The Food and Drug Administration Safety and Innovation Act (FDASIA) added Section 529 to the Federal Food, Drug, and Cosmetic Act. Pursuant to that provision, FDA will award priority review vouchers to sponsors of rare pediatric disease product applications that meet certain criteria. The full text of Section 529 can be found at: http://www.gpo.gov/fdsys/pkg/PLAW-112publ144/pdf/PLAW-112publ144.pdf (see Section 908 of FDASIA on pages 1094-1098 which amends the FD&C Act by adding Section 529). Formal guidance about this program will be published in the future.
Section 529 provides a process by which sponsors who are interested in receiving rare pediatric disease priority review vouchers may request designation of their drug or biological product (“drug”) as a drug for a “rare pediatric disease” (though such designation is not required to receive a voucher). These designation requests should be sent to:
The Office of Orphan Products Development (OOPD)
Food and Drug Administration
10903 New Hampshire Avenue
Silver Spring, MD 20993-0002
Questions regarding designations under this program should be directed to OOPD at email@example.com or 301-796-8660.
Whether or not sponsors receive rare pediatric disease designation, they should include a request for a rare pediatric disease voucher in a cover letter to their NDA/BLA submission, if they are interested in receiving such a voucher and believe they are eligible.
Product-specific questions about rare pediatric disease priority review voucher eligibility should be directed to the appropriate review division within the Center for Drug Evaluation and Research (CDER) or the Center for Biologics Evaluation and Research (CBER). General questions related to the rare pediatric disease priority review voucher program should be directed to OOPD at firstname.lastname@example.org or 301-796-8660.
For information on the rare pediatric disease priority review vouchers issued by FDA, see: Federal Register Notice