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Pharmacoeconomics

A Primer for the Pharmaceutical Industry

Alan M o rriso n Albert 1. W e rth e im er

Ce n ter fo r Ph a rma c euti ca l H ea lth Se r vice s R esea r c h

T e mpl e U ni v er s it y Sc h oo l of Ph a r m ac y

© Temple Uni ve r s it y , 2 002 .

3

TABLE OF CONTE NTS

INTRODU C TION

D E FINITIONS OF PHARMACOECO N O M I CS

5

AN D O U TCOMES

RES EA RCH

 

6

T

Y PES OF PHARMACOECONOMIC

E V A L UA T IO N

7

 

C

OS T ANA L ySIS

 

8

 

Cos t of Car e

8

C

ost of Illne s s and Burd e n of I l l n e s s

 

9

 

C

O S T -OUT CO M ES ANALySIS

10

 

Cos t - Eff ec t i v e n es

s

J O

C

o st-U tilit y

1 2

C

os t- M i ni m i z at i o n

12

C

o s t-Be n e fi t

.

1 3

BASI C C ON C EPTS

 

14

PH A RMAC OE C ONOM I CS

1 4

 

D

e c i s i o n A n a l ys is

1 4

D

e f i ni t i o n o f Ca s e-E ffecti v e n e ss

1 6

D

e f i n i ti o ns o f Costs

1 8

P

e r s p ec t i ve s

2 1

Ti

m e H o ri z o n

22

Di

s c o untin g

23

M

OD E L I NG FRA M E WORKS

2 4

 

Step s i n D ec i s i o n Analy sis

24

I nflu e n ce D iag ram s

3 4

M a r k o v M ode l s

3 6

CLIN I C AL EPIDEMI O L O G y 5T A T I S TI CA L AN A Ly S I S UTIL I Ty

P

S Y C H O M ETR lC S

38

40

4 1

42

V A RIABLE S IN OUTCOMES AND PHARMA C O EC ONOMI CS

P A T I E NT OU T C O M ES

P

h y s i o l og i c al

4 6

. 4 6

46

4

5

Humanistic

M o rtality and M o r b i d it y

ECONOMIC OUTCOMES

No n-Mo netar y C osts

o n e tar y C os t s

M

DA T A SOURCES

Lit e rature Analvs i s

e ta -Analv s i s

M

4 6

4 8

48

4 9

4 9

49

5 0

52

C lini c al Studi e s

5 5

A

d ministra t iv e D ataba ses

5 6

F

inancial Data

59

AC C OUNTING FOR UNCERTAINTy

60

DEFINITION OF ERROR

60

 

PRINCIPAL

REDUCING

SOURCES OF ERROR IN CUNICAL STUDIES

61

ERROR IN CLINICAL STUDIES

62

S

e le c tion

B ias i n Subj ec t s En t e ri ng the Stud y

62

S

e l e c tion

Bias in S ubj e c t s af t e r Ent e ring the St u dy

6 5

PHARI\IACOECONOI\1IC STUDIES

6 5

Se

n s iti v it y An al vsi s

6

5

EV ALUA TING PHARMACOECONOMIC

STUDIES

68

REFERENCES

69

Introduction

The que s tion 'Doe s a medicine work ? ' i s a n s wered in a

controlled clini c al trial that is typicall y financed and de s i g n e d by a pharmac e utical company, carri e d out hy a contract research organiz a tion with the participation of members o f the health c a re professions and pati e nt volunteers , and watch e d over and v etted by an a g e n cy of th e Federal

governm e nt. The randomized controll e d

cornerstone of the pharmaceutical indu s try a nd the m ea n s by which s oci e ty d e termines whether dru g s s h o uld be m a rk e t e d to the public . The randomized co ntroll e d trial doe s n o t.

how e v e r, a n s w e r questions ab o ut th e e ffec t s of the dru g o n the health of th e population once th e drug i s marketed, n o r on the financi a l c on s equences to th e he a lth ca r e system of u s in g

the drug . Two rel a ted discipline s ha ve c om e into bein g

answer th es e qu es tions: outcom e s r e s e ar c h and pharm a coeconomics. Pharmacoeconomi cs , with whi c h thi s handbook i s primarily concerned , i s a s ub se t of outcom es research-that part that deal s s pecifi c ally with drug s a s oppo s ed to health care service s in g ener a l, and that in c lud es economic assessments. Pharm a coeconomics is a nexu s for ideas and methods from clinical epidemiology, economic s , decision analy s i s , psychometric s , stati s ti cs, and other discipline s . Thu s, what may app e ar to be s imple accountin g rests on a complex set of ideas , s ome o f which may b e standard pr a ctic e in one discipline but of questionabl e

c linical trial i s a

to

6

validity from the viewpoint of another. Pharmacoeconomics thus may be difficult for the non-specialist to understand and viewed as controversial. Nevertheless, pharmacoeconornic analysis has become a standard and sometimes required element in the assessment of drugs.

The purpose of this handbook is to make pharmacoeconomics accessible to the more general audience in the pharmaceutical industry by explaining the concepts involved in a concise form. This will help specialists communicate their work to others and enable generalists both to understand pharmacoeconomic studies and to assess their validity.

Definitions of Pharmacoeconomics and Outcomes Research

Pharrnacoeconomics arises from a fusion of pharmacy and economics. Economics is defined as "a social science concerned chiefly with description and analysis of the production, distribution, and consumption of goods and services." In pharmacoeconornics the 'goods and services' are pharmaceutical products and services, and it is the effects of their consumption that is the focus of interest. Thus, we

M e rn.un - Webstcrs

Collegiate Dictionary, Tenth Edition.

may define pharmacoeconomics as: a social science concerned with the description and analysis of the costs of pharmaceutical products and services and their impact on individuals, health care systems, and society. Pharmacoeconomics is a subset of health economics. which deals with health care services in general rather than being restricted specifically to pharmaceuticals.

Because pharmacoeconornics is a social science suh-tantially concerned with events in clinical practice, it overlaps with a

branch of medicine called outcomes research. O u tCl ) l 1 1 CS

research is the study of the clinical (e.g

economic, or humanistic (e.g., patient quality or life) end results (,outcomes') of providing health care services. Pharmacoeconornics is that subset of outcomes research that deals with pharmaceuticals and includes economic outcomes.

presence or disease').

