15101_pgm

13-15 April 2015 Palais des Congrs

Paris

Final Programme

27th Annual EuroMeetingDevelopment, Innovation, Access and Patient Safety

DIA EuroMeeting 2015Development, Innovation, Access and Patient Safety

13-15 April, Palais des Congrs, Paris

2

TABLE OF CONTENTS

Welcome Form DIA Chair, DIA Board of Directors and DIA Global Chief Executive .........................................................................2

Welcome from the EuroMeeting 2015 Co-Chairs & Programme Advisors ................................................................................3

EuroMeeting 2015 Theme Leaders ...................................................................4

Schedule at a glance .............................................................................................5

General Information A-Z ..................................................................................6-7

Award Winners .......................................................................................................9

Professional Posters ............................................................................................ 10

Student Posters / Patient Advocates ............................................................. 11

EuroMeeting Pre-Conference Optional Tutorials ..................................16-18

Clinical Forum Pre-Conference Optional Tutorials .................................... 19

HAS Satellite Symposium ................................................................................. 20

50th Anniversary of the EU Parmaceutical Legislation .......................... 20

50 Years of EDQM .............................................................................................. 20

Opening Plenary .................................................................................................. 21

Theme 1 | New R&D models and innovative clinical trial methodologies. ..................................................................................... 22-23

Theme 2 | Medical Devices and Combination Products .................... 24-25

Theme 3 | Advanced Therapies, Novel Treatments for Rare Diseases .................................................................................. 25-27

Theme 4 | Early Dialogue with Regulators and HTA Bodies on Innovative Medicines .............................................. 27-28

Theme 5 | Collaborative Consortia, IMI and the Way to Horizon 2020 ...................................................................29-30

Theme 6 | Implementing Innovation Globally Conducive Regulatory Policies .......................................................... 31-32

Theme 7 | Special Development Pathways in Paediatrics, the Elderly and in Pregnancy ............................................................33-34

Theme 8 | Translational Medicine and Regulatory Science .............. 35-37

Theme 9 | Big Data, Mobile Health .......................................................... 37-39

Theme 10 | Innovation in Vaccine Development ..................................39-41

Theme 11 | Availability of Medicinal Products: Drug shortages ........41-42

Theme 12 | Pharmacovigilance in 2015 - Poised for convergence of innovation .................................................................43-45

Hot Topics and Stand-Alone Sessions .......................................................... 46

Focus Sessions .................................................................................................... 47

Speaker Index ................................................................................................48-53

Networking Events ............................................................................................ 54

EuroMeeting Session Plan ..........................................................................56-58

Dear Colleagues, It is our pleasure to welcome you to Paris, the hub of European biotech IPOs (international public offerings), and a city that boasts not only centuries of science, but also the highest density of publically traded biotech companies in Europe. Paris is a city that actively engages in translating research into tangible economic realities with a robust regulatory framework. Thank you for joining us at DIAs 27th Annual EuroMeeting. As we come together to share our collective knowledge to address significant challenges in human health, we will reflect on our collective accomplishments in Advancing Innovation to Combat the Global Burden of Disease, the theme of the Opening Plenary. Our two keynote speakers, Olivier Charmeil, Executive Vice President, Sanofi Vaccines, and Andrew Morris, Professor, University of Edinburgh, and of the Farr Institute, will address this subject in view of the need for pharmaceutical innovation. Their focus will be centered on making innovation more efficient and relevant through the use of big data. This EuroMeeting brings together innovators and leaders from all parts of the health care ecosystem. Our scientific offerings are organised into themes and led by experts who will cover a wide range of interest areas, such as access to innovative treatments, advanced therapies, special development pathways, and pharmacovigilance, as well as glocal outlook, collaborative consortia, and dialogue between regulators and HTA bodies to advance translational medicine. Also this DIA 27th EuroMeeting will celebrate the 50th anniversary of the European Pharmaceutical Legislation as well as the 50th anniversary of the EDQM (European Directorate for the Quality of Medicines and Health Care). We will highlight the achievements of these landmark organisations and provide a perspective into what can be expected in the future for health care product development and regulation. Together with DIAs recent 50th anniversary milestone, this EuroMeeting marks an important milestone in our efforts to foster innovation by providing forums for industry, regulators, patients, and other stakeholders to exchange information and discuss health products, technologies, services, and related issues in order to advance our collective mission of developing and delivering effective therapies to patients worldwide. On behalf of the DIA Board of Directors and all of our EuroMeeting programme volunteers, exhibitors, poster presenters, and staff, thank you very much for attending our celebratory DIA 2015 27th EuroMeeting. Enjoy the programme!

Per SpindlerChair, DIA Board of Directors

Barbara Lopez KunzGlobal Chief Executive

DIA EuroMeeting 2015Development, Innovation, Access and Patient Safety13-15 April, Palais des Congrs, Paris

3

WELCOME FROM THE EUROMEETING 2015 CO-CHAIRS

Dear Colleagues,

We are delighted that the 27th Annual DIA EuroMeeting is held in Paris this year.

Our meeting comes at an exciting and challenging time for healthcare innovation. The demand for new therapies and new solutions has never been higher, and the pace of innovation is increasing to meet these needs. This makes Paris an ideal choice for the venue. The city has been at the forefront of scientific developments since the foundation of the Acadmie des Sciences in 1666. Since then, Paris-based scientists have brought us stethoscopes and hypodermic needles, antibiotics and antipsychotics, vaccines against TB and rabies and have successfully identified HIV. Now, in 2014, Paris remains pivotal in healthcare research, as the hub for Biotech IPOs in Europe.

If European companies are to remain competitive, they need a regulatory environment that supports their investment. The current rate of innovation, with new approaches and innovative therapies are testing the limits of the existing system, presents its own challenges for regulators and policymakers. Addressing these will require a collaborative approach between all stakeholders to create a streamlined, harmonised approach to regulation. A future regulatory system should be capable of anticipating potential hurdles and barriers, allowing the fruits of research to reach those people who need them more quickly. This will offer an impetus to companies to continue to invest in research. In addition, it will add momentum to the kind of public-private partnership such as the Innovative Medicines Initiative capable of accelerating effective drug development.

Adaptive pathways approvals, also named Adaptive Licensing or Medicine Adaptive Pathways to Patients, as prospectively planned adaptive approach the regulation of drugs, is without any doubt one of the relevant method to accelerate access to innovative medicines. While it is particularly relevant for medicines to treat serious conditions with unmet medical needs, it should also help developing innovative regulatory strategies for all new drugs and vaccines.

The Annual DIA EuroMeeting provides the ideal opportunity to build the type of collaboration that will underpin future innovation in Europe. An intense 3-day programme will present high quality information and discussion, along with opportunities for face-to-face meetings and professional networking between all the main stakeholders. Participants can exchange knowledge and build the kind of partnerships that will support innovation in drug development and address todays health challenges.

The DIA EuroMeeting 2015 should be the "go to" meeting for all professionals in drug discovery, drug development and regulation. We will be able to add a little more to the reputation of Paris as a city of health innovation. We look forward to seeing you here.

Lidia Retkowska-Mika and Olivier Charmeil

PROGRAMME ADVISORS

Martin Harvey AllchurchHead of Communication, European Medicines Agency, EU

Jan GeisslerDirector, EUPATI, Belgium

Angelika JoosExecutive Director, Global Regulatory Policy, MSD (Europe) Inc., Belgium

Yves JuilletSenior Vice-President, Industrie Sant, France

Spiros VamvakasEuropean Medicines Agency, EU

Christa Wirthumer-HocheHead, Austrian Medicines and Medical Devices Agency (AGES), Austria

Lidia Retkowska-MikaDirector, Legal Department, Office for Registration of Medicinal Products, Medical Devices and Biocidal Products, Poland

Olivier CharmeilExecutive Vice President Vaccines, Sanofi, France

PROGRAMME CO-CHAIRS



4

EUROMEETING 2015 THEME LEADERS

Sabine AtzorHead of EU Regulatory Policies,

F. Hoffmann-La Roche,Switzerland

Salah-Dine ChiboutGlobal Head Discovery &

Investigative Safety, Global Head Preclinical Safety Therapeutic Areas,

Novartis Pharma, Novartis

Anna CielikDirector Department of

Documentation Assessment, Office for Registration of Medicinal

Products, Poland

Isabelle ClamouRegulatory Affairs Director EU Policy, Amgen, Belgium

Susanna Del SignoreAssociate Vice-President Global

Regulatory Affairs, Head of Global Regulatory Policy, Sanofi R&D,

France

Beln Escribano RomeroHead of Department,

Pharmaceutical Inspection and Enforcement Department, Spanish Medicines Agency (AEMPS), Spain

Zaide FriasHead of Regulatory Affairs and Best Evidence, Head of Human Medicines

Research and Development Support (ad interim),

European Medicines Agency, EU

Sabina Hoekstra-van den BoschGlobal Regulations and Standards, Philips Healthcare, the Netherlands

