Insights from the EMA and EORTC Workshop on Developing Treatments for Ultra-Rare Sarcomas

How can we develop new treatments in ultra-rare sarcomas, as a model for ultra-rare tumors?

On January 12, 2024, a workshop between regulators, researchers and patient advocates was held at the European Medicines Agency (EMA) premises in Amsterdam. The objective for the workshop was to discuss strategies to be more flexible in developing and approving new drug treatments for rare cancers, including Sarcoma, which can provide an initial use-case to define paradigms for treatment of other rare cancers.

Delegates from regulators from the EMA, ANSM (French National Agency for Medicines and Health Products Safety) and FDA (US Federal Drug Agency) participated in the workshop, together with representatives from the EORTC, the EORTC’s Soft Tissue and Bone Sarcoma Group (STBSG) and several other research institutions in Europe and the US.

Patient organizations were also represented, including SPAGN, EHE Rare Cancer Charity, Chordoma Foundation, The EHE Foundation and Sarcoma UK.

The presentations and discussions during the panel highlighted common ground for alignment, between the EMA, FDA, ANSM, the research and the patient advocacy community, and identified topics for further study including;

• The need to move on from rigid processes and make better use of available data without wasting resources.
• How to define data of sufficient quality to get regulatory approval.
• How to decide which off-label treatment to allow for compassionate use.
• Endpoints need to be agreed, as well as the way they are measured.
• Quality of Life (QoL) from the perspective of patients from different age groups is seen as an important parameter (the EMA will have a workshop on QoL on the 29th of February).
• There is a need to align with the EMA, FDA and others to define what degree of treatment response constitutes patient benefit.

A further workshop to follow this one this will be arranged before summer to take next steps.