SlideShare a Scribd company logo
Director of Treatment Information and Access
European Organisation for Rare Diseases
Zaragoza, 27-28 April 2017
PATIENTS’ AND CONSUMERS’ WORKING
WITH THE EUROPEAN MEDICINES AGENCY
François Houÿez
Disclaimers
EURORDIS is one of the
signatories of the “Code of
Practice between Patients’
Organisations and the
Healthcare Industry”
Read here
Disclaimers
EURORDIS is one of the
signatories of the “Code of
Practice between Patients’
Organisations and the
Healthcare Industry”
Read here
EMA started its activities in 1995
April 1996: CPMP met with 6 patients’ representatives
• 2002: workshop on how to work together
• 2006: the framework of interaction with patients and consumers adopted by EMA
• 2016: 770 occasions where patients and consumers were involved in EMA activitie
Moving away from a situation where 12 Member States could take different
decisions on marketing authorisations
To a European Union with a marketing authorisation valid in 28 Member States
Norway, Iceland and Liechtenstein, serving 508 millions citizens
• With same product name, same information for patients and healthcare
professionals: all documents for the public are reviewed by patient and consumer
When to consult with patients?
Horizon scanning
How to evaluate medicines? Do we have the right outcome
measurement tools? Patients participate in guidelines
development
A framework of interaction developed with the Patients’
and Consumers’ Working Party (see more here):
Guidance and policy adopted by the EMA management board
The role of patients as members of the EMA human scientific committees
Pilot phase to involve patients in benefit / risk discussions at CHMP meetings
Reporting
EMA’s interaction with patients, consumers, healthcare professionals and their organisations -
2015
Rules
Criteria to be fulfilled by patients’ and consumers’ organisations involved in EMA activities
Evaluation of financial information from patients’ consumers’ and healthcare professionals’
organisations for assessment of EMA ‘eligibility’
Training strategy
Involvement of patient representatives in scientific advice procedures at the EMA
2016: the PCWP celebrating its 10th anniversary!
Involvement across different activities 2009-2016
9
Necessary steps to involve patients
How does it mean in terms of days for EURORDIS?
Members of scientific committees at
the EMA (volunteers)
7
(COMP,
PDCO,CAT, PCWP)
Total days spent in 2016: 286
Average days / volunteer: 41 / year
(7-88)
Members of EURORDIS staff working
on patient engagement / EMA
1.5 FTE 3 different staff
Member of EMA Management board 1 Total days : 22 / year
Number of EMA SA/PA procedures 386 (2015) =154 reviews for EURORDIS (orphan
Numbers of patients invited to
scientific advice
76 (2015) Of whom 25 EURORDIS patients
Number of patients trained
(EURORDIS Summer School) since
2008
300+ 1 FTE for training programme
Other services:
Contact database maintenance, e-learning tools
development, administration, communication to member
EURORDIS Summer School (training): how to minimise
the influence of industry? Example of a case study
CHMP invited 2 patients to an oral
explanation with MAA
Two hours before CHMP meeting, MAA,
patients, clinicians who had been working
on the product for years all together met in
a hotel
Mentor explained the issue
advised not to have the meeting
finally left the room and informed the
EMA about the incident
• What happened • Impact
• CHMP agreed to have the patients
they invited in the room
• CHMP chair explained the situation
before the meeting started
• CHMP agreed to proceed
• It could have stopped there
How? Informing on the evaluation once made? Consulting during the
evaluation? Involving when communicating decision?
1 person? 2? 5?
Patient expert own opinion, or organisation views?
Patient preference elicitations
Extrapolation? 30 or 300 patients? More?
Other deliberative methods?
Panel of patients ? Citizens’ jury ? Focus groups?
Face to face? Video-conferences? In writing?
Patient who participated in clinical trial, or not?
Time for more pilots
Success factors
High quality dialogue
Patients are considered on the same level than other experts
A dedicated unit at the EMA for stakeholders’
involvement
Training materials (videos, face to face…)
Adequate resources and budget
Costs covered
Daily allowance, doubled for volunteers
Rules for involvement defined together with stakeholders
For all aspects
Revised as often as needed
“With a high qualit
dialogue, patients a
regulators can onl
agree.”
Jean-Michel Alexandre
Former CHMP chair, E
Patients’ satisfaction for invitations at CHMP oral explanations
with the applicant (14 patients involved, 6 meetings)
Barriers, obstacles
Patients’ advocates are few (availability, language)
Time commitment (e.g. committee members) and time spent unpaid
Advising the developer or advising the regulators (CofI policy)? Not both
Rapid regulatory timelines, and the time it takes to involve patients
Younger patients gather on social networks, no longer in registered
organisations with statutes, general assemblies, board of directors etc.
No impact assessment when defining a new role for civil society (EU
legislation making): training needs, contact database maintenance,
support, guidance…
The role of patients in EU legislation on medicines
REGULATION EC 726/2004 establishing EMA
Article 65 - The Management Board: Two representatives of patients' organisations shall be appointed by
Council in consultation with EP
Article 14 - The package leaflet shall reflect the results of consultations with target patient groups
REGULATION EC 141/2000 on orphan medicinal products
Article 4 – COMP: Three members nominated by the Commission to represent patients' organisations
REGULATION EC 1901/2006 on medicinal products for paediatric use
Article 4 - Paediatric committee: Three members and three alternates appointed by the Commission in
order to represent patient associations
REGULATION EC 1394/2007 on advanced therapy medicinal products
Article 21 - Committee for Advanced Therapies: The Committee for Advanced Therapies shall be
composed of two members and two alternates to represent patients associations
REGULATION (EU) No 1235/2010 on pharmacovigilance
Article 61a: one member and one alternate member in order to represent patient organisations
What can be improved? Next steps
Public Hearings (PRAC, safety concern and therapeutic context)
Updated patient information: graphic visualisation, changes in patient
leaflet, videos etc.
IMI PREFER project: how to integrate Patient Preferences Elicitations into
the evaluation process?
PRIME: Priority Medicines – Very early scientific advice, with patients
A new status for trained patients participating in CABs/Scientific advice:
“patient investigator”
To conduct research together
E.g “Patient Reporting in the EU: Analysis of EudraVigilance Data”, Drug
Safety, (), 1-17, DOI10.1007/s40264-017-0534-1
IMI projects (EUPATI, PROTECT…)
Synergies with HTA
IMI PROTECT: how to integrate these results in the
package leaflet? (June 2017 PCWP meeting)
Preferred by 54% by 21% by 25%
Patients fully integrated in the centralised procedure
for marketing authorisations in the EU
Bringing the best expertise in the decision
making, including the patients
With equal credibility as other experts
Impact of patient engagement difficult to
assess, but being able to witness the
process is a major achievement
To conclude
Patients at EMA: legal background
EU Treaty (Declaration 17 of the Annex)
“(…) Transparency of the decision-making process
strengthens the democratic nature of the institutions
and the public’s confidence in the administration.”
3

