EMA opens consultation on single-arm trials

The European Medicines Agency (EMA) has opened a public consultation on a reflection paper that discusses key concepts for single-arm clinical trials submitted as pivotal evidence in support of marketing authorization applications.

This is the first guidance document by an international medicine regulator on the considerations and challenges associated with this type of clinical trial. Views are invited via an online form by midnight on September 30.

It tends to be in areas such as rare diseases, including rare cancers, where target populations of new medicines are often very small, that a proportion of marketing authorization applications are submitted to the EMA with clinical data from single-arm trials as pivotal evidence.