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

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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 went on to graduate from medical school in 1975 at the Kaunas Institute of Medicine. He became a surgeon, gaining a specialisation in cardiovascular surgery in 1989 for which he practiced for over 20 years. He completed a degree in History from Vilnius University in 1984.

From 1969 onwards, the Commissioner was active in the anti-Soviet movement, however his political career truly began in 1976. In the 1990’s, Andriukaitis was elected to the Supreme Council of the Republic of Lithuania which preceded Seimas (Lithuanian Parliament). On that same year, he became the co-author and a signatory of the Independence Act of Lithuania of the 11th of March 1990. Vytenis Andriukaitis was also one of the co-authors of the Constitution of the Republic of Lithuania adopted in 1992, and one of the founders of the Lithuanian Social .

Andriukaitis was a Member of the Lithuanian Parliament for six terms. During that time, he served as Chairman of the Committee on European Affairs and was especially active during the accession to the EU in 2004 as he was also a member of the Foreign Affairs Committee. He served as Vice- and later President of the Social Democratic Party; and also as a Deputy Speaker of the Parliament. The Commissioner went on to Head the delegation of Lithuanian to the Convention on the Future of Europe. From 2012 to 2014, Vytenis Andriukaitis was the Minister of Health in the Lithuanian Government.

7 5. Introduction

Beatrice LORENZIN, Italian Minister of Health

Beatrice Lorenzin was appointed Minister of Health in the 62th Government of the Italian Republic led by in April 2013. She maintained the position of Minister of Health in the Government led by in February 2014. In December 2016 she has been reconfirmed Minister of Health in the Government. She was born in Rome in 1971 and started her political career in 1997, with the election to the Council of 13th Municipal district of Rome. In 2001, she was elected member of the Town Council of Rome. In 2005 she was Head of the Technical Secretariat of Undersecretary to the Presidency of the Council of Ministers for the Information and Publishing. Elected to the Chamber of Deputies in 2008, she was reconfirmed in the same Chamber in the 2013 general elections. Beatrice Lorenzin is one of the founders of the NCD political party.

8 SESSION 1

9 6. Moderator

Ilona REISCHL

Head of the Clinical Trials Unit in the Institute Surveillance at the Austrian Medicines and Medical Devices Agency (AGES/MEA)

Ilona Reischl joined the Austrian Medicines and Medical Devices Agency AGES/MEA in March 2006 and is currently the Head of the Clinical Trials Unit in the Institute Surveillance which is responsible for clinical trials with medicinal products or medical devices and GCP inspections.

She holds an initial degree in pharmacy, a PhD in immunology/allergology, postdoctoral experience at an industrial research institute (AT), post-doctoral positions followed at the University of Southampton (UK) and the National Institutes of Health (USA) in immunology. Her current regulatory focus began with a dual research/regulatory position at the US Food and Drug Administration (FDA).

Ilona Reischl is the Austrian member of the European Medicines Agency Committee for Advanced Therapies, currently its vice-chair, and the Biologics Working Party. She is actively involved in procedural and scientific issues pertaining to ATMPs such as clinical trials, GMOs and combination products. More generally, she participates in the EC Expert group on clinical trials and the EU innovation network and is involved in global scientific and regulatory activities.

10 7. Speakers

7.1 Guido RASI

Executive Director of European Medicines Agency (EMA)

