Expert Review of Pharmacoeconomics & Outcomes Research最新文献

Objectives: This study evaluates the cost-effectiveness of adagrasib plus cetuximab in contrast to adagrasib monotherapy in treating colorectal cancer (CRC) patients with mutated KRAS^G12C from the perspective of healthcare payers in the USA.

Methods: An economic evaluation utilizing a 3-state partitioned survival model assessed the cost-effectiveness of adagrasib plus cetuximab versus adagrasib monotherapy. The Kaplan-Meier curves for overall survival (OS) and progression-free survival (PFS) from a clinical trial were digitally extracted, and the Log-Logistic model was employed at the end of the trial to extrapolate the long-term survivals.

Results: The estimated cost for adagrasib plus cetuximab treatment was higher than that of adagrasib monotherapy (290,645.434 USD vs 188,837.346 USD). The estimated utility was decreased compared to that of adagrasib monotherapy treatment (1.094 QALYs vs 1.359 QALYs). The ICER was calculated at -384,674.32 USD/QALY, suggesting the adagrasib plus cetuximab therapy did not demonstrate an economic advantage over adagrasib monotherapy for CRC patients with mutated KRAS^G12C.

Conclusion: Adagrasib plus cetuximab was not cost-effective compared to adagrasib monotherapy as a late-line treatment for advanced or metastatic CRC patients with mutated KRAS^G12C in the USA.

Objectives: Different severities of exacerbations of chronic obstructive pulmonary disease (AECOPD) will lead to different disease progression and economic burden. This study aimed to evaluate the impact of the severity of AECOPD on disease burden.

Methods: Data were from the Tianjin Urban Employee Basic Medical Insurance Database (2016-2020). COPD patients were stratified by severity of AECOPD: (A) no AECOPD, (B) moderate AECOPD only; and (C) ≥1 severe AECOPD. Key outcomes included rate of AECOPD, mortality, COPD-related cost. Sensitivity analysis of reducing the impact of COVID-19 in outcomes was conducted.

Results: 6738 patients were identified, with 22.5% for Group A, 41.1% for Group B and 36.4% for Group C. During 1st follow-up year, Group C experienced an average of 1.43 severe AECOPD, leading to a notably increased all-cause mortality (Group C vs A-B: 17.7% vs. 2.6-3.4%, p < 0.001), highest COPD-related costs (CNY 30,245 vs 1,700-6,923). In the second year, patients in Group C still had a highest rate of severe AECOPD (0.44 vs 0.08-0.11 vs, p < 0.001), as well as increased all-cause mortality.

Conclusion: Severe AECOPD significantly increased mortality rates and economic burden in the current and subsequent year. This underscored the imperative need to enhance severe AECOPD management.

Background: This study assessed the economic burden of hemophilia A, B, and A with inhibitors, including direct medical, non-medical, and indirect costs from both governmental and patient perspectives.

Research design and methods: A retrospective cost-of-illness analysis was conducted at a public hospital in Baghdad, Iraq serving hemophilia patients. Government costs were derived from medical records, while patient out-of-pocket expenses were gathered via interviews. Data were collected from November 2024 to March 2025. One-way ANOVA tested cost differences across hemophilia types and severity levels.

Results: The Ministry of Health spent US$11.30 million annually on 446 hemophilia patients (average $25,312 per patient), with clotting-factor replacement comprising 70.6% of the costs. On-demand treatment accounted for 84.5% of the hospitalization expenses. Patients with hemophilia-A-with-inhibitors incurred the highest annual cost ($113,651), followed by hemophilia-B and hemophilia-A-without-inhibitors cases. Severe cases and comorbidities like hepatitis C further increased spending. Non-medical costs averaged $440.33, and indirect costs $281.42 per patient. School absenteeism totaled 1,753 days across 144 students, averaging 12.2 days each.

Conclusion: Clotting-factor replacement remains the primary cost driver in hemophilia care, with inhibitors significantly increasing expenditures. Non-medical and indirect costs add to the overall burden, underscoring the need for prophylaxis and access to innovative therapies.

Background: Hepatocellular carcinoma (HCC) is a global health issue and the third leading cause of cancer-related deaths. For patients with unresectable HCC (uHCC), Sorafenib and Lenvatinib, are key treatments. This study evaluates the cost-effectiveness of Lenvatinib versus Sorafenib for uHCC in Iran.

Research design and methods: A model-based cost-utility analysis was conducted using a Partitioned Survival Analysis (PartSA) model from the perspective of the Iranian society. Clinical data were sourced from the REFLECT trial, while cost inputs, including treatment, monitoring, and side-effect management, were derived from local healthcare data and expert consultations. Sensitivity analyses and Monte Carlo simulations ensured robustness.

Results: The base-case analysis revealed that Lenvatinib, with a total cost of $9,607, offers a cost saving of $1,551 compared to Sorafenib ($11,158). Lenvatinib also provides an incremental gain of 0.14 Quality-Adjusted Life Years (QALYs) per patient over a 20-years. Probabilistic sensitivity analysis showed a > 99% probability of Lenvatinib being cost-effective. One-way analysis confirmed Lenvatinib's cost-effectiveness if priced below $18/day.

Conclusions: Lenvatinib is a cost-effective alternative to Sorafenib for uHCC treatment in Iran, providing better clinical outcomes and cost savings. These results support its adoption as the preferred treatment, emphasizing the importance of integrating cost-effectiveness analyses into healthcare decision-making.