7

Types of Pharmacoeconomic Evaluation

There are essentially two kinds of health economic .malvsis:

cost analysis and cost -outcomes (or cost -conseq Lienee) analysis. In cost analysis, only the costs of providing health care products or services are considered, without regard to the

outcomes

experienced by the patient or providers. In a cost-

outcomes

analysis, the endpoint of the analysis is a ratio of

8

th e costs of providing health c a re a nd a mea s ure of the

o utcomes of the care. Tabl e 1.

Th e m a in typ es of a naly s i s a re listed in

Table 1. Common Pharma coe c o n o mi c M e th o dologie s

M

e thod of Analysis

Co s t Mea s ure

Outcome Measure

Cost Analysis

 
 

Cost-of-Care

Currency

N/A

Cost-Outcomes

Analysis

 

Cost - Effecti veness

Currency

Natural units (e . g saved)

life-years

Cost-Utility

Currency

Quality-adjusted

life years or

 

other utility

 

Cost-Benefit

Currency

Currency

Cost - Minimization

Currency

Natural units or utility

N/A, not assessed.

COST ANALYSIS

Cost of Care

A c os t of care analysis i s a n e num e r a ti o n of th e he a lth care r eso urces consumed-in thi s c ase dru gs, ph a rm ac y se rvices,

e tc. - a nd

popul a tion o v e r a given time period . Th e o ut co m e s r es ultin g from the c a re a re not considered.

the dollar costs of providing ca r e to a g iv e n pa t i e nt

9

Cost of Illness and Burden of Illness

A cos t of illn ess analysis normall y f a ll s und e r th e um b r e ll a o f

out co m es r ese arch rather than of pharm acoeco n o mi cs.

c l ass ica l cos t of illness analysis, th e tot a l cos t th a t a p a rti c ul a r

di sease imp os e s on society is expre sse d as a s in g l e d o ll a r

a m o unt.

pr ov idin g c a r e for the illness ( in c ludin g dru g th er ap y) , th e

v alu e o f th e lo s t productivit y,

soc i e t y of premature death. Cla ss i c al cos t of illn e ss a n a l y s i s

h as m e tam o rphosed in recent ye ar s into th e burd e n o f illne ss

a n a ly s i s, which in essence is the s am e thin g exce pt t h at th e

e mphas is i s placed on the more tangibl e co mp o n e nt cos t s

rather th a n on a n aggregate dollar figure .

dir ec t medi ca l c osts of treating an illne ss, th e numb e r o f

Thu s, th e t o t a l

In a

In c lud e d in the calculation

mi g h t b e th e c os ts of

and the m o n e t a r y co s t t o

d

e ath s, ho s pit a liz a tions ,

lost work d a y s, et c . , a r e th e va ri a bl es

o

f inte r es t in a burden of illness analy s i s. Th e m os t in fa m o u s

mis u se of co s t of illness and burd e n of illn ess a n a l ys i s i s t o b e found in the opening paragraph of m a n y a m e di ca l eco n o mic

a rtic l e, wh e re future projections of th e s o c i e tal imp ac t o f th e

di sease in que s tion are deli vered for th e ir rh e t o ri ca l e ffec t .

10

COST-OUTCOMES ANALYSIS

The different methodologies for cost-outcomes

essentially

and outcomes:

expressed

analysis are

similar in that the endpoint

(Table

is a ratio of the costs

are

they differ in the way the outcomes

1).

Cost-Effectiveness

Cost-effectiveness analysis compares two (or more)

alternative treatments

monetary costs per unit of effectiveness. The unit of

effectiveness can be any 'natural" unit-e.g

lowering of LDL-C. major coronary

saved. or years of life saved.

year) and effectiveness

compared. Cost-effectiveness

among two or more treatment options. The definition of

for a given condition

in terms of their

percent

events. number of lives

and

The units of cost (currency

must be the same for the treatments

analysis

is used to decide

'cost -effecti

veness ' is discussed

in more detail below.

The Ejjicien t F roll tie r

The cost-effectiveness

but it may be more illuminating to present cost-effectiveness

data graphically as

.-.;110\\S a plot of the costs of treating hypercholesterolemia

ratio may be given as a single number,

Figure

1

a plot of costs versus effects.

11

with statins

treatment (expressed as percent reduction in LDL-C l. The points represent different statins and different dosuge«. The

line connecting those points representing the lowest cost at

any given effectiveness

Figure I. t1uvastatin and atorvastatin are the only two statins

at lesser and atorvastatin

on the efficient frontier-t1uvastatin

at greater effectiveness.

(i.e., the drug costs) versus

the effects

of statiu

describes the 'efficient frontier.'

In

4000 r--

(jJ

::)

~

u;

o

o

3000 r-

OJ 2000 L

;:>

o

ro :J

C

C

«

.

1000

 

P2.

"10

F20·

-- - -- --

F40

L-1C

••

L20

S10

P40

••

520

Al0

.s-w

L50

1\2(i

;".1C

o

20

40

60

Figure 1_ Annual Cost of Starin- versus

frontier'

Percent Reduction 111LDL-C

c·ne'."_

A.

Se,tltc"1 plll\

drugs and do.sagl's that lie' on the

using data of Hillcman et

'efficient

fluvasratin: L. lov.istatin: S. <imvast.uin: and P. prava-t.uin.

lete) .

al. II The line connects

of least cost fur any degree of cffedl\

.uorv :1S\atIlL 1-.

:\ I(). ,11,)l"\asl,111I\ lilll1~:

12

Cost-Utility

s ame w a y a s a cost-

e ff ec tiveness anal y si s exc ept th a t the unit o f e ffectiv eness is

qualit y -adjusted lif e y ear s (QAL Y s) o r a n o ther m e a s ur e of

utilit y. Consider th a t th e

pr o l o nged life but with a d eg re e o f di sabilit y, o r a r e duced pr o bability of disabilit y without prolongation of life . The

v a lu e or 'utility' that individu a l s or soci e ty pl a ce o n different

lif e outcomes can b e qu a ntifi e d u s in g a numb e r o f t e chniques.

S in ce the endpoint i s in p rac ti ce a l ways ex p ressed as co st per

qualit y- adju s ted lif e-ye ar save d , cost - utilit y a n a l ys i s c an, in prin c iple, be used t o co mp a r e n o t ju s t a lt e rn a tive th e rapi es for the sa m e diseas e but th e r a pi es fo r di f f e r e nt di seases, a nd r a nkin gs of the co s t-utiliti es ca n b e dr aw n up . Su c h rankings ca n be useful in sele c tin g p o li c i es wh e n , fo r e x a mple, a

go v e rnment wants t o c h oose a m o n g in s t a llin g hi g hway guard

r a il s, hiring addition a l f oo d in s p ec t o r s , o r v acc in a ting s e niors f o r flu.

