Journal of Medical Economics最新文献

{"title":"Enhanced patient outcomes and improved budget impact from increased PFO diagnostic testing in cryptogenic stroke workup: a US hospital perspective.","authors":"John J Volpi, Giorgia Tiozzo, Jet Neervoort, Timon Louwsma, Anne K Marti, Erik J Landaas, Mitesh Nakum, Ryan J Imhoff","doi":"10.1080/13696998.2026.2630603","DOIUrl":"10.1080/13696998.2026.2630603","url":null,"abstract":"<p><strong>Aim: </strong>Limited research exists on the hospital-level costs and reimbursement associated with PFO diagnostic testing. This study estimates the impact of fully implementing guideline-driven PFO diagnostic evaluation in patients with a history of cryptogenic stroke, from the perspective of an US hospital, focusing on costs, hospital reimbursement, and clinical outcomes.</p><p><strong>Methods: </strong>A budget impact analysis (BIA) was conducted from the perspective of a typical US hospital managing an average of 186 new cryptogenic stroke patients annually. Two scenarios were compared: the Current Diagnostic scenario, with a 54% testing rate, versus an Increased Testing scenario with a 100% testing rate. A hybrid decision tree combined with a Markov health state transition model was used to simulate costs and clinical outcomes over a one-year time horizon. Clinical and diagnostic input parameters were derived from the literature, while cost data were obtained from the Centers for Medicare and Medicaid Services (CMS) and other published sources.</p><p><strong>Results: </strong>Among a cohort of 186 cryptogenic stroke patients, testing 100% of eligible patients (compared to 54%) was expected to result in diagnosing an additional 21 PFO-related strokes (46 vs. 25), and with 8 more PFO closures (17 vs. 9) over a period of one year. This translated to a total expected reimbursement increase of $378,359 ($719,738 vs $1,098,097) over a one-year period. Most reimbursement increases came from PFO closure ($275,546), with contributions from diagnostics ($96,880) and adverse event management ($13,919). The total increase in contribution margin was $191,655 from improving PFO diagnostics.</p><p><strong>Conclusions: </strong>Increased diagnostic testing for PFO-associated stroke is expected to improve patient health outcomes and increase contribution margin from an US hospital perspective. This evidence supports improving PFO testing in US hospitals to reduce recurrent strokes and improve hospital efficiency.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":"29 1","pages":"620-634"},"PeriodicalIF":3.1,"publicationDate":"2026-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147321584","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":"Healthcare burden of mixed aortic valve stenosis and insufficiency disease.","authors":"Ibrahim Sultan, Michael Ryan, Candace Gunnarsson, Godfred Marfo, Prashanth Vallabhajosyula","doi":"10.1080/13696998.2026.2635880","DOIUrl":"10.1080/13696998.2026.2635880","url":null,"abstract":"<p><strong>Objective: </strong>To compare mortality, healthcare utilization, and costs between patients with symptomatic and asymptomatic mixed aortic valve disease (MAVD).</p><p><strong>Methods: </strong>We analyzed Optum United Health Care database for US patients with aortic insufficiency (AI) claims (2017-2024) and prior/concurrent aortic stenosis (AS) claims, requiring 12 months continuous enrollment. Patients with baseline aortic valve replacement (AVR) were excluded. Symptomatic MAVD (SMAVD) was defined as ≥2 baseline visits for heart failure, angina, dyspnea, or syncope. Outcomes included mortality, time to home health/skilled nursing facility (SNF), and annualized healthcare utilization and costs, analyzed using Cox proportional hazard and general linear models.</p><p><strong>Results: </strong>Among 132,361 MAVD patients, 73.62% (<i>n</i> = 97,448) were symptomatic at diagnosis. Of initially asymptomatic patients, 58.94% became symptomatic within 5 years. Only 22% received AVR within 5 years. SMAVD patients had higher mortality (HR 1.48, 95% CI 1.44-1.53), home health utilization (HR 1.32, 95% CI 1.30-1.35), SNF admissions (HR 1.38, 95% CI 1.35-1.41), and $11,120 higher annual costs.</p><p><strong>Conclusions: </strong>SMAVD patients experience significantly worse outcomes than asymptomatic patients. Early detection and timely interventions are critical to reducing MAVD's healthcare burden.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":"29 1","pages":"761-771"},"PeriodicalIF":3.1,"publicationDate":"2026-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147377776","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":"Loss of productivity among commercially insured patients with pulmonary arterial hypertension in the United States.","authors":"Anna Watzker, Christine Ferro, Gabriela Dieguez, Charmaine Girdish, Adnan Alsumali, Dominik Lautsch, Karim El-Kersh","doi":"10.1080/13696998.2026.2642553","DOIUrl":"10.1080/13696998.2026.2642553","url":null,"abstract":"<p><strong>Background: </strong>Despite advances in treatment, pulmonary arterial hypertension (PAH) remains a progressive condition with the onset of disease often in working-aged adults, leading to substantial economic burden and increased healthcare resource utilization despite the rarity of the disease. The aim of this study was to estimate productivity loss for patients with PAH.