TABLE 3.

Definition of a pivotal clinical study.

List of countries or regions	Definition of pivotal clinical study
US	- A human clinical trial intended to be a pivotal trial for obtaining regulatory approval or - Any other clinical trial to confirm the safety and efficacy of the drug’s intended use.
EU	- No official definition for pivotal trials; however, they are consistent with the definition in US and Japan.
Japan	- A critical clinical study for efficacy evaluation among submitted studies categorized as “major sources for evaluation” (“HYOUKA SIRYO” in Japanese), as described in the PMDA review reports. If a clinical study was mentioned as a “reference” (“SANKO SIRYO” in Japanese) in a review report, the study was not classified as a pivotal clinical study (8).
China	- No official definition for pivotal trials; however, they are consistent with the definition in US and Japan