ABSTRACT
Reimbursement is an equally significant hurdle, especially in the world of orphan drugs. Due to the long development trajectory and the specific challenges that affect very rare diseases, the development costs for innovative drugs for such diseases are substantial. In the Netherlands, the minister of health at that time thought that these high-priced orphan drugs could be reimbursed from individual hospital budgets. Close partnerships between companies developing orphan drugs and patient organisations acting in the interests of people with particular diseases are mandatory, but due care and attention must be paid to the degree of independence of the partners. One of the pioneers in the development of orphan drugs for rare diseases was undoubtedly Genzyme, with its early success in gaining approval of a treatment for Gaucher disease. The Orphan Drug Act in the USA and similar legislation in Europe were key drivers in the discovery and development of drugs for rare diseases.
