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[Cost-Effectiveness Analysis of Maribavir in Patients with Post-Transplant Cytomegalovirus Infection or Disease that are Refractory or Resistant to Conventional Therapy]
Full text linkOBJECTIVES: Cytomegalovirus (CMV) infection represents a serious post-transplantation complication, particularly among transplant recipients with resistant or refractory (R/R) infection. Despite the improvement made in CMV prevention and treatment, remains a need for new therapies that are better tolerated and safer for patients, especially for refractory or resistant (R/R) infections. This analysis investigates the cost-effectiveness of maribavir versus investigator-assigned therapy (IAT) for treatment of post-transplant R/R CMV in the Italian context.METHODS: The analysis was conducted from the National Health Service (NHS) perspective using a Markov model previously developed in Excel® to evaluate the cost-effectiveness of maribavir versus IAT for the treatment of post-transplant R/R CMV. Health event rates and transition probabilities were informed by data from observational studies and from SOLSTICE clinical trial, which compared maribavir to conventional antiviral treatments for transplant patients with R/R CMV infections. Utilities were from SOLSTICE and a utility vignette study. Costs inputs were derived from published literature and Italian national tariffs for outpatient and hospital services. Costs and outcomes were discounted by 3%.RESULTS: Maribavir compared to the IAT used for the treatment of post-transplant R/R CMV achieved an incremental cost of € 11,455 for an incremental quality adjusted life year (QALY) gain of 0.313. This resulted in an incremental cost-effectiveness ratio of € 36,626/QALY, for maribavir compared to IAT, which is below the generally accepted, national willingness-to-pay threshold for orphan drugs (€ 37,000/QALY).CONCLUSIONS: This analysis showed that maribavir, an orphan drug treatment, is cost-effective compared to the current antiviral treatments used for treatment of post-transplant R/R CMV
HTA dei dispositivi di controllo urinario artificiale per il trattamento dell’incontinenza urinaria maschile grave post-prostatectomia
Full text linkL’incontinenza urinaria (IU) è un sintomo frequente a seguito di prostatectomia radicale per carcinoma della prostata, dal momento che interessa quote elevate di pazienti, che variano a seconda della definizione, delle tempistiche di valutazione, dell’approccio chirurgico e di chi effettua la rilevazione e che possono anche superare l’80% dei pazienti [1]. Nella maggior parte dei casi, il problema si risolve a distanza di un anno. Tuttavia, l’incontinenza permane anche dopo 12 mesi in una quota non trascurabile di pazienti, che varia a seconda delle statistiche e che si attesta intorno a percentuali pari al 10% [1]. L’IU, oltre a rappresentare un problema di tipo igienico e sociale, compromette fortemente la qualità della vita del soggetto che, dopo aver superato il trauma e la paura della patologia oncologica, vive sentimenti di perdita di autostima, associati ad alterazioni nelle relazioni familiari, sociali e nel rapporto col partner, che interessano anche la vita lavorativa [2,3]. Il tavolo tecnico sull’incontinenza – istituito presso il Ministero della Salute – ha definito un percorso basato su evidenze cliniche, che risponde alla necessità di prendersi cura del paziente con IU dopo chirurgia prostatica [4]. Tra le diverse criticità rilevate sul territorio nazionale, emerge il mancato accesso alla terapia chirurgica dell’IU, che non viene proposta perché non effettuata nel centro di cura oncologica, per la limitata proposta di soluzioni terapeutiche offerte al paziente dal chirurgo e per la limitata disponibilità di presidi chirurgici, anche in relazione ai costi. Nel caso dell’IU da sforzo da incompetenza sfinterica dopo prostatectomia radicale, l’opzione dello sfintere urinario artificiale (AUS) rimane il gold standard per l’IU moderata-grave (raccomandazione B ICI, livello di evidenza 2b EAU). Tale giudizio è confermato anche dalla Regione Veneto che, con il Decreto del Direttore generale dell’Area Sanità e Sociale n. 58 del 15.06.2016, ha emanato le “Linee di indirizzo regionali per l’utilizzo di sfinteri urinari artificiali, sling e neurostimolatori sacrali nell’incontinenza urinaria e fecale”, con cui si stabilisce che «Lo sfintere urinario artificiale deve essere offerto agli uomini con incontinenza da sforzo (SUI) post-prostatectomia da moderata a severa, a seguito di fallimento della terapia conservativa» [5]. Il paradosso dell’impianto di AUS è che, pur essendo una soluzione terapeutica eccellente e costo-efficace per i pazienti con incontinenza urinaria da moderata a grave, la maggioranza dei candidati non ne ha accesso. Come evidenziato dalla analisi delle Schede di Dimissione Ospedaliera (SDO) 2016 [6], in Italia vengono effettuati non più di 240 impianti all’anno a fronte di una stima media di circa 1.000 pazienti/anno che vivono con incontinenza non risolta con terapia conservativa. Tali numeri dimostrano un evidente sottoutilizzo della tecnologia di elezione per questi pazienti. Le barriere all’adozione della soluzione chirurgica sono diverse; tra di esse si annoverano la scarsità di informazione adeguata ai pazienti e la mancata allocazione di risorse – economiche e professionali – adeguate a garantire la procedura: spesso gli ospedali hanno pochissime unità/professionalità disponibili, con lunghe liste d’attesa, e un rimborso inadeguato a coprire i costi della procedura da parte del Servizio Sanitario Regionale/Nazionale (SSR/SSN) [7]. Questi fattori disincentivano il ricorso all’impianto dell’AUS anche laddove clinicamente appropriato, determinando un trattamento insufficiente e inadeguato della popolazione eleggibile. Alla luce dell’analisi HTA condotta, sarebbe auspicabile un intervento normativo che riconosca dignità e valore all’impianto dell’AUS post-prostatectomia radicale, laddove la terapia farmacologica si rivelasse insufficiente alla soluzione del problema. L’analisi ha dimostrato come incrementando il numero di soggetti trattati con AUS (+29 pazienti trattati con il gold standard AMS 800TM [8]) rispetto alla terapia conservativa si verifichi un importante incremento della qualità di vita dei pazienti (rapporto di costo-efficacia altamente positivo e in alcuni casi dominante) accompagnato da una consistente riduzione del costo legato alla terapia conservativa (pannoloni). Si tratterebbe di un numero limitato di procedure, che consentirebbe, però, di migliorare notevolmente la qualità della vita dei pazienti interessati da questa condizione
[Measurement-Based Procurement Approach for Biosimilars in Italy: A Position Paper]
Full text linkThe full potential of biosimilars has not been achieved yet, and this document has been written with the aim of methodizing an effective and measurable procurement system that ensures wider, simplified, and economically sustainable access of biosimilars to patients. The economic and financial sustainability generated by the increased use of biosimilars for the benefit of patients and society has been estimated and confirmed in several observational studies and probabilistic projections conducted in Europe. Despite differences in procurement policies, significant reductions in spending for some biosimilars have been recorded in several European countries between 2015 and 2018 compared with the period preceding the patent’s expiry. An accurate evaluation of the economic outcomes resulting from an extended use of biosimilars has also been conducted in Italy, with the settled purpose of calculating the combined effects that various biosimilars with patent expired have generated and will generate in terms of overall spending reduction. Even with the limitations typically associated with probabilistic models, it is also evident that an expanded use of biosimilars is an effective tool to govern the expenditure and channel resources to support the intrinsic balance of the health system (i.e., sustainability) or to ensure the introduction of innovative drugs or diagnostic procedures (i.e., accessibility). The economic sustainability of the health system must also be achieved through the maintenance of a competitive procurement system. This requires a serious reconsideration of the purchasing criteria. The principle of competitiveness can contribute to the governance, containment, reduction or streamlining of public expenditure on biosimilars, but it is appropriate to establish which factors can feed and support a lawful competition, actually. Competition merely based on price reduction between biosimilar and biosimilar, excluding, undervaluing, or taking for granted that between originator and biosimilar, leads to a price erosion and this condition could have unpredictable and undesirable consequences. The market must have its own balance to be of interest to manufacturing companies and ensure the competitiveness. In the field of biosimilars, as in other areas, the need to move from purchasing criteria based on cost exclusively to those based on overall economic benefit by combining price with quality is generally shared. This approach allows for a balanced competition on multiple parameters and above all enables the best service to be provided to patients and the National Health Service. Hence, what are the quality criteria to be considered in relation to price? The areas relevant to the procurement code regulations, the determination of needs, the technical-market knowledge, the encoding of quality criteria, and their specific parameterization to the contingent dynamics of the market must certainly be identified and examined: this is what the present document aims to do. The intention