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Patient engagement at EMA

20 May 2023

Emphasizing the critical need for patient partnerships in medicines and health devices regulation

Maria Mavris

Public and Stakeholders Engagement Department, European Medicines Agency

An agency of the European Union

Classified as public by the European Medicines Agency

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Patient engagement at EMA

Classified as public by the European Medicines Agency

EMA is a scientific body;
It is a decentralised agency of the EU established in 1995 to evaluate medicines for use in people and animals in the EU;
EMA brings together the best scientific expertise on medicines from across the whole of the EU;
The Agency works with seven scientific committees who evaluate medicines along their life cycle from early stages of development, through marketing authorisation to the safety monitoring once they are on the market; the committees are supported by 28 working parties;
All of this is underpinned by a network of over 4000 scientific experts who are made available to EMA by the national competent authorities (more about the network in the next slide). At EMA’s London headquarters, there are around 900 staff members; EMA staff help to support, coordinate and facilitate the work of the committees;
EMA is a guardian of public health in the EU;
The CHMP (Committee for Medicinal Products for Human Use) plays a vital role in the authorisation of medicines. It is responsible for conducting the initial assessment of EU-wide marketing authorisation applications, their modifications or extensions, suspension or withdrawal;
The CVMP (Committee for Medicinal Products for Veterinary Use) conducts the initial assessment for marketing authorisation applications, post-authorisation and maintenance activities, and monitors the safety of veterinary medicines;
The COMP (Committee for Orphan Medicinal Products) is responsible for evaluating applications for orphan designation. This designation is for medicines to be developed for the diagnosis, prevention or treatment of rare diseases that are life-threatening or very serious. In the EU, a disease is defined as rare if it affects fewer than five in 10,000 people across the EU;
The HMPC (Committee on Herbal Medicinal Products) in responsible for compiling and assessing scientific data on herbal substances, preparations and combinations, to support the harmonisation of the European market;
The PDCO (Paediatric Committee) is responsible for activities on medicines for children and supports the development of such medicines in the EU by providing scientific expertise and defining paediatric needs. It is also responsible for assessing the content of paediatric investigation plans, or PIPS, which describe how a medicine should be studied in children, as well as waivers and deferrals;
The CAT (Committee for Advanced Therapies) is responsible for assessing the quality, safety and efficacy of advanced therapy medicines, including medicines classified as gene therapy, somatic cell therapy or tissue-engineered products, and for following scientific developments in the field;
The PRAC (Pharmacovigilance Risk Assessment Committee) is responsible for assessing and monitoring the safety of medicines for human use.

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Patient engagement at EMA

Classified as public by the European Medicines Agency

First and foremost, EMA protects human and animal health;
EMA facilitates medicines development by giving scientific advice and providing guidance to developers of medicines, including on the development of medicines for children. The Agency has a specific programme in place to incentivise the development of medicines for rare diseases (orphan designation programme);
EMA carries out robust scientific evaluations of medicines for human and veterinary use; the outcomes of our evaluations are the basis of the European Commission’s decision on whether a medicine can be authorised for marketing throughout the EU. The company which produces the medicine can only market the medicine once it has received a marketing authorisation from the Commission;
EMA monitors the safety of all medicines marketed in the EU;
EMA provides information on human and veterinary medicines to healthcare professionals and patients.

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Interaction with patients and consumers: a progressive journey…�

Patient engagement at EMA

Classified as public by the European Medicines Agency

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Patient engagement at EMA

Patients as individual experts in medicine-specific activities

Classified as public by the European Medicines Agency

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Engagement and support

Patient engagement at EMA

Methodologies for engagement

Face to face meeting

oral explanations - scientific advice - SAG

In writing

written responses to scientific advice - surveys

Training and support

EMA training day

Information sheets

Videos on EMA website

Information on webpages

One to one support

One size does not fit all!

Classified as public by the European Medicines Agency

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Patient engagement – added value and impact

Scientific Advice

4 year study published
Added value of patient input quantified and demonstrated

Patient engagement at EMA

CHMP early contact

17 month pilot completed
Positive impact – will be maintained as new methodology

Review of documents

Comments and suggestions by patients incorporated into published documents
Template structure changed

Safety monitoring

Public hearings – recommendations leading to risk minimisation measures

Classified as public by the European Medicines Agency

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Challenges of patient engagement

Patient engagement at EMA

Finding suitable patients (e.g. language barrier, availability)
Ensuring comprehensive, tailored training to facilitate and enhance participation
Provide a clear definition of patients role in the different activities / committees to manage expectations from all angles
Managing potential conflicts of interest
Representativeness
Measuring the value / impact of patients

Classified as public by the European Medicines Agency

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Patient engagement at EMA

Classified as public by the European Medicines Agency

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International cooperation and patient engagement

EMA has various mechanisms in place to work with other regulators such as Australia, Brazil, Canada, Japan, Switzerland and the United States and World Health Organization (WHO).
The US FDA and EMA hold regular ‘cluster’ meetings on all topics including patient engagement.

EMA and African Medicines Agency (AMA) Project

Support the work of AMA
Reinforce scientific and regulatory expertise for evaluation and supervision of medicines
Develop framework for working together as an African regulatory network

Patient engagement at EMA

Classified as public by the European Medicines Agency

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Conclusions

Engaging with patients and their organisations:

Brings everyday aspects of living with a disease into scientific discussions
Helps bridge the gap between clinical trial data and real world data
Increases transparency, awareness and understanding: TRUST

Engage in a stepwise approach; learn together what format works best;

Define roles - manage expectations
Ensure engagement is mutually beneficial

Patient engagement at EMA

Engaging with patients leads to more meaningful outcomes for everyone!

Everyone has a role to play to ensure engagement happens

Classified as public by the European Medicines Agency

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Any questions?

Maria Mavris

Patient Liaison - Public and Stakeholder Engagement Department

maria.mavris@ema.europa.eu

Official address Domenico Scarlattilaan 6 ● 1083 HS Amsterdam ● The Netherlands

Send us a question Go to www.ema.europa.eu/contact

Telephone +31 (0)88 781 6000

Further information

Classified as public by the European Medicines Agency