A c o s t-utility analysi s i s perf o rm e d in th e

out co m e o f a tr ea tment m ay be a

Cost-Minimization

A

cos t-minimization a n a l ys i s i s a cost- e ff e ctiven ess analysis

in

the special case in which the e ff ec tivene ss o f th e

t r ea tments is the same. On ce th e e ff e ctiv e n ess (e xpre s sed in what e ver natural units a re a ppropri a t e) h as b ee n dete rmined t o b e e quivalent for th e a lt e rn a tiv e tr e atm e nt s, it i s n o t

13

con s idered further and the analysis focu s e s entir e ly o n th e

c o s t s, with the aim of determining whi c h tr ea tm e nt minimi zes

co s t s. A co s t-minimization analy s i s i s, in e ffec t. a cos t -o f -

c a r e a naly s i s in which alternative tre a tm e nt s a r e co mp a r e d . Unlike a tru e cos t-of-care anal y si s, how eve r , th e o ut co m e s are tak e n into a ccount and mu s t b e s h ow n t o b e e qui va l e nt .

Cost-Benefit

Lik e c o s t- e f f e c tiveness

co mp a r es the c o s t s and outcome s o f a lt er n a ti ve t h e r ap i es:

unlik e cos t- ef f ec ti v eness a naly s i s , h oweve r. t h e o ut co m es in

a cos t-b e n e fit anal y sis are expr esse d in m o n e t a r y t e r m s . Fo r exa mple, th e outcome of the tre a tment in qu est i o n i s firs t

e xpr ess ed in t e rm s of life-years save d o r qu a lit y-a dju s t e d li fe

analysis , co s t-b e n ef it a n a l ysis

y ea r s sa v e d, a nd this is then tran s l a t e d int o a n e q u i va l e n t

m o n e t a ry a m o unt - under the hum a n c a pit a l a ppr oac h , thi s

amo unt i s the present value of a per so n' s

produ c tivity. Since both the cost s a nd th e effec t s of th e

tr ea tme nt a r e expressed in the s am e ( m o n e t a r y) unit s, t h ey

ca n b e dir ec tly c ompared.

life tim e

Any co s t-b e n e fit r a ti o of l ess th a n

1 . 0 i s co s t - beneficial.

A ratio of 1 : 6 m ea n s th a t o n e r e ce i ves

$6 o f va lu e f o r $1 of investment.

15

Basic Concepts

PHARMACOECONOMICS

Decision Analysis

Deci s ion analy s is provides the basic framework

eff e ct: v e ness a nalysis,

ph.urnacoecon o mic analysi s. Decision analysi s is a

syst e matic. quantitative

value of one or more alternative s.

for co s t- type of

which is the most common

approach to assessin g the relative

,

Use new d.lllg

(I r s t andard C ale ';,

I

Use new' d11 1g.J

f

"

Live

l' Die

LIve

Use s t anderd Cale J

(

" ,

1 DIe

n :::

0.2

n "

• .

0

_'

. 5

<]

---

l·!.J.lr.,,~e

- ] [ · I· · d

.

!:';j

<] i·Jive

<. ]

.

['1 . ad

.!:' ••.

Fi ~ ur t' ~ . Hvpothcticu! D eci si o n

Tr e e. The tree consists of branches (lines) and

nodes ;\ dccrxinu 1" tri.myular.

n o d e ( s quare I. c han c e nodes (circular i, and terminal nodes

14

The basis of decision analysis

illustrates the components

chance, and terminal) and branches.

and branches connect a decision node with terminal

which represent the outcom e s

tree i s structured

begins with a decision

alternative

is the decision

2

tree: n o des (decision.

tre e. figure

of a decision

A series of chance

nodes

node s .

of inter e st

in the analysis.

' 2

The

from left to right.

The tree in Figure

node and two branches

i . e

representing

courses of action .

to u se either a new dru g or

standard care to treat diseas e X. Both c ourse s l e ad to a

chance node that diverges into branches repre se nting

possible outcome s

These branches end in terminal nodes. repres e nting

outcomes of interest

death . Chance node s identify point s at

possible events may occur.

predicted with certainty.

associated with a probability

this case. the probability

the new drug

probabilities must

chance node must exhaust

th e treatment the lif e o r

of survival

or d e ath followin g

i . e

in this decision an a lysis .

Which

which t \\0 (or more)

e vent will o c cur cannot

nod es are

In

be

and so th e chance

for each emergent branch.

of s urvival followin g

tre atm e nt with i s 0.2 ; these

exitin g the Following

is 0 . 8 and the probabilit y

of death

sum to unity and the branches

the possibl e

outcom es .

standard care, the probabilities

both 0.5.

of surviving

and dying ar e

In thi s explanatory drug is superior

surviving patient s .

example, to s tandard

it is easy to see that the new care in terms of the numb e r

of

Definition of Cost-Effectiveness

16

Decision trees such as the hypothetical example shown in Figure 2 are a basic step in cost-effectiveness analysis. Suppose that in the example shown in Figure 2 the cost of providing the new drug therapy to 100 patients was $1,000. This includes the cost of the new drug and the cost the physician's services for diagnosing the condition and prescribing the treatment. Since 80 of the 100 patients given the new drug lived. the cost-effectiveness ratio is $1,000

di vided by 80. or $12.S per life saved. This ratio is referred

to as the average cost-effectiveness

ratio.

The cost-effectiveness ratio of interest is not the average cost- effectiveness ratio but the incremental cost-effectiveness ratio of the new drug relative to standard care. Suppose that, in the example shown in Figure 2, the cost of providing standard care to 100 patients was $300. Standard care is thus less costly than the new drug, but also less effective. The incremental cost-effectiveness of the new drug relative to standard care is the difference in costs divided by the difference in effects.

C /E = G!-Cb

fu-8J

In this case, the difference in costs is $1000 minus $300, or $700. and the difference in effects is 80 minus SO lives, or 30

17

lives. The incremental cost-effectiveness divided by 30, or $23.33 per life saved.

