This document discusses the involvement of patients and consumers in the work of the European Medicines Agency (EMA). It outlines how:
1) EMA has increasingly involved patients and consumers since 1995, with over 770 instances of involvement in 2016 across activities like scientific committees, benefit-risk discussions, and guidelines development.
2) Involving patients requires resources from organizations like EURORDIS, including staff time equivalent to 1.5 full-time employees and over 280 days volunteered in 2016 on EMA scientific committees.
3) While patient involvement has grown, there are still barriers like availability of advocates, balancing industry relationships, and integrating patient perspectives in regulatory timelines. Further steps are exploring ways to better
Presentation in the framework of the International Conference "10th anniversary of the Spanish Network of Health Technology Assessment Agencies. Towads patient and public engagement in HTA" Zaragoza 27-28 April 2017
Presentation in the framework of the International Conference "10th anniversary of the Spanish Network of Health Technology Assessment Agencies. Towads patient and public engagement in HTA" Zaragoza 27-28 April 2017
Presentation in the framework of the International Conference "10th anniversary of the Spanish Network of Health Technology Assessment Agencies. Towads patient and public engagement in HTA" Zaragoza 27-28 April 2017
Presentation in the framework of the International Conference "10th anniversary of the Spanish Network of Health Technology Assessment Agencies. Towads patient and public engagement in HTA" Zaragoza 27-28 April 2017
Presentation in the framework of the International Conference "10th anniversary of the Spanish Network of Health Technology Assessment Agencies. Towads patient and public engagement in HTA" Zaragoza 27-28 April 2017
Presentation in the framework of the International Conference "10th anniversary of the Spanish Network of Health Technology Assessment Agencies. Towads patient and public engagement in HTA" Zaragoza 27-28 April 2017
Presentation in the framework of the International Conference "10th anniversary of the Spanish Network of Health Technology Assessment Agencies. Towads patient and public engagement in HTA" Zaragoza 27-28 April 2017
Presentation in the framework of the International Conference "10th anniversary of the Spanish Network of Health Technology Assessment Agencies. Towads patient and public engagement in HTA" Zaragoza 27-28 April 2017
Presentation in the framework of the International Conference "10th anniversary of the Spanish Network of Health Technology Assessment Agencies. Towads patient and public engagement in HTA" Zaragoza 27-28 April 2017
Presentation in the framework of the International Conference "10th anniversary of the Spanish Network of Health Technology Assessment Agencies. Towads patient and public engagement in HTA" Zaragoza 27-28 April 2017
Presentation in the framework of the International Conference "10th anniversary of the Spanish Network of Health Technology Assessment Agencies. Towads patient and public engagement in HTA" Zaragoza 27-28 April 2017
Presentation in the framework of the International Conference "10th anniversary of the Spanish Network of Health Technology Assessment Agencies. Towads patient and public engagement in HTA" Zaragoza 27-28 April 2017
Presentation in the framework of the International Conference "10th anniversary of the Spanish Network of Health Technology Assessment Agencies. Towads patient and public engagement in HTA" Zaragoza 27-28 April 2017
Presentation in the framework of the International Conference "10th anniversary of the Spanish Network of Health Technology Assessment Agencies. Towads patient and public engagement in HTA" Zaragoza 27-28 April 2017
Presentation in the framework of the International Conference "10th anniversary of the Spanish Network of Health Technology Assessment Agencies. Towads patient and public engagement in HTA" Zaragoza 27-28 April 2017
Presentation in the framework of the International Conference "10th anniversary of the Spanish Network of Health Technology Assessment Agencies. Towads patient and public engagement in HTA" Zaragoza 27-28 April 2017
Presentation in the framework of the International Conference "10th anniversary of the Spanish Network of Health Technology Assessment Agencies. Towads patient and public engagement in HTA" Zaragoza 27-28 April 2017
Presentation in the framework of the International Conference "10th anniversary of the Spanish Network of Health Technology Assessment Agencies. Towads patient and public engagement in HTA" Zaragoza 27-28 April 2017
Presentation in the framework of the International Conference "10th anniversary of the Spanish Network of Health Technology Assessment Agencies. Towads patient and public engagement in HTA" Zaragoza 27-28 April 2017
Presentation in the framework of the International Conference "10th anniversary of the Spanish Network of Health Technology Assessment Agencies. Towads patient and public engagement in HTA" Zaragoza 27-28 April 2017
Is there patient involvement in HTA? Can patients influence HTA decision making?Kathi Apostolidis
Is HTA purely technical?
