OBJECTIVES

Novel therapeutic options for managing vaso-occlusive crises in pediatric sickle cell disease (SCD) have entered the market recently. However, evidence of their cost-effectiveness is lacking. This study presents a cost-effectiveness analysis of L-glutamine oral powder, hydroxyurea (Siklos) and off-label, generic hydroxyurea; compared to standard of care with chronic blood transfusions.

METHODS

Using a decision-tree model, we compared economic and clinical outcomes of novel SCD drugs and generic hydroxyurea over one-year time horizon in a hypothetical cohort of pediatric (≥ 2 years) SCD patients from a US payer perspective. The main outcome is incremental cost per number of SCD-related hospitalizations averted (ICER). Model inputs were derived from published literature and clinical trials. Drug average wholesale price (AWP) was obtained from Wolters Kluwer Medi-Span^®. All costs were converted to 2019 US dollars using the consumer price index (CPI) health care cost component.

RESULTS

Off-label, generic hydroxyurea was associated with both reduced costs and higher SCD-related hospitalizations averted, and dominated the novel drugs. L-glutamine oral powder and Siklos had incremental costs of $23,793 and $35,986; and averted 0.4 versus 0.2 SCD-related hospitalizations/year, respectively when compared to standard care. L-glutamine oral powder dominated Siklos. The ICER for L-glutamine oral powder compared to standard care was $55,816 while Siklos cost $153,634/averted SCD-related hospitalization. Generic hydroxyurea and L-glutamine powder, but not Siklos were cost-effective at a willingness-to-pay threshold of $100,000 per SCD-related hospitalization averted. This model was robust to a deterministic one-way sensitivity analysis wherein the hospitalizations prevented in Siklos users was increased by 50%. Generic hydroxyurea remained dominant and the resultant ICER for Siklos was $137,068/SCD-hospitalization averted.

CONCLUSIONS

The cost-effectiveness of hydroxyurea (SIKLOS) for SCD is dependent on drug cost, budget and willingness-to-pay per sickle cell hospitalization averted. L-glutamine oral powder may represent a cost-effective option for children who fail to respond to generic hydroxyurea.