Gene Replacement Therapy: A Primer for the Health-system Pharmacist

Area of Science:

• Pharmacology
• Biotechnology
• Genetics

Background:

• Over 2600 gene therapy trials are underway globally, with 4 FDA-approved products in the US.
• Existing guidelines from major health organizations for pharmaceutical gene therapy applications are lacking.
• Institutions often develop inconsistent and outdated internal policies for gene therapy handling.

Purpose of the Study:

• To provide comprehensive guidance on gene replacement therapy handling and administration for pharmacists.
• To offer expert opinion on institutional preparedness and the role of pharmacists in managing gene therapies.
• To address the absence of standardized protocols for gene therapy pharmaceutical applications.

Main Methods:

• Expert review and opinion synthesis.
• Analysis of current gene therapy landscape and regulatory gaps.
• Development of a proposed infrastructural model for gene therapy handling.
• Discussion of personnel, patient, and caregiver education strategies.

Main Results:

• Gene therapies require distinct handling procedures compared to conventional drugs.
• Pharmacists are essential in identifying risk levels and establishing safe handling infrastructure.
• Standard operating procedures (SOPs) and institutional policies are critical for safe gene therapy management.

Conclusions:

• Pharmacists are pivotal in the safe handling, administration, and overall management of gene replacement therapies.
• Developing robust infrastructure and clear protocols is essential, especially given the lack of consensus guidelines.
• Expert guidance is needed to ensure institutional preparedness for the growing field of gene therapy.