Paul JansenGlobal Head Medical Devices US,

Sanofi, USA

Jun KitaharaInternational Liaison Officer

stationed in Swissmedic, Pharmaceuticals and Medical

Devices Agency (PMDA), Japan

Maarten LagendijkPharmacovigilance Coordinator,

Medicines Evaluation Board (MEB), the Netherlands

Jordi Llinares GarciaHead of Product Development Scientific Support, European

Medicines Agency, EU

Henrik Kim NielsenCorporate Vice President,

Regulatory Affairs GLP-1 and Obesity, Novo Nordisk, Denmark

Detlef NieseConsultant, Dr. Niese Health Science

and Policy, Germany

Luca PaniDirector General, Italian Medicines

Agency (AIFA), Italy

Richard PilsudskiVice President Global Regulatory

Affairs, Sanofi Pasteur, France

Dorthe PoulsenChief Legal Adviser, EU-Coordinator,

Danish Health and Medicines Authority (DKMA), Denmark

Fatiha SadallahPrincipal Scientific Manager,

Innovative Medicines Initiative (IMI), Belgium

Paula SalmikangasChair CAT, Senior Researcher,

Finnish Medicines Agency (FIMEA), Finland

Duane SchulthessManaging Director, Vital Tranformation, Belgium

Henk SchuringGroup Vice President Regulatory

Affairs - Europe, Genzyme Europe, the Netherlands

Pr TellnerEFPIA ICH Coordinator, Director

Regulatory Affairs, EFPIA, Belgium

Jan Willem van der LaanDepartment of Pharmacology, Toxicology and Biotechnology

(FTBB), Medicines Evaluation Board, the Netherlands

Maria Grazia ZurloQPPV,

Pfizer, Italy


5

SCHEDULE AT-A-GLANCE Sunday, 12 April 2015

Registration Hours:14:00-19:30 Exhibitor Registration and Set-up

14:00-18:00 Attendee and Speaker Registration*

17:30-18:30 Students and Young Professionals Welcome

Reception

* Avoid the rush on Monday by picking up your badge and conference

material on Sunday afternoon

Monday, 13 April 2015

Registration Hours:07:30-11:00 Exhibitor Registration and Set-up

08:00-18:00 Attendee, Speaker and Exhibitor Registration

Schedule:09:00-12:30 Pre-Conference Tutorials*

10:30-11:00 Pre-Conference Tutorials Coffee Break

11:00-12:30 HAS Satellite Symposium

12:00-18:00 Conference and Exhibition Open

12:00-14:00 Lunch & Innovation Theatre Presentation in the

Exhibition Hall

13:30-15:00 Regulatory Town Hall Meeting

14:00-17:30 Optional Clinical Forum Tutorials*

15:00-16:00 Extended Refreshment Break in the Exhibition Hall

16:00-17:45 Opening Plenary Session

18:00-20:30 Bienvenue Paris Reception

*Space is limited for Pre-Conference Tutorials, therefore pre-registration is strongly recommended. Availability for onsite registration is not guaranteed

Tuesday, 14 April 2015

Registration Hours:08:00-18:30 Attendee, Speaker and Exhibitor Registration

Schedule:08:00-09:00 Welcome Coffee in the Exhibition Hall

08:00-18:30 Exhibition Hall Open

09:00-10:30 Parallel Scientific Sessions - Session 1

Choose from Parallel Sessions

10:15-11:00 Coffee Break in the Exhibition Hall




Exhibition Hall

12:30-13:00 Speed Networking

13:00-14:00 DIA Communities - Meet and Eat



14:00-15:30 Exhibition Guest Passes




17:30-18:30 Networking Reception in the Exhibition Hall

17:45-18:15 Student Poster Award Ceremony at the DIA Booth

Wednesday, 15 April 2015

Registration Hours:08:00-17:30 Attendee, Speaker and Exhibitor Registration

Schedule:08:00-09:00 Welcome Coffee

08:00-16:00 Exhibition Hall Open






11:00-12:30 50 Years of EDQM Session


Exhibition Hall






17:30 End of Conference



6

The 27th Annual EuroMeeting takes place at:Le Palais des Congrs de Paris2, Place de la Porte Maillot75017 Paris France

Registration will be located on Level 3. The conference and exhibition will take place on levels 2 and 3.

For the first time ever, the EuroMeeting is organised in parallel to another DIA flagship meeting in Europe, the 8th Annual Clinical Forum. This will afford delegates unparalleled opportunities to interact and network with qualified professionals from throughout Europe and the world, and to meet a wealth of exhibiting companies, all under one roof.

AppDownload the Free DIA Global App Today.

The DIA Global App is designed to enhance your meeting experience and provide valuable information in one place. Create your session agenda, network with attendees and exhibitors, and connect to DIA resources, social media channels, member communities, and more.

To download, search for DIA Global in your app store.Access the EuroMeeting 2015 App: Sign in with the email address you registered for the EuroMeeting 2015 Click on the Events Icon Select 27th Annual DIA EuroMeeting 2015

You can find assistance at the DIA Booth #3.D17 on Level 3

Banking/ATMCash dispensers are available on levels -1 and 0.

Business CentrePlease note that there is no business centre at the Palais des Congrs de Paris. There is a copy/printing shop within 5 minutes walking distance from the Palais des Congrs, located at 62, Avenue de la grande Arme. www.copytop.com/agence-grande-armee-maillot

Certificate of AttendanceA Certificate of Attendance can be printed at the Scan-and-Go desks in Hall Bordeaux on Level 3 on Wednesday, 15 April 2015 from 10:30.

Cloakroom/BaggageThe cloakroom is in the Entrance Hall in Hall Bordeaux on Level 3. There is a charge of 2 per coat/jacket and 3 per piece of luggage.

Monday 08:00 - 21:00Tuesday 08:00 - 19:00Wednesday 08:00 - 18:00

Conference BagsAll attendees with a full meeting registration can collect a conference bag from the Conference Bag Distribution Point in Hall Bordeaux on Level 3. Please bring the bag voucher you received when collecting your badge.

Credits DIA meetings are accredited by the SwAPP (Swiss Association of Pharmaceutical Professionals) Commission for Professional Development (CPD) and SGPM (Swiss Society of Pharmaceutical Medicine).

The 27th Annual EuroMeeting has been awarded 13.75 CPD credits from the Faculty of Pharmaceutical Medicine (FPM) of the Royal College of Physicians (RCP) of the UK. Medical practitioners who are eligible for credits can click on http://www.fpm.org.uk/cpd/registration for more information. If you are already a CPD member, please go directly to http://cpd.fpm.org.uk to claim your credits.

DIA BoothFind out more about all DIA can offer you, how membership can advance your career, how to join a DIA Community, submit an article for publication and lots more, at the DIA Booth # 3.D17. Stop by at the DIA Booth in the Havane Exhibition Hall on Level 3. See Exhibition for opening hours.

DIA Patient BoothDIA actively promotes the involvement of patient representatives in the EuroMeeting. Since 2006, more than 200 patient representatives have been involved as participants, speakers, session chairs, and also in the Programme Committee. The Patient Fellowship Booth #3.D06 is located on Level 3 and acts as a focal point for patient fellows and other stakeholders to meet and network.

ExhibitionVisit the joint EuroMeeting and Clinical Forum Exhibition, with 140+ companies and service providers in a single venue. With many new companies exhibiting this year, the exhibition offers more opportunities than ever to connect with participants.

Monday 12:00 - 18:00Tuesday 08:00 - 18:30 | Public Access 14:00 - 15:30Wednesday 08:00 - 16:00

Please see the exhibition floor plan and list of exhibiting companies in the Exhibition Guide, or use the interactive floor plan in the DIA Global" mobile app.

Exhibitor ServicesThe Exhibitor Services Desks are located in Hall Maillot on Level 2. See Exhibition for opening hours.

First Aid A pharmacy located in the shopping center on Level 0 is open daily from 09:00 to 20:00. For medical assistance during the conference hours, please visit the DIA Onsite Registration Desk. Alternatively, any DIA host/hostess will be more than happy to be of assistance.

Hotel Accommodation InformationIf you have any queries about hotel accommodation, please visit the Hotel Information counter at the DIA Registration Desk located in Hall Bordeaux on Level 3.

Information DesksIf you have any questions about the Clinical Forum or the EuroMeeting, from finding session rooms to networking activities, stop by the DIA Information Desks located on Level 2 and Level 3. Alternatively, any staff member or DIA host/hostess will be more than happy to be of assistance.


GENERAL INFORMATION A-Z


7

Lost and FoundAll items will be stored at the DIA Registration Desk in Hall Bordeaux on Level 3 until the end of the conference.

Members LoungeV.I.P. Feeling for DIA MembersDIA Members can relax in the Members-only Lounge in the Exhibition Hall on level 3 during breaks, or whenever a few minutes in a quiet setting is needed. Coffee and drinks are served throughout exhibition opening hours and charging stations for your electronic devices are available. Make sure you bring your membership card to identify yourself as a DIA Member at the desk. Access exclusively for DIA Members.

Messaging ServicesDownload the DIA Global mobile app and use the messaging function to set appointments or send messages to other attendees.

PostersStudent, professional and patient representative posters will be displayed in the Bordeaux/Havane Hallway on Level 3. Come and talk to our student poster presenters during lunchtime on Tuesday between 13:00 and 14:00. A selected group of professional poster presenters will share their research results on various topics.

Join us at the DIA Booth #3.D17 on Level 3 on the Exhibition Floor for the Student Poster Award Ceremony on Tuesday, 14 April 2015 at 17:45.

Name BadgeName badges must be worn at all times in the Conference Center. Participants will incur a 25 fee for badge reprints. If you have misplaced your badge, you will be required to have a badge reprinted. Please visit Attendee Onsite Registration located in Hall Bordeaux on Level 3. Identification will be required. Please note, allowing exhibitors to scan the barcode on the front of your badge will provide them with your contact information. No children under the age of 18 years will be allowed in the Exhibition Halls due to liability issues.

