By Elisabeth M. Oehrlein, PhD, MS, Senior Director, Research and Programs
The NHC’s clinical outcome assessment (COA) webinar series is designed to prepare patient audiences to engage in conversations about COAs. Our third webinar, Untangling the Terms: Endpoints, Items, Outcomes, PROs, PROMS, PRO PMs, featured Dr. Ashley F. Slagle, Principal at Aspen Consulting. Dr. Slagle provides strategic regulatory and scientific advice to drug product developers on matters related to patient-focused drug development, with a particular focus on patient-relevant endpoints and Clinical Outcome Assessments (COAs). In this webinar, Dr. Slagle introduced formal definitions of COA terminology and interpreted them for patient audiences.
Below are key terms and definitions from the webinar.
- COAs: Clinical outcome assessments are measures that tell you how a patient feels or functions.
- Patient Reported Outcome: A report that comes from a patient about the status of that patient’s health condition without any changes or interpretation of the patient’s report by a clinician or anyone else. The patient’s view of their own health.
- Clinician Reported Outcome: A report that comes from a trained health care professional after observation of a patient’s health condition. The clinician’s view of the patient’s health.
- Observer-Reported Outcome: A report that comes from someone other than the patient or a health professional on the patient’s health. This may be a parent, spouse, or non-clinician caregiver who is in a position to regularly observe and report on a specific aspect of the patient’s health.
- Performance Outcome: A report on a task performed by a patient according to instructions provided by someone trained to observe the patient perform the task. The observer must have training, but does not need to be a health care professional.
- Concept of interest (COI): The actual “thing” we are interested in measuring, such as pain intensity.
- Endpoint: A precise way to describe how we will collect data on the outcome of interest to see if the intervention had an effect.
- Test, Tool, Instruments: These terms are used interchangeably and refer to the questionnaire that will be used to measure the concept of interest. Instruments must have accompanying materials to tell us how to implement the instrument as well as guidelines for helping us to understand what score changes mean.
- Item: A question and the response choices for that question that are used in a COA questionnaire. For example: a survey question and the possible responses.
- Measurement: The value or “score” that results from using a test, tool, or instrument. With patient-reported outcomes, measurements are usually a score.
- Outcome: The outcome is the change in a patient’s health status we are trying to measure, such as pain.