
[Federal Register Volume 79, Number 221 (Monday, November 17, 2014)]
[Notices]
[Pages 68451-68454]
From the Federal Register Online via the Government Printing Office [www.gpo.gov]
[FR Doc No: 2014-27022]


-----------------------------------------------------------------------

DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2014-D-1461]


Rare Pediatric Disease Priority Review Vouchers, Draft Guidance 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

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availability of a draft guidance for industry entitled ``Rare Pediatric 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draft guidance before it begins work on the final version of the 
guidance, submit either electronic or written comments on the draft 
guidance by January 16, 2015.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comments concerning the collection of information proposed in the draft 
guidance by January 16, 2015.

ADDRESSES: Submit written requests for single copies of the draft 
guidance to the Office of Communications, Division of Drug Information, 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,

[[Page 68452]]

Food 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.

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.

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

[[Page 68453]]

about 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
----------------------------------------------------------------------------------------------------------------
                                                     Number of
      Description of burden          Number of     responses per   Total annual   Average burden    Total hours
                                    respondents     respondent       responses     per response
----------------------------------------------------------------------------------------------------------------
Rare pediatric disease                        30               1              30              75           2,250
 designation request............
Rare pediatric disease priority               20               1              20              40             800
 review voucher request.........
Notification of intent to use a                3               1               3               8              24
 voucher........................
Transfer notification...........               2               1               2               8              16
Post-approval report............               2               1               2              20              40
                                 -------------------------------------------------------------------------------
    Total burden hours..........  ..............  ..............  ..............  ..............           3,130
----------------------------------------------------------------------------------------------------------------
\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.

[[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.

    Dated: November 10, 2014.
Leslie Kux,
Assistant Commissioner for Policy.
[FR Doc. 2014-27022 Filed 11-14-14; 8:45 am]
BILLING CODE 4164-01-P


