Economic evaluations of nonpharmacological treatments for drug-resistant epilepsy: A systematic review.

This study was undertaken to systematically identify and critically appraise all published full economic evaluations assessing the cost-effectiveness of nonpharmacological interventions for patients with drug-resistant epilepsy. The Population, Intervention, Comparison, Outcome, Study criteria was used to design search strategies for the identification and selection of relevant studies. Literature search was performed using the MEDLINE (via PubMed), Embase, International Health Technology Assessment, National Institute for Health Research Economic Evaluation Database, and Cost-Effectiveness Analysis Registry databases to identify articles published between January 2000 and May 2023. Web of Science was additionally used to perform forward and backward referencing. Title, abstract, and full-text screening was performed by two independent researchers. The Consensus Health Economic Criteria (CHEC) checklist and Consolidated Health Economic Evaluation Reporting Standards (CHEERS) 2022 were applied for quality assessment. A total of 4470 studies were identified, of which 18 met our inclusion criteria. Twelve of the studies conducted model-based economic evaluation, and others were trial-based. Three studies showed that epilepsy surgery was cost-effective in adults, whereas this remained inconclusive for children (two positive, three negative). Three studies showed negative economic outcome for ketogenic diet in children. One of four studies showed positive results for self-management. For vagus nerve stimulation, one study showed positive results in adults and another one negative results in children. One recent study showed cost-effectiveness of responsive neurostimulation (RNS) in adults. Finally, one study showed promising but inconclusive results for deep brain stimulation (DBS). The mean scores for risk of bias assessment (based on CHEC) and for reporting quality (CHEERS 2022) were 95.8% and 80.5%, respectively. This review identified studies that assessed the cost-effectiveness of nonpharmacological treatments in both adults and children with drug-resistant epilepsy, suggesting that in adults, epilepsy surgery, vagus nerve stimulation, and RNS are cost-effective, and that DBS and self-management appear to be promising. In children, the cost-effectiveness of epilepsy surgery remains inconclusive. Finally, the use of ketogenic diet was shown not to be cost-effective. However, limited long-term data were available for newer interventions (i.e., ketogenic diet, DBS, and RNS).

Assessment of Quality of Life 8-Dimension (AQoL-8D): translation, validation, and application in two Dutch trials in patients with epilepsy and schizophrenia.

BACKGROUND: To translate and linguistically validate the Assessment of Quality of Life 8-dimensions (AQoL-8D) for use in the Netherlands and to compare the psychometric properties of AQoL-8D with the EuroQol 5-dimensions 5-levels (EQ-5D-5L) in two patient samples. METHODS: AQoL-8D was translated from English into Dutch. The translated AQoL-8D was then administered alongside the EQ-5D-5L at baseline and follow-up of two Dutch randomized controlled trials among patients with epilepsy and schizophrenia. These data were subjected to a post-hoc analysis assessing the psychometric properties of AQol-8D vis-à-vis EQ-5D-5L in terms of known-groups construct validity, responsiveness, and floor/ceiling effects. RESULTS: In total, 103 epilepsy patients and 99 schizophrenia patients were included in this study. In both datasets, the two instruments discriminated between known-groups, but in schizophrenia, AQoL-8D showed higher responsiveness than EQ-5D-5L, while both instruments showed equal responsiveness in epilepsy. Ceiling effects were only found for EQ-5D-5L in both epilepsy (26.6%) and schizophrenia (6.1%). CONCLUSION: Our results have shown that, among other things, AQoL-8D presents better ability to discriminate between known-groups and shows no ceiling effect. Based on our results, we would recommend the use of AQoL-8D in addition to EQ-5D-5L in trials assessing patient's quality of life in patients with epilepsy or schizophrenia.

Economic evaluation of deep brain stimulation compared with vagus nerve stimulation and usual care for patients with refractory epilepsy: A lifetime decision analytic model.

