Description
This guidance describes how the Food and Drug Administration (FDA) reviews and evaluates existing, modified, or newly created patient-reported outcome (PRO) instruments used to support claims in approved medical product labeling. A PRO instrument (i.e., a questionnaire plus the information and documentation that support its use) is a means to capture PRO data used to measure treatment benefit or risk in medical product clinical trials. This guidance does not address the use of PRO instruments for purposes beyond evaluation of claims made about a medical product in labeling. This guidance also does not address disease-specific issues. Guidance on clinical trial endpoints for specific diseases can be found on various FDA Web sites.
Scope & Applicability
Product Classes
3Patient-Reported Outcome instruments used in medical product development.; application of PRO instruments to multiple cultures or languages; Incorporating PRO instruments as clinical trial endpoint measures.; Instruments used to support labeling claims
A measurement based on a report that comes directly from the patient about the status of a health condition.
Use in Medical Product Development to Support Labeling Claims
Stakeholders
6Entity responsible for submitting applications under section 524B
Source of safety information in clinical trials
HHS officer or employee who collects an official sample
Veterinarian responsible for selecting anesthetic regimens and conducting field studies
Dosage recommendations for pediatric subpopulations
Assist sponsors in the nonclinical evaluation
Regulatory Context
Attributes
10A characteristic of PRO instruments reviewed by the FDA.; The time period patients are asked to remember when responding to a PRO instrument.; The period of time patients are asked to consider in responding to a PRO item.; A modification to the original instrument that requires rationale
The effect of treatment on how a patient survives, feels, or functions.
Manufacturing scale defined as 200 kg
The point when no new relevant or important information emerges in qualitative research
Attributes relevant to the application of a PRO instrument including content validity and reliability.
Statistical parameter requiring strong control when testing multiple endpoints
The data maintained by the clinical investigator should include an audit trail.
without an electronic audit trail that documents all changes to the data
used to weight items or domains
reliability includes accuracy, completeness, and traceability
Identified Hazards
Hazards
2Undue physical, emotional, or cognitive strain on patients; Methods of missing data imputation should take the patient population, disease progression, and respondent burden into account.
risk of data corruption or loss during the trial
Related CFR Sections (5)
- 21CFR201.56§ 201.56 Requirements on content and format of labeling for human prescription drug and biological products.
(a) General requirements. Prescription drug labeling described in § 201.100(d) must meet the following general requirements:Read full regulation →
- 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 →
- 21CFR312.50§ 312.50 General responsibilities of sponsors.
Sponsors are responsible for selecting qualified investigators, providing them with the information they need to conduct an investigation properly, ensuring proper monitoring of the investigation(s), ensuring that the investigation(s) is conducted in accordance with the general investigational plan Read full regulation →
- 21CFR201.57§ 201.57 Specific requirements on content and format of labeling for human prescription drug and biological products described in § 201.56(b)(1) .
The requirements in this section apply only to prescription drug products described in § 201.56(b)(1) and must be implemented according to the schedule specified in § 201.56(c) , except for the requirement in paragraph (c)(18) of this section to reprint any FDA-approved patient labeling at the end oRead full regulation →
- 21CFR606.122§ 606.122 Circular of information.
A circular of information must be available for distribution if the product is intended for transfusion. The circular of information must provide adequate directions for use, including the following information:Read full regulation →
Related Warning Letters (6)
- 2025-06-03
Bioresearch Monitoring Program
Amy Lightner, MD
- 2023-11-14
CGMP/QSR/Medical Devices/Misbranded
Wello Inc.
- 2022-06-28
CGMP/Active Pharmaceutical Ingredient (API)/Adulterated
Fagron Group B.V.
- 2020-05-05
CGMP/Dietary Supplement/Adulterated/Misbranded
Hawaii Pharm LLC
- 2020-04-07
Investigational Device Exemptions (Clinical Investigator)
Kennedy, Philip R.
- 2020-03-10
Clinical Investigator
James R. Corbett, M.D.
See Also (8)
- Classification Categories for Certain Supplements Under BsUFA III (Status: Final)
- Guidance for Industry for the Evaluation of Combination Vaccines for Preventable Diseases: Production, Testing and Clinical Studies: Guidance for Industry (Status: Final)
- Providing Regulatory Submissions to the Center for Biologics Evaluation and Research (CBER) in Electronic Format - Biologics Marketing Applications: Guidance for Industry (Status: Final)
- FDA Review of Vaccine Labeling Requirements for Warnings, Use Instructions, and Precautionary Information: Guidance for Industry (Status: Final)
- Clinical Studies Section of Labeling for Human Prescription Drug and Biological Products — Content and Format: Guidance for Industry (Status: Final)
- Useful Written Consumer Medication Information (CMI) (Status: Final)
- Hypertension Indication: Drug Labeling for Cardiovascular Outcome Claims (Status: Final)
- PET Drug Applications - Content and Format for NDAs and ANDAs_2011 (Status: Final)