Challenges in Developing Therapies for Rare Diseases Including Pachyonychia Congenita  Roger L. Kaspar  Journal of Investigative Dermatology Symposium Proceedings  Volume 10, Issue 1, Pages 62-66 (October 2005) DOI: 10.1111/j.1087-0024.2005.10208.x Copyright © 2005 The Society for Investigative Dermatology, Inc Terms and Conditions

Figure 1 Orphan drug approval. Cumulative number of orphan drugs approved from 1979 through 1998 as compared to cumulative number of total drugs approved. The Orphan Drug Act was passed in 1983. Graph reproduced with permission from Lichtenberg (Lichtenberg and Waldfogel, 2003). Journal of Investigative Dermatology Symposium Proceedings 2005 10, 62-66DOI: (10.1111/j.1087-0024.2005.10208.x) Copyright © 2005 The Society for Investigative Dermatology, Inc Terms and Conditions

Figure 2 Flowchart of milestones leading to approval of pachyonychia congenita (PC) therapeutic. New orphan drug development process. Application for orphan drug status (for therapeutics being developed for diseases afflicting fewer than 200,000 patients) can be made at any time prior to new drug application (NDA) application. The Food and Drug Administration (FDA) Office of Orphan Products Development (OOPD) helps facilitate orphan disease product development and encourages interaction between sponsor, FDA and OOPD at various stages of the process including prior to application for orphan drug status. Figure was modified (with permission) from the FDA website (http://www.fda.gov/cder/handbook/develop.htm). Journal of Investigative Dermatology Symposium Proceedings 2005 10, 62-66DOI: (10.1111/j.1087-0024.2005.10208.x) Copyright © 2005 The Society for Investigative Dermatology, Inc Terms and Conditions