- ELENCO DEI MEMBRI
- NOTIZIE ED EVENTI
- SOSTIENI L’EGA
Humanitarian Aid has always been a priority for the EGA; we have always pledged to help any Gaucher patient who ask us for help where ever they live in the world. In the past when there was only one enzyme treatment for Gaucher disease all requests for humanitarian aid were passed to Genzyme and the EGA would provide support to the patient and their family throughout the process.
Over the last couple of years with the introduction of new therapies and new companies coming into the market it became clear that it was necessary to look at how best to approach the provision of humanitarian aid to ensure sustainability and security for patients and their families.
At the EWGGD meeting in Cologne in 2010 the EGA made a presentation on the areas of priority for patients and one of these was to look at the development of a global humanitarian aid pathway for Gaucher disease.
After many months of planning a meeting took place in Amsterdam at the Amsterdam Medical Centre on the 5th of October 2011, convened by the EWGGD and EGA. Present at the meeting, co-chaired by Professor Carla Hollak and Professor Timothy Cox, were representatives of all the companies involved in the manufacturing and marketing of treatments for Gaucher disease: Genzyme, Shire, Actelion, Pfizer and Protalix. Genzyme’s humanitarian programs were represented by Dr. Anna Tylki-Symanska and
Dr. Ashok Vellodi. Project Hope was represented by Professor Pramod Mistry. Patients were represented by Rhonda Buyers of the National Gaucher Foundation in the USA and from the EGA Tanya Collin-Histed and Jeremy Manuel.
The meeting agenda was to discuss the development of a global coordinated structure for humanitarian aid for Gaucher patients who are unable to access treatment—wherever they live.
The meeting concluded with the establishment of a working party, led by Tanya Collin-Histed, Rhonda Buyers and Dr Ashok Vellodi, to develop a pilot framework for the distribution of humanitarian aid in India and Pakistan, where there are a large number of children with Gaucher disease who do not have access to any kind of treatment. These two countries were also chosen because their current structures are very different: India has a very good clinical structure, a number of medical centres and an established LSD patient group who are working to support families; Pakistan on the other hand has many challenges, no patient association and a limited clinical structure. For example, families have to pay for infusions and collect the medication from the airport.
During the next five months the working party collected information and data on the current situation in the two countries and presented a report on their findings to the larger group (including representatives from the Pharmaceutical Companies) at a second meeting on Monday 19th March that took place in Cambridge, UK.
Representative Prasanna Shirol from the LSD
Patient association in India and Ati Qureshi, who is the father of a Gaucher child and representing
patients in Pakistan were due to attend the meeting and make presentations on the current situation in their countries however they were unable to travel to the UK as their visa applications were not approved. In their absence Tanya Collin-Histed and Dr Ashok Vellodi made their presentations on their behalf.
At the meeting a number of recommendations were made to the group following the report findings, these included; to improve access to clinical care in India and Pakistan with the development of new centres; to create a Medical Advisory Board in Pakistan; to implement training for doctors in Pakistan; to support the development of a patient association in Pakistan; to look at non-ERT costs; to provide ERT for patients who are currently diagnosed with Gaucher disease but who are currently not receiving treatment; and to look at how to develop a global independent medical advisory board for all Gaucher treatments. The group agreed to form a working party look at how these challenging recommendations could be taken forward and implemented.
The working party will consist of two company representatives, three physicians and two patient representatives; they will meet for the first time by teleconference on Monday 23rd April and will report back to the full group at a meeting on Monday 25th June in Paris, France to coincide with the EWGGD meeting.
This is a unique and exciting but also very challenging opportunity and if it works could also be a model for other LSDs.