Technology Assessment Research Articles

APPROACHES ON SANITATION AND HEALTH IN THE LEGAL AMAZON

The objective of this article is to understand and analyze the approaches employed in study of the relationship between sanitation and health in the Legal Amazon from 2013 to 2023. This article conducts an integrative review of the literature drawing upon the works available in the Scientific Electronic Library Online, Virtual Health Library, and Capes Periodicals. 64 out of 185 studies are analyzed, in line with the research question and after applying the eligibility criteria. The studies are clustered into four thematic categories: (i) occurrence of diseases and injuries; (ii) environmental analysis, which includes studies on the risks of elements present in the soil, water and animals; (iii) technology assessment and proposal of methodologies; and (iv) impact of capitalist expansion. More than half of the studies (56%) refer to the more classic approach, which establishes a relationship between sanitation conditions and the occurrence of diseases and injuries. At the same time, an expansion of theoretical-conceptual approaches to the topic was identified, including a focus on human rights and environmental justice, especially in studies about the impact of capitalist expansion. The results of this research point to the need of improving the critical reflection on the political, economic, and sociocultural determinants inherent to the connections between sanitation and health in Brazil.

The societal burden associated with adolescent idiopathic scoliosis: a cross-sectional burden-of-disease study.

In the general population the prevalence of adolescent idiopathic scoliosis (AIS) is 2-3%. There is growing awareness of how AIS affects the quality of life of patients. However, the extent of the societal burden AIS poses remains poorly understood. Therefore, this study aimed to determine the societal burden of AIS. A cross-sectional burden of disease study was conducted using a bottom-up, prevalence-based approach. Patients with AIS or guardians of a child diagnosed with AIS residing in the Netherlands were eligible for inclusion. The survey was distributed between June - December 2022 and was completed once by each participant. Costs were assessed using the institute for Medical Technology Assessment - Medical Consumption and Productivity Cost Questionnaires. The health-related quality of life (HRQoL) was assessed using the EuroQol 5D-5L/EuroQol 5D Youth and the Scoliosis Research Society-22 revised questionnaires. Costs and HRQoL were identified, measured, and valued. Participants (n = 229) were predominantly female (92%), on average 35 years old, and were employed (65%). The societal cost for a patient with AIS in the Netherlands was €12,275 per year. The largest costs were estimated for the healthcare and productivity losses. The mean utility score for adults was 0.7 (SD 0.20). Severe pain was experienced by 10% of the adult participants and 44% reported to experience moderate pain/discomfort. Statistically significant differences between different age groups were present for the sector costs and HRQoL. AIS negatively impacts societal costs and the HRQoL. Reducing the burden that is posed on the productivity sector by AIS and further improving the HRQoL for AIS patients is needed.

Health Technology Assessment for digital health technologies in India: A framework for action.

Health Technology Assessment for digital health technologies in India: A framework for action.

Patient experiences of treatment-resistant depression (TRD): A systematic review and qualitative meta-synthesis

Treatment-resistant depression (TRD) occurs when depressive symptoms persist after a patient has tried at least two antidepressants at an appropriate dose for an adequate length of time, as judged by their clinician. Understanding what it is like to live with and seek care for TRD can inform treatment planning and contribute to health technology assessments for depression-related therapies. Our objective was to systematically review this evidence through an investigation of qualitative literature about the experiences of people who have TRD. We searched MEDLINE, CINAHL, PsycINFO, and the Web of Science Core Collection. Publications eligible for inclusion were English-language primary empirical qualitative studies or the qualitative component of mixed methods studies examining adults’ experiences with TRD or with specific treatment options for TRD. Two reviewers independently screened and extracted data, as well as critically appraised included studies using the Standards for Reporting Qualitative Research (SRQR) instrument. This review was guided by integrative meta-synthesis. Data analysis consisted of a staged coding process similar to grounded theory. The protocol was registered in PROSPERO prior to the search (record ID: CRD42022356813). Sixteen studies were included from screening 18,568 results. Two studies focused on the overall experience of TRD and 14 explored experiences of specific treatments such as ketamine. Patients described the depressive symptoms they experienced, a sense of futility and desperation to find an effective treatment, and their experiences with new, sometimes experimental, therapies including deriving benefits beyond the clinical. Overall, few studies have examined the patient experience of TRD, reflecting the clinical complexity of this patient population. As most literature coalesced around experiences of specific treatments, greater examination of the overall experience of TRD is needed to inform clinical practice, and administrative and funding policy decisions for interventions that may be effective for this patient population. PROSPERO registration number: CRD42022356813.

