Expert Review of Pharmacoeconomics & Outcomes Research最新文献

Objective: To evaluate the cost-effectiveness of a single-pill combination (SPC) of perindopril/amlodipine/indapamide versus its free equivalent combination (FEC) in adults with hypertension in Italy.

Methods: A Markov model was developed to perform a cost-utility analysis with a lifetime horizon and an Italian healthcare payer's perspective. In the model, the additional effect of the SPC on blood pressure level compared with the FEC was translated into a decreased risk of cardiovascular events and CKD, which was modeled via Framingham risk algorithms. Difference in persistence rates of SPC and FEC were modeled via discontinuation rates.

Results: A perindopril/amlodipine/indapamide SPC is associated with lower cost and better health outcomes compared to its FEC. Over a lifetime horizon, it is associated with a 0.050 QALY gain and cost savings of €376, resulting from lower cardiovascular event rates. In the alternative scenario, where different approach for modeling impact of adherence was considered, incremental gain of 0.069 QALY and savings of €1,004 were observed. Results were robust to sensitivity and scenario analyses, indicating that use of this SPC is a cost-effective strategy.

Conclusions: The findings indicate that a perindopril/amlodipine/indapamide SPC is a cost-saving treatment option for hypertension in Italy, compared to its FEC.

Objective: In the ASTRUM-004 trial, serplulimab plus chemotherapy demonstrated significantly improved survival and controllable safety. This study assessed the cost-effectiveness of serplulimab plus chemotherapy in advanced squamous non-small cell lung cancer (sqNSCLC), considering the perspective of the Chinese healthcare system.

Methods: A decision tree and a Markov model were constructed to simulate the treatment. The interesting results included total cost, life-years (LYs), quality-adjusted life-years (QALYs) and incremental cost-effectiveness ratios (ICERs). Scenario, one-way and probabilistic sensitivity analyses were used to examine model instability.

Results: Compared with placebo plus chemotherapy, serplulimab plus chemotherapy had an ICER of $55,539.46/QALY ($47,278.84/LY). The ICERs were estimated to be $58,706.03/QALY, $48,978.34/QALY and $59,709.54/QALY inpatients with programmed death-ligand 1 expression level of tumor proportion score (TPS) < 1%, 1% ≤ TPS < 50%, and TPS ≥ 50%. The cost-effective prices of serplulimab were $168.276/100 mg, $349.157/100 mg, and $530.039/100 mg at the willingness-to-pay threshold of $12,574.30/QALY, $25,148.60/QALY, and $37,722.90/QALY. Patient weight and price of serplulimab created the most significant impact. Presently, the probability of serplulimab plus chemotherapy being cost-effective was 14.15%.

Conclusion: Compared with placebo plus chemotherapy, serplulimab plus chemotherapy might not be cost-effective in the first-line treatment for advanced sqNSCLC.

Introduction: Multiple sclerosis (MS) is a persistent condition characterized by immune-mediated processes in the central nervous system, affecting around 2.8 million individuals globally. While historically less prevalent in the Middle East and North Africa (MENA) region, recent trends mirror the global rise in MS.

Area covered: The impact of MS is substantial, particularly in the MENA region, with costs per patient surpassing nominal GDP per capita in certain countries. Disease-modifying therapies aim to alleviate MS effects, but challenges persist, especially in managing progressive MS as it shifts from inflammatory to neurodegenerative phases. Limited resources in the MENA region hinder care delivery, though awareness initiatives and multidisciplinary centers are emerging. Contrary to global projections of a decline in the MS market, the MENA region is poised for growth due to increased prevalence, healthcare expenditures, and infrastructure investments.

Expert opinion: This review underscores the urgent necessity for effective treatments, robust disease management, and early diagnosis in tackling MS's repercussions in the MENA region. Bolstering resources tailored to MS patients and elevating the quality of care stand as pivotal strategies for enhancing health outcomes in this context. Taking decisive action holds the key to enhancing the overall well-being of individuals grappling with MS.

