81 FR 26804 - Quantitative Assessment of Assumptions To Support Extrapolation of Efficacy in Pediatrics; Public Workshop
DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration Federal Register Volume 81, Issue 86 (May 4, 2016)
Food and Drug Administration
Federal Register Volume 81, Issue 86 (May 4, 2016)
| Page Range | 26804-26805 | |
| FR Document | 2016-10397 |
[Federal Register Volume 81, Number 86 (Wednesday, May 4, 2016)] [Notices] [Pages 26804-26805] From the Federal Register Online [www.thefederalregister.org] [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 http://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, [email protected]. 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 http://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 [email protected] 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
![26804 Federal Register / Vol. 81, No. 86 / Wednesday, May 4, 2016 / Notices
SUPPLEMENTARY INFORMATION: diseases. The need for natural history exceed the $150,000 or $400,000 total
studies was also emphasized in the cost limit, whichever is applicable.
I. Funding Opportunity Description
recently published (August 17, 2015)
RFA–FD–16–043 B. Length of Support
draft FDA Guidance for Industry, ‘‘Rare
93.103 Diseases: Common Issues in Drug The length of support will depend on
Development,’’ available at http:// the nature of the study. For those
A. Background
www.fda.gov/downloads/Drugs/ studies with an expected duration of
The OOPD was created to identify and GuidanceComplianceRegulatory more than 1 year, all future years of
promote the development of orphan Information/Guidances/ noncompetitive continuation of support
products. Orphan products are drugs, UCM458485.pdf. will depend on the following factors: (1)
biologics, medical devices, and medical Performance during the preceding year;
foods that are indicated for a rare B. Research Objectives (2) compliance with regulatory
disease or condition. The term ‘‘rare The objective of FDA’s Orphan requirements as applicable; and (3)
disease or condition’’ is defined in Products Natural History Grants availability of Federal funds.
section 528 of the Federal Food, Drug, Program is to support studies that
and Cosmetic Act (21 U.S.C. 360ee). III. Electronic Application,
characterize the natural history of rare
FDA generally considers drugs, devices, Registration, and Submission
diseases/conditions, identify genotypic
and medical foods potentially eligible and phenotypic subpopulations, and Only electronic applications will be
for grants under the OOPD grant develop and/or validate clinical accepted. To submit an electronic
program if they are indicated for a outcome measures, biomarkers and/or application in response to this FOA,
disease or condition that has a companion diagnostics. The ultimate applicants should first review the full
prevalence, not incidence, of fewer than goal of these natural history studies is announcement located at http://
200,000 people in the United States. to support clinical development of grants.nih.gov/grants/guide. For all
Diagnostics and vaccines are considered products for use in serious rare diseases electronically submitted applications,
potentially eligible for such grants only or conditions where no current therapy the following steps are required.
if the U.S. population to whom they will exists or where the proposed product • Step 1: Obtain a Dun and Bradstreet
be administered is fewer than 200,000 will be superior to the existing therapy. (DUNS) Number
people in the United States per year. FDA provides grants for natural history • Step 2: Register With System for
The natural history of a disease is the Award Management (SAM) (formerly
studies that will either assist or
natural course of a disease from the time Central Contractor Registration (CCR))
substantially contribute to market
immediately prior to its inception, • Step 3: Obtain Username & Password
approval of these products. Applicants
progressing through its pre-symptomatic on Grants.gov
must include in the application’s
phase and different clinical stages to the • Step 4: Authorized Organization
Background and Significance section
point where the disease has ended Representative (AOR) Authorization
documentation to support that the
without external intervention. Natural • Step 5: Track AOR Status
estimated prevalence of the orphan
history studies track the course of • Step 6: Register With Electronic
disease or condition in the United States
disease over time, identifying Research Administration (eRA)
is less than 200,000 (or in the case of a
demographic, genetic, environmental, Commons
and other variables that correlate with vaccine or diagnostic, information to
its development and outcomes in the support that the product will be Steps 1 through 5, in detail, can be
absence of treatment. Thorough administered to fewer than 200,000 found at http://www07.grants.gov/
understanding of disease natural history people in the United States per year), applicants/organization_
is the foundation upon which a clinical and an explanation of how the proposed registration.jsp. Step 6, in detail, can be
development program for drugs, study will either help support product found at https://commons.era.nih.gov/
biologics, medical foods or medical approval or provide essential data commons/registration/
devices is built. needed for product development. registrationInstructions.jsp. After you
Rare diseases, as defined in the C. Eligibility Information have followed these steps, submit
United States Orphan Drug Act (ODA) electronic applications to: http://
(Pub. L. 97–414), are diseases or The grants are available to any foreign www.grants.gov.
conditions with a prevalence of fewer or domestic, public or private, for-profit
Dated: April 28, 2016.
than 200,000 persons in the United or nonprofit entity (including State and
local units of government). Federal Leslie Kux,
States. Though individually rare, Associate Commissioner for Policy.
together there are approximately 30 Agencies may not apply.
