Methodological Approaches to Cost-Effectiveness Analysis in Saudi Arabia: What Can We Learn? A Systematic Review.

Objective: The recent establishment of the health technology assessment (HTA) entity in the Kingdom of Saudi Arabia (KSA) has resulted in increased interest in economic evaluation. The aim of this study is to evaluate the technical approaches used in published economic evaluations and the limitations reported by the authors of the respective studies that could affect the ability to perform economic evaluations in the KSA. Methods: We conducted a systematic literature review of published economic evaluations performed for the KSA over the past 10 years. An electronic literature search of the PubMed, EMBASE, and Cochrane databases was performed. A CHEERS checklist was used to assess the quality of reporting. Reported limitations were classified into domains including the definition of perspectives, identification of comparators, estimation of costs and resources, and use of the incremental cost-effectiveness ratio threshold. Results: Twelve evaluations were identified; most involved cost-effectiveness analysis (92%). Missing and unclear data were found within the CHEERS criteria. Regardless of the perspective used, most described the perspective as an "institutional" perspective (70%) and almost half were reclassified by the current reviewer (42%). Most did not clearly state the comparator (83%), and published model comparators were commonly used (50%). Resource estimation was mostly performed by the authors of the respective studies (67%), and costs were mostly obtained from hospital institutional data (75%). The lack of an established threshold for the country-specific willingness to pay was observed in 50% of the analyses. Conclusions: Economic evaluations from the KSA are limited. Capacity building and country-specific HTA guidelines could improve the quality of evaluations to better inform decision making. Highlights: Economic analysis of health technology should follow standard guidelines. Unfortunately, these guides are often underutilized, and our findings identify considerable missing, not clearly stated, or incomplete data within the analyses, which can weaken the impact of the recommendations.The limitations reported by the authors of the respective studies emphasize the suboptimal quality of the reporting. A lack of data was frequently identified and resulted in using "institutional" practice as a major source of data input for the analyses.In light of the call for the establishment of an HTA entity in the KSA, framing a standard analytic approach when conducting economic evaluations will support HTA in informing resource allocation decisions. We hope that our findings highlight the need for country-specific guidance to improve practice and enhance future research.

Thromboembolic Events Burden in Patients With Solid Tumors and Their Predisposing Factors.

INTRODUCTION: The relationship between cancers and thromboembolic events is well established. In our study, we aim to determine the burden of thromboembolic events in patients with solid tumors and identify the risk factors related to their development. MATERIALS & METHODS: Data on patients with solid tumors and thromboembolism between January 2013 and September 2014 were collected and analyzed. RESULTS: During the study period 174 patients were identified. Of which, 172 (98.9%) had venous thrombus embolism, 137 (79%) were diagnosed with deep vein thromboses, 67 (38.5%) with pulmonary embolism, 84 (48.3%) were symptomatic and 90 (51.7) were incidental at diagnosis. The most common patients and disease characteristics were female sex, high body mass index (BMI), metastatic stage, colorectal and breast primaries, and anti-neoplastic therapy. CONCLUSION:  Our study confirmed the high burden of thromboembolic events in cancer patients and the relevant factors associated with its development.

Real-World Outcome and Prognostic Factors of Pazopanib in Advanced Soft Tissue Sarcoma.

PURPOSE: Pazopanib has been approved for treating soft tissue sarcomas (STS) after chemotherapy. We aimed to evaluate the prognostic factors, clinical outcomes, and tolerability of pazopanib in patients with STS. PATIENTS AND METHODS: Forty-five patients treated between June 2015 and August 2019 were reviewed. Clinical outcome was measured by assessing the disease control rate (DCR) using Response Evaluation Criteria in Solid Tumors (version 1.1). Progression-free survival (PFS) and overall survival (OS) were estimated using the Kaplan-Meier method. Adverse effects were assessed using the Common Terminology Criteria for Adverse Events (version 5.0). RESULTS: The median age of patients at diagnosis was 28 (interquartile range (IQR), 23-45) years. Pazopanib was used as the second-line treatment in 46.7% and the subsequent line in 53.3% of patients. The overall DCR was 55.6%, and at 8 and 12 weeks, it was 52.3% and 35.5%, respectively; the median duration of response was 7 (IQR: 2-18) months. Pazopanib-induced hypothyroidism was associated with DCR, with an odds ratio of 7 (95% confidence interval [95% CI: 1.7-27.5], p<0.01). The median PFS and OS were 4.1 (95% CI: 0.85-7.42) and 12.4 months (95% CI: 6.5-18.36), respectively. Hypothyroidism and response to pazopanib, better ECOG PS, histological subtypes desmoid tumor/aggressive fibromatosis (DT/AF), and alveolar soft part sarcoma (ASPS) were favorable prognostic factors for PFS. Hypothyroidism and response to pazopanib were significant favorable factors for OS. There was no statistical difference in the OS between patients using pazopanib as the second-line therapy and those using it as the subsequent-line therapy. CONCLUSION: Pazopanib is an effective treatment for STS. However, it showed variability in the clinical outcome in favor of ASPS and an outstanding response in the DT/AF subtype. Pazopanib-induced hypothyroidism is a good prognostic factor for disease control and is associated with prolonged PFS and OS.

Empirical Monetary Valuation of a Quality-Adjusted Life-Year in the Kingdom of Saudi Arabia: A Willingness-to-Pay Analysis.

