The ‘Online elicitation of Personal Utility Functions’ (OPUF) is a new method for valuing health states. It is based on compositional preference elicitation techniques. In contrast to established, decompositional techniques, such as time trade-off or discrete choice experiment (DCE), the OPUF approach does not require hundreds or thousands of respondents, but allows estimating utility functions for small (patient) groups and even on the individual level. The objective of this study was to generate and compare EQ-5D-5L value sets on the societal-, group-, subgroup-, and individual-level.

A demonstration version of the EQ-5D-5L OPUF Tool is available at: https://eq5d5l.me. It broadly consists of three valuation steps: dimension weighting, level rating, and anchoring. Responses were combined on the individual level to construct personal utility functions. Every respondent also completed three conventional DCEs. Preferences were aggregated across individuals to estimate a societal and various group-level preference functions. We then assessed the heterogeneity of preferences between groups using descriptive statistics and k-means cluster analysis.

A representative sample (n = 1,000) of the United Kingdom (UK) population was recruited through the prolific online platform. On average, it took participants about 7 minutes to complete the survey. Data of 874 respondents were included in the analysis. For each respondent, we constructed a personal EQ-5D-5L value set. The derived utility functions predicted respondents’ choices in DCE with an accuracy of 78 percent. On the societal level, the predicted values for the EQ-5D-5L health states ranged from -0.376 to 1. Health state preference varied greatly between groups. This was largely due to differences in the anchoring, while there was near consensus on the relative importance of the five EQ-5D dimensions between groups. Demographic characteristics explained only a small proportion of the variability.

Using the OPUF approach, we were not only able to estimate a new EQ-5D-5L value set for the UK, but also to examine the underlying individual preferences in an unprecedented level of detail.

The SF-6D is a preference-based measure of health, derived from the SF-36 for economic evaluation. No value set exists for the SF-6D in Lebanon and other Arabic speaking countries in the Middle East. The aim of this study was to examine the feasibility and acceptability of using the standard gamble (SG) technique to generate preference-based values for the Arabic version of SF-6D in a Lebanese population.

The SF-6D was translated into Arabic using forward and backward translations. Forty-nine states defined by the SF-6D were selected using an orthogonal design and grouped into seven sets. A gender-occupation stratified sample of 126 Lebanese adults from the American University of Beirut were recruited to value seven states and the pits (worst) SF-6D health states using SG. Mean and individual level multivariate regression models were fitted to estimate preference weights for all SF-6D states. The quality of data and the predictive power of the models were compared with results from the United Kingdom (UK).

All respondents completed the interviews with 25 percent reporting that the SG task was difficult and 21 percent felt some degree of irritation or boredom. A total of 992 (98% out of 1,008 observations) SG valuations were useable for econometric modeling. There was no significant change in the test–retest values of 21 subjects. The mean absolute errors in the mean and individual level models were 0.036 and 0.050, respectively, both of which were lower than the UK results. The random effects model adequately predicts the SG values, with the worst state having a value of 0.322 compared to 0.271 in the UK.

This study confirmed that it was feasible and acceptable to generate preference values with the SG method for the Arabic SF-6D in a Lebanese population. This would be the first step towards developing SF-6D value set for Lebanon to be used in economic evaluation studies and to support resources allocation decisions.

A patient/person-reported outcome measure (PROM) is a questionnaire that measures patient’s health self-assessment (PROs), such as pain/wellbeing. HTx is a Horizon 2020 project supported by the European Union lasting for 5 years from January 2019. The main aim of HTx is to create a framework for the Next Generation Health Technology Assessment (HTA) to support patient-centred, societally oriented, real-time decision-making on access to and reimbursement for health technologies (HTs) throughout Europe. Individual values, needs, and preferences should be taken into account when appraising HTs and in clinical decision-making. Better guidance is needed about how to develop and select generic and/or more specific outcome measures that are relevant to patients and fit-for-purpose. Our goal is to provide that guidance via the PROM toolbox and enable users to select PROMs by using this. The HTx project has received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement N° 825162.

