Journal of Medical Economics最新文献

Aim: Demodex blepharitis (DB) is a chronic eyelid disease caused by an infestation of Demodex mites. The objective of this chart audit study was to characterize the patient journey and burden of illness associated with DB from an eye-care provider's perspective.

Methods: Retrospective medical data for patients with a confirmed diagnosis of DB were anonymously collected via ophthalmologists and optometrists in the US (collectively eye-care providers [ECPs]), in June-July 2022. Each eligible ECP completed a questionnaire and provided chart abstractions of 1-5 medical charts of patients with DB in an electronic case report form.

Results: All ECPs (N = 61) were ophthalmologists or optometrists who worked primarily in office-based private practices (97%). Data from 192 medical charts of patients with an ECP-confirmed diagnosis of DB were abstracted and their demographics included 54% males and 71% Caucasians. The mean time (±SD) since diagnosis was 11 (±9.0) years. Over 40% of patients were not examined for DB at initial presentation, and the average time from initial presentation to a definitive diagnosis for DB was 6.5 months. After DB diagnosis and across the period of observation until the time of the study, patients had a mean (±SD) of 3.2 (±2.8) follow-up visits due to DB and continued to use multiple off-label prescription and over-the-counter therapies for symptom management.

Limitation: Medical records may be incomplete or inconsistently reported and subject to recall bias.

Conclusions: The patient journey is lengthy with multiple challenges, including delay in DB diagnosis and recurring DB symptoms, underlining an urgent need for action to improve diagnosis and treatment of DB.

Introduction: Melanoma, responsible for most skin cancer deaths globally, has mortality rates expected to double by 2040. Pembrolizumab is a highly selective antibody approved for melanoma treatment and other cancers. Despite new treatments for melanoma, high treatment costs and long approval times limit patient access to new therapies. To support decision-making regarding metastatic melanoma therapies, a model was developed to calculate the number needed to treat (NNT) and the cost of preventing an event (COPE) using KEYNOTE-716 (NCT03553836) data.

Method: A cost-per-responder model comparing the clinical and economic impacts of pembrolizumab versus best supportive care (BSC) was developed considering a 52.8-month follow-up for recurrence-free survival (RFS) and distant metastasis-free survival (DMFS) in patients with resected high-risk melanoma. KEYNOTE-716 RFS and DMFS survival curves were used to calculate restricted mean survival time (RMST). The RMST was used to calculate NNT (NNT_RMST). The NNT_RMST calculates the NNT to result in a difference in mean survival time for a death or an event. NNT_RMST is subsequently used to quantify COPE outcomes.

Results: NNT for RFS was 5.3, reflecting the number of patients needed to treat to gain the additional difference observed in the mean RFS for resected high-risk type II (IIB and IIC) melanoma patients treated with pembrolizumab. For DMFS, the NNT_RMST was 7.8. The estimated COPE to prevent an RFS or DMFS event was Mexican Peso (Mex $) 9,554,593 (2024) and Mex $13,961,427, respectively.

Conclusions: NNT values for RFS and DMFS data were both lower than the published average NNT value for current melanoma therapies. This demonstrated that fewer additional patients need to be treated in order to avoid a recurrence or a distant metastases event, compared to currently available melanoma therapies. The NNT and COPE highlight the clinical and economic impact of introducing pembrolizumab therapy for the treatment of patients in resected high-risk stage II melanoma.

Introduction: Hepatocellular carcinoma (HCC), which accounts for about 90% of all primary liver cancer cases, is the fifth most common malignancy and the second leading cause of cancer-related mortality worldwide. This study aims to analyse the differential costs of HCC-related hospital admissions compared to the general population in Spain.

Methods: A retrospective multicenter study analyzed inpatient admissions from a Spanish national discharge database, covering 90% of hospitals between 2010 and 2022. HCC-related admissions were identified using ICD-9 and ICD-10 codes, while control admissions were selected from the general population in the same database without an HCC diagnosis. The direct hospitalization cost was included, covering medical examinations, procedures, medications, surgeries, personnel and equipment. Statistical methods, including nearest-neighbor matching, propensity score matching, and a generalized linear model, were used to estimate differential costs and to ensure comparability based on age, gender, and Charlson Comorbidity Index (CCI).

