Global & Regional Health Technology Assessment最新文献

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Analisi di impatto di budget di un nuovo sistema di cura in pazienti affetti da malattia di Parkinson. 对帕金森患者新治疗系统的预算影响分析。
IF 0.5
Global & Regional Health Technology Assessment Pub Date : 2022-01-01 DOI: 10.33393/grhta.2022.2413
Chiara Bini, Francesco Saverio Mennini, Andrea Marcellusi, Martina Paoletti, Carlo Tomino
{"title":"Analisi di impatto di budget di un nuovo sistema di cura in pazienti affetti da malattia di Parkinson.","authors":"Chiara Bini, Francesco Saverio Mennini, Andrea Marcellusi, Martina Paoletti, Carlo Tomino","doi":"10.33393/grhta.2022.2413","DOIUrl":"https://doi.org/10.33393/grhta.2022.2413","url":null,"abstract":"Objective: To estimate the economic impact of the introduction of a new care system based on apomorphine and Patient Support Program for motor fluctuations (“on-off” phenomena) in patients with Parkinson’s disease which are not sufficiently controlled by oral anti-Parkinson medication in Italy.\u0000Method: A Budget Impact model was developed to evaluate the new care system in patients with Parkinson’s disease over a 3-years’ time horizon. The comparator treatments included in the analysis were treatments based on apomorphine and levodopa + carbidopa. The analysis was conducted from a National Health Service (NHS) perspective. Costs included in the analysis were acquisition costs and device costs. A deterministic sensitivity analysis was carried out to evaluate the uncertainty of the parameters used. A break-even analysis was conducted to identify the minimum number of subjects that would need to be treated with the new care system to obtain a positive Budget Impact (World With – World Without = 0).\u0000Results: The analysis shows that the introduction of the new care system based on apomorphine could generate a cost saving incurred by the NHS of over € 5.7 million in 3 years. Break-even analysis shows that if it were possible to intercept with the new treatment at least 9 patients treated with apomorphine, there would not be an increase in costs for the NHS.\u0000Conclusion: The new care system would respond to the unmet needs of patients with Parkinson’s disease by generating a reduction in the expenditure incurred by NHS.","PeriodicalId":12627,"journal":{"name":"Global & Regional Health Technology Assessment","volume":"9 ","pages":"91-98"},"PeriodicalIF":0.5,"publicationDate":"2022-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/da/5b/grhta-9-91.PMC9768609.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10518583","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: 1
Use of a maturity model for facilitating the introduction of CAR T-cell therapy-Results of the START CAR-T project. 利用成熟度模型促进CAR- t细胞疗法的引入——START CAR- t项目的结果。
IF 0.5
Global & Regional Health Technology Assessment Pub Date : 2022-01-01 DOI: 10.33393/grhta.2022.2340
Stefania Bramanti, Matteo G Carrabba, Alice Di Rocco, Elena Fabris, Luca Gastaldi, Paolo Locatelli, Maria Chiara Tisi
{"title":"Use of a maturity model for facilitating the introduction of CAR T-cell therapy-Results of the START CAR-T project.","authors":"Stefania Bramanti,&nbsp;Matteo G Carrabba,&nbsp;Alice Di Rocco,&nbsp;Elena Fabris,&nbsp;Luca Gastaldi,&nbsp;Paolo Locatelli,&nbsp;Maria Chiara Tisi","doi":"10.33393/grhta.2022.2340","DOIUrl":"https://doi.org/10.33393/grhta.2022.2340","url":null,"abstract":"<p><strong>Introduction: </strong>Chimeric antigen receptor (CAR) T-cell therapies are novel immunotherapies for the treatment of hematologic malignancies. They are administered in specialized centers by a multidisciplinary team and require the careful coordination of all steps involved in manufacturing and using cellular therapies. The Maturity Model (MM) is a tool developed and used for assessing the effectiveness of a variety of activities. In healthcare, it may assist clinicians in the gradual improvement of patient management with CAR T-cell therapy and other complex treatments.