Decoding gene therapy: Current impact and future considerations for health-system and specialty pharmacy practice.

Abstract

To provide health systems with baseline knowledge on existing and pipeline gene therapy treatments, including considerations that health-system pharmacies and specialty pharmacy programs may reference when evaluating and implementing services around gene therapies. Advancements in research and biotechnology have recently led to the development and launch of the first commercially available gene therapy treatments in the United States. These treatments have the ability to significantly alter and even effectively cure diseases. Alongside these significant advances and clinical benefits, these therapies present unique challenges due to their cost and complexity. Given the large number of additional gene therapy treatments that are currently in late-stage clinical development, stakeholders across the healthcare industry must increasingly adapt and ready themselves to meet these challenges. The diagnosis and treatment of patients with diseases being targeted by gene therapies largely occurs within health systems, and judging by the gene therapy pipeline, this trend is likely to continue. To prepare for these novel treatments, health systems must understand and consider the methods in which gene therapies are developed, procured, reimbursed, administered, and monitored. The future of health-system pharmacy practice must include comprehensive gene therapy services and stakeholder engagement strategies to ensure patients have access to these life-changing treatments.