[Horizon Scanning Of Therapeutic Alternatives For Generalized Myasthenia Gravis And Five-Year Healthcare Expenditure Forecast Model]

OBJECTIVE: The objective of the analysis was to assess and quantify the economic impact of generalized Myasthenia Gravis (gMG) in Italy over the next five years (2025—2029) using Horizon Scanning and expenditure forecasting methodologies.METHODS: The analysis is characterized by conducting Horizon Scanning of therapies for gMG that will be reimbursed in the Italian market over the next five years (2025—2029), followed by the development of a disease expenditure model for the same time frame. The Horizon Scanning methodology for gMG included research on ClinicalTrials.gov to identify relevant Phase 2 or 3 studies. A forecasting model was developed to evaluate the impact of new therapies for gMG from the perspective of the Italian National Health Service, considering total patient management costs. The analysis included acquisition and administration costs of treatments, as well as costs for managing exacerbations and myasthenic crises. The assumptions were validated by an expert panel.RESULTS: Horizon Scanning identified 14 new drugs for gMG in clinical development, with 7 of these expected to enter the Italian market between 2025 and 2029. The expenditure forecast analysis results indicate a slight increase in gMG treatment costs, stable administration costs and a slight reduction in costs for managing exacerbations and myasthenic crises. Total expenditure is projected to rise by 10.3%, from approximately €126 million in 2025 to €139 million in 2029.CONCLUSIONS: Through the Horizon Scanning and expenditure forecasting, it was estimated that, with the arrival of new therapeutic alternatives for gMG on the market, despite the high initial cost of these new treatments, their greater effectiveness could reduce the costs for managing exacerbations and myasthenic crises. This analysis provides essential information to improve the clinical management of the disease, optimize the allocation of healthcare resources and ultimately enhance the quality of life of patients with gMG

Treatments of Advanced Non‑Small Cell Lung Cancer (NSCLC) in an Italian Center: Drug Utilization and the Treatment Costs of Innovative Drugs

AIM: To provide an updated picture of the therapies most commonly used in the advanced Non-Small Cell Lung Cancer (NSCLC) setting, together with the relevant costs.METHODS: This study considered the clinical records of patients affected by stage IIIb and IV NSCLC treated in the AORN dei Colli - Plesso Monaldi in Naples during the period January 2016-July 2017 and diagnosed since 2014, as well as the pathology lab database. Multivariate analyses were performed in order to identify the main predictors of time to next treatment and the main cost drivers.RESULTS: Data were collected on 575 patients, who were mainly affected by adenocarcinoma (62%) and squamous cell carcinoma (34%). 64% of patients were reported having been tested for molecular biomarkers (among the patients tested, 13% were EGFR+, 4% Alk t, and 1% ROS1 t). In accordance with the international guidelines, chemotherapy – as single agent or platinum-based doublets – was the prevalent first-line treatment, except among EGFR+ and ROS1 t patients, for whom the target therapy was authorized as first-line therapy. As second-line treatment, the target therapy and immune checkpoint inhibitors (nivolumab) were the most commonly used treatments. Drug expenditure per patient was remarkably higher in mutated patients (€ 29,053) versus wild-type patients, or patients with unknown mutational status (€ 11,854), who received just chemotherapy. The costs sustained in 2017 are proportionally higher than those sustained in 2016, mainlydue to the increasing eligibility to target therapy and immune checkpoint inhibitors and the wider biomarker analysis performed. From multivariate analyses, among the predictors of a longer time to next treatment (TTNT) were a better performance status and target therapy both in first and second line. The therapy for squamous cell carcinoma and other nonadeno histotypes turned out to be less expensive in patients treated just in the first line than that for adenocarcinoma and adenosquamous carcinoma. The use of immune checkpoint inhibitors in the second line results in increased costs compared to the use of chemotherapy. Also the target therapy in the first line results in an increase in the total costs with respect to chemotherapy in patients who received just a first-line therapy.CONCLUSIONS: Generally, in this study population, the treatments administered are in accordance with the international guidelines. The costs borne by the Health Systems are higher for the target therapy and the immune checkpoint inhibitors

Treatments of Advanced Non‑Small Cell Lung Cancer (NSCLC) in an Italian Center: Drug Utilization and the Treatment Costs of Innovative Drugs

