INTRODUCTION: Anti-TNF-α biosimilars (ATB) hold the promise of reducing costs leading in improving access to bio-logical therapies. There is limited insight into how the savings generated by biosimilars may translate into patient benefit in other disease areas. AIMS: To assess the economic savings for Italian National Health System (NHS) due to the expansion of ATB market, together with a reduction in their price and to illustrate how this potential savings can be used by NHS to fund orphan drugs. METHODS: Trend of IMS Health monthly sell-in units (August 2016-December 2019) were used to estimate the current biologic and biosimilar market for rheumatic and inflammatory bowel disease in Italy and its evolution up to 2022. The scenario for 2019-2020 was compared with the future evolution (2021-2022) assuming an increasing uptake of biosimilars in the Italian market. Finally, it was estimated how these savings can potentially fund the treatment of orphan drugs, without increasing the Italian NHS budget. RESULTS: Italian biologic and biosimilar market remains stable in the next years (about 4 million units both in the current scenario and in the future evolution market) with a slight decreasing of less than 2%. However, according to our assump-tions, ATB market is expected to increase of about 33% in the next two years, covering 67% of the total Italian market, mostly due to biosimilar etanercept. Total savings due to biosimilars increases from € 96 million in 2019 to € 161 million in 2022 corresponding to a mean annual savings of about € 130 million. Such savings would permit funding 17.4% of the actual orphan drugs market corresponding to 2,600-4,800 new patients. CONCLUSIONS: The introduction of biosimilars in a range of rheumatic, dermatological and inflammatory bowel disease can be an opportunity to increase patient access to innovative treatments. Potential savings due to biosimilars uptake could lead to a re-allocation of economic resources to fund innovative therapies.
{"title":"Funding Innovation Thanks to Anti-TNF-α Biosimilars Uptake: The Economic Impact in Italy","authors":"M. Povero, L. Pradelli","doi":"10.7175/fe.v21i1.1449","DOIUrl":"https://doi.org/10.7175/fe.v21i1.1449","url":null,"abstract":"INTRODUCTION: Anti-TNF-α biosimilars (ATB) hold the promise of reducing costs leading in improving access to bio-logical therapies. There is limited insight into how the savings generated by biosimilars may translate into patient benefit in other disease areas. AIMS: To assess the economic savings for Italian National Health System (NHS) due to the expansion of ATB market, together with a reduction in their price and to illustrate how this potential savings can be used by NHS to fund orphan drugs. METHODS: Trend of IMS Health monthly sell-in units (August 2016-December 2019) were used to estimate the current biologic and biosimilar market for rheumatic and inflammatory bowel disease in Italy and its evolution up to 2022. The scenario for 2019-2020 was compared with the future evolution (2021-2022) assuming an increasing uptake of biosimilars in the Italian market. Finally, it was estimated how these savings can potentially fund the treatment of orphan drugs, without increasing the Italian NHS budget. RESULTS: Italian biologic and biosimilar market remains stable in the next years (about 4 million units both in the current scenario and in the future evolution market) with a slight decreasing of less than 2%. However, according to our assump-tions, ATB market is expected to increase of about 33% in the next two years, covering 67% of the total Italian market, mostly due to biosimilar etanercept. Total savings due to biosimilars increases from € 96 million in 2019 to € 161 million in 2022 corresponding to a mean annual savings of about € 130 million. Such savings would permit funding 17.4% of the actual orphan drugs market corresponding to 2,600-4,800 new patients. CONCLUSIONS: The introduction of biosimilars in a range of rheumatic, dermatological and inflammatory bowel disease can be an opportunity to increase patient access to innovative treatments. Potential savings due to biosimilars uptake could lead to a re-allocation of economic resources to fund innovative therapies.","PeriodicalId":41585,"journal":{"name":"Farmeconomia-Health Economics and Therapeutic Pathways","volume":"14 1","pages":""},"PeriodicalIF":0.5,"publicationDate":"2020-05-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"84287572","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. N. D. Minno, Lucia S D'Angiolella, P. Cortesi, A. Molinari, L. Mantovani
INTRODUCTION: Hemophilia A is a rare congenital bleeding disorder caused by a deficiency of clotting factor VIII (FVIII). The severe form of the disease is characterized by spontaneous bleeds, especially into the joints. Prophylaxis, based on regularly intravenous administration of the missing factor to avoid hemorrhages, represents the gold standard of treatment. In recent years, new products that significantly improve the treatment management options for patients with hemophilia have become available in the market. OBJECTIVE: To critically evaluate the pivotal studies of recombinant FVIII (rFVIII) products, approved in Europe within the first half of 2018 having obtained the indication for a prophylaxis dosing regimen based also on a twice weekly infusion frequency or even less, highlighting their limitations or strengths. METHODS: A systematic literature search was conducted, and several databases (PubMed and Embase) were consulted. RESULTS: Nine clinical trials involving patients with severe hemophilia A without inhibitor were included in this analysis. Four rFVIII products (Elocta ® , Biogen, Cambridge, MA, USA; Kovaltry ® , Bayer HealthCare Pharmaceuticals, Germany; Afstyla ® , CSL Behring GmbH, Germany; Adynovi ® , Baxalta Innovation GmbH, Austria) with different pharmacokinetic profiles were evaluated. The trials included in this analysis had different designs and heterogeneous methods were utilized to assess the study outcomes. The baseline characteristics of the patients enrolled in the studies were also often different and sometimes not adequately described. LEOPOLD II, a trial to compare prophylaxis to on-demand therapy with an unmodified rFVIII product (Kovaltry ® , octocog alfa), was the only completely randomized trial that enrolled a more critical patient population in terms of compromised joint condition than the other studies. Based on these side-by-side comparison, Octocog alfa reported similar efficacy, in terms of annualized bleeding rate, to the other rFVIII products, including extended half-life. CONCLUSIONS: Even without structural modifications, octocog alfa may be considered a useful treatment option for two times a week prophylaxis in a selected population of haemophilia patients.
