Quantitative Assessment of Assumptions To Support Extrapolation of Efficacy in Pediatrics; Public Workshop, 26804-26805 [2016-10397]
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Federal Register / Vol. 81, No. 86 / Wednesday, May 4, 2016 / Notices
SUPPLEMENTARY INFORMATION:
asabaliauskas on DSK3SPTVN1PROD with NOTICES
I. Funding Opportunity Description
RFA–FD–16–043
93.103
A. Background
The OOPD was created to identify and
promote the development of orphan
products. Orphan products are drugs,
biologics, medical devices, and medical
foods that are indicated for a rare
disease or condition. The term ‘‘rare
disease or condition’’ is defined in
section 528 of the Federal Food, Drug,
and Cosmetic Act (21 U.S.C. 360ee).
FDA generally considers drugs, devices,
and medical foods potentially eligible
for grants under the OOPD grant
program if they are indicated for a
disease or condition that has a
prevalence, not incidence, of fewer than
200,000 people in the United States.
Diagnostics and vaccines are considered
potentially eligible for such grants only
if the U.S. population to whom they will
be administered is fewer than 200,000
people in the United States per year.
The natural history of a disease is the
natural course of a disease from the time
immediately prior to its inception,
progressing through its pre-symptomatic
phase and different clinical stages to the
point where the disease has ended
without external intervention. Natural
history studies track the course of
disease over time, identifying
demographic, genetic, environmental,
and other variables that correlate with
its development and outcomes in the
absence of treatment. Thorough
understanding of disease natural history
is the foundation upon which a clinical
development program for drugs,
biologics, medical foods or medical
devices is built.
Rare diseases, as defined in the
United States Orphan Drug Act (ODA)
(Pub. L. 97–414), are diseases or
conditions with a prevalence of fewer
than 200,000 persons in the United
States. Though individually rare,
together there are approximately 30
million Americans affected by 7,000
known rare diseases. Unlike common
diseases, there is little existing
knowledge on the natural history of
most rare diseases, which makes natural
history studies of particular importance
for rare diseases product development.
In January 2014, the FDA organized a
Public Workshop on Complex Issues in
Developing Drugs for Rare Diseases.
During the workshop, the lack of natural
history studies was reconfirmed by all
stakeholders (patients, industry,
researchers and the FDA) as one of the
most common and urgent issues that
hinder treatment development for rare
VerDate Sep<11>2014
18:44 May 03, 2016
Jkt 238001
diseases. The need for natural history
studies was also emphasized in the
recently published (August 17, 2015)
draft FDA Guidance for Industry, ‘‘Rare
Diseases: Common Issues in Drug
Development,’’ available at https://
www.fda.gov/downloads/Drugs/
GuidanceComplianceRegulatory
Information/Guidances/
UCM458485.pdf.
B. Research Objectives
The objective of FDA’s Orphan
Products Natural History Grants
Program is to support studies that
characterize the natural history of rare
diseases/conditions, identify genotypic
and phenotypic subpopulations, and
develop and/or validate clinical
outcome measures, biomarkers and/or
companion diagnostics. The ultimate
goal of these natural history studies is
to support clinical development of
products for use in serious rare diseases
or conditions where no current therapy
exists or where the proposed product
will be superior to the existing therapy.
FDA provides grants for natural history
studies that will either assist or
substantially contribute to market
approval of these products. Applicants
must include in the application’s
Background and Significance section
documentation to support that the
estimated prevalence of the orphan
disease or condition in the United States
is less than 200,000 (or in the case of a
vaccine or diagnostic, information to
support that the product will be
administered to fewer than 200,000
people in the United States per year),
and an explanation of how the proposed
study will either help support product
approval or provide essential data
needed for product development.
C. Eligibility Information
The grants are available to any foreign
or domestic, public or private, for-profit
or nonprofit entity (including State and
local units of government). Federal
Agencies may not apply.
II. Award Information/Funds Available
exceed the $150,000 or $400,000 total
cost limit, whichever is applicable.
B. Length of Support
The length of support will depend on
the nature of the study. For those
studies with an expected duration of
more than 1 year, all future years of
noncompetitive continuation of support
will depend on the following factors: (1)
Performance during the preceding year;
(2) compliance with regulatory
requirements as applicable; and (3)
availability of Federal funds.
