Expert Review of Pharmacoeconomics & Outcomes Research最新文献

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.

Objective: To evaluate the cost-effectiveness of a single-pill combination (SPC) of perindopril/amlodipine/indapamide versus its free equivalent combination (FEC) in adults with hypertension in Italy.

Methods: A Markov model was developed to perform a cost-utility analysis with a lifetime horizon and an Italian healthcare payer's perspective. In the model, the additional effect of the SPC on blood pressure level compared with the FEC was translated into a decreased risk of cardiovascular events and CKD, which was modeled via Framingham risk algorithms. Difference in persistence rates of SPC and FEC were modeled via discontinuation rates.

Results: A perindopril/amlodipine/indapamide SPC is associated with lower cost and better health outcomes compared to its FEC. Over a lifetime horizon, it is associated with a 0.050 QALY gain and cost savings of €376, resulting from lower cardiovascular event rates. In the alternative scenario, where different approach for modeling impact of adherence was considered, incremental gain of 0.069 QALY and savings of €1,004 were observed. Results were robust to sensitivity and scenario analyses, indicating that use of this SPC is a cost-effective strategy.

Conclusions: The findings indicate that a perindopril/amlodipine/indapamide SPC is a cost-saving treatment option for hypertension in Italy, compared to its FEC.

Objective: In the ASTRUM-004 trial, serplulimab plus chemotherapy demonstrated significantly improved survival and controllable safety. This study assessed the cost-effectiveness of serplulimab plus chemotherapy in advanced squamous non-small cell lung cancer (sqNSCLC), considering the perspective of the Chinese healthcare system.

Methods: A decision tree and a Markov model were constructed to simulate the treatment. The interesting results included total cost, life-years (LYs), quality-adjusted life-years (QALYs) and incremental cost-effectiveness ratios (ICERs). Scenario, one-way and probabilistic sensitivity analyses were used to examine model instability.

Results: Compared with placebo plus chemotherapy, serplulimab plus chemotherapy had an ICER of $55,539.46/QALY ($47,278.84/LY). The ICERs were estimated to be $58,706.03/QALY, $48,978.34/QALY and $59,709.54/QALY inpatients with programmed death-ligand 1 expression level of tumor proportion score (TPS) < 1%, 1% ≤ TPS < 50%, and TPS ≥ 50%. The cost-effective prices of serplulimab were $168.276/100 mg, $349.157/100 mg, and $530.039/100 mg at the willingness-to-pay threshold of $12,574.30/QALY, $25,148.60/QALY, and $37,722.90/QALY. Patient weight and price of serplulimab created the most significant impact. Presently, the probability of serplulimab plus chemotherapy being cost-effective was 14.15%.

Conclusion: Compared with placebo plus chemotherapy, serplulimab plus chemotherapy might not be cost-effective in the first-line treatment for advanced sqNSCLC.

Introduction: Multiple sclerosis (MS) is a persistent condition characterized by immune-mediated processes in the central nervous system, affecting around 2.8 million individuals globally. While historically less prevalent in the Middle East and North Africa (MENA) region, recent trends mirror the global rise in MS.

Area covered: The impact of MS is substantial, particularly in the MENA region, with costs per patient surpassing nominal GDP per capita in certain countries. Disease-modifying therapies aim to alleviate MS effects, but challenges persist, especially in managing progressive MS as it shifts from inflammatory to neurodegenerative phases. Limited resources in the MENA region hinder care delivery, though awareness initiatives and multidisciplinary centers are emerging. Contrary to global projections of a decline in the MS market, the MENA region is poised for growth due to increased prevalence, healthcare expenditures, and infrastructure investments.

Expert opinion: This review underscores the urgent necessity for effective treatments, robust disease management, and early diagnosis in tackling MS's repercussions in the MENA region. Bolstering resources tailored to MS patients and elevating the quality of care stand as pivotal strategies for enhancing health outcomes in this context. Taking decisive action holds the key to enhancing the overall well-being of individuals grappling with MS.

Background: Clostridioides difficile is the most common cause of healthcare-associated diarrhea. Research suggests that treating C. difficile infections (CDI) with fidaxomicin (FDX) is more effective than vancomycin (VAN), with potential cost savings. The objective was to calculate the budget impact of FDX treatment compared to VAN from a German payer perspective.

