Hospital-based health technology assessment of innovative medical devices: insights from a nationwide survey in France.

OBJECTIVES: To better understand the process of hospital acquisition of innovative medical devices (MDs) and the hospital-based health technology assessment (HB-HTA) pathways in France, an in-depth study based on a quantitative approach is needed. The aim of the present study was to assess through a national survey how HB-HTA is currently implemented in French hospitals and to identify its level of formalization. METHODS: A quantitative online survey was conducted among hospitals performing HB-HTA in France, with a focus on the acquisition of innovative MDs for individual use. The survey, conducted between March and June 2022, was developed by a scientific board composed of members of the French-speaking Society for HB-HTA. RESULTS: Sixty-seven out of 131 surveyed hospitals with HB-HTA activities responded, including 29 university hospitals, 24 nonprofit private hospitals, and 14 local hospitals. Sixty-one respondents (91 percent) reported the existence of a process dedicated to evaluating innovative MDs; of these, 16 declared that their hospitals had a formalized unit with HB-HTA activity. These units were more frequently found in larger hospitals with more than 500 inpatient beds (n = 16, p = 0.0160) and in university hospitals (n = 12, p = 0.0158). No hospital reported any collaboration with HAS, the French national HTA agency. CONCLUSION: A diverse range of HB-HTA organizations with different structural levels exist in France for MD procurement linked to the category of hospitals. The study highlights the need for recognition of HB-HTA activity at the regulatory level in France and for direct collaboration between HTA activities performed at local and national levels.

HB-HTA focus in a tertiary hospitals' network in Northern Italy: A three-years experience analysis.

BACKGROUND AND AIM: Nowadays Health Technology Assessment (HTA) is becoming more important in the hospital management, until the conception of Hospital Based HTA (HB-HTA), considered as a tool of HTA that is a multidisciplinary process to determine the value of a health technology in health sector. The aims of this study are the investigation of the current state of the art and of the diffusion of the HTA culture in hospitals (HB-HTA) also the analysis of the thinking of healthcare professionals regarding the usefulness of HTA. METHODS: The study was performed through the administration of two questionnaires respectively to hospitals' HealthCare Workers (HCW) to assess the HTA's knowledge and to hospitals' HCW that have submitted a new technologies or devices' request to Direction between 2017 and 2019. RESULTS: Considering 988 questionnaires send to HCW only 416 (42.1%) answer were collected, with a higher attention towards HTA made by physicians (148; 35,6%). It's fundamental to observe that despite a mean response rate all the population shows interest for the HB-HTA concept as an instrument to help the hospital management. About the 23 questionnaires to HCW that have made request show a higher attention and knowledge to HTA. CONCLUSIONS: In conclusion, in the current welfare landscape and for the future developments that will ensue from it, especially in the Italian context due to the increase of healthcare funds provided by the NRRP (National Recovery and Resilience Plan), the adoption of the HB-HTA method will represent a strategic lever to support clinical and organizational decision-making processes governance.

International practices in health technology assessment and public financing of digital health technologies: recommendations for Hungary.

Background: Evaluating and integrating digital health technologies is a critical component of a national healthcare ecosystem in the 2020s and is expected to even increase in significance. Design: The paper gives an overview of international practices on public financing and health technology assessment of digital health technologies (DHTs) in five European Union (EU) countries and outlines recommendations for country-level action that relevant stakeholders can consider in order to support uptake of digital health solutions in Hungary. A scoping review was carried out to identify and gather country-specific classifications and international practices on the financing DHTs in five pioneering EU countries: Germany, France, Belgium, the United Kingdom and Finland. Results: Several frameworks have been developed for DHTs, however there is no single, unified framework or method for classification, evaluation, and financing of digital health technologies in European context. European countries apply different taxonomy, use different assessment domains and regulations for the reimbursement of DHTs. The Working Group of the Hungarian Health Economic Society recommends eight specific points for stakeholders, importantly taking active role in shaping common clinical evidence standards and technical quality criteria across in order for common standards to be developed in the European Union single market. Conclusion: Specificities of national healthcare contexts must be taken into account in decisions to allocate public funds to certain therapies rather than others.

