Critical Review of Trial Results

Site: EUPATI Open Classroom
Course: Interpretation and Dissemination of Results
Book: Critical Review of Trial Results
Printed by: Guest user
Date: Thursday, 13 June 2024, 12:43 PM

1. What is a clinical study report?

(This section is organised in the form of a book, please follow the blue arrows to navigate through the book or by following the navigation panel on the right side of the page.)

The clinical study report (CSR) is the formal document describing the results of a clinical trial. It therefore represents a fundamental building block in the argument for use of the treatment in humans. The results of all clinical trials conducted in humans must be recorded in a clinical study report.
The E3 Guideline defines the type of report to be used for most clinical trials conducted in three major geographic regions (Europe, Japan, and the United States) (link).
This report usually runs to several hundred pages. Clinical study reports are prepared by the trial sponsor and forwarded to regulatory authorities.
Access to such reports or even summary data will be possible in the future via the EU database: here.

2. Why Access To Clinical Trial Results Is Important?

The concept of evidence-based medicine (EBM) was created in the early 1980s as clinical practice became more data-driven and literature based. EBM is now an essential part of what is taught at medical school. Information on treatment efficacy and safety is important for making informed treatment decisions by the patients and their physicians.

It is important not to limit the search for evidence about a treatment to a single publication. When comparing results coming from different sources, it is important to keep in mind the different levels of evidence. The hierarchy of evidence relates to the strength of the trial design and statistics deployed and not necessarily to the clinical significance. Randomised, controlled, blinded trials provide the best scientific evidence of benefit and risk but sometimes are not available. Meta-analysis can be flawed because of publication bias.

In general, the hierarchy of trials for obtaining evidence is:

Highest level

Adequately powered, randomised controlled trial, or meta-analysis of randomised trials showing statistically consistent results. 

Randomised trials inadequately powered, possibly biased, or showing statistically inconsistent results. 

Non-randomised trials with concurrent controls. 

Non-randomised trials with historical controls (i.e. typical single arm Phase II trial). 

Bottom level

Expert committee review, case reports, retrospective trials. 

Patients can learn about clinical trial results from their doctor and also by accessing the published information directly. Access to clinical trial results for researchers is paramount for improving the efficiency in research by reducing the duplication or replication of research efforts.