Topic

Clinical and patient-reported outcomes

Clinical outcomes record what happens to patients in trials and in practice, such as overall survival and progression-free survival in cancer studies. Patient-reported outcomes describe symptoms and function as patients themselves report them, including activities of daily living. These measures supply the effectiveness evidence that economic models later convert into life years and QALYs.

Concepts in this topic

• Acquired Treatment ResistanceAcquired treatment resistance is the loss of a cancer treatment's effect after initial response or disease control, as the tumour adapts and progresses.
• Activities of Daily LivingActivities of daily living (ADL) are basic self-care tasks, such as bathing, dressing, toileting and eating, used to measure dependence and need for care.
• Activity LimitationActivity limitation is a difficulty a person has in carrying out a task or action, such as walking, washing or communicating, as defined in the WHO ICF.
• Biomarker EndpointA biomarker endpoint is a trial outcome built on a measured biological marker, such as LDL cholesterol, which cost-utility models must convert into QALYs.
• Clinical BenefitA favourable effect of an intervention on how a patient feels, functions or survives relative to an appropriate comparator.
• Clinical Benefit RateClinical benefit rate (CBR) is the share of cancer trial patients with a complete or partial response, or stable disease lasting a set minimum time.
• Clinical Outcome AssessmentA general term for any measure of how a patient feels, functions, or survives, including patient, clinician, and observer-reported outcomes.
• Disease Control RateA cancer outcome measure combining complete response, partial response, and stable disease rates, characterising the proportion whose disease did not progress.
• Event-Free SurvivalEvent-free survival is the time from a specified starting point to the first event in a prespecified endpoint, with incomplete event-free follow-up handled according to the study's censoring rules.
• Minimal Residual DiseaseThe small amount of disease still detectable using highly sensitive techniques after treatment appears, by less sensitive measures, to have achieved complete response.
• Objective Response RateObjective response rate is the proportion of participants in a prespecified analysis population who achieve a complete or partial tumor response according to defined response criteria during the assessment period, with any confirmation requirement stated.
• Overall SurvivalOverall survival is the time from a prespecified origin, such as randomization, diagnosis, or treatment initiation, until death from any cause, with participants not known to have died censored at their last date known alive.
• Patient OutcomeA measurable change in health, function, symptoms, quality of life or survival experienced by a patient.
• Patient-Reported OutcomeAny report of a patient's health status that comes directly from the patient without clinical interpretation, from symptom ratings to full quality of life instruments.
• Progression-Free SurvivalProgression-free survival is the time from a prespecified origin, usually randomisation or treatment initiation, until objectively defined disease progression or death from any cause, whichever occurs first.
• Relapse-Free SurvivalA time-to-event measure defined as the time from treatment completion until disease recurrence or death, used for curative-intent treatments.
• Symptom-Free SurvivalA time-to-event measure defined as the time from treatment start until the onset or return of clinically significant symptoms.