STEM CELL DERIVED BETA CELL TRANSPLANTATION: AN EXAMPLE FOR MODELLING THE COST OF MANUFACTURING NEW AND IMMATURE HEALTHCARE TECHNOLOGIES FOR EARLY PRIORITIZATION OF RESEARCH AND RESOURCES

   Purpose:

   Our study investigates how and to what degree stem-cell-derived transplant tissue would change costs per dose and overall cost-effectiveness of islet beta cell transplantation, a treatment method for patients with type 1 diabetes and hypoglycemia unawareness, which is currently not cost-effective.

   Method:

   We calculated maximum feasible costs per dose using current effectiveness and cost data. A model was developed to estimate cost of cell manufacturing for different production settings. With the results we conducted a cost-effectiveness analysis using a pre-existing state transition Markov model of beta cell transplantation.

   This model had intensive insulin therapy as comparator and took the Alberta Health Services perspective, with cost and outcome data from the University of Alberta hospital in Edmonton, Canada. We use different structural scenarios with the base-case scenario assuming the new treatment technology to be as effective as the current.

   Result:

   When using a strict cost-effectiveness threshold of $50,000 per additional quality-adjusted life year (QALY), the results show that even with no dose costs the extra costs of non-generic immunosuppression prevent it from being cost-effective. Without the need for immunosuppression the maximum cost per dose cost is $19,900 or for a more inclusive cost-effectiveness threshold ($100,000 per QALY) it is $65,500.

   Initial results point to a range for the per-dose costs of $1,500 to $19,000 depending on number cells per dose, production settings and demand. Higher cost estimates lead to lower net benefit of treatment. Additionally it increased the maximum value of information of further research into the cost-effectiveness for the dose-cost parameters.

   Conclusion:

   The feasability of beta cell transplantation becoming a cost-effective treatment depends on lowering the cost of goods through using de novo cells and addressing the need for immunosuppression. Without lowering treatment costs there is no value in further research into the safety and effectiveness.