Farmaci orfani: uno sguardo sulle politiche di produzione e ricerca in ambito europeo

Abstract

Summary. The drug approval process consists of two stages: clinical trials (CT) and new drug application (NDA) approval. Orphan drugs are medicines or vaccines intended to treat, prevent or diagnose a rare disease. New programs in orphan drug development may offer valuable solutions to bring orphan treatments to the market more quickly in despite of hight prices. The aim of this study is to review the range and types of existing policies adopted by Europe to enable the availability and accessibility of orphan drugs.