Drug development for orphan diseases in the context of personalized medicine

摘要: Orphan diseases are that found in less than 200,000 patients the United States, which is cutoff point for number of a drug to be profitable. Because many thousands orphan exist aggregate (about 20 30 million Americans have diseases), these disenfranchised from development by pharmaceutical industry. drugs large part personalized medicine. The often so rare physician may observe only 1 case year or less. So proper treatment encounter between doctor and patient. Academic physician-scientists tried fill this therapy vacuum working on developing drugs. But disincentives involved, include career disincentives, lack funding, multiple areas expertise required. Positive developments formation National Organization Rare Diseases, Drug Act, grant program fund development, Institutes Health Office passage legislation other countries. Progress has increased, but 300 devices approved last 25 years still drop bucket compared with diseases. I believe we must do better. present my own 2 examples positive negative aspects end article giving recommendations how might succeed both more rescuing industry its impending economic collapse.