In a single-arm trial every patient enrolled receives the new medicine. These trials have no separate arm (or group) in which patients are given a placebo or another medicine to allow comparison. There is no specific regulatory guidance on cancer medicine development based on such trials and how to manage the remaining uncertainties optimally.
The workshop will bring together regulators, clinicians, academics, patients, medicine developers and health technology assessment specialists, to discuss situations in which the marketing authorization application for a cancer medicine could be based on results from a single-arm trial.
Workshop participants will discuss experience gained so far with marketing authorizations based on single-arm trials data, the strengths and weaknesses of different approaches, and opportunities from data sharing initiatives.
Situations in which a medicine shows significant anti-cancer activity in an area where patients have no treatment option or where the conduct of standard trials with a comparative arm is difficult, such as in rare cancers or selected populations for example, will be considered.
They will also explore the views of various stakeholders groups and discuss the need for further regulatory guidance with the ultimate aim of optimizing the development of new cancer treatments in these situations.
The workshop will take place at the EMA premises in London, U.K., and will be broadcast live.