Hospital-Based Health Technology Assessment (HB-HTA) is a heterogeneous phenomenon constantly evolving to respond to the needs of decision-makers at the hospital level. In 2023, The HB-HTA Interest Group of Health Technology Assessment International (HTAi) surveyed HB-HTA activities with the aim to provide an updated description of the actual scenario.

An online survey was conducted to gather data on the main characteristics of hospitals, HB-HTA activities, outputs, role in the decision-making processes, dissemination and training activities, and their interaction and collaboration with other stakeholders and HTA-related regulations. Finally, the survey collected feedback on the perception of and current barriers to HB-HTA. Three categories of responders were identified: Both hospitals performing and not performing HTA and policymakers.

Eighty-seven responses were collected from twenty-eight countries. Nearly half of the responders (n = 41) conducted HB-HTA, whereas eighteen consisted of hospitals not performing HTA, and twenty-eight were policy makers. HB-HTA was performed mainly in hospitals with >500 beds. HB-HTA units were organized in 40 percent of cases as an “independent group.” The survey showed that HTA units could contribute to all the steps of the decision-making processes, whereas the impact of the assessments on the decisions was mainly perceived as a medium. Furthermore, HB-HTA was not seen as a duplication of effort, even without specific regulations.

The survey highlighted the role of HB-HTA in hospital decision-making supporting the vision of HB-HTA as one of the actors in the HTA ecosystem, the success of which depends on collaboration with other stakeholders.

The widespread adoption of digital health technologies (DHTs) brings new methodological challenges to health technology assessment (HTA). Different tools, guidelines, and methods have been defined by different stakeholders at national/international levels. In this scenario, the real thread for HTA is the lack of coordination and harmonization in the efforts to finalize the definition of an HTA framework for DHTs.

The European Digital Health Technology Assessment (EDiHTA) project aims to provide the first framework co-created by all stakeholders along the value chain for DHTs. The consortium is comprised of 15 partners from nine countries (Italy, the Netherlands, Denmark, Spain, Germany, Norway, UK, Belgium, and Poland). Stakeholders included are HTA agencies, regulators, hospitals, patient/citizen representatives, academics/universities, notified bodies, and developers. The project covers all types of DHTs and Technology Readiness Levels (TRLs). A multistakeholder, multidomain, and modular approach is adopted with the final goal to provide a validated, ready-to-use digital HTA framework for DHTs for the European ecosystem.

The EDiHTA is articulated in eight working packages and covers four years. Starting from the mapping of the current scenario, after the definition of a common taxonomy, EDiHTA will develop a DHT-dedicated HTA framework. A common digital platform to support the assessment will be developed. Both the HTA framework and platform will be tested, refined, and validated through a piloting process involving disparate DHTs (mApp, telemedicine, artificial intelligence, and others) with different TRLs, available in different countries, with the participation of all stakeholders. As a consequence, evidence requirements will be defined and made more transparent; this will support health technology.

The EDiHTA project will deliver the first European digital HTA framework able to support and inform decision-making (at the macro [policy], meso [management providers], and micro [clinicians] level) towards faster and safer access to DHTs of added value, taking European regulations and legal requirements into account and assessing all HTA domains.

The European parliament is working on a proposal for an Artificial Intelligence (AI) Act. The goal is to guarantee that AI systems used in the European Union are “safe, transparent, traceable, non-discriminatory and environmentally friendly.” The Act is not directly linked to health technology assessment (HTA), but implications for HTA are expected.

The final aim of the AI Act is the adoption of harmonized rules for AI foundation models. Foundation models are designed to produce a wide variety of outputs that are being adopted across various sectors. Therefore, their adaptation to specific (clinical) needs plays a key role and has implications on the required training data. The goal of our analysis was to critically investigate the potential implications of the Act’s requirements for HTA. The analysis moves from the technology (foundation models in health care) to the requirements and adopted terminology.

The definition of methods to assess the adaptation of foundation models has become a priority for HTA. The Act focuses on data quality, transparency, human oversight, and accountability. Available frameworks, such as the one developed by the AI MIND project, include them, but experience is required. HTA must define how to adapt its methods and frameworks as well as the risk levels addressed by the Act. In addition, the reference to energy efficiency standards confirms the need for HTA to clarify the role of and methods for environmental impact assessment. Finally, the patient perspective has great relevance in relation to the risk of discrimination.

