[Federal Register Volume 79, Number 68 (Wednesday, April 9, 2014)]
[Notices]
[Pages 19623-19626]
[FR Doc No: 2014-07908]

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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2014-D-0313]

Draft Guidance for Industry, Researchers, Patient Groups, and
Food and Drug Administration Staff on Meetings With the Office of
Orphan Products Development; Availability

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice.

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SUMMARY: The Food and Drug Administration (FDA) is announcing the
availability of a draft guidance for industry, researchers, patient
groups, and FDA staff entitled ``Meetings With the Office of Orphan
Products Development.'' This draft guidance provides recommendations to
industry, researchers, patient groups, and other stakeholders
(collectively referred to as ``stakeholders'') interested in requesting
a meeting with FDA's Office of Orphan Products Development (OOPD) on
issues related to orphan drug designation requests, humanitarian use
device (HUD) designation requests, rare pediatric disease designation
requests, funding opportunities through the Orphan Products Grants
Program and the Pediatric Device Consortia Grants Program, and orphan
product patient-related topics of concern. This draft guidance document
is intended to assist these groups with requesting, preparing,
scheduling, conducting, and documenting meetings with OOPD.

DATES: Although you can comment on any guidance at any time (see 21 CFR
10.115(g)(5)), to ensure that the Agency considers your comment on this
draft guidance before it begins work on the final version of the
guidance, submit either electronic or written comments on the draft
guidance or proposed collection of information by June 9, 2014.

ADDRESSES: Submit written requests for single copies of the draft
guidance to the Office of Orphan Products Development, Food and Drug
Administration, 10903 New Hampshire Ave., Bldg. 32, Rm. 5271, Silver
Spring, MD 20993. Send one self-addressed adhesive label to assist that
office in processing your requests. See the SUPPLEMENTARY INFORMATION
section for information on electronic access to the draft guidance
document.
    Submit electronic comments on the draft guidance to http://www.regulations.gov. Submit written comments to the Division of Dockets
Management (HFA-305), Food and Drug Administration, 5630 Fishers Lane,
Rm. 1061, Rockville, MD 20852. Identify comments with the docket number
found in brackets in the heading of this document. OMB recommends that
written comments be faxed to the Office of Information and Regulatory
Affairs, OMB, Attn: FDA Desk Officer, FAX:<PHONE_NUMBER>, or emailed to
<EMAIL_ADDRESS>
FOR FURTHER INFORMATION CONTACT: James Bona, Office of Orphan Products
Development, Food and Drug Administration, 10903 New Hampshire Ave.,
Bldg. 32, Rm. 5271, Silver Spring, MD 20993,<PHONE_NUMBER>.

SUPPLEMENTARY INFORMATION:

I. Background

    FDA is announcing the availability of a draft guidance for
industry, researchers, patient groups, and FDA staff entitled
``Meetings With the Office of Orphan Products Development.'' Each year,
OOPD staff participates in meetings with stakeholders who seek guidance
or clarification relating to orphan drug or HUD designation requests,
OOPD grant programs, or other rare disease issues. These meetings can
be ``informal'' or ``formal'' and help build a common understanding on
FDA's thoughts on orphan products, which include drugs, biological
products, devices, or medical foods. These meetings may represent
critical points in the orphan product development process and may even
have an impact on the eventual availability of products for patients
with rare diseases and conditions. It is important that these meetings
be scheduled within a reasonable time, conducted effectively, and
documented where appropriate. This guidance is intended to provide
consistent procedures to promote well managed meetings between OOPD and
stakeholders.
    Topics addressed in this guidance include: (1) Clarification of
what constitutes an ``informal'' or ``formal'' meeting, (2) program
areas within OOPD that may be affected by this draft guidance, (3)
procedures for requesting and scheduling meetings with OOPD, (4)
description of what constitutes a meeting package, and (5) procedures
for the conduct and documentation of meetings with OOPD.
    This draft guidance is being issued consistent with FDA's good
guidance practices regulation (21 CFR 10.115). The draft guidance, when
finalized, will represent the Agency's current thinking on meetings
with OOPD. It does not create or confer any rights for or on any

person and does not operate to bind FDA or the public. An alternative
approach may be used if such approach satisfies the requirements of the
applicable statutes and regulations.

