Italian National Health Service Research Articles

Pricing for Multi-Indication Drugs in the Italian Regulatory Context.

The authorization of new therapeutic indications for drugs already reimbursed by the Italian National Health Service (NHS) represents a matter of importance. This study aims to estimate the additional discount attributed to the extension of indications (EoIs) to explore the potential correlation between spending and negotiated discounts and to find specific factors (determinants) that impact on discount. The study focused on drugs approved in Italy between 2003 and 2017 with at least four therapeutic indications, including the first approved and EoIs, with follow-up extended until 2021 to acquire all the information on the negotiation process that has been completed. Data were obtained from reimbursement and pricing dossiers, and negotiation assessments. Trends in the number of EoIs submitted and the additional discounts negotiated were analyzed, along with the relationship between the negotiated discount and subsequent drug expenditure. Determinants influencing the extent of the negotiated discount were assessed, including drug type, orphan drug designation, innovativeness status, number of EoIs, disease incidence and prevalence, estimated number of patients, revenue projections, availability of therapeutic alternatives, and efficacy outcomes. A Wilcoxon nonparametric test was used to evaluate the associations between determinants and the negotiated additional discount, with a significance level of 0.05. The study identified nine medicines: five of these were used in onco-hematologic therapeutic areas, while the remaining four were immunosuppressants for dermatologic and/or rheumatologic conditions. These nine active substances accounted for 65 approved therapeutic indications, of which 50 were reimbursed by the Italian NHS, including the first indication; the analysis focused only on 40 reimbursed EoIs. The additional discount obtained for EoIs averages approximately 12.5% (95% CI 9.4-16.6%), with a median value of approximately 11%. This latter value was used as the threshold in the analysis of the determinants potentially impacting the negotiated discount amount. Discounts greater than 11% were significantly associated with EoI beyond the fifth and oncology drugs. The additional discount seemed small when compared with the increased spending. AML seems to incur substantial direct economic expenses. Reducing the days of hospitalization can significantly decrease the economic burden of AML in the European Union. Moreover, there is a necessity for studies that comprehensively evaluate the economic implications, particularly concerning total and indirect costs. The study provides valuable insights into the negotiation outcomes for medicines with multiple therapeutic indications, particularly in onco-hematologic and immunosuppressive areas. The analysis revealed that additional discounts for EoIs averaged 12.5%, with a median of 11%, a value used to assess the impact of specific determinants. A discount higher than 11% was statistically correlated with drugs having more than five indications and oncology treatments, showing their influence in negotiations. However, the savings from discounts were modest relative to the increased drug spending as more indications were approved. This suggests an imbalance between the cost control achieved through discounts and the rising expenditure due to expanded drug use.

The Italian Recovery and Resilience Plan (RRP) 2021-2026, the Women, and Their Health

For women, the Italian Recovery and Resilience Plan 2021-2026 has meant the strengthening of important policies for work and maternity. For women’s health, this Plan with the National Health Equity Program 2021-2027 have meant addressing the shortcomings of territorial and gender medicine, the regional gaps in the provision of the Essential Levels of Care of the National Health Service and strengthening the screenings for female cancers. The paper highlights how after the conclusion of these plans, the Italian National Health Service will have to identify a long-term strategy to ensure uniformity of services for women ’s health in all Italian regions.

EE448 The Economic Impact of Polatuzumab Vedotin as First-Line Therapy for High-Risk DLBCL on Subsequent Treatments Within the Italian National Health Service

EE448 The Economic Impact of Polatuzumab Vedotin as First-Line Therapy for High-Risk DLBCL on Subsequent Treatments Within the Italian National Health Service

Policy predictive model for better management of chronic obstructive pulmonary disease: economic-organisational implications in the Italian healthcare system

