50 out of 100
(50%)
Adverse Event Rate
10 out of 100
(10%)
1 out of 100
(1%)
Which medicine would you
prefer?
Prefer 1 Prefer 2
X
Outcome Medicine 1 Medicine 2
Response Rate
90 out of 100
(90%)
50 out of 100
(50%)
Adverse Event Rate
10 out of 100
(10%)
1 out of 100
(1%)
Which medicine would you
prefer?
Prefer 1 Prefer 2
X
Outcome Medicine 1 Medicine 2
Response Rate
90 out of 100
(90%)
50 out of 100
(50%)
Adverse Event Rate
10 out of 100
(10%)
1 out of 100
(1%)
Which medicine would you
prefer?
Prefer 1 Prefer 2
X
DCE Choice Task
Give it a Try
Car A Car B
Color Grey Red
Type 4 door sedan 2 door sport
Safety rating 5 stars 3 stars
Your choice
15
DCE Choice Task
Now what?
Car A Car B
Color Grey Red
Type 4 door sedan 2 door sport
Safety rating 5 stars 3 stars
Price $20,000 $35,000
Your choice
16
Choices Reveal Information about
Preferences
Car A Car B
Color Grey Red
Type 4 door sedan 2 door sport
Safety rating 5 stars 3 stars
Price $20,000 $35,000
Your choice X
17
 Something is only of value if we are willing to
give something up for it
Utility Estimates
Assumes the
utility associated
with an alternative
or profile is a
function of
observed
characteristics
(attributes levels)
and unobserved
characteristics of
the alternative
18
The utility of each medicine is
the sum of the effect of each level
Utility(Medicine 1) > Utility(Medicine 2)
Prefer 1
X
Prefer 1 Prefer 2
X Prefer 1 Prefer 2
X
Outcome Medicine 1 Medicine 2
Response Rate
90 out of 100
(90%)
50 out of 100
(50%)
Adverse Event Rate
10 out of 100
(10%)
1 out of 100
(1%)
Which medicine would you
prefer?
Prefer 1 Prefer 2
X
Using DCEs to Measure Preferences
 DCE is a robust quantitative method
grounded in economic theory to measure the
value of alternative choices and risk-benefit
trade-offs for specific attributes
 Measure how people value components
(attributes) of a product or service
 Eg. For a drug - cost, side effects, delivery mode
 Including non-health outcomes
19
20
At the end of this session participants will be able to:
1. Differentiate between patient reported outcome and experience
measures (PROMs and PREMs) and patient preferences
2. Identify and describe good research practices to develop,
design, and execute a patient preferences survey
3. Describe how patient preferences can be measured and how to
interpret the results from patient preferences studies
4. Identify ways in which patient preferences can be applied in
clinical practice for patients with osteoarthritis
Learning Objectives
ISPOR Task Forces on Good Research
Practices (GRPs)
https://www.ispor.org/workpaper/ConjointAnalysisGRP.asp
22
ISPOR Task Forces on Good Research
Practices (GRPs)
https://www.ispor.org/workpaper/ConjointAnalysisGRP.asp
“Aligning health care policy with patient preferences
could improve the effectiveness of health care
interventions by improving adoption of, satisfaction
with, and adherence to clinical treatments.”
10-point Checklist for Good Research
Practice in CA in Health
The checklist
generally follows
the steps required
to conduct a
conjoint analysis
study
Attributes
and levels
Define Objective
Design and
implement
survey
instrument
Design
experiment
Analyze data
Report Results
However, some of
these categories
require multiple
steps
• Was a well-defined research question stated and
is conjoint analysis an appropriate method for
answering it?
– Was a well-defined research question and/or testable hypothesis
articulated?
– Was the study perspective described and the study placed in any
particular decision-making or policy context described?
– What is the justification for using conjoint analysis to answer the
research question?
Task #1: Defining the Research Question
 Ranking the importance of attributes
 Examining tradeoffs
 Estimating willingness to pay
 Exploring variation in preferences
– Within a population
– Between two groups of stakeholders
 Evaluating potential market share
Possible Research Questions
• Were the attributes and attribute levels
supported by evidence?
– Were all important and relevant attributes identified (that is, supported
by literature reviews, focus groups, or other scientific method)?
– Was the choice of included attributes justified and consistent with
theory?
– Were the range and number of levels for each included attribute
justified?
Task #2:
Determining Attributes and Levels
Qualitative Work as a Foundation to
Stated Preferences…
- Louviere, Hensher and Swait, 2008
- Lanscar and Louviere, 2008
“We cannot overemphasise how important it is to conduct this kind of
qualitative, exploratory work to guide subsequent phases of the
stated preference study.
“It is highly recommended that qualitative work is conducted during
attribute development…the study team should endeavour to
understand the dimensions…along which the product is evaluated by
consumers and how specific levels of these dimensions are
expressed.”
“There is scope to move beyond simplistic and ad hoc uses of
qualitative tools before, alongside and after quantitative data
collection.”
Attributes: Consider all potential attributes, but in context of
plausibility
 What attributes are important to people
 Number of attributes relevant to research question
 Omitted attributes adversely affect study quality
 Understand how people discuss attributes
 What words or phrases do they use?
 Understand any interactions between the attributes
 Are attributes considered together?
 Does the preference for one attribute depend on the level of another
attribute (e.g. route of drug administration and drug regimen)
Levels: Encompass salient range of values, even if
hypothetical or not currently available
Qualitative Methods for Attribute Development
• Was the construction of the conjoint tasks
appropriate?
– Was the number of attributes in each conjoint task
justified?
– Was the number of scenarios in each conjoint task
justified?
– Was the number of conjoint tasks included in the data
collection instrument appropriate?
Task 3 Checklist : Construction of Tasks
Survey Development and Administration
30
Qualitative
Research
Pretest
Pilot Test
Data Collection
Conceptual Framework
Identify Key Attributes
Range of Levels
Cognitive Feasibility
Administrative Feasibility
Administration
How many Attributes and Levels?
31
 # attributes ranged from 3 to 16
 70% with 3 to 7 attributes
 40-50% included cost as an attribute
 # levels ranged from 2 to >6
- Marshall DA et al. The Patient 2010
- deBekker-Grob et al, Health Econ 2010
Number of Valuation Tasks Appropriate?
~ 10-20 valuation tasks in a choice set
Aim to:
– Avoid respondent fatigue
– Maximise information per respondent
– Minimise fractional design
32- Marshall DA et al. The Patient 2010
- deBekker-Grob et al, Health Econ 2010
• Was the choice of experimental design
justified and evaluated?
• Was the choice of experimental design justified?
• Were alternative experimental designs considered?
• Were the properties of the experimental design
evaluated?
• Was the number of conjoint tasks included in the
date-collection instrument appropriate?
Task #4: Experimental Design
Principle of an experiment (the researcher
controls the stimuli):
– To vary one or more attributes with two or
more levels and elicit a behavioural
response
– DCEs systematically vary attributes and
levels to investigate the determinants of
choice in a particular context.
Experimental Design
Full Factorial Design (all possible alternatives)
• Product of # Levels for each attribute (grow quickly!)
• 2 attributes with 2 levels =
• 3 attributes with 3 levels =
Factorial Design
Full Factorial Design (all possible alternatives)
• Product of # Levels for each attribute (grow quickly!)
• Estimate main effects and all interactions
Fractional Factorial Design (subset of all possible alternatives)
– Select subset randomly (potentially biased unless very large sample size) or
systematically (experimental design) to estimate effects
– May not be able to estimate all interactions
Factorial Design
Criteria to Consider Desired Criteria
Correlations among attributes Attributes are independent
Level balance Completely balanced
Number of overlapping attributes Minimal overlap
Efficiency score Higher is better, but relative
Restrictions on implausible
combinations
No implausible
combinations
Cognitive difficulty Low cognitive burden
Complexity to generate design Simple to implement
Considerations in Experimental Design
What is the Right Sample Size?
Effect on Estimate Precision
- Johnson FRJ et al. Value in Health, 2013; Yang JC al J Choice Modelling,2015
...precision
varies with the
inverse of the
square root of
sample size.
39
Task 6 Checklist: Instrument Design
• Was the data collection instrument designed
appropriately?
• Was appropriate respondent information collected
(such as sociodemographic, attitudinal, health
history or status, and treatment experience)?
• Were the attributes and levels defined, and was any
contextual information provided?
• Was the level of burden of the data-collection
instrument appropriate? Were respondents
encouraged and motivated?
An Example of a Survey Outline
Including a DCE
•Confirming inclusion and exclusion criteriaScreening
•Signed informed consent for face-to-face interviews
•Online informed consent (“I agree to participate”) for online
surveys
Informed consent
•Experience with disease
•Experience with disease treatment and managementBackground questions
•Descriptions of each attribute included in the conjoint tasks
•Warm-up questionsInformation treatment
•8-16 DCE Choice Task questions
•# tasks depends on the number of attributes and levels
•Determined by experimental design
Choice Task questions
•Age, gender, martial status, education, etc.Demographic questions
40
41
Task #7 Checklist: Data Collection
• Was the data-collection plan appropriate?
• Was the sampling strategy justified (for
example, sample size, stratification, and
recruitment)?
• Was the mode of administration justified and
appropriate (for example, face-to-face, pen-
and-paper, web-based)?
• Were ethical consideration addressed (for
example, recruitment, information and/or
consent, compensation)?
Mode of Administration
 Pen and paper survey by mail
 Pen and paper survey in-person
 Telephone assisted survey by interviewer
 Computer-based survey in person
 Television-based survey at home
 Internet survey
42
• Were statistical analyses and model
estimations appropriate?
• Were respondent characteristics examined and
tested?
• Was the quality of the responses examined (e.g.
Rationality, validity, reliability)
• Was model estimation conducted appropriately?
Task #8 Checklist: Statistical Analysis
 Data setup
 Coding attribute levels – dummy or effects coding
 Setting up the data for each choice question
 Setting up the data for each respondent
 Estimation
 Conditional logit (the foundation)
 Extensions of conditional logit
 Calculations
 Marginal rates of substitution (tradeoffs)
 Scenario Analysis 44
Analysis Steps
The Utility Function
Ui = V(β,Xi ) + εi
where
• V is the value (utility) function
• X is a vector of attribute levels
• β is a vector or parameters (preference weights)
• ε is a random error term
Conditional Logistic Regression Analysis
• Main effects
