COST-EFFECTIVENESS OF ORPHAN DRUGS FOR LEUKEMIA TREATMENT: A SYSTEMATIC REVIEW

Introduction: Leukemia is a rare disease related to hematologic cancer stemming from the bone marrow. The Vietnam Ministry of Health (MOH) promulgated the Orphan Drugs List, in which there were 37 orphan drugs indicated for leukemia. This study aimed to systematically review all studies on the cost-effectiveness analysis (CEA) of these orphan drugs in leukemia treatment. Materials and methods: This study conducted a systematic review on all studies published till August 2021 on the National Center for Biotechnology Information (NCBI), SpringerLink, and Biomed Central. 23 studies were selected in this systematic review, which were studies that had available full-texts, were written in English, aimed to analyze the cost-effectiveness of leukemia drugs listed on the Vietnam MOH’s Orphan Drugs List. The results were presented by describing CEA findings by five different leukemia types, with a focus on the incremental cost-effectiveness ratio (ICER) of each orphan drug and the comparison to the willingness-to-pay (WTP) threshold. All the cost currency values were converted to USD in 2021 to make comparison. Results: Of 37 drugs on the Vietnam MOH’s orphan drug list, 24 drugs were cost-effectiveness analyzed with available full-texts. Of 23 selected studies in the review, there were 10 studies regarding lymphocytic leukemia (43.5%) and 13 studies regarding myeloid leukemia (56.5%). 60.9% studied on relapsed/refractory patients, 39.1% conducted cost-analyses with a social perspective, and 47.7% used overall survival combined with progression-free survival (OS-PFS) as a clinical endpoint. Quality-adjusted life year (QALY) was used as an effectiveness measurement unit in 65.2% of the total selected studies. 15 studies made a conclusion that their studied orphan drugs were cost-effective. 100% of the studies regarding acute lymphoblastic leukemia showed the orphan drugs of interest were cost-effective (ICER < WTP threshold); while about 50% of the studies regarding other leukemia types showed the orphan drugs of interest were not cost-effective (ICER > WTP threshold) and thus required suitable financial aid. Conclusion: This study provided information on the cost-effectiveness of 24 out of 37 orphan drugs for leukemia treatment listed on the Vietnam MOH's Orphan Drugs List. These orphan drugs could be considered as a financial burden for leukemia patients and other potential payers such as the Vietnam Social Security due to their considerably high cost.