{"title":"Critical Review of the Pivotal Studies of Four rFVIII Products for the Treatment of Hemophilia A Patients: The Role of Octocog Alfa","authors":"M. N. D. Minno, Lucia S D'Angiolella, P. Cortesi, A. Molinari, L. Mantovani","doi":"10.7175/fe.v21i1.1453","DOIUrl":"https://doi.org/10.7175/fe.v21i1.1453","url":null,"abstract":"INTRODUCTION: Hemophilia A is a rare congenital bleeding disorder caused by a deficiency of clotting factor VIII (FVIII). The severe form of the disease is characterized by spontaneous bleeds, especially into the joints. Prophylaxis, based on regularly intravenous administration of the missing factor to avoid hemorrhages, represents the gold standard of treatment. In recent years, new products that significantly improve the treatment management options for patients with hemophilia have become available in the market. OBJECTIVE: To critically evaluate the pivotal studies of recombinant FVIII (rFVIII) products, approved in Europe within the first half of 2018 having obtained the indication for a prophylaxis dosing regimen based also on a twice weekly infusion frequency or even less, highlighting their limitations or strengths. METHODS: A systematic literature search was conducted, and several databases (PubMed and Embase) were consulted. RESULTS: Nine clinical trials involving patients with severe hemophilia A without inhibitor were included in this analysis. Four rFVIII products (Elocta ® , Biogen, Cambridge, MA, USA; Kovaltry ® , Bayer HealthCare Pharmaceuticals, Germany; Afstyla ® , CSL Behring GmbH, Germany; Adynovi ® , Baxalta Innovation GmbH, Austria) with different pharmacokinetic profiles were evaluated. The trials included in this analysis had different designs and heterogeneous methods were utilized to assess the study outcomes. The baseline characteristics of the patients enrolled in the studies were also often different and sometimes not adequately described. LEOPOLD II, a trial to compare prophylaxis to on-demand therapy with an unmodified rFVIII product (Kovaltry ® , octocog alfa), was the only completely randomized trial that enrolled a more critical patient population in terms of compromised joint condition than the other studies. Based on these side-by-side comparison, Octocog alfa reported similar efficacy, in terms of annualized bleeding rate, to the other rFVIII products, including extended half-life. CONCLUSIONS: Even without structural modifications, octocog alfa may be considered a useful treatment option for two times a week prophylaxis in a selected population of haemophilia patients.","PeriodicalId":41585,"journal":{"name":"Farmeconomia-Health Economics and Therapeutic Pathways","volume":"94 1","pages":""},"PeriodicalIF":0.5,"publicationDate":"2020-04-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"82396170","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}
There was an error in the Figure 5 at page 11 in this Supplement by Casolo et al. [Farmeconomia. Health economics and therapeutic pathways 2019; 20(Suppl 1): 3-16; https://doi.org/10.7175/fe.v20i1S.1454]. The online version has been corrected on April 21, 2020.
{"title":"Corrigendum: An Integrated Management Model of Patients With Atrial Fibrillation: The Experience of the Local Health Unit Tuscany North-West","authors":"[No authors listed].","doi":"10.7175/fe.v21i1.1475","DOIUrl":"https://doi.org/10.7175/fe.v21i1.1475","url":null,"abstract":"There was an error in the Figure 5 at page 11 in this Supplement by Casolo et al. [Farmeconomia. Health economics and therapeutic pathways 2019; 20(Suppl 1): 3-16; https://doi.org/10.7175/fe.v20i1S.1454]. The online version has been corrected on April 21, 2020.","PeriodicalId":41585,"journal":{"name":"Farmeconomia-Health Economics and Therapeutic Pathways","volume":"6 1","pages":""},"PeriodicalIF":0.5,"publicationDate":"2020-02-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"75326885","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. Bocchia, A. Bosi, E. Capochiani, S. Ciolli, R. Danesi, S. Galimberti, A. Gozzetti, S. Moretti, U. Occhini, M. Petrini
BACKGROUND: In the last years genomic and somatic alterations have shown to play a pivotal role in the pathogenesis of chronic lymphocytic leukemia (CLL) and new prognostic factors have been identified accordingly. AIM: To describe a real-life diagnostic and therapeutic approach to CLL that takes into account the role of genomic and somatic prognostic factors in the risk stratification of developing progressive disease, and treatment decision. METHODS: This new proposal has been developed and validated by ten key opinion leaders from Tuscany Region during two Expert Meetings. The approach suggested comes from their experience in daily clinical practice and is supported by guidelines recommendations, clinical trials results, and drugs prescribing conditions in Italy. RESULTS: Beside TP53 deletion or mutated status, the Expert Panel highlighted the importance of the IGHV mutation status characterization, since the diagnosis, in order to identify patients who will have a more aggressive progression. Furthermore, just before starting treatment, to obtain useful prognostic information and indication in the selection of the therapy, they recommend cytogenetic analysis for the detection of del(11q), trisomy 12, del(13q), del(17p), conventional karyotyping of stimulated CLL cells, TP53 sequencing, and molecular genetic analysis to detect IGHV mutation status. CONCLUSIONS: The Expert Panel recognized the limitations associated with traditional staging systems in identifying patients who will have a more aggressive disease course and predicting response to treatment and suggested a real-life diagnostic and therapeutic approach to CLL to update the current patient management in light of recent advances that have improved understanding of CLL.
