Journal of Medical Economics最新文献

{"title":"The indirect costs of five cancers in Egypt: years of life lost and productivity costs.","authors":"Karim Abdel Wahab, Ahmed Hassan, Ahmed Morsi, Sneha Amritlal, Anne Meiwald, Robert Hughes, Aimée Fox, Goran Bencina, Bernadette Pöllinger","doi":"10.1080/13696998.2024.2435750","DOIUrl":"10.1080/13696998.2024.2435750","url":null,"abstract":"<p><strong>Background: </strong>In Egypt, there were 150,578 new cancer cases and 95,275 cancer deaths in 2022, indicating a substantial burden on patients and the healthcare system. The analysis aims to support decision-making related to investments in cancer prevention and new treatments, by highlighting the economic burden associated with five types of cancer.</p><p><strong>Methods: </strong>The human capital approach was used to estimate productivity losses from premature mortality due to liver, lung, breast, bladder, and cervical cancer in Egypt in 2019 by calculating years of life lost (YLL), years of productive life lost (YPLL), and present value of future lost productivity (PVFLP). Mortality data were sourced from the World Health Organization (WHO), while life expectancy, retirement age, gross domestic product (GDP) per capita, and labor force participation rates were obtained from the World Bank. Income data, such as annual earnings and minimum wage were sourced from the Wage Indicator database. Deterministic sensitivity analysis (DSA) assessed the sensitivity of results to input variations.</p><p><strong>Results: </strong>In 2019, Egypt had a total of 45,114 deaths, from liver, lung, breast, cervical, and bladder cancers, resulting in a productivity loss of $430,086,636. Liver cancer led to the most male deaths (17,745) and breast cancer to the most female deaths (6,754), with PVFLP of $232,663,468 and $130,745,592, respectively. The five cancers resulted in 551,336 YLL and 235,415 YPLL in Egypt. The total PVFLP was estimated at $217,224,178 for females and $212,862,458 for males, with a total PVFLP/death of $9,533. The DSA showed that the PVFLP was most sensitive to changes in the retirement age.</p><p><strong>Conclusion: </strong>In conclusion, there is a substantial economic burden relating to premature cancer mortality in Egypt, highlighting that policies and treatment advances to decrease cancer are working, however, there is need for continuous prioritization of awareness programs, cancer screening and treatment advancements.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":" ","pages":"36-43"},"PeriodicalIF":2.9,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142769614","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cost-utility analysis of empagliflozin for heart failure in the Philippines.","authors":"Precious Juzenda Montilla, Camilo Oliver Aquino, Elaine Cunanan, Patrick James Encarnacion, Helen Ong-Garcia, Elmer Jasper Llanes, Diana Dalisay Orolfo, Chito Permejo, Mary Joy Taneo, Anthony Russell Villanueva, Dante Salvador, John Añonuevo","doi":"10.1080/13696998.2024.2447180","DOIUrl":"10.1080/13696998.2024.2447180","url":null,"abstract":"<p><strong>Aims: </strong>Empagliflozin confers cardioprotective benefits among patients with heart failure, across the range of ejection fraction (EF), regardless of type 2 diabetes status. The long-term cost-effectiveness of empagliflozin for the treatment of heart failure (HF) in the Philippines remains unclear. This study aims to determine the economic benefit of adding empagliflozin to the standard of care (SoC) vs the SoC alone for HF in the Philippines.</p><p><strong>Methods: </strong>Using a Markov model, we predicted lifetime costs and clinical outcomes associated with treating HF in the Philippine setting. We used estimates of treatment efficacy, event probabilities, and derivations of utilities from the EMPEROR trials. Costs were derived from hospital tariffs and expert consensus. Separate analyses were performed for patients with left ventricular EF > 40%, categorized under mid-range ejection fraction or preserved ejection fraction (HFmrEF/HFpEF), and patients with left EF ≤ 40%, categorized under HF with reduced ejection fraction (HFrEF).</p><p><strong>Results: </strong>Our model predicted an average of 0.09 quality-adjusted life year (QALY) gains among HFmrEF/HFpEF patients and HFrEF patients when empagliflozin was compared to SoC. The addition of empagliflozin in the treatment results in a discounted incremental lifetime cost of PHP 62,692 (USD 1,129.99) and PHP 17,215 (USD 308.67) for HFmrEF/HFpEF and HFrEF, respectively. The incremental cost-effectiveness ratio (ICER) of empagliflozin is PHP 198,270 (USD 3,570.72)/QALY and PHP 742,604 (USD 13,385.08)/QALY for HFrEF and HFmrEF/HFpEF, respectively.