The incremental cost-effecti veness, and not the average cost- effectiveness, is calculated because there is always an alternative to the new drug or whatever therapy is in question. Even if the alternative is literally to do nothing. there are associated costs and/ or effects. Suppose that, in the example we are discussing, if literally nothing is done to treat 100 patients with disease X, then 70 patients die and 30 spontaneously recover and survive. There are no associated medical costs because no treatment is provided. This is illustrated in Figure 3.

ratio is thus $700

Use new drug, standard care, or do nothini'

U se new dmg

r-----------<I

Live

J

I

1 Die

Live

Use standard care J

Do nothing

I

I

1 Dle

Live

J

I

I

1 [He

(I :3

0_]

(1.5

0.5

(1.3

(17

JA~

J De~

~A~

1 De~

~A~

~

De~

Figure 3. Hypothetical DeCISIOn Tree with Three Alternauves [\,,'I1:t the' ailcT1LlliI is literally to do nothing. the outcomes of interest accrue.

 

18

The incremental cost-effectiveness no treatment is:

of the new drug relative

to

($1,000

- $0)/(80 - 30) == $20.00

per life saved

Similarly, the incremental cost-effectiveness of standard therapy relative to no treatment is $IS.OO per life saved.

Relative to no treatment.

less effective but more cost-effective than the new drug.

the standard

therapy

for disease

Definitions of Costs

The definition" of some cost terms commonly

pharmacocconornics

are given in Table 2.

used in

Economists distinguish

average cost from marginal

cost.

EXAMPLE.

The average cost of detecting

a case of

X is

condition

Y is the total cost of all the screening

tests

performed

divided

by the number

of true positive

cases of

Y detected,

condition

85%, the cost of that program

of

If the screen only uncovers

7S% of cases of

this to

cost

Y and a program

the detection

is applied to increase

would be the marginal

increasing

rate by 10%,

In practice the marginal

average cost. The distinction between average and

incremental

cost is usually greater

than the

costs was discussed

above under the definition

of

Table 2. Definitions of Pharmacoeconornic

Cosh

19

Term

Definition

 

Average cost

Total cost divided

bv the number

of unrt , produced

 

Direct cost

The cost of the gc,ods providing a treatment

and

service

thai art' ue-ed In

Incremental

cost

The increased

cost of one

treatment

pr"t:r~lJn relative II'

 

an alternative

Indirect cost

The value

of

the product i . it) Ill" rcsultin"

Irorn :111

illness

 

Intangible cost

The value

of psychosocial

e1lech

,uch as p:l1n and

 

suffering.

 

l\larginal cost

Change

in total Cllst that results

trorn the production

(11

an additional

unit

Mortality cost

The

cost incurred

due to death

 

Opportunity

cost

The

value of all cpslS in an alternative

lIse

Overhead cost

The

coxt of pr()\'idin~ space. PU\\l'l. :ldlllll1i,tr:I\I\('

 

services, etc.

 

Production cost

The total amount something

of resources

used in producinu

 

Productivity

cost

Same as indirect

cost

cost-effectiveness,

of one health care program

incremental

former relates to treatment alternatives

to more of the same treatment.

The incremental

cost is the increased

The

cU:-,l

relative to an alternative.

cost differs from the marginal

cost in that the while the latter refers

The distinction between direct and indirect costs is

particularly relevant to cost-of-illness

analysis.

The direct

cost of an illness, such as asthma, to society is the

providing

the costs of medicines, physician visits, emergency room

visits. and hospitalizations

to society is the value of the productivity prevents people from working.

cost of

all of the health care services to treat it, including

due to asthma.

The indirect cost lost when asthma

Most direct costs considered in phannacoeconomc

are direct medical costs-the costs of physician visits.

analysis

20

hospitalizations.

laboratory tests. drugs. and medical supplies

and equipment.

Non-medical

costs include a variety of out-

of-pocket expenses. such as transportation facilities. special foods, etc.

to health care

Direct costs

= Direct medical COsts + Direct non-medical

costs

Indirect costs. i.e

when

while at work (presenteeism)-and

productivity

costs. arise from morbidity-

people miss work (absenteeism) or are less productive

mortality.

Indirect costs = Morbidity

costs + Mortality

costs

In computing total costs, researchers may include

costs.

not only the IntangiblC'

direct and indirect costs but also intangible

costs include the value placed on pain and suffering.

2 1

T o ta l costs

= Direct cost s + I nd i r ec t c '( hh t · Il 1t : l l 1 ! Ci h k L ' ( l . 'h

Perspectives

Since costs are seen differently

the perspective of any pharmacocconomic

explicitly stated. health care providers

Furthermore. the organization

therefore the appropriate cost perspective. \ urie-. from

country to country and within countries such as the United

States that have mixed systems.

cost-effectiveness

the program.

from different

point« of vie'\\'.

,is

must be

and

~lllaly

Society. health care insurers (payers!.

all have different

perspect: ve s on Cosh.

and

of health care financing.

The usual pl'l\r)t~\.:ti\es III

'\\

.md those ul

anatvsis

arc those of S(h:ii

EXAMPLE.

is hospitalized following an acute myocardial

that subsequently the science writer's cost of hospitalization

hospital under the terms of the health plan. From the

perspective

true cost of providing

A science writer with c o r o n a r x

heart disease

infarction

of

proves fatal. From the perspective

health care insurer (the pay e r). the

is the amount

or money paid to the

the cost is the

From the

of the provider (the hospital).

the hospital s e r v i c es .

perspective

of the science writer's employer.

which (ill

this fictional

example) entirely subsidizes its employees'

health plans. the cost is that part of the iI1S11!dllCe premium

designed to cover coronary

pillS the i ndircc:

heart disease.

22

costs. i.e

science writer was incapacitated, replacement.

the cost of the productivity

lost while the

and the cost of hiring a

Time Horizon

The term 'time horizon' during which the outcomes

The time horizon could be expressed

years (or months or weeks) or relative to study variables

patients' lifetimes,

enrollee! in a clinical trial). More precisely.

is :1 point in the future up to which all costs and effects must

bc accounted for and bevond which evervthinz ignored.

is used to specify a period of time

of an analysis will be considered.

as a fixed number of

(e.g.,

or the amount of time that patients were

o

the time horizon

-'

L

can be

EXAMPLE. A cost-effectiveness

analysis of the ACE

inhibitor

enalapril

based on data from the SOL VD trials

considered

two time horizons: a within-trial

horizon, and

a patients' lifetimes' horizon,6 The within-trial

referred to the actual time period of the SOL YO trial.

Because of staggered enrollment

trial, the durations of enrollment

horizon

and deaths during the varied up to the

maximum

duration of the trial (about five years),

The

within-trial

analysis relied on observed

data of the

effectiveness

of enalapril (cost data were inferred),

The

23

lifetime analysis was a projection future.

of events into the

Discounting

If the time horizon of a pharrnacoeconomic

weeks or months, no adjustment for changes in costs over

time is required.

however,

be brought to same reference time point.

