Health Technology Assessment Framework Research Articles

The Development of a New Approach for the Harmonized Multi-Sectoral and Multi-Country Cost Valuation of Services: The PECUNIA Reference Unit Cost (RUC) Templates.

Increasing healthcare costs require evidence-based resource use allocation for which assessing costs rigorously and comparably is crucial. Harmonized cross-country costing methods for evaluating interventions from a societal perspective are lacking. This study presents the development process and content of the service costing templates developed as part of the European project PECUNIA. The six developmental steps towards technological readiness of the templates included (1) a common conceptual costing framework and review of methodological costing issues, (2) harmonization strategy formulation, (3) proof-of-concept with expert feedback, (4) piloting, (5) validation, and (6) demonstration in six European countries. The PECUNIA Reference Unit Cost (RUC) Templates for service costing are three new self-completion tools to be used with secondary or primary data for top-down micro-costing or top-down gross-costing approaches. Complementary data collection and unit cost aggregation/weighting templates are available. The applications leading to the final versions including (4) piloting through calculation of 15-unit costs, (5) validation within a Health Technology Assessment framework, and (6) RUC calculations mostly based on secondary data demonstrated the templates' general feasibility, with feedback for improved usability incorporated and a supplementary user guide developed. The validated PECUNIA RUC Templates for multi-sectoral and multi-country service costing allow for harmonized RUC development while incorporating flexibility and transparency in the choice of costing approaches, data sources and magnitude of remaining heterogeneity. The templates are expected to significantly improve the quality, comparability and availability of unit costs for economic evaluations, and promote the transferability of service cost information across Europe.

Guiding Principles for Evaluating Vaccines in Joint Health Technology Assessment in the European Union: Preparing for the European Union’s Regulation on Health Technology Assessment for Vaccines

ObjectivesThe appraisal of vaccines in the European Union (EU) currently involves many different decision-making bodies and processes. The objective of this study was to help inform the development of standardized methodology and vaccine-specific processes for use in the EU Regulation on health technology assessment (HTA). MethodsLiterature reviews and expert consultation were conducted to identify current practices and gaps related to vaccine appraisals and to develop guiding principles for the joint clinical assessment of vaccines. ResultsWe found that significant variation exists across the EU member states in the decision-making processes when clinically evaluating vaccines. Three guiding principles consisting of 13 recommendations were developed to help inform the development of decision-making frameworks for the joint clinical assessment of vaccines in the EU: (1) support the creation of appropriate terminology and measurements for clinical appraisals of vaccines; (2) develop inclusive, timely, and transparent vaccine appraisal processes to support stronger evidence generation for vaccine decision making and appraisal; and (3) improve the collection and interoperability of real-world data, including robust surveillance, to foster evidence generation and support the standardization of vaccine clinical appraisals. ConclusionsGiven the significance of vaccines for public health, there is an urgency to develop standardized and vaccine-specific methodologies and processes for use in the EU joint HTA framework. The proposed guiding principles could support the effective implementation of the EU Regulation on HTA for vaccines and have the potential to ensure consistent, transparent, and timely access to new vaccines in the EU.

The development of a roadmap for Health Technology Assessment implementation in Moldova

Abstract* Background Moldova strives for universal health coverage (UHC) and considers health technology assessment (HTA) an important policy instrument to inform the choice of services that should be covered to progressively realize UHC. It plays a key role in determining which technologies are coved, considering various dimensions often including cost-effectiveness, budget impact, and feasibility. This paper reports on work undertaken to develop a roadmap for HTA implementation, using evidence-informed deliberative processes (EDPs) as the guiding framework. Methods Between 2020 and 2022, we undertook several activities that informed the roadmap. We conducted a needs assessment and an assessment of European HTA best practices using a combination of desk research, interviews and surveys. We then conducted a document review of six selected HTA systems, complimented by expert interviews from three neighbouring countries. Results The roadmap provided a suggested HTA process, which included instructions on how to execute the different steps of the EDP framework to enhance the legitimacy of HTA-informed decision-making. The roadmap encompasses several aspects such as how to organize stakeholder involvement, how to identify and operationalize decision criteria, and how to make the decision process transparent. Guidance was given to the country on establishing a legal framework for HTA; a communication strategy; strengthening capacity and leveraging HTA expertise. The country was also given tailored advice on the positioning of the HTA-agency, first as an entity within the Ministry of Health or the National Agency of Public Health but with the long-term vision for it to be independent of any state institution. Conclusions Despite several challenges, including a partial disruption because of the Covid-19 pandemic, the development of the roadmap for HTA implementation was completed and agreed upon by the Ministry of Health in Moldova. This work has helped motivate and support the country in HTA institutionalization.

Suitability of the Current Health Technology Assessment of Innovative Artificial Intelligence-Based Medical Devices: Scoping Literature Review.

Artificial intelligence (AI)-based medical devices have garnered attention due to their ability to revolutionize medicine. Their health technology assessment framework is lacking. This study aims to analyze the suitability of each health technology assessment (HTA) domain for the assessment of AI-based medical devices. We conducted a scoping literature review following the PRISMA (Preferred Reporting Items for Systematic Reviews and Meta-Analyses) methodology. We searched databases (PubMed, Embase, and Cochrane Library), gray literature, and HTA agency websites. A total of 10.1% (78/775) of the references were included. Data quality and integration are vital aspects to consider when describing and assessing the technical characteristics of AI-based medical devices during an HTA process. When it comes to implementing specialized HTA for AI-based medical devices, several practical challenges and potential barriers could be highlighted and should be taken into account (AI technological evolution timeline, data requirements, complexity and transparency, clinical validation and safety requirements, regulatory and ethical considerations, and economic evaluation). The adaptation of the HTA process through a methodological framework for AI-based medical devices enhances the comparability of results across different evaluations and jurisdictions. By defining the necessary expertise, the framework supports the development of a skilled workforce capable of conducting robust and reliable HTAs of AI-based medical devices. A comprehensive adapted HTA framework for AI-based medical devices can provide valuable insights into the effectiveness, cost-effectiveness, and societal impact of AI-based medical devices, guiding their responsible implementation and maximizing their benefits for patients and health care systems.

