Journal of Medical Economics最新文献

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.

Aims: To compare all-cause and Alzheimer's disease (AD)-related healthcare resource utilization (HCRU) by cognitive stage.

Methods and materials: This retrospective study analyzed insurance claims data linked to electronic health records (01/01/2015-12/31/2021). Patients with ≥1 cognitive assessment (Mini-Mental State Examination or Montreal Cognitive Assessment) and ≥1 medical or pharmacy claim for an AD diagnosis or AD medications were included. Inverse probability of treatment weighting (IPTW) was used to address potential confounding. All-cause and AD-related HCRU were summarized per patient per year (PPPY) and compared between early AD and advanced AD cohorts (defined according to cognitive scores) using generalized linear regression models; adjusted incidence rate ratios (IRRs), and 95% confidence intervals (CI) were reported.

Results: A total of 193 patients were included (median age: 82 years; 63.2% female), 108 with early AD and 85 with advanced AD, with similar mean follow up. All-cause HCRU, on average, was similar between early AD and advanced AD cohorts (37.4 PPPY and 38.9 encounters PPPY, respectively). For AD-related HCRU, patients with early AD had fewer encounters PPPY, on average, than patients with advanced AD (1.26 and 3.88 encounters, respectively). Following IPTW adjustment, the advanced AD cohort had significantly higher overall AD-related HCRU (IRR: 3.64 [95% CI: 1.96-6.75], p < 0.001) and outpatient visits (IRR: 2.76 [95% CI: 1.68-4.54], p < 0.001) compared to the early AD cohort.

Limitations: The relatively small sample size of patients with linked claims and cognitive score data limited the ability to assess contribution of all encounter types to HCRU trends, as well as generalizability to the broader AD population.

Conclusions: Although all-cause HCRU was similar, patients with advanced AD incurred higher AD-related HCRU compared to patients living with early AD. Further research is needed to determine whether interventions earlier in disease progression can mitigate the AD-related healthcare burden for patients with advanced AD.

Aims: To quantify preferences for an adult combination vaccine for influenza and COVID-19 (flu + COVID) compared with standalone influenza and COVID-19 vaccines.

Materials and methods: This survey study used a series of direct-elicitation questions to assess preferences for a single-shot combination flu + COVID, standalone influenza, and standalone COVID-19 vaccines among US consumers (N = 601) and healthcare professionals (HCPs) (N = 299). Response frequencies described the proportion of each sample that would prefer a flu + COVID vaccine to standalone influenza and COVID-19 vaccines. A multivariate logit regression model explored how certain characteristics influenced the odds of selecting the flu + COVID vaccine over a standalone influenza vaccine.

Results: Most consumers (398/601; 66.2%) and HCPs (250/298; 83.9%) preferred a flu + COVID vaccine to a standalone influenza vaccine. When not forced to choose between flu + COVID and standalone influenza vaccines, most consumers again selected the flu + COVID vaccine (62.3%); 14.7% would prefer separate standalone influenza and COVID-19 vaccines, 8.3% a standalone influenza vaccine only, 7.3% a COVID-19 vaccine only, and 7.4% neither vaccine. Consumers aged ≥50 years with a body mass index ≥40, those aged ≥65 years who previously received a COVID-19 vaccine, and those who had previously experienced severe impacts from influenza were more likely to choose a flu + COVID vaccine over a standalone influenza vaccine than were consumers without these characteristics. HCPs whose practice stocks high-dose influenza vaccines were more likely to choose the flu + COVID vaccine for patients aged ≥65 with no risk factors and patients aged 18-64 with ≥1 risk factor over the standalone influenza vaccine.

Limitations: Results are subject to potential hypothetical, responder, selection, and information biases.

Conclusions: Most US consumers and HCPs would likely prefer a single-shot combination flu + COVID vaccine compared with standalone influenza and COVID-19 vaccines. Given the low COVID-19 vaccination coverage rates in the US, the availability of a combination flu + COVID vaccine could help increase COVID-19 vaccine coverage.

Objective: von Willebrand Disease (vWD) is the most common congenital bleeding disorder, with an estimated prevalence in Spain of 0.01%. The aim was to assess the cost-utility of Haemate-P compared with present alternatives in the treatment of vWD in Spain.

Methods: A Markov model was developed in Microsoft Excel to estimate the cost-effectiveness of various treatments for vWD over a lifetime horizon. Transition probabilities among health states were based on age and number of bleeding events. Treatment strategies compared included Haemate-P, Fanhdi, and Wilate in long-term prophylaxis (LTP) or on-demand treatment (ODT). Costs and quality-of-life were measured based on patient age, treatment, and number of bleeding events incurred. Both costs and utilities were discounted at 3%. One-way and probabilistic sensitivity analyses were performed.

