Farmeconomia. Health economics and therapeutic pathways
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[Measurement-Based Procurement Approach for Biosimilars in Italy: A Position Paper]
No full textThe 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
[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
[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
Artificial Urinary Sphincters as a Treatment for Post-Prostatectomy Severe Urinary Incontinence in Italy: A Cost-Utility Analysis
Full text linkObjective: This study aimed at evaluating the cost-utility of artificial urinary sphincter (AUS) in men affected by postprostatectomy severe urinary incontinence and identifying the most cost-effective alternative among the various devices analyzed in Italy.Methods: A 5-year cycles Markov model was developed to simulate the disease evolution. The analysis compared conservative therapy, ZSI 375®, single-cuff (SC) AMS 800TM, and double-cuff (DC) AMS 800TM. A Probabilistic Sensitivity Analysis (PSA) was performed. One thousand Monte Carlo simulations were conducted to generate the Cost-Effectiveness Acceptability Curve for each intervention strategy. A sensitivity analysis on the price of the device was conducted.Results: From the Italian National Health Service perspective, DC AMS 800TM was the most cost-effective alternative in comparison with conservative therapy, with an Incremental Cost-Effectiveness Ratio (ICER) value equal to € 12,893. From the NHS + patient perspective, both the AMS 800TM devices (SC and DC) were dominant in comparison with conservative therapy. From the societal perspective, ICER was dominant for all the alternatives considered in terms of cost-effectiveness. The PSA showed that DC AMS 800TM had a greater probability to be cost-effective with respect to the other strategies considered in the analysis. The sensitivity analysis on the price of the device showed that in all the cases analyzed the incremental cost per QALY gained would be below € 25,000.Conclusions: This cost-utility analysis confirms that AUSs are cost-effective options in the Italian context with respect to conservative therapy. Among AUSs, DC AMS 800TM has the greatest probability to be cost-effective
[Cost-Utility Analysis of Dupilumab for the Treatment of Severe Atopic Dermatitis in Children and Adolescents in Italy]
No full textBACKGROUND AND OBJECTIVE: Atopic dermatitis (AD) is a chronic, multifactorial, inflammatory condition characterized by a significant impact on patients’ quality of life. Dupilumab is reimbursed by the Italian Medicines Agency (AIFA) for the treatment of adolescent and adult patients with severe AD (according to AIFA registry criteria). Recently, dupilumab has been reimbursed in the treatment of children with severe AD. The objective of this analysis was to estimate the incremental cost-utility ratio (ICUR) of dupilumab compared to current supportive care (SC), for the treatment of severe AD in children (6-11 years) and adolescents (12-17 years) in Italy.MATERIALS AND METHODS: Cost-effectiveness analysis was conducted using a 1-year decision tree followed by a Markov model over a lifetime period. The base case analysis was performed on the overall population of the LIBERTY AD ADOL (NCT03054428) and LIBERTY AD PEDS (NCT03345914) studies, adopting the National Health Service (NHS) perspective. The following costs were considered: acquisition of treatment, management of disease, adverse events and complications. The robustness of the model was tested through sensitivity analysis. In addition, a scenario analysis adopting the social perspective was performed.RESULTS: In the base case, over a lifetime, dupilumab was more effective than SC in both children and adolescents (+2.44 and +1.62 quality-adjusted life years—QALYs, respectively). The introduction of dupilumab generated an increase in treatment costs (+€ 64,800 and +€ 52,853 € for children and adolescents, respectively), partially offset by a decrease in the costs of disease management and complications. Incremental cost-utility ratios (ICURs) were € 21,189 per QALY gained, for children, and € 26,569 per QALY gained, for adolescents. In both cases, the ICUR was lower than the willingness to pay threshold considered in Italy (€ 50,000 per QALY gained). Both the deterministic and probabilistic sensitivity analysis confirmed the robustness of the base case results. Finally, the scenario analysis, adopting the social perspective, showed coherent results compared to the base case.DISCUSSION: Dupilumab is a cost-effective option for the treatment of children and adolescents with severe AD eligible for systemic treatment in Italy compared to SC, from both the NHS and social perspective, confirming the results obtained in the adult population