Background: Chronic obstructive pulmonary disease (COPD) represents a significant public health problem in Italy, with a high prevalence and a substantial economic burden on the National Health Service (NHS). Effective management of the disease is crucial for reducing exacerbations and improving patients’ quality of life. Methods: This study employs a dynamic predictive model to assess the economic impact of implementing three specific health policies aimed at improving COPD management over a five-year horizon. The analysis is conducted from the perspective of the NHS, focusing on the direct costs associated with disease management. Results: The implementation of the proposed policies demonstrated potential significant savings in direct healthcare costs. The first policy, focused on increasing therapeutic adherence, generated an estimated savings of 202 million €. The second, concerning the wider use of triple therapies, led to savings of about 142 million €. Finally, the third policy, aimed at reducing the delay in prescribing appropriate therapies, contributed to an additional saving of over 175 million € in the time horizon considered in the analysis. Overall, these results suggest a substantial improvement opportunity in COPD management, with significant economic benefits for the Italian NHS. Conclusion: The study confirms the importance of targeted policies in the management of COPD, highlighting how strategic interventions can lead to significant resource savings while simultaneously improving patient care. These results provide a solid foundation for further research and serve as a useful guide for decision-makers in planning effective intervention strategies, aimed at optimizing resources and enhancing disease management in the Italian healthcare context.

Decatecholaminization of Septic Shock Patients in Intensive Care Unit: an Economic Assessment in the Italian Setting

INTRODUCTION: Prolonged administration of norepinephrine to critically ill patients can lead to serious adverse events. In this context, the concept of “decatecholaminization” has emerged over the past decade, involving the association of vasopressin with norepinephrine to reduce catecholamines need. Additionally, beta-blockers can help prevent increased heart rate resulting from sepsis treatments. This study presents an economic analysis evaluating the economic implications of the decatecholaminization use in patients with septic shock treated in intensive care unit (ICU) from the Italian National Health Service (NHS) perspective.METHODS: Two analyses were conducted: (1) a patient-level comparison of costs between two real-world cases, one treated with decatecholaminization and one without this approach, and (2) a cohort-level analysis using a pharmacoeconomic model to project cost differences for the Italian National Health Service (NHS) before and after implementing decatecholaminization.RESULTS: In the patient-level analysis, the use of decatecholaminization results in increased pharmacological expenses (+€210), and cost reduction in resource utilization (-€30,412). Similarly, the cohort-level shows higher pharmacological costs (+€192 per patient) and lower cost for other resources (-€1,264 per patient) in the future vs current scenario, resulting in a cumulative cost reduction of -€1,072 per patient. Considering an eligible population of 39,207 patients, decatecholaminization results in a total cost reduction of approximately €42.4 million.CONCLUSION: This analysis supports the economic viability of decatecholaminization as an effective treatment for comprehensive management of septic shock. Further evaluation in real-world settings is needed to validate these findings and optimize clinical application.

A cost-effectiveness analysis of Navina Smart on adult patients affected by neurogenic bowel dysfunction.

The objective of this study is to evaluate the economic impact of the device Navina Smart on patients affected by neurogenic bowel dysfunction and dependent on transanal irrigation within the Italian context. This study employs the perspective of the Italian National Health Service. The analysis was conducted through a Markov model, comparing two scenarios: standard bowel care vs. transanal irrigation. The model operates on a 30-year time period. The results were reported in terms of net monetary benefit. Transanal irrigation therapy was dominant in all scenarios with lower costs and higher effectiveness. The population was assumed to be composed of 1,000 subjects. Setting the willingness to pay at €35,000.00/QALYs (quality-adjusted life years), the analysis yielded a net monetary benefit of €81,087 and cost savings of €66,101 per patient over 30 years. The results of this study substantiate that transanal irrigation therapy treatment employing the Navina Smart device can significantly benefit patients suffering from neurogenic bowel dysfunction by relieving their symptoms. In addition, this therapy offers important cost savings for the Italian National Health Service by reducing resource utilization.

How Do the Determinants of Collaborative Consumption Influence Its Use in Healthcare? A Managerial Perspective.

