Methodological Challenges Related to Patient Experience Data; Request for Information and Comments, 27521-27522 [2023-09265]

Download as PDF Federal Register / Vol. 88, No. 84 / Tuesday, May 2, 2023 / Notices * 10. Amgen Inc., ‘‘Enbrel (etanercept): Highlights of Prescribing Information,’’ revised April 2021, available at https:// www.pi.amgen.com/∼/media/amgen/ repositorysites/pi-amgen-com/enbrel/ enbrel_pi.pdf, accessed May 16, 2022. * 11. Reuter, E., ‘‘‘Smart Pill’ Startup EtectRx Strikes Partnership with Pear Therapeutics,’’ Med City News, January 14, 2021, available at https:// medcitynews.com/2021/01/smart-pillstartup-etectrx-strikes-partnership-withpear-therapeutics, accessed May 16, 2022. 12. The Medical Futurist, ‘‘The Present and Future of Digital Pills,’’ July 21, 2020, available at https://medicalfuturist.com/ the-present-and-future-of-digital-pills, accessed May 16, 2022. 13. George, C.E., ‘‘Should a Psychiatrist Prescribe a Nanodrug to Help Parents Monitor a Teen’s Adherence?,’’ AMA Journal of Ethics, Vol. 21, Issue 4, Article e317–323, 2019, doi:10.1001/ amajethics.2019.317. * 14. Yang, M., ‘‘A Psychiatrist’s Perspective on the Digital Pill,’’ KevinMD.com, December 2, 2017, available at https:// www.kevinmd.com/blog/2017/12/ psychiatrists-perspective-digitalpill.html, accessed May 16, 2022. Dated: April 27, 2023. Lauren K. Roth, Associate Commissioner for Policy. Notice; establishment of a public docket; request for information and comments. ACTION: A Risk-Based Approach to Monitoring of Clinical Investigations—Questions and Answers; Guidance for Industry; Correction Food and Drug Administration, HHS. Notice of availability; correction. ACTION: ddrumheller on DSK120RN23PROD with NOTICES1 Food and Drug Administration, HHS. [Docket No. FDA–2019–D–0362] The Food and Drug Administration (FDA) is correcting a notice that appeared in the Federal Register of April 12, 2023. The document announced the availability of a final guidance entitled ‘‘A Risk-Based Approach to Monitoring of Clinical Investigations—Questions and Answers; Guidance for Industry.’’ The notice of availability for this final guidance was published with an incorrect OMB control number. This document corrects that error. FOR FURTHER INFORMATION CONTACT: Mona Shing, Center for Drug Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Jkt 259001 DEPARTMENT OF HEALTH AND HUMAN SERVICES AGENCY: Food and Drug Administration 18:14 May 01, 2023 BILLING CODE 4164–01–P Methodological Challenges Related to Patient Experience Data; Request for Information and Comments DEPARTMENT OF HEALTH AND HUMAN SERVICES VerDate Sep<11>2014 [FR Doc. 2023–09264 Filed 5–1–23; 8:45 am] [Docket No. FDA–2023–N–1506] BILLING CODE 4164–01–P SUMMARY: Dated: April 27, 2023. Lauren K. Roth, Associate Commissioner for Policy. Food and Drug Administration [FR Doc. 2023–09268 Filed 5–1–23; 8:45 am] AGENCY: Ave., Bldg. 51, Rm. 3355, Silver Spring, MD 20993–0002, 301–796–0910. SUPPLEMENTARY INFORMATION: In the Federal Register of April 12, 2023 (88 FR 22038), in FR Doc. 2023–07687, the following correction is made: 1. On page 22040, in the first column, in the last sentence of ‘‘II. Paperwork Reduction Act of 1995,’’ the OMB control number 0910–0733 is corrected to read: ‘‘. . .and the collections of information in FDA’s guidance for industry entitled ‘‘Oversight of Clinical Investigations—A Risk-Based Approach to Monitoring’’ have been approved under OMB control number 0910– 0014.’’ The correction changes the OMB control number from a number that was discontinued to an active one. 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: SUMMARY: Electronic Submissions Submit electronic comments in the following way: • Federal eRulemaking Portal: https://www.regulations.gov. Follow the PO 00000 Frm 00091 Fmt 4703 Sfmt 4703 27521 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 E:\FR\FM\02MYN1.SGM 02MYN1 27522 Federal Register / Vol. 88, No. 84 / Tuesday, May 2, 2023 / Notices 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-201509-18/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, Ethan.Gabbour@fda.hhs.gov. SUPPLEMENTARY INFORMATION: ddrumheller on DSK120RN23PROD with NOTICES1 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 VerDate Sep<11>2014 18:14 May 01, 2023 Jkt 259001 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 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. 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. PO 00000 Frm 00092 Fmt 4703 Sfmt 9990 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 DEPARTMENT OF HEALTH AND HUMAN SERVICES Food and Drug Administration [Docket No. FDA–2023–N–1259] Advancing the Utilization and Supporting the Implementation of Innovative Manufacturing Approaches; Public Workshop; Request for Comments; Correction AGENCY: Food and Drug Administration, HHS. Notice of public workshop; request for comments; correction. ACTION: The Food and Drug Administration is correcting a notice entitled ‘‘Advancing the Utilization and Supporting the Implementation of Innovative Manufacturing Approaches’’ that appeared in the Federal Register of April 24, 2023. The document announced a public workshop. The document was published with an incorrect topic for discussion. This document corrects that error. FOR FURTHER INFORMATION CONTACT: Lisa Granger, Office of Policy, Legislation, and International Affairs, Food and Drug Administration, 301–796–9115, Lisa.Granger@fda.hhs.gov. SUPPLEMENTARY INFORMATION: In the Federal Register of April 24, 2023, in FR Doc. 2023–08545 (88 FR 24807), on page 24808, the following correction is made: • On page 24808, in the second column, in Section II, ‘‘Topics for Discussion at the Public Workshop,’’ the fifth topic, ‘‘Science- and risk-based approaches for developing and accessing innovative technologies across platform products and sites to streamline adoption.’’ is corrected to read ‘‘Science- and risk-based approaches for developing and assessing innovative technologies across platform products and sites to streamline adoption.’’ SUMMARY: Dated: April 26, 2023. Lauren K. Roth, Associate Commissioner for Policy. [FR Doc. 2023–09206 Filed 5–1–23; 8:45 am] BILLING CODE 4164–01–P E:\FR\FM\02MYN1.SGM 02MYN1

Agencies

[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]


-----------------------------------------------------------------------

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.

-----------------------------------------------------------------------

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\
---------------------------------------------------------------------------

    \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.
---------------------------------------------------------------------------

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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