Fee for Using a Rare Pediatric Disease Priority Review Voucher in Fiscal Year 2017, 67360-67362 [2016-23624]
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67360
Federal Register / Vol. 81, No. 190 / Friday, September 30, 2016 / Notices
FDA issued a document entitled
‘‘Revised Recommendations for
Reducing the Risk of Human
Immunodeficiency Virus Transmission
by Blood and Blood Products, Guidance
for Industry’’ dated December 2015
(https://www.fda.gov/downloads/
BiologicsBloodVaccines/Guidance
ComplianceRegulatoryInformation/
Guidances/Blood/UCM446580.pdf)
which changed the blood donor
criterion for men who have sex with
men (MSM) from an indefinite
(permanent) deferral to a 12-month
deferral since last MSM contact. The
impact of this change in the deferral
criteria requires a national monitoring
effort as part of TTIMS to assess if the
relative proportions of risk factors for
infection in blood donors have changed
following the adoption of the 12-month
donor deferral for MSM. TTIMS will use
similar procedures as the ones used in
the REDS–II study to monitor and
evaluate risk factors among HIV-positive
donors and recently HCV or HBV
infected donors as well as controls.
This study will help identify the
specific risk factors for TTI and their
prevalence in blood donors, and help
inform FDA on the proportion of
incident (new) infections among all HIV
positive blood donors. Donations with
incident infections have the greatest
potential transmission risk because they
could be missed during routine blood
screening. The study will help FDA
evaluate the effectiveness of screening
strategies in reducing the risk of HIV
transmission from at-risk donors and to
evaluate if there are unexpected
consequences associated with the recent
change in donor deferral policy such as
an increase in HIV incidence among
donors. These data also will inform FDA
regarding future blood donor deferral
policy options to reduce the risk of HIV
transmission, including the feasibility of
moving from the existing time-based
deferrals related to risk behaviors to
alternate deferral options, such as the
use of individual risk assessments, and
to inform the design of potential studies
to evaluate the feasibility and
effectiveness of such alternative deferral
options.
TTIMS will include a comprehensive
interview-based epidemiological study
of risk factor information for viral
infection-positive blood donors at the
American Red Cross (ARC), Blood
Systems, Inc. (BSI), New York Blood
Center (NYBC), and OneBlood that will
identify the current predominant risk
factors and reasons for virus-positive
donations. The TTIMS program
establishes a new, ongoing donor
hemovigilance capacity that currently
does not exist in the United States.
Using procedures developed by the
REDS–II study, TTIMS will establish
this capacity in greater than 50 percent
of all blood donations collected in the
country.
As part of the TTIMS project, a
comprehensive hemovigilance database
will be created that integrates the risk
factor information collected through
donor interviews of blood donor with
the resulting data from disease marker
testing and blood components collected
by participating organizations into a
research database. Following successful
initiation of the risk factor interviews,
the TTIMS network is poised to be
expanded to include additional blood
centers and/or re-focused on other
safety threats as warranted. In this way,
the TTIMS program will maintain
standardized, statistically and
scientifically robust processes for
applying hemovigilance information
across blood collection organizations.
The specific objectives are to:
• Determine current behavioral risk
factors associated with all HIV
infections, incident HBV, and incident
HCV infections in blood donors
(including parenteral and sexual risks)
across the participating blood collection
organizations using a case-control study
design.
• Determine infectious disease
marker prevalence and incidence for
HIV, HBV, and HCV overall and by
demographic characteristics of donors
in the majority of blood donations
collected in the country. This will be
accomplished by forming
epidemiological databases consisting of
harmonized operational data from ARC,
BSI, NYBC, and OneBlood.
• Analyze integrated risk factor and
infectious marker testing data
concurrently because when taken
together these may suggest that blood
centers are not achieving the same
degree of success in educational efforts
to prevent donation by donors with risk
behaviors across all demographic
groups.
The respondents will be persons who
donated blood in the United States and
these participants will be defined as
cases and controls. The estimated
number of respondents is based on an
overall expected participation in the
risk factor survey. We estimate a case to
control ratio of 1:2 (200 to 400) with a
50 percent case enrollment.
FDA estimates the burden of this
collection of information as follows:
TABLE 1—ESTIMATED ANNUAL REPORTING BURDEN 1
Questionnaire/survey
Number of
respondents
Number of
responses per
respondent
Total annual
responses
Average burden
per response
Total
hours
Cases and controls.2 ............................................