PresentationsPresentations will be available to full conference attendees on the DIA web site from 9 April until 15 Ocotber 2015. Presentations are made available to full conference attendees only. To access presentations, visit diahome.org/EM2015 and follow the links for the EuroMeeting presentations.

Press LoungeThe Press Lounge is located in Room 204 on Level 3.DIA welcomes qualified representatives of news organisations for the purpose of reporting and publishing and broadcasting articles and stories. All media must present a copy of their press credentials upon arrival at the DIA Registration Desk. For more information, please contact Jacqueline Bowman on [email protected]


Refreshments/LunchesRefreshments and Lunches will be served each day on the Exhibition Floors on Levels 2 and 3. Enjoy extended refreshment and lunch hours to visit more than 140 exhibiting companies.

Monday12:00 - 14:00 Lunch15:00 - 16:00 Afternoon tea/coffee with snack

Tuesday10:15 - 11:00 Morning tea/coffee with snack12:00 - 14:30 Lunch15:15 - 16:00 Afternoon tea/coffee with snack

Wednesday10:15 - 11:00 Morning tea/coffee with snack12:00 - 14:00 Lunch15:15 - 16:00 Afternoon tea/coffee with snack

Water dispensers are located around the Exhibition Hall and session room hallways.

RegistrationRegistration is located in Hall Bordeaux on Level 3 and will be open as follows:

Sunday 14:00 - 18:00Monday 08:00 - 18:00Tuesday 08:00 - 18:00Wednesday 08:00 - 17:30

SecurityWe take the safety of our participants very seriously. Please help us by cooperating fully with the security personnel on duty and wear your badge at all times. Only participants with a valid conference badge will be allowed into the conference center.

Services for the DisabledAll the rooms at the congress centre are fully accessible to participants with disabilities.

Shopping centreThe Palais des Congrs de Paris has a shopping center with 70 shops: Palais Boutiques (www.lesboutiquesdupalais.com).

Speaker Resource CenterAll speakers are required to visit the Speaker Resource Center located in room 341 on Level 3 and re-check their slides at least 2 hours before the start of their session(s).

Sunday 14:00 - 18:00Monday 08:00 - 18:00Tuesday 08:00 - 18:00Wednesday 08:00 - 16:00

Student CornerA dedicated area is located in Booth #3.A07 in the Exhibition Hall on Level 3 for students to network, plan their day, and meet for lunch.

TwitterTweet about the EuroMeeting using #Euro and @DIA_Europe

WiFi Wireless internet access is available throughout the conference center and is powered by Oracle Health Sciences.

Network: DIA 2015 Password: PARIS2015

GENERAL INFORMATION A-Z



8

Thanks to our Media Partners!

International Pharmaceutical Industry Supporting the industry through communication

JOURNAL FOR

Your Resource for Multisite Studies & Emerging MarketsCLINICAL STUDIESU

from diagnosis to cure

DIA would like to express its sincere thanks to the following exhibitors for their contributions and support of the 27th Annual EuroMeeting & 8th Annual Clinical Forum

Oracle Health Sciences Booth 2.C09

WIFI and Exhibit Hall Signage

Accenture Life ServicesBooth 2.E18

Recharging Stations

APCER PHARMABooth 2.D12

Welcome to Paris Reception

Thanks to our Contributors!


9

DIA AWARD WINNERS

Outstanding Contribution to Health Award Dr June Raine Director of Vigilance and Risk Management of Medicines Medicines and Healthcare products Regulatory Agency (MHRA)

Dr June Raine trained in general medicine in Oxford after completing a Master degree by research in Pharmacology. Her interest in drug safety led to a career in medicines regulation which has spanned a number of roles in assessment, management and strategic development within the UK national authority. Appointed in 1999 to head Pharmacovigilance in the Medicines Control Agency (now Medicines and Healthcare products Regulatory Agency), she was elected in 2005 to chair the CHMPs Pharmacovigilance Working Party and in 2012 as the first chair of the Pharmacovigilance Risk Assessment Committee. She is also a member of the WHO Advisory Committee on Safety of Medicinal Products. Her special interests are in monitoring the outcomes of regulatory action, risk communication and patient involvement in the regulatory process. She has made outstanding contributions towards delivering a world-class European Union pharmacovigilance system. She has served as DIA EuroMeeting Programme co-Chair and has been a welcomed Speaker at DIA.

Excellence in Service Award Dr Birka Lehmann Head of Executive Department EU and International Affairs of the Federal Institute for Drugs and Medical Devices (BfArM)

Dr Birka Lehmann studied Medicines at the Free University Berlin and trained at the Kinderklinik Norderney. Her working experience includes 9 years preclinical assessment in the division Pharmacology and Toxicology of BfArM and she served as head of unit Decentralised Procedure (1996-2002) and as deputy head of EU Division (2000-2002). From 2002 2006 she joined the European Commission, Directorate-General Enterprise and Industry as expert on secondment to in the unit Pharmaceuticals responsible for inter alia Marketing Authorisation and implementation of Clinical Trials Directive. From September 2006 till October 2011 she was head of the division 3 Marketing Authorisation procedure at the BfArM. Since 2007 she is member of the Paediatric Committee at the European Medicines Agency.

She contributed continuously since 1994 to the DIA EuroMeetings, DIA Annual Meetings and several conferences, training courses and workshops.In 2004 she co-chaired with Yves Juillet the DIA EuroMeeting in Prague. Since 2013 she is chair of the Content Committee for DIA Europe, Middle East and Africa and a member of the DIA Advisory Committee for Europe, Middle East & Africa.

Excellence in Service Award Rolf Banholzer, PhDGlobal Head e-Clinical QA, Novartis Development, Basel

Rolf Banholzer has a PhD in Molecular Biology from the University of Basel.His focus on Computer Systems to be understood as an integral part of efficient and compliant clinical processes and must enable end to end data integrity in the clinical research and development process.

He acts as a translator between IT and Business to obtain optimal user requirements as the basis for successful implementation of IT systems. He promotes clear and transparent communication as well as striving for simplicity, imperative to manage the ever increasing complexity of computer systems.

He works for Novartis in Basel since 17 years in roles with increasing responsibility. He started as a Safety System Administrator and then moved to a Business Analyst role in the Pharmacovigilance area before he took over a global team of QA IT Systems and Process Experts responsible for all IT systems in Novartis Pharma Development. Since almost 2 years, he is also providing QA oversight over clinical data management and statistics processes with his team.

He was co-leading the DIA/ISPE CSV workshop recently held in Basel, is actively supporting the organization committee of the DIA EU Clinical Forum and he is a member of the DIA Advisory Committee for Europe, Middle East & Africa.

Excellence in Service Award Breffni MartinDirector, Regintel, Ireland

Breffni Martin was educated at St Micheals College Dublin and Cistercian College Roscrea. At University College Dublin he received his BSc specialising in biochemistry in 1983. Thereafter he worked for a number of major pharmaceutical companies in the area of late stage drug development, in both a scientific, regulatory and information technology context, as well as in environmental management. These include GlaxoSmithKline, Bristol-Myers Squibb, Laboratoire Guerbet and Fermenta (a Pharmacia spin-off). In 1997 he joined IMS Health (IDRAC, a regulatory intelligence company) where he developed regulatory content covering North America, Japan, Australia and elsewhere, including a dedicated FDA Advisory Committee intelligence business. He subsequently set up and operated regintel and CanReg (Europe), regulatory intelligence and services companies.

He is a long-standing member of the DIA a regular programme chair, speaker and contributor to DIAs meetings and publications. He was co-leading the DIA/ISPE CSV workshop recently held in Basel and he served on the DIA Advisory Committee for Europe from 2004 2009.



10

PROFESSIONAL POSTERS

PROFESSIONAL POSTER AUTHORS

A selected group of professional poster presenters will share theirresearch results in various topics. Visit the hallway of Hall Bordeaux on Level 3 to view the Professional Posters.

P1 Tatyana Benichiva, Professor Drug Regulatory Affairs, Faculty of Public Health, Medical University Sofia, Bulgaria

HTA Methodologies of Novel Treatments (T.Benisheva, G.Petrova, A.Stoimenova, A.Savova, D.Sidgimova,

P.Trendafilova, V.Petkova)

P2 Romuald Braun, CEO, Uanotau, Switzerland How IDMP Can Help Structure Product Information/ Labelling

P3 Magadalena Bujar, Research Analyst, Centre for Innovation in Regulatory Science (CIRS), UK

Expediting the Availability of New Medicines: What Role Do Priority Pathways and Special Designations PlayiIn ICH Countries

P4 Daniele De Martini, Academic Professor, Universit Di Milano Biocca, Italy

Evaluations When Adaptation by Design Is Applied

P5 Claudia Hernandez, Associate Director Regulatory Affairs, Sunovion Pharmaceuticals Inc., USA

Clinical Trial Application Approval Rates for Global Paediatric Placebo Controlled Trials

P6 Sherri Hubby, Director, US Quality Assurance, Premier Research Group, USA

A Compare and Contrast of Significant Changes in Clinical Trial Regulation of FDA, EMA and PDMA and Inspectional Readiness

P7 Tatsuya Ito, Visiting Fellow in Research Integrity, Research and Enterprise Development, University of Bristol, UK

Comparison of the Research Governance Frameworks for Research Involving Humans, Their Tissue and/or Data in the UK and Japan