is to provide a guide that can facilitate the implementation of tenders awardable by evaluating the economically most advantageous offer and that will enable us to measure the effect of a “new” procurement system, comparing the differences between what is recorded now and what is detected in the phase following the introduction of quality-price ratio-based tenders for biosimilars in Italy. In conclusion, through the joint work of specialists and experts in the sector, this Position Paper identifies the quality criteria for evaluating biosimilars and proposes a technical specification based on identified criteria, so as to provide central purchasing bodies and institutional payers with a strategy for the actual implementation both at regional and national level of a procurement method focusing on the quality-price ratio.The full potential of biosimilars has not been achieved yet, and this document has been written with the aim of methodizing an effective and measurable procurement system that ensures wider, simplified, and economically sustainable access of biosimilars to patients. The economic and financial sustainability generated by the increased use of biosimilars for the benefit of patients and society has been estimated and confirmed in several observational studies and probabilistic projections conducted in Europe. Despite differences in procurement policies, significant reductions in spending for some biosimilars have been recorded in several European countries between 2015 and 2018 compared with the period preceding the patent’s expiry. An accurate evaluation of the economic outcomes resulting from an extended use of biosimilars has also been conducted in Italy, with the settled purpose of calculating the combined effects that various biosimilars with patent expired have generated and will generate in terms of overall spending reduction. Even with the limitations typically associated with probabilistic models, it is also evident that an expanded use of biosimilars is an effective tool to govern the expenditure and channel resources to support the intrinsic balance of the health system (i.e., sustainability) or to ensure the introduction of innovative drugs or diagnostic procedures (i.e., accessibility). The economic sustainability of the health system must also be achieved through the maintenance of a competitive procurement system. This requires a serious reconsideration of the purchasing criteria. The principle of competitiveness can contribute to the governance, containment, reduction or streamlining of public expenditure on biosimilars, but it is appropriate to establish which factors can feed and support a lawful competition, actually. Competition merely based on price reduction between biosimilar and biosimilar, excluding, undervaluing, or taking for granted that between originator and biosimilar, leads to a price erosion and this condition could have unpredictable and undesirable consequences. The market must have its own balance to be of interest to manufacturing companies and ensure the competitiveness. In the field of biosimilars, as in other areas, the need to move from purchasing criteria based on cost exclusively to those based on overall economic benefit by combining price with quality is generally shared. This approach allows for a balanced competition on multiple parameters and above all enables the best service to be provided to patients and the National Health Service. Hence, what are the quality criteria to be considered in relation to price? The areas relevant to the procurement code regulations, the determination of needs, the technical-market knowledge, the encoding of quality criteria, and their specific parameterization to the contingent dynamics of the market must certainly be identified and examined: this is what the present document aims to do. The intention is to provide a guide that can facilitate the implementation of tenders awardable by evaluating the economically most advantageous offer and that will enable us to measure the effect of a “new” procurement system, comparing the differences between what is recorded now and what is detected in the phase following the introduction of quality-price ratio-based tenders for biosimilars in Italy. In conclusion, through the joint work of specialists and experts in the sector, this Position Paper identifies the quality criteria for evaluating biosimilars and proposes a technical specification based on identified criteria, so as to provide central purchasing bodies and institutional payers with a strategy for the actual implementation both at regional and national level of a procurement method focusing on the quality-price ratio
[Fever of Unknown Origin in a Patient Administered With Bacillus Calmette-Guérin (BCG) to Treat Urothelial Vesical Carcinoma in Situ]
