Economic Evaluations of Treatments for Acquired Hemophilia: A Systematic Literature Review

Author(s)

Ghosh B1, Rai D2, Choudhury A2, Dixit K2, Kaur G2, Aman MS3, Mahon R1
1University of Galway, Galway, Galway, Ireland, 2PharmaQuant Insights Pvt. Ltd., Kolkata, India, 3PharmaQuant Insights Pvt. Ltd., Kolkata , WB, India

OBJECTIVES: Acquired hemophilia (AH) is a rare autoimmune disorder characterized by spontaneous bleeding in individuals with no family history. Due to disease’s rarity and the paucity of emerging treatments there is no definitive approach to the economic evaluation of treatments for AH. This review aims to identify and appraise the health-economic evaluation methods used in treatments for AH.

METHODS: A systematic literature review was conducted using 11 databases including EMBASE, MEDLINE, CINAHL, Web of Science and Scopus, conference abstracts, HTA databases and trial registries through January 2024 to identify publications reporting economic evaluations in AH. This systematic review was based on The Campbell and Cochrane Economics Methods Group (C-CEMG) framework for integrated full systematic review of economic evidence.

RESULTS: Three studies were identified, reporting model-based economic evaluations from the USA perspective. Two of these studies used a cost-effectiveness (CE) and one used a cost-minimization (CM) framework. The CM study used a decision tree analysis, while one CE study used a Markov analysis, and another used discrete event simulation. No inflation adjustment or discounting were reported in any of the studies. Probabilistic sensitivity analyses were used for the CE studies and two-way sensitivity analysis was used for the CM study. The time horizon varied across all the studies ranging from 5 days to lifetime horizon. The common treatments observed across the studies were recombinant pVIII and FVIIa, while other treatments included in the model were aPCC, AICC and bypassing agent. In terms of cost-effectiveness, the recombinant treatment options showed inconsistent results.

CONCLUSIONS: Despite being from the same setting, i.e. USA, there was some heterogeneity in terms of study methodology. Variations regarding the data inputs for the same parameters, time horizon and the included comparator (non-AH) were also recorded. Thus, there are uncertainties for decision makers for identifying the most effective treatment option for AH.

Conference/Value in Health Info

2024-11, ISPOR Europe 2024, Barcelona, Spain

Value in Health, Volume 27, Issue 12, S2 (December 2024)

Code

EE558

Topic

Economic Evaluation, Study Approaches

Topic Subcategory

Literature Review & Synthesis

Disease

Rare & Orphan Diseases, Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)

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