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USFDA Guidance: Submitting Patient-Reported Outcome Data, Clinical Trial Datasets & Documentation

Today (06 November, 2023) the U.S. Food and Drug Administration (FDA) Center for Drug Evaluation and Research (CDER) released two final guidances on "Submitting Patient-Reported Outcome Data in Cancer Clinical Trials" and "Submitting Clinical Trial Datasets and Documentation for Clinical Outcome Assessments Using Item Response Theory".

As part of a marketing application for a medical product in oncology, this document provides technical specifications for the submission of patient-reported outcome (PRO) data collected in cancer clinical trials, which is a type of clinical outcome assessment (COA) that collects patient experience data.


The FDA Patient-Focused Drug Development (PFDD) Glossary defines a PRO as a measurement based on a report that comes directly from the patient (i.e., study subject) about the status of a patient's health condition without interpretation of the patient’s response by a clinician or anyone else, where a PRO can be measured by self-report or by interview, provided that the interviewer records only the patient’s response.


This guidance provides specifications for the submission of Clinical Data Interchange Standards Consortium (CDISC) Study Data Tabulation Model (SDTM) and Analysis Data Model (ADaM) datasets and specifications for recommended tables and figures.


These technical specifications aim to provide general guidelines for

  1. standardized dataset content and structure and

  2. recommended tables and figures to facilitate FDA review of the marketing application that the submitted data and analysis outputs are intended to support.


This guidance provides technical specifications for the submission of clinical outcome assessment (COA) data using Item Response Theory (IRT) and supplements FDA CDER Patient-Focused Drug Development (PFDD) Methodological Guidance Series. IRT is a family of mathematical models that describes the functional relationship between item performance, item characteristics, and the patient’s status on the construct being measured.

As described in the Biomarkers, EndpointS, and other Tools (BEST) Resource glossary, a COA refers to the assessment of a clinical outcome made through a report by a clinician, a patient, a non-clinician observer, or through a performance-based assessment; thus, there are four types of COAs:

  1. clinician-reported outcome (ClinRO),

  2. patientreported outcome (PRO),

  3. observer-reported outcome (ObsRO), and

  4. performance outcome (PerfO) measures.

To know more details about the SDTM Specifications & dataset, click this LINK.

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