Cost Effectiveness and Resource Allocation最新文献

Background: The prominent efficacy in terms of increasing progression-free survival (PFS) of Daratumumab, Lenalidomide and dexamethasone (DRd) triplet therapy versus Carfilzomib, Lenalidomide and dexamethasone (KRd) was proven previously in relapsed-refractory multiple myeloma (RRMM). However, the cost effectiveness of DRd versus KRd is unknown.

Methods: We developed a Markov model by using an Iranian payer perspective and a 10-year time horizon to estimate the healthcare cost, Quality-adjusted life years (QALYs) and life years gain (LYG) for DRd and KRd triplet therapies. Clinical data were obtained from meta-analyses and randomized clinical trials (RCTs). One-way and probabilistic sensitivity analysis were performed to assess model uncertainty. Budget impact analysis of 5 years of treatment under the DRd triplet therapy was also analysed.

Results: DRd was estimated to be more effective compared to KRd, providing 0.28 QALY gain over the modelled horizon. DRd-treated patients incurred $264 in total additional costs. The incremental cost utility ratio (ICUR) and cost effectiveness ratio (ICER) were $956/QALY and $472/LYG respectively. The budget impact analysis indicates that adding Daratumumab to Lenalidomide and dexamethasone regimen, in the first 5 years, will increase the healthcare system's expenses by $6.170.582.

Conclusion: DRd triplet therapy compared to KRd is a cost-effective regimen for RRMM under Iran willingness-to-pay threshold.

Background: The study aimed to examine the direct medical cost and impact of tocilizumab (TOZ) versus adalimumab (ADM) and etanercept (ETC) on reducing the levels of two inflammatory markers (e.g., C-reactive protein (CRP) and erythrocyte sedimentation rate (ESR)) among patients with rheumatoid arthritis (RA) using real-world data from Saudi Arabia.

Method: This was a single-center retrospective cohort study in which data for biologic-naïve RA patients aged ≥ 18 years and treated with TOZ, ADM, ETC were retrieved from the electronic medical records (EMRs) of a university-affiliated tertiary care center in Riyadh, Saudi Arabia. Patients were followed up at least one year after the treatment initiation. Bottom-up microcosting was utilized to estimate the direct medical costs. Additionally, inverse probability treatment weighting and bootstrapping with 10,000 replications were conducted to generate 95% confidence levels for costs and the mean reductions in CRP and ESR levels.

Results: The number of patients who met the inclusion criteria and were included in the analysis was 150 patients (TOZ (n = 56), ADM (n = 41), ETC (n = 53)). Patients on TOZ had 3.96 mg/L (95% CI: -0.229-4.95) and 11.21 mm/hr (95% CI: 10.28-18.11) higher mean reductions in the CRP and ESR levels compared to their counterparts on ADM, ETC, respectively. However, this was associated with mean annual incremental costs of USD 10,087.88 (95% CI: 9494.50-11,441.63) in all cost-effectiveness bootstrap distributions.

Conclusion: Tocilizumab has shown better effectiveness in reducing the levels of CRP and ESR but with higher costs. Future studies should examine whether the reduction of these two inflammatory markers is associated with quality-adjusted life years (QALYs) gains.

Background: There is limited evidence-informed guidance on TISP processes for countries where health technology assessment (HTA) is in a nascent phase. We aimed to explore the range of topic identification, selection and prioritization (TISP) processes and practices for HTA in selected countries and identify aspects relevant to emerging HTA systems.

Methods: This mixed design study included a systematic literature review, an electronic survey, and individual interviews. We conducted a systematic literature review with criteria that were developed a priori to identify countries deemed to have a recently formalized HTA system. Based on the literature review, a twenty-three item online survey was shared with the identified countries, we completed follow-up interviews with ten participants who have experience with HTA. We analyzed documents, survey responses and interview transcripts thematically to identify lessons related to TISP processes and practices.

