In children with sickle cell anemia (SCA) in sub-Saharan Africa, treatment with hydroxyurea at the maximum tolerated dose (MTD), compared with no use of the drug, could lead to substantial net savings per patient, according to research published in Pharmacoeconomics.

Researchers estimated the incremental cost-effectiveness of hydroxyurea as a fixed-dose regimen or MTD regimen vs SCA care without hydroxyurea from the perspective of the health care provider.

Their estimates included the cost of providing outpatient treatment at a pediatric sickle cell clinic in Uganda and were used in a discrete-event simulation model to project mean costs (2021 US$), disability-adjusted life years (DALYs), and consumption of blood products per patient (450 mL units), for patients between 9 months and 18 years of age. The team calculated the cost-effectiveness as the ratio of incremental costs over incremental DALYs averted, discounted at 3% annually.

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The study demonstrated that hydroxyurea at the MTD averted an expected 1.37 DALYs and saved US$ 191 per patient and hydroxyurea at a fixed dose averted 0.80 DALYs per patient at an incremental cost of US$ 2, compared with SCA care without hydroxyurea. It also showed that using the MTD regimen saved 11.2 (95% CI, 11.1-11.4) units of blood per patient, compared with 9.1 (95% CI, 9.0-9.2) units of blood per patient using the fixed-dose regimen.

In threshold analyses for drug price, the investigators showed that hydroxyurea at the MTD was more cost-effective than no hydroxyurea if the cost of hydroxyurea per 500 mg did not exceed US$ 0.36, and the regimen became more cost-effective as the cost of blood for transfusions increased.

“This is the first cost-effectiveness analysis of hydroxyurea in a low-income setting in Africa; however, our findings are consistent with outcomes from wealthier countries,” the authors wrote in their report. “In Tanzania, the drug became substantially cheaper when produced generically in-country, at a cost of US$ 0.09 per 500 mg. With such prices, the net savings per patient should be even larger than in our base case scenario. This underlines previous calls for fair drug pricing and cost-efficient infrastructures for delivering and administering hydroxyurea.”

Disclosures: Some study authors declared affiliations with biotech, pharmaceutical, or device companies. Please see the original reference for a full list of authors’ disclosures. 


Teigen D, Opoka RO, Kasirye P, et al. Cost-effectiveness of hydroxyurea for sickle cell anemia in a low-income African setting: a model-based evaluation of two dosing regimens. Pharmacoeconomics. Published online July 18, 2023. doi:10.1007/s40273-023-01294-3