{"title":"Real-life Diagnostic and Therapeutic Approach to CLL: A New Proposal from an Expert Panel in Tuscany Region","authors":"M. Bocchia, A. Bosi, E. Capochiani, S. Ciolli, R. Danesi, S. Galimberti, A. Gozzetti, S. Moretti, U. Occhini, M. Petrini","doi":"10.7175/fe.v21i1.1452","DOIUrl":"https://doi.org/10.7175/fe.v21i1.1452","url":null,"abstract":"BACKGROUND: In the last years genomic and somatic alterations have shown to play a pivotal role in the pathogenesis of chronic lymphocytic leukemia (CLL) and new prognostic factors have been identified accordingly. AIM: To describe a real-life diagnostic and therapeutic approach to CLL that takes into account the role of genomic and somatic prognostic factors in the risk stratification of developing progressive disease, and treatment decision. METHODS: This new proposal has been developed and validated by ten key opinion leaders from Tuscany Region during two Expert Meetings. The approach suggested comes from their experience in daily clinical practice and is supported by guidelines recommendations, clinical trials results, and drugs prescribing conditions in Italy. RESULTS: Beside TP53 deletion or mutated status, the Expert Panel highlighted the importance of the IGHV mutation status characterization, since the diagnosis, in order to identify patients who will have a more aggressive progression. Furthermore, just before starting treatment, to obtain useful prognostic information and indication in the selection of the therapy, they recommend cytogenetic analysis for the detection of del(11q), trisomy 12, del(13q), del(17p), conventional karyotyping of stimulated CLL cells, TP53 sequencing, and molecular genetic analysis to detect IGHV mutation status. CONCLUSIONS: The Expert Panel recognized the limitations associated with traditional staging systems in identifying patients who will have a more aggressive disease course and predicting response to treatment and suggested a real-life diagnostic and therapeutic approach to CLL to update the current patient management in light of recent advances that have improved understanding of CLL.","PeriodicalId":41585,"journal":{"name":"Farmeconomia-Health Economics and Therapeutic Pathways","volume":"45 1","pages":"11-18"},"PeriodicalIF":0.5,"publicationDate":"2020-02-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"81882075","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}
G. Gancitano, R. Ravasio, L. Cattelino, Paolo Di Procolo, D. Cortinovis
BACKGROUND: A histopathological and mutational diagnosis has become a priority in the correct choice of the most appropriate cancer therapy for NSCLC. In the absence of a molecular analysis, the therapeutic choice will be directed towards platinum-based chemotherapy, thus preventing, in the presence of a specific mutation, the benefits deriving from the administration of a target therapies (TT). AIM: the present analysis was carried out with the aim of estimating the clinical impact, expressed in terms of progression free survival (PFS), associated with the use of the combined strategy (tissue biopsy and liquid biopsy) or the tissue strategy in the EGFR+ mNSCLC population. METHODS: A pre-existing cost-consequence model was adapted to estimate the annual number of mNSCLC patients with or without the EGFR mutation in order to decide the oncological treatment to be administered in first (1L) or second line (2L). In 1L, against the presence of the EGFR mutation, the administration of a Tyrosine Kinase Inhibitor (TKI), such as osimertinib, gefitinib, erlotinib or afatinib, was considered; in the absence of the EGFR mutation, the administration of standard platinum-based chemotherapy was instead considered. With reference to 2L, in the presence of the EGFR T790M mutation, only osimertinib was considered. In the absence of the EGFR T790M mutation, the administration of the standard platinum-based chemotherapy was also considered. The PFS data associated with each of the drugs considered were extrapolated from the respective clinical studies. Key variables were tested in the sensitivity analysis. RESULTS: The adoption of the combined strategy (tissue biopsy and liquid biopsy), by virtue of a greater number of patients treated with TKIs, would make it possible to increase the average PFS in the range of 1.1-3,7 months in the 1L and by 1.4 months in the 2L. CONCLUSION: These results show how the adoption of a correct diagnostic strategy is critical in order to optimize the choice of the therapeutic path in the 1L and 2L of mNSCLC. The addition of the liquid biopsy to the classic diagnostic path (tissue biopsy) would in fact allow to obtain an increase in therapeutic efficacy (average PFS).
