Expert Review of Pharmacoeconomics & Outcomes Research最新文献

Objectives: When health outcomes relevant for economic evaluations are unavailable, algorithms can be developed to map utilities using available clinical outcome measures. This study aims to develop two mapping algorithms estimating EuroQol-5 dimension-3 level (EQ-5D-3 L) utilities using the clinician-rated Health of the Nation Outcome Scores (HoNOS) and Positive and Negative Syndrome Scale (PANNS).

Methods: A dataset with 2,029 observations of patients with psychotic disorders included EQ-5D-3 L, HoNOS, PANSS item scores, and demographics. Correlations between instruments were evaluated. Least Absolute Shrinkage and Selection Operator (LASSO) regression and random forest (RF) algorithms with various predictor variable sets were applied. Model performance was cross-validated using R-squared and Root Mean Square Error (RMSE).

Results: High ceiling effects were observed for EQ-5D-3 L, with weak to moderate negative correlations between EQ-5D and HoNOS (r = -0.34) and PANSS (r = -0.27). Overall, LASSO models outperformed RF models, with individual item models performing best for the HoNOS and PANSS, with the best observed RMSEs of 0.241 and 0.231, respectively.

Conclusions: The HoNOS and PANSS could be mapped onto EQ-5D-3 L utilities but lack accuracy for individual patient predictions. However, in the absence of alternatives, they could adequately predict population-based utility score differences for health economic evaluations.

Background: The addition of atezolizumab to bevacizumab plus platinum regimen has demonstrated notable improvements in treating metastatic, persistent, or recurrent cervical cancer, but its cost-effectiveness requires further investigation. From a US payer perspective, we aimed to evaluate the cost-effectiveness of atezolizumab plus bevacizumab and chemotherapy vs. standard chemotherapy as a first-line treatment for metastatic, persistent, or recurrent cervical cancer.

Methods: A partitioned survival model based on the data from the BEATcc trial was used to calculate the incremental cost-effectiveness ratio (ICER), using cost and health utility information obtained from literature and publicly accessible databases. One-way and probabilistic sensitivity analyses were performed to evaluate the model's responsiveness to variations in parameters.

Results: The addition of atezolizumab resulted in an additional 0.839 quality-adjusted life years (QALY) at an additional cost of $458,237, leading to an ICER of $545,943/QALY. One-way sensitivity analysis indicated that the cost of atezolizumab had the greatest impact on the ICER, followed by the utility value of progression-free survival (PFS) and follow-up costs. Probabilistic sensitivity analysis showed a 0% cost-effectiveness probability at the current willingness-to-pay (WTP) threshold of $150,000 per QALY.

Conclusion: Adding atezolizumab to chemotherapy is cost-prohibitive in the US and may not be cost-effective for patients.

Introduction: Suicide is a major public health concern in low- and middle-income countries (LMICs) due to its substantial psychological, social, and economic impact. There is little synthesized evidence to estimate the economic burden of suicide and suicide attempts in such economies. The present systematic literature review aims to examine existing evidence on the cost of illness (COI) in the case of suicides and suicide attempts and assess their quality.

Methods: A systematic review was carried out using electronic databases, such as Medline, EMBASE, EconLit, PsycINFO, and CINAHL using keywords like 'suicide and suicide attempts,' 'cost of illness,' and economic burden." The quality assessment of studies was conducted along with the per-person cost estimation to understand the variation of methods followed across the studies.

Result: 14 studies qualified for final data extraction and synthesis out of 4,164 studies. The studies showed heterogeneity across objectives, settings, and methods, with cost estimates reflecting a wide range of costings per person in suicide and suicide attempts.

Conclusion: It is challenging to determine and compare the economic estimates of suicide. Intensive research is warranted with standardized cost assessment techniques and wider perspectives to understand the true economic burden of suicide.

Registration: PROSPERO Registration No- CRD42022294080.

Objective: The aim of the current study was to evaluate the cost-effectiveness of serplulimab plus chemotherapy compared chemotherapy alone as first-line strategy for patients with ES-SCLC in China.

Methods: A decision-analytic model that based on the Chinese health-care system perspective was conducted to evaluate the economic benefits for the two competing first-line treatment. The clinical survival and safety data were obtained from the ASTRUM-005 trial, cost and utility values were gathered from the local charges and previously published study. Both cost and utility values were discounted at an annual rate of 5%. Sensitivity analyses and subgroup analyses were performed to examine the robustness of the model results.

Results: Serplulimab plus chemotherapy could bring additional 0.25 QALYs with the marginal cost of $37,569.32, resulting in an ICER of $147,908.74 per additional QALY gained. Sensitivity analyses confirmed that model results were robust. Subgroup analyses revealed that adding serplulimab to first-line chemotherapy were unlikely to be the cost-effective option for all subgroup patients.

Conclusions: Serplulimab plus chemotherapy was unlikely to be the cost-effective first-line strategy compared with chemotherapy alone for patients with ES-SCLC in China. Reduced the price of serplulimab could increase its cost-effective.