[FR Doc. 2016–10398 Filed 5–3–16; 8:45 am]
million Americans affected by 7,000 II. Award Information/Funds Available
BILLING CODE 4164–01–P
known rare diseases. Unlike common
diseases, there is little existing A. Award Amount
knowledge on the natural history of Of the estimated FY 2017 funding DEPARTMENT OF HEALTH AND
most rare diseases, which makes natural ($17.7 million), approximately $2 HUMAN SERVICES
history studies of particular importance million will fund 2 to 5 new awards,
for rare diseases product development. subject to availability of funds. Food and Drug Administration
In January 2014, the FDA organized a Prospective Natural History Studies are
asabaliauskas on DSK3SPTVN1PROD with NOTICES
Public Workshop on Complex Issues in eligible for grants of up to $400,000 per [Docket No. FDA–2016–N–0001]
Developing Drugs for Rare Diseases. year for up to 5 years. Retrospective Quantitative Assessment of
During the workshop, the lack of natural Natural History Studies or Surveys are Assumptions To Support Extrapolation
history studies was reconfirmed by all eligible for grants of up to $150,000 per of Efficacy in Pediatrics; Public
stakeholders (patients, industry, year for up to 2 years. Please note that Workshop
researchers and the FDA) as one of the the dollar limitation will apply to total
most common and urgent issues that costs (direct plus indirect). Budgets for AGENCY: Food and Drug Administration,
hinder treatment development for rare each year of requested support may not HHS.
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![Federal Register / Vol. 81, No. 86 / Wednesday, May 4, 2016 / Notices 26805
ACTION: Notice of public workshop. arthritis will be presented and DEPARTMENT OF HEALTH AND
discussed. HUMAN SERVICES
SUMMARY: The Food and Drug
Administration (FDA), in collaboration FDA has verified the Web site
Food and Drug Administration
with the University of Maryland Center addresses in this document, but FDA is
not responsible for subsequent changes [Docket No. FDA–2013–D–1170]
of Excellence in Regulatory Science and
Innovation (CERSI), is announcing a to the Web site after this document
publishes in the Federal Register. Chronic Hepatitis C Virus Infection:
public workshop entitled ‘‘Quantitative Developing Direct-Acting Antiviral
Assessment of Assumptions to Support Agenda: The agenda is located at Drugs for Treatment; Draft Guidance
Extrapolation of Efficacy in Pediatrics.’’ www.pharmacy.umaryland.edu/ for Industry; Availability
The objective of the workshop is to PedsExtrapolation.
discuss quantitative and qualitative AGENCY: Food and Drug Administration,
Registration: There is a registration fee HHS.
approaches for verifying assumptions
to attend this public workshop in ACTION: Notice of availability.
pertaining to disease and therapeutic
person. Seats are limited and
response similarity between adults and
registration will be on a first-come, first- SUMMARY: The Food and Drug
children. The workshop will also
served basis. To register, please Administration (FDA or Agency) is
provide a forum for discussion on the
complete registration online at announcing the availability of a draft
use of modeling and simulation for
www.pharmacy.umaryland.edu/ guidance for industry entitled ‘‘Chronic
systematic assessment of extrapolation
PedsExtrapolation. There will be no Hepatitis C Virus Infection: Developing
assumptions.
onsite registration. The costs of Direct-Acting Antiviral Drugs for
DATES: The public workshop will be registration, to attend in person, for the Treatment.’’ The purpose of this draft
held on June 1, 2016, from 8 a.m. to 5 different categories of attendees are as guidance is to assist sponsors in all
p.m. follows: phases of development of direct-acting
ADDRESSES: The public workshop will antiviral (DAA) drugs for the treatment
be held at FDA’s White Oak Campus, Category Cost of chronic hepatitis C. This draft
10903 New Hampshire Ave., Building guidance revises the draft guidance of
31 Conference Center, the Great Room Industry Representative .................... $50 the same name that was issued on
(Rm. 1503), Silver Spring, MD 20993. Nonprofit Organization and Aca- October 23, 2013.
