Master Protocol
摘要
The emerging of master protocols is a paradigm shift for drug developers, regulatory agencies, policy makers, payers, health-care providers, and the patients. The innovative trial design framework offers advantages and gains efficiencies over traditional clinical trial designs by using common operating framework, common control when possible, and improved matching rates for patients in clinical trial especially when the prevalence of the biomarkers are low. In this chapter, the definition of master protocol and global regulatory guidance is given first. The practical and operational considerations that are not covered in the global regulatory guidance are provided. Statistical considerations according to different master protocol designs are described in detail, separately for exploratory and confirmatory settings. The master protocol has been implemented successfully and improved the patient care in treating patients with rare cancers and pediatric cancers. One of those successful examples is discussed in the chapter.