Journal of Health Economics and Outcomes Research最新文献

Background: Patients with advanced or recurrent endometrial cancer (EC) have limited treatment options following platinum-based chemotherapy and poor prognosis. The single-arm, Phase I GARNET trial (NCT02715284) previously reported dostarlimab efficacy in mismatch repair-deficient/microsatellite instability-high advanced or recurrent EC. Objectives: The objective of this study was to compare overall survival (OS) and describe time to treatment discontinuation (TTD) for dostarlimab (GARNET Cohort A1 safety population) with an equivalent real-world external control arm receiving non-anti-programmed death (PD)-1/PD-ligand (L)1/2 treatments (constructed using data from a nationwide electronic health record-derived de-identified database and applied GARNET eligibility criteria). Methods: Propensity scores constructed from prognostic factors, identified by literature review and clinical experts, were used for inverse probability of treatment weighting (IPTW). Kaplan-Meier curves were constructed and OS/TTD was estimated (Cox regression model was used to estimate the OS-adjusted hazard ratio). Results: Dostarlimab was associated with a 52% lower risk of death vs real-world treatments (hazard ratio, 0.48; 95% confidence interval [CI], 0.35-0.66). IPTW-adjusted median OS for dostarlimab (N=143) was not estimable (95% CI, 19.4-not estimable) versus 13.1 months (95% CI, 8.3-15.9) for real-world treatments (N = 185). Median TTD was 11.7 months (95% CI, 6.0-38.7) for dostarlimab and 5.3 months (95% CI, 4.1-6.0) for the real-world cohort. Discussion: Consistent with previous analyses, patients treated with dostarlimab had significantly longer OS than patients in the US real-world cohort after adjusting for the lack of randomization using stabilized IPTW. Additionally, patients had a long TTD when treated with dostarlimab, suggesting a favorable tolerability profile. Conclusion: Patients with advanced or recurrent EC receiving dostarlimab in GARNET had significantly lower risk of death than those receiving real-world non-anti-PD-(L)1/2 treatments.

Background: Closed claims are frequently used in outcomes research studies. Lately, the availability of open claims has increased the possibility of obtaining information faster and on a larger scale. However, because of the possibility of missing claims and duplications, these data sets have not been highly utilized in medical research. Objective: To compare frequently used healthcare utilization measures between closed claims and open claims to analyze if the possibility of missing claims in open claims data creates a downward bias in the estimates. Methods: We identified 18 different diseases using 2022 data from 2 closed claims data sets (MarketScan® and PharMetrics® Plus) and 1 open claims database (Kythera). After applying an algorithm that removes possible duplications from open claims data, we compared healthcare utilizations such as inpatient, emergency department, and outpatient use and length of stay among these 3 data sets. We applied standardized differences to compare the medians for each outcome. Results: The sample size of the open claims data sets was 10 to 65 times larger than closed claims data sets depending on disease type. For each disease, the estimates of healthcare utilization were similar between the open claims and closed claims data. The difference was statistically insignificant. Conclusions: Open claims data with a bigger sample size and more current available information provide essential advantages for healthcare outcomes research studies. Therefore, especially for new medications and rare diseases, open claims data can provide information much earlier than closed claims, which usually have a time lag of 6 to 8 months.

Background: Compression therapy is the gold standard for the treatment of chronic venous insufficiency (CVI). Two-layer bandage (2LB) systems have been shown to be a safe and effective treatment option. Objective: To estimate the total cost per response (CPR) for the resolution of edema and wounds in patients with CVI treated with a 2LB system as part of their overall wound healing regimen. Methods: A probabilistic decision tree model was developed to estimate the incremental CPR for a 2LB system. The model simulated 10 000 patients to estimate the CPR for the resolution of edema and wound healing. The analysis was performed using clinical data from a published single-arm, multicenter prospective study of CVI indicated for compression therapy. The response outcomes of interest were resolution of edema and rate of wound healing. The follow-up time was a maximum of 6 weeks, and the perspective of the study was a US outpatient treatment center. Economic data for compression therapy were based on the public prices of a 2LB system. Dressing changes occurred per manufacturer instructions for use. Results: The study comprised 702 patients (56% female), with a total of 414 wounds. The median duration of the wounds was 42 days, and the median size at the initial visit was 3.5 cm2. The average pain reduction fell by 67% using a visual analog score. Bandages were typically changed once or twice a week (51.7%). Wound healing occurred in 128 of the 414 wounds (30.9%). The expected incremental CPR of a 2LB system for the resolution of edema was $65.67 (range, $16.67-$124.32). The expected incremental CPR of a 2LB system for the healing of a wound was $138.71 (range, $35.71-$273.53). Conclusion: This economic evaluation complements previous clinical effectiveness and safety studies of 2LB systems for the treatment of CVI. The results demonstrate that the costs of incorporating 2LB into standard wound-healing protocols are negligible compared with overall treatment costs. Two-layer bandages may be considered a cost-effective first-line system for the treatment of wounds caused by CVI.

