Besides referenced texts and journals, prevalence data for many rare diseases can be found on the internet at government and patient support group websites. Copies of all materials documenting how the prevalence estimate was made should be provided in the designation request. If the reference source is from a website, a hard copy of the document should be included as well as the website address. The date each website was accessed should also be provided for all website sources referenced.
A sponsor is expected to make a good faith effort in finding the most recent prevalence data that refers to a United States population. If only old and/or foreign data are available, the sponsor should explain this in the request. If data are old, the sponsor should explain why the data are still pertinent and, if from a foreign source, why data with that country’s population could also be representative of U.S. population.
The sponsor should be reminded that the prevalence estimate must be current to reflect the prevalence at the time of submission of the request for orphan drug designation (21CFR 316.21 (b)). To update this estimate, the sponsor should use U.S. population data available from the U.S. Census Bureau.
The National Cancer Institute’s Surveillance, Epidemiology and End Results (SEER) Program is one recommended resource for determining cancer statistics in the United States.
