Journal Of Medical Economics[JOURNAL]
Sun
200 papers
Cost per event avoided: adopt or avoid?
Petrou P, Pitsillidou O, Petrou C … +1 more , Postma MJ
J Med Econ · 2026 Dec · PMID 41838865 · Publisher ↗
Abstract loading — click title to view on PubMed.
Employment and productivity losses from cervical spinal cord injury: a 7-year nationwide cohort study in Taiwan, 2012-2018.
Wang WM, Wang JD, Wang F … +1 more , Lien WC
J Med Econ · 2026 Dec · PMID 41831219 · Publisher ↗
OBJECTIVE: To investigate the extent to which employment and productivity losses could be prevented by mitigating cervical spinal cord injury (SCI) and concurrent traumatic brain injury (TBI) in Taiwan. DESIGN: Using Tai...
OBJECTIVE: To investigate the extent to which employment and productivity losses could be prevented by mitigating cervical spinal cord injury (SCI) and concurrent traumatic brain injury (TBI) in Taiwan. DESIGN: Using Taiwan's National Health Insurance Database, we identified individuals with cervical SCI and concurrent TBI, with the 7-year data collection period (2012-2018) and a general population cohort as the reference. Survival and employment status were extrapolated to lifetime and age using rolling extrapolation with restricted cubic spline regression and generalized linear models. RESULTS: This study included 2,852 patients with cervical SCI and 367 with concurrent TBI. In cervical SCI, loss-of-life expectancy, loss-of-lifetime employment duration (LED), and loss-of-lifetime insured salary (LIS; a proxy for productivity) were 19.3 years, 7.5 years, and $122,998, respectively. The relative loss-of-LED of motor-vehicle-related cervical SCI with concurrent TBI was significantly higher than that without TBI (72.7% vs. 64.2%). The relative loss-of-LED in middle-aged cervical SCI was significantly higher than loss-of-LIS (58.2% vs. 63.5%), suggesting that total productivity was more severely affected than working time. CONCLUSION: This 7-year nationwide cohort provides the first lifetime employment and productivity loss estimates for cervical SCI, demonstrating that concurrent TBI and younger age amplify economic burden. The findings shift the policy focus from generic SCI prevention to specific, high-yield interventions: mandatory TBI screening, age-tailored vocational programs, and productivity-targeted workplace accommodations. These evidence-based figures enable precise cost-benefit analyses for preventive measures (e.g. enhanced helmet legislation, road safety) and rehabilitation investments, offering actionable data for Taiwan's health and labor policymakers.
Bleeding rates, healthcare utilization, and costs among patients with hemophilia a without inhibitors treated with concomitant octocog alfa or extended half-life factor VIII while on emicizumab prophylaxis.
Montcrieff C, Caicedo J, Cerretani A … +3 more , Fan AQ, Bullano M, Ponomareva E
J Med Econ · 2026 Dec · PMID 41823988 · Publisher ↗
AIM: Emicizumab has changed prophylactic treatment for HA, but the need for FVIII replacement still exists for the treatment of breakthrough bleeds, perioperative management, and participation in high-risk activities/spo...
AIM: Emicizumab has changed prophylactic treatment for HA, but the need for FVIII replacement still exists for the treatment of breakthrough bleeds, perioperative management, and participation in high-risk activities/sports. The primary objectives of this study were to describe the characteristics of individuals with congenital hemophilia A (HA) without inhibitors receiving emicizumab prophylaxis and to compare clinical outcomes, healthcare resource utilization (HCRU), and costs among those prescribed octocog alfa [Advate, standard half-life factor VIII (FVIII)] extended half-life (EHL) FVIII agents. METHODS: Patients with congenital HA without inhibitors and emicizumab use were identified retrospectively from Inovalon claims data and stratified by concomitant octocog alfa EHL FVIII use. Inverse probability treatment weighting (IPTW) and multivariable regression were used to balance baseline covariates and compare billable annualized rate of bleeding (ABR), healthcare utilization, and costs between subgroups. RESULTS: Among the 1,282 patients receiving emicizumab prophylaxis, 501 received concomitant FVIII prescription for octocog alfa ( = 274) or EHL ( = 227) during follow-up. Following IPTW adjustment, total and medical costs were comparable between groups; however, pharmacy costs associated with concomitant octocog alfa therapy were significantly lower compared with EHL products ($35,381 $61,739; < 0.001). Importantly, ABR (0.35 0.28; = 0.34) and bleed-related healthcare utilization remained similar across cohorts. LIMITATIONS: Findings are limited by the retrospective design and reliance on claims data, which may omit home-treated bleeds, and misrepresent FVIII utilization due to billing complexities and assumptions about dispensed product use. CONCLUSIONS: While the choice of concomitant FVIII does not substantially influence ABR or bleed-related HCRU, significantly lower pharmacy costs with octocog alfa compared with EHL agents highlight its potential cost saving and support its consideration as a preferred on-demand treatment option in patients with HA without inhibitors on emicizumab prophylaxis.
