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To date, the economic burden and patient-reported outcomes associated with carcinoid syndrome (CS) in patients with neuroendocrine tumor (NET) remain largely unknown. The objective of this study was to perform a systematic review of economic and quality-of-life (QOL) studies related to the treatment of CS. Articles included in the review were extracted from PubMed, Embase, and the Cochrane Library. Studies had to be in English and published between 1 January 2000 and 2 July 2020. Other study eligibility criteria included patients with NET with CS receiving treatment for CS, study outcomes of cost or QOL, and clinical trials or population-based studies using claims or other secondary databases. The interventions included somatostatin analogs, telotristat ethyl, or other treatment for CS. To evaluate the quality and bias of the included studies, the 24-item CHEERS and 10-item Gill and Feinstein checklists were used. We report a narrative synthesis of the findings from the selected studies. A total of 12 economic and 12 QOL studies met the inclusion criteria and were included for review. Patients with uncontrolled CS symptoms had 23–92% higher costs than those with controlled CS; mostly, ambulatory/outpatient services were the primary drivers of the costs. The use of telotristat ethyl may be cost effective if the societal willingness to pay is as high as $US150,000 per quality-adjusted life-year in the USA. Of the 12 QOL papers, only three case–control studies assessed QOL at baseline and more than two follow-up time points. Seven studies evaluated QOL at two or more time points but lacked a control group, obscuring direct intervention effects on patients’ well-being. We observed wide variations in the reviewed studies evaluating the economic burden and patient-reported outcomes, in terms of cost and QOL, of patients with CS. Although QOL is consistently impaired and costs are consistently increased by CS, the numbers of both cost and QOL studies among this patient population remain sparse, and many of the existing studies indicated an important need for quality improvement.

We previously reported a significant volume–outcome relationship in mortality rates after gastrectomies for gastric cancer patients in Texas (1999–2001). We aimed to identify whether changes in the volume distribution of gastrectomies occurred, whether volume–outcome relationships persisted, and potential changes in the factors influencing volume–outcome relationships. We performed a population-based study using the Texas Inpatient Public Use Data File between 2010 and 2015. Hospitals were classified as high-volume centers (HVCs, > 15 cases per year), intermediate-volume centers (IVCs, 3–15 cases per year), and low-volume centers (LVCs

The economic burden of renal cell carcinoma (RCC) came into sharp focus when the UK National Institute for Health and Clinical Excellence (NICE) denied coverage (later reversed) of sunitinib for metastatic RCC. In the first of two articles that provide updated reviews and analyses of the economic burden of RCC, we conducted an updated literature review of RCC-related economic studies. We performed a literature search of PubMed, EMBASE and the Cochrane Library for English-language studies published from 1 January 2000 to 15 June 2010. We also performed a separate search for related studies in the Health Technology Assessment (HTA) reports published by the National Institute for Health Research HTA Programme in the UK. Identified articles were classified into three categories: cost studies, cost-effectiveness/cost-utility studies and cost-of-illness studies. All cost estimates were normalized to $US, year 2009 values. We identified 20 articles, including six cost, six cost-utility and eight cost-of-illness studies. In general, the studies found new surgical techniques, such as laparoscopic partial nephrectomy, to be potentially cost saving (in the range of $US181-5842). Targeted agents, such as bevacizumab, sunitinib, sorafenib and temsirolimus, were associated with higher lifetime costs ($US8537-72 254) and were not always considered to be cost effective by authors of the cost-effectiveness studies included in this review (incremental cost-effectiveness ratio [ICER]: $US49 959-272 418 per QALY). The literature reported annual estimates of the US economic burden of RCC between $US0.60 billion and $US5.19 billion, with per-patient costs of $US16 488-43 805. RCC is associated with substantial economic burden, although the estimates are wide ranging. Comparisons of the estimates across studies were hindered by variations in study methodology, choice of database and the associated timeframe, and limitations inherent to each database. More research is needed to assess the quality of the economic studies of RCC and to understand why the estimated costs differ across studies.

