[Federal Register Volume 79, Number 221 (Monday, November 17, 2014)]
[Notices]
[Pages 68451-68454]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2014-27022]
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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Rare Pediatric Disease Priority Review Vouchers, Draft Guidance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.
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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availability of a draft guidance for industry entitled ``Rare Pediatric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draft guidance before it begins work on the final version of the
guidance, submit either electronic or written comments on the draft
guidance by January 16, 2015.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comments concerning the collection of information proposed in the draft
guidance by January 16, 2015.
ADDRESSES: Submit written requests for single copies of the draft
guidance to the Office of Communications, Division of Drug Information,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
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Number of
Description of burden Number of responses per Total annual Average burden Total hours
respondents respondent responses per response
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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
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Total burden hours.......... .............. .............. .............. .............. 3,130
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\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