II. Paperwork Reduction Act of 1995

    Under the Paperwork Reduction Act of 1995 (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 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 comment 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 on respondents, including through the use of automated
collection techniques and other forms of information technology, when
appropriate.
    Title: Draft Guidance for Industry, Researchers, Patients Groups,
and Food and Drug Administration Staff on Meetings With the Office of
Orphan Products Development.
    Description: FDA is issuing a draft guidance on the procedures for
requesting meetings with OOPD on issues related to orphan drug
designation requests, HUD designation requests, rare pediatric disease
designation requests, funding opportunities through the Orphan Products
Grants Program and the Pediatric Device Consortia Grants Program, and
orphan product patient-related topics of concern. The draft guidance
describes procedures for requesting, scheduling, conducting, and
documenting such meetings.
    The draft guidance describes three collections of information: (1)
The submission of a meeting request (for informal and formal meetings),
(2) the submission of a meeting package (for formal meetings), and (3)
the submission of draft meeting minutes (for formal and certain
informal meetings). These collections of information will be used by
the Agency to schedule and prepare for meetings on the issues described
previously in this document and will provide for more productive
meetings with stakeholders. This draft guidance refers to previously
approved collections of information found in FDA regulations. Agency
regulations at part 316 (21 CFR part 316) describe information that
should be submitted in support of an orphan drug designation request.
The information collection provisions of part 316 have been approved
under OMB control number 0910-0167. Agency regulations at Sec.  814.102
(21 CFR 814.102) describe information that should be submitted in
support of a HUD designation request. The information collection
provisions of Sec.  814.102 have been approved under OMB control number
0910-0332.

A. Request for a Meeting

    Under the draft guidance, a stakeholder interested in meeting with
OOPD should submit a meeting request:
     For specific designation requests or grant applications,
by emailing the identified point of contact for the designation request
or grant application with the subject heading ``Meeting Request''; or
     For other issues, by emailing the general OOPD inbox at
<EMAIL_ADDRESS>with the subject heading ``Meeting Request'' or by
emailing the point of contact for each OOPD Program Area listed in the
``Contact FDA'' section of the OOPD's Web site (http://www.fda.gov/orphan), again with the subject heading ``Meeting Request.'' In the
draft guidance, FDA recommends that the meeting request, at a minimum,
include (1) a brief statement of the meeting purpose, (2) whether the
stakeholder prefers an informal or formal meeting, (3) suggested dates
and times for the meeting, (4) preferred format of the meeting, and (5)
the email address(es) to which OOPD should send a response to the
meeting request (if different from the email address from which the
request was sent) and telephone number for the primary contact for the
stakeholder. Before scheduling a meeting, OOPD may ask the stakeholder
for more information about the proposed meeting to help determine
whether an informal or formal meeting is most appropriate and who from
OOPD should attend. For informal meetings, the information in the
meeting request may suffice, although OOPD may ask for supplemental
information via email or telephone.

B. Meeting Package

    If a formal meeting is scheduled, FDA recommends that stakeholders
submit a meeting package to OOPD at least 2 weeks before the meeting.
Stakeholders are encouraged to submit the package electronically by
email to the OOPD program contact who scheduled the meeting. In the
draft guidance, FDA recommends that the meeting package contain the
following information: (1) The date, time, and subject of the meeting;
(2) an explanation of the meeting purposes; (3) basic information about
the product to be discussed (e.g., product name or identifier,
designation or application number (if applicable), proposed rare
disease or condition, brief background about the product); (4) proposed
meeting agenda; (5) any data, information, or presentation materials to
support the discussion (if needed); and (6) a list of all individuals,
with their titles and affiliations, who are expected to participate in
the meeting on behalf of the stakeholder.

C. Draft Meeting Minutes

    Under the draft guidance, a stakeholder should prepare a draft of
summary meeting minutes for all formal meetings and certain informal
meetings. These draft minutes should be sent to the OOPD program
contact by email with the subject heading ``Draft Meeting Minutes.''
The draft minutes should summarize the meeting discussion points,
agreements, disagreements, and action items. OOPD will review and
provide any revisions to the draft meeting minutes via email, and the
stakeholder will then either accept the version as final and notify
OOPD to that effect or will followup with questions and/or further
revisions.
    Description of Respondents: Individuals from industry, researchers,
patient groups, and other stakeholders who seek a meeting with OOPD
regarding orphan drug designation requests, HUD designation requests,
rare pediatric disease designation requests, funding opportunities
through the Orphan Products Grants Program and the Pediatric Device
Consortia Grants Program, and orphan product patient-related issues.
    Burden estimate: Table 1 of this document provides an estimate of
the annual reporting burden for the preparation and submission of
meeting

