Orphan Drugs & Rare Diseases Global Congress 2015 Europe


Background Info

Orphan Drugs & Rare Diseases Global Congress 2015 Europe

Paradigm Global Events are proud to present the Orphan Drugs & Rare Diseases Global Congress 2015 Europe.

Over 7,000 different types of rare diseases and disorders exist, with more being discovered each day, and large pharmaceutical and biotech companies are structuring to participate and leverage the growing orphan market. “Orphan drug sales will make up 19% of the total share of prescription drug sales by 2020, totaling $176 billion. And they’ll grow at an annual rate of nearly 11% per year through the end of the decade, compared with about 4% for drugs treating larger populations.??? (EvaluatePharma’s Orphan Drug Report 2014)

Orphan Drugs & Rare Diseases Global Congress 2015 – Europe provides a unique platform for an intimate & interactive knowledge sharing and convergence of top tier government, pharmaceuticals, biopharmaceuticals, hospitals, non-profit organisations, orphan drugs developers as well as regional and local manufacturers to discuss the driving macroeconomic factors, policies and issues that will steer the development of orphan drugs globally.

Industry Facts

  • There are approximately 7,000 different types of rare diseases and disorders, with more being discovered each day
  • Approximately 50% of the people affected by rare diseases are children
  • 30% of children with rare disease will not live to see their 5th birthday
  • Rare diseases are responsible for 35% of deaths in the first year of life
  • Approximately 50% of rare diseases do not have a disease specific foundation supporting or researching their rare disease


Who Will You Meet

Presidents, Heads/Chiefs, Directors, VPs and Managers of:

  • Commercialisation
  • Regulatory Affairs
  • Market Access
  • Sales & Marketing
  • Research & Development


  • Orphan Drugs Manufacturers – 20%
  • Big Pharmas – 20%
  • Biopharmaceuticals – 20%
  • Governments (Ministries of Health/Drug Authorities) – 10%
  • Regional & Local Contract Manufacturers – 10%
  • Non Governmental Organisations (NGOs) – 5%
  • Hospitals & Patient Group Members – 5%
  • Others – 10%


The two-day Congress will provide an interactive and intimate discussion and networking format led by key industry speakers with intimate knowledge in the industry. Gain practical strategies and best practices on challenges, innovations, technologies and concepts surrounding orphan drugs and rare diseases.

  • Dr Ségolène Aymé, Emeritus Research Director, INSERM, FRANCE
  • David Boothe, Global Commercial Leader – GSK Rare Diseases, GSK, UK
  • Dr. Chia Wen Lee, Head of Emerging Markets – Corporate Market Access, BOEHRINGER INGELHEIM, GERMANY
  • Dr. David King, Global Regulatory Intelligence Director – Regulatory Policies & Intelligence, NOVO NORDISK, DENMARK
  • Barbara McLaughlan, Head of External and Acting Head of Market Access – Oncology, NOVARTIS, UK
  • Dr. Carlos R. Camozzi, Chief Medical Officer, ORPHAZYME, DENMARK
  • Dr. John ZL Gong, Chief Executive Officer, 3D MEDICINES, CHINA
  • Josie Godfrey, Associate Director – Highly Specialised Technologies, NATIONAL INSTITUTE FOR HEALTH AND CARE EXCELLENCE (NICE), UK
  • M. (Ken) Kengatharan, PhD. MBA., President & Chief Operating Officer, ARMETHEON, INC., USA
  • Dr. Hartwig Gajek, Managing Director, HESYRA-MEDICAL GMBH, SWITZERLAND
  • Dr Joanna Cox, Head of Business Partnerships, CRANFIELD UNIVERSITY, UK
  • Prof George Dickson, Chair of Molecular Cell Biology, School of Biological Sciences, ROYAL HOLLOWAY, UNIVERSITY OF LONDON, UK
  • Miriam Gargesi, EuropaBio Healthcare Director, EUROPABIO, BELGIUM
  • Prof Bobby Gaspar, Professor of Paediatrics & Immunology, Institute of Child Health – U.CL, UK
  • Christine Lavery, Chief Executive, MPS Society, UK
  • Didier Caizergues, Head of regulatory Affairs Department, GENETHON, FRANCE
  • Kieron Lewis, Business Development Manager, Clinigen Global Access Programs (GAP) – CLINIGEN
  • Dr. Nicolas Sireau, Chairman and CEO, AKU SOCIETY, UK
  • Timothy R. Cote, MD MPH, Principle / CEO, COTE ORPHAN
  • Prof. Joan – Lluis Vives Corrons, MD, PhD., Head of Project, ENERCA Chief, Red Cell Pathology Unit, Biological Diagnostic Centre, Hospital Clinic. UNIVERSITY OF BARCELONA
  • Donald Macarthur, Independent, Industry Consultant
  • Mark Scott, Director, t & e Consulting and Training


Gain The Latest Insights on:

  • Worldwide Development for Orphan Drugs: Growth and Sustainability for the Industry
  • Global Partnership: Governments, Orphan Drug Developers and CROs
  • Financing Orphan Drug Developments: What Are the Options Available?
  • Role of Academic Institutions
  • Case Studies: Orphan Drug Growth in Asia and Biotech Companies
  • Regulatory Matters: IP Strategy, Licensing and Integrity
  • Patient Access, Pricing and Reimbursements: Challenges and Strategies
  • Future of Orphan Drugs: What Is In Store?

