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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.

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.

Introduction: The economic burden of renal cell carcinoma (RCC) came into sharp focus when the UK's National Institute for Health and Clinical Excellence denied coverage (later reversed) of sunitinib for metastatic RCC. Following an updated review of RCC-related economic studies, we supplemented the costs of RCC reported in the literature with estimates from the latest US databases that capture the utilization of several newly approved targeted agents. Method: We conducted analyses using the 1991-2007 SEER (Surveillance, Epidemiology and End Results)-Medicare and 1996-2007 MarketScan Commercial Claims and Encounter (CCAE) and Medicare Supplemental databases, and based our estimates on a prevalent cohort of patients with RCC or kidney cancer constructed from each database. All cost estimates were normalized to $US, year 2009 values. The incremental costing approach was applied to estimate the annual cost of RCC by treatment phases using a prevalent cohort of patients with RCC identified from the 2005 SEER-Medicare database. We used the method of extended estimation equations to estimate the impact of patients' use of targeted therapies on the annual costs of RCC, while controlling for confounding factors such as patients' age, sex, tumour characteristics, co-morbidity and geographic regions. The method was applied to two elderly cohorts of RCC patients identified from the SEER-Medicare and the MarketScan Medicare Supplemental databases and a non-elderly cohort of patients with RCC identified from the MarketScan CCAE database. Results: Compared with the cost of treating an elderly, non-cancer patient in the matched sample, the average cost of treating an elderly patient with RCC was $US11 169 (95% CI 10 683, 11 655) more per year, based on our analyses of the latest SEER-Medicare data. The annual cost to treat patients with RCC who received targeted therapies was 3- to 4-fold greater than the cost to treat patients with RCC who received other therapies. Results from the multivariate analysis showed that, after controlling for potential confounders, the annual medical cost was $US31 000-65 000 higher for RCC patients treated with targeted therapies, with the largest increase observed among the non-elderly patients. Conclusion: The economic burden of RCC is likely to grow with an increasing use of targeted therapies. Future research is needed to understand the impact of various forces on the economic burden of RCC, such as increased disease incidence, use of minimally invasive surgical techniques and more prevalent adoption of emerging targeted therapies.

Background: Health information technology offers a powerful tool to monitor the performance of a healthcare system. Advances in computer technology and capacity combined with lower start-up costs will allow developing countries to achieve greater impact when they initiate electronic health information systems. We focused on the integrated health information system that was established in Taiwan in conjunction with the launch of the National Health Insurance (NHI) programme. We used data from that health information system to conduct a cost-effectiveness analysis of chemotherapy use among breast cancer patients. We then used this analysis to discuss what policy makers can learn from this type of analysis. Methods: We identified a cohort of patients in the NHI Research Database who had been diagnosed with breast cancer in 2001 and had received chemotherapy following surgical removal of the tumour. We followed these patients for 3 years and conducted a cost-effectiveness analysis from the payer's perspective. Using the net benefit regression approach, we compared the cost effectiveness of the two most commonly prescribed first-line chemotherapy regimens for the treatment of breast cancer in 2001 in Taiwan. The dependent variable of the regression model was the individual-level net benefit, and the independent variables included a binary variable indicating the choice of chemotherapy regimen, the patients' age, co-morbidity, type of surgery, geographic region and type of treatment facility. We employed both frequentist and Bayesian approaches in our net benefit regression analyses. In the Bayesian analysis, we applied non-informative priors to all parameters in the base-case analyses. We then explored the use of informative priors in the sensitivity analysis, using cost-effectiveness data published in the literature to form the prior distributions for the relevant parameters. Results: Over 60% of surgically treated breast cancer patients received either CMF (cyclophosphamide, methotrexate, fluorouracil) or CEF (cyclophosphamide, epirubicin, fluorouracil). A comparison of patient characteristics indicated that patients in the CEF group tended to be younger (47.8 vs 49.1 years; p - 0.016), and were significantly more likely to have undergone a mastectomy (84% vs 76%; p < 0.001) and to have been treated in a teaching hospital (26% vs 13%; p < 0.001). We also observed significant variations in geographic region of the location of facilities between treatment groups. On average, CEF was not cost effective in the treatment of patients with breast cancer in Taiwan, although analyses stratified by geographic region suggested a wide variation across regions. At a societal willingness to pay (WTP) of new Taiwanese dollar (&dollar;NT)1 500 000 (&dollar;US80 000), the probability that CEF was more cost effective than CMF was 0.0%, 0.0%, 0.0% and 3.9% for the Taipei metropolitan area, and the north, middle and the combined south and east region, respectively; the probability became 0.6%, 0.0%, 1.3% and 54.5%, respectively, at a WTP of &dollar;NT5 000 000 (&dollar;US270 000). After co-variate adjustments, the probabilities were 0.0%, 0.0%, 0.0% and 0.8%, respectively at a WTP of &dollar;NT1 500 000, and were 0.0%, 0.0%, 1.4% and 34.7% at &dollar;NT5 000 000. Sensitivity analyses showed that CEF potentially could have been more cost effective than CMF within a reasonable range of societal WTP (i.e. &dollar;NT1 000 000-3 000 000 or &dollar;US55 000-160 000) had the optimal dosage level for CEF been established for breast cancer patients in Taiwan. Conclusions: A population-based, fully integrated electronic health information system provides useful data to assess the cost effectiveness of competing treatments and interventions in current practice. This research may potentially inform policy makers of modifications that can be instituted to improve the cost effectiveness of a new therapy. However, findings from this study need to be interpreted with caution because the study provided information only on the short-term cost effectiveness (i.e. 3 years) of CEF compared with CMF. It is possible that a future analysis will reach a different conclusion when more years of follow-up data become available.