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

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
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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.”
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