Equitable Access to Rare Disease Therapies Workshop
Wednesday, May 20, 2020 | 8:00 AM – 12:00 PM ET
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.
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
Registration, Breakfast, & Networking
Distinguished Welcoming Address
Panel Discussion: Industry Perspectives
Panel Discussion: Provider Perspectives
Distinguished Keynote Address
Panel Discussion: Patient Perspectives