Rare Diseases – Innovation in Funding & Reimbursement, and Insight into Societal Preferences.
Despite the continuing progress made in the R&D of orphan drugs to diagnose and treat rare diseases, the issue of rarity continues to ignite controversies and debates on the scientific, economic, and political grounds. While great efforts are being made both nationally and internationally to collaborate and advance in our knowledge and policies regarding rare diseases, little – albeit growing – attention is paid to societies’ values and preferences when it comes to the trade-offs to be made when allocating public resources.