Full text linkKoch’s Bacillus bovis (Mycobacterium bovis) was made avirulent by special culture conditions, i.e., 230 passages on medium containing potatoes treated with bile salts. It has been used mainly to prevent tuberculosis. The vaccine was named after the bacteriologist Albert Calmette and the veterinarian Camille Guérin, i.e., Bacillus Calmette-Guérin (BCG). These researchers worked at the Pasteur Institute in Lille and launched the vaccine in 1921.Recently, BCG was used also for its non-specific immunostimulant action. Intravesical administration of BCG is an adjunctive therapy for the treatment of bladder cancer, which does not invade the muscle wall.We will discuss the clinical case of a patient who had fever of unknown origin for about 2 months and was resistant to treatment with multiple lines of antibiotics
The Importance of the Pharmacoeconomic Analyses in Drug Negotiation. A Farewell Editorial
Full text link[Burden of Short Bowel Syndrome in Italy: Direct and Indirect Costs and Quality of Life]
Full text linkOBJECTIVE: The Short Bowel Syndrome (SBS) is the most common cause of chronic intestinal failure (CIF) due to benign disease. The prevalence in Europe was estimated about 1.4 cases per million and few information are available for Italy. Home parenteral nutrition (HPN) is the primary and lifesaving treatment for patients with CIF. The parenteral nutrition (PN) has a great impact on the quality of life (QoL) of patients and the role of the caregiver is crucial for the disease management. The aim of this study was to evaluate the economic burden of SBS in Italy and to assess the impact of the disease and the parenteral nutrition on the quality of life of patients.METHODS: The total burden of SBS in Italy was assessed both in terms of costs and QoL using different tools. A prevalence-based cost of illness (COI) model was developed to estimate direct costs (PN cost, central venous catheter insertion cost, monitoring cost, hospitalization cost) and indirect costs (absenteeism, presenteeism, unemployment, abandonment of work due to the disease and economic benefits granted by the National Social Security Institute) associated with patient with SBS in Italy. The total annual costs were calculated considering a micro-costing approach, thus associating the average costs per patient with the prevalence of the disease. A systematic literature review (SLR) was conducted to collect epidemiological and direct cost data related to the patients with SBS. Hospital costs were estimated using the national Hospital Information System. Indirect costs were estimated using a human capital approach; therefore, the productivity loss was estimated both for patients and caregivers. A survey was conducted to obtain data about productivity loss and quality of life of patients and caregivers. The questionnaires were completed by clinicians, who were asked to indirectly report the experience of patients with SBS in parenteral nutrition currently being treated at their referral center. In a subsequent phase, a focus group was conducted to collect further information on QoL for patients and caregivers based on the experience of the KOLs involved. The QoL was evaluated considering a Likert scale.RESULTS: The prevalence of patients with gastrointestinal disease in HPN was estimated equal to 9.4 and 2.3 patients per million inhabitants for adults (age >18) and pediatric (age 0-18 years) patients, respectively. Knowing that SBS is the main cause of CIF due to benign disease, constituting 75% among adults and 56% among children, the number of adults with SBS in HPN present in Italy were 420, while the number of children with SBS in HPN were 77. Regarding direct costs, the mean total annual cost associated with adult and pediatric patient with SBS in Italy was estimated equal to € 36,434 and € 46,682, respectively. Parenteral nutrition accounted for 91% of the mean total cost estimated for the adult and for 87% of the mean total cost estimated for pediatric patient. Concerning indirect costs, the mean total annual cost per adult patient was estimated equal to € 51,093 (81% related to the productivity loss because of the abandonment of work due to the disease), while the mean total cost per pediatric patient was estimated equal to € 3,201 (60% related to caregiver’s presenteeism and 40% attributable to caregiver’s absenteeism). Finally, the average total annual cost per adult SBS patient in Italy was estimated at € 87,527 (42% of direct costs and 58% of indirect costs), whereas for pediatric patients it was estimated at € 49,882 (94% of direct costs and 6% of indirect costs). Overall, the weighted average cost for an SBS patient in Italy was estimated at € 81,712 (47% of direct costs and 53% of indirect costs). The analysis conducted on the QoL of patients with SBS in PN has shown that QoL was perceived as low (mean value equal to 5). The greatest impact on QoL was due to the disease (mean value equal to 9), while PN appeared to have less impact (mean value equal to 6).CONCLUSIONS: The analysis provides an estimate of the total burden associated with patients with SBS in Italy both in terms of cost and QoL. The cost associated with parenteral nutrition and indirect costs represent the main drivers of the total cost estimated for a patient with SBS in Italy. Based on the experience of the KOL involved in this study it was also found that the disease has a great impact on the QoL of these patients
