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Introduction to Effectiveness, Patient Preferences and Utilities Patsi Sinnott, PT, PhD, MPH HERC Economics Course June 13, 2006 Overview Brief review of cost-effectiveness analysis (CEA) and cost utility analysis (CUA) Quality of life and health-related quality of life

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introduction to effectiveness patient preferences and utilities

Introduction to Effectiveness, Patient Preferences and Utilities

Patsi Sinnott, PT, PhD, MPH

HERC Economics Course

June 13, 2006

overview
Overview
  • Brief review of cost-effectiveness analysis (CEA) and cost utility analysis (CUA)
  • Quality of life and health-related quality of life
  • Review of preference/utility measurement
  • Review of the most frequently used preference measurement systems
  • Preference measurement in clinical trials
  • Guidelines on selecting measures

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cea and cua review
CEA and CUA review
  • CEA compares the costs and effectiveness of two (or more) interventions;
    • The effectiveness is defined by the health benefit or outcome achieved with the intervention.
  • All outcomes are defined using natural units,
    • Cost per avoided infection or hospitalization
    • Cost per day “free of anginal pain”
    • Cost per gain in Life Year (LY).

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cea and cua review4
CEA and CUA review
  • CEA and CUA require all outcomes be quantified in a single scale;
    • A day in hospital or an infection avoided vs.
    • A day “free of angina pain”
    • A day of “improved quality of life”.

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defining quality of life
Defining Quality of Life
  • Surveys and questionnaires
  • Domains of various aspects of life
  • Each combination of answers defines a composite “state” or quality of life “status” for that individual

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defining quality of life6
Defining quality of life
  • Quality of life: broad concept, includes all aspects of life; where and how one lives and plays; family circumstances; finances; housing and job satisfaction.

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defining quality of life7
Defining quality of life
  • Health-related quality of life*: narrower concept, that only includes aspects of life dominated or significantly influenced by mental or physical well-being;

* From Ware, et al., SF-36 Health Survey Manual

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defining quality of life8
Defining quality of life
  • Purpose of evaluation will determine the instrument
    • Quality of life measurement tool will define the broad concept of quality of life
    • Health-related quality of life (HRQoL) measurement tool will define an individual’s “health state” or “health status”

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defining health related quality of life
Defining health-related quality of life
  • Health status surveys/instruments – Survey of patient perspectives about their own function, well-being and other important health outcomes.
  • Health status measures describe the health state of an individual, for a specific period, or at a particular time, along various attributes of health.

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defining health related quality of life10
Defining health-related quality of life
  • HRQoL instruments are used to measure
    • Baseline health status
    • Comparative health status
    • Effectiveness/outcomes of clinical intervention

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instruments to measure hrqol
Instruments to measure HRQoL
  • Generic instruments:
    • SF-36: 8 dimensions of health, including physical functioning, bodily pain, social functioning and mental health.

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instruments to measure hrqol12
Instruments to measure HRQoL
  • Disease-specific measures:
    • Asthma Quality of Life Questionnaire (AQLQ)
    • American Urological Association’s Urinary Bother Scale
    • Oswestry Low Back Pain Questionnaire

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cea cua
CEA/CUA
  • CEA compares the costs and effectiveness of two (or more) interventions

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cea cua14
CEA/CUA
  • The effectiveness is defined by the health benefit or outcome achieved with the intervention
  • This effectiveness is defined by a summary measure that combines:
    • Quantity of life, and
    • Quality of life,
    • Weighted by the preference for that quality of life

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cea cua15
CEA/CUA
  • The summary measure of health benefit or outcome in CEA is the QALY
    • Includes both quality and quantity of life;
    • adjusted for the desirability of, or preference for the benefit achieved.

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the quality adjusted life year qaly
The Quality Adjusted Life Year (QALY)
  • QALYs describe years of survival, adjusted for quality of life:
    • 0 = death
    • 1 = perfect health
  • QALYs allow trade-off between length of life with quality of life:
    • 1 QALY = 1 year in perfect health
    • 1 QALY = 2 years with utility of 0.5

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quantifying the qaly or outcome
Quantifying the QALY or outcome
  • Requires:
    • Description or estimation of the health states expected to be experienced by patients with the condition
    • Estimation of the duration of each health state
    • Assessment of patient or community preferences for each health state

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white board exercise
White board exercise
  • In CEA what components of health status will you need to measure ?

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whiteboard summary
Whiteboard summary
  • Health care interventions have impact in many dimensions of life,
  • Those impacts may be more or less desirable.
  • At issue is how to quantify many attributes of outcome into a single measurement scale, which includes a valuation on the outcomes.
  • This valuation is defined as preference

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assessment of patient or community preferences for each health state
Assessment of patient or community preferences for each health state
  • Only health status measures, with preferences/utilities assessed, can be used in economic analysis;
  • Only a few health status measures (generic or specific) have preferences/utilities measured.
  • In this talk, per Gold, et al recommendations, preferences = utilities

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deriving preferences or utilities for health states
Deriving preferences or utilities for health states
  • Basic methodology:
    • Surveys of patients experiencing the condition or health state of interest; or
    • Surveys of a community sample.
  • In both cases, individuals provide a personal reflection on the relative value of different health states experienced or described.

