Core Patient-Reported Outcomes in Cancer Clinical Trials; Draft Guidance for Industry; Availability, 30944-30945 [2021-12166]

Download as PDF 30944 Federal Register / Vol. 86, No. 110 / Thursday, June 10, 2021 / Notices as the date the investigational new drug application (IND) became effective. However, FDA records indicate that the IND effective date was November 26, 2006, which was 30 days after FDA receipt of the IND. 2. The date the application was initially submitted with respect to the human biological product under section 351 of the Public Health Service Act (42 U.S.C. 262): July 19, 2016. FDA has verified the applicant’s claim that the biologics license application (BLA) for EVENITY (BLA 761062) was initially submitted on July 19, 2016. 3. The date the application was approved: April 9, 2019. FDA has verified the applicant’s claim that BLA 761062 was approved on April 9, 2019. This determination of the regulatory review period establishes the maximum potential length of a patent extension. However, the USPTO applies several statutory limitations in its calculations of the actual period for patent extension. In its applications for patent extension, this applicant seeks 1,550 days, 1,575 days or 1,827 days of patent term extension. khammond on DSKJM1Z7X2PROD with NOTICES III. Petitions Anyone with knowledge that any of the dates as published are incorrect may submit either electronic or written comments and, under 21 CFR 60.24, ask for a redetermination (see DATES). Furthermore, as specified in § 60.30 (21 CFR 60.30), any interested person may petition FDA for a determination regarding whether the applicant for extension acted with due diligence during the regulatory review period. To meet its burden, the petition must comply with all the requirements of § 60.30, including but not limited to: Must be timely (see DATES), must be filed in accordance with § 10.20, must contain sufficient facts to merit an FDA investigation, and must certify that a true and complete copy of the petition has been served upon the patent applicant. (See H. Rept. 857, part 1, 98th Cong., 2d sess., pp. 41–42, 1984.) Petitions should be in the format specified in 21 CFR 10.30. Submit petitions electronically to https://www.regulations.gov at Docket No. FDA–2013–S–0610. Submit written petitions (two copies are required) to the Dockets Management Staff (HFA–305), Food and Drug Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852. VerDate Sep<11>2014 17:15 Jun 09, 2021 Jkt 253001 Dated: June 3, 2021. Lauren K. Roth, Acting Principal Associate Commissioner for Policy. [FR Doc. 2021–12199 Filed 6–9–21; 8:45 am] BILLING CODE 4164–01–P DEPARTMENT OF HEALTH AND HUMAN SERVICES Food and Drug Administration [Docket No. FDA–2020–D–2303] Core Patient-Reported Outcomes in Cancer Clinical Trials; Draft Guidance for Industry; Availability AGENCY: Food and Drug Administration, HHS. ACTION: Notice of availability. The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled ‘‘Core Patient-Reported Outcomes in Cancer Clinical Trials.’’ This draft guidance provides recommendations to sponsors regarding the collection of a core set of patient-reported clinical outcomes in cancer clinical trials and related considerations for instrument selection and trial design. This guidance focuses on patient-reported outcome (PRO) measures and is specific to registration trials for anti-cancer therapies intended to demonstrate an effect on survival, tumor response, or delay in the progression of a malignancy. The draft guidance recommendations supplement previous guidance on use of PRO measures in clinical trials by providing additional considerations specific to the cancer clinical trial setting. The draft guidance is intended to facilitate generation of high-quality data on a core set of patient-reported symptom and functional impacts that are important contributors to a patient’s health-related quality of life. DATES: Submit either electronic or written comments on the draft guidance by August 9, 2021 to ensure that the Agency considers your comment on this draft guidance before it begins work on the final version of the guidance. ADDRESSES: You may submit comments on any guidance at any time as follows: SUMMARY: Electronic Submissions Submit electronic comments in the following way: • Federal eRulemaking Portal: https://www.regulations.gov. Follow the instructions for submitting comments. Comments submitted electronically, including attachments, to https:// www.regulations.gov will be posted to PO 00000 Frm 00035 Fmt 4703 Sfmt 4703 the docket unchanged. Because your comment