A framework for the assessment of PROMs was discussed with experts, tested for a subset of PROMs (Linnean menu) and resulted in the PROM-cycle. Multiple literature studies were performed on PROMs, their properties, and characteristics. The general population was studied as well as cancer, diabetes, multiple sclerosis, myelodysplastic syndrome, and (long-lasting) COVID-19. This resulted in an Excel database (PROM-overview) made available via a user-friendly web-application helping users to select PROMs: the PROM select app via https://www.prom-select.eu/proms.

Multiple tools were developed and are available in the PROM toolbox starting with the PROM-guide, Literature review on the use of PROMs, PROM-cycle accompanied by the Linnean menu. In addition, PROM-links, The making of the PROM-overview & PROM-select app, PROM-overview, PROM-select app, its usability test report and PROM toolbox presentation can be found on https://www.zorginzicht.nl/ondersteuning/prom-toolbox-summary-in-english#what-is-the-prom-cycle.

The PROM toolbox guides users and helps them to select PROMs and trustworthy information and finding relevant scientific articles.

The demands for incorporating or excluding health technologies within the Brazilian Public Health System (SUS) can be requested by different stakeholders, including public administration, pharmaceutical companies, specialists’ and patients’ organizations/associations. The Brazilian National Committee for Health Technology Incorporation (CONITEC), part of the Ministry of Health, is the responsible organ to evaluate these demands and emit recommendations. The aim of this study is to show an overview of stakeholders according to the type of technology under request.

On 26 November 2021, a search was performed at CONITEC website. Health Technology Assessment (HTA) incorporation reports from 1 January to 26 November 2021 were extracted. From these reports, data regarding the demanded technology (e.g., medications, diagnostic tests, etc.), demand (technology incorporation, exclusion, or alteration), pharmacological classification according with the Anatomical Therapeutic Chemical (ATC), and requester’ categorization (e.g., pharmaceutical companies, official administration organs, etc.).

Preliminary results showed a total of 77 health technologies, in 63 HTA incorporation submissions, of which 87 percent (n = 67) were medications, and 9 percent (n = 6) were new medical procedures. Only one medical device, one vaccine and two diagnostic tests were requested. Technology incorporation accounted for 94.8 percent of the demands (73), and 4 demands of exclusion. Seventy-one percent of the requested medications were classified, according to first ATC coding level, within groups L (Antineoplastic and immunomodulating agents), N group (Nervous system), A group (Alimentary tract and metabolism), and R group (Respiratory system), accounting 28.3, 17.9, 8.9 and 8.9 percent, respectively. Regarding stakeholders, the Brazilian Ministry of Health and its associated departments were responsible for 57.1 percent of the demands, while pharmaceutical companies requested 37.6 percent of incorporations. Other requesters included the Federal Justice Department, patients’, and specialists’ organizations, summing up with only four demands.

These results present the numerical weight of stakeholders in the Brazilian HTA incorporation system, with special attention to Brazilian bureaucracy and pharmaceutical companies. Further analysis regarding association between demand and other variables such as budgetary impact, costs, and ICD-10, shall deepen our understanding of different stakeholders’ role in Brazilian HTAi.

Multi-stakeholder engagement in the development and evaluation of medical devices is crucial for aligning devices with stakeholders’ views, needs, and values. Focus groups, interviews, and surveys are often used to involve stakeholders, but these methods have rarely been compared to analyse their relative merits. Therefore, we systematically compared these three methods in terms of themes, interaction, and feasibility.

The methods were compared in a case-study on surgery with a new endoscopic device for patients with intracerebral haemorrhage. We asked patients, relatives, healthcare professionals and decision-makers about their perspective on this device, and about their perceived quality of hospital care. We conducted the focus groups and interviews in one explorative and one interactive round. The comparison was made in terms of number and content of themes, who and how participants interact, and in terms of hours that needed to be worked by researchers to apply a method.

