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Orphan Drugs and Rare Diseases Europe
15 May - 16 May 2017
Orphan Drugs and Rare Diseases Europe

ORPHAN DRUGS EUROPE 2017

2 DAY CONFERENCE: 15TH & 16TH MAY

SAE Media Group is proud to announce the return of their 6th annual Orphan Drugs Europe event to Berlin on the 15th -16th May 2017!
The global orphan drug market totaled nearly $123 billion in 2014 and will continue to grow to reach nearly $191 billion by 2019.*
With such growth and innovation witnessed in this field now is the best time to discuss and strategize the best route to market.

Germany is Europe's largest pharma market but is recognised as having one of the toughest pricing policies in Europe.
SAE Media Group 6th annual Orphan Drugs conference will discuss the controversial German pricing mechanism as well as bring together leading industry professionals to discuss the latest regulatory developments, explore how to reduce costs and learn from the latest innovations in the orphan drug landscape.

*http://www.businesswire.com/news/home/20160406005885/en/Global-Orphan-Drug-Market-Set-Grow-191
*http://www.pmlive.com/pharma_news/germany_planning_to_table_pricing_reforms_by_the_summer_1007643

PREVIOUS ATTENDEE TESTIMONIALS FROM ORPHAN DRUGS UK:

"Nice disease/compound specific talks"

“Good schedule, good variety of speakers”

“Very good speakers”

“An intensive and useful conference"
 

FEATURED SPEAKERS

Goran Medic

Goran Medic

Market Access Manager Europe, Horizon Pharma, Inc.
Tony Zbeidy

Tony Zbeidy

General Manager MENA, Orphan Europe

Anders Waas

Chief Executive Officer, Tikomed
Anders Waas

He has three decades of experience in the pharmaceutical, biotech and medical device industries and has significant Business Development, Commercial and R&D experience. Since January 2009, he is CEO of TikoMed. During 2008, he was the Vice-President of Business Development at Actogenix, based in Gent, Belgium. From 2004 to 2008, he was Vice President Business Development at CV Therapeutics in Palo Alto (US). Between 1993 and 2004 he held various senior management positions in product development, marketing and business development at Astra and AstraZeneca. He has also experience in the device industry from WL. Gore. He has since 2009 been engaged in the development of Orphan Drugs both in TikoMed and as a board member of Orphan Drug companies.

Bertram Haussler

Chairman of the Board of Management, IGES Institut
Bertram Haussler

Bertram Häussler is the chairman of the IGES Group, a corporation of research and consulting companies specializing in infrastructure and health care. He is a medical doctor, sociologist, and honorary professor of Economics of the Pharmaceutical Industry at the Technical University (TU) Berlin. His research work focuses mainly on industrial economics, health services research, health economics, benefit assessment in medicine as well as drug development in a national and international context.

Carina Schey

Researcher, University Of Groningen
Carina  Schey

Carina Schey has many years of both pharmaceutical industry and clinical pharmacy experience. She is a consultant health economist based in Switzerland, also currently pursuing research through the University of Groningen, PharmacoEconomics and PharmacoEpidemiology Unit in the Netherlands. The primary focus of her research is developing ways of demonstrating the true value of orphan drugs, as alternative options to standard cost-effectiveness studies.
Carina was a clinical pharmacist with a special interest in rare diseases, having worked at several leading London hospitals. Between 2002 and 2008, Carina designed and implemented patient-focused homecare services on behalf of the UK NHS to patients with rare diseases.
Carina has published several peer-reviewed articles and abstracts. Notable speaking engagements include the European Parliament “Patients’ Rights Day” and “The Chronic Diseases” conference, both in 2015.
Carina sits on the expert judges’ panel for the MassChallenge and on the scientific advisory panels for several charities.
 

Carol McCudden

Patient Ambassador, Ataxia UK
Carol  McCudden

I am Carol McCudden, 52 years old. I am an Occupational Therapist and qualified in 1991. In my clinical career I have worked with numerous conditions, including rare diseases. In November 2007 I was diagnosed with Friedreich’s Ataxia, three years after my younger brother had been diagnosed at the age of 37. I have been representing Ataxia UK, and the interests of people with ataxias, over the past 2 years. Along with other members of the third party sector in the UK, I lobby government to direct policy and delivery of health services to people living with neurological conditions. I also have a keen interest in research, specifically the lived experiences of people with neurological conditions.

Cate Lockhart

Health Economics and Outcomes Research, Omeros Corporation
Cate Lockhart

David Dasberg

Managing Director, Facio Therapies
David Dasberg

David Dasberg is the managing director of Facio Therapies and Facio Intellectual Property. He has over 20 years' experience with the development and commercialization of (bio)pharmaceutical products. As an independent consultant, David performed a feasibility study commissioned by the Dutch FSHD Foundation that laid the foundation for the Facio group of companies. Prior, David held business development positions of increasing responsibility at Pharming Group and Fresenius Kabi, where his last position was at the Vice President level.

Genevieve Michaux

Member of the Brussels and Paris Bar, Partner, mayer brown llp
Genevieve Michaux

Giorgio Iotti

Medical Scientific Manager - Rare Diseases Unit, Chiesi S.p.A.
Giorgio Iotti

Giorgio Iotti, PhD, is Project Leader for Advanced Threapy Medicinal Products and Special Care in the Research and Development Unit in Chiesi.
He leads the Holoclar® project, the first pharmaceutical product containing stem cells receiving marketing authorization in Europe, and gene therapy projects in development in collaboration with uniQure b.v.
He has five years of experience as Medical Manager of Chiesi Rare Diseases Unit, managing medical communication of extremely innovative products such as Glybera® and Holoclar®.
Giorgio was a postdoctoral fellow at the FIRC Institute of Molecular Oncology in Milan, Italy, and Kimmel Cancer Center, Philadelphia, US. He received his PhD in Experimental Medicine from the University of Modena and Reggio Emilia, Italy. He published several peer-reviewed articles and abstracts in the area of molecular oncology and advanced therapies.
 

