Orphan Drugs & Rare Diseases Congress 2016 Europe


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 attention followed by rare genetic disorders, 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 in its 2nd year in July 2016. The conference will provide a unique platform for the convergence of stakeholders in the orphan drugs industry to discuss and network with top tier government, hospitals, pharmaceuticals, biopharmaceuticals, non-profit organisations, orphan drugs developers as well as regional 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 Benefi ts 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


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.

segolene-aymeDr. 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).

edmund-jesspoDr. Edmund Jessop

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.

simon-denegriSimon 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/

malcom-allisonMalcolm 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.


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.

ken-kengatharanM. (Ken) Kengatharan, Ph.D., M.B.A. – President & COO (interim CEO)

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.

barbara-balentaDr. 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.

carlos-camozziDr. 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.

dr-hideo-tonomuraDr. Hideo Tonomura, Director, OHARA PHARMACEUTICAL, JAPAN

• 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

cmaille-metaisCamille 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.

oliver-timmisOliver 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.

miriam-garesiMiriam 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.

virginia-achaDr. 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.

Rick-thompsonDr. 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.

richard-porterRichard Porter, Global Head of Operations Management for Neuroscience, Ophthalmology and Rare Diseases (NORD), ROCHE, SWITZERLAND

Richard has 20+ years’ experience in Pharma/Biotech R&D from working at ASTRA, Vernalis, Roche and 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.

sukhwinder-jossanDr. 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.

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.

martin-andrewsMartin 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 signifi cant operational leadership roles
including. In his current role Martin leads an integrated unit comprising R&D, Medical, Scientifi c 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 Offi cer.
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.

adam-barakAdam Barak, Managing Director, PPi HEALTHCARE CONSULTING LTD

Adam Barak MCIM, Director of PPi Healthcare Consulting Ltd, has 20 years of international pricing and reimbursement (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.

frans-van-andelFrans van Andel, Access of Health Technologies, Board Director PPi Healthcare Consulting Ltd

​Short bio:

​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.

benoit-arnouldBenoit Arnould PhDSenior 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.

steve-bradshawSteve Bradshaw, UK [Affiliation details to be provided]

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 over 30 publications. His career features being Editor at Nature Clinical Reviews, an ophthalmic surgeon in the NHS, and various positions to senior executive level in market access and HEOR consulting firms. He is a peer reviewer for several industry and medical journals and currently holds a position on the Pharma IQ Advisory Board.

jonathon-higginsonJonathan 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.

micheal-rooneyMichael Rooney, Senior Business Support Manager

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.

maria-del-marDr. Maria del Mar Mañú


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
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.

kay-parkinsonKay 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 qualifi ed 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.

flora-raffaiFlóra 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 fi rst hire at Findacure, developing the charity’s projects, funding, and community.
Flóra organises Findacure’s patient group empowerment programmes, runs scientifi c 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.


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 period she developed special interest in Orphan Drugs. Gülce’s entrepreneurial experience already includes
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.

08:30 – 09:00 – Registration

09:00 – 12:30WORKSHOP A

10:30-11:00 – Refreshment Break



What Are the Strategies and Limitations for Commercial, Market and Patient Access for Orphan Drugs?


• 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 justifi ed 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 fi ts all” approach” make sense?

Led by:
Adam Barak, Managing Director, PPi HEALTHCARE CONSULTING LTD
Frans van Andel, Access of Health Technologies, Board Director PPi HEALTCARE CONSULTING LTD

12:30 – 13:30 – Networking Lunch Break

13:30 – 17:00 – WORKSHOP B – MAPI Group

15:00 – 15:30 – Refreshment Break


Workshop B – MAPI Group


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 Scientifi c 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 Scientifi c Offi cer
• 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

08:00 – Registration

08:30Co-Chairperson’s Opening Remarks


08:40Welcome 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 fi nalised

9.40Leadership 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 fi nancing 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?

Dr. M. (Ken) Kengatharan, President, ARMETHEON, INC., USA

Martin Andrews, SVP – Rare Diseases, GSK, UK
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 – MAPI GROUP

13:20Networking Lunch

14:20 – Drug Repurposing for Rare Diseases Using Social Finance
• What are some of the benefi ts 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


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

Adam Barak, Managing Director, PPi HEALTHCARE CONSULTING LTD

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?

Camille Métais, Associate Director, Regulatory Affairs, ALEXION PHARMACEUTICALS, SWITZERLAND

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

17:50Forming 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-profi t research/academic organizations for developing novel drugs in the orphan drugs space
• How can rare disease foundations enable rapid development of orphan drugs?

Dr. M. (Ken) Kengatharan, President, ARMETHEON, INC., USA

Dr. Barbara Valenta-Singer, VP – Global Clinical Development, BAXALTA, AUSTRIA
Martin Andrews, SVP – Rare Diseases, GSK, UK
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

08:00 – Registration

08:30 – Co-Chairperson’s Opening Remarks


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

• What are the challenges in target identifi cation 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 fi nancing 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

09:15How 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

10.25Morning 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?

