Global & Regional Health Technology Assessment

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ISSN / EISSN : 2284-2403 / 2283-5733
Published by: Aboutscience Srl (10.33393)
Total articles ≅ 214
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Jaime Rodríguez Quintosa, Cintia Cristina Ago, Antoni Sicras Mainar, Renata Villoro, Inés Pérez-Román
Global & Regional Health Technology Assessment, Volume 9, pp 133-137;

Background: Impetigo is a common dermatological paediatric infection that can be treated with topical antibiotics: the common are mupirocin (MUP), fusidic acid (FA) and, most recently, ozenoxacin (OZ). Aim: This study assesses the clinical and economic consequences of the use of OZ vs. MUP and vs. FA for the treatment of impetigo in routine clinical practice in Spain. Methods: This is a retrospective observational study using real-life data from electronic medical records of patients with impetigo who started treatment with OZ, MUP or FA (maximum follow-up: 3 months; n = 10,974). We compared treatment duration, comorbidities, use of systemic medication, complications, utilization of resources and associated costs across treatments (p<0.05). Cost-effectiveness of OZ was assessed from a social perspective. Complication rates and treatment duration were the effectiveness measures. Results: Mean age was 12.6 (standard deviation [SD]: 16.6) years; 48.6% were male; treatment: 9.3% (OZ), 56.4% (MUP), 34.5% (FA). The percentage of patients ending treatment after 2 weeks was 87.6% (OZ) vs. 83.2% (MUP) vs. 82.4% (FA); p<0.001; complication rates were 1.8% (OZ), 3.3% (MUP) and 3.2% (FA), p<0.001; mean costs were €158 (OZ), €265 (MUP) and €287 (FA), p<0.001. Conclusions: OZ is a cost-effective and dominant alternative for the treatment of impetigo.
, Marta Mosca, Roberto Ravasio, Pietro Brambilla, Paola Raimondo,
Global & Regional Health Technology Assessment, Volume 9, pp 123-132;

Background: Systemic lupus erythematosus (SLE) is associated with clinical burden for the patient and organ damage. The development of therapies for SLE has been constrained by clinical and biologic heterogeneity. These represent challenges in clinical trial design and endpoint selection. Objective: To identify the most relevant descriptors for efficacy, endpoints, disease activity, organ damage, quality of life (QoL), and Patient Reported Outcome Measures (PROMs) in the treatment of SLE. Methods: A Delphi study was conducted using a national expert panel of clinicians in the treatment of SLE. A steering committee composed of 3 opinion leaders with deep expertise in SLE treatment was defined. The steering committee analyzed and appraised the evidence, designed the Delphi study, defined the statements, and analyzed the expert panel responses. A 2-round Delphi survey was conducted. Participants were asked to rate the statements using a five-point Likert scale. Results: Nine experts participated in the Delphi survey. After the two rounds, the consensus was reached on 18 of the 23 statements: 2 statements were included in the “efficacy” domain, 2 in the “glucocorticoid-sparing” domain, 2 in the “endpoint evaluation” domain, 4 in the “score” domain, 1 in the “disease activity” domain, 1 in the “organ damage” domain, 1 in the “QoL” domain, 2 in the “PROMs” domain, 1 in the “AIFA monitoring” domain and 2 in the “extra” domain. No statements reached consensus within the “onset” domain. Conclusion: In this Delphi study, 18 statements across 11 domains were agreed upon for the treatment of SLE.
Chiara Bini, Martina Paoletti, , , Francesco Saverio Mennini
Global & Regional Health Technology Assessment, Volume 9, pp 105-116;

Objective. To assess the cost-effectiveness and economic sustainability of treosulfan plus fludarabine compared with busulfan plus fludarabine as a conditioning treatment for malignant disease prior to allogeneic haematopoietic stem cell transplantation (alloHSCT) in adult patients in Italy. Method. The two theoretical cohorts of patients aged ≥ 60 years with acute myeloid leukaemia (AML) and myelodysplastic syndrome (MDS) were pooled and followed over time using a partitioned survival model with cycles of 28 days. Patients can transition between a post-HSCT recovery/remission state (Event-Free Survival state, EFS state), a relapsed/progressed disease state, and a death state. A lifetime horizon for cost-effectiveness analysis and a 5-years’ time horizon for budget impact analysis were used. The perspective of the Italian National Health Service was adopted. Utility values were obtained from published sources. Costs included: drug acquisition, HSCT procedure, management and treatment of adverse reactions, graft-versus-host disease (GvHD) and health states, end of life treatment. Discounting of 3% per year was applied for both costs and outcomes according to Italian guidelines. Sensitivity was tested through both one-way and probabilistic analyses. Results. Cost-effectiveness analysis showed that treosulfan is both more effective and less expensive compared with busulfan (+1.11 life-years, +0.96 quality-adjusted life-years per patient and -€ 41,784 per patient). On the side of economic sustainability, the introduction of treosulfan in the market could generate a cumulative decrement of the expense incurred by NHS of about -€ 179,174 over five years. Conclusion. Treosulfan could represent a cost-effective and sustainable treatment alternative from the perspective of the NHS.
Francesca Patarnello, Emiliano Briante,
Global & Regional Health Technology Assessment, Volume 9, pp 117-122;