The incidence and prevalence of neuroendocrine tumors (NETs) have been steadily rising. NETs can arise in various parts of the body and have distinct pathogenesis, clinical manifestations, treatment, and survival compared to other neoplasms. The magnitude of the economic burden of NETs is largely unknown. This study aimed to estimate the cost of illness for NETs among elderly patients based on a large amount of observational data. We estimated the direct medical costs by phase of care using the Surveillance, Epidemiology, and End Results-Medicare data, including claims from January 1, 2002 through to December 31, 2012. Patients’ care was categorized into three phases: initial phase (first year after diagnosis), terminal phase (last year of life), and continuing phase (the period between). We estimated the cost of illness by calculating the difference in medical costs between NET patients and a matched sample from a non-cancer control group. Our study sample included 8409 elderly NET patients in the initial phase, 9218 patients in the continuing phase, and 7897 in the terminal phase. The mean cost of care for the initial phase was $46,462 in 2016 US dollars; mean cost of care for the terminal phase with a cancer-related death was $122,702; while the mean cost of care for the continuing phase was $10,457. The mean 5-year cost was $87,079. This population-based study showed that NET patients had substantial continuing phase costs and 5-year costs. Among elderly NET patients, those with pancreas as the primary cancer site had the highest costs.

Achieving comprehensive patient centricity in cost-effectiveness analyses (CEAs) requires a statistical approach that accounts for patients' preferences and clinical and demographic characteristics. Increased availability and accessibility of patient-level health-related utility data from clinical trials or observational database provide enhanced opportunities to conduct more patient-centered CEA. Regression-based approaches that incorporate patient-level data hold great promise for enhancing CEAs to be more patient centered; this paper provides guidance regarding two CEA approaches that apply regression-based approaches utilizing patient-level health-related utility and costs data. The first approach utilizes patient-reported preferences to determine patient-specific utility. This approach evaluates how individuals' unique clinical and demographic factors affect their utility and cost levels over the course of treatment. The underlying motivation of this approach is to produce CEA estimates that reflect patient-level utilities and costs while adjusting for socio-demographic and clinical factors to aid patient-centered coverage and treatment decision-making. In the second approach, patient utilities are estimated based on the clinically defined health states through which a patient may transition throughout the course of treatment. While this approach is grounded on the widely used Markov transition model, we refine the model to facilitate an enhancement in conducting regression-based analysis to achieve transparent understanding of differences in utilities and costs across diverse patient populations. We discuss the unique statistical challenges of each approach and describe how these analytical strategies are related to non-regression-based models in health services research.

Background Although the diagnosis and treatment of a primary brain tumor present unique challenges to patients and their family caregivers, evidence-based supportive care interventions are generally lacking. The primary aim of this research protocol is to determine the feasibility of implementing a dyadic yoga (DY) versus a caregiver yoga (CY) intervention or a wait-list control (WLC) group using a randomized controlled trial design. Methods Seventy-five glioma patients undergoing radiotherapy and their family caregivers are randomized to the DY, CY, or a WLC group. Patient-caregiver dyads in the DY group and caregivers in the CY group receive 15 sessions (45 min each) over the course of patients’ standard radiotherapy (6 weeks). Patients and caregivers in all groups complete baseline assessments of symptoms, quality of life (QOL), and health utilization outcomes prior to randomization. Follow-up assessments are performed 6 weeks and then again 3 months later. The primary outcome is feasibility (i.e., ≥ 50% of eligible dyads consent, ≥ 70% of enrolled dyads complete all assessments, and ≥ 50% of all practice sessions are attended). We will also perform primarily descriptive analyses of the self-reported outcomes (e.g., fatigue, overall QOL) and explore potential intervention moderators (e.g., performance status) to inform a larger future trial. Conclusion This trial will provide important information regarding the feasibility of a dyadic versus a caregiver yoga intervention regarding symptom, QOL, and health utilization outcomes in glioma patients and their caregivers. Trial registration ClinicalTrials.gov NCT02481349