{"title":"Clinical Impact of two Different Diagnostic Strategies in the First- and Second-Line Treatment of Locally Advanced or Metastatic EGFR-Mutated Non-Small Cell Lung Cancer","authors":"G. Gancitano, R. Ravasio, L. Cattelino, Paolo Di Procolo, D. Cortinovis","doi":"10.7175/fe.v21i1.1450","DOIUrl":"https://doi.org/10.7175/fe.v21i1.1450","url":null,"abstract":"BACKGROUND: A histopathological and mutational diagnosis has become a priority in the correct choice of the most appropriate cancer therapy for NSCLC. In the absence of a molecular analysis, the therapeutic choice will be directed towards platinum-based chemotherapy, thus preventing, in the presence of a specific mutation, the benefits deriving from the administration of a target therapies (TT). AIM: the present analysis was carried out with the aim of estimating the clinical impact, expressed in terms of progression free survival (PFS), associated with the use of the combined strategy (tissue biopsy and liquid biopsy) or the tissue strategy in the EGFR+ mNSCLC population. METHODS: A pre-existing cost-consequence model was adapted to estimate the annual number of mNSCLC patients with or without the EGFR mutation in order to decide the oncological treatment to be administered in first (1L) or second line (2L). In 1L, against the presence of the EGFR mutation, the administration of a Tyrosine Kinase Inhibitor (TKI), such as osimertinib, gefitinib, erlotinib or afatinib, was considered; in the absence of the EGFR mutation, the administration of standard platinum-based chemotherapy was instead considered. With reference to 2L, in the presence of the EGFR T790M mutation, only osimertinib was considered. In the absence of the EGFR T790M mutation, the administration of the standard platinum-based chemotherapy was also considered. The PFS data associated with each of the drugs considered were extrapolated from the respective clinical studies. Key variables were tested in the sensitivity analysis. RESULTS: The adoption of the combined strategy (tissue biopsy and liquid biopsy), by virtue of a greater number of patients treated with TKIs, would make it possible to increase the average PFS in the range of 1.1-3,7 months in the 1L and by 1.4 months in the 2L. CONCLUSION: These results show how the adoption of a correct diagnostic strategy is critical in order to optimize the choice of the therapeutic path in the 1L and 2L of mNSCLC. The addition of the liquid biopsy to the classic diagnostic path (tissue biopsy) would in fact allow to obtain an increase in therapeutic efficacy (average PFS).","PeriodicalId":41585,"journal":{"name":"Farmeconomia-Health Economics and Therapeutic Pathways","volume":"47 1","pages":""},"PeriodicalIF":0.5,"publicationDate":"2020-02-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"87577552","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}
G. Casolo, G. Cavirani, Alessandro Carlo, S. Iannazzo, M. Pardini, Alessandro Squillace
Atrial fibrillation (AF) is the most prevalent form of alteration in cardiac rhythm and is associated with a high economic burden resulting from both clinical consequences and impact on patients’ quality of life. Goals of treatment include symptom control and, in the high-risk patients, the prevention of thromboembolic complications. The advent of novel oral anticoagulant agents (NOACs) has improved the management of patients with non-valvular AF (NVAF) by overcoming limitations associated with traditional oral anticoagulation drugs. NOACs are associated with a lower risk of stroke, systemic embolism, and mortality compared to vitamin K antagonists (VKAs) and with a lower risk of fatal, major, and intracranial bleeding. This supplement aims at sharing the virtuous management model of AF patients in the Local Health Unit Tuscany North-West and promoting the importance of a multidisciplinary management, which involves cardiologists and general practitioners (GPs), not only in terms of clinical outcomes, but also of therapeutic appropriateness and economic sustainability.
