Abstract
BACKGROUND: Advanced therapies, including cell and gene therapies, have shown therapeutic promise in curing life-threatening diseases, such as leukemia and lymphoma. However, these therapies can be complicated and expensive to deliver due to their sensitivity to environment; troublesome tissue, cell, or genetic material sourcing; and complicated regulatory requirements. OBJECTIVE: This study aims to create a novel connected supply chain logistics and manufacturing management platform based on blockchain, with cell and gene therapy as a use case. Objectives are to define the requirements and perform feasibility evaluations on the use of blockchain for standardized manufacturing and establishment of a chain of custody for the needle-to-needle delivery of autologous cell and gene therapies. A way of lowering overall regulatory compliance costs for running a network of facilities operating similar or parallel processes will be evaluated by lowering the monitoring costs through publishing zero-knowledge proofs and product release by exception. METHODS: The study will use blockchain technologies to digitally connect and integrate supply chain with manufacturing to address the security, scheduling, and communication issues between advanced therapy treatment centers and manufacturing facilities in order to realize a transparent, secure, automated, and cost-effective solution to the delivery of these life-saving therapies. An agile software development methodology will be used to develop, implement, and evaluate the system. The system will adhere to the EU and US good manufacturing practices and regulatory requirements. RESULTS: This is a proposed study protocol, and upon acceptance, grant funding will be pursued for its execution in 2021. CONCLUSIONS: The successful implementation of the integrated blockchain solution to supply chain and manufacturing of advanced therapies can push the industry standards toward a safer and more secure therapy delivery process. INTERNATIONAL REGISTERED REPORT IDENTIFIER (IRRID): PRR1-10.2196/17005.