We enrolled 18 participants in the focus groups, 17 in the interviews and 43 in the survey. Focus groups generated 31 and 19 themes, and interviews 58 and 40 themes in the explorative and interactive round. Surveys generated 42 themes. Interviews produced various themes about the device that did not occur in the other methods. In the two rounds of the focus group, 13 and 42 percent of the interactions were directly between participants. In interview round one, 98 percent of the interactions were between the interviewer and participant, whereas 80 percent of the interactions in round two were discussions of other participants’ opinions. In focus groups participants were inclined to emphasise agreement, whereas the interviews generated more in-depth discussions. Interviews took three times as many hours as the focus groups and survey.

Methods for multi-stakeholder involvement in device development vary considerably. These methodological differences should be taken into account when selecting a method for engaging stakeholders in the early stages of the lifecycle of a medical device.

The Institut national d’excellence en santé et en services sociaux (INESSS) makes recommendations regarding introduction, coverage, optimal use or withdrawal of interventions in physical and mental health and social services to support fair and reasonable decisions. The objectives of this work were to develop a statement of principles and ethical foundations for a common appraisal framework across diverse interventions, to enhance legitimacy and coherence of evaluation practices, and develop reflective approaches throughout the institution to operationalize the principles.

To develop this statement, INESSS reviewed its practices across different units, surveyed the literature on innovative practices and the evolution of HTA, and undertook an extensive internal and external consultative process. The principles are used to develop reflective activities as part of a continuous improvement strategy.

The adopted approach to value appraisal considers the contributions of interventions to the Triple Aim of health and social services systems as well as their organizational and sociocultural feasibility and impacts (clinical, populational, economic, organizational, sociocultural dimensions). This approach is articulated around five principles including: (i) evaluating the most relevant objects and adapting evaluation modalities; (ii) mobilizing and integrating diverse types of knowledge; (iii) supporting multidimensional deliberation including diverse perspectives; (iv) developing fair and reasonable recommendations; (v) promoting value creation by supporting the implementation of recommendations and re-evaluation. Although all principles contribute to the legitimacy and credibility of the recommendations, which we aim to implement and consolidate through a set of activities, deliberation is an important part of the process that we are striving to improve. A first set of reflective activities are planned to support its operationalization, including: materials to promote a common understanding of the diverse aspects of the deliberation, reflective workshops on selected past projects, and sharing emerging reflections across INESSS units to further continuous improvement in operationalizing the principles.

Moving forward, INESSS’s strategic intention is to mobilize its staff and collaborators to facilitate the rigorous, agile and coherent application of these principles.

There are many challenges encountered in the health technology assessment (HTA) of treatments for the management of neurodegenerative diseases like Alzheimer’s disease. A recent example where such challenges were visible was the assessment of aducanumab, a medication developed for Alzheimer’s disease. As part of the Innovative Medicines Initiative (IMI) Neuronet project, a series of workshops were held to understand the gaps in neurodegenerative disease research and identify the key research priorities, from the perspective of different stakeholders, that can inform decision-making and enable timely access to effective and safe treatments.

Three workshops were organised by Neuronet to consult with HTA and regulatory experts, people with dementia and their supporters, and academic and industry researchers. The first consultation was with the European Working Group of People with Dementia and their supporters, and their feedback was fed into the following two workshops which were conducted with HTA and regulatory experts, and with researchers. Discussions during the workshops were used to develop a summary of the gaps in research, and priorities from the perspective of each stakeholder group to be submitted to IMI to inform future research funding allocation.

Key priorities that were highlighted from an HTA and regulatory perspective were the collection of longitudinal data to understand the natural history of the disease, the generation of data on long-term clinical outcomes and the use of trial endpoints that are relevant to clinical practice. There is also a need for disease-modifying treatments that is yet to be met in this disease area. Furthermore, the involvement of the public, particularly people with dementia and relevant organisations, in setting research priorities and informing study designs was highlighted as a priority by all stakeholders.