Real-world clinical, safety, and economic evaluation of infliximab biosimilar adoption for crohn's disease.
Shi J, Pan Y, Cao Q … +1 more , Shi L
J Med Econ · 2026 Dec · PMID 41811347 · Publisher ↗
BACKGROUND: Biosimilar infliximab (B-IFX) offers a lower-cost alternative to originator infliximab (O-IFX) for treating Crohn's disease (CD), but real-world data on comparative effectiveness and economic impact in China...
BACKGROUND: Biosimilar infliximab (B-IFX) offers a lower-cost alternative to originator infliximab (O-IFX) for treating Crohn's disease (CD), but real-world data on comparative effectiveness and economic impact in China remain limited. This study assessed clinical outcomes and economic consequences of B-IFX versus O-IFX from a healthcare payer perspective. METHODS: This retrospective cohort study evaluated 473 adult CD patients initiating O-IFX ( = 345) or B-IFX ( = 128) at a tertiary care center for Chron's disease in China between October 2021 and February 2024. The clinical endpoints over 12 months included treatment failure, clinical remission, endoscopic response, and adverse events. A budget impact analysis evaluated direct healthcare costs (Chinese Yuan, CNY) and projected medical insurance fund expenditures under a reference scenario (continued O-IFX use) and a base-case scenario with increased B-IFX uptake for 2025-2027. One-way sensitivity analyses assessed model robustness. RESULTS: Rates of treatment failure, clinical remission, endoscopic response, and adverse events were comparable between O-IFX and B-IFX. Relative to the reference scenario, increased B-IFX adoption was associated with lower annual direct medical costs and reduced insurance fund expenditures, yielding estimated savings of 0.60, 1.09, and 1.75 million CNY in 2025-2027, respectively (cumulative savings: 3.44 million CNY). Sensitivity analyses identified the unit price of O-IFX as the main cost driver; B-IFX remained cost saving across all parameter ranges, with cumulative 3-year savings ranging from 2.49 to 4.39 million CNY. CONCLUSIONS: B-IFX demonstrated comparable real-world effectiveness and safety to O-IFX while generating substantial cost savings for China's medical insurance system. These findings support broader biosimilar adoption as a value-based strategy to enhance the affordability and sustainability of biologic therapy for CD.
Analysis of healthcare resource utilization before and after initiation of maribavir for cytomegalovirus treatment.
Kotton CN, Richards M, Princic N … +3 more , Gelone DK, Schultz BG, Bullano M
J Med Econ · 2026 Dec · PMID 41811308 · Publisher ↗
AIM: This retrospective observational cohort study aimed to assess healthcare utilization (HCRU) and costs before and after initiation of treatment with maribavir using a United States (US) administrative claims database...
AIM: This retrospective observational cohort study aimed to assess healthcare utilization (HCRU) and costs before and after initiation of treatment with maribavir using a United States (US) administrative claims database to further the understanding of the impact of maribavir in the real world setting. METHODS: Inovalon Databases were used to identify post-transplant patients with refractory (with or without resistant) CMV treated with maribavir between November 1, 2021 and March 31, 2024 (index = earliest treatment date). HCRU and costs were assessed during variable length periods both prior to and following initiation of maribavir. McNemar's tests were used to evaluate the statistical significance of differences for categorical variables and paired -tests were used for continuous variables. RESULTS: There were 230 patients eligible for analysis. Compared with the pre-maribavir phase, after the initiation of maribavir there was a significant decrease in the utilization of all-cause acute care services, including inpatient hospital admissions (54.3% v. 30.9%; < 0.001) and emergency room visits (47.0% v. 36.5%; = 0.014). There was a significant decrease in the number of outpatient office visits per patient per month (PPPM) (2.33 v. 1.47; < 0.001) as well as a decrease in the number of all outpatient pharmacy prescriptions PPPM (9.64 v. 7.69; < 0.001). Total medical costs measured PPPM prior to maribavir initiation were $9,986 compared with $5,480 ( < 0.001) after initiation of maribavir. CONCLUSIONS: In real world practice, initiation of maribavir for the treatment of refractory or drug-resistant post-transplant CMV was associated with decreased acute care service utilization, reduced outpatient visits, and lower healthcare costs, resulting in meaningful benefits for both patients and healthcare systems. These reductions in healthcare utilization highlight maribavir's role in optimizing treatment strategies and improving the efficiency of CMV management.
