Potential impact on cost-effectiveness estimates of using immature survival data: a case study based on transcatheter edge-to-edge repair (TEER) used for patients with severe mitral regurgitation at high surgical risk.

OBJECTIVE: To review the survival modelling used in cost-effectiveness studies evaluating an interventional procedure and to discuss implications for decision-makers. DESIGN: A case study of three economic evaluations that each used immature data from the EVEREST II High Surgical Risk (HSR) Study of transcatheter edge-to-edge repair (TEER) for patients with severe mitral regurgitation (MR) who were at high risk of surgery. SETTING: Estimation of patient survival in cost-effectiveness studies. PARTICIPANTS: The EVEREST II HSR Study included 78 patients who had TEER of the mitral valve using the MitraClip device and a retrospectively identified control group of 36 patients who received medical management and were followed up for 12 months. Observed survival (TEER arm only) was updated at 5 years. RESULTS: Two studies used 12-month observed mortality from EVEREST II HSR to model survival over lifetime horizons. Observed and modelled survival were associated with considerable uncertainty due to short follow-up and small numbers of participants. Modelling control patients' survival required an approximate 10-fold extrapolation based on 12-month observation of only 38 patients. Observed 5-year survival in the TEER group differed from that less mature follow-up suggesting that survival modelling based on shorter follow-up was unsatisfactory. No public domain data for the control group are available beyond 12-month follow-up so meaningful estimates using mature data for both arms are currently not possible. A third study developed survival models using incompletely reported transitions between MR grades in EVEREST II HSR and mortality rates observed for different MR grades derived from a study in an unrelated population. CONCLUSIONS: Modelling survival in such small samples followed up for only 12 months is associated with great uncertainty, and cost-effectiveness results based on these analyses should be viewed as premature and used cautiously in reimbursement decisions.

Knowledge of Antibiotics Use among Lebanese Adults: A study on the influence of sociodemographic characteristics.

OBJECTIVES: This study aimed to evaluate the association between sociodemographic characteristics and antibiotic knowledge in the Lebanese population. METHODS: A questionnaire-based survey was conducted in community pharmacies across all Lebanese governorates. Data were collected by well-trained pharmacists through face-to-face interviews from January until March 2017. The survey tool was adapted from a questionnaire developed by the World Health Organization. A knowledge index was computed for comparative purposes and a linear regression model was performed to assess factors associated with knowledge. RESULTS: A total of 623 participants were included in the analysis of this study (response rate: 90.6%). The mean antibiotic knowledge index was 12.5 ± 3.2 (minimum score: 3 and maximum score: 19). Higher knowledge score was inversely correlated with age (r = -0.118; P = 0.003), but no gender differences were reported (females: 12.6 versus males: 12.3; P = 0.191). However, statistically significant differences were found for residence type (P = 0.002), educational level (P <0.001) and total household income categories (P <0.001). The linear regression model showed a significant association between residence type and knowledge (urban versus rural: ß = 0.793; P = 0.011). Furthermore, a higher knowledge index was significantly associated with a higher income combined with higher education (additive scale/ß = 1.590; P = 0.025). Finally, interactions between income and age, gender and residence type were not significant. CONCLUSION: Individuals residing in urban areas, with combined high income and educational levels, are more knowledgeable about antibiotics use and resistance compared to other groups. More studies are needed to assess the interaction of sociodemographic interactions with health literacy.

Ocrelizumab not recommended in France for patients with primary progressive multiple sclerosis while recommended in England: a review comparing the assessment by HAS and NICE.

INTRODUCTION: Ocrelizumab is the first approved drug for primary progressive multiple sclerosis. Following appraisal by health technology assessment (HTA) bodies, this medicine has not been widely covered across European countries. We have compared the HTA process in England and France. AREA COVERED: We undertook an analysis of relevant documents that were published by the two HTA bodies. We analyzed patients' availability of Ocrelizumab at the different stages of the process. EXPERT OPINION: We identified differences in the assessment, one being the use of a different population of the pivotal trial, which has resulted in the consideration of distinct clinical effectiveness estimates. Ocrelizumab became available earlier in France as part of an early access program. However, rapid access was discontinued for newly eligible patients following an opinion concluding that Ocrelizumab yielded no additional benefit over placebo. This opinion was not compatible with the criteria allowing reimbursement in France.In England, there was no early access program and following an appraisal that included cost-effectiveness evaluation combined with pricing agreements, medicine was finally recommended. In conclusion, differences in the HTA process may result in appreciable differences in timing and outcome from marketing authorization to the adoption of newly licensed drugs.

Current Trends in Hospital Pharmacy Practice in Lebanon.

