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An efficacy endpoint is an outcome that is tested during the course of a clinical trial. This assessment is based on a pre-specified event or measure that is expected to change over time. There is a difference between events and measures. An event is something that happens, e.g. losing the ability to walk independently.
Measures are scores given to certain activities. For example, timed function tests measure how fast a child runs/walks a certain distance, how fast he gets off the floor, how fast he can take four steps, etc. Because the natural history (progression) of Duchenne is pretty well understood, researchers at different study sites can test functions over time using testers who do things in the same way (called inter-rater reliability) and end up with quantitative data whether there was improvement.
Types of Clinical-Benefit Efficacy Endpoints
There are two main types of efficacy endpoints: clinically meaningful and surrogate.
There is also an accelerated approval surrogate endpoint that applies to Duchenne. This is a surrogate endpoint that is considered reasonably likely to predict clinical benefit. The surrogate endpoint, combined with primary and secondary endpoints, are used to obtain conditional approval of a drug for a serious or life threatening condition where there is currently little to no treatment. One example of this is the CK blood test, which measures creatine kinase, an enzyme that normally lives inside muscles. Boys with Duchenne often have CK levels 10 to 100 times the normal range. Elevated CK levels indicate muscle damage, although a high CK does not confirm a diagnosis of Duchenne.
What is the ideal event or measure of drug efficacy?
Ideal events or measures have four basic criteria:
What are some of the endpoints (with events or measures) associated with Duchenne?
Learn more about How We Know When Something Works.
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