The AI Act confirms the importance of topics already debated in HTA that still need resolution and testing. Harmonization of rules for AI and approaches for HTA is the main challenge. The AI Act, the HTA regulation, and ongoing European Union projects (i.e., AI-MIND and European Digital Health Technology Assessment) are showing the route to follow in the coming years.

Several organizations specializing in health technology assessment (HTA), at various levels, are exploring avenues to integrate the dimension of environmental sustainability (ES) within their assessments. However, evidence remains scant on how best to do this. Across the different levels, hospital-based HTA (HB-HTA) also needs to comply with the leadership and strategies of the hospital and adapt to the existing resources and established partnerships.

The purpose of this study is threefold: to provide an overview of the progress achieved by hospitals with respect to the integration of ES into HB-HTA; to detect consistencies between HB-HTA activities and hospital ES strategies; and to explore possible approaches and methods for integrating ES into HB-HTA. The methodology is structured in two steps: (i) a literature review on HB-HTA and environmental impact/sustainability to identify emerging approaches and methods for integrating environmental sustainability into HB-HTA; (ii) data collection by means of a questionnaire submitted to hospitals/organizations based on their membership of Health Technology Assessment International.

Preliminary results highlight a widespread awareness of the importance of integrating ES issues into HTA at the different levels at which it is performed. The incorporation of ES issues into HB-HTA is still in its infancy. There are some factors hindering the development of methods, such as the absence of scientific evidence on emerging approaches and the scarce availability of data or difficulty in tracing data back to a specific technology.

The integration of the dimension of ES into HB-HTA still appears to be very immature. Coordination between the different actors in the system seems necessary to overcome the obstacles identified.

The European Digital Health Technology Assessment (EDiHTA), an EU-funded project, aims to deliver the first European digital HTA framework co-created by all relevant stakeholders, validated and ready to use. It responds to the need to harmonize tools, methods, guidelines, and frameworks that are already adopted or under development. All types of digital health technologies (DHTs) and Technology Readiness Levels (TRLs) are covered.

The EDiHTA project adopts a multistakeholder, multidomain, and modular approach to reach a consensus on a common HTA framework for DHTs (e.g., mApp, telemedicine, artificial intelligence). The currently available HTA frameworks refer to different DHTs (e.g., MAST, DiGA, MAS-AI), include different HTA domains, respond to the needs of different stakeholders, and are applicable in specific local and national contexts. In the majority of cases, they are applicable only in advance TRL. In a few cases, they are already adopted in regulatory/HTA processes. The lack of coordination and harmonization has significant implications for all stakeholders, manufacturers and patients included.

The EDiHTA project adopts a holistic approach involving all relevant stakeholders for consensus building at national and European levels. Cross-border collaboration among stakeholders will be reinforced by the definition of a common taxonomy based on consensus building, and the development and validation of common methods and the HTA framework. Evidence requirements will be defined and made more transparent to increase support for health technology manufacturers. In addition, the harmonization effort, together with the development of a digital platform for the HTA framework, will allow faster and safer access to DHTs by patients/citizens.

The goal of the EDiHTA project is to provide the first European digital HTA framework for DHTs that will be adopted by all relevant stakeholders along the lifecycle of digital technologies to promote sustainable health systems. A consortium comprising 15 partners from eight countries, including all relevant stakeholders, works to reach these goals within the next four years.

In Italy, a fixed proportion of health spending is allocated to pharmaceutical expenditure. While the main objective of setting a budget for pharmaceuticals is to control spending, the effectiveness of this ceiling is questionable. This study aims to investigate the determinants of pharmaceutical expenditure for orphan drugs and gather information for effective planning and programming of pharmaceutical spending.

Data analysis relied on pharmaceutical companies’ pricing and reimbursement (P&R) dossiers submitted to the Italian Medicines Agency (AIFA) for drug-reimbursement approval, along with AIFA’s internal procedural documents. The study encompassed all rare disease drugs reimbursed from January 2013 to January 2019. For each drug, a comparison was made between the expected post-negotiation expenditure and the actual spending observed over the three years following reimbursement approval. Potential determinants of the normalized ratio between observed and expected spending were identified using univariate and multivariate beta regression models. The same methodology was replicated to identify potential determinants of the difference between expected spending before and after negotiation.

