Notice2023-26544
Agency Information Collection Activities; Submission for Office of Management and Budget Review; Comment Request; Orphan Drugs
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Metadata and text below are from the Federal Register, a public-domain U.S. government work. Always verify the official published version before relying on it for any legal matter.
Published
December 4, 2023
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 (PRA).
Full Text
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<title>Federal Register, Volume 88 Issue 231 (Monday, December 4, 2023)</title>
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[Federal Register Volume 88, Number 231 (Monday, December 4, 2023)]
[Notices]
[Pages 84148-84149]
From the Federal Register Online via the Government Publishing Office [<a href="http://www.gpo.gov">www.gpo.gov</a>]
[FR Doc No: 2023-26544]
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2023-N-1929]
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 (PRA).
DATES: Submit written comments (including recommendations) on the
collection of information by January 3, 2024.
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, 301-796-5733,
<a href="/cdn-cgi/l/email-protection#b2e2e0f3e1c6d3d4d4f2d4d6d39cdadac19cd5ddc4"><span class="__cf_email__" data-cfemail="d080829183a4b1b6b690b6b4b1feb8b8a3feb7bfa6">[email 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 3671, Common EMEA/FDA
Application for Orphan Medicinal Product, and 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
[[Page 84149]]
<a href="/cdn-cgi/l/email-protection#c1aeb3b1a9a0af81a7a5a0efa9a9b2efa6aeb7"><span class="__cf_email__" data-cfemail="f39c81839b929db3959792dd9b9b80dd949c85">[email 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#f9aa9c9a8c8b9cbc94989095b99f9d98d791918ad79e968f"><span class="__cf_email__" data-cfemail="c596a0a6b0b7a080a8a4aca985a3a1a4ebadadb6eba2aab3">[email 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 June 13, 2023 (88 FR 38513), we
published a 60-day notice soliciting comment on the proposed collection
of information. Although we received one comment, it was not responsive
to the information collection topics solicited and therefore is not
addressed in this notice.
We estimate the burden of this collection of information as follows
based on data from 2022:
Table 1--Estimated Annual Recordkeeping Burden \1\
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Number of
21 CFR part or section; activity Number of records per Total annual Average burden Total hours
respondents recordkeeper records per record
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Part 316 associated records..... 780 1.25 975 135 131,625
Sec. Sec. 316.20, 316.21, 780 1.25 975 32 31,200
316.26 (Form FDA 4035).........
Sec. 316.22; Notifications of 300 1 300 0.5 150
changes in agents..............
Sec. 316.24(a); Deficiency 20 1 20 2 40
letters and granting orphan-
drug designation...............
Sec. 316.27; Submissions to 90 1 90 3 270
change ownership of orphan-drug
designation....................
Sec. 316.30; Annual reports... 2,039 1 2,039 3 6,117
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: 807 1.5 1,211 4 4,842
Meeting requests to OOPD and
related submission packages....
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Total....................... .............. .............. 5,613 .............. 174,289
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\1\ There are no capital costs or operating and maintenance costs associated with this collection of
information.
Our burden estimate includes those activities related 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. Upon a recent evaluation of the information collection, we
adjusted our burden estimate to reflect an overall increase of 50,616
hours and an increase of 766 records annually. We attribute this
adjustment to an increase in the number of submissions, amendments, and
annual reports.
Dated: November 29, 2023.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2023-26544 Filed 12-1-23; 8:45 am]
BILLING CODE 4164-01-P
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