Patient-reportedimpact of myasthenia gravis in the real world: protocol for a digital observational study (MyRealWorld MG).

INTRODUCTION: Myasthenia gravis (MG) is a rare, chronic, autoimmune disease, mediated by immunoglobulin G antibodies, which causes debilitating muscle weakness. As with most rare diseases, there is little patient-reported data with which to understand and address patient needs. This study explores the impact of MG in the real world from the patient perspective. METHODS AND ANALYSIS: This is a 2-year prospective, observational, digital, longitudinal study of adults with MG, resident in the following countries: the USA, Japan, Germany, France, the UK, Italy, Spain, Canada and Belgium. The planned sample size is 2000. Recruitment will be community based, via patient advocacy groups, social media and word of mouth. Participants will use a smartphone application (app) to check eligibility, provide consent and contribute data. Planned data entry is as follows: (1) personal profile on enrollment-covering demographics, MG characteristics and previous care; (2) monthly event tracker-current treatments, healthcare visits, treatment-related adverse events, productivity losses; (3) monthly selection of validated generic and disease-specific patient-reported outcomes instruments: EQ-5D-5L, Myasthenia Gravis Activities of Daily Living, Myasthenia Gravis Quality of Life 15-item revised scale, Hospital Anxiety and Depression Scale and Health Utilities Index III. Analyses are planned for when the study has been running in most countries for approximately 6, 12, 18 and 24 months. ETHICS AND DISSEMINATION: The study protocol has been reviewed and granted ethics approval by Salus IRB for participants resident in the following countries: Germany, the UK and the US. Local ethics approval is being sought for the following study countries: Belgium, Canada, France, Italy, Japan and Spain. Study results will be communicated to the public and participants via conference presentations and journal publications, as well as regular email, social media and in-application communication. TRIAL REGISTRATION NUMBER: NCT04176211.

Cost effectiveness of vildagliptin versus glimepiride as add-on treatment to metformin for the treatment of diabetes mellitus type 2 patients in Greece.

OBJECTIVES: This study was designed to assess the cost-effectiveness of vildagliptin versus glimepiride as add-on to metformin in the management of type 2 diabetes mellitus (T2DM) patients in the Greek healthcare setting. METHODS: A cost-effectiveness model was designed, using MS Excel, to compare two treatment strategies. Strategy 1 consisted of first-line metformin, followed by metformin + vildagliptin in second-line, while strategy 2 consisted of first line metformin, followed by metformin + glimepiride in second line. Subsequent lines were the same in both strategies and consisted of metformin + basal insulin and metformin + basal + rapid insulin. Clinical data and utility decrements relating to diabetes complications were taken from the published literature. Only direct medical costs were included in the analysis (cost base year 2014), and consisted of drug, adverse events and comorbidity costs (taken from local officially published sources and the literature). The perspective adopted was that of the Social Insurance Fund. The time horizon was lifetime, and future costs and outcomes were discounted at 3.5% per annum. RESULTS: Adding vildagliptin to metformin increased drug costs compared with adding glimepiride to metformin (€2853 vs. €2427, respectively). However, this increase was offset by a decrease in the costs of associated comorbidities (€4393 vs. €4539) and adverse events (€2757 vs. €3111), resulting in a lower total cost of €74 in strategy 1 compared with strategy 2. Comorbidities were the largest cost component in both strategies, accounting for 43.9 and 45.0% in strategies 1 and 2, respectively. Strategy 1 was also associated with increased life-years (LYs, 0.11) and quality-adjusted life-years (QALYs, 0.11) compared with strategy 2. Strategy 1 is therefore dominant, as it is associated with both lower overall costs and increased effectiveness. CONCLUSIONS: Vildagliptin as add-on treatment to metformin in the management of T2DM in Greece appears to be dominant versus. glimepiride in terms of both cost per LY and cost per QALY gained.

The direct and indirect costs of managing chronic obstructive pulmonary disease in Greece.

