83_FR_55401 83 FR 55188 - Product Development in Hemophilia; Public Workshop

83 FR 55188 - Product Development in Hemophilia; Public Workshop

DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration

Federal Register Volume 83, Issue 213 (November 2, 2018)

Page Range55188-55189
FR Document2018-23947

The Food and Drug Administration (FDA, the Agency, or we) is announcing the following public workshop entitled ``Product Development in Hemophilia.'' The purpose of the public workshop is to discuss issues related to development and regulation of novel hemophilia products.

Federal Register, Volume 83 Issue 213 (Friday, November 2, 2018) 
[Federal Register Volume 83, Number 213 (Friday, November 2, 2018)]
[Notices]
[Pages 55188-55189]
From the Federal Register Online  [www.thefederalregister.org]
[FR Doc No: 2018-23947]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2018-N-3693]


Product Development in Hemophilia; Public Workshop

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice of public workshop.

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SUMMARY: The Food and Drug Administration (FDA, the Agency, or we) is 
announcing the following public workshop entitled ``Product Development 
in Hemophilia.'' The purpose of the public workshop is to discuss 
issues related to development and regulation of novel hemophilia 
products.

DATES: The public workshop will be held on December 6, 2018, from 8:30 
a.m. to 4:30 p.m. See the SUPPLEMENTARY INFORMATION section for 
registration date and information.

ADDRESSES: The public workshop will be held at FDA's White Oak Campus, 
10903 New Hampshire Ave., Bldg. 31 Conference Center, the Great Room 
(Rm. 1503), 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.
    Docket: For access to the docket to read background documents 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.

FOR FURTHER INFORMATION CONTACT: Joan Ferlo Todd, Food and Drug 
Administration, Center for Drug Evaluation and Research, Office of 
Hematology and Oncology Products, 10903 New Hampshire Ave., Bldg. 22, 
Rm. 2139, Silver Spring, MD 20993-0002, 301-796-6079, 
[email protected].

SUPPLEMENTARY INFORMATION: 

I. Background

    Hemophilia is a bleeding disorder caused by deficiency of 
coagulation factor VIII (hemophilia A) or coagulation factor IX 
(hemophilia B). Hemophilia treatment strategies are intended to prevent 
or control bleeding and the attendant complications. Recently, 
hemophilia treatment strategies have led to the development of factor 
concentrates, recombinant DNA technology products, antibodies, and 
potential curative strategies such as gene therapy. These new emerging 
technologies raise new considerations about trial design, novel 
endpoints, patient-reported outcomes, and long-term safety collection.
    This public workshop is intended to provide a platform for engaging 
in a discussion with experts in hemophilia treatment, patients, and 
caregivers. The purpose of this workshop is to advance further 
development of patient-experience and patient-reported outcomes for use 
in clinical trials, facilitate reliable and interpretable measurements 
of factor VIII/IX activity

[[Page 55189]]

levels for gene therapy products, discuss the need for long-term safety 
assessments in gene therapy clinical trials, and discern when to enroll 
pediatric patients in gene therapy trials.

II. Topics for Discussion at the Public Workshop

    The workshop will feature presentations and panel discussions on 
hemophilia product development. The presentations will include an 
overview of product development in hemophilia, and the regulatory 
challenges in the development of novel hemophilia therapies. Five 
sessions include presentations to frame panel discussions to cover the 
following topics:
    1. Overview of product development in hemophilia;
    2. Efficacy endpoints related to bleeding outcomes and 
considerations for factor activity as a surrogate endpoint;
    3. Patient and caregiver perspectives on developing outcomes for 
clinical trials;
    4. Discrepancies in the factor activity measurements by different 
assays observed in gene therapy trials and root causes for the 
discrepancies; and
    5. Clinical trial design considerations for follow up on safety, 
efficacy, enrollment of pediatric patients in gene therapy trials, and 
the applicability of on-demand treatment as a control group in the 
evolving landscape of treatment options in hemophilia.

