Journal of Health Economics and Outcomes Research最新文献

[This corrects the article DOI: 10.36469/jheor.2026.154974.].

Background: Infections represent the most serious complication associated with cardiac implantable electronic devices (CIEDs). This can result in prolonged hospital stays, high morbidity and mortality, and a significant economic burden for healthcare systems.

Objectives: This study aimed to evaluate the cost-effectiveness of the TYRX absorbable antibacterial envelope for CIED infection prevention from the Spanish Healthcare System perspective.

Methods: A decision tree model with a lifetime horizon was developed to compare standard antibiotic prophylaxis with its combination with TYRX, regardless of infection risk. The model incorporated infection incidence, mortality, and utility values up to 36 months, derived from REINFORCE, AdaptResponse, and WRAP-IT studies. Unit costs (2025 euros) included prevention strategies and infection management. Lifetime costs and quality-adjusted life-years (QALYs) were assigned to survivors beyond 36 months. The incremental cost-effectiveness ratio (ICER) was reported by CIED and weighted by implant distribution (permanent pacemaker [PPM, 76.5%], implantable cardioverter-defibrillator [ICD, 15.2%], cardiac resynchronization therapy with defibrillator [CRT-D, 5.4%], and pacemaker [CRT-P, 2.9%]). A subgroup analysis was performed in high-risk patients (PADIT≥7), modifying infection rates based on PADIT risk stratification, along with sensitivity analyses. Model inputs were validated by an expert panel.

Results: TYRX was the dominant strategy (more effective and less costly) for CRT-D and ICD recipients and cost-effective for those receiving PPM (€17 740/QALY) or CRT-P (€14 647/QALY), considering a willingness-to-pay threshold of €25 000/QALY. Across the spectrum of CIEDs, the ICER was €11 709/QALY. TYRX remained cost-effective in 77% of sensitivity analysis simulations. In high-risk patients, TYRX was dominant for all CIEDs.

Discussion: This study is believed to be the first economic evaluation of TYRX in Spain and provides novel evidence in a broad, unselected population. Previous cost-effectiveness analyses conducted across different healthcare systems have consistently shown that TYRX is cost-effective in patients at elevated risk for device-related infections. Although the populations and healthcare settings differ, our findings are consistent with this body of evidence.

Conclusions: TYRX represents a dominant strategy for infection prevention for CRT-D and ICD and is cost-effective for PPM and CRT-P, based on Spain's willingness to pay.

Background: Pimavanserin is currently the only atypical antipsychotic (AAP) approved by the US Food and Drug Administration for treating hallucinations and delusions in Parkinson's disease psychosis (PDP). Benefit and efficacy of pimavanserin have been demonstrated through clinical trials; however, understanding other real-world outcomes is needed.

Objective: This study evaluated healthcare resource utilization (HCRU) patterns before and after pimavanserin initiation to assess benefit and effectiveness of pimavanserin that may be seen in the real-world setting but not in clinical trials.

Methods: A retrospective pre-post analysis using 100% Medicare claims data (Parts A, B, and D), April 1, 2015-December 31, 2021, was conducted. The study included AAP treatment-naïve PDP patients who initiated continuous pimavanserin monotherapy (index date) within 6 months of their incident PDP diagnosis, April 1, 2016-December 31, 2020 (ie, patient identification period). Outcomes measured during the 6 months before and after pimavanserin initiation included all-cause and psychiatric-related HCRU; further categorized as inpatient, emergency room (ER), outpatient, and office visits. Significant differences in the percentage of patients with at least 1 all-cause and at least 1 psychiatric-related HCRU were evaluated using McNemar's tests at P < .05.

Results: Of the 694 patients newly diagnosed with PDP who initiated pimavanserin within 6 months of PDP diagnosis, mean age was 76.9 (±6.8) years, 54.8% were male, and 78.3% had concomitant dementia. The percentage of patients with at least 1 all-cause HCRU was significantly higher during the 6 months pre-index vs post-index, with inpatient (26.1% vs 20.5%, P < .05), ER (51.3% vs 35.2%, P < .05), outpatient (83.0% vs 79.3%, P < .05), and office visits (97.4% vs 95.8%, P < .05). Similarly, the percentage of patients with at least 1 psychiatric-related HCRU was significantly higher 6 months pre-index vs post-index, with inpatient (7.8% vs 4.9%, P < .05), ER (9.7% vs 3.5%, P < .05), outpatient (23.6% vs 13.0%, P < .05), and office visits (68.7% vs 55.8%, P < .05).