Key Speakers

Prof Joan-Lluis Vives Corrons

Prof. Joan-Lluis Vives Corrons, MD, PhD., Head of Project, ENERCA Chief, Red Cell Pathology Unit, Biological Diagnostic Centre, Hospital University of Barcelona

Professor of Medicine and Head of the Haematology Laboratory Department of the Hospital Clinic i Provincial (University of Barcelona) with a high input in the development of new diagnostic procedures, standardization and quality assessment of Haematology practice and pioneering activities in the development of an External Quality Assessment Scheme (EQAS) for haematology in Spain. Educational and research activities have been carried out at the University of Barcelona and his scientific trajectory is guaranteed by over 400 publications in scientific journals, from which around 300 have a high impact factor (IF). Moreover, a total of 12 books of Haematology (5 as author or editor) have been. Financial support has been received from official sources (Catalonia, Spain and the European Comission) for the accomplishment of 32 different research projects, mainly focused on the study of physiopathology, molecular mechanisms and epidemiology of red blood cell (RBC) disorders due to hereditary enzymopathies, haemoglobinopathies, and membrane defects.
Head of the Red Cell Pathology Unit, a Reference Centre in Spain for the diagnosis and treatment of rare anaemias. Since 2002, Head of ENERCA (European Network for Rare and Congenital Anaemias), a Project funded by the European Commission (DG-SANCO) for the development of European Reference Network (ERN) in Rare Anaemias (anaemias with a prevalence lower than 5 per 10.000 individuals). This Project (2002-2016) is intended to contribute to the better knowledge of Rare Anaemias in Europe, its faster detection, diagnosis, treatment and epidemiological surveillance.
Research topics of main interest
1. Red blood cell pathology and disorders of erythropoiesis
2. Standardization and quality assurance in haematology.
3. Physiopathologic studies of congenital and acquired haemolytic anaemias
4. Haemoglobinopathies and thalassemias
5. Genetic and molecular aspects of erythrocyte enzyme deficiencies
6. Cellular and molecular markers in acute and chronic leukaemias
7. Automation in haematology laboratory.

Dr China Wen Lee

Dr. Chia Wen Lee, Head of Emerging Markets – Corporate Market Access, BOEHRINGER INGELHEIM, GERMANY

Dr. Chia Wen Lee is a trained epidemiologist and statistician; she has significant scientific experience across various delivery systems (clinical, pharmaceutical/ biotechnology, government, and academia) in Europe, Asia and the US.
Dr. Lee joint Boehringer Ingelheim in May 2014 as the Head of Emerging Markets Access, responsible for the entire prescription medicine portfolio.
She began her career in the Pfizer UK office and with technical competencies of evidence based medicine, statistical analysis, population and clinical epidemiology and Health Technology Assessments ( HTA); she was highly effective in providing technical insights into operational planning supporting ophthalmology, pain and CNS therapeutic areas. Being recognized as a talented leader with both academic kudos and business sense, Dr. Lee was assigned the task of setting up and leading the Market Access function (including HEOR&HTA) in China during the year of National Reimbursement Drug List (NRDL), Essential Drug List (EDL) review and the publication of highly anticipated Healthcare Reform Bill in 2009. After accomplishing her assignment securing optimal pricing and access of the portfolio and coaching and developing local talent, Dr. Lee was promoted to the HQs supporting HTA activities for both clinical development assets and in-line products. She was the Head of Emerging Markets Access in Biogen Idec responsible for creating the vision of Emerging Markets Access network, setting up the organization, strategies and details for implantation
Prior to the industry experience, Dr. Lee had extensive experience in Public Health and healthcare policy making and clinical guideline development including consulting to the Department of Health in Taiwan on maternal and child health, to leading a cross-functional team developing clinical guidelines for National Institute of Health and Clinical Excellence (NICE),
Dr. Chia Wen Lee was born and raised in Taiwan and received her PhD in Epidemiology and Biostatistics from London School of Hygiene and Tropical Medicine.

Dr David King

Dr. David King, Global Regulatory Intelligence Director – Regulatory Policies & Intelligence, NOVO NORDISK, DENMARK

David originally trained as a pharmacologist receiving an MSc in Neuropharmacology University of Toronto and a PhD in Cardiovascular Pharmacology from the University of Western Ontario in Canada. Since completing his PhD, David has worked in the pharmaceutical industry for the last 25 years both in Canada and globally based in Europe. During this period he has various roles including clinical research; pharmacovigilance; product development; in-licensing and regulatory affairs. He is currently working in a global regulatory intelligence role with a focus on the Asia Pacific region. David has worked for the last 14 years with the global development and registration of Orphan Medicinal Products. Prior to joining Novo Nordisk, David was the acting medical director working for Swedish Orphan International which specialised in the development of older compounds for rare diseases. He is currently an active member on the EBE/EuropaBio Joint Rare Diseases / Orphan Medicinal Products Task Force.

Mark Scott

Mark Scott, Director, t & e Consulting and Training

Mark Scott graduated with a degree in Law from the London School of Economics in 1993 but changed tack and stepped into healthcare 3 years later and trained as a physiotherapist at Coventry University. Mark practiced as a physiotherapist for 8 years and ran the OP department at Ealing Hospital as well as developing extended scope practice physiotherapy services for Ealing PCT. He was also the academy physiotherapist for London Welsh from 3 years. Following on from his experience developing new clinical models with Ealing Mark moved into management consultancy working with PCTs to review and develop services. He also worked as an interim commissioning manager for London Specialised Commissioning Group which was his fi rst exposure to the challenges of providing services for patients with rarer conditions. This experience later helped Mark secure an interim assignment with the National Specialised Commissioning Team which led to Mark becoming a permanent member of the team and spending 3 1/2 years with them. Whilst with the team Mark was the lead commissioner for contracting and the process for developing new service applications for consideration by AGNSS. Mark left the NHS in January 2013 to join a major pharmaceutical company in a policy role. Mark left this role after 15 months to take up another role in Pharma managing a fi eld team to gain more commercial experience as well as continuing with NHS policy work. Mark has seen healthcare from a wide range of viewpoints gathered from working in different professional roles, in different disease areas and with different types of different organisations.