Pulmonary Complications in a Patient with Common Variable Hypogammaglobulinemia
Full text linkCommon variable immunodeficiency (CVID) is a rare disorder characterized by primary antibody deficiency leading to hypogammaglobulinemia and increased risk of infections. Pulmonary hypertension (PH) is an unusual complication of CVID and may be associated with chronic hypoxemic respiratory failure.We described the case of a 47-year-old female patient hospitalized with worsening dyspnea, which had emerged about 10 days before and was associated with productive cough. 26 years before, she was diagnosed with common variable hypogammaglobulinemia, that was treated with intravenous immunoglobulin infusions for at least 20 years. She had suffered from recurrent secondary infections of the respiratory tract with inveterate bronchiectasis, pulmonary hypertension, and chronic respiratory failure.This case suggests that Internal Medicine wards, due to their global vision of the patient, are well suited to manage subjects with serious, complex, and genetically determined pathologies
Candida guilliermondii Peritonitis During Peritoneal Dialysis. Case Report and Literature Review
Full text linkPeritonitis is the most frequent complication of peritoneal dialysis (PD) and 3–6% of episodes have fungal origin. Candida guilliermondii is an uncommon species of Candida with invasive behavior in patients affected by severe underlying disorders or using indwelling vascular devices.Here we report the case of an 84-year-old woman undergoing outpatient PD for 4 years who had fever, chills, and diffuse increasing abdominal pain. After empiric antimicrobial therapy, based on teicoplanin in the dialytic circuit plus oral ciprofloxacin plus fluconazole, the patient was hospitalized. Afterwards, the culture from the peritoneal fluid showed the presence of C. guilliermondii.PD-related fungal peritonitis is an infrequent event, but the morbidity and mortality rates are significant. In this scenario, appropriate prevention strategies including antifungal prophylaxis during antibiotic treated bacterial peritonitis should be evaluated.
[Budget Impact Analysis of the Adjuvanted Quadrivalent Influenza Vaccine in the Elderly in Italy]
Full text linkBACKGROUND: Vaccination is the most effective way to prevent influenza and its complications. The MF59-adjuvanted quadrivalent (aQIV) and the high-dose quadrivalent (QIV-HD) influenza vaccines have been specifically developed to protect subjects aged ≥65 years. The aim of this study was to evaluate the economic consequences associated with the use of aQIV in the elderly population in Italy.METHODS: An Excel-based budget impact model was developed to estimate the costs of aQIV for the prevention of influenza and its complications in elderly subjects, from the perspective of the Italian National Health Service. In the base-case analysis a current scenario (with aQIV) was compared with a past scenario (without aQIV), in which only the standard quadrivalent influenza vaccine (QIV-STD) was available. In the scenario analysis, a current scenario was compared with a future (hypothetical) scenario, in which the market share of aQIV grows. Efficacy (or effectiveness) data of vaccines, in terms of reduction of influenza-related deaths and hospitalizations for influenza/pneumonia, respiratory, and cardiac complications, were obtained from the scientific literature. Relative effectiveness of aQIV and QIV-HD vs. QIV-STD in preventing laboratory-confirmed influenza cases came from two meta-analysis of real-world studies. Epidemiological data and unit costs are collected from Italian published sources.RESULTS: In the base-case analysis the introduction of aQIV e QIV-STD was associated with a reduction of influenza and influenza-like illness – ILI (-93,171) cases, hospitalization for influenza, respiratory and cardiovascular complications (-6,823), and deaths (-1,841) and a consequent saving of € 1.9 million and € 24 million related to events and hospital admissions avoided, respectively. In the scenario analysis, the growth of the market shares of aQIV was associated with 13,602 fewer cases of influenza and ILI, 996 fewer hospitalizations, and an overall saving equal to € 3.8 million.CONCLUSIONS: aQIV reduces the number of cases of influenza and ILI, hospitalizations for influenza and respiratory or cardiac complications, and deaths in the Italian population aged ≥65 years