ODRD 2016

SPONSORS AND EXHIBITORS
• Dr. Virginia Acha, Executive Director Research, Medical and Innovation, ASSOCIATION OF THE BRITISH PHARMACEUTICAL INDUSTRY (ABPI), UK
• Malcolm Allison, Head of Pulmonology & Anti-Infectives, BAYER HEALTHCARE PHARMACEUTICALS, SWITZERLAND
• Patricia Anderson, Vice President, Regulatory Services, MAPI GROUP
• Martin Andrews, SVP – Rare Diseases, GSK, UK
• Benoit Arnould, Senior Director, Patient-Centered Outcomes, MAPI GROUP
• Dr. Ségolène Aymé, Emeritus Research Director, INSERM, FRANCE
• Adam Barak, Managing Director, PPi HEALTHCARE CONSULTING LTD
• Gulce Belgin, Founder & Director, PROCEUTICA, Turkey
• Dr. Carlos R. Camozzi, Chief Medical Offi cer, ORPHAZYME, DENMARK
• Dr. Steve Bradshaw, Managing Director, VALID INSIGHTS
• Simon Denegri, National Director for Patients and the Public in Research, NATIONAL INSTITUTE FOR HEALTH RESEARCH (NIHR) and Chair, INVOLVE, UK
• Kelly Franchetti, Executive Director, Global Patients Insights and Engagement, MAPI GROUP
• Miriam Gargesi, EuropaBio Healthcare Director, EUROPABIO, BELGIUM
• Dr. Alberto Gimona, VP - Head of Global Clinical Science and Epidemiology, ACTELION PHARMACEUTICALS, SWITZERLAND
• Jonathan Higginson, Director – Global Market Access, SHIRE PHARMACEUTICALS, SWITZERLAND
• Dr. Edmund Jessop, Public Health Adviser, NHS ENGLAND, UK
• Dr. Sukhwinder S. Jossan, Associate VP - Global Regulatory Affairs, FERRING PHARMACEUTICALS, DENMARK
• Will Maier, Chief Scientifi c Offi cer, MAPI GROUP
• Dr. M. (Ken) Kengatharan, President, ARMETHEON, INC., USA
• Dr. Sjef de Kimpe, VP – Early Drug Development, BIOMARIN, NETHERLANDS
• Dr. Maria del Mar Mañú, Project Manager, EUROPEAN NETWORK FOR RARE AND CONGENITAL ANAEMIAS (ENERCA), SPAIN
• Camille Métais, Associate Director, Regulatory Affairs, ALEXION PHARMACEUTICALS, SWITZERLAND
• Kay Parkinson, Chief Executive, CAMBRIDGE RARE DISEASE NETWORK (CRDN) and Founder, ALSTRÖM SYNDROME UK
• Richard Porter, Global Head of Operations Management for Neuroscience, Ophthalmology and Rare Diseases (NORD), ROCHE, SWITZERLAND
• Flóra Raffai, Executive Director, FINDACURE, UK
• Michael Rooney, Senior Business Support Manager, ALMAC GROUP
• Celine Taveau, Project Director - Head of Drug Department MAPI GROUP
• Oliver Timmis, Head of Projects, ALKAPTONURIA SOCIETY (AKU), UK
• Dr. Rick Thompson, Scientifi c Offi cer, FINDACURE, UK
• Dr. Hideo Tonomura, Director, OHARA PHARMACEUTICAL, JAPAN
• Dr. Barbara Valenta-Singer, VP – Global Clinical Development, BAXALTA, AUSTRIA
• Frans van Andel, Access of Health Technologies, Board Director, PPi HEALTHCARE CONSULTING LTD
Driving the Commercialisation and Sustainability of Orphan Drugs to Meet Unmet Needs
14 MARCH 2016: PRE-CONFERENCE WORKSHOP
15 – 16 MARCH 2016: CONFERENCE
Hilton London Olympia Hotel, London UK
TO BOOK CALL: +44 (0) 207 193 3485 • Email: info@paradigmglobalevents.com • www.paradigmglobalevents.com

ORPHAN DRUGS & RARE DISEASES GLOBAL CONGRESS 2016 EUROPE
14 MARCH 2016: PRE-CONFERENCE WORKSHOP • 15 – 16 MARCH 2016: CONFERENCE • Hilton London Olympia Hotel, London UK
Across the globe, the breadth of the rare disease
challenge is immense. It is estimated that 350 million
people worldwide suffer from rare diseases. “While an
individual disease might be labeled as “rare”, the total
number of persons in Europe suffering from one of the
over 6000 different identified rare diseases is estimated
at over 30 million. An individual rare disease may affect
only one person in a million, but all together, rare disease
patients comprise 6% to 8 % of the EU population.”
(EURORDIS 2014).
“In recent years, several new rare diseases are being
included in clinical trials for the development of
orphan drug candidate. Oncology, Genetic diseases,
Autoimmune, pulmonary disease, Central nervous
system and others are main focus of orphan drug
developers. All of these diseases have large patient base
but their rare forms doesn’t have suitable therapeutics
which reflects the marketing potential of orphan drugs
in these segments.
Out of these, oncology segment has got maximum
attentionfollowedbyraregeneticdisorders,neurological
disorder and autoimmune disorders. In a bigger view,
orphan drug segment is expected to grow several
folds in coming years due to several favorable factors.
Introduction of innovative technologies, favourable
pricing, reimbursement, unmet medical necessities and
strong clinical pipeline could be attributed as important
factors responsible for their growth.” (Research and
Markets’ Global Orphan Drug Market Future Outlook
2020 2015)
Orphan Drugs & Rare Diseases Global Congress 2016
Europe is going into its 2nd year in March 2016. The
conference will provide a unique platform for the
convergence of key stakeholders in the orphan drugs
industry to discuss key issues that impact them as
well as network with top tier government, hospitals,
pharmaceuticals, biopharmaceuticals, non-profit
organisations,orphandrugsdevelopersaswellasregional
and local manufacturers. We are putting together an
agenda that address the driving macroeconomic factors,
policies and issues that will steer the development of
orphan drugs globally including commercialisation,
policies, reimbursement, pricing and more. We look
forward to having you be part of the event!
Gain Latest Insights On
• Key Economic Drivers in the Orphan Market: What is
Driving the Industry and the Role of Governments?
• Growing the Market: The Roles of Big Pharmas, Biotechs
and Start-Ups
• Emerging Markets: Challenges and Benefits in Investing
in Emerging Markets
• Europe’s Regulatory Landscape: What Works and What
Doesn’t?
• Strategic Partnerships: What Strategies Are Available to
Ensure Partnership Success
• Pricing Strategy: How to Price Correctly?
• Clinical Trials: From Early Drug Development to Patient
Recruitment and More!
Who Will You Meet
Presidents, Heads/C-Level Executives, Directors, VPs
and Managers from following departments:
• Commercialisation
• Regulatory Affairs
• Market Access
• Sales & Marketing
• Research & Development
• Corporate & Business 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.
Big Pharmas 20%
Governments (Ministries of Health/Drug Authorities) 10%
Non Governmental Organisations (NGOs) 5%
Others 10%
Orphan Drugs Manufacturers 20%
Biopharmaceuticals 20%
Regional & Local Contract Manufacturers 10%
Hospitals & Patient Group Members 5%
MEDIA PARTNERS

