Cost Outcomes For Patients Research Articles

Clinical, Quality of Life and Cost Outcomes in Patients with Early Stage Non Small Cell Lung Cancer Undergoing Surgery or SBRT

Clinical, Quality of Life and Cost Outcomes in Patients with Early Stage Non Small Cell Lung Cancer Undergoing Surgery or SBRT

Burden of illness associated with overweight and obesity in patients hospitalized with COVID-19 in the United States: analysis of the premier healthcare database from April 1, 2020 to October 31, 2020

BackgroundSARS-CoV-2 (COVID-19) continues to be a major public health issue. Obesity is a major risk factor for disease severity and mortality associated with COVID-19.ObjectiveThis study sought to estimate the healthcare resource use and cost outcomes in patients hospitalized with COVID-19 in the United States (US) according to body mass index (BMI) class.MethodsRetrospective cross-sectional study analyzing data from the Premier Healthcare COVID-19 database for hospital length-of-stay (LOS), intensive care unit (ICU) admission, ICU LOS, invasive mechanical ventilator use, invasive mechanical ventilator use duration, in-hospital mortality, and total hospital costs from hospital charge data.ResultsAfter adjustment for patient age, gender, and race, patients with COVID-19 and overweight or obesity had longer durations for mean hospital LOS (normal BMI = 7.4 days, class 3 obesity = 9.4 days, p < .0001) and ICU LOS (normal BMI = 6.1 days, class 3 obesity = 9.5 days, p < .0001) than patients with normal weight. Patients with normal BMI had fewer days on invasive mechanical ventilation compared to patients with overweight and obesity classes 1–3 (6.7 days vs. 7.8, 10.1, 11.5, and 12.4, respectively, p < .0001). The predicted probability of in-hospital mortality was nearly twice that of patients with class 3 obesity compared to patients with normal BMI (15.0 vs 8.1%, p < .0001). Mean (standard deviation) total hospital costs for a patient with class 3 obesity is estimated at $26,545 ($24,433–$28,839), 1.5 times greater than the mean for a patient with a normal BMI at $17,588 ($16,298–$18,981).ConclusionsIncreasing levels of BMI class, from overweight to obesity class 3, are significantly associated with higher levels of healthcare resource utilization and costs in adult patients hospitalized with COVID-19 in the US. Effective treatment of overweight and obesity are needed to reduce the burden of illness associated with COVID-19.

Association between oncology clinical pathway utilization and quality and cost outcomes in patients with metastatic solid tumors.

430 Background: Anticancer drug regimens that are approved by accepted drug compendia and also considered high value based on their efficacy, toxicity, and costs are designated as “on-pathway” for a national commercial payer. This study compared quality and cost of cancer care among patients with metastatic solid tumors treated in the first line setting who were prescribed on- vs. off-pathway regimens. Methods: Using administrative claims data and prior authorization data from a national commercial payer, we identified 8,357 commercially insured or Medicare Advantage adult patients with solid tumor cancers including breast, lung, colorectal, pancreatic, melanoma, kidney, bladder, gastric, or uterine cancer, who were prescribed first-line anti-cancer regimens in the metastatic setting from 2018 to 2021. Patients were classified into on- vs. off pathway group based on the initial anticancer regimen that was prescribed. On-pathway status was prospectively defined by a panel of practicing oncologists based on review of curated evidence and general application of relative clinical value frameworks accepted in the field. We compared post–6-month quality-of-care outcomes including chemotherapy-related avoidable hospitalizations, emergency room (ER) visits, immune-related adverse events (IRAEs) such as endocrinopathies owing to immune checkpoint inhibitors, need for supportive drugs such as granulocyte colony stimulating factor, and cost outcomes between groups. Generalized linear models were used to assess the association between on-pathway regimens and outcomes adjusting for key patient demographics, clinical and provider characteristics. Results: A total of 5,453 (65.3%) patients were prescribed on-pathway regimens. Both groups had similar age (60.1 vs. 59.6, p = 0.06) and ECOG performance status (0.63 vs. 0.62, p = 0.40), with more females in the off-pathway group (54.6% vs. 57.3%, p = 0.02). There was no statistically significant difference in chemotherapy-related avoidable hospitalizations, IRAEs and need for supportive drugs between the two groups after modeling adjustment. However, patients treated on-pathway had higher rates of chemotherapy-related avoidable ER visits (18% vs. 15%, adjusted odds ratio: 1.16, 95% confidence interval (CI): 1.01 to 1.33, p = 0.03). Patients in the on-pathway group had significantly lower 6-month anticancer treatment cost (adjusted cost difference: -$10410, 95% CI: -$14935 to -$5886, p &lt; 0.01), resulting in an overall lower total healthcare costs (adjusted cost difference: -$12826, 95% CI: -$18879 to -$6773, p &lt; 0.01). Conclusions: Pathway regimens for metastatic solid tumors were associated with reduced total healthcare costs and similar quality of care compared with off-pathway regimens. These findings support the use of high value, evidence-based regimens for metastatic cancer patients.

