health technology assessment -...

HEALTH TECHNOLOGY ASSESSMENT

Edmund Jessop

www.eurordis.org

¨Barcelona June 2010

2 Health technology assessment

Session outline

•Assessing benefit

•Assessing cost

•Assessing evidence

3

COSTS


4

Costs

•Exercise – driving to Paris

TITRE DE LA PRESENTATION (A MODIFIER SUR LE MASQUE)

5

Cost types

•Unit and marginal costs

•Incremental costs

•Opportunity costs

•Tangible and intangible costs


6

BENEFITS


7

Benefits

•Survival

•Quality of life


8

Assessing benefit

•Treatment X cures a fatal disease of

childhood

•Costs £100 000

•Extends life by 50 years


9

Assessing benefit

•Treatment Y improves survival in a

fatal cancer

•Costs £10 000

•Extends life by 1 year


10

Assessing benefit

•Treatment Y

•£10 000

•1 extra year of life

•Treatment Z

•£10 000

•1 extra year of life

BUT...

•Patient too ill to get

out of bed


11

Assessing benefit

•Mobility

•Self Care – wash and dress

•Usual activities

•Pain

•Anxious or depressed


12

Health state A

•No problem with mobility

•No problem with self care

•Some problems with usual activities

•Extreme pain

•Moderately depressed


13

Health state B

•Some problems walking about

•Unable to wash or dress


•No pain

•Not anxious or depressed


14

Health state C

•Some problems walking about

•No problems washing or dressing

•Unable to do usual activities

•No pain

•Moderately depressed

TITRE DE LA PRESENTATION (A MODIFIER SUR LE MASQUE)

15

Health state D

•No problems walking about

•No problems washing or dressing


•No pain

•Not anxious or depressed


16

Improvement in health state

•Health state before:

•Extreme pain

•Moderately

depressed

•Some problem with

usual activity

•Health state after:

•No pain

•No depression

•Some problem with

usual activity


17

Health gain

• 20 years in health state without treatment = 20 x

• 20 years in health state with treatment = 20 x

•Gain =


18

Growth hormone

• Extra height as adult: valuation = 0.1

• Adult life span = 50 years

5 QALY gain

• Cost = £10 000

• £2 000 per QALY – cost is justified


19

A note on ethics

•Utilitarianism

•Other ethics


20

STRENGTH OF EVIDENCE


21

Evidence

•Exercise – 5 scenarios


22

Assessing evidence

Stories

Historic comparison

Head-to-head


23

Assessing evidence

•Anecdote, case series

•Placebo effects


24

Assessing evidence

•Historic comparisons


25

0 6 12 18 24 30 36 42 48 54 60

0.0

0.1

0.2

0.3

0.4

0.5

0.6

0.7

0.8

0.9

1.0

Pro

po

rtio

n o

f P

atie

nts

Alive

Age (months)

0 6 12 18 24 30 36 42 48 54 60

0.0

0.1

0.2

0.3

0.4

0.5

0.6

0.7

0.8

0.9

1.0

Pro

po

rtio

n o

f P

atie

nts

Alive

Age (months)

Untreated Infants in

Natural History Study*

Infants on ERT (n=17)*

*Based on n=163 with available data

ERT in Infantile-Onset Pompe Disease:

Survival Comparison by Kaplan Meier Analysis

26

Fig. 3

Source: Neuromuscular Disorders 2007; 17:470-475 (DOI:10.1016/j.nmd.2007.03.002 )

Copyright © 2007 Terms and Conditions

http://www.nmd-journal.com/article/S0960-8966(07)00083-1/abstract



http://www.nmd-journal.com/misc/terms

27

Assessing evidence

•Trials

•Controlled trials

•Placebo controlled trials

•Randomised trials

•Double blind trials


28

Assessing evidence

•Systematic review

•Meta analysis

•Problems in systematic reviews


29

Assessing evidence

•Head-to-head comparison

•Modelled comparison


30

A note on chance

•Effect and cost estimates:

Point estimates

Confidence intervals


31

Variability: 6MWT


health technology assessment -...

Documents