| Popis: |
The needs for rare disease research and orphan drug development have been increasingly recognized in the last few decades, partly due to legislative change, providing incentives for new treatments of rare disease. There are several unique challenges for rare disease therapy development: poor understanding of the disease, clinical trial design with small patient populations, and obstacles to the successful execution of clinical programs. While conventional RCTs have proven to be difficult in this setting, pragmatic randomized clinical trials (pRCTs), by leveraging real-world data and eHealth technologies, may be a unique fit for the special needs of the rare disease field. pRCTs evaluate the performance of a therapy in diverse patient populations with extended follow-up in comparison with standard care and provide evidence for the robust safety and effectiveness of the treatment. This chapter discusses the regulatory advancements and unique challenges in rare disease treatment development, as well as the type of pRCTs that can be considered in addressing these obstacles. |