</p><p><strong>Methods: </strong>The START CAR-T project was initiated to investigate the potential of a MM in the setting of CAR T-cell therapy. Four Italian clinics participated in the creation of a dedicated MM. Following the development and test of this MM, its validity and generalizability were further tested with a questionnaire submitted to 18 Italian centers.</p><p><strong>Results: </strong>The START CAR-T MM assessed the maturity level of clinical sites, with a focus on organization, process, and digital support. For each area, the model defined four maturity steps, and indicated the actions required to evolve from a basic to an advanced status. The application of the MM to 18 clinical sites provided a description of the maturity level of Italian centers with regard to the introduction of CAR T-cell therapy.</p><p><strong>Conclusion: </strong>The START CAR-T MM appears to be a useful and widely applicable tool. It may help centers optimize many aspects of CAR T-cell therapy and improve patient access to this novel treatment option.</p>","PeriodicalId":12627,"journal":{"name":"Global & Regional Health Technology Assessment","volume":"9 ","pages":"1-8"},"PeriodicalIF":0.5,"publicationDate":"2022-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/4d/43/grhta-9-1.PMC9768606.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10525353","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
Regolazione del prezzo e rimborso dei farmaci: comparatori, endpoint e ruolo della costo-efficacia. 药品定价和报销:比较器、终点和成本效益作用。
IF 0.5
Global & Regional Health Technology Assessment Pub Date : 2022-01-01 DOI: 10.33393/grhta.2022.2475
Claudio Jommi, Giovanni Apolone, Giovanna Scroccaro, Valentina Acciai, Antonio Addis, Andrea Ardizzoni, Renato Bernardini, Alberto Bortolami, Alessia Brigido, Giuliano Buzzetti, Pier Luigi Canonico, Francesca Caprari, Stefano Centanni, Chiara Cernetti, Americo Cicchetti, Giorgio Corsico, Francesco Damele, Filippo de Braud, Sara Manurita, Francesco Saverio Mennini, Irene Olivi, Federica Parretta, Lara Pippo, Stefania Pulimeno, Massimo Riccaboni, Giuseppe Rossi, Cecilia Saleri, Alessandra Sinibaldi, Federico Spandonaro, Cristian Stefenoni, Elena Visentin, Pierluigi Viale, Giuseppina Zapparelli, Patrizia Popoli
{"title":"Regolazione del prezzo e rimborso dei farmaci: comparatori, endpoint e ruolo della costo-efficacia.","authors":"Claudio Jommi,&nbsp;Giovanni Apolone,&nbsp;Giovanna Scroccaro,&nbsp;Valentina Acciai,&nbsp;Antonio Addis,&nbsp;Andrea Ardizzoni,&nbsp;Renato Bernardini,&nbsp;Alberto Bortolami,&nbsp;Alessia Brigido,&nbsp;Giuliano Buzzetti,&nbsp;Pier Luigi Canonico,&nbsp;Francesca Caprari,&nbsp;Stefano Centanni,&nbsp;Chiara Cernetti,&nbsp;Americo Cicchetti,&nbsp;Giorgio Corsico,&nbsp;Francesco Damele,&nbsp;Filippo de Braud,&nbsp;Sara Manurita,&nbsp;Francesco Saverio Mennini,&nbsp;Irene Olivi,&nbsp;Federica Parretta,&nbsp;Lara Pippo,&nbsp;Stefania Pulimeno,&nbsp;Massimo Riccaboni,&nbsp;Giuseppe Rossi,&nbsp;Cecilia Saleri,&nbsp;Alessandra Sinibaldi,&nbsp;Federico Spandonaro,&nbsp;Cristian Stefenoni,&nbsp;Elena Visentin,&nbsp;Pierluigi Viale,&nbsp;Giuseppina Zapparelli,&nbsp;Patrizia Popoli","doi":"10.33393/grhta.2022.2475","DOIUrl":"https://doi.org/10.33393/grhta.2022.2475","url":null,"abstract":"This document illustrates the results of a discussion of two multi-disciplinary expert panels on pricing and reimbursement of medicines. Experts work in different organizations. The discussion focused on comparator(s), endpoint(s), negotiation of prices of new medicines and/or indications to include in the List 648, as well as the role of cost-effectiveness in the price and reimbursement negotiation. The debate took place during the fourth edition of the Seminari di Mogliano, organized on the 30th of September/1st of October, 2021.