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COST-EFFECTIVENESS OF ORPHAN DRUGS FOR LEUKEMIA TREATMENT: A SYSTEMATIC REVIEW
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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.
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2. Delea TE, Amdahl J, Boyko D, et al. (2017). Cost-effectiveness of blinatumomab versus salvage chemotherapy in relapsed or refractory Philadelphia-chromosome-negative B-precursor acute lymphoblastic leukemia from a US payer perspective. J Med Econ 20(9): 911-922.
3. Kloos RQH, van Litsenburg RRL, Wolf S, et al. (2019). A cost-effectiveness analysis of Erwinia asparaginase therapy in children with acute lymphoblastic leukemia. Pediatric Blood & Cancer 66(1): e27458.
4. Delea TE, Zhang X, Amdahl J, et al. (2019). Cost Effectiveness of Blinatumomab Versus Inotuzumab Ozogamicin in Adult Patients with Relapsed or Refractory B-Cell Precursor Acute Lymphoblastic Leukemia in the United States. Pharmacoeconomics 37(9): 1177-1193.
5. Hu X, Wildman KP, Basu S, et al. (2019). The cost-effectiveness of pegaspargase versus native asparaginase for first-line treatment of acute lymphoblastic leukaemia: a UK-based cost-utility analysis. Health Econ Rev 9(1): 40.
6. Wakase S, Teshima T, Zhang J, et al. (2021). Cost-Effectiveness Analysis of Tisagenlecleucel for the Treatment of Pediatric and Young Adult Patients with Relapsed or Refractory B Cell Acute Lymphoblastic Leukemia in Japan. Transplant Cell Ther 27(3): 241.e241-241.e211.
7. Casado LF, Hernández JÁ, Jarque I, et al. (2018). Cost-utility analysis of idelalisib in combination with rituximab in relapsed or refractory chronic lymphocytic leukaemia. European Journal of Haematology 100(3): 264-272.
8. Hatswell AJ, Thompson GJ, Maroudas PA, et al. (2017). Estimating outcomes and cost effectiveness using a single-arm clinical trial: ofatumumab for double-refractory chronic lymphocytic leukemia. Cost Eff Resour Alloc 15(1): 8.
9. Barnes JI, Divi V, Begaye A , et al. (2018). Cost-effectiveness of ibrutinib as first-line therapy for chronic lymphocytic leukemia in older adults without deletion 17p. Blood Advances 2(15): 1946-1956.
10. Vreman RA, Geenen JW, Hövels AM, et al. (2019). Phase I/II Clinical Trial-Based Early Economic Evaluation of Acalabrutinib for Relapsed Chronic Lymphocytic Leukaemia. Appl Health Econ Health Policy 17(6): 883-893.
11. Huntington SF, Torsten SM, Masaquel AS, et al. (2019). Cost-effectiveness of a 24-month fixed duration of venetoclax in combination with rituximab in relapsed or refractory chronic lymphocytic leukemia in the United States. Value in Health 22(2): 78-89.
12. Batty N, Yin Y, Wetzler M (2014). Decitabine is more cost effective than cytarabine and daunorubicin in elderly acute myeloid leukemia patients. Journal of Cancer Research & Therapy 2(4): 68-73.
13. Arenaza A, Diez R, Esteve J, et al. (2019). Cost-Effectiveness Of Midostaurin In The Treatment Of Acute Myeloid Leukemia With The FLT3 Mutation In Spain. Clinicoecon Outcomes Res 19(11): 683-694.
14. Hu Y, Charaan M, van Oostrum I, et al. (2021). The cost-effectiveness of glasdegib in combination with low-dose cytarabine, for the treatment of newly diagnosed acute myeloid leukemia in adult patients who are not eligible to receive intensive induction chemotherapy in Canada. J Med Econ 24(1): 150-161.
15. Mareque M, Montesinos P, Font P, et al. (2021). Cost-Effectiveness Analysis of Gemtuzumab Ozogamicin for First-Line Treatment of Patients with Cd-33 Positive Acute Myeloid Leukaemia in Spain. Clinicoecon Outcomes Res 21(13): 263-277.
16. Patel KK, Zeidan MA, Shallis MR , et al. (2021). Cost-effectiveness of azacitidine and venetoclax in unfit patients with previously untreated acute myeloid leukemia. Blood Advances 5(4): 994-1002.
17. Tallman M, Lo-Coco F, Barnes G , et al. (2015). Cost-Effectiveness Analysis of Treating Acute Promyelocytic Leukemia Patients With Arsenic Trioxide and Retinoic Acid in the United States. Clin Lymphoma Myeloma Leuk 15(12): 771-777.
18. Sergio RR, Lorena GT, Maria JDH, et al. (2020). Cost-effectiveness of the regimen proposed by the International Consortium on Acute Promyelocytic Leukemia for the treatment of newly diagnosed patients with Acute Promyelocytic Leukemia. Hematology, Transfusion and Cell Therapy.
19. Gordois A, Scuffham P, Warren E, et al. (2003). Cost-utility analysis of imatinib mesilate for the treatment of advanced stage chronic myeloid leukaemia. Br J Cancer 89(4): 634-640.
20. Hoyle M, Rogers G, Moxham T, et al. (2011). Cost-Effectiveness of Dasatinib and Nilotinib for Imatinib-Resistant or -Intolerant Chronic Phase Chronic Myeloid Leukemia. Value in Health 14(8): 1057-1067.
21. Rochau U, Kluibenschaedl M, Stenehjem D, et al. (2015). Effectiveness and Cost-Effectiveness of Sequential Treatment of Patients with Chronic Myeloid Leukemia in the United States: A Decision Analysis. Leuk Res Treatment 2015: 982395.
22. Li N, Yang X, Fan L, et al. (2017). Nilotinib versus dasatinib as second-line therapy in patients with Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase who are resistant or intolerant to imatinib: a cost-effectiveness analysis based on real-world data. J Med Econ 20(4): 328-336.
23. Bin W, Maobai L, Te L, et al. (2017). An economic analysis of high-dose imatinib, dasatinib, and nilotinib for imatinib-resistant chronic phase chronic myeloid leukemia in China: A CHEERS-compliant article. Medicine (Baltimore) 96(29): e7445.
24. Muresan B, Mamolo C, Cappelleri JC, et al. (2021). Cost-Effectiveness of Bosutinib for the Treatment of Adult Patients with Chronic Phase Chronic Myeloid Leukemia in the Second-Line Setting. Appl Health Econ Health Policy 1179-1896.