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, 21471-21473 [2014-08589]
Download as PDF
<![CDATA[
Federal Register / Vol. 79, No. 73 / Wednesday, April 16, 2014 / Notices
additive petition ranges from $1,600 to
$3,000, depending on the intended use
of the color and the scope of the
requested amendment. A complete
schedule of fees is set forth in § 70.19.
An average of one Category A and one
Category B color additive petition is
expected per year. The maximum color
additive petition fee for a Category A
petition is $2,600 and the maximum
color additive petition fee for a Category
B petition is $3,000. Because an average
of 2 color additive petitions are
expected per calendar year, the
estimated total annual cost burden to
petitioners for this start-up cost would
be less than or equal to $5,600 (1 ×
$2,600 + 1 × $3,000 listing fees =
$5,600). There are no capital costs
associated with color additive petitions.
The labeling requirements for food
and color additives were designed to
specify the minimum information
needed for labeling in order that food
and color manufacturers may comply
with all applicable provisions of the
FD&C Act and other specific labeling
acts administered by FDA. Label
information does not require any
additional information gathering beyond
what is already required to assure
conformance with all specifications and
limitations in any given food or color
additive regulation. Label information
does not have any specific
recordkeeping requirements unique to
preparing the label. Therefore, because
labeling requirements under § 70.25 for
a particular color additive involve
information required as part of the CAP
safety review process, the estimate for
number of respondents is the same for
§ 70.25 and § 71.1, and the burden hours
for labeling are included in the estimate
for § 71.1. Also, because labeling
requirements under parts 172, 173, 179,
and 180 for particular food additives
involve information required as part of
the FAP safety review process under
§ 171.1, the burden hours for labeling
are included in the estimate for § 171.1.
Dated: April 10, 2014.
Leslie Kux,
Assistant Commissioner for Policy.
[FR Doc. 2014–08590 Filed 4–15–14; 8:45 am]
pmangrum on DSK3VPTVN1PROD with NOTICES
BILLING CODE 4160–01–P
VerDate Mar<15>2010
15:20 Apr 15, 2014
Jkt 232001
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA–2014–N–0386]
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
AGENCY:
Food and Drug Administration,
HHS.
ACTION:
Notice.
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).
DATES: Submit written or electronic
comments on the collection of
information by June 16, 2014.
ADDRESSES: Submit electronic
comments on the collection of
information to https://
www.regulations.gov. 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: FDA
PRA Staff, Office of Operations, Food
and Drug Administration, 1350 Piccard
Dr., PI50–400B, Rockville, MD 20850,
PRAStaff@fda.hhs.gov.
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.
SUMMARY:
PO 00000
Frm 00042
Fmt 4703
Sfmt 4703
21471
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.
E:\FR\FM\16APN1.SGM
16APN1
21472
Federal Register / Vol. 79, No. 73 / Wednesday, April 16, 2014 / Notices
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
Number of
respondents
pmangrum on DSK3VPTVN1PROD with NOTICES
21 CFR Section
Number of
responses per
respondent
Total annual
responses
Average
burden per
response
Total hours
316.10, 316.12, and 316.14 .................................................
316.20, 316.21, and 316.26 .................................................
FDA Form 3671 ...................................................................
316.22 ..................................................................................
316.27 ..................................................................................
316.30 ..................................................................................
316.36 ..................................................................................
2
225
50
65
43
450
2
1
2
3
1
1
1
3
2
450
150
65
43
450
6
100
150
45
2
5
3
15
200
67,500
6,750
130
215
1,350
90
Total ..............................................................................
........................
........................
........................
........................
76,235
1 There
are no capital costs or operating and maintenance costs associated with this collection of information.
VerDate Mar<15>2010
15:20 Apr 15, 2014
Jkt 232001
PO 00000
Frm 00043
Fmt 4703
Sfmt 4703
E:\FR\FM\16APN1.SGM
16APN1
Federal Register / Vol. 79, No. 73 / Wednesday, April 16, 2014 / Notices
Dated: April 10, 2014.
Leslie Kux,
Assistant Commissioner for Policy.