AIM: To provide an updated picture of the therapies most commonly used in the advanced Non-Small Cell Lung Cancer (NSCLC) setting, together with the relevant costs.METHODS: This study considered the clinical records of patients affected by stage IIIb and IV NSCLC treated in the AORN dei Colli - Plesso Monaldi in Naples during the period January 2016-July 2017 and diagnosed since 2014, as well as the pathology lab database. Multivariate analyses were performed in order to identify the main predictors of time to next treatment and the main cost drivers.RESULTS: Data were collected on 575 patients, who were mainly affected by adenocarcinoma (62%) and squamous cell carcinoma (34%). 64% of patients were reported having been tested for molecular biomarkers (among the patients tested, 13% were EGFR+, 4% Alk t, and 1% ROS1 t). In accordance with the international guidelines, chemotherapy – as single agent or platinum-based doublets – was the prevalent first-line treatment, except among EGFR+ and ROS1 t patients, for whom the target therapy was authorized as first-line therapy. As second-line treatment, the target therapy and immune checkpoint inhibitors (nivolumab) were the most commonly used treatments. Drug expenditure per patient was remarkably higher in mutated patients (€ 29,053) versus wild-type patients, or patients with unknown mutational status (€ 11,854), who received just chemotherapy. The costs sustained in 2017 are proportionally higher than those sustained in 2016, mainlydue to the increasing eligibility to target therapy and immune checkpoint inhibitors and the wider biomarker analysis performed. From multivariate analyses, among the predictors of a longer time to next treatment (TTNT) were a better performance status and target therapy both in first and second line. The therapy for squamous cell carcinoma and other nonadeno histotypes turned out to be less expensive in patients treated just in the first line than that for adenocarcinoma and adenosquamous carcinoma. The use of immune checkpoint inhibitors in the second line results in increased costs compared to the use of chemotherapy. Also the target therapy in the first line results in an increase in the total costs with respect to chemotherapy in patients who received just a first-line therapy.CONCLUSIONS: Generally, in this study population, the treatments administered are in accordance with the international guidelines. The costs borne by the Health Systems are higher for the target therapy and the immune checkpoint inhibitors.</jats:p

Project R. Evolution for improving clinical research in Italy: challenges and strategies

The R.Evolution project aimed to reach a consensus on the main challenges of conducting clinical research in Italy and possible strategies and approaches to address them and optimize clinical research management. A scientific board of experts initially discussed potentially critical areas in clinical research conduct and further explored them through an online national survey. The survey results were further examined by a group of 35 panelists representing different clinical research stakeholders. A Nominal Group Technique and a Delphi approach (two rounds) were used to generate a consensus on critical factors, tools and strategies in clinical research. Four main critical areas were identified: study feasibility, authorization procedures, operational aspects and patient management. The main issues are scarce awareness of the value of clinical research, lack of trained workforce and excessive complexity of protocols and administrative procedures. The Delphi panel identified six intervention areas: culture and patient involvement; procedures; staff, contracts, training and incentives; organization and infrastructure; administrative procedures; and ethics committee. According to the R.Evolution project, possible strategies to improve clinical research management in Italy include a deeper understanding of the value of clinical research, the creation of long-term plans for hiring, training, organizing and motivating clinical trial staff, the simplification and harmonization of administrative procedures, as well as protocol design, and the development of stronger networks of centers and stakeholders.</p

Colistin Resistance Development Following Colistin-Meropenem Combination Therapy Versus Colistin Monotherapy in Patients With Infections Caused by Carbapenem-Resistant Organisms

Abstract Background We evaluated whether carbapenem-colistin combination therapy reduces the emergence of colistin resistance, compared to colistin monotherapy, when given to patients with infections due to carbapenem-resistant Gram-negative organisms. Methods This is a pre-planned analysis of a secondary outcome from a randomized, controlled trial comparing colistin monotherapy with colistin-meropenem combination for the treatment of severe infections caused by carbapenem-resistant, colistin-susceptible Gram-negative bacteria. We evaluated rectal swabs taken on Day 7 or later for the presence of new colistin-resistant (ColR) isolates. We evaluated the emergence of any ColR isolate and the emergence of ColR Enterobacteriaceae (ColR-E). Results Data were available for 214 patients for the primary analysis; emergent ColR organisms were detected in 22 (10.3%). No difference was observed between patients randomized to treatment with colistin monotherapy (10/106, 9.4%) versus patients randomized to colistin-meropenem combination therapy (12/108, 11.1%; P = .669). ColR-E organisms were detected in 18/249 (7.2%) patients available for analysis. No difference was observed between the 2 treatment arms (colistin monotherapy 6/128 [4.7%] vs combination therapy 12/121 [9.9%]; P = .111). Enterobacteriaceae, as the index isolate, was found to be associated with development of ColR-E (hazard ratio, 3.875; 95% confidence interval, 1.475–10.184; P = .006). Conclusions Carbapenem-colistin combination therapy did not reduce the incidence of colistin resistance emergence in patients with infections due to carbapenem-resistant organisms. Further studies are necessary to elucidate the development of colistin resistance and methods for its prevention. </jats:sec