The Impact of Obstructive Sleep Apnea on Clinical, Perioperative, and Cost Outcomes in Patients Who Underwent Posterior Cervical Decompression and Fusion: A Single-Center Retrospective Analysis From 2008 to 2016.

Obstructive sleep apnea (OSA) is a pervasive problem that can result in diminished neurocognitive performance, increased risk of all-cause mortality, and significant cardiovascular disease. While previous studies have examined risk factors that influence outcomes following cervical fusion procedures, to our knowledge, no study has examined the cost or outcome profiles for posterior cervical decompression and fusion (PCDF) procedures in patients with OSA. All cases at a single institution between 2008 and 2016 involving a PCDF were included. The primary outcome was prolonged extubation, defined as an extubation that took place outside of the operating room. Secondary outcomes included admission to the intensive care unit (ICU), complications, extended hospitalization, nonhome discharge, readmission within 30 and 90 days, emergency room visit within 30 and 90 days, and higher total costs. We reviewed 1191 PCDF cases, of which 93 patients (7.81%) had a history of OSA. At the univariate level, patients with OSA had higher rates of ICU admissions (33.3% vs 16.8%, P < 0.0001), total complications (29.0% vs 19.0%, P = 0.0202), and respiratory complications (12.9% vs 6.6%, P = 0.0217). Multivariate regression analyses revealed no difference in the odds of a prolonged extubation (P = 0.4773) and showed that history of OSA was not predictive of higher costs. However, a significant difference was observed in the odds of having an ICU admission (P = 0.0046). While patients with sleep apnea may be more likely to be admitted to the ICU postoperatively, OSA status a lone is not a risk factor for poor primary and secondary clinical outcomes following posterior cervical fusion procedures. Various deformities of the cervical spine can exert extraluminal forces that partially collapse or obstruct the airway, thereby predisposing patients to OSA; however, no study has examined the cost or outcome profiles for PCDF procedures in patients with OSA. Therefore, this investigation highlights the ways in which OSA influences the risks, outcomes, and costs following PCDF using medical data from an institutional registry.

The PRIME Type 2 Diabetes Model: a novel, patient-level model for estimating long-term clinical and cost outcomes in patients with type 2 diabetes mellitus

Background and Aims The growing burden of diabetes mellitus and recent progress in understanding cardiovascular outcomes for type 2 diabetes (T2D) patients continue to make the disease a priority for healthcare decision-makers around the world. Our objective was to develop a new, product-independent model capable of projecting long-term clinical and cost outcomes for populations with T2D to support health economic evaluation. Methods Following a systematic literature review to identify longitudinal study data, existing T2D models and risk formulae for T2D populations, a model was developed (the PRIME Type 2 Diabetes Model [PRIME T2D Model]) in line with good practice guidelines to simulate disease progression, diabetes-related complications and mortality. The model runs as a patient-level simulation and is capable of simulating treatment algorithms and risk factor progression, and projecting the cumulative incidence of macrovascular and microvascular complications as well as hypoglycemic events. The PRIME T2D Model can report clinical outcomes, quality-adjusted life expectancy, direct and indirect costs, along with standard measures of cost-effectiveness and is capable of probabilistic sensitivity analysis. Several approaches novel to T2D modeling were utilized, such as combining risk formulae using a weighted model averaging approach that takes into account patient characteristics to evaluate complication risk. Results Validation analyses comparing modeled outcomes with published studies demonstrated that the PRIME T2D Model projects long-term patient outcomes consistent with those reported for a number of long-term studies, including cardiovascular outcomes trials. All root mean squared deviation (RMSD) values for internal validations (against published studies used to develop the model) were 1.1% or less and all external validation RMSDs were 3.7% or less. Conclusions The PRIME T2D Model is a product-independent analysis tool that is available online and offers new approaches to long-standing challenges in diabetes modeling and may become a useful tool for informing healthcare policy. HIGHLIGHTS The PRIME Type 2 Diabetes (T2D) Model is a new, product-independent simulation model. The model offers new approaches to long-standing challenges in diabetes modeling. PRIME T2D Model projects outcomes consistent with those from clinical trials. The model is designed to be a useful tool for informing healthcare policy in T2D.