OBJECTIVE: COPD is associated with significant economic burden. The objective of this study was to explore the direct and indirect costs associated with COPD and identify the key cost drivers of disease management in Greece. METHODS: A Delphi panel of Greek pulmonologists was conducted, which aimed at eliciting local COPD treatment patterns and resource use. Resource use was translated into costs using official health insurance tariffs and Diagnosis-Related Groups (DRGs). In addition, absenteeism and caregiver's costs were recorded in order to quantify indirect COPD costs. RESULTS: The total costs of managing COPD per patient per year were estimated at €4,730, with direct (medical and nonmedical) and indirect costs accounting for 62.5% and 37.5%, respectively. COPD exacerbations were responsible for 32% of total costs (€1,512). Key exacerbation-related cost drivers were hospitalization (€830) and intensive care unit (ICU) admission costs (€454), jointly accounting for 85% of total exacerbation costs. Annual maintenance phase costs were estimated at €835, with pharmaceutical treatment accounting for 77% (€639.9). Patient time costs were estimated at €146 per year. The average number of sick days per year was estimated at 16.9, resulting in productivity losses of €968. Caregiver's costs were estimated at €806 per year. CONCLUSION: The management of COPD in Greece is associated with intensive resource use and significant economic burden. Exacerbations and productivity losses are the key cost drivers. Cost containment policies should focus on prioritizing treatments that increase patient compliance as these can lead to reduction of exacerbations, longer maintenance phases, and thus lower costs.

Direct and Indirect Costs of Asthma Management in Greece: An Expert Panel Approach.

OBJECTIVE: Asthma is a major cause of morbidity and mortality and is associated with significant economic burden worldwide. The objectives of this study were to map current resource use associated with the disease management and to estimate the annual direct and indirect costs per adult patient with asthma. METHODS: A Delphi panel with seven leading pulmonologists was conducted. A semistructured questionnaire was developed to elicit data on resource use and treatment patterns. Unit costs from official, published sources were subsequently assigned to resource use to estimate direct medical costs. Indirect costs were estimated as number of work loss days. Cost base year was 2015, and the perspective adopted was that of the National Organization of Health Care Services Provision, as well as the societal. RESULTS: Patients with asthma are mainly managed by pulmonologists (71.4%) and secondarily by general practitioners and internists (28.6%). The annual cost of managing exacerbations was estimated at €273.1, while maintenance costs were estimated at €1,100.2 per year. Total costs of managing asthma per patient per year were estimated at €2,281.8, 64.4% of which represented direct medical costs. Of the direct costs, pharmaceutical treatment was the key driver, accounting for 63.9 and 41.2% of direct and total costs, respectively. Direct non-medical costs (patient travel and waiting time) were estimated at €152.3. Indirect costs accounted for 28.9% of total costs. CONCLUSION: Asthma is a chronic condition, the management of which constrains the already limited Greek health care resources. The increasing prevalence of the disease raises concerns as it could translate per patient costs into a significant burden for the Greek health care system. Thus, the prevention, self-management, and improved quality of care for asthma should find a place in the health policy agenda in Greece.

Dutasteride plus tamsulosin fixed-dose combination first-line therapy versus tamsulosin monotherapy in the treatment of benign prostatic hyperplasia: a budget impact analysis in the Greek healthcare setting.

BACKGROUND: The purpose of this study was to explore the budget impact of dutasteride plus tamsulosin fixed-dose combination (DUT + TAM FDC) versus tamsulosin monotherapy, in the treatment of patients with benign prostatic hyperplasia (BPH) from the perspective of the Greek healthcare insurance system. METHODS: A Microsoft Excel-based model was developed to estimate the financial consequences of adopting DUT + TAM FDC within the Greek healthcare setting. The model, compared six mutually exclusive health states in two alternative treatment options: current standard of care and the introduction of DUT + TAM FDC in the market. The model used clinical inputs from the CombAT study; data on resource use associated with the management of BPH in Greece were derived from expert panel, and unit cost data were derived from official reimbursement tariffs. A payer perspective was taken into account. As patient distribution data between public and private sectors are not available in Greece two scenarios were investigated, considering the whole eligible population in each scenario. A 4 year time horizon was taken into account and included treatment costs, number of transurethral resections of the prostate (TURPs) and acute urinary retention (AUR) episodes avoided. RESULTS: The clinical benefit from the market adoption of DUT + TAM FDC in Greece was 1,758 TURPs and 972 episodes of AUR avoided cumulatively in a four year period. The increase in total costs from the gradual introduction of DUT + TAM FDC to the Greek healthcare system ranges from €1.3 million in the first year to €5.8 million in the fourth year, for the public sector, and €1.2 million to €4.0 million, for the private sector. This represents an increase of 1.91% to 7.94% for the public sector and 1.10% 3.29% in the private sector, during the 4-year time horizon. CONCLUSIONS: Budget impact analysis (BIA) results indicated that the gradual introduction of DUT + TAM FDC, would increase the overall budget of the disease, however providing better clinical outcomes. DUT + TAM FDC drug acquisition cost is partly offset by the reduction in the costs associated with the treatment of the disease.

