[Federal Register Volume 82, Number 187 (Thursday, September 28, 2017)]
[Notices]
[Pages 45291-45293]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2017-20798]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2017-N-0007]


Fee for Using a Rare Pediatric Disease Priority Review Voucher in 
Fiscal Year 2018

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) 2018. 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 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 and that 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 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 2018 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 http://www.fda.gov/downloads/forindustry/userfees/prescriptiondruguserfee/ucm511438.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: http://www.fda.gov/Drugs/DevelopmentApprovalProcess/DevelopmentResources/ucm375479.htm.
    This notice establishes the rare pediatric disease priority review 
fee rate for FY 2018 at $2,830,579 and outlines FDA's procedures for 
payment of rare pediatric disease priority review user fees. This rate 
is effective on October 1, 2017, and will remain in effect through 
September 30, 2018.

II. Rare Pediatric Priority Review User Fee for FY 2018

    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

[[Page 45292]]

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.
    FDA is setting a fee for FY 2018, which is to be based on standard 
cost data from the previous fiscal year, FY 2017. However, the FY 2017 
submission cohort has not been closed out yet, thus the cost data for 
FY 2017 are not complete. The latest year for which FDA has complete 
cost data is FY 2016. 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).
    The standard cost worksheets for FY 2016 show standard costs 
(rounded to the nearest hundred dollars) of $5,929,100 for an NME NDA, 
and $4,887,100 for a BLA. Based on these standard costs, the total cost 
to review the 49 applications in these two categories in FY 2016 (27 
NME NDAs with clinical data and 22 BLAs) was $267,601,900. (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.) 23 of these applications (14 NDAs and 9 BLAs) received 
priority review, which would mean that the remaining 26 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 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 2016 figures, the costs of a priority and standard review are 
estimated using the following formula:

(23 [alpha] x 1.67) + (26 [alpha]) = $267,601,900

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 
$4,154,664 (rounded to the nearest dollar) and the cost of a priority 
review for NME NDAs and BLAs is 1.67 times that amount, or $6,938,289 
(rounded to the nearest dollar). The difference between these two cost 
estimates, or $2,783,625, represents the incremental cost of conducting 
a priority review rather than a standard review.
    For the FY 2018 fee, FDA will need to adjust the FY 2016 
incremental cost by the average amount by which FDA's average costs 
increased in the 3 years prior to FY 2017, to adjust the FY 2016 amount 
for cost increases in FY 2017. That adjustment, published in the 
Federal Register on September 14, 2017 (82 FR 43244), setting the FY 
2018 PDUFA fee, is 1.6868 percent for the most recent year, not 
compounded. Increasing the FY 2016 incremental priority review cost of 
$2,783,625 by 1.6868 percent (or 0.016868) results in an estimated cost 
of $2,830,579 (rounded to the nearest dollar). This is the rare 
pediatric disease priority review user fee amount for FY 2018 that must 
be submitted with a priority review voucher for a human drug 
application in FY 2018, in addition to any PDUFA fee that is required 
for such an application.

III. Fee Schedule for FY 2018

    The fee rate for FY 2018 is set out in Table 1:

  Table 1--Rare Pediatric Disease Priority Review Schedule for FY 2018
------------------------------------------------------------------------
                                                         Fee rate for FY
                      Fee category                             2018
------------------------------------------------------------------------
Application submitted with a rare pediatric disease          $2,830,579
 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 [i.e., section 524 of the FD&C Act] and 360ff [i.e., 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, 2017. 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

[[Page 45293]]

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 https://userfees.fda.gov/pay (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 Pay.gov. 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 with a 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 Treasury, TREAS NYC, 33 Liberty St., New York, 
NY 10045, Account Number: 75060099, Routing Number: 021030004, SWIFT: 
FRNYUS33, Beneficiary: FDA, 8455 Colesville Rd., 14th Floor, Silver 
Spring, MD 20993-0002.

V. Reference

    The following reference is on display in the Dockets Management 
Staff (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 22, 2017.
Anna K. Abram,
Deputy Commissioner for Policy, Planning, Legislation, and Analysis.
[FR Doc. 2017-20798 Filed 9-27-17; 8:45 am]
 BILLING CODE 4164-01-P