Trade Group Study: Hundreds of Rare Disease Drugs in Development

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). A rare disease, also called an orphan disease, is any disease that affects a small percentage of the population. In the United States, the Rare Diseases Act of 2002 defines a rare disease according to prevalence, specifically any disease or condition that affects less than 200,000 individuals in the country (~1 in 1,500 people). The 2002 Act also establishes statutory authorization for the National Institutes of Health (NIH) Office of Rare Diseases Research (ORDR) as a federal entity able to recommend a national research agenda, coordinate research, and provide educational activities for researchers. Recognizing that the high cost of drug developmen...
Source: Highlight HEALTH - Category: Medical Scientists Authors: Source Type: blogs