Global & Regional Health Technology Assessment最新文献

{"title":"Cost minimization analysis of subcutaneous and intravenous immunoglobulin treatment in Italy.","authors":"Chiara Bini, Matteo Scortichini, Silvia Ripoli, Laura Fioravanti, Carmela Papa, Paolo Sciattella","doi":"10.33393/grhta.2026.3642","DOIUrl":"https://doi.org/10.33393/grhta.2026.3642","url":null,"abstract":"<p><strong>Introduction: </strong>Human immunoglobulins are the standard of care for patients with immunodeficiencies and represent a first-line treatment for chronic inflammatory demyelinating polyneuropathy (CIDP). This study aimed to evaluate costs associated with facilitated subcutaneous (fSCIG), conventional subcutaneous (cSCIG), and intravenous (IVIG) administration of immunoglobulin in patients with primary or secondary immunodeficiency (PID and SID, respectively) and in patients with CIDP in Italy.</p><p><strong>Methods: </strong>A cost-minimization analysis from a societal perspective was developed, considering a one-year follow-up. Direct costs included acquisition and administration costs, while indirect costs were evaluated considering productivity loss due to in-hospital IVIG using a Human Capital Approach. The posology considered for immunodeficiency disorders and CIDP was obtained from observational studies and clinical trials, respectively. Subcutaneous and intravenous administration costs were obtained from published literature. Scenario analyses were conducted to test the key assumptions of the model.</p><p><strong>Results: </strong>This study indicates a potential reduction of annual societal expenditure in Italy of approximately €2,145 and €1,605 with fSCIG treatment in PID patients compared to cSCIG and IVIG, respectively. In SID patients, fSCIG would result in a reduction of societal expenditure of approximately €2,145 and €2,071 compared to cSCIG and IVIG. For CIDP patients, fSCIG treatment would result in a reduction of societal expenditure of €8,060 and €15,873 compared to cSCIG and IVIG, respectively.</p><p><strong>Conclusion: </strong>The use of fSCIG for patients with PIDs, SIDs or CIDP could lead to a reduction in the direct and indirect costs associated with the treatment of patients.</p>","PeriodicalId":12627,"journal":{"name":"Global & Regional Health Technology Assessment","volume":"13 ","pages":"109-117"},"PeriodicalIF":0.5,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13136886/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147836970","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A guide to critical reading of influenza vaccine cost-effectiveness analyses.","authors":"Raúl Ortiz de Lejarazu, Esther Redondo Margüello, Ángel Gil de Miguel, Federico Martinón Torres, Javier Díez Domingo, Juan L López-Belmonte Claver, Ariadna Díaz-Aguiló, J Manel Farré Avellà, Paloma I Palomo Jiménez, José M Abellán Perpiñán","doi":"10.33393/grhta.2026.3619","DOIUrl":"https://doi.org/10.33393/grhta.2026.3619","url":null,"abstract":"<p><strong>Introduction: </strong>Influenza vaccines are formulated each year to prevent serious illness in at-risk individuals, including elderly people. Healthcare decision-making is mainly based on the economic evaluations (EEs) (i.e., cost-effectiveness analysis [CEA]) of vaccines; however, understanding the limitations of these models and correctly interpreting the results may be challenging. Here, we provide a practical Guide that will help readers who are not experts in the field of health economics or influenza to critically review influenza vaccine EEs.</p><p><strong>Methods: </strong>This Guide is based on the findings of a systematic review of the literature, a critical analysis of the available EEs published for influenza vaccines for older adults in Spain, and applicable national and international guidelines on EE and influenza modeling. It has been developed by a multidisciplinary board of experts in influenza, vaccines, and health economics.</p><p><strong>Results: </strong>The guide provides tips to help the reader assess whether an EE design is fit for its purpose in terms of comparators, time horizon, perspective of the analysis, population analyzed, and whether appropriate modeling methods were applied. It detects the uncertainty arising from input data and the implications of this uncertainty on the results.