STOA Workshop Therapies for the future Participants' booklet EPRS | European Parliamentary Research Service Scientific Foresight Unit (STOA) PE 603.209 STOA Workshop THERAPIES FOR THE FUTURE Exploring solutions for innovative treatments 11 October 2017, 14:30 – 17:15 European Parliament, Brussels Henri Spaak building, room P7C050 Prepared by Gianluca Quaglio and Nada Alkhayat, STOA Secretariat Available at: http://www.europarl.europa.eu/stoa/cms/home/workshops/therapies Join the conversation on Twitter by using the hashtag #HealthSTOA and by tweeting at @EP_ThinkTank 2 CONTENTS 1. Programme ............................................................................................................................4 2. Introduction and aim of the workshop ...............................................................................5 3. Chair ......................................................................................................................................6 Paul RÜBIG, MEP and STOA 1st Vice-Chair ...........................................................................6 4. Keynote speech......................................................................................................................7 Vytenis ANDRIUKAITIS, European Commissioner for Health and Food Safety ...................7 5. Introduction...........................................................................................................................8 Beatrice LORENZIN, Italian Minister of Health.......................................................................8 Session 1 6. Moderator............................................................................................................................10 Ilona REISCHL .............................................................................................................10 7. Speakers...............................................................................................................................11 7.1 Guido RASI...................................................................................................................11 7.2 Andrea CHIESI .............................................................................................................12 7.3 Bernard MULLIGAN....................................................................................................13 7.4 Yan LE CAM ................................................................................................................14 Session 2 8. Moderator............................................................................................................................16 Elke ANKLAM .............................................................................................................16 9. Speakers...............................................................................................................................17 9.1 Susanne BREMER-HOFFMANN.................................................................................17 9.2 Patrick HUNZIKER ......................................................................................................18 9.3 Beat LOEFFLER ...........................................................................................................19 10. ABOUT STOA ....................................................................................................................20 10.1 Mission.......................................................................................................................20 10.2 Administration ...........................................................................................................21 3 1. Programme Chair: Paul RÜBIG, MEP & STOA 1st Vice-Chair 14:30 – 15:00 Welcome Paul RÜBIG, MEP & STOA 1st Vice-Chair Keynote speech Vytenis ANDRIUKAITIS, European Commissioner for Health & Food Safety Introduction Beatrice LORENZIN, Italian Minister of Health 15:00 - 16:15 PART I – ADVANCED THERAPY MEDICINAL PRODUCTS (ATMPs) Moderator: Ilona G. REISCHL, Austrian Medicines and Medical Devices Agency Advanced therapy medicinal products: from promise to reality Guido RASI, European Medicines Agency Advanced therapy medicinal products: the long journey from the unmet need to the patients Andrea CHIESI, European Biopharmaceutical Companies Supporting research and innovation for advanced therapies Bernard MULLIGAN, DG Research & Innovation, European Commission Now is the time to guarantee access to the therapies of the future Yann LE CAM, European Organisation for Rare Diseases Q&A 16:15 - 17:10 PART II – NANOMEDICINE Moderator: Elke ANKLAM, Joint Research Centre Nanomedicine in EU: enabling innovative therapies Susanne BREMER-HOFFMANN, Joint Research Centre Development of Nanomedicine in the clinic Patrick HUNZIKER, International Society for Nanomedicine Nanomedicine at the international level Beat LOEFFLER, European Foundation for Clinical Nanomedicine Q&A 17:10 - 17:15 CLOSING REMARKS Paul RÜBIG, MEP & STOA 1st Vice-Chair 4 2. Introduction and aim of the workshop Nanomedicine and advanced therapy medicinal products represent two subjects of great innovative impact. Both having significant implications for a number of diseases for which there does not yet exist adequate diagnostic and therapeutic tools; These same areas could also represent a vital boost for the future of pharmaceutical industry development in Europe. By nanomedicine we understand applications of nanotechnology for the treatment, diagnosis, monitoring and control of biological systems. Research into the rational delivery and targeting of pharmaceutical, therapeutic and diagnostic agents is at the forefront of current projects in nanomedicine. These involve the identification of precise targets (cells and receptors) related to specific clinical conditions and choice of the appropriate nano-carriers to achieve the required responses while minimising the side effects. Specific cells (e.g. dendritic cells, endothelial cells, and tumor cells, etc.) are key targets. Today, nanotechnology and nano-science approaches to particle design and formulation are beginning to expand the market for many drugs. Advanced therapy medicinal products constitute an innovative group of heterogeneous, research-driven biopharmaceuticals. This group encompasses gene therapy medicinal products, somatic cell therapy medicinal products, tissue-engineered products, and combined products (tissue or cells associated with a device). - Gene therapy medicinal products are products of biological origin containing recombinant nucleic acids and that have a therapeutic, prophylactic, or diagnostic effect related directly to the recombinant nucleic acid sequence. - Somatic cell therapy medicinal products are biological products that contain or consist of cells or tissues that have been subject to substantial manipulation (or that are not intended to be used for the same essential functions) in the recipient and the donor; the recipient and the donor could be the same person. - Tissue-engineered products contain or consist of engineered cells or tissues and are presented as having properties for, or are used in or administered to, human beings with the aim of regenerating, repairing, or replacing human tissue. The purpose of the workshop is to report on the status of nanomaterial and advanced therapy medicinal products, as well as on possible pathways for the future development of these new therapies in Europe. The event will offer an opportunity to develop a range of options for supporting patient access and transparent information on these therapeutic treatments across Europe. 5 3. Chair Paul RÜBIG, MEP and STOA 1st Vice-Chair Paul Rübig was elected as the STOA Chair for the first half of the European Parliament's 8th legislature and as First Vice-Chair for the second half of the 8th legislature. Previously, he served as STOA Chair from 2009 to 2012 and as First Vice-Chair from 2012 to 2014. Born in Northern Austria, Paul Rübig has been a member of the European Parliament since 1996 and belongs to the European People's Party (EPP). He is the owner of an Austrian blacksmith company and has a degree in Business Administration, Marketing and Production Engineering from the University of Linz, Upper Austria. He is married and has two children. Paul Rübig is a full member of the Committee on Industry, Research and Energy and of the Committee on Budgets. He is Vice-Chair of the Delegation for relations with the Korean Peninsula and substitute member of the Delegation for relations with Switzerland, Norway and of the EU-Iceland Joint Parliamentary Committee. He is also a substitute member of the European Economic Area (EEA) Joint Parliamentary Committee. Furthermore, Paul Rübig is a substitute member in the Committee on Development. Paul Rübig is very active in the field of the small-scale business promotion. He is president of SME Global, a working group of the International Democrat Union (IDU), whose objective it is to support small and medium-sized enterprises (SME) and to improve their business environment. 6 4. Keynote speech Vytenis ANDRIUKAITIS, European Commissioner for Health and Food Safety Vytenis Povilas Andriukaitis was appointed the European Commissioner for Health and Food Safety in November 2014. Vytenis Andriukaitis was born on 9 August 1951 in Kyusyur in Siberia, where his family was deported to in 1941 from Lithuania. He returned to Lithuania in 1957 together with his mother and two brothers. His father was permitted to return only a year later. Andriukaitis