{"title":"An Integrated Management Model of Patients With Atrial Fibrillation: The Experience of the Local Health Unit Tuscany North-West","authors":"G. Casolo, G. Cavirani, Alessandro Carlo, S. Iannazzo, M. Pardini, Alessandro Squillace","doi":"10.7175/fe.v20i1s.1454","DOIUrl":"https://doi.org/10.7175/fe.v20i1s.1454","url":null,"abstract":"Atrial fibrillation (AF) is the most prevalent form of alteration in cardiac rhythm and is associated with a high economic burden resulting from both clinical consequences and impact on patients’ quality of life. Goals of treatment include symptom control and, in the high-risk patients, the prevention of thromboembolic complications. The advent of novel oral anticoagulant agents (NOACs) has improved the management of patients with non-valvular AF (NVAF) by overcoming limitations associated with traditional oral anticoagulation drugs. NOACs are associated with a lower risk of stroke, systemic embolism, and mortality compared to vitamin K antagonists (VKAs) and with a lower risk of fatal, major, and intracranial bleeding. This supplement aims at sharing the virtuous management model of AF patients in the Local Health Unit Tuscany North-West and promoting the importance of a multidisciplinary management, which involves cardiologists and general practitioners (GPs), not only in terms of clinical outcomes, but also of therapeutic appropriateness and economic sustainability.","PeriodicalId":41585,"journal":{"name":"Farmeconomia-Health Economics and Therapeutic Pathways","volume":"34 1","pages":""},"PeriodicalIF":0.5,"publicationDate":"2019-12-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"80936857","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}
F. Mennini, L. Lombardozzi, A. Mecozzi, P. Sciattella
BACKGROUND: In Italy there are an estimated 4 million patients suffering from diabetes mellitus (DM). The most important ocular complication of DM is diabetic retinopathy (DR), which affects about one third of diabetic patients.AIMS: To identify, within the Lazio Region, the cohort of patients starting treatment for DME in the years 2010-2016 and calculating the annual cost of therapy; and to assess the appropriateness of the drugs prescribed.METHODS: From the Health Information System of the Lazio Region were identified all subjects who, in the 2010-2016 period, had received at least one prescription for dexamethasone intravitreal implant (i.i.) or intravitreal triamcinolone or ranibizumab or aflibercept or bevacizumab. For the cohort of users selected, the appropriateness of the treatment were evaluated calculating the number of administrations performed in the first four months of the index prescription and the number of administrations performed during the 12 months of treatment.RESULTS: In 2016, 7,265 patients in the Lazio Region received at least one prescription of ranibizumab (43.0%), aflibercept (37.5%) and dexamethasone i.i. (19.5%). Among the 3,416 patients naïve at 6 months, who started treatment in the 2013-2015 period and who did not switch to different drugs, 78.7% started treatment with ranibizumab, 16.0% with dexamethasone i.i. and 5.3% with aflibercept. The mean annual cost for the treatment of a patient with DME and naïve at 6 months was equal to € 2,388; a total cost for only the naïve patients selected in the 2013-2015 period is therefore estimated at approximately € 8.2 million. The average annual cost of dexamethasone i.i. treatment was € 1,497, lower than that of ranibizumab (€ 2,562) and aflibercept (€ 2,485). The expenditure for patients receiving less than 3 administrations of ranibizumab or aflibercept in the first 10 months of treatment was estimate equal to € 1.3 million.CONCLUSIONS: The administrations of dexamethasone i.i. are in line with what is indicated in the prescribing information, while for ranibizumab and aflibercept a potential under-use has been identified. A greater appropriateness of the drugs prescribed, accompanied by an optimal adherence to therapy, would strongly reduce the current waste of resources.
{"title":"Evaluation of patients treated for diabetic retinopathy: an analysis of the administrative databases of the Lazio Region","authors":"F. Mennini, L. Lombardozzi, A. Mecozzi, P. Sciattella","doi":"10.7175/fe.v20i1.1439","DOIUrl":"https://doi.org/10.7175/fe.v20i1.1439","url":null,"abstract":"BACKGROUND: In Italy there are an estimated 4 million patients suffering from diabetes mellitus (DM). The most important ocular complication of DM is diabetic retinopathy (DR), which affects about one third of diabetic patients.AIMS: To identify, within the Lazio Region, the cohort of patients starting treatment for DME in the years 2010-2016 and calculating the annual cost of therapy; and to assess the appropriateness of the drugs prescribed.METHODS: From the Health Information System of the Lazio Region were identified all subjects who, in the 2010-2016 period, had received at least one prescription for dexamethasone intravitreal implant (i.i.) or intravitreal triamcinolone or ranibizumab or aflibercept or bevacizumab. For the cohort of users selected, the appropriateness of the treatment were evaluated calculating the number of administrations performed in the first four months of the index prescription and the number of administrations performed during the 12 months of treatment.RESULTS: In 2016, 7,265 patients in the Lazio Region received at least one prescription of ranibizumab (43.0%), aflibercept (37.5%) and dexamethasone i.i. (19.5%). Among the 3,416 patients naïve at 6 months, who started treatment in the 2013-2015 period and who did not switch to different drugs, 78.7% started treatment with ranibizumab, 16.0% with dexamethasone i.i. and 5.3% with aflibercept. The mean annual cost for the treatment of a patient with DME and naïve at 6 months was equal to € 2,388; a total cost for only the naïve patients selected in the 2013-2015 period is therefore estimated at approximately € 8.2 million. The average annual cost of dexamethasone i.i. treatment was € 1,497, lower than that of ranibizumab (€ 2,562) and aflibercept (€ 2,485). The expenditure for patients receiving less than 3 administrations of ranibizumab or aflibercept in the first 10 months of treatment was estimate equal to € 1.3 million.CONCLUSIONS: The administrations of dexamethasone i.i. are in line with what is indicated in the prescribing information, while for ranibizumab and aflibercept a potential under-use has been identified. A greater appropriateness of the drugs prescribed, accompanied by an optimal adherence to therapy, would strongly reduce the current waste of resources.","PeriodicalId":41585,"journal":{"name":"Farmeconomia-Health Economics and Therapeutic Pathways","volume":"1 1","pages":""},"PeriodicalIF":0.5,"publicationDate":"2019-11-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"73450943","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}
Azadeh Ahmadi Dashtiyan, I. Asl, L. Riahi, M. Mahmoodi
BACKGROUND: The faster and more accurately Pre-Hospital Emergency Medicine as the first line of care and treatment is done, the less the mortality and disability rates are.AIM: The present study is an attempt to design a model for the establishment of pre-hospital emergency bases in the northern provinces of Iran.METHODS: This quantitative-qualitative (mixed-method) research was conducted in two parts: First, using the literature review, interviews with experts in the field, and a comparative study, the most important factors affecting the establishment were extracted. Second, the obtained data were employed to formulate the development model and to design the questionnaire. The required data for factor analysis were collected through a questionnaire distributed among 200 operational personnel in January 2018. The results were analyzed using confirmatory factor analysis and multiple regression.RESULTS: Five components were identified after the exploratory factor analysis and Varimax with an eigenvalue larger than 1. The effect coefficients calculated for human resources components, service speed, and information and communication system were 0.935, 0.765 and 0.752, respectively. The obtained goodness of fit was very close to one, indicating the one-dimensional strength of the model. The highest parameter estimation in this model was allocated to the human component as 0.935, which has a significant correlation with other components.CONCLUSIONS: In order to reduce the response time, more attention should be paid to the allocation of budget and organizational roles, education, participation from government departments, establishment of an independent medical emergency organization, and appropriate accessibility to reduce the rates of mortality and morbidity.