drivers for patient involvement in HTA
patient participation or tokenism
medicines do not reach patients due to delays in HTA evaluation
need for harmonized HTA
Drug development and treatment strategies may go hand in hand.EUPATI
How early in the development of a new medicine should the discussion between researchers and patients occur? Can such early strategic interaction really maximize the value of the outcome? In this EUPATI webinar, a pharmaceutical industry researcher and a patient expert will present two cases describing the research questions, their interaction and the outcome of this early collaboration. Join us in listening and discussing two very interesting experiences of successful patient involvement in the development of new medicines.
The Observatory of Innovation in Healthcare Management in Catalonia is the instrument used by the Catalan health system to catalogue the efforts made by organisations in innovating in several spheres of management. See more information at http://oigs.gencat.cat
Shared Decision Making in health (Decisions Compartides) is a project of the Catalan Health Ministry of the Generalitat de Catalunya. Physicians and patients are involved in shared medical decisions. Both parties share information (evidence based information about treatment options, cons and pros, patient preferences and values) and an agreement is reached on the treatment to implement.
Essencial "Adding value to clinical practice" is an initiative which identifies low-value clinical practices and promotes recommendations on how to avoid them.
A very important result from the EUPATI project was the development, broad consultation and final release of guidances on the best approach to interaction of patients with pharmaceutical industry-led medicines R&D, regulatory authorities, ethics committees and HTA agencies. This webinar focuses on the Patient Involvement in Industry-led R&D guidance document
Tripartite dimension of interaction of patients, regulators and industry (Jan...jangeissler
Tripartite dimension of interaction of patients, regulators and industry, presented by Jan Geissler as a scene-setting presentation at the EUPATI Workshop on the interaction of patients, regulators and industry on 20 July 2016 in Berlin
How to communicate scientific and medical information to patients, advocates ...jangeissler
How to communicate scientific and medical information to patients, advocates and caregivers, presented by Jan Geissler at the European Medical Writer's Symposium (EMWA) on 12 May 2016
Presentation in the framework of the International Conference "10th anniversary of the Spanish Network of Health Technology Assessment Agencies. Towads patient and public engagement in HTA" Zaragoza 27-28 April 2017
Presentation in the framework of the International Conference "10th anniversary of the Spanish Network of Health Technology Assessment Agencies. Towads patient and public engagement in HTA" Zaragoza 27-28 April 2017
Presentation in the framework of the International Conference "10th anniversary of the Spanish Network of Health Technology Assessment Agencies. Towads patient and public engagement in HTA" Zaragoza 27-28 April 2017
Presentation in the framework of the International Conference "10th anniversary of the Spanish Network of Health Technology Assessment Agencies. Towads patient and public engagement in HTA" Zaragoza 27-28 April 2017
Is there patient involvement in HTA? Can patients influence HTA decision making?Kathi Apostolidis
Is HTA purely technical?
drivers for patient involvement in HTA
patient participation or tokenism
medicines do not reach patients due to delays in HTA evaluation
need for harmonized HTA
Drug development and treatment strategies may go hand in hand.EUPATI
How early in the development of a new medicine should the discussion between researchers and patients occur? Can such early strategic interaction really maximize the value of the outcome? In this EUPATI webinar, a pharmaceutical industry researcher and a patient expert will present two cases describing the research questions, their interaction and the outcome of this early collaboration. Join us in listening and discussing two very interesting experiences of successful patient involvement in the development of new medicines.
The Observatory of Innovation in Healthcare Management in Catalonia is the instrument used by the Catalan health system to catalogue the efforts made by organisations in innovating in several spheres of management. See more information at http://oigs.gencat.cat
Shared Decision Making in health (Decisions Compartides) is a project of the Catalan Health Ministry of the Generalitat de Catalunya. Physicians and patients are involved in shared medical decisions. Both parties share information (evidence based information about treatment options, cons and pros, patient preferences and values) and an agreement is reached on the treatment to implement.
