4th Annual Orphan Drugs and Rare Diseases Conference

Holiday Inn Regents Park Hotel, London, UK (October, 19 - 20, 2015)

SMi Group is delighted to present their 4th annual Orphan Drugs and Rare Diseases Conference, taking place in London, UK, on 19th - 20th October 2015. Through a series of interactive presentations and panel discussions, event attendees will be able to learn how pharmaceutical, biotech, patient organisations and academia are structuring to participate and leverage the growing orphan market.

Key topics include:

• The development of ultra-orphan drugs to prevent, diagnose and treat rare diseases
• Strategies for overcoming the challenges for reimbursement of orphan and ultra-orphan drugs
• How to create workable partnerships for future development of drugs

Benefits of Attending:

• Learn about key strategies and collaborations to accelerate rare disease clinical drug development

• Understand how patients and families are key team players in improving therapy development
• Discover how gene therapy is a very real factor in clinical trials
• Review how big pharma is positioning itself in the emerging orphan drug industry
• Discuss academic and big pharma perspectives on how to overcome the challenges of rare diseases

For further information go to: www.orphandrugs-event.com/gate2bio

Confirmed organisations include: GW Pharmaceuticals, PSR Orphan Experts, PSR Group B.V., Shire Pharmaceuticals Limited, Cello Health Insight - London, Infusion Pharma Consulting LLC, Otsuka Europe Development and Commercialisation

Event Speakers for 2015 include:

• Dr Alastair Kent OBE, Director, Genetic Alliance UK, Chair, Rare Disease UK (RDUK)
• Dr Niclas Sireau, Chairman & CEO, AKU Society
• Dr Tim Miller, President & CEO, Abeona Therapeutics
• Dr Anne Marquet, Principal Clinical Scientist, Rare Diseases, Roche Pharma Research and Early Development
• Josie Godfrey, Associate Director Highly Specialised Technologies and Topic Select, National Institute for Health and Care Excellence
• Christine Lavary, Chief Executive, MPS Society UK
• Dr Carlos R. Camozzi, Chief Medical Officer, Orphazyme
• Michael Skynner, Head of Rare Disease External Alliances, Pfizer
• David Boothe, Global Commercial Leader - GSK Rare Diseases, GSK
• Dr Didier Caizergues, Head of regulatory Affairs Department, International Regulatory Affairs department, GENETHON
• Stephane Demotz, Founder, DORPHAN S.A.
• Tim Miller, President & CEO, Abeona Therapeutics

The event programme also includes two interactive half-day post-conference workshops:

• A: Market access to orphan drugs: Controversies, trends and solutions

Workshop leader: Colette Hamilton, Managing Director, ATP Market Access

• B: The rare disease patient perspective-from regulatory to clinical execution

Workshop leaders: Christa van Kan, Team Lead Clinical Execution, PSR Orphan Experts, Steve Phinder, PhD Director, Envestia Ltd, and Nicolas Sireau, PhD, Chairman and CEO, AKU Society

Sponsorship opportunities available. For more information please contact Alia Malick:

+44 (0) 7827 6168 or amalick@smi-online.co.uk

To register please contact Fateja Begum: +44 (0) 20 7827 6184 or fbegum@smi-online.co.uk

Více: www.orphandrugs-event.com/gate2bio