Agency Information Collection Activities; Submission for Office of Management and Budget Review; Comment Request; Orphan Drugs

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Published

September 21, 2026

Issuing agencies

Health and Human Services DepartmentFood and Drug Administration

Abstract

The Food and Drug Administration (FDA, Agency, or we) is announcing that a proposed collection of information has been submitted to the Office of Management and Budget (OMB) for review and clearance under the Paperwork Reduction Act of 1995.

Full Text

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<title>Federal Register, Volume 91 Issue 181 (Monday, September 21, 2026)</title>
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[Federal Register Volume 91, Number 181 (Monday, September 21, 2026)]
[Notices]
[Pages 59780-59781]
From the Federal Register Online via the Government Publishing Office [<a href="http://www.gpo.gov">www.gpo.gov</a>]
[FR Doc No: 2026-19284]

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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

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

Agency Information Collection Activities; Submission for Office
of Management and Budget Review; 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 that a proposed collection of information has been submitted
to the Office of Management and Budget (OMB) for review and clearance
under the Paperwork Reduction Act of 1995.

DATES: Submit written comments (including recommendations) on the
collection of information by October 21, 2026.

ADDRESSES: To ensure that comments on the information collection are
received, OMB recommends that written comments be submitted to <a href="https://www.reginfo.gov/public/do/PRAMain">https://www.reginfo.gov/public/do/PRAMain</a>. Find this particular information
collection by selecting ``Currently under Review--Open for Public
Comments'' or by using the search function. The OMB control number for
this information collection is 0910-0167. Also include the FDA docket
number found in brackets in the heading of this document.

FOR FURTHER INFORMATION CONTACT: Domini Bean, Office of Operations,
Food and Drug Administration, Three White Flint North, 10A-12M, 11601
Landsdown St., North Bethesda, MD 20852, 240-402-5733,
<a href="/cdn-cgi/l/email-protection#623230233116030404220406034c0a0a114c050d14"><span class="__cf_email__" data-cfemail="81d1d3c0d2f5e0e7e7c1e7e5e0afe9e9f2afe6eef7">[email&#160;protected]</span></a>.

SUPPLEMENTARY INFORMATION: In compliance with 44 U.S.C. 3507, FDA has
submitted the following proposed collection of information to OMB for
review and clearance.

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
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#cca3bebca4ada28caaa8ade2a4a4bfe2aba3ba"><span class="__cf_email__" data-cfemail="cea1bcbea6afa08ea8aaafe0a6a6bde0a9a1b8">[email&#160;protected]</span></a>; or by mailing the required
information to the OOPD at the address

[[Page 59781]]

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#653600061017002008040c09250301044b0d0d164b020a13"><span class="__cf_email__" data-cfemail="fba89e988e899ebe969a9297bb9d9f9ad5939388d59c948d">[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.
In the Federal Register of April 16, 2026 (91 FR 20460), we
published a 60-day notice requesting public comment on the proposed
collection of information. One comment was received encouraging FDA to
offer more incentives toward the development of drug products to treat
specific conditions and we appreciate this comment. At the same time,
the comment did not suggest any modification to the estimates in our
60-day notice nor appear to question the Agency's need for the
information collection.
We estimate the burden of this collection of information as
follows:

Table 1--Estimated Annual Recordkeeping Burden \1\
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Average
Number of Number of Total burden Total
21 CFR part or section; activity respondents records per annual per hours
recordkeeper records record
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Part 316 associated records..................... 864 1 1,080 135 145,800
Sec. Sec. 316.20, 316.21, 316.26 (Form FDA 864 1 1,080 32 34,560
4035)..........................................
Sec. 316.22; Notifications of changes in 305 1 305 0.5 153
agents.........................................
Sec. 316.24(a); Deficiency letters and 756 1 756 2 1,512
granting orphan-drug designation...............
Sec. 316.27; Submissions to change ownership 110 1 110 3 330
of orphan-drug designation.....................
Sec. 316.30; Annual reports................... 2,210 1 2,210 3 6,630
Sec. 316.36; Assurance of the availability of 1 3 3 15 45
sufficient quantities of the orphan drug;
holder's consent for the approval of other
marketing applications for the same drug.......
Guidance Recommendations: Meeting requests to 800 1.5 1,200 4 4,800
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. 2026-19284 Filed 9-18-26; 8:45 am]
BILLING CODE 4164-01-P

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