STOA WorkshopTherapiesfor the futureParticipants' booklet

EPRS | European Parliamentary Research ServiceScientific Foresight Unit (STOA)

PE 603.209

STOA Workshop

THERAPIES FOR THE FUTURE

Exploring solutionsfor innovative treatments

11 October 2017, 14:30 – 17:15European Parliament, Brussels

Henri Spaak building, room P7C050

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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

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CONTENTS

1. Programme ............................................................................................................................ 4

2. Introduction and aim of the workshop ............................................................................... 5

3. Chair ...................................................................................................................................... 6Paul RÜBIG, MEP and STOA 1st Vice-Chair ........................................................................... 6

4. Keynote speech ...................................................................................................................... 7Vytenis ANDRIUKAITIS, European Commissioner for Health and Food Safety ................... 7

5. Introduction........................................................................................................................... 8Beatrice LORENZIN, Italian Minister of Health....................................................................... 8

Session 1

6. Moderator............................................................................................................................ 10Ilona REISCHL ............................................................................................................. 10

7. Speakers ............................................................................................................................... 117.1 Guido RASI ................................................................................................................... 11

7.2 Andrea CHIESI ............................................................................................................. 12

7.3 Bernard MULLIGAN.................................................................................................... 13

7.4 Yan LE CAM ................................................................................................................ 14

Session 2

8. Moderator............................................................................................................................ 16Elke ANKLAM ............................................................................................................. 16

9. Speakers ............................................................................................................................... 179.1 Susanne BREMER-HOFFMANN................................................................................. 17

9.2 Patrick HUNZIKER ...................................................................................................... 18

9.3 Beat LOEFFLER ........................................................................................................... 19

10. ABOUT STOA .................................................................................................................... 2010.1 Mission....................................................................................................................... 20

10.2 Administration ........................................................................................................... 21

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1. Programme

Chair: Paul RÜBIG, MEP & STOA 1st Vice-Chair

14:30 – 15:00WelcomePaul RÜBIG, MEP & STOA 1st Vice-Chair

Keynote speechVytenis ANDRIUKAITIS, European Commissioner for Health & Food Safety

IntroductionBeatrice 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 realityGuido RASI, European Medicines Agency

Advanced therapy medicinal products: the long journey from the unmet need to the patientsAndrea CHIESI, European Biopharmaceutical Companies

Supporting research and innovation for advanced therapiesBernard MULLIGAN, DG Research & Innovation, European Commission

Now is the time to guarantee access to the therapies of the futureYann LE CAM, European Organisation for Rare Diseases

Q&A

16:15 - 17:10 PART II – NANOMEDICINEModerator: Elke ANKLAM, Joint Research Centre

Nanomedicine in EU: enabling innovative therapiesSusanne BREMER-HOFFMANN, Joint Research Centre

Development of Nanomedicine in the clinicPatrick HUNZIKER, International Society for Nanomedicine

Nanomedicine at the international levelBeat LOEFFLER, European Foundation for Clinical Nanomedicine

Q&A

17:10 - 17:15 CLOSING REMARKSPaul RÜBIG, MEP & STOA 1st Vice-Chair

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2. Introduction and aim of the workshop

Nanomedicine and advanced therapy medicinal products represent two subjects of greatinnovative impact. Both having significant implications for a number of diseases for which theredoes not yet exist adequate diagnostic and therapeutic tools; These same areas could alsorepresent 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 targetingof pharmaceutical, therapeutic and diagnostic agents is at the forefront of current projects innanomedicine.

These involve the identification of precise targets (cells and receptors) related to specific clinicalconditions and choice of the appropriate nano-carriers to achieve the required responses whileminimising 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 andformulation 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 recombinantnucleic acids and that have a therapeutic, prophylactic, or diagnostic effect related directly to therecombinant nucleic acid sequence.

- Somatic cell therapy medicinal products are biological products that contain or consist of cellsor tissues that have been subject to substantial manipulation (or that are not intended to be usedfor the same essential functions) in the recipient and the donor; the recipient and the donor couldbe the same person.

- Tissue-engineered products contain or consist of engineered cells or tissues and are presentedas having properties for, or are used in or administered to, human beings with the aim ofregenerating, repairing, or replacing human tissue.

The purpose of the workshop is to report on the status of nanomaterial and advanced therapymedicinal products, as well as on possible pathways for the future development of these newtherapies in Europe. The event will offer an opportunity to develop a range of options forsupporting patient access and transparent information on these therapeutic treatments acrossEurope.

