Journal of Medical Economics最新文献

Background: Patients with refractory multiple myeloma (MM) often progress through lines of therapy (LOTs) comprising multiple drug classes, which may impose severe economic burden. In this retrospective US claims database study, we examined healthcare resource utilization (HCRU) and costs of patients with MM who received 1 to 3 prior LOTs.

Patients and methods: Adults with MM from the IBM Truven MarketScan Claims Database (1 January 2011-22 April 2023) were required to be continuously enrolled in a medical benefit/pharmacy plan for ≥12 months before initial MM diagnosis date and to have received 1 to 3 LOTs (including receiving ≥1 proteasome inhibitor and immunomodulatory drug and receiving and discontinuing lenalidomide) after diagnosis date. Index dates (start of subsequent treatment after fulfilling inclusion criteria) occurred after 1 January 2018, to capture contemporary cost estimates. Primary outcomes included all-cause and MM-related healthcare costs and HCRU after index date.

Results: The primary analysis included 338 patients with MM without post-index stem cell transplant (SCT), with a mean age of 61.1 years (55.3% male). During an average follow-up of 11.5 months, total all-cause healthcare costs averaged US $41,614 per patient per month. MM-related healthcare costs ($39,699) contributed 95% to total all-cause costs. Most MM-related monthly costs were attributed to drug/infusion costs (71%; $28,144). Sensitivity analyses that included patients with post-index SCT (N = 520) yielded similar results.

Conclusions: Patients with MM with 1 to 3 prior LOTs experienced high economic burden largely attributable to MM-related treatment, highlighting the need for more cost-effective therapies.

Objective: The LungFlag risk prediction model uses individualized clinical variables to identify individuals at high-risk of non-small cell lung cancer (NSCLC) for screening with low-dose computed tomography (LDCT). This study evaluates the cost-effectiveness of LungFlag implementation in the Spanish setting for the identification of individuals at high-risk of NSCLC.

Methods: A model combining a decision-tree with a Markov model was adapted to the Spanish setting to calculate health outcomes and costs over a lifetime horizon, comparing two hypothetical scenarios: screening with LungFlag versus non-screening, and screening with LungFlag versus screening the entire population meeting 2013 US Preventive Services Task Force (USPSTF) criteria. Model inputs were obtained from the literature and the clinical practice of a multidisciplinary expert panel. Only direct costs (€of 2023), obtained from local sources, were considered. Deterministic and probabilistic sensitivity analyses were performed to assess the robustness of our results.

Results: A cohort of 3,835,128 individuals meeting 2013 USPSTF criteria would require 2,147,672 LDCTs scans. However, using LungFlag would only require 232,120 LDCTs scans. Cost-effectiveness results showed that LungFlag was dominant versus non-screening scenario, and outperformed the scenario where the entire population were screened since the observed loss of effectiveness (-224,031 life years [LYs] and -97,612 quality-adjusted life years [QALYs]) was largely offset by the significant cost savings provided (€7,053 million). The resulting incremental cost-effectiveness ratio (ICER) for this strategy of screening the whole population versus using LungFlag was €72,000/QALY, showing that LungFlag is cost-effective. Various were described, such as the source of the efficacy or adherence rates, and other limitations inherent to cost-effectiveness analyses.

Conclusions: Using LungFlag for the selection of high-risk individuals for NSCLC screening in Spain would be a cost-effective strategy over screening the entire population meeting USPSTF 2013 criteria and is dominant over non-screening.

Introduction: A cell harvesting device for preparing non-cultured autologous skin cell suspension (ASCS) at the point-of-care is FDA-approved for repigmentation of stable depigmented vitiligo lesions in patients 18 years and older. The pivotal RSVP trial showed ≥80% repigmentation at Week-24 in 36% of lesions treated with laser ablation, ASCS, and narrowband ultraviolet B phototherapy compared to 0% with phototherapy alone (p = 0.012). The objective of this analysis was to evaluate the potential economic impact of laser ablation plus ASCS with phototherapy versus phototherapy alone for repigmentation of stable vitiligo lesions from a US payer perspective.