SPEAKERS
Malcolm Allison, Head of Pulmonology & Anti-Infectives, BAYER HEALTHCARE PHARMACEUTICALS,
SWITZERLAND
Malcolm lives marketing. He started his career in the pharmaceutical Industry in the UK with Glaxo, selling and
marketing in dermatology. He worked with Cyanamid in the field of NSAIDs, before moving to the International field
with Smithkline. Successive moves took him deeper into strategic marketing, including a period in the US running
cardio-vascular, pulmonological and metabolic diseases for the company. He began to get involved with the rarer
diseases when he joined Novartis in Basel, initially with the field of transplantation. Subsequent to his time in Novartis,
Malcolm has worked in Actelion and Bayer, in the field of rare lung diseases. He is motivated by making a difference,
his motto: Try. If you succeed, you lead, if you fail you guide.
Patricia Anderson, Vice President of Regulatory Services MAPI GROUP
Patricia is the Vice President of Regulatory Services and runs the large team that includes US, Canadian, International
Regulatory Services, Medical Devices and Regulatory Operations (Publishing). She has more than 30 years’ experience
in regulatory affairs, including time with Hyal, Miles (Bayer), Novopharm, and as an independent consultant. She has
successfully achieved marketing approvals in Canada, the United States, Europe and smaller markets across a broad
range of therapeutic areas.
Patricia has established long-term relationships with many key decision makers at regulatory agencies around that
world.
Dr. Virginia Acha, Executive Director Research, Medical and Innovation, ASSOCIATION OF THE BRITISH
PHARMACEUTICAL INDUSTRY (ABPI), UK
Dr Virginia Acha is the Executive Director Research, Medical and Innovation and has responsibility for driving the
agenda for innovation in the UK. Dr Acha previously worked for Amgen as Director, Global Regulatory and R&D
Policy – Europe, Middle East and Africa. She holds the chair for the Pharmacovigilance working group in the IFPMA
Bio therapeutics Committee, and until recently was the Vice-Chair for the European Biopharmaceutical Enterprises
(EBE) Biosimilars Task Force and the lead for the EFPIA Regulatory Network supporting Turkey. Previously, she spent
nearly five years at Pfizer working on policy development and engagement in a number of domains, including science,
innovation and access and choice in healthcare. Dr Acha entered the pharmaceutical industry after a decade in
academia where she held posts on innovation strategy at Imperial College London Business School, the Science Policy
Research Unit (SPRU) at the University of Sussex, the Centre for Research in Innovation Management (CENTRIM)
at the University of Brighton, as well as a post-doctoral fellowship at London Business School. She is also a Visiting
Researcher in the Innovation & Entrepreneurship Department at Imperial College Business School London.
Martin Andrews, SVP – Rare Diseases, GSK, UK
A Biology graduate, Martin joined the Pharmaceutical Industry in 1985 as a trainee with Beecham Group Plc. He has
followed a broad Commercial career path working across numerous therapy areas in country, regional and global
positions embracing new product development, commercial strategy and significant operational leadership roles
including. In his current role Martin leads an integrated unit comprising R&D, Medical, Scientific and Commercial
support. GSK’s Rare Disease medicines portfolio includes marketed medicines for PAH, and medicines in development
for Amyloidosis and Gene Therapy :
2014–date Senior Vice President, GSK Rare Diseases
2008-2014 Senior Vice President, Global Vaccines Commercial.
2006-2008 Assistant to the Chief Executive Officer.
2003-2006 Vice President and General Manager, GSK Finland.
2001-2003 Vice President Clinical Development and Product Strategy, Neurology & Gastroenterology.
1999-2001 Vice President Global Commercial Strategy, Anti-Infectives and Acting Head European Marketing.
1997-2001 Marketing Director, UK
1996-1997 National Sales Director, UK
1992-1996 Business Unity Director, Vaccines
Martin was a Member of the Board of Directors of Pharma Industry Finland, the BUPA subsidiary Partnership in
Health and Trustee of the Sir James Black Trust.

SPEAKERS
Benoit Arnould, PhD –Senior Director, Global – Patient-Centered Outcomes, MAPI GROUP
Dr. Benoit Arnould leads the Global Patient-Centered Outcomes research team. Dr. Arnould has been conducting
studies to develop and validate Patient-Reported Outcome and other Clinical Outcomes Assessment instruments for
more than 15 years, measuring a large variety of concepts including Adherence, Acceptance, Satisfaction, Health-
Related Quality of Life, Function and Symptoms. In recent years, he has increasingly been asked by Industry clients to
assist in their Endpoint strategy definition.
Dr. Arnould has experience in a wide variety of conditions, including urinary incontinence, gastrointestinal disorders,
COPD, depression, neuropathic pain, erectile dysfunction, metabolic disorders, ophthalmologic diseases, cancer, and
various rare diseases.
Previously a statistician in a veterinary epidemiological research unit and in the pharmaceutical industry, Dr. Arnould
has sound knowledge of clinical and epidemiological trial design, data analysis and interpretation. He also has extensive
practical experience of a number of different healthcare systems, having worked for several years in various countries
in Africa and Asia.
Dr. Arnould is a Health Economics graduate with a specialty in statistics, and has completed, under the supervision of
Professor Gerard Duru, his PhD on tools for clinical decision making, a subject on which he publishes regularly.
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).
Adam Barak, Managing Director, PPi HEALTHCARE CONSULTING LTD
AdamBarakMCIM,DirectorofPPiHealthcareConsultingLtd,has20yearsofinternationalpricingandreimbursement
(P&R) practice in healthcare for both industry and in consultancy with extensive experience in establishing global P&R
strategies for pharma, biotech and medical device and diagnostics companies and healthcare services.
After an earlier 10 year career of sales, marketing and pricing in the automotive industry with General Motors, Adam
joined GlaxoWellcome’s International P&R division in 1996, in 1997 became Head of European Pricing and developed
international pricing strategies for 4 of the 7 GW therapy areas, including for oncology, cardiovascular, critical care
and in urology, anaesthesiology, diabetes, CNS and metabolic areas. Adam was responsible at GW for developing
international pricing strategies for many lead brands such as telmisartan/Micardis (hypertension), dutasteride/
Avodart (BPH), ondansetron/ Zofran (emesis), troglitazone/Romazin (diabetes), bupropion/Zyban (smoking cessation),
alosetron/Lotronex (IBD) and sumatriptan/Imigran (migraine).
In 2001 Adam joined Oxford GlycoSciences as International Pricing & Reimbursement Manager, developing global
P&R and market access strategies for Orphan technology miglustat/Zavesca for the rare, inherited disorder Gaucher’s
Disease, the company’s first pharmaceutical launch. In 2002 Adam established ABPPC Ltd to provide the healthcare
industry with specialised support in developing pricing strategies, as well as supporting funding and market access
activities. Out of ABPPC grew the international group PPi Healthcare Consulting Ltd which Adam still heads, with
offices in 25 countries. For 13 years PPi has been providing global coverage supporting commercialisation, planning
and implementation for drugs, diagnostics, medical devices and healthcare services.
Adam has experience with a multitude of therapeutic areas, has authored a number of publications, (7 published 2011-
2015 including the one below on pricing and funding for treatments for rare disease), regularly presents to international
audiences on international pricing, funding and market access topics and offers P&R training to the industry as well as
other parties such as health ministries and distributors.
In 2010-15 Adam has presented on pricing and funding in healthcare at 10 separate international conferences,
including the ISPOR 2010 conference in Prague (where he chaired the Educational Symposium on the topic of funding
orphan drugs and led a panel discussing health access in Greece during the Greek economic crisis), the 2014/15 World
Orphan Drug Congresses (Brussels and Geneva) and the last two year’s World Pharma Pricing and Market Access
Congresses in London.
Adam is a Chartered Marketer and a Member of the Chartered Institute of Marketing.