A Systematic Literature Review of Modelling Approaches to Evaluate the Cost Effectiveness of PET/CT for Therapy Response Monitoring in Oncology.

This systematic literature review addresses model-based cost-effectiveness studies for therapy response monitoring with positron emission tomography (PET) generally combined with low-dose computed tomography (CT) for various cancer types. Given the known heterogeneity in therapy response events, studies should consider patient-level modelling rather than cohort-based modelling because of its flexibility in handling these events and the time to events. This review aims to identify the modelling methods used and includes a systematic assessment of the assumptions made in the current literature. This study was conducted and reported following the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) 2020 statement. Information sources included electronic bibliographic databases, reference lists of review articles and contact with experts in the fields of nuclear medicine, health technology assessment and health economics. Eligibility criteria included peer-reviewed scientific publications and published grey literature. Literature searches, screening and critical appraisal were conducted by two reviewers independently. The Consolidated Health Economic Evaluation Reporting Standards (CHEERS) were used to assess the methodological quality. The Bias in Economic Evaluation (ECOBIAS) checklist was used to determine the risk of bias in the included publications. The search results included 2959 publications. The number of publications included for data extraction and synthesis was ten, representing eight unique studies. These studies addressed patients with lymphoma, advanced head and neck cancers, brain tumours, non-small cell lung cancer and cervical cancer. All studies addressed response to chemotherapy. No study evaluated response to immunotherapy. Most studies positioned PET/CT as an add-on modality and one study positioned PET/CT as a replacement for conventional imaging (X-ray and contrast-enhanced CT). Three studies reported decision-tree structures, four studies reported cohort-level state-transition models and one study reported a partitioned survival model. No patient-level models were reported. The simulation horizons adopted ranged from 1 year to lifetime. Most studies reported a probabilistic analysis, whereas two studies reported a deterministic analysis only. Two studies conducted a value of information analysis. Multiple studies did not adequately discuss model-specific aspects of bias. Most importantly and regularly observed were a high risk of structural assumptions bias, limited simulation horizon bias and wrong model bias. Model-based cost-effectiveness analysis for therapy response monitoring with PET/CT was based on cohorts of patients instead of individual patients in the current literature. Therefore, the heterogeneity in therapy response events was commonly not addressed appropriately. Further research should include more advanced and patient-level modelling approaches to accurately represent the complex context of clinical practice and, therefore, to be meaningful to support decision making. This review is registered in PROSPERO, the international prospective register of systematic reviews funded by the National Institute for Health Research, with CRD42023402581.

Time from approval to reimbursement recommendations in healthcare systems with centralized HTA processes. Focus on the Polish HTA agency

ABSTRACT Background To analyze the time from drug registration to reimbursement recommendations, we examined medicinal products, including new clinical indications, registered by the EMA between 2014 and 2019 across various therapeutic areas. Materials and Methods The Polish Agency for Health Technology Assessment and Tariffication (AOTMiT) was compared with 11 agencies in England, Wales, Ireland, Scotland, the Netherlands, Norway, France, Germany, New Zealand, Canada, Australia. A total of 1,942 recommendations published by 12 HTA agencies were analyzed. Results The time from registration to recommendation in Poland was statistically significantly longer than for the other countries. The analysis revealed noticeable differences in the time it takes from drug registration to recommendation across the countries included in this analysis. Analyzing trends from 2014 to 2019 in individual countries, it is noteworthy that in most agencies the median time from registration to recommendation is declining. Conclusions This may suggest improvements in the processes of the recommending authorities and the companies responsible for providing data for assessment. Despite Poland having one of the longest times from registration to recommendation among the countries analyzed, there has been a clear year-over-year decrease in the time to publication of reimbursement recommendations.

Safety and Feasibility of Interventional Hybrid Fluoroscopy and Nuclear Imaging in the Work-up Procedure of Hepatic Radioembolization.