The primary objective of this investigation is to scrutinize the underlying motivations that may prompt those responsible for health to adopt models of collaborative consumption (CC) as business innovation. Furthermore, the study seeks to assess the congruence of determinants influencing the intention to utilize CC in healthcare, comparing perspectives between responsible for health and digital health consumers. Two studies based on the Theory of Planned Behavior (TPB) have been conducted. Study 1 uses a qualitative approach to analyze the determinants in use CC in healthcare of responsible for health of the Italian's National Health Service. Study 2 uses a quantitative approach to analyze a sample of healthcare consumers, their salient beliefs, digital health literacy, and perceived own health status in determining the intention to use CC in healthcare. Responsible for health recognize both the benefits, like improved efficiency, and the drawbacks, such as digital illiteracy and privacy concerns. Consumer data reveals that attitudes, social norms, perceived control, and digital literacy significantly influence the intention to use CC in healthcare, with education and age being moderating factors, whereas income is not impactful. The research ends with a discussion of these findings and their strategic implications for managing decision support systems in healthcare. The research highlights the need for innovation-based strategies in the health system, proposing a new socio-technical health domain to improve management through a participatory approach. The approach emphasizes business innovation, service quality, and cost-efficiency. Finally, the research addresses the gaps highlighted in CC in healthcare adoption, underscoring public-private collaboration and practical strategies for sustainable success.

Cost effectiveness of difelikefalin for the treatment of patients with chronic kidney disease-associated pruritus undergoing hemodialysis in Italy.

Chronic kidney disease (CKD)-associated pruritus is a condition that strongly impacts CKD patients and is associated with increased morbidity/mortality, and worse health-related quality of life (HRQoL). Difelikefalin is currently the only drug approved in Europe specifically for treating moderate to severe CKD-associated pruritus in patients undergoing hemodialysis. The KALM-1 and KALM-2 trials showed better efficacy of difelikefalin vs placebo and best supportive care. The aim of this study was to investigate the cost-effectiveness of difelikefalin according to the Italian National Health Service (NHS) perspective. A cohort model represented by four health states (No, Mild, Moderate, and Severe pruritus) was adapted to the Italian setting. The model used data from the KALM-1 and -2 trials for efficacy, integrated with other publications for HRQoL estimations. To assess the cost of disease management, a recent Italian publication on CKD-associated pruritus was used and a price of €27 per difelikefalin vial was assumed. The base case analysis over a 15-year time horizon, and an additional 10-year scenario analysis, were established. Additionally, both deterministic univariate analysis and probabilistic multivariate sensitivity analyses were developed. Discount rates of 3% were applied. An acceptability threshold of 40,000 €/quality-adjusted life-year (QALY) was considered. The results show that difelikefalin plus best supportive care is cost-effective vs best supportive care alone, with an incremental cost-effectiveness ratio, in the base case, of €35,823/QALY. Both the scenario and sensitivity analyses confirmed the strength of the results. Difelikefalin was found to be a cost-effective treatment for the Italian NHS. These results support its reimbursement and its inclusion in routine clinical practice.

The impact of lactose intolerance diagnosis: costs, timing, and quality-of-life.

Lactose intolerance (LI) is a very common condition but, despite many efforts of the Italian National Health Service to reduce diagnostic delay of chronic disorders and provide prompt treatments, its early diagnosis remains an unmet need. In addition, the COVID-19 pandemic has exacerbated this problem, due to the conversion of many public health services to COVID centers. This study aims to analyze the diagnostic journey of patients suffering from LI, taking into account the duration of the process, associated costs, and impact on quality of life. Anonymous surveys were designed and distributed to patients with LI as well as physicians involved in LI management. The data were analyzed to determine the time required for a reliable LI diagnosis and average costs. Diagnostic delay of LI proved to be longer than 2years, its impact on quality of life proved to be moderate to high in most of participants, especially in their psychosocial domain, and average costs proved to be high. Further investigations are needed to determine the economic burden of maintaining an asymptomatic status in patients with LI.

Ten years of a neonatal screening program for hemoglobinopathies in Friuli-Venezia Giulia: first regional experience in Italy.

