introduction to effectiveness, patient preferences and utilities patsi sinnott, pt, phd, mph herc...

Introduction to Effectiveness, Introduction to Effectiveness, Patient Preferences and UtilitiesPatient Preferences and Utilities

Patsi Sinnott, PT, PhD, MPHPatsi Sinnott, PT, PhD, MPH

HERC Economics CourseHERC Economics Course

May 6, 2009May 6, 2009

Health Economics Resource CenterHealth Economics Resource Center

OverviewOverview Brief review of cost-effectiveness analysis Brief review of cost-effectiveness analysis

(CEA) and cost utility analysis (CUA)(CEA) and cost utility analysis (CUA) Quality of life and health-related quality of life Quality of life and health-related quality of life Review of preference/utility measurementReview of preference/utility measurement Review of the most frequently used preference Review of the most frequently used preference

measurement systemsmeasurement systems Preference measurement in clinical trialsPreference measurement in clinical trials Guidelines on selecting measuresGuidelines on selecting measures


Poll1Poll1


Poll2Poll2


Poll 3Poll 3


Poll 4Poll 4


Poll 5Poll 5


CEA and CUA reviewCEA and CUA review CEA compares the costs and effectiveness of two CEA compares the costs and effectiveness of two

(or more) interventions; (or more) interventions; The effectiveness effectiveness is defined by the health benefit is defined by the health benefit

or outcome achieved with the intervention.or outcome achieved with the intervention. All outcomes are defined using natural units,All outcomes are defined using natural units,

Cost per avoided infection or hospitalizationCost per avoided infection or hospitalization Cost per day “free of anginal pain”Cost per day “free of anginal pain” Cost per gain in Life Year (LY).Cost per gain in Life Year (LY).


CEA and CUA reviewCEA and CUA review

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


Defining Quality of LifeDefining Quality of Life

Surveys and questionnairesSurveys and questionnaires Domains of various aspects of lifeDomains of various aspects of life Each combination of answers defines a Each combination of answers defines a

composite “state” or quality of life “status” for composite “state” or quality of life “status” for that individualthat individual


Defining quality of lifeDefining quality of life

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



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

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



Purpose of evaluation will determine the Purpose of evaluation will determine the instrumentinstrument

Quality of life measurement tool will define Quality of life measurement tool will define the broad concept of quality of lifethe broad concept of quality of life

Health-related quality of life (HRQoL) Health-related quality of life (HRQoL) measurement tool will define an individual’s measurement tool will define an individual’s “health state” or “health status”“health state” or “health status”


Health status surveys/instrumentsHealth status surveys/instruments – Survey of patient – Survey of patient perspectives about their own function, well-being and perspectives about their own function, well-being and other important health outcomes.other important health outcomes.

Health status measuresHealth status measures describe the describe the health state health state of an of an individual, for a specific period, or at a particular individual, for a specific period, or at a particular time, along various attributes of health.time, along various attributes of health.

Defining health-related quality of lifeDefining health-related quality of life


Defining health-related quality of lifeDefining health-related quality of life

HRQoL instruments are used to measureHRQoL instruments are used to measure Baseline health statusBaseline health status Comparative health statusComparative health status Effectiveness/outcomes of clinical Effectiveness/outcomes of clinical

interventionintervention


Instruments to measure HRQoLInstruments to measure HRQoL

Generic instruments:Generic instruments: SF-36: 8 dimensions of health, including SF-36: 8 dimensions of health, including

physical functioning, bodily pain, social physical functioning, bodily pain, social functioning and mental health.functioning and mental health.


Instruments to measure HRQoLInstruments to measure HRQoL

Disease-specific measures:Disease-specific measures: Asthma Quality of Life Questionnaire Asthma Quality of Life Questionnaire

(AQLQ)(AQLQ) American Urological Association’s Urinary American Urological Association’s Urinary

Bother ScaleBother Scale Oswestry Low Back Pain QuestionnaireOswestry Low Back Pain Questionnaire


Poll 6Poll 6


Whiteboard 1Whiteboard 1

What instruments have you used?What instruments have you used?


CEA/CUACEA/CUA

CEA compares the costs and effectiveness of CEA compares the costs and effectiveness of two (or more) interventions two (or more) interventions


CEA/CUACEA/CUA The effectiveness effectiveness is defined by the health is defined by the health

benefit or outcome achieved with the benefit or outcome achieved with the intervention intervention

This effectiveness is defined by a summary This effectiveness is defined by a summary measure that combinesmeasure that combines

Quantity of life, and Quantity of life, and Quality of life, weighted by the Quality of life, weighted by the

preferencepreference for that quality of life for that quality of life


CEA/CUACEA/CUA

The summary measure of health benefit or The summary measure of health benefit or outcome in CEA is the QALYoutcome in CEA is the QALY

includes both quality and quantity of includes both quality and quantity of life;life;

adjusted for the desirability of, or adjusted for the desirability of, or preferencepreference for the benefit achieved. for the benefit achieved.