Economic value of lost productivity attributable to premature cancer mortality and morbidity across Europe.
Brandtmüller A, Meiwald A, Toth P … +2 more , Perera C, Fox A
J Med Econ · 2026 Dec · PMID 41811249 · Publisher ↗
BACKGROUND: Cancer imposes a substantial clinical and economic burden across Europe. This analysis estimated the lost productivity cost of the five cancer types with the highest premature mortality and five cancer types...
BACKGROUND: Cancer imposes a substantial clinical and economic burden across Europe. This analysis estimated the lost productivity cost of the five cancer types with the highest premature mortality and five cancer types with the highest morbidity burden across 28 European countries in 2022. METHODS: Data on cancer deaths, years of life lost (YLL), and years of life lived with disability (YLD) were sourced from the Institute for Health Metrics and Evaluation Global Burden of Disease database. Country- and sex-specific demographic and economic parameters were obtained. Years of productive life lost (YPLL) and present value of future lost productivity (PVFLP) were estimated for individuals dying before retirement, while the value of YLD (VYLD) quantified earnings lost due to cancer-related disability. Outcomes were adjusted for labor force participation and unemployment. Deterministic sensitivity analyses assessed the impact of variations in model inputs. RESULTS: In 2022, tracheal, bronchus, and lung cancer, colon and rectum cancer, breast, pancreatic, and prostate cancers accounted for 899,581 deaths, 17,274,882 YLL, 896,510 YPLL, and €30,749,064,014 PVFLP across Europe. Tracheal, bronchus, and lung cancer contributed the largest share of mortality-related productivity losses with 37.2% of deaths and 43.7% of PVFLP. Breast cancer had the highest PVFLP per death (€49,411). For morbidity, breast, prostate, colon and rectum cancer, tracheal, bronchus, and lung cancer, and non-Hodgkin lymphoma resulted in 993,648 YLD, 210,400 productive YLD (PYLD), and €9,087,096,712 VYLD. Germany, France, and United Kingdom were the largest contributors to both mortality and morbidity burdens. Sensitivity analyses indicated that retirement age was the key driver of results. CONCLUSION: Cancer continues to exert a considerable productivity burden across Europe with substantial variation between countries. These findings highlight the need for equitable cancer prevention, early diagnosis, and timely access to treatment to reduce both health and economic disparities across Europe.
Economic and clinical value of hydrolyzed collagen in the management of spine surgery wounds: a cost-effectiveness analysis.
Mohan V, Meyers JE, Cohen BG … +2 more , Steel P, Padula WV
J Med Econ · 2026 Dec · PMID 41801683 · Publisher ↗
AIMS: To assess the cost-effectiveness of hydrolyzed collagen as an adjunct to standard care for reducing postoperative complications in high-risk spinal surgery patients. METHODS: A decision tree model was developed fro...
AIMS: To assess the cost-effectiveness of hydrolyzed collagen as an adjunct to standard care for reducing postoperative complications in high-risk spinal surgery patients. METHODS: A decision tree model was developed from the U.S. healthcare sector perspective to compare hydrolyzed collagen with standard care alone over a one-year time horizon. The model followed a cohort of high-comorbidity patients undergoing complex spinal procedures, capturing postoperative infections, seromas, hematomas, readmissions, and revision surgeries. Clinical inputs were derived from published evidence, and costs were reported in 2025 U.S. dollars. Outcomes included direct medical costs, QALYs, and net monetary benefit (NMB) at a willingness-to-pay threshold of $100,000/QALY. Univariate, probabilistic, and threshold sensitivity analyses assessed parameter uncertainty. RESULTS: Hydrolyzed collagen dominated no treatment, generating $3,853 in cost savings and a 0.007 QALY gain per patient over one year, yielding an NMB of $4,542. Avoided readmissions and revision procedures were the primary contributors to cost savings. Baseline complication rates, relative risk reduction, and complication persistence rates were the most influential parameters. In probabilistic analysis, hydrolyzed collagen was cost-effective in more than 99% of simulations. LIMITATIONS: The analysis applies simplifying assumptions, a one-year time horizon, and heterogeneous clinical inputs, factors that may limit the generalizability of the findings. CONCLUSIONS: Compared to standard care alone, hydrolyzed collagen demonstrated a dominant strategy associated with cost savings and improved health outcomes for high-risk spinal surgery patients. These findings support its consideration as a value-enhancing component of the spine surgery care pathway, particularly for patients with elevated complication risk.
A systematic literature review of methodologies used in economic evaluations of combination vaccines.