• Assumes relative preferences for each attribute level are
independent of the level of any other attribute in the
profile
• β1*attribute1 + β2*attribute2 + β3*attribute3 + ε
• Interaction effects
• Models preferences for an attribute level as dependent
on the levels of other attributes in the profile
U = …β12*attribute1*attribute2 + β13*attribute1*attribute3 + …
46
Marginal Rates of Substitution to Estimate
Risk Benefit Trade-Offs
 Indirect utility (value) function:
V = α + β1X1 + β2X2 + β3X3
 Marginal rates of substitution:
• - (βk / βj)
•(if X1 and X2 are continuous and linear)
47
10-point Checklist for Good Research
Practice in CA in Health
The checklist
generally follows
the steps required
to conduct a
conjoint analysis
study
Attributes
and levels
Define Objective
Design and
implement
survey
instrument
Design
experiment
Analyze data
Report Results
However, some of
these categories
require multiple
steps
49
At the end of this session participants will be able to:
1. Differentiate between patient reported outcome and
experience measures (PROMs and PREMs) and patient
preferences
2. Identify and describe good research practices to develop,
design, and execute a patient preferences survey
3. Describe how patient preferences can be measured
and how to interpret the results from patient
preferences studies
4. Identify ways in which patient preferences can be applied in
clinical practice for patients with osteoarthritis
Learning Objectives
Osteoarthritis Treatment
Benefits and Risks
Category Attributes Levels
Benefits
Ambulatory pain*
None (0mm)
Mild (25mm)
Moderate (50mm)
Severe (75mm)
Resting pain*
Stiffness*
Difficulty doing daily activities*
Risks
Ulcer risk**
None
10 out of 1,000 (1%)
50 out of 1,000 (5%)
100 out of 1,000 (10%)
Stroke risk***
None
5 out of 1,000 (0.5%)
15 out of 1,000 (1.5%)
30 out of 1,000 (3%)
50
Hauber et al., Osteoarthritis Cartilage, 2013
* After treatment, measured on a 100mm visual analog scale
** Incremental treated-related risk in the next year
*** Incremental treatment-related risk in the next 5 years
Benefits - Most important reductions:
- ambulatory pain and difficulty doing daily activities (both: 6.32)
- resting pain (2.80)
- stiffness (2.65)
Risks – Most Important for incremental changes (3%):
- Risk of MI (10.00)
- Stroke (8.90)
Surgeon Referral and Wait Times for
Total Joint Replacement
- Marshall DA, Deal K, Conner-Spady B, Bohm E, Hawker G, Loucks, L MacDonald KV,
Noseworthy T. How do Patients Trade-Off Surgeon Choice and Waiting Times for Total
Joint Replacement: A Discrete Choice Experiment. Osteoarthrits and Cartilege
2018;26:522-530.
- Damani Z, Spady C, Nash T, Stelfox T, Noseworthy T, Marshall DA. What is the
influence of single-entry models on access to elective surgical procedures?: A
systematic review. BMJ Open Feb 2017;7(2):e012225.
- Connor-Spady BL, Marshall DA, Hawker GA, Bohm E, Dunbar MJ, Frank C,
Noseworthy T. You’ll know when you’re ready. How do patients decide when the time
is right for joint replacement surgery? BMC Health Services 2014;14:454
- Connor-Spady BL, Marshall DA, Bohm E, Dunbar MJ, Loucks L, Hennigar A, Frank C,
Noseworthy T. Patient factors in referral choice for total joint replacement surgery.
Medical Care 2014;52(4):300-306
Arthroplasty is a
Preference-Sensitive Procedure
Next Available Surgeon: Simplified
Example
53
Attributes Levels
Surgeon
reputation
• Excellent
• Good
• Satisfactory
• Don’t know
Surgeon referral
• Selected by you
• Next available surgeon
• Selected by your doctor
Time to surgeon
consultation
• 1 month
• 6 months
• 12 months
• 18 months
Assume all other attributes of surgery (time to surgery and time
to hospital) are the same between options.
*Please note: this example does not use data from the published results (referenced in
previous slides) given the complexity of the full DCE. This is being used as a simplified
example and therefore results do not represent results reported from our published study.
Next Available Surgeon:
Simplified Example Choice Task
54
If you were told at the time of referral to a surgeon that these
were the only scenarios available, which one would you choose?
Scenario A Scenario B
Surgeon reputation Excellent Satisfactory
Surgeon referral Selected by you Selected by your doctor
Time to surgeon
consultation
18 months 6 months
I would choose X
Next Available Surgeon:
Simplified Results
55
Variable Coefficient P value
Surgeon reputation: excellent
(reference = don’t know)
2.0 <0.001
Surgeon referral: surgeon selected by
you
(reference = selected by your doctor)
0.60 <0.001
Wait time (months) -0.20 <0.001
• Excellent reputation preferred to don’t know reputation
• Surgeon selected by you preferred to surgeon selected
by your doctor
• Shorter wait times are preferred to longer wait times
Willingness to Wait for Surgeon with
Excellent Reputation
56
• How long are patients willing to wait to have a
consultation with an excellent surgeon compared to not
knowing their surgeon reputation?
• What does this mean?
Variable Coefficient P value
Surgeon reputation: excellent
(reference = don’t know)
2.0 <0.001
Surgeon referral: surgeon selected by
you
(reference = selected by your doctor)
0.60 <0.001
Wait time (months) -0.20 <0.001
Next Available Surgeon:
Willingness to Wait to Select Surgeon
57
• How long are patients willing to wait to select a
surgeon themselves compared to having their doctor
select a surgeon?
• What does this mean?
Variable Coefficient P value
Surgeon reputation: good
(reference = don’t know)
2.0 <0.001
Surgeon referral: surgeon selected by
you
(reference = selected by your doctor)
0.60 <0.001
Wait time (months) -0.20 <0.001
Next Available Surgeon:
Attributes and Levels
58
Attributes Levels
Surgeon
reputation
• Excellent
• Good
• Satisfactory
• Don’t know
Surgeon referral
• Selected by you
• Next available surgeon
• Selected by your doctor
Time to surgeon
consultation
• 1 month
• 6 months
• 12 months
• 18 months
Time to surgery
• 1 month
• 6 months
• 12 months
• 18 months
Time to hospital
• 1 hour of less
• More than 1 hour
Example DCE Choice Scenario
59
Attributes
Willingness to Wait to Surgeon
with an Excellent Reputation
 Patients are
willing to wait
~10 months
…to see a
surgeon with an
excellent
reputation (vs a
surgeon with a
good reputation)
- Marshall DA et al. Osteoarthritis and Cartilage, 2018
 Patients with the
worst pain are
willing to wait
~7 months
 Patients with the
least pain are
willing to wait
~12 months
…to select the
surgeon themselves
(vs being assigned
the next available
surgeon from a list)
Willingness to Wait to Select Surgeon
verses Next Available Surgeon
- Marshall DA et al. Osteoarthrits and Cartilege, 2018
62
At the end of this session participants will be able to:
1. Differentiate between patient reported outcome and
experience measures (PROMs and PREMs) and patient
preferences
2. Identify and describe good research practices to develop,
design, and execute a patient preferences survey
3. Describe how patient preferences can be measured and how to
interpret the results from patient preferences studies
4. Identify ways in which patient preferences can
be applied in clinical practice for patients with
osteoarthritis
Learning Objectives
What is the Future for Arthritis Care
Informed by Patient Preferences ?
1) Support Patient Centered Care and Personalized
Medicine in Clinical Practice
2) Inform Clinical Practice Guidelines
3) Inform Regulatory Decisions about Therapies
63
64
Identify Preference Phenotypes of Patients with
RA for Treatment Based on Benefit-Risk Profiles
Prefer Triple Therapy
Risk averse (rare),
Cost sensitive, oral
Prefer anti-TNF
- Avoid bothersome side effects
Prefer anti-TNF
- Rapid onset of action
- Fraenkel L et al. Ann Rheum Dis, 2017.
Recognising
heterogeneity in
patient
preferences is
important for
choosing
treatment to
achieve best
outcomes for that
individual patient.
2) Using Patient Preferences to Inform Clinical
Practice Guidelines
65
Grading of Recommendations Assessment,
Development and Evaluation (GRADE)
66
Considerations in formulating guideline
recommendations (in addition to the quality of
the evidence):
• Tradeoffs between benefits and harms
• Uncertainty in the estimates of effects
• Values and preferences of benefits and harms from
those affected
• Translation of evidence into specific setting
• Resource implications
- GRADE working group. BMJ 2004.
√
√
√
Clinical Practice Guidelines: Patient
Preferences Can Differ from Guidelines
- Harrison M et al. BMJ Open 2017
67
- Hazlewood GS et al, Rheumatology, 2016; Hazlewood G et al, J Clin Epi 2018
Treatment preferences of patients with early rheumatoid arthritis:
• On average, patients were risk tolerant, supporting intensive
treatment approaches
• Two classes of patient identified:
a) Patients who were more averse to IV therapies and certain rare risks,
and
b) patients who were highly benefit-driven
Key Messages:
1. There was important heterogeneity in preferences that should
be considered in clinical treatment
2. In contrast to guidelines, many patients with early rheumatoid
arthritis may prefer triple therapy to other treatment options,
a) as initial treatment (78%) or after an inadequate response
to methotrexate (62%)
68
3) Patient Perspectives in Regulatory Decisions
Patient-Focused Benefit-Risk
Analysis to Inform Regulatory
Decisions Value in Health
Themed Issue, October, 2016
- Guest Editor Shelby Reed, Themed Issuue, Value in Health, Oct 2016
Patient-centered movement
Quantitative benefit-risk
Canadian Agency for Drugs
and Technology in Health
(CADTH)
Common Drug Review (CDR)
Process for Patient Input
69
CLINICAL
BENEFIT
ECONOMIC
EVALUATION
ADOPTION
FEASIBILITY
PATIENT INPUT
Drug Evaluation
Recommendations based
on
4 main criteria
https://cadth.ca/about-cadth/what-we-do/products-
services/cdr/patient-input
- Klein AV, Hardy S, Lim R, Marshall DA. Regulatory
decision-making in Canada – Exploring new frontiers in
patient involvement. Value in Health, 2016
Health Canada has an established
practice, albeit implicit and often ad
hoc, for including patient
perspectives in both operational and
policy-based regulatory decision-
making.
Value in Health Themed Issue,
October, 2016
Patient-Focused Benefit-Risk
Analysis to Inform Regulatory
Decisions
Summary of Patient Preferences in Arthritis
• Preferences measure risk benefit trade offs
• Good Research Practice guidance is available for
designing, conducting and analyzing preferences
• Clinical practice - preferences of patients can be
presented by distinct phenotypes to inform decisions
• Clinical guidelines- incorporating patients’ preferences
into clinical guideline development and
recommendations
• Regulatory Approval – Expect that evidence on
patient’s perspective will be part of the regulatory
approval process in the future.
70
71
Join us in Banff at our Next
DCE Workshop!
Applied Workshop – 3 days
September 9-11, 2019
Using Discrete Choice Experiments in
Health Economics: Theoretical and
Practical Issues
Thank you to colleagues and trainees and funders
Arthur J.E. Child Chair Foundation
Canada Research Chair Program
Thank you!
Deborah A Marshall
damarsha@ucalgary.ca
403-210-6377