Donald Macarthur
Donald Macarthur is an independent industry consultant and formerly a report writer on international pharmaceutical business issues. Specialities include pricing & reimbursement (all Europe, Japan/Korea/Taiwan), orphan drugs (Europe, Asia-Pacifi c), GP & hospital market access, funding of medicines, risk sharing, generics/biosimilars, wholesale and retail distribution, EU parallel trade/US importation, and homecare. Qualifying as a pharmacist, 16 years industry experience followed employment in hospital and community pharmacy. In 1996, Mr Macarthur founded the world’s fi rst periodical in its fi eld, Pharma Pricing Review. He has also written over 90 major reports including the best-selling series ‘Orphan Drugs in Europe: Pricing, Reimbursement, Funding & Market Access Issues’ and also ‘Orphan Drugs in Asia-Pacifi c: From Designation to Pricing, Funding & Market Access’.

Kieron Lewis

Kieron Lewis, Business Development Manager, Clinigen Global Access Programs (GAP), CLINIGEN

Kieron joined Clinigen GAP in 2014 and is responsible for sourcing new opportunities for the business, which specializes in the consultancy, development, set up and implementation of access programs on behalf of Biotech and Pharmaceutical industries. 
Prior to joining Clinigen, Kieron spent 15 years within the Pharmaceutical industry fulfilling a range of roles including Head of Cardiovascular Division at Servier Laboratories Ltd contributing to European and Global development plans. Within this time, Kieron has led a number of global pre-launch and launch activities for treatments addressing areas of high unmet medical need. Kieron now focusses his energies on working alongside Pharma and Biotech Companies to consider their strategy for Early Access, developing Global Programs and allowing patients to gain access to treatments that would otherwise be unavailable within their respective countries.

Barbara MCLaughlan

Barbara McLaughlan, Head of External Affairs and Acting Head of Market Access – Oncology, NOVARTIS, UK

Barbara McLaughlan is Head of External Affairs and Acting Head of Market Access at Novartis Oncology. In both roles Barbara’s focus is on shaping the external environment to ensure that all eligible patients in England, Scotland and Wales are able to access the innovative treatments for rare cancers and rare diseases developed by Novartis. Patients benefiting from the company’s commitment to rare diseases include those with Chronic Myeloid Leukaemia, myelofibrosis, pancreatic neuroendocrine tumours, sickle cell, thalassaemia and tuberous sclerosis complex. Before joining Novartis in 2011, Barbara worked as Eye Health Campaigns Manager at RNIB and previously in Government Affairs, education and communications roles in Germany, Belgium and the UK.

Hartwig GAJEK


Hartwig is Managing Director of Hesyra Medical GmbH in Binningen/Switzerland. For approx. 25 years, Hartwig held various positions in international clinical development and medical affairs, mainly in the biopharmaceutical industry. His scope includedclinical development phase I-IV in Europe, US and Asia, product launch and post-launch programmes, scientific communication, negotiations with regulatory authorities, health and industry policy, supporting market access, and developing defence stratgies for loss-of-exclusivity. Hartwig has developed a special interest in rare congenital diseases, and has experience in developing international registries, as well as working with advocacy groups. Before joining the pharmaceutical industry, Hartwig spent 10 years as a practising physician in anaesthesia and intensive care.

Dr Carlos R Camozzi

Dr. Carlos R. Camozzi, Chief Medical Officer, ORPHAZYME, DENMARK

Dr. Carlos Camozzi is the Chief Medical Officer of Orphazyme (Denmark). He has more than 25 years’ experience in the biopharmaceutical industry, most recently (10 years) within the orphan drugs development and paediatric therapeutic options, and successful achievements of products approvals by both the European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA). Prior to Orphazyme, Dr. Camozzi held roles of increasing responsibility at American Cyanamid (Lederle), Roche and Mepha Ltd. He was the Medical Director of Orphan Europe-Recordati and VP-Chief Medical Officer of uniQure BV (ex-AMT) for the development of gene-therapy (GT) for rare diseases, getting the first ever GT approval (Glybera) in western world.

David Boothe

David Boothe is the Global Commercial Lead for Inherited and Metabolic Disorders within the Rare Diseases Unit at GSK

He has commercial responsibility for a broad franchise that has spanned amyloidoses, lysosomal storage disorders (LSDs) and pulmonary arterial hypertension (PAH) from pre-clinical development through to maturity. David has a wealth of experience in the rare and orphan diseases field having joined GSK from Shire Human Genetics Therapies in Switzerland where he served as EMEA Product Director in Fabry Disease. Prior to Shire, David worked in international marketing roles for BioMarin and before that Oxford GlycoSciences, in MPS VI and Gaucher disease respectively.


Didier Caizergues, Head of Regulatory Affairs Department, GENETHON

A Doctor in Pharmacy, who holds a degree in Health law. He enjoyed a long career in the field of Regulatory Affairs.
He began his career as a consultant with International Drug Development (IDD), but soon joined the company SYNTHELABO in its Research and Development center where he worked for nine years. In this office Dr Didier CAIZERGUES oversaw the registration of new molecules that had evolved from cardiovascular, urological and anti-depressive research.
He later headed up the international registration department for the Pierre Fabre group and for the next six years he practices to the fields of anti-cancer drugs, anti-depressants and orphan diseases.
In 2001 he joined the GENETHON as Director of International Regulatory Affairs where he set up a regulatory affairs department specialized in gene and cell therapies in the fields of rare diseases. He obtained several clinical trials authorization in European countries and US for gene therapy products with lentivirus and AAV in the fields of muscular dystrophies and paediatric immunodeficiencies.
He is also in charge of the assessment of new molecules or molecules already marketed still in the field of rare diseases in different working groups in European projects (Treat-NMD).

Dr John ZL Gong

Dr. John ZL Gong, Chief Executive Officer, 3D MEDICINES, CHINA

Prior to 3DMed, Dr. Gong served as VP of Drug Development and Regulatory Affairs of Beigene, CEO of BL Pharma, and CTO of JOINN Laboratories. From 1998 to 2008, Dr. Gong worked at US FDA as Reviewer, he conducted primary and critical reviews of numerous IND and NDA applications for both small molecules and biologics. Dr. Gong is member of the Drug Safety Evaluation Committee of the Chinese Pharmaceutical Association, Council member of AAALAC, and member of editorial board of Chinese Journal of New Drugs. Dr. Gong received his Ph.D. in Toxicology from New York University in 1996 and a medical degree from Beijing Medical College in 1984.

