Federal Drug Administration Qualification
The US Food and Drug Administration (FDA) recognizes the importance of research tools that aid the development and evaluation of medical treatments. These tools include performance measures and patient-reported outcome (PRO) instruments that may serve as clinical outcome assessments (COAs) in clinical trials. To expedite the use of a specific PRO instrument as an outcome in clinical trials, a person or group may submit the PRO for qualification by a center of the FDA, e.g., the Center for Drug Evaluation and Research (CDER). Once a PRO has been qualified, FDA reviewers can be confident that it is valid for its specific context of use, e.g., assessing the effect of treatment relative to placebo on fatigue in children with Crohn’s disease aged 8-17 years old. Hence, the PRO may be employed in drug development trials within the context of use without the need for further review of its validity.
Qualification is a multi-step process that requires substantial qualitative and quantitative evidence to support the reliability, validity, and interpretability of the PRO for its intended use. The current process for qualification by CDER involves (1) submission of a Letter of Intent that describes the PRO and preliminary supporting evidence, (2) submission of a Qualification Plan that provides detailed information about qualitative research conducted with the intended patient population and plans for further research, and (3) submission of a Full Qualification Package with data and results supporting the PRO as well as instructions for administration, scoring, handling missing data, and interpreting scores. At each stage, FDA reviewers provide feedback and may request revisions and additional information. Learn more here.
As of March 2020, the FDA had approved a Letter of Intent for 3 measures developed using PEPR data:
PROMIS Pediatric Crohn’s Disease Short Form – Fatigue
PROMIS Pediatric Chronic Kidney Disease Short Form – Fatigue
PROMIS Pediatric Chronic Kidney Disease Short Form – Sleep Disturbance
For the current status of all COA submissions, visit the FDA’s COA webpage.