There are key priorities for future neurodegenerative research from an HTA and regulatory perspective to address current gaps and challenges. It is important to involve different stakeholders in priority setting and decision-making to produce relevant and satisfactory research outputs.

In the field of drug development and effectiveness evaluation, the use of surrogate endpoints is acceptable if they reliably predict a positive effect on clinical outcomes such as mortality or morbidity. Hemoglobin A1c (HbA1c) is a widely used surrogate endpoint in phase 3 and 4 clinical trials evaluating drugs in patients with diabetes mellitus (DM). The objective of this study was to investigate whether HbA1c is a valid surrogate endpoint for evaluating the effectiveness of antihyperglycemic drugs in DM trials.

We conducted a systematic review of randomized placebo-controlled trials evaluating the effect of a treatment on levels of HbA1c and clinical outcomes in patients with DM. The association between the effects of treatment on HbA1c levels and clinical outcomes was then investigated using regression analysis at the trial level. The correlation coefficients (R) were interpreted using the cut-off points suggested by the German Institute for Quality and Efficiency in Health Care (IQWiG). HbA1c was considered a valid surrogate endpoint if it demonstrated a strong association with clinical outcomes (i.e., the lower limit of the 95% confidence interval [CI] of R≥ 0.85).

Nineteen phase 3 or 4 randomized controlled trials (RCTs) were identified. All studies included adults with type 2 DM. None of the associations evaluated was strong enough to validate HbA1c as a surrogate endpoint for any clinical outcome: mortality (R 0.34, 95% CI: -0.14, 0.69); myocardial infarction (R 0.20, 95% CI: -0.30, 0.61); heart failure (R 0.08, 95% CI: -0.40, 0.53); kidney injury (R -0.04, 95% CI: -0.52, 0.47); and stroke (R 0.81, 95% CI: 0.54, 0.93).

The evidence from multiple placebo-controlled RCTs of antihyperglycemic drugs in patients with type 2 DM suggests that a reduction in levels of HbA1c does not meet the IQWiG criteria for a valid surrogate endpoint. Consequently, the risk-benefit ratio of antihyperglycemic drugs in terms of patient relevant clinical outcomes, regardless of their hypoglycemic effect, should be the focus of therapeutic, regulatory, and reimbursement decisions.

With the aim of reducing patient travel and related costs, physiotherapists and occupational therapists at the Oslo University Hospital began offering video consultations to patients with hand and arm injuries in March 2020. A feasibility study was initiated to describe the first year of using video consultations in the rehabilitation of upper extremity injuries in children and adults, and to assess the acceptability of the service from the perspective of hand therapists. The secondary objective of the study was to investigate the economic effects of using video consultation for this patient group.

The therapists documented the individual consultations in an Excel spreadsheet. Utility and acceptability were analyzed based on the content of each video consultation. The therapists also registered the patient’s municipality in order to calculate costs related to travel, accommodation, and other costs related to in-person consultation. Utility was analyzed using an early economic model based on scenario analysis to compare the costs of video consultations with in-person consultations.

Based on the content analysis from 89 consultations, video consultations were deemed acceptable by the therapists. The total travel distance from patients’ homes to the hospital was 20,190 km, as hand rehabilitation is a national service. The video consultations that replaced the consultations at the hospital were potentially more time efficient, reduced patients’ travel time and absence from home and work, and saved costs for the Oslo University Hospital and society.

Based on early decision support provided by this study, adaptions were made to the delivery of video consultations to improve the cost effectiveness of the service. The findings from this study provided an indication of the potential value of the new service, which may be used for benchmarking purposes to ensure that it meets the needs of users, the health service, and society.

People with a traumatic brain injury and who present with an unsafe or problematic substance use of psychoactive substances (PAS) face specific challenges in social integration resulting from the negative impacts of substance use on the benefits of rehabilitation services and treatments.