[FR Doc. 2014–08589 Filed 4–15–14; 8:45 am]
BILLING CODE 4160–01–P
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA–2014–N–0001]
Advancing the Development of
Pediatric Therapeutics: Pediatric Bone
Health; Public Workshop
AGENCY:
Food and Drug Administration,
HHS.
Notice; rescheduling of public
workshop.
pmangrum on DSK3VPTVN1PROD with NOTICES
ACTION:
The Food and Drug Administration’s
(FDA) Pediatric and Maternal Health
Staff in the Center for Drug Evaluation
and Research and the Office of Pediatric
Therapeutics are announcing the
rescheduling of a 1-day public
workshop entitled ‘‘Advancing the
Development of Pediatric Therapeutics
(ADEPT): Pediatric Bone Health.’’ The
purpose of this initial workshop is to
provide a forum to consider issues
related to advancing pediatric regulatory
science in the evaluation of bone health
in pediatric patients. The workshop
scheduled for March 4, 2014, was
postponed due to unanticipated weather
conditions and rescheduled for June 3,
2014.
Date and Time: The public workshop
will be held on June 3, 2014, from 8 a.m.
to 5:30 p.m. This workshop is being
rescheduled because of a postponed
workshop announced in the Federal
Register of February 6, 2014 (79 FR
7205), originally scheduled for March 4,
2014.
Location: The public workshop will
be held at the FDA White Oak Campus,
10903 New Hampshire Ave., Bldg. 2,
Rm. 2047, Silver Spring, MD 20993.
Entrance for the public workshop
participants (non-FDA employees) is
through Building 1 where routine
security procedures will be performed.
Please visit the following Web site for
location, parking, security, and travel
information: https://www.fda.gov/
AboutFDA/WorkingatFDA/
BuildingsandFacilities/
WhiteOakCampusInformation/
ucm241740.htm.
Contact Person: Denise Pica-Branco,
Center for Drug Evaluation and
Research, Food and Drug
Administration, 10903 New Hampshire
Ave., Silver Spring, MD 20993–0002,
VerDate Mar<15>2010
15:20 Apr 15, 2014
Jkt 232001
301–796–1732, FAX: 301–796–9858,
email: denise.picabranco@fda.hhs.gov.
Registration: There is no fee to attend
the public workshop, but attendees
should register in advance. Space is
limited and registration will be on a
first-come, first-served basis. Persons
interested in attending this workshop
must register online at
PediatricBoneHealth@fda.hhs.gov
before May 23, 2014. If you registered
for the workshop before March 4, 2014,
you must re-register for the workshop.
For those without Internet access, please
contact Denise Pica-Branco (see Contact
Person) to register. Onsite registration
will not be available.
If you need special accommodations
due to a disability, please contact
Denise Pica-Branco (see Contact Person)
at least 7 days in advance.
SUPPLEMENTARY INFORMATION: FDA has
engaged experts to address challenging
issues related to the evaluation of effects
on bone health for products used to treat
pediatric patients. Identification of
signals in animal studies and adult
clinical trials that warrant further
clinical investigation and identification
of biomarkers that may be predictive of
bone health in children will be
discussed. Additionally, strategies and
methods to address the challenges of
assessing long-term bone health for
products used to treat pediatric patients
will be discussed.
Information about this meeting is also
available at https://www.fda.gov/Drugs/
NewsEvents/ucm132703.htm.
Dated: April 10, 2014.
Leslie Kux,
Assistant Commissioner for Policy.
[FR Doc. 2014–08592 Filed 4–15–14; 8:45 am]
BILLING CODE 4160–01–P
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA–2014–N–0406]
Proposed Strategy and
Recommendations for a Risk-Based
Framework for Food and Drug
Administration Safety and Innovation
Act Health Information Technology;
Public Workshop; Request for
Comments
AGENCY:
Food and Drug Administration,
HHS.
Notice of public workshop;
request for comments.
