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La moderna Sanità è sempre maggiormente improntata allo sfruttamento di tecnologie all’avanguardia, indispensabili tanto a garantire la Salute dei Nostri Pazienti quanto alla competizione virtuosa fra Enti ospedalieri. In Italia, dal 2020, la crescente spesa sanitaria pubblica nazionale per i consumi intermedi, ovvero per i fattori produttivi utilizzati nell’erogazione delle prestazioni, ha stabilmente superato la corrispondente da reddito dipendente del Personale sanitario. A questo risultato contribuiscono significativamente le tecnologie sanitarie, i cui importi annui di acquisto e di gestione stanno crescendo 3 - 4 volte più velocemente dell’inflazione media, tanto da essere candidate a principale leva di incremento della spesa complessiva, a discapito della sostenibilità invocata in tutto il mondo ed ormai imprescindibile. Un paradosso, forse, rispetto al costante calo delle prestazioni effettivamente erogate nel nostro Paese; di conseguenza, sembra lapalissiana la necessità di contro-misure proporzionalmente adeguate: più che di spendere meno, si tratta di investire meglio; allo scopo, un’ampia rivisitazione delle attività e delle competenze individuabili all’interno dei nosocomi nazionali. In aggiunta, con particolare riferimento agli Istituti di Ricovero e Cura a Carattere Scientifico (I.R.C.C.S.), si discute dell’opportunità di una concreta distinzione fra la gestione tecnologica in Clinica rispetto a quello nei Laboratori di Ricerca. Per questi ultimi, infatti, differenze negli schemi di finanziamento e nell’approccio organizzativo suggeriscono metodi di governo altrettanto all’avanguardia, così da meglio raggiungere gli obbiettivi di appropriatezza auspicati non solo nei Reparti e negli Ambulatori, ma anche alle frontiere del Sapere. Fra i vari esempi declinati, l’originale proposta di un Research Technology Assessment (alternativo al più noto Health Technology Assessment) e la progettazione di nuove soluzioni hardware direttamente dentro gli Is, Modern healthcare is increasingly based on the exploitation of cutting-edge technologies, which are essential both to guarantee the health of our patients and to virtuous competition between hospital bodies. In Italy, since 2020, the growing national public health expenditure for intermediate consumption, i.e. for the production factors used in the provision of services, has steadily exceeded the corresponding employee income of healthcare personnel. Health technologies contribute significantly to this result, whose annual purchase and management amounts are growing 3 - 4 times faster than average inflation, so much so that they are candidates for the main lever for increasing overall spending, to the detriment of the sustainability invoked in the whole world and is now essential. A paradox, perhaps, compared to the constant decline in services actually provided in our country; consequently, the need for proportionally adequate counter-measures seems obvious: rather than spending less, it is a question of investing better; for this purpose, a broad review of the activities and skills identifiable within national hospitals. In addition, with particular reference to Scientific Hospitalization and Care Institutes (I.R.C.C.S.), we discuss the opportunity of a concrete distinction between technological management in the Clinic compared to that in the Research Laboratories. For the latter, in fact, differences in financing schemes and organizational approaches suggest equally cutting-edge governance methods, so as to better achieve the desired appropriateness objectives not only in the departments and clinics, but also at the frontiers of knowledge. Among the various examples presented, the original proposal for a Research Technology Assessment (alternative to the more well-known Health Technology Assessment) and the design of new hardware solutions directly within the Institutes themselves. This is accompanied by the implementation of good practices both international an

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Introduction: High blood pressure is the main risk factor for global disease. In the Dominican Republic, 31% of the population suffers from arterial hypertension (HTN) and of these, 60% have medical treatment. Objective: To determine blood pressure levels in non-hyper-tensive individuals between 18-65 years of age in the community of Pizarrete Abajo during the period of August - September 2018. Methodology: Prospective, descriptive, and longitudinal study in which 85 individuals residing in the community of Pizarrete Abajo were analyzed, of which 44 resulted in altered blood pressure levels. Results: 51.7% of the individuals presented altered blood pressure levels without previously being diagnosed with hypertension. 24% of people showed altered levels one week after the first blood pressure measurement. Conclusions: In a sample of 85 people, altered blood pres-sure levels were found in 44 individuals. A second blood pressure measurement was carried out one week after the first measurement and it was observed that 56.8% corres-ponded to high blood pressure values, 20.4% corresponded to stage 1 HTN levels, and 18.1% corresponded to stage 2 HTN levels., Introducción: la presión arterial elevada es el principal factor de riesgo de enfermedad global. En la República Domini-cana el 31 % de la población padece de hipertensión arterial (HTA) y de este, un 60 % lleva tratamiento médico. Objetivo: determinar los niveles tensionales en individuos no hipertensos entre 18-65 años en la comunidad de Piza-rrete Abajo, durante el período agosto-septiembre 2018. Metodología: estudio prospectivo, descriptivo y de corte longitudinal, en el que se analizaron 85 individuos que residen en la comunidad de Pizarrete Abajo, de los cuales 44 resultaron con niveles tensionales alterados. Resultados: el 51.7 % de los encuestados presentó niveles de tensión arterial alterados, sin haber sido anteriormente diagnosticado con hipertensión arterial. Un 24 % de las personas mostró niveles alterados una semana después de la primera toma de presión arterial. Conclusiones: en una muestra de 85 personas se encon-traron niveles tensionales alterados en 44 individuos. Se procedió a una segunda toma de presión arterial una semana después de la primera y se observó que un 56.8 % correspondió a valores tensionales elevados, un 20.4 % a valores de HTA estadio 1 y un 18.1 % a valores de HTA estadio 2

La valutazione etica delle terapie digitali nei processi di Health Technology Assessment (HTA)

