OBJECTIVES: A targeted literature review (TLR) was undertaken to collect data inputs and assumptions required to develop a cost-effectiveness model (CEM) for obstructive hypertrophic cardiomyopathy (oHCM).

METHODS: PubMed and EMBASE were searched using key terms including HCM, quality-of-life, cost-effectiveness, cost and resource use, and mortality. Pharmacologic therapies for oHCM were searched on clinicaltrials.gov and clinical evidence was included from associated publications. Relevant analogues were searched to address any oHCM-specific data gaps.

RESULTS: Eight publications were included. Clinical efficacy, demographics, baseline characteristics of oHCM patients, utility values for scenario analysis, and clinical effectiveness of standard-of-care therapy have been reported in clinical trials (PMIDs: 32871100; 34911641; 34915981), and utility values are available from a CEM study (PMID: 27039128). Heart failure has been used as an analogue for oHCM as it provides disaggregated utility values by NYHA class to model health states. A 5-state Markov model (NYHA I-IV, death) was derived from an appraisal of tafamidis (NICE, 2020), which allows for modelling between disease progression and clinical outcomes. Cost and resource use in a real-world oHCM population were found but costs were not broken down by NYHA class (PMIDs: 34493144; 35230625; doi: 10.1016/j.ahjo.2022.100089). Mortality data can be derived from a recent abstract reporting a difference in all-cause mortality by NYHA class, although impact of therapies on NYHA class and associated outcomes was not assessed. Major gaps in published literature for HCM included utility values and costs and resource disaggregated by NYHA I-IV, disutility values for adverse events, healthcare-related and non-healthcare-related resource use and costs, impact of NYHA class on mortality, and impact of therapies on NYHA class and associated outcomes.

CONCLUSIONS: Data from this TLR will be used to develop a CEM for HCM. For identified gaps, research is warranted to generate evidence to help build robust CEMs for newer HCM pharmacotherapies, such as cardiac myosin inhibitors.