Rare Diseases & Orphan Drugs

Patient-Centered Research in Rare Diseases

Patient-centered research in rare diseases prioritizes the needs and perspectives of patients and their families. This approach involves engaging patients in all aspects of research, from study design to dissemination of results. It also aims to address the unique challenges faced by patients with rare diseases, including limited access to care and lack of effective treatments. Patient-centered research can improve the accuracy and relevance of research, ensure that patient perspectives are considered, and enhance patient engagement in the research process. Ultimately, this approach can lead to better outcomes for patients with rare diseases and help to advance our understanding of these complex conditions.