P8 Christa Janko, Project Manager, Medical University of Vienna, Austria

Stay Ahead in Biomedical Sciences Catch the LifeTrain

P9 Heinrich Klech, Medical University of Vienna, Austria The Specialist in Medicines Development (SMD) A Global

Certification Programme by PharmaTrain Federation

P10 George Mathis, CEO, Appletree CI Group AG, Switzerland Clinical Development of Medical Devices in Europe and the USA

Differences and Similarities

P11 Magdalena Matusiak, Manager, Clinical Development, KCR SA, Poland

Safety Monitoring and Reporting in Clinical Trials Ensuring Quality and Effectiveness

P12 Stella Mokiou, Research Scientist, UBC: An Express Scripts Company, UK

Use of Patient-Reported Outcomes in Post-Authorisation Safety Studies: A Targeted Review

P13 Marie Lou Munson, Safety Science Leader, Genentech, USA Application of the DILI Expert Working Group Criteria in the

Assessment of Hepatotoxicity

P14 Toshinori Murayama, Professor, Kanazawa University Hospital, Japan

Toward The Advancement for Investigator- Initiated Clinical Trials A Challenge From Kanazawa/Hokuriku in Japan

P15 Nermina Nakas, Inventiv Health Clinical, USA Pharmacovigilance and Safety: Pediatric Research Challenges

P16 Sue Rees, QPPV, Executive Director Global Safety, Amgen Ltd, UK Pharmacovigilance Process Redesign Changing the Wheels Whilst

the Car Is Moving

P17 Joerg Seebeck, Chief Medical Officer, PrimeVigilance Ltd, UK QT-Prolonging Effects of Biological Drugs in Humans: A Systematic

Review of a Literature and Public Adverse Event Database

P18 Iris Tam, Director, Managed Care Medical Communications, Genentech, USA

A Review of the World Health Organization (WHO) Essential Medicines List (EML) Decision Making Process

P19 Marie Trad, Quintiles, France Epidemiological Data: An Untapped Resource for Developing an

Operational Strategy for Clinical Trials in Rare Diseases

P20 Jasper Van Berkel, National Institute of Public Health and the Environment, the Netherlands

Empowerment of Patients in Online Discussions about Medicine Use

P21 Linda Van Kerkof, National Institute of Public Health and the Environment, the Netherlands

Characterisation of Apps and Other E-Tools for Medication Use

P22 Angela Van der Salm, Director Pharmacovigilance, Dada Consultancy B.V., the Netherlands

Potential for Medication Errors or Off-label Use Practical Challenges

P23 Judy Walker, Drug Safety and Product Advisory Services, Quintiles, USA

Benefit-Risk Assessments (BRAs) of an Established Products Portfolio (M. Mease and J. Walker)

P24 Janine Collins, Senior Director, European Risk Management, United Biosource Corporation, Switzerland

Failure Modes and Effects Analysis strategy to minimise and mitigate risks associated with home administration of drug


11

STUDENT POSTERS | PATIENTS ADVOCATES

STUDENT POSTER AUTHORS

Student abstracts selected by the review committee, addressing similar topics to those in the programme, will be on display in the hallway of Hall Bordeaix on Level 3.

Presenters will be able to discuss their work during the coffee and lunch breaks on Tuesday, 14 April 2015. An awards ceremony will be held on Tuesday, 14 April 2015 at 17:45 at the DIA Booth in the exhibition hall to award the first and second place student poster winners.

Poster Selection Committee:Gaby Danan, Pharmacovigilance Expert, FranceAngelika Joos, Executive Director, Global Regulatory Policy, MSD (Europe) Inc., BelgiumBirka Lehmann, Head of Executive Department EU and International Affairs of the Federal Institute for Drugs and Medical Devices (BfArM), Germany

SP1 Nadia Amaouche, University of Paris-Sud, France European Paediatric Regulation launched in 2007: Feedback since

its implementation and perspective for 2017 and beyond

SP2 Liban Ashkir, University of East Anglia, UK The ECG effects of Moxifloxacin in thorough QT Trials: Systematic

review exploring the impact of design features

SP3 Amandine Beras, Claude Bernard Lyon 1, France The new version of international electronic transmission of adverse

drug reactions: E2B (R3)

SP4 Emeline Blanc, Claude Bernard Lyon 1, France Counterfeit drugs: means and technologies to fight against this peril

in ICH countries

SP5 Ioana-Laura Bogdan, Carol Davila University of Medicine and Pharmacy, Romania

Pharmacy-ethics and economic constraints

SP6 Marija Simaitien, Lithuanian University of Health Sciences, Lithuania

Prevalence, risk factors and self-management strategies of primary dysmenorrhea among Lithuanian women

SP7 Solne Viera de Araujo, Claude Bernard Lyon 1, France What could be publicly retrieved from adverse drug reaction

databases? An overview of 10 pharmacovigilance databases

PATIENT ADVOCATES AT THE EUROMEETING

Vesna Aleksovska, Association of citizens for rare diseases - Life with

Challenges, Republic of Macedonia

Dimitris Athanasiou, Parents Project MDA Hellas, Greece

Claudia Crocione, HHT ONLCUS - Italian Association for Hereditary

Hemorrhagic Telandiectasia, Itlay

Ivana Dragoievic, HRONOS, Serbia

Esraa El-Sherbiny, CanSurvive, Egypt

Elisabetta Gecchele, Acondroplasia Insieme Per Crescere - ONLUS, Italy

Tess Harris, Polycystic Kidney Disease Charity, UK

Alexander Kosenko, Russian Union of Patients and Patients Organisations

of Rare Diseases, Russia

Boris Machavariani, The Alliance of CMI Patients of Georgia, Georgia

Ananda Plate, Myeloma Patients Europe (MPE) AISBL, Belgium

Ieva Plume, PHA Latvia, Latvia

Jill Prawer, LPLD on RareConnect, UK

Ana Sofia, S Cardoso, APCL, Portugal

Antonella Esposito, Angeli Noonan Onlus, Italy

Juan Fuertes-Guillen, Pulmonary Hypertension Association (PHA) Europe,

Spain

Anne Hugon, AFG French Glycogen Storage Disease Association, France

Mary Lynne Van PoelGeest-Pomfret, World Federation For Incontinent

Patients (WFIP), the Netherlands

Nicola Bedlington, EPF, Belgium

Rob Camp, EURORDIS, France

Jan Geissler, EUPATI, Belgium

David Haerry, European AIDS Treatment Goup, EU

Francois Houyez, EURORDIS, France

Alistair Kent, Genetic Alliance, UK

Alison Lightbourne, IAPO, UK

Badri Rengarajan, International Pemphigus and Pemphigoid Foundation,

United States

Bettina Ryll, Melanoma Patient Network Europe, Belgium

Christoph Thalheim, European Multiple Sclerosis Platform, Belgium

Barbro Westerholm, AGE Platform, Sweden

Phil Willan, Healthcare Quality Improvement Partnership (HQIP), UK

Yann Le Cam, Eurordis, France

Durhane Wong-Reider, Canadian Organization for Rare Disorders, Canada

PATIENT WELCOME LUNCHMonday, 13 April 2015 | 12:30 - 14:00 | Room Arlequin Level 3Reserved for patient representatives and patient speakers only

Overview of the EuroMeeting scientific programme and an opportunity for patient representatives to meet and network with each other prior to the conference.

DIA Communities: Get Engaged. Develop Your Horizons.

Together We Can Do So MuchDIAs Communities provide our members with an opportunity to build relationships across disciplines and around the world and at the same time contribute to improving the process of health care product development.

DIA Communities provide a way for members across the globe to interact with their peers and to form cross-disciplinary teams. It is here that members share information, raise concerns, mentor one another, and find answers togetheraccomplishing more as a group than any one person could accomplish alone. DIA members are encouraged to join those Communities that match their interest areas.

DIA Communities Meet & Eat Tuesday, 14 April 2015 | 13:00-14:00Room Arlequin, Level 3

Are you active in a Community? Come attend a relaxed, informal and rare opportunity to meet your fellow Community members face-to-face. Enjoy your lunch and share your experiences with those who are curious about DIA Communities.

New to DIA? Not part of a Community? Youre invited too! This is an opportunity to ask questions and learn more about this member benefit. Look for DIA Community members who will be wearing a special button.

Scan this QR code to learn more about DIA Communities

To learn more about DIA Communities visit the DIA Booth #3.D17 or stop by the Membership Lounge located on Level 3 or scan the QR code below.

DIA Membership

DIA and You: Driving Ideas to Action

Join DIA. Visit DIAGlobal.org/Membership

To learn more about DIA Membership visit the DIA Booth # 3.D17 or stop by the Members Lounge located on Level 3 or scan the QR code below.

DIA Members Lounge Hall Bordeaux Level 3

Are you looking for a space to relax or connect with other DIA members? Stop by the Members Lounge located in the Hall Bordeaux on Level 3.

This exclusive lounge offers members a place to take an important call, get online, charge devices or just relax.

Coffee and drinks are served throughout exhibition opening hours and charging stations for your electronic devices are available. Make sure you bring your membership card to identify yourself as a DIA Member at the desk. Access exclusively for DIA Members.

Note: if you have not yet received your new membership card, have it printed onsite in the Members Lounge.

