RSNN Annual Workshop 2025: One arm, many challenges: the role of single-arm trials in regulatory and clinical context
Villa Jongerius, Utrecht
Single-arm trials
A single-arm trial (SAT) is a type of clinical study where all participants receive the same treatment or intervention. Unlike randomized controlled trials (RCTs), which include both a treatment group and a control group, SATs do not have a comparison group. The RCT is often considered the gold standard for evidence in later stage medicine development. SATs have advantages over RCTs especially in situations where controlled trials are impractical or unethical.
These advantages also come with certain challenges, such as the lack of comparative data which may limit the interpretation of clinical evidence, potential biases may be introduced due to the absence of randomisation and an increased need to collect post-launch real-world evidence to address such issues.
These challenges can be addressed by, for example, guidance on the use of external and synthetic control arms, early involvement of HTA, tailored evidence generation models for HTA (e.g. combining real-world studies and registries) linked to specific reimbursement models, improved statistical designs.
Aim of the workshop
In this workshop, we want to explore the current developments around SATs, their promises, and impact across the value chain. Questions that will be addressed during the workshop include:
- What are key features of SATs, and what are methodological and practical considerations?
- How can results of SATs be contextualised?
- How can challenges arising from SATs in marketing authorisation, HTA and clinical practice be addressed?
- What is on the horizon for SATs?
Participants will understand the current landscape of SATs and how ongoing developments will influence their future application throughout the drug development pathway.