Intelligenza artificiale ed HTA: aspetti etici

Health technology assessment (HTA) is multidisciplinary process that uses explicit methods to determine the value of a health technology at different points in its lifecycle. The purpose is to implement an informed decision-making process in order to promote an equitable, efficient, and high-quality health system. Ethics has been identified as a key element in HTA because health care is a moral endeavour and potential technology presents complex moral dilemmas, especially with the rise of novel technologies. In evaluating health technologies, ethical aspects must be taken into account such as patients’ privacy and informed consent in terms of (re-)use of their personal sensitive data, alongside with the possibility of unintended consequences (e.g., doing more harm than good: Thalidomide case) and the effects on society in terms of justice and equity, with implications for financial sustainability. Without taking account of the ethical dimension in the process, choices may be made without considering the overall context of implementation, thus, compromising the relevance of decisions and policies, with potentially unintended consequences and/or harmful effects to the health and wellbeing of individuals/patients, to public health, to the environment, and to the society at large. Decision-makers should guarantee that adopted health technologies are meaningful and relevant for the society, with due consideration to their development too, to the extent possible, by also taking ethical aspects into consideration when evaluating health technology. Despite many years of recognising ethics as a foundational part of HTA, studies have shown that the ethical dimension is still not systematically and sufficiently addressed by means of ethical analysis. Furthermore, novel technologies, new types of data, and a paradigm shift in terms of how we generate and synthesize evidence, whilst providing us with unique opportunities in terms of HTA, pose concrete challenges and new (and rev

"Evidence" is a key term in medicine and health services research, including Health Technology Assessment (HTA). Randomized clinical trials (RCTs) have undoubtedly dominated the scene of generating evidence for a long period of time, becoming the hallmark of evidence-based medicine (EBM). However, due to a number of misunderstandings, the lay audience and some researchers have sometimes placed too much trust in RCTs compared to other methods of investigation. One of the principal misunderstandings is to consider RCTs findings as isolated and self-apparent pieces of information. In other words, what has been essentially lacking was the awareness of the value-context of the evidence and, in particular, the value- and theory-ladenness (normativity) of scientific knowledge. This paper aims to emphasize the normativity that exists in the production of scientific knowledge, and in particular in the conduct of RCTs as well as in the performance of HTA. The work is based on some lessons learned from Philosophy of Science and the European project "VALIDATE" (VALues In Doing Assessments of healthcare TEchnologies"). VALIDATE was a three-year EU Erasmus+ strategic partnerships project (2018-2021), in which training in the field of HTA was further optimized by using insights from political science and ethics (in accordance with the recent definition of HTA). Our analysis may reveal useful insights for addressing some challenges that HTA is going to face in the future.

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The present study aims at defining the economic and organizational impacts of the introduction of chimeric antigen receptor T-cell therapy (CAR-T) in Italy, for the management of diffuse large B-cell lymphoma (DLBCL) patients in third-line therapy, defining the overall level of sustainability for both hospitals and the National Healthcare System (NHS). The analysis focused on CAR-T and Best Salvage Care (in the following BSC), assuming the Italian hospital and NHS perspectives, over a 36-month time horizon. Process mapping and activity-based costing methodologies were applied to collect the hospital costs related to the BSC and CAR-T pathways, including adverse event management. Anonymous administrative data on services provided (diagnostic and laboratory examinations, hospitalizations, outpatient procedures, and therapies) to 47 third-line patients with lymphoma, as well as any organizational investments required, were collected, in two different Italian Hospitals. The economic results showed that the BSC clinical pathway required less resources in comparison with CAR-T (excluding the cost related to the therapy) (BSC: 29,558.41 vs. CAR-T: EUR 71,220.84, −58.5%). The budget impact analysis depicts that the introduction of CAR-T would generate an increase in costs ranging from 15% to 23%, without considering treatment costs. The assessment of the organizational impact reveals that the introduction of CAR-T therapy would require additional investments equal to a minimum of EUR 15,500 to a maximum of EUR 100,897.49, from the hospital perspective. Results show new economic evidence for healthcare decision makers, to optimize the appropriateness of resource allocation. The present analysis suggests the need to introduce a specific reimbursement tariff, both at the hospital and at NHS levels, since no consensus exists, at least in the Italian setting, concerning the proper remuneration for the hospitals who guarantee this innovative pathway, assuming high risks related t

L’utilizzo di valutazioni economiche in ambito sanitario ha assunto nel corso degli anni un ruolo di crescente importanza, alla luce dell’esigenza di fornire informazioni utili ai decision-maker nel difficile ruolo di continuare a garantire l’innovazione in contesti di risorse scarse all’interno di un sistema universalistico. I modelli decisionali, siano essi alberi decisionali o modelli di Markov, sono strumenti che dimostrano di rispondere il più delle volte alle esigenze di simulare il decorso di una patologia e l’effetto dei trattamenti su di esso, producendo stime su outcome finali e costi nel lungo termine che alimentano indicatori di costo-efficacia/costo-utilità, utilizzati, seppur con logiche differenti, da diverse agenzie di HTA per definire il valore economico di una nuova tecnologia e supportare le negoziazioni di prezzo. Oltre ai modelli di costo-efficacia/utilità, i più semplici modelli di impatto sul budget trovano notevole spazio in questo contesto, fornendo informazioni cruciali rispetto la sostenibilità del sistema.Anche in Italia tali valutazioni sono considerate nei processi di valutazione dei farmaci da parte di AIFA che le analizza e le utilizza all’interno delle istruttorie sottoposte al Comitato Prezzi e Rimborso (CPR) per la definizione delle condizioni di prezzo e rimborso dei farmaci. Tali valutazioni sono inoltre particolarmente raccomandate in quegli ambiti decisionali particolarmente complessi, quali ad esempio il caso di farmaci orfani/per malattie rare.Al fine di leggere come la valutazione economica viene effettivamente utilizzata nei contesti valutativi l’accesso in Italia, è stato pertanto presentato in questo lavoro il caso specifico dell’atrofia muscolare spinale e del medicinale risdiplam, come ambito esemplificativo di una malattia rara in cui l’innovazione è entrata in modo prepotente negli ultimi anni e dove pertanto i decisori si sono trovati (e tuttora si trovano) a dover valutare attentamente gli impatti sia da un p, The use of economic assessments in the health sector has assumed a role of increasing importance over the years, in light of the need to provide useful information to decision-makers in the difficult role of continuing to guarantee innovation in contexts of scarce resources, in a universalistic system. Decision models, both decision trees or Markov models, are tools that demonstrate that they most often respond to the need to simulate the course of a disease and the effect of treatments on it, producing estimates on final outcomes and costs in the long term that feed cost-effectiveness / cost-utility indicators, used, albeit with different logics, by various HTA agencies to define the economic value of a new technology and support price negotiations. In addition to cost-effectiveness / utility models, the simplest budget impact models find considerable space in this context, providing crucial information regarding the sustainability of the system. AIFA which analyzes and uses them within the investigations submitted to the Price and Reimbursement Committee (CPR) for the definition of the price and reimbursement conditions for drugs. These evaluations are also particularly recommended in those particularly complex decision-making areas, such as the case of orphan drugs / for rare diseases. in this work, the specific case of spinal muscular atrophy and the medicinal product risdiplam, as an example of a rare disease in which disruptive innovation has entered in recent years and where therefore the decision-makers have found themselves (and still are) to have to carefully evaluate the impacts both from a clinical point of view and from an organizational and economic point of view, in an HTA logic. The work carried out involved the development of three different types of analyzes whose integrated reading alone allows for an overall picture of the elements of allocative efficiency and sustainability impact required by AIFA for its evaluations of access to drugs. The firs

