An Economic Systematic Literature Review for Hypertrophic Obstructive Cardiomyopathy

Speaker(s)

Krause T1, Slater D2, Boulton E2, Luedke H2, Hurst M3, Maksabedian Hernandez EJ4, Pashley A5
1Bristol Myers Squibb, LONDON, LON, UK, 2Costello Medical, Cambridge, Cambridgeshire, UK, 3Bristol Myers Squibb, Uxbridge, LON, UK, 4Bristol Myers Squibb, Princeton, NJ, USA, 5Costello Medical, Cambridge, CAM, UK

Presentation Documents

OBJECTIVES: Obstructive hypertrophic cardiomyopathy (HCM) is a chronic, progressive disease characterized by left ventricular (LV) hypertrophy and fibrosis due to sarcomeric dysfunction. A systematic literature review (SLR) was conducted to identify economic evaluations (EEs), and studies reporting on health state utility values (HSUVs) and healthcare cost and resource use (HCRU) data associated with obstructive HCM.

METHODS: This SLR was conducted in accordance with Cochrane Collaboration guidance. Literature searches were performed across MEDLINE, Embase, EconLit, PsycINFO and the Health Technology Assessment (HTA) databases from database inception to December 2021 and updated in July 2023. Relevant congress proceedings from 2019–2023, HTA/economic websites and SLR and HTA bibliographies were manually searched. Record screening was performed by 2 independent researchers, using the predefined inclusion and exclusion criteria with any disagreements resolved by a third reviewer.

RESULTS: 954 and 403 unique records were identified from literature searches up to December 2021 and the July 2023 update following deduplication, respectively, which were screened against pre-determined eligibility criteria. Including manual supplementary searches, a total of 8 unique EEs, 2 HSUV studies and 57 HCRU studies were included in this SLR. All 8 EEs assessed mavacamten, a cardiac myosin inhibitor. One HSUV study elicited EQ-5D data from a randomized trial of mavacamten; the remaining elicited EQ-5D pre- and post-septal myectomy. Identified HCRU data were heterogeneous, most commonly reporting on medication use, surgical procedures, hospital stay and readmissions, and permanent pacemaker implantation. No indirect HCRU data were identified.

CONCLUSIONS: The SLR identified a substantial volume of HCRU evidence describing the economic burden of obstructive HCM on different healthcare systems, although data on indirect impacts are needed to characterize the societal impact of the condition. This contrasts with the paucity of cost-effectiveness and HSUV evidence identified, which reflects the historic lack of innovation in treatments for this condition.

Code

EE200

Topic

Economic Evaluation, Study Approaches

Topic Subcategory

Literature Review & Synthesis

Disease

Cardiovascular Disorders (including MI, Stroke, Circulatory), No Additional Disease & Conditions/Specialized Treatment Areas