80 FR 8089 - Identifying Potential Biomarkers for Qualification and Describing Contexts of Use To Address Areas Important to Drug Development; Request for Comments

DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration

Federal Register Volume 80, Issue 30 (February 13, 2015)

The Food and Drug Administration (FDA or Agency) is seeking information to facilitate development and qualification of biomarkers in areas related to human drug therapeutics. Towards this goal, FDA is encouraging interested groups and individuals to submit information on specific medical and biological areas where novel biomarkers can be identified that would meaningfully advance drug development. FDA encourages respondents to describe evidentiary considerations that are important to qualify these biomarkers for a specific context of use. Details of information that should be provided to the Agency are described in the survey.

[Federal Register Volume 80, Number 30 (Friday, February 13, 2015)]
[Notices]
[Pages 8089-8091]
From the Federal Register Online  [www.thefederalregister.org]
[FR Doc No: 2015-02976]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2014-N-2187]


Identifying Potential Biomarkers for Qualification and Describing 
Contexts of Use To Address Areas Important to Drug Development; Request 
for Comments

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice; request for comments.

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SUMMARY: The Food and Drug Administration (FDA or Agency) is seeking 
information to facilitate development and qualification of biomarkers 
in areas related to human drug therapeutics. Towards this goal, FDA is 
encouraging interested groups and individuals to submit information on 
specific medical and biological areas where novel biomarkers can be 
identified that would meaningfully advance drug development. FDA 
encourages respondents to describe evidentiary considerations that are 
important to qualify these biomarkers for a specific context of use. 
Details of information that should be provided to the Agency are 
described in the survey.

DATES: Submit either electronic or written comments by April 14, 2015.

ADDRESSES: You may submit comments by any of the following methods:

Electronic Submissions

    Submit electronic comments in either of the following ways:
     Federal eRulemaking Portal: http://www.regulations.gov. 
Follow the instructions for submitting comments.
     SurveyMonkey Link: https://www.surveymonkey.com/s/RHJLHS7. 
This survey may be used to provide feedback on answers to questions 
regarding potential biomarkers for qualification and to describe 
contexts of use to address areas important to drug development.

Written Submissions

    Submit written submissions in the following ways:
     Mail/Hand delivery/Courier (for paper submissions): 
Division of Dockets Management (HFA-305), Food and Drug Administration, 
5630 Fishers Lane, Rm. 1061, Rockville, MD 20852.
    Instructions: All submissions received must include the Docket No. 
FDA-2014-N-2187 for this document. All comments received may be posted 
without change to http://www.regulations.gov, including any personal 
information provided. It is only necessary to send one set of comments. 
For additional information on submitting comments, see the ``Request 
for Information'' heading of the SUPPLEMENTARY INFORMATION section of 
this document.
    Docket: For access to the docket to read background documents or 
comments received, go to http://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 Division of 
Dockets Management, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852, 
between 9 a.m. and 4 p.m., Monday through Friday.

FOR FURTHER INFORMATION CONTACT: Marianne Noone, Center for Devices and 
Radiological Health, Food and Drug Administration, 10903 New Hampshire 
Ave., Bldg. 21, Rm. 4528, Silver Spring, MD 20993-0002, 301-796-7495.

[[Page 8090]]


SUPPLEMENTARY INFORMATION:

I. Background

    The President signed into law the Food and Drug Administration 
Safety and Innovation Act (FDASIA) (Pub. L. 112-144) on July 9, 2012. 
Title I of FDASIA reauthorizes the Prescription Drug User Fee Act 
(PDUFA) and provides FDA with the user fee resources necessary to 
maintain an efficient review process for human drug and biological 
products. The reauthorization of PDUFA added performance goals and 
procedures for the Agency that represent FDA's commitments during 
fiscal years 2013 through 2017. These commitments are fully described 
in the document entitled ``PDUFA Reauthorization Performance Goals and 
Procedures Fiscal Years 2013 through 2017'' (PDUFA Goals Letter), 
available on FDA's Web site at http://www.fda.gov/downloads/ForIndustry/UserFees/PrescriptionDrugUserFee/UCM270412.pdf. Section IX 
of the PDUFA Goals Letter entitled ``Enhancing Regulatory Science and 
Expediting Drug Development'' references support for the identification 
and advancement of biomarkers.
    A biomarker is an objective characteristic that is measured and 
evaluated as an indicator of normal biologic processes, pathogenic 
processes, or pharmacologic responses to treatment. Biomarkers can 
serve many purposes in clinical drug development, including the 
following: Defining the appropriate patient populations for study, as 
well as those who should receive the drug in clinical practice; 
pharmacodynamic markers for proof of concept and dose selection; and 
pharmacodynamic markers of adverse effects. A subset of pharmacodynamic 
biomarkers can serve as replacements for clinical efficacy endpoints 
that reflect how a patient feels, functions, or survives. The path to 
development of promising therapeutics can be enabled by the 
availability of biomarkers that are analytically validated and 
clinically qualified for a specific context of use (i.e., a 
comprehensive, clear, and precise statement that describes the manner 
of use, interpretation, and purpose of use of a biomarker in drug 
development).
    Qualification is based on a body of evidence that demonstrates that 
the biomarkers are fit for purpose in drug development and evaluation 
(http://www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugDevelopmentToolsQualificationProgram/ucm284076.htm). Further, 
qualification is dependent on the specific proposed context of use. 
Biomarkers that are qualified can help to progressively reduce 
uncertainty about the outcome of clinical development programs.
    Public/private partnerships involving regulatory, academic, and 
industry scientists, collaborating within a precompetitive framework, 
are essential to catalyzing progress. Because of limitations in 
resources, such efforts must be focused on the opportunities that offer 
the greatest potential for impact.
    FDA intends to facilitate identification of the most promising 
biomarkers and the areas important to drug development and to promote 
efforts that will aid in the qualification and regulatory adoption of 
the drug development framework.