BACKGROUND: No willingness-to-pay (WTP) per quality-adjusted life-year (QALY) value exists for the Kingdom of Saudi Arabia (KSA). OBJECTIVE: The primary objective of this study was to determine the WTP for a QALY in the KSA. METHODS: Adult citizens of the KSA, patients with cancer, or members of the general public (MGP) were recruited to participate in a time trade-off survey to elicit health utilities. Cancer was chosen as the disease of interest for patients and the MGP, with a scenario describing stage 3 colorectal cancer, because it is a disease condition that impacts on both quality of life and survival time. In a second step, respondents were asked about their WTP to move from the estimated health state to a state of perfect health for 1 year (QALY). Finally, that amount was processed to generate the WTP for a full QALY. The second step was repeated with a 5-year horizon. Sensitivity analyses were performed without outliers. RESULTS: From 400 participants, data from 378 subjects were obtained and usable: 177 patients, 201 MGP; 278 male, 100 female subjects; 231 aged 26-65 years. Demographic distribution varied widely between the two subgroups for age, education level, and employment status, but with less variation in sex and income. Elicited health utilities were 0.413 (0.472 after adjustment) for the overall group, 0.316 (0.416) for patients, and 0.499 (0.508) for MGP. Overall WTP for a QALY was $US25,600 (adjusted $US32,000) for the 1-year horizon and $US19,200 (adjusted $US22,720) for the 5-year horizon. CONCLUSION: This was the first empirical attempt to estimate the WTP per QALY for the KSA. Results are comparable to those in some other countries and to gross domestic product figures for the KSA. Further research in a country-wide sample is warranted.

International HTA Experience with Targeted Therapy Approvals for Lung Cancer.

OBJECTIVES: The aim of this study was to compare the listing success rates and time incurred to listing of recently approved lung cancer medications across Australia, Canada and England. METHODS: A comparison between the three countries was performed with respect to the listing status, time incurred for listing and differences in recommendations made for cost effectiveness. Major uncertainties and limitations that compromise health technology assessment (HTA) recommendations were identified. RESULTS: The listing success rate was found to be low across all three countries (33% Canada, 17% England and 8% Australia). Across the HTA agencies' reviews, comparators were either dissimilar or altered for effectiveness and/or economic analysis. Overall, limited evidence was found for all indications, and uncertainties were identified due to indirect analyses (70%) and survival extrapolation (100%). Although most of the indications were concluded to be not cost effective, some were subsequently listed (47%) at a reduced price and/or with a specific access programme. CONCLUSIONS: This study demonstrated a low listing success rate for novel lung therapies internationally within different HTA jurisdictions. Major uncertainties that are resistant to available solutions seem to be common across different countries; thus, international solutions would be beneficial.

Utilization and cost of electronic resources in adult cancer center.

OBJECTIVES: This study aims to evaluate the knowledge of healthcare providers and the cost of the current institutional e-resources in an adult oncology setting. To assess the awareness, accessibility, and utilization of the available intranet e-resources, a survey questionnaire was distributed to all oncology healthcare practitioners (physicians, nurses, and pharmacists) in an adult oncology center. The e-resources were divided into two main categories: pre-paid and institution-specific. The cost of the pre-paid e-resources was obtained from the relevant department. The cost of the institution-specific e-resources was calculated based on the human cost spent developing these e-resources; the cost of the information technology (IT) and the organizational overhead were also taken into consideration. RESULTS: Institution-specific e-resources constituted the majority (62%) versus (38%) for pre-paid. The overall awareness, access, and frequent utilization of institution-specific e-resources, as compared to pre-paid e-resources, were low (< 50%). The cost of the institution-specific e-resources was $1,137,196, which was more than ten times higher than the pre-paid e-resources. This study identifies the general lack of awareness and utilization of institutional e-resources. The low utilization coupled with the high cost of the institution-specific e-resources makes pre-paid e-resources an attractive alternative for any institution.

Eliminating the use of intravenous glass bottles using a FOCUS-PDCA model and providing a practical stability reference guide.

OBJECTIVES: To identify the intravenous (IV) medications that are prepared in glass bottles at the institution and establish which of these medications can be prepared in flexible IV bags such as polyvinyl chloride (PVC) or non-PVC instead of glass bottles. The cost implication of switching from glass bottles to flexible IV bags was calculated. METHODS: A study using FOCUS-PDCA model to identify IV medications prepared in glass bottles and establish which of these medications could be prepared in IV bags (PVC or non-PVC). The cost impact of switching from glass bottles to IV plastic bags (including PVC or non-PVC) was calculated. The stability data obtained were used as a reference for updating pharmacy internal IV preparation charts. KEY FINDINGS: A total of 17 IV medications were found to be prepared in IV glass bottles. Of these 17 medications, only 8 (47%) were prepared in IV glass bottles due to incompatibility with PVC bags. For 7 (41%) of the medications, of which 6 were monoclonal antibodies (MABs), the reason for preparation in glass bottles was unclear as these medications are compatible with either PVC or non-PVC or both. The potential cost savings associated with switching all of the identified medications to IV plastic bags (either non-PVC or PVC) exceeded $200 000. CONCLUSIONS: The elimination of glass bottles within the institution resulted in a significant cost saving. The use of FOCUS-PDCA model can help healthcare institution achieve significant improvements in process and realize significant cost savings.

Carotid and brachiocephalic arteries stenosis with long term use of sorafenib.

The risk associated with arterial thromboembolism (ATE) increases with the presence of anti-vascular endothelial growth factor (VEGF). We are reporting a case of transient ischemic attack (TIA) due to stenosis of the carotid and brachiocephalic arteries following long-term treatment with sorafenib for renal cell carcinoma (RCC). The patient is a non-smoker with no known comorbidities and had no history of cardiovascular disease. The patient underwent a right endarterectomy with angioplasty, aortic arch, and brachiocephalic artery angiogram with a stent placed in the brachiocephalic artery.