More Related Content

What's hot

Jornadas #PatientInHTA · Iñaki Imaz
Jornadas #PatientInHTA · Iñaki ImazJornadas #PatientInHTA · Iñaki Imaz
Jornadas #PatientInHTA · Iñaki Imaz
Instituto Aragonés de Ciencias de la Salud - IACS
 
Jornadas #PatientInHTA · Nicholas Brooke
Jornadas #PatientInHTA · Nicholas BrookeJornadas #PatientInHTA · Nicholas Brooke
Jornadas #PatientInHTA · Nicholas Brooke
Instituto Aragonés de Ciencias de la Salud - IACS
 
Jornadas #PatientInHTA · Paloma Casado
Jornadas #PatientInHTA · Paloma CasadoJornadas #PatientInHTA · Paloma Casado
Jornadas #PatientInHTA · Paloma Casado
Instituto Aragonés de Ciencias de la Salud - IACS
 
Jornadas #PatientInHTA · Gaizka Benguria
Jornadas #PatientInHTA · Gaizka BenguriaJornadas #PatientInHTA · Gaizka Benguria
Jornadas #PatientInHTA · Gaizka Benguria
Instituto Aragonés de Ciencias de la Salud - IACS
 
Good practices in patient involvement in HTA
Good practices in patient involvement in HTAGood practices in patient involvement in HTA
Good practices in patient involvement in HTA
EUPATI
 
Is there patient involvement in HTA? Can patients influence HTA decision making?
Is there patient involvement in HTA? Can patients influence HTA decision making?Is there patient involvement in HTA? Can patients influence HTA decision making?
Is there patient involvement in HTA? Can patients influence HTA decision making?
Kathi Apostolidis
 
Consumer involvement: Patient views on participation in HTA
Consumer involvement: Patient views on participation in HTAConsumer involvement: Patient views on participation in HTA
Consumer involvement: Patient views on participation in HTA
Cochrane.Collaboration
 
Drug development and treatment strategies may go hand in hand.
Drug development and treatment strategies may go hand in hand.Drug development and treatment strategies may go hand in hand.
Drug development and treatment strategies may go hand in hand.
EUPATI
 
Patients’ voice in access decisions
Patients’ voice in access decisionsPatients’ voice in access decisions
Patients’ voice in access decisions
EUPATI
 
Health Technology Assessment
Health Technology AssessmentHealth Technology Assessment
Health Technology Assessment
EUPATI
 
Observatory of Innovation in healthcare management in Catalonia (OIGS)
Observatory of Innovation in healthcare management in Catalonia (OIGS)Observatory of Innovation in healthcare management in Catalonia (OIGS)
Observatory of Innovation in healthcare management in Catalonia (OIGS)
Agència de Qualitat i Avaluació Sanitàries de Catalunya (AQuAS)
 
Shared Decision Making in Health in Catalonia
Shared Decision Making in Health in CataloniaShared Decision Making in Health in Catalonia
Shared Decision Making in Health in Catalonia
Agència de Qualitat i Avaluació Sanitàries de Catalunya (AQuAS)
 
Essencial Project in Catalonia. Avoiding low-value clinical practices
Essencial Project in Catalonia. Avoiding low-value clinical practicesEssencial Project in Catalonia. Avoiding low-value clinical practices
Essencial Project in Catalonia. Avoiding low-value clinical practices
Agència de Qualitat i Avaluació Sanitàries de Catalunya (AQuAS)
 
Webinar: Guidance on Industry-led R&D
Webinar: Guidance on Industry-led R&DWebinar: Guidance on Industry-led R&D
Webinar: Guidance on Industry-led R&D
Hibernia College
 
Tripartite dimension of interaction of patients, regulators and industry (Jan...
Tripartite dimension of interaction of patients, regulators and industry (Jan...Tripartite dimension of interaction of patients, regulators and industry (Jan...
Tripartite dimension of interaction of patients, regulators and industry (Jan...
jangeissler
 
In Electronic Health Records We Trust - IPPOSI Outcome Report - March 2017
In Electronic Health Records We Trust - IPPOSI Outcome Report - March 2017In Electronic Health Records We Trust - IPPOSI Outcome Report - March 2017
In Electronic Health Records We Trust - IPPOSI Outcome Report - March 2017
ipposi
 
Brookman final last 5 nov
Brookman final last 5 nov Brookman final last 5 nov
Brookman final last 5 nov
Nowgen
 
The patients' contribution to ethics committees in the Netherlands
The patients' contribution to ethics committees in the NetherlandsThe patients' contribution to ethics committees in the Netherlands
The patients' contribution to ethics committees in the Netherlands
EUPATI
 
How to communicate scientific and medical information to patients, advocates ...
How to communicate scientific and medical information to patients, advocates ...How to communicate scientific and medical information to patients, advocates ...
How to communicate scientific and medical information to patients, advocates ...
jangeissler
 
Mini-Course Starter Kits
Mini-Course Starter KitsMini-Course Starter Kits
Mini-Course Starter Kits
EUPATI
 

What's hot (20)

Jornadas #PatientInHTA · Iñaki Imaz
Jornadas #PatientInHTA · Iñaki ImazJornadas #PatientInHTA · Iñaki Imaz
Jornadas #PatientInHTA · Iñaki Imaz
 
Jornadas #PatientInHTA · Nicholas Brooke
Jornadas #PatientInHTA · Nicholas BrookeJornadas #PatientInHTA · Nicholas Brooke
Jornadas #PatientInHTA · Nicholas Brooke
 
Jornadas #PatientInHTA · Paloma Casado
Jornadas #PatientInHTA · Paloma CasadoJornadas #PatientInHTA · Paloma Casado
Jornadas #PatientInHTA · Paloma Casado
 
Jornadas #PatientInHTA · Gaizka Benguria
Jornadas #PatientInHTA · Gaizka BenguriaJornadas #PatientInHTA · Gaizka Benguria
Jornadas #PatientInHTA · Gaizka Benguria
 
Good practices in patient involvement in HTA
Good practices in patient involvement in HTAGood practices in patient involvement in HTA
Good practices in patient involvement in HTA
 
Is there patient involvement in HTA? Can patients influence HTA decision making?
Is there patient involvement in HTA? Can patients influence HTA decision making?Is there patient involvement in HTA? Can patients influence HTA decision making?
Is there patient involvement in HTA? Can patients influence HTA decision making?
 
Consumer involvement: Patient views on participation in HTA
Consumer involvement: Patient views on participation in HTAConsumer involvement: Patient views on participation in HTA
Consumer involvement: Patient views on participation in HTA
 
Drug development and treatment strategies may go hand in hand.
Drug development and treatment strategies may go hand in hand.Drug development and treatment strategies may go hand in hand.
Drug development and treatment strategies may go hand in hand.
 