TAVR and cancer: machine learning-augmented propensity score mortality and cost analysis in over 30 million patients

IntroductionCardiovascular disease (CVD) and cancer are the top mortality causes globally, yet little is known about how the diagnosis of cancer affects treatment options in patients with hemodynamically compromising aortic stenosis (AS). Patients with cancer often are excluded from aortic valve replacement (AVR) trials including trials with transcatheter AVR (TAVR) and surgical AVR (SAVR). This study looks at how cancer may influence treatment options and assesses the outcome of patients with cancer who undergo SAVR or TAVR intervention. Additionally, we sought to quantitate and compare both clinical and cost outcomes for patients with and without cancer.MethodsThis population-based case-control study uses the most recent year available National Inpatient Sample (NIS (2016) from the United States Department of Health and Human Services’ Agency for Healthcare Research and Quality (AHRQ). Machine learning augmented propensity score adjusted multivariable regression was conducted based on the likelihood of undergoing TAVR versus medical management (MM) and TAVR versus SAVR with model optimization supported by backward propagation neural network machine learning.ResultsOf the 30,195,722 total hospital admissions, 39,254 (0.13%) TAVRs were performed, with significantly fewer performed in patients with versus without cancer even in those of comparable age and mortality risk (23.82% versus 76.18%, p < 0.001) despite having similar hospital and procedural mortality. Multivariable regression in patients with cancer demonstrated that mortality was similar for TAVR, MM, and SAVR, though LOS and cost was significantly lower for TAVR versus MM and comparable for TAVR versus SAVR. Patients with prostate cancer constituted the largest primary cancer among TAVR patients including those with metastatic disease. There were no significant race or geographic disparities for TAVR mortality.DiscussionComparison of aortic valve intervention in patients with and without cancer suggests that interventions are underutilized in the cancer population. This study suggests that patients with cancer including those with metastasis have similar inpatient outcomes to patients without cancer. Further, patients who have symptomatic AS and those with higher risk aortic valve disease should be offered the benefit of intervention. Modern techniques have reduced intervention-related adverse events, provided improved quality of life, and appear to be cost effective; these advantages should not necessarily be denied to patients with co-existing cancer.

Cost and clinical outcomes of postoperative intensive care unit versus general floor management in head and neck free flap reconstructive surgery patients

PurposeTo compare clinical, surgical, and cost outcomes in patients undergoing head and neck free-flap reconstructive surgery in the setting of postoperative intensive care unit (ICU) against general floor management. MethodsRetrospective analysis of head and neck free-flap reconstructive surgery patients at a single tertiary academic medical center. Clinical data was obtained from medical records. Cost data was obtained via the Mayo Clinic Rochester Cost Data Warehouse, which assigns Medicare reimbursement rates to all professional billed services. ResultsA total of 502 patients were included, with 82 managed postoperatively in the ICU and 420 on the general floor. Major postoperative outcomes did not differ significantly between groups (Odds Ratio[OR] 1.54; p = 0.41). After covariate adjustments, patients managed in the ICU had a 3.29 day increased average length of hospital stay (Standard Error 0.71; p < 0.0001) and increased need for take-back surgery (OR 2.35; p = 0.02) when compared to the general floor. No significant differences were noted between groups in terms of early free-flap complications (OR 1.38;p = 0.35) or late free-flap complications (Hazard Ratio 0.81; p = 0.61). Short-term cost was $8772 higher in the ICU (range = $5640–$11,903; p < 0.01). Long-term cost did not differ significantly. ConclusionPostoperative management of head and neck oncologic free-flap patients in the ICU does not significantly improve major postoperative outcomes or free-flap complications when compared to general floor care, but does increase short-term costs. General floor management may be appropriate when cardiopulmonary compromise is not present.

POS-345 ASSOCIATION BETWEEN RELATIVE ESTIMATED GLOMERULAR FILTRATION RATE CHANGES AND DAYS OUT OF THE HOME AND COST OUTCOMES IN PATIENTS WITH TYPE 2 DIABETES

A ≥40% relative decline in estimated glomerular filtration rate (eGFR) has been supported for use as a surrogate endpoint for clinical outcomes. However, the associations between this change and resulting days that a patient spends out of their primary ambulatory residence (e.g., during inpatient admissions and nursing facility stays) and cost outcomes are limited. The purpose of this analysis was to evaluate the associations of relative changes in eGFR with days out of the home and with cost outcomes in patients with type 2 diabetes mellitus (T2DM).

