A new reflection paper and a consultation open up to 30 September 2023 will pave the way to the drafting of a new guideline on single-arm clinical trials submitted to EMA as pivotal evidence in support of marketing authorisation (MA) applications.

The reflection paper published by the European Medicines Agency is the first of this type at the international level. It has been adopted by EMA’s CHMP committee with contributions from the CAT Committee, the Methodology Working Party and the Oncology Working Party. The publication of the final document is planned for 2024.

Single-arm clinical trials are used whereas experimentation occurs in therapeutic areas characterised by small target patients populations, i.e. in rare diseases and rare cancers. In such instances, it often result impossible to enrol a sufficient number of patients to run randomised clinical trials. A proportion of the MA applications are thus submitted to EMA with clinical data from single-arm trials as pivotal evidence. These are trials where there is no randomised comparator, all enrolled patients receive the experimental treatment and only the outcomes under the experimental treatment can be observed.

EMA’s reflection paper outlines considerations on single-arm trials submitted as pivotal evidence to demonstrate efficacy in a MA application. Comments collected from the consultation phase will support the drafting of the new guideline.

Source: EMA