Results: A total of 199,670 HCC-related hospital admissions and 200,000 control admissions were analyzed. Most HCC-related admissions involved male patients (78%) aged 66-85 years, with an average CCI of 5.18. HCC-related admissions incurred significantly higher costs, with an estimated differential cost of €1,303.68 using GLM, €1,804.25 via propensity score matching, and €1,767.77 using nearest-neighbor matching. Total costs per HCC admission ranged between €1,000 and €31,000.

Conclusions: HCC-related hospital admissions impose a significantly higher economic burden due to the complexity of care. Given the high mortality and resource utilization, advancements in early detection, treatment, and cost-effective interventions are needed to improve patient outcomes and reduce healthcare costs.

Aim: Asthma is a heterogeneous respiratory condition often classified into distinct phenotypes. Severe asthma, characterized by uncontrolled symptoms despite optimal treatment, imposes a significant burden on healthcare systems, particularly in low- and middle-income countries. This study evaluates the cost-effectiveness of mepolizumab compared with other interleukin (IL)-5 pathway inhibitors, benralizumab and reslizumab, in treating severe asthma with an eosinophilic phenotype in Chile.

Materials and methods: A Markov cohort model was developed to compare mepolizumab (100 mg subcutaneously every four weeks) with benralizumab (30 mg subcutaneously every four weeks for the first three doses, every eight weeks subsequently) and reslizumab (3 mg/kg intravenously every four weeks), both as add-on therapies to standard care. Data from the Mepolizumab as Adjunctive Therapy in Patients with Severe Asthma (MENSA) clinical trial and a network meta-analysis were used. Utility values were extracted using the EuroQoL 5-Dimension questionnaire (EQ-5D-5L) questionnaire. Probabilistic and one-way sensitivity analyses assessed model robustness.

Results: Mepolizumab demonstrated dominance with probability over 95% when compared with benralizumab and reslizumab. Cost savings ranged from 37,000 United States dollars (USD) to 104,000 USD, with an increase of 0.52 to 0.55 quality-adjusted life years. Mepolizumab was also associated with a lower incidence of exacerbations and asthma-related deaths. Sensitivity analyses confirmed the stability of the model outcomes across key parameters.

Limitations: Limitations of the economic model are related to the lack of direct comparisons between mepolizumab and other biologics. Additionally, the absence of data on continuation criteria required estimating relative risks for the overall population.

Conclusions: Mepolizumab offers greater efficacy and cost savings compared to benralizumab and reslizumab for eosinophilic asthma, providing essential insights for improving asthma management and informing healthcare policies in Chile.

Objective: This study compares the direct healthcare costs of anti-VEGF therapies, including treat-and-extend (T&E) and other durable regimens, for unilateral neovascular age-related macular degeneration (nAMD) and diabetic macular oedema (DMO) in Switzerland.

Methods: An adapted cost-minimisation model estimated healthcare costs over two years for aflibercept 2 mg, aflibercept 8 mg, faricimab, ranibizumab, and ranibizumab biosimilars using clinical trial injection frequencies. Break-even analyses identified the medication prices and injection frequencies required for higher-cost therapies to achieve cost parity with the least expensive options. A one-way sensitivity analysis (OWSA) assessed key drivers of cost outcomes.

Results: Aflibercept 8 mg was estimated to be associated with the lowest treatment costs for both indications (CHF 11,814 for nAMD; CHF 11,242 for DMO). Faricimab (CHF 13,737) and aflibercept 2 mg (CHF 15,243) followed in nAMD and DMO. Ranibizumab and its biosimilars incurred the highest costs: for nAMD, biosimilars ranged from CHF 16,243 to CHF 17,497 and the reference product reached CHF 18,424; for DMO, biosimilars ranged from CHF 18,187 to CHF 19,596, with the reference product at CHF 20,637. Break-even analyses for nAMD showed that prices would need to drop by -22% (faricimab, CHF 644) to -64% (ranibizumab reference, CHF 218) relative to aflibercept 8 mg. For DMO, reductions ranged from -42% (aflibercept 2 mg, CHF 493) to -81% (ranibizumab reference, CHF 114). The OWSA highlighted medication price and injection frequency as primary cost drivers.

