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Outcomes and outcome measurement instruments1115Box 1. Definitions and abbreviations of commonly used terminology with regard to outcomes and outcome measurement instruments, adapted from the Food and Drug Administration (*).1–4Definition Abbreviation ExplanationClinical outcome assessment*COA A clinical outcome assessment describes or reflects how a person feels, functions, or survives and can be reported by the affected individual, a non-clinical observer (such as parent), a health care provider, or through performance of an activity or task.Outcome An outcome refers to a construct or domain. In the context of a clinical trial, it refers to what is being measured on trial participants to examine the effect of exposure to a health intervention (e.g., anxiety). Outcome measurement instrumentAn outcome measurement instrument specifically refers to how the outcome is being measured. It is a tool to measure a quality or quantity of the outcome. It can be used to identify meaningful change for the individual, evaluate the effect of interventions, demonstrate the impact and value of interventions, identify areas for improvement, and benchmark against other interventions. Power calculations are often based on the chosen primary outcome measure. In literature, the term outcome measure has often been inconsistently and interchangeably used to refer to both the outcome and outcome measurement instrument; we consider using ‘outcome measure’ as an abbreviation of ‘outcome measure instrument’. Patient-reported outcomePRO A type of clinical outcome assessment, based on a report that comes directly from the affected individual about the status of the health condition.Patient-reported outcome measure*PROM Instrument or tool utilized to measure PROs to evaluate the affected individuals’ health status from their perspective. For individuals with an intellectual disability who are not able to complete a measure, a PROM can also be a proxyreport provided that it is someone who knows the affected individual well and fills out the PROM from the affected individual’s perspective. Clinician-reported outcome*ClinRO A type of clinical outcome assessment, based on a report that comes from a trained health care professional after observation of a patient’s health condition.Performance outcome*PerfO A type of clinical outcome assessment, based on standardized task(s) actively undertaken by an affected individual according to instructions. Observer-reported outcome*ObsRO A type of clinical outcome assessment, based on a report of observable signs, events or behaviors related to an affected individual’s health condition by someone other than the affected individual or a health care professional, such as a parent, teacher or caregiver.Proxy Someone who reports an outcome as if they were the affected individual themselves. Proxies report on behalf of the affected individual, in contrast to an observer-report in which the informant provides information about the manifestations and condition. Generic outcome measureA measure for a health concept that is relevant to a wide range of patient groups, enabling aggregation and comparison across varied conditions and settings.Annelieke Muller sHL.indd 111 14-11-2023 09:07