Primary Efficacy Variables in Clinical Study Report
ICH E3 section 9.5.3 calls for the primary efficacy variable or variables to be specified and for their role in evaluating efficacy to be clear. This page is limited to the planned primary-efficacy-variable subsection; detailed statistical analysis belongs in the statistical-methods and efficacy-results sections.
ReviewedEvidence1 sourceSectionWriting & Style
Quick answer
Identify the primary efficacy variable or variables and state enough of their planned role to distinguish them from secondary or supportive efficacy measures.
Key details
Core IssueICH E3 section 9.5.3 calls for the primary efficacy variable or variables to be specified and for their role in evaluating efficacy to be clear.
Registerscholarly, professional, or research/report writing
Important caveats
Scope Boundary
This page is limited to the planned primary-efficacy-variable subsection; detailed statistical analysis belongs in the statistical-methods and efficacy-results sections.
Further guidance
Content
Identify the primary efficacy variable or variables and state enough of their planned role to distinguish them from secondary or supportive efficacy measures.
Purpose
Identify the primary efficacy variable or variables and state enough of their planned role to distinguish them from secondary or supportive efficacy measures.
Sources and evidence
Sources are shown with the role they play in this guide. Historical or style-sensitive claims are kept within the evidence boundary described above.
ICH E3 Section 9.5 calls for identification of the efficacy and safety variables assessed, the methods and timing of assessments, and the people or procedures responsible for measurements, often with a schedule or flow chart. This section defines variables and assessment methods; it is not the same artifact as the later efficacy or safety result tables.
ICH E3 section 11.4.7 provides a dedicated place for efficacy conclusions after the detailed efficacy analyses and supporting displays. Do not use the conclusion to overrule the detailed analyses or convert exploratory findings into confirmed primary results.
ICH E3 section 11.4.2.6 calls for efficacy analyses in relevant patient subsets to be presented or discussed when subgroup evidence is important to interpretation. Keep a defined efficacy subset distinct from the primary analysis population and from exploratory subgroup claims that require their own interpretation.
ICH E3 reserves section 14.2 for efficacy tables, figures, and graphs referred to in the report but not included in the main text, allowing detailed displays to support the core efficacy analysis. This page is about supporting ICH E3 efficacy displays, not the broader Efficacy Results section that explains analyses and interprets findings in the report body.
ICH E3 appendix 16.2.6 designates individual efficacy response data as a patient-level listing in the clinical study report supporting material. This is a patient-level listing function, not a substitute for aggregate efficacy tables, statistical analyses, or the broader individual-patient-data appendix overview.
ICH E3 section 12.4.1 points to a by-patient listing of abnormal laboratory values in section 14.3.4 and distinguishes that safety display from the broader listing of individual laboratory measurements. This page is about the dedicated abnormal-value listing under ICH E3 14.3.4; it is distinct from the full individual-measurement appendix under 16.2.8 and from analytical discussion in section 12.4.2.
ICH E3 section 11.4.2.7 identifies special efficacy-analysis considerations for active-control studies intended to support equivalence. This is a CSR efficacy-results reporting issue for active-control equivalence designs; it does not define a universal equivalence margin or replace protocol-level design justification.
ICH E3 section 11.4.2.1 calls for covariate or prognostic-factor selection and adjustment to be explained, including adjustment methods, analysis results, supportive statistical documentation, and differences from planned analyses when relevant. This results-stage statistical issue is narrower than the general statistical-methods section and should not be used as a substitute for documenting the full analysis plan.
