[Federal Register Volume 66, Number 84 (Tuesday, May 1, 2001)]
[Notices]
[Pages 21769-21770]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 01-10711]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. 00N-1503]


Agency Information Collection Activities; Submission for OMB 
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 the 
proposed collection of information listed below has been submitted to 
the Office of Management and Budget (OMB) for review and clearance 
under the Paperwork Reduction Act of 1995.

DATES: Submit written comments on the collection of information by May 
31, 2001.

ADDRESSES: Submit written comments on the collection of information to 
the Office of Information and Regulatory Affairs, OMB, New Executive 
Office Bldg., 725 17th St. NW., rm. 10235, Washington, DC 20503, Attn: 
Wendy Taylor, Desk Officer for FDA.

FOR FURTHER INFORMATION CONTACT: JonnaLynn P. Capezzuto, Office of 
Information Resources Management (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--21 CFR Part 316 (OMB 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: (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 
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 
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 September 19, 2000 (65 FR 56586), the 
agency requested comments on the proposed collections of information. 
No significant 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               No. of         Frequency     Total Annual      Hours per      Total Hours
                                    Respondents     perResponse      Responses       Response
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316.10, 316.12, and 316.14              0               0               0               0               0
316.20, 316.21, and 316.26             90               1.78          160.20          125          20,025
316.22                                  5               1               5               2              10
316.27                                  5               1               5               4              20
316.30                                450               1             450               2             900
316.36                                   .2             3                .6            15               9
Total Burden Hours                                                                                 20,964
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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 10 years, that 90 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 (Secs. 316.10, 316.12, 316.20, and 316.21) is 
generally similar and is estimated to

[[Page 21770]]

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: April 24, 2001.
William K. Hubbard,
Senior Associate Commissioner for Policy, Planning, and Legislation.
[FR Doc. 01-10711 Filed 4-30-01; 8:45 am]
BILLING CODE 4160-01-S