Background: The Complex Drugs Team at Children’s Hospital Colorado has onboarded 10 groundbreaking, high-cost precision therapies. These have been for neuromuscular and neurogenetic diseases. We recognize these therapies are the future and therefore need to have safe and efficient systems in place to allow for a seamless process; from on-boarding through treatment and follow-up.
Objective: To create and implement clinical pathways for the onboarding of delandistrogene moxeparvovec (Elevidys), that demonstrate safe, sustainable and efficient care. Also, being replicable by other colleagues.
Methods: The Complex Drugs Team has navigated the onboarding of these therapies by developing pathways. For delandistrogene moxeparvovec, these include workflows, algorithms, patient/family coordination, and clear communication pathways with the multidisciplinary team across the organization.
Results: The Complex Drugs team has seen successful due to the implementation of systematic clinical pathways. This remains true with the pathway for delandistrogene moxeparvovec. We are currently onboarding three different patients. We have created algorithms and workflows that address pharmaceutical and clinical processes to ensure a well-ordered process and the ability to treat patients in a safe and timely manner. Due to our experience with previous novel therapies, we are confident that these pathways will aid in the successful treatment of these patients and future patients. These processes will also be shared with internal and external colleagues to benefit the greater community.
Conclusions: Complex novel therapies are the future of medicine. Having a dedicated Complex Drugs Team has made it possible to create a streamlined process for delandistrogene moxeparvovec. We will use these to successfully treat patients; safely and efficiently. The Complex Drugs team has served as a national model, and we strive to continue this with delandistrogene moxeparvovec. The knowledge we will share will allow our community to provide safe and timely access to this novel treatment for patients within the Duchenne Muscular Dystrophy world.