Background: Systemic lupus erythematosus (SLE) is a chronic autoimmune disease that can lead to irreversible organ damage (OD). Data describing the patient burden of OD, as compared with SLE without OD, are limited. Objective: To develop a comprehensive conceptual model describing the burden experienced by patients living with SLE-associated OD. Methods: There were three phases to this qualitative study. First, a targeted literature review was conducted to inform a draft conceptual model. Second, key opinion leaders (KOLs) were interviewed to assess the draft conceptual model and help shape patient interview materials. Third, patients of different demographic backgrounds from across the United States were interviewed individually to gather their perspectives on living with SLE-associated OD. Data from concept elicitation interviews with KOLs and patients were coded and analyzed using NVivo software to identify the key concepts of the overall patient burden of SLE-associated OD. Findings from the KOL and patient interviews were used to finalize the conceptual model. Results: KOLs highlighted that SLE-associated OD carried a higher rate of mortality than SLE alone. Participants with SLE-associated OD (n = 40) experienced detrimental impacts across 4 areas of their lives: physical, cognitive, psychosocial functioning, and economic and work-related well-being. Physical impacts were described by all participants, often affecting their ability to perform everyday tasks. Many also described deterioration of cognitive functioning. Almost all participants experienced emotional impacts and challenges to their relationships and social lives resulting from living with SLE-associated OD. Additionally, SLE-associated OD imposed an economic burden including increased healthcare costs. SLE-associated OD had a more severe and debilitating impact on all aspects of the patient's quality of life than SLE prior to OD development, including further limitations in activities of daily living after the development of OD. Discussion: Study findings guided the development of a comprehensive conceptual model that fully represents the patient experience of living with SLE-associated OD, highlighting the additional burden of OD when compared with SLE alone. Conclusions: The conceptual model will inform improvements in disease management, which may result in better patient outcomes and aid development of clinical outcome assessments of disease burden.

Background: Schizophrenia is a serious mental disorder that has greater negative consequences on role functioning than many other severe chronic diseases. Objective: We evaluated the economic impact of long-acting injections of paliperidone palmitate (PP) vs daily oral antipsychotics to treat chronic schizophrenia from a societal perspective over a 2-year period. Methods: A static budget impact model was developed to compare PP with daily oral antipsychotics (risperidone, olanzapine, and aripiprazole) in the treatment of patients with chronic schizophrenia. Our study included treatments used during relapse and hospitalization, validated by an expert panel. The clinical parameters were extracted from the PRIDE trial. Direct medical costs and indirect costs were measured. The unit cost of drug acquisition for all medications was extracted from the public sector. One-way sensitivity analyses were conducted. Results: The target population in our model was estimated to be 142 incident patients. In the first year, the total drug costs in Egyptian pounds (EGP) for PP and oral antipsychotics were £2.7 million and £724 004, respectively, while the total medical costs for PP and oral antipsychotics were £3 million and £5.6 million, respectively. In the second year, the total drug costs for PP and oral antipsychotics were £2.7 million and £724 004, respectively, while the total medical costs for PP and oral antipsychotics were £3 million and £5 million, respectively. The total costs for PP (£11.6 million) over 2 years were less than those of oral antipsychotics without PP (£12.7 million). PP produced an estimated budget savings of £1 046 561 (budget savings per patient per year, £3667). In addition, PP resulted in the avoidance of 18 hospitalizations per year compared with the without-PP arm. Sensitivity analyses showed that the percent of hospitalizations for both oral antipsychotics and PP had the greatest impact on the results. Conclusion: The lower hospitalization rates associated with PP offset the increase in drug costs. PP may potentially be cost-saving compared with the standard of care in chronic schizophrenia in Egyptian representative healthcare settings. Policy makers may consider this approach to improve patient outcomes and budget sustainability.