Goran Medic

Market Access Manager Europe, Horizon Pharma, Inc.
Goran Medic

Jama Nateqi

Co-Founder, SYMPTOMA
Jama  Nateqi

Jama Nateqi is a medical doctor and co-founder of SYMPTOMA.com, a search engine for diseases.

He founded his first successful business in 1999 at the age of 16. Then studied human medicine in Austria and performed the research for his thesis at the Yale University, School of Medicine in New Haven, CT.

Meanwhile he financed his studies himself and co-founded his second company, SYMPTOMA.com.

After investing 10 years in Research & Development, SYMPTOMA probably has established the biggest disease database in the world.

The company is operating at a profit since beginning. The European Commission, the Dutch Ministry of Health and leading experts have acknowledged SYMPTOMA to be the „best & most promising eHealth solution in 2016“.

SYMPTOMA empowers physicians to diagnose even extremely rare diseases.
 

James Mcarthur

Chief Scientific Officer, Cydan
James Mcarthur

James McArthur, PhD has founded four companies including Imara, Vtesse, Cydan, and Synovex (renamed Adheron) and has published and patented extensively in the areas of gene therapy and rare diseases.
He currently serves as President of R&D of Cydan, a rare disease accelerator, and as CEO of Imara, developing therapeutics for the treatment of sickle cell disease. James was an Entrepreneur in Residence at HealthCare Ventures, where he started Synovex and was head of research and the Principal Scientist at Cell Genesys Inc, a pioneer in the field of AAV and lentiviral gene therapy. He began his career in gene therapy at Somatix Therapy. James is a member of the Board of Directors of NightstaRx, a leader in developing AAV gene therapy treatments for rare retinal genetic diseases, and he also works with the Friedreich’s Ataxia Research Alliance (FARA) as a member of its Board of Directors and Scientific Advisory Board, advancing the needs of patients with this rare neurologic disease.
James was a postdoctoral fellow at the Massachusetts Institute of Technology and the University of California, Berkeley. He received his PhD in biochemistry from McGill University.

 

Jana Rautenberg

Co-founder, Decodeon B.V.
Jana Rautenberg

Johan Drott

CEO, Respiratorius AB
Johan Drott

Julie Vallortigara

Research Officer, Ataxia UK
Julie Vallortigara

Dr Julie Vallortigara has been working in Ataxia UK’s research department since November 2013. Before that, she gained a strong knowledge in Neurosciences by obtaining a PhD in Nutrition and Neurosciences at University of Bordeaux (France). Then she specialised in neurological disorders through her research studies on pain and later on dementia at King’s College London. After five years working there as a research associate she decided to move into the charity sector. Since then she has developed a good understanding on the best practice for evaluation of research projects and their ongoing monitoring and support. Dissemination of research information to people living with ataxia and to researchers and healthcare professionals has also been an important element of her role. Finally Dr Vallortigara has gained significant knowledge about the issues and the needs of people living with a rare and long-life condition such as ataxia, by her contribution to the services provided to patients such as the helpline, workshops, and local support groups.
Dr Vallortigara is willing to initiate partnerships with professionals sharing the same interest for rare diseases, and with the ultimate perspective to find new treatments for the ataxias.
 

Leigh Shaw

Head of Regulatory Affairs/Quality Assurance, NightstarX
Leigh Shaw

Leigh Shaw has over 20 years industry experience, principally in the field of Regulatory Affairs. He is currently Head of Regulatory Affairs and QA at NightstaRx, a biotech company with offices in the UK and US which is developing gene therapies for inherited retinal disorders. Prior to joining NightstaRx he was Managing Director of GFA, an expert consultancy providing regulatory and clinical services, where he provided global strategic and operational regulatory support to a variety of pharmaceutical and biotech clients, from discovery to post-licensing. He has also held senior positions in pharma and contract research organisations, and started his career in biotech R&D working on gene therapies and live viral vaccines.
Originally a virologist by training, his first degree is in Microbiology and Virology from the University of Warwick. He also holds an MSc in Molecular and Biochemical Parasitology from the University of Salford and an MBA from the Open University. He is an active member of TOPRA (The Organisation for Professionals in Regulatory Affairs) and a visiting lecturer on Regulatory Affairs at Cambridge University.

 

Mark Connolly

Department of Pharmacy, Unit of Pharmacoeconomics, University Of Groningen
Mark Connolly

Dr. Mark Connolly is a health economist with 18 years of experience in academia and the pharmaceutical industry. Dr. Connolly is a Senior Research Scientist at the University of Groningen, PharmacoEconomics and PharmacoEpidemiology Unit in the Netherlands. Mark is also the Managing Director and Founder of Global Market Access Solutions with licensed offices in Switzerland, UK and United States. His research interests include health technology assessment (HTA), economics of healthcare, health policy, and evaluations of the broader economic and fiscal impacts of investments in healthcare. He has conducted several economic analyses for Europe, South-East Asia, Africa, Australia and the USA. He has published over 25 peer-reviewed articles and abstracts and his work has been reported in major print, television and internet media, including the BBC, The Telegraph, Newsweek, National Journal, Bloomberg and the Economist.
 

Nadia Assenova

Senior Director Regulatory Affairs, EMEA, Alexion Pharma GmbH
Nadia Assenova

Nadia Assenova MPharm, MBA, MSc is Senior Director, Regulatory Affairs – EMEA Region at Alexion Pharma GmbH in Zurich. She has 15 years of experience in global regulatory affairs. She joined Alexion in 2015 focusing on the registration of Orphan Medical products across the globe. Throughout her career, Nadia has been directly involved in multiple regulatory submissions, most recently focusing on development of new indications in ultra-rare diseases and regulatory strategy for the metabolic products within the Alexion pipeline. Nadia received her Master of Pharmacy degree from the University of Medicine in Sofia (Bulgaria). She also holds an MBA from the University of Strathclyde (UK) and M.Sc. in Health Policy from Imperial College, London (UK).