Professor Guido Rasi began his second term as Executive Director of EMA on 16 November 2015. From November 2014 to mid- November 2015, Professor Guido Rasi served as EMA’s Principal Adviser in Charge of Strategy. From November 2011 to November 2014 he was the Executive Director of the European Medicines Agency and a member of its Management Board in the three years prior to this. He was Director-General of the Italian Medicines Agency from 2008 to 2011 and member of the Management Board from 2004 and 2008. He was made full professor of microbiology at the University of Rome 'Tor Vergata' in 2008. From 2005 to 2008 he was Director of Research at the Institute of Neurobiology and Molecular Medicine of the National Research Council (CNR) in Rome. From 1990 to 2005 Professor Rasi worked at the Institute for Experimental Medicine of the National Research Council, . He had teaching and research experience at the University of California, Berkeley in 1999. Professor Rasi holds a degree in medicine and surgery, with specialisations in internal medicine, allergology and clinical immunology from the University of Rome. From 1978 to 1990, he worked as a physician in a hospital, researched and uptained his private practice. He is also the author of more than 100 scientific publications. Prof Rasi was born in Padova, Italy and is married with two children.

Key message Advanced therapy medicinal products (ATMPs) are medicines for human use that are based on genes or cells. They offer ground-breaking new opportunities for the treatment of disease and injury. ATMPs can be classified into four main groups: gene therapy medicinal products, somatic cell therapy medicinal products, tissue engineering products and combined ATMPs. For each of these groups we have seen innovative products authorised in the past few years. Science and technology in ATMPs area is advancing at an exponential pace. New development paradigms are progressing with unprecedented speed. EMA horizon scanning detected a number of highly innovative and promising ATMPs (e.g. CAR-T Cells based products). Although ATMPs have the potential to improve health and deliver economic benefits, the experience acquired in the last decade shows that ATMPs are complex products to develop, manufacture, evaluate and make available to patients. If ATMPs are to fulfil their promise of providing innovative treatments for patients, regulators must nurture a regulatory environment that encourages innovation, safeguards public health and, ultimately, facilitates timely patient access to new therapies. EMA, in collaboration with the European Commission and the EU Regulatory Authorities Network, continues to have an active role in fostering ATMP’s development and access to patients by implementing a collaborative, coordinated action plan.

11 7.2 Andrea CHIESI

Head of R&D Portfolio Management of the Chiesi Group

Andrea Chiesi is head of R&D Portfolio Management of the Chiesi Group. His role entails the coordination of the planning activities of projects from Chiesi’s R&D, contributing to the definition of the R&D strategic plan and yearly budget, tracking of costs, activities and timelines of projects. The role also involves the scientific information, intelligence gathering and diffusion, and the search and management of public grants for the R&D projects. Starting as a bench analyst in the galenic department in 1993, Andrea then becoma a project leader and later on became manager of R&D leading several reorganisation projects and product developments.

He was the Co-founder and CEO of Holostem Terapie Avanzate srl, a spin-off of the University of Modena and Reggio Emilia with scientists Graziella Pellegrini, Michele De Luca and the Chiesi Group until 2015. He specialised in the development and manufacturing of advanced therapies with a particular emphasis on epithelia. The company’s first product Holoclar® was been approved last February as the first ever stem-cell based pharmaceutical product in Europe. Andrea Chiesi is also Vice President of EBE (European Bio-pharmaceutical Enterprises); and Member of IRDIRC’s (International Rare Diseases Research Council) Constituents Committee.

Key message

When it comes to Advanced Therapy Medicinal Products (ATMPs), developing a strong science and a forefront regulatory framework in Europe has been a long journey. Now it’s time to move on and achieve patient access. ATMPs have the potential to be transformative medicines reshaping the treatment of a wide range of conditions, particularly in disease areas where conventional approaches are inadequate. Nearly a decade after Regulation (EC) No 1394/2007 came into force which speeds up patient access to ATMPs, real progress has been made; in regards to novel – living – constructs that bring about entirely new challenges in terms of intellectual property rights, manufacturing, quality, regulatory approvals, pricing, reimbursement, delivery and administration of the finished product that often have a very short shelf-life. However, since 2008, only 8 ATMPs have been authorised, 4 of which have been withdrawn from the market for commercial reasons, not exactly a resounding success. Indeed, despite the unified ATMP framework in Europe that ensures these products are regulated under the central marketing authorisation procedure, there is variation in national legislation relating to some crucial issues as, for instance, clinical trials, good manufactory practices, market access, and ethical oversight. This has been detrimental for European patients but also represents a major challenge for the future of the ATMPs sector in Europe. Europe needs to find the path to ensure a more consistent and more transparent approach in this field.

