Expert Review of Pharmacoeconomics & Outcomes Research最新文献

Background: Higher prices for drugs targeting rarer cancers may be justified, but the extent remains uncertain. This research analyses the determinants of cancer drug prices in Germany and France.

Research design and methods: Cancer drugs approved in EU between 2011 and 2022 with available benefit assessments and prices were analyzed. Monthly treatment cost was the dependent variable; approval year, orphan status, benefit rating, number of comparators, and target population size were independent variables. Univariate and multiple regression analyses were conducted.

Results: The analysis included 107 drugs in Germany and 70 in France. In Germany, univariate analysis showed significance for benefit magnitude, orphan status, number of comparators, target population, and approval year. In France, orphan status, number of comparators, target population, and approval year were significant. Regression models showed that log target population (p < 0.0001), approval year (p = 0.021), and major/considerable benefit (p < 0.0001) were significant in Germany; log target population (p = 0.001) and ASMR II/III (p = 0.017) were significant in France.

Conclusions: Target population and benefit rating are drivers of cancer drug prices in Germany and France. The analysis provides quantitative evidence on the association between cancer rarity and price, which could inform policy.

Introduction: Digitally delivered patient decision aids are becoming more prevalent to support prenatal decision-making. However, the effect of digital decision aids on prenatal outcomes is unclear. The objective of this systematic review and meta-analysis is to synthesize the currently available evidence on the effect of digital decision aids on prenatal outcomes, and quantitatively analyze the effect of interventions on prenatal outcomes across studies.

Methods: Studies that used digital decision aids (I) and measured prenatal care outcomes (O) for pregnant women (P) were eligible for inclusion. Ovid MEDLINE, CINAHL, Scopus, and Cochrane were searched from inception to November 2024. The protocol was registered on Open Science Framework.

Results: 3106 results were identified from databases. After deduplication, 2494 were screened. 11 studies were included in the review. Decision aids were found to improve knowledge and reduce decisional conflict. In a meta-analysis analyzing decisional conflict, interventions were favored (SMD -0.15; 95%CI -0.24, -0.06).

Conclusions: Digital decision aids can improve prenatal decisional quality, though we found less evidence for their effect on changing interventional decisions. Research is still needed on the most effective digital formats and strategies for implementation of decision aids in real-world environments to assess the benefits of digital decision aids for prenatal care.

Registration: The protocol was registered on Open Science Framework (osf.io/p2vb5).

Background: This study evaluates the cost-effectiveness of sotorasib plus panitumumab in contrast to standard care in the treatment of refractory colorectal cancer (CRC) with mutated KRAS^G12Cfrom the perspective of healthcare payers in the U.S.A.

Research design and methods: An economic evaluation utilizing a 3-state partitioned survival model assessed the cost-effectiveness of sotorasib plus panitumumab versus standard care. TheKaplan-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 sotorasib plus panitumumab treatment was higher than that of standard care treatment (77,087.936 USD vs 8905.065 USD). In addition, the estimated utility was relatively augmented than that of standard care treatment (0.876 QALYs vs 0.857 QALYs). The ICER was calculated at 3,551,555554USD/ QALY, suggesting the sotorasib plus panitumumab therapy did not demonstrate an economic advantage over standard care therapy for refractory CRC patients with mutated KRAS^G12C at the threshold of 150,000 USD/ QALY.

Conclusions: Sotorasib plus panitumumab did not demonstrate an economic advantage compared to standard care in the treatment of refractory CRC with mutated KRAS^G12C in the U.S.A.

Objective: To evaluate the cost-effectiveness of ruxolitinib in Singapore for patient with chronic graft-versus-host disease (GVHD) and inadequate response to corticosteroids.

Methods: A three-state partitioned survival model was developed to evaluate the cost-effectiveness of ruxolitinib from the Singapore healthcare system perspective over a five-year time horizon. Clinical data were sourced from the REACH3 trial, health state utilities were retrieved from literature, and direct costs were obtained from public healthcare institutions in Singapore. Sensitivity and scenario analyses were conducted to explore the impact of uncertainties and assumptions on the cost-effectiveness results.

