The objective of this economic evaluation was to compare two alternative therapeutic regimens for the treatment of type 2 diabetes: basal-bolus insulin vs basal insulin and DPP-4. We conducted a ...Cost-Minimization Analysis (CMA) to compare the annual cost of basal-bolus insulin regimen vs basal insulin and DPP-4 regimen from the perspective of the Italian National Health Service (NHS). The CMA considered only direct medical costs (basal insulin, bolus insulin and DPP-4 sitagliptin and self-monitoring of glycemic control). Costs were assessed in 2014 Euros. Sensitivity analysis and threshold analysis on key economic parameters were performed. The expected annual cost (per patient) was €987.60 with basal insulin and DPP-4 and €1,568.23 with basal-bolus insulin. As a consequence of a significant reduction of glycemic control costs, the regimen with DPP-4 was a cost-saving alternative from the perspective of the Italian NHS.
Background: Primary (PID) or secondary (SID) immunodeficiencies are diseases caused by quantitative and/or functional alterations of the different mechanisms involved in the innate and adaptive ...immune response. This economic evaluation was conducted to compare the cost of treatment of HYQVIA® (hyaluronidase-facilitated subcutaneous infusion of immunoglobulin, fSCIG) compared to intravenous immunoglobulin (IVIG) or subcutaneous immunoglobulin (SCIG), currently reimbursed in Italy, in the treatment of PIDs or SIDs.
Methods: A cost-minimisation analysis was carried out, considering the hospital’s perspective. The direct medical costs (cost of immunoglobulins and cost of administration) were assessed. The analysis was conducted considering one year (52 weeks) time horizon. The reference population included adult patients with PID with impaired antibody production or adult patients with SID with severe or recurrent infections, ineffective antimicrobial treatment and documented specific antibody defect (PSAF) or serum IgG level < 4 g/L.
Results: In the maintenance treatment of PID, HYQVIA® (€ 20,020.00) was the therapeutic alternative with the lowest mean annual cost compared to HIZENTRA® (€ 22,165.19) and VENITAL® (€ 24,967.68). Moreover, in the maintenance treatment of SIDs, HYQVIA® (€ 17,160.00) was the cost-saving therapeutic alternative compared to VENITAL® (€ 22,107.68). A sensitivity analysis confirmed the base case results.
Conclusion: Due to lower costs of administration and different scheme of administration, HYQVIA® was a cost-saving alternative to SCIG e IVIG in the treatment of PID and to IVIG in the treatment of SID
Background: The availability of high-efficacy disease-modifying therapy (DMT), including natalizumab, improved treatment efficacy in adults with highly-active relapsing-remitting multiple sclerosis ...(RRMS). Natalizumab patent protection has expired, and the natalizumab biosimilar (Tyruko®) has been recently reimbursed by AIFA. As the price of natalizumab biosimilar is expected to be lower as compared with natalizumab originator’s price, a budget impact analysis was conducted to assess the economic impact associated to the introduction of natalizumab biosimilar for patients with highly-active RRMS. Methods: A budget impact model was developed, considering the INHS perspective and a 5-years time horizon. The number of patients treated with natalizumab was estimated based on historical natalizumab consumption data, disease prevalence rates and natalizumab market share. The budget impact population was divided into prevalent and incident patients. The model assumes that some patients in treatment with natalizumab originator will switch to natalizumab biosimilar and that some naïve patients will directly start treatment with natalizumab biosimilar. The ex-factory price of natalizumab originator (intravenous and subcutaneous) and biosimilar (intravenous) and the corresponding administration costs were included. All assumptions were validated by expert opinion. Results: Eligible population was estimated at 7,552, 7,779, 8,090, 8,494 and 8,834 in years 1, 2, 3, 4 and 5 respectively. The introduction of natalizumab biosimilar, considering a progressive increase in market share from 9.6% (year 1) to 40.5% (year 5), would provide an overall savings (5-years time horizon) over € 47 million to the INHS. The scenario analysis highlights that the lower treatment cost of biosimilar natalizumab compared to originator natalizumab would offset the higher cost associated with intravenous versus subcutaneous administration. Conclusion: Considering the results of this budget impact analysis, it is realistic to expect that the presence of biosimilar natalizumab will contribute to the sustainability of public pharmaceutical expenditure.