[HTA and PTSD: mechanisms and clinical implications]. / HTA et PTSD : mécanismes et implications pratiques.

The causes of arterial hypertension are numerous, but the environment can be a contributing factor. Stress is one of these environmental factors that is difficult to assess. We focus on Post-Traumatic Stress Disorder (PTSD), which is often under-diagnosed and under-recognised in diagnostic and therapeutic strategies. We specifically look at the psychosomatic implications of stress, which put patients with PTSD at a higher risk of developing HTA, and we suggest some therapeutic approaches. Early screening and diagnosis are essential to reduce this major cardiovascular risk factor.

Les causes d'hypertension artérielle (HTA) sont multiples mais l'environnement peut être un facteur favorisant. Le stress fait partie des facteurs environnementaux difficiles à évaluer. Nous nous intéressons plus particulièrement au syndrome de stress post-traumatique (PTSD), souvent sous-diagnostiqué et peu pris en compte dans les stratégies diagnostiques et thérapeutiques. Nous nous intéressons notamment aux implications psychosomatiques engendrées par le stress mettant les personnes atteintes de PTSD plus à risque de développer une HTA et proposons des orientations thérapeutiques. Les enjeux d'un dépistage et d'un diagnostic précoces sont essentiels pour réduire ce facteur de risque cardiovasculaire majeur.

Increasing the efficiency of study selection for systematic reviews using prioritization tools and a single-screening approach.

BACKGROUND: Systematic literature screening is a key component in systematic reviews. However, this approach is resource intensive as generally two persons independently of each other (double screening) screen a vast number of search results. To develop approaches for increasing efficiency, we tested the use of text mining to prioritize search results as well as the involvement of only one person (single screening) in the study selection process. METHOD: Our study is based on health technology assessments (HTAs) of drug and non-drug interventions. Using a sample size calculation, we consecutively included 11 searches resulting in 33 study selection processes. Of the three screeners for each search, two used screening tools with prioritization (Rayyan, EPPI Reviewer) and one a tool without prioritization. For each prioritization tool, we investigated the proportion of citations classified as relevant at three cut-offs or STOP criteria (after screening 25%, 50% and 75% of the citation set). For each STOP criterion, we measured sensitivity (number of correctly identified relevant studies divided by the total number of relevant studies in the study pool). In addition, we determined the number of relevant studies identified per single screening round and investigated whether missed studies were relevant to the HTA conclusion. RESULTS: Overall, EPPI Reviewer performed better than Rayyan and identified the vast majority (88%, Rayyan 66%) of relevant citations after screening half of the citation set. As long as additional information sources were screened, it was sufficient to apply a single-screening approach to identify all studies relevant to the HTA conclusion. Although many relevant publications (n = 63) and studies (n = 29) were incorrectly excluded, ultimately only 5 studies could not be identified at all in 2 of the 11 searches (1x 1 study, 1x 4 studies). However, their omission did not change the overall conclusion in any HTA. CONCLUSIONS: EPPI Reviewer helped to identify relevant citations earlier in the screening process than Rayyan. Single screening would have been sufficient to identify all studies relevant to the HTA conclusion. However, this requires screening of further information sources. It also needs to be considered that the credibility of an HTA may be questioned if studies are missing, even if they are not relevant to the HTA conclusion.

HTA capacity building in Asia: towards one goal.

