Description
The Food and Drug Administration (FDA or Agency) is announcing the availability of a final guidance for industry, FDA staff, and other stakeholders entitled “Patient-Focused Drug Development: Selecting, Developing, or Modifying Fit-for-Purpose Clinical Outcome Assessments.” This guidance (Guidance 3) is the third in a series of four methodological patient-focused drug development (PFDD) guidance documents that describe how stakeholders (patients, researchers, medical product developers, and others) can submit patient experience and other relevant information from patients and caregivers to be used for medical product development and regulatory decision-making. This guidance finalizes the draft guidance of the same title issued on June 30, 2022.
Scope & Applicability
Product Classes
3Includes drugs, devices, and biological products
Medical devices intended for human use; Approved or cleared medical devices
Technologies like mobile sensors used for remote data acquisition.
Stakeholders
10Stakeholders involved in collecting patient experience data
Provides clinical judgment and conducts assessments for ClinROs.
A PRO cannot be completed by a proxy reporter.
entity that creates the COA and defines intended meanings of items
Collaborators encouraged for precompetitive COA development
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
Entity responsible for submitting applications under section 524B
Stakeholders providing relevant information for medical product development; Source of data on the nature and importance of the MAH
Stakeholders using patient experience data for regulatory decision-making
Assist sponsors in the nonclinical evaluation
Regulatory Context
Attributes
8Determining whether a COA is appropriate for its context of use; A COA having sufficient evidence to support its context of use.; COA validation is sufficient to support its context of use; The status of a COA being appropriate for its intended context of use; conclusion that a COA is appropriate for its intended context of use; Determination of a COA's suitability for its intended use; Standard for determining if a COA is appropriate for its intended use; Selecting, Developing, or Modifying Fit
Establishing and Reporting the Evidence in Newly Developed Patient-Reported Outcomes
Improvement in PerfO tasks due to repetition rather than health improvement
Variation in scores that does not reflect the concept of interest
The time frame over which respondents are asked to remember experiences
the specific thing being measured by the COA
MAH targeted by the intervention
The specific environment and population in which a COA is intended to be used; Selecting fit-for-purpose COAs based on the context of use.; The intended context in which the COA will be used
Identified Hazards
Hazards
4Influence on COA scores based on respondent or administrator expectations
A concern that may impact performance on time- or effort-sensitive tasks.
Patients improving performance due to repeated exposure rather than disease change.
Burden due to measure length or complexity affecting data quality
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)
- ANDA Submissions – Prior Approval Supplements Under GDUFA: Guidance for Industry (Status: Final)
- CPG Sec. 400.210, Radiofrequency Identification Feasibility Studies and Pilot Programs for Drugs (Status: Final)
- Format and Content of the Microbiology Section of an Application*: Guidance for Industry (Status: Final)
- Formatting, Assembling and Submitting New Drug and Antibiotic Applications* (Status: Final)
- Format and Content of the Human Pharmacokinetics and Bioavailability Section of an Application (Status: Final)
- Nuclear Pharmacy Guideline Criteria for Determining When to Register as a Drug Establishment (Status: Final)
- Women and Minorities Guidance Requirements (Status: Final)
- Levothyroxine Sodium Products Enforcement of August 14, 2001 Compliance Date and Submission of New Applications (Status: Final)