Agency Information Collection Activities; Proposed Collection; Comment Request; Orphan Drugs

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Published

April 16, 2026

Issuing agencies

Health and Human Services DepartmentFood and Drug Administration

Abstract

The Food and Drug Administration (FDA, Agency, or we) is announcing an opportunity for public comment on the proposed collection of certain information by the Agency. Under the Paperwork Reduction Act of 1995 (PRA), Federal Agencies are required to publish notice in the Federal Register concerning each proposed collection of information, including each proposed extension of an existing collection of information, and to allow 60 days for public comment in response to the notice. This notice solicits comments on information collection associated with orphan drug requirements.

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<title>Federal Register, Volume 91 Issue 73 (Thursday, April 16, 2026)</title>
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[Federal Register Volume 91, Number 73 (Thursday, April 16, 2026)]
[Notices]
[Pages 20460-20462]
From the Federal Register Online via the Government Publishing Office [<a href="http://www.gpo.gov">www.gpo.gov</a>]
[FR Doc No: 2026-07423]

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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2026-N-3400]

Agency Information Collection Activities; Proposed Collection;
Comment Request; Orphan Drugs

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice.

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SUMMARY: The Food and Drug Administration (FDA, Agency, or we) is
announcing an opportunity for public comment on the proposed collection
of certain information by the Agency. Under the Paperwork Reduction Act
of 1995 (PRA), Federal Agencies are required to publish notice in the
Federal Register concerning each proposed collection of information,
including each proposed extension of an existing collection of
information, and to allow 60 days for public comment in response to the
notice. This notice solicits comments on information collection
associated with orphan drug requirements.

DATES: Either electronic or written comments on the collection of
information must be submitted by June 15, 2026.

ADDRESSES: You may submit comments as follows. Please note that late,
untimely filed comments will not be considered. The <a href="https://www.regulations.gov">https://www.regulations.gov</a> electronic filing system will accept comments until
11:59 p.m. Eastern Time at the end of June 15, 2026. Comments received
by mail/hand delivery/courier (for written/paper submissions) will be
considered timely if they are received on or before that date.

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 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-2023-N-1929 for ``Orphan Drug Designation.'' Received comments,
those filed in a timely manner (see ADDRESSES), 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

[[Page 20461]]

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.

FOR FURTHER INFORMATION CONTACT: Amber Barrett, Office of Operations,
Food and Drug Administration, Three White Flint North, 10A-12M, 11601
Landsdown St., North Bethesda, MD 20852, 301-796-8867,
<a href="/cdn-cgi/l/email-protection#18484a594b6c797e7e587e7c793670706b367f776e"><span class="__cf_email__" data-cfemail="dd8d8f9c8ea9bcbbbb9dbbb9bcf3b5b5aef3bab2ab">[email&#160;protected]</span></a>.

SUPPLEMENTARY INFORMATION: Under the PRA (44 U.S.C. 3501-3521), Federal
Agencies must obtain approval from the Office of Management and Budget
(OMB) for each collection of information they conduct or sponsor.
``Collection of information'' is defined in 44 U.S.C. 3502(3) and 5 CFR
1320.3(c) and includes Agency requests or requirements that members of
the public submit reports, keep records, or provide information to a
third party. Section 3506(c)(2)(A) of the PRA (44 U.S.C. 3506(c)(2)(A))
requires Federal Agencies to provide a 60-day notice in the Federal
Register concerning each proposed collection of information, including
each proposed extension of an existing collection of information,
before submitting the collection to OMB for approval. To comply with
this requirement, FDA is publishing notice of the proposed collection
of information set forth in this document.
With respect to the following collection of information, FDA
invites comments on these topics: (1) whether the proposed collection
of information is necessary for the proper performance of FDA's
functions, including whether the information will have practical
utility; (2) the accuracy of FDA's estimate of the burden of the
proposed collection of information, including the validity of the
methodology and assumptions used; (3) ways to enhance the quality,
utility, and clarity of the information to be collected; and (4) ways
to minimize the burden of the collection of information on respondents,
including through the use of automated collection techniques, when
appropriate, and other forms of information technology.