Purpose To evaluate the safety and feasibility of a novel hybrid nuclear and fluoroscopy C-arm scanner to be used during the work-up procedure of hepatic radioembolization. Materials and Methods In this prospective first-in-human clinical study, 12 participants (median age, 67 years [range: 37-78 years]; nine [75%] male, three [25%] female) with liver tumors undergoing work-up for yttrium 90 radioembolization were included (ClinicalTrials.gov NCT06013774). Work-up angiography and technetium 99m-macroaggregated albumin injection were performed in an angiography suite equipped with a hybrid C-arm that could simultaneously perform fluoroscopy and planar nuclear imaging. Technetium 99m-macroaggregated albumin was injected under real-time hybrid imaging, followed by in-room SPECT imaging. Safety and feasibility were studied by assessing adverse events, technical performance, additional x-ray radiation dose, and questionnaires completed by radiologists and technologists. Results No adverse events were attributed to the hybrid C-arm scanner. The additional x-ray radiation dose was low (median, 19 Gy · cm2; minimum: 12 Gy · cm2; maximum: 21 Gy · cm2 for participants who completed all imaging steps). The interventional personnel considered use of the hybrid C-arm scanner safe and feasible, although the additional time spent in the intervention room was considered long (median, 64 minutes; minimum: 55 minutes; maximum: 77 minutes for participants who completed all imaging steps). Conclusion Use of the hybrid C-arm scanner during the work-up procedure of hepatic radioembolization was found to be safe and feasible in this first-in-human clinical study. Keywords: Angiography, Fluoroscopy, Interventional-Vascular, Radionuclide Studies, Radiosurgery, Gamma Knife, Cyberknife, SPECT, Instrumentation, Physics, Technical Aspects, Technology Assessment Supplemental material is available for this article. Published under a CC BY 4.0 license. Clinical trial registration no. NCT06013774.

Health Technology Assessment of Nailfold Capillaroscopy and Digital Retina Imaging in Diabetes Screening

AimThis study aims to identify the criteria for the evaluation of medical technologies in diabetes screening and then perform health technology assessment (HTA) for two technologies: digital retina imaging (DRI) and Nailfold Capillaroscopy (NFC). MethodsA multicriteria decision-making tool measuring attractiveness using a categorical-based evaluation technique (MACBETH) was used to calculate and compare alternatives. A focus group of ten decision-makers with more than ten years of experience in health technology was used for the decision-making. The Delphi Method was used to get a consensus about comparing criteria and alternatives to these criteria. The sampling method used in the study was the nonprobability purposive sampling method. ResultsThe study concluded that clinical efficacy (0.42) is the most important criterion for evaluating medical technologies, followed by cost-effectiveness (0.27). At the same time, ethical considerations were found to be less important (0.03). The sensitivity analysis concluded that the decision is sensitive to clinical efficacy and cost-effectiveness changes. The study found that none of the alternatives dominates others as an option for early detection of diabetes through vascular changes. LimitationsThis study primarily uses systemic literature review methods to identify criteria for evaluating alternatives. There may be additional criteria for evaluation. ConclusionThis study provides an approach for a multicriteria comparison of medical devices. The study findings are useful for public health professionals and health policymakers. Plain Language SummaryThis study assesses nailfold capillaroscopy (NFC) as a substitute for digital retina imaging (DRI) in diabetes screening using the MACBETH multicriteria decision-making tool. The research identifies five key evaluation criteria: clinical efficacy, cost-effectiveness, patient needs, user safety, and ethical considerations. A focus group of experienced health technology decision-makers was used to compare NFC and DRI. Results indicate that clinical efficacy and cost-effectiveness are the most critical factors, with NFC showing potential but not significantly outperforming DRI. Sensitivity analyses highlight the need for further research to validate NFC as an effective diabetes screening tool.

Perceptions of indirect treatment comparisons as an evidence base in oncology decision-making: results of an international survey of health technology assessment and payer decision-makers.

