Pub Date : 2024-05-21DOI: 10.33393/grhta.2024.2735
Claudio Jommi, Federico Pantellini, Giovanni Giuliani, M. Cavazza
Background: Compassionate use programs (CUP) for medicines allow patients, not recruited in trials, accessing medicines before marketing approval. The evidence on the economic impact of CUP is limited to one paper that did not include the possible long-term economic impact of medicines used in CUP compared to alternative treatments, like avoided hospitalization costs. This paper aims at covering this information gap, updating and integrating the existing evidence, with a cost analysis in the perspective of the Italian National Health Service (SSN). Methods: Data were retrieved from the Roche CUP Database, the peer-reviewed literature (e.g. published cost-effectiveness studies, if any), the gray literature (e.g. regional documents for the alternative treatment cost) and unpublished data (e.g. cost-effectiveness analysis provided for price and reimbursement negotiation for medicines in CUP). Results: We retrieved data from 14 CUPs and 3,485 patients (70% affected by tumors) (Jan 2015-August 2022). The alternative treatment mean cost per patient ranged from € 11.1 k to € 18.8 k. The net economic benefit ranged from € 47 million to € 75 million (€ 13.5 k-€ 21.5 k per patient). Avoiding alternative treatments mostly contributes to the economic benefit. The long-term economic impact accounted for 20/30% of the net economic impact. The net economic benefit for cancer drugs ranged from € 12.6 million to € 40.0 million. Conclusions: Despite research limitations, this paper provides additional evidence on the economic impact of CUP in Italy. This could represent the first step of a broader analysis of the economic impact of early access schemes for drugs.
{"title":"The economic impact of 14 compassionate use programs for medicines in Italy, in the perspective of the National Health Service","authors":"Claudio Jommi, Federico Pantellini, Giovanni Giuliani, M. Cavazza","doi":"10.33393/grhta.2024.2735","DOIUrl":"https://doi.org/10.33393/grhta.2024.2735","url":null,"abstract":"Background: Compassionate use programs (CUP) for medicines allow patients, not recruited in trials, accessing medicines before marketing approval. The evidence on the economic impact of CUP is limited to one paper that did not include the possible long-term economic impact of medicines used in CUP compared to alternative treatments, like avoided hospitalization costs. This paper aims at covering this information gap, updating and integrating the existing evidence, with a cost analysis in the perspective of the Italian National Health Service (SSN).\u0000Methods: Data were retrieved from the Roche CUP Database, the peer-reviewed literature (e.g. published cost-effectiveness studies, if any), the gray literature (e.g. regional documents for the alternative treatment cost) and unpublished data (e.g. cost-effectiveness analysis provided for price and reimbursement negotiation for medicines in CUP).\u0000Results: We retrieved data from 14 CUPs and 3,485 patients (70% affected by tumors) (Jan 2015-August 2022). The alternative treatment mean cost per patient ranged from € 11.1 k to € 18.8 k. The net economic benefit ranged from € 47 million to € 75 million (€ 13.5 k-€ 21.5 k per patient). Avoiding alternative treatments mostly contributes to the economic benefit. The long-term economic impact accounted for 20/30% of the net economic impact. The net economic benefit for cancer drugs ranged from € 12.6 million to € 40.0 million.\u0000Conclusions: Despite research limitations, this paper provides additional evidence on the economic impact of CUP in Italy. This could represent the first step of a broader analysis of the economic impact of early access schemes for drugs.","PeriodicalId":228031,"journal":{"name":"Global and Regional Health Technology Assessment","volume":"67 8","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-05-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141116688","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-04-22DOI: 10.33393/grhta.2024.3013
Melania Dovizio, Melania Leogrande, Luca Degli Esposti