600
1
600
0.75 (45 minutes) ..........
450
1 There
2 Cases
are no capital costs or operating and maintenance costs associated with this collection of information.
consist of virus-positive donations, and controls represent uninfected donors.
Dated: September 26, 2016.
Leslie Kux,
Associate Commissioner for Policy.
mstockstill on DSK3G9T082PROD with NOTICES
[FR Doc. 2016–23622 Filed 9–29–16; 8:45 am]
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
[Docket No. FDA–2016–N–0007]
Fee for Using a Rare Pediatric Disease
Priority Review Voucher in Fiscal Year
2017
AGENCY:
Food and Drug Administration,
HHS.
20:49 Sep 29, 2016
Jkt 238001
Notice.
The Food and Drug
Administration (FDA or the Agency) is
announcing the fee rate for using a rare
pediatric disease priority review
voucher for fiscal year (FY) 2017. The
Federal Food, Drug, and Cosmetic Act
(the FD&C Act), as amended by the Food
and Drug Administration Safety and
Innovation Act (FDASIA), authorizes
FDA to determine and collect rare
pediatric disease priority review user
SUMMARY:
Food and Drug Administration
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receipt or filing date, depending on the
type of application. Information
regarding PDUFA goals is available at
https://www.fda.gov/downloads/
forindustry/userfees/
prescriptiondruguserfee/
ucm270412.pdf.
The applicant that uses a rare
pediatric disease priority review
voucher is entitled to a priority review
of its eligible human drug application,
but must pay FDA a rare pediatric
disease priority review user fee in
addition to any user fee required by
PDUFA for the application. Information
regarding the rare pediatric disease
priority review voucher program is
available at: https://www.fda.gov/Drugs/
DevelopmentApprovalProcess/
DevelopmentResources/
ucm375479.htm.
This notice establishes the rare
pediatric disease priority review fee rate
for FY 2017 at $2,706,000 and outlines
FDA’s procedures for payment of rare
pediatric disease priority review user
fees. This rate is effective on October 1,
2016, and will remain in effect through
September 30, 2017.
I. Background
mstockstill on DSK3G9T082PROD with NOTICES
fees for certain applications for review
of human drug or biological products
when those applications use a rare
pediatric disease priority review
voucher. These vouchers are awarded to
the sponsors of rare pediatric disease
product applications that meet all of the
requirements of this program, are
submitted 90 days or more after July 9,
2012, and upon FDA approval of such
applications. The amount of the fee for
using a rare pediatric disease priority
review voucher is determined each FY
based on the difference between the
average cost incurred by FDA in the
review of a human drug application
subject to priority review in the
previous FY, and the average cost
incurred in the review of an application
that is not subject to priority review in
the previous FY. This notice establishes
the rare pediatric disease priority review
fee rate for FY 2017 and outlines the
payment procedures for such fees.
FOR FURTHER INFORMATION CONTACT:
Robert J. Marcarelli, Office of Financial
Management, Food and Drug
Administration, 8455 Colesville Rd.,
COLE–14202F, Silver Spring, MD
20993–0002, 301–796–7223.
SUPPLEMENTARY INFORMATION:
Under section 529(c)(2) of the FD&C
Act, the amount of the rare pediatric
disease priority review user fee is
determined each fiscal year based on the
difference between the average cost
incurred by FDA in the review of a
human drug application subject to
priority review in the previous fiscal
year, and the average cost incurred by
FDA in the review of a human drug
application that is not subject to priority
review in the previous fiscal year.
A priority review is a review
conducted with a PDUFA goal date of 6
months after the receipt or filing date,
depending on the type of application.
Under the PDUFA goals letter, FDA has
committed to reviewing and acting on
90 percent of the applications granted
priority review status within this
expedited timeframe. Normally, an
application for a human drug or
biological product will qualify for
priority review if the product is
intended to treat a serious condition
and, if approved, would provide a
significant improvement in safety or
effectiveness. An application that does
not receive a priority designation will
receive a standard review. Under the
PDUFA goals letter, FDA has committed
to reviewing and acting on 90 percent of
standard applications within 10 months
of the receipt or filing date depending
on the type of application. A priority
review involves a more intensive level
Section 908 of FDASIA (Pub. L. 112–
144) added section 529 to the FD&C Act
(21 U.S.C. 360ff). In section 529 of the
FD&C Act, Congress encouraged
development of new human drugs and
biological products for prevention and
treatment of certain rare pediatric
diseases by offering additional
incentives for obtaining FDA approval
of such products. Under section 529 of
the FD&C Act, the sponsor of an eligible
human drug application submitted 90
days or more after July 9, 2012, for a rare
pediatric disease (as defined in section
529(a)(3)) shall receive a priority review
voucher upon approval of the rare
pediatric disease product application.
