Global and Regional Health Technology Assessment

Cost of illness of Heart Valve Diseases (HVDs): A real-world analysis in Italy

Paolo Sciattella, Matteo Scortichini — Wed, 19 Feb 2025 00:00:00 +0000

Introduction: Heart valve diseases constitute a significant public health challenge with substantial clinical and economic implications. The ageing population, coupled with advancements in surgical techniques, has led to increased hospitalizations for cardiac valve interventions over the past decade. Despite progress, the economic burden of heart valve diseases remains underexplored, particularly in the context of the Italian healthcare system. This study aims to comprehensively assess the economic burden of heart valve diseases in Italy through a real-world analysis.

Methods: Using data from the national Italian Hospital Discharge Records (SDO), we identified the number of adult patients undergoing interventions on the aortic, mitral, and tricuspid valves, along with the associated costs borne by the National Health System. Additionally, we estimated indirect costs by analyzing social security benefit applications related to valve diseases from 2016 to 2019.

Results: A notable rise in hospitalizations for cardiac valve interventions, particularly for the aortic (+53.3%) and mitral valves (+29.5%) was revealed through the study period. In-hospital mortality rates declined, reflecting advancements in perioperative care. The economic burden of heart valve diseases in Italy was substantial, with hospitalization costs reaching €808 million in 2018. Social security benefit applications also increased, adding an annual cost of €29 million.

Conclusions: Heart valve diseases represent a growing clinical and socio-economic challenge in Italy. This study underscores the imperative for a multidisciplinary approach to effectively manage these conditions. Emphasizing prevention, early diagnosis, and timely interventions is crucial to mitigate the economic impact and enhance the quality of life for patients.

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

Wed, 29 Jan 2025 00:00:00 +0000

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). Monthly probabilities of RSV infections, health events, mortality, and complications associated with RSV infections were obtained from the literature. Costs associated with each event were obtained using the available literature and through real-world data analysis of National Hospital Discharge Records.

Results: For one RSV season, in the current SoC, the model estimated 216,100 RSV-MA-LRTIs, 15,121 associated complications, and 16 RSV-deaths–corresponding to an economic burden of approximately €50.5 million related to RSV-MA-LRTIs management, €10.9 million associated with potential complications due to RSV and €3 million in lost productivity due to RSV-deaths. Nirsevimab is expected to prevent 100,208 RSV-MA-LRTIs, 6,969 complications, and 6 deaths due to RSV infections, corresponding to an economic saving of about €23.3, €5, and €1.2 million, respectively.

Conclusion: Nirsevimab is a new prophylaxis strategy that helps to protect all infants against RSV disease and could substantially reduce the clinical and economic burden of RSV in Italy in infants experiencing their first RSV season.

Value contribution of leniolisib in the Treatment of Activated PI3Kδ syndrome (APDS) in Spain using Multi-Criteria Decision Analysis (MCDA)

Mon, 27 Jan 2025 00:00:00 +0000

Background: Activated phosphoinositide 3-kinase (PI3K) δ Syndrome (APDS) is an ultra-rare, potentially life-threatening disease that lacks approved treatments in Spain. This study aimed to apply Multi-Criteria Decision Analysis (MCDA) to assess the value of the first pharmacological treatment for APDS in Spain.

Methods: A multidisciplinary group of 8 experts evaluated the selective PI3Kδ inhibitor leniolisib against Standard of Care (SoC). An MCDA framework tailored for Orphan Drugs (ODs), consisting of 5 comparative and 2 quantitative non-comparative criteria, was used. Re-scoring followed a group discussion.

Results: Leniolisib scored higher than SoC in all criteria, including efficacy and safety. It was deemed highly valuable as the first disease-modifying treatment, with a positive therapeutic impact and potential to improve patients’ quality of life. Additionally, leniolisib may lead to cost savings. The supporting data was considered of high quality.