Objective: This article examined the cost-effectiveness of zanubrutinib and ibrutinib for managing relapsed and refractory chronic lymphocytic leukemia from the viewpoint of payers in China and the US.

Methods: Markov models were employed to conduct comparisons. Baseline characteristics and clinical data were extracted from the ALPINE study. The cost-effectiveness outcome indicators encompassed cost, quality-adjusted life years, and the incremental cost-effectiveness ratio.

Results: The Markov model analysis revealed that the zanubrutinib group incurred an incremental cost per patient of $-24,586.53 compared to the ibrutinib group. The zanubrutinib group exhibited an incremental utility per capita of 0.28 quality-adjusted life years, resulting in an incremental cost-effectiveness ratio of $-88,068.16 per quality-adjusted life year, which is lower than the payment threshold in China. The willingness-to-pay value in China for 2022 was three times the country's gross domestic product per capita. In the US, patients in the zanubrutinib group experienced per capita incremental costs of $-79,421.56, per capita incremental utility of 0.28 quality-adjusted life years, and an incremental cost-effectiveness ratio of $-284,485.45 per quality-adjusted life year.

Conclusion: For Chinese payers, zanubrutinib exhibited superior cost-effectiveness compared to ibrutinib. Zanubrutinib proved to be a more affordable option for US payers when considering the payment threshold.

Introduction: This paper summarizes the results from a forum of healthcare experts, academia representatives, and public agency officials from emerging and established market countries on Value-Based Healthcare (VBHC) and Health Technology Assessment (HTA). Presentations from experts provided insights into current developments and challenges, followed by interactive roundtable discussions. Emerging markets have unique healthcare systems, patient populations, resource constraints and needs.

Areas covered: Each roundtable explored specific topics including the role of HTA and Real-world evidence (RWE) in healthcare decision-making, challenges in biosimilar value assessment and incorporating non-price criteria reflecting context-related specifications of emerging markets such as the multifaceted nature of value in healthcare decision-making, emphasizing stakeholder perspectives and system complexities.

Expert opinion: RWE emerged as important in understanding biosimilar value recognition and decision-making processes, with insights into its applications and challenges. Recommendations were provided for utilizing Multi-Criteria Decision Analysis (MCDA) in pharmaceutical procurement, particularly for off-patent medicines, underscoring the importance of comprehensive evaluation frameworks and adherence to value-based principles. Overall findings suggest avenues for collaboration between industry, academia, and public agencies to address implementation barriers and promote equitable, efficient, and high-quality healthcare systems in emerging markets through public-private partnerships, joint capacity building and training initiatives, and knowledge transfers.

Introduction: Chronic kidney disease (CKD) is a severe, progressive condition with a significant economic burden. We performed a systematic review to assess the cost-effectiveness of sodium-glucose cotransporter 2 (SGLT2) inhibitors in treating CKD.

Methods: A comprehensive search was conducted across PubMed, Embase, Web of Science, Scopus, INAHTA, NHS EED, and relevant websites. Two reviewers independently screened titles and abstracts, extracted data, and assessed study quality using CHEERS 2022 and Phillips's checklist.

Results: Thirteen model-based cost-utility studies met the inclusion criteria, evaluating Empagliflozin (n = 3), Canagliflozin (n = 3), and Dapagliflozin (n = 8). Empagliflozin or Dapagliflozin plus standard care (SoC) was cost-effective compared to SoC alone in CKD patients, regardless of type 2 diabetes (T2D) status. In CKD patients with T2D, SGLT2 inhibitors combined with SoC were cost-saving in high-income countries under health system perspective whereas Dapagliflozin was not cost-effective compared to Canagliflozin. No study met all criteria of the CHEERS 2022 checklist, and most did not fully satisfy Phillips's checklist for economic models.

Conclusion: Adding SGLT2 inhibitors to SoC is cost-saving for treating CKD with T2D and cost-effective for CKD patients with or without T2D.

Registration: The review protocol was registered in the International Prospective Register of Systematic Reviews (PROSPERO) under registration number CRD42023469005.

Background: To analyze the time from drug registration to reimbursement recommendations, we examined medicinal products, including new clinical indications, registered by the EMA between 2014 and 2019 across various therapeutic areas.

Materials and methods: The Polish Agency for Health Technology Assessment and Tariffication (AOTMiT) was compared with 11 agencies in England, Wales, Ireland, Scotland, the Netherlands, Norway, France, Germany, New Zealand, Canada, Australia. A total of 1,942 recommendations published by 12 HTA agencies were analyzed.

Results: The time from registration to recommendation in Poland was statistically significantly longer than for the other countries. The analysis revealed noticeable differences in the time it takes from drug registration to recommendation across the countries included in this analysis. Analyzing trends from 2014 to 2019 across individual countries, there appears to be a slight tendency toward a decrease in the median time from registration to recommendation in many agencies.

Conclusions: This may suggest improvements in the processes of the recommending authorities and the companies responsible for providing data for assessment. Despite Poland having one of the longest times from registration to recommendation among the countries analyzed, there has been a clear year-over-year decrease in the time to publication of reimbursement recommendations.