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Agency Information Collection Activities; Proposed Collection; Comment Request; Orphan Drugs Products: Common European Medicines Agency/Food and Drug Administration Application Form for Orphan Medicinal Product Designation

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Food and Drug Administration, HHS.




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 Drug Products: Common EMEA/FDA Application Form for Orphan Medicinal Product Designation (Form FDA 3671).


Submit written or electronic comments on the collection of information by June 16, 2014.


Submit electronic comments on the collection of information to 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.

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FDA PRA Staff, Office of Operations, Food and Drug Administration, 1350 Piccard Dr., PI50-400B, Rockville, MD 20850,

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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 these topics: (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)—Extension

FDA is amending the 1992 Orphan Drug Regulations, part 316 (21 CFR part 316). The 1992 regulations were issued to implement sections 525 through 528 of the Orphan Drug Act Amendments to the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360aa through 360ee) (the FD&C Act). The 1992 regulations specify the procedures for sponsors of orphan drugs to use in obtaining the incentives provided for in the FD&C Act and set forth the procedures that FDA will use in administering the FD&C Act.

The amendments are intended to clarify regulatory provisions and make minor improvements to address issues that have arisen since the issuance of the regulations in 1992. They are intended to assist sponsors who are seeking and who have obtained orphan drug designations, as well as FDA in its administration of the orphan drug program. Except with respect to the two revisions addressed further, the revisions in this rule clarify existing language and do not constitute a substantive or material modification to the approved collections of information in current part 316 (see 5 CFR 1320.5(g)). The collections of information in current part 316 have been approved by OMB in accordance with the PRA under OMB control number 0910-0167.Start Printed Page 21472

One revision concerns the name of the drug in an orphan-drug designation request. As provided in current § 316.20(b)(2) (Content and format of a request for orphan-drug designation), requests for orphan-drug designation must include the generic and trade name, if any, of the drug. For some products, however, neither a generic or trader name may be available. This can be the case for some large and complicated biological products or for any molecule for which the sponsor has not yet obtained a trade name. Under § 316.20(b)(2) as revised, requests for designation must include a chemical name or a meaningful descriptive name of the drug if neither a generic nor trade name is available. Drug names need to be meaningful to the public because the Orphan Drug Act (Pub. L. 97-414) requires that notice respecting designation of a drug be made available to the public (section 526(c) of the FD&C Act and § 316.28 (Publication of orphan drug designations)). Internal business codes or other similar identifies do not suffice for publication purposes as they do not provide meaningful notice to the public of a designation. By providing a chemical name or a meaningful descriptive name of a drug in a request for designation, if neither a generic nor trade name is available, sponsors would help ensure that the name of the product that FDA ultimately publishes upon designation is accurate and meaningful.

FDA regulations are currently silent on when sponsors must respond to a deficiency letter from FDA on an orphan-drug designation request. FDA sends such deficiency letters when a request lacks necessary information or contains inaccurate information, i.e., miscalculated prevalence estimate. This rule revises § 316.24(a) (Deficiency letters and granting orphan-drug designation) to include a requirement that sponsors respond to deficiency letters from FDA on designation requests within 1 year of issuance of the deficiency letter, unless within that time frame, the sponsor requests an extension of time to respond. FDA will grant all reasonable requests for an extension. In the event the sponsor fails to respond to the deficiency or request an extension of time to respond within the 1 year time frame, FDA may consider the designation request voluntarily withdrawn. This proposal is necessary to ensure that designation requests do not become “stale” by the time they are granted, such that the basis for the initial request may no longer hold.

Sections 525 through 528 of the FD&C Act gives 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 and specify procedures that sponsors of orphan drugs use in availing themselves of the incentives provided for orphan drugs in the FD&C Act and sets forth procedures FDA will use in administering the FD&C 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, 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 3 years, that 275 persons or organizations per year will request orphan-drug designation and none will request formal recommendations on design of preclinical or clinical studies.

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

Table 1—Estimated Annual Reporting Burden 1

21 CFR SectionNumber of respondentsNumber of responses per respondentTotal annual responsesAverage burden per responseTotal hours
316.10, 316.12, and 316.14212100200
316.20, 316.21, and 316.26225245015067,500
FDA Form 3671503150456,750
1 There are no capital costs or operating and maintenance costs associated with this collection of information.
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Dated: April 10, 2014.

Leslie Kux,

Assistant Commissioner for Policy.

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[FR Doc. 2014-08589 Filed 4-15-14; 8:45 am]