The recipient of a rare pediatric disease
priority review voucher may either use
the voucher for a future human drug
application submitted to FDA under
section 505(b)(1) of the FD&C Act (21
U.S.C. 355(b)(1)) or section 351(a) of the
Public Health Service Act (42 U.S.C.
262(a)), or transfer (including by sale)
the voucher to another party. The
voucher may be transferred (including
by sale) repeatedly until it ultimately is
used for a human drug application
submitted to FDA under section
505(b)(1) of the FD&C Act or section
351(a) of the Public Health Service Act.
A priority review is a review conducted
with a Prescription Drug User Fee Act
(PDUFA) goal date of 6 months after the
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II. Rare Pediatric Priority Review User
Fee for FY 2017
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67361
of effort and a higher level of resources
than a standard review.
Section 529 of the FD&C Act specifies
that the rare pediatric disease priority
review voucher fee amount must be
based on the difference between the
average cost incurred by the Agency in
the review of a human drug application
subject to a priority review in the
previous fiscal year, and the average
cost incurred by the Agency in the
review of a human drug application not
subject to a priority review in the
previous fiscal year. FDA is setting a fee
for FY 2017, which is to be based on
standard cost data from the previous
fiscal year, FY 2016. However, the FY
2016 submission cohort has not been
closed out yet, thus the cost data for FY
2016 are not complete. The latest year
for which FDA has complete cost data
is FY 2015. Furthermore, because FDA
has never tracked the cost of reviewing
applications that get priority review as
a separate cost subset, FDA estimated
this cost based on other data that the
Agency has tracked. FDA uses data that
the Agency estimates and publishes on
its Web site each year—standard costs
for review. FDA does not publish a
standard cost for ‘‘the review of a
human drug application subject to
priority review in the previous fiscal
year.’’ However, we expect all such
applications would contain clinical
data. The standard cost application
categories with clinical data that FDA
publishes each year are: (1) New drug
applications (NDAs) for a new
molecular entity (NME) with clinical
data and (2) biologics license
applications (BLAs) with clinical data.
The standard cost worksheets for FY
2015 show standard costs (rounded to
the nearest thousand dollars) of
$5,251,000 for an NME NDA, and
$5,055,000 for a BLA. Based on these
standard costs, the total cost to review
the 56 applications in these two
categories in FY 2015 (32 NME NDAs
and 24 BLAs with clinical data) was
$289,352,000. (Note: These numbers
exclude the President’s Emergency Plan
for AIDS Relief NDAs; no
investigational new drug (IND) review
costs are included in this amount.)
Twenty-five of these applications (18
NDAs and 7 BLAs) received priority
review, which would mean that the
remaining 31 received standard reviews.
Because a priority review compresses a
review schedule that ordinarily takes 10
months into 6 months, FDA estimates
that a multiplier of 1.67 (10 months
divided by 6 months) should be applied
to non-priority review costs in
estimating the effort and cost of a
priority review as compared to a
standard review. This multiplier is
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Federal Register / Vol. 81, No. 190 / Friday, September 30, 2016 / Notices
consistent with published research on
this subject which supports a priority
review multiplier in the range of 1.48 to
2.35 (Ref. 1). Using FY 2015 figures, the
costs of a priority and standard review
are estimated using the following
formula:
(25 a × 1.67) + (31 a) = $289,352,000
Where ‘‘a’’ is the cost of a standard
review and ‘‘a times 1.67’’ is the cost of
a priority review. Using this formula,
the cost of a standard review for NME
NDAs and BLAs is calculated to be
$3,977,000 (rounded to the nearest
thousand dollars) and the cost of a
priority review for NME NDAs and
BLAs is 1.67 times that amount, or
$6,642,000 (rounded to the nearest
thousand dollars). The difference
between these two cost estimates, or
$2,665,000, represents the incremental
cost of conducting a priority review
rather than a standard review.
