[Federal Register Volume 84, Number 146 (Tuesday, July 30, 2019)]
[Notices]
[Pages 36929-36932]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2019-16262]


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

DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

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


Rare Pediatric Disease Pediatric Priority Review Vouchers; Draft 
Guidance for Industry; Availability

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice of availability.

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

SUMMARY: The Food and Drug Administration (FDA or Agency) is announcing 
the availability of a draft guidance for industry entitled ``Rare 
Pediatric Disease Priority Review Vouchers.'' This draft guidance is a 
revision of the guidance of the same title that published in 2014. This 
draft guidance provides information on the rare pediatric disease 
priority review voucher program under the Federal Food, Drug, and 
Cosmetic Act (FD&C Act), under which FDA will award priority review 
vouchers to sponsors of certain rare pediatric disease product 
applications that meet the relevant statutory criteria. These priority 
review vouchers can be used when submitting future human drug marketing 
applications that would not otherwise qualify for priority review. 
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DATES: Submit either electronic or written comments on the draft 
guidance by September 30, 2019 to ensure that the Agency considers your 
comment on this draft guidance before it begins work on the final 
version of the guidance. Submit electronic or written comments on the 
information collection burden by September 30, 2019.

ADDRESSES: You may submit comments on any guidance at any time as 
follows:

Electronic Submissions

    Submit electronic comments in the following way:
     Federal eRulemaking Portal: https://www.regulations.gov. 
Follow the instructions for submitting comments. Comments submitted 
electronically, including attachments, to https://www.regulations.gov 
will be posted to the docket unchanged. Because your comment will be 
made public, you are solely responsible for ensuring that your comment 
does not include any confidential information that you or a third party 
may not wish to be posted, such as medical information, your or anyone 
else's Social Security number, or confidential business information, 
such as a manufacturing process. Please note that if you include your 
name, contact information, or other information that identifies you in 
the body of your comments, that information will be posted on https://www.regulations.gov.
     If you want to submit a comment with confidential 
information that you do not wish to be made available to the public, 
submit the comment as a written/paper submission and in the manner 
detailed (see ``Written/Paper Submissions'' and ``Instructions'').

Written/Paper Submissions

    Submit written/paper submissions as follows:
     Mail/Hand Delivery/Courier (for written/paper 
submissions): Dockets Management Staff (HFA-305), Food and Drug 
Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852.
     For written/paper comments submitted to the Dockets 
Management Staff, FDA will post your comment, as well as any 
attachments, except for information submitted, marked and identified, 
as confidential, if submitted as detailed in ``Instructions.''
    Instructions: All submissions received must include the Docket No. 
FDA-2014-D-1461 for ``Rare Pediatric Disease Priority Review 
Vouchers.'' Received comments will be placed in the docket and, except 
for those submitted as ``Confidential Submissions,'' publicly viewable 
at https://www.regulations.gov or at the Dockets Management Staff 
between 9 a.m. and 4 p.m., Monday through Friday.
     Confidential Submissions--To submit a comment with 
confidential information that you do not wish to be made publicly 
available, submit your comments only as a written/paper submission. You 
should submit two copies total. One copy will include the

[[Page 36930]]

