2018 Speakers


Annemieke Aartsma Rus, Professor of Translational Genetics, Department of Human Genetics, Leiden University Medical Center

Karen Aiach, Chief Executive Officer, Lysogene

Luca Alberici, Chief Business Officer, MolMed

Sofie Alverlind, Project Manager, Swedish Association of Local Authorities and Regions

Sofie Alverlind at World Orphan Drug Congress 2018

Einar Andreassen, Senior Adviser, Norwegian Medicines Agency

Diego Ardigo, R&D Rare Diseases Unit Head, CHIESI Farmaceutici Spa

Francis Arickx, Head of the Directorate Reimbursement of Medicines and Pharmaceutical Policy,, National Institute for Health and Disability Insurance RIZIV-INAMI

Francis Arickx at World Orphan Drug Congress 2018

Dr Segolene Ayme, Founder of Orphanet & Rare Disease Expert in Residence, Inserm

Jasmin Barman, Scientific Advisor, SGP

Jasmin Barman at World Orphan Drug Congress 2018

Denis Belyakov, Executive Director, Union of Patients’ And Patients’ Organizations With Rare Diseases

Marian Bendik, Head of Gene Therapy, Process Development, Shire

Dr Michael Binks, VP, Rare Disease Clinical Research, Pfizer

Dr Michael Binks at World Orphan Drug Congress 2018

Catalin Bobirca, Medical Franchise Head, Rare Diseases, Oncology Region Europe, Novartis

Mathieu Boudes, Coordinator of PARADIGM, European Patients' Forum

Mathieu Boudes at World Orphan Drug Congress 2018

Meriem Bouslouk, Orphan drug policy adviser for Germany

Dr Meriem Bouslouk, Orphan drug policy adviser for Germany

Jacoline Bouvy, Senior Scientific Adviser, National Insitute for Health and Care Excellence

Sophia Brodin, Deputy Head of Department, Department of Value Based Pricing, The Dental and Pharmaceutical Benefits Agency T.L.V.

Sophia Brodin at World Orphan Drug Congress 2018

Nicholas Brooke, Executive Director, Patient Focused Medicines Development

Nicholas Brooke at World Orphan Drug Congress 2018

Anna Bucsics, Project Advisor, Mechanism of Coordinated Access to orphan medicinal products (MoCA)

Anna Bucsics at World Orphan Drug Congress 2018

Dr Andreas Busch, Chief Scientific Officer, Head of Research and Development, Shire Pharmaceuticals

Dr Andreas Busch at World Orphan Drug Congress 2018

Agnese Cangini, Health Economist, AIFA

Agnese Cangini at World Orphan Drug Congress 2018

Nathalie Cartier, Director, Institut National De La sante Et De La Recherche Medicale

Professor María Casado, Director, University of Barcelona

Fleur Chandler, Therapy Area Head of Respiratory Value Evidence and Outcomes, GlaxoSmithKline Biologicals

Fleur Chandler at World Orphan Drug Congress 2018

Maria Chatzou, Co-founder & Senior Advisor, Innovation Forum

James Christie, SVP Manufacturing and Supply Chain, MeiraGTx

Emily Crossley, Co-founder, Duchenne U.K.

Marcin Czech, Vice Minister, Poland, Ministry of Health

Avril Daly, CEO, Retina International

Avril Daly at World Orphan Drug Congress 2018

Dorica Dan, President, Romanian Pader Willi Association

Dorica Dan at World Orphan Drug Congress 2018

Tagle Danilo, Associate Director for Special Initiatives, National Center for Advancing Translational Sciences

Dr Sjef De Kimpe, Co-founder and SVP R&D, Auxesia Orion

Professor Michele De Luca, Scientist, Professor, University of Modena e Reggio Emilia

Dr Renato Dellamano, President, Medical Marketing Economics

Lisa Dilworth, Vice President Rare and Orphan Diseases, Synteract

Scott Dorfman, President & CEO, Odylia Therapeutics

Cinzia Dorigo, Executive Director Clinical Development, Syneos Health

Dr Cynthia Dukes, Vice President, Drug Development Vaccines, Infectious Disease, Women’s Health and Rare Disease, ICON plc

Anne-Virginie Eggimann, Vice President Of Regulatory Science, bluebird bio

Anne-Virginie Eggimann at World Orphan Drug Congress 2018

David Elvira Martinez, Director of the Catalan Health Service, CatSalut, Catalunya Health Service

