Therapeutics for Rare and Neglected Diseases

There are more than 6,500 identified rare and neglected diseases, yet only about 250 treatments are available for these conditions. The limited numbers of patients can make gathering information and designing drug studies difficult. As a result, scientists often know little about the symptoms and biology of these conditions. Also, some private companies may find it difficult to justify the cost of developing drugs for such small rare disease markets. The Therapeutics for Rare and Neglected Diseases (TRND) program is designed to combat these challenges. Its mission is to encourage and speed the development of new treatments for diseases with high unmet medical needs. TRND stimulates therapeutic development research collaborations among NIH and academic scientists, nonprofit organizations, and pharmaceutical and biotechnology companies working on rare and neglected illnesses. The program provides expertise and resources, working with research partners to move therapeutics through pre-clinical testing, including plans for clinical trials and submission of an IND application to the Food and Drug Administration. These efforts effectively “de-risk” therapeutic candidates and make them more attractive for adoption by outside business partners.