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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 8thlegislature 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 1996and belongs to the European People's Party (EPP).

He is the owner of an Austrian blacksmith company and has a degree in BusinessAdministration, Marketing and Production Engineering from the University of Linz, UpperAustria. He is married and has two children.

Paul Rübig is a full member of the Committee on Industry, Research and Energy and of theCommittee on Budgets. He is Vice-Chair of the Delegation for relations with the KoreanPeninsula and substitute member of the Delegation for relations with Switzerland, Norway andof the EU-Iceland Joint Parliamentary Committee.

He is also a substitute member of the European Economic Area (EEA) Joint ParliamentaryCommittee. 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 theirbusiness environment.

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4. Keynote speech

Vytenis ANDRIUKAITIS, European Commissioner for Health and Food Safety

Vytenis Povilas Andriukaitis was appointed the European Commissioner for Health and FoodSafety in November 2014.

Vytenis Andriukaitis was born on 9 August 1951 in Kyusyur in Siberia, where his family wasdeported to in 1941 from Lithuania. He returned to Lithuania in 1957 together with his motherand 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 ofMedicine. He became a surgeon, gaining a specialisation in cardiovascular surgery in 1989 forwhich 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 hispolitical career truly began in 1976. In the 1990’s, Andriukaitis was elected to the SupremeCouncil of the Republic of Lithuania which preceded Seimas (Lithuanian Parliament). On thatsame year, he became the co-author and a signatory of the Independence Act of Lithuania of the11th of March 1990. Vytenis Andriukaitis was also one of the co-authors of the Constitution ofthe Republic of Lithuania adopted in 1992, and one of the founders of the Lithuanian SocialDemocratic Party.

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

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5. Introduction

Beatrice LORENZIN, Italian Minister of Health

Beatrice Lorenzin was appointed Minister of Health in the 62th Government of the ItalianRepublic led by Enrico Letta in April 2013. She maintained the position of Minister of Health inthe Government led by Matteo Renzi in February 2014. In December 2016 she has beenreconfirmed Minister of Health in the Paolo Gentiloni Government. She was born in Rome in1971 and started her political career in 1997, with the election to the Council of 13th Municipaldistrict of Rome. In 2001, she was elected member of the Town Council of Rome. In 2005 she wasHead of the Technical Secretariat of Undersecretary to the Presidency of the Council of Ministersfor the Information and Publishing. Elected to the Chamber of Deputies in 2008, she wasreconfirmed in the same Chamber in the 2013 general elections. Beatrice Lorenzin is one of thefounders of the NCD political party.

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SESSION 1

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6. Moderator

Ilona REISCHL

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

Ilona Reischl joined the Austrian Medicines and Medical Devices Agency AGES/MEA in March2006 and is currently the Head of the Clinical Trials Unit in the Institute Surveillance which isresponsible for clinical trials with medicinal products or medical devices and GCP inspections.

She holds an initial degree in pharmacy, a PhD in immunology/allergology, postdoctoralexperience at an industrial research institute (AT), post-doctoral positions followed at theUniversity 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 andDrug Administration (FDA).

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

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7. Speakers

7.1 Guido RASI

Executive Director of European Medicines Agency (EMA)

Professor Guido Rasi began his second term as Executive Directorof EMA on 16 November 2015. From November 2014 to mid-November 2015, Professor Guido Rasi served as EMA’s PrincipalAdviser in Charge of Strategy. From November 2011 toNovember 2014 he was the Executive Director of the EuropeanMedicines Agency and a member of its Management Board in thethree years prior to this. He was Director-General of the ItalianMedicines Agency from 2008 to 2011 and member of theManagement Board from 2004 and 2008. He was made fullprofessor of microbiology at the University of Rome 'Tor Vergata'in 2008. From 2005 to 2008 he was Director of Research at theInstitute of Neurobiology and Molecular Medicine of the NationalResearch Council (CNR) in Rome. From 1990 to 2005 ProfessorRasi worked at the Institute for Experimental Medicine of the National Research Council, Italy.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 workedas a physician in a hospital, researched and uptained his private practice. He is also the authorof more than 100 scientific publications. Prof Rasi was born in Padova, Italy and is married withtwo children.