Essencial "Adding value to clinical practice" is an initiative which identifies low-value clinical practices and promotes recommendations on how to avoid them.
A very important result from the EUPATI project was the development, broad consultation and final release of guidances on the best approach to interaction of patients with pharmaceutical industry-led medicines R&D, regulatory authorities, ethics committees and HTA agencies. This webinar focuses on the Patient Involvement in Industry-led R&D guidance document
Tripartite dimension of interaction of patients, regulators and industry (Jan...jangeissler
Tripartite dimension of interaction of patients, regulators and industry, presented by Jan Geissler as a scene-setting presentation at the EUPATI Workshop on the interaction of patients, regulators and industry on 20 July 2016 in Berlin
How to communicate scientific and medical information to patients, advocates ...jangeissler
How to communicate scientific and medical information to patients, advocates and caregivers, presented by Jan Geissler at the European Medical Writer's Symposium (EMWA) on 12 May 2016
EUPATI 2013 Conference: Vision on Patient involvement in medicines R&D: Here...EUPATI
"Patient involvement in medicines R&D: Here we are, and where we want to be in 2020" by Nicola Bedlington, Executive Director of European Patients' Forum and Coordinator of the EUPATI project, at the EUPATI 2013 Conference on 19 April 2013.
A Rare International Dialogue (Saturday May 11, 2019)
Drivers of Drug Development – Regulatory Collaboration
European regulatory approaches to drugs for rare diseases - Daniel O’Connor, European Medicines Agency/Medicines and Healthcare products Regulatory Agency, UK
EUPATI Status Update at EMA PCWP Meeting, 26 Nov 2015jangeissler
Overview and Status Quo of the European Patients Academy (EUPATI) project, presented by EUPATI Director Jan Geissler at the EMA Patient and Consumer Working Party (PCWP) meeting in London on 26 Nov 2015
Patient Advocates in Cancer Research: European Patients’ Perspective - Jan ...patvocates
Patient Advocates in Cancer Research: European Patients’ Perspective, presented by Jan Geissler (Twitter @jangeissler) at ISOQOL 19th Annual Conference, Budapast, 26 Oct 2012
EUPATI 2013 Conference: Building Knowledge & Competences for Patients’ Involv...EUPATI
EUPATI 2013 Conference: Building Knowledge & Competences for Patients’ Involvement in Medicines R&D, “Bring to life with EUPATI examples”, by Niels Westergaard, PhD, DSc Biopeople, University of Copenhagen, Denmark
Education of patients (advocates) on R&D: European Patients’ Academy EUPATI...jangeissler
"Education of patients (advocates) on R&D: European Patients’ Academy EUPATI", presented by Jan Geissler at the Careum Congress on 18 March 2014 in Basel
Sannié, member of the patients' consultative group of the French National Agency for Medicines and Health Product Safety, France, for European Patients' Rights Day 2017
Intervención de Elisa Puigdomenech Puig (AQuAS) en la III Jornada Red Española de Agencias de Evaluación de Tecnologías Sanitarias y Prestaciones del SNS. Mesa redonda: Grandes retos presentes y futuros
Intervención de Laura Vallejo Torres (SESCS) en la III Jornada Red Española de Agencias de Evaluación de Tecnologías Sanitarias y Prestaciones del SNS. Mesa redonda: Grandes retos presentes y futuros
More from Instituto Aragonés de Ciencias de la Salud - IACS (18)
- Video recording of this lecture in English language: https://youtu.be/lK81BzxMqdo
- Video recording of this lecture in Arabic language: https://youtu.be/Ve4P0COk9OI
- Link to download the book free: https://nephrotube.blogspot.com/p/nephrotube-nephrology-books.html
- Link to NephroTube website: www.NephroTube.com
- Link to NephroTube social media accounts: https://nephrotube.blogspot.com/p/join-nephrotube-on-social-media.html
micro teaching on communication m.sc nursing.pdfAnurag Sharma
Microteaching is a unique model of practice teaching. It is a viable instrument for the. desired change in the teaching behavior or the behavior potential which, in specified types of real. classroom situations, tends to facilitate the achievement of specified types of objectives.
