Orphan Drugs for the Treatment of Rare Diseases. A Comparative Public Law Perspectives

This article aims to analyse the regulatory framework of the orphan drugs for the treatment of rare diseases. After defining the concepts of “orphan drug” and “rare disease” and reviewing the U.S. and European regulatory regimes, the essay examines the Italian legislation and, in particular, the balance of the current distribution of expenditure between public bodies and private economic operators. The article reaches the conclusion that current Italian framework represents a best practice: it allows the most appropriate constitutional balance between public interest, private economic operators interest and right to health of individuals.