\u0000The two panels agreed on a general need to enhance interaction among the different stakeholders, in the early assessment and negotiation phases, and to increase the transparency/reproducibility of the decisions taken. The experts have also emphasized the need (i) to improve clarity in the evaluation of additional therapeutic value and the place in therapy with respect to comparators and how comparators are identified; (ii) to create work groups to identify the most appropriate endpoint(s), for each therapeutic area and level of unmet needs; (iii) to provide for a systematic use of cost-effectiveness when an added therapeutic value is delivered by a new medicine. With regard to the 648 List, the experts advocated for an overall reorganization of the current rules governing the special uses of drugs.","PeriodicalId":12627,"journal":{"name":"Global & Regional Health Technology Assessment","volume":"9 ","pages":"99-104"},"PeriodicalIF":0.5,"publicationDate":"2022-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/4d/94/grhta-9-99.PMC9768592.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10520998","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
Budget impact analysis sull’introduzione del dispositivo di telemonitoraggio Turbo+ nella gestione dei pazienti asmatici in Italia. 预算影响分析在意大利引入涡轮增压遥测监测系统用于哮喘患者的管理。
IF 0.5
Global & Regional Health Technology Assessment Pub Date : 2022-01-01 DOI: 10.33393/grhta.2022.2290
Filippo Rumi, Michele Basile, Americo Cicchetti, Rosario Contiguglia, Antonella Pentassuglia, Alessandro Oliva, Gianenrico Senna, Marco Bonavia, Francesco Scarpelli, Marco Benvenuto
{"title":"Budget impact analysis sull’introduzione del dispositivo di telemonitoraggio Turbo+ nella gestione dei pazienti asmatici in Italia.","authors":"Filippo Rumi,&nbsp;Michele Basile,&nbsp;Americo Cicchetti,&nbsp;Rosario Contiguglia,&nbsp;Antonella Pentassuglia,&nbsp;Alessandro Oliva,&nbsp;Gianenrico Senna,&nbsp;Marco Bonavia,&nbsp;Francesco Scarpelli,&nbsp;Marco Benvenuto","doi":"10.33393/grhta.2022.2290","DOIUrl":"https://doi.org/10.33393/grhta.2022.2290","url":null,"abstract":"Introduction of the telemonitoring device Turbo+ in the management of patients suffering from asthma in Italy: a budget impact analysis Introduction: Adherence to long-term asthma control drugs is one of the key factors in improving disease management among patients with asthma. Poor adherence to long-term therapies significantly compromises the effectiveness of treatment and represents a problem in controlling the disease in terms of quality of life and economic impact. A budget impact analysis has been developed taking into account that Turbo+ technology (electronic monitoring device) can increase adherence to therapy in patients suffering from asthma. Methods: Coherently with the budget impact model methodology, we developed two different scenarios. One represents the clinical practice assuming a constant market share of 0,99% for Turbo+ and the other one assumes an incremental market share of Turbo+ over the time horizon considered. An increase in adherence to therapy will likely correspond to a reduction of exacerbations and a lower rate of hospitalizations, thus generating savings in terms of use of health resources. Results: The differential analysis shows an incremental saving in terms of resources absorbed by the National Health Service (NHS) over the time horizon considered. In the first year the savings are equal to € 795,658.64 and in the fifth year they are equal to € 3,520,636.34 for a total of resources saved in 5 years of € 10,882,028.50. Conclusions: The model shows how the diffusion of the Turbo+ programme can lead to savings in terms of health resources consumed by the NHS. Further analyses with new real-world data on adherence in asthmatic patients could be useful in confirming the results of the present analysis.","PeriodicalId":12627,"journal":{"name":"Global & Regional Health Technology Assessment","volume":"9 ","pages":"58-67"},"PeriodicalIF":0.5,"publicationDate":"2022-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/dc/83/grhta-9-58.PMC9768613.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10511649","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
Valutazione economica di treosulfan in pazienti sottoposti a trapianto allogenico di cellule staminali ematopoietiche. 造血干细胞异源移植患者对treosulfan的经济评价。