OBJECTIVES: This study was undertaken to estimate the cost-effectiveness of deep brain stimulation (DBS) compared with vagus nerve stimulation (VNS) and care as usual (CAU) for adult patients with refractory epilepsy from a health care perspective using a lifetime decision analytic model. METHODS: A Markov decision analytic model was constructed to estimate the lifetime cost-effectiveness of DBS compared with VNS and CAU. Transition probabilities were estimated from a randomized controlled trial, and assumptions were made in consensus with an expert panel. Primary outcomes were expressed as incremental costs per quality-adjusted life-year (QALY) and per responder. Univariate and probabilistic sensitivity analyses were conducted to characterize parameter uncertainty. RESULTS: In DBS, 28.4% of the patients were responders, with an average of 21.38 QALYs per patient and expected lifetime health care costs of €187 791. VNS had fewer responders (22.3%), fewer QALYs (20.70), and lower lifetime costs (€156 871). CAU had the fewest responders (6.2%), fewest QALYs (18.74), and lowest total health care costs (€64 670). When comparing with CAU, incremental cost-effectiveness ratios (ICERs) showed that costs per QALY gained were slightly lower for DBS (€46 640) than for VNS (€47 155). When comparing DBS with VNS, an incremental cost per additional QALY gained of €45 170 was found for DBS. Sensitivity analyses showed that ICERs were heavily dependent on assumptions regarding loss to follow-up in the respective clinical trial. SIGNIFICANCE: This study suggests that, given current limited evidence, VNS and DBS are potentially cost-effective treatment strategies compared to CAU for patients with refractory epilepsy. However, results for DBS were heavily impacted by assumptions made to extrapolate nonresponse from the original trial. More stringent assumptions regarding nonresponse resulted in an ICER just above an acceptable willingness to pay threshold. Given the uncertainty surrounding the effectiveness of DBS and the large impact of assumptions related to nonresponse, further empirical research is needed to reduce uncertainty.

ZMILE, a multicomponent self-management intervention for adults with epilepsy: Rationale and description of the intervention.

OBJECTIVE: In this paper, we aim to provide a comprehensive description of the multicomponent self-management intervention for adults with epilepsy, ZMILE. RATIONALE OR THEORY: Acquiring self-management skills has been shown to play a vital role in enabling patients with epilepsy overcoming (health-related) struggles in daily life and coping with limitations their condition poses on them. ZMILE is a course consisting of education (to increase concordance to treatment), goal-setting (proactive coping), and self-monitoring. RESOURCES NEEDED: The course is guided by two nurse practitioners and each patient is allowed to bring one family member or friend. Self-monitoring plays an important role and can be done through e-Health tools or written diaries. PROCESSES INVOLVED: During and after the course, patients are required to work toward a personally defined goal using a five-step approach by means of pro-active coping. Moreover, patients are expected to use self-monitoring tools to reflect on their own behavior and identify ways to optimize medication intake when required. QUANTIFICATION: ZMILE is provided in an outpatient setting over five weekly group sessions and one booster session. From the start, patients are encouraged to set individual goals. Each group session will have a different theme but part of every session is reflecting on personal goals and to learn from eachother. CONCLUSIONS: The ZMILE-intervention has been evaluated and may be a promising intervention in terms of effectiveness and feasibility for adults with epilepsy, relatives, and professionals. We present the adapted version which can be implemented in clinical practice.

Economic evaluation of brief cognitive behavioural therapy for social activation in recent-onset psychosis.

BACKGROUND: In schizophrenia spectrum disorders, negative symptoms (e.g. social withdrawal) may persist after initial treatment with antipsychotics, much affecting the quality of life (QOL) of patients. This health-economic study evaluated if a dedicated form of cognitive behaviour therapy for social activation (CBTsa) would reduce negative symptoms and improve QOL in an economically sustainable way. METHODS: A health-economic evaluation was conducted alongside a single-blind randomised controlled trial in two parallel groups: guideline congruent treatment as usual (TAU; n = 50) versus TAU augmented with adjunct CBTsa (n = 49). Outcomes were PANSS negative symptom severity and EQ-5D quality adjusted life years (QALYs) gained. The health-economic evaluation was conducted both from the societal and the health sector perspective. RESULTS: Both conditions showed improvement in the respective outcomes over the follow-up period of six months, but QALY gains were significantly higher in the CBTsa condition compared to the TAU condition. Treatment response rate (i.e. ≥ 5-point decrease on the PANSS) was not significantly different. However, the add-on CBT intervention was associated with higher costs. This did not support the idea that CBTsa is a cost-effective adjunct. Various sensitivity analyses attested to the robustness of these findings. CONCLUSIONS: In the Dutch context where TAU for psychosis is guideline congruent and well implemented there appears no added value for adjunct CBTsa. In other settings where the treatment for the schizophrenia spectrum disorders solely relies on antipsychotics, add-on CBTsa may lead to clinically superior outcomes, but it should still be evaluated if adjunct CBTsa therapy is a cost-effective alternative. TRIAL REGISTRATION: ClinicalTrials.gov registry under NCT03217955.