Single-arm trials (SATs) have led to regulatory approval of oncology products in case of dramatic activity in well-defined populations with high unmet medical need. Further, when randomised-controlled trials are not possible, SATs augmented with statistical approaches may become the standard basis of evidence of efficacy for new applications.
However, what constitutes dramatic activity and high unmet medical need is often debated and there is no regulatory guidance on cancer drug development in these situations and how to manage the remaining uncertainties optimally.
This workshop, organised by the European Medicines Agency (EMA) in partnership with ESMO, debated the experience gained with these types of approvals, the strengths and weaknesses of different approaches, explored the views of different stakeholders and underlying principles, and discussed the need and appropriateness for further regulatory guidance.
View the slide presentations from the workshop
F. Pignatti (EMA) and P. Casali (ESMO)