For the FY 2017 fee, FDA will need
to adjust the FY 2015 incremental cost
by the average amount by which FDA’s
average costs increased in the 3 years
prior to FY 2016, to adjust the FY 2015
amount for cost increases in FY 2016.
That adjustment, published in the
Federal Register on July 28, 2016 (see
81 FR 49674 at 49676), setting the FY
2017 PDUFA fee, is 1.5468 percent for
the most recent year, not compounded.
Increasing the FY 2015 incremental
priority review cost of $2,665,000 by
1.5468 percent results in an estimated
cost of $2,706,000 (rounded to the
nearest thousand dollars). This is the
rare pediatric disease priority review
user fee amount for FY 2017 that must
be submitted with a priority review
voucher for a human drug application in
FY 2017, in addition to any PDUFA fee
that is required for such an application.
III. Fee Schedule for FY 2017
The fee rate for FY 2017 is set out in
table 1:
TABLE 1—RARE PEDIATRIC DISEASE
PRIORITY REVIEW SCHEDULE FOR
FY 2017
mstockstill on DSK3G9T082PROD with NOTICES
Fee category
Application submitted with a
rare pediatric disease priority review voucher in addition to the normal
PDUFA fee ........................
Fee rate for
FY 2017
$2,706,000
IV. Implementation of Rare Pediatric
Disease Priority Review User Fee
Under section 529(c)(4)(A) of the
FD&C Act, the priority review user fee
is due (i.e. the obligation to pay the fee
is incurred) when a sponsor notifies
VerDate Sep<11>2014
20:49 Sep 29, 2016
Jkt 238001
FDA of its intent to use the voucher.
Section 529(c)(4)(B) of the FD&C Act
specifies that the application will be
considered incomplete if the priority
review user fee and all other applicable
user fees are not paid in accordance
with FDA payment procedures. In
addition, section 529(c)(4)(C) specifies
that FDA may not grant a waiver,
exemption, reduction, or refund of any
fees due and payable under this section
of the FD&C Act. Beginning with FDA’s
appropriation for FY 2015, the annual
appropriation language states
specifically that ‘‘priority review user
fees authorized by 21 U.S.C. 360n and
360ff (section 529 of the FD&C Act)
shall be credited to this account, to
remain available until expended.’’ (Pub.
L. 113–235, Section 5, Division A, Title
VI).
The rare pediatric disease priority
review fee established in the new fee
schedule must be paid for any
application that is received on or after
October 1, 2016. In order to comply
with this requirement, the sponsor must
notify FDA 90 days prior to submission
of the human drug application that is
the subject of a priority review voucher
of an intent to submit the human drug
application, including the date on
which the sponsor intends to submit the
application.
Upon receipt of this notification, FDA
will issue an invoice to the sponsor who
has incurred a rare pediatric disease
priority review voucher fee. The invoice
will include instructions on how to pay
the fee via wire transfer or check.
As noted in section II, if a sponsor
uses a rare pediatric disease priority
review voucher for a human drug
application, the sponsor would incur
the rare pediatric disease priority review
voucher fee in addition to any PDUFA
fee that is required for the application.
The sponsor would need to follow
FDA’s normal procedures for timely
payment of the PDUFA fee for the
human drug application.
V. Reference
The following reference is on display
in the Division of Dockets Management
(HFA–305), Food and Drug
Administration, 5630 Fishers Lane, Rm.
1061, Rockville, MD 20852, and is
available for viewing by interested
persons between 9 a.m. and 4 p.m.,
Monday through Friday.
1. Ridley, D.B., H.G. Grabowski, and J.L. Moe,
‘‘Developing Drugs for Developing
Countries,’’ Health Affairs, vol. 25, no. 2,
pp. 313–324, 2006.
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Dated: September 26, 2016.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2016–23624 Filed 9–29–16; 8:45 am]
BILLING CODE 4164–01–P
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
Health Resources and Services
Administration
State Health Departments Coordinating
Center of the Jurisdictional Approach
To Curing Hepatitis C Among HIV/HCV
Coinfected People of Color
Demonstration Project Supported by
the Secretary’s Minority AIDS Initiative
Fund
Health Resources and Services
Administration (HRSA), Department of
Health and Human Services.
ACTION: Notice of a deviation from
competition requirements to make a
single-source award related to the
Jurisdictional Approach to Curing
Hepatitis C (HCV) among HIV/HCV
Coinfected People of Color
demonstration project.
