Document ID: FDA-2011-N-0015-0005
Agency: fda
Document Type: Notice
Title: Agency Information Collection Activities; Submission for Office of
Management and Budget Review; Comment Request; Orphan Products
Development; Food and Drug Administration Orphan Drug Designation Request Form and The Common European Medicines Agency/
Food and Drug Administration Form for Orphan Medicinal Product
Designation
Posted Date: 2017-12-12T05:00Z

[Federal Register Volume 82, Number 237 (Tuesday, December 12, 2017)]
[Notices]
[Pages 58424-58425]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2017-26669]

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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

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

Agency Information Collection Activities; Submission for Office 
of Management and Budget Review; Comment Request; Orphan Products 
Development; Food and Drug Administration Orphan Drug Designation 
Request Form and The Common European Medicines Agency/Food and Drug 
Administration Form for Orphan Medicinal Product Designation

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice.

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SUMMARY: The Food and Drug Administration (FDA) 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.

DATES: Fax written comments on the collection of information by January 
11, 2018.

ADDRESSES: To ensure that comments on the information collection are 
received, OMB recommends that written comments be faxed to the Office 
of Information and Regulatory Affairs, OMB, Attn: FDA Desk Officer, 
Fax: 202-395-7285, or emailed to [email protected]. All 
comments should be identified with the OMB control number 0910-0167. 
Also include the FDA docket number found in brackets in the heading of 
this document.

FOR FURTHER INFORMATION CONTACT: Amber Sanford, Office of Operations, 
Food and Drug Administration, Three White Flint North, 10A-12M, 11601 
Landsdown St., North Bethesda, MD 20852, 301-796-8867, 
[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 Products Development; Food and Drug Administration Orphan Drug 
Designation Request Form and The Common European Medicines Agency/Food 
and Drug Administration Form for Orphan Medicinal Product Designation 
(Formerly Orphan Drugs; Common European Medicines Agency/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-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 83,700 hours of 
human resources annually.
    The Common EMEA/FDA Application for Orphan Medicinal Product 
Designation form for orphan designation of drugs intended for rare 
diseases or conditions (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. 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. Based on past experience, FDA 
estimates there will be 496 respondents using the form requiring 19,840 
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.

[[Page 58425]]

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.
    In the Federal Register of June 19, 2017 (82 FR 27836), FDA 
published a 60-day notice requesting public comment on the proposed 
collection of information. No comments were received.
    FDA estimates the burden of this collection of information as 
follows:

                                 Table 1--Estimated Annual Reporting Burden \1\
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                                                      Annual
     21 CFR section/Form FDA         Number of     frequency per   Total annual      Hours per      Total hours
                                    respondents      response        responses       response
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Content and format when seeking                1               1               1              50              50
 written recommendations;
 results of studies; and
 amendments (Sec.  Sec.
 316.10, 316.12, and 316.14)....
Content and format of a request              496            1.25             620             135          83,700
 for designation; request for
 verification of status;
 amendment to designation.......
Form FDA 3671 or 4035 FDA Orphan            1.25             620              32          19,840
 Drug Designation Request Form
 (Sec.  Sec.   316.20, 316.21,
 and 316.26)....................
Notifications of changes in                   70               1              70               2             140
 agents (Sec.   316.22).........
Deficiency letters and granting               20               1              20               2              40
 orphan-drug designation (Sec.
 316.24(a)).....................
Submissions to change ownership               63               1              63               5             315
 of orphan-drug designation
 (Sec.   316.27)................
Annual reports (Sec.   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 (Sec.   316.36)......
                                 -------------------------------------------------------------------------------
    Total.......................  ..............  ..............  ..............  ..............         106,407
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\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 (Sec.  316.27), (2) the 
number of annual reports (Sec.  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 (Sec.  316.36).

    Dated: December 6, 2017.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2017-26669 Filed 12-11-17; 8:45 am]
 BILLING CODE 4164-01-P