[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
Cost-effectiveness of Empagliflozin, in Addition to Metformin, in Patients with Type 2 Diabetes in Italy
Full text linkBACKGROUND: Cardiovascular diseases represent the main cause of mortality and morbidity in type 2 diabetes mellitus (T2DM) patients. Empagliflozin is used as a treatment for T2DM because of its association with reduced risk of hospitalization for heart failure (hHF). Recently oral semaglutide, in association with metformin, has shown better results. This study analyzes the cost-effectiveness of empagliflozin versus oral semaglutide, in addition to metformin, in patients with T2DM who are inadequately controlled on metformin alone in Italy.METHODS: This analysis was conducted from the Italian National Health Service (SSN) perspective using the IQVIA Core Diabetes Model. For the base case analysis, a 50-year time horizon was chosen to capture the complications, their associated costs, and the final impact on life-years (LYs) and quality-adjusted life-years (QALYs) gained. Cohort baseline characteristics and efficacy data, were mainly sourced from the PIONEER 2 study. Health-state utilities and event disutilities were based on published sources. Drug acquisition and administration costs and patient management inputs were sourced from Italian-specific data. A sensitivity analysis and a range of scenario analyses were carried out.RESULTS: In the base case analysis treatment cost of empagliflozin plus metformin were significantly lower compared to oral semaglutide plus metformin both including and excluding the effect of empagliflozin on hHF (€-13.371/€-13.580; LYs -0.004/0.109 and QALYs -0.037/0.038). The sensitivity analysis confirmed the robustness of the model with empagliflozin plus metformin that was dominant in 63% and in 42% of simulations considering and non-considering the treatment effect on hHF, respectively.CONCLUSIONS: Empagliflozin 25 mg plus metformin is a cost-effective option versus oral semaglutide 14 mg plus metformin for patients with T2DM uncontrolled on metformin alone in Ital
Cost-Effectiveness of Dimethyl Fumarate Compared to Teriflunomide for Relapsing Remitting Multiple Sclerosis Patients in Italy
Full text linkBACKGROUND: The objective of this economic analysis was to compare the cost-effectiveness of dimethyl fumarate vs teriflunomide for the treatment of adult patients with relapsing-remitting multiple sclerosis (RRMS) in the Italian setting. Additionally, the cost-effectiveness analysis was used to predict some patient-relevant outcomes such as burden of relapses and survival with disability over time.METHODS: A Markov model was used to conduct the cost-effectiveness analysis. The model measured health outcomes and costs of RRMS patients treated with either dimethyl fumarate or teriflunomide. Data from a published mixed treatment comparison were used for efficacy and safety input. Local economic data were used to calculate costs. A supplementary analysis was carried out to assess ICER variability over time from the Italian National Healthcare Service (NHS) and societal perspectives. Further analyses were conducted to compare clinical effectiveness of the alternatives over time, in terms of incidence of relapses, proportion of patients with EDSS (Expanded Disability Status Scale) score ≤3 and EDSS score ≥6.RESULTS: In the base-case analysis (lifetime horizon; societal perspective) dimethyl fumarate was dominant over teriflunomide (6.526 vs 5.953 QALYs – quality-adjusted life-years; € 1.01 M vs € 1.03 M). The most relevant cost savings (per-patient) with dimethyl fumarate were related to relapses (-€ 5,096), inpatient care (-€ 5,767), informal care (-€ 9,603), long-term absence/early retirement (-€ 14,187). The additional analysis of ICER by time horizon shows that dimethyl fumarate is cost-effective vs teriflunomide (i.e., ICER <€ 50,000 per QALY gained) at already 6 years and at 15 years in societal or NHS perspectives, respectively. Results favoured dimethyl fumarate vs teriflunomide also for: cumulative burden of relapses (-0.23 and -1.37 relapses saved per patient already at 1 year and 10 years, respectively), proportion of patients with mild disability (+4.0% at 10 years), proportion of patients with severe disability (-4.0% at 10 years).CONCLUSIONS: Dimethyl fumarate is dominant (societal perspective), or cost-effective (NHS perspective), referring to a threshold of € 50,000 per QALY gained, vs teriflunomide for the first-line treatment of RRMS, in the Italian setting
