Global & Regional Health Technology Assessment最新文献

{"title":"Il futuro delle forme di distribuzione dei farmaci in regime di Servizio Sanitario Nazionale.","authors":"Massimo Medaglia, Giuliano Buzzetti, Marco Cossolo, Paola Deambrosis, Giovanna Scroccaro","doi":"10.33393/grhta.2024.2619","DOIUrl":"https://doi.org/10.33393/grhta.2024.2619","url":null,"abstract":"","PeriodicalId":12627,"journal":{"name":"Global & Regional Health Technology Assessment","volume":"11 ","pages":"17-21"},"PeriodicalIF":0.5,"publicationDate":"2024-01-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10792386/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139484939","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 healthcare resource consumption for patients on hemodialysis treated for chronic kidney disease-associated pruritus (CKD-aP).","authors":"Silvia Calabria, Lucio Manenti, Giulia Ronconi, Carlo Piccinni, Letizia Dondi, Leonardo Dondi, Antonella Pedrini, Immacolata Esposito, Alice Addesi, Filippo Aucella, Nello Martini","doi":"10.33393/grhta.2024.2696","DOIUrl":"10.33393/grhta.2024.2696","url":null,"abstract":"<p><strong>Background: </strong>Chronic kidney disease-associated pruritus (CKD-aP) affects patients on hemodialysis. This study identified hemodialysis patients presumably affected or not affected by CKD-aP and integrated healthcare costs, from the perspective of the Italian administrative healthcare data.</p><p><strong>Methods: </strong>Through cross-linkage of Italian administrative healthcare data collected between 2015 and 2017 (accrual period) in the database of Fondazione ReS (Ricerca e Salute), patients undergoing in-hospital/outpatient hemodialysis were selected. Cohorts with and without CKD-aP were created based on the presence/absence of CKD-aP-related treatment (according to common clinical practice and guidelines) supplies and assessed in terms of CKD-aP-related treatments and mean healthcare costs per capita paid by the Italian National Health Service (INHS).</p><p><strong>Results: </strong>Of 1,239 people on hemodialysis for ≥2 years, CKD-aP affected 218 patients. Patients with CKD-aP were older and with more comorbidities. During the follow-up year, on average, the INHS spent €37,065 per case, €31,286 per control and € 35,988 per non-CKD-aP subject. High-efficiency dialytic therapies performed to people on hemodialysis with CKD-aP largely weighed on the overall mean annual cost.</p><p><strong>Conclusions: </strong>This real-world study identified patients on chronic hemodialysis potentially treated for CKD-aP. Interestingly, high-efficiency dialysis seems the most frequent and expensive choice for the treatment of CKD-aP. The discovery of appropriate and effective treatments for this condition might offer cost offsets.</p>","PeriodicalId":12627,"journal":{"name":"Global & Regional Health Technology Assessment","volume":"11 ","pages":"22-30"},"PeriodicalIF":0.5,"publicationDate":"2024-01-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10792387/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139484946","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":"Farmaci orfani in Italia: disponibilità e tempi di accesso a livello regionale.","authors":"Maria Lucia Marino, Eva Alessi, Aurora Di Filippo, Barbara Polistena, Francesco Macchia, Federico Spandonaro, Francesco Trotta","doi":"10.33393/grhta.2023.2641","DOIUrl":"10.33393/grhta.2023.2641","url":null,"abstract":"","PeriodicalId":12627,"journal":{"name":"Global & Regional Health Technology Assessment","volume":"10 ","pages":"89-97"},"PeriodicalIF":0.5,"publicationDate":"2023-12-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10757301/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139073770","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":"Time to market access in Italia: durata del processo di P&R per i farmaci per le malattie rare.","authors":"Andrea Marcellusi, Paola Raimondo, Carlotta Galeone, Pier Luigi Canonico","doi":"10.33393/grhta.2023.2610","DOIUrl":"10.33393/grhta.2023.2610","url":null,"abstract":"Time to market access in Italy: duration of the P&R process for rare disease drugs Objective: This paper aims to investigate the duration of the pricing & reimbursement (P&R) procedures submitted in Italy by pharmaceutical marketing authorization holders (MAH) for drugs indicated for rare diseases. Methods: All the data used in this analysis were publicly available on different sources of the Italian Ministry of Health, the Italian Medicines Agency (AIFA) and other official websites. The information was systematically collected to investigate the timeline (days) needed to complete the P&R process. The process was divided into 6 simplified steps and the median and range of days needed for each phase were estimated based on data reported in official/published