Clinical Research Endpoint
A Clinical Research Endpoint is a clinical outcome measure used in a clinical trial to determine the efficacy and safety of the therapy being studied.
- Context:
- It can (typically) be statistically analyzed to help determine the efficacy and safety of the therapy being studied.
- It can (often) refer to a Disease Occurrence, Symptom Occurence, Medical Sign, or a Laboratory Abnormality.
- It can (often) be referenced in a Clinical Trial Protocol.
- It can range from being a Primary Clinical Endpoint to being a Secondary Clinical Endpoint to being an Exploratory Endpoint.
- …
- Example(s):
- disease relapse, after a surgery (intended to treat the disease).
- chest pain, in a myocardial infarction medication intervention.
- tumor shrinkage, in an oncological clinical trial.
- blood pressure (above a certain threshold), in an hypertension trial.
- a change from baseline at 6 weeks in mean PROMIS Fatigue score (for a PROMIS Fatigue score).
- Clinical Protocol: DS8500-A-U202[1].
- The primary efficacy endpoint is change from baseline in HbA1c at Week 12.
- The secondary endpoints are:
- Changes from baseline in lipids (TC, LDL-C, HDL-C, non-HDL-C and TG) at Week 12.
- …
- Exploratory endpoints are: ...
- …
- Counter-Example(s):
- See: Medical Sign, Clinical Trial, Overall Response Rate (ORR).
References
2021
- (Wikipedia, 2021) ⇒ https://en.wikipedia.org/wiki/Clinical_endpoint Retrieved:2021-11-8.
- Clinical endpoints or clinical outcomes are outcome measures referring to occurrence of disease, symptom, sign or laboratory abnormality constituting a target outcome in clinical research trials. The term may also refer to any disease or sign that strongly motivates withdrawal of an individual or entity from the trial, then often termed a humane (clinical) endpoint.
The primary endpoint of a clinical trial is the endpoint for which the trial is powered. Secondary endpoints are additional endpoints, preferably also pre-specified, for which the trial may not be powered.
Surrogate endpoints are trial endpoints that have outcomes that substitute for a clinical endpoint, often because studying the clinical endpoint is difficult, for example using an increase in blood pressure as a surrogate for death by cardiovascular disease, where strong evidence of a causal link exists.
- Clinical endpoints or clinical outcomes are outcome measures referring to occurrence of disease, symptom, sign or laboratory abnormality constituting a target outcome in clinical research trials. The term may also refer to any disease or sign that strongly motivates withdrawal of an individual or entity from the trial, then often termed a humane (clinical) endpoint.
2021
- (Wikipedia, 2021) ⇒ https://en.wikipedia.org/wiki/Clinical_endpoint#Scope Retrieved:2021-11-15.
- In a general sense, a clinical endpoint is included in the entities of interest in a trial. The results of a clinical trial generally indicate the number of people enrolled who reached the pre-determined clinical endpoint during the study interval compared with the overall number of people who were enrolled. Once a patient reaches the endpoint, he or she is generally excluded from further experimental intervention (the origin of the term endpoint).
For example, a clinical trial investigating the ability of a medication to prevent heart attack might use chest pain as a clinical endpoint. Any patient enrolled in the trial who develops chest pain over the course of the trial, then, would be counted as having reached that clinical endpoint. The results would ultimately reflect the fraction of patients who reached the endpoint of having developed chest pain, compared with the overall number of people enrolled.
When an experiment involves a control group, the proportion of individuals who reach the clinical endpoint after an intervention is compared with the proportion of individuals in the control group who reached the same clinical endpoint, reflecting the ability of the intervention to prevent the endpoint in question.
A clinical trial will usually define or specify a primary endpoint as a measure that will be considered success of the therapy being trialled (e.g. in justifying a marketing approval). The primary endpoint might be a statistically significant improvement in overall survival (OS). A trial might also define one or more secondary endpoints such as progression-free-survival (PFS) that will be measured and are expected to be met. A trial might also define exploratory endpoints that are less likely to be met.
