81 FR 67360 - Fee for Using a Rare Pediatric Disease Priority Review Voucher in Fiscal Year 2017
DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration Federal Register Volume 81, Issue 190 (September 30, 2016)
Food and Drug Administration
Federal Register Volume 81, Issue 190 (September 30, 2016)
| Page Range | 67360-67362 | |
| FR Document | 2016-23624 |
[Federal Register Volume 81, Number 190 (Friday, September 30, 2016)] [Notices] [Pages 67360-67362] From the Federal Register Online [www.thefederalregister.org] [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 http://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: http://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
![67360 Federal Register / Vol. 81, No. 190 / Friday, September 30, 2016 / Notices
FDA issued a document entitled donors. These data also will inform FDA the TTIMS program will maintain
‘‘Revised Recommendations for regarding future blood donor deferral standardized, statistically and
Reducing the Risk of Human policy options to reduce the risk of HIV scientifically robust processes for
Immunodeficiency Virus Transmission transmission, including the feasibility of applying hemovigilance information
by Blood and Blood Products, Guidance moving from the existing time-based across blood collection organizations.
for Industry’’ dated December 2015 deferrals related to risk behaviors to The specific objectives are to:
(http://www.fda.gov/downloads/ alternate deferral options, such as the • Determine current behavioral risk
BiologicsBloodVaccines/Guidance use of individual risk assessments, and factors associated with all HIV
ComplianceRegulatoryInformation/ to inform the design of potential studies infections, incident HBV, and incident
Guidances/Blood/UCM446580.pdf) to evaluate the feasibility and HCV infections in blood donors
which changed the blood donor effectiveness of such alternative deferral (including parenteral and sexual risks)
criterion for men who have sex with options. across the participating blood collection
men (MSM) from an indefinite TTIMS will include a comprehensive organizations using a case-control study
(permanent) deferral to a 12-month interview-based epidemiological study design.
deferral since last MSM contact. The of risk factor information for viral • Determine infectious disease
impact of this change in the deferral infection-positive blood donors at the marker prevalence and incidence for
criteria requires a national monitoring American Red Cross (ARC), Blood HIV, HBV, and HCV overall and by
effort as part of TTIMS to assess if the Systems, Inc. (BSI), New York Blood demographic characteristics of donors
relative proportions of risk factors for Center (NYBC), and OneBlood that will in the majority of blood donations
infection in blood donors have changed identify the current predominant risk collected in the country. This will be
following the adoption of the 12-month factors and reasons for virus-positive accomplished by forming
donor deferral for MSM. TTIMS will use donations. The TTIMS program epidemiological databases consisting of
similar procedures as the ones used in establishes a new, ongoing donor harmonized operational data from ARC,
the REDS–II study to monitor and hemovigilance capacity that currently BSI, NYBC, and OneBlood.
evaluate risk factors among HIV-positive does not exist in the United States. • Analyze integrated risk factor and
donors and recently HCV or HBV Using procedures developed by the infectious marker testing data
infected donors as well as controls. REDS–II study, TTIMS will establish concurrently because when taken
This study will help identify the this capacity in greater than 50 percent together these may suggest that blood
specific risk factors for TTI and their of all blood donations collected in the centers are not achieving the same
prevalence in blood donors, and help country. degree of success in educational efforts
inform FDA on the proportion of As part of the TTIMS project, a to prevent donation by donors with risk
incident (new) infections among all HIV comprehensive hemovigilance database behaviors across all demographic
positive blood donors. Donations with will be created that integrates the risk groups.
incident infections have the greatest factor information collected through The respondents will be persons who
potential transmission risk because they donor interviews of blood donor with donated blood in the United States and
could be missed during routine blood the resulting data from disease marker these participants will be defined as
screening. The study will help FDA testing and blood components collected cases and controls. The estimated
evaluate the effectiveness of screening by participating organizations into a number of respondents is based on an
strategies in reducing the risk of HIV research database. Following successful overall expected participation in the
transmission from at-risk donors and to initiation of the risk factor interviews, risk factor survey. We estimate a case to
evaluate if there are unexpected the TTIMS network is poised to be control ratio of 1:2 (200 to 400) with a
consequences associated with the recent expanded to include additional blood 50 percent case enrollment.
