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Quale efficacia degli strumenti di governance prescrittiva? Risultati dall’applicazione delle note AIFA. 规范性治理工具的有效性如何？应用 AIFA 说明的结果。
IF 0.5
Global & Regional Health Technology Assessment Pub Date : 2024-06-10 eCollection Date: 2024-01-01 DOI: 10.33393/grhta.2024.3027
Giaele Moretti, Benedetta Dal Canto, Francesco Attanasio, Alma Martelli, Francesca Ferrè
{"title":"Quale efficacia degli strumenti di governance prescrittiva? Risultati dall’applicazione delle note AIFA.","authors":"Giaele Moretti, Benedetta Dal Canto, Francesco Attanasio, Alma Martelli, Francesca Ferrè","doi":"10.33393/grhta.2024.3027","DOIUrl":"10.33393/grhta.2024.3027","url":null,"abstract":"","PeriodicalId":12627,"journal":{"name":"Global & Regional Health Technology Assessment","volume":"11 ","pages":"131-137"},"PeriodicalIF":0.5,"publicationDate":"2024-06-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11168302/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141310512","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}
引用次数: 0
Determining value in the treatment of activated PI3Kδ syndrome in Spain: a multicriteria decision analysis from the perspective of key stakeholders. 确定西班牙活化 PI3Kδ 综合征的治疗价值：从主要利益相关者的角度进行多标准决策分析。
IF 0.5
Global & Regional Health Technology Assessment Pub Date : 2024-05-22 eCollection Date: 2024-01-01 DOI: 10.33393/grhta.2024.3041
María Reyes Abad, Carmen Alerany, Laia Alsina, Eduardo López Granados, Olaf Neth, Jose Luis Poveda, Jacques G Rivière, Carlos Rodríguez-Gallego, Joanne B Tutein Nolthenius, Raquel Figueiredo, Silvia Subías Labazuy, Alicia Gil
{"title":"Determining value in the treatment of activated PI3Kδ syndrome in Spain: a multicriteria decision analysis from the perspective of key stakeholders.","authors":"María Reyes Abad, Carmen Alerany, Laia Alsina, Eduardo López Granados, Olaf Neth, Jose Luis Poveda, Jacques G Rivière, Carlos Rodríguez-Gallego, Joanne B Tutein Nolthenius, Raquel Figueiredo, Silvia Subías Labazuy, Alicia Gil","doi":"10.33393/grhta.2024.3041","DOIUrl":"10.33393/grhta.2024.3041","url":null,"abstract":"<p><strong>Introduction: </strong>Activated phosphoinositide 3-kinase (PI3K)δ syndrome (APDS) is an ultra-rare inborn error of immunity (IEI) combining immunodeficiency and immune dysregulation. This study determined what represents value in APDS in Spain from a multidisciplinary perspective applying multicriteria decision analysis (MCDA) methodology.</p><p><strong>Methods: </strong>A multidisciplinary committee of nine experts scored the evidence matrix. A specific framework for orphan drug evaluation in Spain and the weights assigned by a panel of 98 evaluators and decision-makers was used. Re-evaluation of scores was performed.</p><p><strong>Results: </strong>APDS is considered a very severe disease with important unmet needs, including misdiagnosis and diagnostic delay. Current management is limited to treatment of symptoms with off-label use of therapies supported by limited evidence. Therapeutic benefit is partial, resulting in limited disease control. Haematopoietic stem cell transplantation (HSCT), the only potential curative alternative, is restricted to a reduced patient population and without evidence of long-term efficacy or safety. All options present a limited safety profile. Data on patients' quality of life are lacking. APDS is associated with high pharmacological, medical and indirect costs.</p><p><strong>Conclusions: </strong>APDS is considered a severe disease, with limited understanding by key stakeholders of how treatment success is assessed in clinical practice, the serious impact that has on patients and the associated high economic burden. This study brings to light how MCDA methodology could represent a useful tool to complement current clinical and decision-making methods used by APDS experts and evaluators.</p>","PeriodicalId":12627,"journal":{"name":"Global & Regional Health Technology Assessment","volume":"11 ","pages":"124-130"},"PeriodicalIF":0.5,"publicationDate":"2024-05-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11113520/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141087373","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}
