Several thousand different types of rare diseases and disorders exist, with more being discovered each day. As a result, in recent years, the interest for rare diseases has grown throughout the entire medical community. However, the road to an approved orphan drug is a long, challenging and costly one with many challenges on the way including regulatory, financial, sustainability and pricing and reimbursement issues.
Returning for its fourth year and following on from the major success of previous events, we are proud to present the 4th annual
Orphan Drugs and Rare Diseases Conference, 19th – 20th October 2015, London, UK. Through a series of interactive presentations and panel discussions, join us and learn how payers, pharmaceutical, biotech, patient organisations and academia are structuring to participate in and leverage the growing orphan market.
Key topics include:
- Development of ultra-orphan drugs to prevent, diagnose and treat rare diseases
- Creating strategies for overcoming the challenges for reimbursement of orphan and ultra orphan drugs
- Discover how gene therapy is a very real factor in clinical trials
- Creating workable partnerships for future development of drugs
And much more!
Benefits of Attending
This two day networking event will review recent developments in the orphan drug and rare diseases industry. Join us and gain in depth knowledge by:
- Learning about key strategies and collaborations to accelerate rare disease clinical drug development
- Enhance your knowledge on the role of patient organizations in promoting drug development for their disease
- Learn the ins and outs of the changing regional and national regulatory landscape
Plus Two Interactive Half-Day Post-Conference Workshops | Wednesday 21st October 2015
A: Market access to orphan drugs: Controversies; trends and solutionsWorkshop Leader: Colette Hamilton, Managing Director, ATP Market Access
8.30am – 12.30pm
B: The rare disease patient perspective – from regulatory to clinical executionWorkshop Leaders: Christa van Kan, Team Lead Clinical Execution, PSR Orphan Experts
Steve Pinder, PhD Director, Envestia Ltd
Nicolas Sireau, PhD, Chairman and CEO, AKU Society
1.30pm – 5.30pm
Speakers
- Alastair Kent OBE, Director, Director Genetic Alliance UK, Chair of Rare Disease UK (RDUK)
- Anders Waas, Chief Executive Officer, Tikomed
- Christine Lavary, Chief Executive, MPS Society UK
- David Boothe, Global Commercial Leader – GSK Rare Diseases, GSK
- Dr Anne Marquet, Principal Clinical Scientist, Rare Diseases, Roche Pharma Research and Early Development
- Dr Carlos Camozzi, Chief Medical Officer, Orphazyme
- Dr Didier Caizergues, Head of regulatory Affairs Department, International Regulatory Affairs department, GENETHON
- Dr Diego Ardigo, ATMP Project Leader, Corporate Drug Development, R&D, Chiesi Farmaceutici S.p.A.
- Dr Joanna Cox, Head of Business Partnerships, Cranfield University
- Dr Michael Skynner, Head of Rare Disease Alliances, Rare Disease Research Unit, Pfizer
- Dr Nicolas Sireau, Chairman and CEO, AKU Society
- John Maddox, Managing Director, Infusion Pharma Consulting LLC
- Professor Bobby Gasper, Professor of Paediatrics and Immunology, Consultant in Paediatric Immunology, Head, Rare Diseases Theme Head, University College London, Institute of Child Health
- Professor Milan Macek, Head of Department, Department of Biology and Medical Genetics, National Coordination Centre for Rare Diseases, National Center for Cystic Fibrosis, Charles University Prague
- Robert Ryan, President & Chief Executive Officer, Scioderm
- Stephane Demotz, Founder, DORPHAN S.A., Switzerland
- Terence Hoey, CEO, The Ultra Rare Diseases, Disorder & Disabilities Foundation
- Tim Miller, President & CEO, Abeona Therapeutics
Please fill in your name and email to receive the conference agenda of this event.
The agenda is available as PDF under download at the right side of the page.
Venue
The venue is not yet available for this event.
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Venue
Holiday Inn Regents ParkCarburton Street
London
W1W 5EE England
