The European Medicines Agency (EMA) and the European Society for Medical Oncology (ESMO) are co-organizing a workshop on the role of single-arm trials in the approval of new cancer medicines on 30 June 2016.
There is no specific regulatory guidance on cancer medicines development based on such trials and how to manage the remaining uncertainties optimally. In a single-arm trial every patient registered receives the new medicine. These trials have no detached group in which patients are given a placebo or another medicine to permit evaluation.
The workshop will give a platform to regulators, clinicians, academics, patients, medicine developers and health technology assessment specialists to discuss:
- Situations in which the marketing approval application for a cancer medicine could be based on results from a single-arm trial.
- Experience achieved with marketing authorizations based on single-arm trials data, the potency and shortcomings of different perspectives, and chances from data sharing proposals.
- Circumstances in which a medicine shows considerable anti-cancer activity in an area where patients have no treatment option or where the demeanor of standard trials with a relative arm is difficult, such as in rare cancers or selected populations for example, will be measured.
- Look at the views of diverse stakeholders groups and discuss the need for regulatory supervision with the eventual plan of optimizing the progress of new cancer treatments in these situations.
Dated: 27/06/2016