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HEA PTP: M212 Economic Evaluation 1 Session 7: Defining & Assessing Benefits for Economic Evaluation 1. Why, what and how of benefits. 2. Benefit assessment for CEA. 3. Benefit assessment for CUA. 4. Practical exercise in estimating benefits for CUA.

HEA PTP: M212 Economic Evaluation 1 Session 7: Defining & Assessing Benefits for Economic Evaluation 1.Why, what and how of benefits. 2.Benefit assessment

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Page 1: HEA PTP: M212 Economic Evaluation 1 Session 7: Defining & Assessing Benefits for Economic Evaluation 1.Why, what and how of benefits. 2.Benefit assessment

HEA PTP: M212 Economic Evaluation 1

Session 7: Defining & Assessing Benefits for Economic Evaluation

1. Why, what and how of benefits.

2. Benefit assessment for CEA.

3. Benefit assessment for CUA.

4. Practical exercise in estimating benefits for CUA.

Page 2: HEA PTP: M212 Economic Evaluation 1 Session 7: Defining & Assessing Benefits for Economic Evaluation 1.Why, what and how of benefits. 2.Benefit assessment

HEA PTP: M212 Economic Evaluation 2

Why Measure Benefits?

Efficiency

Maximise benefits for given resources

Page 3: HEA PTP: M212 Economic Evaluation 1 Session 7: Defining & Assessing Benefits for Economic Evaluation 1.Why, what and how of benefits. 2.Benefit assessment

HEA PTP: M212 Economic Evaluation 3

Key Features of Economic Evaluation

Economic evaluation is

“The comparative analysis of alternativecourses of action in terms of both their

costs and consequences in orderto assist policy decisions”.

1. Costs and consequences - efficiency!

2. Comparative - relative efficiency

Page 4: HEA PTP: M212 Economic Evaluation 1 Session 7: Defining & Assessing Benefits for Economic Evaluation 1.Why, what and how of benefits. 2.Benefit assessment

HEA PTP: M212 Economic Evaluation 4

Benefit Categories

InterventionIntervention

Direct BenefitsDirect Benefits Indirect BenefitsIndirect Benefits

Savings in productivity.Savings in

productivity.

Improved patient health status / utility.

Improved patient health status / utility.

Reduced health services

resource use eg. LoS.

Reduced health services

resource use eg. LoS.

Family and friends quality

of life.

Family and friends quality

of life.

Page 5: HEA PTP: M212 Economic Evaluation 1 Session 7: Defining & Assessing Benefits for Economic Evaluation 1.Why, what and how of benefits. 2.Benefit assessment

HEA PTP: M212 Economic Evaluation 5

Should Changes in Productivity be Included?

May depend upon viewpoint (govt., societal, NHS)

Main issues are level of ‘true’ loss and comparability• Measurement of value of loss (gross wage, friction cost)• Double-counting, especially with CUA/CBA• Comparability with ‘health’ focus (viewpoint again)• Comparability with other studies (applies to other variables also)

Solution?• Provide a good reason why they should be measured/included• Report separately from other results• Differentiate measurement and valuation

Page 6: HEA PTP: M212 Economic Evaluation 1 Session 7: Defining & Assessing Benefits for Economic Evaluation 1.Why, what and how of benefits. 2.Benefit assessment

HEA PTP: M212 Economic Evaluation 6

Should Benefits be Discounted?

Why not discount?

• Health, unlike resources, cannot be traded over time• Inter-generational equity (cf environmental economics)• If are discounted, may be different rate to cost

Why discount?• Inconsistent treatment costs and benefits• Inconsistent policy, especially in comparison with other sectors• Counter-intuitive conclusions for investment. eg always postpone!• Individuals do trade health over time ((dis)invest in health)

Page 7: HEA PTP: M212 Economic Evaluation 1 Session 7: Defining & Assessing Benefits for Economic Evaluation 1.Why, what and how of benefits. 2.Benefit assessment

HEA PTP: M212 Economic Evaluation 7

Negative And Positive Benefits (and Costs!)

