Developments Related to Orphan Drug Products

Abstract

Historical and current initiatives in orphan product development are reviewed. FDA defines an orphan product as one that is potentially useful but is inadequately tested or unavailable because of limited commercial interest. From 1970-79,24% of 174 new chemicals approved by FDA were drugs of limited commercial value. The FDA Bureau of Drugs convened two interagency task forces in the 1970s to explore problems in developing orphan products. Recent initiatives in the private sector, including the PMA Commission on Drugs for Rare Diseases the Generic Pharmaceutical Industry Association's Institute for Orphan Drugs are described Commitments from manufacturers to develop 14 orphan products have been received by FDA since May 1982. Current activities within the public sector are described, including the responsibilities of the DHHS Orphan Products Board and the FDA Orphan Products Development Office. Provisions of the Orphan Drug Act passed on January 4,1983, that provide assistance to researchers and financial incentives to manufacturers are outlined. Current initiatives in the private and public sectors indicate a willingness to facilitate the development and availability of drugs for rare diseases.