Date
- Friday, 24 May 2024
Location
- OnlineEuropean 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.