C/E ratio = net cost/net benefits

Net cost = positive cost + negative cost

Net benefit = positive benefit + negative benefit

Negative cost = cost saving, eg reduced LoS

Negative benefit = reduced health, eg adverse event

Page 8: HEA PTP: M212 Economic Evaluation 1 Session 7: Defining & Assessing Benefits for Economic Evaluation 1.Why, what and how of benefits. 2.Benefit assessment

HEA PTP: M212 Economic Evaluation 8

Types of Economic Evaluation

Type of Analysis ResultResultConsequencesCosts

Cost Minimisation

Cost BenefitCost Benefit

Cost Utility

Cost Effectiveness

Dollars

Single or multiple effects not necessarily common.

Valued as “utility” eg. QALY

Different magnitude of a common measure

eg., LY’s gained, blood pressure reduction.

Least cost alternative.Least cost

alternative.Identical in all

respects.

DollarsDollars

Dollars

DollarsCost per unit of

consequence eg. cost per LY gained.

Cost per unit of consequence eg.

cost per LY gained.

Cost per unit of consequence eg. cost per QALY.

Cost per unit of consequence eg. cost per QALY.

As for CUA but valued in money. eg

willingness-to-pay

As for CUA but valued in money. eg

willingness-to-pay

Net $ cost: benefit ratio.

Net $ cost: benefit ratio.

Page 9: HEA PTP: M212 Economic Evaluation 1 Session 7: Defining & Assessing Benefits for Economic Evaluation 1.Why, what and how of benefits. 2.Benefit assessment

HEA PTP: M212 Economic Evaluation 9

How Can Health Be Measured?

Length of life• Mortality (numbers, rates, SMRs)

• Life expectancy

• Life years lost

Quality of life• Numerous QoL measures (generic and specific)

• SF-36, Nottingham Health Profile, Guttman Scale, Rotterdam Symptom Checklist, Hospital Anxiety and Depression scale etc….

Page 10: HEA PTP: M212 Economic Evaluation 1 Session 7: Defining & Assessing Benefits for Economic Evaluation 1.Why, what and how of benefits. 2.Benefit assessment

HEA PTP: M212 Economic Evaluation 10

Process of Benefit Assessment

1. Identification:

2. Measurement:

3. Valuation:

Mortality.Quality of life.

Measure in natural physical units (eg. number of deaths averted).

Value benefits if appropriate ie. if performing CUA or CBA.

Page 11: HEA PTP: M212 Economic Evaluation 1 Session 7: Defining & Assessing Benefits for Economic Evaluation 1.Why, what and how of benefits. 2.Benefit assessment

HEA PTP: M212 Economic Evaluation 11

Issues in Assessing Benefits for CEA

1. Efficacy vs effectiveness vs efficiency.

2. Intermediate versus final outcome.

3. Sources of data for CEA.

Page 12: HEA PTP: M212 Economic Evaluation 1 Session 7: Defining & Assessing Benefits for Economic Evaluation 1.Why, what and how of benefits. 2.Benefit assessment

HEA PTP: M212 Economic Evaluation 12

Efficacy Vs Effectiveness Vs Efficiency

Efficacy = measure of effect under ideal conditions.

Effectiveness = effect under ‘real life’ conditions.

Efficacy does not imply effectiveness

Efficiency = relationship between costs & benefits.

Effectiveness does not imply efficiency

Page 13: HEA PTP: M212 Economic Evaluation 1 Session 7: Defining & Assessing Benefits for Economic Evaluation 1.Why, what and how of benefits. 2.Benefit assessment

HEA PTP: M212 Economic Evaluation 13

Intermediate Vs Final Outcome Measures

Final = change in health (status) resulting from the programme.

Intermediate = change in clinical indicator resulting from the programme.

Need to establish causal link betweenintermediate and final outcome measure.

Page 14: HEA PTP: M212 Economic Evaluation 1 Session 7: Defining & Assessing Benefits for Economic Evaluation 1.Why, what and how of benefits. 2.Benefit assessment

HEA PTP: M212 Economic Evaluation 14

Examples of Intermediate Vs Final Outcomes Indicators (PBAC (PBS) Oz)

Condition being Final outcome Surrogate Outcome Indicatorstreated indicator

Coronary thrombosis Quality-adjusted Number surviving Number with specified Number achieving coronary(thrombolysis survival level of left ventricular re-perfusion

function

Stable angina Quality-adjusted Number with Number who can walk Number with adequate(various interventions) survival acceptable a specified distance relief of pain

quality of life

Asthma Quality-adjusted Number surviving Number with adequate Number achieving a target(various drugs) survival control of bronchial level of airways functions

hyperreactivity

Depression Quality-adjusted Number avoiding Quality of life (may be Number achieving a target(various drugs) survival suicide improved by drugs) Hamilton or Montgomery-