</p><p><strong>Limitations: </strong>This study employed parameters derived from short-term clinical trial data, alongside metrics representative of Asian populations, which are not specific to the Philippine cohort.</p><p><strong>Conclusions: </strong>Adding empagliflozin to the SoC in comparison to the SoC is associated with improved clinical outcomes and quality-of-life, at additional costs for both HFrEF and HFmrEF/HFpEF.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":" ","pages":"157-167"},"PeriodicalIF":2.9,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142915033","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Exclusive human milk diet: a challenging innovation in neonatal care.","authors":"Georgina N Marchiori, Elio A Soria","doi":"10.1080/13696998.2024.2445431","DOIUrl":"10.1080/13696998.2024.2445431","url":null,"abstract":"","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":" ","pages":"124-126"},"PeriodicalIF":2.9,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142864563","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Real-world healthcare resource utilization of Alzheimer's disease in the early and advanced stages: a retrospective cohort study.","authors":"Elnara Fazio-Eynullayeva, Marianne Cunnington, Paul Mystkowski, Lei Lv, Abdalla Aly, Christopher W Yee, Raj Desai, Chia-Lun Liu, Mei Sheng Duh, Soeren Mattke","doi":"10.1080/13696998.2024.2442240","DOIUrl":"10.1080/13696998.2024.2442240","url":null,"abstract":"<p><strong>Aims: </strong>To compare all-cause and Alzheimer's disease (AD)-related healthcare resource utilization (HCRU) by cognitive stage.</p><p><strong>Methods and materials: </strong>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.</p><p><strong>Results: </strong>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], <i>p</i> < 0.001) and outpatient visits (IRR: 2.76 [95% CI: 1.68-4.54], <i>p</i> < 0.001) compared to the early AD cohort.</p><p><strong>Limitations: </strong>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.</p><p><strong>Conclusions: </strong>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.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":" ","pages":"81-88"},"PeriodicalIF":2.9,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142818343","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cost-effectiveness of finerenone therapy for patients with chronic kidney disease and type 2 diabetes in England & Wales: results of the FINE-CKD model.","authors":"David Cherney, Aleksandra Drzewiecka, Kerstin Folkerts, Pierre Levy, Aurélie Millier, Stephen Morris, Michał Pochopień, Prabir Roy-Chaudhury, Sean D Sullivan, Paul Mernagh","doi":"10.1080/13696998.2025.2451526","DOIUrl":"10.1080/13696998.2025.2451526","url":null,"abstract":"<p><strong>Objective: </strong>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.</p><p><strong>Methods: </strong>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.</p><p><strong>Results: </strong>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.</p><p><strong>Conclusion: </strong>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.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":" ","pages":"196-206"},"PeriodicalIF":2.9,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142950176","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cost-effectiveness of outpatient COVID-19 antiviral treatment with nirmatrelvir/ritonavir versus usual care in Swedish patients with various risk factors.","authors":"Fredrik Nilsson, Martina Aldvén, Christian Gerdesköld Rappe, Tendai Mugwagwa","doi":"10.1080/13696998.2024.2444836","DOIUrl":"10.1080/13696998.2024.2444836","url":null,"abstract":"<p><strong>Aims: </strong>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.</p><p><strong>Materials and methods: </strong>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.</p><p><strong>Results: </strong>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.</p><p><strong>Limitations: </strong>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.</p><p><strong>Conclusions: </strong>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.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":" ","pages":"186-195"},"PeriodicalIF":2.9,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142864541","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Systematic review of cost-effectiveness modelling studies for haemophilia.","authors":"Niklaus Meier, Daniel Ammann, Mark Pletscher, Jano Probst, Matthias Schwenkglenks","doi":"10.1080/13696998.2024.2444157","DOIUrl":"10.1080/13696998.2024.2444157","url":null,"abstract":"<p><strong>Aims: </strong>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.