
[Federal Register Volume 88, Number 231 (Monday, December 4, 2023)]
[Notices]
[Pages 84148-84149]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2023-26544]


-----------------------------------------------------------------------

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.

-----------------------------------------------------------------------

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 https://www.reginfo.gov/public/do/PRAMain. 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, 
[email protected].

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: 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

[[Page 84149]]

[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.
    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\
----------------------------------------------------------------------------------------------------------------
                                                     Number of
21 CFR part or section; activity     Number of      records per    Total annual   Average burden    Total hours
                                    respondents    recordkeeper       records       per record
----------------------------------------------------------------------------------------------------------------
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....
                                 -------------------------------------------------------------------------------
    Total.......................  ..............  ..............           5,613  ..............         174,289
----------------------------------------------------------------------------------------------------------------
\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


