Informing Clinical Methodology in Orphan Disease Research
Discovering and developing treatments for a rare disease is a complex process that requires a deep understanding of the patient’s experience. In this white paper, experts in rare disease clinical methodology and outcomes provide valuable insights into the importance of incorporating the patient’s voice into the discovery and development process. They discuss various methods for gaining insights into patient and caregiver perspectives, specifically for orphan diseases, and offer an in-depth analysis of their strengths and weaknesses. Check it out below.