{"title":"Developing a Model for the Establishment of Pre-Hospital Emergency Medicine Bases in the Northern Provinces of Iran","authors":"Azadeh Ahmadi Dashtiyan, I. Asl, L. Riahi, M. Mahmoodi","doi":"10.7175/fe.v20i1.1381","DOIUrl":"https://doi.org/10.7175/fe.v20i1.1381","url":null,"abstract":"BACKGROUND: The faster and more accurately Pre-Hospital Emergency Medicine as the first line of care and treatment is done, the less the mortality and disability rates are.AIM: The present study is an attempt to design a model for the establishment of pre-hospital emergency bases in the northern provinces of Iran.METHODS: This quantitative-qualitative (mixed-method) research was conducted in two parts: First, using the literature review, interviews with experts in the field, and a comparative study, the most important factors affecting the establishment were extracted. Second, the obtained data were employed to formulate the development model and to design the questionnaire. The required data for factor analysis were collected through a questionnaire distributed among 200 operational personnel in January 2018. The results were analyzed using confirmatory factor analysis and multiple regression.RESULTS: Five components were identified after the exploratory factor analysis and Varimax with an eigenvalue larger than 1. The effect coefficients calculated for human resources components, service speed, and information and communication system were 0.935, 0.765 and 0.752, respectively. The obtained goodness of fit was very close to one, indicating the one-dimensional strength of the model. The highest parameter estimation in this model was allocated to the human component as 0.935, which has a significant correlation with other components.CONCLUSIONS: In order to reduce the response time, more attention should be paid to the allocation of budget and organizational roles, education, participation from government departments, establishment of an independent medical emergency organization, and appropriate accessibility to reduce the rates of mortality and morbidity.","PeriodicalId":41585,"journal":{"name":"Farmeconomia-Health Economics and Therapeutic Pathways","volume":"1 1","pages":""},"PeriodicalIF":0.5,"publicationDate":"2019-10-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"86492575","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}
L. Mantovani, G. Furneri, R. Bitonti, P. Cortesi, E. Puma, L. Santoni, L. Prosperini
BACKGROUND: Delayed-release dimethyl fumarate (also known as gastro-resistant dimethyl fumarate, hereafter dimethyl fumarate) is an oral disease-modifying therapy used for the treatment of Relapsing-Remitting Multiple Sclerosis (RRMS), an autoimmune chronic inflammatory condition of the central nervous system.OBJECTIVE: The objective of this economic analysis was to compare cost-effectiveness of dimethyl fumarate with the alternatives used as first-line treatment of RRMS in Italy.METHODS: The analysis was conducted from the Italian societal perspective. Health outcomes and costs were evaluated over a 50-year time horizon (equivalent to a lifetime horizon). Both health outcomes and costs were discounted at 3.5%. The cost-effectiveness analysis was conducted by adapting a Markov model, already used in previous similar economic analyses conducted in RRMS, to the Italian context. The Markov model estimated the clinical and economic consequences of treating RRMS patients with the following therapeutic options: dimethyl fumarate; interferon (IFN) beta-1a subcutaneous (SC) at two different doses, 22 mcg and 44 mcg; IFN beta-1b SC; glatiramer acetate (GA) SC 20 mg; oral teriflunomide. Clinical efficacy data were retrieved from an elaboration of an already published mixed treatment comparison (MTC). Both direct and indirect costs (disability, treatment acquisition, administration, monitoring, relapses, adverse events) were included in the analysis. One-way and probabilistic sensitivity analyses were carried out and cost-effectiveness acceptability curves generated.RESULTS: In the base-case analysis, dimethyl fumarate was more efficacious than alternatives, in terms of both survival (19.634 vs. 19.440-19.600 life years for alternatives), and quality-of-life-adjusted survival (6.526 vs. 5.143- 6.189 QALYs for alternatives). The total lifetime cost per patient treated with dimethyl fumarate (€ 954,286) was lower than that of the other DMTs included in the analysis. Therefore, dimethyl fumarate was dominant compared with all analyzed alternatives. Dimethyl fumarate was also the therapeutic option with the highest benefit on disease burden. In fact, costs of disability management were lower than those of all the other first-line drugs included in the analysis. The results of one-way deterministic sensitivity analysis and probabilistic sensitivity analysis confirmed the reliability of base-case results.CONCLUSIONS: The results of the cost-effectiveness analysis confirm that dimethyl fumarate is an optimal first-line treatment for RRMS in Italy, compared with the other first-line alternatives included in the economic analysis, when evaluated from the societal perspective.
