
[Federal Register Volume 84, Number 51 (Friday, March 15, 2019)]
[Notices]
[Pages 9533-9534]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2019-04816]


-----------------------------------------------------------------------

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; 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 final guidance for industry entitled ``Severely 
Debilitating or Life-Threatening Hematologic Disorders: Nonclinical 
Development of Pharmaceuticals.'' This guidance outlines nonclinical 
studies recommended for the development of pharmaceuticals used to 
treat patients with severely debilitating or life-threatening 
hematologic disorders (SDLTHDs) and addresses comments received to the 
docket. This guidance is intended to streamline the development of 
pharmaceuticals used to treat patients with SDLTHDs, other than cancer, 
while protecting patients' safety and avoiding unnecessary use of 
animals, in accordance with the 3R (reduce, refine, replace) 
principles. This guidance applies to pharmaceuticals used both to treat 
the active disease and to prevent the recurrence of a life-threatening 
or debilitating event.

DATES: The announcement of the guidance is published in the Federal 
Register on March 15, 2019.

ADDRESSES: You may submit either electronic or written comments on 
Agency guidances 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.'' 
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.

[[Page 9534]]

    You may submit comments on any guidance at any time (see 21 CFR 
10.115(g)(5)).
    Submit written requests for single copies of this 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 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-
796-7550; 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, 301-796-7550.

SUPPLEMENTARY INFORMATION:

I. Background

    FDA is announcing the availability of a guidance for industry 
entitled ``Severely Debilitating or Life-Threatening Hematologic 
Disorders: Nonclinical Development of Pharmaceuticals.'' The purpose of 
this guidance is to assist sponsors in the design of nonclinical 
studies for the development of pharmaceuticals used to treat patients 
with SDLTHDs. This guidance is intended to streamline the development 
of pharmaceuticals used to treat patients with SDLTHDs, other than 
cancer, while protecting patients' safety and avoiding unnecessary use 
of animals, in accordance with the 3R principles. This guidance applies 
to pharmaceuticals used both to treat the active disease and to prevent 
the recurrence of a life-threatening or debilitating event.
    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. The draft guidance 
for industry entitled ``Rare Diseases: Common Issues in Drug 
Development'' (available at https://www.fda.gov/ucm/groups/fdagov-public/@fdagov-drugs-gen/documents/document/ucm458485.pdf) does not 
specifically facilitate the nonclinical development of pharmaceuticals 
for treatment of SDLTHDs. Because SDLTHDs are not all rare diseases, 
they can fall outside the scope of the draft guidance for rare 
diseases. The present document provides consistent guidance for all 
nononcology SDLTHDs, independent of disease incidence or prevalence.
    This guidance is being issued consistent with FDA's good guidance 
practices regulation (21 CFR 10.115). The guidance represents the 
current thinking of FDA on ``Severely Debilitating or Life-Threatening 
Hematologic Disorders: Nonclinical Development of Pharmaceuticals.'' 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. Paperwork Reduction Act of 1995

    This guidance refers to previously approved collections of 
information that are subject to review by the Office of Management and 
Budget (OMB) under the Paperwork Reduction Act of 1995 (44 U.S.C. 3501-
3520). The collection of information submitted under 21 CFR part 312 
has been approved under OMB control number 0910-0014. The collection of 
information submitted under 21 CFR part 314 has been approved under OMB 
control number 0910-0001.

III. Electronic Access

    Persons with access to the internet may obtain the guidance at 
either https://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/default.htm or 
https://www.regulations.gov.

    Dated: March 11, 2019.
Lowell J. Schiller,
Acting Associate Commissioner for Policy.
[FR Doc. 2019-04816 Filed 3-14-19; 8:45 am]
 BILLING CODE 4164-01-P