Poll 7Poll 7



What instruments have you used (for a study of What instruments have you used (for a study of what medical conditions?)what medical conditions?)


The Quality Adjusted Life Year The Quality Adjusted Life Year (QALY) (QALY)

QALYs describe years of survival, adjusted QALYs describe years of survival, adjusted for quality of life: for quality of life: 0 = death 0 = death 1 = perfect health1 = perfect health

QALYs allow trade-off between length of life QALYs allow trade-off between length of life with quality of life:with quality of life: 1 QALY = 1 year in perfect health1 QALY = 1 year in perfect health 1 QALY = 2 years with utility of 0.51 QALY = 2 years with utility of 0.5


Quantifying the QALY or outcomeQuantifying the QALY or outcomeRequires:Requires: Description or estimation of the health states Description or estimation of the health states

expected to be experienced by patients with expected to be experienced by patients with the condition the condition

Estimation of the duration of each health stateEstimation of the duration of each health state Assessment of patient or community Assessment of patient or community

preferencespreferences for each health state for each health state



In CEA what components of health status will In CEA what components of health status will you need to measure ?you need to measure ?


Whiteboard summaryWhiteboard summary

Health care interventions have impact in many Health care interventions have impact in many dimensions of life,dimensions of life,

Those impacts may be more or less desirable.Those impacts may be more or less desirable. At issue is how to quantify many attributes of At issue is how to quantify many attributes of

outcome into a single measurement scale, outcome into a single measurement scale, which includes a valuation on the outcomes.which includes a valuation on the outcomes.

This valuation is defined as This valuation is defined as preference preference


Assessment of patient or community Assessment of patient or community preferencespreferences for each health state for each health state

Only health status measures, with Only health status measures, with preferences/utilitiespreferences/utilities assessed, can be used in assessed, can be used in economic analysis;economic analysis;

Only a few health status measures (generic or Only a few health status measures (generic or specific) have specific) have preferences/utilitiespreferences/utilities measured. measured.

In this talk, per Gold, et al recommendations, In this talk, per Gold, et al recommendations, preferences = utilitiespreferences = utilities


Deriving preferences or utilities for Deriving preferences or utilities for health stateshealth states

Basic methodology:Basic methodology: Surveys of patients experiencing the Surveys of patients experiencing the

condition or health state of interest; or condition or health state of interest; or Surveys of a community sample.Surveys of a community sample.

In both cases, individuals provide a personal In both cases, individuals provide a personal reflection on the relative value of different reflection on the relative value of different health states experienced or described.health states experienced or described.


Deriving preferences or utilitiesDeriving preferences or utilitiesTwo methods to derive preferences:Two methods to derive preferences: DirectDirect: individuals respond to composite descriptions : individuals respond to composite descriptions

of health states (their own or written descriptions)of health states (their own or written descriptions) IndirectIndirect: individuals respond to questions about : individuals respond to questions about

separately delineated dimensions (or attributes) of a separately delineated dimensions (or attributes) of a health state, and a summary score or utility weight is health state, and a summary score or utility weight is calculated.calculated.

Physical functionPhysical function Social functioningSocial functioning Mental health etc.Mental health etc.


Sample health state description (composite)Sample health state description (composite) You are able to see, hear and speak normallyYou are able to see, hear and speak normally You require the help of another person to walk or get You require the help of another person to walk or get

around; and require mechanical equipment as well.around; and require mechanical equipment as well. You are occasionally angry, irritable, anxious and You are occasionally angry, irritable, anxious and

depressed.depressed. You are able to learn and remember normally.You are able to learn and remember normally. You are able to eat, bathe, dress and use the toilet You are able to eat, bathe, dress and use the toilet

normally.normally. You are free of pain and discomfort.You are free of pain and discomfort.


Methods to assess preferencesMethods to assess preferences

Direct methodDirect method Individuals asked to choose (declare Individuals asked to choose (declare

preferencespreferences) between their current health ) between their current health state and alternative health status scenariosstate and alternative health status scenarios

Individuals make these choices based on Individuals make these choices based on their own comprehensive health state (or the their own comprehensive health state (or the composite described to them).composite described to them).


