83 FR 63166 - Developing and Labeling In vitro

DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration

Federal Register Volume 83, Issue 235 (December 7, 2018)

The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled ``Developing and Labeling In vitro Companion Diagnostic Devices for a Specific Group or Class of Oncology Therapeutic Products.'' This draft guidance describes considerations for the development and labeling of in vitro companion diagnostic devices (referred to as companion diagnostics in this document) to support the indicated uses of multiple drug or biologic oncology products (referred to as therapeutic products or oncology therapeutic products in this document), when appropriate. The draft guidance includes factors for considering when broader labeling (i.e., labeling that is expanded) of a companion diagnostic would be appropriate. Oncology companion diagnostics with broader evidence-based indications will optimally facilitate clinical use.

[Federal Register Volume 83, Number 235 (Friday, December 7, 2018)]
[Notices]
[Pages 63166-63167]
From the Federal Register Online  [www.thefederalregister.org]
[FR Doc No: 2018-26554]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2018-D-3380]


Developing and Labeling In vitro Companion Diagnostic Devices for 
a Specific Group or Class of Oncology Therapeutic Products; Draft 
Guidance for Industry; Availability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the availability of a draft guidance for industry entitled ``Developing 
and Labeling In vitro Companion Diagnostic Devices for a Specific Group 
or Class of Oncology Therapeutic Products.'' This draft guidance 
describes considerations for the development and labeling of in vitro 
companion diagnostic devices (referred to as companion diagnostics in 
this document) to support the indicated uses of multiple drug or 
biologic oncology products (referred to as therapeutic products or 
oncology therapeutic products in this document), when appropriate. The 
draft guidance includes factors for considering when broader labeling 
(i.e., labeling that is expanded) of a companion diagnostic would be 
appropriate. Oncology companion diagnostics with broader evidence-based 
indications will optimally facilitate clinical use.

DATES: Submit either electronic or written comments on the draft 
guidance by February 5, 2019 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-2018-D-3380 for ``Developing and Labeling In vitro Companion 
Diagnostic Devices for a Specific Group or Class of Oncology 
Therapeutic Products.'' 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.
     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.thefederalregister.org/fdsys/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.
    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 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; 
the Office of the Center Director, Guidance and Policy Development, 
Center for Devices and Radiological Health, Food and Drug 
Administration, 10903 New Hampshire Ave., Bldg. 66, Rm. 5431, Silver 
Spring, MD 20993-0002; or 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. The draft guidance may also be 
obtained by mail by calling FDA's Center for Biologics Evaluation and 
Research (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: 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; Reena Philip, Center for

[[Page 63167]]

Devices and Radiological Health, Food and Drug Administration, 10903 
New Hampshire Ave., Bldg. 66, Rm. 5680, Silver Spring, MD 20993-0002, 
301-796-6179; or Julie Schneider, Center for Drug Evaluation and 
Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 
22, Rm. 2208, Silver Spring, MD 20993-0002, 240-402-4658.

SUPPLEMENTARY INFORMATION: 

