Impact of earlier versus later monitoring on disease progression and healthcare costs among patients with chronic myeloid leukemia in the United States.

We evaluated the impact of molecular monitoring earlier as compared to later in the course of chronic myeloid leukemia (CML) on disease progression and healthcare costs in the real-world setting in the US. Patients with a diagnosis of CML were identified from the MarketScan claims databases (1 January 2006 to 30 June 2016). Multivariable regression analyses were used to control for differences in patient cohorts with earlier versus later monitoring. Of the 2730 CML patients in the study population, 60% (n = 1633) received earlier monitoring and 40% (n = 1097) received later monitoring only. After adjusting for differences in patient characteristics, patients with earlier monitoring had a lower likelihood of CML progression during the follow-up period (odds ratio: 0.72, confidence interval: 0.53-0.96; p = .03) and lower total healthcare costs ($6794 versus $9782 per-patient-per-month, p < .001) than patients with later monitoring. Patients who are monitored earlier in the course of CML may have better outcomes and lower total costs of care.

Economic value of regular monitoring of response to treatment among US patients with chronic myeloid leukemia based on an economic model.

BACKGROUND: Regular molecular monitoring with reverse-transcription quantitative PCR (RT-qPCR) analysis of BCR-ABL1 transcripts is associated with reduced disease progression among patients with chronic myeloid leukemia (CML). Molecular monitoring assists in the timely detection of primary or secondary resistance to tyrosine kinase inhibitor (TKI) therapy and is a recommended practice by the National Comprehensive Cancer Network guidelines. An economic model was developed to estimate the potential impact of CML monitoring vs lack of monitoring on patient healthcare costs. METHODS: An Excel-based decision-analytic economic model was developed from a US payer perspective. The model was used to estimate the expected healthcare cost differences between regular molecular monitoring of CML patients and lack of monitoring. CML progression rates among patients with vs without monitoring, the annual cost of CML progression, the average number of monitoring tests per year, and the average cost per RT-qPCR monitoring test were incorporated into the model. Univariate and multivariable sensitivity analyses were conducted. RESULTS: Based on estimates in published literature, disease progression to the accelerated/blast phase occurs among 0.35% of patients with monitoring and 5.12% of patients without monitoring, and the annual cost of CML progression is $136,308 per patient year. The analysis found that total healthcare costs, including the costs associated with CML progression and RT-qPCR monitoring tests (three tests per year), were $1,142 for patients with monitoring and $6,982 for patients without monitoring (difference = $5,840). In a hypothetical cohort of 100 patients with CML, achieving a 100% monitoring rate was associated with a total cost-savings of $584,005 compared to a 0% monitoring rate. This cost-savings remained consistent under both univariate and multivariable sensitivity analyses. CONCLUSION: Regular CML monitoring was associated with improved outcomes among CML patients and, consequently, reduced healthcare costs.

Evaluation of healthcare resource utilization and incremental economic burden of patients with chronic myeloid leukemia after disease progression to blast phase.

AIMS: To evaluate healthcare resource utilization and economic burden of patients with chronic myeloid leukemia (CML) progression to the blast phase. METHODS: Patients (≥ 18 years) with ≥1 inpatient or ≥2 outpatient CML diagnoses were identified from the MarketScan Commercial and Medicare databases (January 1, 2007-June 30, 2015). CML patients were grouped into two study cohorts, those with evidence of disease progression to the blast phase and those without. Patients were required to have continuous medical and prescription coverage during a 12-month baseline period, in which demographics and clinical characteristics were evaluated. All-cause healthcare resource utilization and costs were evaluated during the baseline period, and a variable follow-up period, lasting ≥1 day and up to 1 year. Generalized linear models (GLM) were used to compare the incremental costs of CML patients with vs without progression. RESULTS: Of the overall study population, 587 (7%) experienced disease progression and 7,504 (93%) did not. On the index date, of patients with progression, ∼ 31% were treated with allogeneic hematopoietic cell transplant and 69% with chemotherapy. During the baseline period, mean total healthcare costs, including costs for hospitalizations and outpatient costs, were significantly greater for CML patients with progression as compared to those without progression ($143,778 vs $53,143, p < .001). During the follow-up, mean total healthcare costs, costs for hospitalizations, and outpatient medical service costs were substantially greater for patients with progression as compared to those without progression; however, costs for outpatient prescriptions were less for patients who progressed. When patient characteristics were controlled for, mean incremental 1-year cost for CML patients with vs without progression was $270,925 (confidence interval = $235,290-$311,958, p < .001). CONCLUSIONS: The healthcare burden, in terms of healthcare resource utilization and costs, of patients with CML progression is substantial. Healthcare providers and payers should consider various strategies to minimize the rate of CML progression.