Patients’ voice in access decisions
Patients’ voice in access decisionsPatients’ voice in access decisions
Patients’ voice in access decisions
 
Health Technology Assessment
Health Technology AssessmentHealth Technology Assessment
Health Technology Assessment
 
Observatory of Innovation in healthcare management in Catalonia (OIGS)
Observatory of Innovation in healthcare management in Catalonia (OIGS)Observatory of Innovation in healthcare management in Catalonia (OIGS)
Observatory of Innovation in healthcare management in Catalonia (OIGS)
 
Shared Decision Making in Health in Catalonia
Shared Decision Making in Health in CataloniaShared Decision Making in Health in Catalonia
Shared Decision Making in Health in Catalonia
 
Essencial Project in Catalonia. Avoiding low-value clinical practices
Essencial Project in Catalonia. Avoiding low-value clinical practicesEssencial Project in Catalonia. Avoiding low-value clinical practices
Essencial Project in Catalonia. Avoiding low-value clinical practices
 
Webinar: Guidance on Industry-led R&D
Webinar: Guidance on Industry-led R&DWebinar: Guidance on Industry-led R&D
Webinar: Guidance on Industry-led R&D
 
Tripartite dimension of interaction of patients, regulators and industry (Jan...
Tripartite dimension of interaction of patients, regulators and industry (Jan...Tripartite dimension of interaction of patients, regulators and industry (Jan...
Tripartite dimension of interaction of patients, regulators and industry (Jan...
 
In Electronic Health Records We Trust - IPPOSI Outcome Report - March 2017
In Electronic Health Records We Trust - IPPOSI Outcome Report - March 2017In Electronic Health Records We Trust - IPPOSI Outcome Report - March 2017
In Electronic Health Records We Trust - IPPOSI Outcome Report - March 2017
 
Brookman final last 5 nov
Brookman final last 5 nov Brookman final last 5 nov
Brookman final last 5 nov
 
The patients' contribution to ethics committees in the Netherlands
The patients' contribution to ethics committees in the NetherlandsThe patients' contribution to ethics committees in the Netherlands
The patients' contribution to ethics committees in the Netherlands
 
How to communicate scientific and medical information to patients, advocates ...
How to communicate scientific and medical information to patients, advocates ...How to communicate scientific and medical information to patients, advocates ...
How to communicate scientific and medical information to patients, advocates ...
 
Mini-Course Starter Kits
Mini-Course Starter KitsMini-Course Starter Kits
Mini-Course Starter Kits
 

Similar to Jornadas #PatientInHTA · François Houyez

1 engaging with ema methodology and support
1 engaging with ema methodology and support1 engaging with ema methodology and support
1 engaging with ema methodology and support
EURORDIS Rare Diseases Europe
 
Engaging with EMA methodology
Engaging with EMA methodology Engaging with EMA methodology
Engaging with EMA methodology
EURORDIS Rare Diseases Europe
 
Changes in patient organisations - and how this changes the medical world
Changes in patient organisations - and how this changes the medical worldChanges in patient organisations - and how this changes the medical world
Changes in patient organisations - and how this changes the medical world
jangeissler
 
EUPATI 2013 Conference: Vision on Patient involvement in medicines R&D: Here...
EUPATI 2013 Conference: Vision on Patient involvement in medicines R&D: Here...EUPATI 2013 Conference: Vision on Patient involvement in medicines R&D: Here...
EUPATI 2013 Conference: Vision on Patient involvement in medicines R&D: Here...
EUPATI
 
Changes in patient organisations - and how this changes the medical world
Changes in patient organisations - and how this changes the medical worldChanges in patient organisations - and how this changes the medical world
Changes in patient organisations - and how this changes the medical world
jangeissler
 
Patient involvement
Patient involvementPatient involvement
Patient involvement
EURORDIS Rare Diseases Europe
 
An introduction to the EMA
An introduction to the EMAAn introduction to the EMA
An introduction to the EMA
EURORDIS Rare Diseases Europe
 
STREAM TWO: Daniel O’Connor, European regulatory approaches to drugs for rare...
STREAM TWO: Daniel O’Connor, European regulatory approaches to drugs for rare...STREAM TWO: Daniel O’Connor, European regulatory approaches to drugs for rare...
STREAM TWO: Daniel O’Connor, European regulatory approaches to drugs for rare...
Canadian Organization for Rare Disorders
 
EPIS meeting - Dr Derick Mitchell - October 2017
EPIS meeting - Dr Derick Mitchell - October 2017EPIS meeting - Dr Derick Mitchell - October 2017
EPIS meeting - Dr Derick Mitchell - October 2017
ipposi
 
0107 Jan Geissler - How drug development works and elements of trial protocols
0107 Jan Geissler - How drug development works and elements of trial protocols0107 Jan Geissler - How drug development works and elements of trial protocols
0107 Jan Geissler - How drug development works and elements of trial protocols
Workgroup of European Cancer Patient Advocacy Networks
 
EUPATI Status Update at EMA PCWP Meeting, 26 Nov 2015
EUPATI Status Update at EMA PCWP Meeting, 26 Nov 2015EUPATI Status Update at EMA PCWP Meeting, 26 Nov 2015
EUPATI Status Update at EMA PCWP Meeting, 26 Nov 2015
jangeissler
 
Patient Advocates in Cancer Research: European Patients’ Perspective - Jan ...
Patient Advocates in Cancer Research: European Patients’ Perspective - Jan ...Patient Advocates in Cancer Research: European Patients’ Perspective - Jan ...
Patient Advocates in Cancer Research: European Patients’ Perspective - Jan ...
patvocates
 
MoCA
MoCAMoCA
2023 ISPOR Patient Representatives Roundtable - Europe
2023 ISPOR Patient Representatives Roundtable - Europe2023 ISPOR Patient Representatives Roundtable - Europe
2023 ISPOR Patient Representatives Roundtable - Europe
ipposi
 
eHealth and patient outcomes
eHealth and patient outcomeseHealth and patient outcomes
eHealth and patient outcomes
Anna Kotzeva
 
EUPATI 2013 Conference: Building Knowledge & Competences for Patients’ Involv...
EUPATI 2013 Conference: Building Knowledge & Competences for Patients’ Involv...EUPATI 2013 Conference: Building Knowledge & Competences for Patients’ Involv...
EUPATI 2013 Conference: Building Knowledge & Competences for Patients’ Involv...
EUPATI
 
Joint Rare Diseases / Orphan Medicinal Products Task Force
Joint Rare Diseases / Orphan Medicinal Products Task ForceJoint Rare Diseases / Orphan Medicinal Products Task Force
Joint Rare Diseases / Orphan Medicinal Products Task Force
Dutch Orphan Drug Network
 
