Global & Regional Health Technology Assessment最新文献

{"title":"Regolazione del prezzo e rimborso dei farmaci: comparatori, endpoint e ruolo della costo-efficacia.","authors":"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","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}

{"title":"Budget impact analysis sull’introduzione del dispositivo di telemonitoraggio Turbo+ nella gestione dei pazienti asmatici in Italia.","authors":"Filippo Rumi, Michele Basile, Americo Cicchetti, Rosario Contiguglia, Antonella Pentassuglia, Alessandro Oliva, Gianenrico Senna, Marco Bonavia, Francesco Scarpelli, 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}

{"title":"Valutazione economica di treosulfan in pazienti sottoposti a trapianto allogenico di cellule staminali ematopoietiche.","authors":"Chiara Bini, Martina Paoletti, Andrea Marcellusi, Carlo Tomino, 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}

{"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}

{"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}

{"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}

{"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}

{"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}

{"title":"Number needed to treat for interleukin inhibitors approved for the treatment of moderate-to-severe plaque psoriasis in Italy.","authors":"Roberto Ravasio,&nbsp;Antonio Costanzo,&nbsp;Silvia Antonelli,&nbsp;Alessia Maiorino,&nbsp;Serena Losi","doi":"10.33393/grhta.2021.2222","DOIUrl":"https://doi.org/10.33393/grhta.2021.2222","url":null,"abstract":"<p><strong>Background: </strong>Interleukin (IL) inhibitors achieve greater levels of efficacy than older systemic therapies. We calculated the number needed to treat (NNT) of ixekizumab compared with other IL inhibitors approved in Italy for the treatment of moderate-to-severe plaque psoriasis.</p><p><strong>Methods: </strong>The clinical efficacy was evaluated in terms of NNT, based on the results of a recent network meta--analysis (NMA) by the Cochrane Database of Systematic Reviews. The NMA investigated many systemic and biological treatments, but this analysis compared only the efficacy of the following IL inhibitors - brodalumab, guselkumab, ixekizumab, risankizumab, secukinumab, tildrakizumab and ustekinumab - for patients with moderate-to-severe plaque psoriasis. Drugs were compared and ranked according to effectiveness considering the PASI (Psoriasis Area and Severity Index) 90 score.</p><p><strong>Results: </strong>One-hundred and forty trials (51,749 patients) were included in the NMA. Considering the proportion of patients who achieve PASI90, ixekizumab showed the lowest NNT among all comparators (ixekizumab 2.01 [2.46-3.00]; risankizumab 2.05 [2.50-3-05]; guselkumab 2.16 [2.68-3.36]; secukinumab 2.40 [2.90-3.51]; brodalumab 2.61 [3.18-3.88]; ustekinumab 3.44 [4.12-4.95]; tildrakizumab 3.10 [4.15-5.59].</p><p><strong>Conclusion: </strong>The findings show that ixekizumab is the most effective option (NNT) for the treatment of moderate-to-severe plaque psoriasis.</p>","PeriodicalId":12627,"journal":{"name":"Global & Regional Health Technology Assessment","volume":"8 ","pages":"53-57"},"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/3e/d3/grhta-8-53.PMC9616200.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10574038","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":"Analisi di Budget Impact della formulazione depot di buprenorfina a rilascio prolungato per la gestione di pazienti affetti da disturbo da uso di oppiacei.","authors":"Michele Basile,&nbsp;Lorenzo Somaini,&nbsp;Americo Cicchetti","doi":"10.33393/grhta.2021.2237","DOIUrl":"https://doi.org/10.33393/grhta.2021.2237","url":null,"abstract":"Budget Impact Analysis of prolonged-release buprenorphine depot-formulation for the management of patients affected by opioid use disorder Background: Opioid use disorder (OUD) is a disorder associated with significant rate of morbidity and mortality. Frequent clinic attendance for supervised consumption of sublingual buprenorphine is common. Prolonged-release buprenorphine (PRB) allows a management based on weekly or monthly subcutaneous injections, thus limiting the burdens of clinic attendance and the risks associated with sublingual formulations. Objective: To determine the price level of PRB that allows to obtain a neutral impact from the point of view of the economic resources absorbed, in comparison with the alternatives currently available in the Italian context for the management of patients suffering from OUD. Methods: The analysis assumes a daily PRB cost of € 8.526 (neutral cost). The analysis aims to determine the economic impact associated with the introduction of PRB in the Italian context for the management of OUD patients. Results are expressed in terms of differential resources absorbed in the alternative scenarios. A one-way sensitivity analysis was also carried out to test the robustness of the results. Results: The introduction of PRB implies an increase in the drug acquisition costs over the 5-year time horizon of € 23,016,194.61: such costs are fully compensated by the other cost driver considered in the analysis (drug tests provided, health professionals’ time destined to the provision of the treatment, indirect costs, for savings equal to € 7,255,602.59, € 10,714,320.08 and € 5,046,271.94 respectively) demonstrating its effectiveness in particular by an organizational point of view. Lower price levels for PRB would imply significant savings for the SSN. Conclusions: PRB resulted to be associated to a lower level of resources’ absorption in the Italian sector as compared with the available alternatives thus allowing to re-allocate health founds to other fields of the care sector ensuring greater safety for patients and a decreased misuse and diversion rate.","PeriodicalId":12627,"journal":{"name":"Global & Regional Health Technology Assessment","volume":"8 ","pages":"96-104"},"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/6f/85/grhta-8-96.PMC9616199.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9083778","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}