will be made public, you are solely responsible for ensuring that your comment does not include any confidential information that you or a third party may not wish to be posted, such as medical information, your or anyone else’s Social Security number, or confidential business information, such as a manufacturing process. Please note that if you include your name, contact information, or other information that identifies you in the body of your comments, that information will be posted on https://www.regulations.gov. • If you want to submit a comment with confidential information that you do not wish to be made available to the public, submit the comment as a written/paper submission and in the manner detailed (see ‘‘Written/Paper Submissions’’ and ‘‘Instructions’’). Written/Paper Submissions Submit written/paper submissions as follows: • Mail/Hand Delivery/Courier (for written/paper submissions): Dockets Management Staff (HFA–305), Food and Drug Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852. • For written/paper comments submitted to the Dockets Management Staff, FDA will post your comment, as well as any attachments, except for information submitted, marked and identified, as confidential, if submitted as detailed in ‘‘Instructions.’’ Instructions: All submissions received must include the Docket No. FDA– 2020–D–2303 for ‘‘Core PatientReported Outcomes in Cancer Clinical Trials.’’ Received comments will be placed in the docket and, except for those submitted as ‘‘Confidential Submissions,’’ publicly viewable at https://www.regulations.gov or at the Dockets Management Staff between 9 a.m. and 4 p.m., Monday through Friday, 240–402–7500. • Confidential Submissions—To submit a comment with confidential information that you do not wish to be made publicly available, submit your comments only as a written/paper submission. You should submit two copies total. One copy will include the information you claim to be confidential with a heading or cover note that states ‘‘THIS DOCUMENT CONTAINS CONFIDENTIAL INFORMATION.’’ The Agency will review this copy, including the claimed confidential information, in its consideration of comments. The second copy, which will have the claimed confidential information redacted/blacked out, will be available for public viewing and posted on https://www.regulations.gov. Submit E:\FR\FM\10JNN1.SGM 10JNN1 khammond on DSKJM1Z7X2PROD with NOTICES Federal Register / Vol. 86, No. 110 / Thursday, June 10, 2021 / Notices both copies to the Dockets Management Staff. If you do not wish your name and contact information to be made publicly available, you can provide this information on the cover sheet and not in the body of your comments and you must identify this information as ‘‘confidential.’’ Any information marked as ‘‘confidential’’ will not be disclosed except in accordance with 21 CFR 10.20 and other applicable disclosure law. For more information about FDA’s posting of comments to public dockets, see 80 FR 56469, September 18, 2015, or access the information at: https:// www.govinfo.gov/content/pkg/FR-201509-18/pdf/2015-23389.pdf. Docket: For access to the docket to read background documents or the electronic and written/paper comments received, go to https:// www.regulations.gov and insert the docket number, found in brackets in the heading of this document, into the ‘‘Search’’ box and follow the prompts and/or go to the Dockets Management Staff, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852, 240–402–7500. You may submit comments on any guidance at any time (see 21 CFR 10.115(g)(5)). Submit written requests for single copies of the draft 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, or to the Office of Communication, Outreach and Development, Center for Biologics Evaluation and Research (CBER), Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 71, Rm. 3128, Silver Spring, MD 20993–0002. Send one self-addressed adhesive label to assist that office in processing your requests. The draft guidance may also be obtained by mail by calling CBER at 1– 800–835–4709 or 240–402–8010. See the SUPPLEMENTARY INFORMATION section for electronic access to the draft guidance document. FOR FURTHER INFORMATION CONTACT: Vishal Bhatnagar, Oncology Center of Excellence, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 22, Rm. 2113, Silver Spring, MD 20993–0002, 240–402–3696; or Janice Kim, Center for Drug Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 22, Rm. 2329, Silver Spring, MD 20993–0002, 301–796–9628; or Stephen Ripley, Center for Biologics Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 71, Rm. 7301, VerDate Sep<11>2014 17:15 Jun 09, 2021 Jkt 253001 Silver Spring, MD 20993–0002, 240– 402–7911. SUPPLEMENTARY INFORMATION: I. Background FDA is announcing the availability of a draft guidance for industry entitled ‘‘Core Patient-Reported Outcomes in Cancer Clinical Trials.’’ This draft guidance provides recommendations to sponsors regarding the collection of a core set of PROs in cancer clinical trials and related considerations for instrument selection and trial design. Although this draft guidance focuses on PRO measures, some of the recommendations may be relevant to other clinical outcome assessments (i.e., clinician-reported outcome, observerreported outcome, performance outcome) in cancer clinical trials. The draft guidance is specific to registration trials for anti-cancer therapies intended to demonstrate an effect on survival, tumor response, or delay in the progression of a malignancy. Cancer clinical trials typically employ standardized efficacy assessment using overall survival and tumor measures, and safety assessments provided by clinician reporting of adverse events. FDA acknowledges the potential added value of incorporating PRO measurement of symptoms and functional impacts into the benefit/risk assessment in appropriately designed trials; however, heterogeneity in PRO assessment strategies has lessened the regulatory utility of PRO data from cancer trials. Systematic assessment of a core set of PROs can facilitate high quality data on patient-reported symptoms and functional impacts. FDA has previously described a core set of PROs that may be important contributors to a patient’s health-related quality of life and that may be sensitive to the effect of the disease and treatment under study.1 FDA is issuing this draft guidance to provide FDA’s current thinking on the core PROs, considerations for instrument selection to measure the core PROs, trial design considerations such as assessment frequency, and labeling considerations. The core PROs recommended in the draft guidance are disease-related symptoms, symptomatic adverse events, overall side effect impact summary measure, physical function, and role function. This draft guidance is being issued consistent with FDA’s good guidance 1 Kluetz, P.G., A. Slagle, E.J. Papadopoulos, et al., 2016, ‘‘Focusing on Core Patient-Reported Outcomes in Cancer Clinical Trials: Symptomatic Adverse Events, Physical Function, and DiseaseRelated Symptoms,’’ Clinical Cancer Research, 22(7):1553–1558. PO 00000 Frm 00036 Fmt 4703 Sfmt 4703 30945 practices regulation (21 CFR 10.115). The draft guidance, when finalized, will represent the current thinking of FDA on ‘‘Core Patient-Reported Outcomes in Cancer Clinical Trials.’’ It does not establish any rights for any person and is not binding on FDA or the public. You can use an alternative approach if it satisfies the requirements of the applicable statutes and regulations. II. Paperwork Reduction Act of 1995 While this guidance contains no collection of information, it does refer to previously approved FDA collections of information. Therefore, clearance by the Office of Management and Budget (OMB) under the Paperwork Reduction Act of 1995 (PRA) (44 U.S.C. 3501– 3521) is not required for this guidance. The previously approved collections of information are subject to review by OMB under the PRA. The collections of information in 21 CFR part 312 have been approved under OMB control number 0910–0014; the collections of information in 21 CFR part 314 have been approved under OMB control number 0910–0001; and the collections of information in 21 CFR part 601 have been approved under 0910–0338. III. Electronic Access Persons with access to the internet may obtain the draft guidance at either https://www.fda.gov/drugs/guidancecompliance-regulatory-information/ guidances-drugs, https://www.fda.gov/ vaccines-blood-biologics/guidancecompliance-regulatory-informationbiologics/biologics-guidances, or https:// www.regulations.gov. Dated: June 4, 2021. Lauren K. Roth, Acting Principal Associate Commissioner for Policy. [FR Doc. 2021–12166 Filed 6–9–21; 8:45 am] BILLING CODE 4164–01–P DEPARTMENT OF HEALTH AND HUMAN SERVICES Food and Drug Administration [Docket No. FDA–2019–E–5832] Determination of Regulatory Review Period for Purposes of Patent Extension; XPOVIO AGENCY: Food and Drug Administration, HHS. ACTION: Notice. The Food and Drug Administration (FDA or the Agency) has determined the regulatory review period for XPOVIO and is publishing this notice of that determination as required SUMMARY: E:\FR\FM\10JNN1.SGM 10JNN1

