Expert Review of Pharmacoeconomics & Outcomes Research最新文献

Introduction: Utility values offer a quantitative means to evaluate the impact of novel cancer treatments on patients' quality of life (QoL). However, the multiple methods available for valuing QoL present challenges in selecting the most appropriate method across different contexts.

Areas covered: This review provides cancer clinicians and researchers with an overview of methods to value QoL for economic evaluations, including standalone and derived preference-based measures (PBMs) and direct preference elicitation methods. Recent developments are described, including the comparative performance of cancer-specific PBMs versus generic PBMs, measurement of outcomes beyond health-related QoL, and increased use of discrete choice experiments to elicit preferences. Recommendations and considerations are provided to guide the choice of method for cancer research.

Expert opinion: We foresee continued adoption of the QLU-C10D and FACT-8D in cancer clinical trials given the extensive use of the EORTC QLQ-C30 and FACT-G in cancer research. While these cancer-specific PBMs offer the convenience of eliciting utility values without needing a standalone PBM, researchers should consider potential limitations if they intend to substitute them for generic PBMs. As the field advances, there is a greater need for consensus on the approach to selection and integration of various methods in cancer clinical trials.

Background: The DAPA-CKD study showed that dapagliflozin added to standard treatment reduced the risk of chronic kidney disease progression, and death from renal or cardiovascular causes compared to placebo.

Objective: Assess the cost-effectiveness of dapagliflozin and standard treatment versus standard treatment alone for chronic kidney disease within the Colombian health system.

Methods: We employed a Markov model based on the DAPA-CKD study, tailored to the Colombian scenario. The model forecasted hospitalizations for heart failure, overall and cardiovascular mortality, and chronic kidney disease progression over a 10-year horizon with a 5% discount rate.

Results: Dapagliflozin combined with standard treatment is a cost-effective intervention in treating stage 2-4 CKD. In the base case, the ICER was US $5,366, below 1 GDP (US $6.558) per capita. This was consistent in the sensitivity analyses.

Conclusion: Our study showed that dapagliflozin, when combined with standard treatment, is cost-effective against standard treatment alone, aligning with Colombia's willingness-to-pay threshold.

Introduction: Next-generation sequencing (NGS) identifies genetic variants to inform personalized treatment plans. Insufficient evidence of cost-effectiveness impedes the integration of NGS into routine cancer care. The complexity of personalized treatment challenges conventional economic evaluation. Clearly delineating challenges informs future cost-effectiveness analyses to better value and contextualize health, preference-, and equity-based outcomes.

Areas covered: We conducted a scoping review to characterize the applied methods and outcomes of economic evaluations of NGS in oncology and identify existing challenges. We included 27 articles published since 2016 from a search of PubMed, Embase, and Web of Science. Identified challenges included defining the evaluative scope, managing evidentiary limitations including lack of causal evidence, incorporating preference-based utility, and assessing distributional and equity-based impacts. These challenges reflect the difficulty of generating high-quality clinical effectiveness and real-world evidence (RWE) for NGS-guided interventions.

Expert opinion: Adapting methodological approaches and developing life-cycle health technology assessment (HTA) guidance using RWE is crucial for implementing NGS in oncology. Healthcare systems, decision-makers, and HTA organizations are facing a pivotal opportunity to adapt to an evolving clinical paradigm and create innovative regulatory and reimbursement processes that will enable more sustainable, equitable, and patient-oriented healthcare.

Introduction: In the face of growing patient volumes, health requirements, and economic constraints, modern healthcare systems are in dire need of innovative management solutions. Despite a range of tools available to tackle these challenges, there's a gap in the understanding of how these tools are tailored for health systems management.

Areas covered: This review, conducted in October 2023 across key health administration and technology databases Medline, Embase, IEEE Xplore, and ACM Digital Library, aims to examine the applications, characteristics, and real-world impacts of health systems management tools. From a total of 2,048 records, 12 studies were selected that span a variety of technologies, including decision analysis tools, machine learning algorithms, and data dashboards, all of which have proven critical in optimizing resource allocation, financial management, and patient care.

Expert opinion: These technologies have shown substantial promise in improving decision-making processes, operational efficiency, and overall healthcare outcomes. This review highlights the role these technologies and tools play in enhancing the manageability and sustainability of complex healthcare systems. It also underscores the need for ongoing research to further align technological developments with the evolving needs of the healthcare sector, ultimately aiming to boost system efficiency and improve patient care.

