Italian National Health Service Research Articles

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.

Italian healthcare resource consumptions and direct costs of adults with atopic dermatitis before and after dupilumab treatment.

Atopic dermatitis (AD) is a heterogeneous disease, associated with comorbidities, and high healthcare consumptions and costs. This study assessed the burden before and after treatment with dupilumab in adults with severe AD from 2018 to 2020, from the perspective of the Italian National Health Service (SSN). From Fondazione Ricerca e Salute's administrative healthcare database (~5 million inhabitants/year), adults treated with dupilumab from 09/01/2018 to 31/12/2020 (index date) and a five-year lookback were identified. Age, sex and comorbidities at baseline, concomitant drugs, overnight hospitalizations, outpatient specialist services and direct costs charged to the SSN one year before/after index date were assessed. Of 337 adults treated with dupilumab (5.8x100,000 adult inhabitants/2019; 8.0x100,000/2020; 55% males; mean age 43±19), 68% (228/337) had ≥12-month follow-up available. Asthma was a common comorbidity (23% patients). Rates of patients treated with nearly all concomitant AD-related therapies reduced from 12 months before to 12 months after dupilumab treatment: antibacterials (from 59% to 50%), systemic corticosteroids (55% to 29%), antihistamines (54% to 38%) and cyclosporine (52% to 7%). A similar trend was observed among patients with asthma as comorbidity. Within 12 months before/after dupilumab, patients hospitalized halved from 14% to 7%, and patients receiving outpatient specialist care reduced from 72% to 65%. Annual mean direct total costs per patient treated with dupilumab charged to the SSN, net of dupilumab cost, were €1384 and €773, before and after dupilumab dispensation, respectively. Before dupilumab, observed patients had higher healthcare resource consumptions and direct SSN costs than after dupilumab.

Health and Economic Outcomes of Pembrolizumab in the Treatment of Metastatic Non-small Cell Lung Cancer (mNSCLC) and Melanoma in Italy.

In Italy, the management of metastatic non-small cell lung cancer and melanoma leads to significant healthcare challenges, necessitating cost-effective treatment strategies and offering valuable insights for healthcare policymakers and stakeholders. This study was designed to assess the costs, quality-adjusted life-years (QALYs) and disability-adjusted life-years (DALYs) associated with the health and economic outcomes of (1) pembrolizumab-combined chemotherapy administered as a first-line treatment for metastatic non-squamous and squamous non-small cell lung cancer (NSCLC) where the tumour presents with a programmed death-ligand 1 expression level <50% and of (2) adjuvant pembrolizumab treatment for stage III melanoma. Three cost-effectiveness models developed by MSD were investigated for each treatment indication. A unique model was built to assess the overall effect of pembrolizumab versus chemotherapy or watchful waiting in patients with lung cancer or melanoma, respectively. Theoretical cohorts of patients with metastatic squamous and non-squamous NSCLC were followed over time using a partitioned survival model with weekly cycles. A weekly cycle Markov model was employed for melanoma. The analysis was conducted from the Italian National Health Service perspective, considering a time horizon of 40 years (lifetime). A single closed cohort of treatable patients was followed over time for each indication (4000, 7000 and 900 for NSCLC squamous, non-squamous and melanoma, respectively). The costs evaluated included those for adverse drug events, non-drug disease management, subsequent treatment and terminal care. Drug acquisition and administration costs were excluded. For each treatment indication assessed, pembrolizumab produced downstream direct cost offsets (- €122,498,568, -€133,369,076 and -€32,993,242 for NSCLC squamous, non-squamous and melanoma indications, respectively), increased quality of life (+2088, +5317 and +2307 QALYs for NSCLC squamous, non-squamous and melanoma indications, respectively) and reduced disability (-2658, -7202 and -3029 DALYs for NSCLC squamous, non-squamous and melanoma indications, respectively). Across indications, the total cost offsets of pembrolizumab were -€288,860,885, with 9712 QALYs gained and 12,889 DALYs avoided. The analysis demonstrated that, compared with chemotherapy, pembrolizumab is more cost effective in Italy as a first-line treatment in patients with metastatic squamous or non-squamous NSCLC and, if compared with watchful waiting, as adjuvant treatment in patients with stage III melanoma. The present analysis suggested that pembrolizumab use could lead to important health benefits for patients while offsetting a portion of cancer care costs.

Chronic Kidney Disease Eligible for SGLT2 Inhibitors Through the Integration of Italian Administrative and Primary Care Data.

