8th Orphan Drugs & Rare Diseases
2018 Americas East Coast
25th – 26th September 2018, Hyatt Regency Boston, Boston, MA, USA
Key Industry Speakers:
Lori Shafner, VP, Global Development Team Leader, Alexion Pharmaceuticals, Inc.
Alison Schecter, Global Rare Diseases, Clinical Dev’t and Strategy, Sanofi Genzyme
Tim Miller, Co-Founder, President , Chief Scientific Officer, Abeona Therapeutics Inc.
Kei Kishimoto, Chief Scientific Officer, Selecta Biosciences
Robert Hollowell, VP, Head, Corporate Strategy and Commercial Assessment, Shire
Philip J. Brooks, Program Director, Division of Clinical Innovation , NCATS, NIH
Arndt Rolfs, CEO, Centogene AG – The Rare Disease Company
Brian Bronk, Head of External Innovation, Rare Diseases, Global BD & Licensing, Sanofi
Alvin Shih, Chief Executive Officer, Enzyvant Therapeutics
Matthias P. Schönermark,Managing Director, SKC
Ken Kengatharan, CEO, Auxesia Orion and Managing Partner, Atheneos Ventures
Radhika Tripuraneni, Vice President, Medical Affairs, Prothena Corporation Plc
Anthony J Arleth,Managing Director, Pennside Partners Ltd
Leslie Leahy, VP, Medical Sciences, Ovid Therapeutics
Luke Rosen, Patient Engagement, Ovid Therapeutics
Mike Page, Executive Director, Global Regulatory Affairs , Alexion Pharmaceuticals
Michael A. Swit, Esq., FDA Legal Counsel, Law Offices of Michael A. Swit
David Hogben, Head of Multichannel, Complete HealthVizion
Jodie Gillon, Global Medical Lead, Patient Engagement Rare Diseases, Pfizer Inc.
Khrystal K. Davis, CEO and Patient Advocate, Zebra Leaf Publishing
Robert (Bob) Ward, Chairman, Chief Executive Officer, Eloxx Pharmaceuticals
Tracy Zervakis, President , Patient for Clinical Research , PaCe Member, AstraZeneca
Barry Ticho, Chief Medical Officer, Stoke Therapeutics, Inc.
Carolina Alarco, President, Global Marketing & International Markets , Aegerion
Richard A. Basile, Co Founder and CEO, BioPontis Alliance for Rare Diseases
Jodi Wolff, Head of Patient Advocacy, U.S.,Santhera Pharmaceuticals
Isabelle Lousada, CEO & President, Amyloidosis Research Consortium
Ray Takigiku, Co-Founder, President and CEO, Bexion Pharmaceuticals, Inc.
Dear Colleagues,
Paradigm Global Events is again proud to present our Orphan Drugs and Rare Diseases 2018 Americas East Coast. It’s the 8th in the series of our Flagship tri-annual Orphan Drugs and Rare Diseases event, this congress will provide you with a comprehensive overview of the critical issues shaping the future of Orphan Drugs in the US market. According to a recently published report, Orphan Drugs set to be 21.4% of worldwide prescription sales by 2022. The market for orphan drugs, based on the consensus forecast for the leading 500 pharmaceutical and biotechnology companies, will grow by 11.1% per year (CAGR) between 2017 and 2022 to $209bn. The growth of the orphan drug market is more than double that of the overall prescription drug market, which is set to grow by 5.3% over the period 2017-2022. Orphan drugs are set to account for 21.4% of global prescription sales in 2022, excluding generics, up from 6% in 2000. In 2016 orphan drug sales increased 12.2% to $114bn vs. 2015, while non-orphan drug sales increased by 2.4% to $578bn. (Evaluate Pharma).
The growth of the global orphan drugs market is increasing at a rapid pace due to the growing rare diseases. With further growth anticipated to meet the high unmet demand for more efficacious drugs with very little side effects. Although, the number of people affected by rare diseases are considerably low, the return of investment is high due to the high cost of orphan drugs. Global collaboration is also likely to fuel growth. FDA recognizes the significance of orphan drugs in the treatment of rare debilitating, life-threatening diseases therefore supporting stakeholders to promote research and development in this area.
However, some factors such as high initial investment that leads to higher per patient treatment cost, reimbursement uncertainties and high cost of drug development are hindering the market. North America registered significant growth for the market during the forecast period due to rising healthcare spending, constructive government initiatives, growing occurrence of chronic diseases and small timeline required for orphan drug development. Orphan Drugs & Rare Diseases 2018 Americas 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 organizations, 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 commercialization, policies, reimbursement, pricing and more. We look forward to welcoming you at the congress!
Sincerely yours,
Jocelyn Raguindin
Conference Director
Paradigm Global Events
By attending you will learn:
- What is Orphan Drugs’ role in sustainability of the Pharmaceutical Industry?
- Finding innovative and alternative ways in funding the development of Orphan Drugs
- How commercially viable Is It to Engage in the Development of Orphan Drugs?
- What Do Developers Look for When Looking for an Outsourcing Partner?
- Coming Together in Developing Orphan Drugs and Crossing Borders
- Trends and Deal Structures in Licensing Agreements
- How Can the Developer and the Patient Assist in the Evolution and Development of Orphan Drugs?
- Patient Access: How Can This Continue to Improve?
- What Do Insurance Companies Think About Orphan Drugs? Will They Make Modifications to Their
- Policies to Support Patients with Rare Diseases?