</p><p><strong>Conclusions: </strong>Ultimately, this resource aims to empower decision-makers, particularly those without expertise in health economics or vaccinology, to critically read and interpret EEs, thus favoring evidence-based informed decisions that will improve the efficiency of influenza vaccination programs.</p>","PeriodicalId":12627,"journal":{"name":"Global & Regional Health Technology Assessment","volume":"13 ","pages":"95-108"},"PeriodicalIF":0.5,"publicationDate":"2026-04-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13123513/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147769419","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Ridiscussioni nel processo decisionale dell’Agenzia Italiana del Farmaco: una valutazione quantitativa di cause e implicazioni.","authors":"Michele Caimmi, Beatrice Canali, Anita Ciarlo, Gloria Lombardi, Chiara Vassallo, Laura Candelora","doi":"10.33393/grhta.2026.3717","DOIUrl":"https://doi.org/10.33393/grhta.2026.3717","url":null,"abstract":"","PeriodicalId":12627,"journal":{"name":"Global & Regional Health Technology Assessment","volume":"13 ","pages":"87-94"},"PeriodicalIF":0.5,"publicationDate":"2026-04-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13103655/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147769458","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Italian Medicines Agency's reform and time until pricing and reimbursement decisions: a time-to-event analysis.","authors":"Gian Marco Raspolini, Bruno Federico, Mario Cesare Nurchis, Gianfranco Damiani, Raffaella Cocciolo, Paola Turella, Daniela Pilunni, Pierluigi Navarra","doi":"10.33393/grhta.2026.3671","DOIUrl":"https://doi.org/10.33393/grhta.2026.3671","url":null,"abstract":"<p><strong>Introduction: </strong>The duration of pricing and reimbursement (P&R) negotiations is a key performance indicator for medicines agencies in universal health coverage systems. In early 2024, the Italian Medicines Agency (AIFA) underwent a major reform, including the merging of the previous Scientific-Technical Committee and Price and Reimbursement Committee into a single Scientific and Economic Committee. This study evaluates the reform's impact on time to P&R determinations for new medicines in Italy.</p><p><strong>Methods: </strong>A time-to-event analysis was conducted on 139 new chemical entities authorized by the European Commission (EC) between February 2021 and December 2023. The primary outcome was the time from marketing authorization (MA) to publication of AIFA's P&R determination. Kaplan-Meier curves and Cox proportional hazards models were used to compare reclassification hazards between pre- (before March 2024) and post-reform groups of medicinal products, based on a fixed separation date, adjusting for antineoplastic therapeutic area and pharmaceutical company size. Four sensitivity analyses tested the robustness of the results.</p><p><strong>Results: </strong>The multivariate Cox model, adjusting for antineoplastics, products from major corporations, and orphan medicines, showed that the reform was associated with an 84% increase in reclassification hazards (HR = 1.84, 95% CI 1.20-2.82, p = 0.005). Sensitivity analyses corroborated these findings, showing even greater improvements when focusing on national evaluation timeframes (HR = 11.57 and HR = 3.91).</p><p><strong>Conclusion: </strong>The consolidation of separate committees into a unified structure, as a part of the 2024 AIFA reform, was accompanied by accelerated P&R negotiations for new medicines in Italy, demonstrating that structural optimization of health technology assessment processes may enhance system efficiency.</p>","PeriodicalId":12627,"journal":{"name":"Global & Regional Health Technology Assessment","volume":"13 ","pages":"79-86"},"PeriodicalIF":0.5,"publicationDate":"2026-04-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13071805/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147689606","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Come le Regioni possono utilizzare il Rapporto OsMed per condividere le azioni di miglioramento dell’appropriatezza.","authors":"Silvia Sansone, Maria Cristina Carena, Giorgia Fedele, Roberto Da Cas, Giuseppe Traversa","doi":"10.33393/grhta.2026.3684","DOIUrl":"https://doi.org/10.33393/grhta.2026.3684","url":null,"abstract":"","PeriodicalId":12627,"journal":{"name":"Global & Regional Health Technology Assessment","volume":"13 ","pages":"74-78"},"PeriodicalIF":0.5,"publicationDate":"2026-04-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13055846/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147638587","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Non-profit research: an insight into the management and monitoring of clinical trials in Italy.","authors":"Fatima Tizi, Viviana Appolloni, Barbara Bonifacio, Silvia Tommasi, Sara Lega, Celeste Cagnazzo","doi":"10.33393/grhta.2026.3668","DOIUrl":"https://doi.org/10.33393/grhta.2026.3668","url":null,"abstract":"<p><strong>Introduction: </strong>In January 2022, Italy fully implemented EU Regulation No. 536/2014, revolutionizing the national framework for clinical research on medicinal products. This marked a critical shift for non-profit sponsors, who are now required to align with high-quality standards and ICH-GCP guidelines. Understanding their level of compliance is essential to identify systemic barriers and areas needing support.</p><p><strong>Methods: </strong>In March 2024, a 50-item anonymous pilot survey was distributed via social media to assess the operational and regulatory compliance of non-profit sponsors managing clinical trials in Italy. The survey focused on three domains: data management, risk assessment, and monitoring practices. Responses were collected from 32 non-profit sponsors.</p><p><strong>Results: </strong>The findings revealed substantial compliance gaps. Approximately 91% (n = 29) of respondents reported they rarely or never prepare core GCP-required documents, including Data Management Plans and Statistical Analysis Plans. Only 25% (n = 8) routinely conduct risk assessments before protocol drafting. Monitoring visits, either remote or on-site, were performed by 63% (n = 20) of sponsors. Among those not conducting on-site monitoring (n = 14), the primary constraints reported were staff shortages (29%, n = 4) and financial limitations (57%, n = 8). Alarmingly, within this same subgroup, 29% (n = 4) believed that on-site monitoring was unnecessary, despite regulatory mandates. This attitude extended to remote monitoring activities as well.</p><p><strong>Conclusions: </strong>The survey highlights major challenges faced by non-profit sponsors in meeting regulatory expectations. Limited resources and a lack of trained personnel significantly impair compliance with GCP standards. To ensure research integrity and patient safety, targeted support strategies and policy interventions are urgently needed to empower non-profit research in adapting to the evolving regulatory landscape.</p>","PeriodicalId":12627,"journal":{"name":"Global & Regional Health Technology Assessment","volume":"13 ","pages":"68-73"},"PeriodicalIF":0.5,"publicationDate":"2026-03-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12989811/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147473524","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The societal costs associated with patients with chronic spontaneous urticaria (CSU) in Italy.","authors":"Chiara Bini, Andrea Marcellusi, Virginia Scansetti, Lucia Casoli, Elena Radaelli","doi":"10.33393/grhta.2026.3703","DOIUrl":"https://doi.org/10.33393/grhta.2026.3703","url":null,"abstract":"<p><strong>Introduction: </strong>Chronic spontaneous urticaria (CSU) is a debilitating dermatological disease. The study aimed to estimate the indirect costs and out-of-pocket expenses incurred by CSU patients in Italy.</p><p><strong>Methods: </strong>The economic model was based on data collected through a survey conducted among patients and caregivers with the support of the Association for the Research and Cure of Urticaria (ARCO). Disease severity was classified into three categories: mild, moderate and severe. Indirect costs were estimated using the human capital approach. Out-of-pocket expenses were estimated in terms of direct healthcare costs borne by the patient and direct non-healthcare costs.</p><p><strong>Results: </strong>Data were analyzed for 122 patients; 7% of these reported mild CSU, 16% moderate and 78% severe. The average age was 46.8 years, with an average age at diagnosis of 38.2 years. Overall, 50% of employed patients lost workdays due to CSU, and 63.3% reported reduced productivity at work; 45.1% of patients received caregiver support, with 37.9% of employed caregivers reporting workdays lost. The economic model estimated an average annual indirect cost per patient with CSU of €4,281, mainly due to presenteeism (59%). Indirect costs increase with the severity of the disease. Average annual out-of-pocket expenses was €502.7 per patient.