Fifty-two rare disease drugs admitted for reimbursement during the study period were analyzed. The median expenditure in the first three commercialization years was 7.6 percent lower than the expected post-negotiation spending. Beta regression analysis indicated a significantly lower reduction for innovative drugs (β 0.736, p-value 0.011 univariate, β 0.585, p-value 0.045 multivariate). Similar effects were observed for P&R procedures (β 0.902, p-value 0.007) and the number of indications presented (β 0.754, p-value 0.021), but only in univariate model. Beta regression analysis for the expected expenditure ratio before/after negotiation revealed a significant effect only for the payment-by-result variable (β 1.485, p-value 0.001).

Observed expenditure for orphan drugs aligns with the expected spending post-negotiation. However, in the subgroup of innovative orphan drugs, the observed pharmaceutical spending was higher than estimated. This could be attributed to prescriber preferences and to a prevalent patient pool awaiting innovative treatment. It appears that the recognition of innovativeness favors orphan drugs that are rewarded with faster market access.

Health technologies play a relevant role in environmental sustainability (ES). However, limited evidence exists on approaches and methods to integrate ES into the Health Technology Assessment (HTA).

The purpose of this study is: (i) to provide an overview of global HTA organizations’ progression toward the integration of ES into HTA; (ii) to investigate various paths for this integration, highlighting obstacles, priorities, potential approaches, and methods.

Data were collected via questionnaires from organizations belonging to HTA networks, International Network of Agencies for Health Technology Assessment, and European Network for HTA. To complement the results of the survey, the authors carried out a desk analysis with strategic documents available on institutional websites.

The survey included twenty-six respondents from twenty different countries (thirty-three percent response rate). Among the study’s participants, there is a notable acknowledgment of the importance of integrating ES into HTA. However, only nine organizations are actively engaged in these integration efforts, each employing unique methodologies and perspectives. There is a substantial consensus on the application of life cycle assessment, with a particular emphasis on the use of environmentally extended input–output analysis, and a stronger preference for cost-utility analysis. Nevertheless, evidence on integrating ES into HTA remains scarce. Major challenges identified include data collection difficulties and the necessity for interdisciplinary teams.

Our study represents a preliminary effort to systematize initiatives aimed at integrating ES into HTA. Further research is required to customize methods and tools for appropriately evaluating the environmental impacts of technologies. The findings suggest that achieving ES-HTA integration demands a multi-tiered, interdisciplinary approach.

Artificial intelligence (AI)-based health technologies (AIHTs) have already been applied in clinical practice. However, there is currently no standardized framework for evaluating them based on the principles of health technology assessment (HTA).

A two-round Delphi survey was distributed to a panel of experts to determine the significance of incorporating topics outlined in the EUnetHTA Core Model and twenty additional ones identified through literature reviews. Each panelist assigned scores to each topic. Topics were categorized as critical to include (scores 7–9), important but not critical (scores 4–6), and not important (scores 1–3). A 70 percent cutoff was used to determine high agreement.

Our panel of 46 experts indicated that 48 out of the 65 proposed topics are critical and should be included in an HTA framework for AIHTs. Among the ten most crucial topics, the following emerged: accuracy of the AI model (97.78 percent), patient safety (95.65 percent), benefit–harm balance evaluated from an ethical standpoint (95.56 percent), and bias in data (91.30 percent). Importantly, our findings highlight that the Core Model is insufficient in capturing all relevant topics for AI-based technologies, as 14 out of the additional 20 topics were identified as crucial.

It is imperative to determine the level of agreement on AI-relevant HTA topics to establish a robust assessment framework. This framework will play a foundational role in evaluating AI tools for the early diagnosis of dementia, which is the focus of the European project AI-Mind currently being developed.

Hyperparathyroidism is a medical condition characterized by an excess of parathyroid hormone (PTH) in the blood. This hormone is produced by the parathyroid glands, located behind the thyroid gland. PTH plays a crucial role in regulating calcium levels in the body, which is necessary for muscle activity, nerve transmission, and maintaining bone density. In this study, the authors aimed to determine the parameters necessary for defining the delivery process of interventions for managing hyperparathyroidism.

To achieve this, a group of Key Opinion Leaders (KOLs) was surveyed using a questionnaire to investigate specific drivers such as the duration of interventions, drug therapies employed, and materials required. The authors also estimated the indirect costs associated with patients and caregivers. The economic analysis considered the perspectives of both the Italian National Health Service (SSN) and the community. The analysis was conducted using Activity Based Costing (ABC) methods to determine the full cost sustained for a parathyroidectomy surgery and pharmacological therapies per patient treated, as well as the average resources absorbed by a patient managed through pure surveillance.