Among the functional subpopulations that coexist within the tumor, ‘cancer stem cells’ are characterized by increased self-renewal and the ability to derive all of the other subpopulations of tumor ...cells (‘bulk’). The functional heterogeneity among cancer stem cells and bulk cells must reflect distinct cellular epigenetic landscapes, but — due to the difficulty to isolate bona fide cancer stem cells with a high degree of purity — those different epigenetic landscapes, and the molecular mechanisms underlying them, remain largely unknown. Cues of intratumor phenotypic plasticity complicate the interpretation of the cancer stem cell phenotype: we contend that, however, the concept of cancer stem cell has crucial therapeutic implication, and remains a key target for the exploration of the cancer epigenome.
Background: Enterosorbents are orally administered materials which pass through the gut where they bind various substances. In a randomized clinical trial, the efficacy and safety of enterosorbent ...Enterosgel® in the treatment of non-infectious diseases with diarrhoea have been proved.
Objective: To assess the cost impact of Enterosgel® in the treatment of hospitalized patients with non-infectious diseases with diarrhoea adopting a cost/minimization approach.
Methods: A cost analysis was conducted considering the hospital’s perspective. The analysis compared the treatment costs (drugs and hospital stays) of Enterosgel® in addition to the standard of care (SoC) to SoC alone. The main analysis focused on the comparison between the two therapeutic strategies regarding the economic valorization of hospital stays, while the secondary analysis estimated the hospital organizational efficiency with regard to the annual bed turnover.
Results: Reducing the duration of inpatient admission, compared to SoC alone, Enterosgel® in addition to SoC let the hospital obtain a greater valorization of the hospital stays (range: € 68.54-€ 558.60). The shorter duration of inpatient admission results in an increased number of hospital admissions per year. For example, assuming a 10-bed ward, the total hospital annual gain would be € 82,616.
Conclusions: Enterosgel® in addition to SoC is cost saving, allowing the hospital to achieve greater efficiency in managing patients with non-infectious diseases with diarrhoea.
Caloric Restriction (CR) has established anti-cancer effects, but its clinical relevance and molecular mechanism remain largely undefined. Here, we investigate CR's impact on several mouse models of ...Acute Myeloid Leukemias, including Acute Promyelocytic Leukemia, a subtype strongly affected by obesity. After an initial marked anti-tumor effect, lethal disease invariably re-emerges. Initially, CR leads to cell-cycle restriction, apoptosis, and inhibition of TOR and insulin/IGF1 signaling. The relapse, instead, is associated with the non-genetic selection of Leukemia Initiating Cells and the downregulation of double-stranded RNA (dsRNA) sensing and Interferon (IFN) signaling genes. The CR-induced adaptive phenotype is highly sensitive to pharmacological or genetic ablation of LSD1, a lysine demethylase regulating both stem cells and dsRNA/ IFN signaling. CR + LSD1 inhibition leads to the re-activation of dsRNA/IFN signaling, massive RNASEL-dependent apoptosis, and complete leukemia eradication in ~90% of mice. Importantly, CR-LSD1 interaction can be modeled in vivo and in vitro by combining LSD1 ablation with pharmacological inhibitors of insulin/IGF1 or dual PI3K/MEK blockade. Mechanistically, insulin/IGF1 inhibition sensitizes blasts to LSD1-induced death by inhibiting the anti-apoptotic factor CFLAR. CR and LSD1 inhibition also synergize in patient-derived AML and triple-negative breast cancer xenografts. Our data provide a rationale for epi-metabolic pharmacologic combinations across multiple tumors.
Background: The aim of this economic evaluation was to compare the cost per responder between vedolizumab and ustekinumab in patients with Crohn’s disease (CD) after failure of tumor necrosis ...factor-α inhibitors in Italy.
Methods: Clinical efficacy was assessed using the results of an Italian large multicentre observational retrospective cohort study. The aim of the study was to compare the effectiveness of ustekinumab and vedolizumab as second line therapy in Crohn’s disease patients in which tumour necrosis factor-α inhibitors failed. Clinical efficacy of vedolizumab and ustekinumab was measured by clinical response and clinical remission. Treatment costs were based on the number of administrations at 26 or 52 weeks. Cost per responder, based on clinical efficacy and clinical response, was used as a cost-effectiveness indicator.
