OBJECTIVES: This study aimed to systematically review the cost-effectiveness analyses of epigenetic drugs, an emerging class of therapies in oncology. METHODS: A systematic search was conducted in Medline, Scopus, and Embase databases for references published from January 2000 to November 2024, reporting an economic evaluation on an epigenetic drug regardless of the oncologic or onco-hematologic indication. Eligible studies were cost-effectiveness analysis or cost-utility analysis reporting outcomes in cost per quality-adjusted life-year (QALY) or cost per life-year gained (LYG). The reporting quality was assessed using the Consolidated Health Economic Evaluation Reporting Standards (CHEERS) checklist and the methodological quality with the ISPOR checklist. All costs were converted into 2023 US dollars using purchasing power parity. RESULTS: Of the 541 articles, abstracts, posters, and letters identified, 19 articles were included, encompassing 32 comparisons across 7 epidrugs (azacitidine, decitabine, panobinostat, ivosidenib, tazemetostat, enasidenib, and vorinostat) in 6 distinct indications in oncology. Overall reporting quality was high, 16 of 19 studies meeting at least 70% of CHEERS recommendations and the relevance and credibility were sufficient. Differences in QALYs ranged from -7.71 to 2.6 QALY and cost differences from -$173 300 to $867 500. Base-case results for the incremental cost-effectiveness ratio in terms of cost per QALY ranged from -$129 848/QALY to $1 323 228/QALY. CONCLUSION: This systematic review offers a comprehensive synthesis of current economic evaluations of epidrugs in oncology. The wide variability in incremental cost-effectiveness ratios and methodological approaches underscores the need for more standardized and robust evaluations to clarify the economic value of epigenetic drugs.
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Peretti et al. (2026) studied this question.
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