SPEAKERS
Dr. Steve Bradshaw, Managing Director, VALID INSIGHTS
Steve Bradshaw has had over 16 years in healthcare both as a clinician and as an expert advisor to the pharma industry.
His expertise is in developing value-based market access strategies and solutions, including the implementation and
use of real-world studies. In the rare and orphan disease space he has experience with multiple products and conditions
ranging from Fabry disease through to retinitis pigmentosa.
Dr Bradshaw writes and speaks regularly on the challenges facing the global healthcare environment; he has presented
widely and has over30publications. His careerfeatures being EditoratNatureClinicalReviews,an ophthalmicsurgeon
in the NHS, and various positions to senior executive level in market access and HEOR consulting fi rms. He is a peer
reviewer for several industry and medical journals and currently holds a position on the Pharma IQ Advisory Board.
Dr. Carlos R. Camozzi, Chief Medical Officer, ORPHAZYME, DENMARK
Dr. Camozzi has more than 25 years’ experience in the biopharmaceutical industry, most recently within the orphan
drugs development and paediatric therapeutic options, and successful achievements of products approvals at the
European Medicines Agency (EMA) and U.S. Food and Drug Administration (FDA). Prior to Orphazyme, Dr. Camozzi
held roles of increasing responsibility at Lederle/ American Cyanamid, F Hoffmann La- 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 Genetherapy for rare diseases.
Gülce Belgin, Founder & Director, PROCEUTICA
Mrs Belgin holds a B.Sc degree in Chemistry from Bogaziçi University and an M.B.A from Koç University, Istanbul.
Since completing her MBA she worked as a consultant to pharmaceutical industry for the last 13 years in Turkey.
During this periodshe developedspecial interest in Orphan Drugs. Gülce’sentrepreneurial experiencealreadyincludes
successfully founding and running a business consultancy company since 2008. She is the founder and Director of
Proceutica, a company based in Istanbul providing market access services to global Orphan Drug companies. Gülce’s
experience in the area of Named Patient / Early Access and Compassionate Use Programs enables patients to have
access to therapies as early as possible through existing and innovative early access mechanisms.
Kelly Franchetti, Executive Director, Global Patients Insights and Engagement, MAPI GROUP
Kelly Franchetti has nearly two decades of direct patient experience, gained in both hospital and industry settings
including CROs, healthcare technology, patient research and hands-on patient care across multiple therapeutic areas.
Ms. Franchetti has been involved in the development and moderation of advisory boards, patient HCP and KOL
forums, and has collaborated with Patient and Clinical Advocacy groups.
This positions her to contribute significant insights into how patients regard their treatment options and how to best
approach patient recruitment and retention strategies.
Simon Denegri, National Director for Patients and the Public in Research, NATIONAL INSTITUTE FOR
HEALTH RESEARCH (NIHR) and Chair, INVOLVE, UK
Simon Denegri is National Director for Patients and the Public at the National Institute for Health Research (NIHR),
and Chair of INVOLVE – the national advisory group for the promotion and support of public involvement in research
funded by NIHR. He was Chief Executive of the Association of Medical Research Charities (AMRC) from 2006 until
2011 and, prior to this, Director of Corporate Communications at the Royal College of Physicians from 2003.
He also worked in corporate communications for Procter & Gamble in the United States from 1997 to 2000. He has a
long-standing personal and professional interest in the needs and priorities of people with dementia and their carers
and currently chairs the Lay Champions Group for the national portal on dementia research that is to be launched this
year. He is a member of the NIHR Advisory and Strategy Boards, and a Board member of the UK Clinical Research
Collaboration (UKCRC), Farr Institute and care.data programme respectively. He blogs about the public and health
research at http://simon.denegri.com/ and publishes his poetry at http://otherwiseknownasdotcom.wordpress.com/

SPEAKERS
Dr. Alberto Gimona, VP - Head of Global Clinical Science and Epidemiology, ACTELION
PHARMACEUTICALS, SWITZERLAND
Alberto Gimona, MD is head of the Global Clinical Science and Epidemiology department in Actelion, leading a
team of approximately 50 scientists, epidemiologists and physicians in the therapeutic areas of Pulmonary Arterial
Hypertension, immunology, neurology and antiinfectives. Alberto holds a medical degree from the University of
Pisa in Italy, and a specialization in Clinical Pharmacology from the University of Milan, Italy. Alberto has more
than 25 years of experience in the pharmaceutical industry, including roles as clinical project leader in the areas of
rheumatology, bone and respiratory diseases in Rhône Poulenc Rorer in France and Novartis in Basel. He pursued
his career as Therapeutic Area Lead in Serono, then Merck Serono in Geneva, with responsibilities for immunology,
inflammation and oncology as well as clinical pharmacology, and Therapeutic Area Lead at Novartis in Basel in
Immunology Rheumatology and Psoriasis, before joining Actelion.
Jonathan Higginson, Director, Global Market Access, SHIRE INTERNATIONAL
Jonathan is currently the Regional Market Access Lead responsible for JAPAC & AU/NZ and an interim Global
Value Strategy Lead for a rare disease product, he is based out of Shire International in Switzerland. He has over 10
years of industry experience drawing from tenure at boutique value strategy consultancies PRMA Consulting and
PriceSpective as well as time spent with IMS Consulting (formerly Cambridge Pharma Consultancy). He provides a
unique perspective on pricing strategy, overcoming evidence burdens to gain reimbursement, and optimizing access
from experiences across the US, Europe, and JAPAC markets. Preceding his consulting tenure, Jonathan worked in
cellular biology and monoclonal antibody development R&D. He holds degrees in Molecular Cellular Developmental
Biology and Business Economics.
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.
Dr. Edmund Jessop, Public Health Adviser, NHS ENGLAND, UK
Edmund Jessop has been practising public health for the NHS in England since 1981 in various jobs at district, regional
and national levels. Since 2002, Edmund has been medical adviser to the team which plans, funds and monitors
services for patients with very rare disease. This has included decision making on the so-called “ultra-orphan” drugs
as well as other highly specialised technologies. He was Vice President of the UK Faculty of Public Health (FPH) from
2011 to 2014 and Editor of the Journal of Public Health 1998 - 2007. and has taught courses on public health including
health economics and health technology assessment for over 15 years. Edmund is UK representative to the EU Expert
Group on Rare Disease.