Methods: A 5-year decision-tree model was developed reflecting clinical pathways of adults with stable vitiligo lesions who had an inadequate response to prior topicals and phototherapy. Patients entering the model were treated with ASCS plus phototherapy or phototherapy alone and assessed for treatment response at Weeks-24 and 52 based on the RSVP trial's effectiveness endpoints. Durable response for Year-2 onwards was proxied by melanocyte-keratinocyte transplantation data. Model outcomes included per-patient total and incremental healthcare costs, treatment costs and total costs, cost per-patient per-month (PPPM), and cost per-patient per-year (PPPY). One-way sensitivity analyses assessed model result robustness.

Results: The cumulative total per-patient cost for ASCS plus phototherapy increased from $28,177 to $92,779 between Year-1 and Year-5. Phototherapy alone increased from $21,146 to $101,518 over the same period. Compared to phototherapy alone, ASCS plus phototherapy incurred $7,030 more total per-patient cumulative costs in Year-1 and $8,738 less by Year-5 (-$146 PMPM; -$1,748 PPPY). Breakeven occurred between Years 2-3. Results were most sensitive to changes in ASCS response at Weeks-24 and 52 and healthcare costs.

Conclusion: Among adults with stable vitiligo with prior inadequate response to topicals or phototherapy, ASCS treatment may lead to lower all-cause direct medical costs over 5 years compared to phototherapy alone.

Aim: We assessed the relationship between hospital septal reduction therapy (SRT) procedural volume and clinical outcomes, healthcare resource utilization, and hospital costs.

Methods: This cross-sectional study used 2012-2022 US hospital data from the PINC AI Healthcare Database for adults with hypertrophic cardiomyopathy (HCM) undergoing alcohol septal ablation (ASA) or septal myectomy (SM; with or without mitral valve repair or replacement [MVRR]). We categorized hospital procedural volume into tertiles according to the numbers of procedures performed and made pairwise comparisons of patient characteristics, clinical events, healthcare utilization, and hospital costs between tertiles. We conducted multivariable analyses (adjusted for patient, clinical, and hospital characteristics) for index hospitalization length of stay, cost, and 30-day readmission rates.

Results: Overall, 3,068 patients with HCM (across 315 hospitals) underwent SRT (ASA: 1,400; SM: 1,668). Index visit in-hospital mortality was 1.1-1.5% among individuals undergoing ASA, 3.2-7.4% for SM with MVRR, and 2.8-3.8% for SM without MVRR. There were no significant differences in in-hospital mortality or stroke/transient ischemic attack at index visits between the hospital procedural volume tertiles for ASA or SM. Adjusted hospital length of stay, costs, and readmission rates were significantly greater in low-volume than high-volume hospitals for ASA (p < 0.001). Similar trends were reported for SM for length of stay and costs (p < 0.001).

Limitations: This study relied upon accurate and complete reporting of diagnoses and procedures by hospitals. Patients were not randomly assigned, potentially leading to selection bias. Only in-hospital costs were evaluated. Follow-up events were only captured if they occurred in the same healthcare facility.

Conclusions: Resource utilization and in-hospital costs for patients undergoing SRT are lower in high procedural volume hospitals than low procedural volume hospitals. SRT procedure volume remains low even in hospitals with the highest relative procedural volumes, highlighting a need for globally accessible therapies that improve outcomes.

The rapid evolution of large language models (LLMs) and machine learning (ML) presents both significant opportunities and challenges for market access processes. These sophisticated AI systems, built on transformer architectures and extensive datasets, offer potential to forecast claims and decisions of health technology assessment (HTA) agencies and streamline processes, such as systematic literature reviews and HTA submissions. Furthermore, the analysis of real-world data-also for deriving causal relationships-is being discussed intensively. Despite notable advancements, their adoption in key PRMA processes is still limited at present, with only a small fraction of submissions to HTA bodies incorporating AI. Key barriers include stringent transparency requirements, the necessity of explainability and human oversight in data analyses, and the highly sensitive nature of text drafting-especially in cases where reimbursement decisions or pricing negotiations balance on a knife's edge. These requirements are often not met due to the immaturity of many AI applications, which still lack the necessary precision, reliability, and contextual understanding. Moreover, AI-generated evidence has yet to prove its validity before it can supplement or replace traditional study designs, such as randomized controlled trials (RCTs), which are critical for HTA decisions. Additionally, the environmental and financial costs of training LLMs require careful assessment. This paper explores various current AI applications, their limitations, and future prospects in key PRMA processes from a German perspective while also considering the broader implications of the EU Health Technology Assessment Regulation (HTAR). It concludes that while AI hold transformative potential, its integration into workflows must be approached cautiously, with incremental adoption, and close collaboration between industry, HTA agencies, and academia. Demonstrating robust, unbiased comparative evidence-showcasing superior performance and cost savings over traditional methods-could accelerate the adoption process.

