Managing High-Cost, Technology-Dependent Therapies: Systems for Cellular, Gene, and Advanced Biologics—A Narrative Review
Abstract
Background: The advent of advanced therapy medicinal products (ATMPs), including chimeric antigen receptor T-cell (CAR-T) therapies, in vivo gene therapies, and complex biologics, represents a paradigm shift in treating cancer, genetic disorders, and autoimmune diseases. These "living drugs" and sophisticated molecules present unprecedented challenges, requiring a complete re-engineering of traditional healthcare delivery pathways. Aim: This narrative review synthesizes evidence from 2010-2024 on the integrated, multidisciplinary systems required to safely, effectively, and sustainably manage the clinical and operational lifecycle of high-cost, technology-dependent therapies. Methods: A comprehensive search of PubMed, Scopus, Web of Science, and health policy databases was conducted for peer-reviewed literature and gray literature (white papers, health system reports) addressing the operational, financial, and clinical coordination of advanced therapies. Results: The review identifies five critical, interdependent system pillars: (1) a robust pre-treatment patient and product pathway spanning biomarker screening, cell collection, and manufacturing; (2) a specialized pharmacy and logistics infrastructure for storage, handling, and chain of custody; (3) protocolized clinical delivery and toxicity management anchored by specialized nursing; (4) complex financial navigation and reimbursement models; and (5) coordinated scheduling and data management. Failures in any pillar risk patient harm, therapeutic failure, and catastrophic financial loss. Conclusion: The successful delivery of ATMPs necessitates the creation of dedicated, cross-functional "Advanced Therapy Centers of Excellence." Sustainability demands the development of standardized operational frameworks, novel value-based payment contracts, and continued interdisciplinary research to optimize these complex care ecosystems.
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Authors
Copyright (c) 2024 Abdullah Abdulaziz Ibrahim Alghamdi, Andualrhman Hamad Jaafari, Bander Salem Jaber Almalki, Abdulrhnan Saad Aldawsari, Noor Mohammed Omar Usman, Ashwag Ibrahim Majrashi, Yahya Galib M. Qadhy, Ahmed Mohammed Abu Zibnah, Tirad Rakid Mushih Alruweaili, Rana Ibrahim Al Musabbihi, Sulaf Matir Sultan Alharbi, Saeed Saad Mobark
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