Aim: Health technology assessment (HTA) and payer organizations are often faced with early decision-making in oncology. To design and conduct robust indirect treatment comparisons (ITCs), it is important to better understand HTA and payer decision-maker perceptions of ITCs. Here we aim to describe what individuals with HTA and payer experience see as the acceptability of ITCs for HTA and payer organization coverage and reimbursement decision-making. Materials & methods: This survey included 30 current and former HTA and payer decision-makers from five countries: Australia, France, Germany, the UK (n=5 each) and the US(n=10). Main outcomes included the ratings of acceptance of ITCs and the presence of well-defined methodological guidance for ITCs. Results: ITCs are generally accepted by participants in Australia and the UK but are more likely evaluated on a case-by-case basis in France, Germany and the US. Four of five participants in Germany and the UK, two of five in Australia and one of five in France reported that well-defined and prescribed criteria regarding the use of ITCs were in place. Conclusion: There is a need for harmonization of methods used to assess ITCs by HTA and payers, especially in the rapidly evolving treatment landscape in oncology.

Using QALYs as an Outcome for Assessing Global Prediction Accuracy in Diabetes Simulation Models.

(1) To demonstrate the use of quality-adjusted life-years (QALYs) as an outcome measure for comparing performance between simulation models and identifying the most accurate model for economic evaluation and health technology assessment. QALYs relate directly to decision making and combine mortality and diverse clinical events into a single measure using evidence-based weights that reflect population preferences. (2) To explore the usefulness of Q2, the proportional reduction in error, as a model performance metric and compare it with other metrics: mean squared error (MSE), mean absolute error, bias (mean residual), and R2. We simulated all EXSCEL trial participants (N = 14,729) using the UK Prospective Diabetes Study Outcomes Model software versions 1 (UKPDS-OM1) and 2 (UKPDS-OM2). The EXSCEL trial compared once-weekly exenatide with placebo (median 3.2-y follow-up). Default UKPDS-OM2 utilities were used to estimate undiscounted QALYs over the trial period based on the observed events and survival. These were compared with the QALYs predicted by UKPDS-OM1/2 for the same period. UKPDS-OM2 predicted patients' QALYs more accurately than UKPDS-OM1 did (MSE: 0.210 v. 0.253; Q2: 0.822 v. 0.786). UKPDS-OM2 underestimated QALYs by an average of 0.127 versus 0.150 for UKPDS-OM1. UKPDS-OM2 predictions were more accurate for mortality, myocardial infarction, and stroke, whereas UKPDS-OM1 better predicted blindness and heart disease. Q2 facilitated comparisons between subgroups and (unlike R2) was lower for biased predictors. Q2 for QALYs was useful for comparing global prediction accuracy (across all clinical events) of diabetes models. It could be used for model registries, choosing between simulation models for economic evaluation and evaluating the impact of recalibration. Similar methods could be used in other disease areas. Diabetes simulation models are currently validated by examining their ability to predict the incidence of individual events (e.g., myocardial infarction, stroke, amputation) or composite events (e.g., first major adverse cardiovascular event).We introduce Q2, the proportional reduction in error, as a measure that may be useful for evaluating and comparing the prediction accuracy of econometric or simulation models.We propose using the Q2 or mean squared error for QALYs as global measures of model prediction accuracy when comparing diabetes models' performance for health technology assessment; these can be used to select the most accurate simulation model for economic evaluation and to evaluate the impact of model recalibration in diabetes or other conditions.

4.P. Round table: Advancing genomic diagnostics for rare genetic diseases: a focus on non-clinical domains of HTA

Abstract Genetic rare diseases are a burning global public health issue requiring a thorough assessment of the related burden. Unfortunately, curative therapies are oftentimes unavailable for genetic rare diseases; nevertheless, a prompt diagnosis could allow clinicians to elaborate supportive care plans for patients and their caregivers, determine the best care setting, and potentially consider enrolling them in clinical trials, if suitable. Innovative, effective, and sustainable diagnostic techniques, such as Whole genome sequencing (WGS), are needed to address this growing health concern. At global level, there is a paucity of research that specifically investigates the implementation of WGS into clinical practice which is even more constrained at the European level. Furthermore, given the current challenges impacting pediatric patients suspected of having genetic diseases without a definitive diagnosis, there is an urgent need to improve the diagnostic workflow based on genomic techniques, such as WGS. Delving into this topic could contribute valuable insights that can inform policymakers, at macro-level, and health professionals, at micro-level, on how to develop suited genomic policies and clinical guidelines for the proper adoption of WGS within the daily clinical practice. From an international standpoint, it could be argued that these considerations resonate with the United Nations Sustainable Development Goals (SDGs), particularly SDG 3 (i.e., “Good health and well-being”) and 10 (i.e., “Reduced inequalities”), as a priority for research and action. Notwithstanding, the broader implementation of WGS encounters hindrances owing to its complexity and the multifaceted social, economic, organizational, and ethical implications. In view of these challenges, Health Technology Assessment (HTA), as an integrated and multidisciplinary process, provides a comprehensive framework for the evaluation of these dimensions. This round table is intended to provide attendees with insights into nonclinical domains of HTA of WGS which, unlike clinical domains, demand a nuanced approach within the framework of the HTA. The intended objective is pursued through the initial presentation of a case study on the evaluation of WGS in paediatric genetic disorders from the Italian National Health Service perspectives followed by an open discussion with experts. The discussion will delve into issues related to economics, genomics, health services organization and ethics. Key messages • For pediatric patients suspected of having a genetic disease, WGS could shorten the so-called diagnostic odyssey. • Context-depending aspects of implementing WGS should be properly taken into account through HTA.

