Only 12 weeks to go - World Orphan Drug Congress Europe

Dear Victor Norberto, The 9th annual World Orphan Drug Congress, taking place 6th - 8th November, Barcelona, is fast approaching with only 12 weeks left to go. With our strongest agenda thus far incorporating perspectives from all key stakeholders, I am sure this event is a must attend for you as a professional within the Orphan Diseases sector.  Make sure you have information to all of our latest speakers and agenda amendments by downloading the brand new brochure.

VIEW THE 2018 AGENDA So as to summarise, here are some important reason on why you need to book now:   New speakers announced for 2018! Supported by our 17-strong advisory boardmembers, rest assured that your understanding of the industry will be enlighted by over 150 key speakers  New focus on cell & gene therapies; learn about the developmental challenges, new clinical data, manufacturing challenges and reimbursement models from leaders in the industry like Sparks Therapeutics, bluebird bio, UniQure, Lysogene, Yposkesi, REGENXBIO etc Four new pre-congress workshops on P&R for ATMPS, Drug Repurposing, Managed Access and regulatory expertise for clinical success Understand the latest progress in international HTA collborations from theEuropean Commission’s proposal, BENELUXA, MoCA, Visegrad and the Nordics  Keep up-to-date with the latest developments in ERNs – what are they expected to realistically deliver? Don’t miss our keynote addess and plenary panel fromTill Voigtländer, Vice-President of the Board of Members States on ERNs C-level industry insight: why pharma are chasing after the same diseasesfrom Shire, UniQure and Pfizer. Empowered by patients. Hear the incredible progress made from patient intiatives including PARADIGM and HERCULES as well as the journey of how patient groups have contributed to successful drug development and clinical trial outcomes What’s the impact of the OECD recommendations? Hear what their advise and get active feedback from Nathalie Moll, Director General, EFPIA, Yann Le Cam, CEO, EURORDIS and Pamela Gavin, Chief Strategy Office, NORD live on stage Tailor your drug development trials to meet an evolving orphan drug approval landscape – get expert advice on how small trial populations, varying types of evidence, accelerated approval pathway and use of surrogate outcomes will influence your success or failure How are we going to achieve quicker diagnosis for 7000+ diseases and to solve unsolved diagnostic cases? IRDiRC will be questioned on their research, development, regulatory strategy and healthcare system needs to achieve their target Grow your business; partner with government organisations, public and private funders, new exciting biotechs, big pharma, VCs as well as HTA bodies and payers to advance your orphan products  Now even more networking opportunities across 3 days, you will have more time to meet over 500+ multistakeholder representatives through the extended networking breaks, facilitated by our networking platform and evening drinks receptions

Book Your Place By 24th August To Get 10% Off Your Ticket We expect over 450 attendees. This is your best chance of the year to meet and network with hundreds of like-minded peers so book now to save 10%.  See you in Barcelona! Best wishes, Wing-yun Cheung Project Director World Orphan Drug Congress Email: wing-yun.cheung@terrapinn.com Tel: +44 20 7092 1172 Our Advisory Board

Yann Le Cam, Chief Executive Officer, EURORDIS   Dr Alexander Natz, Secretary General, EUCOPE   Dr Bruno Sepodes, Chair of COMP, EMA   Alastair Kent, Former Director, Genetic Alliance UK

Professor Michael Linden, Former VP Gene Therapy & Head, GMI, Pfizer    Wills Hughes-Wilson, Head of Patient Access & Commercial Planning, Mereo BioPharma   Dr Ségolène Aymé, Founder of Orphanet, Emeritus Research Director, INSERM   Sheela Upadhyaya, Associate Director Highly Specialised Technologies, NICE

Dr Carlo Incerti, SVP, Head of Global Medical Affairs, CMO, Genzyme, A Sanofi Company   Dr Mathew Pletcher, Head of Rare Disease Discovery, Roche    Dr M Ken Kengatharan, Managing Director, Atheneos   Rick Thomspon, CEO, Findacure

Dr Birgitte Volck, SVP, Head of R&D Rare Diseases, GSK   Dr Pedro Franco, Former Scientific Administrator, EMA   Anna Bucsics, Project Advisor, MoCA   Josie Godfrey, Former Associate Director, NICE