Rare Pediatric Disease Pediatric Priority Review Vouchers; Draft Guidance for Industry; Availability, 36929-36932 [2019-16262]
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Federal Register / Vol. 84, No. 146 / Tuesday, July 30, 2019 / Notices
The confidentiality of client data will
be strictly protected as part of the
project. LIHEAP application client
waivers allow grantees to share
information with OCS and its
contractors.
Respondents: 51 (State Governments
and the District of Columbia)
ANNUAL BURDEN ESTIMATES
Instrument
Action Transmittal LIHEAP–AT–2020–04 Extension of
the FY 2015 RECS LIHEAP Administrative Data Matching to FY 2020 ..................................................................
Estimated Total Annual Burden
Hours: 408.
As LIHEAP is a block grant, there is
varying capacity to collect and report
data among grantees. The estimated
burden hours displayed above are for
the average LIHEAP grantee. All
LIHEAP grantees have existing data
systems to collect, maintain, and
analyze this data to complete annual
reporting requirements.
Comments: The Department
specifically requests comments on (a)
whether the proposed collection of
information is necessary for the proper
performance of the functions of the
agency, including whether the
information shall have practical utility;
(b) the accuracy of the agency’s estimate
of the burden of the proposed collection
of information; (c) the quality, utility,
and clarity of the information to be
collected; and (d) ways to minimize the
burden of the collection of information
on respondents, including through the
use of automated collection techniques
or other forms of information
technology. Consideration will be given
to comments and suggestions submitted
within 60 days of this publication.
Authority: 42 U.S.C. 8629(a).
Mary B. Jones,
ACF/OPRE Certifying Officer.
[FR Doc. 2019–16162 Filed 7–29–19; 8:45 am]
BILLING CODE 4184–80–P
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
jspears on DSK3GMQ082PROD with NOTICES
[Docket No. FDA–2014–D–1461]
Rare Pediatric Disease Pediatric
Priority Review Vouchers; Draft
Guidance for Industry; Availability
Food and Drug Administration,
HHS.
ACTION:
Notice of availability.
VerDate Sep<11>2014
16:42 Jul 29, 2019
Jkt 247001
Total
number of
responses
per
respondent
Average
burden hours
per response
Total
burden
hours
Annual
burden
hours
51
1
24
1,224
408
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announcing the availability of a draft
guidance for industry entitled ‘‘Rare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:
SUMMARY:
Electronic Submissions
Food and Drug Administration
AGENCY:
Total
number of
respondents
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
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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
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Federal Register / Vol. 84, No. 146 / Tuesday, July 30, 2019 / Notices
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/201523389.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 selfaddressed adhesive label to assist that
office in processing your requests. See
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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.
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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
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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 postapproval 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
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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 orphandrug 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.
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36931
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.
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FDA estimates the annual reporting
burden for the draft guidance as follows:
TABLE 1—ESTIMATED ANNUAL REPORTING BURDEN 1
Number of
respondents
Activity
Total annual
responses
Average
burden per
response
Total hours
Rare pediatric disease designation request ........................
Rare pediatric disease priority review voucher request ......
Notification of intent to use a voucher .................................
Transfer notification .............................................................
Post-approval report ............................................................
51
20
3
2
2
1
1
1
1
1
51
20
3
2
2
75
40
8
8
20
3,825
800
24
16
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/Guidance
ComplianceRegulatoryInformation/
Guidances/default.htm, https://
www.fda.gov/BiologicsBloodVaccines/
GuidanceCompliance
RegulatoryInformation/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
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
Office of the Secretary
Request for Information: Ensuring
Patient Access and Effective Drug
Enforcement
Office of the Assistant
Secretary for Planning and Evaluation
(ASPE), HHS.
ACTION: Request for Information.
AGENCY:
This Request for Information
(RFI) seeks comment on ensuring
legitimate access to controlled
substances, including opioids, while
also preventing diversion and abuse, as
well as how federal, state, local, and
tribal entities can collaborate to address
these issues.
DATES: Comments must be received at
one of the addresses provided below, no
later than 5 p.m. on August 29, 2019.
ADDRESSES: Written comments can be
provided by email, fax or U.S. mail.
Email: EPAEDEAreport@hhs.gov.
Fax: (202) 690–5882.
Mail: U.S. Department of Health and
Human Services, Office of the Assistant
Secretary for Planning and Evaluation,
SUMMARY:
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Number of
responses
per respondent
VerDate Sep<11>2014
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Jkt 247001
Office of Science and Data Policy, Attn:
EPAEDEA Report Feedback, 200
Independence Avenue SW, Room 434E,
Washington, DC 20201.
FOR FURTHER INFORMATION CONTACT:
Office of the Assistant Secretary for
Planning and Evaluation, 202–690–
7100.
SUPPLEMENTARY INFORMATION:
I. Background
Section 3 of the Ensuring Patient
Access and Effective Drug Enforcement
Act of 2016 (EPAEDEA), Public Law
114–145, called for the Department of
Health and Human Services, acting
through the Commissioner of Food and
Drugs, the Administrator of the
Substance Abuse and Mental Health
Services Administration, the Director of
the Agency for Healthcare Research and
Quality, and the Director of the Centers
for Disease Control and Prevention, and
in coordination with the Administrator
of the Drug Enforcement Administration
and in consultation with the Secretary
of Defense and the Secretary of Veterans
Affairs, to submit a report to Congress
that identifies:
• Obstacles to legitimate patient
access to controlled substances;
• issues with diversion of controlled
substances;
• how collaboration between Federal,
State, local, and tribal law enforcement
agencies and the pharmaceutical
industry can benefit patients and
prevent diversion and abuse of
controlled substances;
• the availability of medical
education, training opportunities, and
comprehensive clinical guidance for
pain management and opioid
prescribing, and any gaps that should be
addressed;
• beneficial enhancements to State
prescription drug monitoring programs,
including enhancements to require
comprehensive prescriber input and to
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expand access to the programs for
appropriate authorized users;
• steps to improve reporting
requirements so that the public and
Congress have more information
regarding prescription opioids, such as
the volume and formulation of
prescription opioids prescribed
annually, the dispensing of such
prescription opioids, and outliers and
trends within large data sets.
II. Solicitation of Comments
EPAEDEA requires that the report
incorporate feedback and
recommendations from the following:
(1) Patient groups; (2) pharmacies; (3)
drug manufacturers; (4) common or
contract carriers and warehousemen; (5)
hospitals, physicians, and other health
care providers; (6) State attorneys
general; (7) Federal, State, local, and
tribal law enforcement agencies; (8)
health insurance providers and entities
that provide pharmacy benefit
management services on behalf of a
health insurance provider; (9) wholesale
drug distributors; (10) veterinarians; (11)
professional medical societies and
boards; (12) State and local public
health authorities; and (13) health
services research organizations.
This RFI is seeking comment from
these stakeholders on the
aforementioned issue areas to be
covered by the report.
III. Response to Comments
Because of the large number of public
comments we normally receive on
Federal Register documents, we are not
able to acknowledge or respond to them
individually. We will consider all
comments we receive by the date and
time specified in the DATES section of
this preamble.
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]]>Agencies
[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