requests, meeting packages, and meeting minutes under the guidance.
    Request for a meeting: Based upon information collected from OOPD
program areas, approximately 2,120 informal and 46 formal meetings were
requested with OOPD in fiscal year (FY) 2013 regarding orphan drug
designation requests, HUD designation requests, rare pediatric disease
designation requests, funding opportunities through the Orphan Products
Grants Program and the Pediatric Device Consortia Grants Program, and
orphan product patient-related issues. FDA anticipates that the number
of meeting requests and stakeholders will remain the same or will only
slightly increase, and therefore estimates the total number of meeting
requests will be 2,166 annually (2120 informal and 46 formal meetings).
The hours per response, which is the estimated number of hours that a
stakeholder would spend preparing the information to be submitted with
a meeting request in accordance with the draft guidance, is estimated
to be approximately 3 hours for informal meetings and approximately 10
hours for formal meetings. Based on FDA's experience, the Agency
expects that it will take stakeholders this amount of time to gather
and copy brief statements about the product and a description of the
purpose and details of the meeting. Therefore, the Agency estimates
that stakeholders will spend 6,820 hours per year (6,360 hours for
informal meetings and 460 hours for formal meetings) preparing meeting
requests to OOPD regarding orphan drug designation requests, HUD
designation requests, rare pediatric disease designation requests,
funding opportunities through the Orphan Products Grants Program and
the Pediatric Device Consortia Grants Program, and orphan product
patient-related issues.
    Meeting package: Based upon information collected from OOPD program
areas, OOPD held approximately 46 formal meetings in FY 2013 regarding
orphan drug designation requests, HUD designation requests, rare
pediatric disease designation requests, funding opportunities through
the Orphan Products Grants Program and the Pediatric Device Consortia
Grants Program, and orphan product patient-related issues. FDA
anticipates that the number of formal meetings, and therefore meeting
packages, may increase only slightly as a result of this guidance;
thus, the Agency estimates that the total responses will be 46
annually. As stated previously, it is current practice for stakeholders
to submit meeting packages to the Agency in advance of any such formal
meeting. The hours per response, which is the estimated number of hours
that a stakeholder would spend preparing the meeting package in
accordance with this draft guidance, is estimated to be approximately
18 hours. Based on FDA's experience, the Agency expects it will take
stakeholders this amount of time to gather and copy brief statements
about the product, a description of details for the anticipated
meeting, and data and information that generally would already have
been compiled for submission to the Agency. Therefore, the Agency
estimates that stakeholders will spend 828 hours per year submitting
meeting packages to the Agency prior to a formal meeting regarding
orphan drug designation requests, HUD designation requests, rare
pediatric disease designation requests, funding opportunities through
the Orphan Products Grants Program and the Pediatric Device Consortia
Grants Program, and orphan product patient-related issues.
    Draft meeting minutes: Based upon information collected from OOPD
program areas, OOPD received approximately 46 draft meeting minutes for
formal meetings and 21 draft meeting minutes for informal meetings in
FY 2013 regarding orphan drug designation requests, HUD designation
requests, rare pediatric disease designation requests, funding
opportunities through the Orphan Products Grants Program and the
Pediatric Device Consortia Grants Program, and orphan product patient-
related issues. FDA anticipates that the number of stakeholders
submitting draft meeting minutes may remain the same or increase only
slightly; thus, the Agency estimates that the total number of
respondents will be 67 annually. As stated previously, it is current
practice for stakeholders to submit draft meeting minutes to the Agency
after all formal meetings and certain informal meetings. The hours per
response, which is the estimated number of hours that a stakeholder
would spend preparing draft meeting minutes in accordance with this
draft guidance, is estimated to be approximately 8 hours. Based on
FDA's experience, the Agency expects it will take stakeholders this
amount of time to summarize the meeting discussion points, agreements,
disagreements, and action items. Therefore, the Agency estimates that
stakeholders will spend 536 hours per year submitting draft meeting
minutes to the Agency documenting the meeting outcomes, agreements,
disagreements, and action items as followup to all formal and certain
informal meetings.
    FDA invites comments on this analysis of information collection
burdens.

                                 Table 1--Estimated Annual Reporting Burden \1\
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                                                     Number of                        Average
 Meeting requests, packages, and     Number of     responses per   Total annual     burden per      Total hours
             minutes               stakeholders     stakeholder      responses       response
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Meeting Requests (informal).....           2,120               1           2,120               3           6,360
Meeting Requests (formal).......              46               1              46              10             460
Meeting Packages................              46               1              46              18             828
Meeting Minutes.................              67               1              67               8             536
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    Total.......................  ..............  ..............  ..............  ..............           8,184
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\1\ There are no capital costs or operating and maintenance costs associated with this collection of
  information.

III. Comments

    Interested persons may submit either electronic comments regarding
this document to http://www.regulations.gov or written comments to the
Division of Dockets Management (see ADDRESSES). It is necessary to send
only one set of comments. Identify comments with the docket number
found in brackets in the heading of this document. Received comments
may be seen in the Division of Dockets Management between 9 a.m. and 4
p.m., Monday through Friday, and will be posted to the docket at http://www.regulations.gov.

IV. Electronic Access

    Persons with access to the Internet may obtain the document at
either http://www.fda.gov/RegulatoryInformation/Guidances/default.htm
or at http://www.regulations.gov.

    Dated: April 2, 2014.
Leslie Kux,
Assistant Commissioner for Policy.
[FR Doc. 2014-07908 Filed 4-8-14; 8:45 am]
BILLING CODE 4160-01-P