Results: Regardless of the clinical efficacy measure used and the treatment duration considered, the cost per responder was consistently lower for vedolizumab compared with ustekinumab on all clinical measures. Considering the clinical response, the cost per responder at 26 weeks was € 15,640 for vedolizumab and € 23,667 for ustekinumab and at 52 weeks was € 23,927 for vedolizumab and € 30,820 for ustekinumab. Considering the clinical remission, the cost per responder at 26 weeks was € 22,832 for vedolizumab and € 33,786 for ustekinumab and at 52 weeks was € 29,488 for vedolizumab and € 46,847 for ustekinumab. Sensitivity analysis confirmed the validity of results.
Conclusion: These results suggest that vedolizumab is a cost-effective option compared with ustekinumab from the perspective of the Italian health service in patients with CD after failure of TNF-α inhibitors.
Background: Targeted systemic therapies, including abrocitinib, baricitinib, dupilumab, tralokinumab and upadacitinib, are new treatments for moderate to severe atopic dermatitis (AD). We evaluated ...the efficacy and the costs of these targeted systemic therapies in the treatment of adult patients with moderate to severe AD. Methods: The clinical efficacy was assessed considering the results of a previous network meta-analysis (NMA). The analysis involved five therapies approved in Italy for the treatment of moderate to severe AD: abrocitinib (ABR), baricitinib (BAR), dupilumab (DUP), tralokinumab (TRA) and upadacitinib (UPA). According to the NMA, the cost of the treatment was based on the number of administrations dispensed at 16 weeks and the clinical efficacy was measured by the number needed to treat (NNT) compared to placebo using the improvement ≥ 75% (EASI-75) or ≥ 90 (EASI-90) from baseline of the eczema area and severity index (EASI). Only the ex-factory price of the targeted systemic therapies was considered. The cost per NNT was adopted as a cost-effectiveness indicator. Results: At 16 weeks, the cost per NNT based on EASI-75 was lower for UPA 15 mg (€ 6,384.00) compared to BAR 4 mg (€ 11,619.73) and 2 mg (€ 14,524.66), ABR 100 mg (€ 16,265.22), DUP 300 mg (€ 16,115.04) and TRA 300 mg (€ 31,710.24). UPA 15 (€ 8,512.00) also showed the lower cost per NNT based on EASI-90 at 16 weeks compared to BAR 4 mg (€ 14,788.75) and 2 mg (€ 20,862.70), ABR 100 mg (€ 25,922.69), DUP 300 mg (€ 25,992.00) and TRA 300 mg (€ 41,067.36). Conclusions: The findings show that upadacitinib is the most cost-effective option (cost per NNT) for the treatment of moderate to severe atopic dermatitis.
In the article “Cost per responder for upadacitinib vs abatacept in patients with moderate-to-severe Rheumatoid Arthritis in Italy”, (1) which appeared in Volume 8, Issue 1 of Global & Regional ...Health Technology Assessment, the values appearing in Fig. 5 C and D, inadvertently repeated the values reported in Fig. 5 A and B. Affected data have been corrected in the article now appearing online. The authors apologize for any inconvenience caused to the readers by these changes, which do not affect the final results of the study.
The final version of this article is available online and includes a reference to this correction.
Objectives: To evaluate the cost-effectiveness analysis (CEA) of caplacizumab in combination with plasmapheresis (PEX) and immunosuppression compared to PEX and immunosuppression in the treatment of ...acute episodes of iTTP.
Methods: A Markov model was used to conduct the CEA from the perspective of the hospital, over a lifetime horizon. Clinical data derived from HERCULES trial and a systematic literature review. Economic input included direct costs only. Utility and disutility values were obtained from literature. Data on healthcare resources and costs were retrieved from HERCULES trial, literature, TTP guidelines and Italian tariffs. A sensitivity analysis was conducted. The cost-effectiveness probability was tested for several options of discount levels considering a suggested willingness to pay (WTP) threshold of € 60,000 in Italy.
Results: The use of caplacizumab in combination with PEX and immunosuppression is associated with a positive difference in survival of 3.27 life years (24.53 vs 21.26) and in quality of life of 3.06 QALY (22.01 vs 18.96) when compared to PEX and immunosuppression. Caplacizumab leads to an ICER per life years of € 41,653 and an ICER per QALY of € 44,572. For the suggested WTP threshold, the probability of caplacizumab being cost-effective is 82.4% (no discount), 92.8% (15% discount), 95.3% (20% discount), 96.9% (25% discount) and 98.2% (30% discount).
Conclusions: Caplacizumab in addition to PEX and immunosuppression is cost-effective, allowing the hospital to achieve greater efficiency in managing the burden of a life-threatening disease such as iTTP.
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