Background External beam radiation therapy (EBRT) is an effective treatment option for patients with localized prostate cancer (LPC). Despite the widespread use of EBRT, evaluation of outcomes based on LPC risk status is limited. Objectives To comprehensively compare clinical and economic outcomes between patients with high-risk vs low/intermediate-risk LPC following EBRT in real-world United States (US) clinical practice. Methods This retrospective study used linked patient-level electronic medical record data from PPS Analytics and administrative claims data from the Komodo Research Database (1 January 2016–31 August 2024). Patients with LPC treated with EBRT (date of the first claim was defined as the index date) were identified and stratified into high-risk and low/intermediate-risk cohorts based on clinical guidelines incorporating information based on localized cancer staging, Gleason score, and prostate-specific antigen level. Baseline characteristics were balanced using inverse probability of treatment weighting. A weighted Cox proportional hazards model was used to compare metastasis-free survival (MFS) and event-free survival (EFS) between cohorts. Weighted ordinary least squares regression was used to compare all-cause and PC-related healthcare costs (2024 US dollars), measured per-patient-per-year (PPPY), in the subgroup of patients with continuous insurance eligibility. Results In total, 5984 and 10 471 patients were included in the high-risk and low/intermediate-risk LPC cohorts, respectively. Patients with low/intermediate-risk vs high-risk LPC had significantly longer MFS (36-month hazard ratio [HR]; 95% confidence interval [CI]: 2.47 [2.15, 2.84], P < .001; 60-month HR: 2.29 [2.05, 2.55], P < .001) and EFS (36-month HR: 1.84 [1.65, 2.06], P < .001; 60-month HR: 1.90 [1.73, 2.08], P < .001). Among the subgroup of patients with continuous insurance eligibility (high-risk: 980 patients; low/intermediate-risk: 1862 patients), patients with high-risk LPC incurred significantly higher mean total all-cause healthcare costs (cost difference [95% CI]: $8269 PPPY [3869, 12 800], P < .001) and mean total PC-related healthcare costs ($9053 PPPY [5232, 13 147], P < .001) post-index compared with those with low/intermediate-risk LPC. Conclusion Patients with high-risk vs low/intermediate-risk LPC had significantly worse survival outcomes and incurred significantly higher costs following EBRT, consistent with the greater disease burden associated with high-risk disease. More effective treatment strategies are needed to further improve clinical outcomes and reduce economic burden among this patient population.
Background Despite the availability of several androgen receptor pathway inhibitors for the treatment of patients with metastatic castration-sensitive prostate cancer (mCSPC), there has been a lack of head-to-head randomized clinical trials to evaluate their comparative efficacy in this population. Objective This real-world study provides a comprehensive and robust comparison of the decline in prostate-specific antigen ≥90% (PSA90) among patients with mCSPC who initiated apalutamide or abiraterone acetate in the United States. Methods Clinical data were collected from US community urology practices by Precision Point Specialty Analytics and linked with administrative claims from the Komodo Health Solutions Research Database. Patients with mCSPC were included in apalutamide or abiraterone acetate cohorts based on their first dispensation/paid claim on or after September 17, 2019 (index date), and followed to the earliest of treatment discontinuation or switch, end of insurance/clinical activity, or end of data availability (Dec. 31, 2023). Inverse probability of treatment weighting balanced potentially confounding patient characteristics. PSA90 response by 6 months post index was compared between the cohorts using weighted Kaplan-Meier analysis and time to PSA90 response was compared using a weighted Cox model with a time-varying treatment effect. Results The study included 1351 patients in the apalutamide cohort and 1003 patients in the abiraterone acetate cohort. By 6 months, 61.4% of the weighted apalutamide cohort and 45.9% of the weighted abiraterone acetate cohort achieved a PSA90 response. Compared with abiraterone acetate, patients treated with apalutamide were 73% more likely to achieve a PSA90 response at 6 months (weighted hazard ratio, 1.73; 95% confidence interval, 1.44-2.08; P < .001). The median time to PSA90 response was 3.8 and 8.7 months in the apalutamide and abiraterone acetate cohorts, respectively. Conclusions The results of this study confirm that patients with mCSPC treated with apalutamide have significantly higher rates of PSA90 response compared with abiraterone acetate after 6 months of treatment. Real-world evidence highlights apalutamide as an important treatment option to improve the prognosis of patients with mCSPC.
