European Conference on Rare Disease & Orphan Products (ECRD)

RVA News

Titled ‘Game Changers in Rare Diseases Delivering 21st Century healthcare to rare disease patients: Together we can change the future!’, the three day conference organised by EURORDIS and Develop Innovate Advance (DIA) was held at the Edinburgh Internatinial Convention Centre.

The program did not disappoint with so many topics, all concerning rare diseases.

There were over 760 attendees from over 40 countries at the event. The conference was attended by a variety of rare disease stakeholders with excellent attendance from the rare disease community. It addressed a range of topics looking at current experiences and exploring various perspectives and reviewing new challenges.

The very full program was preceded by pre-conference tutorials and the second Rare Disease International Meeting.

The conference was opened by co-organisers EURORDIS President; Terkel Anderson, Denmark and DIA Associate Director; and Tamara Kohler. The keynote addresses were delivered by Minister for Mental Health; Maureen Watt (Scottish Government UK) and Alastair Kent; Director, Genetic Alliance UK / RDUK. Alastair gave a brief history lesson reflecting on where we have come since virtually time began until our present day where whole genomes are sequenced for around $1000.

A highlight was the Young Patient Advocates Panel Discussion chaired by Bruno Sepodes, Chair COMP, Prof, University of Lisbon, Portugal. Participants shared unique and personal perspectives about the future of patient advocacy and how young people are creating change in the field of rare diseases.

We were spoilt for choice with six themes that ran concurrently and all very interesting. The themes were:  

Game Changers in Research

The speakers of this theme spoke extensively on the move from research to diagnosis of new technologies with the patient at the centre of new developments. Topics were around the interplay between public and private funding streams for research, and on some of the breakthroughs which have impacted on patient care, some of which have been enabled by innovative funding models. The discussion also covered research in the delivery of new therapies to patients and how this can be enabled in a sustainable manner.

Game Changers in Diagnosis  

For patients, families and carers getting a diagnosis is fundamental to their understanding of the situation they find themselves in. A diagnosis helps to open up potential pathways and answer questions about what has happened to them or my child or my family as well as what may happen in the future. How can I contribute or partner in research? It was an excellent discussion with many perspectives raised by both clinicians, researchers, patient organisations and patients.

Game Changers in Drug Development and Authorisation: Medicines and Adaptive Pathways 

In this theme, the speakers, made up of the European Medicines Agency (EMA), COMP Patient Representative, Global HTA & Patient Access look at how drug development, authorisation and access is no longer a linear path but more a cycle of gathering data and evidence and reviewing as a co-current process. What are the new challenges? How can medicines be accessed earlier? How can this risk be shared amongst stakeholders? How can patient communities better understand the process? Building collaborations and how can we smooth the path from research through to real treatments with real access to real patients.

Game Changers in Care Provision 

This theme looked at how even though we’re living in the ‘connected generation’, surprisingly, expertise in the rare disease field remains isolated and often siloed. The game changer here is bringing together the rare disease networks. Drawing on expertise from across Europe, speakers shared case studies to show what the true potential is in connecting isolated experts and sharing knowledge and information. Also looking at how this is an opportunity for care and how it will change in local hospitals from the creation of European Reference Networks. Simply by enabling centres to share their knowledge and practices, which becomes an opportunity for driving improvements for all. 

Game Changers in Social Policy  

In this theme, the speakers delved both into the current policy scenario as well as into innovative care solutions which are being experimented throughout Europe.

Game Changers in Global Society  

The presentations in this theme drove home the message that rare diseases are truly global, which can greatly accelerate knowledge, public awareness, and drug discovery and development in addition to connecting people. Social media experts shared their knowledge on how to use the internet efficiently. The sessions came out with the message that rare diseases are an international public health priority and a collaborative effort is required to bring this fact to the forefront.

The closing plenary enabled all the Chairs of the six themes to gather and give an overview and report on their sessions.

Although the focus of the meeting was very much about Europe, there was a lot of information that could be applied to our setting in Australia. It was pleasing to see an international focus and other countries featured in the program. There were some Aussies amongst the attendees including Nettie Burke (Cystic Fibrosis Federation), Gareth Baynam (Genetic Services WA), Hugh Dawkins (OPHG WA Health) and Kiwis John Forman (RVA/ICORD), and NZORD’s new CEO Letitia O’Dwyer.

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