The value of a dollar todav is not what it \\;h

years ago. For example.

expressed

of the analysis) by increasing

inflation rate for pharmaceuticals,

analvsis is several

If the time horizon

is several Yl'ars.

at different

then costs that are incurred

time : mu-t

''--:1\. 1\\Cnt\

.

.

the cost of a drug in ll)~) muxt he'

year (or the rdCTl'l1CC

in dollars for the current

y\.'~tr

of a

it according to the annual Conversely. the value

dollar twenty years from now will be less than it:-;present

value. In order to bring future costs to the same frame of reference as present costs, they must be discounted.

EXAMPLE. The cost of a medical

medical service will be utilized

is its present value? into the future is:

service i:-; ')1.0()O, The

five veal'S from now: what

prices

The formula for discounting

Cll

Cprl'SCll!- (1 + r )n

24

25

where cpresentis the current cost, n is the number of years, c, is

EXAMPLE.

National

survey data for 1994 indicate

that

the cost

n years from now, and r is the discount

rate. If the

49% of all pregnancies were unintended:

54(7(, of the

discount

rate is 5 % per annum,

the present cost of the medical

unintended pregnancies ended in abortion. lo

About

half

service I S:

of the women who unintentionally

became pregnant had

Cpr CS C I l l =

I ,(XX) (1 + 0.05)" = $784

MODELING FRAMEWORKS

Steps in Decision Analysis

While the process may be broken down in a number of

different

ways. we will follow previous

authors and describe

a decision

analysis

in terms of five st e p s . i '

I .

Identify and bound the problem

2.

Construct a decision tree

3 .

Collect the information tree

to fill the decision

4. the decision

Analyze

tree

5. a sensitivity analysis

Conduct

been using a regular method of contraception.

Emergency contraception

within 72 hours of unprotected

consequences of a decision whether or not to use

can prevent pregnancy if taken

sex. We can explore

the

emergency

contraception

using decision analysis.

If

emergency

contraception

is used. the probability

of

pregnancy is reduced (but not eliminated).

does occur, a predictable to terminate the pregnancy.

their pregnancies will miscarry. For the sak e of

simplicity,

the Lise of emergency contraception. such as ectopic pregnancies.

we shall ignore the effects

I r pregnancy

proportion

of women

will chose

Some women who continue

of nausea following and complications

I . Id e nti fy a nd bound th e pr ob l e m

The first step in a decision

alternative courses of action.

the decision

following unprotected sex. The consequences

decision

In the example

analysis

is to identify

the

we are using. contraception

of this

is whether

or not to use emergency

that interest us are the numbers

of unwanted

pregnancies,

or more specifically

the number of pregnancy

terminations

and live births that would he avoided

through

26

27

Lise of emergency contraception. The endpoints of the

analysis, therefore, are pregnancy terminations

births.

unprotected sex and its unintended consequences, months. The perspective is that of society.

and live

The time horizon will be limited to an episode of

i.e., nine

' l . C o nstruct a d ec i s io n t r e e

of a decision tree, makes the description

of the problem and its elements

the decision node and branches representing the alternative courses of action. Here, the decision is to use O [ not to use emergency contraception following unprotected sex (Figure

-1-).

Step 2, construction

explicit.

The tree begins with

 

Use Eep

U nprotected sex

 

I

 

Do not use EC:P

F'ilurc 4. Partial dc'chlnn tree wilh decision node. The decision is whether or not to l I " , ' l'n1L'ri,'cnc\ c{ln!r;llcplioll i'l)lIowing unprotected sex. [CPo cmer)lL'nc)

' : \ l Ill1acl']JII vc 1''' I

Following

pregnancy

these alternative outcomes from the decision

the use (or not) of emergency contraception,

mayor

may not occur.

A chance node reflecting

is added to each branch emanating

5).

node (Figure

I

U nprotected sex

I

Use Eep

I.

Plegn;:U:lt

J

J

-1 Not pl'e:?~'l;mt

 

Pregn;mt

Do not use ECP J

"1

i . l

Not pl'epmLt

Figure 5. Partial Decision TrL": with Chance Nudes. likelihood of pregnancy following unprotected s n .

Chance

I Wc l L ' ., reflect the

If pregnancy occurs, some women opt for termination

and

others to continue their pregnancy

individual

the perspective

measurable

the other. This proportion

of women

to term.

While for an

woman this is a decision

proportion

that must be made, from

of women,

a

chose one option over

to the

of an observer of a population

will

might vary according

co mposition

T h e node bran c hin g

c

of th e pop ul at i o n

o f wo m e n

t o e ith e r p r eg n a n cy

o ntinuation

is thu s a c h a nce n o d e .

a n d o th e r factors.

t

e r mi nat i o n

or

A c e rt a in pr o p o rti o n un de r go s p o nt a n eo u s

o f w o m e n co n t inuin g

th e ir p r e g n a n c i es

a

b o r t i o n :

t hi s i s a l so r e f l ect e d

in a

28

c

h a n ce n o d e. Th e bra n c h es n o w in th e m od e l l e a d t o the

e

n d p o ints th a t wer e dec i ded o n in S t e p I - p reg n a nc y

e r mination,

t

c

n

we id e ntifi e d

r e ating

liv e bi r th , a n d n o p r e g n a n cy .

tr ee , t h e r efo r e ,

T h e l ast s tep in

the deci s i o n

i s t o a dd t h e t e rminal

t h e pr o blem

o d es (Figure

6). T h e d e c i s i o n

an d bou n ded

tr ee d esc r i bing

in St e p I i s n o w c o mp l e t e .

"

':.t_I_IJ.;,~~~~d

I

['

I

!

:

~

tic< E· . :

r

" I ' j !;" Ed '

r h'""",~,,r---------

Induced a b c ru o n

Sf~::'=

!

I

f Conunue pregnancv .~ ~ : :ut

- -

F ' t~ g ! ~v : ~_

I ~ I ~€ :n l \ 1\t

-- - --- - -

71tgll-!int

I----"

~ I

lu·1Ul .d ebortror

,----.--

I

------

, : ;p o n l a r~ o " s I t. o n n n u e p r e V' " '' ' '

--------

, I , "

--

' C

-

r - - ' --' .-- . : ~ ~

i

L

o n ' " , ' ",

p r e ; ; n " " " ;!

1 - - NOI pregTlanl

--

T e r m i n an cn

N pI>::?:nal\cy

:,

Lrve bu-l.

