82_FR_44370 82 FR 44188 - Drug Development in Pediatric Heart Failure: Extrapolation, Clinical Trial Design, and Endpoints; Public Workshop

82 FR 44188 - Drug Development in Pediatric Heart Failure: Extrapolation, Clinical Trial Design, and Endpoints; Public Workshop

DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration

Federal Register Volume 82, Issue 182 (September 21, 2017)

Page Range44188-44189
FR Document2017-20106

The Food and Drug Administration (FDA) is announcing the following public workshop entitled ``FDA-University of Maryland CERSI Drug Development in Pediatric Heart Failure: Extrapolation, Clinical Trial Design, and Endpoints.'' The purpose of the public workshop is to address challenges related to the evaluation of products in pediatric heart failure including population to study, endpoints, and extrapolation of adult efficacy data. The workshop will also provide a forum for discussion on the use of registry data, as well as alternative trial designs and statistical methods.

Federal Register, Volume 82 Issue 182 (Thursday, September 21, 2017) 
[Federal Register Volume 82, Number 182 (Thursday, September 21, 2017)]
[Notices]
[Pages 44188-44189]
From the Federal Register Online  [www.thefederalregister.org]
[FR Doc No: 2017-20106]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2017-N-0001]


Drug Development in Pediatric Heart Failure: Extrapolation, 
Clinical Trial Design, and Endpoints; Public Workshop

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice of public workshop.

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SUMMARY: The Food and Drug Administration (FDA) is announcing the 
following public workshop entitled ``FDA-University of Maryland CERSI 
Drug Development in Pediatric Heart Failure: Extrapolation, Clinical 
Trial Design, and Endpoints.'' The purpose of the public workshop is to 
address challenges related to the evaluation of products in pediatric 
heart failure including population to study, endpoints, and 
extrapolation of adult efficacy data. The workshop will also provide a 
forum for discussion on the use of registry data, as well as 
alternative trial designs and statistical methods.

DATES: The public workshop will be held on Friday, October 27, 2017, 
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., Bldg. 31, Rm. 1503A, Silver Spring, MD 20993-
0002. Entrance for the public workshop 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: Jacquline Yancy, Center for Drug 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg. 22, Rm. 6319, Silver Spring, MD 20993-0002, 301-
796-7068, [email protected].

[[Page 44189]]


SUPPLEMENTARY INFORMATION:

I. Background

    The purpose of this public workshop is to provide an opportunity 
for relevant stakeholders, including clinicians, academia, industry, 
and FDA, to discuss alternative trial designs for product development 
in pediatric heart failure.

II. Topics for Discussion at the Public Workshop

    Specifically, the workshop will include application of pediatric 
extrapolation in drug development for pediatric heart failure and a 
discussion of alternative approaches to establishing effectiveness in 
pediatric heart failure, including the use of Bayesian approaches. 
Cases will be presented to exemplify various approaches.
    The agenda is located at http://www.cersi.umd.edu/events/index.php?mode=4&id=12500.

III. Participating in the Public Workshop

    Registration: To register for the public workshop, visit the 
following Web site: http://www.cersi.umd.edu/events/index.php?mode=4&id=12500. Registrants will receive confirmation when 
they have been accepted. There will be no onsite 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. The cost to attend in person is 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
------------------------------------------------------------------------

    If you need special accommodations due to a disability, please 
contact Jacquline Yancy (see FOR FURTHER INFORMATION CONTACT) at least 
7 days in advance.
    Streaming Webcast of the Public Workshop: This public workshop will 
also be webcast. There is no registration fee for attending the 
workshop via the webcast, but registration is still required. 
Information regarding access to the webcast link is available at http://www.cersi.umd.edu/events/index.php?mode=4&id=12500.
    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. FDA has verified 
the Web site addresses in this document, as of the date this document 
publishes in the Federal Register, but Web sites are subject to change 
over time.
    Transcripts: Please be advised that as soon as a transcript of the 
public workshop is available, it will be accessible at https://www.regulations.gov. It may be viewed at the Dockets Management Staff 
Office (HFA-305), Food and Drug Administration, 5630 Fishers Lane, Rm. 
1061, Rockville, MD 20852.