Orphan Drugs--21 CFR Part 316

OMB Control Number 0910-0167--Extension

This information collection helps support implementation of
sections 525, 526, 527, and 528 of the Federal Food, Drug, and Cosmetic
Act (FD&C Act) (21 U.S.C. 360aa, 360bb, 360cc, and 360dd), as well as
related guidance and Agency forms. Sections 525, 526, 527, and 528 of
the FD&C Act pertain to the development of drugs for rare diseases or
conditions, including biological products and antibiotics, otherwise
known or referred to as ``orphan drugs.'' Specifically, section 525 of
the FD&C Act requires written recommendations on studies required for
approval of a marketing application for a drug for a rare disease or
condition. Section 526 of the FD&C Act provides for designation of
drugs as orphan drugs when certain conditions are met; section 527
provides conditions under which a sponsor of an approved orphan drug
enjoys exclusive FDA marketing approval for that drug for the orphan
indication for a period of 7 years; and, finally, section 528 is
intended to encourage sponsors to make investigational orphan drugs
available for treatment of persons in need on an open protocol basis
before the drug has been approved for general marketing. Open protocols
may permit patients who are not part of the formal clinical
investigation to obtain treatment where adequate supplies exist and no
alternative effective therapy is available.
Agency regulations in part 316, subpart A (21 CFR part 316, subpart
A) (Sec. Sec. 316.1 through 316.4) identify the scope of coverage,
applicable definitions, and statutory provisions applicable to orphan
drugs. The regulations in part 316, subpart B (Sec. Sec. 316.10
through 316.14) set forth content and format elements for written
recommendation requests and discuss FDA providing or refusing to
provide the requested written recommendations. Similarly, regulations
in part 316, subpart C (Sec. Sec. 316.20 through 316.30) prescribe
content and format elements for requesting orphan drug designation;
identify submission schedules for requisite information including
amendments, updates, and reports; and provide for publication and
revocation of orphan drug designation. Regulations in part 316,
subparts D and E (Sec. Sec. 316.31 through 316.40) address orphan drug
exclusive approval and open protocols for investigations, respectively.
Finally, regulations in part 316, subpart F (Sec. Sec. 316.50 through
316.52) provide for the issuance of guidance documents that apply to
the orphan drug provisions of the FD&C Act and regulations in part 316.
The list is maintained on the internet and guidance documents are
issued in accordance with our good guidance practices regulation in 21
CFR 10.115, which provide for public comment at any time.
The information collection includes the Agency guidance document
entitled ``Meetings with the Office of Orphan Products Development:
Guidance for Industry, Researchers, Patient Groups, and Food and Drug
Administration Staff'' (July 2015), available for download at: <a href="https://www.fda.gov/regulatory-information/search-fda-guidance-documents/meetings-office-orphan-products-development">https://www.fda.gov/regulatory-information/search-fda-guidance-documents/meetings-office-orphan-products-development</a>. It provides
recommendations to industry, researchers, patient groups, and other
stakeholders interested in requesting a meeting, including a
teleconference, with the Office of Orphan Products Development (OOPD)
on issues related to orphan drug designation requests, humanitarian use
device designation requests, rare pediatric disease designation
requests, funding opportunities through the Orphan Products Grants
Program and the Pediatric Device Consortia Grants Program, and orphan
product patient-related topics of concern. It is also intended to
assist OOPD staff in addressing such meeting requests. The guidance
describes procedures for requesting, preparing, scheduling, conducting,
and documenting such meetings and discusses background information we
recommend be included in such requests.
The information collection includes Form FDA 4035, FDA Orphan Drug

[[Page 20462]]