The results showed that the average annual cost of parathyroidectomy and pharmacological therapies per patient was EUR5,193.20 and EUR1,726.96, respectively. Productivity losses due to the interventions amounted to EUR858.21 and EUR66.80 for the patient and caregiver, respectively. The pure surveillance strategy incurred an average yearly cost of EUR197.42.

The study’s major limitation was the lack of evidence available concerning the therapies under analysis, particularly in the Italian context. Nonetheless, the survey of clinicians provided useful insights into the expenditures associated with implementing interventions for managing hyperparathyroidism. In conclusion, managing hyperparathyroidism involves considerable costs, and healthcare providers must consider the perspectives of both the SSN and the community when estimating the economic impact of interventions. The authors’ analysis provides insights into the cost of different interventions, which could help healthcare providers make informed decisions when managing hyperparathyroidism.

Clinicians are increasingly relying on artificial intelligence (AI) generated technologies for support in diagnosis, therapeutic decision-making, and prediction. Despite the increased focus on AI in health, an agreed HTA model for AI technologies, including consensus on new domains and topics to be assessed, is lacking.

A Delphi survey was sent to a multidisciplinary expert panel asking about the importance of including the nine domains and associated topics presented in the EUnetHTA Core Model, as well as 20 additional topics identified through literature reviews, when assessing AI-supported health technologies. The Delphi survey was repeated twice among the same panelists and a nine-point Likert scale was used to identify the perceived relevance of each domain and topic.

The survey was sent to 87 various experts, with a total 47 of experts completing both Delphi rounds. The majority of panelists was knowledgeable of HTA (80%), familiar with the EUnetHTA Core Model (61%), and had adequate or high-level knowledge of AI (65%). The EUnetHTA domains most often indicated as “critical to include” were clinical effectiveness (82%), ethical aspects (81%), and cost effectiveness (77%), whereas organizational (59%) and social aspects (63%) were less often perceived as critical to assess. For the additional 20 topics identified through literature reviews, bias in data, accuracy in the AI model, appropriateness, and trustworthiness emerged as some of the new topics deemed critical to include in HTAs (all above 85%), whereas there was a lack of agreement on the relevance of including environmental (51%) and social sustainability (55%).

The study investigated in detail which issues should be included in an AI HTA core model. Current models need some adjustment and revision. At the same time, it is essential to open the discussion on including new domains and topics.

Age-related macular degeneration (AMD) is a common condition that affects the middle part of a patient’s vision. Typically, it first appears in people in their 50s and 60s. While it does not cause total blindness, it can make everyday activities, such as reading and recognizing faces, more difficult. This analysis aimed to define the resource absorption and cost-effectiveness profiles of the anti-vascular endothelial growth factor therapies currently available in the Italian healthcare context.

A questionnaire was prepared to gather information on specific drivers involved in the provision pathway. The economic analysis was conducted according to activity-based costing methods. A cost-effectiveness analysis was carried out to provide information on the sustainability profile of the treatments available in the Italian setting. Results were reported in terms of the incremental cost-effectiveness ratio (ICER).

Deterministic and probabilistic sensitivity analyses were carried out to test the robustness of the results.

The average absorption of resources per patient along the whole clinical pathway for aflibercept, bevacizumab, ranibizumab, and brolucizumab was EUR6,858, EUR1,420, EUR7,930, and EUR5,667, respectively. Brolucizumab was characterized by an unacceptable cost-effectiveness profile (ICER EUR43,454) versus bevacizumab, considering a willingness-to-pay threshold of EUR40,000 per quality-adjusted life-year (QALY). Compared with ranibizumab, brolucizumab was associated with lower costs (EUR22,368 versus EUR29,333) and higher QALYs (12.8 versus 12.6). Brolucizumab had a higher level of QALYs (12.8 vs 12.7) and lower resources absorbed than aflibercept, with a saving of EUR4,222. Therefore, brolucizumab was a dominant alternative to ranibizumab and aflibercept.

The analysis underlined how brolucizumab is a cost-saving strategy, compared with aflibercept and ranibizumab, and is likely to be cost-effective relative to bevacizumab in the Italian healthcare context.