A systematic review of grey and scientific literature was conducted. The selection and coding of the included interventions (according to their characteristics), as well as descriptive analyses, resulted in findings which describe the interventions under study. Grades of evidence strength were attributed to the findings based on a range of factors including the methodological characteristics of the studies. Findings resulting from the systematic review were contextualized with information collected from administrative databases, clinical program documentation, and experiential data obtained from rehabilitation professionals. Recommendations were elaborated based on the aggregated data sources and informal deliberations by a committee involving researchers and clinicians.

Out of 5014 selected references, 14 studies examining 13 interventions which targeted social integration of the management of PAS were included. Interventions with the strongest evidence base are user-centered (i.e., support individual choices and personal autonomy), individualized (i.e., considered individual needs and characteristics), and educational (i.e., support the acquisition of strategies and abilities). The evidence base was considered “emerging” for interventions which are systemic in nature (i.e., relate to familial, social, cultural, educational or professional environments), interdisciplinary (i.e., involve clinicians with distinct training, competences and expertise), and ecological (i.e., which involve interventions in the client’s living settings). While additional research is necessary to better support their efficacy, these interventions exhibited promising effects on various outcomes.

The analysis and synthesis of three streams of information resulted in five clinical practice recommendations relating to integration to school and productive activities, psychological adaptation, integration in living settings and independence, social life and sense of belonging, and finally, use of PAS. Recommendations should help support clinicians in rehabilitation settings.

Unadjusted effect modifiers (EM) in indirect treatment comparisons (ITC) can produce biased and uncertain results in health technology assessments (HTA). Even though research on advanced ITC methods to adjust for EMs has attracted much attention, less emphasis has been placed on how these EMs are identified and assessed in the first place. This is surprising given the evidence-based approach underpinning all analyses supporting HTAs. To address this gap, our aim was to identify what HTA guidance exists on the selection process for EMs, and how the selection of EMs has been justified in the context of published ITCs.

A pragmatic review of HTA guidance documents was conducted in 2021 to describe current requirements for the selection of EMs for ITCs. A supplementary Embase and Medline search was conducted to identify primary research on ITCs published between 2015-2021 presenting information on how EMs were selected to inform these analyses.

Our review found that guidance on this topic focusses on developing and testing different methodologies to adjust for EMs. No detailed guidance was identified in any of the reviewed HTAs, although the National Institute for Health and Care Excellence (NICE) briefly mentioned that companies should identify EMs through a topical review or expert discussions. Similar findings were also revealed through the database search; few published ITCs included information on the EM selection process which was either based on evidence highlighted in the literature or findings from prior trial subgroup analyses. No reference to a systematic identification of EMs was found.

To fill the guidance gap identified in our review an extension of current ITC guidelines (including those from HTA bodies) is needed, including (i) indication on how EMs should be identified through systematic reviews, (ii) a quantitative assessment of the EM distribution and (iii) formal expert elicitation prior to the selection of ITC methods. Without these additional steps, ITC results may be biased, potentially negatively impacting decision-making and ultimately patient care.

Although previous studies suggest that fluticasone furoate nasal spray (FFNS) is superior to placebo in reducing symptoms in adolescents and adults with allergic rhinitis (AR), there is still uncertainty about its clinical effectiveness in the pediatric population. The aim of this study was to assess the clinical effectiveness of FFNS, compared with placebo, in reducing nasal symptoms in children with perennial AR.

A systematic review was conducted of studies identified from the MEDLINE and Embase databases that were published up to January 2021. The population of interest was patients aged 2 to 12 years with perennial AR. Included studies were limited to randomized controlled trials (RCTs) comparing FFNS (110 μg once daily) with placebo. The outcomes of interest included the reflective Total Nasal Symptom Score (rTNSS) and safety. Meta-analyses were performed using RevMan 5.4. The Cohen’s guideline was used to assess the minimum clinically important difference for rTNSS; that is, if the pooled standardized mean difference (SMD) and the lower limit of the 95 percent confidence interval (CI) exceed -0.5, the treatment effect was considered clinically significant.

