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Pediatrics: BIO Comments on FDA Draft Guidance on Rare Pediatric Priority Review Vouchers

September 30, 2019

Re: Docket No. FDA–2014-D-1461: FDA Draft Guidance, Rare Pediatric Priority Review Vouchers.

Dear Sir/Madam:

The Biotechnology Innovation Organization (BIO) thanks the Food and Drug Administration (FDA or Agency) for the opportunity to submit comments in response to the FDA’s Draft Guidance on Rare Pediatric Disease Priority Review Vouchers.

BIO is the world's largest trade association representing biotechnology companies, academic institutions, state biotechnology centers and related organizations across the United States and in more than 30 other nations. BIO’s members develop medical products and technologies to treat people afflicted with serious diseases, to delay the onset of these diseases, or to prevent them in the first place.

BIO commends the Agency’s efforts to provide guidance on the implementation of Section 908 of the Food and Drug Administration Safety and Innovation Act (FDASIA), the Rare Pediatric Disease Priority Review Voucher (RPD PRV) Program. The Rare Pediatric Disease Priority Review Voucher Program, along with other incentives, is essential for encouraging investment and research into development for therapies to treat pediatric patients with rare diseases.

As FDA works to improve and streamline the implementation of the RPD PRV process, we recommend that the Agency consider a common application for Orphan Designation and the Rare Pediatric Diseases Designation and streamlining the process for transfer and use of the PRV. We believe that our recommendations not only provide clarity regarding some of the elements in the Draft Guidance but also serve to continue to incentivize the development of therapies for rare pediatric diseases, as intended by Congress.

PRVs are earned or purchased by a sponsor to be redeemed, along with a PRV fee, for Priority Review of an application. As such, we recommend that a voucher and fee would only be needed in a circumstance where the application would not receive a Priority Review based on its own merit. To this end, we request that FDA conduct a routine assessment to determine the review category (e.g., Priority or Standard review) for all applications and supplements.

BIO encourages the Agency to consider a common application pathway for its rare pediatric disease and orphan designations. Although there are instances where a product might be eligible for orphan designation but not rare pediatric disease designation (or eligible for rare pediatric disease designation but not orphan designation), there is significant overlap in the two product categories and information require to be submitted to the FDA. Creating a common application path would serve to reduce administrative review burden by creating greater efficiency for FDA while eliminating duplicative applications for industry.

BIO also requests that the FDA provide additional clarity in the Draft Guidance regarding the transfer procedures for the RPD PRV. The Draft Guidance indicates that FDA must be notified within 30 days of a PRV transfer, and that the notification must be submitted with the NDA/BLA using the PRV. However, the guidance should clarify which FDA division/office should be notified within 30 days of the transfer in situations where the transferee has not decided at that time to which NDA/BLA it will be applying the PRV.

The guidance would also be strengthened if the Agency provided examples of diseases, briefly discussing the Agency’s considerations and rationale when deciding on a product’s eligibility (or ineligibility) for the RPD PRV Program. Examples along with an explanation as to why the particular diseases is or is not eligible for the PRV program can help Sponsors determine if their products are adequate for the RPD PRV, and also avoiding an undue burden on the Agency from the review of non-eligible applications.

BIO appreciates this opportunity to submit comments regarding FDA’s Draft Guidance, Rare Pediatric Priority Disease Review Vouchers.  We would be pleased to provide further input or clarification of our comments, as needed.