83 FR 17825 - Severely Debilitating or Life-Threatening Hematologic Disorders: Nonclinical Development of Pharmaceuticals; Draft Guidance for Industry; Availability
DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration Federal Register Volume 83, Issue 79 (April 24, 2018)
Food and Drug Administration
Federal Register Volume 83, Issue 79 (April 24, 2018)
| Page Range | 17825-17826 | |
| FR Document | 2018-08548 |
[Federal Register Volume 83, Number 79 (Tuesday, April 24, 2018)] [Notices] [Pages 17825-17826] From the Federal Register Online [www.thefederalregister.org] [FR Doc No: 2018-08548] ----------------------------------------------------------------------- DEPARTMENT OF HEALTH AND HUMAN SERVICES Food and Drug Administration [Docket No. FDA-2018-D-1328] Severely Debilitating or Life-Threatening Hematologic Disorders: Nonclinical Development of Pharmaceuticals; Draft Guidance for Industry; Availability AGENCY: Food and Drug Administration, HHS.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SUMMARY: The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled ``Severely Debilitating or Life-Threatening Hematologic Disorders: Nonclinical Development of Pharmaceuticals.'' The purpose of this guidance is to provide information to assist sponsors in the design of an appropriate program of nonclinical studies for the development of pharmaceuticals used to treat patients with severely debilitating or life-threatening hematologic disorders (SDLTHDs). While FDA has guidance for oncology indications (most of which are considered severely debilitating or life-threatening diseases) and for rare diseases (which include some SDLTHD conditions), FDA has no guidance to facilitate nonclinical development specifically for pharmaceuticals used to treat nononcology patients with SDLTHDs. A streamlined approach to drug development is necessary to allow patients with SDLTHDs earlier and continued access to new and potentially effective therapies. DATES: Submit either electronic or written comments on the draft guidance by June 25, 2018 to ensure that the Agency considers your comment on this draft guidance before it begins work on the final version of the guidance. 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-2018-D-1328 for ``Severely Debilitating or Life-Threatening Hematologic Disorders: Nonclinical Development of Pharmaceuticals; Draft Guidance for Industry; Availability.'' 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 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.thefederalregister.org/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 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. 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: John Leighton, Center for Drug Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 22, Rm. 2204, Silver Spring, MD 20993-0002, 301- 796-0750; or Haleh Saber, Center for Drug Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 22, Rm. 2117, Silver Spring, MD 20993-0002, 301-796-0750. [[Page 17826]] SUPPLEMENTARY INFORMATION: I. Background FDA is announcing the availability of a draft guidance for industry entitled ``Severely Debilitating or Life-Threatening Hematologic Disorders: Nonclinical Development of Pharmaceuticals.'' The purpose of this guidance is to provide information to assist sponsors in the design of an appropriate program of nonclinical studies for the development of pharmaceuticals used to treat patients with SDLTHDs. While FDA has guidance for oncology indications (most of which are considered severely debilitating or life-threatening diseases) and for rare diseases (which include some SDLTHD conditions), FDA has no guidance to facilitate nonclinical development specifically for pharmaceuticals used to treat nononcology patients with SDLTHDs. The SDLTHDs include conditions in which life expectancy is short or quality of life is greatly diminished despite available therapies. FDA has defined life-threatening and severely debilitating diseases in regulations (21 CFR 312.81). A streamlined approach to drug development is necessary to allow patients with SDLTHDs earlier and continued access to new and potentially effective therapies. This guidance, when finalized, is expected to reduce the use of animals in accordance with the 3R (refine/reduce/replace) principles and allow faster and continuous access to pharmaceuticals for SDLTHDs. 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 nonclinical development of pharmaceuticals for severely debilitating or life- threatening hematologic disorders. 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. Electronic Access Persons with access to the internet may obtain the draft guidance at either https://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/default.htm or https://www.regulations.gov. Dated: April 19, 2018. Leslie Kux, Associate Commissioner for Policy. [FR Doc. 2018-08548 Filed 4-23-18; 8:45 am] BILLING CODE 4164-01-P
![Federal Register / Vol. 83, No. 79 / Tuesday, April 24, 2018 / Notices 17825
adherence with risk management ADDRESSES: You may submit comments submission. You should submit two
guidance. on any guidance at any time as follows: copies total. One copy will include the
Public comments are requested, information you claim to be confidential
Electronic Submissions
including those expressing support or with a heading or cover note that states
with specific suggestions to improve the Submit electronic comments in the ‘‘THIS DOCUMENT CONTAINS
Research plan. A copy of the draft following way: CONFIDENTIAL INFORMATION.’’ The
Research plan is available at https:// • Federal eRulemaking Portal: Agency will review this copy, including
www.regulations.gov (see Docket https://www.regulations.gov. Follow the the claimed confidential information, in
Number CDC–2018–0038). instructions for submitting comments. its consideration of comments. The
Comments submitted electronically, second copy, which will have the
Dated: April 19, 2018.
