The plight of patients with rare diseases is critically undervalued in healthcare. The statistics are frightening: There are 7000 rare diseases in the world that collectively affect 350,000,000 people. One in 11 Americans has a rare disease. Three-quarters of patients with rare diseases are children. Only half of patients receive an accurate diagnosis. The average delay for a patient to receive a diagnosis of a rare disease is 1.5 years. One in four patients with a rare disease waits 4 years for an accurate diagnosis.
There is an urgent need to advocate for the millions of people worldwide living with rare disease. Advocacy, defined as the public advancement of a cause, has many facets, and includes campaigning for more research funds, activism to eliminate discrimination, lobbying governments to improve access to healthcare, and crusading for the needs of patients’ caregivers.
In this first webinar in the 2023 Science/AAAS Fondation Ipsen series on advocacy in rare disease, we examine the advocacy landscape, asking: what does advocacy entail, who are advocates, what organizations are involved with rare disease advocacy, what determines how umbrella organizations allocate resources, and what is effective—examining success stories of when advocacy has worked.
Join our webinars and let’s collectively advocate for people living with rare disease and their caregivers.
Our guets, all recognized experts in Rare Disease
– Durhane Wong-Rieger, Ph.D. (Canadian Organization for Rare Disorders (CORD), Ontario, Canada)
– Michael Manganiello (Pyxis Partners, Washington, DC)
– Marc C. Patterson, M.D. (Mayo Clinic, Rochester, MN)
– Flaminia Macchia, M.A. (Roche, Brussels, Belgium)
– Erika Gebel Berg, Ph.D, our moderator