International Gaucher Alliance
Many physicians and scientists have played an active role in supporting and encouraging the work of patient groups. In 1994, the first meeting of the European Working Group on Gaucher Disease (EWGGD) was held in Trieste in Italy, and representatives of the known European patient groups, including the Israeli Gaucher Association, were invited to join with the doctors and scientists. While the patient representatives understood relatively little of the detailed science discussed, their presence was seen to encourage the professionals and all parties also learnt much during the coffee, lunch and dinner breaks.
Patient representatives continued to attend EWGGD meetings in Maastricht, Holland (1997) and in Lemnos, Greece (1999). During these meetings, the patient representatives took time to discuss their own priorities and concerns and it became apparent that the patient groups needed more time for discussion, and at the fourth EWGGD meeting in Jerusalem, Israel (2000), a separate day was set aside for a special European Gaucher Alliance (EGA)
meeting. At subsequent EWGGD meetings in Prague, Czech Republic (2002) and Barcelona, Spain (2004), the EGA held their own separate meetings to discuss issues of common interest.
During the late 1990s patients from former Communist countries in central and eastern Europe began to contact members of the EGA, and in 2001, an EGA delegation from the UK and Italy travelled to Bulgaria to hear about the plight of five children with the disease. Susan Lewis (UK) and Fern Torquati (Italy) attended with Dr Ari Zimran from Israel. They were shocked to see the severity of the children’s disease but were pleased that funding had just been obtained for them. Later in the year, Fern Torquati received an email from the sister of a young Yugoslavian boy needing treatment. She arranged for the child and his mother to travel to Italy and obtained enzyme replacement therapy for him. She then went to Yugoslavia to evaluate the situation there and found many more patients in need of treatment.
In the meantime, the UK invited all EGA delegates to their 10th anniversary conference in London in 2001 and patients’ representatives from Russia, Ukraine, Bulgaria, Romania and Yugoslavia arrived to tell their own stories of patients in desperate need of treatment. The EGA took on the role of negotiating for humanitarian aid with the Genzyme Corporation and in 2004, the company agreed the European Cerezyme Access Programme that provides free treatment for severely affected patients whose health services cannot or will not meet the cost of treatment. Genzyme had previously provided humanitarian aid in Egypt, China and parts of India and Pakistan through Project Hope and the International Cerezyme Access Programme.
The patients’ support groups’ focus on the need of the individual patient has put them in a unique position to act as an intermediary in many discussions both on a national and international basis, and in individual countries, patients’ support groups have acted as facilitators between doctors and treatment centres providing the support and structure to focus on future activities and projects. The mere taking on of the responsibility of organising a meeting (freeing the clinicians of the time-consuming details) has resulted in successful outcomes. In the UK,the Gauchers Association commissioned a national research study on bone disease to be a collaboration project between the four national centres.
On an international level, because there are only a relatively small number of clinicians and scientists in the field and due to the initiative to involve patients in scientific and medical meetings, patients’ support group leaders have developed personal relationships with doctors and scientists from around the world and have, through their professional approach, earned their respect and confidence. This has enabled individual patients’ support groups to play an active role in enhancing collaboration between medical centres and individual patient groups in countries where this approach is still novel.
Patients’ support group leaders also appreciate the commercial expectations of the pharmaceutical industry and its drive to develop and market their product. The patients’ support groups have a role to do what they can to ensure that the collaboration between clinicians and scientists with industry focuses on the advancement of treatment most beneficial to patients. Click here to find out more about the EGA