    Dated: September 15, 2017.
Anna K. Abram,
Deputy Commissioner for Policy, Planning, Legislation, and Analysis.
[FR Doc. 2017-20106 Filed 9-20-17; 8:45 a.m.]
 BILLING CODE 4164-01-P

44188 Federal Register / Vol. 82, No. 182 / Thursday, September 21, 2017 / Notices redacted/blacked out, will be available than 2 business days before the meeting. Dated: September 15, 2017. for public viewing and posted on If FDA is unable to post the background Anna K. Abram, https://www.regulations.gov. Submit material on its Web site prior to the Deputy Commissioner for Policy, Planning, both copies to the Dockets Management meeting, the background material will Legislation, and Analysis. Staff. If you do not wish your name and be made publicly available at the [FR Doc. 2017–20105 Filed 9–20–17; 8:45 am] contact information to be made publicly location of the advisory committee BILLING CODE 4164–01–P available, you can provide this meeting, and the background material information on the cover sheet and not will be posted on FDA’s Web site after in the body of your comments and you the meeting. Background material is DEPARTMENT OF HEALTH AND must identify this information as available at https://www.fda.gov/ HUMAN SERVICES ‘‘confidential.’’ Any information marked AdvisoryCommittees/Calendar/default. as ‘‘confidential’’ will not be disclosed htm. Scroll down to the appropriate Food and Drug Administration except in accordance with 21 CFR 10.20 advisory committee meeting link. and other applicable disclosure law. For Procedure: Interested persons may [Docket No. FDA–2017–N–0001] more information about FDA’s posting present data, information, or views, of comments to public dockets, see 80 orally or in writing, on issues pending Drug Development in Pediatric Heart FR 56469, September 18, 2015, or access before the committee. All electronic and Failure: Extrapolation, Clinical Trial the information at: https://www.gpo.gov/ written submissions submitted to the Design, and Endpoints; Public fdsys/pkg/FR-2015-09-18/pdf/2015- Docket (see the ADDRESSES section) on Workshop 23389.pdf. or before September 28, 2017, will be Docket: For access to the docket to AGENCY: Food and Drug Administration, provided to the committee. Oral read background documents or the HHS. presentations from the public will be electronic and written/paper comments scheduled between approximately 1 ACTION: Notice of public workshop. received, go to https:// p.m. and 2 p.m. Those individuals www.regulations.gov and insert the interested in making formal oral SUMMARY: The Food and Drug docket number, found in brackets in the presentations should notify the contact Administration (FDA) is announcing the heading of this document, into the person and submit a brief statement of following public workshop entitled ‘‘Search’’ box and follow the prompts the general nature of the evidence or ‘‘FDA-University of Maryland CERSI and/or go to the Dockets Management arguments they wish to present, the Drug Development in Pediatric Heart Staff, 5630 Fishers Lane, Rm. 1061, names and addresses of proposed Failure: Extrapolation, Clinical Trial Rockville, MD 20852. participants, and an indication of the Design, and Endpoints.’’ The purpose of FOR FURTHER INFORMATION CONTACT: approximate time requested to make the public workshop is to address LaToya Bonner, Center for Drug their presentation on or before challenges related to the evaluation of Evaluation and Research, Food and September 20, 2017. Time allotted for products in pediatric heart failure Drug Administration, 10903 New each presentation may be limited. If the including population to study, Hampshire Ave., Bldg. 31, Rm. 2417, number of registrants requesting to endpoints, and extrapolation of adult Silver Spring, MD 20993–0002, 301– speak is greater than can be reasonably efficacy data. The workshop will also 796–9001, FAX: 301–847–8533, email: accommodated during the scheduled provide a forum for discussion on the DODAC@fda.hhs.gov, or FDA Advisory open public hearing session, FDA may use of registry data, as well as Committee Information Line, 1–800– conduct a lottery to determine the alternative trial designs and statistical 741–8138 (301–443–0572 in the speakers for the scheduled open public methods. Washington, DC area). A notice in the hearing session. The contact person will Federal Register about last minute notify interested persons regarding their DATES: The public workshop will be modifications that impact a previously request to speak by September 21, 2017. held on Friday, October 27, 2017, from announced advisory committee meeting 8 a.m. to 5 p.m. Persons attending FDA’s advisory cannot always be published quickly committee meetings are advised that the ADDRESSES: The public workshop will enough to provide timely notice. Agency is not responsible for providing be held at FDA’s White Oak Campus, Therefore, you should always check the access to electrical outlets. 10903 New Hampshire Ave., Bldg. 31, Agency’s Web site at https://www.fda. Rm. 1503A, Silver Spring, MD 20993– gov/AdvisoryCommittees/default.htm FDA welcomes the attendance of the public at its advisory committee 0002. Entrance for the public workshop and scroll down to the appropriate participants (non-FDA employees) is advisory committee meeting link, or call meetings and will make every effort to accommodate persons with disabilities. through Building 1, where routine the advisory committee information line security check procedures will be to learn about possible modifications If you require special accommodations due to a disability, please contact performed. For parking and security before coming to the meeting. information, please refer to https:// LaToya Bonner at least 7 days in SUPPLEMENTARY INFORMATION: www.fda.gov/AboutFDA/ advance of the meeting. Agenda: The committee will discuss WorkingatFDA/BuildingsandFacilities/ the safety and efficacy of new drug FDA is committed to the orderly WhiteOakCampusInformation/ application (NDA) 208254, for conduct of its advisory committee ucm241740.htm. meetings. Please visit our Web site at asabaliauskas on DSKBBXCHB2PROD with NOTICES netarsudil ophthalmic solution 0.02%, submitted by Aerie Pharmaceuticals https://www.fda.gov/ FOR FURTHER INFORMATION CONTACT: Inc., for the proposed indication to AdvisoryCommittees/AboutAdvisory Jacquline Yancy, Center for Drug reduce elevated intraocular pressure Committees/ucm111462.htm for Evaluation and Research, Food and (IOP) in patients with open-angle procedures on public conduct during Drug Administration, 10903 New glaucoma (OAG) or ocular hypertension advisory committee meetings. Hampshire Ave., Bldg. 22, Rm. 6319, (OHT). Notice of this meeting is given under Silver Spring, MD 20993–0002, 301– FDA intends to make background the Federal Advisory Committee Act (5 796–7068, Jacquline.Yancy@ material available to the public no later U.S.C. app. 2). fda.hhs.gov. VerDate Sep<11>2014 17:52 Sep 20, 2017 Jkt 241001 PO 00000 Frm 00038 Fmt 4703 Sfmt 4703 E:\FR\FM\21SEN1.SGM 21SEN1