</p><p><strong>Conclusions: </strong>The study conducted based on data directly reported by patients could contribute to a better understanding of CSU, thereby promoting a more efficient approach to the care and support of patients and their families.</p>","PeriodicalId":12627,"journal":{"name":"Global & Regional Health Technology Assessment","volume":"13 ","pages":"58-67"},"PeriodicalIF":0.5,"publicationDate":"2026-03-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12983078/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147467792","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Use of artificial intelligence to draft a mini-HTA report on a new medical device belonging to class IIb-III.","authors":"Andrea Messori, Sabrina Beltramini, Paola Crosasso, Claudia Fruttero, Melania Rivano, Marco Chiumente, Maria C Silvani","doi":"10.33393/grhta.2026.3691","DOIUrl":"https://doi.org/10.33393/grhta.2026.3691","url":null,"abstract":"<p><p>The rapid evolution of artificial intelligence (AI) in the pharmaceutical and medical device (MD) sectors has prompted interest in its potential role in supporting health technology assessment (HTA). This editorial presents an innovative project aimed at facilitating and expanding HTA activities for high-risk MDs (Class IIb-III) in Italy, where structured HTA processes for MDs are inconsistently implemented. The project centers on a freely accessible AI-based web tool designed to generate preliminary mini-HTA reports. The tool operates through two steps: users provide essential device information via an online form, and ChatGPT produces a structured draft report, including PICO statements, coverage of the nine EUnetHTA domains, and a preliminary summary of relevant PubMed evidence. Although these AI-generated reports are imperfect and require expert verification and refinement, they offer substantial practical advantages by reducing the initial workload and enabling rapid production of a first draft-within minutes rather than hours. The project includes detailed operational instructions and real application examples, such as an artificial iris device, presented in supplementary appendices. Future developments include the release of an English-language version to support broader international use. While AI cannot replace expert judgment, the editorial highlights its value as an accelerative tool that can streamline early HTA steps and promote more systematic evaluation of MDs across Italian regions. Continued iterative use is expected to improve system performance and enhance integration into HTA workflows.</p>","PeriodicalId":12627,"journal":{"name":"Global & Regional Health Technology Assessment","volume":"13 ","pages":"55-57"},"PeriodicalIF":0.5,"publicationDate":"2026-03-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12955720/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147354662","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Multicriterio e valutazioni economiche: verso una proposta per un nuovo framework per il prezzo dei farmaci in Italia.","authors":"Andrea Marcellusi, Aldo P Maggioni, Anna L Mandorino, Francesca Caprari, Irene Marzona, Claudio Jommi","doi":"10.33393/grhta.2026.3604","DOIUrl":"https://doi.org/10.33393/grhta.2026.3604","url":null,"abstract":"","PeriodicalId":12627,"journal":{"name":"Global & Regional Health Technology Assessment","volume":"13 ","pages":"49-54"},"PeriodicalIF":0.5,"publicationDate":"2026-02-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12908649/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146212892","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Impatto clinico ed economico dell’emicrania in Italia: un’analisi di real-world evidence.","authors":"Valentina Perrone, Simone Leonetti, Luca Degli Esposti, Lisa Giannessi, Simona Guerzoni","doi":"10.33393/grhta.2026.3648","DOIUrl":"10.33393/grhta.2026.3648","url":null,"abstract":"","PeriodicalId":12627,"journal":{"name":"Global & Regional Health Technology Assessment","volume":"13 ","pages":"38-48"},"PeriodicalIF":0.5,"publicationDate":"2026-02-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12903886/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146201282","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}