背景:延迟释放富马酸二甲酯(也称为胃抵抗性富马酸二甲酯，以下简称富马酸二甲酯)是一种用于治疗复发-缓解型多发性硬化症(RRMS)的口服疾病改善疗法，RRMS是一种中枢神经系统自身免疫性慢性炎症。目的:本经济分析的目的是比较富马酸二甲酯与意大利用于RRMS一线治疗的替代品的成本-效果。方法:从意大利社会角度进行分析。在50年的时间范围内(相当于一生的时间范围)评估了健康结果和成本。健康结果和成本均以3.5%折现。成本效益分析是通过调整马尔科夫模型进行的，该模型已经在RRMS中进行的类似经济分析中使用，并适用于意大利的情况。马尔可夫模型估计了用以下治疗方案治疗RRMS患者的临床和经济后果:富马酸二甲酯;干扰素(IFN) β -1a皮下(SC)两种不同剂量，22微克和44微克;IFN β -1b SC;醋酸格拉替默(GA) SC 20 mg;口服teriflunomide。临床疗效数据是从已经发表的混合治疗比较(MTC)的详细阐述中检索的。直接和间接费用(致残、获得治疗、给药、监测、复发、不良事件)均包括在分析中。进行了单向和概率敏感性分析，并生成了成本-效果可接受性曲线。结果:在基本病例分析中，富马酸二甲酯在生存期(19.634 vs. 19.440-19.600生命年)和生活质量调整生存期(6.526 vs. 5.143- 6.189 QALYs)方面都比替代品更有效。用富马酸二甲酯治疗的每位患者的总生命周期成本(954,286欧元)低于分析中包括的其他dmt。因此，与所有被分析的替代品相比，富马酸二甲酯具有优势。富马酸二甲酯也是减轻疾病负担效益最高的治疗选择。事实上，残疾管理的费用低于分析中包括的所有其他一线药物的费用。单向确定性灵敏度分析和概率灵敏度分析的结果证实了基本情况结果的可靠性。结论:从社会角度进行评价时，成本-效果分析结果证实，与经济分析中包括的其他一线替代方案相比，富马酸二甲酯是意大利RRMS的最佳一线治疗方案。
{"title":"Cost-Effectiveness Analysis of Dimethyl Fumarate in the Treatment of Relapsing Remitting Multiple Sclerosis: An Italian Societal Perspective","authors":"L. Mantovani, G. Furneri, R. Bitonti, P. Cortesi, E. Puma, L. Santoni, L. Prosperini","doi":"10.7175/FE.V20I1.1437","DOIUrl":"https://doi.org/10.7175/FE.V20I1.1437","url":null,"abstract":"BACKGROUND: Delayed-release dimethyl fumarate (also known as gastro-resistant dimethyl fumarate, hereafter dimethyl fumarate) is an oral disease-modifying therapy used for the treatment of Relapsing-Remitting Multiple Sclerosis (RRMS), an autoimmune chronic inflammatory condition of the central nervous system.OBJECTIVE: The objective of this economic analysis was to compare cost-effectiveness of dimethyl fumarate with the alternatives used as first-line treatment of RRMS in Italy.METHODS: The analysis was conducted from the Italian societal perspective. Health outcomes and costs were evaluated over a 50-year time horizon (equivalent to a lifetime horizon). Both health outcomes and costs were discounted at 3.5%. The cost-effectiveness analysis was conducted by adapting a Markov model, already used in previous similar economic analyses conducted in RRMS, to the Italian context. The Markov model estimated the clinical and economic consequences of treating RRMS patients with the following therapeutic options: dimethyl fumarate; interferon (IFN) beta-1a subcutaneous (SC) at two different doses, 22 mcg and 44 mcg; IFN beta-1b SC; glatiramer acetate (GA) SC 20 mg; oral teriflunomide. Clinical efficacy data were retrieved from an elaboration of an already published mixed treatment comparison (MTC). Both direct and indirect costs (disability, treatment acquisition, administration, monitoring, relapses, adverse events) were included in the analysis. One-way and probabilistic sensitivity analyses were carried out and cost-effectiveness acceptability curves generated.RESULTS: In the base-case analysis, dimethyl fumarate was more efficacious than alternatives, in terms of both survival (19.634 vs. 19.440-19.600 life years for alternatives), and quality-of-life-adjusted survival (6.526 vs. 5.143- 6.189 QALYs for alternatives). The total lifetime cost per patient treated with dimethyl fumarate (€ 954,286) was lower than that of the other DMTs included in the analysis. Therefore, dimethyl fumarate was dominant compared with all analyzed alternatives. Dimethyl fumarate was also the therapeutic option with the highest benefit on disease burden. In fact, costs of disability management were lower than those of all the other first-line drugs included in the analysis. The results of one-way deterministic sensitivity analysis and probabilistic sensitivity analysis confirmed the reliability of base-case results.CONCLUSIONS: The results of the cost-effectiveness analysis confirm that dimethyl fumarate is an optimal first-line treatment for RRMS in Italy, compared with the other first-line alternatives included in the economic analysis, when evaluated from the societal perspective.","PeriodicalId":41585,"journal":{"name":"Farmeconomia-Health Economics and Therapeutic Pathways","volume":"52 1","pages":""},"PeriodicalIF":0.5,"publicationDate":"2019-07-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"90550066","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}
P. Cortesi, D. Paolicelli, M. Capobianco, P. Cozzolino, L. Mantovani
