The European Medicines Agency (EMA) is seeking industry’s thoughts on a new draft guidance on general considerations and challenges for single-arm trials that serve as the key source of efficacy data.

Randomized controlled trials are generally the best route for reliably evaluating efficacy, EMA says, but in certain circumstances, data from single-arm trials is acceptable.

“In certain areas, such as rare diseases, including rare cancers, where target populations of new medicines are often very small, a proportion of marketing authorization applications are submitted to EMA with clinical data from single-arm trials as pivotal evidence,” the agency noted.

The document offers general considerations for single-arm trial designs, covering endpoint selection, trial population, the role of external information, statistical considerations, sources of bias and ways to mitigate bias.

Interested parties should email comments to RP-SATs@ema.europa.eu using a template included within the guidance. The EMA intends to publish the final version of the reflection paper some time in 2024. Comments are due by Sept. 30.

Read the guidance here: https://bit.ly/3H4io67.