Pathological Complete Response in Neoadjuvant Treatment of High-Risk Early-Stage Breast Cancer: Use as an Endpoint To Support Accelerated Approval; Guidance for Industry; Availability, 60476-60477 [2014-23845]

Download as PDF 60476 Federal Register / Vol. 79, No. 194 / Tuesday, October 7, 2014 / Notices TABLE 1—ESTIMATED ANNUAL REPORTING BURDEN 1 Number of respondents 21 CFR Section/FDA Form Number of responses per respondent Total annual responses Average burden per response Total hours 316.10, 316.12, and 316.14 ................................................. 316.20, 316.21, and 316.26 ................................................. Form FDA 3671 ................................................................... 316.22 .................................................................................. 316.27 .................................................................................. 316.30 .................................................................................. 316.36 .................................................................................. 2 225 50 65 43 450 2 1 2 3 1 1 1 3 2 450 150 65 43 450 6 100 150 45 2 5 3 15 200 67,500 6,750 130 215 1,350 90 Total .............................................................................. ........................ ........................ ........................ ........................ 76,235 1 There are no capital costs or operating and maintenance costs associated with this collection of information. Dated: October 1, 2014. Leslie Kux, Assistant Commissioner for Policy. collection and has assigned OMB control number 0910–0693. The approval expires on August 31, 2017. A copy of the supporting statement for this information collection is available on the Internet at http://www.reginfo.gov/ public/do/PRAMain. [FR Doc. 2014–23846 Filed 10–6–14; 8:45 am] BILLING CODE 4164–01–P DEPARTMENT OF HEALTH AND HUMAN SERVICES Dated: October 1, 2014. Leslie Kux, Assistant Commissioner for Policy. Food and Drug Administration [FR Doc. 2014–23842 Filed 10–6–14; 8:45 am] [Docket No. FDA–2014–N–0222] BILLING CODE 4164–01–P Agency Information Collection Activities; Announcement of Office of Management and Budget Approval; Guidance for Industry on User Fee Waivers, Reductions, and Refunds for Drug and Biological Products AGENCY: Food and Drug Administration, HHS. ACTION: Notice. The Food and Drug Administration (FDA) is announcing that a collection of information entitled ‘‘Guidance for Industry on User Fee Waivers, Reductions, and Refunds for Drug and Biological Products’’ has been approved by the Office of Management and Budget (OMB) under the Paperwork Reduction Act of 1995. FOR FURTHER INFORMATION CONTACT: FDA PRA Staff, Office of Operations, Food and Drug Administration, 8455 Colesville Rd., COLE–14526, Silver Spring, MD 20993–0002, PRAStaff@fda.hhs.gov. SUPPLEMENTARY INFORMATION: On July 16, 2014, the Agency submitted a proposed collection of information entitled ‘‘Guidance for Industry on User Fee Waivers, Reductions, and Refunds for Drug and Biological Products’’ to OMB for review and clearance under 44 U.S.C. 3507. An Agency may not conduct or sponsor, and a person is not required to respond to, a collection of information unless it displays a currently valid OMB control number. OMB has now approved the information asabaliauskas on DSK5VPTVN1PROD with NOTICES SUMMARY: VerDate Sep<11>2014 17:15 Oct 06, 2014 Jkt 235001 DEPARTMENT OF HEALTH AND HUMAN SERVICES Food and Drug Administration [Docket No. FDA–2012–D–0432] Pathological Complete Response in Neoadjuvant Treatment of High-Risk Early-Stage Breast Cancer: Use as an Endpoint To Support Accelerated Approval; Guidance for Industry; Availability AGENCY: Food and Drug Administration, HHS. ACTION: Notice. The Food and Drug Administration (FDA) is announcing the availability of a guidance for industry entitled ‘‘Pathological Complete Response in Neoadjuvant Treatment of High-Risk Early-Stage Breast Cancer: Use as an Endpoint to Support Accelerated Approval.’’ This guidance is intended to assist applicants in designing trials to support marketing approval of drugs to treat breast cancer in the neoadjuvant (preoperative) setting using pathological complete response (pCR) as a surrogate endpoint that could support approval under the accelerated approval regulations. Despite advances in systemic therapy of early-stage breast cancer over the past few decades, there remains a significant unmet medical need for certain high-risk or poor prognosis populations of early-stage SUMMARY: PO 00000 Frm 00033 Fmt 4703 Sfmt 4703 breast cancer patients. This guidance is intended to encourage industry innovation and expedite the development of breakthrough therapies to treat high-risk early-stage breast cancer. This guidance finalizes the draft guidance issued May 30, 2012. DATES: Submit either electronic or written comments on Agency guidances at any time. ADDRESSES: Submit written requests for single copies of this guidance to the Division of Drug Information, Center for Drug Evaluation and Research, Food and Drug Administration, 10001 New Hampshire Ave., Hillandale Building, 4th Floor, Silver Spring, MD 20993– 0002. Send one self-addressed adhesive label to assist that office in processing your requests. See the SUPPLEMENTARY INFORMATION section for electronic access to the guidance document. Submit electronic comments on the guidance to http://www.regulations.gov. Submit written comments to the Division of Dockets Management (HFA– 305), Food and Drug Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852. FOR FURTHER INFORMATION CONTACT: Tatiana Prowell, Center for Drug Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 22, Rm. 2112, Silver Spring, MD 20993–0002, 301– 796–2330. SUPPLEMENTARY INFORMATION: I. Background FDA is announcing the availability of a guidance for industry entitled ‘‘Pathological Complete Response in Neoadjuvant Treatment of High-Risk Early-Stage Breast Cancer: Use as an Endpoint to Support Accelerated Approval.’’ Under the accelerated approval regulations (21 CFR part 314, subpart H, and 21 CFR part 601, subpart E), FDA may grant marketing approval for a new drug on the basis of adequate and well-controlled trials establishing E:\FR\FM\07OCN1.SGM 07OCN1 asabaliauskas on DSK5VPTVN1PROD with NOTICES Federal Register / Vol. 79, No. 194 / Tuesday, October 7, 2014 / Notices that the drug has an effect on a surrogate endpoint that is reasonably likely to predict clinical benefit (e.g., in earlystage breast cancer, an improvement in disease-free or overall survival), provided that the applicant conducts additional trials or collects additional data after approval to verify and describe the predicted clinical benefit. This guidance is intended to assist applicants in designing trials to support marketing approval of drugs to treat breast cancer in the neoadjuvant (preoperative) setting using pCR as a surrogate endpoint that could support approval under the accelerated approval regulations. The guidance provides acceptable definitions of pCR for regulatory purposes. The guidance also describes appropriate patient populations for inclusion in neoadjuvant trials conducted with regulatory intent. Finally, the guidance outlines critical design features of trials for both accelerated approval and confirmation of clinical benefit to support regular approval. FDA recognizes that despite advances in adjuvant systemic therapy of breast cancer over the past few decades, there remains a significant unmet medical need for certain high-risk or poor prognosis populations of early-stage breast cancer patients. Developing highly effective new drugs for these populations is an FDA priority. In providing guidance on the use of pCR as a surrogate endpoint that could support accelerated approval in the neoadjuvant setting, FDA hopes to encourage industry innovation and expedite the development and widespread availability of highly effective novel therapies to treat high-risk early-stage breast cancer. This guidance finalizes the draft guidance issued May 30, 2012 (77 FR 31858). The current version clarifies appropriate trial designs and development strategies to support accelerated approval in the neoadjuvant setting, defines acceptable endpoints for accelerated approval and confirmation of clinical benefit, standardizes the approach to postoperative systemic therapy, includes guidelines for evaluation of the axillary lymph nodes, and provides detailed recommendations for pathology standard operating procedures. This guidance is being issued consistent with FDA’s good guidance practices regulation (21 CFR 10.115). The guidance represents the Agency’s current thinking on use of pCR as an endpoint to support accelerated approval of drug and biological products to treat high-risk early-stage breast cancer patient populations. It VerDate Sep<11>2014 17:15 Oct 06, 2014 Jkt 235001 does not create or confer any rights for or on any person and does not operate to bind FDA or the public. An alternative approach may be used if such approach satisfies the requirements of the applicable statutes and regulations. II. The Paperwork Reduction Act of 1995 This guidance refers to previously approved collections of information that are subject to review by the Office of Management and Budget (OMB) under the Paperwork Reduction Act of 1995 (44 U.S.C. 3501–3520). The collections of information in 21 CFR parts 312 and 314 have been approved under OMB control numbers 0910–0014 and 0910– 0001, respectively. The collections of information for special protocol assessments have been approved under OMB control number 0910–0470. III. Comments Interested persons may submit either electronic comments regarding this document to http://www.regulations.gov or written comments to the Division of Dockets Management (see ADDRESSES). It is only necessary to send one set of comments. Identify comments with the docket number found in brackets in the heading of this document. Received comments may be seen in the Division of Dockets Management between 9 a.m. and 4 p.m., Monday through Friday, and will be posted to the docket at http:// www.regulations.gov. IV. Electronic Access Persons with access to the Internet may obtain the document at either http://www.fda.gov/Drugs/Guidance ComplianceRegulatoryInformation/ Guidances/default.htm or http:// www.regulations.gov. Dated: October 1, 2014. Leslie Kux, Assistant Commissioner for Policy. [FR Doc. 2014–23845 Filed 10–6–14; 8:45 am] BILLING CODE 4164–01–P DEPARTMENT OF HEALTH AND HUMAN SERVICES Food and Drug Administration [Docket No. FDA–2014–N–1208] Laboratory Site Tours Program AGENCY: Food and Drug Administration, HHS. ACTION: Notice. The Food and Drug Administration’s (FDA’s) Center for Tobacco Products’ (CTP) Office of SUMMARY: PO 00000 Frm 00034 Fmt 4703 Sfmt 4703 60477 Science is announcing an invitation for participation in its Laboratory Site Tours Program. This program is intended to give CTP staff an opportunity to visit facilities involved in the testing and analysis of tobacco products and tobacco smoke. These visits are intended to provide CTP staff with the opportunity to gain a better understanding of tobacco science and laboratory operations and are not intended as regulatory inspections or facility visits for the purposes of developing Tobacco Product Manufacturing Practice regulations. The purpose of this notice is to invite parties interested in participating in the Laboratory Site Tours Program to submit their requests to CTP. DATES: Submit either an electronic or written request for participation in this program by December 8, 2014. The request should include a description of your facility, including, as applicable, a list of the types of testing and analyses of tobacco products and tobacco smoke performed. Please specify the physical address(es) of the site(s) for which you are submitting a request, along with a proposed 1-day tour agenda. ADDRESSES: If your facility is interested in offering a site visit, submit either an electronic request to http:// www.regulations.gov or a written request to the Division of Dockets Management (HFA–305), Food and Drug Administration, 5630 Fishers Lane, rm. 1061, Rockville, MD 20852. FOR FURTHER INFORMATION CONTACT: Carolyn Dresler, Center for Tobacco Products, Food and Drug Administration, 10903 New Hampshire Ave., Document Control Center, Bldg. 71, rm. G335, Silver Spring, MD 20993– 0002, 240–402–4067, carolyn.dresler@ fda.hhs.gov. SUPPLEMENTARY INFORMATION: I. Background On June 22, 2009, the Family Smoking Prevention and Tobacco Control Act (Pub. L. 111–31) was signed into law, amending the Federal Food, Drug, and Cosmetic Act (the FD&C Act) and giving FDA authority to regulate tobacco product manufacturing, distribution, and marketing. CTP’s Office of Science is conducting the Laboratory Site Tours Program to provide its scientific and regulatory staff the opportunity to gain a better understanding of tobacco science and laboratory operations, to include tobacco product testing and analysis. CTP’s goal for the Laboratory Site Tours Program is for its staff to gain: (1) Firsthand exposure to laboratories that perform tobacco product testing and (2) E:\FR\FM\07OCN1.SGM 07OCN1