Pharmaceutical industry investments in Italy must be supported with a reform process that promotes simplifications to generate an attractive ecosystem that can enhance innovation. Key actions include facilitating the start-up of clinical trials, promoting public-private partnerships to support technology transfer, integrating data infrastructures to overcome the logic of silos, expanding programs for prevention and early identification of diseases, simultaneous reimbursement for therapy and diagnostic testing, and launching a structural program for early access to therapies. The total contribution of the sector in the last 10 years to the Italian economy was € 315 bn. Looking at employment (67 thousand people employed in the sector in 2020), it is possible to estimate about 280 thousand jobs activated in Italy just in 2020. An Altems study quantified a leverage effect of 2.77 in terms of benefits to the Italian NHS from direct investment in clinical research, which means that for every € 1 invested by companies in clinical trials, € 1.77 of additional savings were generated for the NHS. Applying the multiplier on R&D investments of the entire pharmaceutical sector in 2020 (€ 1.6 bn), we could estimate approximately € 4.4 bn in benefits for the NHS. In addition to these benefits there are the noneconomic benefits of clinical research, including early access for patients to innovative therapies, resulting in improved clinical outcomes and quality of life for patients and caregivers in general.
, , Giovanna Scroccaro, Valentina Acciai, Antonio Addis, Andrea Ardizzoni, , Alberto Bortolami, Alessia Brigido, Giuliano Buzzetti, et al.
Published: 28 September 2022
Global & Regional Health Technology Assessment, Volume 9, pp 99-104;

This document illustrates the results of a discussion of two multi-disciplinary expert panels on pricing and reimbursement of medicines. Experts work in different organizations. The discussion focused on comparator(s), endpoint(s), negotiation of prices of new medicines and/or indications to include in the List 648, as well as the role of cost-effectiveness in the price and reimbursement negotiation. The debate took place during the fourth edition of the Seminari di Mogliano, organized on the 30th of September/1st of October, 2021. The two panels agreed on a general need to enhance interaction among the different stakeholders, in the early assessment and negotiation phases, and to increase the transparency/reproducibility of the decisions taken. The experts have also emphasized the need (i) to improve clarity in the evaluation of additional therapeutic value and the place in therapy with respect to comparators and how comparators are identified; (ii) to create work groups to identify the most appropriate endpoint(s), for each therapeutic area and level of unmet needs; (iii) to provide for a systematic use of cost-effectiveness when an added therapeutic value is delivered by a new medicine. With regard to the 648 List, the experts advocated for an overall reorganization of the current rules governing the special uses of drugs.
Chiara Bini, , , Martina Paoletti,
Published: 19 September 2022
Global & Regional Health Technology Assessment, Volume 9, pp 91-98;

Objective: To estimate the economic impact of the introduction of a new care system based on apomorphine and Patient Support Program for motor fluctuations (“on-off” phenomena) in patients with Parkinson’s disease which are not sufficiently controlled by oral anti-Parkinson medication in Italy. Method: A Budget Impact model was developed to evaluate the new care system in patients with Parkinson’s disease over a 3-years’ time horizon. The comparator treatments included in the analysis were treatments based on apomorphine and levodopa + carbidopa. The analysis was conducted from a National Health Service (NHS) perspective. Costs included in the analysis were acquisition costs and device costs. A deterministic sensitivity analysis was carried out to evaluate the uncertainty of the parameters used. A break-even analysis was conducted to identify the minimum number of subjects that would need to be treated with the new care system to obtain a positive Budget Impact (World With – World Without = 0). Results: The analysis shows that the introduction of the new care system based on apomorphine could generate a cost saving incurred by the NHS of over € 5.7 million in 3 years. Break-even analysis shows that if it were possible to intercept with the new treatment at least 9 patients treated with apomorphine, there would not be an increase in costs for the NHS. Conclusion: The new care system would respond to the unmet needs of patients with Parkinson’s disease by generating a reduction in the expenditure incurred by NHS.
Margarita Majem, , Ana Laura Ortega, Lucía Ruiz de Alda, Rocío Gordo, J Francisco García, Yoana Ivanova-Markova, , Raquel Sánchez San Cristóbal,
Published: 12 September 2022
Global & Regional Health Technology Assessment, Volume 9, pp 82-90;

