Global and Regional Health Technology Assessment

Assessing the economic burden of Waldenström’s Macroglobulinemia (WM), Marginal Zone Lymphoma (MZL), and Chronic Lymphocytic Leukemia (CLL)

2025-03-11T15:35:01+00:00

Introduction: In Italy, Non-Hodgkin Lymphomas (NHL), including Waldenström’s Macroglobulinemia (WM), Marginal Zone Lymphoma (MZL), and Chronic Lymphocytic Leukemia (CLL), are among the most common hematologic cancers. These conditions mainly affect the elderly, who often have multiple comorbidities, complicating management and imposing significant burdens on patient quality of life and healthcare systems.

Objective: This study aimed to estimate the economic burden of selected B-cell lymphomas in Italy, providing insights for decision-makers to improve patient management and resource allocation.

Method: Data from the Italian Hospital Discharges Records (SDO) and the National Institute for Social Security were analyzed to estimate direct healthcare costs and social security costs from 2016 to 2019.

Results: A total of 93,712 hospital discharges were recorded, with MZL being the most common diagnosis, followed by CLL and WM. Most patients were male, and the 70–79 age group was most prevalent. MZL had the highest costs, followed by CLL and WM. Adverse events notably increased total expenditures, with variations across different pathologies. Direct healthcare costs totaled 533.6 million euros, while social security costs amounted to 240.9 million euros.

Conclusion: This study highlights the significant economic burden of selected B-cell lymphomas in Italy. Effective management strategies are crucial for reducing costs and optimizing resource allocation in the healthcare system.

Cost per responder of upadacitinib 30 mg and dupilumab 300 mg in patients with moderate-to-severe atopic dermatitis in italy

2025-03-10T15:34:22+00:00

Purpose: The objective of this economic evaluation was to compare the cost per responder between upadacitinib (Upa) 30 mg and dupilumab (Dup) 300 mg in patients with moderate-to-severe atopic dermatitis in Italy.

Methods: A cost per responder analysis was developed based on results of Heads Up, a head-to-head randomized clinical trial. The primary endpoint was EASI75 at week 16, and the secondary endpoints were EASI90, EASI100, and WP-NRS improvement ≥4 points at week 16. The analysis also assessed a fifth exploratory endpoint from a Heads Up post-hoc analysis: EASI90 & WP-NRS 0/1 at week 16. The cost per responder was based on the perspective of the Italian National Health System (I-NHS). Ex-factory prices were used, excluding mandatory and other hidden discounts. The treatment cost was based on the number of administrations at week 16. The cost per responder was adopted as a cost-effectiveness indicator.

Results: Independently of the endpoint evaluated, upadacitinib always had the lower cost per responder than dupilumab. In the base case, the cost-effectiveness of upadacitinib ranges from a minimum of €361.50 considering EASI75 index at week 16 (Upa: €8,839.78 vs. Dup: €9,201.28) to a maximum of €50,376.18 considering EASI100 index at week 16 (Upa: €22,535.2 vs. Dup: €72,911.39). The difference in cost per responder between upadacitinib and dupilumab increased with higher EASI response levels.

Conclusion: Considering five clinical endpoints, the cost per responder for upadacitinib 30 mg was always lower than for dupilumab 300 mg, highlighting its substantial clinical and economic benefits for patients with moderate-to-severe atopic dermatitis.

D.I.Ri.M.O. project: deprescription, inappropriateness evaluation and therapeutic reconciliation in hospital medicine

2025-03-04T08:16:38+00:00

Background: In the Italian healthcare landscape, the management of chronic pathologies is a priority. Often, the elderly patient suffers from several pathologies at once and is commonly on polytherapy: this can easily bring potentially harmful errors in drug therapy. The D.I.Ri.M.O. project took place in an Internal Medicine department and aimed to reduce medication errors and improve the state of health through the Pharmacological Reconciliation procedure.

