Gaucher's disease is, when left untreated, a progressive and in some subsets even life-threatening lysosomal storage disease. It is caused by a genetically linked deficit of acid beta-glucocerebrosidase. The enzyme can be replaced by Cerezyme®/imiglucerase produced by Genzyme Corp. The therapy has the potential to induce remission and normalise the patient's life. In June 2009 Genzyme had to announce a viral contamination of its bioreactors which led to a sudden stop of the entire production of imiglucerase. Subsequently only 50-20% of the former supply could be provided worldwide. The situation was not normalised until the beginning of the year 2011. Due to this unexpected shortage the relevant actors had to clarify quickly and unprepared which patient groups to prioritise and whom to supply with what quantities of imiglucerase. The shortly enforced prioritisation and rationing provide an opportunity to describe and analyse the spontaneously choosen prioritisation criteria and reveal value preferences shared by clinicians, patients, patient representatives, and company representatives. To reconstruct the chain of events and reactions and the revealed criteria and value preferences partly standardised interviews with representatives of the relevant stakeholder groups were conducted. Very clearly, the actors spontaneously chose to follow a medical need and a social solidarity principle.
|Translated title of the contribution
|Gaucher's disease and imiglucerase in 2009/2010: What leads to a suddenly enforced prioritisation?
|Number of pages
|Published - 02.2015