Cost comparison of two approaches to chiropractic care for patients with acute and sub-acute low Back pain care episodes: a cohort study

BackgroundLow back pain (LBP) imposes a costly burden upon patients, healthcare insurers, and society overall. Spinal manipulation as practiced by chiropractors has been found be cost-effective for treatment of LBP, but there is wide variation among chiropractors in their approach to clinical care, and the most cost-effective approach to chiropractic care is uncertain. To date, little has been published regarding the cost effectiveness of different approaches to chiropractic care. Thus, the current study presents a cost comparison between chiropractic approaches for patients with acute or subacute care episodes for low back pain.MethodsWe employed a retrospective cohort design to examine costs of chiropractic care among patients diagnosed with acute or subacute low back pain. The study time period ranged between 07/01/2016 and 12/22/2017. We compared cost outcomes for patients of two cohorts of chiropractors within health care system: Cohort 1) a general network of providers, and Cohort 2) a network providing conservative evidence-based care for rapid resolution of pain. We used generalized linear regression modeling to estimate the comparative influence of demographic and clinical factors on expenditures.ResultsA total of 25,621 unique patients were included in the analyses. The average cost per patient for Cohort 2 (mean allowed amount $252) was lower compared to Cohort 1 (mean allowed amount $326; 0.77, 95% CI 0.75–0.79, p < .001). Patient and clinician related factors such as health plan, provider region, and sex also significantly influenced costs.ConclusionsThis study comprehensively analyzed cost data associated with the chiropractic care of adults with acute or sub-acute low back pain cared by two cohorts of chiropractic physicians. In general, providers in Cohort 2 were found to be significantly associated with lower costs for patient care as compared to Cohort 1. Utilization of a clinical model characterized by a patient-centered clinic approach and standardized, best-practice clinical protocols may offer lower cost when compared to non-standardized clinical approaches to chiropractic care.

Impact of circulating microRNA test (miRNA-371a-3p) on appropriateness of treatment and cost outcomes in patients with Stage I non-seminomatous germ cell tumours.

To determine whether utilisation of a serum microRNA (miRNA) test could improve treatment appropriateness and cost-effectiveness for patients with Stage I non-seminomatous germ cell tumours (NSGCTs). A decision tree model was built to investigate treatment course, clinical and cost outcomes for patients with Stage IA (T1N0M0S0) and IB (T2-4N0M0S0) NSGCT. The model compared outcomes and cost of standard approach using histopathology, conventional serum tumour markers and radiographic staging (standard model) to a miRNA-based approach using the standard model + post-orchidectomy serum miR-371a-3p (marker model). Probabilities of expected treatment and outcomes were based on presence/absence of cancer upon entering into the model. Overtreatment was defined as adjuvant chemotherapy or primary retroperitoneal lymph node dissection in a patient without cancer. Undertreatment was defined as initial surveillance for a patient with cancer. Utilising the miRNA marker-based approach, 26% of patients avoid overtreatment and 8% avoid undertreatment in Stage IA NSGCT; 27% avoid overtreatment and 23% avoid undertreatment in Stage IB disease. Appropriate treatment decision-making increased from 65% to 94% and 50% to 92% for Stage IA and IB, respectively. The miRNA-based approach remained cost-effective over a wide range of performance characteristics with savings of ~$1400 (American dollars)/patient for both Stage IA and IB disease. A miRNA-based approach may potentially select patients with Stage I NSGCT for correct treatment in a cost-effective manner. Identification of residual teratoma-only remains an issue. Prospective studies are necessary to validate these findings.

Timing, Costs, and Survival Outcome of Specialty Palliative Care in Medicare Beneficiaries With Metastatic Non-Small-Cell Lung Cancer.

ASCO recommends early integration of palliative care in treating patients diagnosed with metastatic lung cancer. Our study sought to examine utilization of timely specialty palliative care (SPC) and its association with survival and cost outcomes in patients diagnosed with metastatic non-small-cell lung cancer (NSCLC). The 2001-2015 SEER-Medicare data were used to determine the baseline characteristics and outcomes of 79,253 patients with metastatic NSCLC. The predictors of early SPC use were examined using logistic regression. Mean and adjusted total and SPC-related costs were calculated using generalized linear regression. We used Cox regression model to determine the survival outcomes by SPC service settings. All statistical tests were two sided. The time from cancer diagnosis to the first SPC use has reduced significantly, from 13.7 weeks in 2001 to 8.3 weeks in 2015 (P < .001). SPC use was associated with lower health care costs compared with those who had no SPC, from -$3,180 in 2011 (P < .001) to -$1,285 in 2015 (P = .059). Outpatient SPC use was associated with improved survival compared with patients who received SPC in other settings (hazard ratio, 0.83; 95% CI, 0.79 to 0.88; P < .001). Patients diagnosed with metastatic NSCLC now have more timely SPC service utilization, which was demonstrated to be a cost-saving treatment. Strategies to improve outpatient palliative care use might be associated with longer survival in patients with metastatic NSCLC.

