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The European Medicines Agency (EMA) and the European Organisation of Research and Treatment of Cancer (EORTC) organised a workshop on soft tissue and bone sarcoma on 12th January 2024 addressing the question on how we can develop new treatments in ultra-rare sarcomas, as a model for ultra-rare tumours.
That workshop brought together academia, learned societies, patients, non-profit organisations and medicines regulators to explore clinical and scientific aspects related to the development of medicines for ultra-rare cancers. The focus was on methodological aspects of clinical studies (such as the use of master protocols), repurposing medicines, and the use of retrospective and real-world data, and prospective registries for further data collection.
This second workshop is organised to discuss more in depth certain aspects as a follow-up discussion. Specifically, the workshop covers topics such as how to support an ecosystem for ultra-rare cancers and discuss lessons learned on specific examples.