The Amyloidosis Research Consortium (ARC) recently attended the European Conference on Rare Diseases and Orphan Products in Vienna, Austria. The conference brings together patient representatives, academics, health care professionals, researchers, industry, payers and regulators around a common interest - to advance diagnosis, treatment and care for patients living with a rare disease in Europe and beyond.
ARC’s objectives in attending were to promote awareness of amyloidosis, share best practice from amyloidosis initiatives and, through networking and learning from other rare disease communities, identify new opportunities for improving the landscape of research, treatment and care in amyloidosis. We were also selected to present our Patient-Focused Drug Development Initiative, an innovative project that ARC hosted in association with the FDA, as a model for improving regulators’ understanding of a rare disease.
This year the focus of the conference covered many relevant areas to ARC’s strategy, with stakeholders discussing the opportunities and challenges in diagnosing rare diseases, the need for better data collection and sharing of this data between specialist centers and countries, and the importance for patient organizations to take the lead in understanding, promoting and communicating what matters to patients and caregivers.
The landscape for diagnosing and treating amyloidosis is changing rapidly but the community needs to make sure the infrastructure is there to ensure patients benefit as quickly as possible. ARC came away inspired by the opportunities to make a tangible impact on the way amyloidosis is diagnosed and treated. We look forward to working with the wider community on putting this into practice.