M (Ken) Kengatharan

M. (Ken) Kengatharan, PhD. MBA., President & Chief Operating Officer, ARMETHEON, INC., USA

Dr. M. (Ken) Kengatharan is a co-founder and President & COO of Armetheon, Inc. a co-founder and former President & CSO of Altheos, Inc., and a co-founder and former Vice President, Pre-Clinical R&D of Athenagen, Inc. (re-named CoMentis, Inc), all three biopharmaceutical companies are based in San Francisco bay area. He is also a founder and General Partner at Atheneos Capital, a San Francisco bay area based evergreen healthcare incubator fund.
During the last 14 years, Dr. Kengatharan has been critical to the development of multiple drug candidates (NCEs and re-purposed drugs) from concept to Phase II. Since setting-up his first company in the late 1990s, Dr. Kengatharan has been critical to raising more than $130 MM in private equity for five start-up companies.
In 2004, Dr. Kengatharan co-founded Athenagen, Inc. (re-named CoMentis, Inc.) which in 2008 completed licensing and drug development collaboration deal with Astellas Pharmaceutical worth $760+ MM with an upfront payment of $100MM. He joined the company as its Vice-President of Pre-Clinical Research and Development and then became Vice-President of Translational Research and Scientific Affairs. His responsibilities included overseeing discovery research and pre-clinical drug development in cognition enhancement, inflammation, and angiogenesis, in addition to managing pre-clinical R&D operations in South San Francisco. Prior to joining CoMentis, he was a staff scientist in the Division of Cardiovascular Medicine at Stanford University, where he continued his affiliation as a visiting scientist until June 2013. Dr. Kengatharan has also served as Director of Corporate Development, and later, Vice President of Pre-Clinical R&D at OxoN Medica Inc., where he spearheaded pre-clinical drug development as well as R&D operations.
He is currently a member of the board of directors at Armetheon, Inc., and previously a board observer at CoMentis, Inc., and Altheos, Inc. He has also served on the boards of EPi3, Athenagen, Inc., and Altheos, Inc., and as a member of the advisory board of Cardinal Free Clinics at Stanford University School of Medicine. In addition, he has been an advisor to Stanford’s SPARK program, a technology translation project for biotech ideas, since it was founded in 2006.
Dr. Kengatharan is an author of several research articles and reviews in the vascular biology, inflammation, and ocular pharmacology areas, and has been invited to present at local and international scientific meetings covering topics in angiogenesis, inflammation, ocular drug delivery and biotech financing and start-ups.
Dr. Kengatharan obtained his PhD in pharmacology from the University of London at the William Harvey Research Institute with Nobel laureate Sir John Vane and his MBA (with Distinction) from Durham University in England, where he focused on Biotech Finance and Entrepreneurship. Thereafter, he held a post-doctoral position at the same institute as a recipient of a British Heart Foundation Fellowship.

Prof George Dickson

Prof George Dickson, Chair of Molecular Cell Biology, School of Biological Sciences, Royal Holloway, University OF LONDON, UK

George Dickson is Professor of Molecular Cell Biology at Royal Holloway – University of London (RHUL). He has spent most of his career studying neuromuscular disease and muscle cell biology, including the first cloning of an intact dystrophin gene, the discovery of the role of cell adhesion molecules in muscle stem cell fusion, the first identification of utrophin, and the first description of exon skipping in Duchenne muscular dystrophy (DMD). Professor Dickson has also conducted notable research into gene therapy for atherosclerosis, and genetic vaccination against HIV/AIDS. He is a member of the UK MDEX Consortium, UK HIV-Vaccine Consortium, and the EU-SKIP-NMD Project, and has been a platform leader in the EU Clinigene Network of Excellence. He is a past President of the European Society of Gene & Cell Therapy, and a past Secretary and founder member of the British Society for Gene Therapy. He has been a member of the European Medicine Agency Committee for Advanced Therapeutics (Gene and Cell Therapies). Professor Dickson is also a Royal Holloway Research Theme Champion for ‘Health, the Human Body and Behaviour’.

Josie Godfrey

Josie Godfrey, Associate Director – Highly Specialised Technologies, National Institute for Health and Care Excellence (NICE), UK

Josie Godfrey is Associate Director at NICE with responsibility for the new Highly Specialised Technologies programme. She is leading work to establish programme which will make recommendation to th NHS in England about the use of new highly specialised technologies.

Before joining NICE, Josie led work to establish the new Advisory Group for National Specialised Services (AGNSS) She developed a decision-making framework to support AGNSS in making these recommendations and developed the process for considering highly specialised services, drugs and technologies.

Josie has worked in health policy development and implementation for the NHS in England and national and local government.

Miriam Gargesi

Miriam Gargesi, EuropaBio Healthcare Director, EUROPABIO, Belgium

Miriam Gargesi is currently Director for Healthcare Biotechnology at EuropaBio, the European Association for Bioindustries. In this role, she leads the advocacy and public affairs of EuropaBio’s healthcare department, in close coordination with the association’s Healthcare Council and Working Groups, and maintains a strong network of contacts with policymakers and other stakeholders. Mrs Gargesi has longstanding experience in the field of European healthcare policies, spanning across major pillars of the sector such as biotechnologies, pharmaceuticals and diagnostics alike. Prior to joining EuropaBio, she served for several years as Director for Public Affairs and Communications at EDMA, the European Diagnostic Manufacturers Association, and she worked for the healthcare practice of a frontrunner international consultancy. She also gained extensive insights into the work of political institutions through her work at the House of Commons in London and the Italian Chamber of Deputies in Rome. Mrs Gargesi holds an MSc in European Political Economy from the London School of Economics and Political Science (LSE), as well as a BSc in International Relations from the University of Wales, Aberystwyth, and McGill University. An Italian national, she is fluent in English and French, and has a working level of Spanish.