Nigel Nicholls

Director and Country Manager UK/Ireland, BioMarin Europe Ltd
Nigel Nicholls

Nigel Nicholls currently serves as the Director and Country Manager UK/Ireland for BioMarin Europe Ltd. He has held senior positions in clinical research, international marketing and country management for Pharmacia, Rhone Poulec Rorer, Schering AG, Orphan Europe, Jerini AG and BioMarin Europe Ltd.

He completed his MSc (Surrey) and MBA (Bath) postgraduate degrees with a focus on the ethics and economics of orphan drugs. He was formerly a trustee but is now a patron of Contact a Family.
 

Peter Ramge

Senior Global Pharma Lead & Orphan Drug Expert. Advocacy Specialist, Freelance
Peter  Ramge

With a pharmacist PhD background I joined the pharmaceutical industry in spring 1999 and now have over 17 years of cross-functional work experience, of which I spent nearly 9 years working in the rare disease space. My key roles and projects include leading the global launch of an ultra-orphan disease drug, building a global public affairs platform in haemo- philia, in-market and pre-launch brand management in the cardiovascular field and heading up a department for the medical management of a wider portfolio of various OTC and pre- scription brands.

My core competencies include

o In depth medical, marketing and public affairs experience incl. medical knowledge across a wide range of indications: haemophilia and rare bleeding disorders, cardio- vascular diseases, dermatology and pain management
o Strategy development and implementation: Marketing, patient advocacy and public af- fairs, communication
o Strong project management skills on a global and local level, very comfortable work- ing in a matrix organisation setting
o Complex stakeholder management, including KOLs and patient organisations
 

WORK EXPERIENCE OVERVIEW
2008 - 2017 Novo Nordisk Health Care AG, Zurich, Switzerland
• Senior Global Product Manager Changing Haemophilia™ and Stra- tegic Operations, Global Product Manager rFXIII (NovoThirteen®)
2005 – 2008 Sanofi-Aventis, Berlin, Germany
• Senior Brand Manager (Multaq® pre-launch, Aprovel®)
1999 – 2005 Boots Healthcare Deutschland, Hamburg, Germany
• Medical Manager & Head of Medical Department

EDUCATION

2003 – 2005 University of Witten/Herdecke, Germany: Postgraduate Study Course in
Pharmaceutical Medicine, M.Sc. Pharmaceutical Medicine
1995 – 1999 Johann Wolfgang Goethe University, Frankfurt/Main, Germany: Doctoral thesis (PhD), including a 3 months’ research project at King’s College, London, UK in 1997
1995 Practical year for pharmacists, Approbation as Pharmacist
1990 – 1994 Johannes Gutenberg-University Mainz, Germany: Pharmacy studies in- cluding exchange semester at Université de Bourgogne, Dijon, France (1993)
 

Richard Porter

Chief Operating Officer, Therachon AG
Richard Porter

Robert Karl

Partner, RBV Capital
Robert Karl

My name is Robert Karl, 49 years old and spend the last 24 years working for global pharmaceutical companies, mainly international, with increasing commercial responsibilities. I worked in Schering Plough, MSD and Mallinckrodt prior to joining RBV Capital as a partner and now represent a group of companies focusing on Turkey, Middle East and Central Eastern Europe, ADEXILIS/MEALIS.

ADEXILIS/MEALIS focuses on 5 therapeutic areas, Medical Imaging, Nuclear Medicine, Women`s Health, Anaesthesia and Orphan drugs. We do represent major global players in these areas for our markets. We offer a full service representation in the outlined geography and strive for maximizing our principal’s assets with highest ethical standards.

RBV Capital, as a small and specialized fund is always interested in Orphan-route /Orphan-product - oriented start-ups - even in Oncology. For us this is an opportunity to work and succeed with good science, cost efficient with companies that are many times not in the spot light of big VC. For many Orphan companies seek investment we might be a very good fit as we invest at earlier stages, we come with high-end expertise, market knowledge and market/industry access.

In my presentation I would like to illustrate the weight of these markets for innovative pharmaceuticals, Orphan Drugs and the opportunity working with companies like ADEXILIS/MEALIS, give an overview of the major market characteristics and outline key success factors for a successful entry strategy. I would also like to give colleagues useful insight on what Venture Capital companies look for, what value they can bring to their company and hopefully give them a good understanding how to approach them for mutual benefit and success. Recently RBV was part of a successful transaction with Retrosense and its deal with Allergan.
 

Tony Zbeidy

General Manager MENA, Orphan Europe
Tony Zbeidy

A globe trotter French-Lebanese national and a US graduate medical doctor who grew up in Lebanon where he obtained a BS in Biology. Travelled to US for his MD and moved to UK training and working in Romford, Harold Wood and Basildon Hospitals. In 2001, moved to France and joined Sanofi (Aventis at that time) as Medical Information Officer. In 2003, joined Orphan-Europe as Medical Advisor and in 2006, established Orphan-Europe (Recordati Group) subsidiary in Dubai Healthcare City taking the responsibility of MENA market as General Manager, representing today 1/3rd of Orphan-Europe total turnover and growing...
A distinguished achievement, having established alone the Middle East Metabolic Group (MEMG) and is organizing its annual meeting since 2004. Since 2011, major pharma companies acting in the field of rare disorders such as Genzyme, Biomarin, Nutricia, Merck-Serono, SOBI, Nestle, Synageva, etc, supported this group of 250 selected metabolic and genetic specialists from the MENA region. The MEMG meeting is still annually organized by Dr. Tony Zbeidy.
 