IF 0.5
Global & Regional Health Technology Assessment Pub Date : 2022-01-01 DOI: 10.33393/grhta.2022.2412
Chiara Bini, Martina Paoletti, Andrea Marcellusi, Carlo Tomino, Francesco Saverio Mennini
{"title":"Valutazione economica di treosulfan in pazienti sottoposti a trapianto allogenico di cellule staminali ematopoietiche.","authors":"Chiara Bini,&nbsp;Martina Paoletti,&nbsp;Andrea Marcellusi,&nbsp;Carlo Tomino,&nbsp;Francesco Saverio Mennini","doi":"10.33393/grhta.2022.2412","DOIUrl":"https://doi.org/10.33393/grhta.2022.2412","url":null,"abstract":"Economic evaluation of treosulfan in patients undergoing allogeneic haematopoietic stem cell transplantation Objective: To assess the cost-effectiveness and economic sustainability of treosulfan plus fludarabine compared with busulfan plus fludarabine as a conditioning treatment for malignant disease prior to allogeneic haematopoietic stem cell transplantation (alloHSCT) in adult patients in Italy. Method: The two theoretical cohorts of patients aged ≥ 60 years with acute myeloid leukaemia (AML) and myelodysplastic syndrome (MDS) were pooled and followed over time using a partitioned survival model with cycles of 28 days. Patients can transition between a post-HSCT recovery/remission state (Event-Free Survival state, EFS state), a relapsed/progressed disease state, and a death state. A lifetime horizon for cost-effectiveness analysis and a 5-years’ time horizon for budget impact analysis were used. The perspective of the Italian National Health Service was adopted. Utility values were obtained from published sources. Costs included: drug acquisition, HSCT procedure, management and treatment of adverse reactions, graft-versus-host disease (GvHD) and health states, end of life treatment. Discounting of 3% per year was applied for both costs and outcomes according to Italian guidelines. Sensitivity was tested through both one-way and probabilistic analyses. Results: Cost-effectiveness analysis showed that treosulfan is both more effective and less expensive compared with busulfan (+1.08 life-years, +0.95 quality-adjusted life-years per patient and –€ 41,388 per patient). On the side of economic sustainability, the introduction of treosulfan in the market could generate a cumulative decrement of the expense incurred by NHS of about –€ 212,063 over five years. Conclusion: Treosulfan could represent a cost-effective and sustainable treatment alternative from the perspective of the NHS.","PeriodicalId":12627,"journal":{"name":"Global & Regional Health Technology Assessment","volume":"9 ","pages":"105-116"},"PeriodicalIF":0.5,"publicationDate":"2022-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/b9/34/grhta-9-105.PMC9768594.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10518582","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
The budgetary impact of alemtuzumab in multiple sclerosis in Quito, Ecuador. Payer's perspective. 厄瓜多尔基多多发性硬化症患者使用阿利珠单抗的预算影响。付款人的观点。
IF 0.4
Global & Regional Health Technology Assessment Pub Date : 2021-10-21 eCollection Date: 2021-01-01 DOI: 10.33393/grhta.2021.2273
Luis J Pastor-Quirós, Edgar P Correa-Díaz
{"title":"The budgetary impact of alemtuzumab in multiple sclerosis in Quito, Ecuador. Payer's perspective.","authors":"Luis J Pastor-Quirós, Edgar P Correa-Díaz","doi":"10.33393/grhta.2021.2273","DOIUrl":"10.33393/grhta.2021.2273","url":null,"abstract":"<p><strong>Introduction: </strong>Multiple sclerosis is a neurological condition that causes disabilities and is most common in young adults. It imposes high financial costs affecting the quality of life of patients, families, and society. It is critical to measure the budgetary impact of new technologies to treat this disease.</p><p><strong>Objective: </strong>The aim of the article is to estimate the budgetary impact of introducing alemtuzumab as an escalation therapy in patients diagnosed with Recurrent Remitting Multiple Sclerosis and treated in Quito, Ecuador.