Two FDA Guidances Present Technical Specifications for Clinical Trial Data
The FDA has issued a pair of final guidances meant to assist sponsors with submitting patient-reported outcome (PRO) data gathered in cancer trials and general clinical outcome assessment (COA) data, respectively, offering technical specifications.
In the 20-page final guidance, “Submitting Clinical Trial Datasets and Documentation for Clinical Outcome Assessments Using Item Response Theory,” the FDA provides technical specifications for submitting COA data that uses Item Response Theory (IRT), a family of mathematical models that describe “the functional relationship between item performance, item characteristics and the patient’s status on the construct being measured.”
Specifically, the guidance covers static, fixed-length COAs that are developed and/or scored using IRT and COAs that are administered using IRT-based Computerized Adaptive Testing, a type of individual testing done by a computer “in which successive items in the COA measure are selected for administration based primarily on the item’s psychometric properties and content in relation to the patients’ responses to previous items.”
The agency notes that the final guidance is meant to supplement CDER’s Patient-Focused Drug Development (PFDD) Methodological Guidance Series.
In the separate 43-page final guidance, “Submitting Patient-Reported Outcome Data in Cancer Clinical Trials,” the FDA provides technical specifications for submitting PRO data from cancer trials used to support a marketing application for an oncological medical product. According to the FDA, the technical specifications are meant to apply to any PRO data used to evaluate safety/tolerability or clinical benefit in randomized oncology trials.
The guidance includes specifications for the Clinical Data Interchange Standards Consortium (CDISC) Study Data Tabulation Model (SDTM), which sponsors are advised to use in support of PRO data tabulation, and the CDISC Analysis Data Model (ADaM), which sponsors should use to support PRO data analyses.
The agency said this final guidance supplements an FDA draft guidance from June 2021, “Core Patient-Reported Outcomes in Cancer Clinical Trials,” as well as the PFDD guidance series.
Read the final guidance, “Submitting Clinical Trial Datasets and Documentation for Clinical Outcome Assessments Using Item Response Theory,” here.
Read the final guidance, “Submitting Patient-Reported Outcome Data in Cancer Clinical Trials,” here.