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Regulatory Update, March 2015

Sunday, March 1, 2015

FDA guidance on developing vaginal Microbicides for Preventing HIV Infection

The FDA has announced the availability of a guidance document titled Vaginal Microbicides: Development for the Prevention of HIV Infection. The purpose of this guidance is to assist sponsors in all phases of development of vaginal microbicides, defined as vaginal drug products that prevent human immunodeficiency virus (HIV) acquisition.

The guidance addresses nonclinical development, early phases of clinical development, phase III trial considerations and safety considerations in vaginal microbicide development, including safety considerations in adolescent and pregnant populations. The guidance also outlines development of combination microbicide products such as drug-drug combinations, drug-device combinations or combination products that include microbicide and are intended for multiple indications. This guidance makes final the draft guidance issued Nov. 23, 2012. The majority of public comments submitted on the draft version were related to clinical trial considerations and nonclinical pharmacology/toxicology issues. This guidance also incorporates FDA responses to the public comments.

Interested persons may submit electronic or written comments on final FDA guidance documents at any time. Submit electronic comments to http://www.regulations.gov or written comments to the Division of Dockets Management (HFA-305), FDA, 5630 Fishers Lane, Room 1061, Rockville, MD 20852. It is only necessary to send one set of written comments. Identify comments with Docket No. FDA-2012-D-1120.

FDA draft guidance on vouchers for priority review of rare pediatric diseases

The FDA has announced availability of a draft guidance document titled Rare Pediatric Disease Priority Review Vouchers. Under the Federal Food, Drug, and Cosmetic Act (the FD&C Act), as amended by the Food and Drug Administration Safety and Innovation Act (FDASIA), the FDA will award priority review vouchers to sponsors of certain rare pediatric disease product applications that meet certain criteria specified in the amendment. Under this amendment, a sponsor who receives approval for a drug or biological product to treat or prevent a rare pediatric disease may, if the statute’s criteria are met, qualify for a voucher which can be used to receive a priority review for a subsequent marketing application for a different product.

The draft guidance is intended to assist developers of rare pediatric disease products in assessing whether their product may be eligible for rare pediatric disease designation and a rare pediatric disease priority review voucher. It also clarifies the process for requesting such designations and vouchers, sponsor responsibilities upon approval of a rare pediatric disease product application and the parameters for using and transferring a rare pediatric disease priority review voucher. These vouchers can be used when submitting future human drug marketing applications that would not otherwise qualify for priority review. They can be sold or transferred for use to another sponsor any number of times before the voucher is used, as long as the sponsor making the transfer has not yet submitted the application. Because a need for products for rare pediatric diseases exists, this program is intended to encourage development of new drug and biological products for prevention and treatment of certain rare pediatric diseases.

The draft guidance defines “rare pediatric disease” as a disease or condition with an entire prevalence of less than 200,000 in the U.S. and with more than 50% of patients living with the disease ages 0 through 18 years. It provides sponsors information on how to calculate and document prevalence in requests for designation. It explains that, in order for an application to qualify for a rare pediatric disease priority review voucher, it must meet several statutory requirements, including being for a human drug that contains no active ingredient (including any ester or salt of the active ingredient) that has been previously approved in any other FDA application as required by various FD&C Act provisions. Key items described in the draft guidance are:

Request for Rare Pediatric Disease Designation. A stakeholder interested in obtaining a rare pediatric disease designation should include information about the drug product and its proposed mechanism of action, a description of the rare pediatric disease for which the drug is being or will be investigated and documentation that the disease or condition for which the drug is proposed is a “rare pediatric disease” as defined in the FD&C Act.

● Request for Rare Pediatric Disease Priority Review Voucher. As described in the draft guidance, the information to be provided in a request for a priority review voucher will depend on whether the sponsor has previously received rare pediatric disease designation. Sponsors that have received rare pediatric disease designation will include the designation letter with the voucher request explaining how the application meets all of the remaining eligibility criteria. Sponsors that have not requested rare pediatric disease designation should include in a voucher request, prevalence estimates at the time of submission with supporting documentation and explain how the application meets all of the remaining eligibility criteria.

Notification of Intent to Use Voucher. The sponsor redeeming a rare pediatric disease voucher must notify the FDA of its intent to submit an application with a priority review voucher at least 90 days before submission of the application, and include the date the sponsor intends to submit the application.

Transfer Notification. Each person to whom a voucher is transferred must notify the FDA of the change of voucher ownership within 30 days after the transfer. This notification should include a letter from the previous owner to the current owner and a letter from the current owner to the previous owner, each acknowledging the transfer. Any sponsor redeeming a voucher should include these transfer letters in the FDA application. A complete record of transfer must be made available to the FDA to redeem a transferred voucher.

Post-Approval Report. The sponsor of an approved rare pediatric disease product application must submit a report to the FDA no later than five years after approval that addresses the following, for each of the first four post-approval years: (1) The estimated population in the U.S. with the rare pediatric disease for which the product was approved (both the entire population and the population ages 0 through 18 years); (2) The estimated demand in the U.S. for the product; and (3) the actual amount of product distributed in the U.S.

Interested persons can comment on draft guidance at any time; submit written or electronic comments on the draft as instructed above and identify comments with Docket No. FDA-2014-D-1461.


The Regulatory Update is excerpted from Research Practitioner, Volume 16, Number 1, January-February 2015.

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