change in donor deferral policy such as centers and/or re-focused on other FDA estimates the burden of this
an increase in HIV incidence among safety threats as warranted. In this way, collection of information as follows:
TABLE 1—ESTIMATED ANNUAL REPORTING BURDEN 1
Number of
Number of Total annual Average burden Total
Questionnaire/survey responses per
respondents responses per response hours
respondent
Cases and controls.2 ............................................ 600 1 600 0.75 (45 minutes) .......... 450
1 There are no capital costs or operating and maintenance costs associated with this collection of information.
2 Cases consist of virus-positive donations, and controls represent uninfected donors.
Dated: September 26, 2016. DEPARTMENT OF HEALTH AND ACTION: Notice.
Leslie Kux, HUMAN SERVICES
Associate Commissioner for Policy. SUMMARY: The Food and Drug
[FR Doc. 2016–23622 Filed 9–29–16; 8:45 am] Food and Drug Administration Administration (FDA or the Agency) is
BILLING CODE 4164–01–P
announcing the fee rate for using a rare
pediatric disease priority review
mstockstill on DSK3G9T082PROD with NOTICES
[Docket No. FDA–2016–N–0007]
voucher for fiscal year (FY) 2017. The
Fee for Using a Rare Pediatric Disease Federal Food, Drug, and Cosmetic Act
Priority Review Voucher in Fiscal Year (the FD&C Act), as amended by the Food
2017 and Drug Administration Safety and
Innovation Act (FDASIA), authorizes
AGENCY: Food and Drug Administration, FDA to determine and collect rare
HHS. pediatric disease priority review user
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![67362 Federal Register / Vol. 81, No. 190 / Friday, September 30, 2016 / Notices
consistent with published research on FDA of its intent to use the voucher. Dated: September 26, 2016.
this subject which supports a priority Section 529(c)(4)(B) of the FD&C Act Leslie Kux,
review multiplier in the range of 1.48 to specifies that the application will be Associate Commissioner for Policy.
2.35 (Ref. 1). Using FY 2015 figures, the considered incomplete if the priority [FR Doc. 2016–23624 Filed 9–29–16; 8:45 am]
costs of a priority and standard review review user fee and all other applicable BILLING CODE 4164–01–P
are estimated using the following user fees are not paid in accordance
formula: with FDA payment procedures. In
(25 a × 1.67) + (31 a) = $289,352,000 addition, section 529(c)(4)(C) specifies DEPARTMENT OF HEALTH AND
Where ‘‘a’’ is the cost of a standard that FDA may not grant a waiver, HUMAN SERVICES
review and ‘‘a times 1.67’’ is the cost of exemption, reduction, or refund of any
a priority review. Using this formula, fees due and payable under this section Health Resources and Services
the cost of a standard review for NME of the FD&C Act. Beginning with FDA’s Administration
NDAs and BLAs is calculated to be appropriation for FY 2015, the annual
$3,977,000 (rounded to the nearest State Health Departments Coordinating
appropriation language states Center of the Jurisdictional Approach
thousand dollars) and the cost of a specifically that ‘‘priority review user
priority review for NME NDAs and To Curing Hepatitis C Among HIV/HCV
fees authorized by 21 U.S.C. 360n and Coinfected People of Color
BLAs is 1.67 times that amount, or 360ff (section 529 of the FD&C Act)
$6,642,000 (rounded to the nearest Demonstration Project Supported by
shall be credited to this account, to the Secretary’s Minority AIDS Initiative
thousand dollars). The difference
remain available until expended.’’ (Pub. Fund
between these two cost estimates, or
L. 113–235, Section 5, Division A, Title
$2,665,000, represents the incremental AGENCY: Health Resources and Services
VI).
cost of conducting a priority review Administration (HRSA), Department of
rather than a standard review. The rare pediatric disease priority Health and Human Services.
For the FY 2017 fee, FDA will need review fee established in the new fee ACTION: Notice of a deviation from
to adjust the FY 2015 incremental cost schedule must be paid for any competition requirements to make a
by the average amount by which FDA’s application that is received on or after single-source award related to the
average costs increased in the 3 years October 1, 2016. In order to comply Jurisdictional Approach to Curing
prior to FY 2016, to adjust the FY 2015 with this requirement, the sponsor must Hepatitis C (HCV) among HIV/HCV
amount for cost increases in FY 2016. notify FDA 90 days prior to submission Coinfected People of Color
That adjustment, published in the of the human drug application that is demonstration project.
