Table 7.
Concerns of member associations
| 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 |
A total of 27 member associations responded (55 responses).