Expert Review of Pharmacoeconomics & Outcomes Research最新文献

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.

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.

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: 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.

Introduction: The standard treatment for gastroesophageal reflux disease (GERD) is proton pump inhibitors (PPIs). In selected cases, Nissen fundoplication is offered as a surgical treatment option, but alternative endoscopic and minimally invasive surgical alternatives are emerging. RefluxStop is a new technology for the treatment of GERD.

Research design and methods: A cost-effectiveness analysis of RefluxStop in comparison to PPI therapy and Nissen fundoplication in the Swedish healthcare setting was conducted using a Markov model and available comprehensive population and clinical trial-based long-term data. Benefits were measured in quality-adjusted life-years (QALYs). Uncertainty was determined by deterministic and probabilistic sensitivity analyses.

Results: The base case incremental cost-effectiveness ratios (ICERs) for RefluxStop in comparison to PPIs and Nissen fundoplications were SEK 48,152 (€ 4,531) and SEK 62,966 (€ 5,925) per QALY gained, respectively. At a cost-effectiveness threshold of SEK 500,000 per QALY gained, RefluxStop has a high likelihood of being cost-effective, with probabilities of 96% and 100% against Nissen fundoplication and PPIs, respectively. The results of the model remained robust with sensitivity analysis.

Conclusions: RefluxStop may offer a highly cost-effective long-term treatment alternative for chronic GERD patients over lifelong PPI therapy, but also in comparison with laparoscopic Nissen fundoplication.

Introduction: Traditional economic evaluations typically focus on direct health effects and costs offset to the healthcare system. However, vaccines offer significant indirect benefits beyond direct health effects, such as herd immunity, reduced force of infection, reduction in antimicrobial resistance, and others. Failure to consider these benefits while evaluating vaccines may undervalue vaccines. Therefore, it is argued that the full value of vaccines should be estimated by incorporating these broader benefits.

Areas covered: This review presents the broader value domains proposed in literature by various frameworks, and their definitions. The review of economic evidence of vaccine use in India to discuss to what extent these broader value domains have been considered in economic evaluations in India has been presented. We also discuss specific considerations that need to be taken care of while developing value frameworks or guidelines for the economic evaluation of vaccines.

Expert opinion: To develop a comprehensive framework tailored to the country needs, prioritize relevant value domains and optimal methodologies based on the country's healthcare context, and data availability. These value domains must align with people's as well as decision-makers preferences to ensure economic assessments are relevant and actionable.

Introduction: We evaluated the comparative efficacy of six later-line (≥3) therapies for metastatic colorectal cancer (mCRC) over placebo. We applied a novel statistical method of reconstructing pseudo-patient-level data (pseudo-IPD) to inform a network meta-analysis of survival curves that considers shape in addition to scale parameters.

Methods: A literature search yielded 10 phase II/III trials. We digitized all survival curves and applied a novel method incorporating curve coordinates, patients-at-risk, and events reported to generate pseudo-IPD. Using fitted random effects lognormal distributions, we estimated the survival proportions and HRs (95CrI) of progression-free (PFS) and overall survival (OS) over 12 months of follow-up.

Results: Compared to placebo, in ascending order, 12-month OS HRs were 0.50 (95% CrI = 0.35, 0.69; PFS = 0.11 (95% CrI = 0.06, 0.14)) for TAS+bevacizumab; 0.71 (95% CrI = 0.51, 0.97; PFS = 0.26 (95% CrI = 0.16, 0.41)) for regorafenib; 0.75 (95% CrI = 0.61, 0.91; (PFS = 0.24 (95% CrI = 0.17, 0.31)) for TAS-102; 0.80 (95% CrI = 0.79, 0.90; PFS = 0.18 (95% CrI = 0.13, 0.24)) for fruquintinib; 0.83 (95% CrI = 0.50, 0.99; PFS = 0.42 (95% CrI = 0.20, 0.75)) for atezolizumab+cobimetinib; and 1.03 (95% CrI = 0.55, 1.65; PFS = 0.67 (95% CrI = 0.29, 1.01)) for atezolizumab.

Conclusion: In this independent NMA of survival data, all later-line mCRC therapies but atezolizumab monotherapy exhibited superiority in 12-month PFS and OS over placebo. TAS+bevacizumab emerged as the most dominant option and may be the preferred choice, with fruquintinib, regorafenib, and TAS-102 monotherapy showing statistically significant but lower PFS and OS benefits.

Registration: PROSPERO: CRD42022371953.

Introduction: Long-term care (LTC) refers to care and support services that are required by individuals who lack the ability to perform important daily routines and may be dependent on others for personal, social, and medical needs over a sustained period of time. LTC may be broadly categorized into formal and informal care, where formal care is provided by professionals who are compensated to provide these services and informal care captures the care services provided without compensation by family members, friends, or other unpaid individuals.

Areas covered: In this narrative review, we identify and synthesize evidence to evaluate the cost of long-term care while balancing the needs of caregivers. We searched Embase and EconLit for studies published from 2010 to November 2023. Our search strategy used a combination of keywords such as 'long-term care,' 'caregiver burden,' 'caregiver support,' 'cost of care,' and 'caregiver wellbeing.' We include both formal and informal LTC, as well as predictors of caregiver wellbeing.

Expert commentary: This review highlights the global variability in LTC costs and the significant burden on caregivers, emphasizing the need for policy interventions and comprehensive insurance schemes. Future research should focus on standardized assessment tools, intervention effectiveness, and integrating caregiver support into healthcare models, ensuring holistic and sustainable LTC solutions.

Introduction: Considering the prospects of increased prevalence and disability due to neck and low back pain, it is relevant to investigate the care processes adopted, to assist future public policies and decision-making for a better allocation of resources. Objective: the aim of this study was to estimate the costs arising from inpatient and outpatient care of individuals with Neck Pain (NP) and Low Back Pain (LBP) in Brazil, between 2010 and 2019.

Methods: This is a cost-of-illness study from the perspective of the Brazilian public health system, based on health conditions with high prevalence (neck and low back pain). Data were presented descriptively using absolute and relative values.

Results: Between 2010 and 2019, the health system spent more than $600 million (R$ 2.3 billion) to treat NP and LBP in adults, and LBP accounted for most of the expenses. Female had higher absolute expenses in inpatient care and in the outpatient system.

Conclusion: Our study showed that the costs with NP and LBP in Brazil were considerable. Female patients had higher outpatient costs and male patients had higher hospitalization costs. Healthcare expenses were concentrated for individuals between 34 and 63 years of age.