including attachments, to https:// claimed confidential information
John J. Howard, www.regulations.gov will be posted to redacted/blacked out, will be available
Director, National Institute for Occupational the docket unchanged. Because your
Safety and Health, Centers for Disease Control
for public viewing and posted on
comment will be made public, you are https://www.regulations.gov. Submit
and Prevention.
solely responsible for ensuring that your both copies to the Dockets Management
[FR Doc. 2018–08472 Filed 4–23–18; 8:45 am] comment does not include any Staff. If you do not wish your name and
BILLING CODE 4163–19–P confidential information that you or a contact information to be made publicly
third party may not wish to be posted, available, you can provide this
such as medical information, your or information on the cover sheet and not
DEPARTMENT OF HEALTH AND anyone else’s Social Security number, or
HUMAN SERVICES in the body of your comments and you
confidential business information, such must identify this information as
Food and Drug Administration as a manufacturing process. Please note ‘‘confidential.’’ Any information marked
that if you include your name, contact as ‘‘confidential’’ will not be disclosed
[Docket No. FDA–2018–D–1328] information, or other information that except in accordance with 21 CFR 10.20
identifies you in the body of your and other applicable disclosure law. For
Severely Debilitating or Life- comments, that information will be more information about FDA’s posting
Threatening Hematologic Disorders: posted on https://www.regulations.gov. of comments to public dockets, see 80
Nonclinical Development of • If you want to submit a comment FR 56469, September 18, 2015, or access
Pharmaceuticals; Draft Guidance for with confidential information that you the information at: https://www.gpo.gov/
Industry; Availability do not wish to be made available to the fdsys/pkg/FR-2015-09-18/pdf/2015-
AGENCY: Food and Drug Administration, public, submit the comment as a 23389.pdf.
HHS. written/paper submission and in the Docket: For access to the docket to
manner detailed (see ‘‘Written/Paper read background documents or the
ACTION: Notice of availability. Submissions’’ and ‘‘Instructions’’). electronic and written/paper comments
SUMMARY: The Food and Drug Written/Paper Submissions received, go to https://
Administration (FDA or Agency) is www.regulations.gov and insert the
Submit written/paper submissions as
announcing the availability of a draft docket number, found in brackets in the
follows:
guidance for industry entitled ‘‘Severely • Mail/Hand delivery/Courier (for heading of this document, into the
Debilitating or Life-Threatening written/paper submissions): Dockets ‘‘Search’’ box and follow the prompts
Hematologic Disorders: Nonclinical Management Staff (HFA–305), Food and and/or go to the Dockets Management
Development of Pharmaceuticals.’’ The Drug Administration, 5630 Fishers Staff, 5630 Fishers Lane, Rm. 1061,
purpose of this guidance is to provide Lane, Rm. 1061, Rockville, MD 20852. Rockville, MD 20852.
information to assist sponsors in the • For written/paper comments You may submit comments on any
design of an appropriate program of submitted to the Dockets Management guidance at any time (see 21 CFR
nonclinical studies for the development Staff, FDA will post your comment, as 10.115(g)(5)).
of pharmaceuticals used to treat patients well as any attachments, except for Submit written requests for single
with severely debilitating or life- information submitted, marked and copies of the draft guidance to the
threatening hematologic disorders identified, as confidential, if submitted Division of Drug Information, Center for
(SDLTHDs). While FDA has guidance as detailed in ‘‘Instructions.’’ Drug Evaluation and Research, Food
for oncology indications (most of which Instructions: All submissions received and Drug Administration, 10001 New
are considered severely debilitating or must include the Docket No. FDA– Hampshire Ave., Hillandale Building,
life-threatening diseases) and for rare 2018–D–1328 for ‘‘Severely Debilitating 4th Floor, Silver Spring, MD 20993–
diseases (which include some SDLTHD or Life-Threatening Hematologic 0002. Send one self-addressed adhesive
conditions), FDA has no guidance to Disorders: Nonclinical Development of label to assist that office in processing
facilitate nonclinical development Pharmaceuticals; Draft Guidance for your requests. See the SUPPLEMENTARY
specifically for pharmaceuticals used to Industry; Availability.’’ Received INFORMATION section for electronic
treat nononcology patients with comments will be placed in the docket access to the draft guidance document.
