[Federal Register Volume 78, Number 113 (Wednesday, June 12, 2013)]
[Notices]
[Pages 35277-35279]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2013-13858]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2013-N-0653]


Agency Information Collection Activities; Proposed Collection; 
Comment Request; Orphan Drugs; Common European Medicines Agency/Food 
and Drug Administration Application Form for Orphan Medicinal Product 
Designation (Form FDA 3671)

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice.

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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

[[Page 35278]]

the procedures by which sponsors of orphan drugs may request 
eligibility for the incentives by implementing a program as outlined in 
the Orphan Drug Act and the joint adoption by FDA and the European 
Medicines Agency (EMA) of the Common EMA/FDA Application Form for 
Orphan Medicinal Product Designation (Form FDA 3671).

DATES: Submit either electronic or written comments on the collection 
of information by August 12, 2013.

ADDRESSES: Submit electronic comments on the collection of information 
to http://www.regulations.gov. Submit written comments on the 
collection of information to the Division of Dockets Management (HFA-
305), Food and Drug Administration, 5630 Fishers Lane, rm. 1061, 
Rockville, MD 20852. All comments should be identified with the docket 
number found in brackets in the heading of this document.

FOR FURTHER INFORMATION CONTACT: Jonna Capezzuto, Office of Information 
Management, Food and Drug Administration, 1350 Piccard Dr., PI50-400B, 
Rockville, MD 20850, 301-796-3794, [email protected].

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 non-clinical 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 two respondents to Sec. Sec.  316.10, 316.12, and 316.14 
requiring 200 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. 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. FDA does not believe the new 
form will result in any increased burden on the respondents and 
therefore we estimate no additional burden. Based on past experience, 
FDA estimates there will be 214 respondents requiring 64,200 hours of 
human resources annually. Section 316.22 specifies requirement of a 
permanent resident agent for foreign sponsors. Based on past 
experience, FDA estimates 55 respondents requiring 110 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 43 respondents requiring 215 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 1,652 requiring 4,956 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 1 respondent 
requiring 45 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 35279]]



                                 Table 1--Estimated Annual Reporting Burden \1\
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                                                     Number of                        Average
   21 CFR Section and Form FDA       Number of     responses per   Total annual     burden per      Total hours
                                    respondents     respondent       responses       response
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Content and format when seeking                2               1               2             100             200
 written recommendations;
 results of studies; amendments
 (Sec.  Sec.   316.10, 316.12,
 316.14)........................
Content and format of a request              214               2             428             150          64,200
 for orphan-drug designation;
 request for verification of
 orphan-drug status; amendments
 (Sec.  Sec.   316.20, 316.21,
 316.26) Form FDA 3671..........
Notifications of changes in                   55               1              55               2             110
 agents (Sec.   316.22).........
Submissions to change ownership               43               1              43               5             215
 of orphan-drug designation
 (Sec.   316.27)................
Annual reports (Sec.   316.30)..           1,652               1           1,652               3           4,956
Assurance of the availability of               1               3               3              15              45
 sufficient quantities of the
 orphan drug; holder's consent
 for the approval of other
 marketing applications for the
 same drug (Sec.   316.36)......
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    Total.......................  ..............  ..............  ..............  ..............          69,726
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\1\ There are no capital costs or operating and maintenance costs associated with this collection of
  information.


    Dated: June 5, 2013.
Leslie Kux,
Assistant Commissioner for Policy.
[FR Doc. 2013-13858 Filed 6-11-13; 8:45 am]
BILLING CODE 4160-01-P