12 7.3 Bernard MULLIGAN

Deputy Head of Unit, Innovative tools, technologies and concepts in health research, Directorate General for Research & Innovation, European Commission

The objective of my unit is to stimulate and support European research & innovation in the development, clinical testing and uptake, including getting products on the market, of novel therapies, devices, tools & technologies and regenerative medicine; and in the establishment of test methods, with regulatory approval, for better human safety testing (including alternatives to animals).

Also important are preventive actions, such as the European Human Biomonitoring Initiative (HBM4EU), in relation to impacts on human health of environmental exposures. Other areas include systems biology, systems medicine and bioinformatics.

Bernard Mulligan received his PhD in Biochemistry from the University of Liverpool, UK in 1980. He carried out postdoctoral research in the USA (Harvard University) and had an academic career at the University of Nottingham before joining the Commission's Research Directorate General in 1998. He worked in the research areas of genomics and infectious diseases before joining his current unit.

Key message

The fast growing and changing areas of cell and gene therapy, tissue engineering and regenerative medicine offer great hope for new therapies for untreatable diseases and potentially a reduction of treatment costs. With the Horizon 2020 research programme, the Health Directorate undertook a major effort to support research and innovation aiming at bringing such new therapies into the clinic and to the market. Thus, in the Horizon 2020 programme, four consecutive calls for proposals resulted in 23 funded projects with a total EU contribution of 133 million Euros. Funded projects include for example new approaches treating ischemic heart diseases with mesenchymal stem cells (SCIENCE) (www.stemcellscience.dk), spinal cord injury using specific antibodies against growth inhibiting factors (NISCI) (www.nisci-2020.eu), knee cartilage injury with autologous nasal chondrocytes (BIO-CHIP) (www.biochip-h2020.eu/), or Stress Urinary Incontinence by using a combination of autologous muscle precursor cells and neuromuscular electromagnetic stimulation (MUSIC) (www.music2020.ch) - all projects at www.cordis.europa.eu/projects. For the last three years of the Work Programme two relevant calls will again support innovative advanced therapies.

13 7.4 Yan LE CAM

Chief Executive Officer of EURORDIS - Rare Diseases Europe

Yann Le Cam is the Chief Executive Officer of EURORDIS – Rare Diseases Europe – since 2001 and was one of its founders in 1997. Yann le Cam is a member of the European Medicines Agency (EMA) Management Board since 2016. He is a member of the Council of Rare Diseases International (RDI).

Yann was Former-Chair of the Therapeutic Scientific Committee of the International Consortium for Research on Rare Diseases IRDiRC (2013-2016); member (2010-2016) and Vice Chair (2010-2013) of the Commission Expert Group on Rare Diseases; and a member of the Committee for Orphan Medicinal Products (COMP) at the EMA as a patient representative (2000-2009) and as Vice-Chairman (2000-2003 and 2003-2006).

Yann has three daughters, the oldest of whom is living with cystic fibrosis.

Key message

Advanced therapies such as gene, cell and somatic therapies represent an incredible opportunity for people living with a rare disease to access life changing treatments, as over 80% of rare diseases are of genetic origin. Science has enormously progressed and is continuing to advance at an incredible pace, but there are challenges on the path to full and equitable access to these innovative treatments for all relevant patients: it is not only the price of the new therapies, but also the logistic impediments for full access to specialised healthcare across borders. We need to address immediately the hurdles that currently impede a true cross border availability of the new, complex therapies to people with rare diseases, a right already enshrined in the EU Charter of Human Rights and legally implemented by the Directive on Patients’ Rights in Cross Border Healthcare. The newly set up European Reference Networks (ERNs) can provide the necessary infrastructure to make sure that patients have the opportunity to access the most advanced, effective and life- changing treatments. There already exists established forums to discuss and agree upon adequate reward for innovation across borders, such as the Mechanism for Coordinated Access (MoCA), the EMA-EUnetHTA dialogue, the BeNeluxA collaboration and other forms of joint procurement. It is a moral obligation of all actors involved to future-proof the access for patients with rare diseases to curative treatments. The tools to do so are in many ways already there. We need the political will to make it happen.