Di Fusco M, Vaghela S, Mateus C … +1 more , Janke K
J Med Econ · 2026 Dec · PMID 41801186 · Publisher ↗
BACKGROUND: Combination vaccines offer simultaneous protection against different diseases or strains of the same pathogen. They could improve vaccine coverage and health outcomes while reducing healthcare visits and inje...
BACKGROUND: Combination vaccines offer simultaneous protection against different diseases or strains of the same pathogen. They could improve vaccine coverage and health outcomes while reducing healthcare visits and injection costs. This systematic literature review (CRD420251135227) aimed to characterise methodologies employed in full economic evaluations of combination vaccines targeting multiple diseases. METHODS: Searches were performed in August 2025 in MEDLINE, Embase, Web of Science, CEA Registry, International HTA Database, and the NHS Economic Evaluation Database. Studied included cost-utility (CUA), cost-effectiveness (CEA) or cost-benefit analyses (CBA) evaluating all the diseases covered by combination vaccines. Given the qualitative research question, findings were narratively synthesised. Quality assessment used CHEERS and the WHO framework for Immunisation Programmes. RESULTS: Seven studies (3 CUAs; 4 CEAs) were included. They targeted five different combination vaccines across four countries and ∼20 years (1999-2019). All employed static models and compared to single vaccines ( = 3) or no vaccination ( = 5). Two studies adopted a societal perspective, three a healthcare perspective, and two adopted both. Four targeted paediatrics, three targeted adults. The efficacy of combination vaccines was assumed to be equivalent to single vaccines, and value was modelled as the incremental reduction in burden and costs of the additional disease covered, or due to higher coverage and fewer visits. There was variation in assessment of severe and long-term outcomes, adverse events, and productivity loss. Cross-disease data integration (e.g. co-infection, interference) was not considered. All studies generally supported cost-effectiveness of the assessed combination vaccines, but these results largely reflected the choice of comparator and modelling assumptions. Methodological and reporting quality varied widely. CONCLUSIONS: Economic evaluations of combination vaccines targeting multiple diseases remain scarce, outdated and simplistic, highlighting opportunities to explore dynamic transmission models, broader societal perspectives, comparisons to single vaccines and extensive sensitivity analyses.
Healthcare burden of mixed aortic valve stenosis and insufficiency disease.
Sultan I, Ryan M, Gunnarsson C … +2 more , Marfo G, Vallabhajosyula P
J Med Econ · 2026 Dec · PMID 41801137 · Publisher ↗
OBJECTIVE: To compare mortality, healthcare utilization, and costs between patients with symptomatic and asymptomatic mixed aortic valve disease (MAVD). METHODS: We analyzed Optum United Health Care database for US patie...
OBJECTIVE: To compare mortality, healthcare utilization, and costs between patients with symptomatic and asymptomatic mixed aortic valve disease (MAVD). METHODS: 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. RESULTS: Among 132,361 MAVD patients, 73.62% ( = 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. CONCLUSIONS: SMAVD patients experience significantly worse outcomes than asymptomatic patients. Early detection and timely interventions are critical to reducing MAVD's healthcare burden.
The indirect economic burden caused by premature mortality from lung cancer in Asia-Pacific countries; years of life lost and productivity costs.
Gambhir M, Subash V, Alexander D … +3 more , Hughes R, Fox A, Gokhale S
J Med Econ · 2026 Dec · PMID 41790053 · Publisher ↗
BACKGROUND: Lung cancer caused 2.2 million new cases and 1.8 million deaths globally in 2020, accounting for 11.4% of global cancer. In the Asia-Pacific (AP) region, lung cancer is the leading cause of cancer mortality,...
BACKGROUND: Lung cancer caused 2.2 million new cases and 1.8 million deaths globally in 2020, accounting for 11.4% of global cancer. In the Asia-Pacific (AP) region, lung cancer is the leading cause of cancer mortality, contributing to 60% of global lung cancer deaths. This study assesses the economic impact of premature lung cancer deaths in 12 AP countries with different economic profiles by estimating productivity losses. METHODS: The human capital approach was used to estimate productivity losses from premature lung cancer deaths (ICD-10 C33-34) across Singapore, Australia, Hong Kong, New Zealand, South Korea, Taiwan, Malaysia, Thailand, Indonesia, Philippines, Vietnam, and India. Years of productive life lost (YPLL) and the present value of future lost productivity (PVFLP) were calculated based on age-specific mortality, wage, and employment data. Results were analyzed by grouping countries into high- and middle-income categories. FINDINGS: In 2019, 221,293 lung cancer deaths led to 617,574 YPLL and over $2.3 billion in productivity losses across the 12 AP countries. High-income countries experienced greater losses ($1.5 billion, $34,359 per death) compared to middle-income countries ($816 million, $4,660 per death). LIMITATIONS: The analysis excluded direct healthcare costs and productivity losses from morbidity and caregiver burden. Assumptions such as uniform labor force participation and mortality distribution may limit precision. INTERPRETATION: Lung cancer imposes a significant burden across the AP region, with economic disparities between high- and middle-income countries. Findings highlight the need for continued investment in prevention, early detection, and equitable access to treatment, especially in middle-income nations.
