Orphanet collaboration

The WHO International Clinical Trials Registry Platform aims to offer a complete view of clinical research, which is accessible to all those involved in health care decision making. Therefore, it is crucial to also include and highlight rare diseases.

Orphanet is a unique resource, providing manually-curated and expert-validated knowledge on rare diseases, and the Orphanet nomenclature of rare diseases, based at the INSERM (French National Institute for Health and Medical Research).

The collaboration between ICTRP and Orphanet, which started in 2018, is aimed at making clinical trials on rare diseases easily identifiable and findable, thus improving knowledge on rare diseases. ICTRP and Orphanet will work to identify and flag all rare disease related clinical trials in the ICTRP database for its users. Clinical trials for rare diseases are exported into the Orphanet database, where additional information is available, in particular the rare disease concerned, the category of clinical trial, and the medicinal product in development, amongst other information. Rare disease related clinical trials are identified using the Orphanet nomenclature of rare diseases.

This will contribute to raising awareness of rare diseases and facilitating access to the most recent information on clinical trials in the field of rare diseases.

• Orphanet
• Orphadata
• EMA Committee on Orphan Medicinal Products
• FDA Office of Orphan Products Development
• European Joint Programme on Rare Diseases
• European Reference Networks