ACTION:
The Food and Drug Administration
(FDA) is announcing the following
public workshop entitled ‘‘Proposed
PO 00000
Frm 00044
Fmt 4703
Sfmt 4703
21473
Strategy and Recommendations for a
Risk-Based Framework for Food and
Drug Administration Safety and
Innovation Act Health Information
Technology.’’ FDA, the Office of the
National Coordinator for Health
Information Technology (ONC), and the
Federal Communication Commission
(FCC) (collectively referred for the
purpose of this notice as ‘‘the
Agencies’’ 1) seek broad input from
stakeholders and experts on the
proposed strategy and recommendations
for a risk-based framework for the Food
and Drug Administration Safety and
Innovation Act (FDASIA) Health
Information Technology (IT). The topic
to be discussed is the FDASIA Health IT
report that contains a proposed strategy
and recommendations on an
appropriate, risk-based framework for
health IT that promotes innovation,
protects patient safety, and avoids
regulatory duplication.
DATES: Dates and Times: The public
workshop will be held on May 13–15,
2014, from 8 a.m. to 5 p.m.
Location: The public workshop will
be held at National Institute of
Standards and Technology, 100 Bureau
Dr., Building 101, Red Auditorium,
Gaithersburg, MD 20899–1070.
Contact Person: Bakul Patel, Center
for Devices and Radiological Health,
Food and Drug Administration, 10903
New Hampshire Ave., Bldg. 66, Rm.
5456, Silver Spring, MD 20993, 301–
796–5528, email: Bakul.patel@
fda.hhs.gov.
Registration: Registration is free and
available on a first-come, first-served
basis. Persons interested in attending
this public workshop must register
online by 4 p.m. on May 2, 2014. Early
registration is recommended because
facilities are limited and, therefore, FDA
may limit the number of participants
from each organization. Onsite
registration on the day of the public
workshop will not be available.
If you need special accommodations
due to a disability, please contact Susan
Monahan, Food and Drug
Administration, Center for Devices and
Radiological Health, 10903 New
Hampshire Ave., Bldg. 66, Rm. 4321,
Silver Spring MD 20993, 301–796–5661,
email: susan.monahan@fda.hhs.gov no
later than April 29, 2014.
To register for the public workshop,
please visit FDA’s Medical Devices
News & Events—Workshops &
Conferences calendar at https://
www.fda.gov/MedicalDevices/
NewsEvents/WorkshopsConferences/
default.htm. (Select this public
1 ONC is not an agency, but an office, within the
Department of Health and Human Services.
E:\FR\FM\16APN1.SGM
16APN1
]]>Agencies
[Federal Register Volume 79, Number 73 (Wednesday, April 16, 2014)]
[Notices]
[Pages 21471-21473]
From the Federal Register Online via the Government Printing Office [www.gpo.gov]
[FR Doc No: 2014-08589]
-----------------------------------------------------------------------
DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2014-N-0386]
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
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 Drug Products: Common
EMEA/FDA Application Form for Orphan Medicinal Product Designation
(Form FDA 3671).
DATES: Submit written or electronic comments on the collection of
information by June 16, 2014.
ADDRESSES: Submit electronic comments on the collection of information
to https://www.regulations.gov. 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: FDA PRA Staff, Office of Operations,
Food and Drug Administration, 1350 Piccard Dr., PI50-400B, Rockville,
MD 20850, PRAStaff@fda.hhs.gov.
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 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.
[[Page 21472]]
One revision concerns the name of the drug in an orphan-drug
designation request. As provided in current Sec. 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 Sec. 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 Sec. 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 Sec. 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\
----------------------------------------------------------------------------------------------------------------
Number of
21 CFR Section Number of responses per Total annual Average burden Total hours
respondents respondent responses per response
----------------------------------------------------------------------------------------------------------------
316.10, 316.12, and 316.14...... 2 1 2 100 200
316.20, 316.21, and 316.26...... 225 2 450 150 67,500
FDA Form 3671................... 50 3 150 45 6,750
316.22.......................... 65 1 65 2 130
316.27.......................... 43 1 43 5 215
316.30.......................... 450 1 450 3 1,350
316.36.......................... 2 3 6 15 90
-------------------------------------------------------------------------------
Total....................... .............. .............. .............. .............. 76,235
----------------------------------------------------------------------------------------------------------------
\1\ There are no capital costs or operating and maintenance costs associated with this collection of
information.
[[Page 21473]]
Dated: April 10, 2014.
Leslie Kux,
Assistant Commissioner for Policy.
[FR Doc. 2014-08589 Filed 4-15-14; 8:45 am]
BILLING CODE 4160-01-P
