A confirmatory strategy for therapeutic equivalence trials

Area of Science:

• Clinical Trials
• Biostatistics
• Pharmacoeconomics

Background:

• Therapeutic equivalence trials require defining a parameter range for practical treatment equality.
• Current methods for equivalence assessment are limited, especially when a new drug proves superior to the standard.

Purpose of the Study:

• To present an alternative approach for therapeutic equivalence trials that allows flexible handling of equivalence and superiority testing.
• To enable testing for superiority alongside equivalence within the same trial framework.

Main Methods:

• Development of a novel statistical approach for hypothesis testing in equivalence trials.
• Utilizing a special hypothesis structure to avoid alpha-adjustments for additional confirmatory analyses.

Main Results:

• The proposed method allows for testing therapeutic equivalence.
• If equivalence is established, the method permits testing all null hypotheses for acceptable equivalence ranges and superiority.
• No alpha-adjustment is required for these additional confirmatory analyses due to the hypothesis structure.

Conclusions:

• The presented approach offers a more flexible and informative method for therapeutic equivalence trials.
• This method enhances the ability to assess drug performance comprehensively, including superiority, while maintaining statistical integrity.