Background and aim Evidence on determinants of prices for orphan medicines is scarce and not available for Italy. The aim of this paper is to provide an evidence on variables affecting the annual treatment cost of orphan drugs in Italy, testing the hypothesis of a negative correlation with the dimension of the target population and a positive correlation with the added therapeutic value of the drug and the quality of the evidence of pivotal studies. Methods Drugs with a European orphan designation reimbursed in Italy in the last 6 years (2014–2019) were considered. Univariate, cluster analysis and multiple regression models were used to investigate the correlation between the annual treatment cost and, as explanatory variables, the dimension of the target population, the existence of Randomized Clinical Trials as a proxy of the quality of the pivotal studies, the added therapeutic value. Results In the univariate analysis prevalence and added therapeutic value, as expected, have a negative and positive correlation with cost respectively. The correlation with RCT is not significant. In the multivariate model, coefficients for prevalence and added value are confirmed but for the latter are not significant anymore. We also found, through an interaction analysis, that the existence of an RCT has a positive impact on annual treatment cost when the target population is very small. Conclusions Our results suggest that value arguments and sustainability (dimension of the target population and its impact on budget impact) issues are considered for orphan drugs pricing: the role played by sustainability is systematically supported by our results. A more transparent and reproducible price negotiation process for orphan drugs is needed in Italy. This paper has contributed to highlight the implicit drivers of this process.
Based on the principle of health equity, the Italian National Health Service is known worldwide for being a universalistic system that guarantees healthcare services for all its population, among which there are undocumented migrants. A commitment for their health needs is further motivated by their lower utilisation rates of healthcare services, which becomes even more crucial when considering chronic conditions such as diabetes that require adherence and continuity of care. However, the need for more official data has resulted in little research documenting these healthcare usage patterns. For this reason, our objective has been to deepen, from the Italian NHS perspective, the quantity, costs, type, preventability and organisation of healthcare services directed to undocumented migrants. We used official healthcare data from the Lombardy Region, which enable the identification of people receiving the STP code (undocumented migrants) and of people with foreign citizenship (documented migrants). After quantifying the average annual amount and expenditure for healthcare services grouped by Italian citizens, documented migrants and undocumented migrants for all clinical conditions (quantity and costs), we performed three primary investigations where we enlightened differences between the three mentioned groups focusing on the diagnosis of diabetes: (i) mapping the types of healthcare services used and their characteristics (type); (ii) quantifying the impact of preventable hospital admissions (preventability); (iii) examining the healthcare patterns linking pharmaceutical prescriptions with hospital accesses (organisation). Our results reveal significant differences among the three groups, such as more urgent hospital admissions, more preventable complications, and a higher recurrence in terms of access and costs to hospital services rather than pharmaceutical prescriptions for undocumented migrants. These findings can represent the leverage to raise awareness toward the emerging challenges of the migrant health burden.
Background The beginning of the Covid-19 pandemic has forced many hospital departments worldwide to implement telehealth strategies for the first time. Telehealth represents the opportunity to increase value for all stakeholders, including patients and healthcare staff, but its success constitutes a challenge for all of them and particularly patients play a crucial role for their needed adherence. This study focuses on the experience of the Rheumatology Unit of Niguarda Hospital in Milan (Italy), where telehealth projects have been implemented for more than a decade with structured design and organized processes. The case study is paradigmatic because patients have experimented personalized mixes of telehealth channels, including e-mails and phone calls, Patient Reported Outcomes questionnaires, and home delivery of drugs. Given all these peculiarities, we decided to deepen patients’ perspective through three main aspects related to the adoption of telehealth: (i) the benefits perceived, (ii) the willingness to enrol in future projects, (iii) the preference on the service-mix between remote contacts and in-person visits. Most importantly, we investigated differences in the three areas among all patients based on the mix of telehealth channels experienced. Methods We conducted a survey from November 2021 to January 2022, enrolling consecutively patients attending the Rheumatology Unit of Niguarda Hospital in Milan (Italy). Our survey comprised an introductory set of questions related to personal, social, clinical and ICT skills information, followed by the central part on telehealth. All the answers were analysed with descriptive statistics and regression models. Results A complete response was given by 400 patients: 283 (71%) were female, 237 (59%) were 40–64 years old, 213 (53%) of them declared to work, and the disease most represented was Rheumatoid Arthritis (144 patients, 36%). Descriptive statistics and regression results revealed that (i) non-users imagined wide-ranging benefits compared to users; (ii) other things being equal, having had a more intense experience of telehealth increased the odds of accepting to participate to future projects by 3.1 times (95% C.I. 1.04–9.25), compared to non-users; (iii) the more telehealth was experienced, the higher the willingness to substitute in-person with online contacts. Conclusions Our study contributes to enlighten the crucial role played by the telehealth experience in determining patients’ preferences.
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