III. Participating in the Public Workshop

    Registration: Persons interested in attending this public workshop 
must register online at https://fdaoce.formstack.com/forms/pdh120618 
before 5 p.m. on December 3, 2018. Please provide complete contact 
information for each attendee, including name, title, affiliation, 
address, email, and telephone.
    Registration is free and based on space availability, with priority 
given to early registrants. Early registration is recommended because 
seating is limited; therefore, FDA may limit the number of participants 
from each organization. Registrants will receive confirmation when they 
have been accepted.
    If you need special accommodations due to a disability, please 
contact Joan Ferlo Todd at [email protected] no later than 5 p.m., 
on November 21, 2018.
    Streaming Webcast of the Public Workshop: This public workshop will 
also be web-streamed on the day of the workshop.
    If you have never attended a webcast event before, test your 
connection at https://collaboration.fda.gov/common/help/en/support/meeting_test.htm. To get a quick overview of the Adobe webcast program, 
visit https://www.adobe.com/go/connectpro_overview. FDA has verified 
the website addresses in this document, as of the date this document 
publishes in the Federal Register, but websites 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 
(see ADDRESSES). A link to the transcript will be available on the 
internet at https://www.fda.gov/NewsEvents/MeetingsConferencesWorkshops/ucm620602.htm.

    Dated: October 29, 2018.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2018-23947 Filed 11-1-18; 8:45 am]
 BILLING CODE 4164-01-P

55188 Federal Register / Vol. 83, No. 213 / Friday, November 2, 2018 / Notices and other applicable disclosure law. For pediatric HBV-infected patients are also DATES: The public workshop will be more information about FDA’s posting included. held on December 6, 2018, from 8:30 of comments to public dockets, see 80 This draft guidance is being issued a.m. to 4:30 p.m. See the FR 56469, September 18, 2015, or access consistent with FDA’s good guidance SUPPLEMENTARY INFORMATION section for the information at: https://www.gpo.gov/ practices regulation (21 CFR 10.115). registration date and information. fdsys/pkg/FR-2015-09-18/pdf/2015- The draft guidance, when finalized, will ADDRESSES: The public workshop will 23389.pdf. represent the current thinking of FDA be held at FDA’s White Oak Campus, Docket: For access to the docket to on ‘‘Chronic Hepatitis B Virus Infection: 10903 New Hampshire Ave., Bldg. 31 read background documents or the Developing Drugs for Treatment.’’ It Conference Center, the Great Room (Rm. electronic and written/paper comments does not establish any rights for any 1503), Silver Spring, MD 20993–0002. received, go to https:// person and is not binding on FDA or the Entrance for the public workshop www.regulations.gov and insert the public. You can use an alternative participants (non-FDA employees) is docket number, found in brackets in the approach if it satisfies the requirements through Building 1 where routine heading of this document, into the of the applicable statutes and security check procedures will be ‘‘Search’’ box and follow the prompts regulations. This guidance is not subject performed. For parking and security and/or go to the Dockets Management to Executive Order 12866. information, please refer to https:// Staff, 5630 Fishers Lane, Rm. 1061, II. Paperwork Reduction Act of 1995 www.fda.gov/AboutFDA/Workingat Rockville, MD 20852. FDA/BuildingsandFacilities/ This guidance refers to previously WhiteOakCampusInformation/ You may submit comments on any approved collections of information that ucm241740.htm. guidance at any time (see 21 CFR are subject to review by the Office of Docket: For access to the docket to 10.115(g)(5)). Management and Budget (OMB) under read background documents go to Submit written requests for single the Paperwork Reduction Act of 1995 copies of the draft guidance to the https://www.regulations.gov and insert (PRA) (44 U.S.C. 3501–3520). The the docket number, found in brackets in Division of Drug