Introduction Drug reimbursement decisions are often made based on a price set by the manufacturer. In some cases, this price leads to public and scientific debates about whether its level can be justified in relation to its costs, including those related to research and development (R&D) and manufacturing. Such considerations could enter the decision process in collectively financed health care systems. This paper investigates whether manufacturers’ costs in relation to drug prices, or profit margins, are explicitly mentioned and considered by health technology assessment (HTA) organisations. Method An analysis of reimbursement reports for cancer drugs was performed. All relevant Dutch HTA-reports, published between 2017 and 2019, were selected and matched with HTA-reports from three other jurisdictions (England, Canada, Australia). Information was extracted. Additionally, reimbursement reports for three cases of expensive non-oncolytic orphan drugs prominent in pricing debates in the Netherlands were investigated in depth to examine consideration of profit margins. Results A total of 66 HTA-reports concerning 15 cancer drugs were included. None of these reports contained information on manufacturer’s costs or profit margins. Some reports contained general considerations of the HTA organisation which related prices to manufacturers’ costs: six contained a statement on the lack of price setting transparency, one mentioned recouping R&D costs as a potential argument to justify a high price. For the case studies, 21 HTA-reports were selected. One contained a cost-based price justification provided by the manufacturer. None of the other reports contained information on manufacturer’s costs or profit margins. Six reports contained a discussion about lack of transparency. Reports from two jurisdictions contained invitations to justify high prices by demonstrating high costs. Conclusion Despite the attention given to manuf

Background and aims: There is an alarming delay in Europe for anticancer medicines becoming accessible for children. Following a paediatric European Union marketing authorisation, national Health Technology Assessment (HTA) agencies evaluate effectiveness, and safety of medicines to support decision on their cost and reimbursement. This study (a SIOPE Access to Medicines project) aimed to evaluate how these HTA evaluations take place for anticancer medicines indicated for paediatric use in Europe and to explore where the delays for market access originate. Methods: We obtained HTA reports from the public domain for nine European countries for blinatumomab, dinutuximab beta and tisagenlecleucel. We evaluated the time elapsed between marketing authorisation for a paediatric indication and a national HTA decision and the nature of the decision. Results: Out of 23 HTA decisions (four countries without blinatumomab report), 18 were positive, two with restrictions, three negative. For blinatumomab, tisagenlecleucel and dinutuximab beta, the median time to an HTA decision after regulatory approval for paediatric use was 353 days (range 193–751), 141 days (range 77–517) and 515 days (range 0–780), respectively, with variability between countries. Dinutuximab beta and tisagenlecleucel were first introduced in children, but did not result in shorter time to HTA decision. For blinatumomab, marketing authorisation followed 1008 days after the indication in adults, with HTA applications submitted a median of 167 days later, and a recommendation after 145 days. Conclusions: This study reveals ample variability in HTA decision making in nine European Union countries. Collaboration and alignment of required evidence is needed to facilitate robust scientific HTA assessments, also considering methodological challenges in paediatric oncology.

Adequate methods are urgently needed to guarantee the good practice of health technology assessment (HTA) for technologies with novel properties. The aim of the study was to construct a conceptual framework to help understand the innovation of HTA methods (IHTAM). The construction of the IHTAM framework was based on two scoping reviews, one on the current practice of innovating methods, that is existing HTA frameworks, and one on theoretical foundations for innovating methods outside the HTA discipline. Both aimed to identify and synthesize concepts of innovation (i.e., innovation processes and roles of stakeholders in innovation). Using these concepts, the framework was developed in iterative brainstorming sessions and subsequent discussions with representatives from various stakeholder groups. The framework was constructed based on twenty documents on innovating HTA frameworks and fourteen guidelines from three scientific disciplines. It includes a generic innovation process consisting of three phases ("Identification," "Development," and "Implementation") and nine subphases. In the framework, three roles that HTA stakeholders can play in innovation ("Developers," "Practitioners," and "Beneficiaries") are defined, and a process on how the stakeholders innovate HTA methods is included. The IHTAM framework visualizes systematically which elements and stakeholders are important to the development and implementation of novel HTA methods. The framework could be used by all stakeholders involved in HTA innovation to learn how to engage dynamically and collaborate effectively throughout the innovation process. HTA stakeholders in practice have welcomed the framework, though additional testing of its applicability and acceptance is essential.

