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Grazie per aver scelto di utilizzare a scopo didattico questo materiale delle Guidelines 2011 libra. Le ricordiamo che questo materiale è di proprietà dell’autore e fornito come supporto didattico per uso personale. Development and validation of new patient related outcomes. Thierry Troosters.

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Grazie per aver scelto di utilizzare a scopo didattico questo materiale delle Guidelines 2011 libra.Le ricordiamo che questo materiale è di proprietà dell’autore e fornito come supporto didattico per uso personale.


Development and validation of new patient related outcomes

Development and validation of new patient related outcomes questo materiale delle Guidelines 2011 libra.

Thierry Troosters


Outline
Outline questo materiale delle Guidelines 2011 libra.

  • What are patient reported outcomes

    • The researchers perspective

    • Regulators perspective

  • PRO science: Validating new PRO’s

  • The example of PROactive


Pros and other outcomes
PROs and other outcomes questo materiale delle Guidelines 2011 libra.

Pathology

LF

What the disease means to the patient

Troosters Respir Medicine 2010

Watz AJRCCM 2008


Pros and other outcomes1
PROs and other outcomes questo materiale delle Guidelines 2011 libra.

Classical outcomes

Lung function

CT-scan

(Bio-markers)

Pathology

LF

Classical outcomes

Exercise capacity

Patient Reported Outcomes

Few are available

What the disease means to the patient


Pro definition
PRO: definition questo materiale delle Guidelines 2011 libra.

A report of the status of a patient’s health condition that comes directly from the patient, without interpretation of the patient’s response by a clinician or anyone else.

FDA guidance document 2009 www.fda.gov


Pros insight in effect
PROs: Insight in effect questo materiale delle Guidelines 2011 libra.

Patient reported outcome tools may help capturing events that happen outside a ‘clinical contact’ (e.g. Exacerbations, side effects)


Pros insight in effect1
PROs: Insight in effect questo materiale delle Guidelines 2011 libra.

Patient reported outcome tools may help capturing events that happen outside a ‘clinical contact’ (e.g. Exacerbations)

48

EXACT PRO

Frequency and severity of exacerbations

44

Mean Daily EXACT total scores

40

36

1

7

14

21

28

Day

Leidy AJRCCM 2011


Pros insight in effect2
PROs: Insight in effect questo materiale delle Guidelines 2011 libra.

Patient reported outcome tools may help capturing events that happen outside a ‘clinical contact’

Some (side-) effects of interventions are only known to the patient Aquadro Value Health 2003


Pros insight in effect3
PROs: Insight in effect questo materiale delle Guidelines 2011 libra.

Patient reported outcome tools may help capturing events that happen outside a ‘clinical contact’

Some (side-) effects of interventions are only known to the patient Aquadro Value Health 2003

PROs often integrate several ‘physiologic’ effects of interventions


Pros insight in effect4
PROs: Insight in effect questo materiale delle Guidelines 2011 libra.

CRDQ vs QF R=0.17

CRDQ vs 6MWD R=0.27


Pros insight in effect5

11 ml.yr questo materiale delle Guidelines 2011 libra.-1

PROs: Insight in effect

42 ml.yr-1 *

1.71 ± 0.24 points.yr-1

1.05 points.yr-1

53 ml.yr-1

0.66 ± 0.23 points.yr-1

Troosters ERJ 2010


Pros insight in effect6
PROs: Insight in effect questo materiale delle Guidelines 2011 libra.

Exacerbations

Mood

Fear

Lungfunction

gas exchange

Symptoms

Musclefunction

PhysicalActivity

HRQoL

ExerciseTolerance

Cardiovascularfunction

Health Beliefs

Body composition

Selfefficacy

Past behavior

Social support


Pros insight in effect7
PROs: Insight in effect questo materiale delle Guidelines 2011 libra.

More integration of different additive/complementary effects

More confounders not directly related to the intervention

Mood

Fear

Lungfunction

gas exchange

Symptoms

Musclefunction

PhysicalActivity

HRQoL

ExerciseTolerance

Cardiovascularfunction

Health Beliefs

Body composition

Selfefficacy

Past behavior

Social support


Pros the regulator s perspective
PROs: The regulator’s perspective questo materiale delle Guidelines 2011 libra.

PROs are nowadays essential to support efficacy

Patient reported outcomes to support labeling claims:

-Very stringent methodology for development

-Subject to regulatory approval

-Intermediate feedback is possible (qualification process)


Outline1
Outline questo materiale delle Guidelines 2011 libra.

  • What are patient reported outcomes

    • The researchers perspective

    • Regulators perspective

  • PRO science: Validating new PRO’s

  • The example of PROactive


Pros very strict guidance on development
PROs: Very strict guidance on development questo materiale delle Guidelines 2011 libra.


Pros very strict guidance on development1
PROs: Very strict guidance on development questo materiale delle Guidelines 2011 libra.

Clear concept and conceptual model

Does the PRO assess something relevant to patients?

Items that are understood by patients and reflect their experience

With specific attention to Content and Scoring

Culturally sensitive translations

Focus groups and patient interviews form the basis

Literature and experts are supportive


Pros very strict guidance on development2
PROs: Very strict guidance on development questo materiale delle Guidelines 2011 libra.