Return on investment of national specific health guidance in Japan: an observational study using "Wellness-Star⋆" health insurance claims database.
Ueda T, Kakinuma A, Kinugawa M … +9 more , Yajima S, Miyamori Y, Kakoi T, Yamamoto S, Zhang T, Takeshima T, Chida A, Iwasaki K, Igarashi A
J Med Econ · 2026 Dec · PMID 41790001 · Publisher ↗
BACKGROUND: In Japan, healthcare insurers are required to conduct Specific Health Checkups (SHC) and Specific Health Guidance (SHG) for insured individuals and their dependents aged 40-74 to mitigate the risk of metaboli...
BACKGROUND: In Japan, healthcare insurers are required to conduct Specific Health Checkups (SHC) and Specific Health Guidance (SHG) for insured individuals and their dependents aged 40-74 to mitigate the risk of metabolic illnesses. This study aims to calculate the return on investment (ROI) of the SHG among individuals insured under employment-based health insurance in Japan by using real-world data (RWD). METHODS: This observational study used RWD from the Wellness-Star⋆ database provided by Nippon Life Insurance Company. Individuals who received SHG were categorized into two intervention groups: motivational support (MS) and active support (AS). Corresponding control groups of individuals who did not receive SHG were identified by matching with each intervention subgroup based on baseline parameters such as metabolic levels and healthcare costs. Healthcare costs were tracked and compared over a 4-year follow-up period between each intervention group and its respective control group. The ROI was calculated by dividing the returns by the investments. Returns were defined as the difference in healthcare costs between the control and SHG intervention groups over the 4-year period and investments were the costs associated with SHG interventions. RESULTS: During the follow-up period, the mean per-member-per-month (PMPM) difference in healthcare costs between the control and intervention groups was 1,107 JPY for MS and 1,171 JPY for AS. The ROI for SHG was estimated to be 3.90 (95% confidence intervals [CIs] = -1.97, 9.77) for MS and 1.36 (-0.73, 3.44) for AS. LIMITATIONS: The evaluation was conducted over a relatively short period. The representativeness may be limited due to characteristics of the data, such as being derived from health insurance mainly associated with large corporations. Unmeasured confounders may also have influenced the outcome. CONCLUSIONS: The mean ROI of SHG is greater than 1, suggesting potential economic benefit, though uncertainty remains.
Budget impact model of nadofaragene firadenovec for the treatment of high-risk non-muscle-invasive bladder cancer that is unresponsive to Bacillus Calmette-Guérin immunotherapy.
Yang M, Chai X, Ye D … +3 more , Zhao A, Moradi A, Lotan Y
J Med Econ · 2026 Dec · PMID 41780023 · Publisher ↗
AIMS: To estimate the budget impact of adopting nadofaragene firadenovec for the treatment of adults with high-risk, Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle-invasive bladder cancer (NMIBC) with carcinoma...
AIMS: To estimate the budget impact of adopting nadofaragene firadenovec for the treatment of adults with high-risk, Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle-invasive bladder cancer (NMIBC) with carcinoma with or without papillary tumors (CIS ± Ta/T1) from a US third-party payer's perspective. METHODS: A budget impact model was developed to compare total healthcare costs under two scenarios: (1) with nadofaragene firadenovec included in the treatment mix, and (2) without it. The model simulated a hypothetical US health plan covering one million members over a 3-year time horizon. Costs assessed included drug acquisition and administration, surveillance and disease management, cystectomy, and treatment-related adverse events (AEs). Base-case inputs were informed by trial data, published literature, and real-world evidence. Scenario analyses explored alternative population definitions, epidemiology and clinical inputs; one-way sensitivity analyses tested parameter uncertainty. RESULTS: In the base-case, the estimated per-member-per-month (PMPM) budget impact of adopting nadofaragene firadenovec ranged from $0.0010 in Year 1 to $0.0084 in Year 3. Increased drug and administration costs were partially offset by cost savings from reduced disease progression, fewer cystectomies, and lower AE rates. Scenario analyses demonstrated that even examining a broader population including papillary disease-only patients, the budget impact remained modest ($0.0064-$0.0548 PMPM from Years 1-3). Sensitivity analyses confirmed that results were most influenced by uptake rate, drug cost, and target population size. LIMITATIONS: Key limitations include reliance on projected market shares for nadofaragene firadenovec, omission of recently approved treatments (nogapendekin alfa inbakicept-pmln and TAR-200) due to the lack of publicly available market share data, and assumptions to derive clinical outcomes for certain treatments due to lack of reported data. CONCLUSIONS: Despite its higher acquisition cost, the inclusion of nadofaragene firadenovec resulted in a minimal budget impact as bladder-sparing option for patients with BCG-unresponsive NMIBC with CIS ± Ta/T1.