Introduction: Hematopoietic stem cell (HSCT) and chimeric antigen receptor T-cell (CAR-T) therapy are crucial in the treatment of hematologic malignancies. While HSCT has been long established as a mainstay of the treatment of these diseases, CAR-T is a newer therapy with the potential to change the treatment landscape. Both cellular therapies are time and resource intensive, which has put stressors on patients that are unique from those associated with other forms of oncologic treatment.

Areas covered: In this review, we discuss the current state of knowledge of psychological symptoms associated with HSCT and CAR-T and their management. In addition, we discuss the models of patient reported outcomes (PROs) used to perform this research. We performed a literature review on PubMed using keywords including 'palliative care, palliative care integration, hematologic malignancy, patient reported outcome, psychological care, and quality of life.' We included papers published before 1 April 2025.

Expert opinion: We propose further investigations into integrated models of oncologic and palliative care teams and their efficacy.

Background: Gaining an understanding of transitioning hemophilia patients to an extended half-life (EHL) agent requires real-world data, encompassing various outcomes, which would help assessing the impact of the switch, for patients and the healthcare system. We investigate the economic implications of switching from standard half-life (SHL) recombinant factor VIII (rFVIII) from either prophylaxis or on-demand, to EHL rFVIII efmoroctocog alfa (FVIIIFc) prophylaxis.

Research design and methods: The study involved 48 patients with hemophilia A from the 5 specialized hemophilia centers in Greece. Patients switched from prophylactic or on-demand treatment using a SHL factor VIII to a rFVIIIFc on prophylaxis only. Data was gathered for the 12-month period before and after switch. Using standard t-tests and regression analysis, we compared the direct treatment cost between the pre and post switch time, focusing on regimens' differential costs.

Results: There is no statistically significant cost increase compared to the core rFVIII regimen used before the switch, when only patients that were previously on prophylaxis were considered. However, there is a statistically significant reduction in cost across treatment styles.

Conclusions: The analysis confirms the advantages offered by prophylaxis with rFVIIIFc from an economic standpoint for the Greek healthcare system, in accordance with other studies.

Introduction: The global obesity epidemic is a complex issue influenced by various factors, including the food system and its environment. Increasingly, interventions targeting systemic changes in the food environment are being implemented to address obesity. Cost-benefit analysis (CBA) is gaining recognition as a valuable tool for evaluating these interventions, due to its ability to capture broader societal impacts beyond health outcomes. However, its application in this context remains poorly understood.

Methods: A systematic review was conducted following PRISMA guidelines, searching academic databases (MEDLINE, EMBASE, PsycINFO, CINAHL, Web of Science, EconLit, CRD). Study quality was assessed using the Making an Early Intervention Business Case checklist, designed to evaluate CBA studies.

Results: Of 6508 references screened, 28 studies met the inclusion criteria. The review identified common methodological approaches, summarized key findings, challenges and implications, and provided clear recommendations for improvement. The review also synthesized evidence to show that food environment interventions offer value for money with positive returns.

Conclusion: This first systematic review of CBAs in food environment interventions identified common methods, challenges, and areas for improvement. While CBA is a valuable tool for evaluating food environment interventions, more robust and standardized methodological approaches are needed to enhance its reliability and applicability.Registration: PROSPERO (CRD42024503615).

Background: Patients with obesity or overweight are at increased risk for obesity-related complications (ORCs), including cardiovascular disease. Treatment with once-weekly (OW) semaglutide 2.4 mg has demonstrated clinical efficacy, however its impact on healthcare resource utilization (HCRU) and costs warrants further investigation.

Research design and methods: This retrospective real-world study evaluated HCRU and costs (excluding pharmacy) among patients with obesity or overweight and ≥1 ORC in the Komodo Healthcare Map database. Baseline characteristics of patients starting OW semaglutide 2.4 mg treatment were matched to obesity medication (OM) non-users. HCRU and medical costs estimates at 12-month follow-up, both all-cause and ORC-related, were compared between the two cohorts.

Results: Patients treated with semaglutide 2.4 mg vs OM non-users had 37% and 21% lower incidence of all-cause inpatient (IP) and emergency room (ER) visits, respectively, and 45% and 29% lower incidence of ORC-related IP and ER visits, respectively. All-cause and ORC-related total medical costs were lower among patients treated with semaglutide 2.4 mg (11% and 15% lower, respectively), translating to an observed savings of $3,342 and $2,408 at 12 months.

Conclusions: In patients with obesity or overweight, OW semaglutide 2.4 mg treatment was associated with decreased total medical HCRU and costs at 12 months.

Objective: To identify the major barriers to and facilitators of implementing the Lebanese Health Economic Evaluation Guideline (LEEG) according to Lebanese stakeholders, supported by their insights.‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬.

Methods: We conducted a survey asking 16 key stakeholders to rate LEEG's importance, its necessity for Lebanon, and its applicability in decision-making, and Likert scale questions to rate barriers and facilitators. Following the survey, an individual face-to-face interview using open-ended questions was arranged to validate the survey's data, to collect further insights regarding LEEG's implementation in Lebanon, and to identify relevant barriers and facilitators.

Results: All participants considered LEEG to be very important for Lebanon. The majority considered it very important for decision-making, while there was some disagreement on the applicability of economic evaluation as a criterion for decision-making. The most important barriers were 'lack of political willingness to adopt economic evaluation and use health technology assessment findings' and 'the absence of policy networks.' The most important facilitators were 'the strong need to implement national guidelines in Lebanon' and 'acceptability to payers.'

Conclusion: We reported on important barriers to and facilitators of implementing LEEG related to the national context and methodological concerns. This will pave the way for developing strategies essential for effective implementation and, consequently, a better healthcare system, advancing progress to universal health coverage.