Hemoglobinopathies are the commonest genetic defect worldwide (7% of the world's population has at least one hemoglobin mutation). Although prenatal screening for hemoglobinopathies is not obligatory during pregnancy in Italy, it is offered to women by the Italian National Health Service in the pre-conception phase. The screening of newborns is a valid alternative, and has been adopted in various European countries, albeit in a piecemeal fashion. Neonatal screening has the advantage of providing early diagnosis of a hemoglobinopathy. Here we report the findings from the experience with neonatal screening in Friuli-Venezia Giulia since 2010. The hemoglobinopathy screening project in Friuli-Venezia Giulia, a Region in north Italy, began in November 2010. High-performance liquid chromatography was performed on dried blood spot samples collected by obstetric nurses from neonates within 5-8 days after birth. From 2010 to 2019, 11,956 newborns were screened, and abnormal hemoglobin was found in 519 of them (4.34%): the variants identified included HbS, HbC, HbD, HbE and HbX. More specifically, the HbS variant was observed in 347 (2.9%) newborns and the homozygous pattern was identified in 24 (0.2%) cases. The screening also detected two cases of β-thalassemia major. We report our experience of 10 years of screening newborns for hemoglobinopathies in the Region of Friuli-Venezia Giulia, in which 7.7% of people come from malaria-endemic areas. Increased mobility and migratory flows bringing in hemoglobinopathy carriers from endemic areas have led to an increase in mutations in non-malarial countries, with a current incidence of around 4% in the newborns we tested. This means that hemoglobinopathies can be described as a rare condition. Our data show that incidence rates are comparable to those of other inherited disorders such as phenylketonuria, thereby justifying the inclusion of the test for hemoglobinopathies into screening programs for rare diseases.

Immigrants' clusters and unequal access to healthcare treatments

We focus on caesarean sections (C-sections) to examine access to appropriate medical care for immigrants in the Italian tax-funded universal National Health Service. We use a detailed micro-dataset to analyse whether non-native women receive different treatments compared to natives and whether there are differences between groups of non-natives defined by citizenship. For identification, we control for hospital fixed effects and maternal characteristics, and we compare the different groups by exploiting the clustering of non-natives of different nationalities in different urban areas. We find no significant differences between natives and non-natives in terms of C-sections and inappropriate C-sections. However, we do find significant differences between different groups of immigrants. In addition, we find that linguistic and socio-cultural distances are significant drivers of inequalities among non-native women. As language, habits, traditions, and beliefs can affect communication between the woman and the medical staff in many ways, we interpret our findings in terms of the ability to process and understand information between the two parties. In support of this interpretation, we find evidence of a “segregation effect”: women linguistically and socio-culturally more distant from Italy experience the greatest difficulties in accessing appropriate care when living in urban areas characterized by the presence of large immigrant communities of the same nationality. Moreover, we find that the role of linguistic and socio-cultural barriers is stronger for first-time mothers and women with non-native partners.

4.P. Round table: Advancing genomic diagnostics for rare genetic diseases: a focus on non-clinical domains of HTA

Abstract Genetic rare diseases are a burning global public health issue requiring a thorough assessment of the related burden. Unfortunately, curative therapies are oftentimes unavailable for genetic rare diseases; nevertheless, a prompt diagnosis could allow clinicians to elaborate supportive care plans for patients and their caregivers, determine the best care setting, and potentially consider enrolling them in clinical trials, if suitable. Innovative, effective, and sustainable diagnostic techniques, such as Whole genome sequencing (WGS), are needed to address this growing health concern. At global level, there is a paucity of research that specifically investigates the implementation of WGS into clinical practice which is even more constrained at the European level. Furthermore, given the current challenges impacting pediatric patients suspected of having genetic diseases without a definitive diagnosis, there is an urgent need to improve the diagnostic workflow based on genomic techniques, such as WGS. Delving into this topic could contribute valuable insights that can inform policymakers, at macro-level, and health professionals, at micro-level, on how to develop suited genomic policies and clinical guidelines for the proper adoption of WGS within the daily clinical practice. From an international standpoint, it could be argued that these considerations resonate with the United Nations Sustainable Development Goals (SDGs), particularly SDG 3 (i.e., “Good health and well-being”) and 10 (i.e., “Reduced inequalities”), as a priority for research and action. Notwithstanding, the broader implementation of WGS encounters hindrances owing to its complexity and the multifaceted social, economic, organizational, and ethical implications. In view of these challenges, Health Technology Assessment (HTA), as an integrated and multidisciplinary process, provides a comprehensive framework for the evaluation of these dimensions. This round table is intended to provide attendees with insights into nonclinical domains of HTA of WGS which, unlike clinical domains, demand a nuanced approach within the framework of the HTA. The intended objective is pursued through the initial presentation of a case study on the evaluation of WGS in paediatric genetic disorders from the Italian National Health Service perspectives followed by an open discussion with experts. The discussion will delve into issues related to economics, genomics, health services organization and ethics. Key messages • For pediatric patients suspected of having a genetic disease, WGS could shorten the so-called diagnostic odyssey. • Context-depending aspects of implementing WGS should be properly taken into account through HTA.