SDLTHDs. A streamlined approach to and, except for those submitted as FOR FURTHER INFORMATION CONTACT: John
drug development is necessary to allow ‘‘Confidential Submissions,’’ publicly Leighton, Center for Drug Evaluation
patients with SDLTHDs earlier and viewable at https://www.regulations.gov and Research, Food and Drug
continued access to new and potentially or at the Dockets Management Staff Administration, 10903 New Hampshire
daltland on DSKBBV9HB2PROD with NOTICES
effective therapies. between 9 a.m. and 4 p.m., Monday Ave., Bldg. 22, Rm. 2204, Silver Spring,
DATES: Submit either electronic or through Friday. MD 20993–0002, 301–796–0750; or
written comments on the draft guidance • Confidential Submissions–To Haleh Saber, Center for Drug Evaluation
by June 25, 2018 to ensure that the submit a comment with confidential and Research, Food and Drug
Agency considers your comment on this information that you do not wish to be Administration, 10903 New Hampshire
draft guidance before it begins work on made publicly available, submit your Ave., Bldg. 22, Rm. 2117, Silver Spring,
the final version of the guidance. comments only as a written/paper MD 20993–0002, 301–796–0750.
VerDate Sep<11>2014 17:03 Apr 23, 2018 Jkt 244001 PO 00000 Frm 00038 Fmt 4703 Sfmt 4703 E:\FR\FM\24APN1.SGM 24APN1](https://thefederalregister.org/doc/83_FR_17904/page/1.svg)
![17826 Federal Register / Vol. 83, No. 79 / Tuesday, April 24, 2018 / Notices
SUPPLEMENTARY INFORMATION: Dated: April 19, 2018. non-profit organizations will submit
Leslie Kux, statewide needs assessment updates in
I. Background response to a forthcoming SIR.
Associate Commissioner for Policy.
FDA is announcing the availability of [FR Doc. 2018–08548 Filed 4–23–18; 8:45 am] The MIECHV Program, authorized by
section 511 of the Social Security Act,
a draft guidance for industry entitled BILLING CODE 4164–01–P
42 U.S.C. 711, and administered by
‘‘Severely Debilitating or Life-
HRSA in partnership with the
Threatening Hematologic Disorders: Administration for Children and
Nonclinical Development of DEPARTMENT OF HEALTH AND
HUMAN SERVICES Families, supports voluntary, evidence-
Pharmaceuticals.’’ The purpose of this based home visiting services during
guidance is to provide information to Health Resources and Services pregnancy and to parents with young
assist sponsors in the design of an Administration children up to kindergarten entry.
appropriate program of nonclinical States, territories, and tribal entities,
studies for the development of Agency Information Collection and nonprofit organizations, in certain
pharmaceuticals used to treat patients Activities: Proposed Collection: Public circumstances, are eligible to receive
with SDLTHDs. While FDA has Comment Request Information funding through MIECHV and have the
guidance for oncology indications (most Collection Request Title: The Maternal, flexibility, within the parameters of the
of which are considered severely Infant, and Early Childhood Home authorizing statute, to tailor the program
debilitating or life-threatening diseases) Visiting Program Statewide Needs to serve the specific needs of their
and for rare diseases (which include Assessment Update communities.
some SDLTHD conditions), FDA has no The statewide needs assessment is a
AGENCY: Health Resources and Services
guidance to facilitate nonclinical critical and foundational resource that
Administration (HRSA), Department of assists awardees in identifying and
development specifically for Health and Human Services.
pharmaceuticals used to treat understanding how to meet the needs of
ACTION: Notice. eligible families living in at-risk
nononcology patients with SDLTHDs.
SUMMARY: In compliance with the communities in their states.