The value of open access and a patient centric approach to oral oncolytic utilization in the treatment of Chronic Myelogenous Leukemia: A U.S. perspective.

INTRODUCTION: Since the introduction of tyrosine kinase inhibitors (TKIs), the treatment of patients with chronic myelogenous leukemia (CML) has resulted in significant improvement in patient survival but at a higher pharmaceutical cost to payers. The recent introduction of generic imatinib presents an opportunity to lower pharmacy costs within a population that is growing due to improved survival. Recent literature has focused on the likely benefits to payers of step therapy through generic imatinib. Areas covered: This review provides a perspective that is broader than the evaluation of financial savings or narrowly defined health economic metrics by incorporating factors such as CML patient heterogeneity, including varying levels of disease progression risk, comorbidities and genetic mutation status, differences in TKI product profiles, clinical guideline recommendations, and the importance of individualized patient care. A focused literature review evaluating the real-world impact of utilization management programs is presented. Expert commentary: The findings indicate that payers can achieve substantial savings without the need to implement utilization management policies. Compromises in the ability to provide individualized patient care and unwanted economic consequences resulting from increased costs of disease progression, adverse events, and lack of response to treatment due to utilization management are summarized.

Using Health Care Claims Data to Assess the Prevalence of Hodgkin Lymphoma and Relapsed or Refractory Hodgkin Lymphoma in the United States.

PURPOSE: Although most patients with Hodgkin lymphoma (HL) respond to primary therapy, some patients experience relapses or are refractory to treatment (RR-HL). The objectives of this study were to investigate the prevalence of HL and RR-HL in the United States by using a large health care claims database. METHODS: Patients with ≥1 diagnosis for HL between January 1, 2013, and September 30, 2014 (prevalence assessment period), in the MarketScan Commercial and Medicare databases were identified. RR-HL patients were identified as any HL patient with any record for either an autologous stem cell transplantation (ASCT) or brentuximab vedotin (BV) treatment between January 1, 2010, and September 30, 2014 (entire study period). Prevalence rates of HL and RR-HL were calculated as the number of patients with HL or RR-HL divided by the total number of persons with insurance enrollment during the prevalence assessment period (January 1, 2013-September 30, 2014) in the MarketScan databases. Age- and sex-specific prevalence rates for HL and RR-HL were estimated. The estimated prevalence rates based on the claims database analysis were applied to the US national population estimates from the US Census Bureau to project the national prevalence of HL and RR-HL in the United States. FINDINGS: Of persons with any insurance enrollment in the MarketScan databases during the prevalence assessment period (N = 58,968,235), 24,812 (0.04%) were identified as having HL (mean age, 48.6 years) between January 1, 2013, and September 30, 2014. Of this HL population, 712 (2.87%) were identified as RR-HL patients, with 432 (1.74%) having received ASCT, 199 (0.80%) having received BV, and 81 (0.33%) having received both ASCT and BV treatments during the study period. According to the national projection according to the US Census population estimate, the overall number of persons with HL in the United States was estimated at 149,615 (469.2 per 1 million) in 2014, with 2.72% (N = 4077; 12.8 per 1 million) having RR-HL. IMPLICATIONS: Among patients in the United States with HL, the proportion of RR-HL patients during the study period was estimated at <3% of the HL population.

The relationship of mean deviation scores and resource utilization among patients with glaucoma: a retrospective United States and European chart review analysis.

