Registration Now Live for the 4th Annual Orphan Drugs and Rare Diseases Conference

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SMi’s 4th Annual Orphan Drugs and Rare Diseases conference will be taking place on the 19th – 20th October 2015 at the Holiday Inn Regents Park Hotel in London, UK

Orphan Drugs and Rare Diseases

Returning for its 4th year and following on from the major success of previous events, SMi is proud to announce that registration is now live for the Orphan Drugs and Rare Diseases Conference taking place on 19th – 20th October 2015 in London, UK.

Through a series of interactive presentations and panel discussions, attendees will learn how payers, pharmaceutical, biotech, patient organisations and academia are structuring to participate in and leverage the growing orphan drugs and rare diseases market.

This two day networking event will review recent developments in the orphan drug and rare diseases industry and provide attendees with the opportunity to gain in-depth knowledge by; learning about key strategies and collaborations to accelerate rare disease clinical drug development; enhancing knowledge on the role of patient organisations in promoting drug development for their disease and discover how gene therapy is a very real factor in clinical trials

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

Key speakers in 2015 include:

  • Alastair Kent OBE, Director, Genetic Alliance UK & Chair of Rare Disease UK (RDUK)
  • David Boothe, Global Commercial Leader - GSK Rare Diseases, GSK
  • Dr Anne Marquet, Principal Clinical Scientist, Rare Diseases, Roche Pharma Research and Early Development
  • Dr Didier Caizergues, Head of regulatory Affairs Department, International Regulatory Affairs department, GENETHON
  • Dr Michael Skynner, Head of Rare Disease Alliances, Rare Disease Research Unit, Pfizer
  • Stephane Demotz, Founder, DORPHAN S.A.
  • Tim Miller, President & CEO, Abeona Therapeutics

In addition to the event, there will be two interactive post-conference workshops taking place:

Market Access to Orphan Drugs: Controversies, Trends and Solutions
Workshop Leader: Colette Hamilton, Managing Director, ATP Market Access
8.30am - 12.30pm

The Rare Disease Patient Perspective - From Regulatory to Clinical Execution
Workshop Leader: Christa van Kan, Team Lead Clinical Execution, PSR Orphan Experts
1.30pm - 5.30pm

For those who are interested in attending there is currently a £400 early bird discount before 30th June 2015

For more information or to register visit or contact Shadey Elkiey on +44 (0) 207 827 6118 or selkiey(at)

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Sarah Watson
SMi Group Ltd
+44 207 827 6134
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since: 10/2011
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