Christian Girard


Christian Girard, MiM, is currently the CEO of ABCrowdFunding Advisors, a global consulting firm with a focus on equity crowdfunding and orphan drugs companies. Concurrently, he is the founder and CEO of Orphan Drugs Industry Insider, a competitive intelligence information provider in the rare diseases area. His entry in life sciences dates back in 1994, when he joined the newly formed French subsidiary of Deknatel, a Pfizer’s medtech spin-off, which was later acquired by Genzyme. He is also a co-founder of gmp-orphan, a biotech repurposing small molecules in orphan indications, and an advisor to research institutions and start-up companies.

CHristine Lavery

Christine Lavery, Chief Executive,
Society for Mucopolysaccharide Diseases, MPS SOCIETY, UK

Christine Lavery was appointed Chief Executive of the Society for Mucopolysaccharide and Related Diseases (MPS) in 1993. During her time at MPS she has taken the charity to new heights, managing a substantial research budget and a unique UK-wide advocacy service providing needs led support to nearly 1300 children and adult sufferers, their families and professionals in areas of home adaptations, special educational needs, access to new therapies, respite care, palliative care and pre- and post-bereavement support. Christine has served on the Department of health Advisory Board on Genetic Testing and is currently a patient representative on the NHS England LSD Specialised Commissioning Advisory Group.

Christine was awarded the Member of the British Empire for her services to Metabolic diseases by the Queen in the New Year’s Honours List for 2002 and at the 2006 International Symposium on Mucopolysaccharide and related diseases received ‘A Life Time Award’ from the International MPS Community. Christine continues to promote the needs of MPS, Fabry and related Lysosomal Storage Diseases across nations and working closely with the MPS International Network.

Dr Joanna Cox

Dr. Joanna Cox, Head of Business Partnerships, CRANFIELD UNIVERSITY, UK

Dr. Joanna Cox is the Research and Business Development Manager for Life Sciences at Royal Holloway, University of London. She has divided her career between academia and industry, previously working in both academic research at the University of Oxford and in early stage biotechnology companies. She has co-founded two companies, both of which continue to be successful. She is currently responsible for research and innovation development in the life sciences at Royal Holloway, University of London. She has formed numerous successful collaborations with industrial partners from multinationals through to mid-cap and SME companies. In technology development she has taken ideas through from early stage proof-of-concept to out-licensing and clinical trials. Her work has often focussed on utilising public/private sector funding initiatives and she has been instrumental in securing over £12million in development funds. In the orphan drugs area her work has focussed on the development of gene therapy technology, with 5 patents currently out-licensed to industry partners and in clinical development.

Dr Segolene Ayme

Dr Ségolène Aymé, Emeritus Research Director, INSERM, FRANCE

Ségolène Aymé is Emeritus Director of Research at the French Institute of Health and Medical Research (INSERM). She was the founder of Orphanet in 1997 and its Executive Manager up to 2011. She chaired the EU Committeee of Experts on rare Diseases (EUCERD 2010-2013) and is now a member of the Commission Expert Group on Rare diseases. She also chairs the WHO Topic Advisory Group for Rare Diseases and serves as Editor-in-Chief of the Orphanet Journal of Rare Diseases (www.ojrd.com). She is the project leader of “Support IRDiRC???, which provides the services of a scientific secretariat to the International Rare Diseases Research Consortium (www.irdirc.org).

Timothy R. Coté, MD MPH, Principle / CEO, COTE ORPHAN

Dr. Coté is the leading national regulatory expert in orphan drug development. With 22 years of Federal service at the FDA, NIH, and CDC, Dr. Coté most recently served as the Director of the FDA Office of Orphan Products Development (OODP) from Sept 2007 – May 2011. Dr. Coté was instrumental in implementing the Orphan Drug Act and personally signed over 800 orphan drug designations in his time at the Agency. From 2002 to 2004 he served as Branch Chief, Therapeutics and Blood Safety division in the CBER Office of Biostatistics and Epidemiology. In 2011 Dr. Coté also served as the Chief Medical Officer for the National Organization for Rare Disorders (NORD) and the Professor of Regulatory Practice at the Keck Graduate Institute in Claremont, California. Currently Dr. Coté serves as the Principle/CEO of Coté Orphan, a regulatory advisory firm in Silver Spring, MD.

Prof Bobby Gaspar

Prof Bobby Gaspar, Professor of Paediatrics and Immunology, Institute of Child Health – U.C.L., UK, Honorary Consultant in Paediatric Immunology, Great Ormond Street Hospital

Bobby Gaspar is a physician/scientist working in paediatric immunology at the Institute of Child Health (ICH) and Great Ormond Street London. He initially trained in paediatrics and then became interested in primary immunodeficiencies at an early stage in his career and undertook a PhD at the Molecular Immunology Unit at ICH. From there he has continued his academic and clinical career. His interests are in many different aspects of primary immunodeficiency including understanding the molecular and cellular defects and disease pathogenesis, bone marrow transplantation for severe immunodeficiencies and the development of gene and cell therapy for these conditions. Over the last decade, his team have conducted clinical trials that have shown that gene therapy can successfully correct the immune defect in specific immunodeficiency conditions. He is leading UK and European initiatives for newborn screening for severe combined immunodeficiency. He is also Director of the Centre for Research in Rare Diseases in Children (CRRDC) a new academic and clinical facility that will open at Great Ormond Street in 2018.

