Description
This guidance (Guidance 4) is the fourth in a series of four methodological patient-focused drug development (PFDD) guidance documents that describe how stakeholders (patients, caregivers, researchers, medical product developers, and others) can collect and submit patient experience data and other relevant information from patients and caregivers to be used for medical product development and regulatory decision-making.
Scope & Applicability
Product Classes
3Defined in section 351 of the Public Health Service Act
Products combining drug, device, or biological constituents; Generally recommended for Enhanced Documentation; Requires 14971-based framework incorporating ICH Q9; A drug-device combination where the device constituent part detects ingestion.
To determine whether a medical product has a positive, meaningful effect; The document discusses determining whether a medical product produces a meaningful effect.; comparing a new product A to a current product B
Stakeholders
6Individuals providing information for medical product development; Participants in facilitated discussions and observational studies
Early engagement with patient communities can improve the patient-centeredness of trial procedures.
sponsors might consider seeking input from members of the patient community
It may be a hardship for patients and/or caregivers to attend in-person; Individuals who may assist patients or provide observations when patients cannot self-report.; conducted with caregivers to obtain similar feedback
Stakeholders who collect and submit patient experience data
Entity responsible for submitting applications under section 524B
Regulatory Context
Attributes
10Determining the significance of changes in COA-based endpoints
Improvement in PerfO tasks due to repetition rather than health improvement
When a COA-based endpoint is on an ordinal scale
Used to evaluate if the overall survival HR excludes a threshold
MSRs are used to interpret the meaningfulness of treatment effects by specifying the meaning of individual COA scores.
MSDs are used to inform the choice of meaningful score differences in COA scores.
FDA evaluates how well results of a COA-based endpoint correspond to a treatment benefit that is meaningful to patients; interpreting the meaningfulness of continuous COA-based endpoints
Endpoint construction method measuring difference from initial state
Prespecified value used for dichotomization of data
statistical significance does not indicate whether the detected effect corresponds to a clinically meaningful effect
Related CFR Sections (2)
- 21CFR812.140§ 812.140 Records.
(a) Investigator records. A participating investigator shall maintain the following accurate, complete, and current records relating to the investigator's participation in an investigation:Read full regulation →
- 21CFR312.57§ 312.57 Recordkeeping and record retention.
(a) A sponsor shall maintain adequate records showing the receipt, shipment, or other disposition of the investigational drug. These records are required to include, as appropriate, the name of the investigator to whom the drug is shipped, and the date, quantity, and batch or code mark of each such Read full regulation →
Related Warning Letters (10)
- 2025-12-23
In Vivo Bioavailability-Bioequivalence Studies – Clinical
Maria A. Carballosa, M.D.
- 2025-12-09
Unapproved New Drug/Unlicensed Biological Product/Biologics License Application (BLA)
Celularity, Inc
- 2025-11-18
Sponsor/Investigator
Verdure Sciences, Inc.
- 2025-09-30
Clinical Investigator (Sponsor)
Pamela K. Den Besten, DDS, MS
- 2025-09-23
Clinical Investigator/Sponsor
Ralph A. DeFronzo, M.D.
- 2025-09-09
Clinical Investigator
Shirish M. Gadgeel, M.D.
- 2025-07-15
Clinical Investigator
Mark J. Savant, M.D
- 2025-07-01
Clinical Investigator
Peter Michael, M.D.
- 2025-06-10
Clinical Investigator (Sponsor)
American Behavioral Research Institute, LLC
- 2025-06-03
Bioresearch Monitoring Program
Amy Lightner, MD
See Also (8)
- Investigator Responsibilities — Protecting the Rights, Safety, and Welfare of Study Subjects: Guidance for Industry (Status: Final)
- Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims: Guidance for Industry (Status: Final)
- In Vitro Diagnostic (IVD) Device Studies - Frequently Asked Questions: Guidance for Industry and FDA Staff (Status: Final)
- Electronic Source Data in Clinical Investigations: Guidance for Industry (Status: Final)
- Use of Electronic Health Record Data in Clinical Investigations Guidance for Industry (Status: Final)
- Considerations for the Conduct of Clinical Trials of Medical Products During Major Disruptions Due to Disasters and Public Health Emergencies (Status: Final)
- Conducting Clinical Trials With Decentralized Elements (Status: Final)
- Current Good Manufacturing Practice for Phase 1 Investigational Drugs: Guidance for Industry (Status: Final)