Objectives: For decades, the role of hospital pharmacists has been instrumental in elevating pharmacy practice worldwide. Recently, the Hospital Pharmacy Section of the International Pharmaceutical Federation (FIP), the European Association of Hospital Pharmacists (EAHP), and the American Society of Health-System Pharmacists (ASHP) updated their statements about the future role and responsibilities of the pharmacy executive in hospitals and health systems. A series of surveys were conducted around the globe to better understand the current state of hospital pharmacy practice. The purpose of these surveys was to identify challenges in hospital pharmacy practice and to develop improvement strategies. The objective of this national survey is to evaluate hospital pharmacy practice in Lebanon. Methods: A cross-sectional observational study was performed among pharmacists working in hospital settings in Lebanon, from January through June 2016. Based on a literature review, a questionnaire to elicit Lebanese hospital pharmacists' practice was developed. Results: The results showed a nonsignificant difference between university teaching and nonuniversity teaching hospitals in the processes of drug procurement, preparation, dispensing, and drug administration. However, statistically significant differences were observed between university teaching and nonuniversity teaching hospitals with respect to having clinical pharmacists (P < .001) and highly qualified personnel (P < .005). Pharmacy services in teaching hospitals seemed to be more advanced cooperating with affiliated medical schools. Furthermore, teaching hospitals were more likely to have pharmacists providing information about the safety of the medications used (P = .029). Although not statistically significant, there was a higher trend toward having a designated champion for medication safety (P = .052). Conclusion: The results of our survey showed that teaching hospitals were more compliant with the International Statements of Hospital Pharmacy Practice compared with nonteaching hospitals. There is room for improvement especially if the application of the accreditation standards for safe hospital pharmacy practice becomes mandatory for all hospitals, which is expected to standardize pharmacy practice and secure both medication and patient safety.

Management of patients with metastatic colorectal cancer in Lebanese hospitals and associated direct cost: a multicentre cohort study.

BACKGROUND: For metastatic colorectal cancer a series of novel therapies has emerged during the last decade but their use in routine clinical practice and their costs are not well documented. AIMS: This study evaluated the clinical effectiveness of metastatic colorectal cancer patients in Lebanese oncologic units and estimated the costs. METHODS: A prospective cohort study was conducted on metastatic colorectal cancer patients during 2008-2013. The type of medical management, overall survival and total costs from diagnosis to death were described. Cost analysis was performed using tariffs from 2013 in US dollars. RESULTS: One hundred and seventy-nine metastatic patients were selected among which 84.9% had colorectal cancer involvement. The average follow-up from diagnosis until death or the latest news was 34.8 months. Around 49.7% were still alive at last follow-up date. Three lines of treatment accounted for 4.5%, 39.6% and 55.9% with an average duration of 14.5, 11.4 and 14.6 months respectively. A 73.2% of patients benefited from targeted therapy. The median overall survival was 20.8 months. The mean total costs of drugs was $22 256 in patients with standard therapy alone whereas the cost increased to $80 396 after the addition of targeted therapy. The mean global total cost was estimated at $64 805 per patient (min $3703; max $304 086). CONCLUSIONS: Targeted therapy associated to standard therapy is highly prevalent in Lebanon in metastatic disease and the associated medical cost substantial. This study is the first to show the clinical effectiveness and costs of targeted therapy in patients with metastatic colorectal cancer.

International normalized ratio self-testing and self-management: improving patient outcomes.

Long term oral anti-coagulation with vitamin K antagonists is a risk factor of hemorrhagic or thromebomlic complications. Periodic laboratory testing of international normalized ratio (INR) and a subsequent dose adjustment are therefore mandatory. The use of home testing devices to measure INR has been suggested as a potential way to improve the comfort and compliance of the patients and their families, the frequency of monitoring and, finally, the management and safety of long-term oral anticoagulation. In pediatric patients, increased doses to obtain and maintain the therapeutic target INR, more frequent adjustments and INR testing, multiple medication, inconstant nutritional intake, difficult venepunctures, and the need to go to the laboratory for testing (interruption of school and parents' work attendance) highlight those difficulties. After reviewing the most relevant published studies of self-testing and self-management of INR for adult patients and children on oral anticoagulation, it seems that these are valuable and effective strategies of INR control. Despite an unclear relationship between INR control and clinical effects, these self-strategies provide a better control of the anticoagulant effect, improve patients and their family quality of life, and are an appealing solution in term of cost-effectiveness. Structured education and knowledge evaluation by trained health care professionals is required for children, to be able to adjust their dose treatment safely and accurately. However, further data are necessary in order to best define those patients who might better benefit from this multidisciplinary approach.

MANAGEMENT OF METASTATIC COLORECTAL CANCER: Current Treatments and New Therapies.

Colorectal cancer represents 8% of metastatic cancers. For decades, the gold standard therapy has been infusional chemotherapy with 5-Fluorouracil (5-FU) associated to folinic acid. The discovery of irinotecan, oxaliplatin and oral forms of 5-FU in the nineties is considered a milestone in the treatment of this disease. Since 2004, targeted therapy with monoclonal antibodies including anti-EGFR and angiogenesis inhibitors showed superiority in terms of mortality compared to conventional therapy. Metastatic colorectal cancer, however, remains an incurable disease. We present the current treatments of metastatic colorectal cancer, the clinical development of these emerging treatments, and their position in the Lebanese health care system.