Background & Objective: The unprecedented value and long­term uncertainties of gene therapies have challenged established health technology assessment (HTA) methods. Real-world data (RWD) and real-world evidence (RWE) may mitigate the encountered uncertainties. However, empirical evidence appears to be lacking in the literature. This research aimed to provide this evidence and identify implications for EU-wide gene therapy joint clinical assessments. Methods: An initial literature review laid the theoretical foundation for the research. A retrospective comparative analysis delivered empirical evidence on RWD/RWE usage in gene therapy HTAs. Preliminary findings were probed in three use cases and verified in semi­structured interviews. Results: Nineteen HTA reports published by the HTA bodies G­BA (Germany) and NICE (England) were identified for the ten in­scope gene therapies. Whereas NICE had an average inclusion of 14 sources per HTA report (with an acceptance rate of 56%), G­BA had 8 (with 32% acceptance rate). While RWD/RWE supporting evidence on an external comparator had the lowest acceptance rate, RWD/RWE on intervention effectiveness had the highest acceptance rate. Insufficient information to substantiate the choice of RWD/RWE and inappropriate RWE study design was the most common exclusion rationales for RWD/RWE. Conclusions: The presented empirical evidence adds to the current knowledge base in two ways; it provides empirical evidence on HTAs of gene therapies (1) and RWD/RWE usage in gene therapy HTAs (2). This work provides insights into how RWD/RWE usage differs between HTA bodies and explicates relevant considerations for gene therapy manufacturers when using RWD/RWE in future HTA submissions. It also proposes that alignment on evidentiary requirements and assessment elements will optimise the transferability of future joint clinical assessment outcomes to national HTA bodies. Knowledge, Management of Technology (MoT)

open, The research activity during the doctorate was focused on an evaluation of HTA for a new drug for the treatment of heart failure. A first pharmaco-economic analysis was conducted before being placed on the market to demonstrate its cost-effectiveness, using data from randomized clinical trials and national rates and is part of the work carried out during the first year of the doctorate. Two new analyzes were subsequently carried out, which aim to demonstrate how pharmaceutical technology, once placed on the market, is still cost-effective and what its impact on the budget of the National Health Service is. In this case, real life data were used as efficacy data and national tariff rates for costs. The budget impact analysis makes it possible to obtain estimates on the economic sustainability of health technologies. The analysis aims to evaluate the financial impact on the Italian National Health Service (NHS) linked to the use of sacubitril / valsartan as therapy for heart failure in NYHA class II and III patients with reduced ejection fraction (FE <35%). The model estimates the target patient pool for the next three years starting from 2021. The target pool is then divided among the therapeutic options currently available. The second health and economic evaluation was conducted with the aim of examining the cost-effectiveness profile of sacubitril / valsartan (in combination with standard therapy), in comparison to enalapril (in combination with standard therapy), in the treatment of chronic heart failure with reduced ejection fraction in Italy A Markov decision model was developed to predict the effects of the two treatment options in comparison, in patients with chronic heart failure and with reduced ejection fraction, in terms of mortality (cardiovascular and all-cause), hospitalizations and quality of life. The model was adapted using data referable to a cohort of patients, specific for the Italian context, obtained from real life studies and subsequently subje, L’attività di ricerca durante il dottorato è stata focalizzata su una valutazione di HTA per uno nuovo farmaco per il trattamento dello scompenso cardiaco. Una prima analisi farmaco-economica era stata condotta prima dell’immissione in commercio per dimostrarne la costo-efficacia, utilizzando i dati degli studi clinici randomizzati e i tariffari nazionali ed è parte del lavoro svolto durante il primo anno di dottorato. Successivamente sono state svolte due nuove analisi, che hanno l’obiettivo di dimostrare come la tecnologia farmaceutica, una volta immessa nel mercato, sia ancora costo-efficace e quale sia il suo impatto sul budget del Servizio Sanitario Nazionale. Sono stati utilizzati in questo caso come dati di efficacia quelli di real life e per i costi i tariffari nazionali. L’analisi di impatto sul budget consente di ottenere stime sulla sostenibilità economica delle tecnologie sanitarie. L’analisi ha l’obiettivo di valutare l’impatto finanziario sul Servizio Sanitario Nazionale (SSN) italiano legato all’utilizzo di sacubitril/valsartan come terapia per lo scompenso cardiaco nei pazienti di classe NYHA II e III con frazione di eiezione ridotta (FE<35%). Il modello stima il pool di pazienti target per i prossimi tre anni a partire dal 2021. Il bacino target è poi suddiviso fra le opzioni terapeutiche attualmente a disposizione. La seconda valutazione economico-sanitaria è stata condotta con lo scopo di esaminare il profilo di costo-efficacia di sacubitril/valsartan (in combinazione con la terapia standard), in confronto ad enalapril (in combinazione con la terapia standard), nel trattamento dell’insufficienza cardiaca cronica con frazione d’eiezione ridotta in Italia È stato sviluppato un modello decisionale di Markov per predire gli effetti delle due opzioni di trattamento a confronto, nei pazienti con insufficienza cardiaca cronica e con frazione d’eiezione ridotta, in termini di mortalità (cardiovascolare e per tutte le cause), ospedalizzazioni e qualità di, 0, open, Ritrovato, D

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.

The goal of this work is to present i* framework as alternative to support design decisions in the Human Computer Interaction Area (HCI), mainly, in the process of task analysis. We show i* framework a modeling language suitable for an early phase of software system modeling to understand the problem domain, also, that provides methods to represent tasks, as well as support design decisions the development that satisfies the process requirements from the beginning until the end. To achieve our goal, approaches addressed to analyze notations aimed at task analysis and their representation. Finally, we present suggestions for improvement in the i* framework through a proposed extension for refining task model, instantiating examples and justifying our proposal through peer review.