Key message

Advanced therapy medicinal products (ATMPs) are medicines for human use that are based ongenes or cells. They offer ground-breaking new opportunities for the treatment of disease andinjury. ATMPs can be classified into four main groups: gene therapy medicinal products, somaticcell therapy medicinal products, tissue engineering products and combined ATMPs. For each ofthese 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 developmentparadigms are progressing with unprecedented speed. EMA horizon scanning detected anumber 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, theexperience 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, regulatorsmust nurture a regulatory environment that encourages innovation, safeguards public healthand, ultimately, facilitates timely patient access to new therapies. EMA, in collaboration with theEuropean Commission and the EU Regulatory Authorities Network, continues to have an activerole in fostering ATMP’s development and access to patients by implementing a collaborative,coordinated action plan.

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7.2 Andrea CHIESI

Head of R&D Portfolio Management of the Chiesi Group

Andrea Chiesi is head of R&D Portfolio Management ofthe Chiesi Group. His role entails the coordination of theplanning activities of projects from Chiesi’s R&D,contributing to the definition of the R&D strategic planand yearly budget, tracking of costs, activities andtimelines of projects. The role also involves the scientificinformation, intelligence gathering and diffusion, and thesearch and management of public grants for the R&Dprojects.Starting as a bench analyst in the galenic department in1993, Andrea then becoma a project leader and later onbecame manager of R&D leading several reorganisationprojects and product developments.

He was the Co-founder and CEO of Holostem Terapie Avanzate srl, a spin-off of the Universityof Modena and Reggio Emilia with scientists Graziella Pellegrini, Michele De Luca and the ChiesiGroup until 2015. He specialised in the development and manufacturing of advanced therapieswith a particular emphasis on epithelia. The company’s first product Holoclar® was beenapproved 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); andMember of IRDIRC’s (International Rare Diseases Research Council) Constituents Committee.

Key message

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

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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 Europeanresearch & innovation in the development, clinical testing anduptake, including getting products on the market, of noveltherapies, devices, tools & technologies and regenerativemedicine; and in the establishment of test methods, withregulatory approval, for better human safety testing (includingalternatives to animals).

Also important are preventive actions, such as the EuropeanHuman Biomonitoring Initiative (HBM4EU), in relation to impactson human health of environmental exposures. Other areas includesystems biology, systems medicine and bioinformatics.

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

Key message

The fast growing and changing areas of cell and gene therapy, tissue engineering andregenerative medicine offer great hope for new therapies for untreatable diseases and potentiallya reduction of treatment costs.

With the Horizon 2020 research programme, the Health Directorate undertook a major effort tosupport research and innovation aiming at bringing such new therapies into the clinic and to themarket. Thus, in the Horizon 2020 programme, four consecutive calls for proposals resulted in23 funded projects with a total EU contribution of 133 million Euros.

Funded projects include for example new approaches treating ischemic heart diseases withmesenchymal stem cells (SCIENCE) (www.stemcellscience.dk), spinal cord injury using specificantibodies against growth inhibiting factors (NISCI) (www.nisci-2020.eu), knee cartilage injurywith autologous nasal chondrocytes (BIO-CHIP) (www.biochip-h2020.eu/), or Stress UrinaryIncontinence by using a combination of autologous muscle precursor cells and neuromuscularelectromagnetic stimulation (MUSIC) (www.music2020.ch) - all projects atwww.cordis.europa.eu/projects. For the last three years of the Work Programme two relevantcalls will again support innovative advanced therapies.

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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 foundersin 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 ScientificCommittee of the International Consortium for Research onRare Diseases IRDiRC (2013-2016); member (2010-2016) andVice Chair (2010-2013) of the Commission Expert Group on

Rare Diseases; and a member of the Committee for Orphan Medicinal Products (COMP) at theEMA 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 opportunityfor people living with a rare disease to access life changing treatments, as over 80% of rarediseases are of genetic origin.Science has enormously progressed and is continuing to advance at an incredible pace, but thereare challenges on the path to full and equitable access to these innovative treatments for allrelevant patients: it is not only the price of the new therapies, but also the logistic impedimentsfor full access to specialised healthcare across borders.We need to address immediately the hurdles that currently impede a true cross borderavailability of the new, complex therapies to people with rare diseases, a right already enshrinedin the EU Charter of Human Rights and legally implemented by the Directive on Patients’ Rightsin Cross Border Healthcare.The newly set up European Reference Networks (ERNs) can provide the necessary infrastructureto 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 uponadequate reward for innovation across borders, such as the Mechanism for Coordinated Access(MoCA), the EMA-EUnetHTA dialogue, the BeNeluxA collaboration and other forms of jointprocurement.It is a moral obligation of all actors involved to future-proof the access for patients with rarediseases to curative treatments. The tools to do so are in many ways already there. We need thepolitical will to make it happen.

