Section 908 of The Food and Drug Administration Safety and Innovation Act (FDASIA) added Section 529 to the Federal Food, Drug, and Cosmetic Act. Pursuant to this, FDA will award priority review vouchers to sponsors of rare pediatric disease product applications that meet certain criteria. FDA has published a draft guidance - Rare Pediatric Disease Priority Review Vouchers Draft Guidance for Industry. Questions regarding designations under this program should be directed to OOPD at email@example.com or 301-796-8660.
Regardless 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.
When sponsors use a priority review voucher to obtain a priority review of a drug application, they are required to pay a fee in order to do so. The Federal Register notice concerning Fee for using a Rare Disease Priority Review Voucher in fiscal year 2015 program is available. Click here to view the FRN.
Under this new program, a sponsor who receives an approval for a drug or biologic for a “rare pediatric disease” may qualify for a voucher which can be redeemed to receive a priority review of a subsequent marketing application for a different product.
This guidance explains how FDA plans to implement section 908 of FDASIA , including the process by which sponsors who are interested in receiving rare pediatric disease priority review vouchers may first request designation of their drug or biological product (“drug”) as a drug for a “rare pediatric disease.” (While such designation is not required to receive a voucher, requesting this in advance will expedite a sponsor’s future request for a priority review voucher).
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.)
Request for designation should be sent to:
The Office of Orphan Products Development (OOPD)
Food and Drug Administration
10903 New Hampshire Avenue
Silver Spring, MD 20993-0002