Methods to assess preferences for Methods to assess preferences for health stateshealth states

Direct MethodsDirect Methods Standard Gamble (SG)Standard Gamble (SG) Time Tradeoff (TTO)Time Tradeoff (TTO)


Direct: Standard Gamble (SG)Direct: Standard Gamble (SG)

Live rest of life in current Live rest of life in current health state; orhealth state; or

““take a pill (with risks) to take a pill (with risks) to be restored to perfect be restored to perfect health”health”

Scale represents risk of Scale represents risk of death respondent is death respondent is willing to bear in order to willing to bear in order to be restored to full health.be restored to full health.


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


How to get the SG & TTOHow to get the SG & TTO

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


Methods to assess preferencesMethods to assess preferences

Indirect methodIndirect method Individuals asked to rate preferences for Individuals asked to rate preferences for

separate domains of health statesseparate domains of health states Scores are aggregated to create a composite Scores are aggregated to create a composite

preference or utility weight for a health statepreference or utility weight for a health state


Sample Questions (EQ-5D)Sample Questions (EQ-5D)

Which statements best describe your own state Which statements best describe your own state of health today?of health today?

Mobility: Mobility: No problems walking aboutNo problems walking about Some problems walking aboutSome problems walking about I am confined to bedI am confined to bed


Sample Questions (EQ-5D)Sample Questions (EQ-5D)

Which statements best describe your own state Which statements best describe your own state of health today?of health today? Pain/discomfortPain/discomfort

No pain or discomfortNo pain or discomfort Moderate pain or discomfortModerate pain or discomfort Extreme pain or discomfortExtreme pain or discomfort


The aggregate health state descriptionThe aggregate health state description You are able to see, hear and speak normallyYou are able to see, hear and speak normally You require the help of another person to walk or get You require the help of another person to walk or get

around; and require mechanical equipment as well.around; and require mechanical equipment as well. You are occasionally angry, irritable, anxious and You are occasionally angry, irritable, anxious and

depressed.depressed. You are able to learn and remember normally.You are able to learn and remember normally. You are able to eat, bathe, dress and use the toilet You are able to eat, bathe, dress and use the toilet

normally.normally. You are free of pain and discomfort.You are free of pain and discomfort.


Indirect preference measurement systemsIndirect preference measurement systems

Individuals respond to questions about the Individuals respond to questions about the separate attributes of a health state, and a separate attributes of a health state, and a summary score or utility weight is calculatedsummary score or utility weight is calculated

Health utility measures vary in: Health utility measures vary in: Dimensions or attributes included; Dimensions or attributes included; The size and nationality of the sample The size and nationality of the sample

population used to establish the weights;population used to establish the weights; Health states defined by the survey; andHealth states defined by the survey; and

How the summary score isHow the summary score is calculated, etc.calculated, etc.


Methods to assess preferences for Methods to assess preferences for health stateshealth states

Indirect MeasuresIndirect Measures Health Utility Index (HUI)Health Utility Index (HUI) EuroQol (EQ-5D)EuroQol (EQ-5D) Quality of Well-Being Scale (QWB)Quality of Well-Being Scale (QWB) SF-6D SF-6D


Indirect measures: Health Utility Index Indirect measures: Health Utility Index (HUI)(HUI) 41 questions (many items can be skipped)41 questions (many items can be skipped)

can derive both HUI Mark 2 and HUI Mark 3 health can derive both HUI Mark 2 and HUI Mark 3 health utility scores.utility scores.

8 domains of health and 972,000 health states8 domains of health and 972,000 health states vision, hearing, speech, ambulation, dexterity, emotion, vision, hearing, speech, ambulation, dexterity, emotion,

cognition, and paincognition, and pain Basis of domain weights:Basis of domain weights:

Canadian community sample rated hypothetical health Canadian community sample rated hypothetical health statesstates

Utility theoryUtility theory


How to get the HUIHow to get the HUI

HUI is copyrighted and can be obtained for a HUI is copyrighted and can be obtained for a fee (~$3,000) from Health Utilities Inc (fee (~$3,000) from Health Utilities Inc (www.healthutilities.comwww.healthutilities.com))

For an overview of the HUI see Horsman, For an overview of the HUI see Horsman, Furlong, Feeny, and Torrance (2003)Furlong, Feeny, and Torrance (2003)

http://www.healthutilities.com/


Indirect measures: EuroQol EQ-5DIndirect measures: EuroQol EQ-5D

5 questions in 5 domains of health5 questions in 5 domains of health Mobility, self-care, usual activity, Mobility, self-care, usual activity,

pain/discomfort, or anxiety/depressionpain/discomfort, or anxiety/depression 245 health states.245 health states.