Conference agenda

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8:30

Registration & Coffee

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9:00

Chairman's Opening Remarks

Mark Connolly, Department of Pharmacy, Unit of Pharmacoeconomics, University Of Groningen

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9:10

First time ever a patients story: Living with Ataxia

  • An introduction to the challenge of the ataxias, a set of rare degenerative neurological condition and the role of patient group, Ataxia UK.
  • Patient experience of being a patient living with ataxia, her strategies for living with the condition, and her engagement as a patient with the NHS.
  • An overview of the challenges for Ataxia UK, as a patient group working with a set of rare conditions with no treatments or cures. 
  • Their  research strategy, partnerships with pharmaceutical companies and others, and the progress which has been made to date
  • Carol McCudden, Patient Ambassador, Ataxia UK

    Julie Vallortigara, Research Officer, Ataxia UK

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    9:50

    Host nation address: AMNOG and Orphan pricing & reimbursement in Germany

    Bertram Haussler, Chairman of the Board of Management, IGES Institut

    - Current situation and implications of the regulation update in 2017 (AMVSG)
    - Key issues in early benefit assessments of Orphan Drugs
    - Outlook  to the future
     

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    10:30

    Morning Coffee

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    11:00

    Case study on the regulatory hurdles and opportunities for development of a gene therapy for a rare inherited retinal disorder

    Leigh Shaw, Head of Regulatory Affairs/Quality Assurance, NightstarX

     • Experience with the regulatory opportunities in terms of what we gained from obtaining EU and US Orphan Drug Designation, and our interactions through regulatory meetings.
    • Hurdles; Lack of information on the natural history of the disease, issues because of limited availability of patients, using state of the art manufacturing and surgical techniques.
    • Differences in approach to gene therapies in different countries.
     

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    11:40

    Setting reimbursement strategies: How to establish a foundation for reimbursement

    Nigel Nicholls, Director and Country Manager UK/Ireland, BioMarin Europe Ltd

    • Analysing how payers are considering reimbursing drugs that are coming on the market for orphan drugs and rare diseases
    • Can workable changes be made to improve current reimbursement strategies?
    • Creating a collaborative reimbursement strategy that satisfies and benefits all parties involved
    • Taking a look at regulatory frameworks and how they influence the reimbursement process

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    12:20

    Networking Lunch

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    13:20

    Panel discussion: The sustainability of orphan drugs

  • Economic tools that improve access to orphan drugs
  • Sustainability of orphan drug reimbursement. How can we ensure the ongoing sustainability of orphan drugs
  • Is there a correlation between sustainability and the cost-effectiveness of orphan drugs? Is there a relationship between cost-effectiveness and affordability?
     
  • Robert Karl, Partner, RBV Capital

    Mark Connolly, Department of Pharmacy, Unit of Pharmacoeconomics, University Of Groningen

    Carina Schey, Researcher, University Of Groningen

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    14:00

    Value Assessment of Orphan Drugs and Treatments for Rare Diseases - a U.S. Perspective

    Cate Lockhart

    Cate Lockhart, Health Economics and Outcomes Research, Omeros Corporation

    • Prices of orphan drugs vs. rarity of disease – is there a link?
    • Are orphan drug prices increasing or decreasing since the launch date?
    • Landscape overview of orphan drug availability in several European countries

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    14:40

    Afternoon Tea

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    15:10

    Case study from an early stage project on achondroplasia and its associated complications

    Richard Porter

    Richard Porter, Chief Operating Officer, Therachon AG

    clock

    15:50

    Uncertainty in early drug development: What are the factors and steps to be taken to ensure success of the drug development?

    James Mcarthur, Chief Scientific Officer, Cydan


    • 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?
     

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    16:30

    VAL001 for Treatment of Diffuse Large B-cell lymphoma - Recently Granted Orphan Drug Designation in EU and US

    Johan Drott

    Johan Drott, CEO, Respiratorius AB

  • Company considerations pursuing an orphan drug designation
  • Commercial, medical and regulatory strategic thoughts to be considered
  • How might the Orphan Drug Designation affect development plans
  • clock

    17:10

    Chairman’s Closing Remarks and Close of Day One

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    8:30

    Registration & Coffee

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    9:00

    Chairman's Opening Remarks

    Carina Schey, Researcher, University Of Groningen

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    9:10

    Orphan Drugs: Criteria, comparison and case studies for US and Europe regulatory landscapes

    Nadia Assenova, Senior Director Regulatory Affairs, EMEA, Alexion Pharma GmbH

  • 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?
  • clock

    9:50

    Identifying the market in an orphan indication – challenges and opportunities

    Peter Ramge, Senior Global Pharma Lead & Orphan Drug Expert. Advocacy Specialist, Freelance

  • Campaign development for small target groups
  • Market access and pricing challenges
  • Reaching the prescriber and patient engagement opportunities
  • clock

    10:30

    Morning Coffee

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    11:00

    Case study Facio Therapies: Breaking Through to an FSHD Therapy

    David Dasberg, Managing Director, Facio Therapies

    • Why we are established
    • Lessons learned translating science into early stage drug discovery
    • How to align with the community you serve
     

    clock

    11:40

    Opportunities and challenges for biotech companies developing orphan drugs

    Anders Waas, Chief Executive Officer, Tikomed

  • Evolution of the orphan/rare disease market
  • Steps in Orphan drug development – From idea to new treatment of patients
  • Unmet need, science hand in hand
  • clock

    12:20

    Networking Lunch

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    13:20

    Orphan drugs in developing (Emerging) Markets

    Robert Karl, Partner, RBV Capital

  • A significant early potential in emerging markets
  • Overview of markets and potential
  • Key players and landscape
  • Fast track access routes
  • Critical success factors
  • clock

    14:00

    Experience on various aspects related EU based companies and MENA market

    Tony Zbeidy, General Manager MENA, Orphan Europe

  • Early Market Access, Market Structure, Current Market Situation
  • Current and Future perspectives
  • Optimal business models, what works and what does not?
  • clock

    14:40

    Afternoon Tea

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    15:10

    Development and commercialisation of advanced therapy medicinal products: The Holoclar case

    Giorgio Iotti, Medical Scientific Manager - Rare Diseases Unit, Chiesi S.p.A.

