Cost-Effectiveness and Budget Impact of Emicizumab Prophylaxis in Haemophilia A Patients with Inhibitors.

Recent evidence demonstrated that weekly prophylaxis with subcutaneous bispecific antibody (emicizumab) has shown higher efficacy in adolescent and adults patients affected by haemophilia A (HA) with inhibitor, compared with patients treated on demand or on prophylaxis with bypassing agents (BPAs). However, no economic evaluations assessing the value and sustainability of emicizumab prophylaxis have been performed in Europe. This study assessed the cost-effectiveness of emicizumab prophylaxis compared with BPA prophylaxis and its possible budget impact from the Italian National Health Service (NHS) perspective. A Markov model and a budget impact model were developed to estimate the cost-effectiveness and budget impact of emicizumab prophylaxis in HA patients with inhibitors. The model was populated using treatment efficacy from clinical trials and key clinical, cost and epidemiological data retrieved through an extensive literature review. Compared with BPAs prophylaxis, emicizumab prophylaxis was found to be more effective (0.94 quality adjusted life-years) and cost saving (-€19.4/-€24.4 million per patient lifetime) in a cohort of 4-year-old patients with HA and inhibitors who failed immune tolerance induction. In the probabilistic sensitivity analysis, emicizumab prophylaxis had always 100% probability of being cost-effective at any threshold. Further, the use of emicizumab prophylaxis was associated to an overall budget reduction of €45.4 million in the next 3 years. In conclusion, the clinically effective emicizumab prophylaxis can be considered a cost-saving treatment for HA with inhibitor patients. Furthermore, emicizumab treatment is also associated to a significant reduction of the health care budget, making this new treatment a sustainable and convenient health care option for Italian NHS.

[Choice of antithrombotic therapy in patients with Non Valvular Atrial Fibrillation in real life]. / Scelta della terapia antitrombotica in pazienti con fibrillazione atriale non valvolare nella reale pratica clinica.

INTRODUCTION: Non-valvular atrial fibrillation (NVAF) is the most common cardiac arrhythmia and it is associated with a 5-fold increase in risk of ischemic stroke. Although clinical guidelines recommend antithrombotic therapy for stroke prevention in patients at moderate or high risk for stroke, little is known on the extent to which the increase of the risks influence the choice of the therapy. AIM: The aim of the study was to assess the level of adherence to the guidelines for the prevention of thromboembolic risk in patients with NVAF. METHODS: A population-based cohort study was conducted using administrative data from a local health authority in the Campania Region (~1,000,000 inhabitants). NVAF was defined as one or more claims for atrial fibrillation between July, 2013 and June, 2014 where none of the claims were associated with cardioversion or cardiac ablation during the identification period and there was no evidence of valve-related diagnoses or procedures. The cohort was classified according to the first drug dispensing during 6 months from the discharge date for atrial fibrillation. Patients were categorized in low ischemic stroke risk (LR, score = 0), moderate-risk (MR, score = 1), high-risk (HR, score≥2) according to the CHA2DS2-VASc score. Multivariable logistic regression was used to evaluate the associations between ischemic stroke risk with the choice of non-vitamin K antagonist oral anticoagulants (NOACs) versus vitamin K antagonists (VKAs) therapy. RESULTS: A total of 1963 patients were identified: 4.9% LR, 7.6% MR and 87.5% HR patients. Overall, 36.4% of patients were not treated (LR: 56.7%, MR: 55.0%, HR: 33.7%patients). The Vitamin K antagonists were prescribed to 17% of the patients (LR: 10.3%, MR: 12,1%, HR: 17,8%), NOAC to 12,7% (LR: 10,3%, MR: 8,1%, HR: 13,2%), low-dose aspirin to 17.5% (LR: 13,4%, MR: 15,4%, HR: 17,9%), other antiplatelet to 12,3% (LR: 7,2%, MR: 6,0%, HR: 13,2%). The ischemic stroke was not significantly associated with the choice of anticoagulant drug. CONCLUSIONS: High proportion of NVAF patients with CHA2DS2-VASc score of 2 or greater not received oral anticoagulant as recommended. In contrast with recent guidelines, aspirin was commonly prescribed even in HR patients. The stroke risk stratification did not influence the choice of anticoagulant drug.

[Health Technology Assessment to evaluate the economic advantage of offering of low-protein diet to pre-dialysis patients in Campania (Italy)]. / Un modello HTA a sostegno dell'offerta della dieta ipoproteica ai nefropatici della Campania.

A low-protein diet is well known to slow the progression of chronic renal failure, delay initiation of dialysis, while achieving significant economic benefits. In the context of a Health Technology Assessment (HTA), a budget impact analysis model was implemented to evaluate the economic advantage of offering of low-protein diet to nephropathic patients in Campania (Italy). The implemented model takes into account only the direct costs to the national healthcare system. In particular, costs related to supplying low-protein foods are compared to dialysis costs avoided, in a scenario that evaluates different indices of Numbers Needed to Treat and compliance to treatment. Results indicate that when compliance to treatment is at least 50% and NNT is £ 50, supplying a low-protein diet to all kidney disease patients in the pre-dialysis phase, namely with an estimated Glomerular filtration rate > 45, in Campania (which in the year 2009 were equal to 25,000 subjects), is economically advantageous. In this perspective, the authors argue that distribution of low-protein foods by local pharmacies could be an appropriate choice as it would allow the products to be offered at a discounted price and create a favorable setting for increasing adherence to treatment.