Identify concepts

& develop conceptual framework

PRO

Modify instrument

(concept, population, method of administration)

Create the instrument

(items, scales, format, pilot)

Assess measurement properties

(Redundancy, revise, MCID, training materials)

PATIENT INPUT IN EVERY STAGE IS VITAL

http://www.fda.gov/cber/gdlns/prolbl.htm


An example proactive capturing pa
An example: PROactive capturing PA questo materiale delle Guidelines 2011 libra.

  • PROactive aims at developing PROs that capture physical activity

  • Question 1

    Do patients consider physical activity or a change thereof relevant/important?

Proactive WP2D 2011


An example proactive capturing pa1
An example: PROactive capturing PA questo materiale delle Guidelines 2011 libra.

Proactive WP2D 2011


An example proactive capturing pa2
An example: PROactive capturing PA questo materiale delle Guidelines 2011 libra.

  • PROactive aims at developing PROs that capture physical activity

  • Question 1

    Do patients consider physical activity or a change thereof relevant/important?

  • Question 2

    What is physical activity from a patient perspective


An example proactive capturing pa3
An example: PROactive capturing PA questo materiale delle Guidelines 2011 libra.

  • Any bodily movement produced by the skeletal muscles that results in energy expenditure

  • Amount and Intensity of physical activity are important to maintain health

  • Physical activity should be considered as a ‘vital sign’

  • WHAT DOES ‘PHYSICAL ACTIVITY’ MEAN TO PATIENTS?

Caspersen Public Health Rep 1985.

Haskell Circulation 2007

CDC Physical activity plan March 2010


Pros very strict guidance on development3
PROs: Very strict guidance on development questo materiale delle Guidelines 2011 libra.

Cognitive debriefing

Country 1

Cognitive debriefing

Country 2

Individual interv.

Country 1

Focus group

Country 1

Cognitive debriefing

Country 3

Focus group

Country 2

Language used by COPD patients

Dimensions relevant to patients with COPD

Cognitive debriefing

Country 4

Individual interv.

Country 2

Initial Item list

Transcribed / translated / Analyzed

Transcribed / translated / Analyzed

Focus group

Country 3

Cognitive debriefing

Country 5

Individual interv.

Country 3

Cognitive debriefing

Country …

Focus group

Country 4

Report/ Adapt where needed

Literature review

Valid and acceptable

Activity monitor

Expert input

Expert input

Item list to initial validation (WP4)


Physical inactivity patient perspective
Physical inactivity: questo materiale delle Guidelines 2011 libra.Patient perspective

Concept Physical Activity

Amount of activity

Walking outdoors

Household Chores

Leisure activities

Dressing

Bathing

Consequences

Emotional

-Feeling sad

-Feeling frustrated

-Feeling dependent

-Embarassement

Influencing factors

Disease related

-Comorbidity

-Exacerbations

-Severity

Symptoms / difficulty

General dyspnea/Fatigue

Symptoms with specific activities

Difficulties with activities

Consequences

Social

-Feeling Isolated

-Others helping

-Feeling dependent

-Others not understanding

Influencing factors

External

-Climate

-Air quality

Need for adaptation

Need for breaks

Slow down

Help from others

Aids


Physical inactivity patient perspective1
Physical inactivity: questo materiale delle Guidelines 2011 libra.Patient perspective

Concept Physical Activity

24 items

Amount of activity

Walking outdoors

Household Chores

Leisure activities

Dressing

Bathing

Symptoms / difficulty

General dyspnea/Fatigue

Symptoms with specific activities

Difficulties with activities

Need for adaptation

Need for breaks

Slow down

Help from others

Aids


Physical inactivity patient perspective2
Physical inactivity: questo materiale delle Guidelines 2011 libra.Patient perspective

Concept Physical Activity

Amount of activity

Walking outdoors

Household Chores

Leisure activities

Dressing

Bathing

Questionnairs available

Monitors available

Symptoms / difficulty

General dyspnea/Fatigue

Symptoms with specific activities

Difficulties with activities

Questionnaires available

Need for adaptation

Need for breaks

Slow down

Help from others

Aids

New dimention


The validation process
The validation process questo materiale delle Guidelines 2011 libra.

  • Content validity of the items

  • Validity of the items

  • Response options preferably linear

  • Sensitivity to change


The validation process1
The validation process questo materiale delle Guidelines 2011 libra.


The validation process an example cat
The validation process; an example CAT questo materiale delle Guidelines 2011 libra.

21 Items

21 items capturing the most important aspects of a patient’s COPD health

Individual interviews

Focus groups

17 Items

Administered in 6 countries in 1490 patients

4 items with

Floor- Ceiling – Poor Item to total correlation

14 Items

3 items with

High inter item correlation (measuring the same thing)

8 Items

6 items

Performing poorly on scoring properties

Leaving 8 items with minimal bias for age, gender, country

Jones Eur Respir J 2009


The validation process2
The validation process questo materiale delle Guidelines 2011 libra.

  • Content validity of the items

  • Validity of the items

  • Sensitivity to change

  • Response options preferably linear

  • MID with several interventions

  • Version control (languages!)


The validation process3
The validation process questo materiale delle Guidelines 2011 libra.

Patient perception of improvement may differ between interventions

Troosters ERJ 2011


Summary
Summary questo materiale delle Guidelines 2011 libra.

  • Patient reported outcomes do highlight unique features of the effects of interventions

  • Methodology to develop PROs has been described recently in much detail by regulators

  • PROs have the benefit of integrating several subtle physiologic changes

  • At the expense of having more potential confounders not directly related to the intervention

  • PROactive is a new PRO that aims at capturing the effects of interventions on PA


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