More Related Content

What's hot

Core Outcome Measures in Effectiveness Trials
Core Outcome Measures in Effectiveness TrialsCore Outcome Measures in Effectiveness Trials
Core Outcome Measures in Effectiveness Trials
HTAi Bilbao 2012
 
CIGARETTE AND OTHER TOBACCO PRODUCTS ACT, (COTPA)
CIGARETTE AND OTHER TOBACCO PRODUCTS ACT, (COTPA)CIGARETTE AND OTHER TOBACCO PRODUCTS ACT, (COTPA)
CIGARETTE AND OTHER TOBACCO PRODUCTS ACT, (COTPA)
SURESH CHAND YADDANAPALLI
 
Study design used in pharmacoepidemiology
Study design used in pharmacoepidemiology Study design used in pharmacoepidemiology
Study design used in pharmacoepidemiology
kamolwantnok
 
Case control study
Case control studyCase control study
Case control study
Timiresh Das
 
Microdosing (Phase 0) studies
Microdosing (Phase 0) studiesMicrodosing (Phase 0) studies
Microdosing (Phase 0) studies
Dr. Ashutosh Tiwari
 
Introduction to clinical research
Introduction to clinical researchIntroduction to clinical research
Introduction to clinical research
Suman Baishnab
 
Clinical trial phases 3,4,5 By Danish Ibrahim Jasnaik
Clinical trial phases 3,4,5 By Danish Ibrahim JasnaikClinical trial phases 3,4,5 By Danish Ibrahim Jasnaik
Clinical trial phases 3,4,5 By Danish Ibrahim Jasnaik
Danish Jasnaik
 
Experimental Study
Experimental StudyExperimental Study
Experimental Study
Mukesh Kumar
 
Different phases of clinical trials
Different phases of clinical trialsDifferent phases of clinical trials
Different phases of clinical trials
Azad Singh
 
PRO (patient reported outcomes)
PRO (patient reported outcomes)PRO (patient reported outcomes)
PRO (patient reported outcomes)
Dr Jagadesh Earla
 
Regulation in clinical trial, Schedule Y and recent amendments
Regulation in clinical trial, Schedule Y and recent amendmentsRegulation in clinical trial, Schedule Y and recent amendments
Regulation in clinical trial, Schedule Y and recent amendments
Dr. Siddhartha Dutta
 
Signal detection and management
Signal detection and managementSignal detection and management
Signal detection and management
sekharbabu41
 
Clincial trials and types
Clincial trials and typesClincial trials and types
Clincial trials and types
Koppala RVS Chaitanya
 
Factorial Design
Factorial DesignFactorial Design
Factorial Design
Lucknow University India
 
PvPI (The Pharmacovigilance Program of India )
PvPI (The Pharmacovigilance Program of India )PvPI (The Pharmacovigilance Program of India )
PvPI (The Pharmacovigilance Program of India )
Anurag Raghuvanshi
 
Clinical trial
Clinical trialClinical trial
Clinical trial
RewariBhavya
 
(I) MEDICAL RESEARCH_ UNIT_III_RESEARCH METHODOLOGY & BIOSTATISTICS.pptx
(I) MEDICAL RESEARCH_ UNIT_III_RESEARCH METHODOLOGY & BIOSTATISTICS.pptx(I) MEDICAL RESEARCH_ UNIT_III_RESEARCH METHODOLOGY & BIOSTATISTICS.pptx
(I) MEDICAL RESEARCH_ UNIT_III_RESEARCH METHODOLOGY & BIOSTATISTICS.pptx
RAHUL PAL
 

What's hot (20)

Core Outcome Measures in Effectiveness Trials
Core Outcome Measures in Effectiveness TrialsCore Outcome Measures in Effectiveness Trials
Core Outcome Measures in Effectiveness Trials
 
CIGARETTE AND OTHER TOBACCO PRODUCTS ACT, (COTPA)
CIGARETTE AND OTHER TOBACCO PRODUCTS ACT, (COTPA)CIGARETTE AND OTHER TOBACCO PRODUCTS ACT, (COTPA)
CIGARETTE AND OTHER TOBACCO PRODUCTS ACT, (COTPA)
 
Sample size estimation
Sample size estimationSample size estimation
Sample size estimation
 
Study design used in pharmacoepidemiology
Study design used in pharmacoepidemiology Study design used in pharmacoepidemiology
Study design used in pharmacoepidemiology
 
Case control study
Case control studyCase control study
Case control study
 
Microdosing (Phase 0) studies
Microdosing (Phase 0) studiesMicrodosing (Phase 0) studies
Microdosing (Phase 0) studies
 
Introduction to clinical research
Introduction to clinical researchIntroduction to clinical research
Introduction to clinical research
 
Clinical trial phases 3,4,5 By Danish Ibrahim Jasnaik
Clinical trial phases 3,4,5 By Danish Ibrahim JasnaikClinical trial phases 3,4,5 By Danish Ibrahim Jasnaik
Clinical trial phases 3,4,5 By Danish Ibrahim Jasnaik
 
Approval proposal format of NHRC
Approval proposal format of NHRCApproval proposal format of NHRC
Approval proposal format of NHRC
 
Experimental Study
Experimental StudyExperimental Study
Experimental Study
 
Different phases of clinical trials
Different phases of clinical trialsDifferent phases of clinical trials
Different phases of clinical trials
 
PRO (patient reported outcomes)
PRO (patient reported outcomes)PRO (patient reported outcomes)
PRO (patient reported outcomes)
 
Regulation in clinical trial, Schedule Y and recent amendments
Regulation in clinical trial, Schedule Y and recent amendmentsRegulation in clinical trial, Schedule Y and recent amendments
Regulation in clinical trial, Schedule Y and recent amendments
 
Survival analysis
Survival analysisSurvival analysis
Survival analysis
 
Signal detection and management
Signal detection and managementSignal detection and management
Signal detection and management
 
Clincial trials and types
Clincial trials and typesClincial trials and types
Clincial trials and types
 
Factorial Design
Factorial DesignFactorial Design
Factorial Design
 
PvPI (The Pharmacovigilance Program of India )
PvPI (The Pharmacovigilance Program of India )PvPI (The Pharmacovigilance Program of India )
PvPI (The Pharmacovigilance Program of India )
 
Clinical trial
Clinical trialClinical trial
Clinical trial
 
(I) MEDICAL RESEARCH_ UNIT_III_RESEARCH METHODOLOGY & BIOSTATISTICS.pptx
(I) MEDICAL RESEARCH_ UNIT_III_RESEARCH METHODOLOGY & BIOSTATISTICS.pptx(I) MEDICAL RESEARCH_ UNIT_III_RESEARCH METHODOLOGY & BIOSTATISTICS.pptx
(I) MEDICAL RESEARCH_ UNIT_III_RESEARCH METHODOLOGY & BIOSTATISTICS.pptx
 

Similar to What are Patient Preferences, How Do You Measure Patient Preferences, and How Do I Use Them?

MedicReS Winter School 2017 Vienna - Importance of Selection of Outcomes - Ma...
MedicReS Winter School 2017 Vienna - Importance of Selection of Outcomes - Ma...MedicReS Winter School 2017 Vienna - Importance of Selection of Outcomes - Ma...
MedicReS Winter School 2017 Vienna - Importance of Selection of Outcomes - Ma...
MedicReS
 
PMED: APPM Workshop: Challenges in Using Bayesian Analysis Approaches for Reg...
PMED: APPM Workshop: Challenges in Using Bayesian Analysis Approaches for Reg...PMED: APPM Workshop: Challenges in Using Bayesian Analysis Approaches for Reg...
PMED: APPM Workshop: Challenges in Using Bayesian Analysis Approaches for Reg...
The Statistical and Applied Mathematical Sciences Institute
 
EBM Therapy Appraisal Template F1
EBM Therapy Appraisal Template F1EBM Therapy Appraisal Template F1
EBM Therapy Appraisal Template F1
Imad Hassan
 
# 9th lect clinical trial analysis
# 9th lect  clinical  trial analysis# 9th lect  clinical  trial analysis
# 9th lect clinical trial analysis
Dr. Eman M. Mortada
 
Drug Discovery & Development Overview
Drug Discovery & Development OverviewDrug Discovery & Development Overview
Drug Discovery & Development Overview
MikeSumner
 
JC SEBMA Prognosis Appraisal Template V1
JC SEBMA Prognosis Appraisal Template V1JC SEBMA Prognosis Appraisal Template V1
JC SEBMA Prognosis Appraisal Template V1
Imad Hassan
 
randomized clinical trials II
randomized clinical trials IIrandomized clinical trials II
randomized clinical trials II
IAU Dent
 
C. Everett Koop National Health Award Update 2014 with Ron Goetzel
C. Everett Koop National Health Award Update 2014 with Ron Goetzel C. Everett Koop National Health Award Update 2014 with Ron Goetzel
C. Everett Koop National Health Award Update 2014 with Ron Goetzel
HPCareer.Net / State of Wellness Inc.
 
Surgical_audit_&_research_iagsisbbbuikbb
Surgical_audit_&_research_iagsisbbbuikbbSurgical_audit_&_research_iagsisbbbuikbb
Surgical_audit_&_research_iagsisbbbuikbb
YassinAdil1
 
The Role of Risk Stratification and Biomarkers in Prevention of CVD
The Role of Risk Stratification and Biomarkers in Prevention of CVDThe Role of Risk Stratification and Biomarkers in Prevention of CVD
The Role of Risk Stratification and Biomarkers in Prevention of CVD
CTSI at UCSF
 
Cadth 2015 e2 dd systemic review-ohtac aug13-2013_2
Cadth 2015 e2 dd systemic review-ohtac aug13-2013_2Cadth 2015 e2 dd systemic review-ohtac aug13-2013_2
Cadth 2015 e2 dd systemic review-ohtac aug13-2013_2
CADTH Symposium
 
Quick introduction to critical appraisal of quantitative research
Quick introduction to critical appraisal of quantitative researchQuick introduction to critical appraisal of quantitative research
Quick introduction to critical appraisal of quantitative research
Alan Fricker
 
Can CER and Personalized Medicine Work Together
Can CER and Personalized Medicine Work TogetherCan CER and Personalized Medicine Work Together
Can CER and Personalized Medicine Work TogetherJohn Cai
 
Eblm pres final
Eblm pres finalEblm pres final
Eblm pres finalprasath172
 
Elaboración de recomendaciones en GPC. Sistema GRADE
Elaboración de recomendaciones en GPC. Sistema GRADEElaboración de recomendaciones en GPC. Sistema GRADE
Elaboración de recomendaciones en GPC. Sistema GRADE
GuíaSalud
 
drug discovery & development
drug discovery & developmentdrug discovery & development
drug discovery & development
Rohit K.
 