Introduction: Heart failure (HF) affects 1% of subjects aged 45-55 and over 10% of subjects aged ≥ 80 and in Italy represents the third leading cause of hospitalization. Objective: To analyse the clinical and economic burden of HF in the Italian real clinical practice. Methods: A retrospective analysis was conducted on the administrative databases of healthcare institutions for 4.2 million health-assisted residents. Between January 2012 and March 2021, patients with a hospital discharge diagnosis for HF were included. Among healthcare utilization and costs, treatments, hospitalizations, and specialist services were evaluated. The HF group was compared with a population without HF (no-HF) similar for age, sex distribution, and cardiovascular risk factors. Results: The same number of patients with (N = 74,085) and without HF (N = 74,085) was included. A profile of cardiovascular comorbidities emerged in the HF group, mainly hypertension (88.6%), cardiovascular disease (61.3%) and diabetes (32.1%). Hospitalizations from any cause were 635.6 vs 429.8/1,000 person-year in the HF vs no-HF group. At one-year follow-up, all-cause mortality was 24.9% in HF patients and 8.4% in no-HF. Resource utilization/patient was respectively 26.8 ± 15.9 vs 17.1 ± 12.5 for medications, 0.8 ± 1.2 vs 0.3 ± 0.8 for hospitalizations, and 9.4 ± 12.6 vs 6.5 ± 9.8 for specialist services. This resource utilization resulted in significantly higher total healthcare costs in the HF group vs no-HF group (€ 5,910 vs € 3,574, p < 0.001), mainly related to hospitalizations (€ 3,702 vs € 1,958). Conclusions: HF patients show a significantly higher clinical and economic burden than no-HF, with total healthcare costs being about 1.7 times the costs of the no-HF group. Keywords: Cardiovascular comorbidities, Healthcare costs, Heart failure, Hospitalizations
{"title":"Heart failure and economic impact: an analysis in real clinical practice in Italy","authors":"Melania Dovizio, Melania Leogrande, Luca Degli Esposti","doi":"10.33393/grhta.2024.3013","DOIUrl":"https://doi.org/10.33393/grhta.2024.3013","url":null,"abstract":"Introduction: Heart failure (HF) affects 1% of subjects aged 45-55 and over 10% of subjects aged ≥ 80 and in Italy represents the third leading cause of hospitalization.\u0000Objective: To analyse the clinical and economic burden of HF in the Italian real clinical practice.\u0000Methods: A retrospective analysis was conducted on the administrative databases of healthcare institutions for 4.2 million health-assisted residents. Between January 2012 and March 2021, patients with a hospital discharge diagnosis for HF were included. Among healthcare utilization and costs, treatments, hospitalizations, and specialist services were evaluated. The HF group was compared with a population without HF (no-HF) similar for age, sex distribution, and cardiovascular risk factors.\u0000Results: The same number of patients with (N = 74,085) and without HF (N = 74,085) was included. A profile of cardiovascular comorbidities emerged in the HF group, mainly hypertension (88.6%), cardiovascular disease (61.3%) and diabetes (32.1%). Hospitalizations from any cause were 635.6 vs 429.8/1,000 person-year in the HF vs no-HF group. At one-year follow-up, all-cause mortality was 24.9% in HF patients and 8.4% in no-HF. Resource utilization/patient was respectively 26.8 ± 15.9 vs 17.1 ± 12.5 for medications, 0.8 ± 1.2 vs 0.3 ± 0.8 for hospitalizations, and 9.4 ± 12.6 vs 6.5 ± 9.8 for specialist services. This resource utilization resulted in significantly higher total healthcare costs in the HF group vs no-HF group (€ 5,910 vs € 3,574, p < 0.001), mainly related to hospitalizations (€ 3,702 vs € 1,958).\u0000Conclusions: HF patients show a significantly higher clinical and economic burden than no-HF, with total healthcare costs being about 1.7 times the costs of the no-HF group.\u0000Keywords: Cardiovascular comorbidities, Healthcare costs, Heart failure, Hospitalizations","PeriodicalId":228031,"journal":{"name":"Global and Regional Health Technology Assessment","volume":"99 41","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-04-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140676445","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-01-09DOI: 10.33393/grhta.2024.2601
Michele Basile, Ilaria Valentini, R. Attanasio, Renato Cozzi, Agnese Persichetti, I. Samperi, Alessandro Scoppola, Renata S. Auriemma, E. Menis, Felice Esposito, Emanuele Ferrante, Giuseppe Iatì, Diego Mazzatenta, Maurizio Poggi, Roberta Rudà, Fabio Tortora, Fabio Cruciani, Z. Mitrova, Rosella Saulle, Simona Vecchi, P. Cappabianca, A. Paoletta, A. Bozzao, Marco Caputo, Francesco Doglietto, Francesco Ferraù, A. Lania, Stefano Laureti, S. Lello, Davide Locatelli, Pietro Maffei, Giuseppe Minniti, A. Peri, Chiara Ruini, Fabio Settanni, Antonio Silvani, Nadia Veronese, Franco Grimaldi, E. Papini, Americo Cicchetti