III. Electronic Application,
Registration, and Submission
Only electronic applications will be
accepted. To submit an electronic
application in response to this FOA,
applicants should first review the full
announcement located at https://
grants.nih.gov/grants/guide. For all
electronically submitted applications,
the following steps are required.
• Step 1: Obtain a Dun and Bradstreet
(DUNS) Number
• Step 2: Register With System for
Award Management (SAM) (formerly
Central Contractor Registration (CCR))
• Step 3: Obtain Username & Password
on Grants.gov
• Step 4: Authorized Organization
Representative (AOR) Authorization
• Step 5: Track AOR Status
• Step 6: Register With Electronic
Research Administration (eRA)
Commons
Steps 1 through 5, in detail, can be
found at https://www07.grants.gov/
applicants/organization_
registration.jsp. Step 6, in detail, can be
found at https://commons.era.nih.gov/
commons/registration/
registrationInstructions.jsp. After you
have followed these steps, submit
electronic applications to: https://
www.grants.gov.
Dated: April 28, 2016.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2016–10398 Filed 5–3–16; 8:45 am]
BILLING CODE 4164–01–P
A. Award Amount
Of the estimated FY 2017 funding
($17.7 million), approximately $2
million will fund 2 to 5 new awards,
subject to availability of funds.
Prospective Natural History Studies are
eligible for grants of up to $400,000 per
year for up to 5 years. Retrospective
Natural History Studies or Surveys are
eligible for grants of up to $150,000 per
year for up to 2 years. Please note that
the dollar limitation will apply to total
costs (direct plus indirect). Budgets for
each year of requested support may not
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DEPARTMENT OF HEALTH AND
HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA–2016–N–0001]
Quantitative Assessment of
Assumptions To Support Extrapolation
of Efficacy in Pediatrics; Public
Workshop
AGENCY:
Food and Drug Administration,
HHS.
E:\FR\FM\04MYN1.SGM
04MYN1
Federal Register / Vol. 81, No. 86 / Wednesday, May 4, 2016 / Notices
ACTION:
Notice of public workshop.
The Food and Drug
Administration (FDA), in collaboration
with the University of Maryland Center
of Excellence in Regulatory Science and
Innovation (CERSI), is announcing a
public workshop entitled ‘‘Quantitative
Assessment of Assumptions to Support
Extrapolation of Efficacy in Pediatrics.’’
The objective of the workshop is to
discuss quantitative and qualitative
approaches for verifying assumptions
pertaining to disease and therapeutic
response similarity between adults and
children. The workshop will also
provide a forum for discussion on the
use of modeling and simulation for
systematic assessment of extrapolation
assumptions.
SUMMARY:
The public workshop will be
held on June 1, 2016, from 8 a.m. to 5
p.m.
ADDRESSES: The public workshop will
be held at FDA’s White Oak Campus,
10903 New Hampshire Ave., Building
31 Conference Center, the Great Room
(Rm. 1503), Silver Spring, MD 20993.
Entrance for the public meeting
participants (non-FDA employees) is
through Building 1 where routine
security check procedures will be
performed. For parking and security
information, please refer to https://
www.fda.gov/AboutFDA/
WorkingatFDA/BuildingsandFacilities/
WhiteOakCampusInformation/
ucm241740.htm.
DATES:
asabaliauskas on DSK3SPTVN1PROD with NOTICES
FOR FURTHER INFORMATION CONTACT:
Audrey Thomas, Office of Regulatory
Science and Innovation, Office of the
Chief Scientist, Food and Drug
Administration, 10903 New Hampshire
Ave., Bldg. 1, Rm. 4220, Silver Spring,
MD 20993–0002, 301–796–3520,
Audrey.Thomas@fda.hhs.gov.
SUPPLEMENTARY INFORMATION: The
purpose of this public workshop is to
provide an opportunity for relevant
stakeholders, including clinicians,
academia, industry, and FDA to discuss
systematic assessment of data needed to
support extrapolation of efficacy in
pediatric product development.
Specifically, the workshop will include:
(1) Presentations on approaches for
assessing disease and therapeutic
response similarity between adults and
pediatrics, and (2) discussion of
alternative approaches to the assessment
of extrapolation assumptions in
pediatric product development,
including the use of clinical trial
simulation and Bayesian approaches.