INTRODUCTION: The availability of ocrelizumab for the relapsing forms of multiple sclerosis (MS) in the Italian markets raised some questions about its economic impact and value compared to the alternative treatment options available.AIM: To assess the cost-effectiveness and budget impact of ocrelizumab compared to the most used second line disease modifying therapies (DMTs) in Italy.METHODS: The study was divided in two phases: Phase 1 – based on the development of a decision analytical Markov model to assess the cost-effectiveness of ocrelizumab compared to natalizumab and fingolimod, and Phase 2 – based on the development of a budget impact model to assess the economic impact of ocrelizumab in Italy. Both models used the National Health System perspective; a lifetime horizon was applied in the cost-effectiveness analysis and a 3-year time horizon in the budget impact. The cost-effectiveness analysis results were reported as incremental cost-effectiveness ratio (ICER) expressed as € per Quality Adjusted Life Year (QALY) gained, the budget impact analysis results were reported as difference in the overall budget (€) between a scenario with and without ocrelizumab.RESULTS: The two analyses reported ocrelizumab as a cost-effective option compared to natalizumab and fingolimod with a positive impact on the overall NHS budget. In the base-case analysis, the ICER was € 2,023 for ocrelizumab compared to fingolimod; while ocrelizumab resulted cost-saving compared to natalizumab. The sensitivity analysis confirmed the base-case analysis results. Further, the use of ocrelizumab was associated to a budget decrease of € 21 million (-2.6%) in a 3-year time horizon.CONCLUSION: The results of our cost-effectiveness and budget impact models reported ocrelizumab as an effective and efficient treatment in patients with relapsing forms of MS who failed a first line DMTs from the Italian NHS perspective.
{"title":"The Value and Sustainability of Ocrelizumab in Relapsing Multiple Sclerosis: A Cost-Effectiveness and Budget Impact Analysis","authors":"P. Cortesi, D. Paolicelli, M. Capobianco, P. Cozzolino, L. Mantovani","doi":"10.7175/FE.V20I1.1435","DOIUrl":"https://doi.org/10.7175/FE.V20I1.1435","url":null,"abstract":"INTRODUCTION: The availability of ocrelizumab for the relapsing forms of multiple sclerosis (MS) in the Italian markets raised some questions about its economic impact and value compared to the alternative treatment options available.AIM: To assess the cost-effectiveness and budget impact of ocrelizumab compared to the most used second line disease modifying therapies (DMTs) in Italy.METHODS: The study was divided in two phases: Phase 1 – based on the development of a decision analytical Markov model to assess the cost-effectiveness of ocrelizumab compared to natalizumab and fingolimod, and Phase 2 – based on the development of a budget impact model to assess the economic impact of ocrelizumab in Italy. Both models used the National Health System perspective; a lifetime horizon was applied in the cost-effectiveness analysis and a 3-year time horizon in the budget impact. The cost-effectiveness analysis results were reported as incremental cost-effectiveness ratio (ICER) expressed as € per Quality Adjusted Life Year (QALY) gained, the budget impact analysis results were reported as difference in the overall budget (€) between a scenario with and without ocrelizumab.RESULTS: The two analyses reported ocrelizumab as a cost-effective option compared to natalizumab and fingolimod with a positive impact on the overall NHS budget. In the base-case analysis, the ICER was € 2,023 for ocrelizumab compared to fingolimod; while ocrelizumab resulted cost-saving compared to natalizumab. The sensitivity analysis confirmed the base-case analysis results. Further, the use of ocrelizumab was associated to a budget decrease of € 21 million (-2.6%) in a 3-year time horizon.CONCLUSION: The results of our cost-effectiveness and budget impact models reported ocrelizumab as an effective and efficient treatment in patients with relapsing forms of MS who failed a first line DMTs from the Italian NHS perspective.","PeriodicalId":41585,"journal":{"name":"Farmeconomia-Health Economics and Therapeutic Pathways","volume":"63 1","pages":""},"PeriodicalIF":0.5,"publicationDate":"2019-07-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"80614995","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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