[Federal Register Volume 83, Number 79 (Tuesday, April 24, 2018)]
[Notices]
[Pages 17825-17826]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2018-08548]


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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Nonclinical Development of Pharmaceuticals; Draft Guidance for 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.

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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the availability of a draft guidance for industry entitled ``Severely 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DATES: Submit either electronic or written comments on the draft 
guidance by June 25, 2018 to ensure that the Agency considers your 
comment on this draft guidance before it begins work on the final 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.gpo.gov/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


