Global & Regional Health Technology Assessment最新文献

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.

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.

Introduction: The infections of multidrug-resistant organisms (MDROs) associated with duodenoscopes during endoscopic retrograde cholangiopancreatography (ERCP) procedure have become a significant cause for concern, especially in fragile patients. While the clinical impacts of these infections are well-documented, their economic implications remain underexplored. This study assesses the incidence and economic burden of post-ERCP infections in Italy using an administrative database.

Methods: We conducted a retrospective observational study using administrative databases from A.O.U. Città della Salute e della Scienza di Torino. The study included all patients who underwent their first inpatient ERCP between 2018 and 2021. Infections were identified using ICD-9-CM codes for Pseudomonas aeruginosa, Clostridium difficile, Escherichia coli, Klebsiella spp., and Staphylococcus aureus. A 1-year follow-up was defined in order to estimate healthcare resource utilization and related costs, including readmissions, drug prescriptions, and outpatient specialist and laboratory services.

Results: The study included 686 inpatient ERCP cases, an overall infection rate of 4.2% was observed. Higher infection rates were observed in women (4.6%), patients over 70 years old (4.6%), emergency admissions (5.1%), and transplant patients (19.1%). The average hospital stay doubled for infected patients (24.3 vs. 11.3 days; p=0.001). Post-ERCP infections led to a 28% increase in average costs (€12,256 vs. €9,561; p=0.048). With an annual volume of 25,000 ERCP procedures, post-ERCP infections in Italy contribute approximately €2.9 million in additional direct costs per year.

Conclusion: Post-ERCP infections impose substantial financial burdens on the healthcare system, underscoring the critical importance of implementing cost-effective prevention strategies to mitigate this public health threat in Italy.

Objective: A per-patient cost of therapy administration model was developed to estimate the cost of mosunetuzumab vs. tisagenlecleucel in patients with relapsing or refractory follicular lymphoma (R/R FL) receiving two or more lines of systemic therapy (3L+) from both the Italian hospital and societal perspectives.

Methods: A per-patient total cost of therapy administration model was developed to compare the resource consumption of two treatments - mosunetuzumab and tisagenlecleucel. The model considered direct costs such as healthcare labor costs for drug preparation and administration, non-drug consumable costs, and drug purchase. Indirect costs such as patient and caregiver's loss of productivity, transportation, and relocation were also considered. The unit costs and resource use data were retrieved from literature and standard Italian tariffs. To appraise the impact of patients' residency on access-to-care and out-of-pocket expenses, three scenario analyses were conducted.

Results: Over 1 year, mosunetuzumab costs approximately one-fourth of tisagenlecleucel per patient. The base-case scenario showed a hospital cost reduction of €158,870 per patient with mosunetuzumab, increasing to €161,974 when including societal costs. Scenario analyses for the societal perspective estimated cost differences of -€161,170, -€166,507, and -€166,811 for scenarios A, B, and C, respectively. Sensitivity analysis indicated that tisagenlecleucel's price had the greatest impact on cost differences, followed by mosunetuzumab's price.

Conclusions: This analysis identifies mosunetuzumab as an accessible therapeutic option for 3L+ R/R FL patients in Italy. Future research should collect real-time data and evaluate long-term outcomes.

Background and objectives: The objective of this study is to evaluate the economic impact of the device Navina Smart on patients affected by neurogenic bowel dysfunction and dependent on transanal irrigation within the Italian context. This study employs the perspective of the Italian National Health Service.

Methods: The analysis was conducted through a Markov model, comparing two scenarios: standard bowel care vs. transanal irrigation. The model operates on a 30-year time period. The results were reported in terms of net monetary benefit.

Results: Transanal irrigation therapy was dominant in all scenarios with lower costs and higher effectiveness. The population was assumed to be composed of 1,000 subjects. Setting the willingness to pay at €35,000.00/QALYs (quality-adjusted life years), the analysis yielded a net monetary benefit of €81,087 and cost savings of €66,101 per patient over 30 years.

Conclusion: The results of this study substantiate that transanal irrigation therapy treatment employing the Navina Smart device can significantly benefit patients suffering from neurogenic bowel dysfunction by relieving their symptoms. In addition, this therapy offers important cost savings for the Italian National Health Service by reducing resource utilization.

Background: Several data support the efficacy/effectiveness, safety and favorable impact on quality of life of triptorelin treatment in patients with prostate cancer. However, little evidence is available concerning triptorelin use in the long term.

Methods: We analyzed data on triptorelin treatment in patients with prostate cancer in an integrated Italian administrative database, covering around 6 million health-assisted subjects throughout the country. Patients with at least one prescription for triptorelin in the period 2010-2020 and with no evidence of metastasis were included and followed up until 2021. Overall survival (OS) and duration of treatment were analyzed using Kaplan-Meier curves, starting from the date of first prescription.

Results: The cohort included a total of 3,411 patients (mean age: 76.8 ± 8.7 years), of whom 1,326 (38.9%) were treated with triptorelin only and 2,085 (61.1%) with triptorelin combined with an anti-androgen. Overall, 847 (24.8%) patients with prostate cancer died and 1,037 (30.4%) had a treatment switch during the follow-up period, and both the median OS and median duration of treatment were not reached in both groups. The mean annual total cost per patient was estimated as 5,574 €, with almost half of the costs related to medication expenses (2,737 €).

Conclusions: We found a long survival and duration of triptorelin treatment in this population of Italian patients with prostate cancer. This study with a long follow-up period further highlights the usefulness of healthcare utilization databases to integrate results obtained from clinical studies with those from everyday clinical practice.