Journal of Health Economics and Outcomes Research最新文献

Background: Proteinuria, a critical marker of glomerulosclerosis, poses a challenge in idiopathic membranous nephropathy (iMN), particularly when standard treatments fail. Acthar® Gel, a US Food and Drug Administration-approved treatment option, may offer an alternative for managing refractory proteinuria in nephrotic syndrome (NS) due to iMN where multiple treatments have failed. Objective: The cost per response of Acthar® Gel vs standard of care (SoC; cyclophosphamide or rituximab) for treatment of proteinuria in NS due to iMN was evaluated among adults who had failed multiple treatments from a US payer perspective over a 1- to 3-year horizon. Methods: A probabilistic, cohort-level state-transition model simulated patient progression through various health states using 6-month cycles. Patients began in a relapse phase and received either Acthar® Gel or SoC. Transition probabilities determined whether patients achieved a response, experienced no response, progressed to renal failure, or remained in relapse. Responders could potentially maintain their response or relapse, while nonresponders risked renal failure, with potential mortality from any state. Clinical, healthcare resource utilization, and cost data were derived from published literature. Drug prices were based on wholesale acquisition costs. Results: Over 1 year, Acthar® Gel showed a lower cost per response ( $377\hspace{0.17em}185\left)thancyclophosphamide\right($ 551 687) and rituximab ($741 373). This cost advantage of Acthar® Gel was maintained over 2 and 3 years. Acthar® Gel had higher drug acquisition costs than cyclophosphamide and rituximab but resulted in lower overall medical costs and higher response rates within 1 year, without additional treatment-related costs. Over 2 and 3 years, Acthar® Gel had a lower overall cost of care and higher response rates than SoC, establishing it as a dominant treatment option. Conclusions: Based on current model assumptions and clinical inputs, Acthar® Gel may potentially be a cost-effective and value-based treatment strategy vs unapproved SoCs for adults with refractory proteinuria in NS due to iMN, particularly for those who have not responded to conventional therapies over a 1- to 3-year period within a US payer context. These results may inform clinical and payer decision-making in cases when other standard therapies fail to achieve desired outcomes for a specific population.

Background: Prostate-specific antigen (PSA) has been used as both a screening tool and a marker for treatment response for advanced prostate cancer. With the introduction of androgen receptor pathway inhibitor (ARPI)-based treatment for metastatic castration-sensitive prostate cancer (mCSPC), there is a need to understand the impact that early treatment response, as measured by PSA, has on long-term clinical outcomes. Objectives: To assess whether long-term indicators of treatment success differ among ARPI-naïve patients with mCSPC who did or did not attain ≥90% reduction in PSA levels within 6 months of treatment initiation. Methods: Patients with mCSPC initiating a first ARPI (ie, apalutamide, enzalutamide, abiraterone acetate, darolutamide) were identified using electronic medical record data linked to insurance claims in the United States (1/1/2016-9/30/2022). Eligible patients were classified based on whether they achieved ≥90% reduction in PSA measured between pre-treatment and a window of 30 to 180 days after ARPI initiation. Cohorts were balanced using inverse probability of treatment weighting. Weighted Kaplan-Meier analysis was used to compare overall survival and castration-resistance-free survival by 36 months post-index between those with and without ≥90% PSA reduction. Results: Weighted cohorts included 1192 patients with early PSA reduction ≥90% and 699 without. By 36 months, significantly better overall survival was observed in those with early PSA reduction ≥90% than in those without (71.5% vs 54.7%; hazard ratio [HR]: 0.40, 95% confidence interval [CI]: 0.31, 0.50; P<.001). Similarly, significantly better castration-resistance-free survival was observed in those with early PSA reduction ≥90% than in those without (53.3% vs 36.8%; HR: 0.51, 95% CI: 0.43, 0.60; P< .001). Discussion: Early reduction of PSA levels by ≥90% within 6 months of ARPI initiation among patients with mCSPC in the real world is a robust indicator of treatment success, with improved long-term clinical outcomes, including survival and reduction in disease progression. Conclusions: These findings corroborate those of clinical trials and highlight the long-term benefits of an early and deep PSA response to ARPIs among real-world patients with mCSPC in the United States.

Background: The prevalence and socioeconomic impact of obesity are increasing in the United Kingdom; however, the effects of body mass index (BMI) and comorbidities on health-related quality of life (HRQoL) and work productivity in people living with overweight or obesity (PLwO) have not been fully described.

Objective: To assess self-reported HRQoL and work productivity in PLwO in the United Kingdom, grouped by BMI and presence of cardiovascular and/or metabolic (CVM) comorbidities.

Methods: Data used were from the Adelphi Obesity Disease Specific Programme™, a real-world cross-sectional survey (October 2023-April 2024). Physicians reported demographic and clinical characteristics for PLwO on a weight management program who were ≥18 years old and had current or previous BMI ≥30 kg/m2, or ≥27 kg/m2 with ≥1 weight-related comorbidity. The same PLwO completed the 36-item Short-Form Health Survey version 2 (SF-36v2) and Work Productivity and Activity Impairment: Specific Health Problem (WPAI:SHP) questionnaire.

Results: Physicians provided data for 904 PLwO, of whom 119 completed the survey questionnaires. Mean age for PLwO was 51.7 years, 54% were women, the majority were White (69%), and 70% had ≥1 CVM comorbidity. SF-36v2 physical functioning scores were lower with higher BMI (P < .05). Scores for physical functioning, role limitations due to physical problems, bodily pain, general health, vitality, and the physical component summary were lower (P < .05) in PLwO with cardiovascular comorbidities than in those without. PLwO with metabolic comorbidities had lower scores than those without for physical problems, general health, social functioning, mental health, and the mental component summary (P < .05). WPAI:SHP scores for activity impairment, presenteeism, and overall work impairment were higher in PLwO with CVM comorbidities than in those without (P < .05), indicating lower productivity.

Discussion: Increasing BMI may be linked to lower physical functioning in PLwO. Comorbidities were associated with reductions in HRQoL, with different domains affected depending on the type of comorbidity. These comorbidities were also associated with impaired work productivity.

Conclusion: The presence of comorbidities, along with BMI, are important considerations for managing obesity. Reducing the prevalence of overweight or obesity is likely to benefit both individuals and wider society.

Background: The efficacy of the combination of bortezomib, lenalidomide, and dexamethasone with daratumumab (DVRd) or isatuximab (IsaVRd) for the frontline treatment of transplant-ineligible (TIE) newly diagnosed multiple myeloma (NDMM) has been demonstrated in clinical trials. However, the treatment cost for DVRd and IsaVRd has not been compared. Objectives: To compare the drug acquisition costs (DAC) of DVRd vs IsaVRd in the first 2 years of frontline treatment for TIE patients with NDMM in the United States. Methods: Dosing schedules from the CEPHEUS and IMROZ clinical US trials were used for this analysis. AnalySource® was utilized to access the First Databank drug pricing database to collect current US DACs. Drug administration time and costs were identified and weighted, assuming 40% and 60% received the drug in a hospital outpatient and community oncology setting, respectively. Total costs were calculated by adding DACs and drug administration costs. Results: The DAC was $200\hspace{0.17em}866inyear1and$ 137 434 in year 2 for daratumumab and $212\hspace{0.17em}421inyear1and$ 144 143 in year 2 for isatuximab. The DAC of daratumumab was $18\hspace{0.17em}264(5.4$ 17 269 and $17\hspace{0.17em}327lessthanIsaVRdinpatients<75yearsand\ge 75yearsold,respectively.Inyear2,thetotalcostofDVRdperpatientwas$ 10 444 and