Chairman, Clinical Working Party of ESID 2004 – 2008
Chairman, ESID BMT and Gene therapy Working party 2008 – 2013
Chairman EBMT Inborn Errors Working party 2008 – 2013

Member, International Committee, American Society of Gene Therapy, 2008 – present day
Chair, IUIS PID – Expert committee 2013 – present day
Chair, International Committee, American Society of Gene Therapy, 2013 – present day
Deputy Editor – Molecular therapy 2013 onwards

Chairman, Medical Advisory Panel – International Patient Organisation for Primary Immunodeficiencies, 2013 onwards
Member, Medical Advisory Panel – PID-UK, 2013 onwards
Member, Medical Advisory Panel – Ataxia Telangiectasia Society, 2013 onwards

Dr Nicolas Sireau

Dr. Nicolas Sireau, Chairman & CEO, AKU Society

Dr Nicolas Sireau is Chairman and CEO of the AKU Society, a medical charity that works to find a cure for and support patients with AKU (also called Black Bone Disease), an ultra rare disease that affects his two sons (www.akusociety.org).

The AKU Society is a fast-growing patient movement, with formal patient groups in the UK, France, Italy, Germany, the Netherlands, Jordan, Belgium, Slovakia, North America and India. It is spearheading an international consortium of biotechs, pharma companies, universities, hospitals and clinical trial centres across Europe, the Middle East, Asia and North America. It works closely with the hospital and university in Liverpool.

Dr Sireau is Co-founder and Chairman of Findacure (www.findacure.org.uk), a new social enterprise that raises awareness into and helps patients with fundamental diseases: extreme and exceptional diseases that advance our understanding of medicine and help us discover potential new treatments (www.findacure.org.uk).

He is a committee member of Rare Disease UK, the national alliance of rare disease groups in the UK, and of the International Rare Disease Research Consortium (IRDiRC). He is also is a founding member of the Cambridge Rare Disease Network and a trustee of DNAdigest, a social enterprise that works on collaborative data cures for genetic diseases.

He is a fellow of the Ashoka fellowship of social entrepreneurs and of the Royal Society of Arts. He is the editor of ‘Rare Diseases: Challenges and Opportunities for Social Entrepreneurs’ (Greenleaf, 2013) and co-editor of ‘The Rare Diseases Patient Group Handbook’ (forthcoming). He is co-founder of the Vincent Foundation for brain injury and a former non-executive Director of GenSeq, a bioinformatics company.

Dr Sireau’s previous career was in international development, where he was CEO of SolarAid and Sunny Money, two award-winning non-profit social enterprises that he set up. Sunny Money is now the largest distributor of solar lanterns in Africa. He has also written several books on international aid.

Dr Sireau has a PhD in Social Psychology. His hobbies include writing novels and producing electronic music.


Day 1

08:00 – 09:00Registration & Coffee

08:45- 09:00 Chairperson’s Opening Remarks
Donald Macarthur, Independent Industry Consultant


09:00 – 09-40Incentives for the Development and Commercialisation of Orphan Drugs in APAC Countries and their Results

  • Japan
  • Korea
  • Taiwan
  • Australia
  • Singapore

Donald Macarthur, Independent, Industry Consultant

Panel Presentation

09:40 – 10:20 Financing Drug Discovery for Orphan Diseases: What Are the Options Available?

  • How can megafunding address the lack of in traditional sources of financing for orphan drugs?
  • How are orphan drugs suited for portfolio financing?
  • Why orphan drug companies are good investments and what risks are associated with investing in orphan drugs?
  • What role do mergers and acquisitions play in the orphan disease investment story?


  • Donald MacArthur, Independent Industry Consultant


  • Miriam Gargesi, EuropaBio Healthcare Director, EUROPABIO, BELGIUM


10:20 – 11:00 Orphan Medicines: our 2020 Vision – A call to Europe

  • What impact did the EU Orphan Medicines Regulation have on pharmaceutical innovation over the last 15 years?
  • What does the orphan drug sector looks like today? Current challenges and future opportunities
  • Going forward: meeting patient’s unmet needs

Marriam Gargesi, Healthcare Director, EuropaBio, Belgium

11:00 – 11:40Morning Break & Networking

Shared Platform

11:40 – 12:20Academic Platform: From the Perspectives of Academics

  • The Role of Academic Institutions in Contributing to the Development of Orphan Drugs

Dr Joanna Cox, Research and Business Development Manager, ROYAL HOLLOWAY, UNIVERSITY OF LONDON, UK

  • Case Study on the Development of a Gene Therapy ATMP Orphan Drug for Duchenne Muscular Dystrophy

Prof George Dickson, Chair of Molecular Cell Biology, School of Biological Sciences, ROYAL HOLLOWAY, UNIVERSITY OF LONDON, UK


12:20 – 13:00The Emerging Markets Perspective in Orphan Drug Development, Market Access And Pricing

All eyes on Asia: Japan and China:

  • Considerations for clinical development in trial design and launch planning
  • Construct relevant value stories for Japan and China : neighbouring countries with very different approach
  • Pricing and access in Japan and China

Orphan Drug Designation in key markets ( including China, Japan, Mexico, Turkey, Argetina, S Korea, Taiwan Brazil and Russia)

  • What is the implication on global launch sequencing?

Dr. Chia Wen Lee, Head of Emerging Markets, Corporate Market Access, BOEHRINGER INGELHEIM, GERMANY

13:00 – 14:00Networking Lunch



14:00 – 14:40GSK’s Adaptive and Flexible Approach to Rare Diseases

  • How GSK continues to adapt to the external environment to ensure patients / HCPs / carers are at the centre of everything we do
  • How GSK’s operating model aims to bring benefit to patients with Rare Diseases
  • How GSK has applied adaptive licensing to help shorten the period to approval and broadening access to medicines: a case study

David Boothe, Global Commercial Leader – GSK Rare Diseases, GSK, UK


14:40 – 15:20 Integrity of Orphan Drugs Legislations: What Benchmark for Globalised Standards Can Regulators Implement Globally and What Can Be Done to Ensure the Integrity of These Standards?

  • How can orphan drug manufacturers help support governments’ initiatives to drive the region’s industry over the next 10 years?
  • What approaches can pharmaceutical companies use to get a new product through development and approval?