Designation Request Form, intended to benefit sponsors who desire to
seek orphan designation of drugs intended for rare diseases or
conditions from FDA. The form is a simplified method for sponsors to
provide only the information required by Sec. 316.20 for FDA decision
making. Orphan drug designation requests and related submissions
(amendments, annual reports, etc.), humanitarian use device
designation, and rare pediatric disease designation requests and
submissions may be submitted electronically by email to the OOPD.
As communicated on our website at <a href="https://www.fda.gov/industry/medical-products-rare-diseases-and-conditions/designating-orphan-product-drugs-and-biological-products">https://www.fda.gov/industry/medical-products-rare-diseases-and-conditions/designating-orphan-product-drugs-and-biological-products</a>, respondents may submit orphan
drug designation requests electronically through the Center for Drug
Evaluation and Research (CDER) NextGen portal, or by emailing the
required information to <a href="/cdn-cgi/l/email-protection#0e617c7e666f604e686a6f2066667d20696178"><span class="__cf_email__" data-cfemail="462934362e272806202227682e2e3568212930">[email&#160;protected]</span></a>; or by mailing the required
information to the OOPD at the address found on our website. New users
of the CDER NextGen Portal must register for an account. For
designation requests submitted by email, the Agency recommends using
automated read receipt to verify receipt of the email.
Sponsors and others who plan to email information to FDA that is
private, sensitive, proprietary, or commercial confidential are
strongly encouraged to send it from an FDA-secured email address so the
transmission is encrypted. The Agency will assume the addresses of
emails received or email addresses provided as a point of contact are
secure when responding to those email addresses. Sponsors and others
can establish a secure email address link to FDA by sending a request
to <a href="/cdn-cgi/l/email-protection#b9eadcdacccbdcfcd4d8d0d5f9dfddd897d1d1ca97ded6cf"><span class="__cf_email__" data-cfemail="550630362027301038343c39153331347b3d3d267b323a23">[email&#160;protected]</span></a>. There may be a fee to a commercial
enterprise for establishing a digital certificate before encrypted
emails can be sent to FDA.
Respondents to the information collection are sponsors who develop
investigational drugs and biologicals for commercial use and who seek
orphan drug designation, and upon approval or licensure, orphan drug
exclusivity.
We estimate the burden of this collection of information as
follows:

Table 1--Estimated Annual Recordkeeping Burden \1\
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Number of Average
21 CFR part or section; activity Number of records per Total annual burden per Total hours
respondents recordkeeper records record
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Part 316 associated records..... 864 1 1,080 135 145,800
Sec. Sec. 316.20, 316.21, 864 1 1,080 32 34,560
316.26 (Form FDA 4035).........
Sec. 316.22; Notifications of 305 1 305 0.5 153
changes in agents..............
Sec. 316.24(a); Deficiency 756 1 756 2 1,512
letters and granting orphan-
drug designation...............
Sec. 316.27; Submissions to 110 1 110 3 330
change ownership of orphan-drug
designation....................
Sec. 316.30; Annual reports... 2,210 1 2,210 3 6,630
Sec. 316.36; Assurance of the 1 3 3 15 45
availability of sufficient
quantities of the orphan drug;
holder's consent for the
approval of other marketing
applications for the same drug.
Guidance Recommendations: 800 1.5 1,200 4 4,800
Meeting requests to OOPD and
related submission packages....
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Total....................... .............. .............. 6,744 .............. 193,830
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\1\ There are no capital costs or operating and maintenance costs associated with this collection of
information.

We base our burden figures on the number of submissions received
and include those activities relating to: (1) requesting orphan drug
designation; (2) responding to deficiencies letters with submissions of
amendments; (3) keeping files current with contact information for
agents and transfer of ownership, when applicable; (4) submitting
annual reports while products have designation status; and (5)
requesting and preparing for both informal and formal meetings. Because
the PRA defines a recordkeeping requirement to include reporting those
records to the Federal government, we account for these activities
cumulatively in table 1 above. Our burden estimate reflects a nominal
increase of approximately 9% annually.

Grace R. Graham,
Deputy Commissioner for Policy, Legislation, and International Affairs.
[FR Doc. 2026-07423 Filed 4-15-26; 8:45 am]
BILLING CODE 4164-01-P

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