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Development of a population-based cost-effectiveness model of chronic graft-versus-host disease in Spain
Carlos Crespo1, José Anton Pérez-Simón, José Manuel Rodríguez
1Statistics Department, University of Barcelona, Barcelona, Spain. carlos.crespo@oblikue.com
Insights
Extracorporeal photopheresis (ECP) is a more cost-effective third-line therapy for chronic graft-versus-host disease (cGvHD) than rituximab or imatinib. ECP offers improved efficacy and long-term benefits for cGvHD patients within the Spanish National Health System.
Area of Science:
- Hematology
- Transplantation Medicine
- Health Economics
Background:
- Chronic graft-versus-host disease (cGvHD) is a significant cause of mortality post-hematopoietic stem cell transplant.
- Efficient resource allocation is crucial given the diverse treatment options for cGvHD.
Purpose of the Study:
- To evaluate the cost-effectiveness of extracorporeal photopheresis (ECP) compared to rituximab (Rmb) and imatinib (Imt) for cGvHD treatment over 5 years.
- Analysis conducted from the perspective of the Spanish National Health System.
Main Methods:
- Microsimulation cost-effectiveness analysis comparing ECP, Rmb, and Imt for 1000 hypothetical patients.
- Utilized literature-based probabilities, clinical opinion, and published reports for treatment pathways and adverse events.
- Validated local cost data (2010 Euros) and resource utilization; employed probabilistic sensitivity analysis for robustness.
Main Results:
- ECP demonstrated greater efficacy, yielding 0.062–0.094 quality-adjusted life-years gained by year 5 compared to Rmb or Imt.
- ECP became dominant (cheaper and more effective) over imatinib after 9 months.
- The incremental cost-effectiveness ratio for ECP versus Rmb was €29,646 per life-year gained and €24,442 per quality-adjusted life-year gained at 2.5 years.
Conclusions:
- Extracorporeal photopheresis (ECP) represents a more cost-effective third-line treatment strategy for cGvHD compared to rituximab or imatinib.
- ECP's higher acquisition costs are offset by its superior efficacy and long-term patient benefits.
Background:
Chronic graft-versus-host disease (cGvHD) is the leading cause of late nonrelapse mortality (transplant-related mortality) after hematopoietic stem cell transplant. Given that there are a wide range of treatment options for cGvHD, assessment of the associated costs and efficacy can help clinicians and health care providers allocate health care resources more efficiently.
Objective:
The purpose of this study was to assess the cost-effectiveness of extracorporeal photopheresis (ECP) compared with rituximab (Rmb) and with imatinib (Imt) in patients with cGvHD at 5 years from the perspective of the Spanish National Health System.
Methods:
The model assessed the incremental cost-effectiveness/utility ratio of ECP versus Rmb or Imt for 1000 hypothetical patients by using microsimulation cost-effectiveness techniques. Model probabilities were obtained from the literature. Treatment pathways and adverse events were evaluated taking clinical opinion and published reports into consideration. Local data on costs (2010 Euros) and health care resources utilization were validated by the clinical authors. Probabilistic sensitivity analyses were used to assess the robustness of the model.
Results:
The greater efficacy of ECP resulted in a gain of 0.011 to 0.024 quality-adjusted life-year in the first year and 0.062 to 0.094 at year 5 compared with Rmb or Imt. The results showed that the higher acquisition cost of ECP versus Imt was compensated for at 9 months by greater efficacy; this higher cost was partially compensated for (€517) by year 5 versus Rmb. After 9 months, ECP was dominant (cheaper and more effective) compared with Imt. The incremental cost-effectiveness ratio of ECP versus Rmb was €29,646 per life-year gained and €24,442 per quality-adjusted life-year gained at year 2.5. Probabilistic sensitivity analysis confirmed the results. The main study limitation was that to assess relative treatment effects, only small studies were available for indirect comparison.
Conclusion:
ECP as a third-line therapy for cGvHD is a more cost-effective strategy than Rmb or Imt.
