Document ID: FDA-2011-N-0015-0004
Agency: fda
Document Type: Notice
Title: Agency Information Collection Activities; Proposed Collection; Comment Request; Orphan Drugs; Common European Medicines Agency/Food and Drug Administration Application Form for Orphan Drug Medicinal Product Designation
Posted Date: 2017-06-19T04:00Z

[Federal Register Volume 82, Number 116 (Monday, June 19, 2017)]
[Notices]
[Pages 27836-27838]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2017-12620]

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

DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2011-N-0015]

Agency Information Collection Activities; Proposed Collection; 
Comment Request; Orphan Drugs; Common European Medicines Agency/Food 
and Drug Administration Application Form for Orphan Drug Medicinal 
Product Designation

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice.

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

SUMMARY: The Food and Drug Administration (FDA) is announcing an 
opportunity for public comment on the proposed collection of certain 
information by the Agency. Under the Paperwork Reduction Act of 1995 
(the 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 Common European Medicines 
Agency (EMA)/FDA Application Form for Orphan Drug Medicinal Product 
Designation (Form FDA 3671).

DATES: Submit either electronic or written comments on the collection 
of information by August 18, 2017.

ADDRESSES: You may submit comments as follows. Please note that late, 
untimely filed comments will not be considered. Electronic comments 
must be submitted on or before August 18, 2017. The https://www.regulations.gov electronic filing system will accept comments until 
midnight Eastern Time at the end of August 18, 2017. 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-2011-N-0015 for ``Orphan Drugs; Common EMA/FDA Application Form for 
Orphan Medicinal Product Designation (Form FDA 3671)--21 CFR part 
316.'' 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.
     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.gpo.gov/fdsys/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://

[[Page 27837]]

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.

FOR FURTHER INFORMATION CONTACT: Amber Sanford, Office of Operations, 
Food and Drug Administration, Three White Flint North, 10A63, 11601 
Landsdown St., North Bethesda, MD 20852, PRAStaff@fda.hhs.gov, 301-796-
8867.

SUPPLEMENTARY INFORMATION: Under the PRA (44 U.S.C. 3501-3520), 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; Common EMA/FDA Application Form for Orphan Medicinal 
Product Designation (Form FDA 3671) 21 CFR Part 316; OMB Control Number 
0910-0167--Extension