Cost-effectiveness analysis of Chagas disease screening in pregnant women and their infants in Italy

Abstract Background Currently, Chagas disease (CD) is widespread in European nations that host notable populations from Latin America. Vertical transmission is a pathway for the spread of the disease in countries where it is not endemic. Due to socio-economic disparities, CD is a public health concern in non-endemic regions, raised by migration phenomena in vulnerable population groups who face similar hindrances as those most at risk in endemic areas. The aim is to evaluate the cost-effectiveness of CD screening versus no screening in pregnant women and their infants in Italy. Methods A Bayesian Markov model was employed to compare the CD screening versus the no screening alternative. Model parameters were sourced from pertinent scientific literature. A lifetime perspective was adopted, aligning with the perspective of the Italian National Health Service. The Eurozone threshold was applied. Costs and benefits were discounted at a rate of 3%. Gibbs sampling served as the algorithm for Bayesian inference. Uncertainty was addressed through a probabilistic sensitivity analysis (PSA) and a value of information analysis (VOI), depicted via the Cost-Effectiveness Acceptability Curve (CEAC) and Expected Value of Perfect Information (EVPI). Results were presented as the Incremental Cost-Effectiveness Ratio (ICER). Results The base case results showed that CD screening was cost-effective, with an ICER of €5,112 [€1,850 - €10,043]. Furthermore, according to the CEAC, the screening option exhibited the greatest probability of being cost-effective across all thresholds. Additionally, the EVPI per mother was €818 based on a threshold of €50,000/diagnosis. Conclusions Implementing a structured CD screening program also in non-endemic countries has the potential for early detection and treatment. This contributes to SDG 3 by improving access to healthcare, preventing transmission, and reducing the disease burden as well as informing public health policies for marginalised populations. Key messages • This study proves that screening for CD in pregnant women and their infants in a non-endemic area is a cost-effective strategy and possibly cost-saving in the long term. • Study findings could support a wider implementation of CD screening in Italy by informing health policy-makers in their decision-making process.

Clinical characteristics, use and switch of drugs for obstructive airway diseases among patients with COPD experiencing an exacerbation: a retrospective analysis of Italian administrative healthcare data