information you claim to be confidential with a heading or cover note 
that states ``THIS DOCUMENT CONTAINS CONFIDENTIAL INFORMATION.'' The 
Agency will review this copy, including the claimed confidential 
information, in its consideration of comments. The second copy, which 
will have the claimed confidential information redacted/blacked out, 
will be available for public viewing and posted on https://www.regulations.gov. Submit both copies to the Dockets Management 
Staff. If you do not wish your name and contact information to be made 
publicly available, you can provide this information on the cover sheet 
and not in the body of your comments and you must identify this 
information as ``confidential.'' Any information marked as 
``confidential'' will not be disclosed except in accordance with 21 CFR 
10.20 and other applicable disclosure law. For more information about 
FDA's posting of comments to public dockets, see 80 FR 56469, September 
18, 2015, or access the information at: https://www.gpo.gov/fdsys/pkg/FR-2015-09-18/pdf/2015-23389.pdf.
    Docket: For access to the docket to read background documents or 
the electronic and written/paper comments received, go to https://www.regulations.gov and insert the docket number, found in brackets in 
the heading of this document, into the ``Search'' box and follow the 
prompts and/or go to the Dockets Management Staff, 5630 Fishers Lane, 
Rm. 1061, Rockville, MD 20852.
    You may submit comments on any guidance at any time (see 21 CFR 
10.115(g)(5)).
    Submit written requests for single copies of the draft guidance to 
the Office of Orphan Products Development, Office of the Commissioner, 
Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 32, Rm. 
5295, Silver Spring, MD 20993-0002; Office of Communication, Outreach 
and Development, Center for Biologics Evaluation and Research, Food and 
Drug Administration, 10903 New Hampshire Ave., Bldg. 71, Rm. 3128, 
Silver Spring, MD 20993-0002; Division of Drug Information, Center for 
Drug Evaluation and Research, Food and Drug Administration, 10001 New 
Hampshire Ave., Hillandale Building, 4th Floor, Silver Spring, MD 
20993-0002; or Office of Pediatric Therapeutics, Office of the 
Commissioner, Food and Drug Administration, 10903 New Hampshire Ave., 
Bldg. 32, Rm. 5126, Silver Spring, MD 20993-0002. Send one self-
addressed adhesive label to assist that office in processing your 
requests. See the SUPPLEMENTARY INFORMATION section for electronic 
access to the draft guidance document.

FOR FURTHER INFORMATION CONTACT: Aaron Friedman, Office of Orphan 
Products Development, Food and Drug Administration, 10903 New Hampshire 
Ave., Bldg. 32, Rm. 5209, Silver Spring, MD 20993, 301-796-2989; 
Stephen Ripley, Center for Biologics Evaluation and Research, Food and 
Drug Administration, 10903 New Hampshire Ave., Bldg. 71, Rm. 7301, 
Silver Spring, MD 20993-0002, 240-402-7911; Althea Cuff, Center for 
Drug Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg. 22, Rm. 6484, Silver Spring, MD 20993-0002, 301-
796-4061; or Terrie Crescenzi, Office of Pediatric Therapeutics, Food 
and Drug Administration, 10903 New Hampshire Ave., Bldg. 32, Rm. 5126, 
Silver Spring, MD 20993, 301-796-8646.

SUPPLEMENTARY INFORMATION: 

I. Background

    FDA is announcing the availability of a draft guidance for industry 
entitled ``Rare Pediatric Disease Priority Review Vouchers.'' This 
draft guidance provides information on implementation of section 529 of 
the FD&C Act (21 U.S.C. 360ff) regarding the awarding of priority 
review vouchers to sponsors of certain rare pediatric disease product 
applications. Under section 529 of the FD&C Act, 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 statutory 
criteria are met, qualify for a voucher that can be used to receive a 
priority review for a subsequent marketing application for a different 
product.
    This draft guidance is a revision of the draft guidance of the same 
title that published November 17, 2014 (79 FR 68451). The revisions 
address updates to the statutory provision on rare pediatric disease 
priority review vouchers made by the Advancing Hope Act of 2016 (Pub. 
L. 114-229) and the 21st Century Cures Act (Pub. L. 114-255), including 
changes made to the definition of rare pediatric disease. When final, 
this draft guidance will provide FDA's thinking regarding the new 
definition of rare pediatric disease and explain the new statutory 
requirement to request a rare pediatric disease priority review 
voucher. This draft guidance also includes revisions based on FDA's 
experience with implementing the rare pediatric disease priority review 
voucher program, including voucher request procedures.
    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, describes the information to 
include in the designation request and the voucher request, and 
describes sponsor responsibilities upon approval of a rare pediatric 
disease product application. Additionally, it describes how FDA will 
respond to requests for rare pediatric disease designation and 
vouchers.
    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 current thinking of FDA on ``Rare 
Pediatric Disease Priority Review Vouchers.'' It does not establish any 
rights for any person and is not binding on FDA or the public. You can 
use an alternative approach if it satisfies the requirements of the 
applicable statutes and regulations. This guidance is not subject to 
Executive Order 12866.

