[Federal Register Volume 85, Number 192 (Friday, October 2, 2020)]
[Notices]
[Pages 62306-62309]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2020-21843]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2014-N-0386]


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 or Agency) 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 provisions of the Federal Food, Drug, and Cosmetic Act 
(FD&C Act) and implementing regulations.

DATES: Submit either electronic or written comments on the collection 
of information by December 1, 2020.

ADDRESSES: You may submit comments as follows. Please note that late,

[[Page 62307]]

untimely filed comments will not be considered. Electronic comments 
must be submitted on or before December 1, 2020. The https://www.regulations.gov electronic filing system will accept comments until 
11:59 p.m. Eastern Time at the end of December 1, 2020. Comments 
received by mail/hand delivery/courier (for written/paper submissions) 
will be considered timely if they are postmarked or the delivery 
service acceptance receipt is 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-2014-N-0386 for ``Agency Information Collection Activities; 
Proposed Collection; Comment Request; Orphan Drugs.'' 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: Jonna 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--Revision

    This information collection supports FDA regulations implementing 
sections 525, 526, 527, and 528 of the FD&C Act, as well as related 
guidance. Sections 525, 526, 527, and 528 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 (21 U.S.C. 360aa) requires 
written recommendations on studies required for approval of a marketing 
application for a drug for a rare disease or condition. The information 
collection in 21 CFR 316.10, 316.12, and 316.14 is approved under OMB 
control numbers 0910-0001 and 0910-0014. Section 526 of the FD&C Act 
(21 U.S.C. 360bb) provides for designation of drugs as orphan drugs 
when certain conditions are met; section 527 (21 U.S.C. 360cc) 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 (21 U.S.C. 
360dd) is

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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.
    We have issued regulations in part 316 (21 CFR part 316) to 
implement the Orphan Drug provisions of the FD&C Act and to set forth 
procedures and requirements related to requesting recommendations for 
investigations of drugs for rare diseases or conditions; requesting 
designation of a drug for a rare disease or condition; or requesting 
exclusive approval for a drug for a rare disease or condition. To 
assist respondents and consistent with 21 CFR part 316.50, our Office 
of Orphan Products Development (OOPD) maintains and makes publicly 
available 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 Practice regulations in 21 CFR part 10.115 which provide 
for public comment at any time. Accordingly, we are revising the 
information collection to include Agency guidance. The document 
entitled, ``Guidance for Industry, Researchers, Patient Groups, and 
Food and Drug Administration Staff on Meetings With the Office of 
Orphan Products Development,'' provides recommendations to industry, 
researchers, patient groups, and other stakeholders interested in 
requesting a meeting, including a teleconference, with 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. This guidance describes 
procedures for requesting, preparing, scheduling, conducting, and 
documenting such meetings, and discusses background information we 
recommend be included in such requests. Information collection 
attendant to recommendations in the guidance are currently approved 
under OMB control number 0910-0787, however for efficiency of Agency 
operations we are consolidating it into this related information 
collection. The guidance is available at https://www.fda.gov/media/92815/download.
    The FDA Orphan Drug Designation Request Form (Form FDA 4035) is 
intended to benefit sponsors who desire to seek orphan designation of 
drugs intended for rare diseases or conditions from only FDA. The form 
is a simplified method for sponsors to provide only information 
required by 21 CFR 316.20 for FDA to make a decision.
    During this public health emergency associated with the COVID-19 
pandemic, the OOPD is providing sponsors with increased flexibility for 
submission of orphan drug designation requests and related submissions 
(amendments, annual reports, etc.). During this public health 
emergency, orphan drug designation, humanitarian use device 
designation, and rare pediatric disease designation requests and 
submissions may be submitted electronically by email to the OOPD. When 
transmitting information to the Orphan Drug Designation Program via 
email, please utilize the mailbox [email protected]. The use of 
automated read receipt is recommended to avoid the need to call to 
verify receipt of the email. Sponsors and others who plan to email 
information to FDA that is considered to be private, sensitive, 
proprietary, or commercial confidential are strongly encouraged to send 
it from an FDA secured email address so the transmission is encrypted. 
The OOPD will assume that the addresses of emails received or email 
addresses provided as a point of contact are FDA secure when responding 
to those email addresses.
    We estimate the burden of the information collection as follows:

                                 Table 1--Estimated Annual Reporting Burden \1\
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                                                      Annual
         21 CFR section              Number of     frequency per   Total annual      Hours per      Total hours
                                    respondents      response        responses       response
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Content and format of a request              534            1.25             668             135          90,180
 for designation; request for
 verification of status;
 amendment to designation.......
Sec.  Sec.   316.20, 316.21,                 534            1.25             668              32          21,376
 316.26 (Form FDA 4035).........
Sec.   316.22; Notifications of              132               1              70               2             264
 changes in agents..............
Sec.   316.24(a); Deficiency                  20               1              20               2              40
 letters and granting orphan-
 drug designation...............
Sec.   316.27; Submissions to                104               1             104               5             520
 change ownership of orphan-drug
 designation....................
Sec.   316.30; Annual reports...             744               1             744               3           2,232
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:                  2,508               1           2,508           3.595           9,016
 Meeting requests to OOPD and
 related submission packages....
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Total...........................  ..............  ..............  ..............  ..............         123,623
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\1\ There are no capital costs or operating and maintenance costs associated with this collection of
  information.


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    Based on our evaluation, we have adjusted the currently approved 
burden estimate we attribute to information collection activities 
associated with our Orphan Drug program to reflect an increase in 
submissions.

    Dated: September 28, 2020.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2020-21843 Filed 10-1-20; 8:45 am]
BILLING CODE 4164-01-P