The RaDiCEA Project: cost of illness (COI) analysis applied to Cryopyrin Associated Periodic Syndromes (CAPS)

When a disease affects only a few individuals in each country (ultra-orphan disease), it can be very hard to establish the costs of illness (COI) and the cost-effectiveness (CE) of treatments (ultra-orphan drugs). Conventional methods for COI and CE of drugs for common conditions do not apply, and additional factors need to be considered. As expensive medications (biologicals) show promising results, it becomes crucial to have detailed information on as many patients as possible.