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SESSION 2

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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. Afterobtaining her PhD from the University Hamburg in Germany, she worked in various EuropeanResearch 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 JointResearch Centre Directorate F (Health, Consumers & Reference Material), located at the JointResearch Centre - Geel and Joint Research Centre - Ispra site.

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9. Speakers

9.1 Susanne BREMER-HOFFMANN

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

Susanne Bremer-Hoffmann is responsible for alaboratory assessing the interaction of nanomaterialswith biological systems which is part of the JRC'sopen Nanobiosciences Laboratory. She isparticipating in various European projects that sheliaises with the regulatory community. Her mainresearch focus is in the identification of regulatoryneeds relevant for progressing nanomedicines to themarket. Susanne Bremer-Hoffmann is a biologist byeducation with a specialisation in cell biology,molecular biology and in vitro toxicology. After having obtained her Ph.D. from the FreeUniversity Berlin in Germany, she worked at the Federal Institute for Risk Assessment inGermany before joining the European Commission’s Joint Research Centre in 1995. SusanneBremer-Hoffmann was involved in the establishment of European Reference Laboratory forAlternatives to Animal Testing (EURL-ECVAM) and contributed to a number of validationstudies related to endocrine disruption and developmental toxicity. She participated in severalEuropean and international research projects, published more than 80 scientific manuscripts andcontributed to various European and international expert groups relevant for theimplementation of European legislations in the field of chemicals and cosmetics.

Key Message

Nanotechnology is considered to be a key technology for the implementation of personalisedmedicine. This emerging scientific field is contributing to the development of innovativetherapies designed to specifically target diseased tissue as well as the development of sensitivediagnostic tools which can assist medical practitioners in the initial selection of the optimumtherapy and subsequently to monitor the efficiency of the treatment. It offers the means to adopta more patient centred approach which becomes even more important for diseases with a poorprognosis where a rapid progress is crucial for therapeutic success.However, the translation of nanomedicine from the laboratory environment into clinicalapplications requires the anticipation of regulatory demands which must be satisfied to promotea smooth translation of nanomedicines to the market. In particular, the issue of additional safetytesting must be carefully monitored addressing also combinations of physical-chemicalproperties which may impact their distribution in the human body or trigger unwantedtoxicological 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 ofnanomedicines has already been stressed by several international regulatory agencies that arebeing increasingly challenged with the regulation of nanomedicines.

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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 workin experimental immunology from the University of Zurich anddid further research in experimental haematology at theUniversity Hospital in Zurich, Switzerland. He earned specialistdegrees in Internal Medicine, Cardiology and Intensive CareMedicine. As a fellow at the Massachusetts General Hospital,Harvard Medical School, he worked on cardiac imaging in ajoint project with the Massachusetts Institute of Technology,Cambridge. In 2008, Patrick Hunziker became professor forCardiology and Intensive Care Medicine at the University ofBasel. His professional activities in Europe, the U.S., Africa andChina gave him a broad insight into the needs for the medicineof 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 inNanomedicine in Switzerland since then. With improved prevention, diagnosis and cure ofcardiovascular disease as his main research topic, he worked in the nanoscience fields of atomicforce microscopy, nanoptics, micro/nanofluidics, nanomechanical sensors and polymernanocarriers for targeting. He is the founding president of the European Society ofNanomedicine, cofounder of the European Foundation for Clinical Nanomedicine and co-initiator of the European Summit for Clinical Nanomedicine and is clinically active as deputyhead of the Clinic for Intensive Care Medicine at the University Hospital Basel, Switzerland. Heis President of the International Society for Nanomedicine, which is unites members from allcontinents 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, anddiseased cells. For the precision medicine of tomorrow, the currently evolving paradigms ofreceptor-specific, targeting of nanomaterial-based drug carriers and a predictive understandingof individual patient responses is needed. This presentation discusses the major obstacles thathinder translation of new ideas through academic research to industrial translation and clinicalapplication. Understanding the clinical need and the preferred use scenarios, i.e. the clinicalapplication view can guide development around the relevant obstacles, contributing to a fasttrack to advance science towards a benefit for society.