Basis of domain weights:Basis of domain weights: Past studies based on British community Past studies based on British community

sample sample New US weights recently publishedNew US weights recently published


How to get the EuroQol EQ-5DHow to get the EuroQol EQ-5D

Nonprofit research can obtain the EQ-5D for Nonprofit research can obtain the EQ-5D for free from the EuroQol Group (free from the EuroQol Group (www.euroqol.orgwww.euroqol.org))

See See Dolan, Gudex, Kind, & Williams (1997) Dolan, Gudex, Kind, & Williams (1997) for British-based EQ-5Dfor British-based EQ-5D

See Shaw, Johnson, & Coons (2005) for US-See Shaw, Johnson, & Coons (2005) for US-based EQ-5Dbased EQ-5D

http://www.euroqol.org/


Indirect measures: the QWBIndirect measures: the QWBQuality of Well-Being ScaleQuality of Well-Being Scale

Two versionsTwo versions Original interviewer-administeredOriginal interviewer-administered More recent self-administered (QWB-SA)More recent self-administered (QWB-SA)

QWB-SA is more feasible, but still takes timeQWB-SA is more feasible, but still takes time 76 questions; 1215 health states defined;76 questions; 1215 health states defined; Includes symptoms, mobility, physical activity, & Includes symptoms, mobility, physical activity, &

social activitysocial activity Basis of domain weights:Basis of domain weights:

Primary care patients in San Diego, CAPrimary care patients in San Diego, CA


How to obtain the QWB-SAHow to obtain the QWB-SA

Contact the UCSD Health Outcomes Contact the UCSD Health Outcomes Assessment Program (Assessment Program (http://www.medicine.ucsd.edu/fpm/hoap/index.htmlhttp://www.medicine.ucsd.edu/fpm/hoap/index.html) ) to register and obtain the QWBto register and obtain the QWB

For interview-administered version see For interview-administered version see Kaplan, Bush, & Berry (1975)Kaplan, Bush, & Berry (1975)

For self-administered version see Kaplan, For self-administered version see Kaplan, Ganiats, & Sieber (1996)Ganiats, & Sieber (1996)

http://www.medicine.ucsd.edu/fpm/hoap/index.html


Indirect measures: SF-6DIndirect measures: SF-6D Converts SF-36 or SF-12 scores to utilitiesConverts SF-36 or SF-12 scores to utilities

When based on SF-36, uses 10 itemsWhen based on SF-36, uses 10 items When based on SF-12, uses 7 itemsWhen based on SF-12, uses 7 items

6 health domains6 health domains physical functioning, role limitations, social physical functioning, role limitations, social

functioning, pain, mental health, and vitalityfunctioning, pain, mental health, and vitality Defines 18,000 health statesDefines 18,000 health states Basis of domain weightsBasis of domain weights

British community sampleBritish community sample


How to obtain SF-6DHow to obtain SF-6D

Both SF-36 and SF-12 can be obtained from Both SF-36 and SF-12 can be obtained from www.sf-36.orgwww.sf-36.org and the scoring algorithm for and the scoring algorithm for the SF-6D can be obtained from its developer, the SF-6D can be obtained from its developer, John Brazier.John Brazier.

For converting the SF-36 into utilities see For converting the SF-36 into utilities see Brazier, Roberts, & Deverill (2002)Brazier, Roberts, & Deverill (2002)

For converting the SF-12 into utilities see For converting the SF-12 into utilities see Ware, Kosinski, & Keller (1996)Ware, Kosinski, & Keller (1996)

http://www.sf-36.org/


Health related quality of life in clinical Health related quality of life in clinical trials (note of caution)trials (note of caution)

Gathering HRQoL (i.e. measuring health Gathering HRQoL (i.e. measuring health status) in clinical trials may have one or more status) in clinical trials may have one or more purposes:purposes: Define the Define the health stateshealth states that that mightmight be be

experienced during the disease progression;experienced during the disease progression; Define the Define the health stateshealth states that that areare experienced experienced

by each participant in a study;by each participant in a study; Establish the Establish the preferencespreferences or utilitiesor utilities for for

each each health state, health state, as defined by the patients as defined by the patients with the medical condition.with the medical condition.


Health related quality of life in clinical Health related quality of life in clinical trialstrials

Define the health states that might occurDefine the health states that might occur – in order – in order to define the physiologic stages of the condition;to define the physiologic stages of the condition;

Define the health states that do occurDefine the health states that do occur – to be used – to be used in modeling QALYs for a CEA, using previously in modeling QALYs for a CEA, using previously established established preferencespreferences for each health state for each health state experienced;experienced;

Establish the preferences of each health stateEstablish the preferences of each health state – to – to compare patient with community samples and compare patient with community samples and other studies.other studies.