Conclusion: Based on MCDA methodology and stakeholder experience in APDS management, leniolisib is seen as a value-added treatment option compared to SoC in Spain.

Towards a Green Health Technology Assessment: embedding Life Cycle Assessment for sustainable choices

Michela Bobini, Eugenio Di Brino, Americo Cicchetti — Fri, 14 Feb 2025 00:00:00 +0000

The healthcare sector significantly contributes to global greenhouse gas emissions. Among the various strategies available, exploring the integration of environmental sustainability into Health Technology Assessment (HTA) presents a potential avenue for addressing these impacts. The HTA Core Model, widely utilized by European HTA agencies, evaluates healthcare technologies across nine domains; however, environmental considerations remain peripheral and are primarily confined to certain safety-related aspects. This paper examines the potential role of Life Cycle Assessment (LCA) in complementing HTA to better address environmental impacts. LCA offers a systematic methodology to evaluate environmental effects across the full lifecycle of a product, from raw material extraction to disposal. Through the analysis of pharmaceuticals, telemedicine, and surgical practices, the study identifies critical environmental impacts at various lifecycle stages, illustrating how LCA could support more informed and sustainable decision-making in healthcare. These findings underscore the diverse environmental impacts associated with healthcare technologies and highlight the need for tailored strategies to mitigate them. This point of view emphasizes the importance of initiating discussions on developing a framework to incorporate environmental impacts into HTA systematically, promoting healthcare decisions that prioritize both human and environmental healths.

What is needed to successfully implement the EU HTA Regulation enabling broad patient access in Europe

Thu, 16 Jan 2025 00:00:00 +0000

There has been a lot of discussion on the technical aspects of the soon to be implemented European Union’s Health Technology Assessment (EU HTA) regulation. However, there has been limited discussion on the implementation aspects and the potential limitations from a policy perspective. In May 2024, a group of HTA experts with previous policy responsibilities met in Rome to propose some policy aspects to be considered.

As a result of the discussion, several proposals were made. Building mutual trust, improving collaboration and engaging all relevant stakeholders seems a must. Equally important are the communication aspects, and ensuring equal commitment by all parties, allocating the appropriate incentives at all levels. Finally, it is noted that the EU HTA regulation has to be seen from the perspective of a wider policy change within the large EU legal framework.

The length of price&reimbursement negotiation procedures in Italy in the period 2021-2023 was independent from the price of medicines

Raffaella Cocciolo, Paola Turella, Daniela Pilunni, Pierluigi Navarra — Wed, 15 Jan 2025 00:00:00 +0000

Background: The duration of price&reimbursement (P&R) procedures is taken as a key performance indicator of Regulatory Agencies in Europe. In this study we have tested the hypothesis that the price of medicines may have negatively affected the duration of P&R negotiations in Italy in the period 2021-2023.

Methods: Data were retrieved from the Intexo database, which included all medicines with EC approval within the period 2021-2023. Biosimilars, generics, gene therapies, Sars-Cov-2 vaccines and medicines with no negotiated price at the datalock of December 2023 were excluded from the analysis. The ex-factory price of the most expensive package was taken as the price of each reimbursed medicine, whereas the time between the publication of EC decision in the European GU and the publication of P&R AIFA decision in the Italian GU (gazette-to-gazette time) as well as the time between the submission of P&R dossier and the publication of AIFA decision in the Italian GU (AIFA negotiation time) were taken as the procedure duration.

Results: Sixty-seven EC approved medicines completed the P&R procedure in Italy within the study period, and had a reimbursed price. The median and mean gazette-to-gazette times were 395 and 442.6 days, respectively, whereas the mean and median AIFA negotiation times were 389.5 and 434.7 days, respectively. The mean price was 10,942 euros. The analysis of correlation between the duration of procedure and the negotiated price showed that there is no correlation whatsoever between the length of procedures and the price of medicines.

Conclusions: We can reasonably exclude that the price of medicines negatively affected the duration of P&R negotiation procedures.