Navigating clinical and operational challenges of adeno‐associated virus gene therapy: A guide for pharmacists
Year: 2025
Type: article
Abstract: Gene therapy provides a novel treatment strategy and potential cure for patients diagnosed with inherited disorders. The integration of gene therapy into clinical care provides a unique opportunity for pharmacists to demonstrate their investment in patient safety and outcomes. The foundation of this integration relies on the education of health care providers rooted in basic classification and understanding of gene therapy, along with the development of policies and procedures to determine pharmacy site readiness. It is critical that health systems recruit key stakeholders within their institution to ensure the safe and effective use of these extremely high‐cost therapies. Pharmacists are well positioned to lead these efforts across the health system. In addition to the challenges in preparation and biosafety regulations, operational considerations exist with the integration of use into technologies throughout the medication use process. Clinical considerations include recognition of both pre‐administration patient selection criteria and post‐administration monitoring, which are both critical to optimizing patient outcomes. Existing literature has been published surrounding biosafety and pharmacy procedures with respect to gene therapy preparation. However, current standardization and best practices are lacking with respect to clinical care models. This clinical pharmacy forum provides an overview of various operational and clinical considerations for adeno‐associated virus therapies and terminology that pharmacists should be aware of as they prepare to meet the demands of this novel therapeutic approach.
DOI: 10.1002/jac5.70036
Authors: Mary Riedy, Ingrid Pan, Jesse Cramer, Lisa Garrity
Language: English
Subfield: Genetics
Domain: Life Sciences
Open Access status: bronze
OpenAlex ID: W4409831141