FDA Draft Guidance Clarifies Criteria and Process for Obtaining Rare Pediatric Disease Priority Review Vouchers. November 2. 5, 2.
Cancer promotes pediatric cancer research by identifying structural impediments at key junctures in the research process - new drugs, tissue donation, and access to funding - and developing strategies to address them. THE CREATING HOPE ACT: THE RARE PEDIATRIC DISEASE PRIORITY REVIEW VOUCHER INCENTIVE PROGRAM. Priority review to encourage treatments for rare pediatric diseases (a) Definitions In this section: (1) Priority review. Beginning on the date that the Secretary awards the third rare pediatric disease priority voucher under this section. Rare pediatric disease priority review voucher incentive program. Practices. FDA Regulatory. Printer- Friendly Version. On November 1. 7, 2. Food and Drug Administration (“FDA”) released a draft guidance entitled Rare Pediatric Disease Priority Review Vouchers, which describes an incentive program for the development of drugs intended to treat rare pediatric diseases. Congress established this program more than two years ago when it enacted the Food and Drug Administration Safety and Innovation Act, adding section 5. Federal Food, Drug, and Cosmetic Act. Section 5. 29 provides that the sponsor of a “rare pediatric disease product application,” upon approval of such application, is eligible for a voucher that can be used to obtain priority review for a subsequent new drug application (“NDA”) or biologics license application (“BLA”). Priority review means that an application has an FDA review clock of six months, rather than the standard review clock of ten months. FDA’s draft guidance clarifies key statutory definitions, explains the processes by which sponsors can request a rare pediatric disease designation and a rare pediatric disease priority review voucher, and elaborates on other aspects of the program, including the transferability of vouchers and the relationship between rare pediatric disease designation and orphan drug designation. Definition of a Rare Pediatric Disease Product Application. Under section 5. 29(a)(4), a rare pediatric disease product application is defined as a human drug application (i. NDA or BLA, with limited exceptions) that meets the following criteria: Is for the prevention or treatment of a “rare pediatric disease,” meaning a disease that satisfies the definition of “rare disease or condition” under the Orphan Drug Act and that primarily affects individuals from 0- 1. Is for a drug that contains no active ingredient (including any ester or salt of the active ingredient) previously approved in another application; Is deemed by FDA as eligible for priority review; Is not an abbreviated new drug application or biosimilar application; Relies on clinical data derived from pediatric studies and dosages of the drug intended for the pediatric population; Does not seek approval for an adult indication in the original rare pediatric disease product application; and Is approved after July 9, 2. FDA’s draft guidance clarifies key aspects of these criteria. FDA interprets the definition of “rare pediatric disease” to require that greater than 5. U. S. Additionally, with regard to the criterion that the original application not seek approval for an adult indication, FDA’s draft guidance states that, to preserve voucher eligibility, the applicant cannot seek approval for a different adult indication (i. If the applicant is seeking approval in both pediatric and adult populations for the same rare pediatric disease, the applicant is still eligible for a voucher. FDA explains that it is adopting this liberal interpretation to avoid incentivizing sponsors to exclude adults from clinical trials for rare pediatric diseases or to exclude adult data from original applications. Designation Request and Voucher Request Processes. The draft guidance also outlines procedures for requesting rare pediatric disease designation and rare pediatric disease priority review vouchers, including the information to include in such requests. Under section 5. 29, any request for rare pediatric disease designation must be submitted at the same time as a request for orphan drug designation or fast track designation. The draft guidance provides that FDA will accept designation requests submitted at other times, so long as they are received before FDA has filed the original marketing application for the drug. FDA emphasizes that the rare pediatric disease designation process is entirely voluntary. Even if a sponsor does not request designation, the sponsor may still request, and is still eligible for, a voucher when submitting the original marketing application. Additionally, although voucher requests by sponsors are not technically required under section 5. FDA notes that the submission of such requests will ensure that FDA has all the necessary information to evaluate voucher eligibility. Recent Developments Under the Existing Program. Although relatively new, the rare pediatric disease priority review voucher program may prove to be a powerful incentive. In February 2. 01. FDA awarded the first rare pediatric disease priority review voucher to Bio. Marin Pharmaceutical Inc. Even though FDA’s approval of Vimizim covered both pediatric and adult use for Morquio A syndrome, FDA determined Bio. Marin’s application was eligible for a priority review voucher, consistent with the liberal interpretation of section 5. In July 2. 01. 4, Biomarin announced that it had sold the voucher to Regeneron Ireland for $6. Ropes & Gray will continue to monitor developments in this area. If you have any questions, please contact any member of Ropes & Gray’s FDA regulatory practice or your usual Ropes & Gray Advisor. Under FDA’s commitments as part of the Prescription Drug User Fee Act V, the FDA review clock begins at the end of a 6.
0 Comments
Leave a Reply. |
Details
AuthorWrite something about yourself. No need to be fancy, just an overview. ArchivesCategories |