Journal of Health Economics and Outcomes Research最新文献

Background: Late-onset Pompe disease (LOPD) is a rare, progressive neuromuscular condition typically characterized by weakness of skeletal muscles, including those involved in respiration and diaphragmatic dysfunction. Individuals with LOPD typically eventually require mobility and/or ventilatory support. Objectives: This study aimed to develop health state vignettes and estimate health state utility values for LOPD in the United Kingdom. Methods: Vignettes were developed for 7 health states of LOPD with states defined in terms of mobility and/or ventilatory support. Vignettes were drafted based on patient-reported outcome data from the Phase 3 PROPEL trial (NCT03729362) and supplemented by a literature review. Qualitative interviews with individuals living with LOPD and clinical experts were conducted to explore the health-related quality-of-life (HRQoL) impact of LOPD and to review the draft vignettes. Vignettes were finalized following a second round of interviews with individuals living with LOPD and used in health state valuation exercises with people of the UK population. Participants rated the health states using the EQ-5D-5L, visual analogue scale, and time trade-off interviews. Results: Twelve individuals living with LOPD and 2 clinical experts were interviewed. Following the interviews, 4 new statements were added regarding dependence on others, bladder control problems, balance issues/fear of falling, and frustration. One hundred interviews with a representative UK population sample were completed. Mean time trade-off utilities ranged from 0.754 (SD = 0.31) (no support) to 0.132 (SD = 0.50) (invasive ventilatory and mobility support-dependent). Similarly, EQ-5D-5L utilities ranged from 0.608 (SD = 0.12) to -0.078 (SD = 0.22). Discussion: The utilities obtained in the study are consistent with utilities reported in the literature (0.670-0.853 for nonsupport state). The vignette content was based on robust quantitative and qualitative evidence and captured the main HRQoL impacts of LOPD. The general public rated the health states consistently lower with increasing disease progression. There was greater uncertainty around utility estimates for the severe states, suggesting that participants found it harder to rate them. Conclusion: This study provides utility estimates for LOPD that can be used in economic modeling of treatments for LOPD. Our findings highlight the high disease burden of LOPD and reinforce the societal value of slowing disease progression.

Background: Gastroesophageal reflux disease (GERD) is a risk factor for Barrett's esophagus (BE) and BE-related neoplasia (BERN). Objectives: This study aimed to evaluate healthcare resource utilization (HRU) and costs associated with GERD, BE, and BERN in the United States. Methods: Adult patients with GERD, nondysplastic BE (NDBE), and BERN (including indefinite for dysplasia [IND], low-grade dysplasia [LGD], high-grade dysplasia [HGD] or esophageal adenocarcinoma [EAC]), were identified from a large US administrative claims database, the IBM Truven Health MarketScan® databases (Q1/2015-Q4/2019). Patients were categorized into the corresponding mutually exclusive EAC-risk/diagnosis cohorts based on the most advanced stage from GERD to EAC using diagnosis codes in medical claims. Disease-related HRU and costs (2020 USD) were calculated for each cohort. Results: Patients were categorized into the following EAC-risk/diagnosis cohorts: 3 310 385 into GERD, 172 481 into NDBE, 11 516 into IND, 4332 into LGD, 1549 into HGD, and 11 676 into EAC. Disease-related annual mean number of inpatient admissions, office visits, and emergency department visits by cohort were 0.09, 1.45, and 0.19 for GERD; 0.08, 1.55, and 0.10 for NDBE; 0.10, 1.92, and 0.13 for IND; 0.09, 2.05, and 0.10 for LGD; 0.12, 2.16, and 0.14 for HGD; and 1.43, 6.27, and 0.87 for EAC. Disease-related annual mean total healthcare costs by cohort were $6955 for GERD, $8755 for NDBE, $9675 for IND, $12 241 for LGD, $24 239 for HGD, and $146 319 for EAC. Discussion: Patients with GERD, BE, and BERN had important HRU and costs, including inpatient admissions and office visits. As patients progressed to more advanced stages, there was substantially higher disease-related resource utilization, with associated costs being 16 times higher in patients with EAC than those with NDBE. Conclusions: Findings suggest the need for early identification of high-risk individuals prior to progression to EAC to potentially improve clinical and economic outcomes in this population.

Background: In hospitalized patients with COVID-19, acute kidney injury (AKI) is associated with higher mortality, but data are lacking on healthcare resource utilization (HRU) and costs related to AKI, community-acquired AKI (CA-AKI), and hospital-acquired AKI (HA-AKI). Objectives: To quantify the burden of AKI, CA-AKI, and HA-AKI among inpatients with COVID-19. Methods: This retrospective cohort study included inpatients with COVID-19 discharged from US hospitals in the Premier PINC AI™ Healthcare Database April 1-October 31, 2020, categorized as AKI, CA-AKI, HA-AKI, or no AKI by ICD-10-CM diagnosis codes. Outcomes were assessed during index (initial) hospitalization and 30 days postdischarge. Results: Among 208 583 COVID-19 inpatients, 30%, 25%, and 5% had AKI, CA-AKI, and HA-AKI, of whom 10%, 7%, and 23% received dialysis, respectively. Excess mortality, HRU, and costs were greater for HA-AKI than CA-AKI. In adjusted models, for patients with AKI vs no AKI and HA-AKI vs CA-AKI, odds ratios (ORs) (95% CI) were 3.70 (3.61-3.79) and 4.11 (3.92-4.31) for intensive care unit use and 3.52 (3.41-3.63) and 2.64 (2.52-2.78) for in-hospital mortality; mean length of stay (LOS) differences and LOS ratios (95% CI) were 1.8 days and 1.24 (1.23-1.25) and 5.1 days and 1.57 (1.54-1.59); and mean cost differences and cost ratios were $7163 and 1.35 (1.34-1.36) and $19 127 and 1.78 (1.75-1.81) (all P < .001). During the 30 days postdischarge, readmission LOS was ≥6% longer for AKI vs no AKI and HA-AKI vs CA-AKI; outpatient costs were ≥41% higher for HA-AKI vs CA-AKI or no AKI. Only 30-day new dialysis (among patients without index hospitalization dialysis) had similar odds for HA-AKI vs CA-AKI (2.37-2.8 times higher for AKI, HA-AKI, or CA-AKI vs no AKI). Discussion: Among inpatients with COVID-19, HA-AKI had higher excess mortality, HRU, and costs than CA-AKI. Other studies suggest that interventions to prevent HA-AKI could decrease excess morbidity, HRU, and costs among inpatients with COVID-19. Conclusions: In adjusted models among COVID-19 inpatients, AKI, especially HA-AKI, was associated with significantly higher mortality, HRU, and costs during index admission, and higher dialysis and longer readmission LOS during the 30 days postdischarge. These findings support implementation of interventions to prevent HA-AKI in COVID-19 patients.

