
[Federal Register Volume 79, Number 194 (Tuesday, October 7, 2014)]
[Notices]
[Pages 60474-60476]
From the Federal Register Online via the Government Printing Office [www.gpo.gov]
[FR Doc No: 2014-23846]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

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


Agency Information Collection Activities; Submission for Office 
of Management and Budget Review; Comment Request; Orphan Drugs; Common 
European Medicines Agency/Food and Drug Administration Application 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 (PRA).

DATES: Fax written comments on the collection of information by 
November 6, 2014.

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 oira_submission@omb.eop.gov. 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: FDA PRA Staff, Office of Operations, 
Food and Drug Administration, 8455 Colesville Rd., COLE-14526, Silver 
Spring, MD 20993-0002, PRAStaff@fda.hhs.gov.

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.

[[Page 60475]]

Orphan Drugs; Common European Medicines Agency/Food and Drug 
Administration Application Form for Orphan Medicinal Product 
Designation--21 CFR Part 316 (OMB Control Number 0910-0167)--Revision

    FDA is amending the 1992 Orphan Drug Regulations, part 316 (21 CFR 
part 316). The 1992 regulations were issued to implement sections 525 
through 528 of the Orphan Drug Act Amendments to the Federal Food, 
Drug, and Cosmetic Act (the FD&C Act) (21 U.S.C. 360aa through 360ee). 
The 1992 regulations specify the procedures for sponsors of orphan 
drugs to use in obtaining the incentives provided for in the FD&C Act 
and set forth the procedures that FDA will use in administering the 
FD&C Act.
    The amendments are intended to clarify regulatory provisions and 
make minor improvements to address issues that have arisen since the 
issuance of the regulations in 1992. They are intended to assist 
sponsors who are seeking and who have obtained orphan drug 
designations, as well as FDA in its administration of the orphan drug 
program. Except with respect to the two revisions addressed further, 
the revisions in this rule clarify existing language and do not 
constitute a substantive or material modification to the approved 
collections of information in current part 316 (see 5 CFR 1320.5(g)). 
The collections of information in current part 316 have been approved 
by OMB in accordance with the PRA under OMB control number 0910-0167.
    One revision concerns the name of the drug in an orphan-drug 
designation request. As provided in current Sec.  316.20(b)(2) (Content 
and format of a request for orphan-drug designation), requests for 
orphan-drug designation must include the generic and trade name, if 
any, of the drug. For some products, however, neither a generic nor 
trade name may be available. This can be the case for some large and 
complicated biological products or for any molecule for which the 
sponsor has not yet obtained a trade name. Under Sec.  316.20(b)(2) as 
revised, requests for designation must include a chemical name or a 
meaningful descriptive name of the drug if neither a generic nor trade 
name is available. Drug names need to be meaningful to the public 
because the Orphan Drug Act (Pub. L. 97-414) requires that notice 
respecting designation of a drug be made available to the public 
(section 526(c) of the FD&C Act and Sec.  316.28 (Publication of orphan 
drug designations)). Internal business codes or other similar 
identifiers do not suffice for publication purposes, as they do not 
provide meaningful notice to the public of a designation. By providing 
a chemical name or a meaningful descriptive name of a drug in a request 
for designation, if neither a generic nor trade name is available, 
sponsors would help ensure that the name of the product that FDA 
ultimately publishes upon designation is accurate and meaningful.
    FDA regulations are currently silent on when sponsors must respond 
to a deficiency letter from FDA on an orphan-drug designation request. 
FDA sends such deficiency letters when a request lacks necessary 
information or contains inaccurate information, i.e., miscalculated 
prevalence estimate. This rule revises Sec.  316.24(a) (Deficiency 
letters and granting orphan-drug designation) to include 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. FDA will grant all reasonable requests for an extension. In 
the event the sponsor fails to respond to the deficiency or request an 
extension of time to respond within the 1-year time frame, FDA may 
consider the designation request voluntarily withdrawn. This proposal 
is necessary to ensure that designation requests do not become 
``stale'' by the time they are granted, such that the basis for the 
initial request may no longer hold.
    Sections 525 through 528 of the FD&C Act 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, specify procedures that sponsors of orphan drugs use in 
availing themselves of the incentives provided for orphan drugs in the 
FD&C Act, and set 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. 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 
316.26 allows an applicant to amend the applications under certain 
circumstances. 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. 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.
    The information requested from respondents, for the most part, is 
an accounting of information already in the possession of the 
applicant. It is estimated, based on frequency of requests over the 
past 3 years, that 275 persons or organizations per year will request 
orphan-drug designation and none will request formal recommendations on 
design of preclinical or clinical studies.
    In the Federal Register of April 16, 2014 (79 FR 21471), 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:

[[Page 60476]]



                                 Table 1--Estimated Annual Reporting Burden \1\
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                                                     Number of
     21 CFR Section/FDA Form         Number of     responses per   Total annual   Average burden    Total hours
                                    respondents     respondent       responses     per response
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316.10, 316.12, and 316.14......               2               1               2             100             200
316.20, 316.21, and 316.26......             225               2             450             150          67,500
Form FDA 3671...................              50               3             150              45           6,750
316.22..........................              65               1              65               2             130
316.27..........................              43               1              43               5             215
316.30..........................             450               1             450               3           1,350
316.36..........................               2               3               6              15              90
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    Total.......................  ..............  ..............  ..............  ..............          76,235
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\1\ There are no capital costs or operating and maintenance costs associated with this collection of
  information.


    Dated: October 1, 2014.
Leslie Kux,
Assistant Commissioner for Policy.
[FR Doc. 2014-23846 Filed 10-6-14; 8:45 am]
BILLING CODE 4164-01-P