Scan this QR code to learn more about

DIA Membership

Its all about connections With DIA, people and ideas come together on a global scale to accelerate innovation and identify solutions. DIA provides invaluable forums to network with your peers, build new relationships, share ideas, and gain knowledge that can improve health care around the world.

As a DIA member, you will:

Be a part of a global network, working to advance health care product development

Work closely with like-minded professionals to solve real issues through DIA Communities and Scientific Task Forces

Enjoy discounts on more than 200 educational opportunities offered worldwide each year

Keep informed of news as it happens with our daily enewsletter

Learn the latest on health care products and regulatory science through our bi-monthly news magazine

Read about recent discoveriesand submit your own papersthrough our peer-reviewed scientific journal

The more you engage, the more you gain.

Together we can create a world where improved health is a reality for everyone

The goal of DIA is simple: To help you gain the knowledge you need to improve health and wellbeing worldwide.



16

EM Tutorial 1 | Monday 13 April 2015, 09:00-12:30

Room 252B Level 2

HOT TOPICS IN PHARMACOVIGILANCE AND ADVERSE REACTION REPORTINGSabine Brosch, Principle Scientific Administrator, European Medicines Agency, EUGaby Danan, Pharmacovigilance Expert, France

Taking into account the implementation experience with GVP module VI, which came into force on 2 July 2012, this tutorial will focus on frequently asked questions from stakeholders with regards to the day-to-day operational aspects.

In addition, practical considerations on the update of GVP module VI focusing on a simplification of the reporting of suspected adverse reactions from non-interventional post-authorisation studies, compassionate use and patient support programmes will be addressed.

At the start of 2015, the European Medicines Agency will launch a new process of monitoring medical literature for selected substances and selected medical literature in line with the provisions set out in Article 27 of Regulation 726/2004. The tutorial will provide the opportunity to discuss first experiences of marketing authorisation holders (MAHs) and to address specific implementation questions.

Article 24 of Regulation 726/2004 also outlines a new approach for marketing authorisation holders to access EU adverse reaction reports directly in EudraVigilance, following the successful outcome of an audit of the European pharmacovigilance database. In preparation of these changes, the EudraVigilance Access Policy will be updated to define the data elements of Individual Case Safety Reports (ICSR) for which access can be provided in compliance with EU personal data protection legislation. The tutorial will provide an opportunity to discuss those data elements in support of the marketing authorisation holders pharmacovigilance obligations.

The tutorial will conclude with a detailed discussion of the reporting principles in line with the new ICH Individual Case Safety Report E2B (3) guideline thereby highlighting new concepts such as causality assessment at event level, drug reaction relatedness, amendment reports and reporting of special situations (e.g. counterfeit medicines, product defects, medication errors).

Learning ObjectivesAt the conclusion of this tutorial, attendees will be able to: Address FAQs on GVP Module VI Management and reporting of adverse

reactions to medicinal products and recent updates Discuss the new process for monitoring of medical literature by the EMA

and the potential impact on MAHs Describe the principles of access to EudraVigilance based on revised

policy Discuss reporting principles based on the new ICH ICSR E2B(R3)

guideline

Target AudienceThis tutorial is designed for Qualified Persons Responsible for Pharmacovigilance (QPPVs) and individuals involved in Pharmacovigilance, clinical development, information management, and safety databases.


Room 253 Level 2

INTERACTIONS BETWEEN REGULATORY AND LEGAL IN INTELLECTUAL PROPERTY, PRODUCT LIABILITY AND COMPETITIONGenevive Michaux, Counsel, Hunton & Williams, Belgium Karina Hellbert, Partner, FIEBINGER POLAK LEON RECHTSANWLTE GmbH, Austria

Interactions between the regulatory regime and intellectual property (for example, SPC, data exclusivity), product liability (e.g., off label information in summary of product characteristics (SmPC), competition (for example, public procurement) and privacy (e.g., clinical trials) rules are increasing. As a result, those matters can no longer be approached in isolation, and a more comprehensive perspective is required when addressing regulatory issues.

Learning ObjectivesAt the conclusion of this tutorial, participants will be able to: Explain and discuss the basics of intellectual property, product liability

and competition rules applicable to medicinal products Identify and better address the regulatory issues that present an

intellectual property, product liability or competition aspect

Target AudienceThe tutorial is designed for non lawyers with good regulatory experience and for regulatory lawyers who want to learn the basics of intellectual property, competition, and product liability rules as they apply to the pharmaceutical sector.


Room 251 Level 2

ANALYSIS OF SAFETY DATA FROM CLINICAL TRIALSJrgen Kbler, Global Head, Clinical Design, Analysis and Reporting, CSL Behring AG, GermanyChristine Wichems, Global Head of Medical Writing and Disclosure, CSL Behring GmbH, Germany

This tutorial is a combination of theory, guidelines, practical considerations, and real-life solutions for those working in the clinical development environment (pharmaceutical, biotech industry, or CRO). The aim of this tutorial is to provide a basic understanding of the underlying methodology and the current guidelines on safety data. Aspects of the planning of clinical trials as well as the problems and pitfalls during the analysis of safety data will be presented. Opportunities for prospective planning of safety analysis at the project level will be discussed. The presentations will also include case studies.

OPTIONAL EUROMEETING PRE-CONFERENCE TUTORIALS

MONDAY, 13 APRIL 2015 | 09:00-12:30


17

Learning ObjectivesAt the conclusion of this tutorial, participants should be able to: Examine relevant guidelines and regulatory requirements for clinical

trials Recognise how to contribute to safety analysis plans Assess statistical safety analysis and identify pitfalls in safety analysis Recognise the impact of benefit/risk assessment in safety data

Target AudienceThis tutorial is designed for biostatisticians, medical writers, clinical researchers, drug safety specialists, project managers, and investigators.


Room 252A Level 2

PRIVACY AND PERSONAL DATA PROTECTION IN DRUG DEVELOPMENTPierre-Yves Lastic, Associate Vice President, Chief Privacy Officer, Sanofi, France

This tutorial addresses issues in data privacy today: Why is personal data protection important? Principles of personal data protection, based on the European

regulations Overview of worldwide regulations and the differences between them Specific regulations for biomedical research and pharmacovigilance

impacting drug development How to comply? Information & consent Communication & training IT security & validation Legal instruments (contracts, Safe Harbor, BCRs) Data Privacy organisation

Learning ObjectivesAt the conclusion of this tutorial, participants should be able to: Understand the principles of personal data protection in the European

Union Understand the differences between European, US and Asian data

privacy regulations Have a basic knowledge on how to comply with regulations in the

specific field of drug development

Target AudienceThis tutorial is designed for: All individuals involved in the organisation and management of clinical

trials and pharmacovigilance, or handling data collected to perform these activities

Professionals working with health data, clinical data, genetic data, tissue samples, medical imaging, mobile health apps, and any kind of personal data in the field of drug development


Room 241 Level 2

MOVING FROM RISK MANAGEMENT TO BENEFIT/RISK MANAGEMENT EMBEDDING PHARMACOVIGILANCE PRINCIPLES INTO THE PRODUCT LIFE CYCLEShelley Gandhi, Director Pharmacovigilance and Drug Safety, NDA Group, UKWilliam Richardson, Medical Advisor, NDA Group, UK

Pharmacovigilance, or the activity of monitoring the safety of medicines in clinical use and taking appropriate action to minimise risk, is governed by a range of new EU legislation, a new Pharmacovigilance Risk Assessment Committee and guidance. The value that can be gained from adopting a benefit/risk management system not only addresses known and potential risks to support the current regulatory status of product but also will feed into the further development of a product as regard new indications and potentially moving from prescription only to over the counter.

This tutorial will discuss how access to robust evidence on emerging risk in post-authorisation phase, good data on how a medicine is used in clinical practice and data on background rates in the exposed population gathering evidence throughout the product lifecycle will help move companies to a benefit/risk system. The ultimate challenge is working towards an integrated regulatory system so you can query across all information within a company, designing safety studies, monitoring the effectiveness of the risk management systems and gather robust evidence from clinical practice.

The lessons learned and our experiences so far with post-authorisation commitments (e.g. BRMPs, PASS, PSURs) will be reviewed as will whether these commitments really do support an acceptable benefit/risk profile. This will include the novel approaches to managing benefit/risk to meet the needs of licensing medicines in biotechnology such as advanced therapies. Communicating benefit/risk will also be discussed as the new legislation will push for greater patient involvement within a benefit/risk system. Better methodologies and tools are required to support this integrated approach and adoption of a quality management system across global enterprise could achieve this.

Learning ObjectivesAt the conclusion of this tutorial, attendees will be able to: Learn about what are effective strategies and the current thinking on risk mitigation in the context of benefit throughout the product lifecycle. Access to robust evidence on emerging risk is critical Discover what the principles are for proportionate risk based assessment Find out about hurdles which get in the way to a systematic approach and how these might be tackled

OPTIONAL EUROMEETING PRE-CONFERENCE TUTORIALSMONDAY, 13 APRIL 2015 | 09:00-12:30



18

Target AudienceProfessionals in companies or regulatory authorities who are involved in pharmacovigilance operations and with responsibilities for post marketing clinical safety including those who are involved in:

Pharmacovigilance Regulatory Clinical research Risk management Medical product safety assessment Data analysis Epidemiology Labelling Quality assurance and compliance


Room 242A Level 2

MODELLING AND SIMULATION (M&S) / MODEL INFORMED DRUG DIS-COVERY AND DEVELOPMENT (MID3)Amy Cheung, Clinical Pharmacometrician, AstraZeneca R&D, UKFlora Musuamba Tshinanu, Research Associate, University of London, UK

The tutorial will be an opportunity to understand better the value of Modelling and Simulation (M&S) / Model Informed Drug Discovery and Development (MID3) in the development of new medicinal products, and the impact of MID3 on regulatory decision making and product labelling.

