Rare diseases (RDs) are an important public-health issue and a challenge for the medical community. They are called ‘health orphans’, as a result of being neglected for many years due to research and development challenges. Dr Duygu Kuyuncu Irmak of MEK Consulting, Hilal Ilbars of the Ministry of Health – Turkish Medicines and Medical Devices Agency – Clinical Trials Department, and Professor Hamdi Akan of Ankara University School of Medicine, review the social and scientific need for the R&D of orphan drugs, discuss the management of challenges, and propose considerations for the future to better meet the medical needs of this area, with status updates from Turkey and the Middle East (ME).