Background. Patients with chronic kidney disease (CKD) can be successfully treated with sodium-glucose cotransporter-2 inhibitors (SGLT2-Is), regardless of diabetes. Fondazione Ricerca e Salute's (ReSD) administrative and Health Search's (HSD) primary care databases were combined in the Database Consortium ReS-HS to quantify and describe patients with CKD potentially eligible for SGLT2-Is and assess costs charged to the Italian National Health Service (SSN). Methods. Patients aged ≥18 with CKD and estimated glomerular filtration rate (eGFR) <60 ml/min in 2018, without dialysis and/or renal transplantation, were included. HSD was used to develop and validate algorithms for estimating eGFR, based on covariates, within the ReSD. Comorbidities, dispensed drugs, and direct healthcare costs were assessed. Results. In 2018, 66,297 (5.0% of HSD population) and 211,494 (4.4% of ReSD population) patients with CKD potentially eligible for SGLT2-Is were identified (females ≥58%). Prevalence increased with age with a peak at 75-84 years. Within HSD and ReSD cohorts, respectively: 31.0% and 41.5% had diabetes; in the observation periods, >82% and >96% received ≥1 pharmacological treatment, of which ≥50% and ≥25% received cardiovascular/blood agents and antidiabetics, respectively. From ReSD, mean per capita direct SSN cost was € 3,825 (CI 95%, € 3,655-€ 4,000): 50.1% due to hospitalizations, and 40.2% to pharmaceuticals (31.6% to cardiovascular drugs and 10.1% to antidiabetics). Conclusion. The Database Consortium ReS-HS methodology found 5% of adult SSN beneficiaries with CKD potentially eligible for SGLT2-Is bringing with them a high cardio-metabolic burden which increases the risk of CKD progression.

The emergence and organizational choices of patient advocacy associations: Evidence from the Italian context.

Within many advanced healthcare systems, patient advocacy associations are gaining a more relevant role in healthcare policy decisions. These associations provide help, representation, and voice to patients and citizens. However, although their role is important, a paucity of research exists analyzing their foundation dynamics, including their organizational decision, from a managerial perspective. To fill this knowledge gap, we formulated two research questions to investigate the motivations underlying the foundation of patient advocacy associations and the institutional pressures influencing the changes that have occurred in these associations throughout their evolution. Using a semi-structured questionnaire developed as part of a larger research project, we collected data about 31 patient advocacy associations operating within the Italian National Health Service. We employed qualitative analysis to examine the associations' birth and evolution as well as the motivations and influences driving change within the sampled organizations. Questionnaire responses provided information about the patient advocacy associations' histories and highlighted their close links with the Italian National Health Service. Our results indicated the motivations for the associations' establishment could be grouped into five categories: support at the system level, patient protection and support, actions to increase awareness, actions for improving patients' health and quality of life, and dissemination of knowledge. We also identified the frequency and nature of the changes within the associations and explored the institutional pressures that influenced these organizational changes. The results can be interpreted considering the organizational theories that focus on organizations' establishment decisions and how external pressures impact organizations' dynamics and evolution.

Early access programs for medicines: a reform proposal for the Italian National Health Service

The present paper illustrates a reform proposal on early access for medicines. In this proposal early access stands for patient’s access, outside of clinical trials and covered by the Italian National Health Service (Servizio Sanitario Nazionale, SSN), to a new medicine or an indication of an already approved medicine before the approval o in between the approval and the decision on price and reimbursement status. The proposal emerged from a multi-stakeholder working group within the Sixth Edition of the “Seminari di Mogliano Veneto”. The reform proposal is aimed at converting the current early access programs (mainly, the programs introduced with Laws 648/96 and 326/03) into a single program inspired by the one introduced in France in 2021. The proposal provides indications on eligibility criteria for drugs and patients, application procedure, evaluation process, economic safeguard clauses, data collection, issues derived from the circumstance that the drug/indication included into the early access program is not reimbursed afterwards and the actions to implement this program.

A cost-effectiveness analysis of community nurse-led self-care education for heart failure patients

BackgroundCommunity nurses delivering heart failure self-care education improve patient outcomes, but the cost-effectiveness of this type of nurse-led intervention has not been recently established. AimTo determine the cost-effectiveness of community nurses’ self-care education for heart failure patients compared with usual care. MethodsWe performed a cost-effectiveness analysis from the perspective of the Italian National Health Service. A Markov model simulated the progression of a cohort of 1000 heart failure patients receiving remote self-care education after hospital discharge or usual care. Outcomes included costs, quality-adjusted life years, and incremental cost-effectiveness ratio. The willingness-to-pay threshold was established at €40,000/quality-adjusted life years. FindingsOver the 20-year time horizon, community nurses’ care incurred an extra cost of €1.3 million while gaining 247 quality-adjusted life years compared with usual care, and the incremental cost-effectiveness ratio was €5490/quality-adjusted life years. ConclusionsThe involvement of community nurses in self-care education is a potential cost-effective way of delivering home self-care education to heart failure patients.

Cost-Effectiveness of Vedolizumab as a First-Line Advanced Therapy Versus Adalimumab Treatment Sequences for Ulcerative Colitis in Italy.