  • Translating stem cell therapy from academic research to industrial production
  • Peculiarities of the regulatory process of Holoclar, the first stem cell based product approved
  • Commercialisation of ATMPs and the challenge of hospital exemption in European countries
     
  • clock

    15:50

    What can be done to actively improve diagnosis of orphan diseases and increase number of patients in care?

    Jama Nateqi, Co-Founder, SYMPTOMA

  • Why most patients with orphan diseases go undiagnosed and others take 10 years from symptom to diagnosis.
  • What new ways can be utilised for physicians to diagnose orphan diseases?
  • How to empower patients and physicians to actively uncover their underlying orphan disease.
  • Why orphan drugs need new techniques of marketing & patient finding.
  • clock

    16:30

    Chairman’s Closing Remarks and Close of Day Two


    Chief Executive Officer
    Tikomed
    Chairman of the Board of Management
    IGES Institut
    Researcher
    University Of Groningen
    Patient Ambassador
    Ataxia UK
    Health Economics and Outcomes Research
    Omeros Corporation
    Managing Director
    Facio Therapies
    Member of the Brussels and Paris Bar, Partner
    mayer brown llp
    Medical Scientific Manager - Rare Diseases Unit
    Chiesi S.p.A.
    Market Access Manager Europe
    Horizon Pharma, Inc.
    Co-Founder
    SYMPTOMA
    Chief Scientific Officer
    Cydan
    Co-founder
    Decodeon B.V.
    CEO
    Respiratorius AB
    Research Officer
    Ataxia UK
    Head of Regulatory Affairs/Quality Assurance
    NightstarX
    Department of Pharmacy, Unit of Pharmacoeconomics
    University Of Groningen
    Senior Director Regulatory Affairs, EMEA
    Alexion Pharma GmbH
    Director and Country Manager UK/Ireland
    BioMarin Europe Ltd
    Senior Global Pharma Lead & Orphan Drug Expert. Advocacy Specialist
    Freelance
    Chief Operating Officer
    Therachon AG
    Partner
    RBV Capital
    General Manager MENA
    Orphan Europe

    Official Publication

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    Interview with Giorgio Iotti, Medical Scientific Manager, Rare Diseases Unit Chiesi Farmaceutici

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    Orphan Drugs UK 2016: Anthony Hall, Therapeutic Area Head Orphan Drugs, Mero BioPharma Presentation

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    Orphan Drugs UK 2016: Tim Guilliams, Founder & CEO, Healx Presentation

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    Orphan Drugs UK 2016: Timothy J. Miller, Ph.D.President & CEO, AbeonaTherapeutics Inc Presntation

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    Orphan Drugs UK 2016: Sheela Upadhyaya, Associate Director, NICE Presentation

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    Interview with Julie Vallortigara, Research Officer, Ataxia UK

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    Orphan Drugs and Rare Diseases 2016 - Past Attendees List

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    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.


    Absave

    Supporters
    http://www.absave.com

    Absave.com is the leading savings website for Antibodies and Immunological Products. Search our extensive database of Antibodies, Kits, Proteins & Peptides, Reagents, etc. to find the best savings!


    Pharmaceutical-Tech

    Supporters
    http://www.pharmaceutical-tech.com

    In the realm of Pharmacy, where innovation is rapid, Pharmaceutical-Tech stands adept at conceptualizing unique ideas relevant to the B2B space. Pharmaceutical-Tech is essentially a B2B online business, technology media platform and global magazine that have under its wraps the largest global database of Pharmacy buyers and suppliers. Pharmaceutical-Tech.com covers in-depth trends that shape industry dynamics and metamorphose global economics. With services like search engine optimization, global magazine, social media marketing, product video showcase, e-mail marketing, e-newsletter sponsorship, banner advertising, event marketing and micro-website within our platform, www.pharmaceutical-tech.com has created a recognition that spans over a global audience, thereby revolutionizing how businesses transact.


    Drug Target Review

    Supporters
    http://www.drugtargetreview.com

    Drug Target Review’s quarterly magazine, website and annual events program provides high quality content with peer-written articles that are submitted exclusively by the world’s most respected scientists in their field. This attracts a committed base of readers, users and delegates made up of senior decision-makers from the life science and drug discovery industries at the top pharmaceutical companies, as well as academics and scientists from the top research institutes across the globe.


    Pharmacircle

    Supporters
    http://www.pharmacircle.com

    PharmaCircle is an innovative knowledge management company serving the current and future global leaders in the Pharmaceutical and Biotechnology related industries. PharmaCircle is a one stop information and analysis source for pipeline, products, clinical trials, drug delivery technologies, deals and acquisitions, company financials, venture capital investments, product sales, pharmaceutical services, news, patents and more….


    CLocate

    Supporters
    http://www.clocate.com

    Clocate.com is a leading international directory for worldwide conferences and exhibitions. Clocate.com is equipped with a unique and comprehensive search that helps you find easily any event in any category or location. Each event includes detailed information, like, description, dates, location, map, prices, link to the official event's website and more...


    International Pharmaceutical Industry

    Supporters
    http://www.ipimediaworld.com

    IPI gives you an insight into all areas of Biopharm, Medical, Pharmaceutical and Healthcare. IPI provides a proven supportive means of communication to the Pharmaceutical, Bio Pharmaceutical, Nutraceutical and Medical Devices industry, incorporating the latest in research and technology innovations, regulatory guidelines, marketing and communication strategies.