CLINICAL AUDIT
CLINICAL AUDITCLINICAL AUDIT
CLINICAL AUDIT
S A Tabish
 
Surgical_audit_&_research_mm (1).ppt
Surgical_audit_&_research_mm (1).pptSurgical_audit_&_research_mm (1).ppt
Surgical_audit_&_research_mm (1).ppt
SofiaJohn5
 

Similar to What are Patient Preferences, How Do You Measure Patient Preferences, and How Do I Use Them? (20)

MedicReS Winter School 2017 Vienna - Importance of Selection of Outcomes - Ma...
MedicReS Winter School 2017 Vienna - Importance of Selection of Outcomes - Ma...MedicReS Winter School 2017 Vienna - Importance of Selection of Outcomes - Ma...
MedicReS Winter School 2017 Vienna - Importance of Selection of Outcomes - Ma...
 
PMED: APPM Workshop: Challenges in Using Bayesian Analysis Approaches for Reg...
PMED: APPM Workshop: Challenges in Using Bayesian Analysis Approaches for Reg...PMED: APPM Workshop: Challenges in Using Bayesian Analysis Approaches for Reg...
PMED: APPM Workshop: Challenges in Using Bayesian Analysis Approaches for Reg...
 
EBM Therapy Appraisal Template F1
EBM Therapy Appraisal Template F1EBM Therapy Appraisal Template F1
EBM Therapy Appraisal Template F1
 
# 9th lect clinical trial analysis
# 9th lect  clinical  trial analysis# 9th lect  clinical  trial analysis
# 9th lect clinical trial analysis
 
Drug Discovery & Development Overview
Drug Discovery & Development OverviewDrug Discovery & Development Overview
Drug Discovery & Development Overview
 
JC SEBMA Prognosis Appraisal Template V1
JC SEBMA Prognosis Appraisal Template V1JC SEBMA Prognosis Appraisal Template V1
JC SEBMA Prognosis Appraisal Template V1
 
randomized clinical trials II
randomized clinical trials IIrandomized clinical trials II
randomized clinical trials II
 
C. Everett Koop National Health Award Update 2014 with Ron Goetzel
C. Everett Koop National Health Award Update 2014 with Ron Goetzel C. Everett Koop National Health Award Update 2014 with Ron Goetzel
C. Everett Koop National Health Award Update 2014 with Ron Goetzel
 
Study Eligibility Criteria
Study Eligibility CriteriaStudy Eligibility Criteria
Study Eligibility Criteria
 
Surgical_audit_&_research_iagsisbbbuikbb
Surgical_audit_&_research_iagsisbbbuikbbSurgical_audit_&_research_iagsisbbbuikbb
Surgical_audit_&_research_iagsisbbbuikbb
 
The Role of Risk Stratification and Biomarkers in Prevention of CVD
The Role of Risk Stratification and Biomarkers in Prevention of CVDThe Role of Risk Stratification and Biomarkers in Prevention of CVD
The Role of Risk Stratification and Biomarkers in Prevention of CVD
 
Cadth 2015 e2 dd systemic review-ohtac aug13-2013_2
Cadth 2015 e2 dd systemic review-ohtac aug13-2013_2Cadth 2015 e2 dd systemic review-ohtac aug13-2013_2
Cadth 2015 e2 dd systemic review-ohtac aug13-2013_2
 
Quick introduction to critical appraisal of quantitative research
Quick introduction to critical appraisal of quantitative researchQuick introduction to critical appraisal of quantitative research
Quick introduction to critical appraisal of quantitative research
 
Can CER and Personalized Medicine Work Together
Can CER and Personalized Medicine Work TogetherCan CER and Personalized Medicine Work Together
Can CER and Personalized Medicine Work Together
 
Eblm pres final
Eblm pres finalEblm pres final
Eblm pres final
 
Elaboración de recomendaciones en GPC. Sistema GRADE
Elaboración de recomendaciones en GPC. Sistema GRADEElaboración de recomendaciones en GPC. Sistema GRADE
Elaboración de recomendaciones en GPC. Sistema GRADE
 
drug discovery & development
drug discovery & developmentdrug discovery & development
drug discovery & development
 
When to Select Observational Studies Quiz
When to Select Observational Studies QuizWhen to Select Observational Studies Quiz
When to Select Observational Studies Quiz
 
CLINICAL AUDIT
CLINICAL AUDITCLINICAL AUDIT
CLINICAL AUDIT
 
Surgical_audit_&_research_mm (1).ppt
Surgical_audit_&_research_mm (1).pptSurgical_audit_&_research_mm (1).ppt
Surgical_audit_&_research_mm (1).ppt
 

More from OARSI

How to Become More Involved in Peer Review
How to Become More Involved in Peer ReviewHow to Become More Involved in Peer Review
How to Become More Involved in Peer Review
OARSI
 
What do we want to see addressed by a reviewer?
What do we want to see addressed by a reviewer?What do we want to see addressed by a reviewer?
What do we want to see addressed by a reviewer?
OARSI
 
So You Want To Be a Reviewer?
So You Want To Be a Reviewer? So You Want To Be a Reviewer?
So You Want To Be a Reviewer?
OARSI
 
Real-life examples of manuscript reviews Comparison and contrast of useful ...
Real-life examples of manuscript reviews  Comparison and contrast of  useful ...Real-life examples of manuscript reviews  Comparison and contrast of  useful ...
Real-life examples of manuscript reviews Comparison and contrast of useful ...
OARSI
 
Real-life examples of manuscript reviews Comparison and contrast of useful ...
Real-life examples of manuscript reviews  Comparison and contrast of  useful ...Real-life examples of manuscript reviews  Comparison and contrast of  useful ...
Real-life examples of manuscript reviews Comparison and contrast of useful ...
OARSI
 
How to write an effective review (and help editors and authors)
How to write an effective review (and help editors and authors)How to write an effective review (and help editors and authors)
How to write an effective review (and help editors and authors)
OARSI
 
Overview of the Editorial Process
Overview of the Editorial ProcessOverview of the Editorial Process
Overview of the Editorial Process
OARSI
 
Real-life examples of manuscript reviews Comparison and contrast of useful ...
Real-life examples of manuscript reviews  Comparison and contrast of  useful ...Real-life examples of manuscript reviews  Comparison and contrast of  useful ...
Real-life examples of manuscript reviews Comparison and contrast of useful ...
OARSI
 
Statistical Review of Basic Science Manuscripts at Osteoarthritis and Cartila...
Statistical Review of Basic Science Manuscripts at Osteoarthritis and Cartila...Statistical Review of Basic Science Manuscripts at Osteoarthritis and Cartila...
Statistical Review of Basic Science Manuscripts at Osteoarthritis and Cartila...
OARSI
 
Imaging of Synovitis in OA
Imaging of Synovitis in OAImaging of Synovitis in OA
Imaging of Synovitis in OA
OARSI
 
Nuts & Bolts of Systematic Reviews, Meta-analyses & Network Meta-analyses
Nuts & Bolts of Systematic Reviews, Meta-analyses & Network Meta-analysesNuts & Bolts of Systematic Reviews, Meta-analyses & Network Meta-analyses
Nuts & Bolts of Systematic Reviews, Meta-analyses & Network Meta-analyses
OARSI
 
Osteoarthriits Imaging: 2018 Year in Review
Osteoarthriits Imaging: 2018 Year in ReviewOsteoarthriits Imaging: 2018 Year in Review
Osteoarthriits Imaging: 2018 Year in Review
OARSI
 
Vincent the Lumper!
Vincent the Lumper!Vincent the Lumper!
Vincent the Lumper!
OARSI
 
OA White Paper: Broader Implications for Advocacy, Health Policy & Research
OA White Paper: Broader Implications for Advocacy, Health Policy & ResearchOA White Paper: Broader Implications for Advocacy, Health Policy & Research
OA White Paper: Broader Implications for Advocacy, Health Policy & Research
OARSI
 
Structural Targets for Prevention of Post Traumatic OA
Structural Targets for Prevention of Post Traumatic OAStructural Targets for Prevention of Post Traumatic OA
Structural Targets for Prevention of Post Traumatic OA
OARSI
 
Building a translational team for impacting public policy Pre-Congress Worksh...
Building a translational team for impacting public policyPre-Congress Worksh...Building a translational team for impacting public policyPre-Congress Worksh...
Building a translational team for impacting public policy Pre-Congress Worksh...
OARSI
 
An industry point of view for building a translational team
An industry point of view for building a translational teamAn industry point of view for building a translational team
An industry point of view for building a translational team
OARSI
 
Osteoarthritis: Structural Endpoints for the Development of Drugs, Devices, a...
Osteoarthritis: Structural Endpoints for the Development of Drugs, Devices, a...Osteoarthritis: Structural Endpoints for the Development of Drugs, Devices, a...
Osteoarthritis: Structural Endpoints for the Development of Drugs, Devices, a...
OARSI
 
Understanding the Accelerated Pathway
Understanding the Accelerated PathwayUnderstanding the Accelerated Pathway
Understanding the Accelerated Pathway
OARSI
 
Approval of Therapeutics for Osteoarthritis in 2019
Approval of Therapeutics for Osteoarthritis in 2019Approval of Therapeutics for Osteoarthritis in 2019
Approval of Therapeutics for Osteoarthritis in 2019
OARSI
 

More from OARSI (20)

How to Become More Involved in Peer Review
How to Become More Involved in Peer ReviewHow to Become More Involved in Peer Review
How to Become More Involved in Peer Review
 
What do we want to see addressed by a reviewer?
What do we want to see addressed by a reviewer?What do we want to see addressed by a reviewer?
What do we want to see addressed by a reviewer?
 
So You Want To Be a Reviewer?
So You Want To Be a Reviewer? So You Want To Be a Reviewer?
So You Want To Be a Reviewer?
 
Real-life examples of manuscript reviews Comparison and contrast of useful ...
Real-life examples of manuscript reviews  Comparison and contrast of  useful ...Real-life examples of manuscript reviews  Comparison and contrast of  useful ...
Real-life examples of manuscript reviews Comparison and contrast of useful ...
 
Real-life examples of manuscript reviews Comparison and contrast of useful ...
Real-life examples of manuscript reviews  Comparison and contrast of  useful ...Real-life examples of manuscript reviews  Comparison and contrast of  useful ...
Real-life examples of manuscript reviews Comparison and contrast of useful ...
 
