Abstract
The diseases that occur in patient populations with a maximum prevalence of 6-8% of the world’s population are defined as rare diseases or orphan diseases, with birth defects, and especially adulthood. The legislation to promote the registration of orphan medicinal products started in the US with the Orphan Drug Act, issued in 1983, and in 1999 the European Union developed Regulation 141/2000 and 847/2000. While progress has been made in the research, development and production of orphan drugs, it is necessary to develop and implement common, coherent and functional policies at national, European and global level in this field. This means involving all relevant decision makers, competent regulatory authorities, appropriate health strategies and policies, engaging academia in research, and setting up national registries for rare diseases for neonatal screening tests and developing and interconnecting European reference networks.
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