Improving the analysis of composite endpoints in rare disease trials

Martina McMenami, Anna Berglind and James M. S. Wason; Orphanet Journal of Rare Diseases 201813:81 doi:10.1186/s13023-018-0819-1

In rare diseases where there are few or no available treatments and limited opportunity to test emerging new treatments, the power to detect an effective treatment is of critical importance. The augmented binary method with small sample adjustments offers a substantial improvement for trials in these populations over methods currently being used, which throw away valuable information. We recommend the use of the augmented binary method in relevant rare disease trials using composite endpoints and supply R code to assist with the implementation.

 Improving the analysis of composite endpoints in rare disease trials