- In a general sense, a clinical endpoint is included in the entities of interest in a trial. The results of a clinical trial generally indicate the number of people enrolled who reached the pre-determined clinical endpoint during the study interval compared with the overall number of people who were enrolled. Once a patient reaches the endpoint, he or she is generally excluded from further experimental intervention (the origin of the term endpoint).
2020
- https://registries.ncats.nih.gov/glossary/endpoint/
- QUOTE: ... An endpoint is a targeted outcome of a clinical trial that is statistically analyzed to help determine the efficacy and safety of the therapy being studied. Endpoints for a clincial trial may include one or more clinical outcome assessment and/or surrogate endpoint. Clinical outcomes assessments measure direct clinical benefit to the participant where as surrogate endpoints, including biomarkers, predict clinical benefit. Endpoints may also be used to throughout a study to determine if a participant’s risk of continuing to be in a study is too great.
Endpoints must be validated prior to using in a clinical trial. Endpoint choice depends on the clinical trial design, the nature of the disorder or condition being treated, and the expected effect of the therapy being tested. Chosen endpoints are usually clearly defined prior to the start of a clinical trial. A clinical study may have one or more primary, secondary, and exploratory endpoints. Primary endpoints will be the basis for determining whether the study met its objective or, in the case of interventional clincal trials, will be the main data evaluated for regulatory approval. Secondary endpoints are those that may provide supportive information about a therapy’s effect on the primary endpoint or demonstrate additional effects on the disease or condition. Exploratory endpoints may include clinically important events that are expected to occur too infrequently to show a treatment effect or endpoints that for other reasons are thought to be less likely to show an effect but are included to explore new hypotheses. ...
- QUOTE: ... An endpoint is a targeted outcome of a clinical trial that is statistically analyzed to help determine the efficacy and safety of the therapy being studied. Endpoints for a clincial trial may include one or more clinical outcome assessment and/or surrogate endpoint. Clinical outcomes assessments measure direct clinical benefit to the participant where as surrogate endpoints, including biomarkers, predict clinical benefit. Endpoints may also be used to throughout a study to determine if a participant’s risk of continuing to be in a study is too great.
2020
- https://rethinkingclinicaltrials.org/chapters/design/choosing-specifying-end-points-outcomes/choosing-and-specifying-endpoints-and-outcomes-introduction/
- QUOTE: ... For an explanatory trial, investigators can specify any outcome or endpoint, define the endpoint, and then measure it. The term outcome usually refers to the measured variable (eg, peak volume of oxygen or PROMIS Fatigue score), whereas an endpoint refers to the analyzed parameter (eg, change from baseline at 6 weeks in mean PROMIS Fatigue score). Even after a specific outcome is selected, it may be challenging to determine the best way to measure the effect of an intervention in terms of an analyzable endpoint, especially with pragmatic research where data are collected as part of routine care.
With pragmatic research, the endpoints and outcomes need to be available as part of routine care. Although the research question regarding the relative risks, benefits, and burdens of a specific intervention or activity will drive the selection of endpoints and outcomes, in a PCT, the selection must be balanced with an understanding of what is available in the electronic health record (EHR) or claims data and what additional resources will be needed to capture information not found in these sources. ...
- Defining endpoints and outcomes for some health phenomena is relatively easy for things like
- acute myocardial infarction
- broken bone
- hospitalization
- However, many outcomes are not routinely recorded as part of healthcare delivery. For example:
- QUOTE: ... For an explanatory trial, investigators can specify any outcome or endpoint, define the endpoint, and then measure it. The term outcome usually refers to the measured variable (eg, peak volume of oxygen or PROMIS Fatigue score), whereas an endpoint refers to the analyzed parameter (eg, change from baseline at 6 weeks in mean PROMIS Fatigue score). Even after a specific outcome is selected, it may be challenging to determine the best way to measure the effect of an intervention in terms of an analyzable endpoint, especially with pragmatic research where data are collected as part of routine care.