Asberg Depression RatingScale

Hypertension Quality-adjusted Number avoiding Quality of life (may be Number achieving a target(various drugs) survival a stroke worsened by drugs) blood pressure

Page 15: HEA PTP: M212 Economic Evaluation 1 Session 7: Defining & Assessing Benefits for Economic Evaluation 1.Why, what and how of benefits. 2.Benefit assessment

HEA PTP: M212 Economic Evaluation 15

Sources of Effectiveness Data

1. Clinical trials, eg RCT’s.

2. Epidemiological studies, eg cohort studies.

3. Synthesis methods, eg meta-analyses.

4. Use of modelling.

Page 16: HEA PTP: M212 Economic Evaluation 1 Session 7: Defining & Assessing Benefits for Economic Evaluation 1.Why, what and how of benefits. 2.Benefit assessment

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Randomised Controlled Trials

‘Gold standard’ - minimal bias and confounding.

Disadvantages:

1. Often establishes efficacy, not effectiveness.2. Selective subjects used.3. Limited opportunity to conduct.4. Limited time horizon.5. Costly to conduct.6. Often unethical and/or unfeasible.

Page 17: HEA PTP: M212 Economic Evaluation 1 Session 7: Defining & Assessing Benefits for Economic Evaluation 1.Why, what and how of benefits. 2.Benefit assessment

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Epidemiological Studies

Real life setting - establish effectiveness

Disadvantages:

1. Potential for significant bias and confounding.2. Causal link can be weak.

Page 18: HEA PTP: M212 Economic Evaluation 1 Session 7: Defining & Assessing Benefits for Economic Evaluation 1.Why, what and how of benefits. 2.Benefit assessment

HEA PTP: M212 Economic Evaluation 18

Decision Rules: CEA

CEA result = CEI (c/e). eg cost per LY gained

Decision rule = adopt lowest CEI

Application = technical efficiency

Qst addressed = “Should we undertake program “X” or

program “Y” to treat condition “A”?

Page 19: HEA PTP: M212 Economic Evaluation 1 Session 7: Defining & Assessing Benefits for Economic Evaluation 1.Why, what and how of benefits. 2.Benefit assessment

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Limitations of Measurements/Need for Valuation

Ambiguity in assessing overall improvement or detriment in health

Allocative efficiency - value of benefits > (opportunity) cost

Page 20: HEA PTP: M212 Economic Evaluation 1 Session 7: Defining & Assessing Benefits for Economic Evaluation 1.Why, what and how of benefits. 2.Benefit assessment

HEA PTP: M212 Economic Evaluation 20

Valuation Versus Measurement

Value is determined by benefits sacrificed elsewhere (weighted preference)

Valuation requires a trade-off between benefits - measurement does not

Page 21: HEA PTP: M212 Economic Evaluation 1 Session 7: Defining & Assessing Benefits for Economic Evaluation 1.Why, what and how of benefits. 2.Benefit assessment

HEA PTP: M212 Economic Evaluation 21

Methods of Valuing Health

‘Utility’ or ‘preference’ assessment• Quality-Adjusted Life Years (QALYs)• Variants on QALY - Years of Health Life (YHL), Health-Adjusted

Person Years (HAPY), Health-Adjusted Life expectancy (HALE)• Healthy-Year Equivalents (HYEs) (based on ‘sequence’ of SG)• Saved-young-life equivalent (SAVE) (based on PTO)

Monetary terms eg WTP• Willingness-to-pay (WTP)• Human Capital

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Quality Adjusted Life Years(QALYs)

Adjusts data on quantity of life years saved to reflect a valuation of the quality of those years

If healthy: QALY = 1

If unhealthy: QALY < 1

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HEA PTP: M212 Economic Evaluation 23

Qol Profile

0 5 10 15

No Life Years = 15

No QALYs = 11

QL Weighting

Page 24: HEA PTP: M212 Economic Evaluation 1 Session 7: Defining & Assessing Benefits for Economic Evaluation 1.Why, what and how of benefits. 2.Benefit assessment

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QALY Procedure

Identify possible health states - cover all important and relevant dimensions of QoL

Derive ‘weights’ for each state

Multiply life years (spent in each state) by ‘weight’ for that state

Page 25: HEA PTP: M212 Economic Evaluation 1 Session 7: Defining & Assessing Benefits for Economic Evaluation 1.Why, what and how of benefits. 2.Benefit assessment

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“Utility” Weight

Utility = satisfaction/well-being - reflects a consumers (weighted) preferences

Utility weights are necessarily subjective - they elicit an individual’s preferences for, or value of, one or more health states.

