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Equitable Access to Rare Disease Therapies Workshop

  • Washington, DC United States (map)

Equitable Access to Rare Disease Therapies Workshop

Thursday, May 23, 2019 | 8:00 AM – 12:00 PM ET
2550 M Street, NW | Washington, DC 20037

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.

Organization represented included: Alnylam Pharmaceuticals, American Autoimmune Related Diseases Association, American Telemedicine Association, AmerisourceBergen, AstraZeneca, Barth Syndrome Foundation, Birmingham VA Medical Center, Boston Scientific, Cardinal Health, Daiichi Sankyo, EveryLife Foundation for Rare Diseases, GE Healthcare, Georgetown University School of Medicine, GSK, Haystack Project: Voices of Ultra Rare, Horizon Pharma, Immune Deficiency Foundation, Ipsen, Janssen Pharmaceuticals, Johns Hopkins University School of Medicine, Medicaid Health Plans of America, Medicines for All Institute, MedImmune, National Alliance for Caregiving, National Center for Advancing Translational Sciences, National Human Genome Research Institute, National Infusion Center Association, Novartis, Organization for Rare Diseases India, Osteogenesis Imperfecta Foundation, Partnership to Fight Chronic Disease, Pfizer, Project HOPE, Rare Access Action Project, Regeneron Pharmaceuticals, RWJBarnabas Health, Sanofi, Shire, Takeda, The ALS Association, The Myositis Association, Triple Negative Breast Cancer Foundation, Vitrisa Therapeutics, and WebMD.

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. Marshall 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 Welcoming Address

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Dr. Jeffrey 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.

Closing Remarks


Julius Pryor III

Chair of the Board of Directors at CHI

Julius Pryor helps companies leverage Diversity & Inclusion to accelerate innovation and drive business results. He’s held executive roles at Johnson & Johnson (J&J), Coca-Cola Enterprises (CCE), Russell Athletic, Abbott Labs and Takeda Pharmaceuticals. He was Vice President of Global Diversity at both J&J and Coke. Most recently, Julius served as Head of Innovation, Diversity & Inclusion at biotech leader Genentech. Julius combines a unique vision for the future of D&I, a strong grounding in sales and management, and insights into what works across different industries and sectors. Julius saw the power of diversity to accomplish results during his 26 years of service in the U.S. Navy. He is a U.S. Navy Captain, Surface Warfare Officer and Instructor for the Navy Officer Leadership Development Program. He’s held numerous Navy leadership roles including, Unit Commanding Officer and Fleet Staff Officer. He had the honor of serving on the re-commissioning crew of the historic USS Missouri (BB-63). Julius graduated from Morehouse College and The Williston Northampton School (Easthampton, MA). He sits on the boards of the Andrew Young Center for Global Leadership and the Center for Healthcare Innovation. He is a member of the Omega Psi Phi Fraternity, having pledged at the legendary Psi Chapter at Morehouse College. Julius is the author of Thriving in a Disruptive World: 6 Critical Concepts for Navigating the 21st Century.

Distinguished Speakers and Panelists





8:00 AM
Registration, Breakfast, & Networking

8:30 AM
Introductory Remarks: Joseph Gaspero, CEO & Co-Founder of CHI

8:35 AM
Distinguished Welcoming Address: Dr. Jeffrey Sherman, MD, Chief Medical Officer at Horizon Pharma

8:45 AM
Panel Discussion: Industry Perspectives

9:45 AM
Panel Discussion: Governmental/NGO Perspectives

10:45 AM
Distinguished Keynote Address: Dr. Marshall Summar, MD, Chairman of the Board of National Organization for Rare Disorders

11:15 AM
Panel Discussion: Patient Perspectives

12:00 PM
Closing Remarks: Julius Pryor III, Chair of the Board of Directors at CHI