The routes to orphan drug designation - Our recent experience at the FDA

摘要

The Orphan Drug Act (ODA) of 1983 provides incentives to advance the development of compounds that hold hope for the treatment of rare diseases [1]. Consequently, the United States (US) Food and Drug Administration (FDA)'s regulations for implementing the ODA contain two criteria which every promising compound must meet to be 'designated' as an 'orphan drug'. First, that the sponsor must demonstrate that there is a clear scientific rationale for believing that the drug has promise for treating the rare disease or condition targeted for study. Second, that the disease or condition of interest affects fewer than 200,000 persons living in the US. FDA's regulations further elaborate on the basic requirements for meeting this first criterion of scientific rationale [2]. Based on these regulations, compounds designated in 2009 were examined and categorized according to the method used for designation. The year investigated was chosen for evaluation based on data completeness and recency at the time of gathering and examining data for this editorial. From the inception of ODA to about the time of writing this paper, the 2433 products that have been orphan-designated represent a heterogeneous collection of compounds ranging from those in the very early stages of their development process to those much further along.