You will have read elsewhere that the first EWGGD was held in Trieste in 1994 under the Chairmanship of Prof. Hans Aerts and Vice Chairmanship of Prof. Tim Cox. They took the unusual step of inviting representatives from the fledgling patient groups that were slowly emerging in Europe to participate, and I had the privilege together with the late Susan Lewis of representing the UK.
It was an inspired move and proved to be the catalyst for the coming together of European Patient groups. Initially, the patient representatives were an informal umbrella group calling themselves the European Gaucher Alliance (EGA), which in 2008 became a formal body and transitioning in 2018 into the International Gaucher Alliance (IGA) with the global reach by Spring 2020 of 57 member organisations. The fact is that it was the invitation in 1994 for patient groups to participate in the EWGGD that was the start of it all.
We didn’t really know what to expect in Trieste. There were seven countries represented, and the patient representatives were not scientists or clinicians. Most had never been at a meeting of this nature and sat at the back of the small meeting room trying to absorb the presentations but when they couldn’t understand the scientific detail they did understand that the people in the room were the world’s experts in Gaucher disease who had come together to share their experiences and their research and to discuss how best to manage and treat their patients.
There was concern expressed by some that it might be upsetting for the representatives (many of whom were patients themselves) to witness the differences in approach to treating patients. However, the opposite was the case. For the patient representative to witness the world’s experts in this rare and not well understood disease debating (and yes, in some cases arguing!) it was a humbling experience because the patients recognised that this group of brilliant and passionate experts had come together to unlock some of the mysteries of the disease and this would ultimately help their members. This remains the case today.
The opportunities for patient representatives to attend such meetings (particularly those from countries where there was little experience of the disease) cannot be underestimated. Initially, the representatives came together as an informal alliance. Whilst patient representatives meeting other patient representatives brought one benefit, representatives having the opportunity of meeting the world’s experts in Gaucher disease (and indeed industry representatives) and being able to engage and build relationships and introduce them to their local doctors, proved to be an altogether different dimension of benefit and the impact of this networking cannot be underestimated.
For patients the threefold benefits of learning of the latest research and into the clinical management of their members’ condition, the time to share experiences with other patient group leaders and the opportunity of networking with the world experts, remains the constant immeasurable gift from the special relationship between the patient representatives and the EWGGD.
As the number of patient representative groups grew it became clear that more time was needed for patients to discuss their issues, and in Autumn 2000 just before the 4th EWGGD in Jerusalem the patient groups (then called the EGA) held their first dedicated patient conference. This pattern has been followed in subsequent years. In the days before each EWGGD workshop, the EGA (now IGA) hold their own patient lead meeting, still sharing experiences and seeking ways by which the patient community members can support each other. Most patient representatives then stay on to participate in the EWGGD workshop.
When the EWGGD itself decided to formalise its own structure in Cologne in 2010 (it was registered as an official body in the Netherlands on the23rd February 2012) it was clear that the relationship between the EGA and the EWGGD was so intertwined that the EGA was given by the constitution of the EWGGD a permanent standing seat on the Board of the EWGGD. It is a convention that although the EGA (IGA) representative is a full member of the board, he/she does not participate in the meetings for the selection of presentations or posters to be given at EWGGD Meetings.
At the 2012 EWGGD Workshop in Paris the EGA were honoured to be shown on all literature as a “Partner of the Congress”, a position it continues to hold.
The EWGGD has since its inception been at the forefront of addressing issues of major concern to Gaucher patients and it is the organisation that the EGA (now IGA) looks to for advice and guidance both generally (seeking to establish consistent management and treatment for patients across Europe and now globally) and specifically at times of special need.
The EWGGD, together with representatives of the EGA, held special meetings of sub-groups leading to the publications of scientific papers, including the paper recording a European Consensus on the treatment of Neuronopathic Gaucher patients.
Crucially the EWGGD played a leading global role in 2009 when the global supply of Cerezyme was compromised by holding an emergency meeting with the subsequent publication of papers on the management of patients during the time of the global shortage of supply of Enzyme Replacement therapy and the priorities for treatment of patients on the reinstatement of limited supplies. The EGA was grateful both for this initiative but also for its ability to participate in the whole process.
Since the very first meeting in Trieste, patient representatives have been given the opportunity of addressing the EWGGD workshop. Over the years the EGA has focussed on unmet need both clinical and practical (patients who cannot access treatment) and has been particularly grateful to have had the opportunity of using the forum to highlight the existence of (and sometimes shortcomings) of the humanitarian aid programmes run in conjunction with industry.
If there is no doubt that the initiative of including the patient group representatives at the EWGGD was a catalyst for the development of the global patient body the special relationship between the EWGGD and the patients has helped shape the Gaucher world for the better.
It is an understatement to say that the Patient community have much to thank the EWGGD for.
Jeremy Manuel O.B.E
Co-founder UK Gaucher Association
Hon President International Gaucher Alliance
