Farmaci orfani e malattie rare: un confronto internazionale delle normative di riferimento

Abstract

Orphan drugs are defined as medicines with low economic returns, so that their production is not a profitable business far pharmaceutical companies. The present study analyses the main characteristics and the role of orphan drugs in four countries (United States of America, .Japan, Australia and European Union), by considering the regulation and the market situation of each State. All countries have introduced a specific legislation on orphan drugs to stimulate the research activity of pharmaceutical industry. The first law was the Orphan Drug Act of the United States of America in 1982. A common limit of all regulations is the strict correlation between “orphan drugs” and “rare diseases”. In fact, the term “orphan” does not refer only to rare disease, but also to other elements that can determine low economic returns for the industry (e.g. drugs with high cost of research and development, drugs that cannot be patented).