A Population Health-Based Approach to Inform the Provisioning of Gene Therapies for Severe Sickle Anemia in a Large, Vertically Integrated, Value-Based Health Care System.

Kaiser Permanente is a large, vertically integrated, health system charged with the care of over 12 million people and approximately 2300 patients with sickle cell anemia(SCA) nationally with close to 50% residing in Southern California (2023 data). We describe a population based approach to the identification and care navigation of severe sickle cell patients in collaboration with contracted centers of excellence to provision access to gene therapies (Lyfgenia & Casgevy). The Southern California Permanente Medical Group created an interdisciplinary working group of pediatric and adult hematologists, case managers, NPs, and social worker to case manage over 1000 sickle cell patients in our regional encatchment. Retrospective data review of total health care utilization in 2023 by region and nationally sorted by ICD-10 Sickle Cell related diagnostic codes. Cost was segregated by age cohorts (0-18yrs, 18-30yrs and >30yrs) and gender. Leveraging our integrated health care network, our analysis revealed severe SCA patients account for 9% of our SCA population but 37% of all encounters and 73% of ED encounters. Furthermore, a focused cost of care analysis stratified by age, and gender identified a 3-fold rise in cost between pediatric (<17yrs) and young adult (18-29yrs). In Southern California, the average duration of coverage for this population was 11.6 years. The highest utilizing pediatric (age >12) and adult patients who meet severe criteria and without HLA matched sibling donors were identified and screened to explore candidacy for gene therapy based on current clinical guidelines. Common reasons for declination or inability to progress beyond screening include: concerns for infertility/poor access to fertility preservation, comorbid liver disease(autoimmune hepatitis/PSC), inability to adhere to rigorous treatment schedule. Providing access to gene therapy for severe sickle cell patients in a vertically integrated health system requires a data informed, multidisciplinary team. Future refinement of our workflow will be to include patient care experience feedback and to focus on patients aged 12-18yrs who may reap the benefits of gene theapy earlier and decrease the expected marked increase in complications and health care cost as they become adolescent/young adults.