Research Nurse Practitioner Stanford University San Francisco, California, United States
Title: Implementation of Commercial Gene Therapy Products – Challenges and Lessons Learned
Abstract : Basis of Inquiry - FDA approval in recent years of various gene therapy products for use in blood, cancer, and immune system disorders are revolutionizing the curative treatment options available to pediatric hematology, oncology, and stem cell transplant patients. Although many pediatric institutions have extensive experience in caring for patients enrolled on clinical trials for gene therapies, translation of these commercially approved treatments into standard of care workflows present a number of potential challenges. To successfully onboard commercial gene therapies into a clinical program, a variety of financial, institutional, and operational hurdles must be identified and addressed.
Purpose/Objective - The purpose of this paper presentation is to review the challenges and lessons learned in the on-boarding of newly approved commercial gene therapy products in a Pediatric Stem Cell Transplant Program at a large academic children’s hospital. It will also describe a single center’s experience in working to develop a structured institutional approach for the implementation of future commercial gene therapies. The presentation will provide a potential framework for other institutions planning to onboard new commercial gene therapies in the future.
Methods - This paper presentation will offer a descriptive overview of the complexities and challenges faced, as well as the efforts required, in the implementation process of the first commercially available gene therapy products for use within a Pediatric Stem Cell Transplant program.
Findings/Outcomes - Identification of key stakeholders, buy-in from hospital leadership, and investment of institutional resources and personnel are essential in the early stages of planning for the on-boarding of commercial gene therapy products. Given the high costs of many of these therapies, discussions with all involved departments should be initiated early in the implementation process. The development of a comprehensive institutional workflow that encompasses all necessary aspects for the successful on-boarding of these complex therapies is essential.