Conclusions: Newly diagnosed PDP patients initiating pimavanserin within 6 months demonstrated significant reductions in 6-month post-index all-cause and psychiatric-related HCRU. Further analysis examining the association between time of pimavanserin initiation and the magnitude of benefits are warranted.

Background: Improved clinical outcomes have been observed in patients with metastatic castration-sensitive prostate cancer (mCSPC) who experience deep prostate-specific antigen (PSA) responses after treatment with androgen receptor pathway inhibitors (ARPIs).

Objective: This retrospective longitudinal study aimed to compare real-world PSA90 response (≥90% reduction from pretreatment levels) in Black patients with mCSPC treated with apalutamide vs abiraterone acetate.

Methods: Electronic medical record (EMR) data from Precision Point Specialty Analytics were linked to claims data from the Komodo Research Database. Black adult patients with mCSPC who initiated apalutamide or abiraterone acetate on or after 9/17/2019 were included. Inverse probability of treatment weighting was used to balance patient characteristics between treatment cohorts. PSA90 was evaluated during the on-treatment period, defined as the time from index date to the earliest of treatment discontinuation, initiation of a new ARPI or radiopharmaceutical, or end of insurance claims activity or clinical activity. Weighted Kaplan-Meier curves and hazard ratios (HRs) were used to compare PSA90 responses between treatment cohorts.

Results: This study included 363 patients, of which 236 initiated apalutamide and 127 initiated abiraterone acetate. At 6 months following treatment initiation, a greater proportion of patients treated with apalutamide (65.4%) vs abiraterone acetate (49.0%) achieved a PSA90 response. Patients who initiated apalutamide vs abiraterone acetate were 66% more likely to achieve a PSA90 response within 6 months of treatment initiation (HR, 1.66; 95% confidence interval, 1.18-2.35; P = .004). The median time-to-PSA90 response was approximately 6 months earlier for patients treated with apalutamide (3.3 months) compared with abiraterone acetate (9.1 months).

Discussion: This study leveraged robust information from combined insurance claims and routinely collected EMR data to evaluate PSA90, a clinically relevant biomarker of treatment response, among Black patients with mCSPC. These results are among the first in this understudied patient population and suggest that a deeper and earlier PSA response achieved with apalutamide relative to abiraterone acetate can extend to Black patients in a real-world US clinical setting.

Conclusion: Black patients treated with apalutamide experienced significantly higher PSA90 response rates than those treated with abiraterone acetate, suggesting possible clinical benefits from early treatment response in this population.

Background: Alopecia areata (AA) is a chronic autoimmune condition characterized by non-scarring hair loss, with significant psychological, social, and economic implications. In Saudi Arabia, AA prevalence ranges from 2.3% to 13.8%, with early onset and strong familial predisposition. Despite its burden, data on the economic impact of AA in the region remain limited.

Objectives: This study assessed the economic burden of AA in the Kingdom of Saudi Arabia from both public payer and societal perspectives, across varying disease severities including mild to moderate, severe, and refractory cases.

Methods: A prevalence-based cost-of-illness model was developed using structured literature review, expert input, and primary data collection via questionnaires. The model estimated direct medical costs (drug acquisition, diagnostics, clinic visits), direct nonmedical costs (travel, accommodation), and indirect costs (productivity loss) over 1 year for mild to severe AA and 2 years for refractory cases. Cost data were sourced from official channels (National Unified Procurement Company and the Saudi Food and Drug Authority) and validated using Saudi Amazon and Al-Dawaa pharmacy platforms. Medical costs, including laboratory tests, diagnostic procedures, and all supportive therapies, were obtained from the Ministry of Health.