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deriving preferences or utilities
Deriving preferences or utilities
  • Two methods to derive preferences:
    • Direct: individuals respond to composite descriptions of health states (their own or written descriptions)
    • Indirect: individuals respond to questions about separately delineated dimensions (or attributes) of a health state, and a summary score or utility weight is calculated.
      • Physical function
      • Social functioning
      • Mental health etc.

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sample health state description composite
Sample health state description (composite)
  • You are able to see, hear and speak normally
  • You require the help of another person to walk or get around; and require mechanical equipment as well.
  • You are occasionally angry, irritable, anxious and depressed.
  • You are able to learn and remember normally.
  • You are able to eat, bathe, dress and use the toilet normally.
  • You are free of pain and discomfort.

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methods to assess preferences
Methods to assess preferences
  • Direct method
    • Individuals asked to choose (declare preferences) between their current health state and alternative health status scenarios
    • Individuals make these choices based on their own comprehensive health state (or the composite described to them).

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methods to assess preferences for health states
Methods to assess preferences for health states
  • Direct Methods
    • Standard Gamble (SG)
    • Time Tradeoff (TTO)

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direct standard gamble sg
Direct: Standard Gamble (SG)
  • Live rest of life in current health state; or
  • “take a pill (with risks) to be restored to perfect health”
  • Scale represents risk of death respondent is willing to bear in order to be restored to full health.

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direct time tradeoff tto
Direct: Time Tradeoff (TTO)
  • How much reduction in total life willing to give up in order to live in perfect health

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how to get the sg tto
How to get the SG & TTO
  • The SG and TTO have are usually administered through interactive computer programs such as
    • U-Titer (Summer, Nease et al., 1991)
    • U-Maker (Sonnenberg FA, 1993)
    • iMPACT I and II(Lenert, Sturley, et al., 2002),
    • ProSPEC (Bayoumi)
    • FLAIR1, FLAIR2, (Goldstein et al.1993)

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methods to assess preferences29
Methods to assess preferences
  • Indirect method
    • Individuals asked to rate preferences for separate domains of health states
    • Scores are aggregated to create a composite preference or utility weight for a health state

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sample questions eq 5d
Sample Questions (EQ-5D)
  • Which statements best describe your own state of health today?
      • Mobility:
        • 1. No problems walking about
        • 2. Some problems walking about
        • 3. I am confined to bed

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sample questions eq 5d31
Sample Questions (EQ-5D)
  • Which statements best describe your own state of health today?
    • Pain/discomfort
      • No pain or discomfort
      • Moderate pain or discomfort
      • Extreme pain or discomfort

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the aggregate health state description
The aggregate health state description
  • You are able to see, hear and speak normally
  • You require the help of another person to walk or get around; and require mechanical equipment as well.
  • You are occasionally angry, irritable, anxious and depressed.
  • You are able to learn and remember normally.
  • You are able to eat, bathe, dress and use the toilet normally.
  • You are free of pain and discomfort.

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indirect preference measurement systems
Indirect preference measurement systems
  • Individuals respond to questions about the separate attributes of a health state, and a summary score or utility weight is calculated
  • Health utility measures vary in:
    • Dimensions or attributes included;
    • The size and nationality of the sample population used to establish the weights;
    • Health states defined by the survey; and
    • How the summary score iscalculated, etc.

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methods to assess preferences for health states34
Methods to assess preferences for health states
  • Indirect Measures
    • Health Utility Index (HUI)
    • EuroQol (EQ-5D)
    • Quality of Well-Being Scale (QWB)
    • SF-6D

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indirect measures health utility index hui
Indirect measures: Health Utility Index (HUI)
  • 41 questions (many items can be skipped)
    • can derive both HUI Mark 2 and HUI Mark 3 health utility scores.
  • 8 domains of health and 972,000 health states
    • vision, hearing, speech, ambulation, dexterity, emotion, cognition, and pain
  • Basis of domain weights:
    • Canadian community sample rated hypothetical health states
    • Utility theory

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how to get the hui
How to get the HUI
  • HUI is copyrighted and can be obtained for a fee (~$3,000) from Health Utilities Inc (www.healthutilities.com)
  • For an overview of the HUI see Horsman, Furlong, Feeny, and Torrance (2003)

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indirect measures euroqol eq 5d
Indirect measures: EuroQol EQ-5D
  • 5 questions in 5 domains of health
    • Mobility, self-care, usual activity, pain/discomfort, or anxiety/depression
    • 245 health states.
  • Basis of domain weights:
    • Past studies based on British community sample
    • New US weights recently published

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how to get the euroqol eq 5d
How to get the EuroQol EQ-5D
  • Nonprofit research can obtain the EQ-5D for free from the EuroQol Group (www.euroqol.org)
  • See Dolan, Gudex, Kind, & Williams (1997) for British-based EQ-5D
  • See Shaw, Johnson, & Coons (2005) for US-based EQ-5D

