[Federal Register Volume 86, Number 35 (Wednesday, February 24, 2021)]
[Notices]
[Pages 11301-11303]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2021-03727]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2021-N-0031]


Best Practices for Development and Application of Disease 
Progression Models; Public Workshop; Establishment of a Public Docket; 
Request for Comments

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice; establishment of a public docket; request for comments.

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SUMMARY: One of the goals of the Prescription Drug User Fee Act of 2017 
(PDUFA VI), part of the FDA Reauthorization Act of 2017 (FDARA), is 
advancing model-informed drug development (MIDD). The ``Best Practices 
for Development and Application of Disease Progression Models'' 
workshop fulfills FDA's performance commitment under PDUFA VI to hold a 
workshop. The Food and Drug Administration (FDA or Agency) is opening a 
docket to solicit public input on topics areas for an upcoming disease 
progression modeling workshop. The purpose of this public workshop is 
to discuss the best practices for developing disease progression models 
and their application to support drug development decisions; share

[[Page 11302]]

experiences and case studies that highlight the opportunities and 
limitations in the development and application of disease progression 
models including models for natural history of disease and clinical 
trial simulations; and discuss the knowledge gaps and research needed 
to advance the development and use of disease progression models.

DATES: To ensure that the Agency considers your input, submit either 
electronic or written comments by March 26, 2021.

ADDRESSES: FDA is establishing a docket for public comment on this 
workshop. The docket number is FDA-2021-N-0031. The docket will close 
on March 26, 2021. Submit either electronic or written comments on this 
public workshop by March 26, 2021. Please note that late, untimely 
filed comments will not be considered. Electronic comments must be 
submitted on or before March 26, 2021. The https://www.regulations.gov 
electronic filing system will accept comments until 11:59 p.m. Eastern 
Time at the end of March 26, 2021. Comments received by mail/hand 
delivery/courier (for written/paper submissions) will be considered 
timely if they are postmarked or the delivery service acceptance 
receipt is on or before that date.
    You may submit comments as follows:

Electronic Submissions

    Submit electronic comments in the following way:
    <bullet> 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.
    <bullet> 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:
    <bullet> 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.
    <bullet> 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-2021-N-0031 for ``Best Practices for Development and Application of 
Disease Progression Models; Public Workshop; Request for Comments.'' 
Received comments, those filed in a timely manner (see ADDRESSES), 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.
    <bullet> 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.

FOR FURTHER INFORMATION CONTACT: Maryanne Dingman, Office of Clinical 
Pharmacology, Center for Drug Evaluation and Research, Food and Drug 
Administration, 10903 New Hampshire Ave., Silver Spring, MD 20993-0002, 
301-796-8777, 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

    Under FDARA, FDA agreed, in accordance with section I of the PDUFA 
VI Performance Goals, ``Ensuring the Effectiveness of the Human Drug 
Review, part J, Enhancing Regulatory Decision Tools to Support Drug 
Development and Review,'' to hold several workshops to identify best 
practices for MIDD. The workshop entitled ``Best Practices for 
Development and Application of Disease Progression Models,'' to be held 
in 2021, fulfills FDA's performance commitment under PDUFA VI. FDA is 
requesting comments from the public to help identify areas of interest 
to be discussed during the workshop given the wide range of approaches 
to data collection, aggregation modeling, model development, 
verification and validation, and potential applications in drug 
development and regulatory review. The outcome will help the Agency 
inform the public on current experience, emerging techniques, and 
limitations to streamline the drug model development and facilitate the 
decision-making process.

II. Request for Information and Comments

    Interested persons are invited to provide detailed information and 
comments on areas of interest to discuss during the upcoming ``Best 
Practices for Development and Application of Disease Progression Models 
'' workshop. FDA is interested in responses about best practice 
considerations including, but not limited to, the following:
    1. The development and application of different types of disease 
progression models (e.g., empirical, semi-mechanistic, and fully 
mechanistic or systems modeling).

[[Page 11303]]

    2. Modeling natural history of disease, specifically methodological 
considerations and challenges in characterizing the natural 
relationship between pharmacodynamic markers and clinical outcomes.
    3. Clinical trial simulations based on disease progression/natural 
history models to support drug development and regulatory decisions.

    Dated: February 16, 2021.
Lauren K. Roth,
Acting Principal Associate Commissioner for Policy.
[FR Doc. 2021-03727 Filed 2-23-21; 8:45 am]
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