Cost Utility Analysis
Dr. Lance I. G. Catedral

T: @lanceoncology
E: lgcatedral@up.edu.ph
Mark 8:36, KJV
“For what shall it profit a man, if he shall gain the whole world, and
lose his own soul?”
A new chemo drug? A new screening modality? A new surgical
technique?
Physicians
Cost Utility Analysis
• special form of cost-effectiveness analysis

• costs per unit of utility are calculated

• compares cost and benefits, where benefits = number of life years saved,
adjusted for loss of quality

• anti-retrovirals vs polio vaccination

• osimertinib vs afatinib as first line treatment for lung cancer

• combines length and quality of life
When is CUA used?
• quality of life is the important outcome

• the program affects both morbidity and mortality

• the programs being compared have a wide range of outcomes

• one of the programs being compared has already been evaluated using
CUA
• Useful for policy makers and reimbursement agencies

• “Cost-effective if:

• UK: £30K per QALY gained

• USA: $50K per QALY gained

• WHO: ICER < 3x per capita gross domestic product for a given
country
Adapted from: Mohammed, Pharmacoeconomics: Cost utility analysis. https://www.slideshare.net/mameclinical/
pharmacoeconomics-cost-utility-analysis?qid=082f5261-353f-4e89-8dcf-2b557e7719c1&v=&b=&from_search=1
When NOT to use CUA
• Only have intermediate outcome data

• Effectiveness data show outcomes are equivalent

• Effectiveness data show dominance
Utility
• “measure of the preference for, or desirability of, a specific level of health status or
specific health outcome

• not “usefulness”

• also: “preference weights”

• quantitative approach for describing preferences for quality of life

• based on 0 to 1 scale

• 0 = death

• 1 = perfect health
Measurable,
quantifiable, and
analyzable
Response method
Question type
Certainty
(“value”)
Uncertainty
(“utility”)
Scale Rating scale
Choice Time trade-off Standard gamble
Standard gamble
Example of the standard gamble
Explanation
As with the time trade-off method, utilities for states of health can be de-
Complete healthAlternative 2:
uncertain outcome
Alternative 1:
certain outcome
Death
Limited health
95%
5%
100%
gamble on the alternatives, complete recovery or death. The probabilities
between the two alternatives are varied until the patient is indifferent to
both alternatives.
“Imagine you have breast cancer stage III. Suppose there is a
surgery that can potentially cure your cancer, but there is a risk for
death. How low does the probability of death have to be for you to be
indifferent between having breast cancer stage III and the gamble of
taking the surgery which can lead to death or cure?”
Standard gamble
Example of the standard gamble
Explanation
As with the time trade-off method, utilities for states of health can be de-
Complete healthAlternative 2:
uncertain outcome
Alternative 1:
certain outcome
Death
Limited health
95%
5%
100%
gamble on the alternatives, complete recovery or death. The probabilities
between the two alternatives are varied until the patient is indifferent to
both alternatives.
Alternative 1:
Breast cancer
Alternative 2:
Breast cancer +
surgery
Limited health
(living with cancer)
85%
15%
Complete health
(cancer remission
or cure)
Standard gamble
• Gold standard in utility elicitation

• respondent must choose between two options

• living with below-optimal health

• a lottery between two uncertain health states

• Limitations

• time-consuming

• cognitively difficult for many people
Time trade-off
The time trade-off method
Explanation
Time trade-off is a method of deriving utility values that can be used in
1.0
Alternative 2
Time
Alternative 1
hi
0
x t
choose between a period that is spent in a specific state of health and a
(shorter) period in perfect health, until they are indifferent to both peri-
ods of time.
“Imagine you have breast cancer stage III and you have a life
expectancy of 10 years. How much of your remaining life
expectancy would you give up to eliminate breast cancer so you
have perfect health?”
The time trade-off method
Explanation
Time trade-off is a method of deriving utility values that can be used in
1.0
Alternative 2
Time
Alternative 1
hi
0
x t
choose between a period that is spent in a specific state of health and a
(shorter) period in perfect health, until they are indifferent to both peri-
ods of time.
Cured of breast
cancer stage III
Breast cancer
stage III
10 y
Rating scale
0
Best possible
state
50 100
Worst possible
state
Rating scale
• Does not involve trade offs

• Simple, easy to administer

• Ordinal, not an interval scale
QALY weights
Source Examples Disadvantages
Literature
Individual studies

CUA databases
Lack of comparability
Indirect measures
EQ-5D-5L

EORTC QLQ-C30

FACT-G
Only common diseases
Direct measures
Expert panel

Special sample survey
Expense

Time 

Representation
Preference weights → QALY
QALY
• Quality-Adjusted Life-Years

• number of years of perfect health

• calculated by estimating the total life-years gained from a treatment and
weighing each year with a quality-of-life score (from 0, representing worst
health, to 1 or 100, representing best health) to reflect the quality of life in
that year. 