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L’attività di ricerca durante il dottorato è stata focalizzata su una valutazione di HTA per uno nuovo farmaco per il trattamento dello scompenso cardiaco. Una prima analisi farmaco-economica era stata condotta prima dell’immissione in commercio per dimostrarne la costo-efficacia, utilizzando i dati degli studi clinici randomizzati e i tariffari nazionali ed è parte del lavoro svolto durante il primo anno di dottorato. Successivamente sono state svolte due nuove analisi, che hanno l’obiettivo di dimostrare come la tecnologia farmaceutica, una volta immessa nel mercato, sia ancora costo-efficace e quale sia il suo impatto sul budget del Servizio Sanitario Nazionale. Sono stati utilizzati in questo caso come dati di efficacia quelli di real life e per i costi i tariffari nazionali. L’analisi di impatto sul budget consente di ottenere stime sulla sostenibilità economica delle tecnologie sanitarie. L’analisi ha l’obiettivo di valutare l’impatto finanziario sul Servizio Sanitario Nazionale (SSN) italiano legato all’utilizzo di sacubitril/valsartan come terapia per lo scompenso cardiaco nei pazienti di classe NYHA II e III con frazione di eiezione ridotta (FE<35%). Il modello stima il pool di pazienti target per i prossimi tre anni a partire dal 2021. Il bacino target è poi suddiviso fra le opzioni terapeutiche attualmente a disposizione. La seconda valutazione economico-sanitaria è stata condotta con lo scopo di esaminare il profilo di costo-efficacia di sacubitril/valsartan (in combinazione con la terapia standard), in confronto ad enalapril (in combinazione con la terapia standard), nel trattamento dell’insufficienza cardiaca cronica con frazione d’eiezione ridotta in Italia È stato sviluppato un modello decisionale di Markov per predire gli effetti delle due opzioni di trattamento a confronto, nei pazienti con insufficienza cardiaca cronica e con frazione d’eiezione ridotta, in termini di mortalità (cardiovascolare e per tutte le cause), ospedalizzazioni e qua, The research activity during the doctorate was focused on an evaluation of HTA for a new drug for the treatment of heart failure. A first pharmaco-economic analysis was conducted before being placed on the market to demonstrate its cost-effectiveness, using data from randomized clinical trials and national rates and is part of the work carried out during the first year of the doctorate. Two new analyzes were subsequently carried out, which aim to demonstrate how pharmaceutical technology, once placed on the market, is still cost-effective and what its impact on the budget of the National Health Service is. In this case, real life data were used as efficacy data and national tariff rates for costs. The budget impact analysis makes it possible to obtain estimates on the economic sustainability of health technologies. The analysis aims to evaluate the financial impact on the Italian National Health Service (NHS) linked to the use of sacubitril / valsartan as therapy for heart failure in NYHA class II and III patients with reduced ejection fraction (FE <35%). The model estimates the target patient pool for the next three years starting from 2021. The target pool is then divided among the therapeutic options currently available. The second health and economic evaluation was conducted with the aim of examining the cost-effectiveness profile of sacubitril / valsartan (in combination with standard therapy), in comparison to enalapril (in combination with standard therapy), in the treatment of chronic heart failure with reduced ejection fraction in Italy A Markov decision model was developed to predict the effects of the two treatment options in comparison, in patients with chronic heart failure and with reduced ejection fraction, in terms of mortality (cardiovascular and all-cause), hospitalizations and quality of life. The model was adapted using data referable to a cohort of patients, specific for the Italian context, obtained from real life studies and subsequently subje

Background: Health care coverage decisions deal with health care technology provision or reimbursement at a national level. The coverage decision report, i.e., the publicly available document giving reasons for the decision, may contain various elements: quantitative calculations like cost and clinical effectiveness analyses and formalised and non-formalised qualitative considerations. We know little about the process of combining these heterogeneous elements into robust decisions. Methods: This study describes a model for combining different elements in coverage decisions. We build on two qualitative cases of coverage appraisals at the Dutch National Health Care Institute, for which we analysed observations at committee meetings (n=2, with field notes taken) and corresponding audio files (n=3), interviews with appraisal committee members (n=10 in seven interviews) and with Institute employees (n=5 in three interviews), and relevant documents (n=4). Results: We conceptualise decisions as combinations of elements, specifically (quantitative) findings and (qualitative) arguments and values. Our model contains three steps: 1) identifying elements; 2) designing the combinations of elements, which entails articulating links, broadening the scope of designed combinations, and black-boxing links; and 3) testing these combinations and choosing one as the final decision. Conclusions: Based on the proposed model, we suggest actively identifying a wider variety of elements and stepping up in terms of engaging patients and the public, including facilitating appeals. Future research could further explore how different actors perceive the robustness of decisions and how this relates to their perceived legitimacy.

Background. Common mental health disorders such as depression and anxiety have severe negative impact on mental wellbeing, quality of life and social and work-related functioning. E-Mental health (eMH) services such as Internet-based Cognitive Behaviour Therapy (iCBT), are proven to be effective in treating common mental health disorders. However, eMH services do not enter routine care as expected and their uptake in practice remains low, slow and costly. The difference between what is known and what is delivered in routine care, is one of the most critical issues in achieving effective, sustainable, and equitable mental health care. This dissertation aimed to contribute to understanding and reducing the research-to-practice gap in clinical mental health care. This thesis focussed on five research questions related to: decision-making in implementing eMH services, a taxonomy of barrier, characterisation of organisational implementation climate, the validity of NoMAD as an instrument to measure and monitor implementation outcomes, and the effectiveness of a toolkit for developing and applying tailored implementation strategies for implementing eMH services. Various methods were applied, including systematic reviews of existing literature, qualitative research using a concept mapping approach, a psychometric Confirmatory Factor Analysis, and an implementation effectiveness trial employing a Stepped Wedge cluster randomised study design with Linear Mixed Modelling with repeated measures. A substantial part of the research was carried out in an international context. Main findings. Implementation is preceded by a decision-making process. Various Health Technology Assessment (HTA) frameworks are adapted and used to assess various aspects of eMH services and by that, support systematic and evidence-informed decision making. In general, outcomes and instruments are not specified by the framework potentially limiting the applicability and comparability of assessments. Once

Objectives: To conduct a formative evaluation of applying the VALIDATE approach in practice by 1) assessing how students appreciated the e-learning course, 2) exploring how, for what purposes and with what outcomes the acquired VALIDATE competences subsequently were used in internships in different institutional contexts, and how this was shaped by these contexts, and 3) what this shows on real-world use of VALIDATE. Methods: Comparative discussion of experiences of applying the VALIDATE approach via a semistructured survey and oral feedback from e-course students; final reports on internships in HTA practice, followed by semi-structured interviews with interns and supervisors to complement and interpret results. Results: All students considered the VALIDATE approach an enlightening and important addition to current HTA knowledge, especially regarding understanding the relation between empirical analysis and normative inquiry, identifying policy relevant questions and using the method of reconstructing interpretive frames for scoping. The latter appeared intellectually challenging and requiring some prior HTA knowledge. The use the VALIDATE approach in practice shows that interns productively re-defined the HTA problem, based on appreciation of different stakeholders definition of the issue; they experienced constraints from retrieving all relevant perspectives from existing literature as well as from institutional rules and routines. Conclusions: Some challenges in applying the VALIDATE approach deserve attention for its future use: currently used research approaches often assume a problem as given ; and the data needed on different perspectives is often not reported in scientific literature. Finally, data gathering on and evaluation of value dimensions was experienced as challenging.