Three RCTs (959 pediatric patients) were included. One study evaluated the short-term use of FFNS, one evaluated the long-term use of FFNS, and the third evaluated both the short- and long-term use of FFNS. FFNS produced a statistically significant reduction in rTNSS (SMD -0.35, 95% CI -0.63, -0.08; p<0.001) relative to placebo in the long-term treatment studies, but not in the short-term studies. However, since the mean reduction did not reach the minimum clinically important difference (SMD -0.5), these results were not considered clinically relevant. The safety outcomes for FFNS were similar to placebo.

The currently available evidence suggests that FFNS does not produce a meaningful clinical effect on nasal symptoms in children with perennial AR, compared with placebo. In the past decade, however, some guidelines have unequivocally endorsed this treatment.

The National Institute for Health and Care Excellence (NICE) is determined to make better use of health and social care data in the development of its guidance. Real-world data (RWD) has the potential to significantly improve our understanding of the value of new and existing health and social care interventions. RWD is already widely used to characterize populations, interventions, and outcomes and to populate economic models, but its use in estimating the effects of interventions remains limited, especially for medicines. Key barriers to its greater use in this context include limited transparency around studies, sometimes a lack of confidence in their integrity, and methodological concerns around how studies have addressed major sources of potential bias.

This abstract focuses on the real-world evidence (RWE) framework developed by NICE to support its ambitions. The framework was developed in an iterative way based on: reviews of best practice approaches to the conduct and assessment of real-world evidence studies; case studies; and workshops with key external stakeholders. The initial version of this living framework focuses on de novo RWE studies using individual patient data.

The RWE framework consists of an overarching research governance framework which describes expectations around the planning, conduct, and reporting of RWE studies across uses of real-world data. Uses are categorized by risk according to their importance to decision-making, the impact of decisions on patient and system outcomes, and their complexity as proxied by risk of bias. Studies of the effects of interventions on patient health and system outcomes are considered the highest risk. The research governance framework is supported by a tool to aid assessment of data suitability for its intended application, and detailed guidance on the conduct and reporting of comparative effect studies using RWD, following the target trial approach.

The RWE Framework underpins NICE’s ambitions to make better use of RWD in its guidance and is intended to improve the quality and utility of RWE studies submitted to NICE enabling more consistent and appropriate evaluation.

Poor nutritional status, a complex consequence of numerous interrelated factors, is poorly characterized in patients with chronic kidney disease (CKD) before they commence dialysis. This study aimed to characterize the risk factors and prevalence of malnutrition among patients with CKD, with or without diabetes mellitus, before they underwent dialysis at a tertiary care public teaching hospital.

This longitudinal observational study utilized a Pt-Global web tool© to assess the nutritional status of patients based on their Subjective Global Assessment score as follows: a score of two to three indicates that patient and family education is required; scores of four to eight mean that intervention is required as indicated by symptoms; and a score of nine or more indicates a critical need for intervention. Glomerular filtration rate calculated using the Chronic Kidney Disease Epidemiology Collaboration equation was used as the measure of kidney function. Multinomial regression analyses were used to ascertain the predictors of poor nutritional status.

A total of 450 patients (265 men and 185 women) who had CKD, with or without diabetes, and were not on dialysis were recruited during the period of study. The average age of the patients was 53.9 years (standard deviation 14.2). ‘Severe’ malnutrition was present in 152 (33.8%) patients, while 140 (31.1%) were ‘mildly or moderately’ malnourished, and 158 (35.1%) were ‘well-nourished’. Patients with CKD and diabetes were more severely malnourished: 68 were rated as mild or moderate (15.1%) and 91 were rated as severe (20.2%). The prevalence of malnutrition increased with the decline of residual renal function. Fatigue, loss of appetite, pain anywhere in the body, constipation, dry mouth, feeling full quickly, and physical and functional inactivity were the most common risk factors for poor nutritional status.