Dr. David King, Global Regulatory Intelligence Director – Regulatory Policies & Intelligence, Novo Nordisk, DENMARK


15:20 – 16:00Early access to Orphan Drugs

  • Current environment
  • Regulatory requirements
  • Practical Considerations

Kieron Lewis, Business Development Manager, Clinigen Global Access Programs (GAP) – Clinigen Group

16:00 – 16:40Afternoon Refreshments Break & Networking


16:40 – 17:20 Ultra Orphan Drugs: What is the Long Term Strategy to support Ultra Orphan Drugs and How Commercially Viable Are These Drugs to Stakeholders?

  • With orphan drugs pricing being questioned, how can companies develop ultra orphan drugs for patients with ultra rare diseases and what can governments do to support theses companies?
  • What are investors take on value for money for pharmaceutical companies seeking to boost their top line sales via M&A in ultra orphan drugs and should regional manufacturers look at developing ultra drugs?
  • Will the classification of some drugs as “ultra -orphan” and others just “orphan” artificially divide services and access to life – saving drugs?



17:20 – 18:00Risks & Rewards of Orphan Drugs Development

M. (Ken) Kengatharan, PhD. MBA., President & Chief Operating Officer, ARMETHEON, INC., USA


Donald Macarthur, Independent, Industry Consultant

18:30 19:00 – Networking Drink Reception

19:00 – 21:00 – Networking Gala Dinner

Day 2

08:00 – 08:45Registration & Coffee

08:45 – 09:00Chairperson’s Opening Remarks


09:00 – 09:40Rising Patient Costs: How Are Payors and Drug Companies Balancing the High Costs of Orphan Drugs?

  • How can manufacturers establish the value of orphan drugs therapies worldwide?
  • What are the criteria that should be used to access the value of a newly approved orphan drug?

Barbara McLaughlan, Head of External Affairs – Oncology, Novartis, UK


09:40 – 10:20The Ideal Partner: What Are the Criteria that Partners for Orphan Drugs Developers Look?

  • How can orphan drug companies partner with governments and what are some of the steps to follow in seeking partnership?
  • What are orphan drug developers looking for when partnering with a CRO and how CROs can meet the needs for external expertise to overcome development hurdles?
  • How to pick the right team and focus on retaining key study personnel for the length of the trial?

Dr. John ZL Gong, Chief Executive Officer, 3D MEDICINES, CHINA


10:20 – 11:00Building Long – Term Sustainability of Clinical Research Networks and Registries

  • How can you prepare healthcare providers to know about the therapy and identify the right patients for your therapy?
  • How can developers make research goals and use of data to be transparent and to be informed about progress or roadblocks in order to build a relationship based on trust with patients?
  • What enhanced ongoing patient support beyond the clinic can be provided to encourage positive relationships between patients and healthcare providers?

Dr. Hartwig Gajek, Managing Director, HESYRA – MEDICAL GMBH, Switzerland

11:00 – 11:40Networking Morning Refreshment Break – Exhibitors Hall


11:40 – 12:20Cross-Border Health Care Services for Patients with Rare Diseases

  • How does reimbursement work under the Directive 2011/24/EU for EU member states?
  • How does the directive allow health professionals assist in the diagnosis of rare diseases and possibly accelerate the treatment for patients?

Dr Ségolène Aymé, Emeritus Research Director, INSERM, FRANCE


12:20 – 13:00Paediatric Drugs and Rare Diseases: How Are Children with Rare Diseases Treated and How Are Their Data Protected?

  • What influences does the European paediatric regulation have on marketing authorisation of orphan drugs?
  • What can be done by healthcare providers to be proactive in monitoring side effects that can put patient safety at risk and effectively communicate results to the developer?
  • How should patient’s information about their treatment experience be collected and how can this steer the evolution of orphan drugs for children with rare diseases?

Christine Lavary, Chief Executive, MPS Society , UK

13:00 – 14:00 – Networking Lunch Break


14:00 – 14:40 – Case Study: The Role Played by the Patients Associations in the Field Of The Rare Diseases in the Development of New Medicinal Products

  • The “Association Francaise centre les Myopathies” a patient group supporting R&D for Rare Disorders from Research to patient access.
  • An expert group the TACT, supported by several patients and associations that created the European program Treat – NMD, in charge to assess several developments of orphan medicinal products?

Dr. Didier Caizergues, Head of regulatory Affairs Department, Genethon, France


14:40 – 15:20 – How to Succeed in Orphan Drug Regulatory Affairs

  • History and the basics of the Orphan Drug Act (ODA)
  • Has the ODA been successful?
  • Top 5 reasons orphan designation application fail
  • Does the FDA cut orphan drugs slack?
  • Why are orphan special?

Timothy R. Cote, MD MPH, Principle / CEO, COTE ORPHAN

15:20 – 16:00 – Afternoon Networking Break


16:00 – 16:40Strategic Corporate Social Responsibility (CSR) and Orphan Drugs Development: Why Is It Important to Integrate CSR Into the Development of Orphan Drugs and How Can This Reinforce the Orphan Market for a Sustainable Future?

  • How can orphan drug companies take a strategic approach to social responsibility by integrating orphan drug development and social responsibility practices with core business activities to obtain both economic and non-economic benefits for the firm and its stakeholders?
  • In what way can companies derive more strategic social responsibility benefits from developing orphan drugs?
  • How can the potential of new technological developments alleviate social responsibility in the development of orphan drugs?

Dr. Carlos R. Camozzi, Chief Medical Officer, Orphazyme, Denmark

17:20 -17:20 – Case Study: How Can ENERCA’s e- Health Tools Ensure the Same Level of Access to Rare Anaemias Services Across Europe?

Prof. Joan Liuis Vives Corrons, Head of Project, ENERCA Chief, Red Cell Pathology Unit, Biological Diagnostic Centre, Hospital Clinic, University of Barcelona, Spain

Panel Discussion

17:10 – 17:50 – What Challenges Still Remain and How Can the Orphan Drugs Marketplace Be Improved?