documents. The analysis was stratified considering every single step of the assessment phase and included segmentation of drugs into indications for rare diseases, Orphan designation, Innovation assessment and Managed entry agreements (MEAs). Results: Overall, 181 first indication procedures were submitted to AIFA in the period considered and, of these, 167 (92.3%) were completed and 129 procedures were considered for the final analysis and the median duration of the entire process (MAH submission to final Gazette publication) was 434 days (range 176.0-918.0). The duration of procedures for rare diseases (n = 53) was longer than those for non-rare-disease procedures (n = 76) (463.0 days vs 407.5 days respectively). Among rare disease procedures, orphan designation and MEAs represent predictors for time prolongation while innovation is associated with a shorter assessment time. Conclusion: The study describes the time spent in each phase of the assessment and the appraisal process and demonstrates that uncertainty represents the main driver for the increment in the overall time.","PeriodicalId":12627,"journal":{"name":"Global & Regional Health Technology Assessment","volume":"10 ","pages":"79-88"},"PeriodicalIF":0.5,"publicationDate":"2023-10-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10628501/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"71524075","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":"Il bisogno insoddisfatto: definizioni, evidenze di letteratura e implicazioni per l’HTA.","authors":"Claudio Jommi,&nbsp;Michela Meregaglia,&nbsp;Carmine Pinto","doi":"10.33393/grhta.2023.2624","DOIUrl":"10.33393/grhta.2023.2624","url":null,"abstract":"Unmet need: definitions, literature evidence and implications for HTA The present paper illustrates the definition of unmet need provided by the peer-reviewed literature and the Health Technology Assessment (HTA) authorities across Europe in the assessment and appraisal process and within the early access schemes for medicines. The analysis relied on a descriptive review of the peer-reviewed literature and HTA documents on the definition of need (disease severity) and the way it is satisfied (existence and validity of alternatives). HTA agencies were found using (i) a narrow definition of need, focused on the clinical impact and the impact on health-related quality of life of the disease and (ii) a broad definition of comparators, including treatments used off-label in the clinical practice. Most of the contributions of the literature advocated for a broader definition of need, including additional dimensions (for example, the socio-economic impact of the disease) and the effects of existing treatments beyond their risk-benefit profile (for example, acceptability to patients). On the one hand, these contributions should be considered by HTA agencies, considering its multi-disciplinary and multi-stakeholder nature. On the other one, the explicit inclusion of the unmet need domains, at present disregarded, should depend on the decisions taken on the ground of the assessment.","PeriodicalId":12627,"journal":{"name":"Global & Regional Health Technology Assessment","volume":"10 ","pages":"70-78"},"PeriodicalIF":0.5,"publicationDate":"2023-10-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/96/f6/grhta-10-70.PMC10551671.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"41139058","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":"Cost-effectiveness analysis of abemaciclib with endocrine therapy (ET) versus ET alone for HR+, HER2-, node-positive, high-risk early breast cancer in Italy.","authors":"Alison Davie,&nbsp;Sory Traoré,&nbsp;Massimo Giovannitti,&nbsp;Giuseppe Pompilio,&nbsp;Mark Lambton,&nbsp;Esra Cakar,&nbsp;Anuja Chatterjee","doi":"10.33393/grhta.2023.2561","DOIUrl":"10.33393/grhta.2023.2561","url":null,"abstract":"<p><strong>Background: </strong>Abemaciclib was recently approved by the European Medicines Agency in combination with adjuvant endocrine therapy (ET) for adult patients with hormone receptor positive (HR+), human epidermal growth factor receptor 2 negative (HER2-), node-positive early breast cancer (EBC) at high risk of recurrence.</p><p><strong>Objective: </strong>To evaluate the cost-effectiveness of abemaciclib plus ET vs. ET alone in patients with HR+, HER2-, node-positive EBC at high risk of disease recurrence, from the Italian healthcare system perspective.</p><p><strong>Methods: </strong>A cohort state transition model was developed with five states: invasive disease-free survival (IDFS), nonmetastatic recurrence, remission, metastatic recurrence, and death. The analysis had a time horizon of 30 years. Individual patient-level data from the monarchE trial (NCT03155997) were used to generate IDFS estimates. Resource use included drug acquisition/administration, best supportive care, terminal care, adverse events, hospitalization, post-progression therapy, and associated resource use in the metastatic disease health state. Health state utilities were derived from monarchE patient-level data and other sources, applying Italian tariffs where feasible.