Aim: The aim of this study is to elucidate the prevalence, incidence, patient characteristics, and recent treatment trends of valvular heart disease (VHD) in Japan using a comprehensive claims database.

Methods: We conducted a cross-sectional study using the national database of health insurance claims in Japan (April 2009-June 2021), which contains data from the entire Japanese population, regardless of the type of medical facility. Descriptive analyses were conducted to examine the prevalence, incidence, and patient characteristics of each valve disease in 2020 based on diagnoses, and the treatment trends from 2009 to 2021.

Results: We identified 28,366,924 patients with VHD over the entire data period, and 2,473,070 patients in 2020, including 711,876 newly diagnosed. The prevalence and annual incidence in the entire Japanese population were 1.96% (1.88% in men and 2.04% in women) and 0.56% (0.53 and 0.60%), respectively, and increased with age in adults. Among the 8 types of VHD in combination with a disordered valve (aortic, mitral, tricuspid, or pulmonic) and type of valve disease (stenosis or regurgitation), mitral regurgitation had the highest prevalence followed by aortic regurgitation and tricuspid regurgitation. Heart failure was diagnosed in ≥50% of patients with aortic, mitral, or tricuspid disease, with the highest rate in mitral stenosis. The number of open-heart surgeries remained constant, while the number of transcatheter surgeries increased over time, particularly between 2016 and 2021. Aortic stenosis prevalence in transcatheter surgeries rose to ≥60% in 2014 and ≥80% in 2016.

Limitations: Diagnoses of VHD and comorbidity were based on claims data, so diagnostic criteria and disease severity are unknown, and misclassification of VHD types might have occurred. Incidence rates were based on the initial VHD diagnosis only, excluding any subsequent diagnoses of different VHD type. Conclusions: We presented basic information, which may provide an understanding of the clinical status of VHD in Japan.

Background: High-dose (HD) influenza vaccine, which has demonstrated superior efficacy and acceptable safety compared to standard-dose (SD), has market authorization in many countries. This study evaluated the public-health impact and cost-effectiveness of HD versus SD in Japanese older-adults (OAs) from healthcare payer-perspective.

Methods: Decision-tree model was employed assessing health outcomes for each vaccination strategy, simulating influenza cases, outpatient/emergency department (ED) visits, hospitalizations, and mortality, over one-year time-horizon. Incremental cost-effectiveness ratios (ICER) were assessed at Japanese willingness-to-pay (WTP) threshold. Base-case analysis considered influenza vaccines effective against influenza hospitalizations only, whereas complementary analyses reflected their efficacies against Pneumonia and Influenza (P&I), respiratory and cardiorespiratory hospitalizations possibly related to influenza among individuals ≥65 years. Scenario analysis extended target population to at-risk individuals aged 60-64 years. Uncertainty was assessed using sensitivity analyses.

Results: In base-case, switching from SD to HD prevented 174,863 influenza cases, 121,084 outpatient and 614 ED visits, annually. Further, 5,777 influenza hospitalizations, and 2,406 deaths related to influenza were avoided with HD vaccine. The HD vaccine was found to be a cost-effective strategy at WTP threshold of ¥5,000,000/Quality-Adjusted-Life-Years (QALY) with ICER of ¥4,876,512/(QALY). Sensitivity analyses confirmed the robustness of these findings. Complementary analyses showed notably improved outcomes, in terms of public-health, economic impact, and ICERs, when considering efficacy of influenza vaccines against P&I, respiratory, and cardiorespiratory hospitalizations possibly related to influenza.

Conclusion: These results indicate that HD vaccine has a high economic value in Japan compared to SD. Implementing HD vaccine could effectively alleviate the burden on healthcare facilities for Japanese OAs.

Aims: CheckMate-77T demonstrated the clinical benefit of perioperative nivolumab plus neoadjuvant platinum-doublet chemotherapy (periNivo + neoCT). This study assessed the cost-effectiveness of periNivo + neoCT as treatment for non-metastatic (Stage IIA-IIIB), resectable non-small cell lung cancer (NSCLC) vs. relevant comparators in the US.

