Pivotal Studies for Drugs About to Be Launched for Rare Diseases: Will They Better Support Health Technology Assessment and Market Access than in the Past?

The designs of clinical trials of drugs for rare diseases are challenged by health technology assessment organisations and payers. Phase II pivotal studies, single-arm or open-label designs, the extensive use of non-final endpoints, and the limited use of patient-reported outcomes (PROs) are the main points of contention. The evidence on the actual design of these trials is limited, but corroborates the concerns of the above.

[Analysis of the reimbursement times of antitumor drugs in Italy in relation to the clinical benefit expressed by the European Society for Medical Oncology (ESMO-MCBS)].

Introduction: Rapid access to safe and effective oncological therapies is crucial. In recent years, many expensive drugs have been commercialized, making it essential to prioritize those with significant clinical benefits. For this purpose, the European Society for Medical Oncology (ESMO) introduced the ESMO-MCBS (Magnitude of Clinical Benefit Scale), which assigns a score to quantify the clinical benefit of treatments.

Estimated impact of nirsevimab prophylaxis on the economic burden of respiratory syncytial virus disease in Northern Italy.

Background: Respiratory syncytial virus (RSV) is a frequent cause of acute lower respiratory tract infection (LRTI) in infants; however, the health and economic burden of infant RSV-associated LRTI in Northern Italy has not been sufficiently assessed. We evaluated the clinical and economic burden of RSV-associated LRTI requiring emergency department (ED) access and/or hospitalization in infants less than 6 months old in two provinces in Northern Italy and estimated the potential impact of universal immunization with a recently approved monoclonal antibody.

Methods: Epidemiological data during the 2021-2022 and 2022-2023 RSV seasons were obtained from administrative records and anonymized to comply with confidentiality requirements.

[Administrative healthcare data to identify and describe patients with rare diseases: the case of Duchenne muscular dystrophy].

Introduction: Duchenne muscular dystrophy (DMD) is a rare disease that causes a progressive loss of muscle function in males, presenting at the age of two years, and involving respiratory and heart function starting from teenage years. This retrospective observational study has identified patients potentially affected by DMD and described their utilization of healthcare resources and direct healthcare costs charged to the Italian National Healthcare Service (INHS).

Methods: From the Foundation Ricerca e Salute (ReS), through a specific algorithm based only on administrative healthcare data of 5.

Cost-effectiveness of cenobamate as a therapeutic alternative for the treatment of focal epilepsy in adults with inadequate seizure control.

Introduction: This study assesses the cost-effectiveness of cenobamate relative to brivaracetam, lacosamide, eslicarbazepine acetate, and perampanel in the management of focal onset seizures (FOS). The objective is to determine whether cenobamate offers enhanced therapeutic benefits and economic viability.

Methods: A comprehensive cost-effectiveness analysis was performed using a lifetime horizon model that encompassed drug acquisition costs, background therapy, monitoring, and seizure management expenses.

[Not Available].

Objective:: Systemic anaplastic large cell lymphoma (sALCL) is a rare and severe non-Hodgkin lymphoma. Despite its clinical relevance, the aetiology of this disease is unknown, not allowing the patient to prevent and counteract it. This study investigates the cost-effectiveness of Brentuximab Vedotin (BV) in combination with cyclophosphamide, doxorubicin and prednisone (CHP) for the front-line treatment of patients with sALCL in Italy.

Burden of disease of Alzheimer disease in Italy: a real-world data analysis.

Background: Alzheimer Disease (AD) represents a growing global health concern with profound socioeconomic implications, with predictions indicating a potential 50% increase in AD cases in Italy over the next 30 years. Timely diagnosis remains challenging due to the slow progression of symptoms and limited accessibility to advanced diagnostic tools, yet it remains one of the few tools available to prevent and alter the clinical course of the disease. The aim of this study is to build a cost-of-illness model to estimate the number of AD patients managed by the National Health Service, analyzing their use of hospital care, and estimating the social costs through real-world data.

Economic and clinical burden associated with respiratory syncytial virus and impact of universal immunization with nirsevimab in Italy.