Rapid health technology assessment to inform policy: a case study with respiratory syncytial virus

Abstract Background Respiratory syncytial virus (RSV) can cause severe acute respiratory tract infection in infants and older adults. However, until recently, no immunisation interventions were available to reduce the burden of RSV disease in the general population. New health technologies have now been authorised in Europe for infants (a long-acting monoclonal antibody (nirsevimab) and a maternal vaccine (RSVpreF)), and two vaccines for adults aged ≥60 years (RSVPreF3 and RSVpreF). A rapid health technology assessment (HTA) was requested by the Irish Department of Health to support an interim decision on RSV immunisation. Methods The rapid HTA commenced in January 2024, and comprised description of technology, a summary of international practice, national and international epidemiology and burden of disease, and a costing analysis. The summary of international practice (including underpinning evidence) was undertaken rather than a systematic review of effectiveness and safety due to the rapidly changing evidence base and short timeline within which information was required. Results While new recommendations regarding immunisation against RSV are emerging internationally in response to recently authorised technologies, implementation and funding strategies are not currently widespread. Compared with a full HTA, a rapid HTA enabled an efficient and flexible approach to producing a high quality evidence synthesis to inform a temporary policy decision, taking approximately 6 months to complete rather than 12 months or longer. A full HTA is planned to inform long-term decision-making on this topic, but would not have been feasible to inform an interim decision. Conclusions This rapid HTA will inform a temporary decision on immunisation policy for a single RSV season in Ireland. Conducting a rapid HTA prior to a full HTA has enabled timely decision-making, with implications for Ireland’s national healthcare system and people at risk of severe RSV-related disease. Key messages • Innovations in immunisation against RSV are leading to changes in policy across Europe. • Rapid HTA can support timely, evidence-based public health decision-making in a fast-evolving field.

A year of rapid reviews: evidence-based decision support for Austrian social insurances & hospitals

Abstract Background The Austrian Institute for Health Technology Assessment (AIHTA) has supported the decisions of Austrian social insurance funds and hospital providers with rapid reviews since 2023. This summary of rapid reviews aims to provide insights on findings, as well as experiences and challenges of conducting the rapid reviews after one year. Methods We included all rapid reviews that were requested to the AIHTA team in 2023. For research questions that were withdrawn, the reasoning was documented. The completed rapid reviews were grouped into medical specialties, depending on the intervention analysed. The conclusions of the rapid reviews were presented in tabular form, with the strength of the available evidence classified as high, moderate, low or insufficient. Results The rapid reviews on interventions (n = 9) cover a variety of medical specialties (orthopaedics, gynaecology, neurology, physical medicine, ophthalmology, vascular surgery and dermatology). In three rapid reviews, the analysed evidence on the intervention was insufficient for benefit (compared to standard of care). In four rapid reviews, the evidence indicated a potential benefit with some limitations (e.g. indication-specific results, or minimal clinically important difference not clearly achieved). In two rapid reviews, the evidence indicated a potential benefit. For three topics, no rapid review was conducted (topic scope too broad for rapid review, change in prioritisation). Challenges include time constraints, as well as narrowing the research questions to be suitable for rapid reviews. Conclusions The overview of rapid reviews highlights evidence-based findings on medical interventions. Further steps include collecting feedback from inquirers, on the extent to which the evidence from rapid reviews was used to support the decision-making. Rapid reviews are a feasible tool but require continuous evaluation and further adaptation of methods. Key messages • The rapid reviews to inform decision-making processes for Austrian social insurance funds and hospitals spanned multiple specialties and revealed varying levels of evidence. • To improve the implementation of rapid reviews, and to overcome challenges of time constraints and narrowing down research questions, feedback from stakeholders should be collected.

