Rare Diseases: Considerations for the Development of Drugs and Biological Products; Guidance for Industry; Availability, 88924-88926 [2023-28310]
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88924
Federal Register / Vol. 88, No. 246 / Tuesday, December 26, 2023 / Notices
Supply Chain Security Act (FASCSA)
Orders. Comments received generally
will be posted without change to
https://www.regulations.gov, including
any personal and/or business
confidential information provided. To
confirm receipt of your comment(s),
please check www.regulations.gov,
approximately two-to-three days after
submission to verify posting.
FOR FURTHER INFORMATION CONTACT:
Marissa Ryba, Procurement Analyst, at
telephone 314–586–1280, or
Marissa.Ryba@gsa.gov.
SUPPLEMENTARY INFORMATION:
A. OMB Control Number, Title, and any
Associated Form(s)
9000–0205, Implementation of
Federal Acquisition Supply Chain
Security Act (FASCSA) Orders
khammond on DSKJM1Z7X2PROD with NOTICES
B. Need and Uses
This clearance covers the information
that offerors and contractors must
submit to comply with the following
FAR requirements:
a. FAR 52.204–29, Federal
Acquisition Supply Chain Security Act
Orders-Representation and Disclosures.
This provision prohibits contractors
from providing or using as part of the
performance of the contract any covered
article, or any products or services
produced or provided by a source, if the
covered article or the source is subject
to an applicable FASCSA order
identified in the clause at FAR 52.204–
30(b)(1).
By submitting an offer, offerors are
representing compliance with the
prohibition. If an offeror cannot
represent compliance with the
prohibition, then the offeror must
disclose the following information in
accordance with 52.204–29(e):
(1) Name of the product or service
provided to the Government;
(2) Name of the covered article or
source subject to an FASCA order;
(3) If applicable, name of the vendor,
including the Commercial and
Government Entity code and unique
entity identifier (if known), that
supplied the covered article or the
product or service to the Offeror;
(4) Brand;
(5) Model number (original equipment
manufacturer number, manufacturer
part number, or wholesaler number);
(6) Item description;
(7) Reason why the applicable
covered article or the product or service
is being provided;
b. FAR 52.204–30, Federal
Acquisition Supply Chain Security Act
Orders-Prohibition. This clause requires
contractors to provide a report to the
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Government within 3 business days if
the contractor identifies that the
contractor or any-tier subcontractor,
delivered or used a covered article or
product or service subject to a FASCSA
order. The report requires the following
information:
(1) Contract number;
(2) Order number(s), if applicable;
(3) Name of the product or service
provided to the Government;
(4) Name of the covered article or
source subject to a FASCSA order;
(5) If applicable, name of the vendor,
including the Commercial and
Government Entity code and unique
entity identifier (if known), that
supplied the covered article or the
product or service to the Contractor;
(6) Brand;
(7) Model number (original equipment
manufacturer number, manufacturer
part number, or wholesaler number);
(8) Item description; and
(9) Any readily available information
about mitigation actions undertaken or
recommended.
The contractor must also submit
additional information within 10 days
of submitting the first report identifying
any further available information about
mitigation actions undertaken or
recommended. Additionally, the
contractor shall describe the efforts it
undertook to prevent submission and
any additional efforts to prevent future
submission of the covered article or the
product or service produced or provided
by a source subject to an applicable
FASCSA order.
FAR provision 52.204–29.
Information collected under will be by
the government to determine whether to
seek a waiver from a FASCSA order
issued under the authority of the
Federal Acquisition Supply Chain
Security Act of 2018.
FAR clause 52.204–30 will
Information collected will be used by
the contracting officer working with the
requirement activity to determine
whether it is necessary to take further
action and modify the contract.
C. Annual Burden
Respondents/Recordkeepers: 6,113.
Total Annual Responses: 1.
Total Burden Hours: 12,226.
Obtaining Copies: Requesters may
obtain a copy of the information
collection documents from the GSA
Regulatory Secretariat Division by
calling 202–501–4755 or emailing
GSARegSec@gsa.gov. Please cite OMB
Control No. 9000–0205, Implementation
PO 00000
Frm 00061
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Sfmt 4703
of Federal Acquisition Supply Chain
Security Act (FASCSA) Orders.