Little is known about the attributes of care that most strongly impact satisfaction in real-world settings where patients’ limited medical knowledge may restrict their ability to ascertain the true quality of care. We therefore examined the association between patient-centered attributes of physician care (thoroughness, explanation, and listening), in-office waiting time, and patient satisfaction. We used the Community Tracking Study Household Survey, a US nationally representative dataset (n = 71,594). Using logistic regression models, we analyzed the association between patient ratings of care attributes and patient satisfaction for the total sample and by subgroups, according to health status, physician type, and visit type. Patients’ perception of excellent or very good care attributes was strongly associated with being very satisfied with care received (thoroughness of care, odds ratio [OR] 2.64, 95 % confidence interval [CI] 2.31–3.02; listening, OR 2.04, 95 % CI 1.77–2.36; explanation, OR 1.63, 95 % CI 1.42–1.86), as was a waiting time of ≤10 min (OR 1.50, 95 % CI 1.39–1.63). The effect magnitude of thoroughness on satisfaction is particularly strong relative to high-quality listening and explanation among respondents in poor health, and for whom the most recent office visit was to see a generalist or for curative care. Thoroughness of care was the strongest determinant of patient satisfaction, followed by physician listening and explanation. Especially with patients’ improved access to current medical information, it is important for physicians to recognize that excellent communication cannot serve as a substitute for high-quality, thorough care.

Over the last 15 years, a paradigm shift in oncology has led to the approval of dozens of targeted oral anti-cancer medications (OAMs), which have become the standard of care for certain cancers. While more convenient for patients than infused drugs, the possibility of non-adherence and the frequently high costs of targeted OAMs have proven controversial. Our objective was to perform the first comprehensive review of cost-effectiveness analyses (CEAs) of targeted OAMs. A literature search in PubMed, The Cochrane Library, and the Health Technology Assessment (HTA) reports published by the National Institute for Health Research HTA Programme in the UK was performed, covering articles published in the 5 years prior to 30 September 2013. Our inclusion criteria were peer-reviewed English-language full-text original research articles with a primary focus on CEA related to targeted OAMs. We categorized these articles by treatment setting (i.e. cancer site/type, line of therapy, and treatment and comparator) and synthesized information from the articles into summary tables. We identified 41 CEAs covering nine of the 18 targeted OAMs approved by the US FDA as of December 2012. These medications were studied in seven cancers, most often as second-line therapy for advanced-stage patients. In over half of treatment settings where a targeted OAM was compared with treatment that was not a targeted OAM, targeted OAMs were considered cost effective. Limitations in interpreting these findings include the risk of bias due to author conflicts of interest, cross-country variation, and difficulties in generalizing clinical trial evidence to community practice. Several types of cost-effectiveness studies remain under-represented in the literature on targeted OAMs, including those for follow-on indications approved after the initial indication for a drug and for off-label indications, head-to-head comparisons of targeted OAMs with other targeted OAMs and targeted intravenous therapies, and studies that adopt a perspective other than the payer’s. Keeping up with the increasing number of approved targeted OAMs will also prove an important challenge for economic evaluation.

Asthma is one of the most common chronic diseases in the US, and its prevalence continues to increase. Despite the availability of effective asthma controller medications, many patients with asthma are still not meeting therapeutic goals because of poor disease management. The high disease prevalence combined with the high costs associated with the poor management of asthma, make patients with asthma a costly group to treat for managed care organisations (MCOs) and this motivates decision makers in MCOs to consider both the clinical and economic value of asthma therapies.To compare the cost effectiveness of first-line controller asthma therapies in patients with mild-to-moderate persistent asthma from an MCO payer perspective.A decision-analysis model was developed to evaluate the cost effectiveness of fluticasone propionate and salmeterol administered in a single inhaler (salmeterol/fluticasone propionate 50/100microg), compared with fluticasone propionate inhaled corticosteroids (FPIC), non-fluticasone propionate inhaled corticosteroids (nFPIC) and leukotriene modifiers. The model estimated costs ($US, year 2005 values) and health outcomes over a 1-year period. Costs and outcomes data were obtained from published clinical trials and observational studies, and model assumptions on the relationship between adherence and effectiveness were evaluated by a panel of experts. Effectiveness measures included symptom-free days and rescue medication-free days. The cost effectiveness of first-line asthma therapies was compared using a step-wise approach, with FPIC as the reference case. Both one-way and probabilistic sensitivity analyses were performed to assess the robustness of results over a range of assumptions.The step-wise comparison found that the additional costs for achieving an incremental effectiveness unit (incremental cost-effectiveness ratio) using single-inhaler salmeterol/fluticasone propionate compared with FPIC was $US9.55 per symptom-free day and $US8.93 per rescue medication-free day. Sensitivity analyses indicated that the model was robust to changes in base-case assumptions. A probabilistic sensitivity analysis showed that, corresponding to a benchmark value of $US14.8 per symptom-free day, the probabilities that single-inhaler salmeterol/fluticasone propionate, n-FPIC and leukotriene modifiers were more cost effective than FPIC were 98%, 30.7% and 2.1%, respectively.Based on our decision analysis, the additional costs for achieving incremental effectiveness with single-inhaler salmeterol/fluticasone propionate treatment compared with FPIC and nFPIC may be lower than the commonly accepted benchmark value for cost effectiveness, based on published estimates of the utility losses associated with asthma symptoms. Single-inhaler salmeterol/fluticasone propionate may also be more cost effective than leukotriene modifiers.

