Randomized Clinical Trials, Analysis Sets, Statistical Analysis, Reporting Issues

Abstract

This chapter addresses types of analysis sets, including the intention to treat analysis (ITT), as well as the per protocol (PP) analysis. Second, statistical principles required for data analysis will be reviewed. It is based on statistical reasoning consisting of (1) statistical estimation, (2) statistical hypothesis testing, and (3) statistical modeling. Other issues include: – stratification, baseline covariates, – missing values, withdrawals, drop-outs (often a PP analysis uses the last observation carried forward principle (LOCF)), – safety & tolerability issues: often analyzed in subgroups with special populations (age, gender, comedication groups). Third, the CONSORT statement (consolidated standards of randomized trials statement), and, fourth, reporting issues, like those of reporting bias are subjects of this chapter. Finally, as special points are mentioned the rules for the SAP (statistical analysis plan), a (blinded) principal features analysis, International Conference of Harmonization (ICH) guidelines, outcome adjustments, routine use of check lists before data lock, the handling of missing data, and the Cochrane risk-of-bias tool.