Must: 1. Have interval properties

2. Be ‘anchored’ at death and ‘good health’

Page 26: HEA PTP: M212 Economic Evaluation 1 Session 7: Defining & Assessing Benefits for Economic Evaluation 1.Why, what and how of benefits. 2.Benefit assessment

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Techniques For Measuring “Utility”

Variety of techniques available, including:

Time Trade off

Person Trade Off

Standard Gamble

Rating Scale

Page 27: HEA PTP: M212 Economic Evaluation 1 Session 7: Defining & Assessing Benefits for Economic Evaluation 1.Why, what and how of benefits. 2.Benefit assessment

HEA PTP: M212 Economic Evaluation 27

Obtaining “Utility” Weights

Two means of obtaining “utility” weights:

1. Evaluation specific/’holistic’ measures - develop evaluation specific (‘holistic’) description of health state and then derive weight for that specific state directly by population survey

2. Use ‘generic’ or ‘multi-attribute’ instruments - use predetermined weights, based on combination of

dimensions of health yielding a finite number of health states/values

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Evaluation Specific/‘holistic’ Measure

Advantages: 1. Sensitive

2. Account for wider QoL(eg process, duration, prognosis)

Disadvantages 1. Cost and time intensive

2. Lack of comparability

Page 29: HEA PTP: M212 Economic Evaluation 1 Session 7: Defining & Assessing Benefits for Economic Evaluation 1.Why, what and how of benefits. 2.Benefit assessment

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Generic (MAU) Instruments

Advantages: 1. Supply weights “off the shelf”2. Comparability

Disadvantages: 1. Insensitive to small changes in health

2. Dimensions may not be

sufficiently comprehensive

3. Weights may not be

transferable across groups

Page 30: HEA PTP: M212 Economic Evaluation 1 Session 7: Defining & Assessing Benefits for Economic Evaluation 1.Why, what and how of benefits. 2.Benefit assessment

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Some Other Issues

Choosing respondents for utility estimation - whose values count?

What constitutes a ‘correct’ health state description?

What is the appropriate ‘measurement’ technique?

Aggregation of values?

Biases - ageist, life enhancing versus life-saving etc.

Page 31: HEA PTP: M212 Economic Evaluation 1 Session 7: Defining & Assessing Benefits for Economic Evaluation 1.Why, what and how of benefits. 2.Benefit assessment

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Decision Rules: CUA

CUA result = CEI (c/e). eg cost per QALY gained

Decision rule = adopt lowest CEI

Application = 1. technical efficiency

2. possibly allocative efficiency within

health care sector

Qst addressed = 1. Should we undertake program “X”

or “Y” to treat condition “Z”?

2. Should we treat condition “A” or “B”?

Page 32: HEA PTP: M212 Economic Evaluation 1 Session 7: Defining & Assessing Benefits for Economic Evaluation 1.Why, what and how of benefits. 2.Benefit assessment

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Decision Rules: Issues

1. Perspective - Health Care Sector

- Purchaser/Provider

- Societal

2. Comparator

3. Budget constraint/indivisibility

4. NPV vs BCI

5. Limited nature of economic evaluation

Page 33: HEA PTP: M212 Economic Evaluation 1 Session 7: Defining & Assessing Benefits for Economic Evaluation 1.Why, what and how of benefits. 2.Benefit assessment

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CUA and Rationing

Market system - price mechanism establishes equilibrium (efficient allocation)

Non-market system - absence of price as allocative tool leads to other, non-price, techniques

Issue is one of: (i) philosophical basis for rationing; and (ii) applied technique for rationing

Page 34: HEA PTP: M212 Economic Evaluation 1 Session 7: Defining & Assessing Benefits for Economic Evaluation 1.Why, what and how of benefits. 2.Benefit assessment

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Methods of Explicit Rationing