II. Request for Information

    FDA is seeking public feedback to identify promising biomarker 
candidates in areas important to drug development and to identify 
considerations for evidence needed to qualify various types of 
biomarkers for specific contexts of use. FDA requests identification of 
specific biomarkers with a proposed context of use and of the type of 
evidence needed to support qualification. After reviewing the 
information provided, FDA will post the collated information on its Web 
site.

A. Information Requirements

    In general, submitted information should include the following for 
each biomarker nominated, as well as any other relevant information:
     Areas that have a critical need for biomarkers to assist 
drug development;
     The name of the biomarker;
     The proposed context of use for the biomarker (if known);
     The reason why the biomarker should be considered, taking 
into account its usefulness as a drug development tool; and
     Any evidence that should be developed to support 
qualification of the biomarker.

B. Questions and Requests

    Specific questions and requests are as follows:
    1. Are there specific aspects of drug development that could be 
enhanced through the development of biomarkers?
    a. Please list the specific applications of biomarkers that address 
areas important to drug development.
    b. Please list the specific areas (for example, a specific disease 
area or an organ toxicity) needed for development of biomarkers 
important to drug development.
    c. Is there information or efforts which could be leveraged to 
advance these areas? If yes, please describe.
    d. Are there areas that appear to be promising for the development 
of new biomarkers and for which collaborative engagement from 
stakeholders offers a path forward? If so, please explain.
     Are there groups positioned to accomplish this? If yes, 
please describe.
    e. Are there barriers that preclude engagement or investment in 
biomarkers for these priority areas? If yes, please explain.
    2. In each of these priority areas that are important to drug 
development, please provide the following information:
    a. Biomarker: What specific biomarkers do you believe represent the 
greatest near-term opportunity to establish utility in drug development 
(i.e., that could be substantially advanced by facilitating discussion 
and consensus building)?
    b. Rationale: Why should the biomarker(s) be included on the list, 
taking into account its usefulness in regulatory decisionmaking as a 
drug development tool?
    c. Context of use: Can you please describe/propose a specific 
context of use for the biomarker(s)?
    d. Evidentiary gaps: To support the proposed context of use, what 
do you see as the largest evidentiary gaps that need to be addressed to 
permit ``fit for purpose'' qualification?
    e. How can these evidentiary gaps be addressed?
    f. Collaborative data sharing: Can any of these gaps be addressed 
by collaborative data sharing of existing data versus prospective 
studies specifically dedicated to addressing the gap?
    3. Please indicate your affiliation from the following list: 
Academia, pharmaceutical sector, biotechnology sector, government, 
professional organization, non-profit organization, clinician, patient 
advocacy group, patient, or other (please provide specifics, if you 
choose other).

III. Paperwork Reduction Act

    This Federal Register notice requests input from biomarker experts 
from academia, the pharmaceutical industry, and government 
organizations on the evidentiary standards for biomarkers or on the 
expectations about data for qualification of different types of 
biomarkers.
    This request is exempt from the Office of Management and Budget's 
review under 5 CFR 1320.3(h)(4): Facts or opinions submitted in 
response to general solicitations of comments from the public, 
published in the Federal Register or other publications, regardless of 
the form or format thereof,

[[Page 8091]]

provided that no person is required to supply specific information 
pertaining to the commenter, other than that necessary for self-
identification, as a condition of the Agency's full consideration of 
the comment.

    Dated: February 5, 2015.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2015-02976 Filed 2-12-15; 8:45 am]
BILLING CODE 4164-01-P