Notice2023-28310
Rare Diseases: Considerations for the Development of Drugs and Biological Products; Guidance for Industry; Availability
Primary source
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Published
December 26, 2023
Issuing agencies
Health and Human Services DepartmentFood and Drug Administration
Abstract
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 This guidance finalizes the draft guidance entitled "Rare Diseases: Common Issues in Drug Development" issued on February 1, 2019.
Full Text
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<title>Federal Register, Volume 88 Issue 246 (Tuesday, December 26, 2023)</title>
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[Federal Register Volume 88, Number 246 (Tuesday, December 26, 2023)]
[Notices]
[Pages 88924-88926]
From the Federal Register Online via the Government Publishing Office [<a href="http://www.gpo.gov">www.gpo.gov</a>]
[FR Doc No: 2023-28310]
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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.
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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 This guidance finalizes the draft guidance entitled ``Rare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:
<bullet> Federal eRulemaking Portal: <a href="https://www.regulations.gov">https://www.regulations.gov</a>.
Follow the instructions for submitting comments. Comments submitted
electronically, including attachments, to <a href="https://www.regulations.gov">https://www.regulations.gov</a>
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
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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 <a href="https://www.regulations.gov">https://www.regulations.gov</a>.
<bullet> 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:
<bullet> 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.
<bullet> 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 <a href="https://www.regulations.gov">https://www.regulations.gov</a> or at
the Dockets Management Staff between 9 a.m. and 4 p.m., Monday through
Friday, 240-402-7500.
<bullet> 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 <a href="https://www.regulations.gov">https://www.regulations.gov</a>.
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: <a href="https://www.govinfo.gov/content/pkg/FR-2015-09-18/pdf/2015-23389.pdf">https://www.govinfo.gov/content/pkg/FR-2015-09-18/pdf/2015-23389.pdf</a>.
Docket: For access to the docket to read background documents or
the electronic and written/paper comments received, go to <a href="https://www.regulations.gov">https://www.regulations.gov</a> 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:
<bullet> 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);
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<bullet> 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
<bullet> 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.
<bullet> Submissions under 21 CFR part 314, subpart H, to grant
accelerated approval of new drugs for serious or life-threatening
illnesses.
<bullet> 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:
<bullet> 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.
<bullet> Submitting under Sec. 312.23(a)(3)(i) a brief
introductory statement and general investigational plan, including the
route of administration of a drug;
<bullet> 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;
<bullet> Submitting under Sec. 312.23(a)(8) and (a)(8)(i)
pharmacology, toxicology, and drug disposition information for rare
diseases;
<bullet> Submitting under 312.23(a)(10)(iii) plans for assessing
pediatric safety and effectiveness;
<bullet> Submitting under Sec. 312.32(c)(1) IND safety reports;
<bullet> 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
<a href="https://www.fda.gov/drugs/guidance-compliance-regulatory-information/guidances-drugs">https://www.fda.gov/drugs/guidance-compliance-regulatory-information/guidances-drugs</a>, <a href="https://www.fda.gov/vaccines-blood-biologics/guidance-compliance-regulatory-information-biologics">https://www.fda.gov/vaccines-blood-biologics/guidance-compliance-regulatory-information-biologics</a>, <a href="https://www.fda.gov/regulatory-information/search-fda-guidance-documents">https://www.fda.gov/regulatory-information/search-fda-guidance-documents</a>, or <a href="https://www.regulations.gov">https://www.regulations.gov</a>.
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
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</html>Indexed from Federal Register on December 26, 2023.
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