    Sections 525 through 528 of the Federal Food, Drug, and Cosmetic 
Act (the FD&C Act) (21 U.S.C. 360aa through 360dd) give FDA statutory 
authority to do the following: (1) Provide recommendations on 
investigations required for approval of marketing applications for 
orphan drugs, (2) designate eligible drugs as orphan drugs, (3) set 
forth conditions under which a sponsor of an approved orphan drug 
obtains exclusive approval, and (4) encourage sponsors to make orphan 
drugs available for treatment on an ``open protocol'' basis before the 
drug has been approved for general marketing. The implementing 
regulations for these statutory requirements have been codified under 
part 316 (21 CFR part 316) and specify procedures that sponsors of 
orphan drugs use in availing themselves of the incentives provided for 
orphan drugs in the FD&C Act and sets forth procedures FDA will use in 
administering the FD&C Act with regard to orphan drugs.
    Section 316.10 specifies the content and format of a request for 
written recommendations concerning the nonclinical laboratory studies 
and clinical investigations necessary for approval of marketing 
applications. Section 316.12 provides that, before providing such 
recommendations, FDA may require results of studies to be submitted for 
review. Section 316.14 contains provisions permitting FDA to refuse to 
provide written recommendations under certain circumstances. Within 90 
days of any refusal, a sponsor may submit additional information 
specified by FDA. Based on past experience, FDA estimates that there 
will be one respondent to Sec. Sec.  316.10, 316.12, and 316.14 
requiring 50 hours of human resources annually.
    Section 316.20 specifies the content and format of an orphan drug 
application which includes requirements that an applicant document that 
the disease is rare (affects fewer than 200,000 persons in the United 
States annually) or that the sponsor of the drug has no reasonable 
expectation of recovering costs of research and development of the 
drug. Section 316.21 specifies content of a request for orphan drug 
designation required for verification of orphan-drug status. Section 
316.26 allows an applicant to amend the applications under certain 
circumstances. Based on past experience, FDA estimates 496 respondents 
to Sec. Sec.  316.20, 316.21 and 316.26, requiring 93,000 hours of 
human resources annually.
    The Common EMA/FDA Application Form for Orphan Medicinal Product 
Designation (Form FDA 3671) is intended to benefit sponsors who desire 
to seek orphan designation of drugs intended for rare diseases or 
conditions from both the European Commission and FDA by reducing the 
burden of preparing separate applications to meet the regulatory 
requirements in each jurisdiction. It highlights the regulatory 
cooperation between the United States and the European Union mandated 
by the Transatlantic Economic Council (TEC). Based on past experience, 
FDA estimates there will be 60 respondents using the form requiring 450 
hours of human resources annually.
    Section 316.22 specifies requirement of a permanent resident agent 
for foreign sponsors. Based on past experience, FDA estimates 70 
respondents requiring 140 hours of human resources annually. Section 
316.24(a) specifies a requirement that sponsors respond to deficiency 
letters from FDA on designation requests within 1 year of issuance of 
the deficiency letter, unless within that time frame, the sponsor 
requests an extension of time to respond. Based on past experience, FDA 
estimates 20 respondents requiring 40 hours of human resources 
annually.
    Section 316.27 specifies content of a change in ownership of 
orphan-drug designation. Based on past experience, FDA estimates 63 
respondents requiring 315 hours of human resources annually. Section 
316.30 requires submission of annual reports, including progress 
reports on studies, a description of the investigational plan, and a 
discussion of changes that may affect orphan status. Based on number of 
orphan-drug designations, the number of respondents is estimated as 744 
requiring 2,232 hours of human resources annually. Finally, Sec.  
316.36 describes information required of sponsor when there is 
insufficient quantity of approved orphan drug. Based on past 
experience, FDA estimates two respondents requiring 90 hours of human 
resources annually.
    The information requested will provide the basis for an FDA 
determination that the drug is for a rare disease or condition and 
satisfies the requirements for obtaining orphan drug status. Secondly, 
the information will describe the medical and regulatory history of the 
drug. The respondents to this collection of information are 
biotechnology firms, drug companies, and academic clinical researchers.
    FDA estimates the burden of this collection of information as 
follows:

[[Page 27838]]

                                 Table 1--Estimated Annual Reporting Burden \1\
----------------------------------------------------------------------------------------------------------------
                                                      Annual
         21 CFR section              Number of    frequency  per   Total annual      Hours per      Total hours
                                    respondents      response        responses       response
----------------------------------------------------------------------------------------------------------------
Content and format when seeking                1               1               1              50              50
 written recommendations;
 results of studies; amendments
 (316.10, 316.12 & 316.14)......
Content and format of a request              496            1.25             620             150          93,000
 for orphan-drug designation;                 60             1.5              90               5             450
 request for verification of
 orphan-drug status; amendments
 (316.20, 316.21 & 316.26) FDA
 Form 3671......................
Notifications of changes in                   70               1              70               2             140
 agents (316.22)................
Deficiency letters and granting               20               1              20               2              40
 orphan-drug designation
 (316.24(a))....................
Submissions to change ownership               63               1              63               5             315
 of orphan-drug designation
 (316.27).......................
Annual reports (316.30).........             744               1             744               3           2,232
Assurance of the availability of               2               3               6              15              90
 sufficient quantities of the
 orphan drug; holder's consent
 for the approval of other
 marketing applications for the
 same drug (316.36).............
                                 -------------------------------------------------------------------------------
    Total.......................  ..............  ..............  ..............  ..............          96,317
----------------------------------------------------------------------------------------------------------------
\1\ There are no capital costs or operating and maintenance costs associated with this collection of
  information.

    FDA has experienced increases in: (1) The number of submissions to 
change ownership of orphan-drug designation (21 CFR 316.27), (2) the 
number of annual reports 21 CFR 316.30, and (3) assurances of the 
availability of sufficient quantities of the orphan drug and the 
holder's consent for the approval of other marketing applications for 
the same drug (21 CFR 316.36). In contrast, however, the use of Form 
FDA 3671, the application form to submit for product designation to the 
European Medicines Agency and to the FDA Office of Orphan Products, has 
decreased from 6,760 to 450 total burden hours.

    Dated: June 13, 2017.
Anna K. Abram,
Deputy Commissioner for Policy, Planning, Legislation, and Analysis.
[FR Doc. 2017-12620 Filed 6-16-17; 8:45 am]
 BILLING CODE 4164-01-P