OBJECTIVES: The aim of the 2022 Health Technology Assessment International (HTAi) Asia Policy Forum (APF) was to discuss experiences and challenges around health technology assessment (HTA) capacity building for both HTA agencies and companies in the Asia region and to identify possible solutions as part of a capacity building roadmap. METHODS: Discussions during the 2022 APF, informed by a pre-meeting survey of HTA agencies and industry attendees from the region, form the basis of this paper. RESULTS: HTA is an essential element of priority-setting in healthcare; however, the scarcity of skilled technical HTA practitioners is a rate-limiting step in the conduct of HTA. The lack of investment in HTA and the political will to mandate the use of HTA in decision-making may be due to a lack of understanding of the value of the HTA process, and how HTA is interpreted and used in the healthcare decision-making process. CONCLUSIONS: Increased demand for HTA is created when the value of HTA is recognized. HTA capacity-building challenges may be mitigated by educating stakeholders, particularly policymakers, on the value of, and the need to invest in, HTA as a transparent process to ensure equitable access to health care for all. Investigating a means of funding and implementing an HTA intern program between agencies, in partnership with industry, to facilitate a supportive environment to foster HTA skills and knowledge, build capacity or strengthen existing capacity should be a priority.

What, Where, and How to Collect Real-World Data and Generate Real-World Evidence to Support Drug Reimbursement Decision-Making in Asia: A reflection Into the Past and A Way Forward.

BACKGROUND: Globally, there is increasing interest in the use of real-world data (RWD) and real-world evidence (RWE) to inform health technology assessment (HTA) and reimbursement decision-making. Using current practices and case studies shared by eleven health systems in Asia, a non-binding guidance that seeks to align practices for generating and using RWD/RWE for decision-making in Asia was developed by the REAL World Data In ASia for HEalth Technology Assessment in Reimbursement (REALISE) Working Group, addressing a current gap and needs among HTA users and generators. METHODS: The guidance document was developed over two face-to-face workshops, in addition to an online survey, a face-to-face interview and pragmatic search of literature. The specific focus was on what, where and how to collect RWD/ RWE. RESULTS: All 11 REALISE member jurisdictions participated in the online survey and the first in-person workshop, 10 participated in the second in-person workshop, and 8 participated in the in-depth face-to-face interviews. The guidance document was iteratively reviewed by all working group members and the International Advisory Panel. There was substantial variation in: (a) sources and types of RWD being used in HTA, and (b) the relative importance and prioritization of RWE being used for policy-making. A list of national-level databases and other sources of RWD available in each country was compiled. A list of useful guidance on data collection, quality assurance and study design were also compiled. CONCLUSION: The REALISE guidance document serves to align the collection of better quality RWD and generation of reliable RWE to ultimately inform HTA in Asia.

Homologous Recombination Deficiency Testing to Inform Patient Decisions About Niraparib Maintenance Therapy for High-Grade Serous or Endometrioid Epithelial Ovarian Cancer: A Health Technology Assessment.