AGENCY:
HRSA’s HIV/AIDS Bureau
(HAB) awarded a non-competitive
single source cooperative agreement to
National Alliance of State and
Territorial AIDS Directors (NASTAD)
for approximately $977,400 in the
Secretary’s Minority AIDS Initiative
Funds (SMAIF) as authorized under the
Consolidated Appropriations Act, 2016
(Pub L. 114–113), Division H, Title II.
Subject to the availability of funds and
NASTAD’s satisfactory performance,
HAB will also issue non-competitive,
single-source awards of approximately
$750,000 each in fiscal years (FY) 2017
and 2018. This will allow NASTAD to
facilitate the participation of up to two
Ryan White HIV/AIDS Program Part B
recipients in the Jurisdictional
Approach to Curing Hepatitis C among
HIV/HCV Coinfected People of Color
demonstration project over its 3-year
project period.
FOR FURTHER INFORMATION CONTACT:
Harold J. Phillips, Director, Office of
Training and Capacity Development,
HAB/HRSA, 5600 Fishers Lane, Room
9N–114, Rockville, MD 20857, by email
at hphillips@hrsa.gov or by phone at
(301) 443–8109.
SUPPLEMENTARY INFORMATION:
Recipient of the Award: National
Alliance of State and Territorial AIDS
Directors.
Period of Supplemental Funding:
September 30, 2016–September 29,
2019.
SUMMARY:
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Agencies
[Federal Register Volume 81, Number 190 (Friday, September 30, 2016)]
[Notices]
[Pages 67360-67362]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2016-23624]
-----------------------------------------------------------------------
DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2016-N-0007]
Fee for Using a Rare Pediatric Disease Priority Review Voucher in
Fiscal Year 2017
AGENCY: Food and Drug Administration, HHS.
ACTION: Notice.
-----------------------------------------------------------------------
SUMMARY: The Food and Drug Administration (FDA or the Agency) is
announcing the fee rate for using a rare pediatric disease priority
review voucher for fiscal year (FY) 2017. The Federal Food, Drug, and
Cosmetic Act (the FD&C Act), as amended by the Food and Drug
Administration Safety and Innovation Act (FDASIA), authorizes FDA to
determine and collect rare pediatric disease priority review user
[[Page 67361]]
fees for certain applications for review of human drug or biological
products when those applications use a rare pediatric disease priority
review voucher. These vouchers are awarded to the sponsors of rare
pediatric disease product applications that meet all of the
requirements of this program, are submitted 90 days or more after July
9, 2012, and upon FDA approval of such applications. The amount of the
fee for using a rare pediatric disease priority review voucher is
determined each FY based on the difference between the average cost
incurred by FDA in the review of a human drug application subject to
priority review in the previous FY, and the average cost incurred in
the review of an application that is not subject to priority review in
the previous FY. This notice establishes the rare pediatric disease
priority review fee rate for FY 2017 and outlines the payment
procedures for such fees.
FOR FURTHER INFORMATION CONTACT: Robert J. Marcarelli, Office of
Financial Management, Food and Drug Administration, 8455 Colesville
Rd., COLE-14202F, Silver Spring, MD 20993-0002, 301-796-7223.
SUPPLEMENTARY INFORMATION:
I. Background
Section 908 of FDASIA (Pub. L. 112-144) added section 529 to the
FD&C Act (21 U.S.C. 360ff). In section 529 of the FD&C Act, Congress
encouraged development of new human drugs and biological products for
prevention and treatment of certain rare pediatric diseases by offering
additional incentives for obtaining FDA approval of such products.
Under section 529 of the FD&C Act, the sponsor of an eligible human
drug application submitted 90 days or more after July 9, 2012, for a
rare pediatric disease (as defined in section 529(a)(3)) shall receive
a priority review voucher upon approval of the rare pediatric disease
product application. The recipient of a rare pediatric disease priority
review voucher may either use the voucher for a future human drug
application submitted to FDA under section 505(b)(1) of the FD&C Act
(21 U.S.C. 355(b)(1)) or section 351(a) of the Public Health Service
Act (42 U.S.C. 262(a)), or transfer (including by sale) the voucher to
another party. The voucher may be transferred (including by sale)
repeatedly until it ultimately is used for a human drug application
submitted to FDA under section 505(b)(1) of the FD&C Act or section
351(a) of the Public Health Service Act. A priority review is a review
conducted with a Prescription Drug User Fee Act (PDUFA) goal date of 6
months after the receipt or filing date, depending on the type of
application. Information regarding PDUFA goals is available at https://www.fda.gov/downloads/forindustry/userfees/prescriptiondruguserfee/ucm270412.pdf.