Agencies

[Federal Register Volume 86, Number 110 (Thursday, June 10, 2021)]
[Notices]
[Pages 30944-30945]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2021-12166]


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

DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2020-D-2303]


Core Patient-Reported Outcomes in Cancer Clinical Trials; Draft 
Guidance for Industry; Availability

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice of availability.

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

SUMMARY: The Food and Drug Administration (FDA or Agency) is announcing 
the availability of a draft guidance for industry entitled ``Core 
Patient-Reported Outcomes in Cancer Clinical Trials.'' This draft 
guidance provides recommendations to sponsors regarding the collection 
of a core set of patient-reported clinical outcomes in cancer clinical 
trials and related considerations for instrument selection and trial 
design. This guidance focuses on patient-reported outcome (PRO) 
measures and is specific to registration trials for anti-cancer 
therapies intended to demonstrate an effect on survival, tumor 
response, or delay in the progression of a malignancy. The draft 
guidance recommendations supplement previous guidance on use of PRO 
measures in clinical trials by providing additional considerations 
specific to the cancer clinical trial setting. The draft guidance is 
intended to facilitate generation of high-quality data on a core set of 
patient-reported symptom and functional impacts that are important 
contributors to a patient's health-related quality of life.

DATES: Submit either electronic or written comments on the draft 
guidance by August 9, 2021 to ensure that the Agency considers your 
comment on this draft guidance before it begins work on the final 
version of the guidance.

ADDRESSES: You may submit comments on any guidance at any time as 
follows:

Electronic Submissions

    Submit electronic comments in the following way:
     Federal eRulemaking Portal: https://www.regulations.gov. 
Follow the instructions for submitting comments. Comments submitted 
electronically, including attachments, to https://www.regulations.gov 
will be posted to the docket unchanged. Because your comment will be 
made public, you are solely responsible for ensuring that your comment 
does not include any confidential information that you or a third party 
may not wish to be posted, such as medical information, your or anyone 
else's Social Security number, or confidential business information, 
such as a manufacturing process. Please note that if you include your 
name, contact information, or other information that identifies you in 
the body of your comments, that information will be posted on https://www.regulations.gov.
     If you want to submit a comment with confidential 
information that you do not wish to be made available to the public, 
submit the comment as a written/paper submission and in the manner 
detailed (see ``Written/Paper Submissions'' and ``Instructions'').

Written/Paper Submissions

    Submit written/paper submissions as follows:
     Mail/Hand Delivery/Courier (for written/paper 
submissions): Dockets Management Staff (HFA-305), Food and Drug 
Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852.
     For written/paper comments submitted to the Dockets 
Management Staff, FDA will post your comment, as well as any 
attachments, except for information submitted, marked and identified, 
as confidential, if submitted as detailed in ``Instructions.''
    Instructions: All submissions received must include the Docket No. 
FDA-2020-D-2303 for ``Core Patient-Reported Outcomes in Cancer Clinical 
Trials.'' Received comments will be placed in the docket and, except 
for those submitted as ``Confidential Submissions,'' publicly viewable 
at https://www.regulations.gov or at the Dockets Management Staff 
between 9 a.m. and 4 p.m., Monday through Friday, 240-402-7500.
     Confidential Submissions--To submit a comment with 
confidential information that you do not wish to be made publicly 
available, submit your comments only as a written/paper submission. You 
should submit two copies total. One copy will include the information 
you claim to be confidential with a heading or cover note that states 
``THIS DOCUMENT CONTAINS CONFIDENTIAL INFORMATION.'' The Agency will 
review this copy, including the claimed confidential information, in 
its consideration of comments. The second copy, which will have the 
claimed confidential information redacted/blacked out, will be 
available for public viewing and posted on https://www.regulations.gov. 
Submit

[[Page 30945]]

both copies to the Dockets Management Staff. If you do not wish your 
name and contact information to be made publicly available, you can 
provide this information on the cover sheet and not in the body of your 
comments and you must identify this information as ``confidential.'' 
Any information marked as ``confidential'' will not be disclosed except 
in accordance with 21 CFR 10.20 and other applicable disclosure law. 
For more information about FDA's posting of comments to public dockets, 
see 80 FR 56469, September 18, 2015, or access the information at: 
https://www.govinfo.gov/content/pkg/FR-2015-09-18/pdf/2015-23389.pdf.
    Docket: For access to the docket to read background documents or 
the electronic and written/paper comments received, go to https://www.regulations.gov and insert the docket number, found in brackets in 
the heading of this document, into the ``Search'' box and follow the 
prompts and/or go to the Dockets Management Staff, 5630 Fishers Lane, 
Rm. 1061, Rockville, MD 20852, 240-402-7500.
    You may submit comments on any guidance at any time (see 21 CFR 
10.115(g)(5)).
    Submit written requests for single copies of the draft 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, or to the 
Office of Communication, Outreach and Development, Center for Biologics 
Evaluation and Research (CBER), Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg. 71, Rm. 3128, Silver Spring, MD 20993-0002. Send 
one self-addressed adhesive label to assist that office in processing 
your requests. The draft guidance may also be obtained by mail by 
calling CBER at 1-800-835-4709 or 240-402-8010. See the SUPPLEMENTARY 
INFORMATION section for electronic access to the draft guidance 
document.