Cost effectiveness of rituximab and mycophenolate mofetil for neuromyelitis optica spectrum disorder in Thailand: Economic evaluation and budget impact analysis.

Neuromyelitis optica spectrum disorder (NMOSD) is an inflammatory condition of the central nervous system. The extent of disability depends on the severity of the disease and the number of relapses. Although azathioprine is currently the main treatment for patients with NMOSD in Thailand, patients often relapse during its use. Hence, it is argued that there are other drugs that would be more effective. The purpose of this study is to evaluate, from a societal perspective and from the economic impact on Thailand's healthcare system, the cost utility of treatment with mycophenolate mofetil (MMF) and rituximab in patients resistant to azathioprine. The Markov model with a one-year cycle length was applied to predict the health and cost outcomes in patients with NMOSD over a lifetime. The results showed that rituximab exhibited the highest quality-adjusted life year (QALY) gains among all the options. Among the rituximab-based treatments, the administration of a rituximab biosimilar with CD27+ memory B cell monitoring proved to be the most cost-effective option. At the willingness-to-pay threshold of 160,000 Thai baht (THB), or 5,289 US dollar (USD), per QALY gained, the treatment exhibited the highest probability of being cost effective (48%). A sensitivity analysis based on the adjusted price of a generic MMF determined that the treatment was cost effective, exhibiting an incremental cost-effectiveness ratio of -164,653 THB (-5,443 USD) and a 32% probability of being cost effective. The calculated budget impact of treating patients resistant to conventional therapy was 1-6 million THB (33,000-198,000 USD) for the first three years, while after the third year, the budget impact stabilized at 3-4 million THB (99,000-132,000 USD). These data indicate that, in Thailand, treatment of drug resistant NMOSD with a rituximab biosimilar with CD27+ memory B cell monitoring or treatment with a generic MMF would be cost effective and would result in a low budget impact. Therefore, the inclusion of both the rituximab biosimilar and a generic MMF in the National Drug List of Essential Medicine for the treatment of NMOSD may be appropriate.

Stratified medicine in schizophrenia: how accurate would a test of drug response need to be to achieve cost-effective improvements in quality of life?

ObjectiveStratified medicine refers to the use of tests that predict treatment response to drive treatment decisions for individual patient. The pharmacoeconomic implications of this approach in schizophrenia are unknown. We aimed to assess the cost-effectiveness of a hypothetical stratified medicine algorithm (SMA) compared with treatment as usual (TAU), for patients with schizophrenia who failed a first-line antipsychotic.MethodsA decision analytic model with embedded Markov process was constructed, which simulated the health and cost outcomes for patients followed SMA or TAU over a lifetime horizon, from healthcare and social care perspective. In the base-case analysis, sensitivity and specificity of the stratifier were both set as 60%. Extensive sensitivity analyses were conducted to test the impact of uncertainty around the value of important parameters. The primary outcome was the incremental cost per quality-adjusted life year (QALY) gained.ResultsWhen both sensitivity and specificity of the stratified test were set at 60%, SMA appeared to be the optimal strategy as it produces more QALYs and incurs lower costs than TAU. This is robust to all scenarios tested. At a willingness-to-pay threshold of £20,000 per QALY, the probability for SMA to be the optimal strategy is 82.4%.ConclusionsOur results suggest that use of any stratifier with a sensitivity and specificity over 60% is very likely to be cost-effective comparing to TAU, for psychotic patients who failed a first-line antipsychotic. This finding, however, should be interpreted with caution due to lack of evidence for clozapine as a second-line antipsychotic.

The Impact of Obstructive Sleep Apnea on Clinical, Perioperative, and Cost Outcomes in Patients Who Underwent Posterior Cervical Discectomy and Fusion: A Single Center Retrospective Analysis from 2008 to 2016

