Orphan Drugs & Rare Diseases Global Congress 2018 Europe

London, UK

In the United States, a rare disease is defined as a condition affecting fewer than 200,000 people.  In EU countries, any disease affecting fewer than 1 in 2,000 people is considered rare. That number may seem small, but it translates into approximately 246 000 people throughout the EU’s 28 member countries. Most patients suffer from even rarer diseases affecting 1 person in 100,000 or more.


There are more than 7,000 rare diseases identified, however, only a fraction of them have approve treatment available. “Orphan drugs” are intended to treat diseases so rare that sponsors are reluctant to develop them under usual marketing conditions. Currently, the regulatory climate is favourable with both FDA and European Medicines Agency, as they offer incentives, tax credits, user waivers and marketing exclusivity. As a result, the number of therapies approved for rare diseases for the past two decades has grown exponentially.


Paradigm Global Events is again proud to present its bi-annual Orphan Drugs & Rare Diseases Global Congress 2018 Europe. The conference will provide a unique platform for the convergence of stakeholders in the orphan drugs industry to discuss and network with top tier government, hospitals, pharmaceuticals, biopharmaceuticals, non-profit organisations, orphan drugs developers as well as regional and local manufacturers. We are putting together an agenda that address the driving macroeconomic factors, policies and issues that will steer the development of orphan drugs globally including commercialisation, policies, reimbursement, pricing and more. We look forward to having you be part of the event!


The two-day Congress will provide an interactive, cutting edge and comprehensive discussion and networking format led by key experts with intimate knowledge in the industry. Gain practical strategies and best practices on challenges, innovations, technologies and concepts surrounding orphan drugs and rare diseases.


Gain Latest Insights On


  • Key Economic Drivers in the Orphan Market: What is Driving the Industry and the Role of Governments?
  • Growing the Market: The Roles of Big Pharmas, Biotechs and Start-Ups
  • Emerging Markets: Challenges and Benefits in Investing in Emerging Markets
  • Europe’s Regulatory Landscape: What Works and What Doesn’t?
  • Medical Devices in the Orphan Realm: Barriers and Opportunities for Medical Devices in Small Populations
  • Strategic Partnerships: What Strategies are available to Ensure Partnership Success
  • Pricing Strategy: How to Price Correctly?
    • Clinical Trials: From Early Drug Development to Patient Recruitment and More


Who will you meet


Presidents, Heads/Chiefs, Directors, VPs and Managers of Research and Development, Regenerative Medicine, External R&D Innovation, Immunology, Clinical Scientist, Cell & Gene, Therapy, Translational Science, Molecular Geneticist, Program Management, Patient Advocacy and Public Affairs, Medical Affairs, Regulatory Affairs, Clinical Field Specialist, Sales and Marketing, Outcomes & Evidence, Commercial Development, Product Specialist, Global Strategic Services, Business Planning and Operations


Featuring Key Industry Experts


Ségolène Aymé, Emeritus Director of Research, INSERM, Founder, Orphanet
Michael Zaiac, Head of Medical Affairs Haematology/Oncology, EMEA, Celgene
Didier Caizergues, Head of regulatory Affairs Department, GENETHON
Otto Schwarz, former COO, Actelion Pharmaceutical Ltd.
Eduardo García-Toledano, Global Health & Rare Diseases, Vice-President & General
Counsel, World Rare Disorders Foundation & Childhood Cancer Foundation
Gilles Vassal, President, European Society for Paediatric Oncology
Ken Kengatharan, CEO, Auxesia Orion and Managing Partner, Atheneos Ventures
Mike Page, Executive Director, Global Regulatory Affairs Portfolio Products, Alexion
Nicolas Sireau, Chair, AKU Society
Richard Thompson, CEO, Findacure
Josie Godfrey, Director, JG Zebra Consulting, Former Associate Director of HST, NICE
Matthew Lumley, Rare Disease Medical Director, Pfizer UK
Raúl Insa, Founder, President and CEO, SOM Biotech
Fred Derosier, Executive Director, Covance Rare Diseases and Orphan Drugs
Carlos R. Camozzi, Orphan Drugs Development and Rare Diseases Expert
Ursula Garczarek, Associate Director of Strategic Consulting, Cytel
Irina Cleemput, Sr. Health Economist, Belgian Health Care Knowledge Centre (KCE)
Jaswinder Singh, Managing Director Americas, WEP Clinical
Amandip Sidhu, EAP Director, WEP Clinical
Leslie Galloway, Chairman, EMIG
Francis Pang, Vice-President of Global Market Access, Amicus
Sjef De Kimpe, CSO and SVP, R&D, Auxesia Orion
Martina Garau, Principal Economist, The Office of Health Economics
Nic Bungay, Director of Campaigns, Care and Information, Muscular Dystrophy UK
Thomas Cawston, Head of Health Policy, Hanover Communications
Farzana Malik, Partner, Cogience
Robin P. Thompson, Director, EU+ Value and Market Access, Biogen
Christian Girard, Senior Advisor, Orphan Drugs Industry Advisory & Intelligence
Anna Bucsics, Project Advisor, Mechanism of Coordinated Access to orphan medicinal products (MoCA)
Simone Boselli, Director of Public Affairs, Eurordis
Dinesh Mistry, Executive Strategist/Global Regulatory Affairs, Covance
Jane Blyth, Scientific Director, Complete HealthVizion
Russell Broome, Account Director, Complete HealthVizion
Maryna Kolochavina, Project Director, Real World & Late Phase, Syneos Health

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Orphan Drugs & Rare Diseases 2017