Trial‐based cost‐effectiveness analyses (CEAs) are an important source of evidence in the assessment of health interventions. In these studies, cost and effectiveness outcomes are commonly measured ...at multiple time points, but some observations may be missing. Restricting the analysis to the participants with complete data can lead to biased and inefficient estimates. Methods, such as multiple imputation, have been recommended as they make better use of the data available and are valid under less restrictive Missing At Random (MAR) assumption. Linear mixed effects models (LMMs) offer a simple alternative to handle missing data under MAR without requiring imputations, and have not been very well explored in the CEA context. In this manuscript, we aim to familiarize readers with LMMs and demonstrate their implementation in CEA. We illustrate the approach on a randomized trial of antidepressants, and provide the implementation code in R and Stata. We hope that the more familiar statistical framework associated with LMMs, compared to other missing data approaches, will encourage their implementation and move practitioners away from inadequate methods.
A recent randomized controlled trial demonstrated an overall survival benefit to the addition of hyperthermic intraperitoneal chemotherapy (HIPEC) to neoadjuvant chemotherapy (NACT) for stage III ...epithelial ovarian cancer (EOC). The objective of the current study was to quantify the cost-effectiveness of HIPEC in this setting.
A decision analytic cost-effectiveness model was designed from a payer perspective to compare 2 surgical management strategies for EOC: (1) interval cytoreductive surgery (ICS); (2) ICS + HIPEC. Overall survival and ostomy rates with HIPEC were modeled from published studies. We assumed that 25% of each arm would later undergo secondary cytoreductive surgery, with the ICS arm eligible for HIPEC at that time. Costs were obtained from Medicare data, published studies, and the financial department of an academic hospital. Quality of life was not different between the arms; we assigned utilities based on a prior time-trade off study of ovarian cancer treatment. A Monte Carlo probabilistic sensitivity analysis was performed in the base case; primary outcome was the incremental cost-effectiveness ratio (ICER), expressed in 2017 US Dollars/quality-adjusted life years (QALYs).
ICS was the least costly strategy at $78,849, compared to ICS + HIPEC at $79,954. ICS + HIPEC was more effective than ICS (2.9 QALYs versus 2.45 QALYs for ICS). ICS + HIPEC was highly cost-effective, with an ICER of $2436/QALY compared to ICS. In one-way sensitivity analyses, probability of ostomy reversal and use of HIPEC at secondary cytoreduction did not substantially impact the cost-effectiveness of ICS + HIPEC.
ICS + HIPEC constitutes cost-effective management of stage III EOC when NACT is performed.
•HIPEC in addition to interval cytoreduction increases cost of care for stage III epithelial ovarian cancer.•The addition of HIPEC at interval cytoreduction for stage III epithelial ovarian cancer is cost-effective.•The ICER for HIPEC at ICS is $2436/QALY compared to ICS alone, which is far below the accepted threshold.
We document three remarkable features of the Opower program, in which social comparison-based home energy reports are repeatedly mailed to more than six million households nationwide. First, initial ...reports cause high-frequency "action and backsliding," but these cycles attenuate over time. Second, if reports are discontinued after two years, effects are relatively persistent, decaying at 10-20 percent per year. Third, consumers are slow to habituate: they continue to respond to repeated treatment even after two years. We show that the previous conservative assumptions about post-intervention persistence had dramatically understated cost effectiveness and illustrate how empirical estimates can optimize program design.
Background
To accelerate the clinical translation of tumor‐infiltrating lymphocytes (TILs) biomarkers for guiding chemotherapy de‐escalation in early‐stage triple‐negative breast cancer (TNBC), ...cost‐effectiveness evidence is essential but has not been investigated. We intend to evaluate the cost‐effectiveness of using TILs to guiding chemotherapy de‐escalation in patients with early‐stage TNBC from the perspective of the Chinese health service system.
Methods
The hybrid decision‐tree‐Markov model was designed to compare the cost‐effectiveness of cytotoxic chemotherapy guided by whether TILs assay was performed in 50‐year‐old female patients with early‐stage TNBC over a lifetime horizon. In Strategy (1), if TILs testing was performed, patients with TILs values exceeding 30% could be spared from chemotherapy. In Strategy (2), where no TILs testing was performed, all patients were administered chemotherapy following China's clinical practices. Based on the algorithm built by Guyot, the individual patient data were reconstructed from the published Kaplan–Meier curves, and the survival functions were calculated by parametric methods. Cost estimates were valued in Chinese yuan (as per rates in 2022).