N

o pregnancy

Te IT:1.ll1.3.tIon

N,) pp-gnoirl.CY

Lrve buth

1< 0p " g w .r u:y

I:igure h. Complete Decision Tree. Complete

"1l1ergenc\ cou na cc pu o n following unprotected sex

tree: for the decision of whether to use

~

3. Co ll e ct th e i nformation

t o fil l th e d ec i sion t r c«

In t h e c ase of th e decis ion

s o u g h t i s a pr o bability

proba bility es tim a tes a r e di s pl a y ed

t

tre e i n F i g ur e

6. t h e i nf o rmat i o n

n

o d e.

Th e

va lu e fo r e ac h c h a n ce

b e n e a th t h e b r an c h es

7.

h e de c i s i o n

tree , as s een in F i g u r e

of

29

n p w t tct~ d : ;f . : ~

Use ECP

r

'

rr~\:illt

1! (I !~;

lnduced

.~b l : I I l l l : ' : 1

.::-Ir'

C o n u n u e iJI':g1\."1r~:y

. ; ~ 4

. ; p ' ~ ; ~ . : ' ': : \ i ' .· ' I : ' ;; ~ , : < , ! ' ,l ( ! ,

-

-.

. - - - ---

~, ;,;.:.

~ : ' : ~! ~ tUd J ' " p l ~ ~ > l l l l : : ~ '

I

~ N ot pre-gni.!.Itt

!

0 9J1

r - !! .'

~

l I [0M' de I e,,::::~, L'" '" : ; , " " . ~~~ :;~

eM

; ~ l J : c: . , ~ "

. " . ' P, t ;; ! l . : " ,, " J

'1 .

1 :' ' , : ,' } l ~ :: :

H ) ) · :. · , ' .1 . , , : . ' " ' . ;

! - ) ; ' , ~' ;, t ,,!

!,,"

' . r

L:-'."I;' 1:,u1i":

LN:~I'!"~_='

 

.

:':

 

(

1 9 3 1 2 5

!

':,: , p J " - P t < l l l ' : '/

Figure 7.

Decision T re e with Probabilitie

s. The decisi

n

tree sht",n

II I th'_' 1'1'," \1' 1: '

figure \ Figure h.l with probabilitie« added.

30

In Figure 7, the probabilities of conception with and without

emergency

contraception

are taken from a clinical trial of

emergency

contraception

versus a control group without

emergency

contraception.

The probabilities of induced and

spontaneous abortion are obtained from state statistics." more extensive description of information sources is given

under 'Data Sources.')

(A

4. Analv;« the decision tree

We will analyze the tree by calculating

reaching the outcome represented by each of the terminal nodes. This is done by tracing the branches from each

terminal node backwards

probabilities

produce the probability of the outcome. These calculations

can be performed using a spreadsheet.

row for each terminal node and a column for each chance node plus a column for the calculated probability of the outcome.

the probability of

to the beginning

of the tree: the

along these branches are multiplied

together to

has a

The spreadsheet

The spreadsheet corresponding

Figure 7 is shown in Table 3. The probability of an induced abortion if emergency contraception is used (corresponding

to the top row of Table 3 and the uppermost branch line of

to the decision analysis ll1

Figure 7) is 0.075 x 0.46 = 0.0086.

cells in the table where a particular

(Note that there are blank chance node does not

"U

8 5 ~

::0

e

co

q c ,<

::.,:J

~

(/)

g~~

p

g. § ~

-

J

(l)

CL

(D

~

00

;l' ~

~

v; ~.

c

-l::O

~--,

.:t;' g

-;

2:.~

~ ~

:;'

-::0

::i

"""'

""Tj

c::

rro

C

J; ri

::r-

n

'f.

r

::

]

r.

(D

("101

-- -

r-,

::::

v

C

,

rt

/.

JC/)

~5

;::;.0

2

o

rn

!)

C)8

'-0

ex:;

-

t-0

'Jl

2

:;

CT1

C)

0

"--'

x'

--J

',J!

,-,'

-

'J)

~

---J

--

-1

2

c

rn

!)

C

8

ex --'

-.J

fJ\

'-'

-

1Jl.

.j

-

t,-->

Jj

2

0

rn

C)

8

---' 'x;

--J

'J!

-

~

~

0'.

tr

rj

m

en :-r

,'-'" ,---

--

~

_

_

---.-.-----:::

:::

--.J

'JI

'Jl

-c

:=.:

tv

'JI

~

-

,::;,

.j

~

::;,

.j

_

:::

--J

'JI

-

;.:.

:J'

-

-

-

]

j

-

1

1

=

;:s

g'

,

w

'1;:;3TO

:.;

,::I

i r:

~

I[

I

II

;:;

'"

~

.;

g-

'"'

"".

g''

:J

~

'-'

!2

12

i::t

I::

'f

;;

~

~'~'

o

.,

0

-'0'

::0

8 18 8 0

8

~

N

~

8

0

WOW

-

~

W

]8

N

~'

0

o

,

.",

(l)

r:ro VJ

:::;

;::;.

(l)

:J'

~

-

::0

C)

0

"""+-,

::0 v

()

'< fD

.')'0

,

CT1s

no

;::'

v

.

~

::0 (l)

:

~

n

tv:

o

,

,

:j

~

'-<'.

::

::

'J;

C~~C:2;:J

7'

~

::::

~

c

~

(;;~

JQ

:::

:::;

-'

~

@

(To

:::

:::; ::

~

~-<'

c.

~

r~

)~

2:

in

I

I

,

[

5

'Y

5" '<

r;

:::;

8

0

.j

~

3

~

~

g

o

.

I

I

1'8 8 8 ::: C

3

3

-

~

8

\:0

'J.,J

2

X

-::J".

-i

::

;;

r,

::J

co

::0

~ ~ §

0'.

p

'-J)

--J

o:

V1

\2

,

21 ~~ ~ ~

C

-

-'

I~

I~

Ie

("';;

JQ

-

~

l~'

[ o

:::

~

("i)

C2.

f"D

nQ

r:

re,

r~.!

n

<

~

1

'"0

2-

2.-

§

~

-:

'7

G

'JJ

»

(--<

J

J.

~

,

H

(l;;

,

,

r:

J:

o

-'

,-

'J:

G

M

:3

r;

iv: r,

r-

'<

r-.

~

G

?

31

Iifili5'Kii35!ft3'1

32

33

occur along

the branch line.) The outcome

for the second and

5. Co ndu c t a sensitivitv analvsi s

 

.