The challenge to health systems is sustainability, not only in the economic and financial sense of compatibility of spending with allocated resources, but also in terms of equity in access to services and care, quality, safety, innovation and research, that is, in terms of the effectiveness of the right to health. Some countries, such as Italy, do not have formal health technology assessment (HTA) or other similar processes that take into account views outside the decision maker.

In the Italian national context, there is currently no single tariff for the reimbursability and pricing of genomic technologies. In fact, although genomic tests have been in clinical practice for many years now, to date they have not yet been included in the LEA (Minimum healthcare provision), especially in view of the fact that a defined and transparent process for updating the Essential Levels of Care has been operational since 2018. With the goal of structuring guidelines for the adaptability of economic evaluations to currently available and developing genomic technologies, a literature review was conducted.

The literature review showed that there are some methodological and practical issues that need to be carefully considered when designing and conducting economic evaluations of genomic tests. In more detail, five key concepts were identified in order to implement the most comprehensive economic evaluation of the technologies under study: the PICO model, the survey perspective, the costs included in the analysis, the effectiveness analysis, the time horizon, and the discount rate.

Adequate definition of these concepts appears to be of paramount importance in view of the fact that genomic testing may have important consequences for future generations as well. For the purpose of sustainability of access of genomic technologies, the use of Budget Impact Analysis (BIA) is recommended in all analysis settings being essential for the regulator to tie access to its available budget capacity.

Health technology assessments (HTAs) of robotic assisted surgery (RAS) face several challenges in assessing the value of robotic surgical platforms. As a result of using different assessment methods, previous HTAs have reached different conclusions when evaluating RAS. While the number of available systems and surgical procedures is rapidly growing, existing frameworks for assessing MedTech provide a starting point, but specific considerations are needed for HTAs of RAS to ensure consistent results. This work aimed to discuss different approaches and produce guidance on evaluating RAS.

A consensus conference research methodology was adopted. A panel of 14 experts was assembled with international experience and representing relevant stakeholders: clinicians, health economists, HTA practitioners, policy makers, and industry. A review of previous HTAs was performed and seven key themes were extracted from the literature for consideration. Over five meetings, the panel discussed the key themes and formulated consensus statements.

A total of ninety-eight previous HTAs were identified from twenty-five total countries. The seven key themes were evidence inclusion and exclusion, patient- and clinician-reported outcomes, the learning curve, allocation of costs, appropriate time horizons, economic analysis methods, and robotic ecosystem/wider benefits.

Robotic surgical platforms are tools, not therapies. Their value varies according to context and should be considered across therapeutic areas and stakeholders. The principles set out in this paper should help HTA bodies at all levels to evaluate RAS. This work may serve as a case study for rapidly developing areas in MedTech that require particular consideration for HTAs.

The objective of the present policy analysis was to understand how a disinvestment approach to the process of health technology assessment (HTA), applied to the field of medical devices, might help Italian policymakers to properly spend the resources in healthcare.

Previous international and national experiences in disinvestment for medical devices were reviewed. Precious insights for the rational expenditure of the resources were derived by assessing the evidence available.

The disinvestment of ineffective or inappropriate technologies or interventions with an inadequate value-for-money ratio has become a growing priority for National Health Systems. Different international disinvestment experiences of medical devices were identified and described through a rapid review. Although most of them have a strong theoretical framework, their practical application remains difficult. In Italy, there are no examples of large and complex HTA-based disinvestment practices, but their importance is becoming increasingly acknowledged, especially given the need to prioritize the funds provided by Recovery and Resilience Plan.

Anchoring decisions on health technologies without reassessing the current technological landscape through a robust HTA model might expose to the risk of not ensuring the best employment of the resources available. Thus, it is necessary to develop a strong HTA ecosystem in Italy through adequate consultation with stakeholders to enable a data-driven and evidence-based prioritization of resources toward choices characterized by high value for both patients and society as a whole.

The role of associations dedicated to patient advocacy has assumed strategic importance within the most advanced health systems, including the Italian NHS. In this period of strong national and international emergency, the associations of citizens and patients have also changed their actions and have implemented others to alleviate the discomforts of sick people in Italy, collaborating with institutions and health services.

Data were collected using a semi-structured survey, with both yes / no and open questions, developed and administered by the Patient Advocacy LAB (ALTEMS- Catholic University of Sacred Heart) to 150 patient advocacy associations. The organizational changes and the initiatives adopted by patient associations during the COVID-19 emergency was investigated.