How to write an effective review (and help editors and authors)
How to write an effective review (and help editors and authors)How to write an effective review (and help editors and authors)
How to write an effective review (and help editors and authors)
 
Overview of the Editorial Process
Overview of the Editorial ProcessOverview of the Editorial Process
Overview of the Editorial Process
 
Real-life examples of manuscript reviews Comparison and contrast of useful ...
Real-life examples of manuscript reviews  Comparison and contrast of  useful ...Real-life examples of manuscript reviews  Comparison and contrast of  useful ...
Real-life examples of manuscript reviews Comparison and contrast of useful ...
 
Statistical Review of Basic Science Manuscripts at Osteoarthritis and Cartila...
Statistical Review of Basic Science Manuscripts at Osteoarthritis and Cartila...Statistical Review of Basic Science Manuscripts at Osteoarthritis and Cartila...
Statistical Review of Basic Science Manuscripts at Osteoarthritis and Cartila...
 
Imaging of Synovitis in OA
Imaging of Synovitis in OAImaging of Synovitis in OA
Imaging of Synovitis in OA
 
Nuts & Bolts of Systematic Reviews, Meta-analyses & Network Meta-analyses
Nuts & Bolts of Systematic Reviews, Meta-analyses & Network Meta-analysesNuts & Bolts of Systematic Reviews, Meta-analyses & Network Meta-analyses
Nuts & Bolts of Systematic Reviews, Meta-analyses & Network Meta-analyses
 
Osteoarthriits Imaging: 2018 Year in Review
Osteoarthriits Imaging: 2018 Year in ReviewOsteoarthriits Imaging: 2018 Year in Review
Osteoarthriits Imaging: 2018 Year in Review
 
Vincent the Lumper!
Vincent the Lumper!Vincent the Lumper!
Vincent the Lumper!
 
OA White Paper: Broader Implications for Advocacy, Health Policy & Research
OA White Paper: Broader Implications for Advocacy, Health Policy & ResearchOA White Paper: Broader Implications for Advocacy, Health Policy & Research
OA White Paper: Broader Implications for Advocacy, Health Policy & Research
 
Structural Targets for Prevention of Post Traumatic OA
Structural Targets for Prevention of Post Traumatic OAStructural Targets for Prevention of Post Traumatic OA
Structural Targets for Prevention of Post Traumatic OA
 
Building a translational team for impacting public policy Pre-Congress Worksh...
Building a translational team for impacting public policyPre-Congress Worksh...Building a translational team for impacting public policyPre-Congress Worksh...
Building a translational team for impacting public policy Pre-Congress Worksh...
 
An industry point of view for building a translational team
An industry point of view for building a translational teamAn industry point of view for building a translational team
An industry point of view for building a translational team
 
Osteoarthritis: Structural Endpoints for the Development of Drugs, Devices, a...
Osteoarthritis: Structural Endpoints for the Development of Drugs, Devices, a...Osteoarthritis: Structural Endpoints for the Development of Drugs, Devices, a...
Osteoarthritis: Structural Endpoints for the Development of Drugs, Devices, a...
 
Understanding the Accelerated Pathway
Understanding the Accelerated PathwayUnderstanding the Accelerated Pathway
Understanding the Accelerated Pathway
 
Approval of Therapeutics for Osteoarthritis in 2019
Approval of Therapeutics for Osteoarthritis in 2019Approval of Therapeutics for Osteoarthritis in 2019
Approval of Therapeutics for Osteoarthritis in 2019
 

Recently uploaded

BRACHYTHERAPY OVERVIEW AND APPLICATORS
BRACHYTHERAPY OVERVIEW  AND  APPLICATORSBRACHYTHERAPY OVERVIEW  AND  APPLICATORS
BRACHYTHERAPY OVERVIEW AND APPLICATORS
Krishan Murari
 
New Directions in Targeted Therapeutic Approaches for Older Adults With Mantl...
New Directions in Targeted Therapeutic Approaches for Older Adults With Mantl...New Directions in Targeted Therapeutic Approaches for Older Adults With Mantl...
New Directions in Targeted Therapeutic Approaches for Older Adults With Mantl...
i3 Health
 
Physiology of Special Chemical Sensation of Taste
Physiology of Special Chemical Sensation of TastePhysiology of Special Chemical Sensation of Taste
Physiology of Special Chemical Sensation of Taste
MedicoseAcademics
 
micro teaching on communication m.sc nursing.pdf
micro teaching on communication m.sc nursing.pdfmicro teaching on communication m.sc nursing.pdf
micro teaching on communication m.sc nursing.pdf
Anurag Sharma
 
BENIGN PROSTATIC HYPERPLASIA.BPH. BPHpdf
BENIGN PROSTATIC HYPERPLASIA.BPH. BPHpdfBENIGN PROSTATIC HYPERPLASIA.BPH. BPHpdf
BENIGN PROSTATIC HYPERPLASIA.BPH. BPHpdf
DR SETH JOTHAM
 
HOT NEW PRODUCT! BIG SALES FAST SHIPPING NOW FROM CHINA!! EU KU DB BK substit...
HOT NEW PRODUCT! BIG SALES FAST SHIPPING NOW FROM CHINA!! EU KU DB BK substit...HOT NEW PRODUCT! BIG SALES FAST SHIPPING NOW FROM CHINA!! EU KU DB BK substit...
HOT NEW PRODUCT! BIG SALES FAST SHIPPING NOW FROM CHINA!! EU KU DB BK substit...
GL Anaacs
 
ARTIFICIAL INTELLIGENCE IN HEALTHCARE.pdf
ARTIFICIAL INTELLIGENCE IN  HEALTHCARE.pdfARTIFICIAL INTELLIGENCE IN  HEALTHCARE.pdf
ARTIFICIAL INTELLIGENCE IN HEALTHCARE.pdf
Anujkumaranit
 
ARTHROLOGY PPT NCISM SYLLABUS AYURVEDA STUDENTS
ARTHROLOGY PPT NCISM SYLLABUS AYURVEDA STUDENTSARTHROLOGY PPT NCISM SYLLABUS AYURVEDA STUDENTS
ARTHROLOGY PPT NCISM SYLLABUS AYURVEDA STUDENTS
Dr. Vinay Pareek
 
Novas diretrizes da OMS para os cuidados perinatais de mais qualidade
Novas diretrizes da OMS para os cuidados perinatais de mais qualidadeNovas diretrizes da OMS para os cuidados perinatais de mais qualidade
Novas diretrizes da OMS para os cuidados perinatais de mais qualidade
Prof. Marcus Renato de Carvalho
 
Triangles of Neck and Clinical Correlation by Dr. RIG.pptx
Triangles of Neck and Clinical Correlation by Dr. RIG.pptxTriangles of Neck and Clinical Correlation by Dr. RIG.pptx
Triangles of Neck and Clinical Correlation by Dr. RIG.pptx
Dr. Rabia Inam Gandapore
 
ACUTE SCROTUM.....pdf. ACUTE SCROTAL CONDITIOND
ACUTE SCROTUM.....pdf. ACUTE SCROTAL CONDITIONDACUTE SCROTUM.....pdf. ACUTE SCROTAL CONDITIOND
ACUTE SCROTUM.....pdf. ACUTE SCROTAL CONDITIOND
DR SETH JOTHAM
 
Superficial & Deep Fascia of the NECK.pptx
Superficial & Deep Fascia of the NECK.pptxSuperficial & Deep Fascia of the NECK.pptx
Superficial & Deep Fascia of the NECK.pptx
Dr. Rabia Inam Gandapore
 
KDIGO 2024 guidelines for diabetologists
KDIGO 2024 guidelines for diabetologistsKDIGO 2024 guidelines for diabetologists
KDIGO 2024 guidelines for diabetologists
د.محمود نجيب
 
Cervical & Brachial Plexus By Dr. RIG.pptx
Cervical & Brachial Plexus By Dr. RIG.pptxCervical & Brachial Plexus By Dr. RIG.pptx
Cervical & Brachial Plexus By Dr. RIG.pptx
Dr. Rabia Inam Gandapore
 
For Better Surat #ℂall #Girl Service ❤85270-49040❤ Surat #ℂall #Girls
For Better Surat #ℂall #Girl Service ❤85270-49040❤ Surat #ℂall #GirlsFor Better Surat #ℂall #Girl Service ❤85270-49040❤ Surat #ℂall #Girls
For Better Surat #ℂall #Girl Service ❤85270-49040❤ Surat #ℂall #Girls
Savita Shen $i11
 
Physiology of Chemical Sensation of smell.pdf
Physiology of Chemical Sensation of smell.pdfPhysiology of Chemical Sensation of smell.pdf
Physiology of Chemical Sensation of smell.pdf
MedicoseAcademics
 
Alcohol_Dr. Jeenal Mistry MD Pharmacology.pdf
Alcohol_Dr. Jeenal Mistry MD Pharmacology.pdfAlcohol_Dr. Jeenal Mistry MD Pharmacology.pdf
Alcohol_Dr. Jeenal Mistry MD Pharmacology.pdf
Dr Jeenal Mistry
 
Evaluation of antidepressant activity of clitoris ternatea in animals
Evaluation of antidepressant activity of clitoris ternatea in animalsEvaluation of antidepressant activity of clitoris ternatea in animals
Evaluation of antidepressant activity of clitoris ternatea in animals
Shweta
 
263778731218 Abortion Clinic /Pills In Harare ,
263778731218 Abortion Clinic /Pills In Harare ,263778731218 Abortion Clinic /Pills In Harare ,
263778731218 Abortion Clinic /Pills In Harare ,
sisternakatoto
 
Hemodialysis: Chapter 3, Dialysis Water Unit - Dr.Gawad
Hemodialysis: Chapter 3, Dialysis Water Unit - Dr.GawadHemodialysis: Chapter 3, Dialysis Water Unit - Dr.Gawad
Hemodialysis: Chapter 3, Dialysis Water Unit - Dr.Gawad
NephroTube - Dr.Gawad
 

Recently uploaded (20)

BRACHYTHERAPY OVERVIEW AND APPLICATORS
BRACHYTHERAPY OVERVIEW  AND  APPLICATORSBRACHYTHERAPY OVERVIEW  AND  APPLICATORS
BRACHYTHERAPY OVERVIEW AND APPLICATORS
 
New Directions in Targeted Therapeutic Approaches for Older Adults With Mantl...
New Directions in Targeted Therapeutic Approaches for Older Adults With Mantl...New Directions in Targeted Therapeutic Approaches for Older Adults With Mantl...
New Directions in Targeted Therapeutic Approaches for Older Adults With Mantl...
 