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9.3 Beat LOEFFLER

Chief Executive Officer of CLINAM - European Foundation for ClinicalNanomedicine

Beat Löffler, MD h.c., MA studied after a learning-year in the USAPhilosophy, Communication Sciences and Politics at the FreieUniversität in Berlin, graduating with a Master of Arts. Workingfor years in the medical field, he received in 2014 an MD h.c. fromthe University of Basel. In 1984, he co-founded an Agency for NewMedia. From 1988 to 1994 he was Managing Director of theInternational Hightech Forum Basel where he organisedcongresses on new technologies in mobility, energy, CFD andmedical technology. In 1994, he founded his present company"L&A - Concept Engineering" for translation of science-basedvisions into applications and establishment of worldwidenetworks. He was for 6 years secretary general and coach of thetrinational BioValley Promotion Team, with the mission ofestablishing the trinational Upper-Rhine Biotechnology network.From 2003 to 2006, he worked for NEC Hightech Performance Computing as Consultant leadingLife Sciences Business Development in Biology and Medicine. He founded the EuropeanFoundation for Clinical Nanomedicine in 2007 together with Patrick Hunziker. The aim of thefoundation is the research and development of nanomedicine with regard to its use as aninnovative technology, better medical care in the future and the establishment of an internationalnetwork 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 placefor the international regulatory authorities from all continents in the field of nanotechnologies inhealth. Presently CLINAM is the worldwide largest network for clinical nanomedicine debatesand has become a meeting forum between all stakeholders in Nanomedicine and related fields.The foundation launched the European Journal of Nanomedicine, the European Society forNanomedicine and the International Society for Nanomedicine, which realises every year aNanomedicine Summer school.

Key message

Nanomedicine is the use of nanotechnology at the scale of one-billionth of a meter and is theapplication of nanotechnology to the discipline of medicine: the use of nanoscale materials forthe diagnosis, monitoring, control, prevention, and treatment of diseases - The NanomedicineMarket and its expectation of growth relates to the Technology Pipeline for Development ofNanomedicines. Although nanomedicine is often claimed to be not rapidly developing asexpected, in realitythis is not the case. Research is extremely high and any novel technologyneeds up to 30 to 35 years to get an accepted flourishing market. (Example is the wind energysector that now spends 10% of the European Energy after the research on feasibility to build largefarms started approximately 35 years ago). What are the predictions seen by industry, how doesglobal Nanotechnology develops in Drug delivery by technology and by therapeuticapplications? How big are the successes in Nanomedicine globally and how do they develop inEurope? What is the position towards Nanomedicine of the large pharmaceutical companies(Big Players in Pharmacy)? What are the market opportunities, Restraints and Drivers forNanomedicine and what are the proposals for an accelerating of Nanomedicine market inEurope? The short intervention will give answers to these questions.

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10.ABOUT STOA

10.1 Mission

The Science and Technology Options Assessment (STOA) Panel forms an integral part of thestructure of the European Parliament. Launched in 1987, STOA is tasked with identifying andindependently 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 EuropeanParliament (MEPs) in developing options for long-term, strategic policy-making.

The STOA PanelThe STOA Panel consists of 24 MEPs nominated from the eight permanent parliamentarycommittees: 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 (LegalAffairs) and TRAN (Transport & Tourism).

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

The STOA ApproachSTOA fulfils its mission primarily by carrying out science-based projects. Whilst undertakingthese projects, STOA assesses the widest possible range of options to support evidence-basedpolicy decisions. A typical project investigates the impacts of both existing and emergingtechnology options and presents these in the form of studies and options briefs. These arepublicly 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, withthe aim to support MEPs in anticipating the consequences of developments in science. Alongsideits production of 'hard information', STOA communicates its findings to the EuropeanParliament by organising public events throughout the year.

Focus areasSTOA activities and products are varied and are designed to cover as wide a range of scientificand 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; healthand life sciences; science policy, communication and global networking.

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10.2 Administration

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

Director, Directorate C, Impact Assessment & European Added ValueWolfgang Hiller

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

Head of Service - STOA SecretariatZsolt Pataki

Head of Service - Scientific ForesightLieve Van Woensel

AdministratorsPhilip BoucherMihalis KritikosNera KuljanicChristian KurrerSilvia PolidoriGianluca Quaglio – Seconded National Expert

AssistantsEmilia Bandeira MoraisSerge EvrardRachel ManirambonaMarie MassaroDamir Plese

TraineesNada AlkhayatCarys Lawrie

PE 603.209

This is a publication of the STOA SecretariatDirectorate for Impact Assessment and European Added ValueDirectorate-General for Parliamentary Research Services, European Parliament