This study presents real-world evidence of poor nutritional status in patients with CKD and confirms that it is more common in individuals who are diabetic and have poor kidney function and hypoalbuminemia. Emphasis on nutrition in patients with CKD is important for improving their health.

A Cardiology Evaluation Unit was established in 2004 within Québec’s Institut national d’excellence en santé et en services sociaux (INESSS) with a novel mandate to collect real-world evidence (RWE) to complement literature-based health technology assessment. In 2010 following publication of the seminal PARTNER trial, INESSS was mandated by the health ministry to review the evidence on transcatheter aortic valve intervention (TAVI) for patients with aortic stenosis. Herein we show how RWE was used to evaluate health system performance throughout the technology’s life cycle and inform organizational and clinical decisions.

Various products were diffused by INESSS over the years: a guidance (2012), an updated literature review (2017) and provincial standards (2017), in parallel with RWE reports covering TAVI use from 2013-2015, from 2013-2018, and a 2021 RWE report combined with administrative data covering transcatheter and surgical treatment of aortic stenosis from 2013-2019.

Based on the guidance’s review of evidence, TAVI was initially recommended for patients considered at too high risk for the surgical approach, under the condition of continued evidence generation to address uncertainty. The subsequent literature review update highlighted that the indication for TAVI had been extended to patients at moderate surgical risk. INESSS produced standards in collaboration with clinical experts to optimize and harmonize the use of TAVI in designated centers. Evaluation of structures, processes and outcomes by INESSS continued until 2019, showing a continuous increase in the use of TAVI, improved short-term survival, and careful patient selection via a multidisciplinary process. RWE also highlighted the impact of TAVI on the overall organization of care for patients with aortic stenosis, as selection criteria further expanded to patients at lower surgical risk, raising important issues regarding patient selection processes, wait times, and longer-term outcomes.

TAVI clinical practice is constantly evolving and leads to changes in the management of aortic stenosis. RWE provided essential organizational and clinical input to inform clinical guidance and decision-making by Québec policy-makers, clinicians and patients.

Breast Units (BU) represent an opportunity to adequately manage and improve quality of care for patients with breast cancer. The presence of specific national guidelines should promote safe and good quality, integrated care. In fact, the presence of a multidisciplinary team of specialists whose workflows follow specific guidelines, set to the highest European standards, should ensure that patients in Breast Units receive appropriate care at diagnosis and throughout the course of the disease.

Two surveys were developed and administered to Breast Unit coordinators and patients throughout Italy. The surveys investigated the provision of healthcare services and the implementation of a new organizational model over two time periods: the first wave of the coronavirus disease 2019 (COVID-19) pandemic from March 2020 to August 2020 and the second wave from September 2020 to December 2020. The surveys aimed to assess the continuity of care during the pandemic from the clinician and patient perspective.

Patients observed a reduction in the postponement of care between the first wave of COVID-19 and the second wave. Some services were delayed, particularly in the second wave where a large number of services were not rescheduled. Management of the COVID-19 pandemic resulted in many healthcare professionals being reallocated to other duties and hospital departments. The effect of this was particularly relevant for both patients and clinicians, with few healthcare professionals having the capacity to focus on conditions other than COVID-19. In the first wave of the pandemic 42 percent of professionals were redeployed, compared with 27 percent in the second wave.

This study demonstrates how COVID-19 affected the care of patients with breast cancer in Italy. Patients and coordinators from Breast Units across the country highlighted many important aspects that should be considered when assessing the effects of the COVID-19 pandemic on the entire healthcare system in order to be better prepared for future pandemics.

Qualitative research is being increasingly integrated in heath technology assessments (HTA) within the Spanish Network of Agencies for Assessing National Health System Technologies and Performance (RedETS). Qualitative research methodological guidelines are given in RedETS HTA guidelines and the Patient Involvement Strategy. A specific methodological guideline to systematically review qualitative studies was published in 2007 and is pending its update. The impact of their implementation is unknown. The aim of this work is to analyze the techniques, impact and reporting of qualitative research (primary and secondary) in HTAs.