HOT NEW PRODUCT! BIG SALES FAST SHIPPING NOW FROM CHINA!! EU KU DB BK substit...GL Anaacs
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New Directions in Targeted Therapeutic Approaches for Older Adults With Mantl...i3 Health
i3 Health is pleased to make the speaker slides from this activity available for use as a non-accredited self-study or teaching resource.
This slide deck presented by Dr. Kami Maddocks, Professor-Clinical in the Division of Hematology and
Associate Division Director for Ambulatory Operations
The Ohio State University Comprehensive Cancer Center, will provide insight into new directions in targeted therapeutic approaches for older adults with mantle cell lymphoma.
STATEMENT OF NEED
Mantle cell lymphoma (MCL) is a rare, aggressive B-cell non-Hodgkin lymphoma (NHL) accounting for 5% to 7% of all lymphomas. Its prognosis ranges from indolent disease that does not require treatment for years to very aggressive disease, which is associated with poor survival (Silkenstedt et al, 2021). Typically, MCL is diagnosed at advanced stage and in older patients who cannot tolerate intensive therapy (NCCN, 2022). Although recent advances have slightly increased remission rates, recurrence and relapse remain very common, leading to a median overall survival between 3 and 6 years (LLS, 2021). Though there are several effective options, progress is still needed towards establishing an accepted frontline approach for MCL (Castellino et al, 2022). Treatment selection and management of MCL are complicated by the heterogeneity of prognosis, advanced age and comorbidities of patients, and lack of an established standard approach for treatment, making it vital that clinicians be familiar with the latest research and advances in this area. In this activity chaired by Michael Wang, MD, Professor in the Department of Lymphoma & Myeloma at MD Anderson Cancer Center, expert faculty will discuss prognostic factors informing treatment, the promising results of recent trials in new therapeutic approaches, and the implications of treatment resistance in therapeutic selection for MCL.
Target Audience
Hematology/oncology fellows, attending faculty, and other health care professionals involved in the treatment of patients with mantle cell lymphoma (MCL).
Learning Objectives
1.) Identify clinical and biological prognostic factors that can guide treatment decision making for older adults with MCL
2.) Evaluate emerging data on targeted therapeutic approaches for treatment-naive and relapsed/refractory MCL and their applicability to older adults
3.) Assess mechanisms of resistance to targeted therapies for MCL and their implications for treatment selection
TEST BANK for Operations Management, 14th Edition by William J. Stevenson, Ve...kevinkariuki227
TEST BANK for Operations Management, 14th Edition by William J. Stevenson, Verified Chapters 1 - 19, Complete Newest Version.pdf
TEST BANK for Operations Management, 14th Edition by William J. Stevenson, Verified Chapters 1 - 19, Complete Newest Version.pdf
Title: Sense of Smell
Presenter: Dr. Faiza, Assistant Professor of Physiology
Qualifications:
MBBS (Best Graduate, AIMC Lahore)
FCPS Physiology
ICMT, CHPE, DHPE (STMU)
MPH (GC University, Faisalabad)
MBA (Virtual University of Pakistan)
Learning Objectives:
Describe the primary categories of smells and the concept of odor blindness.
Explain the structure and location of the olfactory membrane and mucosa, including the types and roles of cells involved in olfaction.
Describe the pathway and mechanisms of olfactory signal transmission from the olfactory receptors to the brain.
Illustrate the biochemical cascade triggered by odorant binding to olfactory receptors, including the role of G-proteins and second messengers in generating an action potential.
Identify different types of olfactory disorders such as anosmia, hyposmia, hyperosmia, and dysosmia, including their potential causes.
Key Topics:
Olfactory Genes:
3% of the human genome accounts for olfactory genes.
400 genes for odorant receptors.
Olfactory Membrane:
Located in the superior part of the nasal cavity.
Medially: Folds downward along the superior septum.
Laterally: Folds over the superior turbinate and upper surface of the middle turbinate.
Total surface area: 5-10 square centimeters.
Olfactory Mucosa:
Olfactory Cells: Bipolar nerve cells derived from the CNS (100 million), with 4-25 olfactory cilia per cell.
Sustentacular Cells: Produce mucus and maintain ionic and molecular environment.