Results: When comparing LTP regimens, Haemate-P was less costly and numerically more effective than both Fanhdi (incremental costs = -€1,313,845; incremental quality-adjusted life-years [QALY] = 0.13) and Wilate (incremental costs = -€2,233,940; incremental QALY = 0.29). For ODT, Haemate-P was assumed to have equal effectiveness as Fanhdi and Wilate but reduced the costs by €696,857 and €1,145,780, respectively. Haemate-P prophylaxis was more effective and less costly compared with Haemate-P on-demand in the base case (incremental costs = -€633,317; incremental QALY = 0.90). Results were generally robust to sensitivity analyses.

Conclusions: In patients with severe vWD experiencing a high bleed rate, Haemate-P prophylaxis is a less costly and potentially more effective treatment strategy and Haemate-P is cost-saving among on-demand strategies.

Aim: To compare all-cause and mental health (MH)-related short-term and long-term disability leaves and associated costs among patients in the United States with bipolar disorder (BP), major depressive disorder (MDD), or schizophrenia spectrum disorders (SCZ) before versus after cariprazine initiation.

Methods: Merative MarketScan Commercial and Health and Productivity Management (HPM) databases (January 2016 to December 2021) were utilized to identify adults diagnosed with BP, MDD, or SCZ with ≥2 pharmacy cariprazine claims (first claim = index), ≥3 months of cariprazine use (adjunctively for MDD), and continuous commercial insurance coverage and HPM eligibility during baseline (12 months pre-index) and ≥3 months post-index. Observation continued until cariprazine discontinuation, insurance or HPM eligibility end, 1 year post-index, or HPM data availability end. All-cause and MH-related disability claims, days, and costs were evaluated. Baseline versus post-index rates of disability claims (events) and days were compared using rate ratios (RR); costs were compared using mean cost differences. Comparisons were calculated from generalized estimating equation models. Analyses were replicated separately across indications.

Results: There were 489 patients overall (BP = 238, MDD = 233, SCZ = 18; mean age = 43.3 years; 60.7% female; mean follow-up = 7.6 months). All-cause rates of disability events and days following cariprazine initiation were 29% (RR = 0.71 [95% CI = 0.57, 0.86]) and 28% (0.72 [0.53, 0.94]) lower than baseline, respectively (both p < .05). MH-related rates of disability events and days were 40% (0.60 [0.43, 0.80]) and 43% (0.57 [0.34, 0.84]) lower, respectively (both p < .01). All-cause disability costs were $2,917 lower and MH-related disability costs were $2,482 lower than baseline (40% and 51% decrease, respectively; both p < .01). Results were similar for indication-specific analyses.

Limitations: Limited generalizability to patients who are unemployed, uninsured, or have public insurance.

Conclusions: Rates of disability events, days, and mean costs were significantly lower after versus before cariprazine initiation. These results can help contextualize cariprazine's role in managing disability for these patients.

Background: Biosimilars to originator bevacizumab (Avastin), such as bevacizumab-bvzr (Zirabev), can deliver substantial savings and/or expanded access to biologic therapy for patients with metastatic colorectal (mCRC) and non-squamous non-small cell lung cancer (mNSCLC). The objective of this study is to explore the cost-efficiency and budget-neutral expanded access of bevacizumab-bvzr in mCRC and mNSCLC in Medicare.

Methods: We developed a Medicare payer perspective simulation model of patients treated for mCRC and mNSCLC to estimate cost-savings from converting bevacizumab (originator) to bevacizumab-bvzr or alternative biosimilars such as bevacizumab-awwb, -maly, and -abcd. The target patient population receiving annual first-line systemic therapy was calculated using Medicare enrollment data, SEER cancer incidence rates in patients age ≥65, and an assumption that 39.3% and 77.2% of new diagnoses receive systemic therapy for mCRC and mNSCLC respectively based on recent evidence. 76.0% of systemically treated mCRC patients and 11.4% of incident mNSCLC patients were expected to be treated with bevacizumab-based regimens based on recent evidence. Costs were derived from the 2024 Average Sales Price (ASP). Results include per-patient per-month (PPPM) cost savings (vs. originator), total monthly savings in the cohort, and number needed to convert (NNC) to biosimilar to fund the treatment of an additional 100 patients.

