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Table 7 Concerns of member associations

From: The European Gaucher Alliance: a survey of member patient organisations’ activities, healthcare environments and concerns

Concern No. of responses Member association's country
Medical care (Total 19 responses from 12 member associations)
Lack of information/limited knowledge among healthcare professionals/lack of Gaucher disease specialists 7 Finland, Latvia, Lithuania, Macedonia, Mexico PPuDm, Slovenia, Switzerland
Lack of consensus on management approach for Gaucher disease 2 Slovenia, Switzerland
Unmet needs: neuronopathic Gaucher disease, bone, mental health services/psycho-social support for patients 3 Slovenia, UK & RoI, USA
Lack of information on new therapies 1 Germany
Need for home infusions 1 Mexico PPuDM
Need for diagnostic centre/issues with delayed diagnosis/finding undiagnosed patients, need for specialist centre 4 Mexico AGdM, Slovenia, USA, Russia
Lack of a national Gaucher disease registry 1 Slovenia
Access to treatment (Total 25 responses from 20 associations)
Treatment access/inequalities in access 9 Bosnia & Herzegovina, Canada, Greece, Jordan, Latvia, Mexico PPuDM, Serbia, South Africa
Low treatment doses and delayed time to treatment 3 Norway, Romania, Spain
Imiglucerase shortage/way shortage was managed 4 Italy, Poland, South Africa, Spain
Changing economic environments/concerns for future reimbursement/specialised services 7 Denmark, the Netherlands, Spain, Sweden, Switzerland, UK & RoI, USA
Lack of collaboration with pharmaceutical companies regarding humanitarian aid 1 Jordan
Lack of dialogue with administrative authorities/hospitals/payers 1 Poland
Living with Gaucher disease (Total 5 responses from 5 associations)
Difficulties for Gaucher disease patients in finding jobs 1 Bosnia & Herzegovina
Patients' fear of repercussions if disease is known for insurance/barrier to diagnosis 1 USA
Obtaining disability status for patients 1 Russia
Lack of information on Gaucher disease for the general public 2 Macedonia, Switzerland
Patient organisations (Total 6 responses from 5 associations)
Lack of Gaucher disease patient interest in patient organisation 3 Belgium, Germany, Slovenia
Need to collaborate (between patient organisations) 1 Mexico PPuDM
Changing financial situation and lack of funds to support patients 1 USA
Lack of umbrella organisation for rare diseases 1 Slovenia
  1. A total of 27 member associations responded (55 responses).