Declaration of Intent “Better Access to Promising Orphan Drugs”  

On March 4^th, SCN attended the presentation of the Declaration of Intent “Better Access to Promising Orphan Drugs” to Karla Van Rooijen, Director of Medicines and Medical Technology (Directeur Geneesmiddelen en Medische Technologie, GMT), on behalf of Spierziekten Nederland, Patiëntenfederatie Nederland, Prinses Beatrix Spierfonds, Spieren voor Spieren, VSOP, Spierziekten Centrum Nederland, and the VRA. To highlight the progress and challenges in this area, Erik Niks (LUMC) discussed recent developments in treatments for Duchenne muscular dystrophy while Anja Horemans from Spierziekten Nederland shared the issues and experiences related to other neuromuscular diseases. During the subsequent dialogue with the Ministry of Health, Welfare and Sport (VWS), several initiatives aimed at ensuring faster and better access to orphan drugs at socially acceptable prices were discussed. We concluded that a prompt engagement with all relevant parties, such as Zorginstituut Nederland, is essential to pave the way for new treatments. We thank VWS for the conversation and the informative discussion.