[Federal Register: February 24, 2004 (Volume 69, Number 36)]
[Page 8447-8448]
From the Federal Register Online via GPO Access [wais.access.gpo.gov]



Food and Drug Administration

[Docket No. 2004N-0046]

Agency Information Collection Activities; Proposed Collection; 
Comment Request; Orphan Drugs

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice.


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 orphan drugs.

DATES: Submit written or electronic comments on the collection of 
information by April 26, 2004.

ADDRESSES: Submit electronic comments on the collection of information 
to: http://frwebgate.access.gpo.gov/cgi-bin/leaving.cgi?from=leavingFR.html&log=linklog&to=http://www.fda.gov/dockets/ecomments. 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: JonnaLynn P. Capezzuto, Office of 
Management Programs (HFA-250), Food and Drug Administration, 5600 
Fishers Lane, Rockville, MD 20857, 301-827-4659.

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: (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--21 CFR Part 316 (OMB Control Number 0910-0167)

    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 
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

[[Page 8448]]

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.
    FDA estimates the burden of this collection of information as 

             Table 1.--Estimated Annual Reporting Burden\1\
                      No. of    Frequency    Total      Hours     Total
  21 CFR Section   Respondents      per      Annual      per      Hours
                                 Response  Responses  Response
316.10, 316.12,            3           1          3       130       390
 and 316.14
316.20, 316.21,          138         2.0        276       130    35,880
 and 316.26
316.22                    22           1         22         2        44
316.27                     5           1          5         4        20
316.30                   500           1        500         2     1,000
316.36                    .2           3         .6        15         9
Total              ...........  .........  .........  ........   37,343
\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: February 13, 2004.
Jeffrey Shuren,
Assistant Commissioner for Policy.
[FR Doc. 04-3860 Filed 2-23-04; 8:45 am]