</p><p><strong>Methods: </strong>This was a retrospective cohort study of the Milliman Consolidated Health Cost Guidelines Source Data and the Merative MarketScan Commercial dataset between 01/01/2018 and 09/30/2023. Adult patients diagnosed and treated for PAH between 01/01/2019 and 08/31/2023 were identified and followed through the earliest of end of enrollment, or end of data. Outcomes were described as average workdays lost to receive healthcare services per-patient per-year (PPPY) and further assessed by healthcare setting. The cost of productivity loss was calculated based on the annual median household income.</p><p><strong>Results: </strong>The study included 1,588 commercially insured patients (mean age 52 years, 63% female). Between 2019-2023, the mean number of workdays lost ranged between 21 and 23 PPPY, equating to over 8% of annual workdays. The cost of loss of productivity ranged from $8,828 to $9,599 PPPY (in 2023 dollars). Across all study years, the largest proportion of workdays lost were attributed to emergency room (ER)/observation, followed by office visits, and outpatient facilities, altogether comprising two-thirds of total workdays lost. Inpatient hospitalization-related workday loss was approximately 11% of total workdays lost. Over the study period, the largest change in workdays lost was observed for ER/observation.</p><p><strong>Conclusion: </strong>This study demonstrates substantial workdays lost due to health services utilization among patients with PAH, further adding to the overall economic burden of PAH. Quantifying productivity loss in PAH patients provides critical insight into the broader societal costs of the disease, supporting the inclusion of indirect costs in future economic evaluations and healthcare policy decisions.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":"29 1","pages":"909-918"},"PeriodicalIF":3.1,"publicationDate":"2026-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147491170","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":"Addressing the challenges of health economic modelling in the context of suboptimal evidence base - case study based on a comparison between photodynamic diagnosis and narrow band imaging in non-muscle invasive bladder cancer.","authors":"Jonathan Belsey, Wojciech Chrosny, Jane Lapon","doi":"10.1080/13696998.2026.2661553","DOIUrl":"10.1080/13696998.2026.2661553","url":null,"abstract":"<p><strong>Introduction: </strong>Standard economic models, which have been developed to reflect typical data availability for pharmaceutical products, often struggle with non-pharmaceutical interventions like diagnostics and medical devices, where clinical trial data is more likely to be short-term or non-comparative. This paper explores methodological challenges in evaluating Photodynamic Diagnosis (PDD) <i>via</i> blue light cystoscopy (BLC) versus Narrow Band Imaging (NBI) for non-muscle invasive bladder cancer (NMIBC).</p><p><strong>Methods: </strong>A semi-Markov state-transition cost-utility model was developed for a health technology appraisal (HTA) by the Danish Treatment Council. The basis of the model was a differential effect of the two technologies on the risk of early local cancer recurrence. Due to a lack of direct comparative trials, an indirect hazard-function-based approach was used. Using TreeAge Pro software, baseline hazard curves were derived from ten-year survival data for conventional white light cystoscopy. Hazard ratios (HRs) from meta-analyses were then applied to these curves to derive simulated time-to-event curves for BLC and NBI.</p><p><strong>Results: </strong>The base-case analysis yielded an incremental cost-effectiveness ratio (ICER) of DKK 70,707/QALY for BLC-TURBT versus NBI-TURBT. Sensitivity analyses confirmed results remained robust and well below the DKK 500,000/QALY willingness-to-pay threshold. The model was most sensitive to HRs for time to first recurrence. Scenario analyses, including traditional parametric extrapolation, yielded consistent ICERs between DKK 36,775 and DKK 215,965/QALY.</p><p><strong>Discussion: </strong>The hazard-based workflow effectively integrated survival data from disparate sources, using a software-based method was quicker, simpler and more intuitive to use than conventional statistical methods. The approach used is equally applicable to a partitioned survival structure. The alignment between hazard-based and traditional parametric methods suggests this is a valid, efficient alternative for developing models in the face of evidence gaps.