SPEAKERS
Dr. Sukhwinder S. Jossan, Associate VP - Global Regulatory Affairs, FERRING PHARMACEUTICALS,
DENMARK
Sukhwinder has an extensive research background with among others 25 original publications, as well as several
overview publications and abstracts. His background includes many years as a Research Scientist at the Medical
Pharmacology University of Uppsala in Sweden and several years with the Swedish Medical Products Agency. He
joined Ferring in 2001 as Director of Regulatory Intelligence and has since had different management positions within
Regulatory Affairs. He has also been course leader for Medicademy; an international educational program established
in 2002 by The Danish Association of the Pharmaceutical Industry. As of 2010 he has been Associate Vice President
within Global Regulatory Affairs with responsibility for development projects for all therapeutic areas within Ferring.
Dr. William C Maier, Chief Scientific Officer, MAPI GROUP
Mapi’s Chief Scientific Officer, Dr. Will Maier provides scientific leadership to Mapi’s research and consulting services
across a range of scientific areas.
Dr. Maier has more than 20 years of drug development and commercialization experience.
He served as Senior Director of Epidemiology at GlaxoSmithKline and Elan Pharmaceuticals and led research
groups conducting observational research to support reimbursement, marketing and drug safety investigations of
pharmaceuticals.
Dr. Maier is a member of the EMA’s European Network of Centres for Pharmacoepidemiology and Pharmacovigilance.
Dr. M. (Ken) Kengatharan, President, ARMETHEON, INC., USA and General Partner,
ATHENEOS CAPITAL, 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.

SPEAKERS
Camille Métais, Associate Director, Regulatory Affairs, ALEXION PHARMACEUTICALS, SWITZERLAND
Camille Métais is Associate Director, Regulatory Affairs in Alexion Pharma GmbH, Zürich, Switzerland. Camille
is responsible for the management of projects involving the design and implementation of European and global
regulatory strategies for the development, registration, and maintenance of pharmaceutical and biotechnology
medicinal products developed by Alexion. Her expertise is in development of regulatory strategies for drugs and
biologics, especially including orphan and pediatric diseases.
Before joining Alexion, Camille was Director with Voisin Consulting Life Sciences and was helping small to mid-size
companies in the design and implementation of their regulatory strategies. With Voisin Consulting, Camille was
heavily involved in the discussions around the implementation of the pediatric regulations. She focused on orphan
products, design of Pediatric Investigation Plans and first-in-human clinical developments.
Camille is an engineer by training and graduated from the Ecole Polytechnique, Paris, France where she specialized in
biology and biochemistry.
Kay Parkinson, Chief Executive, CAMBRIDGE RARE DISEASE NETWORK (CRDN) and Founder,
ALSTRÖM SYNDROME UK
Kay Parkinson was the mother of two children who were diagnosed with the ultra rare disease Alström Syndrome
when they were aged 18 & 15, having had four previous mis-diagnosis. Both children died following heart and heart/
kidney transplantations aged 25 and 29 respectively. She qualified as a lawyer in 1996 as a mature student, specialising
in charity law.
In 1998 she founded the charity Alström Syndrome UK (ASUK). Kay served as their CEO for 15 years before stepping
down in 2013 to start up Alström Europe (AS EU) charity. In 2015 Kay joined the Cambridge Rare Disease Network as
Chief Executive she believes all rare diseases need to work together for better diagnosis, treatments, services and a
much needed higher public profile.
Richard Porter, Global Head of Operations Management for Neuroscience, Ophthalmology and
Rare Diseases (NORD), ROCHE, SWITZERLAND
Richard has 20+ years’ experienceinPharma/BiotechR&Dfromworking atASTRA,Vernalis,Rocheand Shire.Richard
is currently responsible for ensuring the strategy, governance and operating model of the rare diseases unit within the
department of neuroscience ophthalmology and rare diseases of pRED, Roche. Richard has extensive experience of
leading projects in neurodevelopmental , genetic and orphan diseases across multiple therapeutic areas.
Flóra Raffai, Executive Director, FINDACURE, UK
Flóra Raffai is the Executive Director at Findacure, a UK charity building the rare disease community to drive research
and develop treatments. She was the first hire at Findacure, developing the charity’s projects, funding, and community.
Flóra organises Findacure’s patient group empowerment programmes, runs scientific community engagement
projects, develops the charity strategy, and oversees major funding applications and online communications. She also
line manages other members of staff. Flóra graduated from the London School of Economics and Political Science
with a BSc (hons) in International Relations. She is a founding member of the Cambridge Rare Diseases Network and
volunteers as Co-Organiser for the Cambridge Chapter of Good for Nothing.
Dr. Maria del Mar Mañú, Project Manager, EUROPEAN NETWORK FOR RARE AND CONGENITAL
ANAEMIAS (ENERCA), SPAIN
Dr. Maria del Mar Mañú-Pereira, obtained her degree in Biology at the University of Barcelona (Spain) in 2002 and
specialized in Human Genetics. She obtained her PhD in 2009, with a thesis on “Haemoglobinopathies and Glucose-6-
phosphate dehydrogenase in Catalonia: Epidemiological and molecular genetics studies in the newborn population.”
Since 2002, she has been working as a molecular biologist at the Red Cell Pathology Laboratory of Hospital Clínic
- Barcelona. Dr. Mañú-Pereira performs diagnostic tests for red cell disorders, and is leading the development of
methodologies for genetic characterization. Dr. Mañú-Pereira also collaborates as professor with the University of
Barcelona.
Her research activity is devoted to haemoglobin, membrane and enzyme disorders; particularly to the molecular
characterization and genotype-phenotype correlation. She has been involved in several national and international
projects. She is member of the Coordination Team of ENERCA (European Network for Rare and Congenital Anaemias)
since 2007.