14 SESSION 2

15 8. Moderator

Elke ANKLAM

Director of the JRC-Geel site and Director of JRC Directorate F (Health, Consumers & Reference Material) at the JRC-Geel and JRC-Ispra site

Elke Anklam is a chemist, with specialisation in food, organic and radiation chemistry. After obtaining her PhD from the University Hamburg in Germany, she worked in various European Research Institutions and was a Teaching Professor at the Applied University of Fulda (Germany).

Since 1991, she has been working at the European Commission's Joint Research Centre (EC-JRC) and since 2006, she holds a Director position in the JRC.

At present, she is the Director of the Joint Research Centre - Geel site and Director of Joint Research Centre Directorate F (Health, Consumers & Reference Material), located at the Joint Research Centre - Geel and Joint Research Centre - Ispra site.

16 9. Speakers

9.1 Susanne BREMER-HOFFMANN

Sector Head for the group "Interaction of Nanomaterials with Biological Systems" at JRC's Directorate for Health, Consumers and Reference Materials

Susanne Bremer-Hoffmann is responsible for a laboratory assessing the interaction of nanomaterials with biological systems which is part of the JRC's open Nanobiosciences Laboratory. She is participating in various European projects that she liaises with the regulatory community. Her main research focus is in the identification of regulatory needs relevant for progressing nanomedicines to the market. Susanne Bremer-Hoffmann is a biologist by education with a specialisation in cell biology, molecular biology and in vitro toxicology. After having obtained her Ph.D. from the Free University Berlin in Germany, she worked at the Federal Institute for Risk Assessment in Germany before joining the European Commission’s Joint Research Centre in 1995. Susanne Bremer-Hoffmann was involved in the establishment of European Reference Laboratory for Alternatives to Animal Testing (EURL-ECVAM) and contributed to a number of validation studies related to endocrine disruption and developmental toxicity. She participated in several European and international research projects, published more than 80 scientific manuscripts and contributed to various European and international expert groups relevant for the implementation of European legislations in the field of chemicals and cosmetics.

Key Message

Nanotechnology is considered to be a key technology for the implementation of personalised medicine. This emerging scientific field is contributing to the development of innovative therapies designed to specifically target diseased tissue as well as the development of sensitive diagnostic tools which can assist medical practitioners in the initial selection of the optimum therapy and subsequently to monitor the efficiency of the treatment. It offers the means to adopt a more patient centred approach which becomes even more important for diseases with a poor prognosis where a rapid progress is crucial for therapeutic success. However, the translation of nanomedicine from the laboratory environment into clinical applications requires the anticipation of regulatory demands which must be satisfied to promote a smooth translation of nanomedicines to the market. In particular, the issue of additional safety testing must be carefully monitored addressing also combinations of physical-chemical properties which may impact their distribution in the human body or trigger unwanted toxicological effects. The unique characteristics of nanomedicines require a thorough review of existing guidelines, guidance and standards to evaluate their suitability for use with these novel products. Furthermore, the need to develop new standards addressing the quality and safety of nanomedicines has already been stressed by several international regulatory agencies that are being increasingly challenged with the regulation of nanomedicines.