Abstract INTRODUCTION Obstructive sleep apnea (OSA) is a chronic condition that results from upper airway narrowing during sleep, with an increasing prevalence estimated to be around 9% to 24%. The combination of an unstable, recently fused or immobile cervical spine and OSA may reasonably lead to special considerations, although this topic has not been well studied. METHODS We retrospectively obtained 1191 posterior cervical discectomy and fusion (PCDF) cases, of which 93 subjects (7.81%) had a history of OSA and 1098 subjects (92.19%) did not. The primary outcome selected was prolonged intubation. Secondary outcomes included intensive care unit (ICU) admission, overall complications, extended hospitalization, nonhome discharge, readmission within 30 and 90 d, emergency room visit within 30 and 90 d, and higher total costs. Univariate and multivariate logistic regression analyses were conducted to assess OSA-affected clinical and perioperative outcome measures. RESULTS Compared to the control cohort, the OSA cohort had more subjects with body mass index (BMI) &gt;30 (P &lt; .0001) and ASA status &gt;2 (P &lt; .0001). After controlling for age, sex, obesity, and ASA status, multivariate regression analyses revealed no difference in the odds of a prolonged intubation (P = .4092). However, a difference was noted in the odds of ICU admission (P = .0038), extended hospitalization (P = .0223), and nonhome discharge (P = .0218). Variables predictive of higher direct cost included a higher ASA status (&lt;0.0001), higher Elixhauser Comorbidities Index (ECI) score (P = .004), and more segments involved in the procedure (P &lt; .0001). Conversely, our models suggested that the 2 cohorts did not differ significantly in the odds of increased complications, readmission at 30 and 90 d, and ER visit at 30 or 90 d. CONCLUSION This study is the largest retrospective review, to our knowledge, of patients who have undergone PCDF with a specific focus on OSA. The results from this study suggest that OSA status is an important determinant of primary and secondary clinical outcomes following posterior cervical fusion procedures.

Optimizing Clinical and Cost Outcomes for Patients on Enteral Nutrition Support for Treatment of Exocrine Pancreatic Insufficiency: Proceedings from an Expert Advisory Board Meeting.

Overview of exocrine pancreatic insufficiency (EPI) and related issues The pancreas, an essential part of the gastrointestinal (GI) system, is functionally composed of exocrine and endocrine constituents. Because there is minimal anatomic separation of the exocrine and endocrine pancreatic function, disease states that cause disruption or damage in one component of the organ may lead to defects in the other component.8 EPI is a relatively uncommon but severe condition that is associated with a variety of complex disorders such as cystic fibrosis (CF), pancreatic cancer, gastric/pancreatic surgery, short bowel syndrome, and chronic pancreatitis. Physiological and biochemical effects of EPI include decreased production and secretion of lipase, increased lipase destruction and degradation, and GI motility disorders. The deficiency in pancreatic enzymes (lipase, amylase, protease) results in impaired digestion and subsequent absorption of all nutrients, fats and fat-soluble vitamins being the most clinically relevant.8 Steatorrhea, diarrhea, weight loss, abdominal discomfort, and bloating are hallmarks of EPI. In addition to lifestyle modification (eg, alcohol abstinence, frequent low-volume meals), the typical treatment for EPI is pancreatic enzyme replacement therapy (PERT), the clinical efficacy of which is inconsistent.9 Left untreated, or inadequately controlled, the condition leads to increasingly severe GI symptoms, reduced caloric intake, and inability to gain and/or maintain weight, placing the patient at risk for malnutrition. Malabsorption of fats dramatically increases the risk for malnutrition in patients with EPI.10 Because malnutrition further compromises treatment for the medical conditions underlying EPI, enteral nutrition (EN) becomes a vital therapeutic intervention for stabilizing body mass index (BMI) in some cases.3 EN formulas contain fats as well as carbohydrates, proteins, vitamins, and minerals to help prevent or address nutritional deficiencies related to malabsorption. Nutrition support therapy is more complex than commonly appreciated, particularly when prescribed for patients with EPI. Severely pancreatic insufficient patients are unable to digest and absorb long-chain triglycerides contained in EN formulas – particularly healthy fats such as docosahexanoic acid (DHA) and eicosapentaenoic acid (EPA). Currently, there is no published evidence to support the use of any Food and Drug Administration (FDA)-approved PERT products in enteral feedings, and existing clinical guidelines do not support administration of enzymes by mixing them into EN formulas.3 Factors such as care settings and nutritional formula types must be considered on a case-by-case basis, and complications must be anticipated and avoided; for example, the use of crushed PERT capsule contents in enteral feedings, or capsule contents administered directly though the feeding tube, causes tube clogging and requires frequent monitoring.11 From the patient perspective, some with CF rely on nocturnal EN for adequate caloric intake to maintain and hopefully improve BMI and lung function. Following pancreatic and other GI surgeries, EN is essential for patients who experience gastroparesis and are unable to tolerate oral meals. Lacking an appetite for food, patients with pancreatic cancer often use EN to prevent cachexia. EN is helpful for some patients with cerebral palsy who experience pain while eating solid food.