Background: Prolactinoma, the most common pituitary adenoma, is usually treated with dopamine agonist (DA) therapy like cabergoline. Surgery is second-line therapy, and radiotherapy is used if surgical treatment fails or in relapsing macroprolactinoma. Objective: This study aimed to provide economic evidence for the management of prolactinoma in Italy, using a cost-of-illness and cost-utility analysis that considered various treatment options, including cabergoline, bromocriptine, temozolomide, radiation therapy, and surgical strategies. Methods: The researchers conducted a systematic literature review for each research question on scientific databases and surveyed a panel of experts for each therapeutic procedure’s specific drivers that contributed to its total cost. Results: The average cost of the first year of treatment was €2,558.91 and €3,287.40 for subjects with microprolactinoma and macroprolactinoma, respectively. Follow-up costs from the second to the fifth year after initial treatment were €798.13 and €1,084.59 per year in both groups. Cabergoline had an adequate cost-utility profile, with an incremental cost-effectiveness ratio (ICER) of €3,201.15 compared to bromocriptine, based on a willingness-to-pay of €40,000 per quality-adjusted life year (QALY) in the reference economy. Endoscopic surgery was more cost-effective than cabergoline, with an ICER of €44,846.64. Considering a willingness-to-pay of €40,000/QALY, the baseline findings show cabergoline to have high cost utility and endoscopic surgery just a tad above that. Conclusions: Due to the favorable cost-utility profile and safety of surgical treatment, pituitary surgery should be considered more frequently as the initial therapeutic approach. This management choice could lead to better outcomes and an appropriate allocation of healthcare resources.
{"title":"A pharmacoeconomic analysis from Italian guidelines for the management of prolactinomas","authors":"Michele Basile, Ilaria Valentini, R. Attanasio, Renato Cozzi, Agnese Persichetti, I. Samperi, Alessandro Scoppola, Renata S. Auriemma, E. Menis, Felice Esposito, Emanuele Ferrante, Giuseppe Iatì, Diego Mazzatenta, Maurizio Poggi, Roberta Rudà, Fabio Tortora, Fabio Cruciani, Z. Mitrova, Rosella Saulle, Simona Vecchi, P. Cappabianca, A. Paoletta, A. Bozzao, Marco Caputo, Francesco Doglietto, Francesco Ferraù, A. Lania, Stefano Laureti, S. Lello, Davide Locatelli, Pietro Maffei, Giuseppe Minniti, A. Peri, Chiara Ruini, Fabio Settanni, Antonio Silvani, Nadia Veronese, Franco Grimaldi, E. Papini, Americo Cicchetti","doi":"10.33393/grhta.2024.2601","DOIUrl":"https://doi.org/10.33393/grhta.2024.2601","url":null,"abstract":"Background: Prolactinoma, the most common pituitary adenoma, is usually treated with dopamine agonist (DA) therapy like cabergoline. Surgery is second-line therapy, and radiotherapy is used if surgical treatment fails or in relapsing macroprolactinoma.\u0000Objective: This study aimed to provide economic evidence for the management of prolactinoma in Italy, using a cost-of-illness and cost-utility analysis that considered various treatment options, including cabergoline, bromocriptine, temozolomide, radiation therapy, and surgical strategies.\u0000Methods: The researchers conducted a systematic literature review for each research question on scientific databases and surveyed a panel of experts for each therapeutic procedure’s specific drivers that contributed to its total cost.\u0000Results: The average cost of the first year of treatment was €2,558.91 and €3,287.40 for subjects with microprolactinoma and macroprolactinoma, respectively. Follow-up costs from the second to the fifth year after initial treatment were €798.13 and €1,084.59 per year in both groups. Cabergoline had an adequate cost-utility profile, with an incremental cost-effectiveness ratio (ICER) of €3,201.15 compared to bromocriptine, based on a willingness-to-pay of €40,000 per quality-adjusted life year (QALY) in the reference economy. Endoscopic surgery was more cost-effective than cabergoline, with an ICER of €44,846.64. Considering a willingness-to-pay of €40,000/QALY, the baseline findings show cabergoline to have high cost utility and endoscopic surgery just a tad above that.\u0000Conclusions: Due to the favorable cost-utility profile and safety of surgical treatment, pituitary surgery should be considered more frequently as the initial therapeutic approach. This management choice could lead to better outcomes and an appropriate allocation of healthcare resources.","PeriodicalId":228031,"journal":{"name":"Global and Regional Health Technology Assessment","volume":"6 6","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-01-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139441368","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2023-12-27DOI: 10.33393/grhta.2023.2970
Marcello Pani
Hospital and community pharmacists play a crucial role in ensuring the seamless continuity of care for individuals managing epilepsy. They contribute to medication management, providing education, monitoring drug interactions, and fostering collaboration between healthcare providers. Their involvement is essential for optimizing treatment outcomes and enhancing the overall well-being of patients with epilepsy.
