London, United Kingdom – 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 2016.
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, what constitutes “dramatic activity” and “high unmet medical need” is often debated. 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; explore the views of different stakeholders and debate on 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. Our meeting with ESMO - a long time partner in our quest to answer the urgent needs of cancer patients - and other stakeholders to review SATs as an effective tool, is part of this effort.”
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. So, we are honoured to host this workshop with the EMA. It brings us a step further to supporting meaningful decisions, based on treatment outcomes, even in small patient populations. This is part of our commitment to sustainable oncology and equal access to new treatments, as described in ESMO’s 2020 Vision (1). We are also pleased that patients are represented, as their say on these issues is obviously fundamental.”
Furthermore, ESMO together with the European Organisation for Research and Treatment of Cancer (EORTC), have expressed their views on the EU Clinical Trials Regulation in an official position paper recently published in the Annals of Oncology (2).
In single-arm (or non randomised) trials, all patients enrolled receive the experimental therapy. SATs are used when target populations are very small, making randomised clinical trials (RCT) impractical. In SATs, researchers will use endpoints such as response rate to demonstrate clinical impact, in the absence of direct comparison data.
Single-arm trials are smaller, faster and make it easier to enrol specific tumour types. Weaknesses include the lack of randomised comparison; possible bias in selecting patients; lack of info on long-term outcomes and lack of good comparative safety data.
Dr Casali concluded: “We hope discussions at the workshop will help us improve the reliability of single arm trials in oncology and help us accelerate access to better treatment and care for cancer patients.”
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Notes to Editor
The event will be broadcast live on 30 June 2016 09:30-16:30 UK time (GMT +1) via the EMA website. Further details can be found here.
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.
ESMO’s educational resources support an integrated, multi-professional approach to cancer care. We have European roots and a global reach: we welcome oncology professionals from around the world and we seek to erase boundaries in cancer care as we pursue our mission: across oncology, worldwide.
About the European Medicines Agency (EMA):
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.