[Federal Register Volume 88, Number 84 (Tuesday, May 2, 2023)]
[Notices]
[Pages 27521-27522]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2023-09265]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2023-N-1506]


Methodological Challenges Related to Patient Experience Data; 
Request for Information and Comments

AGENCY: Food and Drug Administration, HHS.

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

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SUMMARY: The Food and Drug Administration (FDA or Agency) is 
establishing a public docket to collect comments on methodological 
challenges related to patient experience data in the context of the 
benefit-risk assessment and product labeling, and other areas of 
greatest interest or concern to public stakeholders. Public comments 
will help FDA plan two public workshops focused on methodological 
challenges and identify priorities for future work.

DATES: Although you can comment at any time, to ensure the Agency 
considers your comment in our development of the workshops, submit 
either electronic or written information and comments by July 3, 2023.

ADDRESSES: You may submit comments and information 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-2023-N-1506 for ``Methodological Challenges Related to Patient 
Experience Data.'' 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

[[Page 27522]]

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: Ethan Gabbour, Center for Drug 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg. 51, Rm. 6306, Silver Spring, MD 20993, 301-796-
9208, [email protected].

SUPPLEMENTARY INFORMATION: 

I. Background

    Under the seventh iteration of the Prescription Drug User Fee Act 
(PDUFA VII), incorporated as part of the FDA User Fee Reauthorization 
Act of 2022, FDA committed to continue to strengthen capacity to 
facilitate development and use of patient-focused methods to inform 
drug development and regulatory decisions, including issuing this 
Request for Information (RFI) to elicit public input on methodologic 
challenges encountered by stakeholders, and other areas of greatest 
interest or concern to public stakeholders. These methodologic 
challenges may be related to the collection and analysis of patient 
experience data, generally, or they may be related more specifically to 
the submission and evaluation of patient experience data in the context 
of FDA's benefit-risk assessment or product labeling.
    The feedback received as part of this RFI will be summarized in a 
subsequent Federal Register document and will help to inform future 
public workshops focused on methodologic challenges related to patient-
focused drug development. The Federal Food, Drug, and Cosmetic Act, as 
amended by the 21st Century Cures Act (Pub. L. 114-255) and the FDA 
Reauthorization Act of 2017 (FDARA) (Pub. L. 115-52), defines patient 
experience data as: ``data that (1) are collected by any persons 
(including patients, family members, and caregivers of patients, 
patient advocacy organizations, disease research foundations, 
researchers, and drug manufacturers); and (2) are intended to provide 
information about patients' experiences with a disease or condition, 
including (A) the impact (including physical and psychosocial impacts) 
of such disease or condition, or a related therapy or clinical 
investigation, on patients' lives; and (B) patient preferences with 
respect to treatment of such disease or condition.'' \1\
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    \1\ Patient experience data is defined for purposes of this 
guidance in Title III, section 3001 of the 21st Century Cures Act, 
as amended by section 605 of FDARA, https://www.congress.gov/115/plaws/publ52/PLAW-115publ52.pdf.
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II. Request for Information and Comments

    Interested persons are invited to provide detailed information and 
comments on methodological challenges relating to patient experience 
data, including the submission and evaluation of patient experience 
data in the context of the benefit-risk assessment and product 
labeling. Please provide the rationale for any suggestions and include 
supporting data if available. FDA is particularly interested in 
information related to the following:
    (1) Describe any perceived barriers to the use of patient 
experience data for regulatory decision making (e.g., benefit-risk 
assessment, product labeling).
    (2) Describe any challenges and limitations experienced when 
selecting, modifying, or developing fit-for-purpose Clinical Outcome 
Assessment measures.
    (3) Describe any challenges and statistical analysis considerations 
when constructing and selecting endpoints of interest and in 
understanding whether an estimated treatment effect corresponds to a 
real difference in patients' lives.
    (4) Describe any challenges and limitations experienced when 
developing and conducting patient preference studies to support 
regulatory submissions.
    (5) Describe any challenges and limitations when submitting patient 
experience data to FDA.
    The public comments collected will help FDA plan two workshops 
focused on methodological challenges with patient experience data and 
will identify opportunities for future work.

    Dated: April 27, 2023.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2023-09265 Filed 5-1-23; 8:45 am]
BILLING CODE 4164-01-P