</p><p><strong>Methods: </strong>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.</p><p><strong>Results: </strong>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.</p><p><strong>Limitations: </strong>It was not possible to perform a quantitative synthesis of the results due to the heterogeneity of the underlying studies.</p><p><strong>Conclusion: </strong>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.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":" ","pages":"89-104"},"PeriodicalIF":2.9,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142854539","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cost-effectiveness of a machine learning risk prediction model (LungFlag) in the selection of high-risk individuals for non-small cell lung cancer screening in Spain.","authors":"Juan Carlos Trujillo, Joan B Soriano, Mercè Marzo, Oliver Higuera, Luis Gorospe, Virginia Pajares, María Eugenia Olmedo, Natalia Arrabal, Andrés Flores, José Francisco García, María Crespo, David Carcedo, Carolina Heuser, Milan M S Obradović, Nicolò Olghi, Eran N Choman, Luis M Seijo","doi":"10.1080/13696998.2024.2444781","DOIUrl":"10.1080/13696998.2024.2444781","url":null,"abstract":"<p><strong>Objective: </strong>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.</p><p><strong>Methods: </strong>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.</p><p><strong>Results: </strong>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.</p><p><strong>Conclusions: </strong>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.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":" ","pages":"147-156"},"PeriodicalIF":2.9,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142854530","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cost-utility analysis of newborn screening for spinal muscular atrophy in Japan.","authors":"Akira Hata, Akihito Uda, Satoru Tanaka, Diana Weidlich, Walter Toro, Laetitia Schmitt, Ataru Igarashi, Matthias Bischof","doi":"10.1080/13696998.2024.2439734","DOIUrl":"10.1080/13696998.2024.2439734","url":null,"abstract":"<p><strong>Aims: </strong>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.</p><p><strong>Materials and methods: </strong>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.</p><p><strong>Results: </strong>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.</p><p><strong>Limitations: </strong>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.</p><p><strong>Conclusion: </strong>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.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":" ","pages":"44-53"},"PeriodicalIF":2.9,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142785717","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Real-world psoriasis treatment patterns and disease burden in Germany, with a focus on biologics and apremilast: data from a German statutory health insurance database.","authors":"Andreas Pinter, Marcus Schulte, Nils Kossack, Marc Pignot, Michael Schultze, Andrea Feldhus","doi":"10.1080/13696998.2025.2452054","DOIUrl":"10.1080/13696998.2025.2452054","url":null,"abstract":"<p><strong>Background: </strong>Psoriasis is a chronic, systemic, inflammatory skin disease, with increasing prevalence; however, few studies have reported real-world prescription patterns and healthcare burden.</p><p><strong>Objectives: </strong>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.</p><p><strong>Methods: </strong>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.</p><p><strong>Results: </strong>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% (<i>n</i> = 101) received index drug apremilast, 10.2% (<i>n</i> = 287) anti-IL, 6.8% (<i>n</i> = 191) anti-TNF, and 79.4% (<i>n</i> = 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 <i>vs.</i> anti-IL and anti-TNF groups (24.8% <i>vs.</i> 59.6% and 53.9%; 36.6% <i>vs.</i> 66.9% and 57.6%, respectively). During a 12-month baseline period, psoriasis-related hospitalization was lower for apremilast <i>vs.</i> anti-IL and anti-TNF groups (4.95% <i>vs.</i> 15.68% and 14.14%) and higher during 12 months' follow-up (5.94% <i>vs.</i> 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 <i>vs.</i> apremilast, respectively, and the main driver for lower overall apremilast costs.</p><p><strong>Conclusion: </strong>Given variation in treatment adherence/persistence, HCRU, and costs between apremilast and biologics, these findings could be key considerations during treatment selection.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":" ","pages":"207-220"},"PeriodicalIF":2.9,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142978947","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}