[Federal Register Volume 85, Number 134 (Monday, July 13, 2020)]
[Notices]
[Pages 41993-41995]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2020-14996]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2019-D-0359]


Cancer Clinical Trial Eligibility Criteria: Patients With Organ 
Dysfunction or Prior or Concurrent Malignancies; Guidance for Industry; 
Availability

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice of availability.

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SUMMARY: The Food and Drug Administration (FDA or Agency) is announcing 
the availability of a final guidance for industry entitled ``Cancer 
Clinical Trial Eligibility Criteria: Patients with Organ Dysfunction or 
Prior or Concurrent Malignancies.'' This guidance is one in a series of 
guidances that provide recommendations regarding eligibility criteria 
for clinical trials of drugs or biological products regulated by the 
Center for Drug Evaluation and Research (CDER) and the Center for 
Biologics Evaluation and Research (CBER) for the treatment of cancer. 
Specifically, this guidance includes recommendations on the inclusion 
of patients with organ dysfunction or with prior or concurrent 
malignancies. This guidance finalizes the draft guidance of the same 
title that published on March 13, 2019.

DATES: The announcement of the guidance is published in the Federal 
Register on July 13, 2020.

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ADDRESSES: You may submit either electronic or written comments on 
Agency guidances 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-2019-D-0359 for ``Cancer Clinical Trial Eligibility Criteria: 
Patients with Organ Dysfunction or Prior or Concurrent Malignancies.'' 
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 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 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; or to the Office of 
Communication, Outreach and Development, Center for Biologics 
Evaluation and Research, 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. See the SUPPLEMENTARY INFORMATION section for electronic 
access to the guidance document.

FOR FURTHER INFORMATION CONTACT: Julia Beaver, Center for Drug 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg. 22, Rm. 2100, Silver Spring, MD 20993-0002, 240-
402-0489; 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 guidance for industry 
entitled ``Cancer Clinical Trial Eligibility Criteria: Patients with 
Organ Dysfunction or Prior or Concurrent Malignancies.'' This guidance 
provides recommendations on the inclusion of patients with organ 
dysfunction or prior or concurrent malignancies in clinical trials of 
drugs or biological products regulated by CDER and CBER for the 
treatment of cancer.
    A clinical trial's eligibility criteria (for inclusion and 
exclusion) are essential components of the trial, defining the 
characteristics of the study population. Because there is variability 
in investigational drugs and trial objectives, eligibility criteria 
should be developed taking into consideration the mechanism of action 
of the drug, the targeted disease or patient population, the 
anticipated safety of the investigational drug, the availability of 
adequate safety data, and the ability to recruit trial participants 
from the patient population to meet the objectives of the clinical 
trial. However, some eligibility criteria have become commonly accepted 
over time or used as a template across trials without clear scientific 
or clinical rationale. Unnecessarily restrictive eligibility criteria 
may slow patient accrual, limit patients' access to clinical trials, 
and lead to trial results that do not fully represent treatment effects 
in the patient population that will ultimately use the drug. Broadening 
cancer trial eligibility criteria can maximize the generalizability of 
trial results and the ability to understand the therapy's benefit-risk 
profile across the patient population likely to use the drug in 
clinical practice and should be considered to avoid jeopardizing 
patient safety.
    The recommendations in this guidance for clinical trial eligibility 
criteria for patients with organ dysfunction focus on renal function, 
cardiac function, and hepatic function. This guidance also includes 
recommendations for eligibility criteria for patients with cancer who 
have prior or concurrent malignancies.
    In the Federal Register of March 13, 2019 (84 FR 9129), FDA 
announced the

[[Page 41995]]

availability of the draft guidance of the same title. FDA received 
comments and considered those comments as the guidance was finalized. 
The final guidance clarifies the recommendations regarding eligibility 
criteria related to renal function, cardiac function, and hepatic 
function. For example, recommendations regarding the equation used to 
assess renal function for eligibility were clarified and 
recommendations regarding population pharmacokinetic analyses of 
patients with renal impairment were added. In addition, recommendations 
regarding QTc prolongation were clarified and the recommendation on 
patients with asymptomatic elevations in unconjugated bilirubin was 
removed because it is out of the scope of organ dysfunction.
    This guidance is being issued consistent with FDA's good guidance 
practices regulation (21 CFR 10.115). The guidance represents the 
current thinking of FDA on ``Cancer Clinical Trial Eligibility 
Criteria: Patients with Organ Dysfunction or Prior or Concurrent 
Malignancies.'' 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

    This guidance refers to previously approved FDA collections of 
information. These collections of information are subject to review by 
the Office of Management and Budget (OMB) under the Paperwork Reduction 
Act of 1995 (44 U.S.C. 3501-3521). 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 201.56 and 201.57 have been 
approved under OMB control number 0910-0572.

III. Electronic Access

    Persons with access to the internet may obtain the 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: July 7, 2020.
Lowell J. Schiller,
Principal Associate Commissioner for Policy.
[FR Doc. 2020-14996 Filed 7-10-20; 8:45 am]
BILLING CODE 4164-01-P