Examples in partial onset seizures,
inflammatory bowel diseases, and
polyarticular juvenile idiopathic
VerDate Sep<11>2014
18:44 May 03, 2016
Jkt 238001
arthritis will be presented and
discussed.
FDA has verified the Web site
addresses in this document, but FDA is
not responsible for subsequent changes
to the Web site after this document
publishes in the Federal Register.
Agenda: The agenda is located at
www.pharmacy.umaryland.edu/
PedsExtrapolation.
Registration: There is a registration fee
to attend this public workshop in
person. Seats are limited and
registration will be on a first-come, firstserved basis. To register, please
complete registration online at
www.pharmacy.umaryland.edu/
PedsExtrapolation. There will be no
onsite registration. The costs of
registration, to attend in person, for the
different categories of attendees are as
follows:
26805
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA–2013–D–1170]
Chronic Hepatitis C Virus Infection:
Developing Direct-Acting Antiviral
Drugs for Treatment; Draft Guidance
for Industry; Availability
AGENCY:
Food and Drug Administration,
HHS.
ACTION:
Notice of availability.
The Food and Drug
Administration (FDA or Agency) is
announcing the availability of a draft
guidance for industry entitled ‘‘Chronic
Hepatitis C Virus Infection: Developing
Direct-Acting Antiviral Drugs for
Treatment.’’ The purpose of this draft
guidance is to assist sponsors in all
phases of development of direct-acting
antiviral (DAA) drugs for the treatment
of chronic hepatitis C. This draft
Category
Cost
guidance revises the draft guidance of
Industry Representative ....................
$50 the same name that was issued on
October 23, 2013.
Nonprofit Organization and Academic other than University of
DATES: Although you can comment on
Maryland .......................................
50 any guidance at any time (see 21 CFR
University of Maryland, College Park
10.115(g)(5)), to ensure that the Agency
and Baltimore ................................
0 considers your comment on this draft
Federal Government .........................
0 guidance before it begins work on the
final version of the guidance, submit
Streaming Webcast of the Public
either electronic or written comments
Workshop: This public workshop will
on the draft guidance by July 5, 2016.
also be Webcast. There is no registration ADDRESSES: You may submit comments
fee for access to the workshop via the
as follows:
Webcast, but registration is still
Electronic Submissions
required. Information regarding access
Submit electronic comments in the
to the Webcast link is available at
following way:
www.pharmacy.umaryland.edu/
• Federal eRulemaking Portal: https://
PedsExtrapolation. If you have never
www.regulations.gov. Follow the
attended a Connect Pro event before,
instructions for submitting comments.
test your connection at https://
Comments submitted electronically,
collaboration.fda.gov/common/help/en/
including attachments, to https://
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www.regulations.gov will be posted to
quick overview of the Connect Pro
the docket unchanged. Because your
program, visit https://www.adobe.com/
comment will be made public, you are
go/connectpro_overview.
solely responsible for ensuring that your
comment does not include any
Accommodations: Attendees are
confidential information that you or a
responsible for their own hotel
third party may not wish to be posted,
accommodations. If you need special
such as medical information, your or
accommodations while at FDA’s White
anyone else’s Social Security number, or
Oak Campus due to a disability, please
confidential business information, such
contact Shari Solomon at
as a manufacturing process. Please note
Shari.Solomon@fda.hhs.gov at least 7
that if you include your name, contact
days in advance.
information, or other information that
Dated: April 26, 2016.
identifies you in the body of your
Leslie Kux,
comments, that information will be
posted on https://www.regulations.gov.
Associate Commissioner for Policy.
• If you want to submit a comment
[FR Doc. 2016–10397 Filed 5–3–16; 8:45 am]
with confidential information that you
BILLING CODE 4164–01–P
do not wish to be made available to the
public, submit the comment as a
written/paper submission and in the
PO 00000
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SUMMARY:
E:\FR\FM\04MYN1.SGM
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]]>Agencies
[Federal Register Volume 81, Number 86 (Wednesday, May 4, 2016)]
[Notices]
[Pages 26804-26805]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2016-10397]
-----------------------------------------------------------------------
DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2016-N-0001]
Quantitative Assessment of Assumptions To Support Extrapolation
of Efficacy in Pediatrics; Public Workshop
AGENCY: Food and Drug Administration, HHS.
