Today the Gaucher community continues to face a crisis because there is an ever-growing number of patients without treatment in countries where governments are not willing or able to pay for it, with the EGA receiving requests for Humanitarian Aid from many countries.
Genzyme, Pfizer and Shire’s Charitable Access programmes either remain closed to new applications or with limited opportunities available to treat these patients.
Despite this situation the EGA will do everything it can to advocate on behalf of those seeking treatment. Please download this form and return it to firstname.lastname@example.org. By completing this form it gives the EGA permission to share the patient’s details with all of the pharmaceutical companies in the hope that they may be able to help.
These details will also be added to the EGA’s database of patients who are seeking treatment so that if any of the companies do open their programmes up we will be able to inform patients, their families/advocates and clinicians immediately.
The EGA is continuously working with the pharmaceutical companies, clinicians and patient groups worldwide to improve the lives and access to treatment for Gaucher patients.
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 few 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.
In October 2011, a meeting took place at the Amsterdam Medical Centre, convened by the EWGGD and EGA to discuss the development of a global coordinated structure for humanitarian aid for Gaucher patients who are unable to access treatment, wherever they live. This meeting was co-chaired by Professor Carla Hollak and Professor Timothy Cox and involved representatives from the EGA and all the companies involved in the manufacturing and marketing of treatments for Gaucher disease: Genzyme, Shire, Actelion, Pfizer and Protalix.
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.
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 in March 2012 in Cambridge, UK.
At this meeting a number of recommendations were made to the group following the report findings, including: 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 that the working party should look at how these challenging recommendations could be taken forward and implemented and to meet again in June 2012 in Paris, France.
Unfortunately in December 2012 one of the pharmaceutical companies decided to pull out of the programme and develop their own charitable access programme and so we were unable to move forward.
Despite this, the EGA continued to advocate on behalf of all patients seeking treatment and remained in regular contact with families and clinicians.
During this time, the EGA also developed a database of patients seeking treatment so that if any of the companies do open their programmes up we will be able to inform patients, their families/advocates and clinicians immediately.
In November 2014, the EGA convened a meeting in London, UK attended by representatives from all of the pharmaceutical companies and several Key Opinion Leaders (KOLs) in the Gaucher field. It was agreed that closer communication was essential to support patients and their families. Possible ways to support this were identified as: the development of a common application form; sharing of data with the EGA to generate a registry of patients; revisit how ‘we’ as a Gaucher community could seek to address this issue through a ‘global pathway’.
The EGA together with treating physicians discussed how to develop a consensus to support physicians where ERT is not available. These guidelines would be a ‘stop-gap’, to be used alongside seeking to access ERT and developing a new business model. This area of work is ongoing, however it has been challenging to get a consensus from the KOLs on this as there is a strong feeling that patients should not be discriminated against by offering different treatment options depending on where they live in the world.
For more information on the EGA’s humanitarian aid efforts please contact Tanya Collin-Histed email@example.com