Date

Friday, 24 May 2024

Location

Online
European Medicines Agency, Amsterdam, the Netherlands

Event summary

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 follow-up meeting is organised to:

  • discuss the feedback obtained through the EU survey from the participants who attended the workshop on 12th January 2024;
  • discuss a proposed framework for developing medicines in rare disease indication using sarcoma as a model;
  • cover important topics such as how to support an ecosystem for ultra-rare cancers, providing methodological guidance, identifying core data elements to collect in registries and compassionate use programs.

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