Journal of Medical Economics最新文献

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.

Objective: Chronic kidney disease (CKD) is the leading cause of kidney failure, end-stage kidney disease (ESKD), and cardiovascular (CV) events in patients with type 2 diabetes (T2D). The FIDELIO-DKD trial demonstrated that finerenone lowered the risk of renal and CV events in patients with CKD and T2D, regardless of cardiovascular disease history. This study evaluated the cost-effectiveness of finerenone added to background treatment (finerenone + BT) versus background treatment (BT) alone in patients with CKD and T2D from the perspective of the National Health Service in England and Wales.

Methods: A lifetime Markov model assessed the indicated usage of finerenone for the treatment of stage 3 or 4 CKD with albuminuria associated with T2D in adults, as per the relevant marketing authorization. The model structure considered kidney disease progression and CV risk, with health states encompassing patients' kidney disease stage and CV event profiles, using patient-level data from the FIDELIO-DKD trial. Model outcomes were life years, quality-adjusted life years (QALYs), per-patient costs, incremental costs, and incremental cost-effectiveness ratio (ICER). Sensitivity and scenario analysis were performed, including an analysis exploring the impact of real-world data which suggests more frequent sodium-glucose co-transporter-2 (SGLT2) inhibitor use in the United Kingdom since FIDELIO-DKD.

Results: Patients receiving finerenone experienced kidney and CV benefits, including reduced rates of nonfatal CV events and CV deaths, translating to improvements in survival and quality-adjusted life years (QALYs) of 6.11 and 5.97 per patient for finerenone + BT versus BT, respectively. Total discounted per-patient costs were £48,940 for finerenone + BT and £47,716 for BT alone, resulting in an incremental cost-effectiveness ratio of £8,808 per QALY gained for finerenone + BT versus BT.

Conclusion: Sensitivity and scenario analyses, including more frequent SGLT2 inhibitor use consistent with real-world data, indicate a robust ICER that remains within the bounds of what is typically considered cost-effective.

Aims: Nirmatrelvir/ritonavir (NMV/r) is an orally administered antiviral indicated for the outpatient treatment of adult patients with mild-to-moderate COVID-19 at high risk for disease progression to severe illness. We estimated the cost-effectiveness of NMV/r versus best supportive care for 54 patient cohorts, specified according to age, vaccination status and comorbidity burden.

Materials and methods: A previously published and validated cost-effectiveness model was utilized and adapted to the Swedish setting. The model used a short-term decision-tree (1 year) followed by a lifetime 2-state Markov model. The short-term decision-tree captured costs and outcomes associated with the primary infection. Post-acute COVID-19 syndrome was only considered in terms of quality-of-life decrements for one year. Baseline hospitalization and mortality risks were taken from a Swedish, nationwide, uniquely granular, Omicron-era, real-world study. NMV/r effectiveness were taken from an Omicron-era US real-world study. Remaining inputs were informed by previous COVID-19 studies and publicly available Swedish sources.

Results: The incremental cost-effectiveness ratios (ICERs) showed a large variation ranging from almost nine million SEK for some of the youngest cohorts to being dominant (i.e. cost-saving with higher gains in quality-of-life vs standard of care) for twelve elderly cohorts. In general, higher age in combination with non-recent (>180 days) or no vaccination led to lower ICERs. Specifically, NMV/r was cost-effective for all but one patient cohorts at least 70 years old, and for most patient cohorts 60-69 years old.

Limitations: As the COVID-19 landscape changes, symptom burden and baseline risks constantly change. Thus, the cost-effectiveness of NMV/r will change with time. However, the future risks could be related to the risks in the current study, and thus remain useful for decision makers.

Conclusions: This study shows that NMV/r is a cost-effective or even cost-saving treatment option for many patient cohorts, including most elderly and not-recently vaccinated patients with at least some comorbidity burden.

Aims: Haemophilia is a rare genetic disease that hinders blood clotting. We aimed to review model-based cost-effectiveness analyses (CEAs) of haemophilia treatments, describe the sources of clinical evidence used by these CEAs, summarize the reported cost-effectiveness of different treatment strategies, and assess the quality and risk of bias.

Methods: We conducted a systematic literature review of model-based CEAs of haemophilia treatments by searching databases, the Tufts Medical Center CEA registry, and grey literature. We summarized and qualitatively synthesized the approaches and results of the included CEAs, without a meta-analysis due the diversity of the studies.

Results: 32 eligible studies were performed in 12 countries and reported 53 pairwise comparisons. Most studies analysed patients with haemophilia A rather than haemophilia B. Comparisons of prophylactic versus on-demand treatment indicated that prophylaxis may not be cost-effective, but there was no clear consensus. Emicizumab was generally cost-effective compared with clotting factor treatments and was always dominant for patients with inhibitors. Immune tolerance induction following a Malmö protocol was found to be cost-effective compared to bypassing agents, while there was no consensus for the other protocols. Gene therapies as well as treatment with extended half-life coagulation factors were always cost-effective over their comparators. Studies were highly heterogenous regarding their time horizons, model structures, the inclusion of bleeding-related mortality and quality-of-life impacts. This heterogeneity limited the comparability of the studies. 19 of the 32 included studies received industry funding, which may have biased their results.

Limitations: It was not possible to perform a quantitative synthesis of the results due to the heterogeneity of the underlying studies.

Conclusion: Differences in results between previous CEAs may have been driven by heterogeneity in modelling approaches, clinical input data, and potential funding biases. A more consistent evidence base and modelling approach would enhance the comparability between CEAs.