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indirect measures the qwb quality of well being scale
Indirect measures: the QWBQuality of Well-Being Scale
  • Two versions
    • Original interviewer-administered
    • More recent self-administered (QWB-SA)
  • QWB-SA is more feasible, but still takes time
    • 76 questions; 1215 health states defined;
    • Includes symptoms, mobility, physical activity, & social activity
  • Basis of domain weights:
    • Primary care patients in San Diego, CA

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how to obtain the qwb sa
How to obtain the QWB-SA
  • Contact the UCSD Health Outcomes Assessment Program (http://www.medicine.ucsd.edu/fpm/hoap/index.html) to register and obtain the QWB
  • For interview-administered version see Kaplan, Bush, & Berry (1975)
  • For self-administered version see Kaplan, Ganiats, & Sieber (1996)

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indirect measures sf 6d
Indirect measures: SF-6D
  • Converts SF-36 or SF-12 scores to utilities
    • When based on SF-36, uses 10 items
    • When based on SF-12, uses 7 items
  • 6 health domains
    • physical functioning, role limitations, social functioning, pain, mental health, and vitality
  • Defines 18,000 health states
  • Basis of domain weights
    • British community sample

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how to obtain sf 6d
How to obtain SF-6D
  • Both SF-36 and SF-12 can be obtained from www.sf-36.org and the scoring algorithm for the SF-6D can be obtained from its developer, John Brazier.
  • For converting the SF-36 into utilities see Brazier, Roberts, & Deverill (2002)
  • For converting the SF-12 into utilities see Ware, Kosinski, & Keller (1996)

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health related quality of life in clinical trials note of caution
Health related quality of life in clinical trials (note of caution)
  • Gathering HRQoL (i.e. measuring health status) in clinical trials may have one or more purposes:
    • Define the health states that might be experienced during the disease progression;
    • Define the health states that are experienced by each participant in a study;
    • Establish the preferencesor utilities for each health state, as defined by the patients with the medical condition.

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health related quality of life in clinical trials
Health related quality of life in clinical trials
  • Define the health states that might occur – in order to define the physiologic stages of the condition;
  • Define the health states that do occur – to be used in modeling QALYs for a CEA, using previously established preferences for each health state experienced;
  • Establish the preferences of each health state – to compare patient with community samples and other studies.

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health related quality of life in clinical trials note of caution45
Health related quality of life in clinical trials (note of caution)
  • Be sure your purpose is clear, before you choose your measurement tool

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which method to use
Which method to use?
  • Trade-off between sensitivity and burden
  • Start with a literature search

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hierarchy of methods
Hierarchy of methods
  • Going from least burdensome to most:
    • Off-the-shelf utility values
    • Indirect Measures
      • (HUI, EQ-5D, QWB, SF-6D)
    • Use a disease-specific survey during the trial and transform at a later time to preferences
    • Direct measure (SG, TTO)

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off the shelf values
Off-the-shelf values
  • Use preference weight determined in another study for health state of interest
    • Not all health states have been characterized
  • Useful in decision modeling

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indirect measures hui eq 5d qwb sf 6d
Indirect measures (HUI, EQ-5D, QWB, SF-6D)
  • Standard surveys that are widely used
  • Review published studies on psychometric properties in the population of interest
  • May not reflect changes in health states caused by intervention (or of interest)
    • May lack “responsiveness ”

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using disease specific survey
Using disease-specific survey
  • If consequences of the treatment or disease are not captured with a generic measure
  • Use disease specific quality of life instrument
  • Have community respondents value health states with a direct measure at a later time

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using disease specific survey51
Using disease-specific survey
  • Key methods issues:
    • Difficult to describe health state to community respondent
    • Difficult to establish values when there are a large number of possible health states
  • Expensive, but potentially sensitive to variations in quality of life for this disease
  • Often used in addition to generic measure

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direct method sg tto
Direct Method (SG, TTO)
  • May be necessary if effects of intervention are complex:
    • Multiple domains
    • Effects not captured in disease-specific instrument

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direct method sg tto53
Direct Method (SG, TTO)
  • High variance in estimates from respondents
    • Reflect risk aversion, feeling about disability
    • High variance = large sample size
  • Not the “community value” specified by Gold et al

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important resources
Important Resources
  • Harvard Center for Risk Assessment
    • http://www.hcra.harvard.edu/
  • Brazier J, Deverill M, Green C, Harper R, Booth A. A Review of the use of health status measures in economic evaluation. Health Technol Assess 1999;3(9).
    • http://www.hta.nhsweb.nhs.uk/
  • Table of published utility weights (preferences) for different health states
    • http://www.tufts-nemc.org/cearegistry/

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slide55
HERC
  • PL Sinnott, Joyce, JR, Barnett, PG. Preference Measurement in Economic Analysis. Guidebook. Menlo Park, CA. VA Palo Alto Health Economics Resource Center. 2007

http://www.herc.research.va.gov/files/BOOK_419.pdf

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slide56
QUESTIONS and COMMENTS

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