Comparing deep learning and classical regression approaches for predicting healthcare expenditure and spending: a systematic review.
Lee JT, Yeh MH, Li VC … +4 more , Chen HH, Liu YH, Chen YC, Wu DB
J Med Econ · 2026 Dec · PMID 41779998 · Publisher ↗
AIMS: This study compares deep learning architectures with traditional regression and tree-based models for individual-level healthcare cost prediction, with particular attention to performance differences across data co...
AIMS: This study compares deep learning architectures with traditional regression and tree-based models for individual-level healthcare cost prediction, with particular attention to performance differences across data contexts. METHODS: We conducted a preregistered systematic review (PROSPERO CRD420251129440). Web of Science, PubMed, Embase, and Scopus were searched through August 2025. Eligible studies used real-world individual-level data (claims, electronic health records, or registries) to predict cost-related outcomes with at least one deep learning architecture and one classical regression comparator, and reported quantitative performance. Data were extracted on population, predictors, outcome horizon, model type, validation strategy, performance metrics, calibration, and interpretability. RESULTS: Eight studies met inclusion criteria, spanning the United States, Europe, and Asia. In longitudinal designs-such as multi-year claims prediction and medication or hospitalization time-series forecasting-sequential deep learning models, particularly LSTM and CNN-LSTM hybrids, consistently outperformed regression and tree-based algorithms. Reported gains included approximately 10-20% reductions in RMSE/MAE, R improvements of 0.01-0.15, and AUROC values up to 0.78 for high-risk classification. Across studies, prior costs and utilization were consistently the strongest predictors, while social determinants and free-text features were rarely incorporated. In contrast, for low-dimensional, structured, cross-sectional medical data, generalized linear models and tree-based approaches remain robust baseline models due to their interpretability and calibration stability. LIMITATIONS: Evidence is based on a small and heterogeneous set of eight studies, with limited external or temporal validation, short prediction horizons, and sparse assessment of calibration, economic interpretability, and fairness, warranting cautious interpretation. CONCLUSIONS: Deep learning offers clear gains for longitudinal, sequence-rich cost forecasting, whereas tree-based methods remain highly competitive for cross-sectional tabular prediction. Overall, these findings are consistent with the proposed Complexity-Performance Hypothesis, which posits that the predictive advantages of deep learning emerge primarily when model capacity is well matched to data complexity.
Clinical and methodological uncertainty in the economic evaluation of suzetrigine.
Alcántara Montero A
J Med Econ · 2026 Dec · PMID 41779973 · Publisher ↗
Abstract loading — click title to view on PubMed.
Reply: clinical and methodological uncertainty in the economic evaluation of suzetrigine.
Garrison K, Ellis AG, Curtice TG … +4 more , Rosen MM, Sussman M, Lee J, Lopez A
J Med Econ · 2026 Dec · PMID 41779957 · Publisher ↗
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Management of moderate-to-highly exuding leg ulcers with polyacrylate superabsorbent wound dressings versus foam dressings in spanish settings: an early-stage cost-effectiveness and budget-impact analysis.
Veličković V, Torra I Bou JE, Cegri F … +1 more , Llatas FP
J Med Econ · 2026 Dec · PMID 41776171 · Publisher ↗
AIM: This study evaluates the incremental cost‑effectiveness and budget impact of polyacrylate Superabsorbent (SAP) dressings compared with foam dressings for the management of moderate‑to‑highly exuding leg ulcers in th...
