Patient Involvement in Clinical ResearchIn Relationship with Biobanking

BBMRI - 15 December 2009

Cor OosterwijkProject Coordinator PatientPartner

Dutch Genetic Alliance VSOPEuropean Genetic Alliances’ Network EGAN

Challenges

• Administrative burden, costs, delays (European competitiveness)• Risk/benefit assessment, especially i.r.t. advanced therapies• Patient recruitment • Personalised medicine• Ethical and regulatory issues related to

– patient registries / biobanking– paediatric research– stakeholder relationships

• Public perception and awareness

Public perception of clinical research

Added value of patient participation

Before– Identification of indications, therapy features, patient population– Patient perspective on ethical and risk/benefit dilemmas – Defining patient-oriented outcome measuresDuring– Managing of expectations: hope or hype– Patient inclusion and compliance– Data quality– Patient and public confidence in clinical researchAfter:– Patients’ quality of life – Better compliance to therapies– (Cost-)effectiveness of healthcare

PatientPartnerDG Research – Health Directorate, Call identifier FP7-HEALTH-2007-A, 22/12/2006 – 19/4/2007. Coordination and support actions across the health theme: Health-2007-4.1-4:

Identifying patients’ needs in the clinical trial context• How can patients be better mobilized and

empowered in the clinical trials landscape• Which are the clinical outcomes that really matters

to patients, • how can they be integrated into clinical trials.

PatientPartner

• Objectives– identification of patients needs (yr 1)

– dialogue with other stakeholders on those needs and consensus development(yr 2)

– identification of regional differences (yr 2)

– development of (regional) strategies and recommendations for implementation (yr 3)

Consortium

Patient Alliances• Genetic Interest Group (GIG)• Dutch Genetic Alliance (VSOP)• European Genetic Alliances’ Network (EGAN)

Multi-Stakeholder NGO• European Forum for Good Clinical Practice (EFGCP)

PatientPartner

Best practices

Literature search

Regional Workshop Nord-West Europe

Start-Up Central Workshop

Regional Workshop Central/East Europe

Regional Workshop Southern Europe

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European Network of Patients Partnering in Clinical Research / Working Groups

Final Central Workshop

Interviews

Recommandations

Investigators

Policy makers

Regulators

May 2008 – May 2011

ENPCR: www.enpcrtest.forumotion.com•patient registries and biobanks •pediatric clinical research •ethical issues (stakeholder relationship, transparency)•information and education

Dissemination• Guides and Recommendations• At European and national levels • For:

– Patients Organizations – Academic and Biopharmaceutical Investigators, Sponsors

and CROs– Regulators, (Ethics) Committee’s, etc

The PatientPartner website will be transformed to facilitate contacts and partnerships between academic / biopharmaceutical stakeholders and patient organizations

Biobanks, clinical research and PatientPartner

• Validated biobanks increase the (cost)effectiveness of clinical research

• P.o. are a trusted third party to– Connect the basic and the clinical research field– Connect and integrate separate data/biobanks, both basic and clinical – Connect with individuals to obtain actual medical data and/or biological

samples • PatientPartner is about creating equal relationships. • PatientPartner pays special attention to the role of p.o. concerning

data/biobanks that are used in (pre)clinical research.• BBMRI Patient Working Group and PatientPartner can work

together on recommendations related to the role of p.o. in biobanks for (pre)clinical research.

Levels of Patient Participation in Clinical Research

11/04/23 11

Research subject

Information provider

Advisor

ReviewerReviewer

Co-researcherCo-researcher

Driving forceDriving force

11/04/23 12

Outcome PatientPartner survey

Co-researcher

Advisor

Information Provider

Research subject

Driving Force

Reviewer

Patient entrepreneurship in biobanking

Genetic Alliance BioBank Functioning

• Enable IRB approved investigator research that otherwise would not happen

• Ensure appropriate use of samples & data • Enable ethical re-contact and follow-up for

phenotype/genotype correlations, natural history and longitudinal studies

• Advocacy organization control & benefit sharing

www.biobank.org By: Patrick F. Terry ©

EGAN-EPPOSI conference report ‘Biobanks, the entrepreneurial role of patient organizations (2006)– Pseudoxanthoma Elasticum (PXE)– Alternating Hemiplegia of Childhood (www.enrah.org)– Hemophilia (NL) – Pompe’s disease (www.worldpompe.org)– Inflammatory bowel / Crohn’s disease (IBD warehouse)

www.egan.euwww.epposi.org

PatientPartner preliminary findings

• Much agreement on the need for pp in clinical research• No one-fits-all model: different needs and capacities

within patient organisations• Minor differences within Europe regarding needs, major

differences regarding possibilities• How to find each other and how to match

infrastructures and cultures?• Some research professionals fear delay and additional

bureaucracy

PatientPartner preliminary outcomes

• Stimulate and facilitate both virtual and ftf contact between clinical research professionals and patient organisations

• Basic, uniform training and information for the patient field is needed.

• P.o. want to be involved at a governance level: in the setting of research priorities, protocol development and ethical review

• User friendly databases are needed for research professionals on p.o. capacities irt clinical research and for p.o. on actual clinical research opportunities

• More efforts are needed to address the media and the public on the nature of clinical research and the position of patients in it.

Take home message

Patient partnership in biomedical and clinical research is not (only) a right (“Nothing about us without us”)

but a medical, scientific, ethical, and even economic necessity

that also brings responsibilities to p.o.

Start partnering there where everybody involved is convinced of the added value

and explore further possibilities from thereon.

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www.patientpartner-europe.eu

www.enpcrtest.forumotion.com

www.biomedinvo4all.com

BBMRI Patient Working Group and PatientPartner can work together on recommendations related to the role of p.o. in biobanks for (pre)clinical research.