Results: Compared to best available therapy, ruxolitinib yielded a base-case incremental cost-effectiveness ratio (ICER) of S$776,653 (US$574,724) per quality-adjusted life-years gained. Sensitivity analyses revealed that the ICER was particularly sensitive to utilities in failure-free and progressed disease states. Scenario analyses confirmed that the ICERs remained high even under favorable assumptions, and a substantial price reduction was necessary to lower the ICER.

Conclusion: At its current price, ruxolitinib is not cost-effective for treating chronic GVHD in Singapore. This finding helps to inform funding decision-making, which also considers other factors such as clinical effectiveness, safety, and budget impact, in addition to cost-effectiveness.

Objective: To evaluate the cost-effectiveness of osimertinib compared to dacomitinib for advanced non-small cell lung cancer with EGFR mutation from the perspective of China's health system.

Methods: Based on the FLAURA clinical trial and network meta-analysis, a partitioned survival model was constructed with a model simulation timeframe of 10 years and a 3-week cycle. Cost and quality-adjusted life years (QALYs) were used as model output indicators, and the incremental cost-effectiveness ratio was calculated to determine the economic feasibility of osimertinib compared to dacomitinib through cost-utility analysis. Sensitivity analysis was applied to test the robustness of the model.

Results: The basic analysis results indicate that the osimertinib group incurred an additional cost of 138,487 Chinese Yuan (CNY) compared to the dacomitinib group, but gained an additional 0.32 QALYs. The incremental cost-effectiveness ratio is 436,203 CNY, which is higher than three times China's per capita GDP.

Conclusion: Under the threshold of three times China's per capita GDP, osimertinib appears not to be cost-effective compared to dacomitinib.

Objectives: We first evaluated the cost-effectiveness of the FOLFOXIRI plus bevacizumab versus mFOLFOX6 plus bevacizumab as the first-line treatment of metastatic colorectal cancer from healthcare system perspective.

Methods: A partitioned survival model was developed to assess the costs and effects of FOLFOXIRI plus bevacizumab versus mFOLFOX6 plus bevacizumab. Health outcomes were measured in quality-adjusted life years (QALYs), and incremental cost-effectiveness ratios (ICERs). The evaluation of the Chinese healthcare payer perspective was performed across a lifetime horizon, encompassing direct medical expenses.

Results: The estimated cost for FOLFOXIRI plus bevacizumab treatment was 9306.364USD, which was higher than 8218.436 USD estimated for mFOLFOX6 plus bevacizumab, leading to an ICER of 1961.857 USD per QALY. One-way sensitivity analysis suggested the body surface area (BSA), the cost of irinotecan, and the cost of fluorouracil had the largest impact on the ICER. The cost-effectiveness acceptability curves showed the probability of FOLFOXIRI plus bevacizumab being cost-effective was100% at a threshold of 12 300 USD per QALY.

Conclusion: FOLFOXIRI plus bevacizumab had an economic advantage compared to mFOLFOX6 plus bevacizumab as the first-line treatment of metastatic CRC in China.

Background: In 2024, Alzheimer's disease affected approximately 6.9 million Americans aged 65 and older. Current therapies include acetylcholinesterase inhibitors, N-methyl-D-aspartate receptor inhibitors, and monoclonal antibodies. With an economic burden surpassing $345 billion, $222 billion borne by Medicare and Medicaid, this study evaluates lecanemab's budgetary impact for early AD, including subgroup analyses by gender and race.

Methods: A budget impact model evaluated LECA for early Alzheimer's disease, comparing scenarios with and without the therapy. Inputs included market share, costs, duration, and compliance. Gender and race subgroup analyses, annual costs, PMPM, PTMPM, and sensitivity analysis outcomes were assessed to explore parameter-driven variability comprehensively.

Results: Introducing LECA for over 3.5 million eligible early AD patients in the U.S. may generate a three-year budget impact of $4.1 billion for Medicare. Incremental PMPM savings were $1.4, and PTMPM savings reached $24.1. Subgroup analyses revealed no significant gender or racial differences in PMPM and PTMPM, with variability only in overall budget impact. Sensitivity analyses indicate that enhanced healthcare resource utilization, reduced disease severity, and improved cost-efficiency among males contribute to strengthening Medicare's budget sustainability.

