[email protected]/cemdc/files/2016/12/Dubois_BM6_QALYs.pdf · Cost-utility analysis: current methodological issues and future perspectives. Front. Pharmacol. 2:29

QALYs: The Holy Grail?

Dominique Dubois MD FFPM FBCPMPhysician Specialist in Pharmaceutical Medicine

Vice-Chairman Pharmed post-graduate programme in Pharmaceutical Medicine and Medicines Development Sciences, Brussels, Belgium

[email protected]

mailto:[email protected]

Agenda

• Measuring utilities: strengths and weaknesses

• Where is the light(house)?

11/17/2016 Quality Adjusted Life Years 2

QALYs = Quality Adjusted Life Years

Quality Adjusted Life Years11/17/2016 3

One QALY is equal to 1 year of life in perfect health

TIME

(Years)

1

0

4

0.5

INDEX (“utility level”)

2

0.6+0.4

5

+0.5

Lieven Annemans

Health economics for non-economists

Gent, Academia Press, 2008 (www.academiapress.be)

Quality Adjusted Life Years

1

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The State of the Debate on QALYs

“Different methods for valuing health yield different results and this needs to be better understood”


Toward a Consensus on the QALYMichael Drummond, Diana Brixner, Marthe Gold, Paul Kind, Alistair McGuire,

Erik Nord, (Consensus Development Group)

V A L U E I N H E A L T H Volume 12 • Supplement 1 • 2009 S31–S35

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Nuijten MJC and Dubois DJ (2011) Cost-utility analysis: current methodological issues and

future perspectives. Front. Pharmacol. 2:29. doi: 10.3389/fphar.2011.00029

“Cost–effectiveness of a new pharmaceutical may depend heavily on underlying methodological choices for measurement of QALYs.”

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QALY Assessment for Healthcare Decision Making

Should Be Abandoned”

http://www.echoutcome.eu/index.php

http://www.echoutcome.eu/images/Echoutcome__Leaflet_Guidelines___final.pdf

Final conference of the ECHOUTCOME project held in Brussels on January 25th, 2013

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“Given the overwhelming methodological limitations,

http://www.echoutcome.eu/index.php

http://www.echoutcome.eu/images/Echoutcome__Leaflet_Guidelines___final.pdf

Relative importance of decision criteria according to Belgian citizens

Added value of new interventions:

The general public gives the highest weight to

the impact on quality of life.


Cleemput I, Devriese S, Kohn L, Devos C, van Til J, Groothuis-Oudshoorn K, Vandekerckhove P, Van de Voorde C. Incorporating

societal preferences in reimbursement decisions – Relative importance of decision criteria according to Belgian citizens. Health

Services Research (HSR) Brussels: Belgian Health Care Knowledge Centre (KCE). 2014. KCE Reports 234. D/2014/10.273/91.

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INCORPORATING SOCIETAL PREFERENCES INREIMBURSEMENT DECISIONS

RELATIVE IMPORTANCE OF DECISION CRITERIA ACCORDING TO BELGIAN CITIZENS

• In the appraisal of therapeutic need, the general public as well as the policy makers gave the highest weight to the current quality of life. For the general public, the inconvenience of current treatment is more important than the impact of the disease on life expectancy, despite current treatment. This is the other way around for the decision makers.

• In the appraisal of the added value of new interventions, the general public gives the highest weight to the intervention’ impact on quality of life, followed by its impact on the prevalence of the disease and on life expectancy. Decision makers have the same preference order, but their weight for the impact on life expectancy is relatively larger than in the general public.


Cleemput I, Devriese S, Kohn L, Devos C, van Til J, Groothuis-Oudshoorn K, Vandekerckhove P, Van de Voorde C. Incorporating

societal preferences in reimbursement decisions – Relative importance of decision criteria according to Belgian citizens. Health

Services Research (HSR) Brussels: Belgian Health Care Knowledge Centre (KCE). 2014. KCE Reports 234.

D/2014/10.273/91.

How to Measure?

Types of HRQL measures

1. Generic profile measures: SF-36

2. Condition/Disease-specific measures

3. Direct preference measures

4. Indirect preference measures


Medical Outcomes Study Short Form-36 (SF-36)

• Physical functioning (10 items)

• Role limitations/physical (4 items)

• Bodily Pain (2 items)

• General health perceptions (5 items)

• Vitality (4 items)

• Social functioning (2 items)

• Role limitations/emotional (3 items)

• Mental Health (5 items)

• Reported Health Transition

(How health is now compared to 1 year ago) (1 item)


SF-36® Health Survey


Types of HRQL measures

1. Generic profile measures: SF-36

2. Condition/Disease-specific measures

3. Direct preference measures

4. Indirect preference measures


Direct Utility- or Preference based measures

- Standard gamble

- Time trade-off

- Visual analogue scales



Visual Analogue Scale

How good or bad is your health state today?

