BACKGROUND

Sickle cell disease is a burdensome disease with limited treatment options. Given how inconclusive the evidence is for some of the newer agents, clinicians seem to only have hydroxyurea and crizanlizumab as treatment options for preventing vaso-occlusive events in patients with SCD.

OBJECTIVES

To estimate the cost-utility of preventing vaso-occlusive events in patients with sickle cell disease with crizanlizumab since the age 16 in the United States.

METHODS

We used a Markov model with two health states (sickle cell disease at baseline and dead) and two transition states (vaso-occlusive events and vaso-occlusive events requiring hospitalization). Utilities were presented as Quality-adjusted Life Years. A societal perspective was taken when calculating incremental cost-utility. Costs were elicited via macro-costing with model inputs being adjusted to 2020 US Dollars.

RESULTS

Using crizanlizumab to prevent vaso-occlusive events in patients with SCD resulted in an incremental 2.527 quality-adjusted life-years with $725,917 in added costs (ICER = $287,263/QALY) over a lifetime period. Effectiveness of crizanlizumab and its drug price were the parameters to which the ICER was most sensitive. To achieve a willingness to pay threshold of $150,000/QALY, crizanlizumab costs could not amount to more than $58,000 per year to be considered cost-effective.

CONCLUSIONS

Crizanlizumab is not cost-effective in this model in preventing vaso-occlusive events in patients with SCD. Drug price and relative effectiveness of crizanlizumab when compared to hydroxyurea were key drivers of this cost-effectiveness analysis.