II. Paperwork Reduction Act of 1995

    Under the Paperwork Reduction Act of 1995 (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 comments on these topics: (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

[[Page 36931]]

collection of information on respondents, including through the use of 
automated collection techniques, when appropriate, and other forms of 
information technology.
    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.
    This draft guidance also refers to previously approved collections 
of information found in FDA regulations and guidance. The collections 
of information in 21 CFR part 314 have been approved under OMB control 
number 0910-0001, the collections of information in 21 CFR part 601 
have been approved under OMB control number 0910-0338, the collections 
of information in 21 CFR part 316 have been approved under OMB control 
number 0910-0167, and the collections of information in the guidance 
for industry entitled ``Expedited Programs for Serious Conditions--
Drugs and Biologics'' have been approved under OMB control number 0910-
0765.
    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 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 and its proposed mechanism of action, a description of the rare 
pediatric disease for which the drug is being or will be investigated, 
whether or not the sponsor is requesting orphan-drug designation or 
fast track designation at the same time, 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 
(including evidence supporting whether the serious or life-threatening 
manifestations of the disease or condition primarily affect children or 
adults).
    FDA estimates that annually a total of approximately 51 respondents 
will complete one rare pediatric disease designation request as 
described in question 9 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 requested rare pediatric disease 
designation. Sponsors who have requested rare pediatric disease 
designation should include the latest designation correspondence from 
FDA (e.g., designation letter, deficiency letter, etc.) with the 
voucher request. Sponsors who have not requested rare pediatric disease 
designation should include in a voucher request prevalence estimates as 
of the time of new drug application/biologics license application 
submission, with supporting documentation. All sponsors requesting a 
voucher should explain how the application meets each of the 
eligibility criteria described in question 2 of the draft guidance.
    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 15 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 (section 
529(b)(4)(B)(i) of the FD&C Act).
    FDA estimates that annually a total of approximately three 
respondents will complete one Notification of Intent to Use a Voucher 
as described in response to question 19 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 two 
respondents will complete Transfer Notifications as described in 
response to question 21 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 4 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 (section 
529(e)(2) of the FD&C Act).
    FDA estimates that annually a total of approximately two 
respondents will complete post-approval reports, as described in 
response to question 7 of the draft guidance. We estimate that each of 
these post-approval reports will take about 20 hours to complete.

[[Page 36932]]

    FDA estimates the annual reporting burden for the draft guidance as 
follows:

                                 Table 1--Estimated Annual Reporting Burden \1\
----------------------------------------------------------------------------------------------------------------
                                                     Number of
            Activity                 Number of     responses per   Total annual   Average burden    Total hours
                                    respondents     respondent       responses     per response
----------------------------------------------------------------------------------------------------------------
Rare pediatric disease                        51               1              51              75           3,825
 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.......................  ..............  ..............  ..............  ..............           4,705
----------------------------------------------------------------------------------------------------------------
\1\ There are no capital costs or operating and maintenance costs associated with this collection of
  information.

III. Electronic Access

    Persons with access to the internet may obtain the draft guidance 
at either https://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/default.htm, https://www.fda.gov/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/default.htm, or 
https://www.regulations.gov.

    Dated: July 24, 2019.
Lowell J. Schiller,
Principal Associate Commissioner for Policy.
[FR Doc. 2019-16262 Filed 7-29-19; 8:45 am]
 BILLING CODE 4164-01-P