Jingjing Ye - Böcker

Drug development is at a crossroads. As personalized medicine and targeted therapies expand, traditional clinical trial models have become too slow, costly, and inefficient. To meet the urgent need for faster, reliable evidence generation and decision making, researchers and regulators alike have turned to innovative trial designs. Among these, master protocols stand out as a transformative approach, allowing multiple therapies, disease subtypes, or objectives to be studied under a single, integrated framework. Proven successful in oncology, COVID-19, and beyond, they improve efficiency, reduce participant exposure to ineffective treatments, and accelerate meaningful outcomes with patient centric approach, prompting regulatory guidance and global adoption.This book provides a multidisciplinary exploration of master protocol clinical trials, blending clinical, statistical, regulatory, operational, economic insights and patients perspectives. It examines both opportunities and challenges in design and implementation, and provides in-depth summary and discussion on real-world examples such as I-SPY, ROAR, and RECOVERY. A practical guide for researchers, statisticians, regulators, and industry professionals, it demonstrates how master protocols can reshape drug development and bring innovative therapies to patients faster.● Provides comprehensive, theoretical and practical aspects for master protocol trials● Includes most recent development in the master protocol clinical trials● Covers broad topics such as trial designs, governance oversight, software implementation, database and data pipeline, multi-regional trials, risk and benefit with operational solutions, economic considerations, regulatory perspectives, patient engagement and diversity● Deep dives into numerous real world case studies, their underlying thinking, challenges and success.● Includes global perspective in multi-regional clinical development

Drug development is at a crossroads. As personalized medicine and targeted therapies expand, traditional clinical trial models have become too slow, costly, and inefficient. To meet the urgent need for faster, reliable evidence generation and decision making, researchers and regulators alike have turned to innovative trial designs. Among these, master protocols stand out as a transformative approach, allowing multiple therapies, disease subtypes, or objectives to be studied under a single, integrated framework. Proven successful in oncology, COVID-19, and beyond, they improve efficiency, reduce participant exposure to ineffective treatments, and accelerate meaningful outcomes with patient centric approach, prompting regulatory guidance and global adoption.This book provides a multidisciplinary exploration of master protocol clinical trials, blending clinical, statistical, regulatory, operational, economic insights and patients perspectives. It examines both opportunities and challenges in design and implementation, and provides in-depth summary and discussion on real-world examples such as I-SPY, ROAR, and RECOVERY. A practical guide for researchers, statisticians, regulators, and industry professionals, it demonstrates how master protocols can reshape drug development and bring innovative therapies to patients faster.● Provides comprehensive, theoretical and practical aspects for master protocol trials● Includes most recent development in the master protocol clinical trials● Covers broad topics such as trial designs, governance oversight, software implementation, database and data pipeline, multi-regional trials, risk and benefit with operational solutions, economic considerations, regulatory perspectives, patient engagement and diversity● Deep dives into numerous real world case studies, their underlying thinking, challenges and success.● Includes global perspective in multi-regional clinical development

This book discusses a multidisciplinary and multi-stakeholder collaborative approach to small population clinical trials. It provides and illustrates a systematic framework that depends upon a collaborative infrastructure between the patient community, patient advocacy groups, researchers and investigators, pharmaceutical companies, global regulatory agencies, and customers. The book also highlights the considerations of natural history and patient registry, diagnostic criteria determination, identification of clinically meaningful endpoints, and global regulatory guidance. The authors detail the nuances of clinical trial design as specific to small population disease, drawing from real examples.

This book discusses a multidisciplinary and multi-stakeholder collaborative approach to small population clinical trials. It provides and illustrates a systematic framework that depends upon a collaborative infrastructure between the patient community, patient advocacy groups, researchers and investigators, pharmaceutical companies, global regulatory agencies, and customers. The book also highlights the considerations of natural history and patient registry, diagnostic criteria determination, identification of clinically meaningful endpoints, and global regulatory guidance. The authors detail the nuances of clinical trial design as specific to small population disease, drawing from real examples.