The applicant that uses a rare pediatric disease priority review
voucher is entitled to a priority review of its eligible human drug
application, but must pay FDA a rare pediatric disease priority review
user fee in addition to any user fee required by PDUFA for the
application. Information regarding the rare pediatric disease priority
review voucher program is available at: https://www.fda.gov/Drugs/DevelopmentApprovalProcess/DevelopmentResources/ucm375479.htm.
This notice establishes the rare pediatric disease priority review
fee rate for FY 2017 at $2,706,000 and outlines FDA's procedures for
payment of rare pediatric disease priority review user fees. This rate
is effective on October 1, 2016, and will remain in effect through
September 30, 2017.
II. Rare Pediatric Priority Review User Fee for FY 2017
Under section 529(c)(2) of the FD&C Act, the amount of the rare
pediatric disease priority review user fee is determined each fiscal
year based on the difference between the average cost incurred by FDA
in the review of a human drug application subject to priority review in
the previous fiscal year, and the average cost incurred by FDA in the
review of a human drug application that is not subject to priority
review in the previous fiscal year.
A priority review is a review conducted with a PDUFA goal date of 6
months after the receipt or filing date, depending on the type of
application. Under the PDUFA goals letter, FDA has committed to
reviewing and acting on 90 percent of the applications granted priority
review status within this expedited timeframe. Normally, an application
for a human drug or biological product will qualify for priority review
if the product is intended to treat a serious condition and, if
approved, would provide a significant improvement in safety or
effectiveness. An application that does not receive a priority
designation will receive a standard review. Under the PDUFA goals
letter, FDA has committed to reviewing and acting on 90 percent of
standard applications within 10 months of the receipt or filing date
depending on the type of application. A priority review involves a more
intensive level of effort and a higher level of resources than a
standard review.
Section 529 of the FD&C Act specifies that the rare pediatric
disease priority review voucher fee amount must be based on the
difference between the average cost incurred by the Agency in the
review of a human drug application subject to a priority review in the
previous fiscal year, and the average cost incurred by the Agency in
the review of a human drug application not subject to a priority review
in the previous fiscal year. FDA is setting a fee for FY 2017, which is
to be based on standard cost data from the previous fiscal year, FY
2016. However, the FY 2016 submission cohort has not been closed out
yet, thus the cost data for FY 2016 are not complete. The latest year
for which FDA has complete cost data is FY 2015. Furthermore, because
FDA has never tracked the cost of reviewing applications that get
priority review as a separate cost subset, FDA estimated this cost
based on other data that the Agency has tracked. FDA uses data that the
Agency estimates and publishes on its Web site each year--standard
costs for review. FDA does not publish a standard cost for ``the review
of a human drug application subject to priority review in the previous
fiscal year.'' However, we expect all such applications would contain
clinical data. The standard cost application categories with clinical
data that FDA publishes each year are: (1) New drug applications (NDAs)
for a new molecular entity (NME) with clinical data and (2) biologics
license applications (BLAs) with clinical data.
The standard cost worksheets for FY 2015 show standard costs
(rounded to the nearest thousand dollars) of $5,251,000 for an NME NDA,
and $5,055,000 for a BLA. Based on these standard costs, the total cost
to review the 56 applications in these two categories in FY 2015 (32
NME NDAs and 24 BLAs with clinical data) was $289,352,000. (Note: These
numbers exclude the President's Emergency Plan for AIDS Relief NDAs; no
investigational new drug (IND) review costs are included in this
amount.) Twenty-five of these applications (18 NDAs and 7 BLAs)
received priority review, which would mean that the remaining 31
received standard reviews. Because a priority review compresses a
review schedule that ordinarily takes 10 months into 6 months, FDA
estimates that a multiplier of 1.67 (10 months divided by 6 months)
should be applied to non-priority review costs in estimating the effort
and cost of a priority review as compared to a standard review. This
multiplier is
[[Page 67362]]
consistent with published research on this subject which supports a
priority review multiplier in the range of 1.48 to 2.35 (Ref. 1). Using
FY 2015 figures, the costs of a priority and standard review are
estimated using the following formula:
(25 [alpha] x 1.67) + (31 [alpha]) = $289,352,000
Where ``[alpha]'' is the cost of a standard review and ``[alpha]
times 1.67'' is the cost of a priority review. Using this formula, the
cost of a standard review for NME NDAs and BLAs is calculated to be
$3,977,000 (rounded to the nearest thousand dollars) and the cost of a
priority review for NME NDAs and BLAs is 1.67 times that amount, or
$6,642,000 (rounded to the nearest thousand dollars). The difference
between these two cost estimates, or $2,665,000, represents the
incremental cost of conducting a priority review rather than a standard
review.