Validation of the NEED assessment framework: an EU level Delphi study

Abstract The NEED (Needs Examination, Evaluation and Dissemination) assessment framework was developed to identify and measure health-related patient and societal unmet needs across different health conditions, supporting the creation of needs-driven healthcare policy and innovation. It includes 25 criteria of needs and 46 related indicators allowing to estimate if the needs are met. The current study aims to validate this framework on the EU level using a modified Delphi approach. The selection of national and European institutes invited to participate in the Delphi process was based on their respective roles; specifically, public health, health technology assessment (HTA), medicine authorisation, pricing and reimbursement (P&R), and patient advocacy. Through a modified Delphi approach, the experts were able to express their opinion on the framework structure (dimensions and domains), health-related needs criteria and indicators. Two Delphi rounds were performed using online questionnaires. Consensus was reached when 85% of the respondents agree or totally agree with a proposal and less than 5% of the respondents totally disagree. A panel of 27 experts participated in the first round of the study. The majority of the experts worked in HTA agencies (39%) and P&R agencies (24%), followed by national medicine authorities (9%) and umbrella patient organisations (9%). Seventeen EU Member States were represented, with a higher representation of experts from Austria, Belgium, France, Germany, The Netherlands, Spain and Sweden. Consensus was reached for 22 out of the 25 criteria and 41 out of the 46 indicators after the first round. The modified Delphi technique was an adequate approach to aid the validation of the NEED framework at the EU level. This represents an important first step in achieving consensus on how to define and identify unmet health-related needs and move towards a more needs-driven healthcare policy and innovation landscape. Key messages • Consensus on the definition and identification of unmet health-related needs is crucial to transform public health innovation. • Expert consultation by means of the Delphi technique assumed a pivotal role to reach consensus on the NEED framework.

Productivity loss due to acute COVID-19 and post COVID-19 condition in the Northern Netherlands

Abstract Introduction The COVID-19 pandemic caused significant socioeconomic and health shocks worldwide. Yet, research is still scarce on how acute COVID-19 and post COVID-19 condition (PCC) have impacted workers productivity in terms of absenteeism, presenteeism and reduced unpaid work. This study examines the effect of acute COVID-19 and PCC on Dutch workers’ loss of work productivity in standardized hourly costs compared to those with neither disease. Methods This study included data from 31 waves (March 2020 - October 2022) of the Lifelines COVID-19 cohort, a populational cohort from the Netherlands. Productivity was measured in 7 waves in terms of absenteeism, presenteeism, and loss of unpaid work using the Medical Technology Assessment Productivity Costs Questionnaire. We selected workers aged 18 to 64 in the first wave (n = 10,950). Using administrative data, average hourly costs of productivity were calculated as 38.67€ for absenteeism and presenteeism and 15.94€ for unpaid work. The friction cost method was used. To compare group differences, a Kruskal-Wallis rank sum test with Holms-Bonferroni p-value adjustment was performed. Results In total, 33.4% of participants had acute COVID-19, 5.7% had PCC, and 60.9% had neither disease. The mean total combined productivity loss per person of those with PCC was 7,118€, compared to 3,410€ for those with acute COVID-19, and 3,076€ for those with neither. When comparing groups, significant differences in costs were found for all aspects of productivity, except for unpaid work loss between acute COVID-19 and those with neither disease. Discussion Although the number of people with acute COVID-19 represents a large societal cost in terms of accumulated loss, those with PCC incurred on average twice as much productivity losses per person across all domains. This study highlights the high costs associated with PCC, which continues to chronically burden workers. Policies should be aimed at lessening this burden. Key messages • Post COVID-19 condition is associated with significantly higher average productivity costs per person than acute COVID-19, which highlights the individual burden of this chronic disease. • Acute COVID-19, despite relatively lower average productivity costs, represents a large societal cost in terms of accumulated loss.