Physiology of Special Chemical Sensation of Taste
Physiology of Special Chemical Sensation of TastePhysiology of Special Chemical Sensation of Taste
Physiology of Special Chemical Sensation of Taste
 
micro teaching on communication m.sc nursing.pdf
micro teaching on communication m.sc nursing.pdfmicro teaching on communication m.sc nursing.pdf
micro teaching on communication m.sc nursing.pdf
 
BENIGN PROSTATIC HYPERPLASIA.BPH. BPHpdf
BENIGN PROSTATIC HYPERPLASIA.BPH. BPHpdfBENIGN PROSTATIC HYPERPLASIA.BPH. BPHpdf
BENIGN PROSTATIC HYPERPLASIA.BPH. BPHpdf
 
HOT NEW PRODUCT! BIG SALES FAST SHIPPING NOW FROM CHINA!! EU KU DB BK substit...
HOT NEW PRODUCT! BIG SALES FAST SHIPPING NOW FROM CHINA!! EU KU DB BK substit...HOT NEW PRODUCT! BIG SALES FAST SHIPPING NOW FROM CHINA!! EU KU DB BK substit...
HOT NEW PRODUCT! BIG SALES FAST SHIPPING NOW FROM CHINA!! EU KU DB BK substit...
 
ARTIFICIAL INTELLIGENCE IN HEALTHCARE.pdf
ARTIFICIAL INTELLIGENCE IN  HEALTHCARE.pdfARTIFICIAL INTELLIGENCE IN  HEALTHCARE.pdf
ARTIFICIAL INTELLIGENCE IN HEALTHCARE.pdf
 
ARTHROLOGY PPT NCISM SYLLABUS AYURVEDA STUDENTS
ARTHROLOGY PPT NCISM SYLLABUS AYURVEDA STUDENTSARTHROLOGY PPT NCISM SYLLABUS AYURVEDA STUDENTS
ARTHROLOGY PPT NCISM SYLLABUS AYURVEDA STUDENTS
 
Novas diretrizes da OMS para os cuidados perinatais de mais qualidade
Novas diretrizes da OMS para os cuidados perinatais de mais qualidadeNovas diretrizes da OMS para os cuidados perinatais de mais qualidade
Novas diretrizes da OMS para os cuidados perinatais de mais qualidade
 
Triangles of Neck and Clinical Correlation by Dr. RIG.pptx
Triangles of Neck and Clinical Correlation by Dr. RIG.pptxTriangles of Neck and Clinical Correlation by Dr. RIG.pptx
Triangles of Neck and Clinical Correlation by Dr. RIG.pptx
 
ACUTE SCROTUM.....pdf. ACUTE SCROTAL CONDITIOND
ACUTE SCROTUM.....pdf. ACUTE SCROTAL CONDITIONDACUTE SCROTUM.....pdf. ACUTE SCROTAL CONDITIOND
ACUTE SCROTUM.....pdf. ACUTE SCROTAL CONDITIOND
 
Superficial & Deep Fascia of the NECK.pptx
Superficial & Deep Fascia of the NECK.pptxSuperficial & Deep Fascia of the NECK.pptx
Superficial & Deep Fascia of the NECK.pptx
 
KDIGO 2024 guidelines for diabetologists
KDIGO 2024 guidelines for diabetologistsKDIGO 2024 guidelines for diabetologists
KDIGO 2024 guidelines for diabetologists
 
Cervical & Brachial Plexus By Dr. RIG.pptx
Cervical & Brachial Plexus By Dr. RIG.pptxCervical & Brachial Plexus By Dr. RIG.pptx
Cervical & Brachial Plexus By Dr. RIG.pptx
 
For Better Surat #ℂall #Girl Service ❤85270-49040❤ Surat #ℂall #Girls
For Better Surat #ℂall #Girl Service ❤85270-49040❤ Surat #ℂall #GirlsFor Better Surat #ℂall #Girl Service ❤85270-49040❤ Surat #ℂall #Girls
For Better Surat #ℂall #Girl Service ❤85270-49040❤ Surat #ℂall #Girls
 
Physiology of Chemical Sensation of smell.pdf
Physiology of Chemical Sensation of smell.pdfPhysiology of Chemical Sensation of smell.pdf
Physiology of Chemical Sensation of smell.pdf
 
Alcohol_Dr. Jeenal Mistry MD Pharmacology.pdf
Alcohol_Dr. Jeenal Mistry MD Pharmacology.pdfAlcohol_Dr. Jeenal Mistry MD Pharmacology.pdf
Alcohol_Dr. Jeenal Mistry MD Pharmacology.pdf
 
Evaluation of antidepressant activity of clitoris ternatea in animals
Evaluation of antidepressant activity of clitoris ternatea in animalsEvaluation of antidepressant activity of clitoris ternatea in animals
Evaluation of antidepressant activity of clitoris ternatea in animals
 
263778731218 Abortion Clinic /Pills In Harare ,
263778731218 Abortion Clinic /Pills In Harare ,263778731218 Abortion Clinic /Pills In Harare ,
263778731218 Abortion Clinic /Pills In Harare ,
 
Hemodialysis: Chapter 3, Dialysis Water Unit - Dr.Gawad
Hemodialysis: Chapter 3, Dialysis Water Unit - Dr.GawadHemodialysis: Chapter 3, Dialysis Water Unit - Dr.Gawad
Hemodialysis: Chapter 3, Dialysis Water Unit - Dr.Gawad
 

What are Patient Preferences, How Do You Measure Patient Preferences, and How Do I Use Them?