Results
In 50‐year‐old female patients with early‐stage TNBC, Strategy (1), which employs TILs testing to guide cytotoxic chemotherapy yielded an additional 0.47 quality‐adjusted life years (QALYs) and saved 40,976 yuan, with an incremental cost‐effectiveness ratio (ICER) of −87,182.98 yuan per QALY gained compared with Strategy (2). This indicates that compared with Strategy (2), Strategy (1) is the dominant scheme. The results were sensitive to utility parameters, discount rates, and treatment costs after relapse. At a willingness‐to‐pay threshold of 85,700 yuan (based on GDP per capita) per QALY, the probability of TILs being cost‐effective was almost 100%.
Conclusions
The application of biomarkers (TILs) to guide decisions for chemotherapy de‐escalation is a cost‐effective strategy for early‐stage TNBC patients and deserves to be widely promoted in clinical practice.
Tumor‐infiltrating lymphocytes (TILs) testing is a cost‐effective strategy for guiding chemotherapy de‐escalation in early‐stage triple‐negative breast cancer (TNBC) patients, according to a study from Chinese health care providers. Using TILs testing can spare patients unnecessary chemotherapy while adding an additional 0.47 quality‐adjusted life years (QALYs) and saving costs. The study found that using TILs testing to guide chemotherapy in early‐stage TNBC patients was almost 100% cost‐effective at a willingness‐to‐pay threshold of 85,700 yuan per QALY. This highlights the importance of incorporating cost‐effectiveness evidence into clinical decisions for biomarker‐guided chemotherapy de‐escalation.
To report the cost of target lesion revascularisation procedures (TLR) for femoropopliteal peripheral artery disease (PAD) following stenting, from a healthcare payer’s perspective.
European ...multicentre study involving consecutive patients requiring femoropopliteal TLR (January 2017 – December 2021). The primary outcome was overall cost (euros) associated with a TLR procedure from presentation to discharge. Exact costs per constituent, clinical characteristics, and early outcomes were reported.
This study included 482 TLR procedures (retrospectively, 13 hospitals, six countries): 56% were female, mean age was 75 ± 2 years, 61% were Rutherford class 5 or 6, 67% had Tosaka class 3 disease, and 16% had common femoral or iliac involvement. A total of 52% were hybrid procedures and 6% involved open surgery only. Technical success was 70%, 30 day mortality rate was 1%, and the 30 day major amputation rate was 4%. Most costs were for operating time during the TLR (healthcare professionals’ salaries, indirect and estate costs), with a mean of: €21 917 ± €2 110 for all procedures; €23 337 ± €8 920 for open procedures; €12 903 ± €3 108 for endovascular procedures; and €22 806 ± €3 977 for hybrid procedures. In a regression analysis, procedure duration was the main parameter associated with higher overall TLR costs (coefficient, 2.77; standard error, 0.88; p < .001). The mean cost per operating minute of TLR (indirect, estate costs, all salaried staff present included) was €177 and the mean cost per night stay in hospital (outside intensive care unit) was €356. The mean cost per overnight intensive care unit stay (minimum of 8 hours per night) was €1 193.
The main driver of the considerable peri-procedure costs associated with femoropopliteal TLR was procedure time.
Despite the central role of the threshold incremental cost-effectiveness ratio (ICER), or lambda (
λ), in the methods and application of cost-effective analysis (CEA), little attention has been given ...to the determining the value of
λ. In this paper we consider ‘what explains the silence of the
λ’? The concept of the threshold ICER is critically appraised. We show that there is ‘silence of the
λ’ with respect to justification of the value of ICER thresholds, their use in decision-making and their relationship to the opportunity cost of marginal resources. Moreover, the ‘sound of silence’ extends to both ‘automatic cut-off’ and more sophisticated approaches to the use of
λ in determining recommendations about health care programs. We argue that the threshold value provides no useful information for determining the efficiency of using available resources to support new health care programs. On the contrary, the threshold approach has lead to decisions that resulted in increased expenditures on health care programs and concerns about the sustainability of public funding for health care programs without any evidence of increases in total health gains. To improve efficiency in resource allocation, decision-makers need information about the opportunity costs of programs.
PDF
