BUDGET IMPACT ANALYSIS OF EXAGAMGLOGENE AUTOTEMCEL FOR PATIENTS WITH SICKLE CELL DISEASE AND RECURRENT VASO-OCCLUSIVE EVENTS IN THE UNITED STATES
Author(s)
Poonam S. Bhatjire, MS1, Khashayar Eshtiaghi, MBA2, Enrique Seoane-Vazquez, PhD2, Marc Fleming, BS, MPH, RPh, PhD2;
1Chapman University School of Pharmacy, Ph.D. Student, Irvine, CA, USA, 2Chapman University School of Pharmacy, Irvine, CA, USA
1Chapman University School of Pharmacy, Ph.D. Student, Irvine, CA, USA, 2Chapman University School of Pharmacy, Irvine, CA, USA
OBJECTIVES: Gene therapy holds significant clinical promise; however, affordability is a major barrier to access, and evidence on budget impact is limited. This study aimed to estimate the budget impact and per-patient cost implications of exagamglogene autotemcel for patients with sickle cell disease (SCD) and recurrent vaso-occlusive events from the United States (U.S.) payer perspective.
METHODS: A budget impact model was developed from the U.S. payer perspective to compare exagamglogene autotemcel with lovotibeglogene autotemcel in patients with SCD and recurrent vaso-occlusive events. Cost inputs were obtained from IBM Micromedex Red Book, the Centers for Medicare and Medicaid Services (CMS), and published literature. The analysis assumed a hypothetical 1-million-member health plan over a 3-year period. Because both therapies are one-time treatments, the model estimated total per-patient treatment episode costs occurring within the analytic period, including drug acquisition, administration, and grade 3-4 adverse event management. The incremental cost difference was calculated as the difference in cost between therapies. One-way sensitivity analyses were conducted on key cost inputs to assess robustness.
RESULTS: Within the 3-year analytic period, the total per-patient cost of treatment with exagamglogene autotemcel was $2,699,094 compared with $3,599,094 for lovotibeglogene autotemcel, corresponding to an approximate per-patient cost savings of $900,000 in favor of exagamglogene autotemcel. The drug acquisition cost was ($3,100,000) for lovotibeglogene autotemcel and ($2,200,000) for exagamglogene autotemcel. Administration costs and grade 3-4 adverse event management costs per patient were $470,800 for exagamglogene autotemcel and $28,300 for lovotibeglogene autotemcel. One‑way sensitivity analyses showed that results were most sensitive to drug acquisition and administration costs, while adverse event costs had minimal impact.
CONCLUSIONS: Exagamglogene autotemcel was associated with lower modeled per-patient total treatment cost than lovotibeglogene autotemcel, primarily due to lower drug acquisition cost. Administration and grade 3-4 adverse event costs were higher for exagamglogene autotemcel.
METHODS: A budget impact model was developed from the U.S. payer perspective to compare exagamglogene autotemcel with lovotibeglogene autotemcel in patients with SCD and recurrent vaso-occlusive events. Cost inputs were obtained from IBM Micromedex Red Book, the Centers for Medicare and Medicaid Services (CMS), and published literature. The analysis assumed a hypothetical 1-million-member health plan over a 3-year period. Because both therapies are one-time treatments, the model estimated total per-patient treatment episode costs occurring within the analytic period, including drug acquisition, administration, and grade 3-4 adverse event management. The incremental cost difference was calculated as the difference in cost between therapies. One-way sensitivity analyses were conducted on key cost inputs to assess robustness.
RESULTS: Within the 3-year analytic period, the total per-patient cost of treatment with exagamglogene autotemcel was $2,699,094 compared with $3,599,094 for lovotibeglogene autotemcel, corresponding to an approximate per-patient cost savings of $900,000 in favor of exagamglogene autotemcel. The drug acquisition cost was ($3,100,000) for lovotibeglogene autotemcel and ($2,200,000) for exagamglogene autotemcel. Administration costs and grade 3-4 adverse event management costs per patient were $470,800 for exagamglogene autotemcel and $28,300 for lovotibeglogene autotemcel. One‑way sensitivity analyses showed that results were most sensitive to drug acquisition and administration costs, while adverse event costs had minimal impact.
CONCLUSIONS: Exagamglogene autotemcel was associated with lower modeled per-patient total treatment cost than lovotibeglogene autotemcel, primarily due to lower drug acquisition cost. Administration and grade 3-4 adverse event costs were higher for exagamglogene autotemcel.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
EE212
Topic
Economic Evaluation
Topic Subcategory
Budget Impact Analysis
Disease
SDC: Rare & Orphan Diseases, STA: Genetic, Regenerative & Curative Therapies