PURPOSE: To determine the relationship between mean deviation (MD) scores and resource utilization among glaucoma patients in the United States and Europe. METHODS: A retrospective analysis of patient in the United States (June 1990 to January 2003, N=151) and Europe (June 1994 to July 2003, N=194) was conducted. Office visits, pressure-lowering medications, visual field examinations, and glaucoma surgeries were recorded with age, intraocular pressure (IOP), and MD. Patients with missing MD and IOP were excluded; final sample sizes were 130 in the United States and 161 in Europe. Fixed effects and logistic regression models assessed the associations between MD and visits, medication, examinations, and surgery. RESULTS: Number of office visits and visual field examinations significantly increased but the number of glaucoma medications decreased as MD worsened and IOP increased. Odds of trabeculoplasty within 2 years decreased by 8.1% in the United States and 9.9% in Europe with every 1.0 dB improvement in MD. Odds of trabeculectomy within 2 years increased by 13.9% in the United States and by 18.6% in Europe with every 1 mm Hg increase in IOP. In the United States, odds of trabeculectomy within 5 years increased by 12.2% with each 1.0 dB worsening in MD. CONCLUSIONS: MD is associated with resource utilization in glaucoma patients. Evidence supporting use of MD to predict surgery is less conclusive, possibly due to the relative low frequency of surgeries. Therapies aimed at stabilizing visual field deterioration may reduce resource use and costs associated with glaucoma; however, more research is necessary to establish whether such treatments meet common standards of cost-effectiveness.

Association between intraocular pressure variation and glaucoma progression: data from a United States chart review.

PURPOSE: To evaluate whether greater intraocular pressure (IOP) variation between visits was associated with higher likelihood of glaucoma progression. DESIGN: Cohort study. METHODS: A five-year minimum of data (June 1, 1990 through January 22, 2002) was collected on 151 patients (302 eyes) from 12 United States specialty centers. A post hoc analysis of visual field (VF) progression, glaucoma medication, intraocular pressure (IOP), and other ocular data was conducted for two nonmutually exclusive cohorts based on retrospective data abstracted well after actual patient visits. Mean IOP and standard deviations (SD) were calculated before treatment (medication or surgery) or progression, whichever occurred first, and before progression regardless of treatment. IOP variables were assessed in a univariate fashion; Cox proportional hazards models evaluated glaucoma progression as an outcome measure and IOP SD as a main predictor, controlling for covariates. RESULTS: In cohort 1 (55 patients; 84 eyes), mean age was 63 years (range, 37 to 85 years), 58% were female, and 19% of eyes underwent VF progression. In cohort 2 (129 patients; 251 eyes), mean age was 66 years (range, 19 to 88 years), 55% were female, and 27% of eyes underwent VF progression. Mean IOP was 16.5 mm Hg (IOP SD, 2.0 mm Hg), and 16.4 mm Hg (IOP SD, 2.7 mm Hg) in cohorts 1 and 2, respectively. Controlling for age, mean IOP, VF stage, and other covariates, each unit increase in IOP SD resulted in a 4.2 times and 5.5 times higher risk of glaucoma progression for cohort 1 (95% confidence interval [CI], 1.3 to 12.9) and cohort 2 (95% CI, 3.4 to 9.1), respectively. CONCLUSIONS: IOP variability is an important predictor of glaucoma progression; SD is a convenient measure of variability to assess glaucoma progression risk.

Glaucoma in the United States and europe: predicting costs and surgical rates based upon stage of disease.