Aims: Iron deficiency anemia (IDA) is among the most common extraintestinal sequelae of inflammatory bowel disease (IBD). Intravenous iron is often the preferred treatment in patients with active inflammation with or without active bleeding, iron malabsorption, or intolerance to oral iron. The aim of the present study was to evaluate the cost-utility of ferric derisomaltose (FDI) versus ferric carboyxymaltose (FCM) in patients with IBD and IDA in Norway.

Materials and methods: A published patient-level simulation model was used to evaluate the cost-utility of FDI versus FCM in patients with IBD and IDA from a Norwegian national payer perspective. Iron need was modelled based on bivariate distributions of hemoglobin and bodyweight combined with simplified tables of iron need from the FDI and FCM summaries of product characteristics. Patient characteristics and disease-related quality of life data were obtained from the PHOSPHARE-IBD trial. Cost-utility was evaluated in Norwegian Kroner (NOK) over a five-year time horizon.

Results: Patients required 1.64 fewer infusions of FDI than FCM over five years (5.62 versus 7.26), corresponding to 0.41 fewer infusions per treatment course. The reduction in the number of infusions resulted in cost savings of NOK 5,236 (NOK 35,830 with FDI versus NOK 41,066 with FCM). The need for phosphate testing in patients treated with FCM resulted in further cost savings with FDI (no costs with FDI versus NOK 4,470 with FCM). Total cost savings with FDI were therefore NOK 9,707. FDI also increased quality-adjusted life expectancy by 0.071 quality-adjusted life years (QALYs) driven by reduced incidence of hypophosphatemia and fewer interactions with the healthcare system.

Conclusions: FDI resulted in cost savings and improved quality-adjusted life expectancy versus FCM in patients with IDA and IBD in Norway. FDI therefore represents the economically preferable iron formulation in Norwegian patients with IBD and IDA in whom it is indicated.

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.

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.

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.

Aims: Spinal muscular atrophy (SMA) is a rare genetic disorder characterized by progressive muscle weakness, atrophy, respiratory failure, and in severe cases, infantile death. Early detection and treatment before symptom onset may substantially improve outcomes, allowing patients to achieve age-appropriate motor milestones and longer survival. We assessed the cost-utility of newborn screening (NBS) for SMA in Japan.

Materials and methods: A cost-utility model (decision tree and Markov model) compared lifetime health effects and costs between "NBS" for SMA (presymptomatic treatment) or "no NBS" (treatment initiated at symptom onset). Model inputs were sourced from literature, local data, and expert opinion. Sensitivity and scenario analyses were conducted to assess model robustness and data validity.

Results: Based on the 1:10,000 SMA incidence, it was estimated that 43 newborns/year would have SMA, and a total of 39 patients with SMA would initiate presymptomatic treatment after NBS. An estimated 736 quality-adjusted life-years were gained per annual birth cohort with NBS. NBS for SMA was dominant compared with no NBS (i.e. less costly and more effective), with ¥8,856,960,096 reduced total costs with NBS versus no NBS (base-case). Sensitivity and scenario analyses supported cost effectiveness of NBS for SMA versus no NBS. A greater percentage of patients was estimated to enjoy longer survival and be without permanent assisted ventilation with NBS versus no NBS.

Limitations: Real-world observations may differ from single-arm clinical trial outcomes. It was assumed that patients with SMA identified via NBS were asymptomatic and would receive treatment prior to symptoms. Best supportive care was not considered, and Japan-specific variations in gene replacement therapy protocol were not fully reflected.

Conclusion: NBS for SMA allows for early identification of patients with SMA and treatment initiation before symptom onset, improving health outcomes and reducing total costs than without NBS.

Background: Psoriasis is a chronic, systemic, inflammatory skin disease, with increasing prevalence; however, few studies have reported real-world prescription patterns and healthcare burden.

Objectives: This retrospective, observational cohort study used statutory health insurance claims data (January 2014-December 2019) to estimate prevalence/incidence of moderate-to-severe psoriasis in Germany. Patient characteristics, treatment patterns/compliance, and healthcare resource utilization (HCRU)/costs were evaluated, focusing on apremilast and anti-interleukin (IL), and anti-tumor necrosis factor (TNF) biologics.

Methods: The epidemiology population included adults with psoriasis; 1-year prevalence/incidence rates were extrapolated to the statutory health insurance population. The HCRU/costs population included adults with psoriasis and a first prescription for a drug of interest (index date). Baseline periods were 12 or 48 months before the index date, with 12‑month follow-up.

Results: In 2019, the estimated psoriasis prevalence/incidence was 2,672.9 per 100,000 individuals/508.7 per 100,000 person-years. Of 2,809 patients in the HCRU/costs population, 3.6% (n = 101) received index drug apremilast, 10.2% (n = 287) anti-IL, 6.8% (n = 191) anti-TNF, and 79.4% (n = 2,230) traditional/other systemic therapy. Patients initiating apremilast were older and were more often biologic-naïve than those initiating anti-IL/TNF biologics. Twelve months after treatment initiation, drug adherence (medication possession rate >80%) and persistence (<60 days between prescriptions/no switch) were lower for apremilast vs. anti-IL and anti-TNF groups (24.8% vs. 59.6% and 53.9%; 36.6% vs. 66.9% and 57.6%, respectively). During a 12-month baseline period, psoriasis-related hospitalization was lower for apremilast vs. anti-IL and anti-TNF groups (4.95% vs. 15.68% and 14.14%) and higher during 12 months' follow-up (5.94% vs. 2.44% and 3.14%). Adjusted index drug costs during follow-up were €4,105, €3,498, and €13,777 higher for adalimumab, other anti-TNF and anti-IL biologics vs. apremilast, respectively, and the main driver for lower overall apremilast costs.

Conclusion: Given variation in treatment adherence/persistence, HCRU, and costs between apremilast and biologics, these findings could be key considerations during treatment selection.