Dr. Sjef de Kimpe, VP – Early Drug Development, BIOMARIN, NETHERLANDS
Camille Métais, Associate Director, Regulatory Affairs, ALEXION PHARMACEUTICALS, SWITZERLAND
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?

Dr. Sjef de Kimpe, VP – Early Drug Development, BIOMARIN, NETHERLANDS

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 benefi ts, 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 INSIGHT

12:45Is 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
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
• 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

Dr. Virginia Acha, Executive Director Research, Medical and Innovation, ASSOCIATION OF THE BRITISH

13:15 – Networking Lunch
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

14:50How 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

15.20Pricing 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

15:55Tea 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 confi dentiality and privacy protections for patients contributing their information?
• How can patient advocacy groups infl uence regulation and corporate strategy?
• How can developers make transparent the research goals and use of data to patient and continue to gain patients’ trust?

Dr. Sjef de Kimpe, VP – Early Drug Development, BIOMARIN, NETHERLANDS

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
Dr. Virginia Acha, Executive Director Research, Medical and Innovation, ASSOCIATION OF THE BRITISH

17:15Coverage 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

17:50Registry Implementation for Rare Diseases in Europe: The Rare Anaemias Model

18.20Chairperson’s Closing Remark & End of Conference


ppiPPI Healthcare Consulting
Adding value to commercialisation strategies through local expertise and insight.

Mission – Our mission is to deliver value through expertise and local market understanding to ensure successful market entry and optimise global business opportunities for our clients in the pharmaceutical, medical device and biotech sectors. We respect ethical principles, accelerating patients’ access to better treatments while delivering a fair added value to our clients.

History – In 2002 PPi Healthcare Consulting Ltd was established to provide the healthcare industry with specialised support in developing pricing strategies, as well as supporting funding and market access activities. We have offices in 25 countries and for 13 years have been providing global coverage supporting commercialisation, planning and implementation for drugs, diagnostics,medical devices and healthcare services. Our team includes experts who have been working in healthcare market access for many years, and together we have completed around 400 assignments in a multitude of therapy areas.

Services – Our market access services cover a broad range of areas which companies encounter in bringing technologies to the
market. Our main services include:

A. Pricing and Reimbursement (P&R) Consulting Services:
• Developing national and international pricing strategy throughout product planning process and post-launch
• 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:
• Market landscaping and segmentation
• Product positioning and messaging
• Stakeholder identification, mapping and decision analysis
• Payer panels and Advisory board meetings
• Qualitative and quantitative research

C. Health Economic Consulting Services:
• Develop core and country-tailored value / submission dossiers
• Economic modelling addressing national and sub-national payer requirements
• Provide value and clinical evidence-generation needs including supportive studies design
• Supporting Health Technology Assessment (HTA) submissions
• Systematic reviews

D. Other Market Access Consulting Services:
• e.g. launch sequencing assessment, in-licencing and transaction support, ATC (WHO medicines classification system) guidance


Almac Group

With over 40 years’ experience, Almac is an FDA and EU approved outsourcing partner to the global pharmaceutical and biotechnology industries. Having successfully partnered in the commercial launch of many of the industry’s recent first in class, innovative orphan drug products in areas such as Cystic Fibrosis, Muscular Dystrophy, Hemolysis and Short 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 experience of launching products into the various EU and ROW markets and manage a multidisciplinary project team in quality, packaging design, regulatory and distribution, ensuring launch timelines are met and market entry strategy is successful.

Assisting client partners to achieve launch timelines and maximise product flexibility to serve global markets, Almac offer a
full range of product launch support services including:

• Product launch consulting services
• Regulatory support for EU filings
• EU import testing and QP market release
• Packaging design and artwork generation
• Customised packaging solutions
• Order processing and Financial services
• Storage and end-user distribution (Ambient, Cold, Frozen & Controlled Drugs)

Benefiting from integrated pharmaceutical and distribution from a single supply team, clients minimise effort and maximise return.

Contact Details:

Mapi Group

Mapi Group has over 40 years of experience supporting Life-Science companies in utilizing Patient Reported Outcomes measures, Value communications and Commercialization support, Strategic Regulatory Services, and gathering Real-World Evidence on Pharmaceuticals, Biologicals, and Medical devices. Mapi Group is the premier provider of Health Research and Commercialization services to Life-Science companies enabling Market Authorization, Market Access and Market Adoption of novel therapeutics.



logo-valid-insightValid Insight™: Strength. Strategy. Solutions.

Delivering real knowledge, valid perspectives and trusted evidence solutions: Valid Insight is here to help conquer your toughest challenges crossing the life science landscape.

Our in-house advisory board – and unique network of payers, HTA advisors, clinical key opinion leaders and stakeholders in over 40 markets – gives you direct access to some of the world’s prominent advisors and thought leaders in the life science industry.