Budget impact analysis of ustekinumab in the management of moderate to severe psoriasis in Greece.

BACKGROUND: The purpose of this study was to estimate the annual and per-patient budget impact of the treatment of moderate to severe psoriasis in Greece before and after the introduction of ustekinumab. METHODS: A budget impact model was constructed from a national health system perspective to depict the clinical and economic aspects of psoriasis treatment over 5 years. The model included drug acquisition, monitoring, and administration costs for both the induction and maintenance years for patients in a treatment mix with etanercept, adalimumab, infliximab, with or without ustekinumab. It also considered the resource utilization for non-responders. Greek treatment patterns and resource utilization data were derived from 110 interviews with dermatologists conducted in February 2009 and evaluated by an expert panel of 18 key opinion leaders. Officially published sources were used to derive the unit costs. Costs of adverse events and indirect costs were excluded from the analysis. Treatment response was defined as the probability of achieving a PASI 50, PASI 75, or PASI 90 response, based on published clinical trial data. RESULTS: The inclusion of ustekinumab in the biological treatment mix for moderate to severe psoriasis is predicted to lead to total per-patient savings of €443 and €900 in years 1 and 5 of its introduction, respectively. The cost savings were attributed to reduced administration costs, reduced hospitalizations for non-responders, and improved efficacy. These results were mainly driven by the low number of administrations required with ustekinumab over a 5 year treatment period (22 for ustekinumab, compared with 272 for etanercept, 131 for adalimumab, and 36 for infliximab). CONCLUSIONS: The inclusion of ustekinumab in the treatment of moderate to severe psoriasis in Greece is anticipated to have short- and long-term health and economic benefits, both on an annual and per-patient basis.

Costs and effects of paliperidone extended release compared with alternative oral antipsychotic agents in patients with schizophrenia in Greece: a cost effectiveness study.

BACKGROUND: To compare the costs and effects of paliperidone extended release (ER), a new pharmaceutical treatment for the management of schizophrenia, with the most frequently prescribed oral treatments in Greece (namely risperidone, olanzapine, quetiapine, aripiprazole and ziprasidone) over a 1-year time period. METHODS: A decision tree was developed and tailored to the specific circumstances of the Greek healthcare system. Therapeutic effectiveness was defined as the annual number of stable days and the clinical data was collected from international clinical trials and published sources. The study population was patients who suffer from schizophrenia with acute exacerbation. During a consensus panel of 10 psychiatrists and 6 health economists, data were collected on the clinical practice and medical resource utilisation. Unit costs were derived from public sources and official reimbursement tariffs. For the comparators official retail prices were used. Since a price had not yet been granted for paliperidone ER at the time of the study, the conservative assumption of including the average of the highest targeted European prices was used, overestimating the price of paliperidone ER in Greece. The study was conducted from the perspective of the National Healthcare System. RESULTS: The data indicate that paliperidone ER might offer an increased number of stable days (272.5 compared to 272.2 for olanzapine, 265.5 f risperidone, 260.7 for quetiapine, 260.5 for ziprasidone and 258.6 for aripiprazole) with a lower cost compared to the other therapies examined (euro 7,030 compared to euro 7,034 for olanzapine, euro 7,082 for risperidone, euro 8,321 for quetiapine, euro 7,713 for ziprasidone and euro 7,807 for aripiprazole). During the sensitivity analysis, a +/- 10% change in the duration and frequency of relapses and the economic parameters did not lead to significant changes in the results. CONCLUSION: Treatment with paliperidone ER can lead to lower total cost and higher number of stable days in most of the cases examined.