BackgroundChronic obstructive pulmonary disease (COPD) represents an important health challenge, despite being preventable and manageable thanks to up-to-date recommendations. In Italy, the pharmaceutical care of COPD patients is still ill-timed and inaccurate. This study aimed to describe the treatment of COPD patients in Italy and possible switches following an exacerbation.MethodsThis observational retrospective analysis of Italian administrative healthcare data from the Fondazione Ricerca e Salute (ReS) database identified patients aged ≥ 45 years with COPD in 2019 and 2020. At least 6 years of look-back period and absence of concomitant asthma were required. COPD patients were categorized by treatment (SI–single/MI–multiple inhalers, TT–triple therapy, DT–dual therapy, other respiratory treatments, untreated) at index date (first dispensation during accrual period). Occurrence of moderate/severe exacerbation during one-year preceding index date and treatments during one-year preceding the exacerbation (possible switch) were evaluated.ResultsFrom ~ 4.7 million beneficiaries of the Italian National Health Service in 2019 and 2020, respectively, 105,828 and 103,729 (43 and 41 × 1,000 inhabitants aged ≥ 45 years) were identified as having COPD. Of 2019/2020 patients: 3.4%/5.2% received SI-TT, 20.7%/17.5% MI-TT, 35.9%/38.1% DT, 33.0%/33.1% other treatments, and 7.0%/6.0% were untreated. Males were prevalent and median age was > 73 years for all groups. Of 2019/2020 cohorts, heart failure and coronary artery disease affected 24/20%, 18/17%, and 11%/16% patients with SI-TT, MI-TT, DT, and other treatments, respectively. A previous moderate/severe exacerbation (2019/2020 patients) occurred to 60.5%/56.6%, 39.9%/37.4%, 30.8%/29.2% and 31.9%/29.7% patients treated with SI-TT, MI-TT, DT, and other treatments, respectively. Of 2019/2020 patients experiencing moderate/severe exacerbation: 6.0%/7.0% receiving DT, 5.1%/7.0% receiving other treatments and 4.5%/10.0% untreated, switched to SI-TT; 23.7%/16.9% receiving DT, 21.4%/17.7% receiving other treatments and 15.4%/12.0% untreated, switched to MI-TT.ConclusionsCOPD patients receiving TT were older and had more comorbidities, especially cardiovascular diseases, than patients receiving DT or other treatments. The limited number of patients switching after exacerbation suggests that many COPD patients may be inappropriately treated. Ensuring early and adequate treatment, combination of in-hospital and outpatient management, and integration of specialist and primary care is pivotal for the appropriate clinical management of COPD patients.

Outcomes and prognostic factors in 3306 patients with relapsed/refractory chronic lymphocytic leukemia treated with ibrutinib outside of clinical trials: A nationwide study.

We performed a cohort study that included all patients with relapsed/refractory chronic lymphocytic leukemia (R/R CLL) who received ibrutinib in the Italian National Health Service. With a median follow-up of 42.2 months (IQR 30.8-54.6 months), the study involved 3306 patients with a median age of 72.1 years, of whom 42.6% had received ≥2 previous lines of treatment. The estimated 24-month probabilities of being on treatment and alive were 57.9% (95% confidence interval [CI]: 59.6-56.2) and 76.6% (95% CI: 75.2-78.1), respectively. The median time to treatment discontinuation (TTD) was 31.3 months (95% CI: 29.5-33.5). Out of 3306 patients, 2015 (60.9%) discontinued treatment, with 993 cases attributed to death or disease progression (30.0% of all cases). Among the 1022 patients who discontinued treatment for reasons other than progression or death, 564 (17.1%) patients did so due to toxicity or medical decision, while 458 patients (13.8%) were lost to follow-up. Multivariable analysis revealed that age, Eastern Cooperative Oncology Group Performance Status, the number of previous lines of therapy, refractoriness to the last treatment, and reduced renal function were associated with shorter TTD and overall survival (OS). The coexistence of 17p- and TP53 mutations had an independent unfavorable impact on TTD and OS. Nonstandard doses were associated with shorter TTD and advanced stage with shorter OS. The median OS postprogression and postdiscontinuation for other reasons were estimated at 12.9 (95% CI: 11.3-16.2) and 22.7 months (95% CI: 20.2-28.3), respectively. This large real-world study shows that ibrutinib is an effective treatment for R/R CLL. Baseline patient characteristics and double-hit TP53 aberrations were associated with inferior prognosis, and discontinuation due to CLL progression portended a poor outcome.