引用次数: 0
Patient-reported outcomes in epilepsy: a case study exploring their usage and impact. 癫痫患者报告结果：探索其用途和影响的案例研究。
IF 0.5
Global & Regional Health Technology Assessment Pub Date : 2024-05-03 eCollection Date: 2024-01-01 DOI: 10.33393/grhta.2024.3020
Simona Lattanzi, Angela La Neve
{"title":"Patient-reported outcomes in epilepsy: a case study exploring their usage and impact.","authors":"Simona Lattanzi, Angela La Neve","doi":"10.33393/grhta.2024.3020","DOIUrl":"10.33393/grhta.2024.3020","url":null,"abstract":"<p><strong>Background: </strong>This study aimed to obtain insights from epilepsy specialists on the use of Patient-Reported Outcome (PRO) measures and how they can affect the management of people with epilepsy and healthcare resource utilization.</p><p><strong>Methods: </strong>The heads of two referral units for people with epilepsy at one tertiary care hospital were invited to respond to a structured survey.</p><p><strong>Results: </strong>Paper-based questionnaires and face-to-face interviews were the main modalities used to measure the quality of life of people with epilepsy. The Quality of Life in Epilepsy Inventory-31 (QOLIE-31), the Adverse Event Profile (adult centre), the Generalized Anxiety Disorder-7, Short-Form Health Survey 36, PSY-Flex, SAFA and Child Behavior Checklist (paediatric centre) were the most used scales. There was consensus about the favourable impact of PRO upon patient management, disease management and measurement of the success of a treatment. Both respondents considered the PRO as important as other main indicators like efficacy and tolerability of the treatment. Lack of time, personnel and economic resources was identified as a barrier on the use of PRO. The PRO could reduce the number of visits, exams and treatments, and increase the time spent on each patient and the number of neuropsychological, psychological and rehabilitation services. The standardized use of PRO was considered useful and the increase in human resources was considered a priority to achieve this goal.</p><p><strong>Conclusions: </strong>Despite the heterogeneity in the actual collection of PRO, there was a uniform perception about their role to optimize the care of people with epilepsy.</p>","PeriodicalId":12627,"journal":{"name":"Global & Regional Health Technology Assessment","volume":"11 ","pages":"108-114"},"PeriodicalIF":0.5,"publicationDate":"2024-05-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11074701/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140876252","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}
引用次数: 0
Costs and benefits in patients with NYHA class III heart failure treated with CardioMEMS in Italy. 意大利 NYHA III 级心力衰竭患者接受 CardioMEMS 治疗的成本和收益。
IF 0.4
Global & Regional Health Technology Assessment Pub Date : 2024-05-02 eCollection Date: 2024-01-01 DOI: 10.33393/grhta.2024.3018
Andrea Messori, Maria Rita Romeo, Valeria Fadda, Francesca Collini, Sabrina Trippoli