•
QALY’s advantage over CEA
• combines more than one measure of effectiveness

• mortality + morbidity in one single measure
Determining QALYs using the example of dialysis and kidney transplants
Explanation
Initial
situation
Death
Dialysis
Kidney transplant
Full health
1
0
Life expectancy
(years)
Qualityoflife(standardised) Area A:
QALYs lost
Area B:
QALYs gained
A
B
Surgery
Lumpectomy + RT
QALY
• Dapat ang years of life, may quality!

• Example: If a treatment prolongs the
remainder of the patient’s life by an
average of 3 years and the quality of life
is 0.5 on a scale of 0 (= poorest state of
health) to 1 (= best state of health)

• The number of QALYs is 3 × 0.5 =
1.5, without discounting. 

Photo credit: Dr. RE King
Exercise
• In your hospital in Cavite, you have been using chemo A as
standard of care. Chemo A prolongs life by 1 year but reduces the
quality of life of your patients due to its side effects. The new
chemo B prolongs life by 1.5 years at estimated utility value of 0.5.
Calculate the QALY for chemo A and chemo B.
• Chemo B (standard)
• Life expectancy = 1. 5 y

• Utility = 0.5

• QALY = 1.5 x 0.5 = 0.6=5 years

• Chemo A adds 0.75 QALY to the patient’s
life
• Chemo A (standard)
• Life expectancy = 1 y

• Utility = 1–0.35=0.65

• QALY = 1 x 0.65 = 0.65 years

• Chemo A adds 0.65 QALY to the patient’s life
League tables
Extract from a league table
Explanation
Measures Current cost of an additional QALY
(pounds sterling; 1985 prices)
Fitting a pacemaker 700
Hip replacement 750
Heart transplant 5000
Haemodialysis in hospital 14000
Source: Briggs A, Gray A. Using cost effectiveness information. BMJ 2000; 320: 246.
Brief definition
League tables rank medical treatments according to their relative
cost:utility ratios as a guide to resource-allocation decisions. In this way,
conclusions can be drawn about which healthcare services should be
given preference if resources are tight (or which procedures should be
removed from the programme of services).
l
• Cost:utility ratios (CUR)

• Used for allocating health resources with a limited budget
ICUR
Incremental Cost Utility Ratio
• ICUR = [Cost A – Cost B] / [QALY A – QALY B]
Exercise
• In your hospital in Cavite, you have been using chemo A as standard of
care. Chemo A prolongs life by 1 year but reduces the quality of life of
your patients due to its side effects. The new chemo B prolongs life by
1.5 years at estimated utility value of 0.5. Calculate the QALY for chemo
A and chemo B.
• Suppose a full course of treatment costs are as follows:
• $1,200 for chemo A
• $1,500 for chemo B
• Calculate ICUR.
• ICUR =
1500 – 1200
0.75 – 0.65
cost of chemo B – cost of chemo A
QALY chemo B – QALY chemo A
• ICUR =
= 3000 per QALY
On the average, it costs $3000 to add one year of perfect health onto the life of the patient if chemo B is used
DALY
• Disability-Adjusted Life-Years

• indicator developed to quantify the global burden of disease

• calculated by adjusting a standard life expectancy for loss of healthy life
resulting from disability and premature death

• scale: 0 = perfect health, 1 = death

• adjusts for time and age preferences
DALY
YLL
(years of life lost)=
N x L
where N is number of deaths, L
is standard life expectancy at
age of death
YLD
(years of life lived with
disability)=
I x DW x L
where I is incidence, DW is
disease weight (0 = perfect
health, 1= dead), L is average
disease duration
+
DALY
• Useful in:

• defining burden of disease

• evaluating interventions and programs

• setting health service and research priorities
Murthy et al. Disability Adjusted Life Years for Cancer Patients in India.
Asian Paci c Journal of Cancer Prevention, Vol 11, 2010 