Claims data analyses unable to properly characterize the value of neurologists in epilepsy care.

To determine the association of a neurologist visit with health care use and cost outcomes for patients with incident epilepsy. Using health care claims data for individuals insured by United Healthcare from 2001 to 2016, we identified patients with incident epilepsy. The population was defined by an epilepsy/convulsion diagnosis code (ICD codes 345.xx/780.3x, G40.xx/R56.xx), an antiepileptic prescription filled within the succeeding 2 years, and neither criterion met in the 2 preceding years. Cases were defined as patients who had a neurologist encounter for epilepsy within 1 year after an incident diagnosis; a control cohort was constructed with propensity score matching. Primary outcomes were emergency room (ER) visits and hospitalizations for epilepsy. Secondary outcomes included measures of cost (epilepsy related, not epilepsy related, and antiepileptic drugs) and care escalation (including EEG evaluation and epilepsy surgery). After participant identification and propensity score matching, there were 3,400 cases and 3,400 controls. Epilepsy-related ER visits were more likely for cases than controls (year 1: 5.9% vs 2.3%, p < 0.001), as were hospitalizations (year 1: 2.1% vs 0.7%, p < 0.001). Total medical costs for epilepsy care, nonepilepsy care, and antiepileptic drugs were greater for cases (p ≤ 0.001). EEG evaluation and epilepsy surgery occurred more commonly for cases (p ≤ 0.001). Patients with epilepsy who visited a neurologist had greater subsequent health care use, medical costs, and care escalation than controls. This comparison using administrative claims is plausibly confounded by case disease severity, as suggested by higher nonepilepsy care costs. Linking patient-centered outcomes to claims data may provide the clinical resolution to assess care value within a heterogeneous population.

Treating Type 1 Diabetes Mellitus with a Rapid-Acting Analog Insulin Regimen vs. Regular Human Insulin in Germany: A Long-Term Cost-Effectiveness Evaluation.

The aim of the present study was to evaluate the cost effectiveness of rapid-acting analog insulin relative to regular human insulin in adults with type 1 diabetes mellitus in Germany. The PRIME Diabetes Model, a patient-level, discrete event simulation model, was used to project long-term clinical and cost outcomes for patients with type 1 diabetes from the perspective of a German healthcare payer. Simulated patients had a mean age of 21.5years, duration of diabetes of 8.6years, and baseline glycosylated hemoglobin of 7.39%. Regular human insulin and rapid-acting analog insulin regimens reduced glycosylated hemoglobin by 0.312 and 0.402%, respectively. Compared with human insulin, hypoglycemia rate ratios with rapid-acting analog insulin were 0.51 (non-severe nocturnal) and 0.80 (severe). No differences in non-severe diurnal hypoglycemia were modeled. Discount rates of 3% were applied to future costs and clinical benefits accrued over the 50-year time horizon. In the base-case analysis, rapid-acting analog insulin was associated with an improvement in quality-adjusted life expectancy of 1.01 quality-adjusted life-years per patient (12.54 vs. 11.53 quality-adjusted life-years). Rapid-acting analog insulin was also associated with an increase in direct costs of €4490, resulting in an incremental cost-effectiveness ratio of €4427 per quality-adjusted life-year gained vs. human insulin. Sensitivity analyses showed that the base case was driven predominantly by differences in hypoglycemia; abolishing these differences reduced incremental quality-adjusted life expectancy to 0.07 quality-adjusted life-years, yielding an incremental cost-effectiveness ratio of €74,622 per quality-adjusted life-year gained. Rapid-acting analog insulin is associated with beneficial outcomes in patients with type 1 diabetes and is likely to be considered cost effective in the German setting vs. regular human insulin.

Stratified Medicine in Schizophrenia - How Accurate Would a Test of Drug Response Need to Be to Achieve Cost-Effective Improvements in Quality of Life?

