5^th International Conference on Rare Diseases and Orphan Drugs

Clinical Trials in Rare Diseases: Clinical trials evaluate the safety, effectiveness, and potential risks of new medicines, medical interventions, and treatment strategies. Conducting trials for rare diseases can be challenging because eligible patients may be geographically dispersed, patient populations may be small, and disease progression may be difficult to predict. This session addresses innovative trial designs, patient recruitment, outcome measurements, real-world evidence, ethical considerations, and approaches to generating reliable clinical evidence for rare disease treatments.