Following a public consultation, the Methodology Working Party (MWP) has finalised the Reflection paper on establishing efficacy based on single-arm trials submitted as pivotal evidence in a marketing authorisation.
The reflection paper discusses key issues and requirements for single-arm clinical trials that are submitted as pivotal evidence in support of marketing authorisation applications for medicines in the European Union (EU).
During the public consultation phase comments from 45 stakeholders were received. The comments were taken into careful consideration and have resulted in the following major changes compared to the draft version:
- The reflection paper includes a clearer statement that as a regulatory standard, randomised controlled evidence is expected and that deviation from this standard by proposing a SAT needs justification.
- The scope of the reflection paper has been clarified to exclude guidance on the use of external controls. This led to a rephrased wording in the introductory section and the removal of the section on the role of external information and external-control focused biases from table 1…