  • 1. What Are Patient Preferences, How Do You Measure Patient Preferences, and How Do I Use Them? Deborah A Marshall, PhD Professor and Arthur J.E. Child Chair in Rheumatology Research Cumming School of Medicine OARSI, Toronto, May 3, 2019
  • 2. 2 At the end of this session participants will be able to: 1. Differentiate between patient reported outcome and experience measures (PROMs and PREMs) and patient preferences 2. Identify and describe good research practices to develop, design, and execute a patient preferences survey 3. Describe how patient preferences can be measured and how to interpret the results from patient preferences studies 4. Identify ways in which patient preferences can be applied in clinical practice for patients with osteoarthritis Learning Objectives
  • 3. What are Patient Reported Outcome Measures (PROMs) and Patient Reported Experience Measures (PREMs)? PROMs PREMs Umbrella term that includes outcome data reported directly by the patient Encompasses range of interactions that patients have with health care system - care from doctors, nurses, and staff in hospitals, health care facilities Includes global impressions, functional status, well-being, symptoms, health-related quality of life, satisfaction ACR core set of 3 PROMs: physical function, pain, global assessment of disease activity Communication with doctors and nurses; Pain management; Timeliness of assistance; Explanation of medications administered; Different from patient satisfaction (which relates to meeting expectations) Example: Health Assessment Questionnaire (HAQ) Example: Hospital Consumer Assessment of Healthcare Providers and Systems Survey (HCAHPS) - Van Tuyl et al. Rheum Dis Clin N Am 2016; Felson et al. Arth Rheum 1993; Felson et al. Arth Rheum 1995 ; Manery et al. N Engl J Med 2013
  • 4. Beyond PROMS and PREMS – Preferences Consider Choices and Trade-Offs • Experimental survey methods that ask respondents to express the relative desirability or acceptability of features that differ amongst alternatives …which reflects their underlying utility for that alternative Attributes Treatment A Treatment B Functional Improvement 20% improvement 40% improvement Side Effects Mild Moderate Mode of Administration Oral Injection Which Treatment would you choose? □ □ - Medical Device Innovation Consortium Framework for Patient-Centered Benefit-Risk Assessment, 2015
  • 5. Arthritis is a ‘preference sensitive’ condition Treatments are preference sensitive with a key issue of compliance and adherence to therapy. 5
  • 6. Patient Preference Methods Medical Device Innovation Consortium Catalogue of Methods Source: MDIC PCBR Framework Report Release Event, May 13, 2015. Available at: http://mdic.org/pcbr-framework-report-release/ Group Method Structured- weighting • Simple direct weighting • Ranking exercises • Swing weighting • Point allocation • Analytic hierarchy process • Outranking methods Health-state utility • Time tradeoff • Standard gamble Stated- preference • Direct-assessment questions • Threshold technique • Discrete-choice experiments • Best-worst scaling exercises Revealed- preference • Patient-preference trials • Direct questions in clinical trials
  • 7. Preference Methods • For uni-dimensional decisions (i.e., consider one attribute or outcome at a time) – Standard Gamble – Time tradeoff – Contingent valuation • For multi-dimensional decisions (i.e., consider multiple attributes or outcomes simultaneously) – ConjointAnalysis – Best-Worst Scaling (BWS) – Discrete-Choice Experiments (DCE)* – Analytic Hierarchy Process (AHP) 7
  • 8. – Respondents choose amongst a set of alternatives – Each alternative is a profile defined by attributes – (e.g., efficacy, tolerability, mode of administration, cost, etc…) – Each attribute can take on different levels – For example, if efficacy attribute is defined as a response rate, then levels could be – 60 out of 100 (60%) – 75 out of 100 (75%) – 85 out of 100 (85%) Direct Preference Elicitation with Discrete- Choice Experiment (DCE)
  • 9. – Profiles are combined into sets in each choice task – Alternative choice formats can include • two or more active alternatives (forced choice), • opt-out or status quo (neither or none) • Each respondents completes a series of choice tasks • Each choice task has a different set of profiles determined by an experimental design • The key to a DCE is that one alternative is chosen in each choice task Direct Preference Elicitation with Discrete- Choice Experiment (DCE)
  • 10. Anatomy of a DCE Choice Task Attributes Prefer 1 Prefer 2 X Prefer 1 Prefer 2 X Outcome Medicine 1 Medicine 2 Response Rate 90 out of 100 (90%) 50 out of 100 (50%) Adverse Event Rate 10 out of 100 (10%) 1 out of 100 (1%) Which medicine would you prefer? Prefer 1 Prefer 2 Attributes Benefit Risk
  • 11. Anatomy of a DCE Choice Task Attribute Levels Prefer 1 Prefer 2 X Prefer 1 Prefer 2 X Outcome Medicine 1 Medicine 2 Response Rate 90 out of 100 (90%) 50 out of 100 (50%) Adverse Event Rate 10 out of 100 (10%) 1 out of 100 (1%) Which medicine would you prefer? Prefer 1 Prefer 2 Attribute Levels
  • 12. Anatomy of a DCE Choice Task Profile Prefer 1 Prefer 2 X Prefer 1 Prefer 2 X Outcome Medicine 1 Medicine 2 Response Rate 90 out of 100 (90%) 50 out of 100 (50%) Adverse Event Rate 10 out of 100 (10%) 1 out of 100 (1%) Which medicine would you prefer? Prefer 1 Prefer 2 Profile Attributes Benefit Risk
  • 13. Anatomy of a DCE Choice Task Choice Prefer 1 Prefer 2 X Prefer 1 Prefer 2 X Outcome Medicine 1 Medicine 2 Response Rate 90 out of 100 (90%) 50 out of 100 (50%) Adverse Event Rate 10 out of 100 (10%) 1 out of 100 (1%) Which medicine would you prefer? Prefer 1 Prefer 2 X Choice
  • 14. Anatomy of a DCE Choice Task Series of Choice Tasks Outcome Medicine 1 Medicine 2 Response Rate 90 out of 100 (90%) 50 out of 100 (50%) Adverse Event Rate 10 out of 100 (10%) 1 out of 100 (1%) Which medicine would you prefer? Prefer 1 Prefer 2 X Outcome Medicine 1 Medicine 2 Response Rate 90 out of 100 (90%) 50 out of 100 (50%) Adverse Event Rate 10 out of 100 (10%) 1 out of 100 (1%) Which medicine would you prefer? Prefer 1 Prefer 2 X Outcome Medicine 1 Medicine 2 Response Rate 90 out of 100 (90%) 50 out of 100 (50%) Adverse Event Rate 10 out of 100 (10%) 1 out of 100 (1%) Which medicine would you prefer? Prefer 1 Prefer 2 X
  • 15. DCE Choice Task Give it a Try Car A Car B Color Grey Red Type 4 door sedan 2 door sport Safety rating 5 stars 3 stars Your choice 15
  • 16. DCE Choice Task Now what? Car A Car B Color Grey Red Type 4 door sedan 2 door sport Safety rating 5 stars 3 stars Price $20,000 $35,000 Your choice 16
  • 17. Choices Reveal Information about Preferences Car A Car B Color Grey Red Type 4 door sedan 2 door sport Safety rating 5 stars 3 stars Price $20,000 $35,000 Your choice X 17  Something is only of value if we are willing to give something up for it
  • 18. Utility Estimates Assumes the utility associated with an alternative or profile is a function of observed characteristics (attributes levels) and unobserved characteristics of the alternative 18 The utility of each medicine is the sum of the effect of each level Utility(Medicine 1) > Utility(Medicine 2) Prefer 1 X Prefer 1 Prefer 2 X Prefer 1 Prefer 2 X Outcome Medicine 1 Medicine 2 Response Rate 90 out of 100 (90%) 50 out of 100 (50%) Adverse Event Rate 10 out of 100 (10%) 1 out of 100 (1%) Which medicine would you prefer? Prefer 1 Prefer 2 X
  • 19. Using DCEs to Measure Preferences  DCE is a robust quantitative method grounded in economic theory to measure the value of alternative choices and risk-benefit trade-offs for specific attributes  Measure how people value components (attributes) of a product or service  Eg. For a drug - cost, side effects, delivery mode  Including non-health outcomes 19
  • 20. 20 At the end of this session participants will be able to: 1. Differentiate between patient reported outcome and experience measures (PROMs and PREMs) and patient preferences 2. Identify and describe good research practices to develop, design, and execute a patient preferences survey 3. Describe how patient preferences can be measured and how to interpret the results from patient preferences studies 4. Identify ways in which patient preferences can be applied in clinical practice for patients with osteoarthritis Learning Objectives
  • 21. ISPOR Task Forces on Good Research Practices (GRPs) https://www.ispor.org/workpaper/ConjointAnalysisGRP.asp
  • 22. 22 ISPOR Task Forces on Good Research Practices (GRPs) https://www.ispor.org/workpaper/ConjointAnalysisGRP.asp “Aligning health care policy with patient preferences could improve the effectiveness of health care interventions by improving adoption of, satisfaction with, and adherence to clinical treatments.”
  • 23. 10-point Checklist for Good Research Practice in CA in Health The checklist generally follows the steps required to conduct a conjoint analysis study Attributes and levels Define Objective Design and implement survey instrument Design experiment Analyze data Report Results However, some of these categories require multiple steps
  • 24. • Was a well-defined research question stated and is conjoint analysis an appropriate method for answering it? – Was a well-defined research question and/or testable hypothesis articulated? – Was the study perspective described and the study placed in any particular decision-making or policy context described? – What is the justification for using conjoint analysis to answer the research question? Task #1: Defining the Research Question
  • 25.  Ranking the importance of attributes  Examining tradeoffs  Estimating willingness to pay  Exploring variation in preferences – Within a population – Between two groups of stakeholders  Evaluating potential market share Possible Research Questions
  • 26. • Were the attributes and attribute levels supported by evidence? – Were all important and relevant attributes identified (that is, supported by literature reviews, focus groups, or other scientific method)? – Was the choice of included attributes justified and consistent with theory? – Were the range and number of levels for each included attribute justified? Task #2: Determining Attributes and Levels
  • 27. Qualitative Work as a Foundation to Stated Preferences… - Louviere, Hensher and Swait, 2008 - Lanscar and Louviere, 2008 “We cannot overemphasise how important it is to conduct this kind of qualitative, exploratory work to guide subsequent phases of the stated preference study. “It is highly recommended that qualitative work is conducted during attribute development…the study team should endeavour to understand the dimensions…along which the product is evaluated by consumers and how specific levels of these dimensions are expressed.” “There is scope to move beyond simplistic and ad hoc uses of qualitative tools before, alongside and after quantitative data collection.”
  • 28. Attributes: Consider all potential attributes, but in context of plausibility  What attributes are important to people  Number of attributes relevant to research question  Omitted attributes adversely affect study quality  Understand how people discuss attributes  What words or phrases do they use?  Understand any interactions between the attributes  Are attributes considered together?  Does the preference for one attribute depend on the level of another attribute (e.g. route of drug administration and drug regimen) Levels: Encompass salient range of values, even if hypothetical or not currently available Qualitative Methods for Attribute Development
  • 29. • Was the construction of the conjoint tasks appropriate? – Was the number of attributes in each conjoint task justified? – Was the number of scenarios in each conjoint task justified? – Was the number of conjoint tasks included in the data collection instrument appropriate? Task 3 Checklist : Construction of Tasks
  • 30. Survey Development and Administration 30 Qualitative Research Pretest Pilot Test Data Collection Conceptual Framework Identify Key Attributes Range of Levels Cognitive Feasibility Administrative Feasibility Administration
  • 31. How many Attributes and Levels? 31  # attributes ranged from 3 to 16  70% with 3 to 7 attributes  40-50% included cost as an attribute  # levels ranged from 2 to >6 - Marshall DA et al. The Patient 2010 - deBekker-Grob et al, Health Econ 2010
  • 32. Number of Valuation Tasks Appropriate? ~ 10-20 valuation tasks in a choice set Aim to: – Avoid respondent fatigue – Maximise information per respondent – Minimise fractional design 32- Marshall DA et al. The Patient 2010 - deBekker-Grob et al, Health Econ 2010
  • 33. • Was the choice of experimental design justified and evaluated? • Was the choice of experimental design justified? • Were alternative experimental designs considered? • Were the properties of the experimental design evaluated? • Was the number of conjoint tasks included in the date-collection instrument appropriate? Task #4: Experimental Design
  • 34. Principle of an experiment (the researcher controls the stimuli): – To vary one or more attributes with two or more levels and elicit a behavioural response – DCEs systematically vary attributes and levels to investigate the determinants of choice in a particular context. Experimental Design
  • 35. Full Factorial Design (all possible alternatives) • Product of # Levels for each attribute (grow quickly!) • 2 attributes with 2 levels = • 3 attributes with 3 levels = Factorial Design
  • 36. Full Factorial Design (all possible alternatives) • Product of # Levels for each attribute (grow quickly!) • Estimate main effects and all interactions Fractional Factorial Design (subset of all possible alternatives) – Select subset randomly (potentially biased unless very large sample size) or systematically (experimental design) to estimate effects – May not be able to estimate all interactions Factorial Design
  • 37. Criteria to Consider Desired Criteria Correlations among attributes Attributes are independent Level balance Completely balanced Number of overlapping attributes Minimal overlap Efficiency score Higher is better, but relative Restrictions on implausible combinations No implausible combinations Cognitive difficulty Low cognitive burden Complexity to generate design Simple to implement Considerations in Experimental Design
