Trials in rare diseases: the need to think differently
نویسندگان
چکیده
Background In general, large-scale clinical trials are needed to make definitive conclusions regarding changes in treatment practice. This is because treatment effects are often modest and large numbers of patients are needed to detect such differences with sufficient power for robust conclusions. Such trials are difficult and often impossible in rare diseases and their worth in this setting could be questioned and this drives the need to consider a different approach.
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