Information, Center for collections of information in 21 CFR Drug Evaluation and Research, Food the heading of this document, into the parts 312 and 314 have been approved ‘‘Search’’ box and follow the prompts and Drug Administration, 10001 New under OMB control numbers 0910–0014 Hampshire Ave., Hillandale Building, and/or go to the Dockets Management and 0910–0001, respectively. The Staff, 5630 Fishers Lane, Rm. 1061, 4th Floor, Silver Spring, MD 20993– submission of prescription drug labeling 0002. Send one self-addressed adhesive Rockville, MD 20852. under 21 CFR 201.56 and 201.57 has label to assist that office in processing FOR FURTHER INFORMATION CONTACT: Joan been approved under OMB control your requests. See the SUPPLEMENTARY Ferlo Todd, Food and Drug number 0910–0572. INFORMATION section for electronic Administration, Center for Drug access to the draft guidance document. III. Electronic Access Evaluation and Research, Office of Persons with access to the internet Hematology and Oncology Products, FOR FURTHER INFORMATION CONTACT: may obtain the draft guidance at either 10903 New Hampshire Ave., Bldg. 22, Poonam Mishra, Center for Drug https://www.fda.gov/Drugs/Guidance Rm. 2139, Silver Spring, MD 20993– Evaluation and Research, Food and ComplianceRegulatoryInformation/ 0002, 301–796–6079, Joan.Todd@ Drug Administration, 10903 New Guidances/default.htm or https:// fda.hhs.gov. Hampshire Ave., Bldg. 22, Rm. 6100, Silver Spring, MD 20993, 301–796– www.regulations.gov. SUPPLEMENTARY INFORMATION: 1500. Dated: October 29, 2018. I. Background SUPPLEMENTARY INFORMATION: Leslie Kux, Hemophilia is a bleeding disorder Associate Commissioner for Policy. caused by deficiency of coagulation I. Background [FR Doc. 2018–23951 Filed 11–1–18; 8:45 am] factor VIII (hemophilia A) or FDA is announcing the availability of BILLING CODE 4164–01–P coagulation factor IX (hemophilia B). a draft guidance for industry entitled Hemophilia treatment strategies are ‘‘Chronic Hepatitis B Virus Infection: intended to prevent or control bleeding Developing Drugs for Treatment.’’ The DEPARTMENT OF HEALTH AND and the attendant complications. purpose of this guidance is to assist HUMAN SERVICES Recently, hemophilia treatment sponsors in the clinical development of strategies have led to the development Food and Drug Administration drugs and biologics for the treatment of of factor concentrates, recombinant chronic HBV infection from the initial [Docket No. FDA–2018–N–3693] DNA technology products, antibodies, IND through the NDA/BLA and and potential curative strategies such as postmarketing phases. The guidance Product Development in Hemophilia; gene therapy. These new emerging includes general considerations for Public Workshop technologies raise new considerations nonclinical toxicology and virology AGENCY: Food and Drug Administration, about trial design, novel endpoints, studies, early phase clinical HHS. patient-reported outcomes, and long- development, clinical pharmacology ACTION: Notice of public workshop. term safety collection. assessments, and phase 3 safety and This public workshop is intended to efficacy trials. The guidance discusses SUMMARY: The Food and Drug provide a platform for engaging in a phase 3 trial design considerations and Administration (FDA, the Agency, or discussion with experts in hemophilia efficacy endpoints for the development we) is announcing the following public treatment, patients, and caregivers. The of combination therapies for the workshop entitled ‘‘Product purpose of this workshop is to advance treatment of chronic HBV infection. Development in Hemophilia.’’ The further development of patient- Drug development considerations for purpose of the public workshop is to experience and patient-reported specific subpopulations such as patients discuss issues related to development outcomes for use in clinical trials, coinfected with hepatitis D virus or and regulation of novel hemophilia facilitate reliable and interpretable human immunodeficiency virus and products. measurements of factor VIII/IX activity VerDate Sep<11>2014 17:57 Nov 01, 2018 Jkt 247001 PO 00000 Frm 00047 Fmt 4703 Sfmt 4703 E:\FR\FM\02NON1.SGM 02NON1