How much digital public health is taught in German public health programs? A qualitative analysis

Abstract Background Digital public health (DiPH) professionals are crucial for a successful digital transformation in social and health care. However, the extent to which academic professionals are taught DiPH-related content in their public health studies in Germany remains unclear. Methods We used a systematic module handbook analysis to assess accredited full-time public health-oriented degree programs at German Universities for DiPH-related modules. Programs were identified through the Hochschulkompass platform and among the German Public Health Association member programs. We used MAXQDA for the content analysis of the included module handbooks. Results We identified 79 public health programs. Of these, 10 Bachelor’s and 6 Master’s offered at least one DiPH module, all being highly heterogeneous in their overall addressed public health subfields. Regarding DiPH, 6 programs lectured on the ethics of digitalizing health and data protection, 5 programs each offered modules on DiPH in health policy, on data science in healthcare and software used for eHealth, or on the digital determinants of health. 4 programs each included modules on digital health promotion and alternative evaluation methods. 3 programs each addressed digital science communication and health technology assessment. Finally, 2 programs had modules on digital epidemiology. We found differences between the common understanding of DiPH in academia and the content in the module handbooks. The content identified in the analysis focused mainly on technical areas. Social and health science content was only marginally present. Conclusions The diverse DiPH study programs allow academic public health specialists in Germany to develop specific profiles. There is a need for further development of modules with relevance to the respective degree program to achieve comprehensive competencies in DiPH. We encourage the uptake of our analysis to develop a core curriculum in DiPH among European public health programs. Key messages • Digitalization affects all areas of life, including healthcare systems and public health. Preparing the future workforce already during their training is crucial to enable them to lead the change. • German public health programs are not fit to prepare students for their professions. Developing DiPH curricula with pre-defined and updated competencies will strengthen programs in Germany and beyond.

What are the challenges in the Health Technology Assessment of genetic and genomic applications?

Abstract Introduction Evaluating genetic/genomic applications (GGAs) is crucial for their implementation in clinical practice but hindered by several issues, such as rapid development processes and unclear benefits. Since these challenges lack comprehensive discussion, this study aims to identify and analyze all barriers emerged in the Health Technology Assessment (HTA) of genetic/genomic tests, filling a gap in the current literature. Methods PubMed, Scopus and Web of Science were searched to identify studies that specifically discussed any challenge or barrier in the HTA evaluation of GGAs. No restriction was applied on evaluation aspect or study type. Challenges/barriers were then grouped into the domains outlined in the EUnetHTA Core Model. A narrative synthesis of the main findings was performed. This study was supported by the EC and MUR- PNRR-M4C2-I1.3 Project PE_00000019 ‘HEAL ITALIA’. Results 19 articles were included: one third involved experts from different countries, and about 50% were author perspectives. Articles either focused on one aspect only (37%) or were more general (47%). The most challenging domain was economic aspects (69%), followed by clinical effectiveness (47%) and social impact (42%), but issues were found in all domains. The lack of a standardized HTA approach, the paucity of evidence on clinical outcomes, the challenges in capturing all health benefits, and the difficulties in identifying the healthcare pathways triggered by the test were consistently mentioned across the HTA domains. Conclusions Our study systematically summarized challenges in the HTA evaluation of GGAS, providing a thorough analysis and categorization of these issues. Various challenges surfaced, notably related to the identification of costs, as well as clinical and non-clinical benefits. There is a need for exhaustive discussion on potential solutions to facilitate the assessment process of these technologies to ultimately foster their implementation in clinical practice. Key messages • There are multiple challenges in the HTA evaluation of genetic and genomic tests in clinical practice that impact every HTA domain. • There is a need for concrete efforts in generating further evidence in relation to the definition of costs and benefits of the healthcare pathways triggered by genetic/genomic applications.