SPEAKERS
Oliver Timmis, Head of Projects, ALKAPTONURIA SOCIETY (AKU), UK
Oliver Timmis is the Head of Projects for the AKU Society, an entrepreneurial patient organisation supporting those
diagnosed with a rare disease, alkaptonuria (AKU). Oliver leads on funding applications for new projects, allowing for
the creation of a National AKU Centre for UK patients, and international phase III clinical trials called DevelopAKUre.
He graduated with a BA(hons) in Natural Science (Physiology, Development and Neuroscience) from Cambridge
University. Oliver volunteers at Eurordis (Rare Diseases Europe) on their DITA (Drug Information, Transparency
and Access) taskforce, sits on the Patient Advisory Council at the RD Connect registries project, and is a member of
the Patient-centered Special Interest Group at ISPOR. He also volunteers for Findacure, the Fundamental Diseases
Partnership.
Dr. Rick Thompson, Scientific Officer, FINDACURE, UK
Dr Rick Thompson works primarily on Findacure’s drug repurposing programme. This aims to develop a social
funding model for pharmaceutical repurposing research into rare diseases. Findacure aim to use this programme to
help patient groups to identify and fund early proof of concept clinical trials into existing generic drugs that could be
used to treat rare diseases. Rick holds a PhD in Evolutionary Biology from Cambridge University, and has experience
in palaeontology, genetics and evolutionary theory. He is working to apply his scientific experience to the treatment
of rare diseases, and help communicate the science behind these illness to both the rare disease community and the
general public.
Dr. Hideo Tonomura, Director, OHARA PHARMACEUTICAL, JAPAN
MAJOR ACCOMPLISHMENTS
• Deeply involved in Pariet® (Proton pump inhibitor) NDA submission and launch activities in Japan.
• Promoted Pariet® on Japan market from 1997-2000 and 2003-2006 as a product manager. Pariet® achieved more
than 30 billion yen sales performance in fiscal year 2006.
• Established regulatory and clinical study plan and conducted the meeting with Japanese authority (PMDA) for the
new indication of Helicobacter pylori eradication in Japan in 2001.
• Temporary transfer from HQs to the US branch company in 2002. Involved in establishing GI franchise committee
by building pipeline proposals for new GI products as one of GIFC members.
• Presented data of cost effectiveness in patient with GERD on Pariet on May 2006 at DDW/AGA in Chicago
• Involved in the contract agreement for transfer of the two ethical drugs from Minophagen Pharmaceutical Co., Ltd.
• Involved in the contract agreement for the ethical drugs from Lion Corporation on Japan marketplace in 2008.
• Contracted with J&J for an OTC out-licensing right of Pariet® in Nov., 2010.
• Presented interim results of a PK/PD study with two posters on May 2012 at DDW/AGA in San Diego.
• Data Management of the study result with PAREXEL for finalizing the CSR in 2012
• Conducted in approval of the indication for patient with ulcerative colitis on Humira in 2013
• Conducted in approval of additional indication for H.pylori eradication on Pariet in 2013
• Conducted in submission of additional indication for low dose aspirin induced peptic ulcers on Pariet in 2013
• Conducted in managements of clinical studies on GI, Inflammatory and Immunology therapeutic programs in
Japan at Eisai from 2011 to 2014
Michael Rooney, Senior Business Support Manager, ALMAC GROUP
Michael has over 15 years experience working in the Pharmaceutical industry. In this time he has been responsible
for leading multi-disciplinary teams consisting of personnel from QA, QC, Packaging Design, Production and Logistics,
in order to support a number of challenging, but ultimately successful product launches/site transfers and on-going
commercial supply to global markets.
After graduating from University of Ulster with a degree in Applied Biochemical Science, Michael went on to spend
3 years as an Analytical Chemist for a major US Pharmaceutical company, where he gained valuable experience with
analytical transfers, validation, stability and EU Import analysis.

SPEAKERS
Frans van Andel, Access of Health Technologies, Board Director PPi Healthcare Consulting Ltd
Dutch nationality. Board Director of PPi Healthcare Consulting Ltd. Degrees in health economics (Universities of
Groningen and Utrecht) and public health (Harvard, USA). PhD in pharmaceutical economics. More than 25 years
of experience in market access issues involving pharmaceuticals and medical devices with work experience in the
pharmaceutical industry, CRO’s and WHO. In addition, investment and healthcare restructuring projects (especially
rehabilitation facilities for handicapped persons) for the public and private sector in Central and Eastern Europe,
Africa, Middle East and Asia. Assignments from the health insurance sector, medical technology and pharmaceutical
industries as well as institutional agencies such as the European Union, the World Bank and the Netherlands’
Government. Expert in market access issues involving pharmaceuticals and medical devices, project development
sourcing, health financing and insurance and public/private mix. Fluent in Dutch, English, German. Conversant in
French and Russian.
Céline Taveau, Project Director - Head of Drug Department, MAPI GROUP
Céline Taveau has 8 years of experience in market access. Céline provides senior leadership for projects on strategic
market access and HTA’s for pharmaceutical, vaccine and medical device companies. Céline has extensive experience
with orphan drugs and has worked on projects in oncology, immunology, cardiovascular and infectiology. Her
experience is wide working on projects for market access strategies, value proposition development, French HTA
submissions and payer advisory boards.
Dr. Barbara Valenta-Singer, VP – Global Clinical Development, BAXALTA, AUSTRIA
Barbara Valenta-Singer, M.D, Vice President Clinical Development at Baxalta (formerly Baxter) since June 2013,
manages with passion and enthusiasm a global team driving the development of compounds Phase I-IV across the
franchises and shapes the Clinical Development organization by engaging in innovative partnerships.
Barbara joined Baxalta in 2007, where she has been entrusted roles with increasing responsibilities, from Medical
Director Technical Assessment, then Senior Director Global Clinical Operations, to her current position as Vice
President Clinical Development.
Before joining Baxalta, Barbara worked clinically and non-clinically at the University of Vienna, gained experience
as assessor in the Austrian Regulatory Agency and built her solid experience and knowledge in the areas of Medical,
Clinical, Regulatory, Pharmaco-Vigilance, Market Access, and Quality, in a country, regional, and global environment
in organizations such as Serono, Pharmacia, Pharmacia-Upjohn, Pfizer and Wyeth Whitehall Export.
Barbara holds a Medical Doctor degree as well as a Medical Specialist degree in Pharmacology and Toxicology from the
University of Vienna.

DAY 1 | PRE-CONFERENCE WORKSHOP | MONDAY, 14 MARCH 2016
08:30 - 09:00 Registration
09:00 - 12:30 WORKSHOP A
10:30-11:00 Refreshment Break
Workshop A
What Are the Strategies and Limitations for Commercial, Market and Patient Access for Orphan Drugs?
TOPICS DISCUSSED AT THE WORKSHOP ARE:
• Pricing of OD’s usually follows the same logic as drug pricing in general - Is this still justified?
• Is economic evaluation of OD’s as basic input for reimbursement fl awed? - Is there optimal input for reimbursement of
OD’s?
• What is the effect of societal considerations on pricing and reimbursement of OD’s?
• Is there a justified role for patient advocacy groups and the media in pricing and reimbursement of OD’s?
• Is there an optimal approach of the industry with respect to pricing and reimbursement of OD’s - Does a “one size fits all”
approach” make sense?
Led by:
Frans van Andel, Access of Health Technologies, Board Director PPi HEALTCARE CONSULTING LTD
12:30 - 13:30 Lunch Break
13:30 - 17:00 WORKSHOP B
15:00 - 15:30 Refreshment Break
Workshop B
Overcoming Challenges in Rare Disease Health Research and Orphan Drug Program Commercialization
Opening Introductions:
Will Maier, PhD, MPH, Chief Scientific Officer
• The Process of Understanding the Person with the Indication
Kelly Franchetti, RN, CCRN, CEN, Executive Director, Global Patients Insights and Engagement
• Market Access Challenges for Rare Diseases – Recent Development in HTA and Reimbursement Decision-Making
Considerations for Orphan Drugs in Europe
Céline Taveau, Associate Scientific Director, Real World Strategy & Analytics
• Orphan Drug and Rare Diseases – Requirements for Real World Data: Generating Data to Gain and Keep Market Access
Will Maier, PhD, MPH, Chief Scientific Officer
• Regulatory Hurdles Orphan Drug – Are They Increasing?
Patricia Anderson, Vice President, Regulatory Services
• How to Capture the Patients and Caregivers Perspectives in Rare Diseases? A Challenge for Orphan Drug Developers
Benoit Arnould, Senior Director, Patient-Centered Outcomes
Led By: Mapi Group
Workshop A
Workshop B