[[Page 26805]]
ACTION: Notice of public workshop.
-----------------------------------------------------------------------
SUMMARY: The Food and Drug Administration (FDA), in collaboration with
the University of Maryland Center of Excellence in Regulatory Science
and Innovation (CERSI), is announcing a public workshop entitled
``Quantitative Assessment of Assumptions to Support Extrapolation of
Efficacy in Pediatrics.'' The objective of the workshop is to discuss
quantitative and qualitative approaches for verifying assumptions
pertaining to disease and therapeutic response similarity between
adults and children. The workshop will also provide a forum for
discussion on the use of modeling and simulation for systematic
assessment of extrapolation assumptions.
DATES: The public workshop will be held on June 1, 2016, from 8 a.m. to
5 p.m.
ADDRESSES: The public workshop will be held at FDA's White Oak Campus,
10903 New Hampshire Ave., Building 31 Conference Center, the Great Room
(Rm. 1503), Silver Spring, MD 20993. Entrance for the public meeting
participants (non-FDA employees) is through Building 1 where routine
security check procedures will be performed. For parking and security
information, please refer to https://www.fda.gov/AboutFDA/WorkingatFDA/BuildingsandFacilities/WhiteOakCampusInformation/ucm241740.htm.
FOR FURTHER INFORMATION CONTACT: Audrey Thomas, Office of Regulatory
Science and Innovation, Office of the Chief Scientist, Food and Drug
Administration, 10903 New Hampshire Ave., Bldg. 1, Rm. 4220, Silver
Spring, MD 20993-0002, 301-796-3520, Audrey.Thomas@fda.hhs.gov.
SUPPLEMENTARY INFORMATION: The purpose of this public workshop is to
provide an opportunity for relevant stakeholders, including clinicians,
academia, industry, and FDA to discuss systematic assessment of data
needed to support extrapolation of efficacy in pediatric product
development. Specifically, the workshop will include: (1) Presentations
on approaches for assessing disease and therapeutic response similarity
between adults and pediatrics, and (2) discussion of alternative
approaches to the assessment of extrapolation assumptions in pediatric
product development, including the use of clinical trial simulation and
Bayesian approaches. Examples in partial onset seizures, inflammatory
bowel diseases, and polyarticular juvenile idiopathic arthritis will be
presented and discussed.
FDA has verified the Web site addresses in this document, but FDA
is not responsible for subsequent changes to the Web site after this
document publishes in the Federal Register.
Agenda: The agenda is located at www.pharmacy.umaryland.edu/PedsExtrapolation.
Registration: There is a registration fee to attend this public
workshop in person. Seats are limited and registration will be on a
first-come, first-served basis. To register, please complete
registration online at www.pharmacy.umaryland.edu/PedsExtrapolation.
There will be no onsite registration. The costs of registration, to
attend in person, for the different categories of attendees are as
follows:
------------------------------------------------------------------------
Category Cost
------------------------------------------------------------------------
Industry Representative........................................ $50
Nonprofit Organization and Academic other than University of 50
Maryland......................................................
University of Maryland, College Park and Baltimore............. 0
Federal Government............................................. 0
------------------------------------------------------------------------
Streaming Webcast of the Public Workshop: This public workshop will
also be Webcast. There is no registration fee for access to the
workshop via the Webcast, but registration is still required.
Information regarding access to the Webcast link is available at
www.pharmacy.umaryland.edu/PedsExtrapolation. If you have never
attended a Connect Pro event before, test your connection at https://collaboration.fda.gov/common/help/en/support/meeting_test.htm. To get a
quick overview of the Connect Pro program, visit https://www.adobe.com/go/connectpro_overview.
Accommodations: Attendees are responsible for their own hotel
accommodations. If you need special accommodations while at FDA's White
Oak Campus due to a disability, please contact Shari Solomon at
Shari.Solomon@fda.hhs.gov at least 7 days in advance.
Dated: April 26, 2016.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2016-10397 Filed 5-3-16; 8:45 am]
BILLING CODE 4164-01-P