.

fourth rows

of Table 3 is the same ('No pregnancy')

and the

probabilities are added together: the probability of no

pregnancy if emergency contraception

0 . 98 1 3 = 0.9836. Similarly,

pregnancy" if emergency contraception

+ 0 . 9 250 = 0.9 3 4 3. The probability of an unwanted

pregnancy (sum of pregnancy

births) is 0.0164 if emergency

(U)657 if it is not

is used is 0 . 00 2 3 +

the probability

is

terminations

contraception

of 'No not used is: 0.009 3

and unplanned is usee! and

(Table 3).

The consequences.

unplanned

emergency' contraception

in terms of induced abortions

and

births. of the decision

to LIse or not to use

for a hypothetical

population of

10 . 000 women are shown in Table 4 . The use o f emergency

contraception

v ould prevent

2 5 9 induced

abortions

and 234

unplanned

births per 10 . oon women wh o had had unprotected

sex.

Table 4. Outcomes

Contraception

of Decision to Use Emergency

per 10,000 Women

Induced Abortions

EC

86

:\0 EC

. \4 5

D

iff e rence

--259

E

C . e m e r ge n c y c ont ra ce p t io n

Unplanned Births

78

."112

2) +

Any measurement

estimate and an indication

descriptive

standard deviation, or 95'1t confidence interval rnav be

provided.

a point estimate

prevented by emergency contraception.

should be expressed

in terms of a point For instance.

and range.

of its reliability.

in

statistics

a mean (point estimate)

The decision analysis described

of the number

"bu\"l' has yielded pregn.mcies

o f

of unintended

Th e r e li a bili t y

such a point estimate

(usually) large number of probabilities

A point estimate

model, but of course there is a range of likcl , values for each

is made difficult

to calculate

in v o l ve d

by the

in the m o d e l .

in the

was us e d for each o f the probabilities

of the probabilities.

Sensitivity

analysis determines

the effect

on the result of varying

the probability

estimates

through th-

range of their possible or likely values. In a (111e\\ ay

sensitivity analysis. the probabilities

the decision

a time. This process determines

to changes in the assumptions the most critical assumptions

at eac h c h an ce

node in

tree are varied across their range of values one at

the scnsitivirv

of the results

in the moue! ~lnu can identi fy

in the model.

i .c., those that

have the

greatest

effect on the results.

The following

is an

example of one-way sensitivity analysis.

Two-way and

three-way and other forms of sensitivity analysis are

discussed below.

EXAMPLE.

emergency contraception,

In our decision

analysis

of the use of

of the

the point estimate

35

34

probability of spontaneous

of values for this probability is 0.17-0.29. 15

the decision tree shows that changing the probability of

spontaneous abortion does not affect the number of induced abortions but does affect the number of live

births. Substituting

and then the lower limit estimate (0.17) for the value 0.23

used in the initial calculation,

in the number of unplanned births (without emergency contraception minus with emergency contraception)

varies between 2 1 6 and 252 (the point estimate was 234).

The spontaneous

abortion

was 0.23. The range

Inspection of

first the upper limit estimate (0.29)

we find that the difference

abortion rate, thus. does not critically

affect the reduction in the number of unplanned births attributable to the use of emergency contraception.

Influence Diagrams

It is sometimes constructing

makes specific the decision to be taken, the outcome of

interest, and the chance clements that influence the outcome. Figure 8 shows an influence diagram corresponding to the

decision

usefu I to draw an influence

diagram before

a detailed decision

tree. An influence diagram

tree in Figure 7. The only outcome of interest in

Figure 8 is live births following unprotected

abortion was also an endpoint

in Figure 8 J. The

occurrences of pregnancy, induced abortion. and spontaneous

sex (induced

in the decision analysis shown

outcome is affected by the chance

abortion.

The decision, chance elements.

and outcome

are

presented

as a square, circles,

and a lozenge

shape.

respectively.

EC P

Figure 8. Influence Diagram.

Diagram representing

I nduced abort 1 0 1 1

the CkCl,l \)1 1

c l c r n c nt

and c l i: l 11 cl '

clements influencing

the outcome

of unwanted birth

rep. e l 1l c r ~e n ( \

c\ l[ l l r a c 'c l ' t i ll'

pill

36

Markov Models

The decision analysis

shown in Figure 7 represents

a single.

linear chain of C\l'nts tran"piring

SOl1ll' disl'asL's, however. progrl'ss gradually

Years. whil e the ri x k o f the outcome

coronary death. increases

appropriate for such problems,

Markov analyses

simple decision analysis.

problem

an influence diagram,

representing. the progression

over , I single time period,

over a period of for instance.

o f interest.

with age,

\1<lrKO\ analysi • is

similar

u se tree diagrams

to those used ill

of the

similar

diagram

heart failure,

to

The

However, the elements

diagram

arc first mapped out in a Markov

Figure l) shows a Marko:

or congestive

M

a rk r »

model consists

o f states (o val s) and transitions

(aITO\\S),

heart failure.

failure.

represented

III Figure <J. there are four stales:

late stage heart failure,

well, early stage

and dead from heart

where early and late stage heart failure

by New York Heart Association

Time i~ broken

are (N'{HA) class d o wn into a

I

l l! and IIIIIV, respectively,

series of sequential

p e ri o d s or cycles:

individual

must be in one of the four

within each cycle, an states; transitions

between

the states occur at the end of each cycle,

Individuals

in the 'well" state can transition

C L IS S states or remain

dlT( ) \ \ e.\iting

Similarlv. individuals

that state. progre."s into the NYHA-IfI/I\/

into either of the NYHA

b y an

in the well state (represented

from and circling

buck into the well state),

stall' (:111 remain

in the N't'H,-\-UII

in

state. or enter the

'dead'

state at the end of each cycle.

Needless

to ~~ l y ,

37

individuals

cannot

exit the dead state.