Education of patients (advocates) on R&D: European Patients’ Academy EUPATI...
Education of patients (advocates) on R&D: European Patients’ Academy EUPATI...Education of patients (advocates) on R&D: European Patients’ Academy EUPATI...
Education of patients (advocates) on R&D: European Patients’ Academy EUPATI...
jangeissler
 
Thomas Sannié, Association francaise des hemophilies
Thomas Sannié, Association francaise des hemophiliesThomas Sannié, Association francaise des hemophilies
Thomas Sannié, Association francaise des hemophilies
Cittadinanzattiva onlus
 
CURE OM Patient Registry Update - 2019 CURE OM Symposium
CURE OM Patient Registry Update - 2019 CURE OM SymposiumCURE OM Patient Registry Update - 2019 CURE OM Symposium
CURE OM Patient Registry Update - 2019 CURE OM Symposium
Melanoma Research Foundation
 

Similar to Jornadas #PatientInHTA · François Houyez (20)

1 engaging with ema methodology and support
1 engaging with ema methodology and support1 engaging with ema methodology and support
1 engaging with ema methodology and support
 
Engaging with EMA methodology
Engaging with EMA methodology Engaging with EMA methodology
Engaging with EMA methodology
 
Changes in patient organisations - and how this changes the medical world
Changes in patient organisations - and how this changes the medical worldChanges in patient organisations - and how this changes the medical world
Changes in patient organisations - and how this changes the medical world
 
EUPATI 2013 Conference: Vision on Patient involvement in medicines R&D: Here...
EUPATI 2013 Conference: Vision on Patient involvement in medicines R&D: Here...EUPATI 2013 Conference: Vision on Patient involvement in medicines R&D: Here...
EUPATI 2013 Conference: Vision on Patient involvement in medicines R&D: Here...
 
Changes in patient organisations - and how this changes the medical world
Changes in patient organisations - and how this changes the medical worldChanges in patient organisations - and how this changes the medical world
Changes in patient organisations - and how this changes the medical world
 
Patient involvement
Patient involvementPatient involvement
Patient involvement
 
An introduction to the EMA
An introduction to the EMAAn introduction to the EMA
An introduction to the EMA
 
STREAM TWO: Daniel O’Connor, European regulatory approaches to drugs for rare...
STREAM TWO: Daniel O’Connor, European regulatory approaches to drugs for rare...STREAM TWO: Daniel O’Connor, European regulatory approaches to drugs for rare...
STREAM TWO: Daniel O’Connor, European regulatory approaches to drugs for rare...
 
EPIS meeting - Dr Derick Mitchell - October 2017
EPIS meeting - Dr Derick Mitchell - October 2017EPIS meeting - Dr Derick Mitchell - October 2017
EPIS meeting - Dr Derick Mitchell - October 2017
 
0107 Jan Geissler - How drug development works and elements of trial protocols
0107 Jan Geissler - How drug development works and elements of trial protocols0107 Jan Geissler - How drug development works and elements of trial protocols
0107 Jan Geissler - How drug development works and elements of trial protocols
 
EUPATI Status Update at EMA PCWP Meeting, 26 Nov 2015
EUPATI Status Update at EMA PCWP Meeting, 26 Nov 2015EUPATI Status Update at EMA PCWP Meeting, 26 Nov 2015
EUPATI Status Update at EMA PCWP Meeting, 26 Nov 2015
 
Patient Advocates in Cancer Research: European Patients’ Perspective - Jan ...
Patient Advocates in Cancer Research: European Patients’ Perspective - Jan ...Patient Advocates in Cancer Research: European Patients’ Perspective - Jan ...
Patient Advocates in Cancer Research: European Patients’ Perspective - Jan ...
 
MoCA
MoCAMoCA
MoCA
 
2023 ISPOR Patient Representatives Roundtable - Europe
2023 ISPOR Patient Representatives Roundtable - Europe2023 ISPOR Patient Representatives Roundtable - Europe
2023 ISPOR Patient Representatives Roundtable - Europe
 
eHealth and patient outcomes
eHealth and patient outcomeseHealth and patient outcomes
eHealth and patient outcomes
 
EUPATI 2013 Conference: Building Knowledge & Competences for Patients’ Involv...
EUPATI 2013 Conference: Building Knowledge & Competences for Patients’ Involv...EUPATI 2013 Conference: Building Knowledge & Competences for Patients’ Involv...
EUPATI 2013 Conference: Building Knowledge & Competences for Patients’ Involv...
 
Joint Rare Diseases / Orphan Medicinal Products Task Force
Joint Rare Diseases / Orphan Medicinal Products Task ForceJoint Rare Diseases / Orphan Medicinal Products Task Force
Joint Rare Diseases / Orphan Medicinal Products Task Force
 
Education of patients (advocates) on R&D: European Patients’ Academy EUPATI...
Education of patients (advocates) on R&D: European Patients’ Academy EUPATI...Education of patients (advocates) on R&D: European Patients’ Academy EUPATI...
Education of patients (advocates) on R&D: European Patients’ Academy EUPATI...
 
Thomas Sannié, Association francaise des hemophilies
Thomas Sannié, Association francaise des hemophiliesThomas Sannié, Association francaise des hemophilies
Thomas Sannié, Association francaise des hemophilies
 
CURE OM Patient Registry Update - 2019 CURE OM Symposium
CURE OM Patient Registry Update - 2019 CURE OM SymposiumCURE OM Patient Registry Update - 2019 CURE OM Symposium
CURE OM Patient Registry Update - 2019 CURE OM Symposium
 

More from Instituto Aragonés de Ciencias de la Salud - IACS

Jornadas #PatientInHTA · Gaizka Benguria
Jornadas #PatientInHTA · Gaizka BenguriaJornadas #PatientInHTA · Gaizka Benguria
Jornadas #PatientInHTA · Gaizka Benguria
Instituto Aragonés de Ciencias de la Salud - IACS
 
Sesiones Clínicas de Investigación e Innovación 2017. Huesca
Sesiones Clínicas de Investigación e Innovación 2017. HuescaSesiones Clínicas de Investigación e Innovación 2017. Huesca
Sesiones Clínicas de Investigación e Innovación 2017. Huesca
Instituto Aragonés de Ciencias de la Salud - IACS
 
Tipos de proyectos que deben ser evaluados por un CEIC
Tipos de proyectos que deben ser evaluados por un CEICTipos de proyectos que deben ser evaluados por un CEIC
Tipos de proyectos que deben ser evaluados por un CEIC
Instituto Aragonés de Ciencias de la Salud - IACS
 