Background: Health Technology Assessment (HTA) is a multidisciplinary process that synthesizes, with a systematic, transparent, impartial and robust methodological approach, the main information on the medical, economic, ethical and social implications of the use and dissemination of a health technology. Its aim is to support decision-makers in identifying safe, effective, patient-centered and best-value health policies, in order to promote an equitable, efficient, and high-quality health system. Given the continued application of innovative technologies into clinical practice, healthcare professionals need to be able to adequately evaluate these technologies using evidence-based approaches such as HTA. Therefore, the implementation of training in HTA is crucial. The aim of this study was to investigate existing HTA training initiatives for healthcare professionals provided by international HTA agencies and organizations around the world. Methods: From March to November 2020, the websites of HTA agencies and organizations belonging to the European network for HTA (EUnetHTA) and to the International Network of Agencies for HTA (INAHTA), and the website of the HTA International (HTAi), were explored for identifying the HTA training initiatives directed to healthcare professionals. In addition, we screened the training initiatives proposed at European level by EUnetHTA as part of its Joint Actions and conducted in collaboration with its public-private partners. Specific keywords were searched in English and adapted to French, Portuguese, Spanish, Italian and German. Data extraction of the retrieved training initiatives was conducted from November 2020 to February 2021 and considered the following information: agency, country, website, coordinator, type of initiative, target, topic, main contents, and language. Results: Out of 124 agencies/organizations/EUnetHTA public-private partners screened, only 21 provided training initiatives for healthcare professionals. A total

Background. The elderly, commonly defined as subjects aged ≥65 years, are among the at-risk subjects recommended for annual influenza vaccination in European countries. Currently, two new vaccines are available for this population: the MF59-adjuvanted quadrivalent influenza vaccine (aQIV) and the high-dose quadrivalent influenza vaccine (hdQIV). Their multidimensional assessment might maximize the results in terms of achievable health benefits. Therefore, we carried out a Health Technology Assessment (HTA) of the aQIV by adopting a multidisciplinary policy-oriented approach to evaluate clinical, economic, organizational, and ethical implications for the Italian elderly. Methods. A HTA was conducted in 2020 to analyze influenza burden; characteristics, efficacy, and safety of aQIV and other available vaccines for the elderly; cost-effectiveness of aQIV; and related organizational and ethical implications. Comprehensive literature reviews/analyses were performed, and a transmission model was developed in order to address the above issues. Results. In Italy, the influenza burden on the elderly is high and from 77.7% to 96.1% of influenza-related deaths occur in the elderly. All available vaccines are effective and safe; however, aQIV, such as the adjuvanted trivalent influenza vaccine (aTIV), has proved more immunogenic and effective in the elderly. From the third payer’s perspective, but also from the societal one, the use of aQIV in comparison with egg-based standard QIV (eQIV) in the elderly population is cost-effective. The appropriateness of the use of available vaccines as well as citizens’ knowledge and attitudes remain a challenge for a successful vaccination campaign. Conclusions. The results of this project provide decision-makers with important evidence on the aQIV and support with scientific evidence on the appropriate use of vaccines in the elderly.

Standard outcome sets developed by the International Consortium for Health Outcomes Measurement (ICHOM) facilitate value-based health care in healthcare practice and have gained traction from regulators and Health Technology Assessment (HTA) agencies that regularly assess the value of new medicines. We aimed to assess the extent to which the outcomes used by regulators and HTA agencies are patient-relevant, by comparing these to ICHOM standard sets. We conducted a cross-sectional comparative analysis of ICHOM standard sets, and publicly available regulatory and HTA assessment guidelines. We focused on oncology due to many new medicines being developed, which are accompanied by substantial uncertainty regarding the relevance of these treatments for patients. A comparison of regulatory and HTA assessment guidelines, and ICHOM standard sets showed that both ICHOM and regulators stress the importance of disease-specific outcomes. On the other hand, HTA agencies have a stronger focus on generic outcomes in order to allow comparisons across disease areas. Overall, similar outcomes are relevant for market access, reimbursement, and in ICHOM standard sets. However, some differences are apparent, such as the acceptability of intermediate outcomes. These are recommended in ICHOM standard sets, but regulators are more likely to accept intermediate outcomes than HTA agencies. A greater level of alignment in outcomes accepted may enhance the efficiency of regulatory and HTA processes, and increase timely access to new medicines. ICHOM standard sets may help align these outcomes. However, some differences in outcomes used may remain due to the different purposes of regulatory and HTA decision-making.

This chapter is focused on the so-called “fact/value dichotomy”. In the current practice of HTA, value judgments are often considered external to HTA or separate from it and are addressed by experts (in ethics) and decision makers after the “real” HTA is finished. In other words, facts and values seem to be two completely different “substances” that come from two completely different sources. Is this dichotomy attainable? 2) is it desirable? This chapter will argue that a strict fact-value distinction is not so easy to maintain, based on insights from Philosophy and HTA practice. Key messages of this chapter: HTA cannot be considered a matter of collecting the facts about a certain health technology, but rather “a matter of collecting facts that are considered plausibly associated with the use of the technology, relevant, and amenable to accepted methods of scientific inquiry”.

The COVID-19 pandemic is challenging how healthcare technologies are evaluated, as new, more dynamic methods are required to test the cost effectiveness of alternative interventions during use rather than before initial adoption. Currently, health technology assessment (HTA) tends to be static and a priori: alternatives are compared before launch, and little evaluation occurs after implementation. We suggest a method that builds upon the current pre-launch HTA procedures by conceptualizing a mean-variance approach to the continuous evaluation of attainable portfolios of interventions in health systems. Our framework uses frontier analysis to identify the desirability of available health interventions so decision makers can choose diverse portfolios based upon information about expected returns and risks. This approach facilitates the extension of existing methods and assessments beyond the traditional concern with pre-adoption data, a much-needed innovation given the challenges posed by COVID-19.

