Cost-Effectiveness of Epigenetic Drugs in Oncology: A Systematic Review of Economic Evaluations

Author(s)

Lou-Anne Peretti, PharmD1, Isabelle BORGET, PharmD, PhD2, Sophie Postel-Vinay, PhD3, Christophe Willekens, PhD3, JULIA BONASTRE, PhD4, Arnaud Pagès, PharmD, PhD5.
1Student/Trainee, Gustave Roussy, Villejuif, France, 2Master2 Market-Access and Economic Evaluation, ORSAY, France, 3Gustave Roussy, Villejuif, France, 4Gustave roussy, VILLEJUIF, France, 5Gustave roussy, Villejuif, France.
OBJECTIVES: Epigenetic drugs (epidrugs) are a novel class of targeted therapies, primarily used in oncology, offering promising therapeutic advances. However, their cost-effectiveness remains uncertain; the methodologies and quality of related economic evaluations are poorly characterized. This study aimed to systematically review the cost-effectiveness analyses (CEAs) of epidrugs used in oncology.
METHODS: A systematic search was conducted in Medline, Scopus, Embase and the ISPOR database for articles, abstracts, letters and posters 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, written in English or French were cost-effectiveness analysis (CEA) or cost-utility analysis (CUA) reporting outcomes in cost/QALY (Quality Adjusted Life Years) or cost/LYG (Life Years Gained). Study quality was assessed using the CHEERS checklist. All costs were converted into 2023 US dollars using PPA (Purchasing Power Parity).
RESULTS: Of 541 articles, abstracts, posters and letters identified, 27 analyses were included, encompassing 45 comparisons across seven epidrugs (azacitidine, decitabine, panobinostat, ivosidenib, tazemetostat, enasidenib, vorinostat) in six distinct indications (acute myeloid leukemia, myelodysplastic syndromes, multiple myeloma, cholangiocarcinoma, follicular lymphoma, and sezary syndrome and mycosis fungoides). Overall reporting quality was high, with 17 out of 27 studies meeting at least 70% of CHEERS recommendations. Differences in QALYs ranged from -7.71 to 2.26 QALY and cost differences from -$173,300 to $867,500. Base-case results for the incremental cost-effectiveness ratio (ICER) in terms of cost per QALY ranged from -$129,848/QALY to $1,323,228/QALY for full articles and from -$342,177/QALY to $582,459/QALY for congress abstracts, posters, and letters.
CONCLUSIONS: This systematic review offers a comprehensive synthesis of current economic evaluations of epidrugs in oncology. The wide variability in ICERs and methodological approaches underscores the need for more standardized and robust evaluations to clarify the economic value of this innovative drug class.

Conference/Value in Health Info

2025-11, ISPOR Europe 2025, Glasgow, Scotland

Value in Health, Volume 28, Issue S2

Code

EE248

Topic

Economic Evaluation

Disease

No Additional Disease & Conditions/Specialized Treatment Areas, Oncology

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