Journal of Health Economics and Outcomes Research最新文献

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.

Background: Demonstrating the cost-effectiveness of new treatments for multiple myeloma (MM) often relies on the extrapolation of overall survival (OS) trial data. This method can introduce uncertainty in long-term survival estimates if OS data are immature, as is often the case in newly diagnosed MM (NDMM). We explore the use of the relationship between minimal residual disease (MRD) status and OS to reduce uncertainty of long-term survival outcomes. Objectives: To evaluate if uncertainty in long-term modeled outcomes in NDMM is reduced using a response-based partitioned survival model (PSM), whereby patients were categorized as MRD-positive or -negative, relative to a standard PSM, when OS data are immature. Methods: Standard and response-based PSMs, estimating patient life-years (LYs) over a lifetime horizon, were developed for NDMM patients treated with bortezomib, thalidomide, and dexamethasone (BTd) with or without daratumumab as induction and consolidation therapy. In the standard PSM, LYs were determined by extrapolations from individual patient data from CASSIOPEIA. In the response-based PSM, survival was dependent on MRD status at the time of the response assessment via a landmark analysis. Cox-proportional hazard ratios from external sources and CASSIOPEIA informed the relationship for OS between MRD-positive and MRD-negative, and between patients receiving BTd and daratumumab plus BTd, respectively. Uncertainty was assessed by comparing LYs and OS extrapolations from deterministic and probabilistic analyses. Results: This response-based PSM demonstrated reduced uncertainty in long-term survival outcomes compared with the standard PSM (range across extrapolations of 3.4 and 7.7 LYs for daratumumab plus BTd and BTd, respectively, vs 14.8 and 11.8 LYs for the standard PSM). It also estimated a narrower interquartile range of LYs in the probabilistic analyses for the majority of parametric extrapolations. Discussion: Alternative methods to estimate long-term survival outcomes, such as a response-based PSM, can reduce uncertainty in modeling predictions around cost-effectiveness estimates for health technology assessment bodies and payers, thereby supporting faster market access for novel therapies with immature survival data. Conclusions: Use of MRD status in a response-based PSM reduces uncertainty in modeling long-term survival in patients with NDMM and provides a greater number of clinically plausible extrapolations compared with a standard PSM.

The authors respond to the comments raised in the letter regarding Adeniji and Obembe's article on catastrophic health expenditures in sub-Saharan Africa.

The Institute for Clinical and Economic Review (ICER), a nonprofit, nongovernmental organization, is the predominant independent price assessor in the United States. ICER's cost effectiveness assessments are increasingly being used to support health insurance coverage and healthcare policy decisions. ICER often does not apply rigorous data quality and inclusion criteria to either the assumptions embedded within their cost-effectiveness models or the data inputted into the models. Poor quality assumptions and data can lead to poor quality assessments. ICER should re-evaluate their reliance on quality adjusted life-years and equal value of life years gained as measures of drug effectiveness, establish data quality and inclusiveness minimum standards, produce cost-effectiveness assessments only when the minimum data is available, and prominently report data quality and inclusion limitations. These changes will increase the rigor and inclusiveness of drug price assessments and support sustainable access to high-value care for all Americans.

Background: The effect of gestational age (GA) on comorbidity prevalence, healthcare resource utilization (HCRU), and all-cause costs is significant for extremely premature (EP) infants in the United States. Objectives: To characterize real-world patient characteristics, prevalence of comorbidities, rates of HCRU, and direct healthcare charges and societal costs among premature infants in US Medicaid programs, with respect to GA and the presence of respiratory comorbidities. Methods: Using International Classification of Diseases, Ninth/Tenth Revision, Clinical Modification codes, diagnosis and medical claims data from 6 state Medicaid databases (1997-2018) of infants born at less than 37 weeks of GA (wGA) were collected retrospectively. Data from the index date (birth) up to 2 years corrected age or death, stratified by GA (EP, ≤28 wGA; very premature [VP], >28 to <32 wGA; and moderate to late premature [M-LP], ≥32 to <37 wGA), were compared using unadjusted and adjusted generalized linear models. Results: Among 25 573 premature infants (46.1% female; 4462 [17.4%] EP; 2904 [11.4%] VP; 18 207 [71.2%] M-LP), comorbidity prevalence, HCRU, and all-cause costs increased with decreasing GA and were highest for EP. Total healthcare charges, excluding index hospitalization and all-cause societal costs (US dollars), were 2 to 3 times higher for EP than for M-LP (EP $74 436 vs M-LP $27 541 and EP $28 504 vs M-LP $15 892, respectively). Conclusions: Complications of preterm birth, including prevalence of comorbidities, HCRU, and costs, increased with decreasing GA and were highest among EP infants during the first 2 years in this US analysis.