incorporating patients in research what have we done and how did we do it maarten de wit-04112014

Incorporating the patient perspective in outcome research. A best practice case from the field of rheumatology

Oslo November 4, 2014

Maarten de Wit

Researchers can easily overlook the complexity and capriciousness of living with a chronic disease, reducing the meaning of life experiences to abstract themes and models.

Schipper, K. (2011). Patient participation & knowledge [thesis]. VU University, Amsterdam (p.232)

Introducing EULAR’s recommendations for patient involvement

Developing a PROM for Psoriatic Arthritis

Multiple levels of patient involvement

Added value of patient involvement

Take home messages

Overview

Introducing EULAR’s recommendations for patient involvement

Developing a PROM for Psoriatic Arthritis

Multiple levels of patient involvement

Added value of patient involvement

Take home messages

Overview

The European League Against Rheumatism (EULAR) is the organisation which represents the patient, health professional and

scientific societies of rheumatology of all the European nations. EULAR endeavours to stimulate, promote, and support the research, prevention, treatment and rehabilitation of rheumatic diseases. In line with UEMS, EULAR defines rheumatology as including rheumatic diseases of the connective tissue, locomotor and musculoskeletal systems.

5

EULAR

EULAR initiative: Patients as research partners

Introducing EULAR’s recommendations for patient involvement

Developing a PROM for Psoriatic Arthritis

Multiple levels of patient involvement

Added value of patient involvement

Take home messages

Overview

June 2014, Volume 73, Issue 6

A EULAR case study

The development and validation of the EULAR PsAID

(Psoriatic Arthritis Impact of Disease score)

Development and validation of the Psoriatic Arthritis Impact of Disease (PsAID) questionnaire.

A 13-country EULAR initiative

New, short questionnaire to better assess all patient-perceived impact of PsA

Both for clinical practice as well as for clinical trials

Developed with patient-partners

PsA Impact of Disease – participants

Participation of patient research partners is strongly recommended for clinical research projects and for the development of recommendations and guidelines, and should be considered for all other research projects. WHEN

1

Participation of patient research partners should be considered in all phases of the project to provide experiential knowledge, with the aim of improving the relevance, quality and validity of the research process. WHEN

2

Methods and phases in the research project

Elaboration of domains

into questions

Formulations of items

Translation of items

Cognitive debriefing

on translation

Identification and selection of

domains of health

Initial

identification

of domains

Domains

prioritised for

importance

Ranking order

Priority (yes/no)

VA

LID

AT

ION

Filling in

qu

estion

naires

Steering Committee

A minimum of two patient research partners should be involved in each project. NUMBER

Identification of potential patient research partners should be supported by a clear description of expected contributions. RECRUITMENT

3 4

Tasks and numbers

Identification /

elaboration of

questions

Physician and

Patient-partner

opinion on wording

65 patients pre-testing

translated items

Identification / selection of

domains of health

Initial

identification

11 patient-

partners

Domains

prioritised for

importance

139 patients

Ranking order

Priority (yes/no)

VA

LID

AT

ION

49

9 re

spo

nd

en

ts

Steering Committee: 2 patient experts

The selection process of patient research partners should take into account communication skills, motivation and constructive assertiveness in a team setting. SELECTION

5

Introducing EULAR’s recommendations for patient involvement

Developing a PROM for Psoriatic Arthritis

Multiple levels of patient involvement

Added value of patient involvement

Take home messages

Overview

Levels of participation

© Teunissen 2009

Consultation

Advise

Collaboration

Control

4) Study participants to prioritize important domains (n=139) and to fill in questionnaires (n=499)

3) Patient respondents in cognitive debriefing (n=65)

2) National patient research partners (n=11)

1) Patient steering Group members (n=2)

Levels of participation

The principal investigator must facilitate and encourage the contribution of patient research partners, and consider their specific needs. SUPPORT

