EURORDIS Summer School Virginie Hivert Therapeutic Development - - PowerPoint PPT Presentation

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EURORDIS Summer School

Virginie Hivert Therapeutic Development Director, EURORDIS PCWP meeting, 26th November, EMA, London, UK

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Who are we?

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An international non-profit, non-governmental umbrella rare disease patients’ organisation representing an estimated 30 million individuals in Europe. Our Mission:

• To build a strong pan-European community of patient
• rganisations and people living with rare diseases
• To be their voice at the European level
• To directly or indirectly fight against the impact of rare

diseases on their lives

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Training programmes at EURORDIS

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2008 2016 ExPRESS 2016 ExPRESS 2015 2015 2010 EMA/HTA advanced course 2012 EUPATI start EUPATI 1st cohort EUPATI 2nd cohort EURORDIS Summer School 4 days/each year

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Objectives of the Summer School

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• Capacity building programme
• Patient engagement all along the life cycle of product

development

• Content is continuously adapted in line with evolution

in the field of regulation and processes

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Format & Content

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• 4-days F2F in Barcelona (formal lectures, breakout

sessions, mini COMP, mini PDCO, etc.) coupled with

• nline training (quizzes, video recordings, webinars)
• Content not Rare Disease specific, collaboration with

ECRIN (participants from other medical fields, medical devices, nutrition, etc)

• Evidence-based medicine, clinical research, clinical trials

methodology, ethics, statistics, regulatory principles & processes in the EU, EMA organisation and opportunities for patients, HTA appraisal, pharmacovigilance, etc.

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Organisation of four-day programme

• Pre-Summer School preparation
• Four-day programme
• General organisation
• Days 1 and 2 – Clinical trials and Drug development
• Days 3 and 4 – EMA overview and committees, HTA, etc.
• Specific organisation
• Mix of small group sessions and large group formal

presentations

• Problem-based learning model
• Small group sessions of 10 -12 individuals (maximum) to

encourage interactions and exchanges

• Case-based tutorials

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Summer School started back in 2008

01/12/2015

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1 3 8 3 2 1 5 3 2 1 21 2 17 2 7 4 3 19 2 1 20 4 3 7 2 5 2 1 19 7 1 4 30 16 5 10 15 20 25 30 Armenia Austria Belgium Bulgaria Canada Croatia CzechRepublic Denmark Estonia Finland France Georgia Germany Greece Hungary Iceland Ireland Italy Lithuania Malta Netherlands Poland Portugal Romania Russia Serbia Slovakia SouthAfrica Spain Sweden Switzerland Ukraine UnitedKingdom EURORDIS

Distribution / Countries of the participants 2008-2014

• From 2008 to 2014, 227 patient

representatives have been trained, coming from 33 different countries and representing more than 75 diseases

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ExPRESS 2015!

01/12/2015

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• 30 patient representatives
• 12 academic researchers
• 20 different countries

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All rare Child with Cystic Fibrosis Esophageal Atresia and Tracheo Esophogeal Fistula Fabry Gaucher Juvenile Idiopathic arthritis Malattie metaboliche ereditarie Melenomas Uveal Morbus Osler Mucopolysaccaridosis Mucopolysaccharidosis type IV Myeloma Neurofibromatosis Neuromuscular and Mitochondrial Pathology Niemann Pick Type A B & C Ocular Melanoma Osteogenesis imperfecta Pulmonary arterial hypertension Pulmonary hypertension Rare and Inherited retina degenerations

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ExPRESS 2015!

01/12/2015

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• This Summer School was co-organised by:
• COST Action BM2107
• European Medicines Agency
• EURORDIS
• Platforma Malaties Minoritàries
• Programme Committee:

Annemieke Aartsma-Rus Monica Ensini Nathalie Goemans Elizabeth Vroom Nathalie Bere Maria Mavris Nancy Hamilton Virginie Hivert François Houÿez Yann Le Cam Josep Torrent-Farnell

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Also thanks to:

01/12/2015

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• Funding from:
• Coordination and local organisation:
• Nancy Hamilton (EURORDIS)
• Iolanda Arbiol (Platforma Malaties Minoritàries)
• Our Faculty: Great thanks!! Highly committed people,

academics, regulators, industry and expert patients – some of them since the beginning

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What is it about? & What is new?

01/12/2015

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• First Training Programme with Patients and

Researchers being trained together

• Blended approach with plenary/formal lectures, small

groups sessions, round table discussion and practical exercises (‘mock’ COMP & SAWP, review of product information, etc.)

• Capacity building Programme  increasing

involvement in the R&D processes, Health Technology Assessment, information and access

to medicines

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eurordis.org CHMP CAT PRAC COMP CHMP- SAWP CHMP PRAC

Orphan designation/ ATMP classification

Scientific Advice Protocol assistance Paediatric Investigation Plan Post Marketing Authorisation Marketing Authorisation Application Evaluation

COMP/ CAT PDCO CAT

PRE-SUBMISSION EVALUATION POST AUTHORISATION

SAG SAG

Public Summaries

• f Opinion

Package Leaflets (PL) EPAR summaries Safety Communications

Documents for the Public Regulatory Procedure Committee s and Working Parties

Package Leaflets (PL) (renewal)

Patient input Patient input Patient input Patient input Patient input Patient input Patient input Patient input Patient input Patient input Patient input Patient input

Opportunities for Patient involvement along the medicine lifecycle at EMA

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Patient involvement all along the product life cycle: interaction with all the stakeholders

Patients

Academics Regulators

…

Healthcare professionals

HTA bodies

Payers

Industry

12/1/2015

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Feedback from patients

• Question - what did you think about having the researchers with us in

the training:

• In discussions with the researchers, I expected more disagreement.
• We never get the chance to exchange with researchers, so it was very

special to be able to approach them and realize that they are also normal people!

• In the sessions, it was nice to listen to someone looking at diseases at a

totally different perspective.

• It was nice to make personal connections with them as people and see how

they relate to our diseases in a different way.

• It was interesting to learn about what they are doing and about what other

diseases are doing. The networking opportunity was appreciated.

12/1/2015

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Feedback from academics

• The best thing/what they liked most

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• Learned a LOT
• Interaction with patients
• Small group sessions (some said this was where they learned most)
• This is a very unique opportunity to get this type of training
• Participants noted that they have a changed view on the regulatory

process and regulators; they understand more why regulation is needed and were pleasantly surprised about how nice and approachable the regulators are

• Patient engagement was made much more tangible, researchers more

convinced now that patients can be involved in research every step of the way

• How cultural differences became apparent … and can be better

understood

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We are now working on ExPRESS 2016

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• Online content accumulated from previous editions of the

EURORDIS Summer School such as webcasts and slide presentations as well as webinars, and interactive training modules.

• Even more hands-on/practical approach related to
• pportunities offered to be engaged into therapeutic

development and regulatory processes (Scientific Advice/Protocol Assistance, Early dialogue with HTA bodies, Benefit/Risk assessment at CHMP, Adaptive Pathways, etc.)

• Reinforcement of networking and interactions between

patients and researchers - sharing experiences & peer-to- peer training

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Summer School  Emergence of leadership + Network of alumnis

From 2008 to 2013

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2014 2015

T H A N K Y O U !