Janet Fry,
Director, Federal Acquisition Policy Division,
Office of Governmentwide Acquisition Policy,
Office of Acquisition Policy, Office of
Governmentwide Policy.
[FR Doc. 2023–28419 Filed 12–22–23; 8:45 am]
BILLING CODE 6820–EP–P
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA–2015–D–2818]
Rare Diseases: Considerations for the
Development of Drugs and Biological
Products; Guidance for Industry;
Availability
AGENCY:
Food and Drug Administration,
HHS.
ACTION:
Notice of availability.
The Food and Drug
Administration (FDA or Agency) is
announcing the availability of a final
guidance for industry entitled ‘‘Rare
Diseases: Considerations for the
Development of Drugs and Biological
Products.’’ This guidance is intended to
assist sponsors of drugs and biological
products for treatment of rare diseases
in conducting efficient and successful
drug development programs through a
discussion of selected issues commonly
encountered in rare disease drug
development. This guidance finalizes
the draft guidance entitled ‘‘Rare
Diseases: Common Issues in Drug
Development’’ issued on February 1,
2019.
DATES: The announcement of the
guidance is published in the Federal
Register on December 26, 2023.
ADDRESSES: You may submit either
electronic or written comments on
Agency guidances at any time as
follows:
SUMMARY:
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
E:\FR\FM\26DEN1.SGM
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Federal Register / Vol. 88, No. 246 / Tuesday, December 26, 2023 / Notices
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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–
2015–D–2818 for ‘‘Rare Diseases:
Considerations for the Development of
Drugs and Biological Products.’’
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, 240–402–7500.
• 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
VerDate Sep<11>2014
20:25 Dec 22, 2023
Jkt 262001
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.govinfo.gov/content/pkg/FR-201509-18/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, 240–402–7500.
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; or the Office of Communication,
Outreach, and Development, Center for
Biologics Evaluation and Research,
Food and Drug Administration, 10903
New Hampshire Ave., Bldg. 71, Rm.
3128, 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:
Andrea Bell-Vlasov, Center for Drug
Evaluation and Research, Food and
Drug Administration, 10903 New
Hampshire Ave., Bldg. 22, Rm. 6484,
Silver Spring, MD 20993–0002, 240–
402–4977; or Anne Taylor, Center for
Biologics Evaluation and Research,
Food and Drug Administration, 10903
New Hampshire Ave., Bldg. 71, Rm.
7301, Silver Spring, MD 20993–0002,
240–402–7911.
SUPPLEMENTARY INFORMATION:
I. Background
FDA is announcing the availability of
a final guidance for industry entitled
‘‘Rare Diseases: Considerations for the
Development of Drugs and Biological
Products.’’ This guidance is intended to
assist sponsors of drugs and biological
products for treatment of rare diseases
in conducting efficient and successful
product development programs through
a discussion of selected issues
PO 00000
Frm 00062
Fmt 4703
Sfmt 4703
88925
commonly encountered in rare disease
drug development. This final guidance
addresses important aspects of drug and
biological product development to
support the proposed clinical
investigation(s), including nonclinical
pharmacology/toxicology; trial design
and endpoint considerations to ensure
quality and interpretability of data;
standard of evidence to establish safety
and effectiveness; and drug
manufacturing considerations during
drug development.
This guidance finalizes the draft
guidance entitled ‘‘Rare Diseases:
Common Issues in Drug Development’’
issued February 1, 2019 (84 FR 1156).
Changes made from the draft to the final
guidance took into consideration
comments received. Major changes
include the removal of the natural
history section (because this is
addressed in a separate guidance),
additional considerations regarding
nonclinical studies, information on the
use of external controls and early
randomization, a section discussing
safety considerations, information on
changes to drug substance or
manufacturing process, and sections
discussing participation of patients/
patient groups in drug development
programs and pediatric considerations
in rare disease drug development.
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 ‘‘Rare Diseases:
Considerations for the Development of
Drugs and Biological Products.’’ 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.
II. Paperwork Reduction Act of 1995
While this guidance contains no
collection of information, it does refer to
previously approved FDA collections of
information. The previously approved
collections of information are subject to
review by the Office of Management and
Budget (OMB) under the Paperwork
Reduction Act of 1995 (PRA) (44 U.S.C.
3501–3521).