</p><p><strong>Conclusions: </strong>A hazard-function approach provides a transparent, practical solution for building robust economic models when clinical data is limited or incompatible with standard approaches.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":"29 1","pages":"1341-1354"},"PeriodicalIF":3.1,"publicationDate":"2026-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147773854","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":"Estimating the budget impact of introducing dostarlimab as first line in treating endometrial cancer in Saudi Arabia.","authors":"Mohamed Aseafan, Faisal Azam, Ahmed S Alanazi, Shaheed Alhumaid, Nawaf H Almalki, Mohamed Masoud, Zainab Alsharef, Hany Soliman, Mohamed El-Khedr Hassan","doi":"10.1080/13696998.2026.2706988","DOIUrl":"https://doi.org/10.1080/13696998.2026.2706988","url":null,"abstract":"<p><strong>Objective: </strong>The budget impact of introducing dostarlimab plus carboplatin-paclitaxel (CP) as a first-line treatment option for primary advanced or recurrent endometrial cancer (EC) in Saudi Arabia was evaluated from the perspective of the Saudi national payer.</p><p><strong>Methods: </strong>The budget impact model (BIM) utilized epidemiological, clinical, and cost and resource use inputs to evaluate the total costs associated with a market scenario without dostarlimab versus a market scenario with dostarlimab over a three-year time horizon (2026-2028). Currently available comparator treatments in Saudi Arabia were evaluated based on associated market share and relevance as comparable treatment regimens to dostarlimab plus CP. A one-way sensitivity analysis was performed to identify the sensitivity of the BIM to changes in input parameters.</p><p><strong>Results: </strong>Out of 250 eligible patients, 58 were estimated to receive dostarlimab plus CP as a first-line EC treatment during its first year on the market, increasing to 242 out of 569 eligible patients in the third year. The resulting annual budget impact was -$326,607 in the first year and -$668,830 in the third year of market availability, with a total cumulative budget impact of -$1,650,171 over the three-year time horizon. The mean annual budget impact per patient treated with dostarlimab plus CP was -$4,388.57, a decrease of -1.84% compared with the scenario without dostarlimab.</p><p><strong>Conclusions: </strong>The introduction of dostarlimab plus CP to healthcare settings for first-line treatment of primary advanced and recurrent EC was found to be budget neutral in this model, with lower treatment acquisition costs and decreased second-line subsequent treatment costs compared with the other currently available treatment options.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":"29 1","pages":"2063-2075"},"PeriodicalIF":3.1,"publicationDate":"2026-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148630613","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":"Italian policies and incentives introduced to tackle antimicrobial resistance: an emerging model.","authors":"Andrea Zovi","doi":"10.1080/13696998.2026.2708509","DOIUrl":"https://doi.org/10.1080/13696998.2026.2708509","url":null,"abstract":"","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":"29 1","pages":"2086-2090"},"PeriodicalIF":3.1,"publicationDate":"2026-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148630627","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":"Clinical and economic outcomes of cabazitaxel versus Lu-PSMA in mCRPC: a US perspective.","authors":"Braydon Nault, David Garcia, Heather Cameron, Andrew Revel, Kevin Steele, Kyle Flannery, Pedro Barata","doi":"10.1080/13696998.2026.2709297","DOIUrl":"https://doi.org/10.1080/13696998.2026.2709297","url":null,"abstract":"<p><strong>Background: </strong>The TheraP trial compared cabazitaxel to Lutetium-177-labeled PSMA-617 (Lu-PSMA) in metastatic castration-resistant prostate cancer (mCRPC) following docetaxel and an androgen receptor pathway inhibitor (ARPI). Lu-PSMA demonstrated improved PSA response (66% vs 37%; <i>p</i> < 0.0001) and progression-free survival (HR = 0.63; <i>p</i> = 0.0028), while overall survival (OS; median 16.4 vs. 19.4 months) and rates of Grade 3 (0.88 vs. 0.96 events/patient) and Grade 4 adverse events (AE; 0.11 vs. 0.08 events/patient) were comparable. The comparative economic implications of these two therapies in the United States (US) remain uncertain.</p><p><strong>Methods: </strong>A cost-consequence Excel model was developed from the US Medicare perspective to evaluate the direct cost outcomes for mCRPC patients receiving cabazitaxel or Lu-PSMA. Inputs were derived from TheraP, supplemented by literature and clinical expert validation. Costs included PSMA testing, drug acquisition and administration, supportive care, AE management, and end-of-life care. Outcomes (derived from TheraP inputs) included OS, progression-free survival (PFS), PSA-PFS, and radiographic-PFS (rPFS). An 18-month time horizon aligned with clinical follow-up. Costs were reported in 2025 USD.