Tipos de CEI
Tipos de CEITipos de CEI
Marco normativo de los ensayos clínicos. Novedades RD 1090/2015
Marco normativo de los ensayos clínicos. Novedades RD 1090/2015Marco normativo de los ensayos clínicos. Novedades RD 1090/2015
Marco normativo de los ensayos clínicos. Novedades RD 1090/2015
Instituto Aragonés de Ciencias de la Salud - IACS
 
Aspectos críticos en un proyecto de investigación clínica. Consideraciones al...
Aspectos críticos en un proyecto de investigación clínica. Consideraciones al...Aspectos críticos en un proyecto de investigación clínica. Consideraciones al...
Aspectos críticos en un proyecto de investigación clínica. Consideraciones al...
Instituto Aragonés de Ciencias de la Salud - IACS
 
Comités de ética en la investigación con animales
Comités de ética en la investigación con animalesComités de ética en la investigación con animales
Comités de ética en la investigación con animales
Instituto Aragonés de Ciencias de la Salud - IACS
 
Integridad científica
Integridad científicaIntegridad científica
Cómo elaborar una hoja de información para recabar el consentimiento informado
Cómo elaborar una hoja de información para recabar el consentimiento informadoCómo elaborar una hoja de información para recabar el consentimiento informado
Cómo elaborar una hoja de información para recabar el consentimiento informado
Instituto Aragonés de Ciencias de la Salud - IACS
 
Qué hace que una investigación sea ética
Qué hace que una investigación sea éticaQué hace que una investigación sea ética
Qué hace que una investigación sea ética
Instituto Aragonés de Ciencias de la Salud - IACS
 
Biobancos y colecciones de muestras biologicas
Biobancos y colecciones de muestras biologicasBiobancos y colecciones de muestras biologicas
Biobancos y colecciones de muestras biologicas
Instituto Aragonés de Ciencias de la Salud - IACS
 
Aspectos éticos. Novedades RD 1090/2015
Aspectos éticos. Novedades RD 1090/2015Aspectos éticos. Novedades RD 1090/2015
Aspectos éticos. Novedades RD 1090/2015
Instituto Aragonés de Ciencias de la Salud - IACS
 
Manejo de datos en investigacion clinica. Aspectos éticos y legales
Manejo de datos en investigacion clinica. Aspectos éticos y legalesManejo de datos en investigacion clinica. Aspectos éticos y legales
Manejo de datos en investigacion clinica. Aspectos éticos y legales
Instituto Aragonés de Ciencias de la Salud - IACS
 
Compra Publica de Innovacion: Proyecto Piloto "Descontaminacion barranco de B...
Compra Publica de Innovacion: Proyecto Piloto "Descontaminacion barranco de B...Compra Publica de Innovacion: Proyecto Piloto "Descontaminacion barranco de B...
Compra Publica de Innovacion: Proyecto Piloto "Descontaminacion barranco de B...
Instituto Aragonés de Ciencias de la Salud - IACS
 
Compra Pública de Innovacion. Estructura de gobernanza
Compra Pública de Innovacion. Estructura de gobernanzaCompra Pública de Innovacion. Estructura de gobernanza
Compra Pública de Innovacion. Estructura de gobernanza
Instituto Aragonés de Ciencias de la Salud - IACS
 
Estrategia de Compra Publica de Innovación en Aragón: Formación
Estrategia de Compra Publica de Innovación en Aragón: FormaciónEstrategia de Compra Publica de Innovación en Aragón: Formación
Estrategia de Compra Publica de Innovación en Aragón: Formación
Instituto Aragonés de Ciencias de la Salud - IACS
 
8. Evaluar tecnologías basadas en mHealth. ¿Es posible un marco común de eval...
8. Evaluar tecnologías basadas en mHealth. ¿Es posible un marco común de eval...8. Evaluar tecnologías basadas en mHealth. ¿Es posible un marco común de eval...
8. Evaluar tecnologías basadas en mHealth. ¿Es posible un marco común de eval...
Instituto Aragonés de Ciencias de la Salud - IACS
 
7. Análisis coste-efectividad: ¿un umbral para determinar si el coste de una ...
7. Análisis coste-efectividad: ¿un umbral para determinar si el coste de una ...7. Análisis coste-efectividad: ¿un umbral para determinar si el coste de una ...
7. Análisis coste-efectividad: ¿un umbral para determinar si el coste de una ...
Instituto Aragonés de Ciencias de la Salud - IACS
 

More from Instituto Aragonés de Ciencias de la Salud - IACS (18)

Jornadas #PatientInHTA · Gaizka Benguria
Jornadas #PatientInHTA · Gaizka BenguriaJornadas #PatientInHTA · Gaizka Benguria
Jornadas #PatientInHTA · Gaizka Benguria
 
Sesiones Clínicas de Investigación e Innovación 2017. Huesca
Sesiones Clínicas de Investigación e Innovación 2017. HuescaSesiones Clínicas de Investigación e Innovación 2017. Huesca
Sesiones Clínicas de Investigación e Innovación 2017. Huesca
 
Tipos de proyectos que deben ser evaluados por un CEIC
Tipos de proyectos que deben ser evaluados por un CEICTipos de proyectos que deben ser evaluados por un CEIC
Tipos de proyectos que deben ser evaluados por un CEIC
 
Tipos de CEI
Tipos de CEITipos de CEI
Tipos de CEI
 
Marco normativo de los ensayos clínicos. Novedades RD 1090/2015
Marco normativo de los ensayos clínicos. Novedades RD 1090/2015Marco normativo de los ensayos clínicos. Novedades RD 1090/2015
Marco normativo de los ensayos clínicos. Novedades RD 1090/2015
 
Aspectos críticos en un proyecto de investigación clínica. Consideraciones al...
Aspectos críticos en un proyecto de investigación clínica. Consideraciones al...Aspectos críticos en un proyecto de investigación clínica. Consideraciones al...
Aspectos críticos en un proyecto de investigación clínica. Consideraciones al...
 
Comités de ética en la investigación con animales
Comités de ética en la investigación con animalesComités de ética en la investigación con animales
Comités de ética en la investigación con animales
 
Integridad científica
Integridad científicaIntegridad científica
Integridad científica
 
Cómo elaborar una hoja de información para recabar el consentimiento informado
Cómo elaborar una hoja de información para recabar el consentimiento informadoCómo elaborar una hoja de información para recabar el consentimiento informado
Cómo elaborar una hoja de información para recabar el consentimiento informado
 
Qué hace que una investigación sea ética
Qué hace que una investigación sea éticaQué hace que una investigación sea ética
Qué hace que una investigación sea ética
 
Biobancos y colecciones de muestras biologicas
Biobancos y colecciones de muestras biologicasBiobancos y colecciones de muestras biologicas
Biobancos y colecciones de muestras biologicas
 
Aspectos éticos. Novedades RD 1090/2015
Aspectos éticos. Novedades RD 1090/2015Aspectos éticos. Novedades RD 1090/2015
Aspectos éticos. Novedades RD 1090/2015
 
Manejo de datos en investigacion clinica. Aspectos éticos y legales
Manejo de datos en investigacion clinica. Aspectos éticos y legalesManejo de datos en investigacion clinica. Aspectos éticos y legales
Manejo de datos en investigacion clinica. Aspectos éticos y legales
 
Compra Publica de Innovacion: Proyecto Piloto "Descontaminacion barranco de B...
Compra Publica de Innovacion: Proyecto Piloto "Descontaminacion barranco de B...Compra Publica de Innovacion: Proyecto Piloto "Descontaminacion barranco de B...
Compra Publica de Innovacion: Proyecto Piloto "Descontaminacion barranco de B...
 
