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Published on: September 18, 2013
Development of a policy model for pediatric acute lymphoblastic leukemia to facilitate economic evaluation
Petros Pechlivanoglou1,2,3, Linda Luu1, Qing Li2
1Child Health Evaluative Sciences, The Hospital for Sick Children Research Institute, Toronto, ON M5G0A4, Canada.
Insights
The ALL Policy model estimates long-term outcomes for pediatric acute lymphoblastic leukemia (ALL). Blinatumomab may offer a cost-effective treatment for relapsed B-cell ALL.
Area of Science:
- Pediatric Oncology
- Health Economics
- Biostatistics
Background:
- Novel immunotherapies for relapsed pediatric acute lymphoblastic leukemia (ALL) offer high efficacy but their long-term clinical and economic impact remains unclear.
- The ALL Policy microsimulation model was developed to address this uncertainty for pediatric ALL patients in Ontario, Canada.
- The model's utility is demonstrated through a cost-effectiveness analysis of blinatumomab in relapsed B-cell ALL.
Purpose of the Study:
- To develop and validate the ALL Policy microsimulation model for estimating long-term clinical and economic outcomes in pediatric ALL.
- To assess the cost-effectiveness of blinatumomab compared to standard chemotherapy for relapsed B-cell ALL.
Main Methods:
- The ALL Policy model integrates health administrative data, chart data from Ontario, Canada, and published literature.
- The model simulates lifetime risk of relapse, bone marrow transplant (BMT), conditional life expectancy, quality-adjusted life years (QALYs), and total healthcare costs.
- Stratification by clinical characteristics (e.g., B-cell or T-cell lineage) is possible, with a specific subset for relapsed B-cell ALL analysis.
Main Results:
- Simulated patients diagnosed between 2002-2012 have a projected conditional life expectancy of 64.9 years.
- The estimated lifetime risk of BMT is 12.5%, with average lifetime healthcare costs of $244,433 CAD.
- Blinatumomab demonstrated an additional 1.08 QALYs at an incremental cost of $59,410 CAD per QALY, indicating potential cost-effectiveness.
Conclusions:
- The ALL Policy model provides a robust framework for timely economic evaluations in pediatric ALL.
- The introduction of blinatumomab for relapsed B-cell ALL appears to be a potentially cost-effective treatment strategy.
Background:
New highly effective, but expensive, immunotherapies have revolutionized the treatment of relapsed pediatric acute lymphoblastic leukemia (ALL) but their long-term clinical and economic impact is unclear. We developed the ALL Policy microsimulation model to estimate long-term clinical and economic outcomes for patients with pediatric ALL aged 0-17 in Ontario, Canada. We also illustrate the model's clinical utility through a cost-effectiveness analysis of blinatumomab in relapsed B-cell ALL.
Methods:
The ALL Policy model is informed using health administrative data and chart abstracted data from Ontario, Canada, and published literature. The model estimates lifetime risk of relapse, bone marrow transplant (BMT), conditional life expectancy, quality-adjusted life years (QALYs), and total health-care costs for individuals with pediatric ALL and can be stratified by relevant clinical characteristics (eg, B-cell or T-cell lineage). Additionally, we subset the model to patients with relapsed B-cell ALL to illustrate use of the model in estimating the cost-effectiveness of blinatumomab vs standard chemotherapy.
Results:
Simulated pediatric ALL patients diagnosed from 2002 to 2012 had a projected conditional life expectancy of 64.90 years. The lifetime risk of BMT was estimated at 12.5%. Lifetime health-care costs were $244 433 Canadian Dollars (CAD) (95% confidence interval = $213 314 to $303 430). Treatment with blinatumomab compared to standard chemotherapy post-relapse was estimated to result in 1.08 additional QALYs and an additional cost of $59 410 CAD (incremental cost-effectiveness ratio of $54 885/QALY).
Conclusion:
The ALL policy model can serve as a modeling foundation for timely economic evaluation. Introduction of blinatumomab in relapsed B-cell ALL may be a cost-effective strategy.
