Cost‐effectiveness of implantable ventricular assist devices in older children with stable, inotrope‐dependent dilated cardiomyopathy

Abstract

Background: In a stable, inotrope-dependent pediatric patient with dilated cardiomyopathy, we evaluated the cost-effectiveness of continuous-flow VAD implantation compared to a watchful waiting approach using chronic inotropic therapy. Methods:We used a state-transition model to estimate the costs and outcomes of 14-year-old (INTERMACS profile 3) patients receiving either VAD or watchful waiting.We measured benefits in terms of lifetime QALYs gained. Model inputs were taken from the literature. We calculated the ICER, or the cost per additional QALY gained, of VADs and performed multiple sensitivity analyses to test how our assumptions influenced the results.Results: Compared to watchful waiting, VADs produce 0.97 more QALYs for an additional $156 639, leading to an ICER of $162 123 per QALY gained from a healthcare perspective. VADs have 17% chance of being cost-effective given a cost-effectiveness threshold of $100 000 per QALY gained. Sensitivity analyses suggest that VADs can be cost-effective if the costs of implantation decrease or if hospitalization costs or mortality among watchful waiting patients is higher.Conclusions: As a bridge to transplant, VADs provide a health benefit to children who develop stable, inotrope-dependent heart failure, but immediate implantation is not yet a cost-effective strategy compared to watchful waiting based on commonly used cost-effectiveness thresholds. Early VAD support can be cost-effective in sicker patients and if device implantation is cheaper. In complex conditions such as pediatric heart failure, cost-effectiveness should be just one of many factors that inform clinical decision-making.

A systematic literature review of economic evaluations and cost-of-illness studies of inherited cardiomyopathies

“…A total of 11 EEs were identified, of which four studies focused on DCM and five on HCM [ 24 – 34 ]. Two studies included both types of CMs, of which one mainly focussed on DCM patients [ 32 , 34 ].…”

“…Other perspectives were a healthcare system perspective ( n = 2), and a payer ( n = 2) or a provider perspective ( n = 2). Two studies analysed cost-effectiveness from both the societal and healthcare system perspective [ 24 , 31 ]. The most frequently used study design was a model-based analysis ( n = 9); the exceptions were two trial-based analyses [ 32 , 34 ].…”

“…One trial-based analysis additionally included a model simulation to account for a longer time period than investigated in their trial [ 32 ]. As the primary outcome, QALYs were used in eight studies [ 24 – 29 , 31 , 32 ]. Two studies chose different outcomes, such as life years saved/gained (LYS/LYG) ( n = 1) or perceived personal control and patient satisfaction ( n = 1) [ 33 , 34 ].…”

“…Figure 3 shows the cost-effectiveness plane, including all reported ICERs using QALYs as outcome. Most studies chose a WTP threshold of either € 100,000 or € 50,000 per additional QALY gained [ 24 – 30 ]. The results of most interventions lie in the upper quadrant, indicating more effects but also higher costs relative to the comparator.…”

“…Avanceña et al. looked at VADs in children with stable, inotrope-dependent HF and compared the surgical addition of VADs to watchful waiting, indicating that VADs are neither cost-effective from a healthcare nor from a societal perspective at a WTP of $100,000/QALY [ 24 ].…”