DAY 2 | 1st DAY OF CONGRESS | TUESDAY, 15 MARCH 2016
08:00 Registration
08:30 Co-Chairperson’s Opening Remarks
MACRO OUTLOOK & TRENDS
08:40 Welcome Address: The Right Honourable Professor the Lord Kakkar, BSc, MBBS, PhD, FRCS, member of the House of
Lords and Director of the Thrombosis Research Institute (London)
09:00 Keynote Presentation/Interview: Regulatory Pathway for Approval in Europe Past, Present and Future
Speaker being finalised
9.40 Leadership Panel: What Currently Prevails for the Growth of the Orphan Drugs Market and What Must
Stakeholders Do to Continue to Propel Market Growth?
• What has driven the change in the landscape for financing orphan and rare disease drug development?
• How are Big Pharmas performing in the orphan drugs market landscape and what should be done to ensure
the viability of the development of orphan drugs?
• How can drug manufacturers collaborate with governments, payers and patients to continue meeting unmet needs?
• What are the critical success factors that will continue to drive growth in the orphan and rare disease drugs space?
Moderator:
Dr. M. (Ken) Kengatharan, President, ARMETHEON, INC., USA
Panellists:
Malcolm Allison, Head of Pulmonology & Anti-Infectives, BAYER HEALTHCARE PHARMACEUTICALS, SWITZERLAND
10:20 What Barriers Remain in the Development of Orphan Drugs for the Paediatric Population?
• What are the changes to the regulatory requirements that are likely to impact development of orphan drugs for this
patient population?
• How will changes to clinical development design impact bringing orphan drugs to the market for paediatric patients?
• What are some of the challenges when developing orphan drugs for children with rare diseases?
Dr. Carlos R. Camozzi, Chief Medical Officer, ORPHAZYME, DENMARK
10:55 Morning Break & Networking
11:35 Key considerations for the successful commercial launch of an orphan drug product into Europe
Michael Rooney, Senior Business Support Manager, ALMAC GROUP
12:10 Case Study: How Can Emerging Markets Participate in the Development of Orphan Drugs With the Rest of the
World and How Will This Impact the Pharmaceutical Industry in the Region?
• What are the key drivers for manufacturers to invest in the manufacturing of orphan drugs in emerging markets?
• What major challenges will developers face when distributing orphan drugs in emerging markets and how can these
challenges be overcome?
Dr. Hideo Tonomura, Director, OHARA PHARMACEUTICAL, JAPAN
12:45 <Sponsor Spotlight Presentation> MAPI GROUP
13:20 Networking Lunch
14:20 Drug Repurposing for Rare Diseases Using Social Finance
• What are some of the benefits of repurposing existing generic pharmaceuticals to treat rare diseases with an unmet
medical need?
• How does repurposing reduce the investment required in drug discovery, and leverage on known information on
drug behaviour and safety in diverse patient populations?
• What is Findacure’s outline model and progress in turning this concept into a reality in collaboration with patient
v groups, the NHS and consultancies?
Dr. Rick Thompson, Scientific Officer, FINDACURE, UK
MACRO OUTLOOK & TRENDS

POLICY FRAMEWORKS & COLLABORATIONS
14:55 Evaluation of Ultra Orphan Drugs: How Can Stakeholders Collaborate to Steer the Development of Orphan Drugs?
• How can stakeholders evaluate factors leading to high costs and strategise on the changes needed to policies?
• What are some of the long term strategies in supporting ultra orphan drugs?
• How are governments collaborating with stakeholders to make ultra orphan drugs available to patients?
Dr. Edmund Jessop, Public Health Adviser, NHS ENGLAND, UK
15:30 Considerations for Securing Early Advice in Europe
• Regulatory and commercial (market access) guidance
• Timing: when to seek guidance
• Questions to ask and considerations
• Formal and informal guidance approaches, pros and cons
• Examples of formal EAP schemes in Europe
16:05 Regulatory Success: Navigating the Regulatory Landscape for Orphan Drugs in Europe
• What is the current regulatory framework in the EU in terms of pre-market requirements and how do these
regulations affect time to market for orphan drugs?
• What submission requirements are expected during the drug registration process?
• What regulatory challenges will manufacturers need to confront during the drug registration process?
16:40 Tea Break & Networking
17:15 Orphan Drugs: Criteria, Comparison and Case Studies for US and Europe Regulatory Landscapes
• What are the criteria for orphan drugs in EU and US?
• What are the incentives available to companies developing orphan drugs?
• What are the differences in the regulatory landscape for orphan drugs in EU and US?
• Review case studies and future initiatives
Dr. Sukhwinder S. Jossan, Associate VP - Global Regulatory Affairs, FERRING PHARMACEUTICALS, DENMARK
17:50 Forming Strategic Partnerships: How Can Partnerships Improve the Orphan Disease Investment Story?
• What are the critical aspects of making a multi-stakeholder partnership successful?
• How can biotech companies partner with big pharmas to construct and deliver robust clinical trials from a broad
patient population?
• How can drug development collaboration/partnership improve the chance of bringing orphan drugs to the market?
• What is the role for non-profit research/academic organizations for developing novel drugs in the orphan drugs space
• How can rare disease foundations enable rapid development of orphan drugs?
Moderator:
Dr. M. (Ken) Kengatharan, President, ARMETHEON, INC., USA
Panellists:
Dr. Barbara Valenta-Singer, VP – Global Clinical Development, BAXALTA, AUSTRIA
Malcolm Allison, Head of Pulmonology & Anti-Infectives, BAYER HEALTHCARE PHARMACEUTICALS, SWITZERLAND
Flóra Raffai, Executive Director, FINDACURE, UK
18:30 Chairperson’s Closing Remark & End of Day 1
18:30 Drink Reception
19:00 Networking Gala Dinner
POLICY FRAMEWORKS & COLLABORATIONS