17 9.2 Patrick HUNZIKER

President of the International Society for Nanomedicine

Patrick Hunziker studied Medicine at the University of Zurich, Switzerland. He received a doctoral decree based on thesis work in experimental immunology from the University of Zurich and did further research in experimental haematology at the University Hospital in Zurich, Switzerland. He earned specialist degrees in Internal Medicine, Cardiology and Intensive Care Medicine. As a fellow at the Massachusetts General Hospital, Harvard Medical School, he worked on cardiac imaging in a joint project with the Massachusetts Institute of Technology, Cambridge. In 2008, Patrick Hunziker became professor for Cardiology and Intensive Care Medicine at the University of Basel. His professional activities in Europe, the U.S., Africa and China gave him a broad insight into the needs for the medicine of the future in a variety of settings. Hunziker became involved in medical applications of Nanoscience in the late nineties and has been the pioneer physician in Nanomedicine in Switzerland since then. With improved prevention, diagnosis and cure of cardiovascular disease as his main research topic, he worked in the nanoscience fields of atomic force microscopy, nanoptics, micro/nanofluidics, nanomechanical sensors and polymer nanocarriers for targeting. He is the founding president of the European Society of Nanomedicine, cofounder of the European Foundation for Clinical Nanomedicine and co- initiator of the European Summit for Clinical Nanomedicine and is clinically active as deputy head of the Clinic for Intensive Care Medicine at the University Hospital Basel, Switzerland. He is President of the International Society for Nanomedicine, which is unites members from all continents in the world and organizes every year a Nanomedicine Summer school.

Key message

A major limitation of today’s medicine is the lack of precision in targeting patients, organs, and diseased cells. For the precision medicine of tomorrow, the currently evolving paradigms of receptor-specific, targeting of nanomaterial-based drug carriers and a predictive understanding of individual patient responses is needed. This presentation discusses the major obstacles that hinder translation of new ideas through academic research to industrial translation and clinical application. Understanding the clinical need and the preferred use scenarios, i.e. the clinical application view can guide development around the relevant obstacles, contributing to a fast track to advance science towards a benefit for society.

18 9.3 Beat LOEFFLER

Chief Executive Officer of CLINAM - European Foundation for Clinical Nanomedicine Beat Löffler, MD h.c., MA studied after a learning-year in the USA Philosophy, Communication Sciences and Politics at the Freie Universität in Berlin, graduating with a Master of Arts. Working for years in the medical field, he received in 2014 an MD h.c. from the University of Basel. In 1984, he co-founded an Agency for New Media. From 1988 to 1994 he was Managing Director of the International Hightech Forum Basel where he organised congresses on new technologies in mobility, energy, CFD and medical technology. In 1994, he founded his present company "L&A - Concept Engineering" for translation of science-based visions into applications and establishment of worldwide networks. He was for 6 years secretary general and coach of the trinational BioValley Promotion Team, with the mission of establishing the trinational Upper-Rhine Biotechnology network. From 2003 to 2006, he worked for NEC Hightech Performance Computing as Consultant leading Life Sciences Business Development in Biology and Medicine. He founded the European Foundation for Clinical Nanomedicine in 2007 together with Patrick Hunziker. The aim of the foundation is the research and development of nanomedicine with regard to its use as an innovative technology, better medical care in the future and the establishment of an international network in nanomedicine and related fields. CLINAM shaped in the last decade a neutral high- level debate platform organized by the nonprofit foundation, which serves also as meeting place for the international regulatory authorities from all continents in the field of nanotechnologies in health. Presently CLINAM is the worldwide largest network for clinical nanomedicine debates and has become a meeting forum between all stakeholders in Nanomedicine and related fields. The foundation launched the European Journal of Nanomedicine, the European Society for Nanomedicine and the International Society for Nanomedicine, which realises every year a Nanomedicine Summer school. Key message Nanomedicine is the use of nanotechnology at the scale of one-billionth of a meter and is the application of nanotechnology to the discipline of medicine: the use of nanoscale materials for the diagnosis, monitoring, control, prevention, and treatment of diseases - The Nanomedicine Market and its expectation of growth relates to the Technology Pipeline for Development of Nanomedicines. Although nanomedicine is often claimed to be not rapidly developing as expected, in realitythis is not the case. Research is extremely high and any novel technology needs up to 30 to 35 years to get an accepted flourishing market. (Example is the wind energy sector that now spends 10% of the European Energy after the research on feasibility to build large farms started approximately 35 years ago). What are the predictions seen by industry, how does global Nanotechnology develops in Drug delivery by technology and by therapeutic applications? How big are the successes in Nanomedicine globally and how do they develop in Europe? What is the position towards Nanomedicine of the large pharmaceutical companies (Big Players in Pharmacy)? What are the market opportunities, Restraints and Drivers for Nanomedicine and what are the proposals for an accelerating of Nanomedicine market in Europe? The short intervention will give answers to these questions.