A manual search of the HTAs published in the last 5 years in RedETS was conducted to locate assessments that include qualitative research. To ensure a complete identification, RedETS agencies and units were consulted to provide information about the assessments that have used qualitative techniques in their development over the past 5 years. A content analysis of the selected assessments was conducted to analyze the techniques, impact and reporting of qualitative research in HTA.

In the past five years, focus groups, semi-structured interviews, evidence synthesis of observational studies including qualitative studies have been used and integrated in HTA in RedETS. Most of them have been linked to patient involvement facilitation or the inclusion of patient perspectives in HTA. Qualitative research has been used to analyze patient’s experiences and values, to elicit and select important outcome measures for patients, to research for barriers-facilitators for technology implementation and to inform evidence to decision frameworks.

Qualitative primary and secondary research is being used in HTA in Spain. It is mainly linked to patient involvement strategies both to elicit patient perspectives directly or to collect patient-based evidence. The impact of qualitative research in HTA is broad and diverse, extending from the scope of the assessments to the drafting of the recommendations.

The outbreak of the COVID-19 global pandemic in 2020 has been a major challenge for the world’s population and governments. The lack of vaccines, the saturation of health systems, and its rapid spread forced governments to take non-pharmacological interventions (NPI) that had a high impact on the population. Assessing the efficacy of these measures is a challenge for health technology assessment bodies.

The main NPIs for which assessment was required were: mobility restrictions, social distancing, cancellation of events or reduction of seating capacity, closure or reduction of seating capacity in non-essential businesses, closure or limitation of seating capacity in educational establishments, and promotion of teleworking in potential jobs. The implementation of these measures at a global level provides a large population for the study of the impact of these measures. However, the challenges for their evaluation are numerous:

• • The joint implementation of these measures makes it difficult to evaluate them in an isolated manner.

• • The heterogeneity between countries and regions of the pandemic situation at the time when these measures are initiated and terminated.

• • The different accuracy in the application of the measures.

• • Heterogeneity in the quality and accessibility of public health services for citizens.

Outcome variables to assess the effectiveness of these measures should include parameters related to:

• • Incidence variables: the number of new or accumulated cases in a given time range, the variation in the number of cases in a given time range and the proportion of positive tests.

• • Transmission variables: the basic reproductive number (R0) and the effective reproductive number (Rt).

• • Severity and mortality variables: the number or variation of hospitalizations, the number or variation of intensive care unit (ICU) hospitalizations and the number or variation of deaths.

The large number of available data, the heterogeneity of the measures, the differences between populations, the numerous outcome variables and the possible inclusion of mathematical modelling studies, are a methodological challenge for the HTA bodies.

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 emergence of advanced therapy medicinal products (ATMPs), a disruptive class of health technologies, is generating important challenges in terms of value assessment, and their high prices introduce critical access and affordability concerns.

The aim of this oral presentation is to expose the challenges of traditional value assessment and pricing and reimbursement methods in the evaluation of ATMPs, and to characterize the current and prospective financing solutions that may ensure patient access to and affordability for these health technologies.

Standard health technology assessment (HTA) is not designed for assessing ATMPs and may delay access to these therapies; thus, a broader concept of value is required. As a result, value-based pricing methodologies have been gaining prominence as a way to cope with the specific challenges of ATMPs. The pricing and reimbursement framework should ensure a balance between encouraging innovation and maximizing value for money for payers through the attribution of a fair price to new health technologies. The provision of early scientific advice to developers by regulatory and HTA bodies is key, as it will help diminish the perspective gap between developers, regulators, and payers.

The high efficacy and high price dynamic of many ATMPs necessitates novel financing models, both in the European Union and in the USA. Managed entry agreements, where financing is conditional upon the submission of additional evidence, linked with leased payments may offer effective strategies to address the uncertainties caused by the evidence gap associated with ATMPs, ensuring affordable and sustained access to these therapies.