Notice2021-21329
Fee Rate for Using a Rare Pediatric Disease Priority Review Voucher in Fiscal Year 2022
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Published
September 30, 2021
Issuing agencies
Health and Human Services DepartmentFood and Drug Administration
Abstract
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) 2022. The Federal Food, Drug, and Cosmetic Act (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 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 sponsors of rare pediatric disease product applications that meet all the requirements of this program and are submitted 90 days or more after July 9, 2012, 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 to review a human drug application designated as 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 2022 and outlines the payment procedures for such fees.
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<title>Federal Register, Volume 86 Issue 187 (Thursday, September 30, 2021)</title>
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[Federal Register Volume 86, Number 187 (Thursday, September 30, 2021)]
[Notices]
[Pages 54217-54219]
From the Federal Register Online via the Government Publishing Office [<a href="http://www.gpo.gov">www.gpo.gov</a>]
[FR Doc No: 2021-21329]
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2021-N-0982]
Fee Rate for Using a Rare Pediatric Disease Priority Review
Voucher in Fiscal Year 2022
AGENCY: Food and Drug Administration, HHS.
ACTION: Notice.
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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) 2022. The Federal Food, Drug, and
Cosmetic Act (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 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 sponsors of rare pediatric disease
product applications that meet all the requirements of this program and
are submitted 90 days or more after July 9, 2012, 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 to review a human
drug application designated as 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 2022 and
outlines the payment procedures for such fees.
FOR FURTHER INFORMATION CONTACT: Tim Davidson, Office of Financial
Management, Food and Drug Administration, 4041 Powder Mill Rd., Rm.
61077A, Beltsville, MD 20705-4304, 301-796-3254.
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 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 current PDUFA goals is available at: <a href="https://www.fda.gov/media/99140/download">https://www.fda.gov/media/99140/download</a>.
The sponsor 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: <a href="https://www.fda.gov/Drugs/DevelopmentApprovalProcess/DevelopmentResources/ucm375479.htm">https://www.fda.gov/Drugs/DevelopmentApprovalProcess/DevelopmentResources/ucm375479.htm</a>.
This notice establishes the rare pediatric disease priority review
fee rate for FY 2022 at $1,266,651 and outlines FDA's payment
procedures for rare
[[Page 54218]]
pediatric disease priority review user fees. This rate is effective on
October 1, 2021, and will remain in effect through September 30, 2022.
II. Rare Pediatric Priority Review User Fee Rate for FY 2022
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. As described in 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 receives a standard review. As described in 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.
FDA is setting a fee for FY 2022, which is to be based on standard
cost data from the previous fiscal year, FY 2021. However, the FY 2021
submission cohort has not been closed out yet, thus the cost data for
FY 2021 are not complete. The latest year for which FDA has complete
cost data is FY 2020. 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. The Agency expects all applications that received
priority review would contain clinical data. The application categories
with clinical data for which FDA tracks the cost of review are: (1) New
drug applications (NDAs) for a new molecular entity (NME) with clinical
data and (2) biologics license applications (BLAs).
The total cost for FDA to review NME NDAs with clinical data and
BLAs in FY 2020 was $227,248,467. There was a total of 86 applications
in these two categories (53 NME NDAs with clinical data and 33 BLAs).
(Note: These numbers exclude the President's Emergency Plan for AIDS
Relief NDAs; no investigational new drug review costs are included in
this amount.) Of these applications 55 (35 NDAs and 20 BLAs) received
priority review and the remaining 31 (18 NDAs and 13 BLAs) 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 / 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
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 2020 figures, the costs of a priority and standard review are
estimated using the following formula:
(55 [alpha] x 1.67) + (31 [alpha]) = $227,248,467
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
$1,849,804 (rounded to the nearest dollar) and the cost of a priority
review for NME NDAs and BLAs is 1.67 times that amount, or $3,089,173
(rounded to the nearest dollar). The difference between these two cost
estimates, or $1,239,369, represents the incremental cost of conducting
a priority review rather than a standard review.
For the FY 2022 fee, FDA will need to adjust the FY 2020
incremental cost by the average amount by which FDA's average costs
increased in the 3 years prior to FY 2021, to adjust the FY 2020 amount
for cost increases in FY 2021. That adjustment, published in the
Federal Register on August 16, 2021 (86 FR 45732), setting the FY 2022
PDUFA fees, is 2.2013 percent for the most recent year, not compounded.