Van Zyl Engelbrecht, Clinical Project Director EU, UBC

Van Zyl Engelbrecht at World Orphan Drug Congress 2018

Karen Facey, Senior Research Fellow, University of Edinburgh

Karen Facey at World Orphan Drug Congress 2018

Kelly Franchetti, Vice President, Global Patients Insights and Engagement, ICON plc

Pedro Franco, Director for Europe for Global Regulatory and Scientific Policy, Merck Serono

Pedro Franco at World Orphan Drug Congress 2018

Michela Gabaldo, Head of Alliance Management & Regulatory Affairs, Fondazione Telethon

Benjamin Gannon, Head of International Government Affairs, Public Policy & Patient Engagement, Vertex Pharmaceuticals

Pamela Gavin, Chief Strategy Officer, National Organization for Rare Disorders

Josie Godfrey, Director, National Institute for Health and Care Excellence

Josie Godfrey at World Orphan Drug Congress 2018

Prof Nathalie Goemans, Field Of StudyPediatric Neurology, Rehabilitation Medicine, University Hospitals Leuven

Rory Graham, Senior Director, Regulatory Affairs, EU and International Region, ICON plc

Dr Tim Guilliams, CEO, Healx

Dr Femida Gwadry-Sridhar, Co‐Founder and CEO, Pulse Infoframe Inc

Denis Horgan, Executive Director, EAPM (European Alliance for Personalised Medicine)

Denis Horgan at World Orphan Drug Congress 2018

Annie Hubert, Director, European Public Policy, Alliance for Regenerative Medicine

Pedro Huertas, Chief Medical Officer, Eloxx Pharmaceuticals

Adam Hutchings, Managing Director, Dolon

Edmund Jessop, Medical Adviser, National Health Service

Alex Johnson, Co-founder and joint CEO, Duchenne U.K.

Daria Julkowska, Scientific Coordinator, French National Research Agency

Dr Panos Kanavos, Associate Professor in International Health Policy, Department of Social Policy Deputy Director, LSE Health, London School of Economics and Political Science

Vikram Karnani, Chief Commercial Ofiicer, Horizon Pharma

M Ken Kengatharan, Managing Partner, Atheneos Ventures and CEO, Auxesia Orion

Alastair Kent Obe, Former Director, Genetic Alliance UK, Retired

Alastair Kent Obe at World Orphan Drug Congress 2018

Rabia Khan, Health Division, Directorate for Employment, Labour and Social Affairs, O.E.C.D.

Rabia Khan at World Orphan Drug Congress 2018

Diane Kleinermans, Advisor to the Ministry of Health and Social Affairs, Belgian Federal Government

Maryna Kolochavina, Project Director, Real World & Late Phase, Syneos Health

Nerissa Kreher, Chief Medical Officer, Avrobio

Dr Alexander Kuta, Senior Vice President Regulatory Affairs, Uniqure N.V.

Jennifer Lambe, Senior Project Manager, ICON plc

Dr Greg Larosa, SVP & CSO, Rare Disease Research Unit, Pfizer

Dr Greg Larosa at World Orphan Drug Congress 2018

Yann Le Cam, Chief Executive Officer, EURORDIS

Michael Linden, Former VP Gene Therapy & Head, GMI, Pfizer Ltd

Sune Lindgaard, Chief of Business Intelligence, Amgros, Denmark

Sune Lindgaard at World Orphan Drug Congress 2018

Alastair Mackinnon, CMO, Mereo BioPharma

Alastair Mackinnon at World Orphan Drug Congress 2018

Dr Will Maier, Chief Scientific Officer, ICON plc

Marc Martinell, CEO, Minoryx Therapeutics

Marc Martinell at World Orphan Drug Congress 2018

Henrique Martins, Chief Executive Officer, Servicos Partilhados Do Ministerio Da SaOde

Ana Mingorance, Chief Development Officer, Loulou Foundation

Eva Molero, Ceo, Synapse Research Management Partners

Nathalie Moll, Director General, EFPIA

Nathalie Moll at World Orphan Drug Congress 2018

Dr Marisol Montolio Del Olmo, Scientific Director, Duchenne Parent Project Spain

Karl-Johan Myren, Head of Patient Access, Wilson Therapeutics

Dr Alexander Natz, Secretary General, EUCOPE

Daniel O'Connor, Medical Assessor, Medicines and Healthcare Products Regulatory Agency