</p><p><strong>Materials and methods: </strong>A cohort of 85 patients receiving treatment with disease-modifying therapies was used, within a 5-year timeframe, between 2021 and 2025. The baseline scenario, including the percentages of administration of the different drugs, is compared with the alternative scenario, including alemtuzumab. The cost assessment included only direct medical resources. To obtain local resources for management of the disease, a neurologist and clinical expert who treats most of the patients in Quito was consulted.</p><p><strong>Results: </strong>Considering a cohort of 85 patients with active Recurrent Remitting Multiple Sclerosis, the average global budget impact in 5 years would be USD 10,603,230.00 in the base case and USD 9,995,817.00 in the alemtuzumab scenario.</p><p><strong>Conclusion: </strong>The inclusion of alemtuzumab as escalation therapy represents budgetary savings over the next 5 years (2021-2025).</p>","PeriodicalId":12627,"journal":{"name":"Global & Regional Health Technology Assessment","volume":"8 ","pages":"140-146"},"PeriodicalIF":0.4,"publicationDate":"2021-10-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/2d/55/grhta-8-140.PMC9616185.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10869816","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
Real-world data: come possono aiutare a migliorare la qualità dell’assistenza. 真实世界的数据:如何帮助提高医疗质量。
IF 0.4
Global & Regional Health Technology Assessment Pub Date : 2021-09-20 eCollection Date: 2021-01-01 DOI: 10.33393/grhta.2021.2286
Giovanni Corrao, Giovanni Alquati, Giovanni Apolone, Andrea Ardizzoni, Giuliano Buzzetti, Giorgio W Canonica, Pierfranco Conte, Elisa Crovato, Francesco Damele, Carlo La Vecchia, Aldo P Maggioni, Alberto Mantovani, Michele Marangi, Walter Marrocco, Andrea Messori, Alessandro Padovani, Alessandro Rambaldi, Walter Ricciardi, Francesco Ripa di Meana, Federico Spandonaro, Valeria Tozzi, Giuseppe Mancia
{"title":"Real-world data: come possono aiutare a migliorare la qualità dell’assistenza.","authors":"Giovanni Corrao, Giovanni Alquati, Giovanni Apolone, Andrea Ardizzoni, Giuliano Buzzetti, Giorgio W Canonica, Pierfranco Conte, Elisa Crovato, Francesco Damele, Carlo La Vecchia, Aldo P Maggioni, Alberto Mantovani, Michele Marangi, Walter Marrocco, Andrea Messori, Alessandro Padovani, Alessandro Rambaldi, Walter Ricciardi, Francesco Ripa di Meana, Federico Spandonaro, Valeria Tozzi, Giuseppe Mancia","doi":"10.33393/grhta.2021.2286","DOIUrl":"10.33393/grhta.2021.2286","url":null,"abstract":"","PeriodicalId":12627,"journal":{"name":"Global & Regional Health Technology Assessment","volume":"8 ","pages":"134-139"},"PeriodicalIF":0.4,"publicationDate":"2021-09-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/4a/f8/grhta-8-134.PMC9616184.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10574037","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
Comments on "Continuity of care between hospital pharmacies and community pharmacies, and costs avoided: a pilot experience in times of COVID-19 in Spain". 关于 "医院药房与社区药房之间的持续护理以及避免的成本:西班牙 COVID-19 期间的试点经验 "的评论
IF 0.4
Global & Regional Health Technology Assessment Pub Date : 2021-05-04 eCollection Date: 2021-01-01 DOI: 10.33393/grhta.2021.2261
David Gómez Gómez
{"title":"Comments on \"Continuity of care between hospital pharmacies and community pharmacies, and costs avoided: a pilot experience in times of COVID-19 in Spain\".","authors":"David Gómez Gómez","doi":"10.33393/grhta.2021.2261","DOIUrl":"10.33393/grhta.2021.2261","url":null,"abstract":"","PeriodicalId":12627,"journal":{"name":"Global & Regional Health Technology Assessment","volume":"8 ","pages":"67"},"PeriodicalIF":0.4,"publicationDate":"2021-05-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/cc/21/grhta-8-67.PMC9616193.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9083776","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
Price and reimbursement for orphan medicines and managed entry agreements: does Italy need a framework? 孤儿药的价格和报销以及管理准入协议:意大利是否需要一个框架?