    Drug Discovery Today

    Supporters
    http://www.drugdiscoverytoday.com/

    Drug Discovery Today covers the whole of the preclinical drug discovery process. The reviews are cutting edge, written by experts in their respective fields and cover all aspects of drug discovery from genomic and proteomic approaches, computational drug design, medicinal chemistry and the translation of these sciences to therapies.


    pharmaphorum

    Supporters
    http://www.pharmaphorum.com

    pharmaphorum drives innovation within the pharmaceutical industry, by bringing healthcare together through a suite of media services that help produce and disseminate thought leadership, combined with an online platform for communicating messages to a global audience.Visit www.pharmaphorum.com


    Drug Development Technology

    Supporters
    http://www.drugdevelopment-technology.com

    Drugdevelopment-technology.com covers every aspect of the drug development and research process, from conception to pre-FDA approval. Our global network of journalists updates the site with the latest and most significant industry developments. This coverage allows us to provide everyone - from senior management through to research and QA staff – with in-depth reviews of the latest projects, advance notice of new product releases and analysis of the latest procedural and legislative developments.


    RareBase

    Supporters
    http://www.rarebase.co.uk

    The RareBase Networking Portal links people and companies throughout the world with an interest in rare and paediatric diseases.


    ATAXIA UK

    Supporters
    https://www.ataxia.org.uk/

    Ataxia UK is the national charity for people affected by ataxia, a group of rare, neurological conditions, for which there is currently no cures. Ataxia UK works in partnership to facilitate, promote and fund research to develop much needed treatments for this neglected group of conditions. The charity also provides support services to the ataxia community


    Bentham Science

    Supporters
    http://www.benthamscience.com/

    Bentham Science Publishers is a major STM journal publisher of 130 plus print and online journal. Out of these, 40 journals have already registered good IMPACT FACTORS as per Journal Citation Reports® 2017. These titles have extensive readership mostly in Europe and North America. For a detailed profile please visit our website at http://www.benthamscience.com. Besides, Bentham Science publishes eBooks in all areas of Science, Technology and Medicine. Our eBooks provide professionals, academicians, corporate researchers, graduates and undergraduates worldwide with the most current information in their subject areas of interest. Our eBooks are also available in the ePub and Kindle formats besides the PDF edition here http://ebooks.benthamscience.com/. Bentham is offering attendees of this conference discounts on its publication. For more information click here


    Pharmavision

    Supporters
    http://www.pharmavision.co.uk

    PharmaVision offers a consultancy service providing independent pharmaceutical thematic research to the healthcare industry, the investment community and competitive intelligence specialists. We perform due diligence research and provide expert commentary in healthcare. Our reports combine scientific analysis in drug delivery, R&D technologies and pharmaceutical products including patient modelling, product/technology forecasts and market trends evaluation.


    Technology Networks

    Supporters
    http://go.technologynetworks.com/subscribe-to-newsletters

    Founded in 2000, Technology Networks is established as the leading news provider for life science and drug discovery professionals. In addition, we provide unique content including webcasts, videos, application notes and posters from recent conferences. Our portfolio now includes around 30 focussed scientific communities, all of which are accessible free of charge within TechnologyNetworks.com


    Labsave

    Supporters
    http://www.labsave.com

    Labsave is the leading savings website for Laboratory Equipment and Lab Supplies. We work closely with the top suppliers in the world to bring you the best products at the most competitive prices. We are constantly striving to secure the biggest and most exclusive offers that you won’t find anywhere else! http://www.labsave.com/


    Pharmiweb

    Supporters
    http://www.pharmiweb.com

    Established in October 2003, PharmiWeb Solutions is an energetic, innovative provider of web-based solutions for the pharmaceutical sector, with customers such as AstraZeneca, Roche, Servier Laboratories, and Merck With solutions in three key areas: Online Publishing, e-Business and Mobile Computing, we deliver connectivity – between people and applications – unlocking islands of data and knowledge. We own some of the sector’s most innovative Online Publications: PharmiWeb.com, HospitalPharma.com and Detail-Direct.com. We are actively developing solutions for Mobile Computing platforms. For example, our PharmiTabTM e-pharmacy solution runs real-time patient data and prescribing transactions in a busy hospital environment, on a TabletPC, wireless client/server platform. e-Business solutions range from e-Detailing, to Online Market Research, all designed to increase reach, increase sales, and to reduce administration and costs.


    European Biopharmaceutical Review

    Supporters
    http://www.samedanltd.com/magazine/12

    European Biopharmaceutical Review (EBR) is a magazine dedicated to the biopharmaceutical and biotech industries. Each quarterly edition features articles from key opinion leaders on research, drug discovery and development, biomanufacturing, outsourcing and more on the science and business of the sectors. EBR also features insightful interviews, reviews and event information to form a strong B2B platform. Read EBR in print, online, or via the Samedan app.


    CanBiotech

    Supporters
    http://www.canbiotech.com

    CanBiotech - A Portal and B2B Outsourcing Marketplace for the Biotech and Pharmaceutical Industry. The Marketplace features the Outsourcing Services Showcase; the Portal features our biopharmaceutical and venture capital directories and databases. Publications include the BioMedical Outsourcing Report and the Bio Outsourcing Asia© Publication.


    BioChem Adda

    Supporters
    https://www.biochemadda.com/

    BioChem Adda is a Not-just-for-Profit organization which aims to bring out the most reliable and transparent information about everything related to jobs, careers, education, news, articles, and events from the field of BioSciences and Chemistry!


    Pharmalicensing

    Supporters
    http://www.pharmalicensing.com

    Pharmalicensing (www.pharmalicensing.com) is the premier biopharmaceutical Open Innovation resource designed for professionals involved with partnering, licensing and business development worldwide. Actively supporting all forms of partnering and in- and out-licensing activities, Pharmalicensing utilizes the unique online Showcase Profiling & Discover services, as well as its renowned Partnering Search service leveraging its global network of industry experts, to enable companies to identify and engage with appropriate partners around the world. Pharmalicensing is actively utilized by many tens of thousands of industry professionals each month. Pharmalicensing is a division of Cognis Group, Inc.