FOR FURTHER INFORMATION CONTACT: Vishal Bhatnagar, Oncology Center of 
Excellence, Food and Drug Administration, 10903 New Hampshire Ave., 
Bldg. 22, Rm. 2113, Silver Spring, MD 20993-0002, 240-402-3696; or 
Janice Kim, Center for Drug Evaluation and Research, Food and Drug 
Administration, 10903 New Hampshire Ave., Bldg. 22, Rm. 2329, Silver 
Spring, MD 20993-0002, 301-796-9628; or Stephen Ripley, Center for 
Biologics Evaluation and Research, Food and Drug Administration, 10903 
New Hampshire Ave., Bldg. 71, Rm. 7301, Silver Spring, MD 20993-0002, 
240-402-7911.

SUPPLEMENTARY INFORMATION:

I. Background

    FDA is announcing the availability of a draft guidance for industry 
entitled ``Core Patient-Reported Outcomes in Cancer Clinical Trials.'' 
This draft guidance provides recommendations to sponsors regarding the 
collection of a core set of PROs in cancer clinical trials and related 
considerations for instrument selection and trial design. Although this 
draft guidance focuses on PRO measures, some of the recommendations may 
be relevant to other clinical outcome assessments (i.e., clinician-
reported outcome, observer-reported outcome, performance outcome) in 
cancer clinical trials. The draft guidance is specific to registration 
trials for anti-cancer therapies intended to demonstrate an effect on 
survival, tumor response, or delay in the progression of a malignancy.
    Cancer clinical trials typically employ standardized efficacy 
assessment using overall survival and tumor measures, and safety 
assessments provided by clinician reporting of adverse events. FDA 
acknowledges the potential added value of incorporating PRO measurement 
of symptoms and functional impacts into the benefit/risk assessment in 
appropriately designed trials; however, heterogeneity in PRO assessment 
strategies has lessened the regulatory utility of PRO data from cancer 
trials. Systematic assessment of a core set of PROs can facilitate high 
quality data on patient-reported symptoms and functional impacts. FDA 
has previously described a core set of PROs that may be important 
contributors to a patient's health-related quality of life and that may 
be sensitive to the effect of the disease and treatment under study.\1\
---------------------------------------------------------------------------

    \1\ Kluetz, P.G., A. Slagle, E.J. Papadopoulos, et al., 2016, 
``Focusing on Core Patient-Reported Outcomes in Cancer Clinical 
Trials: Symptomatic Adverse Events, Physical Function, and Disease-
Related Symptoms,'' Clinical Cancer Research, 22(7):1553-1558.
---------------------------------------------------------------------------

    FDA is issuing this draft guidance to provide FDA's current 
thinking on the core PROs, considerations for instrument selection to 
measure the core PROs, trial design considerations such as assessment 
frequency, and labeling considerations. The core PROs recommended in 
the draft guidance are disease-related symptoms, symptomatic adverse 
events, overall side effect impact summary measure, physical function, 
and role function.
    This draft guidance is being issued consistent with FDA's good 
guidance practices regulation (21 CFR 10.115). The draft guidance, when 
finalized, will represent the current thinking of FDA on ``Core 
Patient-Reported Outcomes in Cancer Clinical Trials.'' It does not 
establish any rights for any person and is not binding on FDA or the 
public. You can use an alternative approach if it satisfies the 
requirements of the applicable statutes and regulations.

II. Paperwork Reduction Act of 1995

    While this guidance contains no collection of information, it does 
refer to previously approved FDA collections of information. Therefore, 
clearance by the Office of Management and Budget (OMB) under the 
Paperwork Reduction Act of 1995 (PRA) (44 U.S.C. 3501-3521) is not 
required for this guidance. The previously approved collections of 
information are subject to review by OMB under the PRA. The collections 
of information in 21 CFR part 312 have been approved under OMB control 
number 0910-0014; the collections of information in 21 CFR part 314 
have been approved under OMB control number 0910-0001; and the 
collections of information in 21 CFR part 601 have been approved under 
0910-0338.

III. Electronic Access

    Persons with access to the internet may obtain the draft guidance 
at either https://www.fda.gov/drugs/guidance-compliance-regulatory-information/guidances-drugs, https://www.fda.gov/vaccines-blood-biologics/guidance-compliance-regulatory-information-biologics/biologics-guidances, or https://www.regulations.gov.

    Dated: June 4, 2021.
Lauren K. Roth,
Acting Principal Associate Commissioner for Policy.
[FR Doc. 2021-12166 Filed 6-9-21; 8:45 am]
BILLING CODE 4164-01-P
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