Background: Ovarian cancer affects the cells of the ovaries, and epithelial cancer is the most common type of malignant ovarian cancer. The homologous recombination repair pathway enables error-free repair of DNA double-strand breaks. Damage of key genes associated with this pathway leads to homologous recombination deficiency (HRD), which results in unrepaired DNA and can lead to cancer. Tumours with HRD are believed to be sensitive to treatment with poly-adenosine diphosphate (ADP)-ribose polymerase (PARP) inhibitors, such as niraparib. We conducted a health technology assessment to evaluate the clinical utility and cost-effectiveness of HRD testing to inform patient decisions about the use of niraparib maintenance therapy for patients with high-grade serous or endometrioid epithelial ovarian cancer. We also evaluated the efficacy and safety of niraparib maintenance therapy in patients with HRD or homologous recombination proficiency (HRP), the cost-effectiveness of HRD testing, the budget impact of publicly funding HRD testing, and patient preferences and values. Methods: We performed a systematic literature search of the clinical evidence. We assessed the risk of bias of each included study using the Cochrane risk-of-bias tool for randomized trials version 2, and the quality of the body of evidence according to the Grading of Recommendations Assessment, Development, and Evaluation (GRADE) Working Group criteria. We performed a systematic economic literature search and conducted a cost-utility analysis with a 5-year time horizon from a public payer perspective. We also analyzed the budget impact of publicly funding HRD testing in people with ovarian cancer in Ontario. We performed a literature search for quantitative evidence of patient and provider preferences with respect to HRD testing and maintenance therapy with PARP inhibitors. To contextualize the potential value of HRD testing, we spoke with people with ovarian cancer. Results: The clinical evidence review included two studies in high-grade epithelial ovarian cancer (one in patients with newly diagnosed advanced cases and one in patients with recurrent cancer). The studies evaluated niraparib maintenance therapy compared with no maintenance therapy and used HRD testing to group patients according to HRD status. Compared to placebo, niraparib maintenance therapy improved progression-free survival in patients with newly diagnosed and recurrent ovarian cancer, and in tumours with HRD or HRP (GRADE: High), but the studies did not compare the results between the HRD and HRP groups. The frequency of adverse events was higher in the niraparib group. We identified no studies that evaluated the clinical utility of HRD testing.We conducted a primary economic evaluation to evaluate the cost-effectiveness of HRD testing for people with newly diagnosed ovarian cancer in an Ontario setting. Our analysis used a 5-year time horizon. HRD testing (for all eligible people or only for people with BRCA wild type) resulted in a lower proportion of patients receiving niraparib maintenance therapy, leading to lower costs and fewer quality-adjusted life-years (QALYs). The average total cost per patient was $131,375 for no HRD testing, $126,867 for HRD testing only in people with BRCA wild type, and $127,746 for HRD testing in all eligible people. The average total QALYs per patient were 2.087 for no HRD testing, 1.971 for HRD testing only in people with BRCA wild type, and 1.971 for HRD testing in all eligible people. Our budget impact analysis suggested that assuming a high uptake rate, publicly funding HRD testing for people with newly diagnosed ovarian cancer would lead to a total saving of $9.00 million (if HRD testing were funded for all) to $12.67 million (if HRD testing were funded for people with BRCA wild type) over the next 5 years. Publicly funding HRD testing for people with recurrent cancer would lead to a total saving of $16.31 million (if HRD testing were funded for all) to $21.67 million (if HRD testing were funded for people with BRCA wild type) over the next 5 years.We identified no studies that evaluated quantitative preferences for HRD testing. Based on two studies that evaluated patients and oncologists' preferences for maintenance therapy with a PARP inhibitor in the recurrent setting, a decrease in moderate to severe adverse events was more important for patients than an improvement in progression-free survival; however, improvement in progression-free survival was more important for oncologists. Both patients and oncologists accepted some trade-offs between efficacy and safety. The people with ovarian cancer we spoke with demonstrated a shared value for access to information, prevention of cancer recurrence, and overall survival with minimal adverse effects. This was consistent with findings from another survey in patients with ovarian cancer and at least one episode of recurrence, which suggest that patients prioritize treatment benefit over some treatment adverse events in the context of niraparib maintenance therapy. Interviewees also emphasized the importance of the patient-doctor partnership, access to local health care services, and patient education. Conclusions: In patients with newly diagnosed (advanced) or recurrent high-grade serous or endometrioid ovarian cancer, niraparib maintenance therapy improved progression-free survival compared with no maintenance therapy in tumours with HRD or HRP (GRADE: High). Because we identified no studies on the clinical utility of HRD testing, we cannot comment on how it would affect patient decisions and clinical outcomes.Over a 5-year time horizon, HRD testing for people with BRCA wild type could save $4,509 per person and lead to a loss of 0.116 QALY. The findings of our economic analyses are dependent on assumptions about the use of niraparib following HRD testing. We estimate that publicly funding HRD testing would lead to a total saving of $9 million to $12.67 million for newly diagnosed cancer, and a total saving of $16.31 million to $21.67 million for recurrent cancer over 5 years, assuming the use of niraparib maintenance therapy would be reduced following HRD testing.Patients prioritized decreasing the risk of moderate to severe adverse events of maintenance therapy with PARP inhibitors over improving progression-free survival, and oncologists prioritized improving progression-free survival over decreasing the risk of moderate to severe adverse events. However, both patients and oncologists were open to accepting certain trade-offs between treatment efficacy and toxicity. The people we interviewed, who had lived experience with ovarian cancer and genetic testing, valued the potential clinical benefits of HRD testing for themselves and their family members. They emphasized patient education as an important consideration for public funding in Ontario.