This study explored the relationship between the M. D. Anderson Symptom Inventory (MDASI), an instrument measuring the severity of symptoms common to patients with cancer, and utility derived from the SF-36.Cancer patients from Tianjin Cancer Hospital in China (n = 249) completed a demographic questionnaire and Chinese versions of the MDASI and SF-36. Using a published algorithm converting SF-36 scores to standard gamble (SG) utilities, we examined the association between utility and individual symptoms using Spearman's rank correlation, and explored the association between utility and aggregate symptom scores through multivariate regression analyses.The mean SG utility was 0.81 (SD = 0.11); utilities were significantly but moderately correlated with the majority of symptoms, especially those of distress, sadness, fatigue, and pain. Regression models showed a significantly negative association between the total symptom score and the utility. After controlling for sociodemographics, cancer stage and performance status, a significantly negative association between the total symptom scores and utility was found in the multivariate analyses. We also found the total number of severe symptoms to be a stronger predictor of "disutility."Symptom measures were significantly albeit moderately associated with utility derived from the SF-36 scores, suggesting that a full study with rigorously collected utilities is worth exploring.

Health impairment often leads to work impairment in the form of both absenteeism and presenteeism (i.e. reduced productivity while at work). Several self-report productivity instruments have been designed over the past few years to measure the impact of illness on productivity at work and/or in non-work activities. In a review of the literature we identified six generic subjective instruments - the Endicott Work Productivity Scale, Health and Labor Questionnaire, Health and Work Questionnaire, Health and Work Performance Questionnaire, Work Limitations Questionnaire (WLQ) and the Work Productivity and Activity Impairment Questionnaire (WPAI) - that could theoretically be used in any working population. These instruments were usually validated against other subjective measures (such as health-related QOL). Each productivity instrument has benefits in certain research settings, but the psychometric properties of the WPAI have been assessed most extensively. It was the most frequently used instrument and has also been modified to measure productivity reductions associated with specific diseases (e.g. allergic rhinitis, gastro-oesophageal reflux disease, chronic hand dermatitis). The WLQ has also been tested extensively to measure the general health impact and impact of specific conditions. Two migraine-specific subjective instruments were also identified: the Migraine Disability Assessment questionnaire and the Migraine Work and Productivity Loss Questionnaire, of which the latter was found to have better psychometric properties. Productivity outcomes are useful in that they characterise the impact of an illness in the workplace and show the effect of treatment on productivity. Evidence of psychometric properties and generalisability of different instruments was found to a varying degree. Thus, further research is needed to assess the accuracy and usefulness of individual instruments in certain research settings. Health-related productivity has been increasingly recognised as an important component of the burden of illness associated with a given disease; without it, one cannot reliably assess this burden.

This study uses national databases to examine the impact of irritable bowel syndrome (IBS) on resource utilization in the United States. Approximately 1.5–2.7 million physician visits (599–1043 per 100,000) yearly were related to IBS, with 45.3% seen by gastroenterologists, and 89% prescribed medications. Rates of physician visits by women were approximately 2.4–3.3 times higher than that for men. The average number of medication prescribed per visit was 1.83. Approximately 89% of the visits were prescribed with medications. The rate of hospitalization (5.1 per 100,000 in 1997) decreased by 60% and length of stay decreased from 5.5 to 3.1 days in the past decade. The average charges of IBS-related hospitalization were US$7,882. Our study found an apparent decreasing trend of IBS-related hospitalizations and no marked increase in office consultations in the past decade. However, a better case identification criterion is necessary to estimate the true disease burden.

