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.

FDA approved