The FDA regulates all the drug clinical development and approval in the U.S, and provide support and incentive to promote the interest in developing rare pediatric diseases. All the sponsors can apply for rare pediatric disease designation and entitled its associated benefits including, may not limited to, clinical development tax credit, potential waive of the marketing application fee, and 7 years of marketing exclusivity.
Most of all, the sponsor of a human drug for a rare pediatric disease may be eligible for a valuable voucher that can be used to obtain a priority review for a subsequent human drug.
For updated information for incentive for rate pediatric diseases, please check FDA website at
https://www.fda.gov/industry/developing-products-rare-diseases-conditions
Request for Rare Pediatric Disease designation should be sent to:
The Office of Orphan Products Development (OOPD)
Food and Drug Administration
WO32-5295
10903 New Hampshire Avenue
Silver Spring, MD 20993-0002
Or using email orphan@fda.hhs.gov during the Covid-19 pandemic time.