  • Josie Godfrey, Associate Director, Highly Specialised Technologies, National Institute for Health and Care Excellence (NICE), UK
  • Dr. Carlos R. Camozzi, Chief Medical Officer, Orphazyme, Denmark
  • Prof Bobby Gasper, Professor of Paediatrics & Immunology, Institute OF Child Health – U.C.L., UK

18:00Chairperson’s Closing Remark & End of Conference

Donal Macarthur, Independent Industry Consultant

Post - Conference Workshops

Wednesday 1st July 2015

08:30 – Registration and Coffee


  • How do we fund an orphan drug candidate in the Death Valley?
  • Can the crowd fund the rare?
  • Why and how did patients associations contribute to the funding of orphan drugs companies (the Kalydeco case)?
  • How do patients associations contribute to the funding of orphan drugs companies, today?
  • Can patients, families and relatives directly contribute to the funding of orphan drugs companies, and what are the pros and cons?
  • How to reconcile donation and ownership?

Led By:

12:30 – 17:00 Lunch Break


  • How can manufacturers build strong relationships with patients?
  • How can we bridge power and knowledge with patients to bring them as partners into the drug development process which continue to remain complicated for rare diseases?
  • How can secured patient registries infrastructure provide assured confidentiality and privacy protections for patients contributing their information?
  • How can patient advocacy groups influence regulation and corporate strategy?
  • How can developers make transparent the research goals and use of data to patient and continue to gain patients’ trust?

Led By:
Dr Nicolas Sireau, Chairman and CEO, AKU Society
Mark Scott, Director, t & e Consulting and Training




About Clinigen Group (Clinigen GAP)

The Clinigen Group is a specialty global pharmaceutical company headquartered in the UK, with offices in the US and Japan. The Group, dedicated to delivering ‘the right drug, to the right patient at the right time’, has three operating businesses; Specialty Pharmaceuticals (Clinigen SP), Clinical Trials Supply (Clinigen CTS), and Global Access Programs (Clinigen GAP). Clinigen GAP develops and implements global access programs for biotechnology and pharmaceutical companies and has provided access to unlicenced, licenced and end-of-lifecycle products to thousands of patients. Clinigen has experience in the implementation of more than 30 access programs worldwide.

For more information, please visit www.clinigengroup.com



Coté Orphan, LLC Coté Orphan, LLC

About Coté Orphan, LLC Coté Orphan, LLC, a regulatory affairs advisory firm based in Silver Spring, MD, provides valuable strategic planning and execution services to companies developing or seeking to develop orphan drugs. Under the leadership of Dr. Timothy R. Coté, an orphanology expert and former Food and Drug Administration (FDA) Director for the Office of Orphan Products Development (OOPD), Coté Orphan demystifies the FDA regulatory process. Coté Orphan knows the FDA hurdles unique to orphan products and forges successful pathways to orphan product designation. For more information on Coté Orphan, please call 202.957.9599 or visit www.coteorphan.com



Caligor Rx

Caligor Rx partners with pharmaceutical and biotechnology companies worldwide to develop, implement, and manage ethical and compliant Global Access Programs. These programs, often referred to as Compassionate Use or Expanded Access, provide physicians and patients with access to unlicensed medicines where there is critical need and the medicines are currently not available in their home countries. Our leadership team leverages deep experience in the rare disease space to help companies “bridge the gap??? between medicines with limited availability and patients in need on a global scale.
Partnering with Caligor Rx to manage a Global Access Program for rare diseases allows sponsors to have an ethical and compliant provision for demand while relieving them of the significant resources required to handle these complex and unpredictable requests so that they can remain focused on clinical development which ultimately brings innovative and life-saving medicines to market.


Healthcare’s dynamics demand innovative, strategic partnerships.

ASD Healthcare’s innovative products and exceptional customer service make us the largest distributor of oncology and supportive care products to healthcare facilities and specialty pharmacies. Our knowledge, reach and partnerships position us as the number one distributor of blood derivatives, nephrology products and critical specialty pharmaceutical products in the U.S. We maintain solid relationships with all major specialty pharmaceutical manufacturers, and are the trusted partner of all major GPOs. That’s why 95 percent of U.S. hospitals are our active customers, and why other healthcare providers depend on us to deliver innovative products and services.


Media Partners

PharmaVOICE magazine provides commentary about the challenges and trends impacting the life-sciences industry, covering a range of issues from molecule through market. PharmaVOICE’s more than 25,000 BPA-qualified subscribers are also kept abreast of the latest trends through additional media resources, including WebSeminars, Podcasts, Videocasts, and White Papers. For further information please visit: www.pharmavoice.com

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SelectScience.net is the leading online product and application publication for laboratory scientists featuring the latest news, application articles, videos, product directory and product reviews. Membership is fast, free and provides access to all of the SelectScience services. SelectScience.tv is a unique video news channel for laboratory application and product information. For further information please visit: www.selectscience.net

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BIOTECHNOLOGY EUROPE is owned by BIOTECHNOLOGY WORLD. It is based and located in Warsaw, Poland. Biotechnology World was founded in 2007 to provide the world’s biotech and pharma information and market to make it universally accessible and useful for scientific and business processes. Its first step to fulfilling that mission was building the BIOTECHNOLOGY EUROPE platform that will allow a quick spread of information in different channels. BIOTECHNOLOGY EUROPE offers companies completed internet public relations, publication and marketing solutions. One of the mains goals of BIOTECHNOLOGY EUROPE is to integrate the Biotech and Pharma Sector in Europe to global biotechnology, pharmaceutical and life science activities. For further information please visit www.biotechnology-europe.com

Future Pharmaceuticals has forged powerful relationships with key industry leaders to provide a platform for successful brand recognition, and for senior decision- makers to have the means to procure and plan implementation strategies based on the topics covered. Positioned to be an authoritative resource within top pharma companies as well as small, specialty, and biotech, Future Pharmaceuticals magazine is geared to create a deep penetration into a highly targeted and responsive audience, bridging the gap between the industries’ top issues and the solutions top-tier vendors can provide. For further information please visit: www.futurepharmaus.com