Abstract
In addition to having an acceptable safety profile, an investigational drug needs to display beneficial therapeutic effects for it to receive marketing approval, that is, it needs to have a favorable benefit–risk profile. Therapeutic benefit, operationalized as efficacy, is evaluated in therapeutic exploratory and therapeutic confirmatory trials, trials in which the subjects have the disease or condition of biological concern.
In addition to having an acceptable safety profile, an investigational drug needs to display beneficial therapeutic effects for it to receive marketing approval.
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Turner, J.R. (2011). Efficacy Assessment. In: New Drug Development. Springer, New York, NY. https://doi.org/10.1007/978-1-4419-6418-2_13
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DOI: https://doi.org/10.1007/978-1-4419-6418-2_13
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