Agencies

[Federal Register Volume 79, Number 194 (Tuesday, October 7, 2014)]
[Notices]
[Pages 60476-60477]
From the Federal Register Online via the Government Printing Office [www.gpo.gov]
[FR Doc No: 2014-23845]


-----------------------------------------------------------------------

DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2012-D-0432]


Pathological Complete Response in Neoadjuvant Treatment of High-
Risk Early-Stage Breast Cancer: Use as an Endpoint To Support 
Accelerated Approval; Guidance for Industry; Availability

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice.

-----------------------------------------------------------------------

SUMMARY: The Food and Drug Administration (FDA) is announcing the 
availability of a guidance for industry entitled ``Pathological 
Complete Response in Neoadjuvant Treatment of High-Risk Early-Stage 
Breast Cancer: Use as an Endpoint to Support Accelerated Approval.'' 
This guidance is intended to assist applicants in designing trials to 
support marketing approval of drugs to treat breast cancer in the 
neoadjuvant (preoperative) setting using pathological complete response 
(pCR) as a surrogate endpoint that could support approval under the 
accelerated approval regulations. Despite advances in systemic therapy 
of early-stage breast cancer over the past few decades, there remains a 
significant unmet medical need for certain high-risk or poor prognosis 
populations of early-stage breast cancer patients. This guidance is 
intended to encourage industry innovation and expedite the development 
of breakthrough therapies to treat high-risk early-stage breast cancer. 
This guidance finalizes the draft guidance issued May 30, 2012.

DATES: Submit either electronic or written comments on Agency guidances 
at any time.