The majority of the initiatives adopted by patient advocacy associations during the COVID-19 pandemic have been introduced during the first wave (March- June 2020), and that most of them have been maintained to (December 2020). These initiatives included improvements and updating of the communication tools aimed at reaching the higher number of patients. Thanks to these new approaches, the empathy and the assistance to patients have been increased. In addition, a number of training initiatives have been developed online and they have been followed by a large number of patients and caregivers. At an operative level, during the pandemic, many patient advocacy associations have provided their support to distribute masks, gloves, and therapies to patients. Unfortunately the fundraising activities suffered due to the lockdown, and the main problems were related to social distancing and lack of public campaigns. The institutional relationships have increased during the COVID-19 pandemic and they mainly regarded the participation in legislative interventions. Among the initiatives adopted, our sampled associations affirmed that they would like to maintain the social support provided to patients experienced during the pandemic.

Finally we calculated a resiliency score and we discovered that the majority of the sampled associations developed an intermediate level of resiliency and that it is positively correlated with their tenure.

Our results provide a fresh view about the role of patient advocacy associations during the pandemic indicate their important role within the NHS.

To provide a complete picture of the economic impact of the coronavirus disease 2019 (COVID-19) emergency for the Italian National Health System (NHS), an estimate was made of the costs to the NHS of vaccination hesitation. The concept of economic impact was investigated with reference to the volume of hospitalizations and days of intensive care required for patients with COVID-19 related to missed vaccinations, considering a vaccine efficacy of less than 100 percent.

Data from the Istituto Superiore di Sanità were analyzed with respect to the number of people vaccinated in the general population, and the number of people with severe acute respiratory syndrome coronavirus 2 infection who were hospitalized or died in a one-month period stratified by vaccination status. The costs for unvaccinated patients admitted to a general hospital ward (Medical Area) or the intensive care unit were calculated.

Based on the number of preventable hospitalizations among unvaccinated people, the economic impact of missed vaccinations on the NHS in the 30-day period from 13 August 2021 to 12 September 2021 was estimated. Among the unvaccinated hospitalized patients, 5,932 would have avoided hospitalization in the Medical Area and 715 would have avoided admission to the intensive care unit. Thus, each unvaccinated hospitalized patient had an average per capita cost of EUR 17,408. The total costs amounted to EUR 69,894,715, comprising EUR 51,166,079 for hospitalizations in the Medical Area and EUR 18,728,636 for hospitalizations in intensive care.

By evaluating the weekly incidence of hospitalizations per 100,000 people stratified by vaccination status (unvaccinated, partially vaccinated, and fully vaccinated), it is possible to see that we are facing two distinct pandemics running together.

The continuous and pressing challenge that the drug regulatory authorities in Italy and in Europe are facing is that of guaranteeing patients’ quick access to new drugs, ensuring the economic sustainability of the system at the same time. In recent years, flexible and diversified approaches have been developed known as Managed Entry Agreements (MEA).

We performed an analysis of the Italian legislative and regulatory aspects in reference to a new Value-based Managed Entry Agreement (VBMEA) pathway. Thus, we tried to investigate the rationale for a new pathway analyzing three main dimensions related to the new medical product (MP): value; time to entry access; and, data quality and registry design. Moreover, we shared the discussion of the proposal with an international experts’ panel.

The proposal for a new pathway of VBMEA from a procedural point of view shows the novelty related to the possibility to organize joint CTS (Technical Scientific Committee) and CPR (Price and Reimbursement Committee) assessment. A 24-month contract with an ex-factory price (PP) equal to X EUR per dose and a transfer price to the National Public Health System (NPHS), following application of a confidential discount for public structures (-X%), of X EUR per dose. After 24-months, an analysis of VBMEA is carried out. The price of the MP is therefore established based on AIFA registries and VBMEA results. The cost value incurred by the NPHS, intended as the difference between the price in market (entry) access phase and the price negotiated (PVB) in the light of the VBMEA results, shall be returned by the pharmaceutical company in the form of a payback.

Currently, MEAs represent one of the main topics of discussion between the European National Payers Authorities. There is very little information on product performance that results from MEAs. This research project could provide advice to policy makers to decrease negotiation time by ensuring earlier access to innovation for patients.

The integrated patient-centered, evidence-based approach to care recognizes the role of patient preferences. A discrete choice experiment (DCE) was developed with the aim of identifying the preferences of men with prostate cancer in Italy regarding the different risk-benefit factors of various treatment options.

