LONDON, June 27, 2016 /PRNewswire/ --
The European Medicines Agency (EMA) and the European Society for Medical Oncology (ESMO) will host a workshop on single-arm trials (SATs) at the EMA headquarters in London, UK, on June 30th.
Single-arm trials are used when standard randomised trials are not possible, for example in rare cancers, where patient populations are very small. SATs have led to regulatory approval of oncology products showing dramatic activity in well-defined patient populations with high unmet medical needs. SATs, together with statistical approaches, could become the standard basis of evidence of efficacy for new applications. However, consensus is needed on what constitutes "dramatic activity" and "high unmet medical needs". Regulatory guidance on cancer drug development is needed in these situations, as well as on managing the remaining uncertainties optimally.
Participants at the EMA-ESMO workshop will examine the experience gained with past approvals based on SATs; the strengths and weaknesses of different approaches; the views of different stakeholders and the need and appropriateness of further regulatory guidance.
Dr Francesco Pignatti, Head of EMA's Oncology, Haematology and Diagnostics Office and Co-Chair of the workshop said: "Our role is to ensure that all medicines available on the EU market are safe, effective and of high quality. The EMA is aware of the urgent need for therapies for certain cancers and is committed to supporting access of promising anticancer medicines without compromising patients' safety."
Dr Paolo G. Casali, Chair of ESMO Public Policy Committee and Co-Chair of the workshop, explained that single arm trials could be very important for the development of new drugs in rare cancers and in small subgroups. "Sometimes the numbers for randomised trials are not available and at times the impact of new agents is huge in selected patients. This has immediate implications for drug development, provided methodological difficulties are addressed."
Casali added: "We hope this workshop will help us improve the reliability of single arm trials in oncology and help us accelerate access to better treatment and care for patients."
About the European Society for Medical Oncology
ESMO is the leading professional organisation for medical oncology. With more than 13,000 members representing oncology professionals from over 130 countries, ESMO is the society of reference for oncology education and information. We are committed to supporting our members to develop and advance in a fast-evolving professional environment.
About the European Medicines Agency
The European Medicines Agency (EMA) is a decentralised agency of the European Union (EU), located in London. It is responsible for the scientific evaluation, supervision and safety monitoring of medicines developed by pharmaceutical companies for use in the European Union.
SOURCE European Society for Medical Oncology (ESMO)