[Federal Register: May 27, 2004 (Volume 69, Number 103)]
[Notices]               
[Page 30314-30315]
From the Federal Register Online via GPO Access [wais.access.gpo.gov]
[DOCID:fr27my04-66]                         

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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. 2004N-0046]

 
Agency Information Collection Activities; Submission for Office 
of Management and Budget Review; Comment Request; Orphan Drug Products

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 June 
28, 2004

ADDRESSES: OMB is still experiencing significant delays in the regular 
mail, including first class and express mail, and messenger deliveries 
are not being accepted. 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: Fumie 
Yokota, Desk Officer for FDA, FAX: 202-395-6974.

FOR FURTHER INFORMATION CONTACT: Jonna Capezzuto, Office of Management 
Programs (HFA-250), Food and Drug Administration, 5600 Fishers Lane, 
Rockville, MD 20857, 301-827-4659.

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 Drug Products--(OMB Control Number 0910-0167)--Extension

    Sections 525 through 528 of the Federal Food, Drug, and Cosmetic 
Act (the 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 act and sets forth procedures FDA will use in 
administering the 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 than 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 
application 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.
    In the Federal Register of February 24, 2004 (69 FR 8447) FDA 
published a 60-day notice requesting public comment on the information 
collection provisions. No comments were received.
    FDA estimates the burden of this collection of information as 
follows:

[[Page 30315]]



                                 Table 1.--Estimated Annual Reporting Burden\1\
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                                                 Annual Frequency    Total Annual     Hours per
      21 CFR Section        No. of Respondents      of response        Responses      Response      Total Hours
----------------------------------------------------------------------------------------------------------------
316.10, 316.12, and 316.14          3                   1                 3            130            390
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316.20, 316.21, and 316.26        138                   2.0             276            130         35,880
----------------------------------------------------------------------------------------------------------------
316.22                             22                   1                22              2             44
----------------------------------------------------------------------------------------------------------------
 316.27                             5                   1                 5              4             20
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 316.30                           500                   1               500              2          1,000
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316.36                               .2                 3                  .6           15              9
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Total                       ..................  ..................  ..............  ............   37,343
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\1\ There are no capital costs or operating and maintenance costs associated with this collection of
  information.

    The information requested from respondents represents, for the most 
part, an accounting of information already in possession of the 
applicant. It is estimated, based on the frequency of requests over the 
past 13 years that 138 persons or organizations per year will request 
orphan drug designation and that no requests for recommendations on 
design of preclinical or clinical studies will be received. Based upon 
FDA experience over the last decade, FDA estimates that the effort 
required to prepare applications to receive consideration for sections 
525 and 526 of the act (Sec. Sec.  316.10, 316.12, 316.20, and 316.21) 
is generally similar and is estimated to require an average of 95 hours 
of professional staff time and 30 hours of support staff time per 
application. Estimates of annual activity and burden for foreign 
sponsor nomination of a resident, agent, change in ownership or 
designation, and inadequate supplies of drug in exclusivity, are based 
on total experience by FDA with such requests since 1983.

    Dated: May 21, 2004.
Jeffrey Shuren,
Assistant Commissioner for Policy.
[FR Doc. 04-12010 Filed 5-26-04; 8:45 am]

BILLING CODE 4160-01-S