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Equitable Access to Rare Disease Therapies Workshop

  • New York, NY United States (map)

Equitable Access to Rare Disease Therapies Workshop

Thursday, May 23, 2019 | 8:00 AM – 4:00 PM EST

About the Workshop

With just 5% of the over 7,000 rare diseases addressed by some form of drug treatment, 95% of rare diseases remain without a therapeutic option. Rare diseases affect 1 in 10 Americans, yet many of these debilitating conditions impact a very small number of patients. The biopharmaceutical industry is delivering tremendous innovation, from disease-modifying medications to curative gene therapies, to address previously unmet medical needs. It is imperative that these innovations be accessible to those who deserve them. This workshop brings together patients, families, patient advocacy groups, biopharmaceutical firms, providers, policymakers, and other NGOs to discuss how to achieve equitable access to care for rare diseases. Topics will include current barriers to care, ensuring earlier access, and drug pricing. Our goal is to empower patients and families who are affected by rare diseases.

Why Attend?

LEARN how rare diseases impact millions of Americans and how new therapies are developed

UNDERSTAND the major barriers to care for rare diseases

DISCUSS the complexities of drug pricing and why drug pricing has become such a crucial issue

FOCUS on how to empower patients and families who are affected by rare diseases

NETWORK with other patients, families, patient advocacy groups, biopharmaceutical firms, providers, policymakers, and NGOs

Distinguished Keynote Address

Dr. Summar.jpeg

Dr. Marhall Summar, MD

Director, Rare Disease Institute at Children's National Health System
Chairman of the Board of National Organization for Rare Disorders

Dr. Summar is well-known for his pioneering work in caring for children diagnosed with rare diseases. He joined Children’s National in 2010 where he leads the Division of Genetics and Metabolism. He founded and Directs the Rare Disease Institute in 2017, the first dedicated home for the clinical care of patients with genetic rare diseases and The National Organization for Rare Disorders first designated Rare Disease Clinical Center of Excellence. Dr. Summar’s laboratory works on both devices and treatments for patients with genetic rare conditions and adapting knowledge from rare diseases to mainstream medicine. His work has resulted in new drugs in FDA clinical trials for patients with congenital heart disease and premature birth. He has over 30 patents and patent applications. His laboratory is best known for its work in the rare diseases affecting nitrogen, ammonia, and amino acid metabolism. Dr. Summar has also organized and led a number of international work groups to develop and publish standards of care and treatment for rare diseases resulting in significant improvements in outcomes. He has built remote/telemedicine programs to reach patients currently without genetic care access. Dr. Summar is board-certified in Pediatrics, Clinical Genetics, and Biochemical Genetics and has been listed with Best Doctor’s in America since 2004. He serves as President of the Board of Directors of the National Organization for Rare Disorders and is the past president of the Society for Inherited Metabolic Disorders. At NORD he is spearheading an effort that has created digital registries for families to collect long-term information about poorly understood diseases. He is very active in newborn screening policy issues and in developing testing and follow-up systems.

Distinguished Speakers

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Dr. Jeff Sherman, MD

Chief Medical Officer at Horizon Pharma

Dr. Sherman serves as Chief Medical Officer and Executive Vice President at Horizon Pharma, and has more than 25 years of pharmaceutical industry experience. He also serves as an Adjunct Assistant Professor of Medicine at the Northwestern University Feinberg School of Medicine, as a member of numerous professional societies, and as a Diplomat of the National Board of Medical Examiners and the American Board of Internal Medicine. A former DIA President and member of the DIA Board of Directors, Dr. Sherman was also the 2008 DIA Annual Meeting Chair, received the DIA Outstanding Service Award, is an inaugural Fellow of DIA, and serves as the DIA liaison to the US Food and Drug Administration’s Clinical Trial Transformation Initiative Steering Committee.





8:00 AM
Registration, Breakfast, & Networking

8:45 AM
Opening Remarks

9:00 AM
Panel Discussion

10:00 AM
Case Study

11:00 AM
Panel Discussion

12:00 PM
Panel Discussion

12:30 PM

1:15 PM
Distinguished Keynote Address

2:15 PM

2:45 PM
Panel Discussion

3:45 PM
Concluding Remarks

4:00 PM
Reception & Networking