Health related quality of life in clinical Health related quality of life in clinical trials (note of caution)trials (note of caution)

Be sure your purpose is clear, before you choose your measurement tool


Which method to use?Which method to use?

Trade-off between sensitivity and burdenTrade-off between sensitivity and burden Start with a literature search Start with a literature search


Hierarchy of methods Hierarchy of methods

Going from least burdensome to most:Going from least burdensome to most: Off-the-shelf utility valuesOff-the-shelf utility values Indirect Measures Indirect Measures

(HUI, EQ-5D, QWB, SF-6D)(HUI, EQ-5D, QWB, SF-6D) Use a disease-specific survey during the Use a disease-specific survey during the

trial and transform at a later time to trial and transform at a later time to preferencespreferences

Direct measure (SG, TTO)Direct measure (SG, TTO)


Off-the-shelf valuesOff-the-shelf values

Use preference weight determined in another Use preference weight determined in another study for health state of intereststudy for health state of interest Not all health states have been characterizedNot all health states have been characterized

Useful in decision modelingUseful in decision modeling


Indirect measures (HUI, EQ-5D, Indirect measures (HUI, EQ-5D, QWB, SF-6D)QWB, SF-6D)

Standard surveys that are widely usedStandard surveys that are widely used Review published studies on psychometric Review published studies on psychometric

properties properties in the population of interestin the population of interest May not reflect changes in health states caused May not reflect changes in health states caused

by intervention (or of interest) by intervention (or of interest) May lack “responsiveness ” May lack “responsiveness ”


Using disease-specific surveyUsing disease-specific survey

If consequences of the treatment or disease are If consequences of the treatment or disease are not captured with a generic measurenot captured with a generic measure

Use disease specific quality of life instrumentUse disease specific quality of life instrument Have community respondents value health Have community respondents value health

states with a direct measure at a later timestates with a direct measure at a later time


Using disease-specific surveyUsing disease-specific survey

Key methods issues: Key methods issues: Difficult to describe health state to Difficult to describe health state to

community respondentcommunity respondent Difficult to establish values when there are a Difficult to establish values when there are a

large number of possible health states large number of possible health states Expensive, but potentially sensitive to Expensive, but potentially sensitive to

variations in quality of life for this disease variations in quality of life for this disease Often used in addition to generic measureOften used in addition to generic measure


Direct Method (SG, TTO)Direct Method (SG, TTO)

May be necessary if effects of intervention are May be necessary if effects of intervention are complex:complex: Multiple domainsMultiple domains Effects not captured in disease-specific Effects not captured in disease-specific

instrumentinstrument


High variance in estimates from respondentsHigh variance in estimates from respondents Reflect risk aversion, feeling about Reflect risk aversion, feeling about

disability disability High variance = large sample sizeHigh variance = large sample size

Not the “community value” specified by Gold Not the “community value” specified by Gold et al et al

Direct Method (SG, TTO)Direct Method (SG, TTO)


Important Resources Important Resources Harvard Center for Risk AssessmentHarvard Center for Risk Assessment

http://http://www.hcra.harvard.eduwww.hcra.harvard.edu//

Brazier J, Deverill M, Green C, Harper R, Brazier J, Deverill M, Green C, Harper R, Booth A. A Review of the use of health status Booth A. A Review of the use of health status measures in economic evaluation. Health measures in economic evaluation. Health Technol Assess 1999;3(9).Technol Assess 1999;3(9).http://www.hta.nhsweb.nhs.uk/http://www.hta.nhsweb.nhs.uk/

Table of published utility weights Table of published utility weights (preferences) for different health states (preferences) for different health states http://www.tufts-nemc.org/cearegistry/http://www.tufts-nemc.org/cearegistry/

http://www.hcra.harvard.edu/



http://www.hta.nhsweb.nhs.uk/


HERCHERC

PL Sinnott, Joyce, JR, Barnett, PG. Preference PL Sinnott, Joyce, JR, Barnett, PG. Preference Measurement in Economic Analysis. Measurement in Economic Analysis. GuidebookGuidebook. Menlo Park, CA. VA Palo Alto . Menlo Park, CA. VA Palo Alto Health Economics Resource Center. 2007Health Economics Resource Center. 2007http://www.herc.research.va.gov/files/BOOK_419.pdfhttp://www.herc.research.va.gov/files/BOOK_419.pdf

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


QUESTIONS and COMMENTSQUESTIONS and COMMENTS

introduction to effectiveness, patient preferences and utilities patsi sinnott, pt, phd, mph herc...

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