Probubiluic-.

must

hI.'

assigned for each transition,

Since time is modeled

Cis ,1

series of cycles

different

of equal length.

thl' probabilitie-,

at each c y cle. so that th c , c a n h e 111'Ii

can h l ' kd"jWIH.il'nt

()11

the age of individual-

entering

the m o d e l .

l\brK()\

IllUdl'ls i ll

which probabilities

arc time dependent

ar l ' l ,~ t1 k d \LtrK(I\

process models,

~

~

"Y1L·\·I,1I

NYH : \ . II I ' I V ~ '

Q

< ,

~

C - ~ ~"

» >:

( " i: l , : r < l111h : l s, ' J O i l Stl l l ! \ h ; x a u H ou t

\ 1 i : I I T h ,', ' 1 ! combined. ' \ ,s l , , ' I , ll i l l l l , · l ; h S

as h:I\L' 1 \YIL\

F i p l r c I ) . : ' \ l a rk\ )\ Tr.msiuon

1,,1' hc.ut

Stal e Di a; ,: r : 1 I l 11I > II ':! l i " l I l . ' 1 1 1 11I k1,l i I liulur.

c I , d :-

[ ' , ' I ,il l l l ' l k i l \

e Ll ss, ' , III :llld 1 \ '

l.ulur , '

\ I. I I ~ '"

\ Y I 1 I . · 1 : 1 " 1 . " 1 : 1 1 1 < 11 1

0 1 ' 1 1 York ll e' , l: 1

j\;Y II . \.

38

CLINICAL EPIDE MIOLOGY

Pharmacocconomic and cff e cti veuess. epidemiological

every medical research t ' C\\ fundamental mutual"

studies usually require data both on costs The effecti veness data arc taken from

The design of

to a

or medical research studies.

study can be classified according

exclusive dichotomies (see Table

.5 l.

Table 5. Concepts

l - '

in Cliniral Study Design

S r u d v D e ~ i ( r! l

.

h

I n\- i.'ql~ J t l

In'

I

I: \" . :

I I "

~ I ~ r :

l

i m«

I \T ' ; '\ . ·(i l\ · ~ ·

T : ~ ~ L · :- J : : ~ l ' l il j

!

i

!

1 -

I

l

~

I :

! ) r l )' I H. : C l l \ ' l '

1.( Jfl:

c ~.(''''

,l.l!

lI\

\[r:~d

I

"~'~.!

" ]1 .

I n .i j

q "

Ii :,\:

I \ Ltr . "p ~ · \ · t i \ t ·

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.1; 1.L:il"1.l\.hn"~

First. a study may be either observational or experimental.

clinical trial is an experiment-a

which the investigator interferes with the normal course of

l'\l'Jlts. usually by providing

they would nut otherwise have received. In an observational

test of an intervention-s-in

certain people with a treatment

.A

39

study. the investigator

interfere with them. There cannot be. therefore. :111

'observational

merely observes

Second.

events al1d dues 110t

studied m:ly is :1 in which prospcct ivc l y

trial'

the events heing

have alreudv occurred.

retrospective

case they will be studied as they happen.

An observational study may be either rctroxpcctiv« ()1'

prospective hut an experimental study can only be

prospective-unless.

machine in which he visits the past to perform his

intervention

results. Third. the observations and are called cross-sectional.

time. and are then called longitudinal.

study may be longitudinal or cross-sectional

may be either prospective or retrospective. study, however, Gill only be longitudinal

brief in duration

in which case the study

.

.

one. or may nut yet h:I\'C occurred.

i.c

that is. the investigator

to the present

possesses

l o colk\'[

a time

the

before returning

ma~ refer to a poin: in time. or to two or more points in

All observational

and in both case'> An experimeutal because. however of CIU S C and

it might be. it is an analysis

effect-the

'cause' being the intervention

being tested.

and

the 'effect'

being the clinical outcome

being ohsenl'd-

which cannot by definition

Thus. a clinical trial is a prospective, longitudinal.

experimental study.

occur at the same point ill time.

STATISTI C AL

C

l u- . x i ca l statislics

A N ALYSI S

is based o n hypothesis

testing.

The

40

hypothesi"

is made that the observations

to be cxpluincd

are

the result purc l. of chancc-e-thix

is the null hypothesis.

A

calculation

is then made of the probahility

that the

observations

would arise under the null hypothesis

and. if th.u

probability

is b e l o w an arbitrary

threshold

(1110-;t often

I in

2(). or O.(5). ihc null hypothesis

is rejected.

The results

cannot be explained

purely by chance

and

are said to he

"-;uti-;tic;tlly significant."

\ o t L' that the above procedure

chance

explores

the role of random

and in it-elf docs nothing

to assl'S-; the role of

,,~ sicmatic error {discussed

belowi.

which is often more

important than random error.

Hypothesis

testing

is not

parucularly useful in decision

analysis.

where we need to

KIlO\\ the prohahility

of a certain

event occurring

(such as

death from a myocardial

infarction)

under a certain

set of

,-:lrCLlmst~lI1cl'" 1 such as when a patient has already had one

heart auack l. The calculation

of such conditional

probabilities

i " referred

to as Bayesian

analyxis.

To the non-

preconditioned

mind. the Bayesian

approach

intuitive, if k"" conceptually

sophisticated,

may be more than hypothesi"

t l ' - ;till g .

UTILITY

41

The effectiveness

of many medical

C;\I1 he

expressed

in terms of prolongation

treatments of life. c.g

: \ S the

(average)

number

of years of life s(\\l'd.

SUIl1\' trc.umcntx.

owe v e r. may prevent without actually extending

extend

of life. These

placing

The utility of normal

utility of not being alive is set at 0: a <tate of rcduc.d

has a value between

by the number

order to arrive at the number (QALYs).

reduces the quality

h

a wors e nin g ill the quality

it. Similarly.;\

tll life

may

trc.umcm

life hut with the presence

a value on the quality

health

of significant

sitLutioll"

disahility

that

; 1 1 \ ' dc:alt with h , if ' . ; u i i li i .

of life. i.c

is given a value of I. n . while the

health

(l') is m u ltipli ed

in

() and 1.0. This utility

of years of life ( Y ) saved b y till' trc.nment

of quality-adjusted

life' years

QALY

= Y x li

A related concept is the disability-adjusted

The DALY

and injury on societies Studyl7) and represents of life due to disease.

life year I. D""\L Y:

t)f disease

of Discasl'

was developed

to quantify

the burden

Burden

(as in the Global the reduction

weighted

in the 1111111hl'lof years

(l r lif due i n

hy the quality

the presence

of the disease.

Utilitv values

fur QALY

culcul.u ions call he f ( lli nd II I th«

medical literature.

A detailed description

of' 11\1\1,ulility IS

42

determined

i~ beyond

the scope of this primer.

There arc

cxxcntially

two way».

First, one can determine

people." s

p

r e f e ren ces

I o r different

health states by m e th ods

based on

decision analysis using techniques

~amble or the time trade-off.

particular

<calc-.

such as the standard

Secondly,

the utility o f a using psychometric

- r . u c o f health can be estimated

PSYCHOMETRICS