  • 38. What is the Right Sample Size? Effect on Estimate Precision - Johnson FRJ et al. Value in Health, 2013; Yang JC al J Choice Modelling,2015 ...precision varies with the inverse of the square root of sample size.
  • 39. 39 Task 6 Checklist: Instrument Design • Was the data collection instrument designed appropriately? • Was appropriate respondent information collected (such as sociodemographic, attitudinal, health history or status, and treatment experience)? • Were the attributes and levels defined, and was any contextual information provided? • Was the level of burden of the data-collection instrument appropriate? Were respondents encouraged and motivated?
  • 40. An Example of a Survey Outline Including a DCE •Confirming inclusion and exclusion criteriaScreening •Signed informed consent for face-to-face interviews •Online informed consent (“I agree to participate”) for online surveys Informed consent •Experience with disease •Experience with disease treatment and managementBackground questions •Descriptions of each attribute included in the conjoint tasks •Warm-up questionsInformation treatment •8-16 DCE Choice Task questions •# tasks depends on the number of attributes and levels •Determined by experimental design Choice Task questions •Age, gender, martial status, education, etc.Demographic questions 40
  • 41. 41 Task #7 Checklist: Data Collection • Was the data-collection plan appropriate? • Was the sampling strategy justified (for example, sample size, stratification, and recruitment)? • Was the mode of administration justified and appropriate (for example, face-to-face, pen- and-paper, web-based)? • Were ethical consideration addressed (for example, recruitment, information and/or consent, compensation)?
  • 42. Mode of Administration  Pen and paper survey by mail  Pen and paper survey in-person  Telephone assisted survey by interviewer  Computer-based survey in person  Television-based survey at home  Internet survey 42
  • 43. • Were statistical analyses and model estimations appropriate? • Were respondent characteristics examined and tested? • Was the quality of the responses examined (e.g. Rationality, validity, reliability) • Was model estimation conducted appropriately? Task #8 Checklist: Statistical Analysis
  • 44.  Data setup  Coding attribute levels – dummy or effects coding  Setting up the data for each choice question  Setting up the data for each respondent  Estimation  Conditional logit (the foundation)  Extensions of conditional logit  Calculations  Marginal rates of substitution (tradeoffs)  Scenario Analysis 44 Analysis Steps
  • 45. The Utility Function Ui = V(β,Xi ) + εi where • V is the value (utility) function • X is a vector of attribute levels • β is a vector or parameters (preference weights) • ε is a random error term
  • 46. Conditional Logistic Regression Analysis • Main effects • Assumes relative preferences for each attribute level are independent of the level of any other attribute in the profile • β1*attribute1 + β2*attribute2 + β3*attribute3 + ε • Interaction effects • Models preferences for an attribute level as dependent on the levels of other attributes in the profile U = …β12*attribute1*attribute2 + β13*attribute1*attribute3 + … 46
  • 47. Marginal Rates of Substitution to Estimate Risk Benefit Trade-Offs  Indirect utility (value) function: V = α + β1X1 + β2X2 + β3X3  Marginal rates of substitution: • - (βk / βj) •(if X1 and X2 are continuous and linear) 47
  • 48. 10-point Checklist for Good Research Practice in CA in Health The checklist generally follows the steps required to conduct a conjoint analysis study Attributes and levels Define Objective Design and implement survey instrument Design experiment Analyze data Report Results However, some of these categories require multiple steps
  • 49. 49 At the end of this session participants will be able to: 1. Differentiate between patient reported outcome and experience measures (PROMs and PREMs) and patient preferences 2. Identify and describe good research practices to develop, design, and execute a patient preferences survey 3. Describe how patient preferences can be measured and how to interpret the results from patient preferences studies 4. Identify ways in which patient preferences can be applied in clinical practice for patients with osteoarthritis Learning Objectives
  • 50. Osteoarthritis Treatment Benefits and Risks Category Attributes Levels Benefits Ambulatory pain* None (0mm) Mild (25mm) Moderate (50mm) Severe (75mm) Resting pain* Stiffness* Difficulty doing daily activities* Risks Ulcer risk** None 10 out of 1,000 (1%) 50 out of 1,000 (5%) 100 out of 1,000 (10%) Stroke risk*** None 5 out of 1,000 (0.5%) 15 out of 1,000 (1.5%) 30 out of 1,000 (3%) 50 Hauber et al., Osteoarthritis Cartilage, 2013 * After treatment, measured on a 100mm visual analog scale ** Incremental treated-related risk in the next year *** Incremental treatment-related risk in the next 5 years Benefits - Most important reductions: - ambulatory pain and difficulty doing daily activities (both: 6.32) - resting pain (2.80) - stiffness (2.65) Risks – Most Important for incremental changes (3%): - Risk of MI (10.00) - Stroke (8.90)
  • 51. Surgeon Referral and Wait Times for Total Joint Replacement - Marshall DA, Deal K, Conner-Spady B, Bohm E, Hawker G, Loucks, L MacDonald KV, Noseworthy T. How do Patients Trade-Off Surgeon Choice and Waiting Times for Total Joint Replacement: A Discrete Choice Experiment. Osteoarthrits and Cartilege 2018;26:522-530. - Damani Z, Spady C, Nash T, Stelfox T, Noseworthy T, Marshall DA. What is the influence of single-entry models on access to elective surgical procedures?: A systematic review. BMJ Open Feb 2017;7(2):e012225. - Connor-Spady BL, Marshall DA, Hawker GA, Bohm E, Dunbar MJ, Frank C, Noseworthy T. You’ll know when you’re ready. How do patients decide when the time is right for joint replacement surgery? BMC Health Services 2014;14:454 - Connor-Spady BL, Marshall DA, Bohm E, Dunbar MJ, Loucks L, Hennigar A, Frank C, Noseworthy T. Patient factors in referral choice for total joint replacement surgery. Medical Care 2014;52(4):300-306
  • 53. Next Available Surgeon: Simplified Example 53 Attributes Levels Surgeon reputation • Excellent • Good • Satisfactory • Don’t know Surgeon referral • Selected by you • Next available surgeon • Selected by your doctor Time to surgeon consultation • 1 month • 6 months • 12 months • 18 months Assume all other attributes of surgery (time to surgery and time to hospital) are the same between options. *Please note: this example does not use data from the published results (referenced in previous slides) given the complexity of the full DCE. This is being used as a simplified example and therefore results do not represent results reported from our published study.
  • 54. Next Available Surgeon: Simplified Example Choice Task 54 If you were told at the time of referral to a surgeon that these were the only scenarios available, which one would you choose? Scenario A Scenario B Surgeon reputation Excellent Satisfactory Surgeon referral Selected by you Selected by your doctor Time to surgeon consultation 18 months 6 months I would choose X
  • 55. Next Available Surgeon: Simplified Results 55 Variable Coefficient P value Surgeon reputation: excellent (reference = don’t know) 2.0 <0.001 Surgeon referral: surgeon selected by you (reference = selected by your doctor) 0.60 <0.001 Wait time (months) -0.20 <0.001 • Excellent reputation preferred to don’t know reputation • Surgeon selected by you preferred to surgeon selected by your doctor • Shorter wait times are preferred to longer wait times
  • 56. Willingness to Wait for Surgeon with Excellent Reputation 56 • How long are patients willing to wait to have a consultation with an excellent surgeon compared to not knowing their surgeon reputation? • What does this mean? Variable Coefficient P value Surgeon reputation: excellent (reference = don’t know) 2.0 <0.001 Surgeon referral: surgeon selected by you (reference = selected by your doctor) 0.60 <0.001 Wait time (months) -0.20 <0.001
  • 57. Next Available Surgeon: Willingness to Wait to Select Surgeon 57 • How long are patients willing to wait to select a surgeon themselves compared to having their doctor select a surgeon? • What does this mean? Variable Coefficient P value Surgeon reputation: good (reference = don’t know) 2.0 <0.001 Surgeon referral: surgeon selected by you (reference = selected by your doctor) 0.60 <0.001 Wait time (months) -0.20 <0.001
  • 58. Next Available Surgeon: Attributes and Levels 58 Attributes Levels Surgeon reputation • Excellent • Good • Satisfactory • Don’t know Surgeon referral • Selected by you • Next available surgeon • Selected by your doctor Time to surgeon consultation • 1 month • 6 months • 12 months • 18 months Time to surgery • 1 month • 6 months • 12 months • 18 months Time to hospital • 1 hour of less • More than 1 hour
  • 59. Example DCE Choice Scenario 59 Attributes
  • 60. Willingness to Wait to Surgeon with an Excellent Reputation  Patients are willing to wait ~10 months …to see a surgeon with an excellent reputation (vs a surgeon with a good reputation) - Marshall DA et al. Osteoarthritis and Cartilage, 2018
  • 61.  Patients with the worst pain are willing to wait ~7 months  Patients with the least pain are willing to wait ~12 months …to select the surgeon themselves (vs being assigned the next available surgeon from a list) Willingness to Wait to Select Surgeon verses Next Available Surgeon - Marshall DA et al. Osteoarthrits and Cartilege, 2018
  • 62. 62 At the end of this session participants will be able to: 1. Differentiate between patient reported outcome and experience measures (PROMs and PREMs) and patient preferences 2. Identify and describe good research practices to develop, design, and execute a patient preferences survey 3. Describe how patient preferences can be measured and how to interpret the results from patient preferences studies 4. Identify ways in which patient preferences can be applied in clinical practice for patients with osteoarthritis Learning Objectives
  • 63. What is the Future for Arthritis Care Informed by Patient Preferences ? 1) Support Patient Centered Care and Personalized Medicine in Clinical Practice 2) Inform Clinical Practice Guidelines 3) Inform Regulatory Decisions about Therapies 63
  • 64. 64 Identify Preference Phenotypes of Patients with RA for Treatment Based on Benefit-Risk Profiles Prefer Triple Therapy Risk averse (rare), Cost sensitive, oral Prefer anti-TNF - Avoid bothersome side effects Prefer anti-TNF - Rapid onset of action - Fraenkel L et al. Ann Rheum Dis, 2017. Recognising heterogeneity in patient preferences is important for choosing treatment to achieve best outcomes for that individual patient.
  • 65. 2) Using Patient Preferences to Inform Clinical Practice Guidelines 65
  • 66. Grading of Recommendations Assessment, Development and Evaluation (GRADE) 66 Considerations in formulating guideline recommendations (in addition to the quality of the evidence): • Tradeoffs between benefits and harms • Uncertainty in the estimates of effects • Values and preferences of benefits and harms from those affected • Translation of evidence into specific setting • Resource implications - GRADE working group. BMJ 2004. √ √ √
  • 67. Clinical Practice Guidelines: Patient Preferences Can Differ from Guidelines - Harrison M et al. BMJ Open 2017 67 - Hazlewood GS et al, Rheumatology, 2016; Hazlewood G et al, J Clin Epi 2018 Treatment preferences of patients with early rheumatoid arthritis: • On average, patients were risk tolerant, supporting intensive treatment approaches • Two classes of patient identified: a) Patients who were more averse to IV therapies and certain rare risks, and b) patients who were highly benefit-driven Key Messages: 1. There was important heterogeneity in preferences that should be considered in clinical treatment 2. In contrast to guidelines, many patients with early rheumatoid arthritis may prefer triple therapy to other treatment options, a) as initial treatment (78%) or after an inadequate response to methotrexate (62%)
  • 68. 68 3) Patient Perspectives in Regulatory Decisions Patient-Focused Benefit-Risk Analysis to Inform Regulatory Decisions Value in Health Themed Issue, October, 2016 - Guest Editor Shelby Reed, Themed Issuue, Value in Health, Oct 2016 Patient-centered movement Quantitative benefit-risk
  • 69. Canadian Agency for Drugs and Technology in Health (CADTH) Common Drug Review (CDR) Process for Patient Input 69 CLINICAL BENEFIT ECONOMIC EVALUATION ADOPTION FEASIBILITY PATIENT INPUT Drug Evaluation Recommendations based on 4 main criteria https://cadth.ca/about-cadth/what-we-do/products- services/cdr/patient-input - Klein AV, Hardy S, Lim R, Marshall DA. Regulatory decision-making in Canada – Exploring new frontiers in patient involvement. Value in Health, 2016 Health Canada has an established practice, albeit implicit and often ad hoc, for including patient perspectives in both operational and policy-based regulatory decision- making. Value in Health Themed Issue, October, 2016 Patient-Focused Benefit-Risk Analysis to Inform Regulatory Decisions
  • 70. Summary of Patient Preferences in Arthritis • Preferences measure risk benefit trade offs • Good Research Practice guidance is available for designing, conducting and analyzing preferences • Clinical practice - preferences of patients can be presented by distinct phenotypes to inform decisions • Clinical guidelines- incorporating patients’ preferences into clinical guideline development and recommendations • Regulatory Approval – Expect that evidence on patient’s perspective will be part of the regulatory approval process in the future. 70
  • 71. 71 Join us in Banff at our Next DCE Workshop! Applied Workshop – 3 days September 9-11, 2019 Using Discrete Choice Experiments in Health Economics: Theoretical and Practical Issues
  • 72. Thank you to colleagues and trainees and funders Arthur J.E. Child Chair Foundation Canada Research Chair Program Thank you! Deborah A Marshall damarsha@ucalgary.ca 403-210-6377