For the FY 2017 fee, FDA will need to adjust the FY 2015
incremental cost by the average amount by which FDA's average costs
increased in the 3 years prior to FY 2016, to adjust the FY 2015 amount
for cost increases in FY 2016. That adjustment, published in the
Federal Register on July 28, 2016 (see 81 FR 49674 at 49676), setting
the FY 2017 PDUFA fee, is 1.5468 percent for the most recent year, not
compounded. Increasing the FY 2015 incremental priority review cost of
$2,665,000 by 1.5468 percent results in an estimated cost of $2,706,000
(rounded to the nearest thousand dollars). This is the rare pediatric
disease priority review user fee amount for FY 2017 that must be
submitted with a priority review voucher for a human drug application
in FY 2017, in addition to any PDUFA fee that is required for such an
application.
III. Fee Schedule for FY 2017
The fee rate for FY 2017 is set out in table 1:
Table 1--Rare Pediatric Disease Priority Review Schedule for FY 2017
------------------------------------------------------------------------
Fee rate for FY
Fee category 2017
------------------------------------------------------------------------
Application submitted with a rare pediatric disease $2,706,000
priority review voucher in addition to the normal
PDUFA fee.............................................
------------------------------------------------------------------------
IV. Implementation of Rare Pediatric Disease Priority Review User Fee
Under section 529(c)(4)(A) of the FD&C Act, the priority review
user fee is due (i.e. the obligation to pay the fee is incurred) when a
sponsor notifies FDA of its intent to use the voucher. Section
529(c)(4)(B) of the FD&C Act specifies that the application will be
considered incomplete if the priority review user fee and all other
applicable user fees are not paid in accordance with FDA payment
procedures. In addition, section 529(c)(4)(C) specifies that FDA may
not grant a waiver, exemption, reduction, or refund of any fees due and
payable under this section of the FD&C Act. Beginning with FDA's
appropriation for FY 2015, the annual appropriation language states
specifically that ``priority review user fees authorized by 21 U.S.C.
360n and 360ff (section 529 of the FD&C Act) shall be credited to this
account, to remain available until expended.'' (Pub. L. 113-235,
Section 5, Division A, Title VI).
The rare pediatric disease priority review fee established in the
new fee schedule must be paid for any application that is received on
or after October 1, 2016. In order to comply with this requirement, the
sponsor must notify FDA 90 days prior to submission of the human drug
application that is the subject of a priority review voucher of an
intent to submit the human drug application, including the date on
which the sponsor intends to submit the application.
Upon receipt of this notification, FDA will issue an invoice to the
sponsor who has incurred a rare pediatric disease priority review
voucher fee. The invoice will include instructions on how to pay the
fee via wire transfer or check.
As noted in section II, if a sponsor uses a rare pediatric disease
priority review voucher for a human drug application, the sponsor would
incur the rare pediatric disease priority review voucher fee in
addition to any PDUFA fee that is required for the application. The
sponsor would need to follow FDA's normal procedures for timely payment
of the PDUFA fee for the human drug application.
V. Reference
The following reference is on display in the Division of Dockets
Management (HFA-305), Food and Drug Administration, 5630 Fishers Lane,
Rm. 1061, Rockville, MD 20852, and is available for viewing by
interested persons between 9 a.m. and 4 p.m., Monday through Friday.
1. Ridley, D.B., H.G. Grabowski, and J.L. Moe, ``Developing Drugs
for Developing Countries,'' Health Affairs, vol. 25, no. 2, pp. 313-
324, 2006.
Dated: September 26, 2016.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2016-23624 Filed 9-29-16; 8:45 am]
BILLING CODE 4164-01-P