Introduction: This study aims to investigate healthcare resource utilization and related costs for influenza virus infections in adults admitted into secondary care in England across four influenza seasons.

Methods: This study includes all influenza admissions from the Hospital Episode Statistics database (HES) across England, September 2016 to March 2020. Descriptive analyses and comparative modeling techniques were used to assess the impact of influenza on risk groups of interest.

Results: Influenza admissions were estimated to cost £401 M. Average admission costs were heavily impacted by age and comorbid status, with comorbid patients representing 91% of costs. Additionally, patients with comorbidities treated in secondary care cost twice as much as non-comorbid patients, after adjusting for age and gender. Comorbid patients also had increased length of stay (LOS) and admissions to ICU, with patients' LOS being a core contributing factor to higher costs.

Conclusion: The study documents the substantial burden of influenza in England, emphasizing the impact of age and comorbid status on healthcare resource utilization (HCRU) and related costs. The data presented offers critical information to healthcare systems evaluating new strategies to alleviate the winter pressures on the NHS; highlighting the need to implement vaccination campaigns with enhanced vaccinations and increased vaccine coverage.

Introduction: Mucormycosis is a rare invasive fungal infection (IFI) which is characterized by prolonged antifungal therapy, high morbidity and mortality rates, as well as increased treatment costs.

Areas covered: Appropriate diagnosis of mucormycosis is a fundamental component of successful treatment, however, evidence about health economic expenses does not exist. Based on an international guideline approach for diagnosis of mucormycosis, we calculated costs for imaging-based and laboratory procedures and susceptibility testing from the German statutory health insurance perspective. We therefore analyzed the diagnostic recommendations for patients at increased progression risk, i.e. neutropenia, previous solid organ transplantation or hematopoietic stem cell transplantation.

Expert opinion: From the health economic point of view, our analysis underlines the relevance of appropriate guideline-based diagnosis of mucormycosis. The overall costs are relatively low (€499.40 per case) compared to other components in the management of mucormycosis, such as cost-intensive treatment with antifungal agents. Nevertheless, it is important to bear in mind that the level of diagnostic accuracy in line with the global guidelines by the European Confederation of Medical Mycology and the Mycoses Study Group Education and Research Consortium requires substantial resources, which may not be available in all countries or centers, especially in those with low income.

Background: The research aims to the costs and contents of waste pharmaceuticals collected through pharmacies for disposal. This study adds significant value to the country's economy in terms of cost and number of medicines.

Methods: The study was conducted with waste pharmaceuticals collected from 29 pharmacies in Erbaa district of Tokat province in Turkey. In the study, the total of waste pharmaceuticals, ATC classification and cost data of these medicines were obtained from the Eczanem automation program. Microsoft Office Excel and IBM SPSS programs were used in the analysis of the data.

Results: In the study, 1178 waste pharmaceuticals were evaluated. It was determined that approximately 88% of the waste pharmaceuticals were never used and 30% of them had not expired. Finally, it was determined that the total monthly cost of the collected waste pharmaceuticals was 4,908.88 USD. When this amount is calculated nationwide, the annual estimated cost of waste pharmaceuticals is 58,281,272.36 USD.

Conclusion: As a result of the research, it was revealed that waste pharmaceuticals constitute a serious national burden and waste in terms of both number and cost. Therefore, it is important to investigate the causes of waste pharmaceuticals in detail and take measures against them.

Objectives: To assess the economic value of lorlatinib and crizotinib in the first-line treatment of anaplastic lymphoma kinase-positive non-small cell lung cancer at medical insurance negotiation prices from the viewpoint of China's health system.

Methods: Based on data from the phase III clinical trial, a three-state partitioned survival model was established. In combination with parameters such as treatment costs, utility values, incidence of adverse reactions, and discount rates, the total incremental cost-effectiveness ratio (ICER) was simulated.

Results: The results of the fundamental analysis indicated that the patients in the lorlatinib group expended 709,671 yuan more than did the patients in the crizotinib group did but gained 3.09 quality-adjusted life years (QALYs). The ICER value was 229,782.6 yuan/QALY, which was lower than three times the per capita gross domestic product (GDP) of China.

Conclusions: Compared with crizotinib, lorlatinib may be a cost-effective first-line treatment choice for advanced ALK-positive NSCLC in China.