Conclusions: Utilizing LECA as a treatment for early AD is expected to be cost saving with respect to Medicare budgets in the U.S.

Introductions: The benefits and risks of each vaccine must be compared prior to their recommendation and again when new evidence emerges.

Areas covered: A narrative scoping review was conducted by searching MEDLINE via PubMed on 8 October 2024 and 12 September 2025. The implications of benefit-risk assessments of vaccines are discussed. A quantitative benefit-risk assessment may be conducted from an individual perspective, which only considers direct health effects, or from a societal perspective, which considers both direct and indirect health effects (e.g. a reduction of transmissibility through community immunity). Whether the results of quantitative benefit-risk assessment of vaccination encompass an individual or societal perspective should be made clear as this changes the meaning for stakeholders. A societal perspective is fundamental to evaluating vaccine benefits and risks as a population. However, showing a beneficial impact from a societal perspective may not be sufficient motivation to vaccinate oneself or their children.

Expert opinion: Benefit-risk assessments of vaccination from an individual perspective may be helpful for these individuals in their vaccine decision-making. Ideally, timely vaccine benefit-risk assessments from both societal and individual perspectives are conducted in parallel, and the results of both are clearly communicated to the public to aid in vaccine decision-making.

Background: Precision medicine's emphasis on genetic profiles rather than diseases has reshaped oncology trials, prioritizing biomarker-specific approaches named basket trials. They involve heterogeneous populations, necessitating new economic evaluation methods, as traditional approaches fail to capture this diversity. This study introduced a methodology for economic evaluations based on basket trials, exemplified through pembrolizumab's cost-effectiveness analysis using Keynote-158 data as a case study and compared the results to disease-specific methods.

Research design and methods: A regression-based approach was developed to aggregate costs and QALYs and calculate transition probabilities for economic evaluations in populations sharing a biomarker and various cancers. The proposed method involved five steps: designing a general model, selecting comparator arms, comparing Kaplan-Meier curves and conducting network analysis to evaluate treatment responses, calculating disease-specific coefficients to aggregate QALYs, and aggregating costs using weighted averages.

Result: All the steps have been followed for conducting cost-effectiveness of Pembrolizumab in patients with dMMR/MSI-H biomarker. The results revealed significant differences in ICERs calculated by the proposed method versus disease-specific evaluations.

Conclusion: This study introduces a biomarker-focused model for estimating ICERs across diverse diseases, enabling informed decision-making in healthcare resource allocation. Further research is recommended to broaden the model's applicability and address additional population heterogeneity factors.

Background: In the phase III DUO-E trial, durvalumab with or without olaparib significantly increased survival outcomes compared to chemotherapy alone among patients with endometrial cancer. The aim of this study was to assess the cost-effectiveness of durvalumab in patients with advanced/recurrent dMMR endometrial cancer.

Research design and methods: A Markov model was used to compare the cost-effectiveness of durvalumab with or without olaparib compared to chemotherapy alone in the treatment of advanced/recurrent dMMR endometrial cancer, using the data from phase III DUO-E trial. The outcomes evaluated were total costs, quality-adjusted life years (QALYs), and incremental cost-effectiveness ratios (ICER). One-way sensitivity and probabilistic sensitivity analyses were performed to account for uncertainty.

Results: The base case analysis found that durvalumab and chemotherapy had an ICER of $239,884/QALY, whereas durvalumab with olaparib and chemotherapy had an ICER of $259,651/QALY compared to chemotherapy alone. The model was most sensitive to the cost of durvalumab. Probabilistic sensitivity analysis revealed that durvalumab had a 0% likelihood of being considered cost effective at a WTP threshold of $150,000 per QALY.

Conclusions: Durvalumab was not cost-effective as a first-line treatment of patients with advanced/recurrent dMMR subtype of endometrial cancer. Reducing the cost of durvalumab might improve economic outcomes.