Background: Clostridioides difficile is the most common cause of healthcare-associated diarrhea. Research suggests that treating C. difficile infections (CDI) with fidaxomicin (FDX) is more effective than vancomycin (VAN), with potential cost savings. The objective was to calculate the budget impact of FDX treatment compared to VAN from a German payer perspective.

Research design and methods: The analysis used real-world data of patients discharged from University Hospital Cologne between Jan-01-2018 and Dec-31-2019. We identified recurrent and non-recurrent CDI cases and calculated direct treatment costs based on G-DRG flat rates. To calculate average costs per treatment and the budget impact, recurrence probabilities for VAN and FDX were taken from published evidence (28-day and 90-day scenarios).

Results: Totally, 475 cases were analyzed, thereof 421 non-recurrent, causing mean costs of €32,901 per case (95% CI: 27.752-38.050). Thirty-two patients experienced a recurrence within 28 days, yielding mean costs of €10,952 (95% CI: 5.627-16.277) for their additional hospital stay. The resulting budget impact was €1,303 (95% CI: 670-1.937) in favor of FDX, ranging from €148.34 to €2,190.30 in scenario analyses.

Conclusion: The analysis indicates FDX treatment can lead to cost savings compared to VAN. Future research should focus on specific patient groups, such as refractory CDI patients.

Objective: The current study aims to develop an algorithm for mapping the WHODAS 2.0 to the EQ-5D-5 L for patients with mental disorders.

Methods: This cross-sectional study was conducted at the Institute of Mental Health and Community Wellness Clinics in Singapore between June 2019 and November 2022. We included four regression methods including the Ordinary Least Square (OLS) regression, the Tobit regression model (Tobit), the robust regression with MM estimator (MM), and the adjusted limited dependent variable mixture model (ALDVMM) to map EQ-5D-5 L utility scores from the WHODAS 2.0.

Results: A total of 797 participants were included. The mean EQ-5D-5 L utility and WHODAS 2.0 total scores were 0.615 (SD = 0.342) and 11.957 (SD = 8.969), respectively. We found that the EQ-5D-5 L utility score was best predicted by the robust regression model with the MM estimator. Our findings suggest that the WHODAS 2.0 total scores were significantly and inversely associated with the EQ-5D-5 L utility scores.

Conclusion: This study provides a mapping algorithm for converting the WHODAS 2.0 scores into EQ-5D-5 L utility scores which can be implemented using a simple online calculator in the following web application: https://eastats.shinyapps.io/whodas_eq5d/.

Objective: This study aims to conduct a cost-effectiveness analysis of pembrolizumab in combination with chemotherapy for HER2-negative advanced gastric cancer in China.

Methods: A partitioned survival approach model was constructed to simulate the progression of HER2-negative advanced gastric cancer and evaluate the outcomes of different treatment strategies. We calculated incremental cost-effectiveness ratios (ICER) to assess the cost associated with each quality-adjusted life-year (QALY) gained. One-way sensitivity analysis and probabilistic sensitivity analysis were performed to assess robustness and reliability.

Results: The analysis conducted in the base case demonstrated that the ICER associated with pembrolizumab was $177405.83/QALY gained in all population. In the subgroup analysis, it was found that individuals with a PD-L1 CPS ≥ 1 and those with a PD-L1 CPS ≥ 10 had ICERs of $152397.06/QALY and $109534.13/QALY, respectively. All ICER values for both the all population groups and the subgroups exceeded the WTP threshold in China. Our analysis shows the robustness of these results, as they remained consistent when input parameters were varied within a ± 25% range.

Conclusion: The findings of this cost-effectiveness analysis suggest that pembrolizumab in combination with chemotherapy is not a cost-effective treatment option for HER2-negative advanced gastric cancer in China.

Background: We propose a framework to assess the value of pharmaceutical innovations, with explicit clinical and methodological parameters, based on the therapeutic value and health needs.

