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Rare Pediatric Disease Priority Review Vouchers, Draft Guidance for Industry; Availability

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AGENCY:

Food and Drug Administration, HHS.

ACTION:

Notice.

SUMMARY:

The Food and Drug Administration (FDA) is announcing the availability of a draft guidance for industry entitled “Rare Pediatric Disease Priority Review Vouchers.” Under the Federal Food, Drug, and Cosmetic Act (the FD&C Act), FDA will award priority review vouchers to sponsors of certain rare pediatric disease product applications that meet the criteria specified in that section. These vouchers can be used when submitting future human drug marketing applications that would not otherwise qualify for priority review. These vouchers 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 there exists a need for products for rare pediatric diseases, this program is intended to encourage development of new drug and biological products for prevention and treatment of certain rare pediatric diseases.

DATES:

Although you can comment on any guidance at any time (see 21 CFR 10.115(g)(5)), to ensure that the Agency considers your comment on this draft guidance before it begins work on the final version of the guidance, submit either electronic or written comments on the draft guidance by January 16, 2015. Submit either electronic or written comments concerning the collection of information proposed in the draft guidance by January 16, 2015.

ADDRESSES:

Submit written requests for single copies of the draft guidance to the Office of Communications, Division of Drug Information, Center for Drug Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 51, Rm. 2201, Silver Spring, MD 20993-0002, or Office of Communication, Outreach, and Development, Center for Biologics Evaluation and Research, Food and Drug Administration, Bldg. 71, Rm. 3128, 10903 New Hampshire Ave., Silver Spring, MD 20993-0002; or Office of Orphan Products Development, Office of Special Medical Programs, Start Printed Page 68452Food and Drug Administration, 10903 New Hampshire Ave., Silver Spring, MD 20993. Send one self-addressed adhesive label to assist the office that will be processing your requests. See the SUPPLEMENTARY INFORMATION section for electronic access to the draft guidance document.

Submit electronic comments on the draft guidance to http://www.regulations.gov. Submit written comments to the Division of Dockets Management (HFA-305), Food and Drug Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852.

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FOR FURTHER INFORMATION CONTACT:

Henry Startzman, Food and Drug Administration, Office of Orphan Products Development, Bldg. 32, Rm. 5295, 10903 New Hampshire Ave., Silver Spring, MD 20993-0002, 301-796-8660.

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SUPPLEMENTARY INFORMATION:

I. Background

FDA is announcing the availability of a draft guidance for industry entitled “Rare Pediatric Disease Priority Review Voucher.”

This draft guidance clarifies FDA's plans to implement section 908 of the Food and Drug Administration Safety and Innovation Act (FDASIA), which added section 529 to the Federal Food Drug and Cosmetic Act (21 U.S.C. 360ff) (the FD&C Act). Under this statutory section, a sponsor who receives an approval for a drug or biological product to treat or prevent a rare pediatric disease (as defined by statute) 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.

The draft guidance provides FDA's interpretation of a variety of terms in the statute. It defines “rare pediatric disease” as a disease or condition with an entire prevalence of less than 200,000 in the United States and with more than 50 percent of patients living with the disease aged 0 through 18 years. It provides sponsors information on how to calculate and document prevalence in their 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 application under section 505(b)(1), 505(b)(2), or 505(j) of the FD&C Act (21 U.S.C. 355(b)(1), 355(b)(2), or 355(j)) or section 351(a) or 351(k) of the Public Health Service Act (42 U.S.C. 262(a) or 42 U.S.C. 262(k).

The draft guidance also outlines for sponsors the procedures for requesting rare pediatric disease designation and rare pediatric disease priority review vouchers and describes the information to include in the designation request and the voucher request. Additionally, it describes how FDA will respond to requests for rare pediatric disease designation and vouchers.

Finally, the draft guidance describes the processes by which a rare pediatric disease priority review voucher is to be awarded, used, and transferred to another sponsor. This draft guidance is being issued consistent with FDA's good guidance practices regulation (21 CFR 10.115). The draft guidance, when finalized, will represent the Agency's current thinking on Rare Pediatric Disease Priority Review Vouchers. It does not create or confer any rights for or on any person and does not operate to bind FDA or the public. An alternative approach may be used if such approach satisfies the requirements of the applicable statutes and regulations.

II. Paperwork Reduction Act of 1995

Under the Paperwork Reduction Act of 1995 (the PRA) (44 U.S.C. 3501-3520), Federal Agencies must obtain approval from the Office of Management and Budget (OMB) for each collection of information they conduct or sponsor. “Collection of information” is defined in 44 U.S.C. 3502(3) and 5 CFR 1320.3(c) and includes Agency requests or requirements that members of the public submit reports, keep records, or provide information to a third party. Section 3506(c)(2)(A) of the PRA (44 U.S.C. 3506(c)(2)(A)) requires Federal Agencies to provide a 60-day notice in the Federal Register concerning each proposed collection of information before submitting the collection to OMB for approval. To comply with this requirement, FDA is publishing notice of the proposed collection of information set forth in this document.

With respect to the following collection of information, FDA invites comment on: (1) Whether the proposed collection of information is necessary for the proper performance of FDA's functions, including whether the information will have practical utility; (2) the accuracy of FDA's estimate of the burden of the proposed collection of information, including the validity of the methodology and assumptions used; (3) ways to enhance the quality, utility, and clarity of the information to be collected; and (4) ways to minimize the burden of the collection on respondents, including through the use of automated collection techniques and other forms of information technology, when appropriate.