Background: The US population includes 24 million to 29 million people with diagnosed and undiagnosed chronic obstructive pulmonary disease (COPD). Studies have demonstrated the safety and efficacy of single-inhaler triple therapy (SITT) in reducing COPD exacerbations. Long-term population implications of SITT use have not been quantified. Objectives: This simulation-based projection aimed to estimate the potential impact of widespread SITT use on the US COPD population. Methods: Exacerbation and all-cause mortality reductions reported in the Efficacy and Safety of Triple Therapy in Obstructive Lung Disease trial (ETHOS; NCT02465567) were used to project clinical outcomes in US patients meeting ETHOS trial eligibility criteria (ETHOS-Eligible) and patients meeting a practical definition of SITT eligibility (Expanded ETHOS-Eligible). The US COPD population was modeled with 1000 simulations of patient progression over 10 years. Agent characteristics were based on literature and claims analysis of the 2016-2018 Medicare 100% fee-for-service and IBM MarketScan^® databases. Agent annual characteristics reflected incident cases, changes in COPD severity, treatment, mortality, and exacerbations under status quo treatment patterns and scenarios for the adoption of SITT. The scenarios assumed the reduced exacerbation and mortality rates associated with SITT according to ETHOS trial outcomes mean values. Results: Higher than current SITT adoption over 10 years would be expected to substantially reduce COPD exacerbation-associated hospitalizations by 2 million. Applying mean improvements reported in ETHOS for SITT would extend average patient life expectancy 2.2 years for ETHOS-Eligible patients and 1.7 years for Expanded ETHOS-Eligible patients. The number needed to treat to extend the average patient life by 1 year was 8 for the ETHOS-Eligible population and 10 for the Expanded ETHOS-Eligible population. Discussion: Widespread SITT adoption may be impeded by competitive pressures from generic treatments and nonadherence, and efficacy observed in clinical trials may not occur in real-world populations. Conclusions: Assuming ETHOS treatment effects and adherence translate to clinical practice, higher than current use of SITT can substantially reduce COPD exacerbations and hospitalizations and extend survival. These results should be viewed cautiously, because the improved outcomes for SITT in the ETHOS final retrieved vital statistics data were not statistically significant for all comparator therapy groups.

Background: With advances in antiretroviral therapy (ART), people with HIV infection are living longer. Pre-exposure prophylaxis (PrEP) to reduce HIV infection risk continues to be underutilized in high-risk individuals. Recent data on economic burden for patients with newly diagnosed HIV-1 or initiated with PrEP are limited. Objectives: To assess characteristics, healthcare resource utilization (HRU), and costs among adults and adolescents either with newly diagnosed HIV-1 or initiated with PrEP. Methods: This retrospective observational study utilized data from the IBM MarketScan® Commercial Claims and Encounters database. Adults with newly diagnosed HIV-1 or those initiated with PrEP were included (index date was the first HIV diagnosis or PrEP prescription, respectively, between January 1, 2016, and April 30, 2021). Corresponding cohorts of adolescents were considered exploratory. Descriptive analyses were conducted to assess baseline demographics and clinical characteristics, and all-cause and HIV-related HRU and costs per patient per month (PPPM) during follow-up. Results: Data from 18 154 adults and 220 adolescents with newly diagnosed HIV and 34 123 adults and 175 adolescents initiated with PrEP were included. Approximately 70% of adolescents and 9% of adults receiving PrEP were female. Baseline depression/anxiety was present in 16.1% and 24.6% of adults and 14.5% and 45.1% of adolescents in the HIV and PrEP cohorts, respectively. Substance abuse in the HIV and PrEP cohorts, respectively, was reported in 10.1% and 7.0% of adults, and 2.7% and 17.7% of adolescents. During follow-up, among adults with newly diagnosed HIV, mean (SD) total all-cause and HIV-related PPPM costs were $2657 ($5954) and $1497 ($4463), respectively; pharmacy costs represented 47% of all-cause costs and 67% of HIV-related costs, but only 37% of patients had an HIV-related prescription. All-cause costs PPPM for adults with PrEP were $1761 ($1938), with pharmacy costs accounting for 71%. Conclusions: Despite advances in ART, patients with newly diagnosed HIV and at-risk patients receiving PrEP continue to incur HRU costs. The chronic nature of HIV warrants further exploration of factors contributing to disease burden and opportunities to improve prevention strategies.