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Introduction: Pulmonary arterial hypertension (PAH) is a rare disease involving small pulmonary arteries and is characterized by vascular proliferation and remodeling. The financial impact of therapies for the treatment of PAH in the Italian context is unknown. Objective: The objective of this study was to carry out a budget impact analysis comparing ambrisentan with bosentan for patients with class II or III PAH in the Italian context over a 3-year timeline. Methods: Data on efficacy and rate of treatment discontinuation were taken from two separate double-blind studies (no head-to-head studies were available) and supported by expert’s opinion. The analysis was conducted from the perspective of the National Health System and included drug acquisition cost, direct medical costs associated to ambrisentan or bosentan treatment, and direct medical costs associated to the treatment of adverse events. Results: Over a 3-year timeline, total costs for the cohort of patients considered would be €86,482,146 with a...

Objectives: The study evaluates the costs of systemic lupus erythematosus (SLE) and the budget impact due to the introduction of belimumab in the Italian setting.Methods: Adaptation to the Italian setting of a budget impact model with a time horizon of 4 years (year 0 without belimumab, years 1–3 with belimumab) to compare treatment, administration, and clinical monitoring costs of standard therapy and of the alternative scenario in which belimumab is administered in addition to the standard therapy to the subgroup of patients selected according to the label approved by the European Medicines Agency. The model takes also into account the costs of flares.Results: Over 3 years, belimumab is able to prevent cumulatively 1,111 severe flares and 3,631 nonsevere flares with a total saving for the Italian National Health System (NHS) of approximately €6.2 million. Budget impact ranges from €4.4 million in the first year to €20.3 million in the third year.Conclusions: The decrease in the number of flare partially counterbalances the costs of the new technology (impact attenuation of approximately 16 percent). These data elucidate the importance to control and monitor the disease progression and to prevent exacerbations, which are the major causes of the increase in costs paid by the NHS and by the society. The financial impact could be replicate on a regional basis, to inform local decision makers. Further developments are possible as the model does not consider the additional clinical and economic benefits of reduced damage accrual and slowed disease progression.

Objective The purpose of this analysis is to evaluate the cost-effectiveness of belimumab, a new biological treatment specifically developed for the treatment of Systemic Lupus Erythematosus (SLE), in the Italian setting. SLE is a chronic non-organ specific autoimmune disease characterized by a disregulation of the immune system that involves many organs and systems. Methods A cost-effectiveness micro-simulation model with a lifetime horizon originally developed for the UK was adapted to the Italian setting. The analysis compared Standard of Care (SoC) alone vs belimumab plus SoC from a National Healthcare Service (NHS) and societal perspective. Health-economic consequences of treatments and organ damage progression were calculated. When available, Italian data were used, otherwise UK costs were converted using Purchasing Power Parities (PPPs). Utility values were based on the EQ-5D™ assessments in the belimumab clinical trials (BLISS 52 and 76). Results were discounted with 3% for costs and effects. A maximum belimumab treatment duration of 6 years was assumed and wastage costs were considered. Results Cost per life year gained (Incremental Cost-Effectiveness Ratio, ICER) and cost per Quality Adjusted Life Year (QALY) (Incremental Cost-Utility Ratio, ICUR) were €22,990 and €32,859, respectively. These values reduced to €20,119 and €28,754, respectively, when indirect costs were included. Conclusions It may be concluded that in the Italian setting and according to the guidelines of the Italian Association of Health Economics (IAHE), belimumab was shown to be cost-effective, in terms of both ICER and ICUR, (€25–40,000/QALY).

Objective.Systemic lupus erythematosus (SLE) is treated with anti-inflammatory and immunosuppressive drugs and off-label biologics. Belimumab is the first biologic approved after 50 years as an add-on therapy for active disease. This paper summarizes a health technology assessment performed in Italy.Methods.SLE epidemiology and burden were assessed using the best published international and national evidences and efficacy and safety of belimumab were synthesized using clinical data. A cost-effectiveness analysis was performed by a lifetime microsimulation model comparing belimumab to standard of care (SoC). Organizational and ethical implications were discussed.Results.Literature review showed that SLE affects 47 per 100,000 people for a total of 28,500 patients in Italy, 50% of whom are affected by active form of the disease despite SoC. These patients, if autoantibodies and anti-dsDNA positive with low complement, are eligible for belimumab. SLE determines work disability and a 2–5-fold increase in mortality. Belimumab with SoC may prevent 4,742 flares in three years being cost-effective with an incremental cost-effectiveness ratio of €32,859 per quality adjusted life year gained. From the organizational perspective, the development of clear and comprehensive clinical pathways is crucial.Conclusions.The assessment supports the use of belimumab into the SLE treatment paradigm in Italy.

Community-acquired pneumonia (CAP) is an infectious disease with high morbidity and mortality and a major social and economic impact. A budget impact model was developed to estimate the impact on hospital direct costs of different antibiotic therapies suggested by international and national (FADOI) guidelines on treatment of patients hospitalized with CAP. The model includes the costs of drugs, staffing, consumables and in-patient stays in two different scenarios: intravenous therapy only and switch therapy; it compares levofloxacin (monotherapy) versus other combination therapies as suggested by the guidelines and includes the cost of failure of first-line treatment. Budget impact analysis shows that the cost of CAP-hospitalized patients in Italy consists mainly in the cost of treatment failure while that of antibiotics is just a small component of total direct costs incurred by hospitals.

A focused exploration targeting conformationally restricted analogues of Vestipitant, resulted in the discovery of novel, in vitro potent NK 1 antagonists. In particular, two of the compounds reported exhibited a good pharmacokinetic (PK) profile and produced anxiolytic-like effects in the gerbil foot tapping (GFT) in vivo model.

We have studied the effects of chromomycin and of a triple-helix-forming oligonucleotide (TFO) that recognizes Sp1 binding sites on protein–DNA interactions and HIV-1 transcription. Molecular interactions between chromomycin, the Sp1 TFO and target DNA sequences were studied by gel retardation, triplex affinity capture using streptavidin-coated magnetic beads and biosensor technology. We also determined whether chromomycin and a TFO recognizing the Sp1 binding sites of the HIV-1 long terminal repeat (LTR) inhibit the activity of restriction enzyme HaeIII, which recognizes a sequence (5′-GGCC-3′) located within these Sp1 binding sites. The effects of chromomycin and the TFO on the interaction between nuclear proteins or purified Sp1 and a double-stranded oligonucleotide containing the Sp1 binding sites of the HIV-1 LTR were studied by gel retardation. The effects of both chromomycin and TFO on transcription were studied by using an HIV-1 LTR-directed in vitro transcription system. Our results indicate that low concentrations of chromomycin potentiate the effects of the Sp1 TFO in inhibiting protein–DNA interactions and HIV-1-LTR-directed transcription. In addition, low concentrations of chromomycin do not affect binding of the TFO to target DNA molecules. The results presented here support the hypothesis that both DNA binding drugs and TFOs can be considered as sequence-selective modifiers of DNA–protein interactions, possibly leading to specific alterations of biological functions. In particular, the combined use of chromomycin and TFOs recognizing Sp1 binding sites could be employed in order to abolish the biological functions of promoters (such as the HIV-1 LTR) whose activity is potentiated by interactions with the promoter-specific transcription factor Sp1.