Carrier Screening Programs for Cystic Fibrosis, Fragile X Syndrome, Hemoglobinopathies and Thalassemia, and Spinal Muscular Atrophy: A Health Technology Assessment.

Background: We conducted a health technology assessment to evaluate the safety, effectiveness, and cost-effectiveness of carrier screening programs for cystic fibrosis (CF), fragile X syndrome (FXS), hemoglobinopathies and thalassemia, and spinal muscular atrophy (SMA) in people who are considering a pregnancy or who are pregnant. We also evaluated the budget impact of publicly funding carrier screening programs, and patient preferences and values. Methods: We performed a systematic literature search of the clinical evidence. We assessed the risk of bias of each included study using the Cochrane Risk of Bias tool and the Risk of Bias Assessment tool for Non-randomized Studies (RoBANS), and the quality of the body of evidence according to the Grading of Recommendations Assessment, Development, and Evaluation (GRADE) Working Group criteria. We performed a systematic economic literature search and conducted cost-effectiveness analyses comparing preconception or prenatal carrier screening programs to no screening. We considered four carrier screening strategies: 1) universal screening with standard panels; 2) universal screening with a hypothetical expanded panel; 3) risk-based screening with standard panels; and 4) risk-based screening with a hypothetical expanded panel. We also estimated the 5-year budget impact of publicly funding preconception or prenatal carrier screening programs for the given conditions in Ontario. To contextualize the potential value of carrier screening, we spoke with 22 people who had sought out carrier screening. Results: We included 107 studies in the clinical evidence review. Carrier screening for CF, hemoglobinopathies and thalassemia, FXS, and SMA likely results in the identification of couples with an increased chance of having an affected pregnancy (GRADE: Moderate). Screening likely impacts reproductive decision-making (GRADE: Moderate) and may result in lower anxiety among pregnant people, although the evidence is uncertain (GRADE: Very low).We included 21 studies in the economic evidence review, but none of the study findings were directly applicable to the Ontario context. Our cost-effectiveness analyses showed that in the short term, preconception or prenatal carrier screening programs identified more at-risk pregnancies (i.e., couples that tested positive) and provided more reproductive choice options compared with no screening, but were associated with higher costs. While all screening strategies had similar values for health outcomes, when comparing all strategies together, universal screening with standard panels was the most cost-effective strategy for both preconception and prenatal periods. The incremental cost-effectiveness ratios (ICERs) of universal screening with standard panels compared with no screening in the preconception period were $29,106 per additional at-risk pregnancy detected and $367,731 per affected birth averted; the corresponding ICERs in the prenatal period were about $29,759 per additional at-risk pregnancy detected and $431,807 per affected birth averted.We estimated that publicly funding a universal carrier screening program in the preconception period over the next 5 years would require between $208 million and $491 million. Publicly funding a risk-based screening program in the preconception period over the next 5 years would require between $1.3 million and $2.7 million. Publicly funding a universal carrier screening program in the prenatal period over the next 5 years would require between $128 million and $305 million. Publicly funding a risk-based screening program in the prenatal period over the next 5 years would require between $0.8 million and $1.7 million. Accounting for treatment costs of the screened health conditions resulted in a decrease in the budget impact of universally provided carrier screening programs or cost savings for risk-based programs.Participants value the perceived potential positive impact of carrier screening programs such as medical benefits from early detection and treatment, information for reproductive decision-making, and the social benefit of awareness and preparation. There was a strong preference expressed for thorough, timely, unbiased information to allow for informed reproductive decision-making. Conclusions: Carrier screening for CF, FXS, hemoglobinopathies and thalassemia, and SMA is effective at identifying at-risk couples, and test results may impact preconception and reproductive decision-making.The cost-effectiveness and budget impact of carrier screening programs are uncertain for Ontario. Over the short term, carrier screening programs are associated with higher costs, and also higher chances of detecting at-risk pregnancies compared with no screening. The 5-year budget impact of publicly funding universal carrier screening programs is larger than that of risk-based programs. However, accounting for treatment costs of the screened health conditions results in a decrease in the total additional costs for universal carrier screening programs or in cost savings for risk-based programs.The people we spoke with who had sought out carrier screening valued the potential medical benefits of early detection and treatment, particularly the support and preparation for having a child with a potential genetic condition.

