[Federal Register Volume 88, Number 113 (Tuesday, June 13, 2023)]
[Notices]
[Pages 38513-38516]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[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 https://www.regulations.gov 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:
     Federal eRulemaking Portal: https://www.regulations.gov. 
Follow the instructions for submitting comments. Comments submitted 
electronically, including attachments, to https://www.regulations.gov 
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 https://www.regulations.gov.
     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:
     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.
     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 https://www.regulations.gov or at the Dockets 
Management Staff between 9 a.m. and 4 p.m., Monday through Friday, 240-
402-7500.
     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 https://www.regulations.gov. 
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: https://www.govinfo.gov/content/pkg/FR-2015-09-18/pdf/2015-23389.pdf.
    Docket: For access to the docket to read background documents or 
the electronic and written/paper comments received, go to https://www.regulations.gov 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, 
[email protected].

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

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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: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/meetings-office-orphan-products-development. 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 https://www.fda.gov/industry/medical-products-rare-diseases-and-conditions/designating-orphan-product-drugs-and-biological-products, 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 [email protected]; 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 [email protected]. 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.

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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]
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