The prevalence of depression and the high costs associated with its treatment have increased interest in pharmacoeconomic evaluations of drug treatment, particularly in the 1990s as the use of selective serotonin (5-hydroxytryptamine; 5-HT) reuptake inhibitors (SSRIs) expanded substantially. This review presents results from specific studies representing the key study designs used to address the pharmacoeconomics of SSRI use: retrospective administrative database analyses, clinical decision analysis models, and randomised clinical trials. Methodological considerations in interpreting results are highlighted. In retrospective administrative database analyses, most comparisons have been made between SSRIs and tricyclic antidepressants (TCAs). A few studies have addressed differences between SSRIs. The studies focused on healthcare cost (to payer) and cost-related outcomes (e.g. treatment duration, drug switching). Although SSRIs are generally associated with higher drug acquisition costs than are TCAs, total healthcare costs are at least offset, if not decreased, by reductions in costs associated with use of SSRIs. Although studies from the early 1990s show some advantage for fluoxetine, the results are limited by use of data from shortly after the introduction of paroxetine and sertraline; studies from the mid- 1990s on that compare drugs within the SSRI class show general equivalence in terms of cost. Important methodological advances are occurring in retrospective studies, with selection bias and other design limitations being addressed statistically. Clinical decision analysis models permit flexibility in terms of ability to specify different alternative treatment scenarios and varying durations. Sensitivity analysis aids interpretability, although model inputs are limited by data availability. Results from short term (1 year duration or less) studies comparing SSRIs and TCAs suggest that SSRIs are more cost effective or that there is no difference. Longer term studies (lifetime Markov models) focus more on the impact of maintenance antidepressant therapy and show more mixed results, generally favouring SSRIs over TCAs. The results indicate that the effect of SSRIs is mainly through prevention of relapse. Important assumptions of these models include fewer serious adverse effects and lower treatment discontinuation rates with SSRIs. Naturalistic clinical trials provide greater generalisability than traditional randomised clinical trials. One naturalistic trial found that nearly half of TCA-treated patients switched to another antidepressant within 6 months; only 20% of SSRI-treated patients switched. Cost differences between groups were minimal. These studies indicate few differences in medical costs, depression outcomes and health-related quality of life between TCAs and fluoxetine, although fewer fluoxetine-treated patients switched treatment.

The choice of evidence used in decision modelling of healthcare interventions divides analysts into 2 groups: (i) those who favour randomised clinical trial (RCT) data; and (ii) those who prefer 'real world' data. This preference may have serious consequences if the end result is to inform healthcare policy. This paper uses Medicare coverage of epoetin-alpha [erythropoietin (EPO)] as a case study to illustrate a technique which can be used to overcome some of the bias inherent in RCT data while avoiding some of the common pitfalls associated with the use of observational data.Cost analysis of 2 treatments for anaemia of renal failure primarily in an outpatient setting is modelled in a decision tree. This method can be used to analyse healthcare interventions or policies in any setting.Patients with nontransplanted end-stage renal disease (ESRD) who received either EPO or blood transfusion for treatment of anaemia at any time during the 1-year study period (July 1989 to June 1990) were included in the sample.Outcome effects in the natural setting are decomposed into 2 parts: a treatment effect and a population effect. This is then extended to the special case of policy analysis. Logistic and multiple regression are used to estimate branch probabilities and payoffs, respectively, for 2 treatment options.Under standard methods of decision analysis, an increase of $US7032 per patient following EPO coverage is observed. With the decomposition technique, the policy effect is estimated to be less, $US6172, the difference coming from the population effect.Failure to remove population effects from observed outcome effects may lead to biased decision-making. Although not directly observable, the population effect can be imputed from secondary data. The decomposition and imputting technique allows for a more meaningful interpretation of the results for the purpose of policy analysis.