Title: Rare Pediatric Disease Priority Review Vouchers, Draft Guidance for Industry.

Description of Respondents: Respondents to this collection of information are sponsors that develop drugs and biological products.

Burden Estimate: This draft guidance on Rare Pediatric Disease Priority Review Vouchers 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.

The draft guidance 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.

FDA has OMB approval under the PRA for the submission of new drug applications (NDAs) and related submissions under 21 CFR part 314 (OMB control number 0910-0001), biologics license applications (BLAs) and related submissions under 21 CFR part 601 (OMB control number 0910-0338), and orphan-drug designation requests and related submissions under 21 CFR part 316 (OMB control number 0910-0167). The draft guidance describes five collections of information that are not currently approved by OMB under the PRA: (1) The request for a rare pediatric disease designation, (2) the request for a rare pediatric disease priority review voucher, (3) the notification of intent to use a voucher, (4) the notification to transfer a voucher, and (5) the post-approval report.

These collections of information will be used by the Agency to issue rare pediatric disease designations and vouchers, prepare for an incoming priority review, and maintain awareness Start Printed Page 68453about which sponsors currently hold vouchers.

A. Request for Rare Pediatric Disease Designation

Under the draft guidance, 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 section 529(a)(3) of the FD&C Act.

FDA estimates that annually a total of approximately 30 respondents will complete one rare pediatric disease designation request as described in question 8 of the draft guidance. FDA estimates that preparing these designation requests will take approximately 75 hours for each designation request. This includes the time that may be needed to respond to FDA actions and requests.

B. Request for Rare Pediatric Disease Priority Review Voucher

As described more fully 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 who 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 who have not requested rare pediatric disease designation should include in a voucher request prevalence estimates as of the time of NDA/BLA submission, with supporting documentation, and explain how the application meets all of the remaining eligibility criteria.

We estimate that annually a total of approximately 20 respondents will complete one rare pediatric disease priority review voucher request as described in response to question 14 of the draft guidance. We estimate that preparing these designation requests will take approximately 40 hours for each rare pediatric disease priority review voucher request. This includes the time that may be needed to respond to FDA actions and requests.

C. Notification of Intent To Use Voucher

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

FDA estimates that annually a total of approximately 3 respondents will complete one Notification of Intent to Use a Voucher as described in response to question 18 of the draft guidance. We estimate that preparing each of these Notifications of Intent to Use a Voucher will take approximately 8 hours.

D. Transfer Notification

Each person to whom a voucher is transferred must notify 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 application submitted to FDA. A complete record of transfer must be made available to FDA to redeem a transferred voucher.

FDA estimates that annually a total of approximately 2 respondents will complete Transfer Notifications as described in response to question 20 of the draft guidance. We estimate that preparing each of these Transfer Notifications will take approximately 8 hours.

E. Post-Approval Report

The sponsor of an approved rare pediatric disease product application must submit a report to FDA no later than 5 years after approval that addresses the following, for each of the first four post-approval years: (1) The estimated population in the United States with the rare pediatric disease for which the product was approved (both the entire population and the population aged 0 through 18 years); (2) The estimated demand in the United States for the product; and (3) the actual amount of product distributed in the United States.

FDA estimates that annually a total of approximately 2 respondents will complete post-approval reports, as described in response to question 6 of the draft guidance. We estimate that each of these post-approval reports will take about 20 hours to complete.

The total estimated annual reporting burdens for the draft guidance are as follows:

Table 1—Estimated Annual Reporting Burden

Description of burdenNumber of respondentsNumber of responses per respondentTotal annual responsesAverage burden per responseTotal hours
Rare pediatric disease designation request30130752,250
Rare pediatric disease priority review voucher request2012040800
Notification of intent to use a voucher313824
Transfer notification212816
Post-approval report2122040
Total burden hours3,130
1 There are no capital costs or operating and maintenance costs associated with this collection of information.

Send comments regarding this burden estimate or suggestions for reducing this burden to: Office of Orphan Products Development, Food and Drug Administration, Bldg. 32, Rm. 5295, 10903 New Hampshire Ave., Silver Spring, MD 20993.

III. Comments

Interested persons may submit either electronic comments regarding this document to http://www.regulations.gov or written comments to the Division of Dockets Management (see ADDRESSES). It is only necessary to send one set of comments. Identify comments with the docket number found in brackets in the heading of this document. Received comments may be seen in the Division of Dockets Management between 9 a.m. and 4 p.m., Monday through Friday, and will be posted to the docket at http://www.regulations.gov. Start Printed Page 68454

IV. Electronic Access

Persons with access to the Internet may obtain the document at: http://www.fda.gov/​Drugs/​GuidanceComplianceRegulatoryInformation/​Guidances/​default.htm, http://www.fda.gov/​BiologicsBloodVaccines/​GuidanceComplianceRegulatoryInformation/​Guidances/​default.htm, http://www.fda.gov/​ForIndustry/​DevelopingProductsforRareDiseasesConditions/​default.htm, or http://www.regulations.gov.

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Dated: November 10, 2014.

Leslie Kux,

Assistant Commissioner for Policy.

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[FR Doc. 2014-27022 Filed 11-14-14; 8:45 am]

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