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

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COSTS

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Costs

•Exercise – driving to Paris

TITRE DE LA PRESENTATION (A MODIFIER SUR LE MASQUE)

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Cost types

•Unit and marginal costs

•Incremental costs

•Opportunity costs

•Tangible and intangible costs

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BENEFITS

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Benefits

•Survival

•Quality of life

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Assessing benefit

•Treatment X cures a fatal disease of

childhood

•Costs £100 000

•Extends life by 50 years

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Assessing benefit

•Treatment Y improves survival in a

fatal cancer

•Costs £10 000

•Extends life by 1 year

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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

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Assessing benefit

•Mobility

•Self Care – wash and dress

•Usual activities

•Pain

•Anxious or depressed

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Health state A

•No problem with mobility

•No problem with self care

•Some problems with usual activities

•Extreme pain

•Moderately depressed

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Health state B

•Some problems walking about

•Unable to wash or dress

•Some problems with usual activities

•No pain

•Not anxious or depressed

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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)

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Health state D

•No problems walking about

•No problems washing or dressing

•Some problems with usual activities

•No pain

•Not anxious or depressed

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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

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Health gain

• 20 years in health state without treatment = 20 x

• 20 years in health state with treatment = 20 x

•Gain =

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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

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A note on ethics

•Utilitarianism

•Other ethics

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STRENGTH OF EVIDENCE

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Evidence

•Exercise – 5 scenarios

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Assessing evidence

Stories

Historic comparison

Head-to-head

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Assessing evidence

•Anecdote, case series

•Placebo effects

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Assessing evidence

•Historic comparisons

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0 6 12 18 24 30 36 42 48 54 60

0.0

0.1

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0.9

1.0

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Alive

Age (months)

0 6 12 18 24 30 36 42 48 54 60

0.0

0.1

0.2

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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

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Fig. 3

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

Copyright © 2007 Terms and Conditions

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Assessing evidence

•Trials

•Controlled trials

•Placebo controlled trials

•Randomised trials

•Double blind trials

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Assessing evidence

•Systematic review

•Meta analysis

•Problems in systematic reviews

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Assessing evidence

•Head-to-head comparison

•Modelled comparison

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A note on chance

•Effect and cost estimates:

Point estimates

Confidence intervals

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Variability: 6MWT

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