AIM: This study evaluates the incremental cost‑effectiveness and budget impact of polyacrylate Superabsorbent (SAP) dressings compared with foam dressings for the management of moderate‑to‑highly exuding leg ulcers in the Spanish healthcare setting. METHODS: We developed a weekly-cycle microsimulation state-transition model over 24 weeks from the Spanish National Health System perspective. Clinical effectiveness inputs for SAP dressings were derived from IPD from two observational clinical studies ( = 84, 2-week follow-up). Foam-dressing effectiveness was informed by a systematic review and meta-analysis of three eligible studies reporting 14-day wound-area reduction. The modelled cohort represented patients with moderate-to-highly exuding leg ulcers managed with compression therapy as standard of care; baseline severity was parameterised from the IPD (mean ulcer duration 15.1 months; baseline wound size 5,765 mm). RESULTS: This early-stage model predicts an absolute incremental improvement in healing rate of 2.41 percentage points, an absolute incremental gain of 0.135 quality‑adjusted life weeks (QALWs) over a 24‑week period, and an absolute per‑patient direct cost reduction of €729 when SAP dressings are compared with foam dressings. When these incremental results are extrapolated to a hypothetical scenario of 100% adoption, the Spanish National Health System could achieve annual cost savings of €55.557 million. These estimates should be interpreted cautiously given the early‑stage, non‑comparative nature of the underlying evidence. LIMITATIONS: Treatment‑effect estimates were derived from non‑randomized studies, which may introduce unmeasured confounding. As a result, the magnitude of the incremental effects is uncertain. To evaluate robustness, we conducted deterministic and probabilistic sensitivity analyses, both of which showed that the direction of the base‑case findings remained consistent across all plausible parameter ranges. CONCLUSIONS: Preliminary model predictions suggest that polyacrylate wound dressings may provide additional health benefits and potentially reduce costs compared with foam dressings within the Spanish Health Care System.
Annual pharmacy cost per patient achieving composite treatment endpoints: a cost to target analysis of tirzepatide versus subcutaneous semaglutide 1 mg in patients with type 2 diabetes in the UK.
Kanumilli N, Osumili B, Evans J … +6 more , Webb J, Buckingham M, Debackere N, Nowakowski P, Cattin J, Hunt B
J Med Econ · 2026 Dec · PMID 41771166 · Publisher ↗
INTRODUCTION: Tirzepatide is a treatment for type 2 diabetes associated with improvements in glycemic control and weight loss, and a low risk of hypoglycemia when not used in combination with insulin or insulin secretago...
INTRODUCTION: Tirzepatide is a treatment for type 2 diabetes associated with improvements in glycemic control and weight loss, and a low risk of hypoglycemia when not used in combination with insulin or insulin secretagogues. A cost to target analysis of tirzepatide 5, 10 and 15 mg versus subcutaneous semaglutide 1 mg in the UK setting was performed, calculating the annual pharmacy cost per patient treated to six composite endpoints combining glycemic control (glycated hemoglobin [HbA1c] ≤ 6.5% [48 mmol/mol] and <7.0% [53 mmol/mol]) with weight loss (≥5%, ≥10%, ≥15%) and avoidance of hypoglycemia. The costing analysis was based on the tirzepatide costs appraised by NICE as part of TA924. METHODS: The proportions of patients achieving composite treatment targets with tirzepatide and semaglutide (both in combination with metformin) were taken from a post-hoc analysis of the SURPASS-2 clinical trial ( = 1,845). Costs per patient treated to target were calculated by dividing the annual treatment costs associated with each intervention by the proportion of patients achieving the treatment target with each intervention. RESULTS: Tirzepatide 5, 10 and 15 mg were associated with a lower pharmacy cost per patient achieving treatment target than semaglutide 1 mg for the majority of endpoints evaluated. Differences became greater at more strict treatment targets. For example, the cost per patient achieving HbA1c ≤6.5%, weight loss ≥15%, and no hypoglycemia was GBP 5,650, GBP 8,665 and GBP 9,462 lower with tirzepatide 5, 10 and 15 mg, respectively, compared with semaglutide 1 mg over a 1-year time horizon. The only endpoint where semaglutide 1 mg was associated with a lower cost per patient achieving target was HbA1c <7.0%, weight loss ≥5%, and no hypoglycemia. CONCLUSIONS: Compared to semaglutide, tirzepatide was associated with a lower annual pharmacy cost per patient with diabetes achieving treatment target in the UK for the majority of endpoints, with greater differences at more strict treatment targets.
Clinical and healthcare burden of myotonic dystrophy type 1 (DM1) in the United States: a claims-based study.
Hamel JI, Samnaliev M, Do L … +4 more , Ito D, Pan Y, Dugar A, Novack A
J Med Econ · 2026 Dec · PMID 41764034 · Publisher ↗
OBJECTIVES: Myotonic dystrophy type 1 (DM1) is a multi-systemic disease affecting skeletal and smooth muscle, the eye, brain, and heart. No disease-modifying therapies are available. Prior studies on clinical care and ec...
