An article published by Giannuzzi V. et al, in Orphanet Jounal of Rare Diseases, analysed the status of orphan drugs designation that have not yet received a marketing authorisation or already marketed for patients affected by rare diseases in both the European Union (EU) and the United States (US). For each drug, the authors extracted data such as designation date, active substance(s), orphan condition and indication, trade name, approved therapeutic indication, approved ages, genetic nature of disease and if it affects children.
The authors used the EuOrphan database which compiles information on drugs aimed to diagnose, prevent or treat a rare disease. There is an important difference between the two markets in term of orphan designations and marketing authorisation per year. To date, the US has more designations as well as approvals than the EU (around three times more). Regarding therapeutic needs, oncology is the most represented while a majority of rare genetic diseases has still an unmet therapeutic need. In addition, despite the interest and the need for drugs approved for children, about half of the drugs approved in the EU and US for a rare disease affecting children was not granted a paediatric indication.
The authors concluded that, by merging all the existing approvals, patients would benefit of substantial advantages in both geographic areas. Efforts and cooperation between EU and US seem the only way to expedite the development and marketing of drugs for rare diseases.
Orphanews May 15, 2017