Notice2023-12547
Agency Information Collection Activities; Proposed Collection; Comment Request; Orphan Drugs
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Published
June 13, 2023
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.
Full Text
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<title>Federal Register, Volume 88 Issue 113 (Tuesday, June 13, 2023)</title>
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[Federal Register Volume 88, Number 113 (Tuesday, June 13, 2023)]
[Notices]
[Pages 38513-38516]
From the Federal Register Online via the Government Publishing Office [<a href="http://www.gpo.gov">www.gpo.gov</a>]
[FR Doc No: 2023-12547]
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2023-N-1929]
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
[[Page 38514]]
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 August 14, 2023.
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 August 14, 2023. 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 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: JonnaLynn Capezzuto, Office of
Operations, Food and Drug Administration, Three White Flint North, 10A-
12M, 11601 Landsdown St., North Bethesda, MD 20852, 301-796-3794,
<a href="/cdn-cgi/l/email-protection#9ececcdfcdeafff8f8def8faffb0f6f6edb0f9f1e8"><span class="__cf_email__" data-cfemail="8ededccfddfaefe8e8cee8eaefa0e6e6fda0e9e1f8">[email 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
[[Page 38515]]
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 <a href="/cdn-cgi/l/email-protection#f8978a88909996b89e9c99d690908bd69f978e"><span class="__cf_email__" data-cfemail="bdd2cfcdd5dcd3fddbd9dc93d5d5ce93dad2cb">[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#5e0d3b3d2b2c3b1b333f37321e383a3f7036362d70393128"><span class="__cf_email__" data-cfemail="3c6f595f494e5979515d55507c5a585d1254544f125b534a">[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.
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 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..... 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.
[[Page 38516]]
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: June 7, 2023.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2023-12547 Filed 6-12-23; 8:45 am]
BILLING CODE 4164-01-P
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