El objetivo de este estudio es el de conocer si la propuesta de un panel de control optimizado reduce el nivel de carga mental al compararlo con el panel de control actual de un centro de lavado Frigidaire mediante un análisis jerárquico de tareas (HTA) en conjunto con un estudio TAFEI y un estudio NASA-TLX. Como resultado de este trabajo, en el que participaron 20 personas de Ciudad Juárez, Chihuahua, México, se encontró que la tarea de lavado de ropa y selección de ciclo con el panel de control actual genera un nivel de carga mental de 89 y 53 puntos al utilizar el panel de control actual. No se han encontrado otros estudios en los que se realice un análisis de carga cognitiva de paneles de control de los centros de lavado. En base a los resultados se puede determinar que los usuarios sienten una mejora en el nivel de carga mental de 28 % y 45 % con las nuevas propuestas de paneles de control.

The definition of criteria and processes for the submission of price and reimbursement requests (P&R) of a drug in Italy cannot be separated from the definition of an overall “Pharmaceutical Policy” that includes, in an organic vision: (i) the governance related to the research, marketing and monitoring of drugs in the Italian market; (ii) the availability of drugs on the national territory as an element included in the essential levels of care (LEA) and the related conditions in terms of timing and equity of access between different regions, as well as towards other European countries; (iii) the assessment criteria used. The decree published in the Italian Official Journal in the summer of 2020, which defines the new criteria for the regulation of P&R of medicines in Italy, focuses on the final part of the process, i.e. the price negotiation. It would be necessary to frame this last step within a broader and more organic structure of drug policies aimed at: 1. optimising healthcare funding by encouraging competition between healthcare technologies; 2. reducing assessment time by simplifying processes; 3. improving early access to drugs for unmet need; 4. increasing the quality of P&R dossiers by improving interaction with the companies; 5. encouraging innovative agreements and complementary elements to the price; 6. encouraging the reproducibility of assessment methodologies in a value-based pricing system; 7. considering cost-benefit analyses as tools for the definition of price and conditions of reimbursability; 8. creating a place for discussion on drug policies.

The introduction of modern Information and Communication Technologies (ICT) was one of the most remarkable innovations of recent decades. ICT brings with it a remarkable technological background that conveys all kinds of information and multimedia content with a significant change in human-technology interaction and significant implications also in the health sector. The constant process of digitization is increasingly affecting national health systems (SSN) and they turn out to be influenced by the process itself, where the literature shows itself in favor of the use of technologies in health, improving their effectiveness and efficiency. These include eHealth, Telemedicine, Electronic Health File, Big Data, Virtual Reality, Augmented Reality, ePrescription. The technologies allow, even remotely, to have an always active and direct contact, between the various professionals, and between professionals and users, and are also useful for the training of both healthcare professionals and users themselves. The use of technology in the healthcare sector should therefore be encouraged as it allows direct contacts between users and healthcare personnel, speed and correlation of data analysis, tracking, time and cost savings, reduction of errors and a positive environmental impact with a reduction in the use of printed paper. For all the points listed, the technological revolution in hospital and territorial care can no longer be postponed., The introduction of modern Information and Communication Technologies (ICT) was one of the most remarkable innovations of recent decades. ICT brings with it a remarkable technological background that conveys all kinds of information and multimedia content with a significant change in human-technology interaction and significant implications also in the health sector. The constant process of digitization is increasingly affecting national health systems (SSN) and they turn out to be influenced by the process itself, where the literature shows itself in favor of the use of technologies in health, improving their effectiveness and efficiency. These include eHealth, Telemedicine, Electronic Health File, Big Data, Virtual Reality, Augmented Reality, ePrescription. The technologies allow, even remotely, to have an always active and direct contact, between the various professionals, and between professionals and users, and are also useful for the training of both healthcare professionals and users themselves. The use of technology in the healthcare sector should therefore be encouraged as it allows direct contacts between users and healthcare personnel, speed and correlation of data analysis, tracking, time and cost savings, reduction of errors and a positive environmental impact with a reduction in the use of printed paper. For all the points listed, the technological revolution in hospital and territorial care can no longer be postponed.

La severidad de la Covid-19 se ve influenciada por la genética del huésped. Conocer los mecanismos que rigen esa interacción puede tener múltiples aplicaciones, ya sean dianas para nuevos fármacos o los factores genéticos que definen la respuesta individual a la infección. Para ello, en primer lugar, se llevó a cabo un análisis del GWAS del consorcio SCOURGE en el que se halló relación estadísticamente significativa entre la severidad de la Covid-19 y la edad avanzada (p < 2.2e-16), el sexo masculino (p < 2.2e-16), la obesidad (p < 2.2e-16), la EPOC (p = 7.80e-09), la diabetes mellitus (p = 6.72e-10) y la HTA (p = 7.52e-15). A continuación, se buscó una hipótesis sobre la base genética etiopatogénica común entre la gravedad provocada por el Covid-19 y las enfermedades relacionadas a través de una exploración de la bibliografía existente. Se consideró la inflamación crónica de bajo grado como el nexo entre la etiopatogenia genética de las diferentes patologías y se elaboró una puntuación de riesgo poligenético (PRS) con loci implicados en su actividad. El análisis de las diferencias para este PRS fue significativo en menores de 65 años en los análisis “casos/resto” en hospitalizados (p = 0.03861) y en pacientes con fenotipo grave y crítico de Covid-19 (p = 0.03401), así como, en los análisis “casos/población general” en hospitalizados (p = 0.04883) y en pacientes con fenotipo grave y crítico de Covid-19 (p = 0.03675). Los resultados sugieren relación entre la inflamación crónica de bajo grado y la severidad de la Covid-19, aportando una información valiosa para estudios posteriores