</p><p><strong>Results: </strong>At 18 months in the modeled cohort of 100 patients, cabazitaxel was associated with nine additional modeled survivors; this should be interpreted cautiously, as TheraP reported no statistically significant difference in OS between cabazitaxel and Lu-PSMA. In contrast, Lu-PSMA demonstrated improved PFS outcomes in the modeled cohort, with eight more patients remaining progression-free and 15 more radiographic progression-free. From the Medicare perspective, cabazitaxel was associated with a per-patient cost of $107,729, versus $303,338 per patient for Lu-PSMA (-$195,608). Cost differences were driven primarily by lower drug acquisition costs for cabazitaxel.</p><p><strong>Conclusions: </strong>Cabazitaxel provides a clinically validated and economically favorable treatment option post-docetaxel and ARPI, providing substantial cost savings across payer scenarios with no observed difference in OS. Lu-PSMA offers improved PSA-based outcomes and progression-related endpoints at higher treatment costs. These findings support clinical-economic trade-off considerations when sequencing therapies for mCRPC.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":"29 1","pages":"2107-2120"},"PeriodicalIF":3.1,"publicationDate":"2026-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148684911","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":"Annual vaccination with BNT162b2 in Germany can avoid substantial clinical and economic burden of COVID-19 disease.","authors":"Amy Barber, Julie Roiz, Sam Greenall, Edward Church, Andrea Schmetz, Stefan Riebel, Jingyan Yang, Oliver Witzke, Afschin Gandjour","doi":"10.1080/13696998.2025.2604970","DOIUrl":"https://doi.org/10.1080/13696998.2025.2604970","url":null,"abstract":"<p><strong>Aims: </strong>To quantify the clinical and economic burden of Coronavirus disease 19 (COVID-19), and burden potentially avoided with annual vaccination, in German adults in an endemic setting.</p><p><strong>Materials and methods: </strong>A decision tree model was constructed to estimate the clinical and economic impact of COVID-19 and projected burden avoided with BNT162b2, a seasonally adapted mRNA vaccine against severe COVID-19 disease. The majority of cost inputs and clinical event probabilities were informed by German real-world evidence from seasons 2022/23 and 2023/24. Vaccine efficacy was derived from US and German studies. Long/Post COVID impact was assessed in scenario analysis.</p><p><strong>Results: </strong>The model estimated up to 20.8 million symptomatic infections per year in adults, including 7.5 million in people aged 60+, and 6.0 million in comorbid adults. This translates to an estimated 198,598 hospitalizations and 24,626 COVID-attributable deaths, with 93.6% of deaths occurring in people aged 60+. The total economic burden including productivity loss was estimated at €1.4 billion in people aged 60+, €2.0 billion in comorbid adults, and €3.9 billion in all working adults. Long/Post COVID increased the economic burden by 1.7 times.</p><p><p>Assuming vaccination of 100% of recommended groups, 11.0 million symptomatic infections could be prevented, with the greatest impact in people aged 60+ (estimated 110,362 hospitalizations and 13,685 deaths avoided). For people aged 60+, comorbid adults, and all working adults, the number needed to vaccinate to prevent one symptomatic infection was 7, 4, and 4 people; to prevent one hospitalization, 231, 2,363, and 4,549; and to avoid one death, 1,862, 19,055, and 36,683, respectively.</p><p><strong>Conclusion: </strong>COVID-19 imposes a substantial clinical and economic burden on the German population, which could be mitigated with an expanded COVID-19 vaccination program. Further research into Long/Post COVID is needed. Our study presents considerations highlighting the value of broad vaccination especially for working adults.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":"29 1","pages":"16-28"},"PeriodicalIF":3.0,"publicationDate":"2026-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145846571","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 pasireotide long-acting release in acromegaly: a systematic literature review and methodology assessment.","authors":"Aleksandra Gilis-Januszewska, Małgorzata Bronikowska, Grzegorz Binowski, Michał Jachimowicz, Beatrice Gueron, Fabian Schmidt, Sika Dédé Kossi","doi":"10.1080/13696998.2025.2609506","DOIUrl":"10.1080/13696998.2025.2609506","url":null,"abstract":"<p><strong>Background: </strong>Acromegaly is a rare and progressive condition caused by excessive secretion of growth hormone and insulin-like growth factor type 1. Pasireotide long-acting release (LAR) is indicated as a second-line therapy for adults with acromegaly who are unsuitable for or unresponsive to surgery and inadequately controlled with first-generation somatostatin receptor ligands (FGSRLs). Although its efficacy and safety have already been established, its cost-effectiveness remains unclear. The primary objective of this study was to systematically assess the cost-effectiveness of pasireotide LAR compared to other second-line medical options, particularly pegvisomant.