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Orphan Drugs and Rare Diseases

October 21, 2014 - October 22, 2014

SMi’s 3rd Annual Orphan Drugs and Rare Diseases Conference will be held October 21-22, 2014 in London, United Kingdom.

The Orphan Drugs market is set to rise as a result of pharmaceutical companies increasingly looking to orphan drugs as an essential revenue stream. As of January 2014, the European Medicines Agency (EMA) lifted the restriction on providing fee reductions to large companies developing new orphan drugs and the Early Access to Medicines Scheme was introduced. Key implementation of this will be a factor into further commercialization and developments.

With collaboration being a key topic of discussion, the conference will enable all those involved to discuss Market Access, Pricing and Reimbursement, Patient Engagement and Recruitment, with an insight into Regulatory Requirements.

SMi are providing a platform for all stakeholders, inclusive of small biotechs, pharmaceutical companies, patient advocacy organizations, researchers and regulators to share perspectives and expertise to drive the field of Orphan Drugs and Rare Diseases forward.

Featured speakers at the event include:

  • Bertram Häussler, Chairman of the Board of Management, IGES Institut
  • Camille Metais, Associate Director, Regulatory Affairs Manager, Alexion Pharmaceuticals Inc.
  • Christopher Hart, Information Practice Leader, AstraZeneca
  • Dario DiSimone, Sr. Director, Head of Rare Disease Europe, Pfizer
  • Didier Caizergues, Director, Department of International Regulatory Affairs, GENETHON
  • Dominic Nutt, Director of Communications, M&C Saatchi
  • Emily Crossley, Patient Representative, Duchene Children’s Trust
  • Gunter Harms, Market Access & Public Affairs Director, Shire Pharmaceuticals
  • Ian Hudson, CEO, MHRA
  • Josie Godfrey, Associate Director, Highly Specialized Technologies, NICE
  • Professor Sir Peter Lachmann, Emeritus Professor of Immunology, University of Cambridge
  • Tim Miller, President and CEO, Abeona Therapeutics
  • Tony Hall, Co-Founder, Findacure Foundation

Please visit SMI’s Orphan Drugs and Rare Diseases website for more information and to register.

Global Biodefense is an official media partner of SMi’s Orphan Drugs and Rare Diseases 2014 conference.


October 21, 2014
October 22, 2014
Event Tags:


SMi Group
+44 (0) 20 7827 6000
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Holiday Inn Regents Park
Carburton Street
London, W1W 5EE United Kingdom
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+44 871 942 9111
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