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ackground and Aim: Digital health technologies (DHTs) are one of the most promising perspectives in healthcare. On the other hand, the investment in of DHTs has an impact on the (ethical, economic, organizational, environmental) sustainability of National Healthcare Systems (NHSs). A relevant tool for supporting healthcare decision-makers is Health Technology Assessment (HTA), ‘a multidisciplinary process that summarizes information about the medical, social, economic and ethical issues related to the use of a health technology in a systematic, transparent, unbiased, robust manner’ (EUnetHTA 2007). The aim of our contribution is to deal with HTA around DHTs, particularly ethical, legal and social implications (ELSI). Materials and Methods: The literature/documentation review on HTA and DHTs was carried out on PubMed database through a query, merging the following MESH terms: “digital technolog*”; healthcare; health technology assessment; HTA (last access. August 17, 2021). Results: 90 items were found: 4 were considered relevant for our analysis. The literature on our focus is at the very beginning. Early contributions underline as follows: 1. current HTA frameworks are not well equipped for assessing DHTs. The development of DHT-specific content for HTA frameworks is hampered by DHTs having varied benefit and risk profiles (von Huben et al., 2021); 2. existing HTA process should be adapted for assessing DHTs, including to make provisions for an assessment of app cybersecurity, the impact on DHT clinical utility of software updates, and compatibility issues (Moshi et al., 2020) as well as the need for standardization of reporting characteristics of eHealth services, and specifying assessment outcomes and methods following a stepped-approach tailored to the functional characteristics of eHealth services (Vis et al., 2020); 3. attention should also be paid to ethical issues, such as data privacy, and technology specific characteristics (Moshi et al., 2018).

This workshop will deepen understanding the role of Hospital Based HTA on the decision making; participants will learn the steps for developing an assessment in hospital-based context and tools to perform a rapid assessment; it will be presented the implementation process of its recommendations. For each section there is an analysis of the impact of the pandemic. The coronavirus pandemic has changed the global scenario regarding healthcare and the use of tools such as HB-HTA. Operational processes have become much faster and there has been a greater need for information on technologies for diagnosing and treating COVID. Which of these had a rationale, some potential evidence, which impact on the organization and which costs. All questions to which the Hospital Based HTA was asked to give answers that required its reorganization both of a methodological and operational type. The workshop aims to illustrate these changes resulting from real experiences lived in different parts of the globe. 1. Main role of HB-HTA – functions 2. Has HB-HTA role changed as a consequence of the pandemic? 3. Steps for a HB-HTA, relying on AdHopHtA 4. Rapid Assessment? Criteria 4.a. Assessment of clinical outcomes, evidence level and safety 4.b. Assessment with real world HTA 4.c. Assessment of patient perception 4.d. Assessment of economic consequences 4.e. Assessment of organizational consequences 4.f. Assessment of other potential aspects: Strategical, legal, ethical etc. 4.g. How to ensure the quality of HB HTA? 5. Implementation process of the HB-HTA recommendations into the hospital decision making. Impact of pandemic 6. Discussion and closing • To give the participants: - an understanding of how HB HTA can be organized, produced and implemented at hospitals as the basis for decisions on investment in new health technologies - skills to produce high quality HB HTAs on their hospitals on the basis of the evidence from the AdHopHTA project. • To show potentials of HB-HTA to push for va

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The need to optimize drug development and facilitate faster access for patients has ignited discussions around the importance of improving interactions between health technology assessment (HTA) bodies and regulatory agencies. In this study, we conducted a systematic review to examine processes, progress, outcomes, and challenges of harmonization/interaction initiatives between HTA bodies and regulatory agencies. MEDLINE, EMBASE, and the International Pharmaceutical Abstracts database were searched up to 21 October 2019. Searches for gray literature (working papers, commissioned reports, policy documents, etc.) were performed via Google scholar and several institutional websites. An online cross-sectional survey was also conducted among HTA (n = 22) and regulatory agencies (n = 6) across Europe to supplement the systematic review. Overall, we found that while there are areas of divergence, there has been progress over time in narrowing the gap in evidentiary requirements for HTA bodies and regulatory agencies. Most regulatory agencies (4/6; 67%) and half (11/22, 50%) of the HTA bodies reported having a formal link for "collaborating" with the other. Several mechanisms such as early tripartite dialogues, parallel submissions (reviews), adaptive licensing pathways, and postauthorization data generation have been explored as avenues for improving collaboration. A number of pilot initiatives have shown positive effects of these models to reduce the time between regulatory and HTA decisions, which may translate into faster access for patients to life-saving therapies. Thus, future approaches aimed at improving harmonization/interaction between HTA bodies and regulatory agencies should build on these existing models/mechanisms while examining their long-term impacts. Several barriers including legal, organizational, and resource-related factors were also identified, and these need to be addressed to achieve greater alignment in the current regulatory and reimbursement

Introduction: The new European Union (EU) Regulations on medical devices and on in vitro diagnostics provide manufacturers and Notified Bodies with new tools to improve pre-market and post-market clinical evidence generation especially for high-risk products but fail to indicate what type of clinical evidence is appropriate at each stage of the whole lifecycle of medical devices. In this paper we address: i) the appropriate level and timing of clinical evidence throughout the lifecycle of high-risk implantable medical devices; and ii) how the clinical evidence generation ecosystem could be adapted to optimize patient access. Areas covered: The European regulatory and health technology assessment (HTA) contexts are reviewed, in relation to the lifecycle of high-risk medical devices and clinical evidence generation recommended by international network or endorsed by regulatory and HTA agencies in different jurisdictions. Expert opinion: Four stages are relevant for clinical evidence generation: i) pre-clinical, pre-market; ii) clinical, pre-market; iii) diffusion, post-market; and iv) obsolescence & replacement, post-market. Each stage has its own evaluation needs and specific studies are recommended to generate the appropriate evidence. Effective lifecycle planning requires anticipation of what evidence will be needed at each stage

The evaluation of medicines and medical technologies – also called Health Technology Assessment (HTA) – underpins market access, price negotiations and reimbursement policies of health technologies. In Europe, HTA is characterised by a diversity of methodologies and practices. European HTA networks have sought to establish a sustainable cooperation framework to reach convergence. This process has led to a European Commission proposal for a Regulation in HTA cooperation which adoption process is still running. HTA cooperation has been analysed in relation to European health policy. As EU competences regarding HTA are limited to support-lending and coordination policies, the thesis has examined to what extent cooperation and convergence of European HTA practices can be structured through soft governance applied in networks. Factors favouring convergence in this area have been identified. The conclusions of this thesis bring to the fore that soft governance, by offering flexibility and gradualism, can be a powerful instrument to instil convergence in policy areas of limited EU competences. Lack of effectiveness of soft governance in HTA cooperation processes has been attributed to the manner in which soft governance instruments have been implemented rather than inherent weaknesses of soft governance itself. The thesis