- 14th - 15th November 2024
- Boston, MA, USA
22nd Orphan Drugs & Rare Diseases Global Congress 2024 Americas
Enhance Information Sharing, Improve Collaboration to Increase Drug Approval for Rare Diseases
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Dear Colleagues,
Facilitate Live is again proud to present our Orphan Drugs and Rare Diseases
Global Congress 2024 Americas. It’s the 20th in the series of our Flagship Orphan Drugs &
Rare Diseases event. This congress will provide you with a comprehensive overview of the
critical issues shaping the future of Orphan Drugs and Rare Diseases.
In the United States, 1 in 10,000 people, or around 30 million Americans are affected by more
than 10,000 known rare diseases. Rare disease are classified as any disease which affects less
than 200,000 Americans. Most rare diseases are life-threatening and many does not have
treatment. Half of the sufferers are children and 30 percent of them will die before the age of 5,
due to lack of available treatment. The complex biology and lack of understanding of the
natural history of rare diseases makes drug, biologic, and device development in rare diseases
very challenging.
Accurate diagnosis and treatment for these diseases has also been teeming with regulatory and
logistical barriers. There is an urgent need for a regulatory framework that encourages safe
therapeutic innovations and helps ensure their timely access to patients.
According to reports (Fortune Business Insights), the global Orphan Drugs Market size is
projected to reach USD 368.43 billion in 2030, at a CAGR of 11.6% during the forecast period,
2023-2030. Biological Orphan drug which has been use in treating rare diseases and in oncology
therapy area possesses a substantially higher share in the global market as new cancer-related
disease emerges and series of FDA approvals will result in increased commercialization of these
drugs. The growing demand for immunomodulators will also contribute to the orphan drugs
market growth. Increasing R&D initiatives and investments by prominent players and patient
advocacy involvement have resulted in major pharmaceutical breakthroughs, and the
development of blockbuster drugs for the treatment of several rare diseases will create
opportunities and contribute exponentially to the overall market growth.
The two-day Congress aims to offer insights into how to improve the diagnostic process and
access to treatments. Strategies and best practices, innovations, technologies and concepts that can promote rare diseases product development will be discussed in an interactive, thought-
provoking and uplifting manner.
We look forward to welcoming you to the congress!
Jocelyn is Conference Director at Paradigm Global Events (PGE). She has over 15+ years experience in organizing pharma-industry conferences and has been at the helm of every PGE conference since 2013. Jocelyn is based in London, UK.
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WHO SHOULD ATTEND?
This congress is specially created for valued stakeholders in the Rare Disease community:
Presidents, Heads/Chiefs, VPs, Directors, and Managers in the area of:
Senior Vice President, General Manager North America
President, Research & Development
Head of Patient Advocacy
Chief Strategy Officer
Founder and CEO
Chief Scientific Officer & Co-Founder
Vice President
President & GM, North America
Co-founder
Co-Founder/President- Stronger Than Sarcoidosis
Chief Executive Officer
CEO
Sr Research Analyst
Executive Director
Founder
CEO
Head, Business Development and Patient Advocacy
Pulse infoframe Inc., Co- founder, CATS Foundation
Head, Business Development and Patient Advocacy
Content Rich Program Agenda! Featuring Keynote Presentations, Stream Sessions, Panel, and Round Table Discussions.
Moderator:
Panelist:
Erica Barnes, Executive Director of the Minnesota Rare Disease Advisory Council
Bennett M. Smith, SVP, GM, North America, Orchard Therapeutics
Dr. Femida Gwadry-Sridhar, Founder and CEO, Pulse infoframe Inc.
Mohamed H. Ladha, President & GM, North America, RECORDATI RARE DISEASES
Moderator:
Alison Schecter, Chief Executive Officer, Molecules to Medicine Advisory Group
Panellist:
David Young, President, Research & Development, Processa Pharmaceuticals, Inc
Shane Hegarty, Chief Scientific Officer & Co-Founder, AXONIS Therapeutics, Inc.
Sanjeev Luther, President & CEO, Cornerstone Pharmaceuticals
Amanda Moore, CEO, Angelman Syndrome Foundation
Christian Rubio, Executive Director, EverythingALS
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Jennifer McNary, Founder, One Rare
Tricha Shivas, Chief of Staff and Strategy, Foundation for Sarcoidosis Research
Joslyn Crowe, Vice President, International Niemann-Pick Disease Alliance (INPDA)
Stuart Siedman, Global Head Patient Advocacy, Rare Diseases, Chiesi
Daniel Chung, Chief Medical Officer, SparingVision
Chisa Nosamiefan, Co-founder, The Labalaba Foundation for Lupus
Luke Rosen, CEO, Rescue7, CEO, KIF1A.ORG
Frank Rivera, Co-Founder/President, Stronger Than Sarcoidosis
Moderator:
Panelist:
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