{"title":"Costs and benefits in patients with NYHA class III heart failure treated with CardioMEMS in Italy.","authors":"Andrea Messori, Maria Rita Romeo, Valeria Fadda, Francesca Collini, Sabrina Trippoli","doi":"10.33393/grhta.2024.3018","DOIUrl":"10.33393/grhta.2024.3018","url":null,"abstract":"<p><strong>Background: </strong>CardioMEMS is a device suitable for telemedicine that is currently being evaluated by the Regional Health Technology Assessment (HTA) Committee of Tuscany. Two detailed HTA reports are available in the specialized literature, the results of which need to be transferred to our regional setting. These decisions in Tuscany are made by the so-called Centro Operativo HTA.</p><p><strong>Aim: </strong>To validate, with local cost-effectiveness data, the decision on CardioMEMS that will be made in the Tuscany region.</p><p><strong>Methods: </strong>Two detailed international HTA reports were rearranged and adapted to our regional setting to generate a simplified analysis that could form the basis of our decision. Two willingness-to-pay (WTP) thresholds of €20,000/quality-adjusted life year (QALY) and €50,000/QALY were considered.</p><p><strong>Results: </strong>Based on epidemiological and regulatory information, the target population in Tuscany for this device is 166 cases. The value-based price of CardioMEMS is estimated to be €4,332 and €16,662 at WTP thresholds of 20,000/QALY and 50,000/QALY, respectively. Its current price in Italy is €12,000.</p><p><strong>Conclusion: </strong>In our region, the introduction of CardioMEMS is likely to be gradual, around 50 patients/year (or €0.60 million/year at current price). This example highlights the need to adapt the information published in the international literature to the local context in which the approval decision is made. In this context, simplified analyses are easier to apply than complex Markov models.</p>","PeriodicalId":12627,"journal":{"name":"Global & Regional Health Technology Assessment","volume":"11 ","pages":"105-107"},"PeriodicalIF":0.4,"publicationDate":"2024-05-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11067868/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140862640","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}
引用次数: 0
Dati regionali e loro ruolo nella pianificazione, nella programmazione e nel controllo a livello regionale. 地区数据及其在地区一级的规划、计划编制和控制中的作用。
IF 0.5
Global & Regional Health Technology Assessment Pub Date : 2024-04-25 eCollection Date: 2024-01-01 DOI: 10.33393/grhta.2024.3024
Irene Marzona, Paolo Stella, Gianluca Trifirò, Giovanni Corrao
{"title":"Dati regionali e loro ruolo nella pianificazione, nella programmazione e nel controllo a livello regionale.","authors":"Irene Marzona, Paolo Stella, Gianluca Trifirò, Giovanni Corrao","doi":"10.33393/grhta.2024.3024","DOIUrl":"https://doi.org/10.33393/grhta.2024.3024","url":null,"abstract":"","PeriodicalId":12627,"journal":{"name":"Global & Regional Health Technology Assessment","volume":"11 ","pages":"101-104"},"PeriodicalIF":0.5,"publicationDate":"2024-04-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11056689/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140855318","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}
引用次数: 0
Prontuari Terapeutici Regionali in Italia: stato dell’arte e prospettive future. 意大利的地区治疗方案：技术水平和未来前景。
IF 0.5
Global & Regional Health Technology Assessment Pub Date : 2024-03-08 eCollection Date: 2024-01-01 DOI: 10.33393/grhta.2024.2677
Alberto Bortolami, Claudio Jommi, Filippo Bresciani, Luca Piccoli, Elisa Sangiorgi, Giovanna Scroccaro
{"title":"Prontuari Terapeutici Regionali in Italia: stato dell’arte e prospettive future.","authors":"Alberto Bortolami, Claudio Jommi, Filippo Bresciani, Luca Piccoli, Elisa Sangiorgi, Giovanna Scroccaro","doi":"10.33393/grhta.2024.2677","DOIUrl":"https://doi.org/10.33393/grhta.2024.2677","url":null,"abstract":"","PeriodicalId":12627,"journal":{"name":"Global & Regional Health Technology Assessment","volume":"11 ","pages":"68-74"},"PeriodicalIF":0.5,"publicationDate":"2024-03-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10949060/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140178661","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}
引用次数: 0
Costo per Number Needed to Treat (NNT) di upadacitinib nel trattamento dei pazienti bio-exposed con rettocolite ulcerosa attiva da moderata a grave. 奥达帕替尼治疗生物暴露的中重度活动性溃疡性直肠结肠炎患者的单位治疗人数成本（NNT）。