Research design and methods: The analysis used real-world data of patients discharged from University Hospital Cologne between Jan-01-2018 and Dec-31-2019. We identified recurrent and non-recurrent CDI cases and calculated direct treatment costs based on G-DRG flat rates. To calculate average costs per treatment and the budget impact, recurrence probabilities for VAN and FDX were taken from published evidence (28-day and 90-day scenarios).

Results: Totally, 475 cases were analyzed, thereof 421 non-recurrent, causing mean costs of €32,901 per case (95% CI: 27.752-38.050). Thirty-two patients experienced a recurrence within 28 days, yielding mean costs of €10,952 (95% CI: 5.627-16.277) for their additional hospital stay. The resulting budget impact was €1,303 (95% CI: 670-1.937) in favor of FDX, ranging from €148.34 to €2,190.30 in scenario analyses.

Conclusion: The analysis indicates FDX treatment can lead to cost savings compared to VAN. Future research should focus on specific patient groups, such as refractory CDI patients.

Objective: The current study aims to develop an algorithm for mapping the WHODAS 2.0 to the EQ-5D-5 L for patients with mental disorders.

Methods: This cross-sectional study was conducted at the Institute of Mental Health and Community Wellness Clinics in Singapore between June 2019 and November 2022. We included four regression methods including the Ordinary Least Square (OLS) regression, the Tobit regression model (Tobit), the robust regression with MM estimator (MM), and the adjusted limited dependent variable mixture model (ALDVMM) to map EQ-5D-5 L utility scores from the WHODAS 2.0.

Results: A total of 797 participants were included. The mean EQ-5D-5 L utility and WHODAS 2.0 total scores were 0.615 (SD = 0.342) and 11.957 (SD = 8.969), respectively. We found that the EQ-5D-5 L utility score was best predicted by the robust regression model with the MM estimator. Our findings suggest that the WHODAS 2.0 total scores were significantly and inversely associated with the EQ-5D-5 L utility scores.

Conclusion: This study provides a mapping algorithm for converting the WHODAS 2.0 scores into EQ-5D-5 L utility scores which can be implemented using a simple online calculator in the following web application: https://eastats.shinyapps.io/whodas_eq5d/.

Objective: This study aims to conduct a cost-effectiveness analysis of pembrolizumab in combination with chemotherapy for HER2-negative advanced gastric cancer in China.

Methods: A partitioned survival approach model was constructed to simulate the progression of HER2-negative advanced gastric cancer and evaluate the outcomes of different treatment strategies. We calculated incremental cost-effectiveness ratios (ICER) to assess the cost associated with each quality-adjusted life-year (QALY) gained. One-way sensitivity analysis and probabilistic sensitivity analysis were performed to assess robustness and reliability.

Results: The analysis conducted in the base case demonstrated that the ICER associated with pembrolizumab was $177405.83/QALY gained in all population. In the subgroup analysis, it was found that individuals with a PD-L1 CPS ≥ 1 and those with a PD-L1 CPS ≥ 10 had ICERs of $152397.06/QALY and $109534.13/QALY, respectively. All ICER values for both the all population groups and the subgroups exceeded the WTP threshold in China. Our analysis shows the robustness of these results, as they remained consistent when input parameters were varied within a ± 25% range.

Conclusion: The findings of this cost-effectiveness analysis suggest that pembrolizumab in combination with chemotherapy is not a cost-effective treatment option for HER2-negative advanced gastric cancer in China.

Background: While medical costs of chickenpox have been researched, little is known about indirect costs. Understanding total costs is important for decisions about vaccination. This study estimated the value of lost productivity of adults missing work to care for children with chickenpox.

Research design and methods: It comprised an international literature review, online survey of 1,526 parents of children aged 1 to 11 years, and computation of indirect costs of chickenpox in the UK. The survey covered chickenpox episodes amongst respondents' children, time children took off school/nursery, and work absenteeism by parents/caregivers caring for them.

Results: Respondents reported on 2,283 children, of whom 52% (1185/2283) experienced chickenpox. Almost half (591/1185) missed days of school/nursery, averaging 5.6 days missed. In 260 cases of 542 adults providing data with such a child, an adult missed work to care for the child. The daily value of this lost productivity was £170. There were approximately 200,000 GP consultations for chickenpox and 625,000 births annually, suggesting annual chickenpox incidence lies between these figures. The estimated annual UK productivity loss due to chickenpox is £20 -£70 million ($25-$90 million).

Conclusions: Annual value of lost productivity due to chickenpox is in range £20 to £70 million.