OBJECTIVES: Myotonic dystrophy type 1 (DM1) is a multi-systemic disease affecting skeletal and smooth muscle, the eye, brain, and heart. No disease-modifying therapies are available. Prior studies on clinical care and economic burden combined DM1 and DM type 2. This study describes the US DM1 population aged ≥12 years, assessed organ system involvement over time, estimated healthcare resource utilization (HCRU), associated costs, and identified predictors of high-cost status. METHODS: Linked open claims and EHR data (Clarivate) from 1 January 2015 to 25 August 2023 were analyzed. Eligible individuals had a DM1 diagnosis (index), ≥6 months pre-index data, and were aged ≥12 years; congenital DM was excluded. Organ system involvement rates up to 7 years post-index were estimated using the Kaplan-Meier method. Annual HCRU rates and costs to payers (2023 USD) were summarized by setting, type of care and specialty and compared by organ involvement. Predictors of high-cost status were evaluated using multivariable logistic regression. RESULTS: The cohort included 1,343 people with DM1. Over 7 years, most experienced neurological, gastrointestinal, cardiac, pulmonary, and/or musculoskeletal system involvement. Annually post-index, approximately 24% of patients had emergency room visits, and 15% had inpatient visits. Common specialty visits included rehabilitation (13%), cardiology (13%), and neurology (11%). Frequent device use included noninvasive positive-pressure ventilation (12%), assistive devices (7%), and oxygen therapy (4%). Mean annual total cost of care was $20,687 (SD = $122,082), primarily driven by inpatient and outpatient hospital visits. Cardiac and pulmonary complications were significant predictors of high-cost status. CONCLUSION: DM1 imposes significant clinical and economic burden, marked by extensive organ involvement and variable healthcare costs. As any patient may progress to costly cardiac or pulmonary complications, early proactive management is critical. The complexity and heterogeneity of DM1 highlight ongoing unmet needs and the importance of developing effective treatments.
Enhanced patient outcomes and improved budget impact from increased PFO diagnostic testing in cryptogenic stroke workup: a US hospital perspective.
Volpi JJ, Tiozzo G, Neervoort J … +5 more , Louwsma T, Marti AK, Landaas EJ, Nakum M, Imhoff RJ
J Med Econ · 2026 Dec · PMID 41764033 · Publisher ↗
AIM: 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 patie...
AIM: 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. METHODS: 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. RESULTS: 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. CONCLUSIONS: 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.
Impact of offering blood-based testing alongside existing modalities for colorectal cancer screening among those who previously declined screening: an economic evaluation.
Forbes SP, Yay Donderici E, Zhang N … +3 more , Raymond VM, Das AK, Liang PS
J Med Econ · 2026 Dec · PMID 41764032 · Publisher ↗
AIM: Inadequate adherence to colorectal cancer screening reduces individual and population level health benefits. Blood-based tests offer a new modality that may help patients overcome barriers, but there are concerns ab...
AIM: Inadequate adherence to colorectal cancer screening reduces individual and population level health benefits. Blood-based tests offer a new modality that may help patients overcome barriers, but there are concerns about the impact of patients switching from existing guideline-recommended screening modalities. This study estimates the population health outcomes and cost-effectiveness of offering blood-based testing using a validated individual-level simulation model based on patient preference evidence from randomized controlled trials. MATERIALS AND METHODS: In this study, a validated discrete-event simulation model was used to evaluate the performance of different combinations of colorectal cancer screening strategy preferences per 10,000 screened individuals beginning at age 45. Preferences for screening options were informed by randomized controlled trials of patients with and without the option of blood-based testing. Adherence to initial patient preferences over a simulated lifetime was modeled as: (1) assumed 100% adherence and (2) longitudinal using a calibrated model. Simulated outcomes included clinical outcomes and cost-effectiveness from a healthcare sector perspective. A strategy was deemed cost-effective at a willingness-to-pay threshold of $100,000 per quality-adjusted life-year gained. RESULTS: The introduction of blood-based testing to an unscreened population with evidence from randomized controlled trials is projected to increase colorectal cancer deaths averted by 35% to 116% and from 68% to 247% relative to no screening, for stated preference and revealed preference scenarios, respectively. These outcomes are cost-effective, with incremental cost-effectiveness ratios ranging from $63,994 to $85,497 and from $30,464 to $54,764 across stated preference and revealed preference scenarios, respectively. LIMITATIONS: Given limited data, natural history and real-world longitudinal adherence to screening are based on evidence-informed assumptions. CONCLUSIONS: Using a simulation model to extrapolate data from two recent trials, we demonstrate that the introduction of blood-based tests has the potential to lead to cost-effective increases in the number of CRC deaths averted among the unscreened population.
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