Federal Register / Vol. 83, No. 213 / Friday, November 2, 2018 / Notices 55189 levels for gene therapy products, discuss If you have never attended a webcast to provide T/TA addressing the critical the need for long-term safety event before, test your connection at health needs of MSAW in alignment assessments in gene therapy clinical https://collaboration.fda.gov/common/ with HRSA priorities. T/TA provided at trials, and discern when to enroll help/en/support/meeting_test.htm. To the Migrant Stream Forums is targeted pediatric patients in gene therapy trials. get a quick overview of the Adobe to a broad range of health center staff webcast program, visit https:// positions, and covers diverse topics that II. Topics for Discussion at the Public www.adobe.com/go/connectpro_ address the needs of migrant health Workshop overview. FDA has verified the website centers and the patients they serve. The workshop will feature addresses in this document, as of the Supplemental funds are necessary to presentations and panel discussions on date this document publishes in the support their timely and successful hemophilia product development. The Federal Register, but websites are implementation. presentations will include an overview subject to change over time. This supplemental funding will of product development in hemophilia, Transcripts: Please be advised that as augment the current NCA investment and the regulatory challenges in the soon as a transcript of the public for these T/TA opportunities through development of novel hemophilia workshop is available, it will be support of enhanced personnel therapies. Five sessions include accessible at https:// presence, the availability of continuing presentations to frame panel discussions www.regulations.gov. It may be viewed education unit-bearing educational to cover the following topics: at the Dockets Management Staff (see sessions to meet the diverse needs of 1. Overview of product development ADDRESSES). A link to the transcript will multidisciplinary health center staff, in hemophilia; be available on the internet at https:// and speaker and participant stipends 2. Efficacy endpoints related to www.fda.gov/NewsEvents/Meetings that underscore the unique value these bleeding outcomes and considerations ConferencesWorkshops/ in-person regional T/TA sessions for factor activity as a surrogate ucm620602.htm. provide. endpoint; Dated: October 29, 2018. FOR FURTHER INFORMATION CONTACT: 3. Patient and caregiver perspectives Leslie Kux, Tracey Orloff, Strategic Partnerships on developing outcomes for clinical Associate Commissioner for Policy. Division Director in the Bureau of trials; Primary Health Care, Office of Quality [FR Doc. 2018–23947 Filed 11–1–18; 8:45 am] 4. Discrepancies in the factor activity Improvement, at TOrloff@hrsa.gov. BILLING CODE 4164–01–P measurements by different assays Dated: October 26, 2018. observed in gene therapy trials and root George Sigounas, causes for the discrepancies; and DEPARTMENT OF HEALTH AND Administrator. 5. Clinical trial design considerations HUMAN SERVICES [FR Doc. 2018–24008 Filed 11–1–18; 8:45 am] for follow up on safety, efficacy, BILLING CODE 4165–15–P enrollment of pediatric patients in gene Health Resources and Services therapy trials, and the applicability of Administration on-demand treatment as a control group Health Center Program DEPARTMENT OF HEALTH AND in the evolving landscape of treatment HUMAN SERVICES options in hemophilia. AGENCY: Health Resources and Services III. Participating in the Public Administration (HRSA), Department of National Institutes of Health Workshop Health and Human Services. Eunice Kennedy Shriver National ACTION: Announcement of Supplemental Registration: Persons interested in Institute of Child Health & Human attending this public workshop must Award. Development; Notice of Closed register online at https:// SUMMARY: HRSA provided supplemental Meeting fdaoce.formstack.com/forms/ grant funds to a currently funded pdh120618 before 5 p.m. on December Pursuant to section 10(d) of the National Training and Technical Federal Advisory Committee Act, as 3, 2018. Please provide complete Assistance Cooperative Agreement contact information for each attendee, amended, notice is hereby given of the (NCA) award recipient to coordinate following meeting. including name, title, affiliation, and provide training and technical address, email, and telephone. The meeting will be closed to the assistance (T/TA) to health centers that public in accordance with the Registration is free and based on serve migrant and seasonal agricultural provisions set forth in sections space availability, with priority given to workers (MSAW) and their families 552b(c)(4) and 552b(c)(6), Title 5 U.S.C., early registrants. Early registration is through three regional forums. as amended. The grant applications and recommended because seating is SUPPLEMENTARY INFORMATION: the discussions could disclose limited; therefore, FDA may limit the Recipient of the Award: The National confidential trade secrets or commercial number of participants from each Center for Farmworker Health, Inc. property such as patentable material, organization. Registrants will receive Amount of Non-Competitive Awards: and personal information concerning confirmation when they have been $150,000. individuals associated with the grant accepted. Period of Supplemental Funding: applications, the disclosure of which If you need special accommodations Fiscal years 2018 and 2019 (contingent would constitute a clearly unwarranted due to a disability, please contact Joan upon available funding and satisfactory invasion of personal privacy. Ferlo Todd at Joan.Todd@fda.hhs.gov performance). no later than 5 p.m., on November 21, CFDA Number: 93.129. Name of Committee: National Institute of 2018. Authority: Section 330(l) of the Public Child Health and Human Development Health Service Act, as amended. Special Emphasis Panel. Streaming Webcast of the Public Date: November 20, 2018. Workshop: This public workshop will JUSTIFICATION: The award recipient will Time: 1:00 p.m. to 3:00 p.m. also be web-streamed on the day of the lead the coordination and management Agenda: To review and evaluate grant workshop. of three regional Migrant Stream Forums applications. VerDate Sep<11>2014 17:57 Nov 01, 2018 Jkt 247001 PO 00000 Frm 00048 Fmt 4703 Sfmt 4703 E:\FR\FM\02NON1.SGM 02NON1


Document Created: 2018-11-02 01:09:41
Document Modified: 2018-11-02 01:09:41
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 December 6, 2018, from 8:30 a.m. to 4:30 p.m. See the SUPPLEMENTARY INFORMATION section for registration date and information.
ContactJoan Ferlo Todd, Food and Drug Administration, Center for Drug Evaluation and Research, Office of Hematology and Oncology Products, 10903 New Hampshire Ave., Bldg. 22, Rm. 2139, Silver Spring, MD 20993-0002, 301-796-6079, [email protected]
FR Citation83 FR 55188 
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