6

Pre-meeting material for patient-research-partners

Zurich, November 2012

Table of Contents Introduction 1 Logistic information 2 Reminder on the PsAID project: objectives 3 Reminder on the PsAID project: previous steps 4 First steps: elaboration of the questionnaire 4 Second step: finalization and validation of the

questionnaire 6 What does validation mean? 6 The meeting in Zurich: what will happen there? 7 PsAID glossary: some terms you may be hearing at the

meeting 8

Introducing EULAR’s recommendations for patient involvement

Developing a PROM for Psoriatic Arthritis

Multiple levels of patient involvement

Added value of patient involvement

Take home messages

Overview

Added value of patients´ involvement

Identification /

elaboration of

questions

Physician and

Patient-partner

opinion on wording

65 patients pre-testing

translated items

Identification / selection of

domains of health

Initial

identification

11 patient-

partners

Domains

prioritised for

importance

139 patients

Ranking order

Priority (yes/no)

VA

LID

AT

ION

49

9 re

spo

nd

en

ts

Steering Committee: 2 patient experts

16 domains

of health

12 domains

of health

9 domains

of health

Ranking

Mean rank Priority Priority

order

In top 8

ranks

Lowest

4 ranks

1 Pain 2.56 84% 1 95% 0

2 Skin problems 6.2 53% 2 65% 13%

3 Fatigue 6.43 43% 5 74% 6%

4 Ability to work / leisure 6.67 50% 3 67% 9

5 Disability 7.23 46% 4 64% 12%

6 Feeling of discomfort 7.58 26% 10 64% 7%

7 Sleep disturbance 7.96 36% 6 56% 25%

8 Anxiety, fear and uncertainty 8.42 33% 8 50% 19%

9 Coping 8.45 35% 7 53% 17%

10 Embarrassment and/or shame 9.74 24% 11 40% 35%

11 Social participation 10.01 23% 13 33% 30%

12 Depression 10.06 24% 12 39% 32%

13 Relationship with family 10.51 30% 9 34% 44%

14 Concentration difficulties 10.61 19% 14 32% 37%

15 Rejection and discrimination 11.60 12% 16 22% 53%

16 Sexual life 11.61 15% 15 25% 52%

Ranking

Mean rank Priority Priority

order

In top 8

ranks

Lowest

4 ranks

1 Pain 2.56 84% 1 95% 0

2 Skin problems 6.2 53% 2 65% 13%

3 Fatigue 6.43 43% 5 74% 6%

4 Ability to work / leisure 6.67 50% 3 67% 9

5 Disability 7.23 46% 4 64% 12%

6 Feeling of discomfort 7.58 26% 10 64% 7%

7 Sleep disturbance 7.96 36% 6 56% 25%

8 Anxiety, fear and uncertainty 8.42 33% 8 50% 19%

9 Coping 8.45 35% 7 53% 17%

10 Embarrassment and/or shame 9.74 24% 11 40% 35%

11 Social participation 10.01 23% 13 33% 30%

12 Depression 10.06 24% 12 39% 32%

13 Relationship with family 10.51 30% 9 34% 44%

14 Concentration difficulties 10.61 19% 14 32% 37%

15 Rejection and discrimination 11.60 12% 16 22% 53%

16 Sexual life 11.61 15% 15 25% 52%

Results: Patients’ contributions

Identification /

elaboration of

questions

Physician and

Patient-partner

opinion on wording

65 patients pre-testing

translated items

Identification / selection of

domains of health

Initial

identification

11 patient-

partners

Domains

prioritised for

importance

139 patients

Ranking order

Priority (yes/no)

VA

LID

AT

ION

49

9 re

spo

nd

en

ts

Steering Committee: 2 patient experts

16 domains

of health

12 questions

9 questions

2 validated

questionnaires

12 domains

of health

9 domains

of health

The principal investigator must ensure that patient research partners receive information and training appropriate to their roles. EDUCATION

The contribution of patient research partners to projects should be appropriately recognised, including co-authorship when eligible. ACKNOWLEDGEMENT

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Introducing EULAR’s recommendations for patient involvement

Developing a PROM for Psoriatic Arthritis

Multiple levels of patient involvement

Added value of patient involvement

Take home messages

Overview

Take home messages

Patient involvement should be considered in every phase of the research process

Patient involvement should be considered on multiple levels

The role of the principal investigator is key in enabling patients to contribute to the research

Thanks for listening martinusdewit@hotmail.com

Oslo November, 4 2014

M. de Wit L. Gossec

T. Kvien