The Patient s Voice at the European Medicines Agency (EMA

5th European Patients Rights Day Brussels
Dr Mary Baker, MBE
President, European Brain Council President, European Federation of Neurological Associations

With grateful acknowledgement to: Isabelle Moulon Head of Medical Information, European Medicines Agency

EMA: What does that mean?
The EMA is responsible for:
The evaluation of the Marketing Authorisation applications submitted by pharmaceutical companies, for certain types of products of level Provision of scientific advice on the development medicines Coordination of pharmacovigilance at European (supervision of the medicines on the market) Coordination of the inspection activities Provision of information to the public

The EMA Network
A unique structure:
EMA partners with -More than 40 national competent authorities -4000 EU Experts -European Parliament -European Commission Relation with non-EU regulatory authorities, international health organisations, industry academia, and the general public

Representation of patients at the EMA 

Management Board: 2 representatives of patients organisations Committee for Orphan Medicinal Products (COMP): 2 representatives of patients organisations + 2 observers Paediatric Committee (PDCO): 3 representatives of patients organisations Committee for Advanced Therapy (CAT): 2 representatives EMA Patients and Consumers Working Party (PCWP)   

Other participation of patients to EMA activities
‡ Quality-checking of product information for medicines and EPAR summaries (European Public Assessment Report) ‡ Proactive involvement in guideline preparation ‡ Responding to specific requests from EMA scientific committees, working parties, scientific advisory groups etc

EMA Patients & Consumer Working Party

10 PCWP Members out of 22 eligible Organisations + representatives from scientific committees


4 Meetings a year

‡ Co-chair elected: Nikos Dedes ‡ 1 joint meeting a year with Healthcare Professionals Working Party ‡ 1 meeting a year with all 22 eligible organisations

Criteria to be fulfilled by Patients & Consumers Organisations involved in EMA Activities

‡ Not-for-profit ‡ Focus on patient/consumer care ‡ Representative of patients/consumers

Which are the eligible Patients and Consumers Organisations?
‡ ‡ ‡ ‡ ‡ ‡ ‡ Legitimacy Mission Objectives Activities Representativeness Structure Accountability Transparency

Role of the PCWP
To provide recommendations to the EMA and its Scientific Committees from patients on all matters directly and indirectly related to medicines:
Implementation of the recommendations and proposals for action Provision of information on medicines Development of appropriate communication tools Increasing awareness of patients on the rational use of medicines Training patients and consumers Provision of advice to Scientific Committees Interaction with other interested parties: e.g. healthcare professionals, pharmaceutical industry

Role of the Members
Each member represents his/her organisation:
± He/she is responsible for presenting the organisation s position on the topics discussed ± He/she is responsible for informing his/her organisation about the PCWP s activities

Each member fills in a declaration of interest None of the discussions/topics is confidential

The PCWP Work Plan
Interactions EMA Patients and Consumers Organisations
Explore further involvement

Pharmacovigilance Support to EC Strategy to Strengthen the Community System
Direct patients reporting of adverse drug reactions Benefit/risk communication

Transparency and dissemination of information
To provide input on ongoing restructuration of the EMA website

Product information
Review of Patients leaflets and EPAR summaries

Involvement of patients during the evaluation process
Ad hoc involvement:
Consultation of PCWP on the wording of the Package Leaflets (e.g. NSAIDs): how to express the warnings on cardiovascular risk. ‡ Consultation of patients and thalidomide victims during the evaluation of Revlimid (lenalinomide) on the Package Leaflet and the risk management plan. ‡ Consultation of patients: - during the evaluation of Tysabri (natalizumab) - for the recall of Viracept (nelfinavir)

Other activities in which patients & consumers are involved
‡ Workshop (User Testing, Transparency) ‡ Website design (corporate & paediatric) ‡ EudraPharm development ‡ Observer in the Pharmacovigilance Working Party

Benefit of involving patients
‡ They bring unique input and a different perspective highlighting real-life implications of regulatory decisions ‡ Their contribution is very often taken into account and improves the regulatory outcome ‡ Their involvement brings more transparency in the regulatory process and as a consequence helps to build trust in the regulatory process

Issues to be considered
Need to define the roles of the patients Lack of resources in the organisations Need for training to understand the regulatory environment

HTA Summer School for patient groups
Collaboration between EFNA and London School of Economics: ‡ September 2009 ‡ May 2010 ‡ June 2010 ‡ September 2010 ‡ June 2011 ‡ September 2011
Working for people living with brain disorders

Patient Experience Network National Awards 2010
Access to Information:
Criteria Innovation Your Score 220

Ranked Second Place
Description Winner s Score 190

Demonstrates a very high level of innovation and ambition, well above that expected in their sector Is easily transferable as a complete programme to other organisations/ settings with little or no adaptation required.




The new world require more and more decision making to be evidence based by those as close to patient care / the patient as an entity themselves. The use of material to get patients to take such responsibility is the right way forwards. Extract from Benchmark Scores and Judges remarks

Societal responsibility

‡ Understanding benefit/risk ‡ An informed patient is a cost
effective patient (Wanless)

The Patient s journey

Information is needed from:

‡ ‡ ‡

Clinician - about the disease Industry - about the medication Patient NGOs - about fellow travellers

From the bench to the bedside

New drug application & review ~ 2 years Preclinical & clinical development ~ 8 years HTA review ~ 2 years


From the bench to the bedside

New drug application & review ~ 2 years Preclinical & clinical development ~ 8 years HTA review ~ 2 years


From the bench to the bedside

New drug application & review ~ 2 years Preclinical & clinical development ~ 8 years HTA review ~ 2 years


From the bench to the bedside

New drug application & review ~ 2 years Preclinical & clinical development ~ 8 years HTA review ~ 2 years


The biggest threat


Partnership is the only way

It is not the strongest of the species that survives nor the most intelligent that survives. It is the one that is most adaptable to change.
Charles Darwin 1809 - 1882

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