IF 0.5
Global & Regional Health Technology Assessment Pub Date : 2024-03-07 eCollection Date: 2024-01-01 DOI: 10.33393/grhta.2024.2658
Flavio Caprioli, Massimo Claudio Fantini, Francesca Marando, Dario Scaduto, Roberto Ravasio
{"title":"Costo per <i>Number Needed to Treat</i> (NNT) di upadacitinib nel trattamento dei pazienti <i>bio-exposed</i> con rettocolite ulcerosa attiva da moderata a grave.","authors":"Flavio Caprioli, Massimo Claudio Fantini, Francesca Marando, Dario Scaduto, Roberto Ravasio","doi":"10.33393/grhta.2024.2658","DOIUrl":"10.33393/grhta.2024.2658","url":null,"abstract":"","PeriodicalId":12627,"journal":{"name":"Global & Regional Health Technology Assessment","volume":"11 ","pages":"55-67"},"PeriodicalIF":0.5,"publicationDate":"2024-03-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10926000/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140101425","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}
引用次数: 0
Regolamento HTA: come si sta muovendo l’Italia? HTA 法规：意大利在做什么？
IF 0.5
Global & Regional Health Technology Assessment Pub Date : 2024-03-05 eCollection Date: 2024-01-01 DOI: 10.33393/grhta.2024.3025
Francesca Patarnello
{"title":"Regolamento HTA: come si sta muovendo l’Italia?","authors":"Francesca Patarnello","doi":"10.33393/grhta.2024.3025","DOIUrl":"https://doi.org/10.33393/grhta.2024.3025","url":null,"abstract":"","PeriodicalId":12627,"journal":{"name":"Global & Regional Health Technology Assessment","volume":"11 ","pages":"51-54"},"PeriodicalIF":0.5,"publicationDate":"2024-03-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10915632/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140051473","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}
引用次数: 0
Costo per NNT di upadacitinib nel trattamento dei pazienti con dermatite atopica da moderata a severa in Italia. 意大利奥达帕替尼治疗中重度特应性皮炎患者的每 NNT 成本。
IF 0.5
Global & Regional Health Technology Assessment Pub Date : 2024-02-16 eCollection Date: 2024-01-01 DOI: 10.33393/grhta.2024.2728
Andrea Chiricozzi, Antonio Costanzo, Anna Levi, Federica Parretta, Roberto Ravasio
{"title":"Costo per NNT di upadacitinib nel trattamento dei pazienti con dermatite atopica da moderata a severa in Italia.","authors":"Andrea Chiricozzi, Antonio Costanzo, Anna Levi, Federica Parretta, Roberto Ravasio","doi":"10.33393/grhta.2024.2728","DOIUrl":"10.33393/grhta.2024.2728","url":null,"abstract":"","PeriodicalId":12627,"journal":{"name":"Global & Regional Health Technology Assessment","volume":"11 ","pages":"38-50"},"PeriodicalIF":0.5,"publicationDate":"2024-02-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10877702/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139912464","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}
引用次数: 0
Il burden clinico ed economico delle complicanze della cute peristomale: un’analisi di Activity Based Costing. 肛周皮肤并发症的临床和经济负担：基于活动成本的分析。
IF 0.4
Global & Regional Health Technology Assessment Pub Date : 2024-01-23 eCollection Date: 2024-01-01 DOI: 10.33393/grhta.2024.2639
Agostino Fortunato, Filippo Rumi, Massimo Zazzetta, Marco Della Valle, Vincenzo Pedace, Americo Cicchetti
{"title":"Il burden clinico ed economico delle complicanze della cute peristomale: un’analisi di Activity Based Costing.","authors":"Agostino Fortunato, Filippo Rumi, Massimo Zazzetta, Marco Della Valle, Vincenzo Pedace, Americo Cicchetti","doi":"10.33393/grhta.2024.2639","DOIUrl":"10.33393/grhta.2024.2639","url":null,"abstract":"","PeriodicalId":12627,"journal":{"name":"Global & Regional Health Technology Assessment","volume":"11 ","pages":"31-37"},"PeriodicalIF":0.4,"publicationDate":"2024-01-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10813189/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139570373","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}
引用次数: 0
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