DAY 3 | 2nd DAY OF CONGRESS | WEDNESDAY, 16 MARCH 2016
08:00 Registration
08:30 Co-Chairperson’s Opening Remarks
ORPHAN DRUGS DEVELOPMENT – CLINICAL TRIALS
Session Moderator: TBD
08:40 Uncertainty in Early Drug Development: What Are the Factors and Steps to be Taken to Ensure Success of the Drug
Development?
• What are the challenges in target identification and target validation for a particular rare disease?
•What are some of the payer issues that should be addressed early on?
•How does market and financing landscape factor into early drug development?
•Is there a major role for repurposing of old drugs for new uses in orphan and rare disease space?
Richard Porter, Global Head of Operations Management for Neuroscience, Ophthalmology and Rare Diseases (NORD),
ROCHE, SWITZERLAND
09:15 How Patient Networks and Advocacy Groups Can Help in Design of Clinical Trials and Patient Recruitment?
• How can understanding and identifying patients’ experience plays out and where patients are getting their
information help in clinical development?
Simon Denegri, National Director for Patients and the Public in Research, NATIONAL INSTITUTE FOR HEALTH RESEARCH
(NIHR) and Chair, INVOLVE, UK
09:50 The Challenge of Outcome Trials in Rare Diseases: From Short Terms Symptomatic Endpoints to Long Term
Morbidity Mortality Endpoints; The Pulmonary Arterial Hypertension Experience
• How can manufacturers show clinically meaningful outcomes (hospitalisation, death, irreversible disease progression)
which have an important impact on the life of the patients and not only just a symptomatic effect?
Dr. Alberto Gimona, VP - Head of Global Clinical Science and Epidemiology, ACTELION PHARMACEUTICALS,
SWITZERLAND
10.25 Morning Break & Networking
11:05 How Can Developers and Regulators Collaborate to De-Risk Clinical Development?
• How can manufacturers work with regulators in understanding regulatory issues and challenges to minimise risks
during clinical development?
• Are regulators providing enough guidance for development and approval of agents in the orphan drugs space?
• Should regulators give breaks in the regulatory requirements for expedited drug development and approval in orphan
disease space, if yes, what are currently available and what more could be done?
Moderator:
TBC
Panellists:
Dr. Sjef de Kimpe, VP – Early Drug Development, BIOMARIN, NETHERLANDS
Dr. Sukhwinder S. Jossan, Associate VP - Global Regulatory Affairs, FERRING PHARMACEUTICALS, DENMARK
11:40 Cell and Gene Therapy for Rare Diseases: From Research to Regulatory
• What are the challenges in identifying a novel target and validating it in the orphan drugs space?
• Special considerations for developing novel cell and gene therapies for rare diseases?
12:15 Evaluation and funding challenges for gene therapies: exploring potential frameworks for reimbursement
• Gene therapies are at the frontier of translational medicine, promising a hopeful future, and in some cases even a cure,
for many patients with otherwise untreatable orphan and rare diseases
• The waters are as yet relatively unchartered: uncertainty in long-term clinical benefits, plus high-cost and budget
impact of these novel technologies raise concerns among payer and other decision-makers
• Over the next decade many new gene therapies will launch: how they are evaluated, and how they are paid for, will
set the precedent for those that follow
• How to actively navigate to overcome payer uncertainty and prepare for funding challenges are the key questions
Dr. Steve Bradshaw, Managing Director, VALID INSIGHTS
12:45 Is the UK the Right Place for Expanding Treatments for Rare Diseases?
• Genomics England has raised the potential for identifying and delivering treatment for rare disease.
How can the UK deliver on this potential?
• Innovation support through translational research networks, academic research and collaboration in the UK can
make a difference
ORPHAN DRUGS DEVELOPMENT – CLINICAL TRIALS

• More can be done to bring together rare disease patient groups, drug discovery teams in industry and academia,
healthcare services and policy makers to deliver new treatments for unmet need in the UK
13:15 Networking Lunch
MARKET ACCESS, PRICING & REIMBURSEMENT
Session Moderator: TBD
14:15 Outcome Measures for Rare Diseases: From Biomarkers to Patient-Relevant Outcome Measures
• Appropriate and validated patient-centered outcome measures are key to the assessment of new therapies for
rare diseases
• Most rare diseases lack such outcome measures and there development is too costly and to lengthy to be
considered by many sponsors
• Developing outcome measures for rare diseases should be approached as a pre-competitive activity in the best
interest of all stakeholders
• Initiatives to reach this goal will be presented
Dr. Ségolène Aymé, Emeritus Research Director, INSERM, FRANCE
14:50 How rare is it to achieve access to Orphan Drugs in Turkey ?
• Overview of the Orphan Drugs Market in Turkey
• Policy and practice associated with orphan drugs and treatment of rare diseases
• Market access strategies for Orphan Drugs
Gulce Belgin, Founder and Director, PROCEUTICA, TURKEY
15.20 Pricing and Return on Investment (ROI) of Orphan Drugs: What is the Right Price Strategy to Obtain Access
for Patients and Remain Commercially Viable?
• Cost drivers and changes in rare disease drug pricing
• HTA in rare diseases and payer budget management
Jonathan Higginson, Director – Global Market Access, SHIRE PHARMACEUTICALS, SWITZERLAND
15:55 Tea Break & Networking
16:35 Advocating For and Engaging 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?
Moderator:
Panellists:
Simon Denegri, National Director for Patients and the Public in Research, NATIONAL INSTITUTE FOR HEALTH RESEARCH
(NIHR) and Chair, INVOLVE, UK
Oliver Timmis, Head of Projects, ALKAPTONURIA SOCIETY (AKU), UK
Kay Parkinson, Chief Executive, CAMBRIDGE RARE DISEASE NETWORK (CRDN) and Founder, ALSTRÖM SYNDROME
UK
17:15 Coverage and Reimbursement: Can Change to the Current Policies to Encourage Companies to Continue Responding
to Incentives and Meeting Unmet Needs?
• What criteria are driving Health Technology Assessments (HTA) assessments in European countries and how are
these assessments speeding up patients’ needs for orphan drugs?
• How “pragmatic” have European HTA bodies become in considering available clinical data, recognising the limitations
of cost-effectiveness analysis and including wider elements of value/societal preferences into their P&R
recommendations for orphan drugs?
• What more could HTAs do to enhance the environment to enable faster and more cost-effective development and
approval of orphan drugs?
Miriam Gargesi, EuropaBio Healthcare Director, EUROPABIO, BELGIUM
17:50 Registry Implementation for Rare Diseases in Europe: The Rare Anaemias Model
Dr. Maria del Mar Mañú, Project Manager, EUROPEAN NETWORK FOR RARE AND CONGENITAL ANAEMIAS (ENERCA),
SPAIN
18.20 Chairperson’s Closing Remark & End of Conference
MARKET ACCESS, PRICING & REIMBURSEMENT

