Today is the sixth annual Rare Disease Day, an international advocacy day held on the last day of February — a rare day for rare people. On this day, millions of patients and their families from more than 60 countries and regions around the world will share their story to promote awareness of the challenges of living with a rare disease and bring widespread recognition of rare diseases as a global health challenge. This year’s slogan is “Rare Disorders without Borders”, which emphasizes the need for international cooperation.
According to a new report released last month by the trade group Pharmaceutical Research and Manufacturers of America (PhRMA), the biopharmaceutical pipeline is innovative and robust, with a high percentage of potential first-in-class medicines (meaning a new treatment where nothing currently exists) targeting diseases with limited treatment options. In addition to identifying medicines in development for conditions and diseases such as septic shock, ovarian cancer, sickle cell disease, and Lou Gehrig’s disease (amyotrophic lateral sclerosis), which haven’t had any new product approvals in the last ten years, the report offers positive news for the rare disease community: one third of the products currently in clinical development have a rare disease designation by the U.S. Food and Drug Administration (FDA).