Biostatistics

The European Medicines Agency's scientific guidelines on biostatistics help medicine developers prepare marketing authorisation applications for human medicines.

For a complete list of scientific guidelines currently open for consultation, see Public consultations.

Guidelines

• Adjustment for baseline covariates in clinical trials
• Choice of a non-inferiority margin
• Clinical trials in small populations
• Data monitoring committees
• Investigation of subgroups in confirmatory clinical trials
• Missing data in confirmatory clinical trials

Points to consider

• Impact of the war in Ukraine on methodological aspects of ongoing clinical trials
• Implications of coronavirus disease (COVID-19) on methodological aspects of ongoing clinical trials
• Application with 1. Meta-analyses; 2. One pivotal study
• Multiplicity issues in clinical trials
• Switching between superiority and non-inferiority

Reflection papers

• Establishing efficacy based on single-arm trials submitted as pivotal evidence in a marketing authorisation
• Extrapolation of efficacy and safety in medicine development
• Methodological issues in confirmatory clinical trials planned with an adaptive design
• Need for active control in therapeutic areas where use of placebo is deemed ethical and one or more established medicines are available
• Statistical methodology for the comparative assessment of quality attributes in drug development

Concept papers

• Platform trials
• Non-inferiority and equivalence comparisons in clinical trials

ICH guidelines

• ICH E9 statistical principles for clinical trials
• ICH E10 Choice of control group in clinical trials

Questions and answers

• Adjustment for cross-over in estimating effects in oncology trials
• Data Monitoring Committees issues
• The adequacy of the Mahalanobis distance to assess the comparability of drug dissolution profiles