Harmonising Assistive Technology Assessment Data: A Case Study in Nepal.

There is a practical demand to maximise existing data to understand and meet the assistive technology (AT) needs in dynamic populations. Harmonisation can generate new insight by integrating multiple datasets that were not previously comparable into a single longitudinal dataset. We harmonised AT assessment data from three population-based surveys collected several years apart in Nepal: the Living Conditions of Persons with Disabilities (2014-2015), the Multiple Indicator Cluster Survey (2019), and the rapid Assistive Technology Assessment (2022). The harmonised dataset demonstrates a method that can be used for unifying AT surveys in other settings and conducting trend analyses that are necessary for monitoring a population's dynamic AT needs. We set out to explore AT data's potential for harmonisation, and learned there is indeed value in this approach for situating disparate datasets, though the methodology proposed will need further validation.

Perspectives on how to build bridges between regulation, health technology assessment and clinical guideline development: a qualitative focus group study with European experts.

OBJECTIVE: Improving synergy among regulation, health technology assessment (HTA) and clinical guideline development is relevant as these independent processes are building on shared evidence-based grounds. The two objectives were first to assess how convergence of evidentiary needs among stakeholders may be achieved, and second, to determine to what extent convergence can be achieved. DESIGN: Qualitative study using eight online dual-moderator focus groups. SETTING: Discussions had a European focus and were contextualised in four case studies on head and neck cancer, diabetes mellitus, multiple sclerosis and myelodysplastic syndromes. PARTICIPANTS: Forty-two experienced (over 10 years) European regulators, HTA representatives and clinicians participated in the discussion. INTERVENTIONS: Participants received information on the case study and research topic in advance. An introductory background presentation and interview guide for the moderators were used to steer the discussion. RESULTS: Convergence may be achieved through improved communication institutionalised in multistakeholder early dialogues, shared definitions and shared methods. Required data sets should be inclusive rather than aligned. Deliberation and decision-making should remain independent. Alignment could be sought for pragmatic clinical trial designs and patient registries. Smaller and lower-income countries should be included in these efforts. CONCLUSION: Actors in the field expressed that improving synergy among stakeholders always involves trade-offs. A balance needs to be found between the convergence of processes and the institutional remits or geographical independence. A similar tension exists between the involvement of more actors, for example, patients or additional countries, and the level of collaboration that may be achieved. Communication is key to establishing this balance.

Moral and Social Values in Evidence-Informed Deliberative Processes for Health Benefit Package Design Comment on "Evidence-Informed Deliberative Processes for Health Benefit Package Design - Part II: A Practical Guide".

An evidence-informed deliberative process (EDP) is defined as "a practical and stepwise approach for health technology assessment (HTA) bodies to enhance legitimate health benefit package design based on deliberation between stakeholders to identify, reflect and learn about the meaning and importance of values, informed by evidence on these values." In this commentary, I discuss some considerations for EDPs that arise from acknowledging the difference between social and moral values. First, the best practices for implementing EDPs may differ depending on whether the approach is grounded in moral versus social values. Second, the goals of deliberation may differ when focused on moral versus social values. I conclude by offering some considerations for future research to support the use of EDPs in practice, including the need to assess how different approaches to appraisal (eg, more quantitative versus qualitative) impact perceptions of the value of deliberation itself.