I. Background

    FDA is announcing the availability of a draft guidance for industry 
entitled ``Developing and Labeling In vitro Companion Diagnostic 
Devices for a Specific Group or Class of Oncology Therapeutic 
Products.'' This draft guidance describes considerations for the 
development and labeling of companion diagnostics to support the 
indicated uses of multiple therapeutic oncology products, when 
appropriate. This draft guidance expands on existing policy, 
surrounding broader labeling, which notes that in some cases, if 
evidence is sufficient to conclude that the companion diagnostic is 
appropriate for use with a specific group or class of therapeutic 
products (as discussed in the draft guidance), the companion 
diagnostic's intended use/indications for use should name the specific 
group or class of therapeutic products, rather than specific products. 
To describe FDA's thinking on the topic, the draft guidance discusses a 
specific example of companion diagnostics for a specific biomarker, 
disease, and specimen type (specific epidermal growth factor receptor 
mutations in tumors of patients with nonsmall cell lung cancer in 
tissue specimens).
    Trials designed to support approval of a specific therapeutic 
product and a specific companion diagnostic have led to companion 
diagnostic labels that reference only a specific therapeutic 
product(s). Such specificity in labeling can limit a potentially 
broader use of a companion diagnostic that may be scientifically 
appropriate. In clinical practice, an oncologist generally considers 
the mutation profile of the tumor along with other factors when 
determining the treatment for a patient, such as the toxicity profile 
of the therapeutic product, the patient's preference, and formulary 
options. When a companion diagnostic is labeled for use with a specific 
therapeutic product, the clinician may need to order a different 
companion diagnostic (i.e., one that includes other therapeutic 
products in the labeling), obtain an additional biopsy(ies) from a 
patient, or both, to have additional therapy treatment options.
    The draft guidance describes considerations for when broader 
labeling may be scientifically appropriate and when it may not. FDA 
recommends developers of therapeutic oncology products and associated 
companion diagnostics collaboratively consider development programs 
that may result in broader labeling of companion diagnostics that are 
most clinically useful. Developers are encouraged to discuss 
development programs that could result in broader labeling with the 
CBER, Center for Devices and Radiological Health (CDRH), or Center for 
Drug Evaluation and Research, in coordination with the Oncology Center 
of Excellence, as appropriate, early to determine if the approach 
described in this guidance is appropriate for consideration. Developers 
whose approved companion diagnostics may be appropriate for broader 
labeling are encouraged to contact CDRH or CBER, as appropriate, to 
discuss.
    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 ``Developing 
and Labeling In vitro Companion Diagnostic Devices for a Specific Group 
or Class of Oncology Therapeutic Products.'' 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. This guidance is not subject to 
Executive Order 12866.

II. Other Issues for Consideration

    In addition to providing stakeholders an opportunity to comment on 
the draft guidance, the Agency is interested in responses from 
stakeholders to the following:
    1. Please describe any specific challenges with developing the 
evidence needed to identify in labeling a companion diagnostic for use 
with a specific group or class of oncology therapeutic products, rather 
than a specific therapeutic product. For example, please describe any 
challenges resulting from industry or business practices, including 
business agreements. What actions can FDA take to address the 
challenge(s)?
    2. Please describe any specific challenges with submitting a 
premarket approval (PMA) supplement to FDA to expand the labeling for 
an approved companion diagnostic for use with a specific group or class 
of oncology therapeutic products. What actions can FDA take to address 
the challenge(s)?
    3. Please describe any additional actions FDA can take to 
facilitate or encourage broader, evidence-based labeling that supports 
the use of a specific group or class of oncology therapeutic products 
with a companion diagnostic.
    4. The guidance notes that variations in defined cut-points 
established for specific biomarkers for companion diagnostics can lead 
to challenges in implementing broader labeling for a specific group or 
class of oncology therapeutic products. Are there actions that FDA, or 
the broader scientific community, can take to facilitate 
standardization in this area?

III. Paperwork Reduction Act of 1995

    This draft guidance refers to previously approved collections of 
information found in FDA regulations and guidance. 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-
3520). The collections of information in 21 CFR parts 801 and 809 have 
been approved under OMB control number 0910-0485; the collections of 
information in 21 CFR part 807, subpart E, have been approved under OMB 
control number 0910-0120; the collections of information in 21 CFR part 
814, subparts A through E, have been approved under OMB control number 
0910-0231; the collections of information in 21 CFR part 814, subpart 
H, have been approved under OMB control number 0910-0332; the 
collections of information in the guidance document ``Requests for 
Feedback on Medical Device Submissions: The Pre-Submission Program and 
Meetings with Food and Drug Administration Staff'' have been approved 
under OMB control number 0910-0756; and the collections of information 
in the guidance ``De Novo Classification Process (Evaluation of 
Automatic Class III Designation)'' have been approved under OMB control 
number 0910-0844.

IV. Electronic Access

    Persons with access to the internet may obtain the draft guidance 
at either https://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/default.htm or 
https://www.regulations.gov.

    Dated: December 3, 2018.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2018-26554 Filed 12-6-18; 8:45 am]
 BILLING CODE 4164-01-P