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.
Archives for February 2013
Older Brains Get Too Full for New Information
According to new research, learning becomes more difficult as we get older because our brains get too full for new information. This may be due, in part, to finding that with advanced age we get less sleep during the stage in which we don’t dream. Both studies are reported in the prestigious Nature family of journals.
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).
2012: Banner Year for New Drugs
Fueled by new cancer therapeutics, last year the annual new molecular and biological entity approval count from the U.S. Food and Drug Administration (FDA) saw its highest year since 1997. One-third of the novel products approved by the FDA’s Center for Drug Evaluation and Research (CDER) are used to treat cancers of the blood, breast, colon, prostate, skin and thyroid.