Toolkit Outline
Analyse
The subsection below includes two parts. The subsection opens with an explanation of the subject and guidance on how to address it. This is followed by a list of tools that have been identified in relation to this subsection. The tools listed may not be unique to clinical trials in rare diseases, this is specified in the table to better understand the use of the tool. You can visit the different section by clicking on the buttons above or return to the full toolkit here.
STATISTICAL ANALYSIS PLAN (SAP)
The SAP is intended to be a comprehensive document that contains a detailed and technical description of the principal features of the analysis outlined in the protocol. It also includes detailed procedures for executing the statistical analysis of the primary and secondary endpoints and other data. As compared to the protocol, the SAP should contain an in-depth description of the statistical methods to be used and a definition of the statistical output which will be included in the clinical study report.
The SAP is generally developed as a separate document and written after the protocol has been finalised. Ideally, a biostatistician should develop the SAP with the help of the principal investigator and in alignment with the protocol. The SAP should be finalised prior to data analysis and before treatment un-blinding. Any changes between the methods in the protocol and analysis plan, should be explained in the SAP. The SAP must include a brief summary of the trial, which includes the aims and objectives, trial population, design of the trial, sample size calculations (including the justifications and assumptions), and the randomisation method.
The SAP must properly explain the statistical analysis following the aims and primary objectives, secondary objective, exploratory objectives, primary/secondary/exploratory endpoints, trial population, design of the trial, sample size calculations with justifications/assumptions and the randomization methods. Additionally, an SAP must describe in detail the statistical methodology i.e. efficacy analysis, safety data analysis, interim analyses, handling of missing data, reporting conventions, etc
| Tool Name | Relevance | Year | RD/ Paediatric specific | Type |
| ICH Statistical Principles for clinical trials E9 | This guidance is intended to give direction to sponsors in the design, conduct, analysis, and evaluation of clinical trials of an investigational product in the context of its overall clinical development. The document will also assist scientific experts charged with preparing application summaries or assessing evidence of efficacy and safety, principally from clinical trials in later phases of development. | 1998 | No | Guideline |
| Guideline for the Content of Statistical Analysis Plans in Clinical Trials | A checklist of 32 minimum items for inclusion in SAPs that was developed with the primary intention of being applicable to the final analyses of later-phase randomized clinical trials addressing the minimum recommended content of a SAP | 2017 | No | Guideline |
| ECRIN Metadata Repository | Search of metadata (including protocols and Statistical Analysis Plans) on published clinical trials. | 2020 | No | Repository |
| Clinical Trials Registry (NIH) | Database of publicly and privately funded clinical studies conducted around the world. The Study Documents tab allows search for SAP (Statistical Analysis Plan), protocols and Informed consents | Regular updates | No | Registry |