Combined-Indications Significance Level of Multiple Related Indications Developed Simultaneously.
BACKGROUND
Typically, regulatory requirements include 2 confirmatory studies, each at a 1-sided .025 significance level, for a medicine to be approved for a specific indication. When the same medicine has been approved in related indications, 1 confirmatory study at a 1-sided .025 significance level could constitute adequate evidence of efficacy for a new indication.


METHODS
This article does not contain any studies with human or animal subjects performed by any of the authors. For multiple related indications developed simultaneously to constitute sufficient evidence of clinical efficacy, the combined-studies significance level can be set at the same level as if those indications are developed sequentially.


RESULTS
This article establishes possible strategies to develop a few related indications at the same time for marketing registration approval, maintaining a desired combined-studies significance level; for example, 1-sided .0000156 for 2 indications, with 1 option having each indication assessed with 1 confirmatory study at .00395 significance level.


CONCLUSION
It is possible to develop a few indications at the same time for marketing registration approval, where the combinedstudies significance level is less stringent than that of the usual paradigm with 2 confirmatory studies each at 1-sided .025 significance level for every indication.
['Yuan|Jiacheng|J|0000-0002-2762-242X', 'Zhu|Ray|R|', 'Jia|Daniel|D|', 'Palm|Ulo|U|', 'Koch|Gary|G|']
D000818:Animals D017277:Drug Approval D006801:Humans D040541:Marketing
confirmatory studies combined-studies significance level combined-studies evidence strength borrowing