</p><p><strong>Results: </strong>The estimated total discounted costs (€39,249 vs. €16,806; difference: €22,443) and quality-adjusted life years (QALYs) (11.49 vs. 10.50; difference: 0.99) were higher for abemaciclib plus ET compared with ET alone. The incremental cost-effectiveness ratio was €22,651 per QALY gained. The likelihood of abemaciclib plus ET being cost-effective vs. ET alone was 99% at a willingness-to-pay threshold of €30,000 per QALY gained.</p><p><strong>Conclusion: </strong>Abemaciclib plus ET is a cost-effective treatment option vs. ET alone for those with HR+, HER2- node-positive EBC at high risk of recurrence in Italy.</p>","PeriodicalId":12627,"journal":{"name":"Global & Regional Health Technology Assessment","volume":"10 ","pages":"62-69"},"PeriodicalIF":0.5,"publicationDate":"2023-09-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/22/ef/grhta-10-62.PMC10551672.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"41101679","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":"COVID-19 impact on the decision process of the Italian Medicine Agency: a quantitative assessment.","authors":"Beatrice Canali, Laura Candelora, Francesca Fiorentino, Tom Halmos, Paola La Malfa, Francesca Massara, Chiara Vassallo, Duccio Urbinati","doi":"10.33393/grhta.2023.2560","DOIUrl":"10.33393/grhta.2023.2560","url":null,"abstract":"<p><strong>Background: </strong>Since the COVID-19 pandemic has placed more attention on drugs' approval process and the importance of rapid decision-making in the healthcare sector, it is crucial to assess how time to market (TTM) of drugs varied.</p><p><strong>Objective: </strong>To estimate the impact of the COVID-19 pandemic on TTM of drugs in Italy.</p><p><strong>Methods: </strong>An IQVIA database was used to retrieve information on drugs that obtained positive opinion from the Committee for Medicinal Products for Human Use between January 2015 and December 2021. The available observations were divided into three groups (Pre COVID, Partially COVID, and Fully COVID) according to the timing of their negotiation process. Differences in average TTM among the three groups were analyzed in three steps: (1) descriptive statistics; (2) univariate analysis; (3) multivariate analysis, using a matching estimator.</p><p><strong>Results: </strong>A total of 363 unique combinations of molecule and indication met the inclusion criteria: 174 in the Pre COVID group, 69 in the Partially COVID group, and 123 in the Fully COVID group. Descriptive statistics and univariate analysis found a statistically significant difference in TTM among the three periods, with average TTM increasing during the pandemic (+136 days, p = 0.00) and then decreasing afterward (-23 days, p = 0.09). In the matching analysis, results for the Partially COVID period were confirmed (+108 days, p = 0.00) while results for the Fully COVID period lost significance but maintained a negative sign.</p><p><strong>Conclusions: </strong>The results suggest that after an adjustment phase in the Partially COVID period, a return to the <i>status quo</i> was reached.</p>","PeriodicalId":12627,"journal":{"name":"Global & Regional Health Technology Assessment","volume":"10 ","pages":"46-52"},"PeriodicalIF":0.4,"publicationDate":"2023-05-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/0c/76/grhta-10-46.PMC10238912.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9939860","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 impatto sul budget sanitario italiano di enzalutamide per il trattamento del carcinoma prostatico metastatico ormono-sensibile.","authors":"Francesca Fiorentino, Paolo Di Rienzo","doi":"10.33393/grhta.2023.2507","DOIUrl":"10.33393/grhta.2023.2507","url":null,"abstract":"","PeriodicalId":12627,"journal":{"name":"Global & Regional Health Technology Assessment","volume":"10 ","pages":"29-39"},"PeriodicalIF":0.4,"publicationDate":"2023-04-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/d7/c2/grhta-10-29.PMC10105321.