Federal Register / Vol. 82, No. 182 / Thursday, September 21, 2017 / Notices 44189 SUPPLEMENTARY INFORMATION: go/connectpro_overview. FDA has systems. Attendance is free. Non-USDA verified the Web site addresses in this employees must enter through the Wing I. Background document, as of the date this document 3 entrance on Independence Avenue. The purpose of this public workshop publishes in the Federal Register, but Attendees must be pre-registered for the is to provide an opportunity for relevant Web sites are subject to change over meeting (and check-in outside the day stakeholders, including clinicians, time. of the meeting) and show a valid photo academia, industry, and FDA, to discuss Transcripts: Please be advised that as ID to enter the building. Only registered alternative trial designs for product soon as a transcript of the public attendees will be permitted to enter the development in pediatric heart failure. workshop is available, it will be building. For parking and security II. Topics for Discussion at the Public accessible at https:// information, please refer to https:// Workshop www.regulations.gov. It may be viewed smithsonianassociates.org/ticketing/ at the Dockets Management Staff Office help/locations/jefferson.htm. Specifically, the workshop will (HFA–305), Food and Drug You may submit comments as include application of pediatric Administration, 5630 Fishers Lane, Rm. follows. Please note that late, untimely extrapolation in drug development for 1061, Rockville, MD 20852. filed comments will not be considered. pediatric heart failure and a discussion Electronic comments must be submitted Dated: September 15, 2017. of alternative approaches to establishing on or before November 24, 2017. The effectiveness in pediatric heart failure, Anna K. Abram, Deputy Commissioner for Policy, Planning, https://www.regulations.gov electronic including the use of Bayesian filing system will accept comments approaches. Cases will be presented to Legislation, and Analysis. [FR Doc. 2017–20106 Filed 9–20–17; 8:45 a.m.] until midnight Eastern Time at the end exemplify various approaches. of November 24, 2017. Comments The agenda is located at http:// BILLING CODE 4164–01–P received by mail/hand delivery/courier www.cersi.umd.edu/events/ (for written/paper submissions) will be index.php?mode=4&id=12500. DEPARTMENT OF HEALTH AND considered timely if they are III. Participating in the Public HUMAN SERVICES postmarked or the delivery service Workshop acceptance receipt is on or before that Registration: To register for the public Food and Drug Administration date. workshop, visit the following Web site: [Docket No. FDA–2017–N–5056] Electronic Submissions http://www.cersi.umd.edu/events/ index.php?mode=4&id=12500. 2017 Scientific Meeting of the National Submit electronic comments in the Registrants will receive confirmation Antimicrobial Resistance Monitoring following way: when they have been accepted. There System; Public Meeting; Request for • Federal eRulemaking Portal: will be no onsite registration. Comments https://www.regulations.gov. Follow the There is a registration fee to attend instructions for submitting comments. this public workshop in person. Seats AGENCY: Food and Drug Administration, Comments submitted electronically, are limited and registration will be on HHS. including attachments, to https:// a first-come, first-served basis. The cost ACTION: Notice of public meeting; www.regulations.gov will be posted to to attend in person is as follows: request for comments. the docket unchanged. Because your comment will be made public, you are Category Cost SUMMARY: The Food and Drug solely responsible for ensuring that your Administration (FDA, the Agency, or comment does not include any Industry Representative ................ $50 we), together with the NARMS partner confidential information that you or a Nonprofit Organization and Aca- agencies, is announcing a public third party may not wish to be posted, demic Other Than University of meeting entitled ‘‘2017 Scientific such as medical information, your or Maryland ................................... 50 Meeting of the National Antimicrobial anyone else’s Social Security number, or University of Maryland, College Resistance Monitoring System.’’ The Park and Baltimore ................... 0 confidential business information, such purpose of the public meeting is to as a manufacturing process. Please note Federal Government ..................... 0 discuss the current status of the that if you include your name, contact If you need special accommodations National Antimicrobial Resistance information, or other information that due to a disability, please contact Monitoring System (NARMS) and identifies you in the body of your Jacquline Yancy (see FOR FURTHER directions for the future. comments, that information will be INFORMATION CONTACT) at least 7 days in DATES: The public meeting will be held posted on https://www.regulations.gov. advance. on October 24 and 25, 2017, from 8:30 • If you want to submit a comment Streaming Webcast of the Public a.m. to 5 p.m. Eastern Time. Submit with confidential information that you Workshop: This public workshop will either electronic or written comments do not wish to be made available to the also be webcast. There is no registration on this public meeting by November 24, public, submit the comment as a fee for attending the workshop via the 2017. See the SUPPLEMENTARY written/paper submission and in the webcast, but registration is still INFORMATION section for registration date manner detailed (see ‘‘Written/Paper required. Information regarding access and information. Submissions’’ and ‘‘Instructions’’). asabaliauskas on DSKBBXCHB2PROD with NOTICES to the webcast link is available at http:// ADDRESSES: The public meeting will be Written/Paper Submissions www.cersi.umd.edu/events/ held at the Jefferson Auditorium in the index.php?mode=4&id=12500. South Building, U.S. Department of Submit written/paper submissions as If you have never attended a Connect Agriculture (USDA), 14th and follows: Pro event before, test your connection at Independence Avenue SW., • Mail/Hand Delivery/Courier (for https://collaboration.fda.gov/common/ Washington, DC 20250. The South written/paper submissions): Dockets help/en/support/meeting_test.htm. To Building is a Federal facility, and Management Staff (HFA–305), Food and get a quick overview of the Connect Pro attendees should plan adequate time to Drug Administration, 5630 Fishers program, visit https://www.adobe.com/ pass through the security screening Lane, Rm. 1061, Rockville, MD 20852. VerDate Sep<11>2014 17:52 Sep 20, 2017 Jkt 241001 PO 00000 Frm 00039 Fmt 4703 Sfmt 4703 E:\FR\FM\21SEN1.SGM 21SEN1


Document Created: 2018-10-24 14:33:50
Document Modified: 2018-10-24 14:33:50
CategoryRegulatory Information
CollectionFederal Register
sudoc ClassAE 2.7:
GS 4.107:
AE 2.106:
PublisherOffice of the Federal Register, National Archives and Records Administration
SectionNotices
ActionNotice of public workshop.
DatesThe public workshop will be held on Friday, October 27, 2017, from 8 a.m. to 5 p.m.
ContactJacquline Yancy, Center for Drug Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 22, Rm. 6319, Silver Spring, MD 20993-0002, 301- 796-7068, [email protected]
FR Citation82 FR 44188 
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