Our experts can help you understand value, clinical and marketplace opportunities for new products and therapies. We can then help you build strategy, evidence and marketplace solutions for each step of your product’s research, development and commercialisation lifecycle, which can be used to drive your decision making and help you overcome your toughest challenges in the real world.

We’re proud that this proven approach not only enables your commercial success, but also supports the delivery of better
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Durbin is one of the world’s leading specialist medical suppliers. Established more than 50 years ago in 1963, we source and distribute pharmaceuticals, medical equipment and consumable supplies to healthcare professionals in over 180 countries.

Our main divisions encompass everything from international sales, sales to NGO’s and charities, clinical trials supply, early access programmes and imported unlicensed medicines to sexual health supplies, and the storage and distribution of medical
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Durbin. Saving lives by saving time.


biocompareBiocompare is the most comprehensive, in-depth, and objective website for life science product information. Thousands of scientists use Biocompare daily to quickly find the right product for their experiments rather than looking through multiple print catalogs. Visit www.biocompare.com to find the products you need, stay informed of new technologies, read product reviews, watch product videos, and keep up-to-date on life science news. For further information please visit:www.biocompare.com



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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BioProcess International

BioProcess International™ is a monthly, controlled-circulation magazine devoted to the development, scale-up, and manufacture of biotherapeutics and biodiagnostics. Each issue provides the global industrial biotherapeutic community with up-to-date, peer-reviewed information detailing the business, politics, ethics, applications, products, and services required to successfully drive biopharmaceuticals, vaccines, and biodiagnostics through the development and manufacturing process. www.bioprocessintl.com


Global Events List

Global Events List is an online directory of scientific and medical events from around the world, brought to you by Elsevier. Attending these types of events facilitates the proliferation of scientific discoveries and the building of productive relationships. We hope to play our part by providing the most comprehensive, user-friendly, reliable, and high quality listing of events available. www.globaleventslist.elsevier.com


Manufacturing Chemist Pharma

Manufacturing Chemist is among the longest established and most respected information sources in the global pharmaceutical manufacturing industry. Manufacturing Chemist reports on current and future trends in the global marketplace, including the latest developments in drug discovery, biotechnology, drug delivery, ingredients, processing technologies and equipment, packaging, business strategies and regulatory updates. – See more at: www.manufacturingchemist.com



FiercePharma is the pharma industry’s daily monitor, with a special focus on pharmaceutical company news and the market development of FDA approved products.www.fiercepharma.com


Pharma Phorum

pharmaphorum.com is the premier digital publication for thought leadership relating to the pharmaceutical industry. The news, opinions and insights published every day help industry executives better understand the broader healthcare landscape within which they operate, including regular contributions from their peers, innovative service providers, physicians, payers / policy makers, patient organisations and patients. www.pharmaphorum.com



Pharmafile.com is a leading portal for the pharmaceutical industry, providing industry professionals with pharma news, pharma events, and pharma service company listings. Site content is produced by our editorial team for Pharmafile.com and our industry newspaper Pharmafocus. Service company profiles and listings are taken from our pharmaceutical industry directory, Pharmafile, and presented in a unique Find and Compare format to ensure the most relevant matches.www.pharmafile.com


My Event Guru

My Event Guru is an interactive portal that allows individuals and businesses to set up, market and promote events, sell tickets and collect RSVP’s, instantly process payments for events online, obtain feedback via sophisticated surveys and generate additional revenue by selling event content.
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Bio-equip is a professional website which provides the information of equipments, consumables, chemical reagents and experimental animals in life science. Our website which takes full advantage of the most up-to-date computer technologies and networks offers the most all-round and considerate service in the information of biology equipments for not only institutes, universities, labs in hospitals but also scientists in bio-engineering and bio-medicine industries and suppliers of lab equipments who would be exposed to comprehensive and well-timed market information to compare the different products in function and price and promote the development of scientific research and industry. www.bio-equip.cn



Pharmaceutical-technology.com is the only website focusing specifically on issues relevant to pharmaceutical professionals working with technology, be it development, engineering, IT or production. Pharmaceutical-technology.com brings you the latest in industry projects and updates, along with the news, views and trends that leading professionals – from senior executives to manufacturing managers and heads of procurement – require to stay on top of their field. Our international scope allows us to examine all pharmaceutical markets closely, from biotechnology to therapeutics, vaccines and more.www.pharmaceutical-technology.com



Conference Alerts is an online conference calendar for academics and professionals. Organizers post conference information on www.conferencealerts.com and subscribers receive alerts about conferences in their areas of interest via email.www.conferencealerts.com



Conferize is the world’s biggest social platform for events. You’re invited.We connect the world of events with speakers, people and media from any industry. We help event organizers build communities, websites and mobile experiences to increase the quality and attendance of their events. www.conferize.com



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