Journal of Medical Economics最新文献

Objective: The Institute for Clinical and Economic Review (ICER) publishes annual Unsupported Price Increase (UPI) reports on prescription drugs, which have gained attention from policymakers and healthcare stakeholders. These reports do not adjust for inflation in their analyses of net price changes. This study aimed to evaluate the economic context of reported drug price increases in ICER's UPI reports by applying inflation adjustments to the estimated impact on healthcare spending.

Methods: Separate inflation adjustments using the Medical Consumer Price Index (Medical CPI) and the Consumer Price Index for All Urban Consumers (CPI-U) were applied to unadjusted UPI findings. The study analyzed data from four most recent UPI reports spanning pricing data from 2018 to 2019 (report year 2020) to 2021 to 2022 (report year 2023), focusing on drugs classified as having "unsupported" price increases.

Results: Inflation-adjusted analyses showed lower estimates of real changes in spending compared to ICER's unadjusted figures across all drugs analyzed. Across the four UPI reports, the average report-year difference in Medical CPI inflation adjustment spending versus unadjusted findings was 53%, whereas the average report-year difference in CPI-U inflation adjustment spending versus unadjusted findings was 80%. In the most recent report year of 2023, ICER's unadjusted estimate of a $1.3 billion increase in healthcare spending transformed to a $134 million decrease when adjusted by Medical CPI and a $1.51 billion decrease when adjusted by CPI-U.

Conclusion: This study demonstrates that inflation adjustment can alter not only the findings but also the interpretation of drug spending changes in UPI reports. The findings highlight the importance of considering economic context in evaluating drug pricing trends and suggest that unadjusted figures may overstate the impact of real price changes. Given the influence of these reports on policy discussions, incorporating inflation adjustments into analyses is one of many steps toward informing evidence-based policy decisions.

Aims and background: The current report details transition of outsourced conventional dialysis therapy in the ICU services to an in-house prolonged intermittent renal replacement therapy (PIRRT) service model as a quality improvement project using the Tablo Hemodialysis System, Outset Medical, Inc. The goals were aimed at maintaining or improving clinical outcomes, while also reducing dialysis-related nursing staff burden and dialysis-related treatment costs.

Methods: A descriptive comparative analysis was conducted of renal replacement therapy (RRT) of ≥6 hours in duration performed in the 1 year prior and 1 year after the ICU's in-house program launch using a PIRRT model including sequential 24-h treatments when medically necessary.

Results: Overall, there were 145 intensive care unit (ICU) stays among 145 patients with 13,641 h of conventional ICU dialysis in the year prior to program transition. In the year post, there were 116 ICU stays among 116 patients with 5,098 h of PIRRT. By employing a PIRRT and sequential 24-h treatment strategy vs. the prior outsourced model, the mean dialysis treatment hours per patient were reduced (Pre, 94.1 h with 214 treatment starts; Post, 43.9 h with 370 treatment starts), increasing ICU nurse productivity by 50.2 h per patient. Overall, ICU length of stay and ICU mortality declined post-service transition by 4.8 days and 9.8 percentage points (pp), respectively, overall, and in the non-COVID subset by 1.6 days and 3.1 pp, respectively.

Conclusions: Insourcing RRT with an innovative technology that can provide both PIRRT and 24-h sequential treatments can maintain or improve clinical outcomes in critically ill patients requiring RRT in the ICU, while reducing dialysis-related costs.

Background: Ebola virus disease (EVD) continues to be a major public health threat globally, particularly in the low-and-middle-income countries (LMICs) of Africa. The social and economic burdens of EVD are substantial and have triggered extensive research into prevention and control. We aim to highlight the impact and economic implications, identify research gaps, and offer recommendations for future economic studies pertaining to EVD.

Method: We conducted a comprehensive librarian-led search in PubMed/Medline, Embase, Google Scholar, EconLit and Scopus for economic evaluations of EVD. After study selection and data extraction, findings on the impact and economics of EVD were synthesized using a narrative approach, while identifying gaps, and recommending critical areas for future EVD economic studies.

Results: The economic evaluations focused on the burden of illness, vaccine cost-effectiveness, willingness-to-pay for a vaccine, EVD funding, and preparedness costs. The estimated economic impact of the 2014 EVD outbreak in Guinea, Liberia, and Sierra Leone across studies ranged from $30 billion to $50 billion. Facility construction and modification emerged as significant cost drivers for preparedness. The EVD vaccine demonstrated cost-effectiveness in a dynamic transmission model; resulting in an incremental cost-effectiveness ratio of about $96 per additional disability adjusted life year averted. Individuals exhibited greater willingness to be vaccinated if it incurred no personal cost, with a minority willing to pay about $1 for the vaccine.