The following collections of
information in the final guidance have
been approved under OMB control
number 0910–0001:
• Submitting under 21 CFR
314.50(c)(1)(iv) and (d)(3)
(§ 314.50(c)(1)(iv) and (d)(3)) a summary
of the nonclinical pharmacology and
toxicology section and the human
pharmacokinetics and bioavailability
section of new drug application (NDAs);
E:\FR\FM\26DEN1.SGM
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88926
Federal Register / Vol. 88, No. 246 / Tuesday, December 26, 2023 / Notices
• Submitting under § 314.50(d)(1)(i)
chemistry, manufacturing, and controls
information, including the drug
substance, for the content and format of
an NDA for rare diseases; and
• Submitting under § 314.50(d)(5) and
(d)(5)(iv) clinical data of a drug,
including a description of any other
data information relevant to an
evaluation of the safety and
effectiveness of a drug.
• Submissions under 21 CFR part
314, subpart H, to grant accelerated
approval of new drugs for serious or
life-threatening illnesses.
• Submissions under §§ 312.47 and
312.82 for requesting meetings with
FDA about drug development programs.
The following collections of
information in the final guidance have
been approved under OMB control
number 0910–0014:
• Submitting under 21 CFR
312.23(a)(6)(i) (§ 312.23(a)(6)(i)) a
protocol for the duration of a trial and
the criteria to enter a trial and under
§ 312.23(a)(6)(i), (a)(6)(iii)(d) and (g) a
description of an estimate of patients
that will be involved in a trial,
including a description of the safety
exclusions and a description of clinical
procedures, laboratory, or other
methods.
• Submitting under § 312.23(a)(3)(i) a
brief introductory statement and general
investigational plan, including the route
of administration of a drug;
• Submitting under § 312.23(a)(7) and
(a)(7)(iv)(a) chemistry, manufacturing,
and controls information for the content
and format of an investigational new
drug application (IND) and the safety
and effectiveness of such information;
• Submitting under § 312.23(a)(8) and
(a)(8)(i) pharmacology, toxicology, and
drug disposition information for rare
diseases;
• Submitting under 312.23(a)(10)(iii)
plans for assessing pediatric safety and
effectiveness;
• Submitting under § 312.32(c)(1) IND
safety reports;
• Submissions under §§ 312.305(b)
and 312.310(b) for expanded access uses
and treatment of an individual patient.
The collections of information in 21
CFR part 316 for submitting the content
and format of NDAs for orphan drugs
have been approved under OMB control
number 0910–0167. The collections of
information pertaining to postmarketing
adverse drug experience reporting have
been approved under OMB control
number 0910–0230. The collections of
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20:25 Dec 22, 2023
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information pertaining to expedited
review programs for serious conditions,
accelerated approval, breakthrough
therapy-designation, and fast-track
designation, have been approved under
OMB control number 0910–0765. The
collections of information in 21 CFR
part 58 pertaining to good laboratory
practices have been approved under
OMB control number 0910–0119. The
collection of information pertaining to
current good manufacturing practices
have been approved under OMB control
number 0910–0139.
III. Electronic Access
Persons with access to the internet
may obtain the guidance at https://
www.fda.gov/drugs/guidancecompliance-regulatory-information/
guidances-drugs, https://www.fda.gov/
vaccines-blood-biologics/guidancecompliance-regulatory-informationbiologics, https://www.fda.gov/
regulatory-information/search-fdaguidance-documents, or https://
www.regulations.gov.
Dated: December 18, 2023.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2023–28310 Filed 12–22–23; 8:45 am]
BILLING CODE 4164–01–P
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
[Document Identifier: OS–0990–30D]
Agency Information Collection
Request. 30-Day Public Comment
Request
Office of the Secretary, HHS.
Notice.
AGENCY:
ACTION:
In compliance with the
requirement of the Paperwork
Reduction Act of 1995, the Office of the
Secretary (OS), Department of Health
and Human Services, is publishing the
following summary of a proposed
collection for public comment.
DATES: Comments on the ICR must be
received on or before January 25, 2024.
ADDRESSES: Submit your comments to
Sherrette.Funn@hhs.gov or by calling
(202) 264–0041 and PRA@HHS.GOV
FOR FURTHER INFORMATION CONTACT:
When submitting comments or
requesting information, please include
the document identifier 0990–0313 and
project title for reference, to Sherrette A.