Compra Pública de Innovacion. Estructura de gobernanza
Compra Pública de Innovacion. Estructura de gobernanzaCompra Pública de Innovacion. Estructura de gobernanza
Compra Pública de Innovacion. Estructura de gobernanza
 
Estrategia de Compra Publica de Innovación en Aragón: Formación
Estrategia de Compra Publica de Innovación en Aragón: FormaciónEstrategia de Compra Publica de Innovación en Aragón: Formación
Estrategia de Compra Publica de Innovación en Aragón: Formación
 
8. Evaluar tecnologías basadas en mHealth. ¿Es posible un marco común de eval...
8. Evaluar tecnologías basadas en mHealth. ¿Es posible un marco común de eval...8. Evaluar tecnologías basadas en mHealth. ¿Es posible un marco común de eval...
8. Evaluar tecnologías basadas en mHealth. ¿Es posible un marco común de eval...
 
7. Análisis coste-efectividad: ¿un umbral para determinar si el coste de una ...
7. Análisis coste-efectividad: ¿un umbral para determinar si el coste de una ...7. Análisis coste-efectividad: ¿un umbral para determinar si el coste de una ...
7. Análisis coste-efectividad: ¿un umbral para determinar si el coste de una ...
 

Recently uploaded

Cervical Disc Arthroplasty ORSI 2024.pptx
Cervical Disc Arthroplasty ORSI 2024.pptxCervical Disc Arthroplasty ORSI 2024.pptx
Cervical Disc Arthroplasty ORSI 2024.pptx
LEFLOT Jean-Louis
 
NAVIGATING THE HORIZONS OF TIME LAPSE EMBRYO MONITORING.pdf
NAVIGATING THE HORIZONS OF TIME LAPSE EMBRYO MONITORING.pdfNAVIGATING THE HORIZONS OF TIME LAPSE EMBRYO MONITORING.pdf
NAVIGATING THE HORIZONS OF TIME LAPSE EMBRYO MONITORING.pdf
Rahul Sen
 
Tele Optometry (kunj'sppt) / Basics of tele optometry.
Tele Optometry (kunj'sppt) / Basics of tele optometry.Tele Optometry (kunj'sppt) / Basics of tele optometry.
Tele Optometry (kunj'sppt) / Basics of tele optometry.
Kunj Vihari
 
Ophthalmic drugs latest. Xxxxxxzxxxxxx.pdf
Ophthalmic drugs latest. Xxxxxxzxxxxxx.pdfOphthalmic drugs latest. Xxxxxxzxxxxxx.pdf
Ophthalmic drugs latest. Xxxxxxzxxxxxx.pdf
MuhammadMuneer49
 
K CỔ TỬ CUNG.pdf tự ghi chép, chữ hơi xấu
K CỔ TỬ CUNG.pdf tự ghi chép, chữ hơi xấuK CỔ TỬ CUNG.pdf tự ghi chép, chữ hơi xấu
K CỔ TỬ CUNG.pdf tự ghi chép, chữ hơi xấu
HongBiThi1
 
Pollen and Fungal allergy: aeroallergy.pdf
Pollen and Fungal allergy: aeroallergy.pdfPollen and Fungal allergy: aeroallergy.pdf
Pollen and Fungal allergy: aeroallergy.pdf
Chulalongkorn Allergy and Clinical Immunology Research Group
 
Hemodialysis: Chapter 5, Dialyzers Overview - Dr.Gawad
Hemodialysis: Chapter 5, Dialyzers Overview - Dr.GawadHemodialysis: Chapter 5, Dialyzers Overview - Dr.Gawad
Hemodialysis: Chapter 5, Dialyzers Overview - Dr.Gawad
NephroTube - Dr.Gawad
 
Skin Diseases That Happen During Summer.
 Skin Diseases That Happen During Summer. Skin Diseases That Happen During Summer.
Skin Diseases That Happen During Summer.
Gokuldas Hospital
 
Know the difference between Endodontics and Orthodontics.
Know the difference between Endodontics and Orthodontics.Know the difference between Endodontics and Orthodontics.
Know the difference between Endodontics and Orthodontics.
Gokuldas Hospital
 
June 2024 Oncology Cartoons By Dr Kanhu Charan Patro
June 2024 Oncology Cartoons By Dr Kanhu Charan PatroJune 2024 Oncology Cartoons By Dr Kanhu Charan Patro
June 2024 Oncology Cartoons By Dr Kanhu Charan Patro
Kanhu Charan
 
pharmacology for dummies free pdf download.pdf
pharmacology for dummies free pdf download.pdfpharmacology for dummies free pdf download.pdf
pharmacology for dummies free pdf download.pdf
KerlynIgnacio
 
Artificial Intelligence Symposium (THAIS)
Artificial Intelligence Symposium (THAIS)Artificial Intelligence Symposium (THAIS)
Artificial Intelligence Symposium (THAIS)
Josep Vidal-Alaball
 
Post-Menstrual Smell- When to Suspect Vaginitis.pptx
Post-Menstrual Smell- When to Suspect Vaginitis.pptxPost-Menstrual Smell- When to Suspect Vaginitis.pptx
Post-Menstrual Smell- When to Suspect Vaginitis.pptx
FFragrant
 
Physical demands in sports - WCSPT Oslo 2024
Physical demands in sports - WCSPT Oslo 2024Physical demands in sports - WCSPT Oslo 2024
Physical demands in sports - WCSPT Oslo 2024
Torstein Dalen-Lorentsen
 
Recent advances on Cervical cancer .pptx
Recent advances on Cervical cancer .pptxRecent advances on Cervical cancer .pptx
Recent advances on Cervical cancer .pptx
DrGirishJHoogar
 
CHEMOTHERAPY_RDP_CHAPTER 2 _LEPROSY.pdf1
CHEMOTHERAPY_RDP_CHAPTER 2 _LEPROSY.pdf1CHEMOTHERAPY_RDP_CHAPTER 2 _LEPROSY.pdf1
CHEMOTHERAPY_RDP_CHAPTER 2 _LEPROSY.pdf1
rishi2789
 