Research design and methods: The study was based on the adaptation of health technology assessment methods documented in the literature, which was applied to a sample of oncological drugs. Difficulties and issues during the application of those tools were identified and addressed to develop a new framework with new and revised domains and clear classification criterion for each domain. Scores were assigned to each level and domain according to their relevance to generate the final score of innovativeness.

Results: The Pharmaceutical Innovation Index (PII) includes four domains, two related to clinical and social dimensions - Therapeutic Need and Added Therapeutic Value - and other two about methodological features - Study Design and Quality (risk of bias). The scores combined after assigned to each domain results Index of the Innovativeness of the medicines represents the degree of pharmaceutical innovation.

Conclusion: This work proposes a transparent methodology with well-defined criteria and script; the algorithm developed with authors' weightings and criteria may be switched to best adjust to other applications, perspective or clinical indications, while keeping the transparency and objectiveness.

Objectives: The objective of this study is to elicit health care preferences of people with diabetes and identify classes of people with different preferences.

Methods: A discrete choice experiment was conducted among people with diabetes in Germany comprising attributes of role division in daily diabetes care planning, type of lifestyle education, support for correct medication intake, consultation frequency, emotional support, and time spent on self-management. A conditional logit model and a latent class model were used to elicit preferences toward diabetes care and analyze preference heterogeneity.

Results: A total of 76 people with diabetes, recruited in two specialized diabetes care centers in Germany (mean age 51.9 years, 37.3% women, 49.1% type 2 diabetes mellitus, 50.9% type 1 diabetes mellitus), completed the discrete choice experiment. The most important attributes were consultation frequency, division in daily diabetes care planning, and correct medication intake. The latent class model detected preference heterogeneity by identifying two latent classes which differ mainly with respect to lifestyle education and medication intake.

Conclusion: While the majority of people with diabetes showed preferences in line with current health care provision in Germany, a relevant subgroup wished to strengthen lifestyle education and medication intake support with an aid or website.

Introduction: Health economic evaluation (HEE) provides guidance for decision-making in the face of scarcity but ignores ecological scarcities as long as they involve external costs only. Following the imperative to account for planetary health, this study explores how this blind spot can be addressed.

Areas covered: The study is based on a critical review of relevant work, particularly in the fields of HEE and life cycle assessment (LCA). LCA can provide information on a technology's environmental impacts which can be accounted for on both the effect and cost sides of HEE. Cost-benefit analyses can incorporate environmental impacts in case vignettes used for eliciting consumers' willingness to pay. Existing LCA impact models can be used to estimate human health risks associated with environmental impacts and add them to the health benefits in cost-utility analyses. Many jurisdictions offer lists of shadow prices that can be used to incorporate environmental impacts on the cost side of HEE. Also, environmental impacts can be reported in a disaggregated manner.

Expert opinion: Accounting for planetary boundaries is likely to become a key field of methodological innovation in HEE. Decision relevance is likely to be highest for technologies with similar cost-effectiveness but different ecological impacts.

Introduction: This systematic review study investigated the cost-effectiveness of stereotactic radiotherapy (SRT) and stereotactic radiosurgery (SRS) for treatment of various types of cancers.

Methods: PubMed, Scopus, and Web of Science were searched from 30 December 1990 to 1 January 2023. The entered studies were screened in accordance with the inclusion criteria. The inclusion criteria encompassed all types of economic evaluation studies that investigated SRT/SRS technologies in the treatment of various cancers.

Results: A total of 47 articles were included in the review. The findings suggest that the use of Linear accelerator technology for the treatment of lung cancer (8 out of 12 studies) and prostate cancer (4 out of 5 studies) was a cost-effective strategy. Linear accelerator was found to be cost-effective in the treatment of liver metastases and liver cancer (2 out of 5 studies). All of the included studies that used Gamma Knife technology in brain metastases reported Gamma-Knife was a cost-effective treatment. Furthermore, in the treatment of prostate and liver cancer, proton therapy was identified as a cost-effective option than other treatments.

Conclusions: This study confirms that SRT/SRS is a cost-effective procedure for the treatment of various types of cancers. Therefore, it is recommended to use SRT/SRS technology for optimal use of resources.