    BioPharm Insight

    Supporters
    http://www.biopharminsight.com

    BioPharm Insight is your definitive guide to the global life sciences community. Subscribers take action on forward-looking intelligence uncovered by an independent team of investigative journalists, and make strategic business decisions using the most comprehensive and powerful real-time database of market analytics and key contacts. Featuring an intuitive online interface and exclusive Active IntelT relational content technology, BioPharm Insight provides an unrivaled capability to segment and analyze the industry with detailed and searchable profiles.


    Samedan

    Supporters
    http://www.samedanltd.com/magazine/13.

    International Clinical Trials (ICT) is a quarterly magazine edited by Dr Graham Hughes, and written by specialists at the forefront of clinical research. ICT’s coverage of operational developments ranges from adaptive designs in early phase trials through to post marketing research. ICT’s targeted readership consists of key decision makers across the pharmaceutical and biotech industries, along with contract partners and consultants. The magazine is distributed to pharmaceutical professionals in Europe and North America, and is accessed globally online. Read the latest issues, explore the ICT archive and subscribe at www.samedanltd.com/magazine/13.


    ASD MEDIA

    Supporters
    http://www.asd-network.com

    Professionals in the Aerospace & Defence market use the ASD Media internet platforms to:
    • Be informed on the latest market developments; www.asd-network.com
    • Find the latest business news; www.asd-network.com
    • Find the upcoming events; www.asd-network.com
    • Find companies and organizations; www.asdsource.com
    • Distribute news globally. www.asdwire.com
    The ASD Media platforms are well known and used intensively, your company is able to benefit from these large numbers of users. Be informed with ASD-Network; Create exposure for your company with ASDSource, distribute your news with ASDWire or advertise with us, build your brand and increase traffic to your company’s website.

    For more detailed information please contact with: ASD MEDIA
    Stefan.koopman@asdmedia.nl


    PMR

    Supporters
    http://www.pmrcorporate.com

    PMR (www.pmrcorporate.com) is a British-American company providing market information, advice and services to international businesses interested in Central and Eastern European countries as well as other emerging markets. PMR's key areas of operation include business publications (through PMR Publications), consultancy (through PMR Consulting) and market research (through PMR Research ). Being present on the market since 1995, offering high international standards in projects and publications, as well as providing one of the most frequently visited and top-ranked websites, PMR is one of the largest companies of its type in the region.


    AntibodyChain

    Supporters
    http://www.antibodychain.com

    The complete and transparent marketplace for your research antibodies, kits, proteins and small molecules. The sophisticated search engine, with more than 800.000 products, transparency in suppliers, easy comparison, the one stop shop for your research antibodies in combination with interesting news and events plus a CMO exchange makes us a full service partner for you.


    Farmavita

    Supporters
    https://farmavitar.com

    FarmavitaR+ is the professional network of experts and service providers. Network is gathering local consultants from 90 countries in Europe, Asia, North America, Latin America, Australia and Africa. Management of international, multi-centre projects is our core competence. FarmavitaR+ is providing solutions related to pharmaceutical, medical device, food supplement and cosmetic products. Scope of services is related to solutions for product development, quality assurance, clinical trials, product registration, portfolio analysis, lifecycle management, vigilance/risk management, pricing/reimbursing, market access and promotional compliance. FarmavitaR+ is brand name of Farmavita Regulanet Ltd. Visit https://farmavitar.com for more information. Outsource anything you can think of!


    European Biotechnology Network

    Supporters
    http://european-biotechnology.net/

    The European Biotechnology Network is dedicated to facilitating co-operation between professionals in biotechnology and the life sciences all over Europe. The non-profit organisation brings research groups, universities, SMEs, large companies and indeed all actors in biotechnology together to build and deliver partnerships.


    Biosave

    Supporters
    http://www.Biosave.com

    Biosave.com brings together the best Life Science promotions, product releases, featured products, publications, videos and events from 100’s of leading Life Science suppliers. Updated 24 hours a day, Biosave is constantly uploading special promotions and exciting product news that you won’t find anywhere else. Our goal is to provide our users with the most exclusive product information to help reach that all important purchasing decision.


    Labiotech.eu

    Supporters
    https://goo.gl/DRE2Gp

    Labiotech.eu is the leading digital media covering the European Biotech industry. Over 100,000 monthly visitors use it to keep an eye on the business and innovations in biotechnology. Hope you'll enjoy reading our stories!


    Copybook - Pharmaceutical

    Supporters
    http://www.copybook.com/pharmaceutical

    Copybook is a global business network with many pharmaceutical events, exhibitions and seminars. We encourage business-to-business transactions and work tirelessly to gain our customers new business contracts worldwide. You can add events, companies and associations for free. When you are a member, you can add your company to the events listed in order to encourage other visitors to arrange meetings and appointments at those events to facilitate new business contracts. Want to see how we can increase your business network and transactions? SIGN UP FOR FREE today! Copybook – Your Global Business Network


    Select Science

    Supporters
    http://www.selectscience.net/

    SelectScience is an independent, online information resource for the worldwide scientific community, and the home of trusted information for laboratory scientists. Discover impartial, expert opinion and trusted reviews about latest laboratory equipment and techniques; plus videos, application notes and science news from around the world. Become a member for free today.


    One Nucleus

    Supporters
    http://www.onenucleus.com

    Established in May 2010, One Nucleus is the result of the merger of ERBI and London Biotechnology Network. One Nucleus is a not-for-profit membership organisation for international life science and healthcare companies and the largest of its kind in Europe. The company is based in Cambridge UK and London, at the heart of Europe's largest cluster. The 470 members include pharmaceutical, biotech, medical device and diagnostic companies and associated technical and commercial Service Providers.