Daniel O'Connor at World Orphan Drug Congress 2018

Tuyen Ong, Chief Development Officer, Nightstar Therapeutics

Dr Emanuele Ostuni, Head of Europe, Cell and Gene Therapy, Novartis

Dr Emanuele Ostuni at World Orphan Drug Congress 2018

Dr Pan Pantziarka, Program Director, Anticancer Fund

Dr Pan Pantziarka at World Orphan Drug Congress 2018

Samantha Parker, Chief Patient Access Officer, Lysogene

Andrew Parker, Executive Vice President and Chief Science Officer, Zealand Pharma

Doug Paul, partner, Medical Marketing Economics

Doug Paul at World Orphan Drug Congress 2018

Tapani Piha, Head Of Unit, European Commission

Tapani Piha at World Orphan Drug Congress 2018

Sarah Pitluck, Head, Global Pricing & Reimbursement, Spark Therapeutics

Silvia Ragno, Senior Director External Asset Lead, Takeda

Dr Bernard Ravina, Chief Medical Officer, Voyager Therapeutics

Kirsty Reid, Senior Manager Science Policy and Animal Welfare, EFPIA

Frederic Revah, Chief Executive Officer, Genethon

Owen Roberts, Chief Executive Officer, M4K Pharma

Dr Paul Robinson, Executive Director, Patient Perspective, Merck Sharp and Dohme

Gregory Robinson, Chief Scientific Officer, Nightstar Therapeutics

Dr Jerome Rossert, Vice President, General Medicine, Global Clinical Development, Vertex Pharmaceuticals

Delphine Roulland, Senior Manager Public Affairs chez, EUCOPE

Delphine Roulland at World Orphan Drug Congress 2018

Jude Samulski, Director, Professor of Pharmacology, University of North Carolina at Chapel Hill, Gene Therapy Center

Jude Samulski at World Orphan Drug Congress 2018

Luca Sangiori, Head of Medical Genetics and Rare Orthopedic Diseases, Rizzoli Orthopaedic Institute

Scott Schliebner, Vice President, Scientific Affairs, Rare Diseases, PRA

Dr Yvonne Schmidt, Scientific Advisor, Pharmaceuticals Department, G-BA

Sophie Schmitz, President & Partner, Partners4Access

Ad Schuurman, Senior Advisor International Affairs, National Health Care Institute (ZIN), the Netherlands

Raghuram Selvaraju, Managing Director, Senior Analyst, Healthcare, Rodman & Renshaw

Daniela Settesoldi, Technical Scientific Committee and Pricing and Reimbursement Committee, AIFA

Daniela Settesoldi at World Orphan Drug Congress 2018

Nick Sireau, Chairman, AKU Society

Dr Kari Stefansson, Chief Executive Officer, Decode Genetics

Leo Strican, Head of Access Value & Evidence Strategy, Europe, Bluebird Bio

Leo Strican at World Orphan Drug Congress 2018

Rick Thompson, CEO, Findacure

Dr Josep Torrent-Farnell, Medicines Department, Catalan Health Service

Sheela Upadhyaya, Associate Director Highly Specialised Technologies, National Institute For Health and Care Excellence

Dr Giuliana Vallanti, Director Of Development, Quality Control And Qualified Person, Molmed SpA

Dr Sander Van Deventer, Chief Scientific Officer And General Manager, uniQure

Dr Andreas Vogel, Head of European Regulatory, Bluebird Bio

Till Voigtlander, Vice-President of the Board of Members States on ERNs, Medical University of Vienna

Till Voigtlander at World Orphan Drug Congress 2018

Dr Birgitte Volck, Senior Vice President, Head of R&D Rare Diseases, GlaxoSmithKline

Misha Votruba, Director, Gradus R.S.J. Life Sciences

Misha Votruba at World Orphan Drug Congress 2018

Darren Walsh, Senior Director - Market Access & Govt. Affairs – EU, Orchard Therapeutics

Darren Walsh at World Orphan Drug Congress 2018

Dr Mary Wang, Global Partnerships & Project Manager, Fondazione Telethon

Dr Richard Wenzel, Principal, Charles River Associates

Pawel Wozniak, Managing Director, Komtur Polska

Pawel Wozniak at World Orphan Drug Congress 2018

Entela Xoxi, Former AIFA, Universita Cattolica Del Sacro Cuore

Stefano Zancan, Head Clinical Development and Operations, Fondazione Telethon

Martine Zimmermann, Senior Vice President, Head of Global Regulatory Affairs, Alexion Pharmaceuticals





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