Funn, email: Sherrette.Funn@hhs.gov,
SUMMARY:
PO 00000
Frm 00063
Fmt 4703
Sfmt 4703
PRA@HHS.GOV or call (202) 264–0041
the Reports Clearance Officer.
Interested
persons are invited to send comments
regarding this burden estimate or any
other aspect of this collection of
information, including any of the
following subjects: (1) The necessity and
utility of the proposed information
collection for the proper performance of
the agency’s functions; (2) the accuracy
of the estimated burden; (3) ways to
enhance the quality, utility, and clarity
of the information to be collected; and
(4) the use of automated collection
techniques or other forms of information
technology to minimize the information
collection burden.
Title of the Collection: National Blood
Collection & Utilization Survey
(NBCUS)
Type of Collection: Revision
OMB No. 0990–30D–0313 Office of
the Assistant Secretary for Health/HHS
SUPPLEMENTARY INFORMATION:
Abstract
The Office of the Assistant Secretary
for Health (OASH) is requesting
approval for a three-year revised
information collection request (ICR)
titled ‘‘National Blood Collection &
Utilization Survey (NBCUS).’’ The
NBCUS is a biennial survey that
includes a core of standard questions on
blood collection, processing, and
utilization practices. Questions on
transfusion-transmitted infections,
transfusion associated circulatory
overload, acute hemolysis, delayed
hemolysis, and severe allergic reactions
are also included in the survey. The
rapidly changing environment in blood
supply and demand makes it important
to have regular, periodic data describing
the state of U.S. blood collections and
transfusions for understanding the
dynamics of blood safety and
availability. In 2023, two sections were
removed from the survey related to the
impact of the COVID–19 pandemic on
the blood supply during the course of
2020.
Survey respondents will consist of
blood collection centers and hospitals
that perform blood transfusions, except
those reporting fewer than 100 inpatient
surgeries per year. For the purposes of
this ICR, federal burden is only being
placed on facilities located within the
fifty states and the District of Columbia.
The total estimated burden is 5,106
hours.
E:\FR\FM\26DEN1.SGM
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]]>Agencies
[Federal Register Volume 88, Number 246 (Tuesday, December 26, 2023)]
[Notices]
[Pages 88924-88926]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2023-28310]
=======================================================================
-----------------------------------------------------------------------
DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2015-D-2818]
Rare Diseases: Considerations for the Development of Drugs and
Biological Products; 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 ``Rare
Diseases: Considerations for the Development of Drugs and Biological
Products.'' This guidance is intended to assist sponsors of drugs and
biological products for treatment of rare diseases in conducting
efficient and successful drug development programs through a discussion
of selected issues commonly encountered in rare disease drug
development. This guidance finalizes the draft guidance entitled ``Rare
Diseases: Common Issues in Drug Development'' issued on February 1,
2019.
DATES: The announcement of the guidance is published in the Federal
Register on December 26, 2023.
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
[[Page 88925]]
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-2015-D-2818 for ``Rare Diseases: Considerations for the Development
of Drugs and Biological Products.'' 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, 240-402-7500.
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.govinfo.gov/content/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, 240-402-7500.
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; or the Office of
Communication, Outreach, and Development, Center for Biologics
Evaluation and Research, Food and Drug Administration, 10903 New
Hampshire Ave., Bldg. 71, Rm. 3128, 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: Andrea Bell-Vlasov, Center for Drug
Evaluation and Research, Food and Drug Administration, 10903 New
Hampshire Ave., Bldg. 22, Rm. 6484, Silver Spring, MD 20993-0002, 240-
402-4977; or Anne Taylor, Center for Biologics Evaluation and Research,
Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 71, Rm.
7301, Silver Spring, MD 20993-0002, 240-402-7911.
SUPPLEMENTARY INFORMATION:
I. Background
FDA is announcing the availability of a final guidance for industry
entitled ``Rare Diseases: Considerations for the Development of Drugs
and Biological Products.'' This guidance is intended to assist sponsors
of drugs and biological products for treatment of rare diseases in
conducting efficient and successful product development programs
through a discussion of selected issues commonly encountered in rare
disease drug development. This final guidance addresses important
aspects of drug and biological product development to support the
proposed clinical investigation(s), including nonclinical pharmacology/
toxicology; trial design and endpoint considerations to ensure quality
and interpretability of data; standard of evidence to establish safety
and effectiveness; and drug manufacturing considerations during drug
development.