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9317186","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":"Buone pratiche per lo sviluppo dei modelli di budget impact a livello regionale.","authors":"Andrea Marcellusi,&nbsp;Angela Ragonese,&nbsp;Andrea Marinozzi,&nbsp;Alberto Bortolami,&nbsp;Sara Mucherino,&nbsp;Carolina Moreno,&nbsp;Amalia Antenori,&nbsp;Matteo Ferrario,&nbsp;Claudia Simonelli,&nbsp;Matteo Zanuzzi,&nbsp;Marco Cicoira,&nbsp;Ruggero Lasala,&nbsp;Francesco Russoniello,&nbsp;Francesco Attanasio,&nbsp;Caterina Donati,&nbsp;Chiara Roni,&nbsp;Fabrizio Gemmi,&nbsp;Francesco Saverio Mennini,&nbsp;Pierluigi Russo,&nbsp;Giovanna Scroccaro,&nbsp;Pier Luigi Canonico","doi":"10.33393/grhta.2023.2582","DOIUrl":"https://doi.org/10.33393/grhta.2023.2582","url":null,"abstract":"Good practices for the development of budget impact models at regional level Introduction: The present work aims to discuss the current scenario of procedures and regulations regarding budget impact analysis/models (BIA/BIM) at regional level in Italy and to provide a standardized approach and detailed recommendations for developing these analyses. Method: A systematic review of the literature was conducted in order to collect existing guidelines or specific regional procedures for budget impact analysis in Italy. All the records were analysed in qualitative terms according to a pre-specified analytical framework, based on the ISPOR BIA guidelines. At the end of the analysis, a consensus questionnaire was developed to establish agreed approaches and to provide possible solutions to any critical issues. A list of 39 statements was developed. The survey was distributed to 69 experts who rated their level of agreement with each statement on a 5-point Likert scale. Consensus was predefined as more than 66% of the panel agreeing/disagreeing with any given statement. Results: Sisty-nine experts answered the questionnaire; a total of 30/39 statements achieved consensus. There was agreement on most of the statements. Time horizon to consider and costs were the issues on which no agreement was found. The results allowed the working group to define a list of good practices. Conclusion: While the structure and development of BIM are now well-known and well-applied at national level, there remains a great diversity of management of BIM tools at regional level. Consensus was reached among participating experts, as to the main characteristics, determinants and features of regional BIA/BIM in the perspective of the Italian payer.","PeriodicalId":12627,"journal":{"name":"Global & Regional Health Technology Assessment","volume":"10 ","pages":"53-61"},"PeriodicalIF":0.5,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/e8/b0/grhta-10-53.PMC10318586.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10180422","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":"Costo-efficacia di IMI/CIL/REL rispetto a CMS+IMI per il trattamento di infezioni batteriche Gram-negative non sensibili ai carbapenemi.","authors":"Martina Paoletti,&nbsp;Andrea Marcellusi,&nbsp;Joe Yang,&nbsp;Francesco Saverio Mennini","doi":"10.33393/grhta.2023.2488","DOIUrl":"https://doi.org/10.33393/grhta.2023.2488","url":null,"abstract":"Cost-effectiveness of IMI/CIL/REL for the treatment of carbapenem non-susceptible Gram-negative bacterial infections Objective: The objective of this analysis was to evaluate the cost-effectiveness of imipenem/cilastatin/relebactam compared to colistin-imipenem in the treatment of hospitalized patients with Gram-negative bacterial infections caused by imipenem-resistant pathogens. The perspective was both that of the National Health Service (NHS) and the social one. Methodology: A mixed model was developed to simulate a cohort of patients capable of highlighting the impacts of the disease on the quality of life and the absorption of economic resources of the patients in analysis. Modelled patients were those with hospital-acquired bacterial pneumonia/ventilator-associated bacterial pneumonia (HABP/VABP), complicated intra-abdominal infection (cIAI) or complicated urinal tract infection (cUTI) caused by carbapenem-resistant Gram-negative (GN) pathogens. The model begins with a short-term decision tree describing possible treatment routes and outcomes for patients during the hospitalization period. Patients who are healed in the decision tree enter the long-term Markov model, designed to capture the follow-up costs and health-related quality of life (HRQL) of patients healed over their lifetime. Results: The analysis, conducted on a hypothetical cohort of 1,000 patients, highlights how the use of imipenem/cilastatin/relebactam is advantageous both in terms of diagnosis and treatment in the short term and in terms of cost-effectiveness. In fact, it is dominant compared to colistin-imipenem both in the NHS and in the social perspective since, compared to an average saving of € 2,800.15 and € 3,174.63 respectively, it would generate an increase of 4.76 years of life and of 4.12 QALYs per patient.","PeriodicalId":12627,"journal":{"name":"Global & Regional Health Technology Assessment","volume":"10 ","pages":"18-28"},"PeriodicalIF":0.5,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/0e/55/grhta-10-18.PMC10105322.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9317188","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}