Breast cancer: Post menopausal endocrine therapy
Breast cancer: Post menopausal endocrine therapyBreast cancer: Post menopausal endocrine therapy
Breast cancer: Post menopausal endocrine therapy
Dr. Sumit KUMAR
 
Top Travel Vaccinations in Manchester
Top Travel Vaccinations in ManchesterTop Travel Vaccinations in Manchester
Top Travel Vaccinations in Manchester
NX Healthcare
 
Lecture 6 -- Memory 2015.pptlearning occurs when a stimulus (unconditioned st...
Lecture 6 -- Memory 2015.pptlearning occurs when a stimulus (unconditioned st...Lecture 6 -- Memory 2015.pptlearning occurs when a stimulus (unconditioned st...
Lecture 6 -- Memory 2015.pptlearning occurs when a stimulus (unconditioned st...
AyushGadhvi1
 
Osteoporosis - Definition , Evaluation and Management .pdf
Osteoporosis - Definition , Evaluation and Management .pdfOsteoporosis - Definition , Evaluation and Management .pdf
Osteoporosis - Definition , Evaluation and Management .pdf
Jim Jacob Roy
 

Recently uploaded (20)

Cervical Disc Arthroplasty ORSI 2024.pptx
Cervical Disc Arthroplasty ORSI 2024.pptxCervical Disc Arthroplasty ORSI 2024.pptx
Cervical Disc Arthroplasty ORSI 2024.pptx
 
NAVIGATING THE HORIZONS OF TIME LAPSE EMBRYO MONITORING.pdf
NAVIGATING THE HORIZONS OF TIME LAPSE EMBRYO MONITORING.pdfNAVIGATING THE HORIZONS OF TIME LAPSE EMBRYO MONITORING.pdf
NAVIGATING THE HORIZONS OF TIME LAPSE EMBRYO MONITORING.pdf
 
Tele Optometry (kunj'sppt) / Basics of tele optometry.
Tele Optometry (kunj'sppt) / Basics of tele optometry.Tele Optometry (kunj'sppt) / Basics of tele optometry.
Tele Optometry (kunj'sppt) / Basics of tele optometry.
 
Ophthalmic drugs latest. Xxxxxxzxxxxxx.pdf
Ophthalmic drugs latest. Xxxxxxzxxxxxx.pdfOphthalmic drugs latest. Xxxxxxzxxxxxx.pdf
Ophthalmic drugs latest. Xxxxxxzxxxxxx.pdf
 
K CỔ TỬ CUNG.pdf tự ghi chép, chữ hơi xấu
K CỔ TỬ CUNG.pdf tự ghi chép, chữ hơi xấuK CỔ TỬ CUNG.pdf tự ghi chép, chữ hơi xấu
K CỔ TỬ CUNG.pdf tự ghi chép, chữ hơi xấu
 
Pollen and Fungal allergy: aeroallergy.pdf
Pollen and Fungal allergy: aeroallergy.pdfPollen and Fungal allergy: aeroallergy.pdf
Pollen and Fungal allergy: aeroallergy.pdf
 
Hemodialysis: Chapter 5, Dialyzers Overview - Dr.Gawad
Hemodialysis: Chapter 5, Dialyzers Overview - Dr.GawadHemodialysis: Chapter 5, Dialyzers Overview - Dr.Gawad
Hemodialysis: Chapter 5, Dialyzers Overview - Dr.Gawad
 
Skin Diseases That Happen During Summer.
 Skin Diseases That Happen During Summer. Skin Diseases That Happen During Summer.
Skin Diseases That Happen During Summer.
 
Know the difference between Endodontics and Orthodontics.
Know the difference between Endodontics and Orthodontics.Know the difference between Endodontics and Orthodontics.
Know the difference between Endodontics and Orthodontics.
 
June 2024 Oncology Cartoons By Dr Kanhu Charan Patro
June 2024 Oncology Cartoons By Dr Kanhu Charan PatroJune 2024 Oncology Cartoons By Dr Kanhu Charan Patro
June 2024 Oncology Cartoons By Dr Kanhu Charan Patro
 
pharmacology for dummies free pdf download.pdf
pharmacology for dummies free pdf download.pdfpharmacology for dummies free pdf download.pdf
pharmacology for dummies free pdf download.pdf
 
Artificial Intelligence Symposium (THAIS)
Artificial Intelligence Symposium (THAIS)Artificial Intelligence Symposium (THAIS)
Artificial Intelligence Symposium (THAIS)
 
Post-Menstrual Smell- When to Suspect Vaginitis.pptx
Post-Menstrual Smell- When to Suspect Vaginitis.pptxPost-Menstrual Smell- When to Suspect Vaginitis.pptx
Post-Menstrual Smell- When to Suspect Vaginitis.pptx
 
Physical demands in sports - WCSPT Oslo 2024
Physical demands in sports - WCSPT Oslo 2024Physical demands in sports - WCSPT Oslo 2024
Physical demands in sports - WCSPT Oslo 2024
 
Recent advances on Cervical cancer .pptx
Recent advances on Cervical cancer .pptxRecent advances on Cervical cancer .pptx
Recent advances on Cervical cancer .pptx
 
CHEMOTHERAPY_RDP_CHAPTER 2 _LEPROSY.pdf1
CHEMOTHERAPY_RDP_CHAPTER 2 _LEPROSY.pdf1CHEMOTHERAPY_RDP_CHAPTER 2 _LEPROSY.pdf1
CHEMOTHERAPY_RDP_CHAPTER 2 _LEPROSY.pdf1
 
Breast cancer: Post menopausal endocrine therapy
Breast cancer: Post menopausal endocrine therapyBreast cancer: Post menopausal endocrine therapy
Breast cancer: Post menopausal endocrine therapy
 
Top Travel Vaccinations in Manchester
Top Travel Vaccinations in ManchesterTop Travel Vaccinations in Manchester
Top Travel Vaccinations in Manchester
 
Lecture 6 -- Memory 2015.pptlearning occurs when a stimulus (unconditioned st...
Lecture 6 -- Memory 2015.pptlearning occurs when a stimulus (unconditioned st...Lecture 6 -- Memory 2015.pptlearning occurs when a stimulus (unconditioned st...
Lecture 6 -- Memory 2015.pptlearning occurs when a stimulus (unconditioned st...
 
