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Gene therapies for sickle cell disease (SCD) may offer meaningful benefits for patients and society. This study evaluated the cost-effectiveness of lovotibeglogene autotemcel (lovo-cel), a one-time gene therapy administered via autologous hematopoietic stem cell transplantation, compared with common care for patients in the United States (US) with SCD aged ≥ 12 years with ≥ 4 vaso-occlusive events (VOEs) in the past 24 months. We developed a patient-level simulation model accounting for lovo-cel and SCD-related events, complications, and mortality over a lifetime time horizon. The pivotal phase 1/2 HGB-206 clinical trial (NCT02140554) served as the basis for lovo-cel efficacy and safety. Cost, quality-of-life, and other clinical data were sourced from HGB-206 data and the literature. Analyses were conducted from US societal and third-party payer perspectives. Uncertainty was assessed through probabilistic sensitivity analysis and extensive scenario analyses. Patients treated with lovo-cel were predicted to survive 23. 84 years longer on average (standard deviation SD, 12. 80) versus common care (life expectancy, 62. 24 versus 38. 40 years), with associated discounted patient quality-adjusted life-year (QALY) gains of 10. 20 (SD, 4. 10) and direct costs avoided of 1, 329, 201 (SD, 1, 346, 446) per patient. Predicted societal benefits included discounted caregiver QALY losses avoided of 1. 19 (SD, 1. 38) and indirect costs avoided of 540, 416 (SD, 262, 353) per patient. Including lovo-cel costs (3, 282, 009 SD, 29, 690 per patient) resulted in incremental cost-effectiveness ratios of 191, 519 and 124, 051 per QALY gained from third-party payer and societal perspectives, respectively. In scenario analyses, the predicted cost-effectiveness of lovo-cel also was sensitive to baseline age and VOE frequency and to the proportion of patients achieving and maintaining complete resolution of VOEs. Our analysis of lovo-cel gene therapy compared with common care for patients in the US with SCD with recurrent VOEs estimated meaningful improvements in survival, quality of life, and other clinical outcomes accompanied by increased overall costs for the health care system and for broader society. The predicted economic value of lovo-cel gene therapy was influenced by uncertainty in long-term clinical effects and by positive spillover effects on patient productivity and caregiver burden.
Herring et al. (Mon,) studied this question.