Introduction: In recent years, target therapies to specific molecular alterations in advanced non–small cell lung cancer (NSCLC) have been identified and have shown superior efficacy compared to non-targeted treatments. Anaplastic lymphoma kinase (ALK) is one of the therapeutic targets; nevertheless, ALK diagnosis is not performed in all NSCLC patients in Spain. The objective of this study is to estimate in monetary terms the benefit for the Spanish society of ALK diagnosis in advanced NSCLC patients. Methods: A cost-benefit analysis of ALK diagnosis vs. non-diagnosis in advanced NSCLC patients was carried out from the Spanish social perspective, with a time horizon of 5 years. Costs, benefits and the cost-benefit ratio weremeasured. The analysis has considered the overall survival in advanced NSCLC patients treated with the ALKtyrosine kinase inhibitor (TKI) alectinib. The natural history of NSCLC was simulated using a Markov model. A 3% discount rate was applied to both costs and benefits. The result was tested using a deterministic sensitivity analysis. Results: The cost of ALK diagnosis vs. non-diagnosis in the base case would be €10.19 million, generating benefits of €11.71 million. The cost-benefit ratio would be €1.15. In the sensitivity analysis, the cost-benefit ratio could range from €0.89 to €2.10. Conclusions: The results justify the universal application of ALK diagnosis in advanced NSCLC, which generates a benefit for Spanish society that outweighs its costs and allows optimal treatment with targeted therapies for these patients.
Arianna Bertolani, Roberto Ravasio, Paola Raimondo, Claudio Jommi
Global & Regional Health Technology Assessment, Volume 9, pp 73-81;

Background. Several health care services are required to get eligibility to PCSK9-inhibitors medicines and the follow-up of patients being treated. The ultimate goal is making prescriptions appropriate and monitoring the effects of these drugs. Some recent papers (opinion / consensus documents) highlighted the necessity to make simpler this clinical pathway. Our paper illustrates the cost of this pathway incurred by patients (direct healthcare and non-healthcare costs, productivity losses by patients and their possible care-giver due to the time dedicated to healthcare services). Methods. The study relied on a retrospective data collection through a structured questionnaire administered to 240 patients, being on treatments with PCSK9-inhibiotrd drugs for at least one year. Patients were recruited in 4 Italian healthcare centres from June 2020 to July 2021. Results. Recruited patients are 64 years old on average. 64% of patients are males and 36% are actively employed and working. Mean cost incurred by patients amounts to € 926,1. Direct healthcare costs, direct non-healthcare costs and productivity losses equal to € 463,5 (50%), € 136,7 (15%) and € 325,9 (35%) respectively. Healthcare services fully covered by the National Health Service account for 56% of the total. Co-payments are applied to 26% of healthcare services, whereas patients pay the full price for 18% healthcare services. Discussion. Getting eligibility to PCSK9-inhibitors and managing patients’ follow-up generate important costs incurred by patients. Furthermore, these costs are very different across healthcare centres. We are fully aware that appropriateness of prescriptions and patients’ follow-up are very important. However, simplifying the clinical pathway would bring economic advantages and could make more homogenous the way this pathway is managed by healthcare centres.
Angela La Neve, Giovanni Falcicchio
Global & Regional Health Technology Assessment, Volume 9, pp 4-9;

Epilepsy is a diffuse chronic neurological disease affecting around 50 million people worldwide. The diagnostic criteria by the International League against Epilepsy must be fulfilled to diagnose the disease, which is characterized by brief and transient episodes of abnormal neuronal activity involving one or both hemispheres, depending on the epilepsy type. The diagnosis of epilepsy should be properly and timely made because patients suffering from the disease are affected not only by seizure recurrence but also by epilepsy-related psychiatric and/or cognitive comorbidities that may have a huge impact with severe professional and social implications. It is of vital importance to define a specific governance model that has to be virtuously applied into the different phases of the clinical pathway of the patients with epilepsy in order to guarantee them the best model of care possible.
Global & Regional Health Technology Assessment, Volume 9, pp 14-19;

Anti-seizure medications (ASMs) represent the pillar of the treatment of epilepsy. The rate of drug-resistant epilepsy remained substantially unchanged over time and there is still the need for new and more effective treatment options. Brivaracetam, cenobamate, eslicarbazepine acetate, lacosamide and perampanel are ‘third-generation’ ASMs. The aim of this article is to summarize the currently available evidence about the relative efficacy and tolerability of the ‘third-generation’ ASMs as adjunctive treatment of focal-onset seizures in adults. So far, no randomized controlled study directly compared these ASMs, and their comparative efficacy and tolerability have been indirectly evaluated by one network meta-analysis. Sixteen trials were included in the network meta-analysis. The efficacy endpoints were the rates of seizure response and seizure freedom, defined as ≥ 50% and 100% reduction in baseline monthly seizure frequency. The tolerability endpoints were the rate of patients who developed any treatment emergent adverse events (TEAEs) and any TEAE leading to drug discontinuation. Cenobamate had the greatest likelihood of being the best option for the ≥ 50% and 100% seizure frequency reduction. Brivaracetam and lacosamide had the greatest likelihood to rank as the best-tolerated treatments for the occurrence of any TEAE and TEAE leading to discontinuation. Although network meta-analyses are not substitutes of direct comparisons, they can provide valuable evidence about the hierarchy of interventions. Additional real-world data can be useful complement to characterize the clinical profile and therapeutic potentialities of third-generation ASMs.
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