We are thrilled to share a new publication that includes participation of  EHE advocacy organizations: “How to foster new treatment development in ultra-rare tumours? Joint EMA-EORTC multi-stakeholder workshops on ultra-rare sarcomas as a model for rare cancers.” 

Leaders of the EHE Rare Cancer Charity (UK), the EHE Foundation, and EHE ITALIA Associazione Non Solo Laura ODV offer our sincerest appreciation to Dr. Silvia Stacchiotti for her vision, tenacity, and leadership throughout these workshops. 

Alongside Dr. Stacchiotti, numerous leading clinician-scientists, researchers, advocates, and regulators have dedicated countless hours to collaboratively defining a framework that supports the development of new treatments for patients with ultra-rare sarcomas, serving as a model for rare cancers. During these workshops, EHE was used as an example of the enormous unmet medical needs and unrealistic regulatory challenges facing the development of treatments for rare and ultra-rare diseases—challenges that are not typically experienced by common, larger population cancers. 

As advocates, we look forward to continued dialogue with regulators while working with clinician-scientists and industry to bring new and repurposed treatments to EHE patients.

Hugh Leonard, Chair of Trustees, EHERCC (UK) commented,

“This publication marks the beginning of an impressive and enlightening process, demonstrating to us, as advocates, the extraordinary challenges involved in developing a drug for an ultra-rare disease. It truly takes all of us working together – clinicians, researchers, patients, advocates, regulators, and industry partners – to make a difference. The process is far from easy, but we are making progress. Together, we will continue moving forward with the shared goal of improving the lives of people with EHE and other rare tumors.”