SOME OBSERVATIONS ON THE MAKUCH/SIMON APPROACH TO SAMPLE SIZE DETERMINATION IN CLINICAL TRIALS WITH HISTORICAL CONTROLS

Abstract

The widely implemented Makuch and Simon method for using historical control data to determine sample size for efficacy studies is carefully explained. An extensive Monte Carlo study of the method clearly delineates its performance characteristics. A second Monte Carlo study describes the poor overall performance achieved when the Makuch and Simon method is used with the natural two-sample statistic for detecting a difference in proportions and results in the unequivocal recommendation that the composition of the angular and square root transformations be used in the context of the historical control problem.

Keywords:

• Monte Carlo
• Simulations

Acknowledgments