IF 0.5
Global & Regional Health Technology Assessment Pub Date : 2021-01-01 DOI: 10.33393/grhta.2021.2278
Claudio Jommi, Antonio Addis, Nello Martini, Elena Nicod, Marcello Pani, Annalisa Scopinaro, Sabine Vogler
{"title":"Price and reimbursement for orphan medicines and managed entry agreements: does Italy need a framework?","authors":"Claudio Jommi,&nbsp;Antonio Addis,&nbsp;Nello Martini,&nbsp;Elena Nicod,&nbsp;Marcello Pani,&nbsp;Annalisa Scopinaro,&nbsp;Sabine Vogler","doi":"10.33393/grhta.2021.2278","DOIUrl":"https://doi.org/10.33393/grhta.2021.2278","url":null,"abstract":"<p><p>This article illustrates a consensus opinion of an expert panel on the need and usefulness of a framework for price and reimbursement (P&R) process and managed entry agreements (MEAs) for orphan medicines in Italy. This opinion was gathered in three rounds: an introductory document was sent to the panel and discussed during a recorded online meeting. A second document was sent to the panel for their review. In the third step the final document was validated. Members of the expert panel are the authors of the article. The panel agreed that Italy does not need a specific value framework for orphan medicines, driving the P&R process. Rather, a more structured value framework for all medicines tailored to the specific drugs can be useful. For orphan drugs, the panel advocated for a multidisciplinary approach and the contribution of different stakeholders to value assessment, and acknowledged the importance of addressing, more than for other drugs, unmet needs, equity issues and societal value. The panel raised the need of increasing the importance of patient-reported outcomes. Experts, acknowledging the growing criticisms in implementation of outcome-based agreements in Italy, expressed their position against their abandonment in favour of discounts only and supported orphan medicines as natural candidates for these agreements. Finally, the panel made some recommendations on the appraisal process for orphan medicines, including an early discussion on the uncertainty of the evidence generated and the adoption of a structured approach to identify the agreement, which better responds to the uncertainty.</p>","PeriodicalId":12627,"journal":{"name":"Global & Regional Health Technology Assessment","volume":"8 ","pages":"114-119"},"PeriodicalIF":0.5,"publicationDate":"2021-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/21/66/grhta-8-114.PMC9616180.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9083771","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: 3
A comparative analysis of international health technology assessments for novel gene silencing therapies: patisiran and inotersen. 新型基因沉默疗法的国际卫生技术评估的比较分析:patisiran和intertersen。
IF 0.5
Global & Regional Health Technology Assessment Pub Date : 2021-01-01 DOI: 10.33393/grhta.2021.2193
Sergio Iannazzo
{"title":"A comparative analysis of international health technology assessments for novel gene silencing therapies: patisiran and inotersen.","authors":"Sergio Iannazzo","doi":"10.33393/grhta.2021.2193","DOIUrl":"https://doi.org/10.33393/grhta.2021.2193","url":null,"abstract":"<p><strong>Objectives: </strong>Using the case study of patisiran and inotersen, we conducted a narrative comparative analysis of the health technology assessment (HTA) agency appraisals of these two first-in-class transthyretin gene silencers, which represent exceptional advances in the treatment of hereditary transthyretin-mediated (hATTR) amyloidosis, a rare and multisystemic disease. Despite the impact of each product on the treatment landscape, the majority of HTAs are only considered standard of care as a comparator, resulting in a void of information and limited comprehension of the clinical and pharmacoeconomic differences between the two treatments.</p><p><strong>Methods: </strong>A search was conducted internationally for HTA reports, and only instances where assessment decisions for both treatments were publicly available were included in the present analysis. The HTA reports were analyzed broadly for the assessment of clinical and pharmacoeconomic evidence. Only economic models considering both patisiran and inotersen were included in this analysis.</p><p><strong>Results: </strong>A total of nine agencies with public assessment reports for both treatments were identified. HTA agency assessments for both treatments were essentially positive; however, differences were noted in the final recommendations, place in treatment or reimbursed indications, and in the narrative of the evaluations. Only the Canadian Agency for Drugs and Technologies in Health (CADTH) assessment for patisiran evaluated an economic model comparing the two treatments.</p><p><strong>Conclusions: </strong>The differences summarized in this comparative analysis may provide a more comprehensive overview of the two treatments.</p>","PeriodicalId":12627,"journal":{"name":"Global & Regional Health Technology Assessment","volume":"8 ","pages":"14-21"},"PeriodicalIF":0.5,"publicationDate":"2021-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/3c/92/grhta-8-14.PMC9616187.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10574039","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}
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