Economic Consequences of Administering Obinutuzumab as a Short Duration Infusion in Italian Patients with Advanced Follicular Lymphoma: A Cost Analysis
Full text linkOBJECTIVE: To assess time- and cost-savings in relation to active time of health care professional (HCP) and resource consumption of administering obinutuzumab as a short duration infusion (SDI) in patients in treatment for Follicular Lymphoma (FL).METHODS: A cost-minimization model was developed to compare resource consumption and cost of the obinutuzumab SDI relative to obinutuzumab regular infusion rate (RIR) for the previously untreated and rituximab-refractory FL. Monetary valuation of resource and time allocated to treatment as a whole was carried out from the Italian Hospital and the societal perspective. Direct costs included HCP costs for drug preparation and administration activities, non-drug consumable costs, drug acquisition costs, and formal care costs. Indirect costs included the lost productivity of patients and informal caregivers. All costs (updated to 2021-value) were estimated by multiplying resource use by the unit cost of each resource. Evidence on resource use and unit costs were retrieved from scientific literature and standard Italian tariffs. A deterministic sensitivity analysis was used to test the results.RESULTS: The administration time of obinutuzumab SDI is shorter than with obinutuzumab RIR, with a difference of 102 minutes per patient and for every cycle of administration beyond the first one. On average, the cost of HCP time invested in the preparation and administration of obinutuzumab RIR is € 92 during cycle 2 and from cycle 2 onwards, compared to € 54 per cycle of obinutuzumab SDI. Overall, the cost from the societal perspective is estimated to be € 38,698 for obinutuzumab RIR and € 37,692 for obinutuzumab SDI, resulting in a cost-saving per patient of € 1,007 (2.6%).CONCLUSIONS: The application of obinutuzumab SDI schedule allows substantial reduction of hospital stay, improving quality of life of patient and caregiver and reducing costs and health care system burden. The time-savings with obinutuzumab SDI may improve clinical unit capacity by optimizing chair utilization and/or allowing rearrangements of the nurse residual time into valuable supplementary activities, spanning from more patient-centered clinical support to research and learning activit
Budget Saving Potential of Pegfilgrastim Biosimilar for the Treatment of Chemotherapy-Induced Febrile Neutropenia, in Italy
Full text linkINTRODUCTION: Current Italian guidelines recommend prophylaxis with granulocyte colony-stimulating factors (G-CSFs) to reduce the risk of chemotherapy-induced febrile neutropenia (FN). The availability of G-CSF biosimilars represents an opportunity for savings in the Italian National Healthcare Service (NHS) delivery of care.OBJECTIVE: To assess the cost saving potential associated with the introduction of pegfilgrastim biosimilars to local formularies, compared to the current G-CSF standard practice in Italy.METHODS: A budget impact model was developed to compare the current standard practice of long-acting (LA) and short-acting (SA) G-CSFs use, with a future scenario in which the market share of LA G-CSFs grows due to the more advantageous administration schedule and price of pegfilgrastim biosimilar. The analysis included G-CSF treatment schedules, drug acquisition costs and costs of patient management including hospitalization and ambulatory care.RESULTS: The introduction of pegfilgrastim biosimilar resulted in cumulative 3-year cost savings of € 59,650 and € 41,539 for FN prophylaxis in a potential cohort of 1000 patients with solid tumors and lymphomas, respectively.CONCLUSIONS: The results indicate that the introduction of pegfilgrastim biosimilar is potentially associated with substantial cost savings for the Italian healthcare system