Objectives: To describe the seasonal respiratory syncytial virus (RSV) burden in Italy considering the current prophylaxis strategy with palivizumab recommended only for high-risk infants (representing only 4.4% of an estimated birth cohort) and to evaluate the potential benefits of a new prophylaxis strategy targeting all infants with nirsevimab.

Methods: A static decision analytic model previously used in the US was adapted to evaluate the RSV-related health and cost outcomes associated with nirsevimab versus standard of care (SoC) for the prevention of RSV medically attended lower respiratory tract infections (RSV-MA-LRTIs).

Long-Term Effects of Direct-Acting Antivirals on Hepatitis C: Trends in Liver Disease-Related Hospitalisations in Italy.

This study aimed to evaluate the effectiveness of direct-acting antivirals (DAAs) on hepatitis C virus (HCV) hospitalisation trends in Italy, the country with not only the highest burden of HCV-related disease but also the highest number of patients treated for chronic HCV infection in Europe. Incident hospital discharge records in Italy from 2012 to 2019 that included a liver cirrhosis diagnosis without mention of alcohol, hepatocellular carcinoma (HCC), HCV and liver cirrhosis without mention of alcohol and/or HCC, cirrhosis with mention of alcohol, as defined by the International Classification of Diseases (ICD-9-CM) were reviewed. An interrupted time series analysis compared the incidence of cirrhosis and HCC before and after the introduction of DAAs (Year 2015).

The economic burden of asthma in Italy: evaluating the potential impact of different treatments in adult patients with severe eosinophilic asthma.

Introduction: Asthma is a prevalent chronic respiratory condition that significantly impacts public health, with severe asthma subtypes, such as severe eosinophilic asthma, imposing substantial socioeconomic burdens.

Methods: Real-world data from the Italian Health Information System were analyzed to evaluate the economic consequences of asthma in Italy. An in-depth comparative analysis was conducted to investigate the economic implications of various asthma subtypes, focusing on severe eosinophilic asthma.

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

Background: 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.

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

Unlabelled: BACKGROUND AND OBJECTIVE: Untreated spinal muscular atrophy (SMA) is the leading genetic cause of death in children younger than 2 years of age. Early detection through newborn screening allows for presymptomatic diagnosis and treatment of SMA. With effective treatments available and reimbursed by the National Health Service, many regions in Italy are implementing newborn screening for SMA.

An Analysis of the Distribution of Direct Cost of Diabetes Care in Selected Districts in Italy.

Introduction: This study aims to define the distribution of direct healthcare costs for people with diabetes treated in two healthcare regions in Italy, based on number of comorbidities and treatment regimen.

Methods: This was a retrospective analysis using data from two local health authority administrative databases (Campania and Umbria) in Italy for the years 2014-2018. Data on hospital care, pharmaceutical and specialist outpatient and laboratory assistance were collected.

Screening strategy to advance HCV elimination in Italy: a cost-consequence analysis.

Background And Aims: Italy has the greatest burden of hepatitis C virus (HCV) infection in Western Europe. The screening strategy represents a crucial prevention tool to achieve HCV elimination in Italy. We evaluated the cost-consequences of different screening strategies for the diagnosis of HCV active infection in the birth cohort 1948-1968 to achieve the HCV elimination goal.

Impact of Chronic Coronary Artery Disease and Revascularization Strategy in Patients with Severe Aortic Stenosis Who Underwent Transcatheter Aortic Valve Implantation.

The prognostic impact of coronary artery disease (CAD) after transcatheter aortic valve implantation (TAVI) is controversial. The aim of this study is to investigate the impact of CAD and different revascularization strategies on clinical outcomes in patients who underwent TAVI with third generation devices. Patients enrolled in the national observational Observational Study of Effectiveness of SAVR-TAVI Procedures for Severe Aortic Stenosis Treatment II study were stratified according to the presence of CAD (CAD+, n = 1,130) versus no CAD (CAD-, n = 1,505), and compared using a propensity matched analysis.