EURORDIS Summer School Virginie Hivert Therapeutic Development - PowerPoint PPT Presentation

EURORDIS Summer School Virginie Hivert Therapeutic Development Director, EURORDIS PCWP meeting, 26 th November, EMA, London, UK eurordis.org eurordis.org 1

Who are we? 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  organisations 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 2 eurordis.org

Training programmes at EURORDIS EUPATI 2nd EMA/HTA cohort EUPATI advanced start EUPATI 1st course cohort EURORDIS Summer School ExPRESS ExPRESS 4 days/each year 2015 2016 2012 2008 2010 2015 2016 3 eurordis.org

Objectives of the Summer School • 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 4 eurordis.org

Format & Content • 4-days F2F in Barcelona (formal lectures, breakout sessions, mini COMP, mini PDCO, etc.) coupled with online 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. 5 eurordis.org

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 eurordis.org

30 Summer School started back in 2008 30 25 • From 2008 to 2014, 227 patient 21 20 19 19 20 17 representatives have been 16 15 trained, coming from 33 different 10 8 countries and representing more 7 7 7 5 5 4 3 4 3 4 5 3 3 2 1 3 2 1 than 75 diseases 2 2 2 1 2 2 1 1 1 0 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 01/12/2015 7 eurordis.org

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All rare Child with Cystic Fibrosis ExPRESS 2015! Esophageal Atresia and Tracheo Esophogeal Fistula Fabry Gaucher • 30 patient representatives Juvenile Idiopathic arthritis Malattie metaboliche ereditarie 1 1 1 1 1 • 12 academic researchers Melenomas Uveal 9 Morbus Osler 1 1 Mucopolysaccaridosis • 20 different countries 1 Mucopolysaccharidosis type IV 1 1 1 Myeloma 1 1 1 1 Neurofibromatosis 1 1 1 1 9 1 1 Neuromuscular and Mitochondrial 1 1 Pathology 8 Niemann Pick Type A B & C 7 Ocular Melanoma 6 5 Osteogenesis imperfecta 4 Pulmonary arterial hypertension 3 2 Pulmonary hypertension 1 Rare and Inherited retina 0 degenerations 01/12/2015 9 eurordis.org

ExPRESS 2015! • This Summer School was co-organised by:  COST Action BM2107  European Medicines Agency  EURORDIS  Platforma Malaties Minoritàries • Programme Committee: Nancy Hamilton Josep Torrent-Farnell Annemieke Aartsma-Rus Nathalie Bere Virginie Hivert Monica Ensini Maria Mavris François Houÿez Nathalie Goemans Yann Le Cam Elizabeth Vroom 01/12/2015 10 eurordis.org

Also thanks to: • 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 01/12/2015 11 eurordis.org

What is it about? & What is new? • 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 01/12/2015 12 eurordis.org

Opportunities for Patient involvement along the medicine lifecycle at EMA Documents for the Public Public Summaries Package Leaflets (PL) Safety Package Leaflets (PL) of Opinion (renewal) Communications EPAR summaries Patient Patient Patient Patient input input input input POST PRE-SUBMISSION EVALUATION AUTHORISATION Regulatory Procedure Paediatric Scientific Advice Marketing Authorisation Post Marketing Orphan designation/ Investigation ATMP classification Protocol assistance Application Evaluation Authorisation Plan CHMP Committee s and Working Parties COMP/ CHMP- CAT CHMP PDCO CAT SAWP PRAC PRAC COMP Patient Patient Patient Patient Patient input input input input input CAT SAG SAG Patient Patient Patient input input input 13 eurordis.org eurordis.org

Patient involvement all along the product life cycle: interaction with all the stakeholders Academics Industry Regulators Patients Payers … HTA Healthcare bodies professionals 12/1/2015 14 eurordis.org

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 15 eurordis.org

Feedback from academics • The best thing/what they liked most - 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 12/1/2015 16 eurordis.org

We are now working on ExPRESS 2016 • 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 opportunities 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 17 eurordis.org

Summer School  Emergence of leadership + Network of alumnis From 2008 to 2013 eurordis.org

T H 2014 A N K Y O 2015 U ! 19 eurordis.org