Is genomic testing properly evaluated? A systematic review of health technology assessment reports

Abstract Background The last decade has witnessed a steady embrace of Personalized Medicine. However, the evaluation of genetic/genomic tests is not straightforward. The purpose of this systematic review is to identify health technology assessment (HTA) reports assessing genetic and genomic tests, to summarize the methodologies used, the maturity level of the evidence included in it and the highlighted gaps in research. Methods PubMed, Scopus, and Web of Science were searched. Additionally, a desk research was performed on the main HTA reports repositories. HTA reports expressly created to assess genetic/genomic technologies including at least three core evaluation components (analytic validity, clinical validity, clinical utility, economic evaluation, organizational aspects, ethical, legal, and social implications) were included. This study was supported by the EC and MUR under PNRR - M4C2-I1.3 Project PE_00000019 ‘HEAL ITALIA’. Results Overall, 27331 unique records were retrieved, 55 of which were included in the systematic review. The reports were mainly from Australia (29%), Canada (27%) and UK (25%), regarded pharmacogenomics (36%) and oncology (35%), and analysed test use for treatment choice (29%) and diagnosis (13%). The most reported evaluation components were economic evaluation (87%), clinical utility (76%), and clinical validity (67%). Personal utility (7%), ethical (15%), legal (11%) and social (24%) implications and the patient’s perspective (27%) were poorly represented. Analytical validity, safety, and organizational aspects were included in about half of the reports. Discussion Although these are only preliminary results, the substantial lack of a shared standard in the evaluation of genetic/genomic applications appears clear, given the heterogeneity of the dimensions addressed between reports, as well as the need to strengthen the evaluation of the neglected dimensions, often of primary importance in the definition of the value and risks of personalized medicine. Key messages • The findings highlight the lack of a standard in evaluating genetic/genomic applications. • A common methodology should be developed to comprehensively evaluate genetic/genomic applications.

Human Rights System Guidelines on the Benefit from Scientific Progress in Access to Medicines

Abstract Health innovation and research has become an economic and profitable activity. Market practices have exposed inequalities, scarcities and inequities in access to treatments and have significantly reduced the decision-making power of governments in the field of public health. Documentary research on the human right (HR) to health (Art. 12) and its interrelationship with the right to benefit from scientific progress (Art. 15, b) of the International Covenant on Economic, Social and Cultural Rights (CESCR) on access to medicines in three bodies of the UN system - the ICESCR Monitoring Committee (CESCR), the Human Rights Council (HRC) and World Health Organisation - over the last 10 years. The documents address three main topics. Market: marketing restrictions, price controls and using flexibility in pharmaceutical patents to reduce costs and produce generics. Innovation: patent protection without barriers to access to medicines for people in need. Health system interventions: giving priority to economic efficiency and sustainability by institutionalising health technology assessment, selecting essential medicines, developing clinical guidelines and integrating and linking regulatory agencies. These normative guidelines minimise the ethical, political and legal dimensions of the human right to enjoy scientific progress in access to medicines as a priority value that should guide public and private action, and emphasise technocratic and financial measures that seek to reconcile the liberal and social logic of the ICESCR. Key messages • In order to maximise the guarantee of human rights in access to medicines, economic and financial considerations should play only a residual role in human rights. • To give countries greater decision-making power at the global health, the scope of the right to benefit from scientific progress and its applications in health care needs to be expanded.

Health technology assessment and innovation: here to help or hinder?

Innovative health technologies offer much to patients, clinicians, and health systems. Policy makers can, however, be slow to embrace innovation for many reasons, including a less robust body of evidence, perceived high costs, and a fear that once technologies enter the health system, they will be difficult to remove. Health technology funding decisions are usually made after a rigorous health technology assessment (HTA) process, including a cost analysis. However, by focusing on therapeutic value and cost-savings, the traditional HTA framework often fails to capture innovation in the assessment process. How HTA defines, evaluates, and values innovation is currently inconsistent, and it is generally agreed that by explicitly defining innovation would recognize and reward and, in turn, stimulate, encourage, and incentivize future innovation in the system. To foster innovation in health technology, policy needs to be innovative and utilize other HTA tools to inform decision making including horizon scanning, multicriteria decision analysis, and funding mechanisms such as managed agreements and coverage with evidence development. When properly supported and incentivized, and by shifting the focus from cost to investment, innovation in health technology such as genomics, point-of-care testing, and digital health may deliver better patient outcomes. Industry and agency members of the Health Technology Assessment International Asia Policy Forum (APF) met in Taiwan in November 2023 to discuss the potential of HTA to foster innovation, especially in the Asia region. Discussions and presentations during the 2023 APF were informed by a background paper, which forms the basis of this paper.