The U.S. Food and Drug Administration’s Office of Orphan Products Development (OOPD) has established a valuable resource for drug developers - a database of products that:
- have received orphan status designation (i.e. they’ve been found “promising” for treating a rare disease) AND;
- are already market-approved for the treatment of some other diseases up through June 2010.
The ‘Repurposing’ of Products for Rare Diseases
While the data included in the RDRD is a re-configuration/cross-indexing of already FDA-released information, it offers sponsors a useful tool for finding special opportunities to develop niche therapies that are already well-advanced through development. For example, these drugs have already been subjected to pre-clinical (e.g., pharmacokinetic and toxicologic) testing and are already deemed to be pharmacologically active, effective and safe in some clinical context. The opportunities tabulated in the RDRD represent a far “easier lift” to drug developers than beginning with an untested new therapy compound.
The RDRD is being released as a Beta version of FDA’s cross-tabulation efforts, and does not represent any formal regulatory action. Our due diligence notwithstanding, as the first version of an initiative designed to enable developers to identify new opportunities for making treatments for people with rare diseases, it is subject to the same potential for errors and inaccuracies as any other first version of an initiative.
The RDRD has three sections:
Tables are Excel Files. See below for instructions on downloading viewers and players
Table 1. Orphan-designated products with at least one marketing approval for a common disease indication(XLS - 102KB) Table 2. Orphan-designated products with at least one marketing approval for a rare disease indication(XLS - 172KB) Table 3. Orphan-designated products with marketing approvals for both common and rare disease indication(XLS - 156KB)