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BACKGROUND: A histopathological and mutational diagnosis has become a priority in the correct choice of the most appropriate cancer therapy for NSCLC. In the absence of a molecular analysis, the therapeutic choice will be directed towards platinum-based chemotherapy, thus preventing, in the presence of a specific mutation, the benefits deriving from the administration of a target therapies (TT).AIM: the present analysis was carried out with the aim of estimating the clinical impact, expressed in terms of progression free survival (PFS), associated with the use of the combined strategy (tissue biopsy and liquid biopsy) or the tissue strategy in the EGFR+ mNSCLC population.METHODS: A pre-existing cost-consequence model was adapted to estimate the annual number of mNSCLC patients with or without the EGFR mutation in order to decide the oncological treatment to be administered in first (1L) or second line (2L). In 1L, against the presence of the EGFR mutation, the administration of a Tyrosine Kinase Inhibitor (TKI), such as osimertinib, gefitinib, erlotinib or afatinib, was considered; in the absence of the EGFR mutation, the administration ofstandard platinum-based chemotherapy was instead considered. With reference to 2L, in the presence of the EGFR T790M mutation, only osimertinib was considered. In the absence of the EGFR T790M mutation, the administration of the standard platinum-based chemotherapy was also considered. The PFS data associated with each of the drugs considered were extrapolated from the respective clinical studies. Key variables were tested in the sensitivity analysis.RESULTS: The adoption of the combined strategy (tissue biopsy and liquid biopsy), by virtue of a greater number of patients treated with TKIs, would make it possible to increase the average PFS in the range of 1.1-3,7 months in the 1L and by 1.4 months in the 2L.CONCLUSION: These results show how the adoption of a correct diagnostic strategy is critical in order to optimize the choice of the therapeutic path in the 1L and 2L of mNSCLC. The addition of the liquid biopsy to the classic diagnostic path (tissue biopsy) would in fact allow to obtain an increase in therapeutic efficacy (average PFS).

Background Chronic kidney disease (CKD) is an important public health problem. Most of the evidence on its costs relates to patients receiving dialysis or kidney transplants, which shows that, in these phases, CKD poses a high burden to payers. Less evidence is available on the costs of the predialytic phase. Objective The aim of this study was to estimate the annual cost of patients with CKD not receiving dialysis treatment, using the Italian healthcare system perspective and a prospective approach. Methods A 3-year observational study (December 2010–September 2014) was carried out to collect data on resource consumption for 864 patients with CKD. Costs were estimated for both patients who completed the follow-up and dropouts. Results The mean annual total (healthcare) cost per patient equalled €2723 (95% confidence interval 2463.0–2983.3). Disease severity (higher CKD stage), multiple comorbidities, dropout status and belonging to the southern region are predictive of higher costs. Pharmaceuticals, hospitalisation, and outpatient services account for 71.5, 18.8 and 9.7% of total healthcare expenditure, respectively. Recent estimates of Italian costs of patients receiving dialysis are nine times the unit costs of CKD for patients estimated in this study. Unit costs at stage 5 CKD (the highest level of severity) equals 4.7 times the costs for patients at stage 1 CKD. Conclusion Despite its limitations, this study provides further evidence on the opportunity to invest in the first phases of CKD to avoid progression and an increase in healthcare costs.

Multiple sclerosis is the most frequent cause of non-traumatic neurologic disability in young adults with over 3400 newly diagnosed cases annually in Italy. The research objective was to assess the cost-effectiveness of alemtuzumab in comparison with other disease-modifying therapies in the management of relapsing-remitting multiple sclerosis, from a payer perspective in Italy. A Markov model was created to assess cost-effectiveness of alemtuzumab in comparison with subcutaneous IFN β-1a, natalizumab and fingolimod. Treatment effects were derived from a network meta-analysis. Economic input included cost of therapies, their administration and follow-up, cost of adverse events and cost of relapse. Data on health care resource utilization and their costs were retrieved from published sources. Cost-effectiveness was measured as incremental cost (€, 2017) per quality-adjusted life year while the robustness of the results was demonstrated in sensitivity analyses. Over a lifetime horizon, alemtuzumab yielded more quality-adjusted life years and less costs compared to the other disease-modifying therapies in all base-case analyses. Treatment with alemtuzumab yielded an incremental quality-adjusted life years of 1.62, 1.03 and 1.36 with savings of €4312, €81,562 and €54,067 versus IFN β-1a, fingolimod and natalizumab, respectively. Results on the multiple cost-effectiveness acceptability curve showed alemtuzumab carries the highest likelihood of being below the accepted willingness-to-pay threshold (€40,000) compared to IFN β-1a, natalizumab and fingolimod. Based on the current analysis, alemtuzumab is likely to be cost-effective versus IFN β-1a, natalizumab and fingolimod in the treatment of relapsing-remitting multiple sclerosis patients in Italy.

This paper scrutinises pipelines for Neglected Diseases (NDs), through freely accessible and at-least-weekly updated trials databases. It updates to 2012 data provided by recent publications, and integrates these analyses with information on location of trials coordinators and patients recruitment status. Additionally, it provides (i) disease-specific information to better understand the rational of investments in NDs, (ii) yearly data, to understand the investment trends. The search identified 650 clinical studies. Leishmaniasis, Arbovirus infection, and Dengue are the top three diseases by number of clinical studies. Disease diffusion risk seems to be the most important driver of the clinical trials target choice, whereas the role played by disease prevalence and unmet need is controversial. Number of trials is stable between 2005 and 2010, with an increase in the last two years. Patient recruitment was completed for most studies (57.6%), and Phases II and III account for 35% and 28% of trials, respectively. The primary purpose of clinical investigations is prevention (49.3%), especially for infectious diseases with mosquitoes and sand flies as the vector, and treatment (43.2%), which is the primary target for parasitic diseases Research centres and public organisations are the most important clinical studies sponsors (58.9%), followed by the pharmaceutical industry (24.1%), foundations and non-governmental organisations (9.3%). Many coordinator centres are located in less affluent countries (43.7%), whereas OECD countries and BRICS account for 34.7% and 17.5% of trials, respectively. Information was partially missing for some parameters. Notwithstanding, and despite its descriptive nature, this research has enhanced the evidence of the literature on pipelines for NDs. Future contributions may further investigate whether trials metrics are consistent with the characteristics of the interested countries and the explicative variables of trials location, target (disease) choice, and the object of the trials., Author Summary Neglected diseases lead to illness, long-term disability and affect economic development in poor populations. There is evidence that clinical research on neglected diseases has increased starting from the second half of the '90s. This paper aims at updating this evidence to 2012 and at integrating available data (groups of target of the clinical projects, phase in the clinical development process, sponsors) with other data that have not been investigated or published so far (recruitment status of patients, and trials location of the trials coordinator). Our study has confirmed previous findings on the important investment in NDs, highlighting, thanks to a disease-specific approach, a particular focus on diseases with a higher diffusion risk, but not necessarily the higher prevalence and the most unmet need. In most studies, patients' recruitment has been completed, and many trials are also in the very terminal phases: this means a high probability that new treatments will be available in the next years. In addition, trial coordinator centres are increasingly located in low income countries; as a consequence, the investment in clinical research has become an opportunity to further enhance clinical and organisational expertise in these countries.