A retrospective observational analysis of the real-world care pathway of people with hereditary transthyretin amyloidosis with polyneuropathy in Italy.

Background: This retrospective observational study described the epidemiology and the burden on the Italian healthcare service (SSN) of patients with polyneuropathy (PN) associated to the hereditary transthyretin amyloidosis (ATTRv).

Research Design And Methods: From the Fondazione ReS (Ricerca e Salute) administrative healthcare database (~5.5 million inhabitants in 2021), patients were identified as having ATTRv-PN in 2021 if they had received treatments for ATTRv-PN under SSN reimbursement (i.

[Not Available].

The role of Real-World Evidence (RWE) concerns the entire drug lifecycle; despite widely recognised in the post-marketing, it is still debated in the pre-marketing, mainly as per the identification and analysis of target populations (TPs) for new drugs or indications. Through administrative healthcare databases, Research and Health Foundation (ReS), in collaboration with experts, develops algorithms to select and analyse TPs. As of March 2024, 85 TPs in 15 clinical areas have been analysed, of which oncology is the most represented.

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.

Background: Chronic 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.

Symptom Burden in Patients on Maintenance Hemodialysis: Prevalence and Severity 17 Years Apart.

: The aim of this study is to compare data from two cohorts separated by a 17-year interval. We assessed the prevalence and severity of symptoms with the "dialysis symptom index" in these two groups, recruited in 2007 and 2024, to determine how advancements in dialysis therapy have influenced the symptom burden's prevalence and severity. : End-stage renal diseases patients receiving maintenance hemodialysis three times a week in the hemodialysis unit of the university hospital were recruited between February and March 2007.

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

Introduction: 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).

The Early Access and the Potential Cost Savings by the Compassionate Use of Onco-haematological Drugs: Results from the Italian Study Compass-O.

Background: Compassionate drug use (CDU) provides early access to not yet authorised medicines and is funded by pharmaceutical companies. The observational retrospective study Compass-O monitored the CDU of onco-haematological drugs, managed by seven Italian units for cytotoxic drug preparations (Unità Farmaci Antiblastici [UFA]), between 1 January, 2016 and 31 December, 2021.

Objective: We aimed to evaluate the CDU of onco-haematological drugs managed by seven Italian UFA, between 2016 and 2021.

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

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). Patients aged ≥18 with CKD and estimated glomerular filtration rate (eGFR) <60 ml/min in 2018, without dialysis and/or renal transplantation, were included.

[Update of the Pdta Net Observatory of the ReS Foundation with all regional Care Pathways approved until 2023.].

Pdta Net, established and managed by Research and Health Foundation (ReS), is a database aimed at gathering and analysing the Regional Care Pathways (CPs) approved in Italy. A comprehensive search was conducted within institutional websites to retrieve all CPs approved by Italian Regions and Autonomous Provinces until December 2023, by utilizing specific keywords. Compared to the previous year, 51 new approvals were recorded.

[The domino effect of drug distribution channels on the management of chronicity.].

Drug distribution channels affect all actors in the drug supply chain: healthcare institutions, healthcare professionals, patients, pharmacies, pharmaceutical companies and distributors. They should be redesigned in line with the new proximity care model planned in the Pnrr and DM 77, while avoiding a negative domino effect on the management of chronic diseases. A guide recently published by the MaCroScopio project is useful to shed light on this issue and to understand the relationship between the distribution channels present in Italy (distribuzione diretta - DD; distribuzione per conto - Dpc; distribuzione convenzionata) and the management of chronic diseases.

Italian healthcare resource consumption for patients on hemodialysis treated for chronic kidney disease-associated pruritus (CKD-aP).

Background: Chronic kidney disease-associated pruritus (CKD-aP) affects patients on hemodialysis. This study identified hemodialysis patients presumably affected or not affected by CKD-aP and integrated healthcare costs, from the perspective of the Italian administrative healthcare data.

Methods: Through cross-linkage of Italian administrative healthcare data collected between 2015 and 2017 (accrual period) in the database of Fondazione ReS (Ricerca e Salute), patients undergoing in-hospital/outpatient hemodialysis were selected.

To retrieve values of albuminuria to define the size of CKD patients eligible to SGLT-2Is: An explorative analysis using a primary care database.

Purpose: To address missingness of albuminuria values, which establish the eligibility to SGLT-2Is for patients with CKD, using the multiple imputation (MI) method.

Methods: We selected patients aged 18 or older and diagnosed with CKD in a primary care database. Those with severe CKD and/or previously treated with SGLT-2Is were excluded.

[The population affected by neovascular age-related macular degeneration and treated with anti-Vegf through Italian administrative healthcare data.].

Introduction: The neo-vascular age-related macular degeneration (nAmd) is a frequent cause of vision loss, although the intravitreal (Ivt) injections of anti-Vegf (vascular endothelial growth factor) have improved functional outcomes. This study has assessed the healthcare and economic burden on the Italian national health service (Inhs) for patients with nAmd and new users of anti-Vegf.

Methods: From the database of Fondazione Ricerca e Salute (ReS), people aged ≥55 and with an in-hospital diagnosis of nAmd and/or an injection of anti-Vegf (aflibercept, ranibizumab, pegaptanib; index date) in 2018 are selected.

To support the use of NT-proBNP to better detect heart failure in patients with type 2 diabetes.

Purpose: Heart failure (HF) is a chronic disease that causes approximately 300,000 and 250,000 deaths per year in Europe and United States, respectively. Type 2 Diabetes Mellitus (T2DM) is one the major risk factors of HF, and the investigation of NT-proBNP might support the early identification of HF in T2DM sufferers. Nevertheless, this parameter is poorly investigated.