Results: PPPM savings from conversion to bevacizumab-bvzr were $4,205 in mCRC and $8,410 in mNSCLC. In 100% conversion scenarios, full cohort monthly savings were $27,664,432 in mCRC (n = 6,579) and $32,319,323 in mNSCLC (n = 3,843), respectively. At 100% conversion, monthly savings from biosimilar conversion could fund up to 13,887 additional mCRC patient-months of treatment with bevacizumab-bvzr + FOLFOX, and up to 8,959 additional mNSCLC patient-months of treatment with bevacizumab-bvzr + paclitaxel + carboplatin. In mCRC and mNSCLC the biosimilar NNC from the originator was 47 and 43, respectively. The biosimilar NNC from other biosimilars ranged from 60-4,564 and 55-4,422 for mCRC and NSCLC, respectively.

Conclusion: In the first cost-efficiency and expanded access study of biosimilar bevacizumab in mCRC and mNSCLC, we find that bevacizumab-bvzr-based regimens can result in substantial cost savings relative to originator-based first line treatment in Medicare. These cost savings could be reinvested to treat a substantial number of additional patients with mCRC or mNSCLC, or fund other costs of care in Medicare, on a budget-neutral basis.

Background and objectives: Chronic Kidney Disease-associated Pruritus (CKD-aP) is a disabling condition that affects around 60% of patients with end-stage kidney disease undergoing dialysis. Current off-label treatment options are neither effective nor appropriate for all dialysis patients, leaving a clear unmet need. This study aimed to evaluate the cost-effectiveness of difelikefalin - the only drug approved in Europe for the treatment of moderate to severe CKD-aP adult patients in haemodialysis - compared to the best supportive care (BSC) from the Spanish NHS perspective.

Methods: A Markov model was developed with seven health states: five health states representing levels of pruritus intensity over time (No, Mild, Moderate, Severe and Very severe CKD-aP), kidney transplant and death as the absorbing state. The model included patients with moderate to severe CKD-aP at baseline, in line with difelikefalin approved indication and clinical trials. Local costs, utilities, mortality rates and kidney transplant probabilities were obtained from published literature. Costs and quality-adjusted life-years (QALYs) were discounted at a 3% annual rate with a lifetime horizon (36 years).

Results: Difelikefalin was associated with an increased in QALYs (+0.49) and higher costs (+12,300€) compared to the BSC over a lifetime horizon. At a provisional cost estimate of 270.6€per 28-days for difelikefalin (based on a tentative list price for Spain), the incremental cost-utility ratio was 25,000€/QALY. The sensitivity analysis (DSA) confirmed the robustness of the results. The probabilistic sensitivity analysis (PSA), undertaken with 1000 iterations, indicated a 50% and 83% probability of difelikefalin being cost-effective at a willingness-to-pay (WTP) thresholds of 25,000 €/QALY and 30,000 €/QALY, respectively.

Conclusions: Difelikefalin could be a cost-effective option compared to BSC for the management of CKD-aP in adult haemodialysis patients within the Spanish NHS setting. Considering the unmet needs, these results support the convenience of incorporating difelikefalin in routine clinical practice in Spain.

Objective/aim: In 2009, dronedarone was approved by the United States Food and Drug Administration based on results from the ATHENA trial (NCT00174785), which showed significant reduction of cardiovascular (CV) hospitalization and death in patients with atrial fibrillation (AF) randomized to dronedarone versus placebo. In 2020, a retrospective study by Goehring et al. showed CV hospitalizations and deaths were lower in clinical practice following initiation of dronedarone compared to other antiarrhythmic drugs (AADs) in patients with AF and atrial flutter. However, the economic impact associated with dronedarone use has not been fully assessed. The objective of this study was to estimate the cost associated with CV outcomes reported by Goehring et al. (2020).

Methods: National average Medicare payments in the Centers for Medicare and Medicaid Services (CMS) database (www.data.CMS.gov) were used to assign cost estimates to CV outcomes evaluated in Goehring et al. (2020) by diagnosis-related grouping. When costs were unavailable in the CMS database, a literature search was performed to identify publications reporting hospitalization costs.

Results: The weighted average cost for CV hospitalization was calculated to be $20,508. When multiplied by the event rate reported in Goehring et al. (2020), cost per person year for CV hospitalization was 14% lower with dronedarone versus other AADs ($3,679 vs $4,272, respectively). For hospitalizations due to heart failure, cost was 31% lower with dronedarone compared with other AADs ($324 vs $472, respectively).

Limitations: Costs have been calculated based on national averages reported by CMS (Medicare perspective) and are estimates. Regional differences may be present.

Conclusions: Patients with AF taking dronedarone had lower costs associated with CV hospitalization compared with patients taking other AADs.