Adaptive Designs

The use of adaptive designs can be particularly useful or at times necessary when a clinical trial in rare disorders can only enroll a small number of patients. The global regulatory agencies have issued guidance and have shown encouragement on the use of adaptive designs in evaluating scientific evidence for approvals in rare and pediatric diseases. The types and key principles of adaptive designs are illustrated, and those useful designs that are either been used to support rare and pediatric diseases drug approvals or promising to further make the drug development feasible or efficient are discussed in the chapter. Practical considerations are given when implementing adaptive designs to support drug approval. Successful case examples are discussed throughout the chapter.