Methods: From June to October 2022, the team archived therapies for 70 hospitalized patients aged over 65 years and suffering from two or more chronic diseases. For each patient enrolled, the team developed a reconciliation board; afterward, the physician and the pharmacist proceeded to remodulate therapies, especially in those patients with serious interactions.

Results: The team collected 287 drug interactions and then classified them according to the Intercheck Web software classification: 36 class D (very serious), 49 class C (major), 174 class B (moderate), and 28 class A (minor). The modified therapies at discharge were 77.14%. This restriction brought about the removal of unnecessary drugs. After six months, the team observed an improvement in the health conditions of the patients enrolled.

Conclusions: By increasing the patient’s awareness and reducing the number of potentially inappropriate prescriptions, it is possible to improve the effectiveness of therapies. It is also possible to look at a saving policy to make the economic resources better allocated.

Optimization in the HIV treatment: economic and organizational impact analysis of Bictegravir/Emtricitabine/Tenofovir Alafenamide

2025-03-04T08:16:40+00:00

Introduction: Given the availability of a growing number of HIV treatment options, it becomes essential to have a clear understanding of the related economic-organizational evidence, to operate informed and conscious choices. The study aims to define the economic and organizational impact related to a consolidated use of Bictegravir/Emtricitabine/Tenofovir Alafenamide (BIC/FTC/TAF), within the Italian National Healthcare Service (NHS), for the treatment of both naïve and experienced HIV individuals.

Materials and methods: A budget impact analysis was developed assuming the NHS perspective and considering a 36-month time horizon. Scenario A, representative of the current situation of consumption of the different therapeutic alternatives (derived from the most update guidelines), was compared with Scenario B, assuming a greater adoption of BIC/FTC/TAF. An organizational impact analysis was conducted to define any advantages for hospitals, devoted to the management of any ART-related adverse events.

Results: The BIA revealed an economic saving of 0.97% (26,040,271.36 €) given a higher penetration rate for BIC/FTC/TAF, for the treatment of HIV individuals assuming ART in Italy. From an organizational perspective, a greater BIC/FTC/TAF administration would generate a reduction in the overall hospital accesses devoted to the management of adverse events, generating an overall saving of 245,938 hours, considering the time spent by the healthcare professionals involved in the care and treatment of individuals with HIV.

Conclusions: BIC/FTC/TAF represent an interesting possibility for the rapid initiation of ART, as well as for switches, being able to optimize the clinical pathway of a patient with HIV, from an economic and organizational perspective.

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

2025-02-19T10:16:55+00:00

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

2025-01-29T14:08:34+00:00

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)

2025-01-29T14:08:37+00:00

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.

Assessment and value of drugs: report of the focus groups from the XXII National Conference on Pharmaceuticals

2025-03-04T08:16:42+00:00

The XXII National Conference on Pharmaceuticals, held in Catania from 29 February to 1 March 2024, involved the participation of representatives from more than forty pharmaceutical industries, national authorities, academia, clinicians and clinical pharmacists. The 22^nd edition represented a key forum for the analysis of value assessment of medicines, focusing on the impact of new European and National regulations, including the Italian Medicines Agency (AIFA) reform and the HTA regulation. This point of view summarizes insights from focus groups discussions that took place during the Conference, highlighting the pillar role of the new HTA regulation for homogenizing the evaluation across Europe, and the importance to strengthen the collaboration among the parties involved from an early phase, and implementing adaptive and flexible assessment, especially for orphan medicines. The re-evaluation of the innovativeness criteria and framework of the Pricing and Reimbursement (P&R) dossier are points raised among the different focus groups, especially for aligning these tools to the upcoming HTA regulation. The proposals emerged could be useful for AIFA, which is facing a reorganization aimed to optimizing the reimbursement process in Italy.

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

2025-02-19T10:16:57+00:00

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

2025-01-16T12:54:29+00:00

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

2025-01-15T13:20:10+00:00

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.