Results: The average annual per-patient cost was SAR 20 703 for mild to moderate AA and SAR 76 957 for severe AA, translating into SAR 5.827 billion and SAR 3.352 billion total burden, respectively. Refractory AA cases incurred cumulative 2-year costs ranging from SAR 102 117 to SAR 167 615 per patient. Indirect costs, primarily due to productivity loss, were the dominant cost driver in mild to moderate AA and remained substantial across all severities.

Discussion: Indirect costs, mainly productivity loss, drive the economic burden of AA in Saudi Arabia, with drug costs rising in severe cases. This pattern mirrors findings from global studies.

Conclusions: AA imposes a significant financial burden in Saudi Arabia, driven largely by productivity losses and drug acquisition costs. These findings underscore the need for early diagnosis, standardized treatment protocols, and improved access to innovative therapies. Integrated care pathways and national registries are essential to optimize resource allocation and improve patient outcomes.

Background: Prostate cancer is one of the most common malignancies in men, and radiation therapy, most often stereotactic body radiation therapy or conventionally fractionated radiotherapy, is a standard curative treatment for advanced cases. Although genitourinary toxicity is a known side effect, with acute symptoms typically resolving and late toxicity causing lasting harm, individual risk varies substantially. However, no validated tool exists to predict patient-specific toxicity across radiation modalities, leaving treatment decisions to be made without personalized risk insights.

Objective: The goal of this analysis was to estimate changes in cost and quality of life associated with reductions in late genitourinary toxicity attributable to personalized radiation therapy in individuals with prostate cancer. Implementation of PROSTOX ultra, a novel microRNA-based assay for toxicity risk assessment with stereotactic body radiation therapy, is expected to enable the personalization of radiation therapy, helping patients avoid toxic effects from specific types of radiation therapy.

Methods: This study utilizes a hybrid decision-tree and Markov model approach to estimate the 5-year cost-impact and lifetime cost-effectiveness in prostate cancer patients of PROSTOX ultra vs current treatment standards that do not include toxicity risk assessment. High or low risk assessment influences whether patients receive stereotactic body radiation therapy, conventionally fractionated radiotherapy, or prostatectomy, impacting toxicity-related treatment costs and quality-of-life decrements.

Results: Over 5 years, PROSTOX ultra patients totaled $47\hspace{0.17em}683incostsvs$ 67 298 with standard-care risk assessment. Cumulative savings over 5 years were $19 615 per tested patient. Over a lifetime, PROSTOX ultra was expected to save about $24 777 per tested patient while adding 0.24 quality-adjusted life-years compared with patients tested using standard assessment.

Conclusions: Model results indicate that toxicity risk assessment with PROSTOX ultra would be cost saving over 5 years compared with standard risk assessment. Cost-effectiveness results show cost savings and quality-adjusted life-year gains over a lifetime, indicating that PROSTOX ultra would be a dominant strategy compared with treatment without risk assessment.

Background: Atrial fibrillation (AF) affects 2% to 4.5% of the population. Catheter ablation, a key strategy for paroxysmal AF management, can be achieved through radiofrequency (RFA), cryoablation (CBA), or pulsed field ablation (PFA). While clinical outcomes are well studied, their environmental impact remains underexplored.

Objectives: This study modeled the environmental impact of CBA and PFA ablation techniques in Europe, aiming to provide evidence to guide sustainable practices in AF treatment.

Methods: An early environmental analysis compared pentaspline PFA (Farapulse system, Boston Scientific) with CBA using a decision-analytic model. The model simulated the patient care pathway from a hospital perspective over a 1-year time horizon, considering index and redo procedures, and complications. The environmental impact, linked to resource use, was measured in kilograms of CO₂ equivalents (kg CO_2eq), incorporating length of stay, intervention time, anesthetic use, and complications. Probabilistic and scenario analyses, including a comparison with RFA, were performed to assess uncertainty and robustness of the results.

Results: The environmental analysis showed that PFA resulted in total emissions of 13 899 kg CO_2eq, compared with 16 383 kg CO_2eq for CBA (-2483 kg CO_2eq, -15.2%) per 100 patients. Monte Carlo simulation results confirmed these findings, showing median savings of 2409 kg CO_2eq (95% credible interval: 581-4312 kg). Parameters, such as anesthesia time and anesthetic drug use, were key drivers of the results. In the RFA scenario analysis, PFA yielded a saving potential of -4640 kg (-25%). In Germany, for example, with approximately 24 000 CBA procedures annually, PFA adoption was projected to reduce emissions by 509 723 kg CO_2eq.