PURPOSE: Primary open-angle glaucoma is a significant health-economic burden in both the United States and Europe that is likely to increase. This study compared treatment patterns and cost among patients with primary open-angle glaucoma in these locations. METHODS: Retrospective medical chart reviews were conducted in the United States (1990 to 2002) and Europe (1995 to 2003). A total sample of 151 US charts and 194 European charts was studied, and patients were assigned a baseline intraocular pressure (IOP) and baseline stage, using a 6-stage visual functional glaucoma staging algorithm. Resource utilization and direct costs were assessed by stage of disease using publicly available United States and European costs. Cox Proportional Hazards modeling were used to examine covariates predicting glaucoma surgery. Total cost was predicted, adjusting for covariates using Generalized Linear Models, with baseline stage as the independent variable. RESULTS: Glaucoma surgery requirement was highly associated with baseline disease stage and IOP increase before surgery in the United States and somewhat associated with these factors in Europe. Within both locations, baseline IOP was highly associated with glaucoma surgery requirement. Controlling for covariates, patients at higher baseline stages incurred greater costs in the United States (P=0.0017) and Europe (P=0.0715). Surgery and medication were also highly predictive of increased cost (P<0.0001). Cost of care differed greatly between the European countries, with costs lowest in Italy. CONCLUSIONS: Increases in annual cost were related to higher baseline IOP, higher baseline stage, medication, and surgery. Thus, significant potential savings and reductions in annual healthcare burden are possible if patients are diagnosed and treated at earlier stages of glaucoma.

Costs and utilization of end-stage glaucoma patients receiving visual rehabilitation care: a US multisite retrospective study.

PURPOSE: Glaucoma is a prevalent ophthalmologic disease and leading cause of blindness. A retrospective analysis was conducted to evaluate resources and costs for end-stage glaucoma patients receiving visual rehabilitation care (VRC). MATERIALS AND METHODS: A chart review was conducted in 3 United States VRC centers. Charts of patients with primary open-angle glaucoma as the primary cause of vision loss (1998 to 2003) were selected, yielding 81 records. Data were collected from patient-level billing and reimbursement records (ophthalmologist/optometrist visits, glaucoma medications, procedures, and specialized low-vision and glaucoma-related services). Visual rehabilitation services included utilization of low-vision devices, assessment of daily functioning, orientation and mobility training, and patient counseling. RESULTS: Mean age at baseline was 72.7 years [standard deviation (SD)=17.2, range: 29 to 95]. Of those with known sex (n=77), 55.8% were women. Medicare was the payer type for most patients (59.3%), whereas 20% had Medicaid. Mean number of visits was 7.1 (SD=6.1) in year 1 and 3.7 (SD=4.2) in year 2, for an annual mean of 5.4 (SD=5.0) visits overall. Total mean cost per patient in year 1 was greater than year 2 [$2170 (SD=$2252) vs. $1202 (SD=$1080), respectively]; of the total 2-year costs, 15% were VRC, 37% ophthalmology care, and 48% pharmacy. Analysis of nonpharmacy costs revealed that VRC accounted for 28% and ophthalmology for 72%. CONCLUSIONS: End-stage glaucoma is associated with appreciable resource utilization and costs, because of both vision rehabilitation and ophthalmology care. Advanced primary open-angle glaucoma has a substantial cost-of-illness, warranting improved management in early stages of disease.

Categorizing the stage of glaucoma from pre-diagnosis to end-stage disease.

PURPOSE: To provide a reliable, comprehensive staging system to assess glaucoma stage in the absence of an universally accepted glaucoma staging system (GSS) on the basis of visual field results. DESIGN: Literature review and GSS adaptation. METHODS: After a review of published GSSs was conducted, the Bascom Palmer (Hodapp-Anderson-Parrish) GSS was selected as an appropriate platform for a retrospective GSS on the basis of visual fields. The system was modified by a panel of glaucoma specialists, and additional modifications were made after pilot testing to cover the full range of disease progression, from preglaucoma diagnosis to complete blindness; the ordered stages reflect the typical progression of glaucoma. RESULTS: The GSS is comprised of six ordered stages and is on the basis of the Humphrey visual field. The completed GSS was validated by reviewing patient charts from 12 US glaucoma centers. CONCLUSIONS: The GSS allows accurate staging of 100% of glaucoma on the basis of visual fields and other data, enabling evaluation of disease progression and resource utilization at various glaucoma stages. Additionally, treatment costs may be assigned to determine cost-effectiveness of treatment. Research utilizing the GSS has found that cost of care increases with increasing disease severity. The GSS may be used as the basis for creating treatment guidelines, which have the potential to delay glaucoma progression and lower treatment costs.