A methodology to assess the population size and estimate the needed resources for new licensed medications by combining clinical and administrative databases: The example of glycated haemoglobin in type 2 diabetes.

Purpose: To develop and validate a model to estimate glycated haemoglobin (HbA1c) values in patients with type 2 diabetes mellitus (T2DM) using a clinical data source, with the aim to apply this equation to administrative databases.

Methods: Using a primary care and administrative Italian databases, namely the Health Search database (HSD) and the ReS (Ricerca e Salute) database, we selected all patients aged 18 years or older on 31 December 2018 being diagnosed with T2DM and without prior prescription of sodium-glucose cotransporter-2 (SGLT-2) inhibitors. We included patients prescribed with and adherent to metformin.

Do more targets allow more cancer treatments, or not?

The three current oncology models (histological, agnostic and mutational) mainly differ in clinical, technological and organisational aspects, leading to different regulatory procedures and implications in antineoplastic therapy access by patients. Within the histological and agnostic models, Regulatory Agencies authorise target therapies and define their price, reimbursement, prescription and access based on results from clinical trials including patients affected by the same tumour (histological) or subjects with specific genetic mutations regardless of the tumour site or the histology (agnostic). The mutational model has been developed to identify specific actionable molecular alterations found by next-generation sequencing test-based large platforms on solid and liquid biopsies.

[Community acquired pneumoniae and chronic obstructive pulmonary disease: the RWE analysis of events that required hospitalizations, and healthcare integrated costs.].

Introduction: The community-acquired pneumonia (Cap) and the acute exacerbation of chronic obstructive pulmonary disease (Aecopd) frequently receive wrong therapies, leading to the increase of healthcare consumption resources, direct and indirect costs, and antimicrobial resistance. This study identified Cap and Aecopd hospitalized events, and analyzed them in terms of comorbidities, antibiotic use, re-hospitalizations, diagnostics and costs, from the perspective of the Italian national health service (Inhs).

Methods: From the database of Fondazione Ricerca e Salute (ReS), hospitalizations for Cap and Aecopd from 2016 to 2019.

[Inclusion of vaccination into clinical pathways for COPD and asthma: current challenges and future perspectives in Italy].

Objectives: to describe the importance given to vaccination as a preventive measure in the clinical pathways (CPWs) of patients affected by chronic obstructive pulmonary disease (COPD) and asthma in the Italian regional healthcare services.

Design: a comparative analysis was conducted to assess the presence/absence of vaccination recommendations among the available regional CPWs for the management of COPD and asthma.

Setting And Participants: all the regional CPWs for COPD and asthma available in the "Fondazione ReS" database between 2008 and 2019 have been analysed.

Acute myeloid leukemia: Incidence, transplantation and survival through Italian administrative healthcare data.

Objective: To identify newly diagnosed patients with acute myeloid leukemia in 2017 treated with intensive chemotherapy or unfit for intensive chemotherapy, and to assess their probability of receiving allogeneic stem cell transplantation and survival, from the Italian National Health Service perspective.

Patients And Methods: From the Ricerca e Salute database, adults with an in-hospital diagnosis of acute myeloid leukemia (International Classification of Disease-9th version-Clinical Modification code 205.0x) in 2017 (index date), without any identifying acute myeloid leukemia criteria within the preceding year, were selected.

Safety of drugs used for the treatment of migraine during pregnancy: a narrative review.

Introduction: Migraine is common in females of childbearing age and negatively impacts quality of life. The majority of those with migraine who become pregnant see an improvement in their condition but not all do. Providing evidence-based recommendations for the pharmacological management of migraine in pregnancy is challenging.

[Occurrence of cardiovascular events within a large population of patients affected by rheumatoid arthritis with or without diabetes.].

Introduction: Rheumatoid arthritis (Ra) and diabetes are often associated with chronic multimorbidity and share the high risk of development of major cardiovascular events (Mace). This study aimed to identify and analyse patients with only Ra, Ra + diabetes, and only diabetes, in terms of comorbidities and new occurrence of Cv events, from the perspective of the Italian national health service (Inhs).

Methods: Starting from the Fondazione ricerca e salute (ReS)'s database, through the record linkage of administrative healthcare data, cohorts with only Ra, Ra + diabetes and only diabetes have been selected, characterized (age and sex), and analysed by comorbidity (depression, dyslipidemia, hypertension, hemorrhagic stroke and ischemic stroke/transient ischemic attack - Tia, coronary artery disease - Cad, heart failure - Hf, chronic liver disease, periphery artery disease - Pad, chronic kidney disease, asthma/chronic obstructive pulmonary disease - Copd, neoplasia) and by new Cv events (Hf, Cad and ischemic stroke/Tia) within two follow-up years (Kaplan-Meier curves).

[A view of care pathways approved by Italian Regions, to face the challenge of the community-based healthcare: a quali-quantitative analysis of the Pdta Net database.].

Introduction: The adoption of a Care Pathway (CP) allows the healthcare management of patients suffering from high-epidemiological impact chronic diseases. The continuity of care of these patients is one of the main purposes of the community-based healthcare reform, foreseen in the 6th Mission of the National recovery and resilience plan. Fondazio-ne Ricerca e Salute (ReS) collects and analyses regional CPs approved in Italy, through the Pdta Net database.

How many and who are patients with heart failure eligible to SGLT2 inhibitors? Responses from the combination of administrative healthcare and primary care databases.

Background: Recent successful findings (i.e. DAPA-HF trial) in patients with heart failure (HF) with/without diabetes treated with sodium-glucose co-transporter inhibitors (SGLT2-I) have fostered real-world data analyses.