This guidance finalizes the draft guidance entitled ``Rare
Diseases: Common Issues in Drug Development'' issued February 1, 2019
(84 FR 1156). Changes made from the draft to the final guidance took
into consideration comments received. Major changes include the removal
of the natural history section (because this is addressed in a separate
guidance), additional considerations regarding nonclinical studies,
information on the use of external controls and early randomization, a
section discussing safety considerations, information on changes to
drug substance or manufacturing process, and sections discussing
participation of patients/patient groups in drug development programs
and pediatric considerations in rare disease drug development.
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 ``Rare Diseases: Considerations for the
Development of Drugs and Biological Products.'' 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.
II. Paperwork Reduction Act of 1995
While this guidance contains no collection of information, it does
refer to previously approved FDA collections of information. The
previously approved collections of information are subject to review by
the Office of Management and Budget (OMB) under the Paperwork Reduction
Act of 1995 (PRA) (44 U.S.C. 3501-3521).
The following collections of information in the final guidance have
been approved under OMB control number 0910-0001:
Submitting under 21 CFR 314.50(c)(1)(iv) and (d)(3) (Sec.
314.50(c)(1)(iv) and (d)(3)) a summary of the nonclinical pharmacology
and toxicology section and the human pharmacokinetics and
bioavailability section of new drug application (NDAs);
[[Page 88926]]
Submitting under Sec. 314.50(d)(1)(i) chemistry,
manufacturing, and controls information, including the drug substance,
for the content and format of an NDA for rare diseases; and
Submitting under Sec. 314.50(d)(5) and (d)(5)(iv)
clinical data of a drug, including a description of any other data
information relevant to an evaluation of the safety and effectiveness
of a drug.
Submissions under 21 CFR part 314, subpart H, to grant
accelerated approval of new drugs for serious or life-threatening
illnesses.
Submissions under Sec. Sec. 312.47 and 312.82 for
requesting meetings with FDA about drug development programs.
The following collections of information in the final guidance have
been approved under OMB control number 0910-0014:
Submitting under 21 CFR 312.23(a)(6)(i) (Sec.
312.23(a)(6)(i)) a protocol for the duration of a trial and the
criteria to enter a trial and under Sec. 312.23(a)(6)(i),
(a)(6)(iii)(d) and (g) a description of an estimate of patients that
will be involved in a trial, including a description of the safety
exclusions and a description of clinical procedures, laboratory, or
other methods.
Submitting under Sec. 312.23(a)(3)(i) a brief
introductory statement and general investigational plan, including the
route of administration of a drug;
Submitting under Sec. 312.23(a)(7) and (a)(7)(iv)(a)
chemistry, manufacturing, and controls information for the content and
format of an investigational new drug application (IND) and the safety
and effectiveness of such information;
Submitting under Sec. 312.23(a)(8) and (a)(8)(i)
pharmacology, toxicology, and drug disposition information for rare
diseases;
Submitting under 312.23(a)(10)(iii) plans for assessing
pediatric safety and effectiveness;
Submitting under Sec. 312.32(c)(1) IND safety reports;
Submissions under Sec. Sec. 312.305(b) and 312.310(b) for
expanded access uses and treatment of an individual patient.
The collections of information in 21 CFR part 316 for submitting
the content and format of NDAs for orphan drugs have been approved
under OMB control number 0910-0167. The collections of information
pertaining to postmarketing adverse drug experience reporting have been
approved under OMB control number 0910-0230. The collections of
information pertaining to expedited review programs for serious
conditions, accelerated approval, breakthrough therapy-designation, and
fast-track designation, have been approved under OMB control number
0910-0765. The collections of information in 21 CFR part 58 pertaining
to good laboratory practices have been approved under OMB control
number 0910-0119. The collection of information pertaining to current
good manufacturing practices have been approved under OMB control
number 0910-0139.
III. Electronic Access
Persons with access to the internet may obtain the guidance at
https://www.fda.gov/drugs/guidance-compliance-regulatory-information/guidances-drugs, https://www.fda.gov/vaccines-blood-biologics/guidance-compliance-regulatory-information-biologics, https://www.fda.gov/regulatory-information/search-fda-guidance-documents, or https://www.regulations.gov.
Dated: December 18, 2023.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2023-28310 Filed 12-22-23; 8:45 am]
BILLING CODE 4164-01-P