Osteoporosis - Definition , Evaluation and Management .pdf
Osteoporosis - Definition , Evaluation and Management .pdfOsteoporosis - Definition , Evaluation and Management .pdf
Osteoporosis - Definition , Evaluation and Management .pdf
 

Jornadas #PatientInHTA · François Houyez

  • 1. Director of Treatment Information and Access European Organisation for Rare Diseases Zaragoza, 27-28 April 2017 PATIENTS’ AND CONSUMERS’ WORKING WITH THE EUROPEAN MEDICINES AGENCY François Houÿez
  • 2. Disclaimers EURORDIS is one of the signatories of the “Code of Practice between Patients’ Organisations and the Healthcare Industry” Read here
  • 3. Disclaimers EURORDIS is one of the signatories of the “Code of Practice between Patients’ Organisations and the Healthcare Industry” Read here
  • 4. EMA started its activities in 1995 April 1996: CPMP met with 6 patients’ representatives • 2002: workshop on how to work together • 2006: the framework of interaction with patients and consumers adopted by EMA • 2016: 770 occasions where patients and consumers were involved in EMA activitie Moving away from a situation where 12 Member States could take different decisions on marketing authorisations To a European Union with a marketing authorisation valid in 28 Member States Norway, Iceland and Liechtenstein, serving 508 millions citizens • With same product name, same information for patients and healthcare professionals: all documents for the public are reviewed by patient and consumer
  • 5. When to consult with patients? Horizon scanning
  • 6. How to evaluate medicines? Do we have the right outcome measurement tools? Patients participate in guidelines development
  • 7. A framework of interaction developed with the Patients’ and Consumers’ Working Party (see more here): Guidance and policy adopted by the EMA management board The role of patients as members of the EMA human scientific committees Pilot phase to involve patients in benefit / risk discussions at CHMP meetings Reporting EMA’s interaction with patients, consumers, healthcare professionals and their organisations - 2015 Rules Criteria to be fulfilled by patients’ and consumers’ organisations involved in EMA activities Evaluation of financial information from patients’ consumers’ and healthcare professionals’ organisations for assessment of EMA ‘eligibility’ Training strategy Involvement of patient representatives in scientific advice procedures at the EMA
  • 8. 2016: the PCWP celebrating its 10th anniversary!
  • 9. Involvement across different activities 2009-2016 9
  • 10. Necessary steps to involve patients
  • 11. How does it mean in terms of days for EURORDIS? Members of scientific committees at the EMA (volunteers) 7 (COMP, PDCO,CAT, PCWP) Total days spent in 2016: 286 Average days / volunteer: 41 / year (7-88) Members of EURORDIS staff working on patient engagement / EMA 1.5 FTE 3 different staff Member of EMA Management board 1 Total days : 22 / year Number of EMA SA/PA procedures 386 (2015) =154 reviews for EURORDIS (orphan Numbers of patients invited to scientific advice 76 (2015) Of whom 25 EURORDIS patients Number of patients trained (EURORDIS Summer School) since 2008 300+ 1 FTE for training programme Other services: Contact database maintenance, e-learning tools development, administration, communication to member
  • 12. EURORDIS Summer School (training): how to minimise the influence of industry? Example of a case study CHMP invited 2 patients to an oral explanation with MAA Two hours before CHMP meeting, MAA, patients, clinicians who had been working on the product for years all together met in a hotel Mentor explained the issue advised not to have the meeting finally left the room and informed the EMA about the incident • What happened • Impact • CHMP agreed to have the patients they invited in the room • CHMP chair explained the situation before the meeting started • CHMP agreed to proceed • It could have stopped there
  • 13. How? Informing on the evaluation once made? Consulting during the evaluation? Involving when communicating decision? 1 person? 2? 5? Patient expert own opinion, or organisation views? Patient preference elicitations Extrapolation? 30 or 300 patients? More? Other deliberative methods? Panel of patients ? Citizens’ jury ? Focus groups? Face to face? Video-conferences? In writing? Patient who participated in clinical trial, or not? Time for more pilots
  • 14. Success factors High quality dialogue Patients are considered on the same level than other experts A dedicated unit at the EMA for stakeholders’ involvement Training materials (videos, face to face…) Adequate resources and budget Costs covered Daily allowance, doubled for volunteers Rules for involvement defined together with stakeholders For all aspects Revised as often as needed “With a high qualit dialogue, patients a regulators can onl agree.” Jean-Michel Alexandre Former CHMP chair, E
  • 15. Patients’ satisfaction for invitations at CHMP oral explanations with the applicant (14 patients involved, 6 meetings)
  • 16. Barriers, obstacles Patients’ advocates are few (availability, language) Time commitment (e.g. committee members) and time spent unpaid Advising the developer or advising the regulators (CofI policy)? Not both Rapid regulatory timelines, and the time it takes to involve patients Younger patients gather on social networks, no longer in registered organisations with statutes, general assemblies, board of directors etc. No impact assessment when defining a new role for civil society (EU legislation making): training needs, contact database maintenance, support, guidance…
  • 17. The role of patients in EU legislation on medicines REGULATION EC 726/2004 establishing EMA Article 65 - The Management Board: Two representatives of patients' organisations shall be appointed by Council in consultation with EP Article 14 - The package leaflet shall reflect the results of consultations with target patient groups REGULATION EC 141/2000 on orphan medicinal products Article 4 – COMP: Three members nominated by the Commission to represent patients' organisations REGULATION EC 1901/2006 on medicinal products for paediatric use Article 4 - Paediatric committee: Three members and three alternates appointed by the Commission in order to represent patient associations REGULATION EC 1394/2007 on advanced therapy medicinal products Article 21 - Committee for Advanced Therapies: The Committee for Advanced Therapies shall be composed of two members and two alternates to represent patients associations REGULATION (EU) No 1235/2010 on pharmacovigilance Article 61a: one member and one alternate member in order to represent patient organisations
  • 18. What can be improved? Next steps Public Hearings (PRAC, safety concern and therapeutic context) Updated patient information: graphic visualisation, changes in patient leaflet, videos etc. IMI PREFER project: how to integrate Patient Preferences Elicitations into the evaluation process? PRIME: Priority Medicines – Very early scientific advice, with patients A new status for trained patients participating in CABs/Scientific advice: “patient investigator” To conduct research together E.g “Patient Reporting in the EU: Analysis of EudraVigilance Data”, Drug Safety, (), 1-17, DOI10.1007/s40264-017-0534-1 IMI projects (EUPATI, PROTECT…) Synergies with HTA
  • 19. IMI PROTECT: how to integrate these results in the package leaflet? (June 2017 PCWP meeting) Preferred by 54% by 21% by 25%
  • 20. Patients fully integrated in the centralised procedure for marketing authorisations in the EU Bringing the best expertise in the decision making, including the patients With equal credibility as other experts Impact of patient engagement difficult to assess, but being able to witness the process is a major achievement To conclude
  • 21. Patients at EMA: legal background EU Treaty (Declaration 17 of the Annex) “(…) Transparency of the decision-making process strengthens the democratic nature of the institutions and the public’s confidence in the administration.” 3