Conclusions: This study estimated that the potentially minimized injection frequency of aflibercept 8 mg in a clinical trial regimen may result in the lowest treatment costs for nAMD and DMO, followed by faricimab and aflibercept 2 mg, respectively.

Aims: To predict the budget impact of Symvess (Symvess is a trademark of Humacyte Global, Inc.) (acellular tissue engineered vessel-tyod [ATEV]) for extremity arterial trauma repair when autologous vein repair is not feasible.

Materials and methods: The 3-year budget impact of adding ATEV as a repair option alongside autologous vein, prosthetic graft, and "non-autologous other" grafts was evaluated from the perspectives of a Level I trauma center and third-party commercial payers. Conduit-specific complication rates were obtained from two clinical studies for ATEV and from the published literature and analysis of the PROOVIT registry for other conduits. Costs were compared pre- and post-ATEV availability. Conduit-related costs and complications included conduit infections, amputations, vein harvest site infection, surgical re-interventions, rehabilitation after amputation, and 12-month post-discharge costs. Impact on operating room (OR) time and readmissions was evaluated. A sensitivity analysis was conducted to evaluate parameter uncertainty.

Results: With introduction of ATEV, there was a 29.8% reduction in amputations and a 29.5% reduction in graft infections over 3 years. From a Level I trauma center perspective, seven patients were expected to receive an ATEV over 3 years, with cumulative cost savings of $80,650 (2.3% decrease). OR time would decrease by 8.6 h, and readmission-related costs would be reduced by 16.7% with ATEV availability. From the third-party commercial payer perspective, 35 patients were expected to receive ATEV, with a budget impact showing a savings of -$0.08 per member per month after 3 years. For trauma centers, sensitivity analysis showed that cost drivers were amputation risk associated with "non-autologous other" graft types and market share of autologous vein (short ischemia time).

Limitations: Uncertainty surrounding model parameters.

Conclusions: ATEV was projected to be cost-saving over 3 years for both trauma centers and third-party payers due to reductions in the costs related to amputations and conduit infections.

Aims: This study aimed to establish a real-world benchmark of recovery following rotator cuff repair (RCR) using healthcare claims data. Secondary objectives included determining the effect of comorbidities and complications such as joint contracture, additional procedures, and rehospitalizations on the recovery timeline and costs.

Materials and methods: Healthcare claims data from the IBM MarketScan Commercial Claims and Encounters Database (2015-2018) were reviewed to determine costs and recovery time after RCR. Costs and recovery duration (index surgery to last therapy claim) were calculated. Subgroup analyses assessed the effects of comorbidities (diabetes, obesity, peripheral vascular disease, cardiovascular disease) and postoperative events (revision, motion restoring surgery (MRS), complication-related surgery, and nonoperative hospitalization) on outcomes. Perioperative complications including joint fibrosis/contracture, infection, and pulmonary embolus were also reported. Descriptive statistics including medians with interquartile ranges (IQR) were reported.

Results: In the 14,947 patients included in analysis, median index surgery cost was $11,454 (IQR = $8,169-$17,204). Median recovery was 153 days (IQR = 79-683). Development of postoperative shoulder contracture or adhesive capsulitis added a median of 162 recovery days and nearly doubled costs. Patients requiring surgery for a complication had 3.5-fold longer recoveries and 5-fold higher costs than those without complications. MRS increased recovery time and costs nearly 3-fold, and patients undergoing MRS were 7 times more likely to require arthroplasty. Comorbidities extended recovery by 30-90 days, modestly increased costs, and were associated with a 2-3 times higher frequency of pulmonary embolism.

Limitations: Claims data may be affected by coding inconsistencies, lack of clinical detail, and inability to capture medication costs or outcomes beyond the last therapy claim.

Conclusions: This study defined a benchmark for recovery after RCR and found that complications including contracture and motion restoring surgery substantially increased recovery time and costs. These benchmarks can guide earlier identification of patients at risk for delayed recovery and help in evaluating strategies to reduce economic burden and improve outcomes.

