RE:RE:RE:RE:RE:RE:RE:RE:RE:RE:RE:RE:Clinical Trial Size in Orphan and Rare Diseases The U.S. Food and Drug Administration (FDA), in collaboration with Duke University’s Duke-Margolis Center for Health Policy, held a virtual public meeting on Thursday, Dec. 14 aimed towards ultimately improving patient community engagement in the development of rare disease treatments.
Traditional clinical trials are challenging to conduct for therapies targeting these small populations due to a limited understanding of disease natural history, difficulty powering traditional clinical trials given small population size and disease heterogeneity, and challenges with identifying and recruiting participants. Together, these factors significantly impact the rare disease drug development space. Given these challenges, understanding the experiences and priorities of patients and working closely with rare disease or condition experts, and experts on small population studies can be important for informing and supporting the drug development process for products intended for patients with rare diseases.
Understanding the experiences and priorities of patients and working closely with rare disease or condition experts, and experts on small population studies can be important for informing and supporting the drug development process for products intended for patients with rare diseases.
https://aadcnews.com/news/fda-meeting-thursday-focus-rare-disease-patient-engagement/