Background: Stratified medicine refers to the use of tests that predict treatment response to drive treatment decisions for individual patient. The pharmacoeconomic implications of this approach in schizophrenia is unknown. We aimed to assess the cost-effectiveness of a hypothetical stratified medicine algorithm (SMA) compared with treatment as usual (TAU), for patients with schizophrenia who failed a first-line antipsychotic. Methods: A decision analytic model with embedded Markov process was constructed based on the best available evidence. This model simulated the health and cost outcomes for patients followed SMA or TAU over a lifetime horizon, from healthcare and social care perspective. In the base-case analysis, sensitivity and specificity of the stratifier were both set as 60%. Extensive sensitivity analyses were conducted to test the impact of uncertainty around the value of all important parameters, including but not limited to sensitivity and specificity of the stratifier. The primary outcome was the incremental cost per quality-adjusted life year (QALY) gained. Outcomes: When both sensitivity and specificity of the stratified test were set at 60%, SMA appeared to be the optimal strategy as it produces more QALYs and incurs less costs than TAU. This is robust to all scenarios tested. At a willingness-to-pay threshold of £20,000 per QALY, the probability for SMA to be the optimal strategy is 82.4%.. Interpretation: Our results suggest that SMA is very likely to be cost-effective comparing to TAU, even with the relatively modest specificity and sensitivity in recently reported pharmacogenetic studies. Funding Statement: Medical Research Council, UK (grant number MR/L011794/1), and the National Institute for Health Research (NIHR) Biomedical Research Centre at South London and Maudsley NHS Foundation Trust and King's College London. Declaration of Interests: The authors declare no competing interests.

Consequences of delaying treatment intensification in type 2 diabetes: evidence from a UK database

Objective: Type 2 diabetes mellitus (TD2M) treatment focuses on achieving glycemic control, with HbA1c targeted at 6.5–7.5%. Clinicians commonly delay treatment intensification despite patients failing glycemic targets. This study evaluated longitudinal clinical and cost outcomes in patients failing metformin monotherapy using electronic medical records. Research design and methods: Adults with incident T2DM were identified in the UK Clinical Practice Research Datalink (CPRD) from 1 January 2000 to 31 March 2014. Patients were initiated on metformin monotherapy but had not reached target (HbA1c <7%). Patients were grouped by time to intensification of second-line therapy from first recorded HbA1c ≥7%: Group A, rapid intensification within 365 days; Group B, delayed intensification days 366–1824; Group C, never intensified. Patients were followed from day 366 for 5 years until end of study, switch to insulin, migration or death. Main outcome measures: The study evaluated baseline clinical and medication characteristics which were re-evaluated each year, including HbA1c, weight, cholesterol and concomitant prescribing. Results: A total of 6710 patients were included (Group A 2647, Group B 2452, Group C 1611). Group A achieved a significant decline in HbA1c at 1 year post-index date compared to Groups B and C (−1.13% Group A; +0.26% Group B, +0.16% Group C). A significantly higher proportion of patients achieved HbA1c target < 7% in Group A (Group A [45.8%]; Group B [19.1%], p < 0.0001). Using an adjusted hazard model, Group A was found to achieve the HbA1c target from the index date significantly faster than Group B (hazard ratio 3.25 [95% CI 2.87–3.69]). The most commonly prescribed second-line medications were sulfonylureas in Groups A and B throughout observation and were associated with significant weight gain (+1.3 kg per patient) in the adjusted models. Conclusions: Patients who were rapidly intensified achieved a maintained reduction in HbA1c faster than those with delayed intensification or no second-line therapy, despite a higher baseline HbA1c.

Clinical and Economic Impact of Formulary Conversion From Inhaled Flolan to Inhaled Veletri for Refractory Hypoxemia in Critically Ill Patients.

Flolan (iFLO) and Veletri (iVEL) are 2 inhaled epoprostenol formulations. There is no published literature comparing these formulations in critically ill patients with refractory hypoxemia. To compare efficacy, safety, and cost outcomes in patients who received either iFLO or iVEL for hypoxic respiratory failure. This was a retrospective, single-center analysis of adult, mechanically ventilated patients receiving iFLO or iVEL for improvement in oxygenation. The primary end point was the change in the PaO2/FiO2 ratio after 1 hour of pulmonary vasodilator therapy. Secondary end points assessed were intensive care unit (ICU) length of stay (LOS), hospital LOS, duration of study therapy, duration of mechanical ventilation, mortality, incidence of adverse events, and cost. A total of 104 patients were included (iFLO = 52; iVEL = 52). More iFLO patients had acute respiratory distress syndrome compared with the iVEL group (61.5 vs 34.6%; P = 0.01). There was no difference in the change in the PaO2/FiO2 ratio after 1 hour of therapy (33.04 ± 36.9 vs 31.47 ± 19.92; P = 0.54) in the iFLO and iVEL groups, respectively. Patients who received iVEL had a shorter duration of mechanical ventilation (P < 0.001) and ICU LOS (P < 0.001) but not hospital LOS (P = 0.86) and duration of therapy (P = 0.36). No adverse events were attributed to pulmonary vasodilator therapy, and there was no difference in cost. We found no difference between iFLO and iVEL when comparing the change in the PaO2/FiO2 ratio, safety, and cost in hypoxic, critically ill patients. There were differences in secondary outcomes, likely a result of differences in underlying indication for inhaled epoprostenol.