Cost-Effectiveness of Influenza Vaccination in Healthy Children: A 10-Year Population-Based Study

Background/Objectives: Seasonal influenza annually puts a significant burden on the pediatric population, especially the youngest, causing severe illness and death. Additionally, associated healthcare costs cause a significant financial strain on healthcare systems. While vaccination is the most effective prevention method, its cost-effectiveness for healthy children remains unassessed. Methods: Using the Pedianet database spanning from 2009 to 2019, we analyzed influenza cases among 6-month-olds to 14-year-olds in Italy. Data included influenza-related medical visits, prescriptions, exams, emergency visits, hospitalizations, and costs. Adverse events and quality-adjusted life years (QALYs) were considered from the existing literature. A static decision-tree model compared annual vaccination strategies, assessing probabilities for influenza or influenza-like illnesses by vaccination status. Incremental cost-effectiveness ratios (ICERs) were calculated, along with sensitivity analyses and cost-effectiveness acceptability curve generation. Results: Mean total influenza costs for vaccinated children averaged EUR 18.6 (range 0–3175.9, including EUR 15.79 for the influenza vaccination), whereas costs for unvaccinated children were consistently lower at around EUR 4.6 (range 0–3250.1). The average ICER for years where vaccine and virus strains are matched was EUR 29,831 per QALY, which is below the EUR 40,000 threshold set by the Italian National Health Services. The ICER values range from EUR 13,736 (2017/2018) to EUR 72,153 (2013/2014). Averted influenza costs averaged EUR 23 per case, with fluctuations over the years. In most observed years, influenza vaccination was cost-effective from the healthcare providers’ standpoint. The exception was 2009–2010, due to a mismatch between vaccine and virus strains. Conclusions: This study highlights the economic viability of influenza vaccination, especially when virus and vaccine strains align. It demonstrates the potential of vaccination programs in preserving children’s health and well-being while managing healthcare costs.

The care pathway of patients with diffuse large B-cell lymphoma described through Italian administrative healthcare data.

The diffuse large B-cell lymphoma (Dlbcl) is the most common non-Hodgkin lymphoma and at highest incidence among the elderly. Despite the improved outcomes of patients treated with the first-line (1L) standard of care until the end of 2022, composed by rituximab and polychemotherapy (R-Chop), during the last 20 years, the rate of relapsed and refractory Dlbcl (rrDlbcl) remains elevated. This study has identified and analyzed patients newly diagnosed with Dlbcl and treated with 1L, from the perspective of the Italian National Health Service (Ssn). From the administrative database of Fondazione Ricerca e Salute (ReS) including ~5.5 million inhabitants/year in Italy, adults with a new in-hospital Dlbcl diagnosis (index date) and treated with 1L in 2018, 2019, 2020 and 2021 were identified and characterized in terms of demographics and comorbidities during a period (from 4 to 8 years) preceding index date. From 1 to 4 years following index date (follow-up), overall survival (Kaplan-Meier curves), percentage distribution of patients by line of therapy including dispensation/administration of chemo-immunotherapy, hemopoietic stem cell transplantation (Hsct), and direct healthcare costs charge to the Ssn, were evaluated. Overall, from the ReS database, 206 patients newly diagnosed with Dlbcl and treated with 1L from 2018 to 2021 in Italy (incidence from 0.9 to 1.7 x100,000 adult inhabitants) were identified. They were mainly older (median age 68 [56; 75] years), males (56%) and affected by ≥2 comorbidities (52%), mostly cardiometabolic. During 4 years of follow-up, 56% of cases in 2018 survived. During the first follow-up year: 73%, 80%, 100% and 35% of cases in 2018, 2019, 2020 and 2021, respectively, received a 2L; 42% and 64% of cases in 2018 and 2020, respectively, received a 3L. At least one Hsct was found as a 2L among cases in 2018, 2020 and 2021. On average, each patient newly diagnosed with Dlbcl and treated with 1L from 2018 to 2021 caused a total expenditure directly charged to the Ssn ranging from € 20,000 to € 30,000 during the first follow-up year (chemo-immunotherapy accounted for 40-53%), which reduced with time in favor of other drugs and Hsct. This analysis confirms the high rate of rrDlbcl and the high economic impact charged to the SSN to support first the chemo-immunotherapy, then the chronic care and the absence of standardized further lines of therapy for patients with rrDlbcl.