Materials and methods: Following the natural history of non-metastatic NSCLC, a four-state Markov model was developed. Modeled health states were event-free survival, locoregional recurrence, distant metastasis, and death. CheckMate-77T informed time to progression estimates for periNivo + neoCT and neoCT; mortality estimates leveraged longer-term follow-up available from CheckMate-816. Indirect treatment comparison informed efficacy of comparator treatments not considered in CheckMate-77T. Comparators were neoadjuvant treatment strategies (neoadjuvant nivolumab + chemotherapy [neoNivo + CT], neoadjuvant chemotherapy [neoCT], and neoadjuvant chemoradiotherapy [neoCRT]), adjuvant chemotherapy (adjCT), and perioperative immuno-therapy (IO) strategies (perioperative durvalumab + neoadjuvant chemotherapy [periDurva + neoCT] and perioperative pembrolizumab + neoadjuvant chemotherapy [periPembro + neoCT]). Cost inputs were obtained from published literature and standard US sources and expressed in 2024 USD. The base-case analysis adopted the perspective of a commercial payer with a lifetime time horizon and discounted cost and health outcomes by 3% annually.

Results: Model results showed that periNivo + neoCT is more effective and costly than comparators. Deterministic incremental cost-effectiveness ratios were $84,921, $153,557, $77,976, $60,826, $74,252, $32,069, and $21,974 vs. neoCT, neoNivo + CT, neoCRT, adjCT, surgery, periPembro + neoCT, and periDurva + neoCT, respectively. In probabilistic sensitivity analysis, periNivo + neoCT resulted in an ICER below $150,000/QALY in 93.3%, 58.2%, 82.4%, 95.1%, 98.3%, 69.9%, and 82.1% of iterations vs. neoCT, neoNivo + CT, neoCRT, adjCT, surgery only, periPembro + neoCT, and periDurva + neoCT, respectively.

Limitations: Uncertainty in the survival extrapolations reflected the limited body of evidence informing the indirect treatment comparison. ICERs vs. perioperative IO treatment strategies were sensitive to small changes in predicted costs and QALYs, given low incremental base case costs and QALYs.

Conclusion: PeriNivo + neoCT is a cost-effective treatment option for patients with resectable, non-metastatic NSCLC.

Background: Underdiagnosis and the absence of a condition-specific diagnostic code have made the economic burden of adult growth hormone deficiency (AGHD) difficult to capture. This study measured all-cause and disease-specific healthcare utilization and costs of AGHD among individuals stratified by diagnosis status and receipt of growth hormone (GH) treatment.

Methods: Adults meeting ≥1 of the following criteria (1/1/2017-12/31/2021): diagnosis of hypopituitarism or related condition, ≥3 pituitary hormone deficiencies, ≥3 pituitary hormone treatments, or ≥1 GH prescription were identified in the Veradigm Network EHR linked to claims. Individuals were stratified by GH level on or before the earliest qualifying event: confirmed (GH < 3 ng/mL), at-risk (no test result), ruled-out (GH ≥ 3 ng/mL). Confirmed and at-risk individuals were segmented by GH treatment. An age and gender-matched control cohort without AGHD was identified. Healthcare utilization and costs were measured in the 12-month post-index period. Multivariable modeling compared all-cause and AGHD-related healthcare costs, excluding cost of GH, among diagnosis- or treatment status-stratified cohorts while adjusting for baseline characteristics.

Results: Among 54,310 individuals at risk for AGHD and 268 with confirmed AGHD, 3.1% and 9.7% received GH treatment, respectively. Study subjects were, on average, 50 years old and majority female. Adjusted all-cause healthcare costs were higher among at-risk individuals (cost ratio [95% confidence interval]: 2.37 [2.26-2.49]) and among confirmed individuals (2.11 [1.52-3.43]) compared to controls. Adjusted annual AGHD-related costs were lower among confirmed individuals compared to at-risk individuals (0.62 [0.52-0.76]) and among treated individuals compared to untreated individuals (0.55 [0.47-0.64]) for those initiating GH therapy.

Conclusions: All-cause healthcare costs were higher among individuals with confirmed AGHD or at risk for AGHD than among adults without GHD. After excluding the cost of GH therapy, lower adjusted AGHD-related costs were associated with both a confirmed AGHD diagnosis and receipt of GH treatment.