    Contract Biotechnology

    Supporters
    http://www.contract-biotechnology.com

    Contract-Biotechnology.com is a web-based platform for laboratory outsourcing solutions. It is an online R&D matching tool that connects Scientifics and service and product providers worldwide. The platform Contract-Biotechnology.com would help you in the process of finding the right partner saving time and money, because with one single and secure application you would be able to receive multiple quotes quickly, keeping your contact information confidential. Contract-Biotechnology.com is the new Collaboration Network Model for Discovery Research and Development. Contract-Biotechnology.com team has extensive experience working for pharmaceuticals, biotechs, universities and academic research institutes and can help you addressing your key gaps.


    Gate2Biotech

    Supporters
    http://www.gate2biotech.com

    Gate2Biotech is a portal that unites biotechnological community in Central Europe. It covers all the news in the field of biotechnology. Thanks to the the portal the companies engaged in the field can easily search for research institutions and other partners to solve technical problems they are facing and here they can also present their services offer to potential partners from the Czech Republic and abroad. Gate2Biotech portal serves as a bridge connecting scientific and commercial sector. It incorporates offices and support organizations dedicated to promoting transfer of innovative technologies into a unified communication platform of Czech and international biotechnologies. As a mediator of information it draws attention to biotechnologies and support of their application and transfer into practice. It also functions as a tool for encouraging non-professionals (especially scientists and students) towards innovative business or applied research.


    Swiss Biotech Association

    Supporters
    http://www.swissbiotech.org/

    The Swiss Biotech Association (SBA) is the national industry association for biotechnology, including pharmaceuticals, diagnostics, agriculture, food, cosmetics, environmental biotechnology, and specialty chemicals. Members are companies active in modern biotechnology, such as R&D, Production, Marketing and Sales, Finance, Services and Consulting. SBA provides a networking platform for Life Science clusters, academic and federal institutions the like. Founded in March 1998, the Association grows steadily.


    Mednous

    Supporters
    http://www.MedNous.com

    MedNous is a print publication and website about medical innovation in Europe. It carries exclusive interviews with companies that are at the forefront of medical technology, as well as contributor articles from prominent practitioners. Our mission is to identify significant advances in medicine and to explain how this innovation is being commercialised. In doing so, we talk to venture capitalists about what products and platforms they are supporting. We report on how regulators cope with the accelerating pace of innovation. And we regularly cover the latest developments in the discovery and development of new medical concepts in the area of antibodies, vaccines, small molecules, regenerative medicine and nanomedicines. MedNous combines the English word for medicine with the Greek word for intellect. And those with nous are readers of our publication. Visit our website: www.mednous.com

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    Crowne Plaza Berlin - Potsdamer Platz

    Hallesche Str. 10
    Berlin 10963
    Germany

    Crowne Plaza Berlin - Potsdamer Platz

    The Crowne Plaza Berlin - Potsdamer Platz offers Berlin Zeitgeist and a big city feeling. The listed building of the former Postpalais exudes the high-class elegance reminiscent of the Bohemian circles of the 1930s. Broad corridors with natural stone floors, high ceilings, wood panelling and an open fire in the lobby convey glamorous Berlin city life. Art Déco elements and a well thought out design ensure freshness and modernity. Generous rooms and suites, an elegant restaurant with a show kitchen and the stylish THE POST Bar take the Berlin hotel scene to a new level.

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    WHAT IS CPD?

    CPD stands for Continuing Professional Development’. It is essentially a philosophy, which maintains that in order to be effective, learning should be organised and structured. The most common definition is:

    ‘A commitment to structured skills and knowledge enhancement for Personal or Professional competence’

    CPD is a common requirement of individual membership with professional bodies and Institutes. Increasingly, employers also expect their staff to undertake regular CPD activities.

    Undertaken over a period of time, CPD ensures that educational qualifications do not become obsolete, and allows for best practice and professional standards to be upheld.

    CPD can be undertaken through a variety of learning activities including instructor led training courses, seminars and conferences, e:learning modules or structured reading.

    CPD AND PROFESSIONAL INSTITUTES

    There are approximately 470 institutes in the UK across all industry sectors, with a collective membership of circa 4 million professionals, and they all expect their members to undertake CPD.

    For some institutes undertaking CPD is mandatory e.g. accountancy and law, and linked to a licence to practice, for others it’s obligatory. By ensuring that their members undertake CPD, the professional bodies seek to ensure that professional standards, legislative awareness and ethical practices are maintained.

    CPD Schemes often run over the period of a year and the institutes generally provide online tools for their members to record and reflect on their CPD activities.

    TYPICAL CPD SCHEMES AND RECORDING OF CPD (CPD points and hours)

    Professional bodies and Institutes CPD schemes are either structured as ‘Input’ or ‘Output’ based.

    ‘Input’ based schemes list a precise number of CPD hours that individuals must achieve within a given time period. These schemes can also use different ‘currencies’ such as points, merits, units or credits, where an individual must accumulate the number required. These currencies are usually based on time i.e. 1 CPD point = 1 hour of learning.

    ‘Output’ based schemes are learner centred. They require individuals to set learning goals that align to professional competencies, or personal development objectives. These schemes also list different ways to achieve the learning goals e.g. training courses, seminars or e:learning, which enables an individual to complete their CPD through their preferred mode of learning.

    The majority of Input and Output based schemes actively encourage individuals to seek appropriate CPD activities independently.

    As a formal provider of CPD certified activities, SAE Media Group can provide an indication of the learning benefit gained and the typical completion. However, it is ultimately the responsibility of the delegate to evaluate their learning, and record it correctly in line with their professional body’s or employers requirements.

    GLOBAL CPD

    Increasingly, international and emerging markets are ‘professionalising’ their workforces and looking to the UK to benchmark educational standards. The undertaking of CPD is now increasingly expected of any individual employed within today’s global marketplace.

    CPD Certificates

    We can provide a certificate for all our accredited events. To request a CPD certificate for a conference , workshop, master classes you have attended please email events@saemediagroup.com

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    Opening Hours: 9.00 - 17.30 (local time)
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