The DCE was developed with the support of prostate cancer patients and oncologists and was based on a targeted scoping review. The final DCE included 26 choice sets divided into two blocks. The first block focused on all prostate cancer patients (both metastatic and non-metastatic), while the second block aimed to assess preferences for patients with metastatic hormone-sensitive prostate cancer (mHSPC). Patients were asked to choose from ten attributes in the first block and six in the second block. The aim was to identify attributes and levels with a statistically significant impact on patient preferences. Preference estimates were calculated using a conditional logit regression model and the results were stratified by cancer stage (metastatic or non-metastatic) in the first block.

A total of 202 patients (mean age 72 years) completed the DCE. In the first block, the most important attribute was quality of life (QoL), particularly for patients with metastatic cancer. The other three attributes found to be significant, in order of relevance to patients, were the risks of experiencing cognitive impairment, hematologic complications, and fatigue. For patients with mHSPC, QoL was the strongest determinant of preference. The risk of experiencing fatigue was also a relevant attribute, followed by skin irritation.

This study shows that the effect of treatment on QoL was the most important attribute for patients diagnosed with prostate cancer. Specific risk factors play a different role in the choice of treatment depending on cancer type, with the risk of experiencing fatigue being valued by all groups. Identifying and understanding patients’ preferences related to treatments for prostate cancer will help physicians identify the best treatment strategy.

Neurogenic bowel dysfunction occurs in people with central nervous system disease or injury and causes loss of bowel control and severe constipation. These problems involve a lot of anxiety and discomfort and can reduce the quality of life of those who suffer from them, therefore the management of symptoms is very important. Peristeen Plus is a transanal irrigation (TAI) system for bowel dysfunction management, used to empty the rectum and distal sigmoid colon, to prevent uncontrolled bowel movements or to relieve and prevent constipation.

A literature review was conducted. A total of 14 records were included to evaluate the benefits in terms of efficacy and safety associated with the adoption of the medical device. To assess the economic impact, two different budget impact models have been implemented. The first aimed at evaluating an incremental diffusion in a short-term time horizon (3 years) of the home distribution of the device compared to the direct and indirect distribution methods in the Italian context. The second model aimed at assessing the impact of the diffusion of the device in the clinical practice.

Overall, most studies demonstrate improved endpoints related to the severity of fecal incontinence, constipation and intestinal disorders in patients using the device. The economic assessments conducted estimate that the increase in the Italian care setting is associated with a saving of resources in each year under analysis. The diffusion of home distribution of the device would potentially be able to offer a lower absorption of resources compared to other distribution methods. In addition to this, there is an incremental saving correlated to the degree of diffusion of Peristeen Plus.

TAI is considered a safe and more effective method than conventional treatments for reducing fecal incontinence, constipation and improving quality of life. The results of our study confirm the benefits of TAI as a second-line treatment in case of failure of conventional medical therapy in the management of the neurogenic gut.

Treatments for coronary heart disease patients have had major developments in recent decades, both in the pharmacological and interventional fields, and this has helped to prolong the survival of these patients. However, the growing number of patients who show persistent and disabling symptoms of angina proves that at the same time their quality of life has not been equally improved.

We conducted a multidimensional assessment coherent with health technology assessment methodology on the Coronary Sinus Reducer System (CSRS). CSRS is the latest line of therapy for patients with coronary artery disease who are ineligible for revascularization, demonstrate reversible ischemia, and have refractory angina pectoris (AP) despite optimal standard medical therapy. We performed a literature review in order to gather evidence on efficacy and safety of the device and on the economic and organizational impact of the procedure. In the economic domain we developed a cost-utility model based on a decision tree and a five-year time horizon budget impact model.

Several studies in the literature have shown that this therapy is related to an increase in quality of life and an improvement in symptoms of refractory angina. The economic evaluations conducted show how the therapy, despite an increase in the resources absorbed in the first years of implementation, reaches a cost saving profile in the medium term due to positive outcomes, while leading to an increase in the quality of life in patients suffering from refractory angina.

The treatment of refractory angina remains a challenge for today’s medicine. Patients suffering from this condition are often described as “no option” patients. Thus, despite there is a need of further evidence to establish even more robustly the economic sustainability of the device, especially on its effectiveness in the medium-long term, the device should be taking into account in those patients who could benefit from it in terms of relieving the symptoms of angina and improving their quality of life.