Basal Cells: Replace worn-out olfactory cells with an average lifespan of 1-2 months.
Bowman’s Gland: Secretes mucus.
Stimulation of Olfactory Cells:
Odorant dissolves in mucus and attaches to receptors on olfactory cilia.
Involves a cascade effect through G-proteins and second messengers, leading to depolarization and action potential generation in the olfactory nerve.
Quality of a Good Odorant:
Small (3-20 Carbon atoms), volatile, water-soluble, and lipid-soluble.
Facilitated by odorant-binding proteins in mucus.
Membrane Potential and Action Potential:
Resting membrane potential: -55mV.
Action potential frequency in the olfactory nerve increases with odorant strength.
Adaptation Towards the Sense of Smell:
Rapid adaptation within the first second, with further slow adaptation.
Psychological adaptation greater than receptor adaptation, involving feedback inhibition from the central nervous system.
Primary Sensations of Smell:
Camphoraceous, Musky, Floral, Pepperminty, Ethereal, Pungent, Putrid.
Odor Detection Threshold:
Examples: Hydrogen sulfide (0.0005 ppm), Methyl-mercaptan (0.002 ppm).
Some toxic substances are odorless at lethal concentrations.
Characteristics of Smell:
Odor blindness for single substances due to lack of appropriate receptor protein.
Behavioral and emotional influences of smell.
Transmission of Olfactory Signals:
From olfactory cells to glomeruli in the olfactory bulb, involving lateral inhibition.
Primitive, less old, and new olfactory systems with different path
Ethanol (CH3CH2OH), or beverage alcohol, is a two-carbon alcohol
that is rapidly distributed in the body and brain. Ethanol alters many
neurochemical systems and has rewarding and addictive properties. It
is the oldest recreational drug and likely contributes to more morbidity,
mortality, and public health costs than all illicit drugs combined. The
5th edition of the Diagnostic and Statistical Manual of Mental Disorders
(DSM-5) integrates alcohol abuse and alcohol dependence into a single
disorder called alcohol use disorder (AUD), with mild, moderate,
and severe subclassifications (American Psychiatric Association, 2013).
In the DSM-5, all types of substance abuse and dependence have been
combined into a single substance use disorder (SUD) on a continuum
from mild to severe. A diagnosis of AUD requires that at least two of
the 11 DSM-5 behaviors be present within a 12-month period (mild
AUD: 2–3 criteria; moderate AUD: 4–5 criteria; severe AUD: 6–11 criteria).
The four main behavioral effects of AUD are impaired control over
drinking, negative social consequences, risky use, and altered physiological
effects (tolerance, withdrawal). This chapter presents an overview
of the prevalence and harmful consequences of AUD in the U.S.,
the systemic nature of the disease, neurocircuitry and stages of AUD,
comorbidities, fetal alcohol spectrum disorders, genetic risk factors, and
pharmacotherapies for AUD.
These simplified slides by Dr. Sidra Arshad present an overview of the non-respiratory functions of the respiratory tract.
Learning objectives:
1. Enlist the non-respiratory functions of the respiratory tract
2. Briefly explain how these functions are carried out
3. Discuss the significance of dead space
4. Differentiate between minute ventilation and alveolar ventilation
5. Describe the cough and sneeze reflexes
Study Resources:
1. Chapter 39, Guyton and Hall Textbook of Medical Physiology, 14th edition
2. Chapter 34, Ganong’s Review of Medical Physiology, 26th edition
3. Chapter 17, Human Physiology by Lauralee Sherwood, 9th edition
4. Non-respiratory functions of the lungs https://academic.oup.com/bjaed/article/13/3/98/278874
Couples presenting to the infertility clinic- Do they really have infertility...Sujoy Dasgupta
Dr Sujoy Dasgupta presented the study on "Couples presenting to the infertility clinic- Do they really have infertility? – The unexplored stories of non-consummation" in the 13th Congress of the Asia Pacific Initiative on Reproduction (ASPIRE 2024) at Manila on 24 May, 2024.
Ozempic: Preoperative Management of Patients on GLP-1 Receptor Agonists Saeid Safari
Preoperative Management of Patients on GLP-1 Receptor Agonists like Ozempic and Semiglutide
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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
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
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