{"title":"The role of the hospital and community pharmacist in continuity of care for the management of epilepsy","authors":"Marcello Pani","doi":"10.33393/grhta.2023.2970","DOIUrl":"https://doi.org/10.33393/grhta.2023.2970","url":null,"abstract":"Hospital and community pharmacists play a crucial role in ensuring the seamless continuity of care for individuals managing epilepsy. They contribute to medication management, providing education, monitoring drug interactions, and fostering collaboration between healthcare providers. Their involvement is essential for optimizing treatment outcomes and enhancing the overall well-being of patients with epilepsy.","PeriodicalId":228031,"journal":{"name":"Global and Regional Health Technology Assessment","volume":"61 5","pages":""},"PeriodicalIF":0.0,"publicationDate":"2023-12-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139154638","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2023-12-27DOI: 10.33393/grhta.2023.2888
Luca Buscioni, Giancarlo Di Gennaro, Claudia Passamonti
Epilepsy is one of the most common serious chronic neurological diseases affecting around 50 million people globally and 500,000 patients in Italy. It causes uncontrollable, unprovoked, and unpredictable seizures that reduce the quality of life of those afflicted. It is widely recognized that the most important strategy for controlling seizures and enhance well-being includes receiving and adhering to anti-seizure medications as well as making appropriate lifestyle adjustments. However it is estimated that 30–60% of patients do not adhere to their antiepileptic medication regimens. EpiOnApp is a digital Patients Support Program (PSP) designed to empower patients with epilepsy who are taking Cenobamate for their chronic condition to take control of their health care pathway, from inception throughout the duration of the treatment, and to have a meaningful dialogue with their neurologist by sharing health records and emotional states. Moreover, as patients may have limited mobility, they can order their therapy and have it home delivered in a very easy way, and they can benefit from continuous human support through the figure of a dedicated Care Manager. The advantage for the neurologist is that the platform stores seizure data, facilitating the drug titration and better assessing at the time of the follow-up visit. The efficacy of the program is currently being evaluated. So far, the level of satisfaction expressed by patients and neurologists shows that a well-designed and tailored PSP can address the challenge of improving long-term adherence, helping people with epilepsy through the on-boarding and initial treatment period, reducing discontinuation, and ultimately, delivering a better health outcome.