Today, there are many treatment options available for the management of ulcerative colitis, creating challenges in selecting the most efficacious and cost-effective treatment sequences. Treatments in the anti-tumor necrosis factor alpha (TNFα) therapeutic class, as well as vedolizumab, are widely used and endorsed as first-line options according to treatment guidelines. The aim of this study was to compare treatment sequences involving vedolizumab and the anti-TNFα treatment adalimumab in terms of cost-effectiveness in the treatment of moderately to severely active ulcerative colitis in Italy. A cost-effectiveness model comparing treatment sequences within the Italian National Health Service in terms of costs and quality-adjusted life years (QALYs) with a lifetime horizon was developed. The analysis focused on the relative positioning of vedolizumab and adalimumab, leveraging the results of the landmark head-to-head VARSITY clinical trial as key inputs. The robustness of the results was investigated through a range of sensitivity and scenario analyses. The strategy of vedolizumab as a first-line advanced therapy followed by adalimumab was associated with higher costs and health benefits compared with first-line adalimumab followed by vedolizumab. The incremental cost-effectiveness ratio was €16,146/QALY, which was found to be robust to changes to inputs associated with areas of high uncertainty. This economic evaluation estimated a 94% probability that vedolizumab as a first-line advanced therapy is cost-effective at a threshold of €33,004/QALY when compared with first-line adalimumab sequences. Using clinical trial evidence to inform the efficacy of second-line treatments estimated that the effectiveness of anti-TNFα treatments is not substantially reduced by vedolizumab exposure.

A new organizational model of primary healthcare in Liguria, Italy. Insights and implications.

After years of cost-containment policies, the Italian National Health Service (NHS) has now the chance to change and improve, especially thanks to the National Recovery and Resilience Plan (NRRP). The plan serves as a catalyst for reform, allocating substantial funds to reinforce proximity networks, facilities, and telemedicine for territorial healthcare. Mission 6, specifically dedicated to health, focuses on integrating primary healthcare, hospital, and specialty care networks, underscoring the importance of a robust primary healthcare system. In alignment with NRRP objectives, the Ligurian model introduces innovative structures, such as Community Houses (CdCs), Community Hospitals (OdCs), and Territorial Operation Centres (COTs). These interconnected components form a dynamic network designed to enhance healthcare accessibility, prevent inappropriate hospital admissions, and facilitate efficient patient transitions. The model prioritizes multidisciplinary collaboration, community engagement, and the integration of socio-healthcare services. Despite substantial NRRP funding for infrastructure, challenges related to staffing and human resources persist. The social and epidemiological context highlights concern about the economic feasibility of the reform, potential workforce shortages, and the imperative for updated regulatory frameworks. The strategic reallocation of personnel from acute hospitals to new facilities is crucial, requiring meticulous workforce planning, role definitions, and trainingIn conclusion, the Ligurian model emerges as a proactive response to the structural vulnerabilities exposed by the pandemic, aligning with international trends in emphasizing primary care, prevention, and community-based services.

Patients with metastatic cholangiocarcinoma in Italy and their healthcare resource consumptions and related costs.

e16139 Background: Patients with cholangiocarcinoma (CCA) are often diagnosed at late-stage disease. Few or any therapeutic options are possible for older patients with metastases (mCCA) and comorbidities. This analysis aimed to identify and characterize newly mCCA diagnosed patients in Italy in terms of clinical features, healthcare resource consumptions and related costs charged to the Italian National Health Service (SSN). Methods: From the Fondazione ReS (Ricerca e Salute) database (~5 million inhabitants/year), among adults with primary/secondary in-hospital diagnosis of CCA in 2018 and analysable look-back period until 2013, patients newly diagnosed with lymph nodes/distant metastases (index date) were selected. They were characterized by age, sex, and comorbidities (cardiometabolic, respiratory and cerebrovascular disorders, depression, chronic kidney disease and inflammatory bowel diseases). Rate of antineoplastic treatment dispensations and hepatic metastasectomy, overall and progression-free survivals - through Kaplan-Meier curves - among patients treated (vs untreated), and healthcare costs charged to the SSN were assessed during 2 years following index date. Results: In 2018, 176 newly mCCA diagnosed patients (incidence: 4.4x100,000 adult inhabitants; 31% of CCA patients) were identified. Males were 52.3%. Mean age was 72±10 years, and 65% of patients had ≥2 comorbidities. During 1 year following index date, 45.5% of newly mCCA diagnosed patients underwent antineoplastic treatment and/or surgery (mean age was 69±9 years), of whom, 61% were males, 67% had ≥2 comorbidities. During 2 years following index date: 34 patients were lost from 1st to 2nd year; 77% of treated patients died (vs 93.8% untreated; p &lt; 0.01); 87.5% of treated patients relapsed, on average after 6.3 months following index date. On average, during 1st and 2nd follow-up year, respectively: each mCCA patient weighed on the SSN € 12,699 and € 8,298; hospitalizations accounted 87% and 78% of the respective total expenditure. From 1st to 2nd follow-up year, respectively, the expenditure for hospitalizations and pharmaceuticals decreased from € 11,017 to € 6,498 and from € 700 to € 590, while costs for local outpatient services increased from € 982 to € 1,210. Conclusions: This study suggests that newly mCCA diagnosed patients strongly impact on the SSN, and that antineoplastic treatments and/or surgery, if applicable, can reduce mortality with substantial survival benefit.

