Role of healthcare databases and registries for surveillance of orphan drugs in the real-world setting: the Italian case study

Orphan drugs are medicines intended to treat, prevent, or diagnose a rare and serious condition. The collection of pre-marketing safety and efficacy data for orphan drugs is very challenging for several reasons related to the rarity of the diseases. This highlights the need for robust post-marketing evidence generation. Real-world data sources, such as claims databases, electronic healthcare records, and disease and drug registers, have an important role in studying orphan drugs. The availability and usefulness of such resources vary from country to country.