Results: The literature review identified 29 nine candidate countries as having a "potential" recently formalized HTA system. Twenty-one survey responses were analyzed and supplemented with ten individual interviews. We found variation in countries' approaches to TISP - particularly between pharmaceutical and non-pharmaceutical interventions. Results indicate that TISP is heavily driven by policy makers, expert involvement, and to a lesser extent, relevant stakeholders. The use of horizon-scanning and early warning systems is uncommon. Interviewee participants provided further insight to the survey data, reporting that political awareness and an institutional framework were important to support TISP. TISP can be optimized by stronger national regulations and legislative structures, in addition to education and advocacy about HTA among politicians and decision-makers. In some settings regional networks have been useful, particularly in the development of TISP guidelines and methodologies. Additionally, the technical capacity to conduct TISP, and access to relevant local data were factors limiting TISP in national settings. Increased network collaboration and capacity building were reported as future needs.

Conclusions: This study provides current insights into a topic where there is limited published peer reviewed literature. TISP is an important first step of HTA, and topics should be selected and prioritized based on local need and relevance. The limited capacity for TISP in settings where HTA is emerging may be supported by local and international collaboration to increase capacity and knowledge. To succeed, both TISP and HTA need to be embedded within national health care priority setting and decision-making. More in-depth understanding of where countries are situtated in formalizing the TISP process may help others to overcome factors that facilitate or hinder progress.

Preference elicitation is widely used within health economic evaluations to inform coverage decisions. However, coverage decisions involve questions of social justice and it is unclear what role empirical evidence about preferences can play here. This study reviews the prevalent normative frameworks for using population-based preference elicitation and the criticisms they face, and proposes an alternative based on constitutional economics. The frameworks reviewed include a supposedly value-neutral framework of preferences as predictors of choice, preference utilitarian frameworks that aim to maximize preference satisfaction, and substantive consequentialist frameworks that aim to maximize happiness, health, or capabilities. The proposed alternative implements the idea that indices of social value are tools for conflict resolution, rather than tools for maximization. Preference elicitation is used for validating values generated by multi-criteria decision analysis results within representative processes of stakeholder deliberation.

Background: Combined serplulimab and chemotherapy demonstrated improved clinical survival outcomes in patients with advanced esophageal squamous cell carcinoma (ESCC) and PD-L1 combined positive scores (CPS) ≥ 1. The present study aimed to evaluate the economic viability of integrating serplulimab in combination with chemotherapy as a potential therapeutic approach for treating ESCC in China.

Methods: A Markov model was constructed to evaluate the economic and health-related implications of combining serplulimab with chemotherapy. With the incremental cost-effectiveness ratio (ICER), costs and results in terms of health were estimated. For assessing parameter uncertainty, one-way and probabilistic sensitivity studies were carried out.

Results: The combination of serplulimab and chemotherapy yielded incremental costs and QALYs of $3,163 and 0.14, $2,418 and 0.10, and $3,849 and 0.15, respectively, for the overall population as well as patients with PD-L1 CPS1-10 and PD-L1 CPS ≥ 10. This corresponds to ICER values per QALY of $23,657, $23,982, and $25,134. At the prespecified WTP limit, the probabilities of serplulimab with chemotherapy being the preferred intervention option were 74.4%, 61.3%, and 78.1% for the entire patient population, those with PD-L1 1 ≤ CPS < 10, and those with PD-L1 CPS ≥ 10, respectively. The stability of the presented model was confirmed through sensitivity studies.

Conclusions: In conclusion, the combination of Serplulimab and chemotherapy showed excellent cost-effectiveness compared to chemotherapy alone in treating PD-L1-positive patients with ESCC in China.

Background: Programmed cell death protein 1 (PD-1) monoclonal antibody, pembrolizumab, is a promising drug for platinum-pretreated, recurrent or metastatic nasopharyngeal cancer (NPC). We aimed to assess the cost-effectiveness of pembrolizumab compared with chemotherapy for Chinese patients in this NPC.

Methods: The cost-effectiveness of pembrolizumab versus chemotherapy was evaluated using a partitioned survival model with a 5-year boundary. Efficacy and toxicity data were derived from the KEYNOTE-122 trials. Economic indicators including life-years (LYs), quality-adjusted life-years (QALYs), incremental cost-effectiveness ratio (ICER), and lifetime cost were used. One-way analysis and probabilistic sensitivity analysis (PSA) were performed to explore the uncertainties. Additionally, various scenario analyses, including different pembrolizumab price calculations and discount rates were performed.