The SDLTHDs include conditions in Need and Proposed Use of the
requirement for opportunity for public
which life expectancy is short or quality Information: Congress, through
comment on proposed data collection
of life is greatly diminished despite enactment of the Social Security Act,
projects of the Paperwork Reduction Act
available therapies. FDA has defined Title V, Section 511 (42 U.S.C. 711), as
of 1995, HRSA announces plans to
life-threatening and severely debilitating amended, established the MIECHV
submit a Supplemental Information
diseases in regulations (21 CFR 312.81). Program. The MIECHV Program is
Request (SIR), described below, to the
A streamlined approach to drug designed to: (1) Strengthen and improve
Office of Management and Budget
development is necessary to allow the programs and activities carried out
(OMB). Prior to submitting the SIR to
patients with SDLTHDs earlier and under Title V of the Social Security Act;
OMB, HRSA seeks comments from the
continued access to new and potentially (2) improve coordination of services for
public regarding the burden estimate,
effective therapies. This guidance, when at risk communities; and (3) identify
below, or any other aspect of the SIR.
finalized, is expected to reduce the use and provide comprehensive services to
DATES: Comments on this SIR should be improve outcomes for families who
of animals in accordance with the 3R received no later than June 25, 2018. reside in at risk communities. Section
(refine/reduce/replace) principles and ADDRESSES: Submit your comments to 50603 of the Bipartisan Budget Act of
allow faster and continuous access to paperwork@hrsa.gov or mail the HRSA 2018 (Pub. L. 115–123) amended section
pharmaceuticals for SDLTHDs. Information Collection Clearance 511(b)(1) of the Social Security Act, and
This draft guidance is being issued Officer, 14N39, 5600 Fishers Lane, requires that states review and update
consistent with FDA’s good guidance Rockville, MD 20857. their statewide needs assessments
practices regulation (21 CFR 10.115). FOR FURTHER INFORMATION CONTACT: To (which may be separate from, but in
The draft guidance, when finalized, will request more information on the coordination with, the Title V statewide
represent the current thinking of FDA proposed project or to obtain a copy of needs assessment) no later than October
on nonclinical development of the data collection plans and draft 1, 2020, as a condition of receiving
pharmaceuticals for severely instruments, email paperwork@hrsa.gov payments from Title V Block Grant
debilitating or life-threatening or call Lisa Wright-Solomon, the HRSA allotments.
hematologic disorders. It does not Information Collection Clearance Officer In response to the forthcoming SIR,
establish any rights for any person and at (301) 443–1984. state and territory awardees will be
is not binding on FDA or the public. SUPPLEMENTARY INFORMATION: required to submit an updated statewide
You can use an alternative approach if Information Collection Request Title: needs assessment that identifies all of
The Maternal, Infant, and Early the following information, as required
it satisfies the requirements of the
Childhood Home Visiting Program by the MIECHV authorizing statute:
applicable statutes and regulations. This (1) Communities with concentrations
Needs Assessment Update
guidance is not subject to Executive OMB No.: 0906–XXXX, New. of (a) premature birth, low-birth weight
Order 12866. Abstract: HRSA is requesting infants, and infant mortality, including
II. Electronic Access approval to collect updated statewide infant death due to neglect, or other
needs assessments from Maternal, indicators of at-risk prenatal, maternal,
daltland on DSKBBV9HB2PROD with NOTICES
Persons with access to the internet Infant, and Early Childhood Home newborn, or child health; (b) poverty; (c)
may obtain the draft guidance at either Visiting (MIECHV) Program awardees. crime; (d) domestic violence; (e) high
https://www.fda.gov/Drugs/Guidance The previous statewide needs rates of high-school drop-outs; (f)
ComplianceRegulatoryInformation/ assessment that was approved under substance abuse; (g) unemployment; or
Guidances/default.htm or https:// OMB control number 0915–0333 has (h) child maltreatment.
www.regulations.gov. been discontinued. Eligible entities that (2) The quality and capacity of
are states, the District of Columbia, and existing programs or initiatives for early
VerDate Sep<11>2014 17:03 Apr 23, 2018 Jkt 244001 PO 00000 Frm 00039 Fmt 4703 Sfmt 4703 E:\FR\FM\24APN1.SGM 24APN1](https://thefederalregister.org/doc/83_FR_17904/page/2.svg)
Document Created: 2018-04-24 00:39:11
Document Modified: 2018-04-24 00:39:11
| 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 availability. | |
| Dates | Submit either electronic or written comments on the draft guidance by June 25, 2018 to ensure that the Agency considers your comment on this draft guidance before it begins work on the final version of the guidance. | |
| Contact | John Leighton, Center for Drug Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 22, Rm. 2204, Silver Spring, MD 20993-0002, 301- 796-0750; or Haleh Saber, Center for Drug Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 22, Rm. 2117, Silver Spring, MD 20993-0002, 301-796-0750. | |
| FR Citation | 83 FR 17825 |
2025 Federal Register | Disclaimer | Privacy Policy
USC | CFR | eCFR