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

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Food and Drug Administration, HHS.




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.


Fax written comments on the collection of information by June 28, 2007.


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, Start Printed Page 29516OMB, Attn: FDA Desk Officer, FAX: 202-395-6974. 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.

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Jonna Capezzuto, Office of the Chief Information Officer (HFA-250), Food and Drug Administration, 5600 Fishers Lane, Rockville, MD 20857,301-827-4659.

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In compliance with 44 U.S.C. 3507, FDA has submitted the following proposed collection of information to OMB for review and clearance.

Orphan Drugs (OMB Control Number 0910-0167)—Extension

Sections 525 through 526 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 non-clinical 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 represents, for the most part, an accounting of information already in the possession of the applicant. It is estimated, based on frequency of requests over the past 5 years, that 171 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 October 30, 2006 (71 FR 63325), FDA published a 60-day notice requesting public comment on the information collection provisions. FDA received one comment related to the information collection.

(Comment 1) The comment suggested that our burden estimate to prepare an Orphan Drug Annual Report is too low.

(Response 1) Section 316.30 pertains to annual reporting, a brief progress report which is a requirement after orphan designation has been granted to a sponsor. We estimate this takes 1 hour professional time and 1 hour support time.

(Comment 2) The comment suggested that our estimate of 130 hours to prepare and submit an orphan drug application is too high.

(Response 2) We disagree with the comment because some sponsors have more experience with submitting Orphan Drug Designation applications/requests and, therefore, may require less human resource hours to compile all required information. Many other sponsors, which include foreign sponsors, do not have such experience.

The estimated 130 hours pertains to §§ 316.20, 316.21, and 316.26. These apply primarily to initial applications/requests seeking orphan drug designation. Many applications/requests received in the Office of Orphan Products Development contain multiple volumes; include an exact duplicate copy of the original; and may include 50 or more documented references. Additional information is requested when an application/request is denied. The sponsor usually supplies the requested information in the form of an amendment.

FDA estimates the burden of this collection of information as follows:

Table 1.—Estimated Annual Reporting Burden1

21 CFR SectionNo. of RespondentsAnnual Frequency per ResponseTotal Annual ResponsesHours per ResponsesTotal Hours
316.10, 316.12, & 316.14515130650
316.20, 316.21, & 316.26171234213044,460
1 There are no capital costs or maintenance costs associated with this collection of information.
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Dated: May 22, 2007.

Jeffrey Shuren,

Assistant Commissioner for Policy.

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[FR Doc. E7-10271 Filed 5-25-07; 8:45 am]