May 7, 2013
The first IRDiRC conference was held on April 16-17 2013 in the charming city of Dublin, Ireland. Thought leaders from all over the world captured the audience with stimulating, inspirational and highly informative talks on the past, present and future of rare disease research. IRDiRC was unanimously hailed as need of the time to advance the cause of rare disease research. IRDIRC promises to contribute in development of 200 therapies for rare disease and means to diagnose all of them by 2020. Rare disease stakeholders discussed the ways and means to help reach these goals. They provided examples of success stories as well as suggestions of the innovative ways in which stakeholders can come together to fulfill this goal.
This truly international conference was attended by more than 400 participants representing Europe, North America, Australia and Asia. It was recognised by one and all that expertise in rare disease, like the patients, are scattered across the globe. It is thus imperative for the global rare disease community to put their heads together to solve the great big jigsaw of rare disease, of which everyone holds a piece. This meeting boasted of attendance from policy makers, industry leaders, academicians as well as patient organisations from around the world. Representatives from regulatory bodies such as FDA, EMA, the Canadian and Australian regulatory agencies shared their expertise and pushed the need for more regulatory success for orphan drugs. Patient organisations such as Rare Voices Australia, Eurordis and Genetic Alliance US, gave an overview of the current need of patients and how coming together with a common agenda is urgent, but also achievable. Industry partners emphatically expressed the need to work with academicians, patient organisations and regulatory bodies to significantly increase the number and quality of drugs that is accessible to all. The buzz word was collaboration, collaboration, collaboration.
Mirroring the scientific committees of IRDiRC, the conference panels were divided into 3 tracks- therapies, diagnostic and interdisciplinary track-with experts in each area presenting the current outlook and the way forward. The therapies track addressed issues that deal with providing better treatment for patients such as drug repurposing, developing innovative therapies and ensuring an open dialogue with the regulatory bodies so that the treatments developed painstakingly see the light of day. The diagnostic track articulated the current projects that are underway to identify the causes of the rare diseases, the tremendous advancements in Next Generation Sequencing including the use of data generated from it and the crucial understanding of the Human Phenome. The speakers in the Interdisciplinary track highlighted the means by which successful collaborations can lead to successful treatments and diagnostics. They also endeavoured to delineate how future challenges in the extremely complex world of rare disease research can be overcome. Economic and ethical issues such as those that have cropped up due to recent advancements in technology, especially next generation sequencing, were also discussed at length.
Dr Ruxandra Draghia-Akli who is a champion of the rare disease cause and served IRDiRC as its chairperson with impressive results, passed on the baton to Dr. Paul Lasko, a distinguished member in the field of rare disease research. The success of this conference promises a much brighter future for rare disease patients. IRDiRC has 32 members representing research funding bodies. Organisations interested in becoming members of IRDiRC are encouraged to apply.