The tutorial will be divided in three parts.

First, the basic concepts and principles of modelling and simulation will be recalled and the added value over two stage approach will be discussed using examples.

The second part will be dedicated to the use of M&S and MID3 in the context of drug development and the impact it can have in key decision making: study design optimization, data analysis, dose selection and extrapolation will be discussed.

The last part will be dedicated to model evaluation given the purpose. For each of the application of M&S/MID3 in drug development, the relevant criteria for model evaluation will be presented and discussed. Focus will be made on data needed, model assumptions and uncertainties and fitting performances and examples will be provided for good and bad practices. Challenges in current practices (given the current regulatory requirements) will also be discussed.

Learning ObjectivesAt the conclusion of this tutorial, attendees will be able to: Recall the basic concepts of Modelling and Simulation and its advantages

over other approaches for Pharmacokinetics (PK) / Pharmacodynamcis (PD) description and predictions

Discuss/explain the added-value and importance of Modelling and Simulation Model Informed Drug Discovery and Development in the context of drug development

Outline/discuss the criteria for model evaluation given the purpose in drug development setting

Target AudienceModeller, biostatisticians, medical writers, clinical researchers, drug safety specialists, project managers, investigators, clinical practitioners, physicians, regulators, with basic experience/awareness of Modelling and Simulation (M&S)/Model Informed Drug Discovery and Development (MID3).

OPTIONAL EUROMEETING PRE-CONFERENCE TUTORIALS

MONDAY, 13 APRIL 2015 | 09:00-12:30

21-23 SEP VIENNA, AUSTRIA | #15543Quality by Design - New concepts for chemical and biotech product development and optimisation

Participants from pharmaceutical, biotech and generic industry as well as regulators will learn how to use QRM, Process Characterisation, DoE, Development of a Design Space and Control Strategy, as well as the tools of Knowledge Management (KM).

EUROPE, MIDDLE EAST & AFRICA

2015 TRAINING COURSESCHEMISTRY, MANUFACTURING & CONTROLS

CF Tutorial 1 | Monday 13 April 2015, 14:00-17:30

Room 242A Level 2

A LEGAL UPDATE: RECENT AND CURRENT DEVELOPMENTS IN EUROPEAN PHARMACEUTICAL LAWJohn Lisman, Lawyer, Lisman Legal Life Sciences, NetherlandsKoosje van Lessen Kloeke, Life Sciences Lawyer Partner, Leijnse Artz advocaten, Netherlands

Pharmaceutical law is at the basis of daily business in the pharmaceutical and biotech industry. In the last few years many developments have occurred in the field of pharmaceutical law, as well as in the marketing authorisation practice.

This tutorial brings you up to date in respect to marketing authorisation for Biosimilars - Paediatric Regulation - Advanced Therapy Medicinal Products Regulation - Clinical Trials Regulation - Early Access to promising new medicines - Adaptive licensing - Transparency. For each of these topics the most relevant highlights will be presented in an interactive manner. Furthermore, recent ECJ case law relevant for the audience will be discussed.

Learning ObjectivesAt the conclusion of this tutorial, attendees will be able to: Apply pharmaceutical legislation in their daily practice. Explain the changes in pharmaceutical legislation in their companies. Participate in an interactive way in the DIA Clinical Forum sessions

dealing with new legislation, e.g. Clinical Trial Regulation. Recognise recent case law of the Court of Justice and the General Court.

Target AudienceNon-lawyers who work in a regulated (clinical or medical) environment from industry, government agencies and competent authorities. Lawyers from industry, government agencies and competent authorities who have recently started in this position.

CF Tutorial 2 | Monday 13 April 2015, 14:00-17:30

Room 242B Level 2

ENSURING DATA QUALITY AND DETECTING POTENTIAL FRAUD/GCP MISCONDUCTMarc Buyse, Chairman, IDDI (International Drug Development Institute) Inc., USAStephen George, Department of Biostatistics and Bioinformatics, Duke University School of Medicine, USA

A large number of options are available to operationalise the recommendations outlined in the FDA Guidance on Risk-Based Monitoring (RBM) and the EMA Reflection Paper on Quality by Design. TransCelerates position paper provides a detailed view of some of the tools and processes that can be used for a successful implementation of RBM. This tutorial session will review the landscape of current practices with a focus on data quality and integrity. The session will demonstrate how analytical tools can support an RBM strategy by analysing clinical data to identify risks in some sites arising from lack of training, misunderstanding, carelessness, negligence or even fraud.

The session will cover the tenets of CSM (Central Statistical Monitoring) and KRI (Key Risk Indicators). The types of findings that can be detected by these two approaches will be discussed and illustrated with concrete examples. Importance will be given on how the cause of the findings can be identified and classified by seriousness, from poor protocol understanding to intent to cheat. The operational implications of the findings in terms of corrective actions to take will also be discussed.

Learning ObjectivesAt the conclusion of this tutorial, attendees will be able to: Implement the recommendations outlined in the FDA Guidance on Risk-

Based Monitoring Demonstrate how the use of objective, analytical tools can be used to

identify sites at risk and detect potential fraud Interpret findings from KRIs and CSM to drive operational corrective

actions

Target AudienceProfessionals involved in the management of clinical trials: Data Managers, Statisticians, Medical Reviewers, Study Physicians, Project Managers, Study Coordinators, Quality Managers and Senior Management.

SAVE TIME AND TRAVEL COSTS with IN-HOUSE TRAININGAll DIA Training Courses can be held on your premises and tailored to your needs.

Get the best value and train your whole team!

OPTIONAL CLINICAL FORUMPRE-CONFERENCE TUTORIALSMONDAY, 13 APRIL 2015 | 14:00-17:30

The 8th Annual Clinical Forum will take place in parallel of the 27th Annual EuroMeeting.Participants have the opportunity to register for optional tutorials for both conferences.



20

EU REGULATORY TOWN HALL MEETING

50th Anniversary of the EU Pharmaceutical Legislation

MONDAY, 13 APRIL | 13:30-15:00ROOM BORDEAUX LEVEL 3

Moderator: Fernand Sauer, French Academy of Pharmacy, Former Executive Director of the European Medicines, France

The Regulatory Town Hall will celebrate this unique occasion with a panel discussion on how we can build on past achievements to address the challenges ahead.

The European Commission will make an opening address followed by the panel discussion covering these subjects: Building the European Medicines Regulatory Network. How

is the house looking today and in the future? What can regulatory authorities do to support innovation? Implementing new legislation - what are the challenges,

what are the solutions?

Panellists:Xavier De Cuyper, Chief Executive Officer, Federal Agency for Medicines and Health Products, BelgiumViola Macoli arini, Head of Agency, Agency for Medicinal Products and Medical Devices (HALMED), Croatia Guido Rasi, Principal Adviser, European Medicines Agency, EUAndrzej Rys, Director of Health Systems and Products, European CommissionChristoph Thalheim, Director External Affairs, European MS Platform, BelgiumChrista Wirthumer-Hoche, Head, Austrian Medicines and Medical Devices Agency (AGES), Austria

50 YEARS OF EDQM Preparing for the Future to Continuously Ensure the Quality of Medicines

WEDNESDAY, 15 APRIL | 11:00-12:30ROOM MAILLOT LEVEL 2Session Chair: Susanne Keitel, Director, EDQM/Council of Europe

The European Directorate for the Quality of Medicines and HealthCare (EDQM) turned 50 in 2014. While EDQM and its 37 Member States are proud of the tremendous achievements over the past 50 years, globalisation and technical and scientific developments in all fields pose constant challenges.

See page 46 for details.

HAS SATELLITE SYMPOSIUM

MONDAY, 13 APRIL | 11:00-12:30 ROOM MAILLOT LEVEL 2

Introduction of Innovative Drugs and Devices in France- Recent Advancements in HTA, Pricing and Reimbursement

Moderator: Franois Meyer, Advisor to the President, International Affairs, Haute Authorit de Sant (HAS), France

How does France, one of Europes largest countries, deal with the assessment and introduction of Innovative Drugs and Devices? The session will provide an overview by HAS with regard to its first-year experience in health economic assessments of new health products, examine the current situation and challenges of pricing for innovative drugs and devices and describe the evaluation and decision-making process at the level of hospital committees.

Introduction of Health Economics in the HTA Process for New Drugs and Medical Devices in France: First-year experienceJean-Luc Harousseau, President, Haute Autorit de Sant (HAS)

Price Determination for New Drugs and Medical Devices in France: Current situation and perspectives Dominique Giorgi, President, Comit Economique des Produits de Sant (CEPS)

Role of Institutional Committees for Admission of New Medicines and Innovative Medical Devices in French Hospitals (COMEDIMS)Philippe Lechat, French Society of Pharmacologie and Therapeutics (SFPT)

Experience of Early Dialogue Pilots Conducted within SEED and EUnetHTA: Coordinators perspective on the feasibility of implementing a permanent model in Europe and the promise of improving clinical data collection prior to market approvalMira Pavlovic, Deputy Director DEMESP, Haute Autorit de Sant (HAS)

The session will conclude with HASs experience in coordinating the early dialogue/scientific advice activity in Europe through the EUnetHTA and SEED projects, highlighting the benefits of a collaborative approach between HTA agencies, regulatory and health product developers.