Discussion: PFA showed potential for reducing emissions by approximately 25 kg CO_2eq per patient compared with CBA, driven by lower resource use. These findings aligned with studies identifying operating rooms and anesthetic drug use as major contributors to hospital emissions. A study limitation was the lack of data on catheter manufacturing and disposal.

Conclusions: PFA was expected to reduce emissions compared with CBA in AF patients. Conscious medical device choices can foster more sustainable hospital practices. A full life-cycle analysis of catheters is needed to validate these findings.

Background: Since 2019, Japan has implemented health technology assessments (HTAs) for selected drugs and medical devices. In the HTA system, the incremental cost-effectiveness ratio (ICER), calculated using quality-adjusted life-years (QALYs), is employed to guide price adjustments. However, the current system does not incorporate a quantitative assessment of disease severity.

Objectives: This study aimed to evaluate whether severity modifiers based on QALY shortfalls correspond to conditions currently granted special consideration, that is, those eligible for a higher ICER reference value (1.5× the standard), and to explore their implications for Japan's HTA system.

Methods: We retrospectively analyzed 32 drugs assessed under Japan's HTA up to March 2025. Absolute shortfall (AS) and proportional shortfall (PS) were calculated using age, sex distribution, and comparator quality-adjusted life expectancy estimates from manufacturer assessments and public assessments. Severity categories were defined as ×1.0 (AS ≤ ×12 or PS ≤0.85), ×1.2 (12 < AS < 18 or 0.85 < PS < 0.95), and ×1.7 (AS ≥18 or PS ≥0.95). The concordance between severity classification and policy-based special consideration was then examined.

Results: Twenty-five matched target populations were identified. Mean AS and PS values did not differ significantly between manufacturer and public assessments, although manufacturers tended to report higher shortfalls. All cancer and pediatric cases were classified as ×1.2 or ×1.7, whereas 1 designated intractable disease was classified as having low severity (×1.0). Chronic and infectious diseases fell into higher severity categories despite not currently being subject to special consideration. Weighted mean severity values were comparable to those used in the UK's National Institute for Health and Care Excellence benchmarks.

Discussion: The findings revealed both alignment and misalignment between Japan's current HTA policy and severity classification. While cancer and pediatric diseases were consistent with the existing system, some serious diseases might have been overlooked, and the designated intractable disease might not align with quantitative severity criteria.

Conclusions: QALY shortfalls may serve as a complementary approach to identifying unmet health needs within Japan's HTA system. To ensure methodological robustness and social acceptance, broader validation, standardized estimation methods, and stakeholder consensus are necessary for effective decision-making.

Background: Lyme disease, the most common vector-borne disease in Minnesota, is estimated to be underreported by a factor of 10. Delayed diagnosis and misdiagnosis may lead to health complications and increased personal and societal costs. Environmental factors can help to predict high disease years, allowing for early intervention to decrease disease burden. Objective: To estimate the health and cost burdens of Lyme disease and the extent to which they could be diminished by public health intervention when high-incidence Lyme disease years are forecasted. Methods: We used 5 two-dimensional Monte Carlo simulations to estimate (1) average annual expected burden of Lyme disease, (2 and 3) average burden in low- and high-incidence years, and (4 and 5) the expected burden saved with public health educational interventions preceding high-incidence years. We employed cases reported to the Minnesota Department of Health adjusted for estimates of underreporting found in the literature. Results: Among an average of 8436 Lyme disease cases annually, 6074 of them were unidentified. High-incidence years saw over 3700 more cases than low-incidence years, with incremental costs to patients and society exceeding $3million.Weestimatedthatpublichealtheducationbeforehigh-incidenceyearscouldreduceLymediseasecasesby390to787annually,savingupto$ 1.9 million in societal costs. Discussion: The simulations presented revealed substantial health and cost burden from Lyme disease, including hidden impacts from undiagnosed and unreported cases. Burden varied widely between high- and low-incidence years, highlighting the need to prioritize prevention when peak years are predicted. While we estimated the effects of individual prev