Conclusions: The severe impact of EVD puts pressure on governments and the international community for better resource utilization and re-allocation. Several technical and methodological issues related to economic evaluation of EVD remain to be addressed, especially for LMICs. We recommend conducting cost-of-sequelae and cost-of-distribution analyses in addition to adapting existing economic analytical methods to EVD. Characteristics of the affected regions should be considered to provide evidence-based economic plans and economic-evaluation of mitigations that enhance resource allocation for prevention and treatment.

Objective: The sodium-glucose co-transporter-2 inhibitor empagliflozin was approved for treatment of adults with chronic kidney disease (CKD) on the basis of its demonstrated ability to slow CKD progression and reduce the risk of cardiovascular death. This analysis was performed to assess the cost-effectiveness of empagliflozin plus standard of care (SoC) vs SoC alone in the treatment of CKD in the UK.

Methods: A comprehensive, patient-level CKD progression model that simulates the evolution of risk factors for disease progression based on CKD-specific equations and clinical data was used to project a broad range of CKD-related complications. Patient baseline characteristics, distribution across Kidney Disease Improving Global Outcomes (KDIGO) health states, and changes in estimated glomerular filtration rate (eGFR), urine albumin-creatinine ratio (uACR), and other parameters while on treatment were derived from the EMPA-KIDNEY trial. UK cost and utilities/disutilities were sourced from the literature. Univariate and probabilistic sensitivity analyses were conducted. Annual discounting of 3.5% was applied on costs and outcomes.

Results: Over a 50-year horizon, SoC resulted in per-patient costs, life years, and QALYs of £95,930, 8.55, and 6.28, respectively. Empagliflozin plus SoC resulted in an incremental gain in life years (+1.04) and QALYs (+0.84), while decreasing per-patient costs by £6,019. Empagliflozin was more effective and less costly (dominant) with a net monetary benefit of £22,849 at the willingness-to-pay threshold of £20,000. Although treatment cost was higher for empagliflozin, this was more than offset by savings in kidney replacement therapy. Empagliflozin remained highly cost-effective in patients with and without diabetes, and across scenario and sensitivity analyses.

Limitations: This analysis is limited by reliance on short-term clinical trial data and by uncertainties in modelling CKD progression.

Conclusions: Empagliflozin as an add-on to SoC for treatment of adults with CKD represents cost-effective use of UK National Health Service (NHS) resources.

Aims: To compare healthcare resource utilization (HRU) and costs between patients with or without melanoma recurrence and between patients with distant or locoregional melanoma recurrence.

Methods: Patients aged ≥65 years with completely resected, stage IIB/IIC or III melanoma were identified from Surveillance, Epidemiology, and End Results-Medicare data and stratified based on whether they experienced a recurrence, and whether it was distant or locoregional (separately for each stage). The index date was the date of recurrence (recurrence group) or a randomly assigned date (non-recurrence group). Patients in the recurrence and non-recurrence groups were propensity score-matched 1:1 based on patient characteristics; HRU and healthcare costs were compared between the 2 groups and between patients with distant or locoregional recurrence during the ≤24 months following index.

Results: After matching, 507 pairs of patients with recurrent or non-recurrent stage IIB/IIC melanoma (236 patients with distant recurrence, 271 with locoregional) and 141 pairs of patients with recurrent or non-recurrent stage III melanoma (50 patients with distant recurrence, 91 with locoregional) were included. During the first year following recurrence, unadjusted HRU was generally higher in patients with versus without recurrence and patients with distant versus locoregional recurrence among both stage IIB/IIC and III cohorts. Patients who experienced recurrence incurred $6,474 (stage IIB/IIC) or $6,112 (stage III) per patient per month (PPPM) more in unadjusted, all-cause, total healthcare costs than patients without recurrence (both p < 0.001). Patients with distant recurrence incurred $7,292 (stage IIB/IIC) or $5,436 (stage III) PPPM more in unadjusted, all-cause, total healthcare costs than patients with locoregional recurrence (both p < 0.05).

Limitations: Melanoma recurrence was identified using a claims-based algorithm.

Conclusions: Economic burden is higher in patients with versus without melanoma recurrence and patients with distant versus locoregional recurrence. There is a high unmet need for adjuvant therapies that may help to prevent or delay recurrence.