Patterns and determinants of patient sharing in end-of-life health services: Empirical evidence from the Italian National Health Service

ABSTRACT End-of-life (EoL) care is critical for cancer patients, who tend to have high service needs that are dispersed across organizations and different levels of care. Although EOL has been widely studied, little is known about the patterns of coordination among EoL health care providers and how they contribute to the care of cancer patients. This study adopts a network perspective to examine the complex patterns of patient sharing among health care providers involved in EoL care, using data on the use of EoL health care services by 266 cancer patients in a large Local Health Authority in Italy. We conducted a social network analysis of the structural properties of the emerging network and used logistic regression-quadratic assignment procedures (LR-QAP) to explore how characteristics of health care providers, their collaborative network, and their distances predict the likelihood of observing patient sharing relationships. Our results show that complementarities in terms of medical specialization and co-location of services positively predict the likelihood of cancer patient sharing. This probability is also positively related to the difference in terms of eigenvector centrality as well as the degree of network transitivity. We discuss the policy implications of our findings.

Cost-Effectiveness Analysis of Newborn Screening for Spinal Muscular Atrophy in Italy.

A decision-analytic model assessed the cost effectiveness of newborn screening from the National Health Service perspective in 400,000 newborns. Newborn screening enabling early identification and presymptomatic treatment of SMA was compared with no newborn screening, symptomatic diagnosis, and treatment. Transition probabilities between health states were estimated from clinical trial data. Higher-functioning health states were associated with increased survival, higher utility values, and lower costs. Long-term survival and utilities were extrapolated from scientific literature. Health care costs were collected from official Italian sources. A lifetime time horizon was applied, and costs and outcomes were discounted at an annual rate of 3%. Deterministic and probabilistic sensitivity analyses were conducted. Newborn screening followed by presymptomatic treatment yielded 324 incremental life-years, 390 incremental quality-adjusted life-years, and reduced costs by €1,513,375 over a lifetime time horizon compared with no newborn screening. Thus, newborn screening was less costly and more effective than no newborn screening. Newborn screening has a 100% probability of being cost effective, assuming a willingness-to-pay threshold of >€40,000. Newborn screening followed by presymptomatic SMA treatment is cost effective from the Italian National Health Service perspective.

Developing Physiotherapy in Primary Health Care: A First Snapshot from the Italian Metropolitan City of Milan.

Since the COVID-19 pandemic, the Italian National Health Service (NHS) has been undergoing a structural reform shifting focus from hospital-centered care to smaller, intermediate, or primary health facilities closer to the community (e.g., community hospitals and community houses). This reorganization should include rehabilitation and physiotherapy, but the actual spread of these services is still unclear. This study explored the number and characteristics of community-based physiotherapy services in the Metropolitan City of Milan (Italy). Between April and May 2024, we distributed a structured, anonymous online survey about community physiotherapy services and users to all Directors of the Health and Social Care Professions Departments (DAPSS) in the Metropolitan City of Milan. We used descriptive statistics to analyze the number of community houses offering physiotherapy services, the specific intervention areas, and access modalities. Six out of seven DAPSS Directors completed the survey (87%). Thirty-seven community houses were reported in the area, with fourteen of these offering physiotherapy services. In most of them, physiotherapy was a primary reason for access following a general practitioner's prescription. Five out of six responders reported that rehabilitation needs were mainly assessed by specialists in Physical and Rehabilitation Medicine, with physiotherapists involved in the assessment process in two cases. Physiotherapists primarily handled the intervention phase, dealing mainly with orthopedic and neurological conditions. DAPSS Directors noted that additional physiotherapy initiatives focusing on prevention will be implemented. Physiotherapy services are becoming available in the Metropolitan City of Milan. However, more efforts are needed to facilitate access and ensure tailored assessment and effective interventions, particularly in preventive care. Future investigations should help to better define the number and the characteristics of the patients who can most benefit from this type of care, the number of sessions they need, and with what types of intervention; it would be also necessary to better define the communication network in the area that allows doctors, health professionals, and patients to be informed about this possibility.