Background Primary biliary cholangitis (PBC) is a chronic autoimmune liver disease in which long-term clinical outcomes are difficult to assess within conventional trial durations. Alkaline phosphatase (ALP) and bilirubin are established biochemical endpoints in regulatory and clinical practice, and their relationship with long-term clinical outcomes has been demonstrated through a robust literature of analyses across different datasets. However, the magnitude and consistency of their association with long-term clinical outcomes have not been systematically quantified through meta-analysis. Objective This systematic literature review and meta-analysis aimed to identify, evaluate, and summarize evidence on the correlation between surrogate biochemical endpoints and long-term clinical outcomes in patients with PBC. Methods A comprehensive search of key biomedical databases and reporting was conducted, in accordance with Cochrane, NICE and PRISMA guidelines. Meta-analysis was performed using Stata 19.5 SE software, with the DerSimonian and Laird (DL) random-effects model employed in the base-case, inverse-variance fixed-effect and DL + Hartung-Knapp-Sidik-Jonkman random effects model were used in sensitivity analyses. Other sensitivity analyses were performed based on study/follow-up duration, study quality, publication type, and removal of heterogeneous studies. Results Overall, 129 studies were included in the systematic literature review, among which 89 studies met criteria for meta-analysis. Failure to achieve ALP normalization was associated with significantly increased risks of composite clinical outcomes (HR; 95% CI: 1.46; 1.26-1.69), liver-related events (1.82; 1.41-2.34), and overall death and/or liver transplantation (1.52; 1.30-1.79). Similar findings were reported for other ALP response definitions. Nonresponse to composite criteria was also associated with significantly worse long-term outcomes. Sensitivity analyses yielded directionally consistent results. Discussion To our knowledge, this research represents the first comprehensive meta-analytic evaluation integrating multiple surrogate biochemical endpoints with a broad spectrum of long-term clinical outcomes in PBC across a global study population. Associations were directionally consistent across ALP-, bilirubin-, and composite-based definitions and across all analyses. Conclusion This meta-analysis provides robust evidence that ALP normalization, ALP-based response criteria, and composite biochemical response measures (comprising ALP and total bilirubin), were consistently associated with long-term clinical outcomes in PBC and may inform HTA, reimbursement, payer decision-making, and economic evaluation when mature long-term outcome data are unavailable.
# Background TAPS® therapy (Cala Health) delivers transcutaneous afferent patterned stimulation through a noninvasive, wrist-worn neuromodulation device for the treatment of hand tremor in patients with essential tremor (ET). Although previous studies have demonstrated its effectiveness and safety, real-world evidence regarding its long-term clinical outcomes and economic impact remains limited. # Objectives To evaluate the long-term clinical effectiveness and the healthcare resource utilization and cost impact of TAPS in patients with ET treated in real-world settings. # Methods Patients who completed a 1-month randomized pragmatic phase comparing TAPS with standard of care entered 12 months of open-label home use. Clinical outcomes were assessed via device-based motion sensors and the Bain & Findley Activities of Daily Living (BF-ADL) upper-limb scale at baseline and every 3 months. Healthcare resource utilization and costs over the 12-month TAPS treatment period were compared with those during the 12 months before TAPS initiation and with a propensity-score-matched external control arm (ECA) derived from a large US commercial and Medicare Advantage claims database. # Results Among 225 patients who used TAPS therapy for 12 months, daily functioning improved, with the mean BF-ADL score decreasing from 17.2 to 15.8 (_P_ < .003). Device-measured tremor power decreased by more than 50% (_P_ < .001). No serious device-related adverse events were reported. In the ECA analysis, 153 TAPS-treated patients were matched to 306 ECA patients. Compared with baseline, TAPS-treated patients experienced significant reductions in ET-related healthcare costs ($440 decrease; _P_ < .0001), ET-related medication use (15% fewer patients; _P_ < .01), and ambulatory and office visits (~0.3 fewer each; _P_ < .05). These reductions were significantly greater than those observed in the ECA (all _P_ < .05). # Discussion This is the largest and longest prospective ET study to jointly assess tremor severity, daily function, and healthcare economic outcomes. The sustained clinical benefits observed with TAPS therapy, together with reductions in ET-related healthcare utilization and costs, support TAPS as a noninvasive treatment option for adults with ET. # Conclusions Over 12 months of real-world use, TAPS reduced tremor severity, improved daily functioning, and lowered ET-related healthcare resource utilization and costs.
# Background Evidence on the economic burden of post-COVID syndrome in Germany remains limited. This study quantifies post-COVID-associated healthcare resource utilization (HCRU), sick leave, and costs. # Objectives To compare HCRU, sick leave, and costs borne by the statutory health insurance (SHI) among incident pediatric and adult post-COVID patients with matched post-COVID-free controls. # Methods This retrospective cohort study analyzed an anonymized German SHI claims database provided by GWQ ServicePlus AG. The observation period spanned October 2021 to June 2025 and included all individuals with incident COVID-19 diagnosis until September 2024. Incident post-COVID was defined as the first ICD-10-GM code (U07.4!; U09.9!) recorded ≥90 days after first COVID-19 diagnosis. Post-COVID cases were exactly matched 1:2 to post-COVID–free COVID-19 controls by calendar quarter of incident COVID-19 diagnosis, acute severity, cohort entry quarter, 5-year birth interval, sex, and Elixhauser Comorbidity Index. We estimated mean differences in HCRU, sick leave, and direct SHI-borne costs per case. Rate ratios of HCRU, sick leave, and costs per person-year were estimated using Poisson (HCRU/sick leave) and linear (costs) regression. Analyses covered 365-day, 730-day, and any follow-up period. # Results We matched 25 485 identified post-COVID cases with 49 671 controls. Matched cases and controls were comparable in age, sex, and comorbidities. Overall, during 365 days of follow-up, post-COVID patients showed higher HCRU and sick leave rates across all sectors, especially sick leave days. Mean differences per case were greatest for sick leave days (365 days: +27 days) and outpatient physician contacts (365 days: 6.9 contacts). HCRU and sick leave rates remained significantly increased over extended follow-up periods. Total SHI-borne costs per person-year were higher among post-COVID patients across all follow-up periods (all _P_ < .001). Sector-specific costs per person-year were increased primarily in the outpatient sector and for sickness benefits (all _P_ < .001). Mean total cost differences per case were +€1806 per case (365 days) and mean excess sickness benefits per case were +€1077. # Conclusions Documented post-COVID was associated with a substantial and sustained economic burden on the German healthcare system and society. Targeted strategies are needed to mitigate potential long-term impacts on HCRU and labor productivity.