Background: Triple Negative Breast Cancer (TNBC) is an aggressive subtype of breast cancer that can impact patients' employment and workforce participation. This study estimates how the employment effects of TNBC impact government tax revenue and public benefits expenditure in Switzerland, representing the fiscal burden of disease (FBoD), and likely consequences of introducing new treatment options.

Methods: A four-state cohort model was used to calculate fiscal effects for two treatments: Neoadjuvant pembrolizumab plus chemotherapy followed by adjuvant pembrolizumab monotherapy (P + C→P) and neoadjuvant chemotherapy alone (C). Lifetime present values of tax revenue, social benefit payments, and healthcare costs were calculated for the average population and those undergoing treatment to assess the FBoD.

Results: An average TNBC patient treated with C and P + C→P is expected to generate CHF128,999 and CHF97,008 less tax than the average population, respectively, and require increased social benefit payments. Compared to C, 75% of the incremental healthcare costs of P + C→P are estimated to be offset through tax revenue gains.

Conclusions: This analysis demonstrates that 75% of the additional costs of a new TNBC treatment option can be offset by gains in tax revenue. Fiscal analysis can be a useful tool to complement existing methods for evaluating new treatments.

Aims: To provide an update on the cost-effectiveness of the chimeric antigen receptor (CAR) T-cell therapies axicabtagene ciloleucel (axi-cel) and tisagenlecleucel (tisa-cel) for the treatment of relapsed/refractory (r/r) large B-cell lymphoma (LBCL) among patients who have previously received ≥2 lines of systemic therapy using more mature clinical trial data cuts (60 months for axi-cel overall survival [OS] and 45 months for tisa-cel OS and progression-free survival [PFS]).

Methods: A partitioned survival model consisting of three health states (pre-progression, post-progression and death) was used to estimate quality-adjusted life years (QALYs) and costs associated with axi-cel and tisa-cel over a lifetime horizon. PFS and OS inputs for axi-cel and tisa-cel were based on a previously published matching-adjusted indirect treatment comparison (MAIC). Long-term OS and PFS were extrapolated using parametric survival mixture cure models (PS-MCMs). Costs of CAR-T cell therapy drug acquisition and administration, conditioning chemotherapy, apheresis, CAR T-specific monitoring, stem cell transplant, hospitalization, adverse events, routine care, and terminal care were sourced from US cost databases. Health state utilities were derived from previous publications. Model inputs were varied using a range of sensitivity and scenario analyses.

Results: Compared with tisa-cel, axi-cel resulted in 2.51 additional QALYs and $50,185 additional costs (an incremental cost-effectiveness ratio [ICER] of $19,994 per QALY gained). In probabilistic sensitivity analysis (PSA), the ICER for axi-cel versus tisa-cel was ≤$50,000/QALY in 99.4% of simulations and ≤$33,500 in 99% of simulations. Axi-cel remained cost-effective versus tisa-cel (assuming a willingness-to-pay threshold of $150,000 per QALY) across a range of scenarios.

Conclusions: With longer-term survival data, axi-cel continues to represent a cost-effective option versus tisa-cel for treatment of r/r LBCL among patients who have previously received ≥2 lines of systemic therapy, from a US payer perspective.

Background and purpose: The incidence of end-stage renal disease (ESRD) in Sudan is increasing, affecting the economic status of patients, caregivers and society. This study aimed to measure ESRD's costs, including direct and morbidity indirect expenditures, and to investigate any associated factors and financial consequences.

Materials and methods: This cross-sectional study used a standardized questionnaire to collect data from 150 ESRD patients who had been receiving dialysis for at least one year before the time of data collection at 13 specialized renal centres in Khartoum state. Data about sociodemographic, clinical, and economic factors were gathered, and their relationship to the cost of ESRD was examined using both bivariate (Man Whitney test, Kruskal Wallis test and Spearman correlation) and multivariate analytical procedures (multivariate linear regression).

Results: This study reported a median direct per capita ESRD cost of 38 600 SDG ($1 723.2 PPP) annually with an interquartile range of 69 319.3 SDG ($3 094.6 PPP). The median morbidity indirect cost was estimated to be 0.0 ± 3 352 SDG ($ 0.0 ± 149.6 PPP) per annum. In 28.8% of cases, the patients were their family's primary income earner and over 85% were covered by medical insurance. Our study found that none of the study variables were significantly associated with the total cost of ESRD.

Conclusion and limitations: Our findings point out considerable direct out-of-pocket expenses and productivity losses for patients and their households. However, these results should be carefully applied for comparison between the different countries due to differences in the cost of medical interventions and insurance coverage. Further longitudinal studies and studies on health finance and insurance policies are recommended.