Aims: Olaparib is approved for the treatment of germline BRCA mutant (gBRCAm) high-risk early breast cancer (eBC) following treatment with neoadjuvant or adjuvant chemotherapy. The potential long-term outcomes of widespread germline BRCA testing and treatment with olaparib in the US have not been quantified.

Methods: We developed a decision-analytic model comparing a scenario with BRCA testing and olaparib treatment to a scenario with no testing and no treatment in an olaparib-eligible population. Olaparib-eligible population estimates were derived from published literature; long-term treatment outcomes were based on a published cost-effectiveness analysis. Breast cancer recurrences and life-years were projected over a lifetime. Scenario analyses were conducted to test different high-risk and testing uptake assumptions.

Results: We estimated that 3,983 eBC patients in the US were eligible for olaparib in 2024. Compared with no testing and no treatment, testing and olaparib treatment resulted in 272 (22% reduction) and 68 (22% reduction) fewer metastatic breast cancer (mBC) and non-metastatic breast cancer (non-mBC) recurrences, respectively, over a lifetime. A 9% increase in life expectancy would be achieved from adopting testing and olaparib treatment. Over 10 years, we estimated 40,094 olaparib-eligible eBC patients. 2,496 mBC (22% reduction) and 618 non-mBC recurrences (22% reduction) would be prevented, and 78,672 life-years would be saved vs no testing. Scenario analyses with different high-risk definitions and testing assumptions demonstrated a maintained clinical benefit (range of 82 - 552 mBC and 20 - 137 non-mBC recurrences avoided over a lifetime).

Limitations: Results were based on long-term outcomes modeled on the results of the OlympiA clinical trial; these uncertainties were evaluated using sensitivity analyses.

Conclusion: BRCA testing and subsequent treatment with olaparib results in fewer recurrences in eBC and a longer life expectancy vs no testing and no treatment, suggesting there is substantial clinical value in widespread BRCA testing for this population.

Aims: This study hypothesized that greater continuity of care (CoC) would be associated with lower all-cause healthcare resource utilization and improved overall survival (OS) among patients with chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL) among patients who received covalent Bruton tyrosine kinase inhibitor (cBTKi)-based therapy.

Methods: Optum's de-identified Clinformatics^® Data Mart Database was used for this retrospective study. Patient-level CoC measured by continuity of hematologist/oncologist provider care was evaluated using published measures; the Herfindahl-Hirschman Index (HHI) was the primary measure (range 0 = no continuity to 1.0 = complete continuity). Outcomes included all-cause emergency room (ER) visits, inpatient hospitalizations, and OS. Multivariable regression models (logistic, negative binomial, and Cox proportional hazards), adjusted for baseline covariates, were conducted to evaluate the relationship of CoC with outcomes.

Results: In total, 5,990 patients were included in the analysis; median follow-up was 31.8 months. Median HHI was 0.7210 (interquartile range = 0.4749, 1.0000). With higher CoC, there were lower odds of having an ER visit (HHI odds ratio [OR] = 0.89; 95% confidence interval [CI]: 0.87-0.91; p < 0.0001), lower number of ER visits (HHI rate ratio [RR] = 0.93; 95%CI 0.92-0.94; p < 0.0001), lower odds of inpatient hospitalization (HHI OR = 0.85; 95%CI: 0.84-0.87; p < 0.0001), and lower number of hospitalizations (HHI RR = 0.89; 95%CI: 0.88-0.90; p < 0.0001). There was no significant difference in OS (HHI hazard ratio = 0.99 (95%CI: 0.97-1.01) p = 0.18.

Limitations: Causality cannot be inferred in this retrospective study.

Conclusions: Greater CoC was significantly associated with reduced ER visits and reduced hospitalization, among patients diagnosed with CLL who received cBTKi therapy. While interpretation may be limited in the retrospective design, sensitivity and post-hoc analyses support this relationship. The findings from this study suggest the importance of maintaining a consistent oncologist/hematologist for the patient with CLL to reduce these healthcare events; however, causality cannot be inferred from this study.