{"title":"Personalized support programs with innovative digital tools to manage and monitor patients suffering from epilepsy: EpiOnApp case study","authors":"Luca Buscioni, Giancarlo Di Gennaro, Claudia Passamonti","doi":"10.33393/grhta.2023.2888","DOIUrl":"https://doi.org/10.33393/grhta.2023.2888","url":null,"abstract":"Epilepsy is one of the most common serious chronic neurological diseases affecting around 50 million people globally and 500,000 patients in Italy. It causes uncontrollable, unprovoked, and unpredictable seizures that reduce the quality of life of those afflicted. It is widely recognized that the most important strategy for controlling seizures and enhance well-being includes receiving and adhering to anti-seizure medications as well as making appropriate lifestyle adjustments. However it is estimated that 30–60% of patients do not adhere to their antiepileptic medication regimens. EpiOnApp is a digital Patients Support Program (PSP) designed to empower patients with epilepsy who are taking Cenobamate for their chronic condition to take control of their health care pathway, from inception throughout the duration of the treatment, and to have a meaningful dialogue with their neurologist by sharing health records and emotional states. Moreover, as patients may have limited mobility, they can order their therapy and have it home delivered in a very easy way, and they can benefit from continuous human support through the figure of a dedicated Care Manager. The advantage for the neurologist is that the platform stores seizure data, facilitating the drug titration and better assessing at the time of the follow-up visit. The efficacy of the program is currently being evaluated. So far, the level of satisfaction expressed by patients and neurologists shows that a well-designed and tailored PSP can address the challenge of improving long-term adherence, helping people with epilepsy through the on-boarding and initial treatment period, reducing discontinuation, and ultimately, delivering a better health outcome.","PeriodicalId":228031,"journal":{"name":"Global and Regional Health Technology Assessment","volume":"13 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2023-12-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139154115","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2023-12-27DOI: 10.33393/grhta.2023.2889
Oriano Mecarelli
Epilepsy is a widespread social disease that affects all ages of life and often involves both diagnostic and therapeutic difficulties. Beyond seizure control, it is necessary to ensure People with Epilepsy a good quality of life and respect for human rights, seeking to increase self-management capacity and break down stigma. People with Epilepsy should have privileged access to specialized Epilepsy Centers, where multidisciplinary care is possible. These Centers, organized by different levels of complexity, should be uniformly distributed throughout the country and networked together. The scientific community and healthcare organizations must therefore design all necessary strategies so that knowledge about Epilepsy improves among the general population and the most effective pathways of care are effectively implemented.
{"title":"Models of care and relevance of territorial management in assisting Persons with Epilepsy","authors":"Oriano Mecarelli","doi":"10.33393/grhta.2023.2889","DOIUrl":"https://doi.org/10.33393/grhta.2023.2889","url":null,"abstract":"Epilepsy is a widespread social disease that affects all ages of life and often involves both diagnostic and therapeutic difficulties. Beyond seizure control, it is necessary to ensure People with Epilepsy a good quality of life and respect for human rights, seeking to increase self-management capacity and break down stigma. People with Epilepsy should have privileged access to specialized Epilepsy Centers, where multidisciplinary care is possible. These Centers, organized by different levels of complexity, should be uniformly distributed throughout the country and networked together. The scientific community and healthcare organizations must therefore design all necessary strategies so that knowledge about Epilepsy improves among the general population and the most effective pathways of care are effectively implemented.","PeriodicalId":228031,"journal":{"name":"Global and Regional Health Technology Assessment","volume":"67 2","pages":""},"PeriodicalIF":0.0,"publicationDate":"2023-12-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139154609","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2023-12-27DOI: 10.33393/grhta.2023.2890
Marcello Pani
{"title":"New organizational and governance strategies in the management of epilepsy in Italy","authors":"Marcello Pani","doi":"10.33393/grhta.2023.2890","DOIUrl":"https://doi.org/10.33393/grhta.2023.2890","url":null,"abstract":"","PeriodicalId":228031,"journal":{"name":"Global and Regional Health Technology Assessment","volume":"96 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2023-12-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139153655","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Patients’ Associations and HTA for medicines: actual and future role in Italy","authors":"Claudio Jommi, MSc, Arianna Bertolani, Phd","doi":"10.33393/grhta.2018.445","DOIUrl":"https://doi.org/10.33393/grhta.2018.445","url":null,"abstract":"","PeriodicalId":228031,"journal":{"name":"Global and Regional Health Technology Assessment","volume":"3 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2018-04-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"134210395","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
E. Ballatori, S. Palazzo, B. Ruggeri, D. Morale, A. Filice, Carmela Oriolo, A. Vergati, M. Napoletano, I. Mazzoni
A new payment-by-results method, published in a previous issue of this Journal was applied in two Italian oncological centers to show how it is easily implemented. This allowed us to make some considerations concerning clinical oncological research, as well as to draw some conclusions on the possible savings that this approach can deliver to the Italian National Health Service.
