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Objectives: Using the case study of patisiran and inotersen, we conducted a narrative comparative analysis of the health technology assessment (HTA) agency appraisals of these two first-in-class transthyretin gene silencers, which represent exceptional advances in the treatment of hereditary transthyretin-mediated (hATTR) amyloidosis, a rare and multisystemic disease. Despite the impact of each product on the treatment landscape, the majority of HTAs are only considered standard of care as a comparator, resulting in a void of information and limited comprehension of the clinical and pharmacoeconomic differences between the two treatments. Methods: A search was conducted internationally for HTA reports, and only instances where assessment decisions for both treatments were publicly available were included in the present analysis. The HTA reports were analyzed broadly for the assessment of clinical and pharmacoeconomic evidence. Only economic models considering both patisiran and inotersen were included in this analysis. Results: A total of nine agencies with public assessment reports for both treatments were identified. HTA agency assessments for both treatments were essentially positive; however, differences were noted in the final recommendations, place in treatment or reimbursed indications, and in the narrative of the evaluations. Only the Canadian Agency for Drugs and Technologies in Health (CADTH) assessment for patisiran evaluated an economic model comparing the two treatments. Conclusions: The differences summarized in this comparative analysis may provide a more comprehensive overview of the two treatments.

IntroductionBevacizumab is a monoclonal antibody, which, in association with combination chemotherapy regimens, has been shown to be active in metastatic colorectal cancer. Other biologic agents active in the same setting are cetuximab and panitumumab, both of which are monoclonal antibodies directed against the antiepidermal growth factor receptor. The objective of this study was to compare treatment costs of first-line regimens for metastatic colorectal cancer in Italy.MethodsA set of first-line regimens was considered, according to the Italian Association of Medical Oncology and the European Society for Medical Oncology guidelines. A targeted review of the literature was undertaken to identify clinical study references for treatment regimens. The total cost of a regimen was calculated in the perspective of the Italian healthcare system summing up drugs, administration, and adverse event costs, based on year 2016 prices and tariffs.ResultsBevacizumab 7.5 mg + capecitabine was the least expensive regimen, with a total cost of €16,754 per patient. When we consider regimens based on FOLFOX, bevacizumab 5 mg + FOLFOX4 was the least expensive (€32,709 per patient), compared to panitumumab + FOLFOX4 (€42,815), cetuximab + FOLFOX4 (€42,725), and cetuximab + FOLFOX (€37,995). If we consider combination regimens based on FOLFIRI, the association of FOLFIRI and bevacizumab was less expensive than regimens that included cetuximab (€28,389 for bevacizumab 5 mg + FOLFIRI and €35,310 for cetuximab + FOLFIRI).ConclusionsFrom the perspective of the Italian health care system, bevacizumab appears to be a convenient option among the first-line regimens for metastatic colorectal cancer. Further study, based on real-world evidence, would be necessary to confirm this result.

Budget impact analysis of empagliflozin in T2D patients with a previous cardiovascular diseaseIntroductionThe EMPA-REG OUTCOME study demonstrated that empagliflozin is effective in reducing cardiovascular morbidity and mortality in T2D patients with high risk linked to a previous cardiovascular condition. The objective of this study is the budget impact analysis of the use of empagliflozin in this population in Italy.MethodsThe analysis was conducted from the cost perspective of the Italian NHS with a 3-year time horizon. The target population, defined as T2D patients with a known cardiovascular disease, was estimated based on epidemiologic and market data. An uptake of empagliflozin growing from 10% to 30% was considered to add up or substitute combinations of metformin, insulin, glitazones and DPP-4. The uptake of 80-100% was considered to substitute sulfonylureas and repaglinide. Costs, including drugs, glycaemia monitoring, management of severe hypoglycaemia events, and cardiovascular and renal events, were estimated based on published literature and current tariffs.ResultsThe target population was estimated in 243,693 in the first year. The incremental cost for drugs (17.6 million Euro) is entirely offset by the savings in glycaemia monitoring (8.1 million Euro), cardiovascular and renal events (8.8 million Euro) and severe hypoglycaemic events (3.1 million Euro), with a net saving of 2.3 million Euro, approximately corresponding to -0.7% of the total expenditure. The cumulative budget impact in the 3 years was 4.9 million Euro (-0.5%).ConclusionsThe progressive adoption of empagliflozin to treat T2D patients with known cardiovascular disease in Italy, would provide significant clinical benefit without entailing additional expense for the Italian NHS.