.
DALY: criticisms
• Data used to estimate DALY are inadequate

• DALYs used standardized maximum life expectancies

• may not be true for the developing countries

• Preferential treatment: young adults > infants or elderly; present
generations > future ones

• Obscures too much information for resource allocation
Cost Utility AnalysisThe additional quality of life and years of life gained with two
alternative treatments
Explanation
Time
QALYs lost through B
QALYs won through B
Treatment A
Treatment B
Utility(e.g.QALYs)
being) are calculated. The most commonly used unit of utility is quality-
adjusted life-years (QALYs). The additional costs of a treatment are compared
with the utility gained as a result of the treatment (e.g. cost per QALY).
ICER
• ICER = [Cost A – Cost B] / [QALY A – QALY B]
Incremental Cost Effectiveness Ratio
Cost-effectiveness of Osimertinib in the First-Line
Treatment of Patients With EGFR-Mutated Advanced
Non–Small Cell Lung Cancer
Pedro N. Aguiar Jr, MD, MSc; Benjamin Haaland, PhD; Wungki Park, MD; Pui San Tan, MPharm, PhD;
Auro del Giglio, MD, PhD; Gilberto de Lima Lopes Jr, MD, FAMS, MBA
IMPORTANCE The survival of patients with advanced non–small cell lung cancer (NSCLC) with
epidermal growth factor receptor (EGFR) gene mutations has improved substantially in the
last decade with the development of targeted tyrosine kinase inhibitors (TKIs). Osimertinib, a
third-generation TKI that is approved by the US Food and Drug Administration for the
treatment of patients who develop EGFR T790M mutations, has recently shown improved
clinical outcomes compared with gefitinib and erlotinib for treatment-naive patients.
Supplemental content
Research
JAMA Oncology | Original Investigation
JAMA Oncology, 4(8), 1080. doi:10.1001/jamaoncol.2018.1395
Summary of results and findings
• Is osimertinib a cost-effective first-line therapy for treatment-naive, epidermal
growth factor receptor gene (EGFR)-mutated, non–small cell lung cancer?

• Results:

• Incremental QALY gained with osimertinib versus 1st and 2nd generation
TKIs: 0.594

• US: Incremental cost per QALY → $225,000; in Brazil, $172,000.

• Not cost-effective either in the US or Brazil

• cost-effective if cost is $12,5000 in US and %3000 in Brazil
Yes
Yes
Yes
Yes
Yes
Yes
Yes
No
Yes
Yes

