Discover the Science of Health
Today is the ninth annual Rare Disease Day, an international advocacy day held on the last day of February — a rare day for rare people. Rare Disease Day 2016 recognizes the crucial role that patients play in voicing their needs and instigating change that improves their lives and the lives of their families and caregivers.
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).