21

OPENING PLENARYMONDAY, 13 APRIL 2015 | 16:00 - 17:45 | BLUE ROOM LEVEL 2

ADVANCING INNOVATION TO COMBAT THE GLOBAL BURDEN OF DISEASE: A CALL TO ACTION

A panel of respected leaders from academia, industry, health agencies, patient advocacy, and the payer

community will share perspectives on the dichotomy between evolving diseases, well-known conditions,

and neglected and rare diseases, contrasted with the challenges that exist in understanding the physiology

of these conditions and developing therapies to treat them.

The challenges include topics such as the efficiency of R&D, access to health care, ethics and transparency

issues related to the clinical trials, and the increasing challenges related to investments required to bring

safe, effective, economically-viable, and accessible products to patients worldwide.

All stakeholders see the challenges, but each from a different perspectives: health authorities in terms of

the public health preservation, industry in terms of ROI, academics in terms of generating new knowledge,

payers in terms of risk management, patients in terms of managing their disease.

During this provocative and stimulating discussion, the panellists will work to converge on a shared vision

and call to action.

Keynote Speakers:

Olivier Charmeil, Executive Vice President Vaccines, Sanofi, France

Andrew Morris, Professor, University of Edinburgh, Chairman and Centre Director, Farr Institute, UK

Panellists:

David Haerry, Board of Directors, European AIDS Treatment Group, EU

Hugo Hurts, Executive Director, Medicines Evaluation Board (MEB), the Netherlands

Guido Rasi, Principal Adviser, European Medicines Agency, EU

Moderator:

Maureen Kenny

Editor, Scrip Regulatory Affairs, Informa Business Intelligence, UK



22

EM0101 | Tuesday, 14 April, 09:00-10:30 | Blue Room Level 2

THE ROLE OF THE NEW CLINICAL TRIALS LEGISLATIONSession Chair:Lidia Retkowska-Mika, Director, Legal Department, Office for Registration of Medicinal Products, Poland

The objective of the session is to present and discuss major changes in the overall approach, assessment, approval and conduct of clinical trials in the EU introduced in the new legislation. New definitions, new procedures for sponsors and Member States, the role of ethics committees, new IT tools introduced in the legislation will be presented with the view to inspire discussion on the forthcoming challenges and the use of new legal instruments to make the EU a more attractive area for the conduct of clinical trials.

Key Content of the New Clinical Trials Legislation- Setting the sceneLidia Retkowska-Mika, Director, Legal Department, Office for Registration of Medicinal Products, Poland

EMA Preparedness for the Implementation of the New LegislationAna Rodriguez, Head of Clinical and Non-Clinical Compliance, European Medicines Agency, EU

Challenges in the Ethical Review of Clinical Trial ApplicationWojciech Maselbas, Vice President, Association for Good Clinical Practice, Poland


CLEARING THE PATH FOR INNOVATIONS IN DRUG DEVELOPMENTSession Chair:John Lisman, Lawyer, Lisman Legal Life Science, the Netherlands

This session will focus on the activities stakeholders perform to prepare and implement the novelties in the Clinical Trials Regulation. What will Member States do to lift their new responsibilities? How will industry adapt to the new regulatory set-up and where do patients foresee strengths and challenges?

Pathways for Early Access to Medicines - How can the use of existing tools be improved from an industry perspective? Sabine Atzor, Head of EU Regulatory Policies, F. Hoffmann-La Roche, Switzerland

Member States Activities for Implementing Innovative Initiatives in Respect of Clinical TrialsChristian Schneider, Senior Medical Officer, Danish Health and Medicines Authority (DKMA), Denmark

Patients Expectations in Respect of the Development of New MedicinesBettina Ryll, Founder, Melanoma Patient Network Europe, Sweden


PRIMARY FACTORS FOR TRIAL LOCATIONSession Chair:Dorthe Poulsen, Chief Legal Adviser, EU-Coordinator, Danish Health and Medicines Authority, Denmark

Which factors are decisive for the decision to conduct a clinical trial in a certain part of the world? There is no doubt that the EU competes with other parts of the world such as the BRIC countries. In this session, well have a look at the EU in a more global context.

Country Allocation in Global PerspectiveHenrik Troelsen, Director, Global Trial Allocation, Novo Nordisk, Denmark

A Brazilian Perspective Speaker invited

The Swedish Way to Foster InnovationChrister Backman, EU Coordinator & Senior Expert, Medical Products Agency (MPA), Sweden

Theme 1 Access to Innovative Treatments: New R&D models and innovative clinical trial methodologies. The role of the new clinical trials legislation

Susanna Del Signore, Associate Vice-President Global Regulatory Affairs, Head of Global Regulatory Policy, Sanofi R&D, FranceDorthe Poulsen, Chief Legal Adviser, EU-Coordinator, Danish Health and Medicines Authority (DKMA), Denmark

Researching and developing new medicines demands high levels of innovation. This in turn delivers insights that benefits both society and patients. This research is dependent on clinical trials; without these, there would be no new medicines, no improvement of existing medicines and no evidence-based improvement in how we use current treatments.

Despite this, the number of clinical trials undertaken in Europe is decreasing. Clearly, if we wish to stimulate healthcare R&D, we need a robust, operative and cost-effective EU framework for clinical trials.

This theme will examine how we can use the new legal framework for clinical trials to create this environment. How can we make the best use of available tools whilst safeguarding the rights and safety of the clinical trial subject? What are the considerations for clinical trial design, such as location and disclosure requirements? Practically, how can we have a regulatory system that lets us respond quickly to the demands of an ageing population and a rapid rise in chronic conditions?


23

EM0105 | Wednesday, 15 April, 09:00-10:30 | Room 251 Level 2

IMPACT OF THE CLINICAL TRIAL REGULATION ON CMC FILING AND RELEVANT GMP REQUIREMENTSChair invited

The new Clinical Trial Regulation will be a key step towards supporting medical research in Europe, covering multiple aspects of the clinical trial, from the organisation, through implementation and the reporting process. The quality of CT material needs to be properly designed and appropriately manufactured. Which quality system should be applied? What are the expectations from regulators at this stage? What consequences for the industry?

This session will be a good opportunity, with appropriate timing, to go through it and clarify the legal and regulatory impact (legislation and delegated act) as well as identifying the challenges and opportunities for the pharmaceutical industry.

How CTR May Change IMP Supply and How Ready Are We? Anthony Moult, Director Clinical Supplies Operations, Daiichi Sankyo Development, UK

Impact of the CTR on Investigational Product Supply - Potential Challenges for the Industry Sabine Pfister, Senior Compliance Professional, Quality Compliance & Auditing, Novartis, Switzerland

EM0106 | Wednesday, 15 April, 11:00-12:30 | Blue Room Level 2

MEDICINES ADAPTIVE PATHWAYS TO PATIENTS (MAPPS) - HOLDING HANDS ACROSS THE LIFECYCLESession Chair:James Anderson, Head of Corporate Government Affairs, GlaxoSmithKline, UK

MAPPs refer to flexible development and access pathways within the current regulatory framework that balance early patient access, public health and societal benefits. To afford patients faster access to innovative medicines a fundamental and co-ordinated change is now required across all elements of the bioscience sector, with collaboration and partnership becoming a core capability for success. The requirements and expectations for sustainable change will be discussed from various perspectives.

Hans-Georg Eichler, Senior Medical Officer, European Medicines Agency, EU

Gigi Hirsch, Executive Director, CBI and Program Director, NEWDIGS, MIT, USA

Yann Le Cam, CEO, EURORDIS, France EM0107 | Wednesday, 15 April, 14:00-15:30 | Blue Room Level 2

MAPPS: FOSTERING COLLABORATIVE MODELS FOR CLINICAL DEVELOPMENT Susanna Del Signore, Associate Vice-President Global Regulatory Affairs, Head of Global Regulatory Policy, Sanofi R&D, France

Chris Chinn, VP and Head of Health Investment Evidence, GSK; Project Coordinator IMI GETREAL

Alastair Kent, Director General, Genetic Alliance UK

Nathalie Seigneuret, Senior Scientific Project Manager, Innovative Medicines Initative (IMI), Belgium

EM0108 | Wednesday, 15 April, 16:00-17:30 | Blue Room Level 2

DISCLOSURE OF INFORMATION - WHAT WILL BE DISCLOSED ACCORDING TO THE NEW SCHEME IN THE CLINICAL TRIALS LEGISLATION? Alexander Roediger, Director European Union Affairs, MSD (Europe) Inc., Belgium

The Clinical Trials Regulation requires that the EU database shall be publicly accessible unless, for all or part of the data and information contained therein, confidentiality is justified for certain reasons. Some factors that may justify confidentiality is the protection of commercially confidential information, the protection of confidential communication between Member States in relation to the preparation of the assessment report and the protection of personal data.

This session will take a closer look at the circumstances under which confidentiality is justified as listed in Art. 81(4), in particular commercially confidential information. It will also address the questions what constitutes an overriding public interest in disclosure and which legitimate factors from various stakeholders need to be taken into account? Finally, it will put these requirements into the broader context, in particular regarding EMAs Policy 70 On publication of clinical data for medicinal products for human use.

The presentations/discussion will shed light on controversial issues and a panel debate will follow.