ADDRESSES: Submit written requests for single copies of this guidance 
to the Division of Drug Information, Center for Drug Evaluation and 
Research, Food and Drug Administration, 10001 New Hampshire Ave., 
Hillandale Building, 4th Floor, Silver Spring, MD 20993-0002. Send one 
self-addressed adhesive label to assist that office in processing your 
requests. See the SUPPLEMENTARY INFORMATION section for electronic 
access to the guidance document.
    Submit electronic comments on the guidance to http://www.regulations.gov. Submit written comments to the Division of Dockets 
Management (HFA-305), Food and Drug Administration, 5630 Fishers Lane, 
Rm. 1061, Rockville, MD 20852.

FOR FURTHER INFORMATION CONTACT: Tatiana Prowell, Center for Drug 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg. 22, Rm. 2112, Silver Spring, MD 20993-0002, 301-
796-2330.

SUPPLEMENTARY INFORMATION: 

I. Background

    FDA is announcing the availability of a guidance for industry 
entitled ``Pathological Complete Response in Neoadjuvant Treatment of 
High-Risk Early-Stage Breast Cancer: Use as an Endpoint to Support 
Accelerated Approval.'' Under the accelerated approval regulations (21 
CFR part 314, subpart H, and 21 CFR part 601, subpart E), FDA may grant 
marketing approval for a new drug on the basis of adequate and well-
controlled trials establishing

[[Page 60477]]

that the drug has an effect on a surrogate endpoint that is reasonably 
likely to predict clinical benefit (e.g., in early-stage breast cancer, 
an improvement in disease-free or overall survival), provided that the 
applicant conducts additional trials or collects additional data after 
approval to verify and describe the predicted clinical benefit. This 
guidance is intended to assist applicants in designing trials to 
support marketing approval of drugs to treat breast cancer in the 
neoadjuvant (preoperative) setting using pCR as a surrogate endpoint 
that could support approval under the accelerated approval regulations. 
The guidance provides acceptable definitions of pCR for regulatory 
purposes. The guidance also describes appropriate patient populations 
for inclusion in neoadjuvant trials conducted with regulatory intent. 
Finally, the guidance outlines critical design features of trials for 
both accelerated approval and confirmation of clinical benefit to 
support regular approval.
    FDA recognizes that despite advances in adjuvant systemic therapy 
of breast cancer over the past few decades, there remains a significant 
unmet medical need for certain high-risk or poor prognosis populations 
of early-stage breast cancer patients. Developing highly effective new 
drugs for these populations is an FDA priority. In providing guidance 
on the use of pCR as a surrogate endpoint that could support 
accelerated approval in the neoadjuvant setting, FDA hopes to encourage 
industry innovation and expedite the development and widespread 
availability of highly effective novel therapies to treat high-risk 
early-stage breast cancer.
    This guidance finalizes the draft guidance issued May 30, 2012 (77 
FR 31858). The current version clarifies appropriate trial designs and 
development strategies to support accelerated approval in the 
neoadjuvant setting, defines acceptable endpoints for accelerated 
approval and confirmation of clinical benefit, standardizes the 
approach to postoperative systemic therapy, includes guidelines for 
evaluation of the axillary lymph nodes, and provides detailed 
recommendations for pathology standard operating procedures.
    This guidance is being issued consistent with FDA's good guidance 
practices regulation (21 CFR 10.115). The guidance represents the 
Agency's current thinking on use of pCR as an endpoint to support 
accelerated approval of drug and biological products to treat high-risk 
early-stage breast cancer patient populations. It does not create or 
confer any rights for or on any person and does not operate to bind FDA 
or the public. An alternative approach may be used if such approach 
satisfies the requirements of the applicable statutes and regulations.

II. The Paperwork Reduction Act of 1995

    This guidance refers to previously approved collections of 
information that are subject to review by the Office of Management and 
Budget (OMB) under the Paperwork Reduction Act of 1995 (44 U.S.C. 3501-
3520). The collections of information in 21 CFR parts 312 and 314 have 
been approved under OMB control numbers 0910-0014 and 0910-0001, 
respectively. The collections of information for special protocol 
assessments have been approved under OMB control number 0910-0470.

III. Comments

    Interested persons may submit either electronic comments regarding 
this document to http://www.regulations.gov or written comments to the 
Division of Dockets Management (see ADDRESSES). It is only necessary to 
send one set of comments. Identify comments with the docket number 
found in brackets in the heading of this document. Received comments 
may be seen in the Division of Dockets Management between 9 a.m. and 4 
p.m., Monday through Friday, and will be posted to the docket at http://www.regulations.gov.

IV. Electronic Access

    Persons with access to the Internet may obtain the document at 
either http://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/default.htm or http://www.regulations.gov.

    Dated: October 1, 2014.
Leslie Kux,
Assistant Commissioner for Policy.
[FR Doc. 2014-23845 Filed 10-6-14; 8:45 am]
BILLING CODE 4164-01-P