Evidence-Informed Deliberative Processes for UHC: Progress, Potential and Prudence Comment on "Evidence-Informed Deliberative Processes for Health Benefit Package Design - Part II: A Practical Guide".

In their recent article on evidence-informed deliberative processes (EDPs) for health benefit package decisions, Oortwijn et al examine how the different steps of EDP play out in eight countries with relatively mature institutions for using health technology assessment (HTA). This commentary examines how EDP addresses stakeholder involvement in decision-making for equitable progress towards universal health coverage (UHC). It focuses on the value of inclusiveness, the need to pay attention to trade-offs between desirable features of EDP and the need to broaden the scope of processes examined beyond those specifically tied to producing and using HTAs . It concludes that EDPs have contributed to significant progress for health benefit design decisions worldwide and holds much potential in further application. At the same time, this commentary calls for prudence: investments in EDPs should be efficiently deployed to enhance the pre-existing legislative, institutional and political framework that exist to promote fair and legitimate healthcare decisions.

Access in all areas? A round up of developments in market access and health technology assessment: part 1.

In this new series reviewing recent developments in market access, we highlight publications investigating health technology assessment (HTA) guidance, review processes and outcomes across the world and discuss how forthcoming changes in the HTA and regulatory environment in the European Union may allow for more consistency in decision making.

Integration of real-world evidence from different data sources in health technology assessment.

Real-world evidence (RWE) is being increasingly used by a wide range of stakeholders involved in the therapeutic product lifecycle but remains underutilized in the health technology assessment (HTA) process. RWE aims to fill the current evidence gaps, reduce the uncertainty around the benefits of medical technologies, and better understand the long-term impact of health technologies in real-world conditions. Despite the minimal use of RWE in some elements of HTA, there has been a larger push to further utilize RWE in the HTA processes. HTA bodies, as other stakeholders, work towards developing more robust means to leverage RWE from various data sources in the HTA processes. However, these agencies need to overcome important challenges before the broader incorporation of RWE into their routine practice. This paper aims to explore the extensive integration of RWE utilizing diverse sources of RWD. We discuss the utilization of RWE in HTA processes, considering aspects such as when, where, and how RWE can be effectively applied. Additionally, we seek the potential challenges and barriers associated with the utilization of different data sources.

Patient and citizen participation at the organizational level in health technology assessment: an exploratory study in five jurisdictions.

OBJECTIVE: While patient participation in individual health technology assessments (HTAs) has been frequently described in the literature, patient and citizen participation at the organizational level is less described and may be less understood and practiced in HTA bodies. We aimed to better understand its use by describing current practice. METHOD: To elicit descriptive case studies and insights we conducted semi-structured interviews and open-ended questionnaires with HTA body staff and patients and citizens participating at the organizational level in Belgium, France, Quebec, Scotland, and Wales. RESULTS: We identified examples of organizational participation in managerial aspects: governance, defining patient involvement processes, evaluation processes and methods, and capacity building. Mechanisms included consultation, collaboration, and membership of standing (permanent) groups. These were sometimes combined. Participants were usually from umbrella patient organizations and patient associations, as well as individual patients and citizens. DISCUSSION: Although the concept, participation at the organizational level, is not well-established, we observed a trend toward growth in each jurisdiction. Some goals were shared for this participation, but HTA bodies focused more on instrumental goals, especially improving participation in HTAs, while patients and citizens were more likely to offer democratic and developmental goals beyond improving participation processes. CONCLUSION: Our findings provide rationales for organizational-level participation from the perspectives of HTA bodies and patients. The case studies provide insights into how to involve participants and who may be seen as legitimate participants. These findings may be useful to HTA bodies, the patient sector, and communities when devising an organizational-level participation framework.

