[Federal Register Volume 63, Number 95 (Monday, May 18, 1998)]
[Notices]
[Pages 27299-27300]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 98-13042]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration
[Docket No. 97N-0513]


Agency Information Collection Activities; Orphan Drugs: 
Submission for OMB Review; Comment Request

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 (the PRA).

DATES: Submit written comments on the collection of information by June 
17, 1998.
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, 
Attention: Desk Officer for FDA.

FOR FURTHER INFORMATION CONTACT: Denver Presley, Office of Information 
Resources Management (HFA-250), Food and Drug Administration, 5600 
Fishers Lane, Rockville, MD 20857, 301-827-1472.

SUPPLEMENTARY INFORMATION: In compliance with section 3507 of the PRA 
(44 U.S.C. 3507), FDA has submitted the following proposed collection 
of information to OMB for review and clearance.

Orphan Drugs--21 CFR Part 316--(OMB No. 0910-0167--Reinstatement)

    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 
specify procedures that sponsors of orphan drugs use in availing 
themselves of the incentives provided for orphan drugs in the act and 
set 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

[[Page 27300]]

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 ophan 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:

                                  Table 1.--Estimated Annual Reporting Burden1                                  
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                                                      Annual                                                    
         21 CFR Section               No. of       Frequency per   Total Annual      Hours per      Total Hours 
                                    Respondents      Response        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 5 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 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 8, 1998.
William K. Hubbard,
Associate Commissioner for Policy Coordination.
[FR Doc. 98-13042 Filed 5-15-98; 8:45 am]
BILLING CODE 4160-01-F