</p><p><strong>Methods: </strong>A systematic literature review was conducted in May 2024 in Medline, Medline In Process, Web of Science, and the Centre for Reviews and Dissemination (CRD), York. Studies were eligible if they were full or partial economic analyses of pasireotide LAR as a second-line pharmacological treatment for adult patients with acromegaly. Studies not available in English and publications prior to 2009 were excluded. Included articles were assessed for transparency using the appropriate checklists. Data extraction focused on costs, incremental cost-effectiveness ratios and quality-adjusted life years. Formal data synthesis of outcomes was not undertaken due to the small number of studies and notable heterogeneity between them. Methodological assessment involving the evaluation of model inputs, data sources, and their alignment with the evidence on the course of disease and clinical practice was performed to examine the main factors contributing to discrepancies in cost-effectiveness outcomes and assess the credibility of the results.</p><p><strong>Results: </strong>Of 160 records identified, six publications met the inclusion criteria. Their findings demonstrated variability. The critical assessment highlighted considerable variability in methodological rigor among the included studies.</p><p><strong>Conclusions: </strong>Evidence on pasireotide LAR's cost-effectiveness versus pegvisomant regimens remains inconclusive. Although two studies indicated that pegvisomant is more cost-effective, the study with the highest methodological credibility found that pasireotide LAR is a cost-effective alternative.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":"29 1","pages":"135-154"},"PeriodicalIF":3.0,"publicationDate":"2026-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145949003","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":"Rett syndrome and real-world treatment patterns of trofinetide in the United States.","authors":"Nazia Rashid, Vinod Kumar Yakkala, Safiuddin Shoeb Syed, Krithika Rajagopalan","doi":"10.1080/13696998.2026.2682668","DOIUrl":"10.1080/13696998.2026.2682668","url":null,"abstract":"<p><strong>Background: </strong>Trofinetide (TROF) remains the first and only FDA-approved pharmacologic treatment for Rett syndrome (RTT). There is limited real-world evidence on TROF use, restarts, dosing patterns and predictors of non-persistence. This study evaluated long-term treatment patterns and baseline predictors of non-persistence among RTT individuals initiating TROF.</p><p><strong>Methods: </strong>A retrospective claims analysis of linked IQVIA Anonymized Patient Level Data and TROF pharmacy data (1 January 2021 to 30 September 2024) was performed. RTT individuals initiating 1^st TROF prescription (RX) between 1 April 2023 and 31 March 2024 with ≥6 months of pre- and post-index continuous enrollment were classified as persistent (gap ≤90 days) and non-persistent. Median (IQR) time on treatment was analyzed, and Kaplan-Meier assessed time to non-persistence. Restarts among non-persistent were examined. Dosing patterns such as mean dose in milligrams (mg) and percentage target daily dose (%TDD) at each RX were evaluated. Predictors of non-persistence were assessed using logistic regression.</p><p><strong>Results: </strong>Among 1,175 TROF initiators, 54.9% were persistent and 45.1% were non-persistent; 7.6% of non-persistent individuals restarted. Mean ± SD age was 14.7 ± 10.8 vs. 16.5 ± 11.6 years and median ± IQR time on TROF was 14.3 ± 4.6 vs. 3.0 ± 3.8 months, respectively. Over 75% remained on treatment beyond 3 months, and more than half continued through study follow-up. Mean twice daily (BID) TROF dose was lower among persistent (7422.0 mg) vs. non-persistent (7850.8 mg) at RX1 and increased to 7953.7 mg vs 8423.4 mg, respectively at RX2. Thereafter, the persistent individuals maintained stable dosing, while the non-persistent showed greater fluctuation over time. Similar patterns were observed for %TDD. Older age and history of infectious disorders were significantly associated with non-persistence.</p><p><strong>Conclusions: </strong>In this real-world analysis, more than half of RTT individuals remained persistent on TROF for ≥14 months. Stable dosing patterns were observed among persistent individuals, while greater dose variability was observed among non-persistent individuals. Thus, these findings may provide guidance to clinicians about long-term TROF use in real-world practice.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":"29 1","pages":"1715-1727"},"PeriodicalIF":3.0,"publicationDate":"2026-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148239402","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}