Federal Register on July 28, 2016 (see the subject of a priority review voucher
81 FR 49674 at 49676), setting the FY of an intent to submit the human drug SUMMARY: HRSA’s HIV/AIDS Bureau
2017 PDUFA fee, is 1.5468 percent for application, including the date on (HAB) awarded a non-competitive
the most recent year, not compounded. which the sponsor intends to submit the single source cooperative agreement to
Increasing the FY 2015 incremental application. National Alliance of State and
priority review cost of $2,665,000 by Territorial AIDS Directors (NASTAD)
1.5468 percent results in an estimated Upon receipt of this notification, FDA
for approximately $977,400 in the
cost of $2,706,000 (rounded to the will issue an invoice to the sponsor who Secretary’s Minority AIDS Initiative
nearest thousand dollars). This is the has incurred a rare pediatric disease Funds (SMAIF) as authorized under the
rare pediatric disease priority review priority review voucher fee. The invoice Consolidated Appropriations Act, 2016
user fee amount for FY 2017 that must will include instructions on how to pay (Pub L. 114–113), Division H, Title II.
be submitted with a priority review the fee via wire transfer or check. Subject to the availability of funds and
voucher for a human drug application in As noted in section II, if a sponsor NASTAD’s satisfactory performance,
FY 2017, in addition to any PDUFA fee uses a rare pediatric disease priority HAB will also issue non-competitive,
that is required for such an application. review voucher for a human drug single-source awards of approximately
III. Fee Schedule for FY 2017 application, the sponsor would incur $750,000 each in fiscal years (FY) 2017
the rare pediatric disease priority review and 2018. This will allow NASTAD to
The fee rate for FY 2017 is set out in facilitate the participation of up to two
table 1: voucher fee in addition to any PDUFA
fee that is required for the application. Ryan White HIV/AIDS Program Part B
The sponsor would need to follow recipients in the Jurisdictional
TABLE 1—RARE PEDIATRIC DISEASE Approach to Curing Hepatitis C among
PRIORITY REVIEW SCHEDULE FOR FDA’s normal procedures for timely
payment of the PDUFA fee for the HIV/HCV Coinfected People of Color
FY 2017 demonstration project over its 3-year
human drug application.
project period.
Fee rate for V. Reference
Fee category FOR FURTHER INFORMATION CONTACT:
FY 2017
Harold J. Phillips, Director, Office of
The following reference is on display
Application submitted with a Training and Capacity Development,
in the Division of Dockets Management
rare pediatric disease pri- HAB/HRSA, 5600 Fishers Lane, Room
ority review voucher in ad- (HFA–305), Food and Drug
9N–114, Rockville, MD 20857, by email
dition to the normal Administration, 5630 Fishers Lane, Rm. at hphillips@hrsa.gov or by phone at
PDUFA fee ........................ $2,706,000 1061, Rockville, MD 20852, and is (301) 443–8109.
mstockstill on DSK3G9T082PROD with NOTICES
available for viewing by interested
SUPPLEMENTARY INFORMATION:
IV. Implementation of Rare Pediatric persons between 9 a.m. and 4 p.m., Recipient of the Award: National
Disease Priority Review User Fee Monday through Friday. Alliance of State and Territorial AIDS
Under section 529(c)(4)(A) of the 1. Ridley, D.B., H.G. Grabowski, and J.L. Moe, Directors.
FD&C Act, the priority review user fee ‘‘Developing Drugs for Developing Period of Supplemental Funding:
is due (i.e. the obligation to pay the fee Countries,’’ Health Affairs, vol. 25, no. 2, September 30, 2016–September 29,
is incurred) when a sponsor notifies pp. 313–324, 2006. 2019.
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Document Created: 2018-02-09 13:34:11
Document Modified: 2018-02-09 13:34:11
| Category | Regulatory Information | |
| Collection | Federal Register | |
| sudoc Class | AE 2.7: GS 4.107: AE 2.106: | |
| Publisher | Office of the Federal Register, National Archives and Records Administration | |
| Section | Notices | |
| Action | Notice. | |
| 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. | |
| FR Citation | 81 FR 67360 |
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