0

Worst imaginable

Health state

100

Best imaginable

Health state

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Time Trade-off

17

Alternative 1

Alternative 2HEALTHY 1.0

TIME

STATE i hi

DEAD 0x t

VALUE

Alternative 1: State i for time t (life expectancy followed by death)Alternative 2: Healthy for time x < t followed by deathPreference score for state i: hi = x/t

Quality Adjusted Life Years11/17/2016

Standard Gamble

18

Health State 1

Full Health

Immediate Death

Probability P (x%)

Probability 1-P

Certainty

Gamble

11/17/2016 Quality Adjusted Life Years

Indirect Utility- or Preference Based Measures

– SF-6D

– EQ-5D (EuroQol)

– Health Utility Index (HUI)

– Quality of Well-Being (QWB)


SF-6D

Physical functioning

Role limitations

Social functioning

Pain

Mental health

Vitality


EQ-5D


Mobility

Self-care

Usual activities

Pain/

discomfort

Anxiety/

depression

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National Institute for Health and Care Excellence


EQ-5D Descriptive system and EQ 5D VAS


EQ-5D descriptive system

• Mobility

• Self-care

• Usual activities (eg work, study, housework, family or leasure activities)

• Pain/discomfort

• Anxiety/Depression

• Each (single item) dimension has 3 levels:

– No problems

– Some problems

– Severe problems

17-nov-16 24

EQ-5D Descriptive System1. Mobility

I have no problems in walking about

I have some problems in walking about

I am confined to bed

2. Self-care

I have no problems with self-care

I have some problems washing or dressing myself

I am unable to wash or dress myself

3.Usual Activities

I have no problems with performing my usual activities

(e.g. work, study, housework, family or leisure activities)

I have some problems with performing my usual activities

I am unable to perform my usual activities

4.Pain/Discomfort

I have no pain or discomfort

I have moderate pain or discomfort

I have extreme pain or discomfort

5.Anxiety/Depression

I am not anxious or depressed

I am moderately anxious or depressed

I am extremely anxious or depressed


Please tick one

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EQ-5D Scoring Algorithm

Full health 1.00 Constant term (for any dysfunctional state): -0.081 Mobility:

Level 2 -0.069 Level 3 -0.314

Self-care: Level 2 -0.104 Level 3 -0.214

Usual activities: Level 2 -0.036 Level 3 -0.094

Pain or discomfort: Level 2 -0.123 Level 3 -0.386

Anxiety or depression Level 2 -0.071 Level 3 -0.236

N3 (level 3 occurs within at least one dimension) -0.269


Dolan P, Medical Care 35 (11): 1095-1108, (1997)

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EQ-5D Estimated value for state 12123

Full health 1.00

Constant term (for any dysfunctional state): -0.081

Mobility: Level 1 -0

Self-care: Level 2 -0.104

Usual activities: Level 1 -0

Pain or discomfort: Level 2 -0.123

Anxiety or depression: Level 3 -0.236

N3 (level 3 occurs within at least one dimension) -0.269

Estimated Value for state 12123: 0,187


Dolan P, Medical Care 35 (11): 1095-1108, (1997

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Patient Reported Outcome Measures (PROMs)


http://www.hscic.gov.uk/catalogue/PUB17201

Provisional Monthly Patient Reported Outcome Measures (PROMs) in England – April 2014 to November 2014

Publication date: April 09, 2015

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http://www.hscic.gov.uk/catalogue/PUB17201

5 Things You Should Do with EQ5D Data

https://www.ohe.org/news/5thingsyoushoulddoeq5ddata

Friday, 29 January 2016Written by Professor Nancy Devlin, Director of Research at

OHE and member of the EuroQol Group

https://www.ohe.org/news/5thingsyoushoulddoeq5ddata


1. What not to do – don’t skip straight to ‘utilities’ or use of scores of any kind to summarise patients’ data

2. Look at patients’ responses to the question items (dimensions). Good, old fashioned descriptive statistics …



3. Summarising change without using value sets

– Their health state is better

– Their health state is worse

– Their health state is exactly the same

– The changes in health are ‘mixed’: better on one dimension, but worse on another.



4. Use your EQ-VAS data!

5. Last but not least: if you do need to use a value set to summarise your EQ5D data – e.g., for estimating QALYs – make sure you do sensitivity analysis to alternative value sets.



European Network for Health Technology Assessment

Endpoints used for relative effectiveness assessment of pharmaceuticals:

HEALTH-RELATED QUALITY OF LIFE and UTILITY MEASURES


EUnetHTA Joint Action WP5 – Relative Effectiveness Assessment (REA) of

Pharmaceuticals – Model for Rapid Relative Effectiveness Assessment of

Pharmaceuticals, February 2013, Final version

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Health-related quality of life and utility measures

• A general recommendation applicable to all types of Relative Effectiveness Assessment (REA), irrespective of their particular purpose, is to require the inclusion of a disease or population specific AND a generic HRQL measure for most adequately capturing the impact of a disease on daily life.

• In case there is a need for the calculation of QALYs, a utility measure (Time Trade-Off or Standard Gamble) OR generic HRQL instrument associated with a reference set of utility values is recommended.



Health-utility data may be generated using one of the following categories of instruments

1. A generic preference-based measure (PBM) (e.g., EQ-5D, Health Utilities Index [HUI], Short-Form Six-Dimensions [SF-6D, derived from SF-36], 15D, Assessment of Quality of Life [AQoL], or Quality of Well-being [QWB])

2. A condition-specific PBM (e.g., the Asthma Quality of Life Utility

Index [AQL-5D] or the European Organization for Research and

Treatment of Cancer eight dimensions [EORTC-8D]);

3. A non–preference-based, condition-specific patient-reported

outcome measure mapped onto a generic, preference-based

measure


V A L U E IN HE A L T H 1 9 ( 2 0 1 6 ) 7 0 4 – 7 1 9


QALY: The Holy Grail?

MEASURING IS KNOWING ?

IT’s ALL ABOUT THE PATIENT(S) !

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PHARMED

http://www.frontiersin.org/Pharmaceutical_Medicine_and_Outcomes_Research

THANK YOU !


[email protected]

11/17/2016 40

http://www.frontiersin.org/Pharmaceutical_Medicine_and_Outcomes_Research

mailto:[email protected]

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[email protected]/cemdc/files/2016/12/Dubois_BM6_QALYs.pdf · Cost-utility analysis: current methodological issues and future perspectives. Front. Pharmacol. 2:29