Cost Utility Analysis

  • 1.
    Cost Utility Analysis Dr.Lance I. G. Catedral T: @lanceoncology E: [email protected]
  • 2.
    Mark 8:36, KJV “Forwhat shall it profit a man, if he shall gain the whole world, and lose his own soul?” A new chemo drug? A new screening modality? A new surgical technique? Physicians
  • 3.
    Cost Utility Analysis •special form of cost-effectiveness analysis • costs per unit of utility are calculated • compares cost and benefits, where benefits = number of life years saved, adjusted for loss of quality • anti-retrovirals vs polio vaccination • osimertinib vs afatinib as first line treatment for lung cancer • combines length and quality of life
  • 4.
    When is CUAused? • quality of life is the important outcome • the program affects both morbidity and mortality • the programs being compared have a wide range of outcomes • one of the programs being compared has already been evaluated using CUA
  • 5.
    • Useful forpolicy makers and reimbursement agencies • “Cost-effective if: • UK: £30K per QALY gained • USA: $50K per QALY gained • WHO: ICER < 3x per capita gross domestic product for a given country Adapted from: Mohammed, Pharmacoeconomics: Cost utility analysis. https://www.slideshare.net/mameclinical/ pharmacoeconomics-cost-utility-analysis?qid=082f5261-353f-4e89-8dcf-2b557e7719c1&v=&b=&from_search=1
  • 6.
    When NOT touse CUA • Only have intermediate outcome data • Effectiveness data show outcomes are equivalent • Effectiveness data show dominance
  • 7.
    Utility • “measure ofthe preference for, or desirability of, a specific level of health status or specific health outcome • not “usefulness” • also: “preference weights” • quantitative approach for describing preferences for quality of life • based on 0 to 1 scale • 0 = death • 1 = perfect health Measurable, quantifiable, and analyzable
  • 8.
  • 9.
    Standard gamble Example ofthe standard gamble Explanation As with the time trade-off method, utilities for states of health can be de- Complete healthAlternative 2: uncertain outcome Alternative 1: certain outcome Death Limited health 95% 5% 100% gamble on the alternatives, complete recovery or death. The probabilities between the two alternatives are varied until the patient is indifferent to both alternatives.
  • 10.
    “Imagine you havebreast cancer stage III. Suppose there is a surgery that can potentially cure your cancer, but there is a risk for death. How low does the probability of death have to be for you to be indifferent between having breast cancer stage III and the gamble of taking the surgery which can lead to death or cure?”
  • 11.
    Standard gamble Example ofthe standard gamble Explanation As with the time trade-off method, utilities for states of health can be de- Complete healthAlternative 2: uncertain outcome Alternative 1: certain outcome Death Limited health 95% 5% 100% gamble on the alternatives, complete recovery or death. The probabilities between the two alternatives are varied until the patient is indifferent to both alternatives. Alternative 1: Breast cancer Alternative 2: Breast cancer + surgery Limited health (living with cancer) 85% 15% Complete health (cancer remission or cure)
  • 12.
    Standard gamble • Goldstandard in utility elicitation • respondent must choose between two options • living with below-optimal health • a lottery between two uncertain health states • Limitations • time-consuming • cognitively difficult for many people
  • 13.
    Time trade-off The timetrade-off method Explanation Time trade-off is a method of deriving utility values that can be used in 1.0 Alternative 2 Time Alternative 1 hi 0 x t choose between a period that is spent in a specific state of health and a (shorter) period in perfect health, until they are indifferent to both peri- ods of time.
  • 14.
    “Imagine you havebreast cancer stage III and you have a life expectancy of 10 years. How much of your remaining life expectancy would you give up to eliminate breast cancer so you have perfect health?”
  • 15.
    The time trade-offmethod Explanation Time trade-off is a method of deriving utility values that can be used in 1.0 Alternative 2 Time Alternative 1 hi 0 x t choose between a period that is spent in a specific state of health and a (shorter) period in perfect health, until they are indifferent to both peri- ods of time. Cured of breast cancer stage III Breast cancer stage III 10 y
  • 16.
    Rating scale 0 Best possible state 50100 Worst possible state
  • 17.
    Rating scale • Doesnot involve trade offs • Simple, easy to administer • Ordinal, not an interval scale
  • 18.
    QALY weights Source ExamplesDisadvantages Literature Individual studies CUA databases Lack of comparability Indirect measures EQ-5D-5L EORTC QLQ-C30 FACT-G Only common diseases Direct measures Expert panel Special sample survey Expense Time Representation
  • 19.
  • 20.
    QALY • Quality-Adjusted Life-Years •number of years of perfect health • calculated by estimating the total life-years gained from a treatment and weighing each year with a quality-of-life score (from 0, representing worst health, to 1 or 100, representing best health) to reflect the quality of life in that year. •
  • 21.
    QALY’s advantage overCEA • combines more than one measure of effectiveness • mortality + morbidity in one single measure
  • 22.
    Determining QALYs usingthe example of dialysis and kidney transplants Explanation Initial situation Death Dialysis Kidney transplant Full health 1 0 Life expectancy (years) Qualityoflife(standardised) Area A: QALYs lost Area B: QALYs gained A B Surgery Lumpectomy + RT
  • 23.