{"title":"On the New Payment-by-Results Method for Determining the Fair Price of New Drugs. An Application in Oncology","authors":"E. Ballatori, S. Palazzo, B. Ruggeri, D. Morale, A. Filice, Carmela Oriolo, A. Vergati, M. Napoletano, I. Mazzoni","doi":"10.5301/GRHTA.5000233","DOIUrl":"https://doi.org/10.5301/GRHTA.5000233","url":null,"abstract":"A new payment-by-results method, published in a previous issue of this Journal was applied in two Italian oncological centers to show how it is easily implemented. This allowed us to make some considerations concerning clinical oncological research, as well as to draw some conclusions on the possible savings that this approach can deliver to the Italian National Health Service.","PeriodicalId":228031,"journal":{"name":"Global and Regional Health Technology Assessment","volume":"33 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2016-07-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"123050245","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
M. De Francesco, M. Lamotte, P. Ascierto, P. Di Rienzo, Y. Asukai
Background Ipilimumab, a fully human monoclonal antibody that blocks CTLA-4 to promote anti-tumour immunity, was the first treatment in metastatic melanoma to show a significant survival benefit. Methods A three-health-state partitioned survival model was developed to assess ipilimumab 3 mg/kg compared to dacarbazine and vemurafenib in first line therapy of advanced melanoma treatment-naive patients in Italy. The outcomes considered were costs, life years (LYs) and quality-adjusted life years (QALYs). Given the lack of trials assessing ipilimumab 3 mg/kg in this subgroup of patients, the efficacy was derived from a dataset of chemo-naive patients. Patient's management costs were estimated based on a micro-costing approach and the cost of adverse events based on both outpatient and inpatient care. Utilities considered were elicited from ipilimumab's clinical trials. Results Basecase results showed that ipilimumab was both more costly and more effective than dacarbazine, with ratios of €38,345/LYs and €49,466/QALYs. By contrast, results vs. vemurafenib showed a marginal increase in health outcomes accompanied by a saving of €32,999, thus making ipilimumab the dominant strategy over vemurafenib in the base-case analysis. Sensitivity analysis showed overall robustness of the model. Conclusions Treatment with ipilimumab showed better results in terms of LYs and QALYs against both comparators. Moreover, ipilimumab was the dominant strategy compared to vemurafenib, thus highly likely to bring both health benefits and cost savings in the Italian setting.
{"title":"Economic Evaluation of Ipilimumab in First Line Treatment of Advanced Melanoma in Italy","authors":"M. De Francesco, M. Lamotte, P. Ascierto, P. Di Rienzo, Y. Asukai","doi":"10.5301/GRHTA.5000230","DOIUrl":"https://doi.org/10.5301/GRHTA.5000230","url":null,"abstract":"Background Ipilimumab, a fully human monoclonal antibody that blocks CTLA-4 to promote anti-tumour immunity, was the first treatment in metastatic melanoma to show a significant survival benefit. Methods A three-health-state partitioned survival model was developed to assess ipilimumab 3 mg/kg compared to dacarbazine and vemurafenib in first line therapy of advanced melanoma treatment-naive patients in Italy. The outcomes considered were costs, life years (LYs) and quality-adjusted life years (QALYs). Given the lack of trials assessing ipilimumab 3 mg/kg in this subgroup of patients, the efficacy was derived from a dataset of chemo-naive patients. Patient's management costs were estimated based on a micro-costing approach and the cost of adverse events based on both outpatient and inpatient care. Utilities considered were elicited from ipilimumab's clinical trials. Results Basecase results showed that ipilimumab was both more costly and more effective than dacarbazine, with ratios of €38,345/LYs and €49,466/QALYs. By contrast, results vs. vemurafenib showed a marginal increase in health outcomes accompanied by a saving of €32,999, thus making ipilimumab the dominant strategy over vemurafenib in the base-case analysis. Sensitivity analysis showed overall robustness of the model. Conclusions Treatment with ipilimumab showed better results in terms of LYs and QALYs against both comparators. Moreover, ipilimumab was the dominant strategy compared to vemurafenib, thus highly likely to bring both health benefits and cost savings in the Italian setting.","PeriodicalId":228031,"journal":{"name":"Global and Regional Health Technology Assessment","volume":"12 1 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2016-07-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"132407387","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
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