A real-world picture of patients with a new diagnosis of acute myeloid leukemia in Italy.

6520 Background: Acute myeloid leukemia (AML) is a blood malignancy with poor prognosis. Antineoplastic strategies are still mostly restricted to fit patients. This analysis of administrative healthcare data identified and described newly AML diagnosed patients, their antineoplastic treatment and survival from the perspective of the Italian National Health Service (SSN). Methods: From the Fondazione ReS (Ricerca e Salute) database (~5 million inhabitants/year), among adults with primary/secondary in-hospital diagnosis of AML in 2018 (index date) and analysable look-back period until 2013, patients newly AML diagnosed were selected. They were characterized by age, sex and comorbidities (cardiometabolic, thyroid and cerebrovascular disorders, chronic kidney and liver disease, and inflammatory bowel diseases). During 1 year following index date, antineoplastic treatment and allogeneic stem cell transplantation (alloSCT) were assessed. Among patients undergoing antineoplastic therapy, those fit and unfit for intensive chemotherapy (ICHT) were categorized according to proxies based on the setting of chemotherapy administration (i.e., overnight hospitalization=fit, while daily hospitalization or local outpatient ambulatory=unfit). The 2-year overall survival (OS) of treated patients was assessed through a Kaplan Meyer analysis. Results: In 2018, 228 newly AML diagnosed patients (incidence: 5.7x100,000 adult inhabitants) were identified. Males were 56.6%. Mean age was 69±16 years, and 61% patients had ≥2 comorbidities. During 1 year following index date, 75% of newly AML diagnosed patients received antineoplastic treatment (males were 57%, mean age was 66±15 years, and 49% had ≥2 comorbidities). Of them, 19% underwent alloSCT (mean age was 50±14 years). On average, remaining untreated patients (57/228; 25%) were older (77±14 years) and affected by more comorbidities (79% with ≥2 comorbidities) than treated patients. At 2 years following index date, 97% of treated patients survived (vs 65% of untreated patients; p&lt;0.01). Among treated patients, 51% (mean age was 64±15 years) were considered fit for ICHT, and 24% were considered unfit for ICHT (mean age was 71±15 years). At 2 years following index date, 38% of patients fit for ICHT survived (vs 29% of patients unfit for ICHT; p&lt;0.01). Conclusions: This real-word analysis suggests that patients receiving antineoplastic treatments, regardless of a following alloSCT, were younger and affected by less comorbidities than untreated patients. Also, older patients are likely to be excluded from the ICHT option, as recommended by most of guidelines. However, these findings suggest that being treated with chemotherapy and/or alloSCT can increase survival probabilities.

The economic impact of 14 compassionate use programs for medicines in Italy, in the perspective of the National Health Service

Background: Compassionate use programs (CUP) for medicines allow patients, not recruited in trials, accessing medicines before marketing approval. The evidence on the economic impact of CUP is limited to one paper that did not include the possible long-term economic impact of medicines used in CUP compared to alternative treatments, like avoided hospitalization costs. This paper aims at covering this information gap, updating and integrating the existing evidence, with a cost analysis in the perspective of the Italian National Health Service (SSN). Methods: Data were retrieved from the Roche CUP Database, the peer-reviewed literature (e.g. published cost-effectiveness studies, if any), the gray literature (e.g. regional documents for the alternative treatment cost) and unpublished data (e.g. cost-effectiveness analysis provided for price and reimbursement negotiation for medicines in CUP). Results: We retrieved data from 14 CUPs and 3,485 patients (70% affected by tumors) (Jan 2015-August 2022). The alternative treatment mean cost per patient ranged from € 11.1 k to € 18.8 k. The net economic benefit ranged from € 47 million to € 75 million (€ 13.5 k-€ 21.5 k per patient). Avoiding alternative treatments mostly contributes to the economic benefit. The long-term economic impact accounted for 20/30% of the net economic impact. The net economic benefit for cancer drugs ranged from € 12.6 million to € 40.0 million. Conclusions: Despite research limitations, this paper provides additional evidence on the economic impact of CUP in Italy. This could represent the first step of a broader analysis of the economic impact of early access schemes for drugs.