    QALY • Dapat angyears of life, may quality! • Example: If a treatment prolongs the remainder of the patient’s life by an average of 3 years and the quality of life is 0.5 on a scale of 0 (= poorest state of health) to 1 (= best state of health) • The number of QALYs is 3 × 0.5 = 1.5, without discounting. Photo credit: Dr. RE King
  • 24.
    Exercise • In yourhospital in Cavite, you have been using chemo A as standard of care. Chemo A prolongs life by 1 year but reduces the quality of life of your patients due to its side effects. The new chemo B prolongs life by 1.5 years at estimated utility value of 0.5. Calculate the QALY for chemo A and chemo B. • Chemo B (standard) • Life expectancy = 1. 5 y • Utility = 0.5 • QALY = 1.5 x 0.5 = 0.6=5 years • Chemo A adds 0.75 QALY to the patient’s life • Chemo A (standard) • Life expectancy = 1 y • Utility = 1–0.35=0.65 • QALY = 1 x 0.65 = 0.65 years • Chemo A adds 0.65 QALY to the patient’s life
  • 25.
    League tables Extract froma league table Explanation Measures Current cost of an additional QALY (pounds sterling; 1985 prices) Fitting a pacemaker 700 Hip replacement 750 Heart transplant 5000 Haemodialysis in hospital 14000 Source: Briggs A, Gray A. Using cost effectiveness information. BMJ 2000; 320: 246. Brief definition League tables rank medical treatments according to their relative cost:utility ratios as a guide to resource-allocation decisions. In this way, conclusions can be drawn about which healthcare services should be given preference if resources are tight (or which procedures should be removed from the programme of services). l • Cost:utility ratios (CUR) • Used for allocating health resources with a limited budget
  • 26.
    ICUR Incremental Cost UtilityRatio • ICUR = [Cost A – Cost B] / [QALY A – QALY B]
  • 27.
    Exercise • In yourhospital in Cavite, you have been using chemo A as standard of care. Chemo A prolongs life by 1 year but reduces the quality of life of your patients due to its side effects. The new chemo B prolongs life by 1.5 years at estimated utility value of 0.5. Calculate the QALY for chemo A and chemo B. • Suppose a full course of treatment costs are as follows: • $1,200 for chemo A • $1,500 for chemo B • Calculate ICUR.
  • 28.
    • ICUR = 1500– 1200 0.75 – 0.65 cost of chemo B – cost of chemo A QALY chemo B – QALY chemo A • ICUR = = 3000 per QALY On the average, it costs $3000 to add one year of perfect health onto the life of the patient if chemo B is used
  • 29.
    DALY • Disability-Adjusted Life-Years •indicator developed to quantify the global burden of disease • calculated by adjusting a standard life expectancy for loss of healthy life resulting from disability and premature death • scale: 0 = perfect health, 1 = death • adjusts for time and age preferences
  • 30.
    DALY YLL (years of lifelost)= N x L where N is number of deaths, L is standard life expectancy at age of death YLD (years of life lived with disability)= I x DW x L where I is incidence, DW is disease weight (0 = perfect health, 1= dead), L is average disease duration +
  • 31.
    DALY • Useful in: •defining burden of disease • evaluating interventions and programs • setting health service and research priorities
  • 32.
    Murthy et al.Disability Adjusted Life Years for Cancer Patients in India. Asian Paci c Journal of Cancer Prevention, Vol 11, 2010 .
  • 33.
    DALY: criticisms • Dataused to estimate DALY are inadequate • DALYs used standardized maximum life expectancies • may not be true for the developing countries • Preferential treatment: young adults > infants or elderly; present generations > future ones • Obscures too much information for resource allocation
  • 34.
    Cost Utility AnalysisTheadditional quality of life and years of life gained with two alternative treatments Explanation Time QALYs lost through B QALYs won through B Treatment A Treatment B Utility(e.g.QALYs) being) are calculated. The most commonly used unit of utility is quality- adjusted life-years (QALYs). The additional costs of a treatment are compared with the utility gained as a result of the treatment (e.g. cost per QALY).
  • 35.
    ICER • ICER =[Cost A – Cost B] / [QALY A – QALY B] Incremental Cost Effectiveness Ratio
  • 36.
    Cost-effectiveness of Osimertinibin the First-Line Treatment of Patients With EGFR-Mutated Advanced Non–Small Cell Lung Cancer Pedro N. Aguiar Jr, MD, MSc; Benjamin Haaland, PhD; Wungki Park, MD; Pui San Tan, MPharm, PhD; Auro del Giglio, MD, PhD; Gilberto de Lima Lopes Jr, MD, FAMS, MBA IMPORTANCE The survival of patients with advanced non–small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) gene mutations has improved substantially in the last decade with the development of targeted tyrosine kinase inhibitors (TKIs). Osimertinib, a third-generation TKI that is approved by the US Food and Drug Administration for the treatment of patients who develop EGFR T790M mutations, has recently shown improved clinical outcomes compared with gefitinib and erlotinib for treatment-naive patients. Supplemental content Research JAMA Oncology | Original Investigation JAMA Oncology, 4(8), 1080. doi:10.1001/jamaoncol.2018.1395
  • 37.
    Summary of resultsand findings • Is osimertinib a cost-effective first-line therapy for treatment-naive, epidermal growth factor receptor gene (EGFR)-mutated, non–small cell lung cancer? • Results: • Incremental QALY gained with osimertinib versus 1st and 2nd generation TKIs: 0.594 • US: Incremental cost per QALY → $225,000; in Brazil, $172,000. • Not cost-effective either in the US or Brazil • cost-effective if cost is $12,5000 in US and %3000 in Brazil
  • 38.
  • 39.