Notice2023-09265
Methodological Challenges Related to Patient Experience Data; Request for Information and Comments
Primary source
Metadata and text below are from the Federal Register, a public-domain U.S. government work. Always verify the official published version before relying on it for any legal matter.
Published
May 2, 2023
Issuing agencies
Health and Human Services DepartmentFood and Drug Administration
Abstract
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.
Full Text
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<title>Federal Register, Volume 88 Issue 84 (Tuesday, May 2, 2023)</title>
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[Federal Register Volume 88, Number 84 (Tuesday, May 2, 2023)]
[Notices]
[Pages 27521-27522]
From the Federal Register Online via the Government Publishing Office [<a href="http://www.gpo.gov">www.gpo.gov</a>]
[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:
<bullet> Federal eRulemaking Portal: <a href="https://www.regulations.gov">https://www.regulations.gov</a>.
Follow the instructions for submitting comments. Comments submitted
electronically, including attachments, to <a href="https://www.regulations.gov">https://www.regulations.gov</a>
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 <a href="https://www.regulations.gov">https://www.regulations.gov</a>.
<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-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 <a href="https://www.regulations.gov">https://www.regulations.gov</a> 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
[[Page 27522]]
claimed confidential information redacted/blacked out, will be
available for public viewing and posted on <a href="https://www.regulations.gov">https://www.regulations.gov</a>.
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: <a href="https://www.govinfo.gov/content/pkg/FR-2015-09-18/pdf/2015-23389.pdf">https://www.govinfo.gov/content/pkg/FR-2015-09-18/pdf/2015-23389.pdf</a>.
Docket: For access to the docket to read background documents or
the electronic and written/paper comments received, go to <a href="https://www.regulations.gov">https://www.regulations.gov</a> 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, <a href="/cdn-cgi/l/email-protection#4401302c252a6a032526262b3136042220256a2c2c376a232b32"><span class="__cf_email__" data-cfemail="2b6e5f434a45056c4a4949445e596b4d4f4a05434358054c445d">[email protected]</span></a>.
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, <a href="https://www.congress.gov/115/plaws/publ52/PLAW-115publ52.pdf">https://www.congress.gov/115/plaws/publ52/PLAW-115publ52.pdf</a>.
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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
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