Entrance for the public meeting demic other than University of DATES: Although you can comment on
participants (non-FDA employees) is Maryland ....................................... 50 any guidance at any time (see 21 CFR
through Building 1 where routine University of Maryland, College Park 10.115(g)(5)), to ensure that the Agency
security check procedures will be and Baltimore ................................ 0 considers your comment on this draft
performed. For parking and security Federal Government ......................... 0 guidance before it begins work on the
information, please refer to http:// final version of the guidance, submit
www.fda.gov/AboutFDA/ Streaming Webcast of the Public either electronic or written comments
WorkingatFDA/BuildingsandFacilities/ Workshop: This public workshop will on the draft guidance by July 5, 2016.
WhiteOakCampusInformation/ also be Webcast. There is no registration ADDRESSES: You may submit comments
ucm241740.htm. fee for access to the workshop via the as follows:
FOR FURTHER INFORMATION CONTACT:
Webcast, but registration is still
Electronic Submissions
Audrey Thomas, Office of Regulatory required. Information regarding access
to the Webcast link is available at Submit electronic comments in the
Science and Innovation, Office of the following way:
Chief Scientist, Food and Drug www.pharmacy.umaryland.edu/
PedsExtrapolation. If you have never • Federal eRulemaking Portal: http://
Administration, 10903 New Hampshire www.regulations.gov. Follow the
Ave., Bldg. 1, Rm. 4220, Silver Spring, attended a Connect Pro event before,
instructions for submitting comments.
MD 20993–0002, 301–796–3520, test your connection at https://
Comments submitted electronically,
Audrey.Thomas@fda.hhs.gov. collaboration.fda.gov/common/help/en/
including attachments, to http://
SUPPLEMENTARY INFORMATION: The support/meeting_test.htm. To get a www.regulations.gov will be posted to
purpose of this public workshop is to quick overview of the Connect Pro the docket unchanged. Because your
provide an opportunity for relevant program, visit http://www.adobe.com/ comment will be made public, you are
stakeholders, including clinicians, go/connectpro_overview. solely responsible for ensuring that your
academia, industry, and FDA to discuss Accommodations: Attendees are comment does not include any
systematic assessment of data needed to responsible for their own hotel confidential information that you or a
support extrapolation of efficacy in accommodations. If you need special third party may not wish to be posted,
pediatric product development. accommodations while at FDA’s White such as medical information, your or
Specifically, the workshop will include: Oak Campus due to a disability, please anyone else’s Social Security number, or
(1) Presentations on approaches for contact Shari Solomon at confidential business information, such
assessing disease and therapeutic Shari.Solomon@fda.hhs.gov at least 7 as a manufacturing process. Please note
response similarity between adults and days in advance. that if you include your name, contact
pediatrics, and (2) discussion of information, or other information that
asabaliauskas on DSK3SPTVN1PROD with NOTICES
alternative approaches to the assessment Dated: April 26, 2016. identifies you in the body of your
of extrapolation assumptions in Leslie Kux, comments, that information will be
pediatric product development, Associate Commissioner for Policy. posted on http://www.regulations.gov.
including the use of clinical trial [FR Doc. 2016–10397 Filed 5–3–16; 8:45 am] • If you want to submit a comment
simulation and Bayesian approaches. BILLING CODE 4164–01–P
with confidential information that you
Examples in partial onset seizures, do not wish to be made available to the
inflammatory bowel diseases, and public, submit the comment as a
polyarticular juvenile idiopathic written/paper submission and in the
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Document Created: 2016-05-04 01:23:15
Document Modified: 2016-05-04 01:23:15
| Category | Regulatory Information | |
| Collection | Federal Register | |
| sudoc Class | AE 2.7: GS 4.107: AE 2.106: | |
| Publisher | Office of the Federal Register, National Archives and Records Administration | |
| Section | Notices | |
| Action | Notice of public workshop. | |
| Dates | The public workshop will be held on June 1, 2016, from 8 a.m. to 5 p.m. | |
| 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, [email protected] | |
| FR Citation | 81 FR 26804 |
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