May 2 -- 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. To ensure the Agency considers your comment in our development of the workshops, submit information and comments by July 3, 2023.
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.”
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.
FRN:
https://www.federalregister.gov/d/2023-09265