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

Area of Science:

• Biotechnology and Pharmaceutical Sciences
• Health Systems Management
• Genetics and Genomics

Background:

• Recent advancements have introduced the first commercial gene therapies in the U.S., offering potential cures for diseases.
• These novel treatments present significant challenges related to high costs and complex logistical requirements.
• A robust pipeline of gene therapies in late-stage development necessitates proactive adaptation by the healthcare industry.

Purpose of the Study:

• To equip health systems with essential knowledge regarding current and emerging gene therapy treatments.
• To outline key considerations for health-system pharmacies and specialty pharmacy programs in evaluating and implementing gene therapy services.

Main Methods:

• This study involved a comprehensive review of the current gene therapy landscape, including commercially available treatments and those in clinical development.
• Analysis focused on the unique challenges posed by gene therapies, such as cost, complexity, and logistical demands.
• Considerations for health system integration were examined, encompassing development, procurement, reimbursement, administration, and patient monitoring.

Main Results:

• Gene therapies have the potential to significantly alter disease trajectories and offer cures.
• The increasing prevalence of gene therapies within health systems requires a thorough understanding of their lifecycle.
• Proactive planning and adaptation are essential for health systems to manage the complexities of these advanced treatments.

Conclusions:

• Health-system pharmacy practice must evolve to incorporate comprehensive gene therapy services.
• Effective stakeholder engagement strategies are vital to ensure patient access to these transformative treatments.
• Preparing health systems for gene therapies is critical for future healthcare delivery and patient outcomes.