L ayp a rticip a tion

M e d ica lp a te rn a lism

P o lit ica lp ro cesses

E q u ity E ff ic ie n cy

T e ch n ica lm e th o ds

E xp lic itra tion ing

(Coast et al, Priority setting: the health care debate, John Wiley, 1996)

Page 35: HEA PTP: M212 Economic Evaluation 1 Session 7: Defining & Assessing Benefits for Economic Evaluation 1.Why, what and how of benefits. 2.Benefit assessment

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Explicit Rationing: Technical Methods

Single principle

Little distinction between setting priorities at different levels

Examples

• maximising health gain• need-based rationing• lotteries• age-based rationing

Page 36: HEA PTP: M212 Economic Evaluation 1 Session 7: Defining & Assessing Benefits for Economic Evaluation 1.Why, what and how of benefits. 2.Benefit assessment

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Technical Method: ‘QALY League Tables’

Economic evaluation produces information on cost-effectiveness

If using comparable outcomes (eg QALY) can ‘rank’ according to c/e

Can use resultant ‘league table’ to allocate resource to most c/e first

Page 37: HEA PTP: M212 Economic Evaluation 1 Session 7: Defining & Assessing Benefits for Economic Evaluation 1.Why, what and how of benefits. 2.Benefit assessment

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League Tables: Handle With Care!

Studies show differences in methodology

• choice of discount rate• method of estimating utility values• range of costs included• choice of comparator

Requires consistent methodology, ‘admission criteria’ for inclusion, applicability in local decision context

Page 38: HEA PTP: M212 Economic Evaluation 1 Session 7: Defining & Assessing Benefits for Economic Evaluation 1.Why, what and how of benefits. 2.Benefit assessment

HEA PTP: M212 Economic Evaluation 38

The Oregon Plan

1987 - decision to stop funding for organ transplantation

1989 - Oregon Health Services Commission begins work

1990 - List 1

1991 - List 2

1994 - plan begins

Page 39: HEA PTP: M212 Economic Evaluation 1 Session 7: Defining & Assessing Benefits for Economic Evaluation 1.Why, what and how of benefits. 2.Benefit assessment

HEA PTP: M212 Economic Evaluation 39

Oregon List Version 1

Efficiency principle

1600 condition/treatment pairs

Cost/QALY gained

• social values• outcome• cost

Page 40: HEA PTP: M212 Economic Evaluation 1 Session 7: Defining & Assessing Benefits for Economic Evaluation 1.Why, what and how of benefits. 2.Benefit assessment

HEA PTP: M212 Economic Evaluation 40

Oregon List Version 1

“... looked at the first two pages of that list and threw it in the trash can”

“... the presence of numerous flaws, aberrations and errors”

(Harvey Klevit, member, Oregon Health Services Commission)

Page 41: HEA PTP: M212 Economic Evaluation 1 Session 7: Defining & Assessing Benefits for Economic Evaluation 1.Why, what and how of benefits. 2.Benefit assessment

HEA PTP: M212 Economic Evaluation 41

Oregon List Version 2

Equal treatment for equal need

709 condition/treatment pairs

Method:

• Development & ranking of categories• Ranking C/T pairs within categories

– Public preferences– Outcome

• Professional judgement

Page 42: HEA PTP: M212 Economic Evaluation 1 Session 7: Defining & Assessing Benefits for Economic Evaluation 1.Why, what and how of benefits. 2.Benefit assessment

HEA PTP: M212 Economic Evaluation 42

Oregon List Version 2

Top Five C/T pairs

1 Pneumonia - medical

2 Tuberculosis - medical

3 Peritonitis - medical/surgical

4 Foreign body - removal

5 Appendicitis - surgical

Bottom Five C/T pairs

705 Aplastic anaemia - medical

706 Prolapsed urethral mucosa - surgical

707 Central retinal artery occlusion - paracentesis of aqueous

708 Extremely low birth weight, < 23 weeks - life support

709 Anencephaly - life support

Page 43: HEA PTP: M212 Economic Evaluation 1 Session 7: Defining & Assessing Benefits for Economic Evaluation 1.Why, what and how of benefits. 2.Benefit assessment

HEA PTP: M212 Economic Evaluation 43

Summary

1. Benefits must be assessed to establish efficiency.

2. Breadth and depth of benefits measured (& valued) varies across type of economic evaluation.

3. Difference between valuation and measurement.

4. Debate on role of CUA (& CEA) in allocative efficiency

5. Beware ‘league tables’!