The EMA Proposal The Agencys Attempt to Strike a Balance between Disclosure and Protection of Clinical Trials InformationAna Rodriguez, Head of Clinical and Non-Clinical Compliance, European Medicines Agency, EU

Disclosing Information about Clinical Trials- Where are the limits? The Industry Research PerspectiveFabien Peuvrelle, Director, Clinical Trials and Labelling Translations Group, Regulatory Affairs Europe, Celgene R&D, Switzerland

Public Access to Clinical Trials Data: The consumer perspectiveIlaria Passarani, Health Policy Officer, European Consumers Organisation (BEUC), Belgium

To What Extent Will the European Citizen Benefit from the Clinical Trial Information? Experiences from Empirical ResearchFrederic Bouder, Associate Professor, Maastricht University, the Netherlands



24

EM0201 | Tuesday, 14 April, 09:00-10:30 | Room 241 Level 2

NEW MEDICAL DEVICE REGULATIONS IN THE EUSession Chair:Sabina Hoekstra-van den Bosch, Global Regulations and Standards, Philips Healthcare, the Netherlands.

The Strengthening of the EU Medical Devices Regulatory SystemErik Hansson, Deputy Head of Unit, Health Technology and Cosmetics, European Commission, EU

New Legislation on Medical Devices Update on progress and future prospectsGraeme Tunbridge, Head of Medical Devices EU Policy, Medicines and Healthcare Products Regulatory Agency (MHRA), UK

A Notified Bodys Perspective on the Impact on Stakeholders of the Immediate Action Plan and the Proposed Medical Device Regulation Neil Adams, Director Operations and Delivery, Medical Devices, BSI, UK


IMPACT OF THE REGULATIONS IN PRACTICE ACROSS REGIONSSession Chair:Michelle OConnor, Director Regulatory Consulting, Icon Clinical Research, Ireland

Medical Devices Regulatory Pathways across the EU and US regions Michelle OConnor, Director Regulatory Consulting, Icon Clinical Research, Ireland

Ning Li, Vice President and Head, Asia/China Regulatory Affairs, Sanofi

Medical Device Requirements in India Daniel Verstappen, Vice President Quality & Regulatory Affairs Europe - EAGM India, GE Healthcare, Belgium


IMPACT OF THE REGULATIONS ON SUBSTANCE-BASED MEDICAL DEVICESSession Chair:Miranda Moussa, Manager for Medical Devices, AESGP, Belgium

In light of the growing interest in substance-based medical devices across Europe, the session will look into their particularities and consider ways to ensure that safe, effective and innovative substance-based medical devices continue to reach citizens. The latest European developments on the proposed regulation on medical devices and their potential impact on this category will be discussed.

A Notified Bodys Perspective on the Impact of the Proposed Medical Device Regulation on Substance-based Medical DevicesNeil Adams, Director Operations and Delivery, Medical Devices, BSI, UK

Adjusting the Medical Device Legislation to Substance-based Products- The perspective of one competent authority Judite Neves, Head of Medical Devices, Infarmed, Portugal

George Jessen, Member of the AESGP Committee on Medical Devices; Regulatory Affairs Manager, Procter & Gamble, UK

EM0205 | Wednesday, 15 April 09:00-10:30 | Room 242 Level 2

NEW IVD REGULATIONS IN THE EUSession Chair:Jess Rueda Rodrguez, Regulatory Affairs Director, European Diagnostic Manufacturers Association

MSdialog: A Novel System for Engaging Patients with Multiple Sclerosis in Routine Health Outcomes Reporting and MonitoringMark Thristan, Associate Director, IS - R&D - Embedded IT, EMD Serono, USA

View from a Notified BodyCatherine Holzmann, IVD department Manager, GMED Certification Division

Theme 2 Medical Devices and Combination Products

Paul Jansen, Global Head Medical Devices US, Sanofi, USASabina Hoekstra-van den Bosch, Global Regulations and Standards, Philips Healthcare, the Netherlands.

The healthcare industry is increasingly embracing the opportunities offered by medical devices, in vitro diagnostics combination products and health information technology.

This theme examines the trends in this rapidly evolving field. It will examine the regulatory challenges posed by these novel combination therapies, particularly the use of the biologics used in drug / device combinations. It will also consider the regulatory aspects of how information technology and health apps collect and use the data generated by modern medical devices. How can we ensure we that have a regulatory environment capable of reacting quickly to the needs of this dynamic sector and maximizing its benefits for the patient? The theme will also provide the latest news on the upcoming European Medical Device Regulation and In Vitro Diagnostic Regulation, highlight the latest trends in international medical device regulation and analyse consequences of the future European legislative changes for substance-based medical devices.


25

Theme 3 Advanced Therapies, Novel Treatments for Rare Diseases

Paula Salmikangas, Chair CAT, Senior Researcher, Finnish Medicines Agency (FIMEA), FinlandHenk Schuring, Group Vice President Regulatory Affairs - Europe, Genzyme Europe B.V., the Netherlands

We now have five years of experience of the Advanced Therapy Medicinal Products (ATMP) legislation. It is time to review whether this legislation has met expectations. Has it produced the numbers and effectiveness - of treatments expected? Although some products have already reached patients, the success rate has been low and there have been several issues over effectiveness.

This session will examine what we have learned from the ATMP legislation and whether it now requires revision. Will the new regulatory pathways developed for ATMP, such as adaptive licensing, change how we approach drug development? What lessons have the work with rare diseases and orphan drugs taught us, and how do we leverage those insights more widely?


APPROVAL PROCESS AND EXPERIENCE TO DATE/CHALLENGES AND SUCCESSFUL OUTCOMES OF ATMP DEVELOPMENT Session Chair: Beatriz Silva Lima, Advisory Board, NDA, UK

The progress on gene and cell therapies from regulation implementation will be addressed from European perspective. The rapid knowledge evolution will be illustrated with examples of products history from their research until their current stage.

An Overview to MAA Process for ATMPs and CAT Experience from Five First YearsPatrick Celis, Scientific Administrator, Committee for Advanced Therapies (CAT) Secretariat, European Medicines Agency, EU

Commercially Viable Options for ATMP ManufactureKlaus Maleck, CEO, TETEC AG, Germany

International Regulatory Co-Operation for ATMPsLiz Anne Gillham-Eisen, A/Director, Health Products and Food Branch, Health Canada, Canada

Anticipation of the Coming Changes in the EU Regulatory Framework for Companion DiagnosticsSylvie Le Gledic, Director, IVD/CDx, Voisin Consulting Life Sciences, France


HEALTH APPS: DEFINITION, REGULATION & USESession Chair: Erik Vollebregt, Partner, AXON Lawyers, the Netherlands

Erik Vollebregt, Partner, AXON Lawyers, the Netherlands

Brad Thompson, Member of the Firm, Epstein, Becker & Green

Pierre Leurent, CEO, Voluntis, France


CLINICAL, QUALITY & REGISTRATION CONSIDERATIONS FOR BIOLOGICS COMBINATION PRODUCTS DEVELOPMENT Session Chair: Paul Jansen, Global Head Medical Devices US, Sanofi, USA

There are a growing number of Biologics in active development, many of them in late stage development. This session will explore some of the key considerations and unique challenges of gaining approval of Biologics combination products.

EU/US Registration Dossier Challenges for Biologics/Device CombinationsMarielle Fournier, Director, Combination and Borderline Products, Voisin Consulting Life Sciences, France

Risk-Based Quality and Compliance Management in Clinical Trials with Combination ProductsMarina Malikova, Executive Director, Surgical Translational Research Operations and Compliance, Boston University School of Medicine, USA

The EU Framework for Combination Products A Regulatory ViewChrista Wirthumer-Hoche, Head, Austrian Medicines and Medical Devices Agency, Austria



26


QUALITY ASPECTS FOR AN ATMP (M3 OF MAA)Session Chair: Valerie Pimpaneau, Voisin Consulting Life Sciences, France

The development of ATMP comes with a complex set of CMC challenges that developers need to overcome in order to design an adapted control strategy. The complex nature of cell and gene therapy products combined with mode of actions that may not always be fully understood and often involve multiple pathways lead to quality assessment approaches likely to be unique to each product. This session will provide an overview of major quality issues encountered during the development of cell based medicinal products, discuss the difficulties encountered in developing potency assays and the value of using risk assessment to support development choices and quality assessment strategies.

Major Quality Issues for CBMPsMargarida Menezes-Ferreira, Senior Assessor and National Scientific Advice Coordinator, INFARMED, Portugal

The Challenges of Potency Assay Development of Cell and Gene Therapy ProductValerie Pimpaneau, Voisin Consulting Life Sciences, France

New Developments for GTMPsBastien Calmels, Quality Control Senior Manager, Transgene, France EM0303 | Tuesday, 14 April, 14:00-15:30 | Room 243 Level 2

ADAPTIVE LICENSING FOR ATMPS Session Chair: Hans Ovelgnne, Member SAWP, CAT; Medicines Evaluation Board, the Netherlands

In March 2014 the EMA launched a pilot project into adaptive pathways. The aim was to investigate a better use of real-world data in the licensing of medicinal products, in conjunction with HTAs. Several companies submitted products into this pilot, including a larger than expected proportion of ATMPs. What is special about ATMPs? ATMPs and the EMA Pilot Project o

15101_pgm

Documents

theme leaders

convergence of innovation

patient safetydia euromeeting

patient safety13

vaccine development

conference optional

dia board of directors

special development