Ethical soundness of health technology assessment reports in Islamic Republic of Iran.

Background: Health technology assessment (HTA) is a conventional method for evaluating reasonable use of health technologies in many countries. Aims: To investigate the ethical soundness of HTA studies in Islamic Republic of Iran. Methods: All HTA reports published by the HTA office until 2020 were reviewed using the HTA Core Model and the Q-SEA questionnaires. Results: We evaluated 91 reports for ethical soundness. The research question, literature search and inclusion/exclusion criteria were included in 91.2%, 83.5% and 82.4% of the HTA reports, respectively. Only 13.2% of the reports explicitly stated the objective of the analysis and 6.6% stated the ethics framework. Only 2.2%, 4.4%, 9.9%, 9.9%, 14.3%, and 2.2%, respectively, of the reports, complied with the completeness, bias, policy implications, other implications, conceptual clarification, and conflicting values. Conclusions: HTA reports in the Islamic Republic of Iran require coordinated and integrated framework acceptable to all stakeholders to ensure their compliance with sound ethical requirements.

Evaluating the quality of health technology assessment reports in a developing country.

Background: No single method of health technology assessment (HTA) can meet all the policy- and decision-making needs in a country. However, there should be minimum criteria for performing HTA worldwide, and many HTA agencies have reached a consensus on this. Aim: This study aimed to assess the quality of HTA reports in the Islamic Republic of Iran. Method: We examined all the HTA research reports published by the Iranian HTA office up to 2020, using the International Network of Agencies for Health Technology Assessment checklist for quality assessment. Results: A total of 97 reports were examined, of which only 10.0% provided complete and appropriate contact details for further information and 5.6% clearly stated a conflict of interest. In 87.78% of the reports, the scope of assessment was clearly determined. The quality of the reports was relatively appropriate as well as details of the sources of information and text search strategies. Some 7.8%, 74.4%, 11.1%, 8.9%, and 4.4%, respectively, of the reports considered legal aspects, economic analysis, ethical implications, social implications, and other stakeholder perspectives. Conclusion: We recommend that minimum standards be established for the HTA process so that healthcare policy- and decision-makers can make reliable decisions on the basis of the HTA reports.

Do France, Germany, and Italy agree on the added therapeutic value of medicines?

OBJECTIVES: The Health Technology Assessment (HTA) of medicines is performed separately at the country level with some differences, but Italy, France, and Germany have implemented price and reimbursement systems strongly focused on the Added Therapeutic Value (ATV). This study investigates the level of agreement on ATV assessments by Agenzia Italiana del Farmaco (AIFA), Haute Autorité de Santé (HAS), and Gemeinsamer Bundesausschuss (G-BA). METHODS: A database was created collecting all information about drugs with innovativeness status requests in Italy from July 2017 to December 2022 and populated with the corresponding HAS and G-BA ATV assessments. The primary comparative analysis was conducted by grouping the ATV ratings into "higher added value" and "lower or no added value", while a secondary analysis considered the Italian innovativeness status as a criterion to include the quality of evidence assessment. The concordance between ATV assessments was investigated through percentage agreement and unweighted Cohen k-value. RESULTS: 189 medicines/indications were included. The greatest agreement was found when comparing G-BA versus HAS (82 percent; k = 0.61, substantial agreement). Lower levels of agreements were observed for AIFA versus HAS and AIFA versus G-BA (respectively 52 percent; k = 0.17 and 57 percent; k = 0.25). The secondary analysis led to a reconciliation to moderate agreement for AIFA versus HAS (72 percent; k = 0.45) and AIFA versus G-BA (74 percent; k = 0.47). CONCLUSIONS: A high degree of concordance between HTA organizations is reached when considering jointly ATV and quality of evidence, suggesting that the system is extensively mature to make a Joint Clinical Assessment, avoiding duplications and reducing access inequalities.