SILVER SPONSORS
Almac Group
With over 40 years’ experience, Almac is an FDA and EU approved out-
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Bowel Syndrome, Almac leads the CMO field in integrating its services to
support orphan drugs.
Almac’s dedicated product supply team are experts in navigating market
requirements and supporting client partners to successfully launch their orphan drug products. They have extensive practical
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Assisting client partners to achieve launch timelines and maximise product flexibility to serve global markets, Almac offer a
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History - In 2002 PPi Healthcare Consulting Ltd was established to provide the healthcare industry with specialised support in
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Services - Our market access services cover a broad range of areas which companies encounter in bringing technologies to the
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• Implementing and facilitating P&R submissions and revisions
• Direct stakeholder and secondary research to inform positioning and value demonstration strategies
• Supporting pre-approval usage / Early Access
B. Market Research Consulting Services:
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C. Health Economic Consulting Services:
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D. Other Market Access Consulting Services:
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www.ppi.consulting

SILVER SPONSORS
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11
Biosimilars Global Congress 2014 Europe
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provide our services to you, to respond to your inquiries about our offerings, and to offer you
other products, programs or services that we believe may be of interest to you. We
sometimes use such information to communicate with you. If you choose to submit content for
event publication or event program, we may publish your name and other information you
have provided to us. If you are a previous or existing customer, we may contact you with
information about products and services similar to those, which were the subject of a previous
sale to you. Furthermore, we may use your data, or permit selected third parties to use your
data, so that you can be provided with information about unrelated products and services
which we consider may be of interest to you. We or they may contact you about these
products and services by any methods that you consented at the time your information was
collected. As set out above, we are committed to safeguarding your personal information.
Whenever you provide such information, we are legally obliged to use your information in line
with all laws concerning the protection of personal information, including the Data Protection
Act 1998 (these laws are referred to collectively in this Privacy Policy as the “data protection
laws”
Fee:
The event fee includes lunch, refreshments and conference documents provided on the day.
This fee does not include travel, hotel accommodation, transfers or insurance.
Payment of Invoice
Our aim is to establish and maintain a long-term business relationship with all our clients and
customers, however failure to pay in accordance with clause causes us significant problems
and expense in chasing late payers. For late payers we will take action to enforce full
payment of the invoice, and without prejudice any other rights, to terminate the contract with
you in respect of any future supplies and to compensation for debt recovery cost under “The
Late Payment of Commercial Debts (Interest)” Act 1998 as amended and supplemented by
“The Late Payment of Commercial Debts Regulations 2002”. In jurisdictions where this may
not apply you accept that equivalent interest (8% above UK base rate) and debt recovery fees
(minimum £40) will be payable to offset our costs in this matter. We may set and vary credit
limits from time to time and may not accept any further orders if such credit limits are
exceeded. If you have a problem with any product or service please contact us immediately
and we will try and resolve the issue. You should however pay the invoice in full by the due
date to prevent further action!
Delegate Details
Terms and Conditions of Booking
Formation and Incorporation:
There is a 50% liability on all bookings once made, whether by fax, email or by post. Receipt
of this booking form, inclusive or exclusive of payment constitutes formal agreement to attend
and acceptance of the terms and conditions stated and are sold on a firm sales basis.
Payment Terms
Paradigm Global Events Ltd requires full payment of the amount to be made at the time of
booking via credit card. We reserve the right to decline any booking for the event. If payment
is not made at the time of the booking an event, then an invoice will be issued and must be
paid in full within 14 days from the date of the booking stated on the invoice and immediately
prior to the event. A credit card guarantee may be requested if the full payment has not been
received in full before the event and payment will be taken before entry to the event. We are
unable to allow a delegate to attend an event or have access to the Document Portal unless
payment has been received in full by us prior to commencement of the event. Paradigm
Global Events reserves the right to charge interest on unpaid invoices.
Cancellation
The event organized by Paradigm Global Events Ltd, a limited liability company formed under
English company law and registered in the UK. Cancellations received in writing more than 45
days before the conference date, will be eligible for 50% of the due less a £75 administration
fee. If you are unable to send substitution then Cancellations must be made in writing.
Cancellations made less than 45 days before the event date will not be entitled to a refund.
Substitutions / Name Changes
If you are unable to attend you may nominate, in writing, another delegate to take your place
any time prior to the start of the events. Two or more delegates may not share a place at an
event. Please make separate bookings for each delegate.
Alterations
Paradigm Global Events Ltd reserves the right to make alterations to the event / executive
briefing content, timing, speakers or program and venues beyond our control. The event may
be postponed or cancelled due to unforeseen events beyond the control of Paradigm Global
Events Ltd. We have no liability for any loss of trade or profit occurring to the customer as a
result of a charge or cancellation. Paradigm Global Events Ltd will not accept liability for any
transport disruption or individual transport delays and in such circumstances the normal
cancellation restrictions apply.
Data Protection
The information that we collect and store relating to you is primarily used to enable us to
provide our services to you, to respond to your inquiries about our offerings, and to offer you
other products, programs or services that we believe may be of interest to you. We
sometimes use such information to communicate with you. If you choose to submit content for
event publication or event program, we may publish your name and other information you
have provided to us. If you are a previous or existing customer, we may contact you with
information about products and services similar to those, which were the subject of a previous
sale to you. Furthermore, we may use your data, or permit selected third parties to use your
data, so that you can be provided with information about unrelated products and services
which we consider may be of interest to you. We or they may contact you about these
products and services by any methods that you consented at the time your information was
collected. As set out above, we are committed to safeguarding your personal information.
Whenever you provide such information, we are legally obliged to use your information in line
with all laws concerning the protection of personal information, including the Data Protection
Act 1998 (these laws are referred to collectively in this Privacy Policy as the “data protection
laws”
Fee:
The event fee includes lunch, refreshments and conference documents provided on the day.
This fee does not include travel, hotel accommodation, transfers or insurance.
Payment of Invoice
Our aim is to establish and maintain a long-term business relationship with all our clients and
customers, however failure to pay in accordance with clause causes us significant problems
and expense in chasing late payers. For late payers we will take action to enforce full
payment of the invoice, and without prejudice any other rights, to terminate the contract with
you in respect of any future supplies and to compensation for debt recovery cost under “The
Late Payment of Commercial Debts (Interest)” Act 1998 as amended and supplemented by
“The Late Payment of Commercial Debts Regulations 2002”. In jurisdictions where this may
not apply you accept that equivalent interest (8% above UK base rate) and debt recovery fees
(minimum £40) will be payable to offset our costs in this matter. We may set and vary credit
limits from time to time and may not accept any further orders if such credit limits are
exceeded. If you have a problem with any product or service please contact us immediately
and we will try and resolve the issue. You should however pay the invoice in full by the due
date to prevent further action!
Delegate Details
Card Billing Address:
Direct Tel: Direct Mobile:
Email address:
Card Holder’s Signature:
By Electronic Bank Transfer: Please transfer the full funds to Paradigm Global
Events Ltd bank account.
Methods of Payment: All payment must be made in Sterling.

ODRD 2016

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ODRD 2016