Increasing the FY 2020 incremental priority review cost of $1,239,369
by 2.2013 percent (or 0.022013) results in an estimated cost of
$1,266,651 (rounded to the nearest dollar). This is the rare pediatric
disease priority review user fee amount for FY 2022 that must be
submitted with a priority review voucher for a human drug application
in FY 2022, in addition to any PDUFA fee that is required for such an
application.
III. Fee Rate Schedule for FY 2022
The fee rate for FY 2022 is set in table 1:
Table 1--Rare Pediatric Disease Priority Review Schedule for FY 2022
------------------------------------------------------------------------
Priority review
Fee category fee rate for
FY 2022
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Application submitted with a rare pediatric disease $1,266,651
priority review voucher in addition to the normal
PDUFA fee.............................................
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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.
The rare pediatric disease priority review fee established in the
new fee schedule must be paid for applications submitted with a
priority review voucher received on or after October 1, 2021. 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 estimated submission date.
Upon receipt of this notification, FDA will issue an invoice to the
sponsor for the rare pediatric disease priority review voucher fee. The
invoice will include instructions on how to pay the fee via
[[Page 54219]]
wire transfer, check, or online payments.
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.
Payment must be made in U.S. currency by electronic check, check,
bank draft, wire transfer, credit card, or U.S. postal money order
payable to the order of the Food and Drug Administration. The preferred
payment method is online using electronic check (Automated Clearing
House (ACH) also known as eCheck). Secure electronic payments can be
submitted using the User Fees Payment Portal at <a href="https://userfees.fda.gov/pay">https://userfees.fda.gov/pay</a> (Note: Only full payments are accepted. No partial
payments can be made online). Once you search for your invoice, select
``Pay Now'' to be redirected to <a href="http://Pay.gov">Pay.gov</a>. Note that electronic payment
options are based on the balance due. Payment by credit card is
available for balances that are less than $25,000. If the balance
exceeds this amount, only the ACH option is available. Payments must be
made using U.S bank accounts as well as U.S. credit cards.
If paying by paper check the invoice number should be included on
the check, followed by the words ``Rare Pediatric Disease Priority
Review.'' All paper checks must be in U.S. currency from a U.S. bank
made payable and mailed to: Food and Drug Administration, P.O. Box
979107, St. Louis, MO 63197-9000.
If checks are sent by a courier that requests a street address, the
courier can deliver the checks to: U.S. Bank, Attention: Government
Lockbox 979107, 1005 Convention Plaza, St. Louis, MO 63101. (Note: This
U.S. Bank address is for courier delivery only. If you have any
questions concerning courier delivery, contact the U.S. Bank at 314-
418-4013. This telephone number is only for questions about courier
delivery). The FDA post office box number (P.O. Box 979107) must be
written on the check. If needed, FDA's tax identification number is 53-
0196965.
If paying by wire transfer, please reference your invoice number
when completing your transfer. The originating financial institution
may charge a wire transfer fee. If the financial institution charges a
wire transfer fee, it is required to add that amount to the payment to
ensure that the invoice is paid in full. The account information is as
follows: U.S. Dept. of the Treasury, TREAS NYC, 33 Liberty St., New
York, NY 10045, Account Number: 75060099, Routing Number: 021030004,
SWIFT: FRNYUS33.
V. Reference
The following reference is on display at the Dockets Management
Staff (HFA-305), Food and Drug Administration, 5630 Fishers Lane, Rm.
1061, Rockville, MD 20852, 240-402-7500, and is available for viewing
by interested persons between 9 a.m. and 4 p.m., Monday through Friday;
it is not available electronically at <a href="https://www.regulations.gov">https://www.regulations.gov</a> as
this reference is copyright protected. FDA has verified the website
address, as of the date this document publishes in the Federal
Register, but websites are subject to change over time.
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, available at: <a href="https://www.healthaffairs.org/doi/full/10.1377/hlthaff.25.2.313">https://www.healthaffairs.org/doi/full/10.1377/hlthaff.25.2.313</a>.
Dated: September 27, 2021.
Lauren K. Roth,
Acting Principal Associate Commissioner for Policy.
[FR Doc. 2021-21329 Filed 9-29-21; 8:45 am]
BILLING CODE 4164-01-P
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