In early January, Hugh Leonard, Chair of the EHERCC was invited to participate in a workshop hosted by the European Medicines Agency (EMA) and the European Organisation for Research and Treatment of Cancer (EORTC), to discuss the challenges faced by ultra-rare sarcomas, defined as sarcomas with a incidence rate of less than 1 in 1,000,000 of the population. Hugh explained:
“I was delighted to be part of the team, led by Dr Silvia Stacchiotti from Milan, who participated in a workshop looking at how we can encourage and improve the pathway with regulators to get new treatments approved for ultra-rare sarcomas. Not only did we have the EMA participating but also the Food and Drug Administration (FDA) from the USA.”
Hugh Leonard and Dr Josh Sommer from the Chordoma Foundation in the US jointly presented the perspectives of patients with ultra-rare sarcomas. Senior clinicians from Europe, the UK and USA also participated, addressing a number of key areas. In addition, over 300 people participated on line, including clinicians, health care professionals, researchers, patient advocates and members of the public.
The meeting was very positive with strong alinement between the clinicians, patient groups and the regulators, all keen to continue the dialogue and to find improved ways to approve new drugs or the repurposing of existing drugs for ultra rare cancers. This is of particular importance for EHE as the EHE group continue to seek approval of sirolimus for the treatment of EHE.
Hugh is confident that further meetings will be held and looked forward to updating the EHE community at a later date.
Recordings of the presentations and a narrative report from the January workshop can be found at:
www.ema.europa.eu/en/events/ema-eortc-multi-stakeholder-workshop-soft-tissue-bone-sarcoma