Table 6.

Safety, Efficacy, and Effectiveness

Safety determines the highest tolerable dose or optimal dose needed to achieve the desired clinical benefit and potential adverse effects in that exposure range.

Efficacy determines whether a drug has a positive clinical benefit over placebo or other intervention. Efficacy tests involve “ideal,” that is, strictly controlled conditions.

Effectiveness describes a drug's clinical benefits in a “real world” situation, for example, where patients may have comorbid conditions or other medications that interact with the drug, and where drug administration may not following strict study guidelines.