19 10. ABOUT STOA

10.1 Mission

The Science and Technology Options Assessment (STOA) Panel forms an integral part of the structure of the European Parliament. Launched in 1987, STOA is tasked with identifying and independently assessing the impact of new and emerging science and technologies.

The goal of its work is to assist, with independent information, the Members of the European Parliament (MEPs) in developing options for long-term, strategic policy-making.

The STOA Panel The STOA Panel consists of 24 MEPs nominated from the eight permanent parliamentary committees: AGRI (Agriculture & Rural Development), CULT (Culture & Education), EMPL (Employment & Social Affairs), ENVI (Environment, Public Health & Food Safety), IMCO (Internal Market & Consumer Protection), ITRE (Industry, Research & Energy), JURI (Legal Affairs) and TRAN (Transport & Tourism).

Mr Ramon Luis Valcarcel Siso MEP is the European Parliament Vice-President responsible for STOA and member of the Panel. The STOA Chair for the second half of the 8th legislature is Eva Kaili, with Paul Rübig and Evžen Tošenovský elected as 1st and 2nd Vice-Chairs.

The STOA Approach STOA fulfils its mission primarily by carrying out science-based projects. Whilst undertaking these projects, STOA assesses the widest possible range of options to support evidence-based policy decisions. A typical project investigates the impacts of both existing and emerging technology options and presents these in the form of studies and options briefs. These are publicly available for download via the STOA website: www.europarl.europa.eu/stoa/.

Some of STOA's projects explore the long-term impacts of future techno-scientific trends, with the aim to support MEPs in anticipating the consequences of developments in science. Alongside its production of 'hard information', STOA communicates its findings to the European Parliament by organising public events throughout the year.

Focus areas STOA activities and products are varied and are designed to cover as wide a range of scientific and technological topics as possible, such as nano-safety, e-Democracy, e-Health and m-Health, bio-engineering, assistive technologies for people with disabilities, waste management, cybersecurity, smart energy grids, responsible research & innovation, etc.

They are grouped in five broad focus areas: eco-efficient transport and modern energy solutions; sustainable management of natural resources; potential and challenges of the Internet; health and life sciences; science policy, communication and global networking.

20 10.2 Administration

Director-General, Directorate-General for Parliamentary Research Services (EPRS) Anthony Teasdale

Director, Directorate C, Impact Assessment & European Added Value Wolfgang Hiller

Head of Unit - Scientific Foresight Unit (STOA) Theo Karapiperis

Head of Service - STOA Secretariat Zsolt Pataki

Head of Service - Scientific Foresight Lieve Van Woensel

Administrators Philip Boucher Mihalis Kritikos Nera Kuljanic Christian Kurrer Silvia Polidori Gianluca Quaglio – Seconded National Expert

Assistants Emilia Bandeira Morais Serge Evrard Rachel Manirambona Marie Massaro Damir Plese

Trainees Nada Alkhayat Carys Lawrie

21 This is a publication of the STOA Secretariat Directorate for Impact Assessment and European Added Value Directorate-General for Parliamentary Research Services, European Parliament

PE 603.209