Results: Pembrolizumab or chemotherapy alone respectively yielded 2.82 QALYs (3.96 LYs) and 2.73 QALYs (3.93 LYs) with an ICER of $422,535 per QALYs ($1,232,547 per LYs). This model was primarily influenced by the price of pembrolizumab. Furthermore, PSA indicated that pembrolizumab had none probability of being cost-effective compared with chemotherapy at a willingness-to- pay (WTP) of $38223. Scenario analyses revealed that irrespective of any potential price reduction or adjustments in the discount rate, no discernible impact on the ultimate outcome was observed.

Conclusion: Pembrolizumab was less cost-effective for patients with platinum-pretreated, recurrent or metastatic NPC compared with chemotherapy in China.

Background: Talent is a crucial resource for economic and social development, serving as the driving force behind urban progress. As China experiences rapid growth in digital city construction, the capability of e-services continues to improve incessantly. In China, the new-generation highly educated migrants (NGHEMs) account for ~ 20-30% of the total floating populations. This study aimed to explore the settlement intention of new-generation highly educated migrations in China from the new perspective of urban e-service capabilities. Furthermore, the mechanism of the urban e-services on the settlement intention on the NGHEMs will be proved.

Methods: This paper employed data of China Migrants Dynamic Survey in 2017 and Evaluation Report of Government E-service Capability Index (2017). Descriptive analyses were conducted to investigate the factors influencing the settlement intention of NGHEMs in the destination city. Based on the principle of utility maximization, LASSO regression was employed to select individual and city characteristics that determined the settlement intention of NGHEMs. The impact of urban e-services on settlement intention was analyzed by using ordinal logit model. Additionally, robustness check, endogeneity analysis, and heterogeneity analysis were performed to validate the benchmark regression results. Finally, mediation model was employed to examine whether urban e-services enhance the settlement intention of NGHEMs by improving urban livability and urban innovation.

Results: The results indicate that the urban e-services promote the NGHEMs' settlement intention in the destination cities. Moreover, the results are still robust through a series of robustness tests. Furthermore, from the perspective of individual and regional heterogeneity, urban e-services significantly enhances the settlement intention of NGHEMs with male and female, married and urban household registration, and urban e-services can promote the settlement intention of NGHEMs with over 3 million inhabitants and those in the eastern regions of China. Finally, the intermediary effect test shows that urban e-services promote the settlement intention of NGHEMs through urban livability and urban innovation.

Conclusion: This study highlights the important impact of urban e-services on the settlement intention of new-generation highly educated migrants. The conclusions of this study provide suggestions for the government to use when designing policies to enhance the settlement intention of the NGHEMs and to improve the development of urban e-services.

Add-on therapy with tiotropium was cost-effective when added to usual care in patients who remain uncontrolled despite treatment with medium or high-dose ICS/LABA in a middle-income country.

Background: A significant proportion of asthma patients remain uncontrolled despite inhaled corticosteroids and long-acting beta-agonists. Some add-on therapies, such as tiotropium bromide, have been recommended for this subgroup of patients. This study aimed to assess the cost-effectiveness of tiotropium as an add-on therapy to inhaled corticosteroids and long-acting b2 agonists for patients with severe asthma.

Methods: A probabilistic Markov model was created to estimate the cost and quality-adjusted life-years (QALYs) of patients with severe asthma in Colombia. Total costs and QALYs of two interventions include standard therapy with inhaled corticosteroids and long-acting bronchodilators versus add-on therapy with tiotropium. Multiple sensitivity analyses were conducted. Cost-effectiveness was evaluated at a willingness-to-pay value of $5180.

Results: The expected incremental cost per QALY (ICER) is estimated at US$-2637.59. There is a probability of 0.77 that tiotropium + ICS + LABA is more cost-effective than ICS + LABA at a threshold of US$5180 per QALY. The strategy with the highest expected net benefit is Tiotropium, with an expected net benefit of US$800. Our base-case results were robust to parameter variations in the deterministic sensitivity analyses.

Conclusion: Add-on therapy with tiotropium was cost-effective when added to usual care in patients who remain uncontrolled despite treatment with medium or high-dose inhaled corticosteroids and long-acting bronchodilators. Our study provides evidence that should be used by decision-makers to improve clinical practice guidelines and should be replicated to validate their results in other middle-income countries.