Use of Single-Arm Trials in Rare Diseases – Clinical Trial Design and Protocol Development
Use of Single-Arm Trials in Rare Diseases – Clinical Trial Design and Protocol Development “Implementing Single-Arm Trials for Rare Disease Treatment” Introduction The complexities of conducting clinical trials in rare diseases are numerous, including small patient populations, heterogeneity of disease presentation, and a lack of existing treatment options. One approach that has gained traction in…