Implementing the EU HTA regulation: Insights from semi-structured interviews on patient expectations, Belgian and European institutional perspectives, and industry outlooks.

Introduction: The goal of the Health Technology Assessment (HTA) Regulation 2021/2282 is to establish a more harmonized HTA framework, fostering member states cooperation and enabling equal patient access to innovative health technologies in Europe. This research aimed to assess the impact of the regulation on national HTAs, the strategic implications for health technology developers, and its influence on price and reimbursement negotiations. Methods: A scoping literature review encompassing peer-reviewed literature as well as grey literature was conducted. Between February and March 2023, semi-structured interviews (n = 20) were performed with stakeholders from Belgian governmental institutions, European institutions, advanced therapy medicinal product developers, academics, and sickness funds. The interviews were analyzed using the framework analysis method. Results: Numerous steps, such as the development of implementing acts and procedural guidelines remain to be taken. At member state level, national/regional HTA bodies and payers must act to adopt the new concepts of Joint Scientific Consultations (JSC) and Joint Clinical Assessments (JCA) within their national legislation, as well as revise their timelines and prepare for interactions at a European level. Compiling a harmonized PICO (Population, Intervention, Comparator, and Outcome), adapting local procedures, and increasing capacity to actively take part in the JSC and JCA are seen as primary barriers by several stakeholders. Training and education will help HTA bodies, payers, and health technology developers to participate in the European processes. Conclusion: While practical and legal challenges were identified, recommendations (such as actively preparing for the upcoming changes and increasing capacity while providing training) were provided to adapt national and European procedures to the needs of the HTA Regulation 2021/2282. The importance of fostering collaborations and aligning local HTA procedures with the new way of working set out by the Regulation was demonstrated with this study.

A blueprint for health technology assessment capacity building: lessons learned from Malta.

The development and strengthening of health technology assessment (HTA) capacity on the individual and organizational level and the wider environment is relevant for cooperation on HTAs. Based on the Maltese case, we provide a blueprint for building HTA capacity. A set of activities were developed based on Pichler et al.'s framework and the starting HTA capacity in Malta. Individual level activities focused on strengthening epidemiological and health economic skills through online and in-person training. On the organizational level, a new HTA framework was developed which was subsequently utilized in a shadow assessment. Awareness campaign activities raised awareness and support in the wider environment where HTAs are conducted and utilized. The time needed to build HTA capacity exceeded the planned twoyears accommodating the learning progress of the assessors. In addition to the planned trainings, webinars supplemented the online courses, allowing for more knowledge exchange. The advanced online course was extended over time to facilitate learning next to the assessors' daily tasks. Training sessions were added to implement the new economic evaluation framework, which was utilized in a second shadow assessment. Awareness by decision-makers was achieved with reports, posters, and an article on the current and developing HTA capacity. It takes time and much (hands-on) training to build skills for conducting complex assessment such as HTAs. Facilitating exchange with knowledgeable parties is crucial for succeeding as well as the buy-in of local managers motivating staff. Decision-makers need to be on-boarded for the continued success of HTA capacity building.

Commissioning [Integrated] Care in England: An Analysis of the Current Decision Context

Background: The emergence of Integrated Care Systems (ICSs) across England poses an additional challenge and responsibility for local commissioners to accelerate the implementation of integrated care programmes and improve the overall efficiency across the system. To do this, ICS healthcare commissioners could learn from the experience of the former local commissioning structures and identify areas of improvement in the commissioning process. 
 Aim & Audience: This study describes the investment decision process in integrated care amid the transition toward ICSs, highlights challenges, and provides recommendations to inform ICSs in their healthcare commissioning role. It is expected that this study encourages an open discussion within the emerging ICSs on the barriers and enablers of the local commissioning of integrated care. We also believe that findings of this study are likely to be of international interest as countries are gradually moving towards integrated care, and local purchasers of healthcare from high-income economies are likely to encounter similar challenges as the ones faced by local commissioners in England.
 Methods: We conducted twenty-six semi-structured interviews with local commissioners and other relevant stakeholders in South East England in 2021. Interviews were supplemented with existing evidence from the literature.
 Results: England’s local healthcare commissioning has made the transition towards a new organisational architecture, with some integrated care programmes running, and a dual top-down and bottom-up prioritisation process in place. The commissioning and consequent development of integrated care programmes have been hindered by various barriers, including difficulties in accessing and using information, operational challenges, and resource constraints. Investment decisions have mainly been driven by national directives and budget considerations, with a mixture of subjective and objective approaches. This contrasts with what seems to be a set of desirable prioritisation criteria, with the triple aim framework at the front. A systematic and data-driven framework could replace this ad-hoc prioritisation of integrated care and contribute to a more rational and transparent commissioning process. To develop such a framework, a good reference point could be the health technology assessment framework established at national level to guide priority-setting decisions. 
 Conclusion & next-steps: The local healthcare commissioning structures in England are complex, the evidence available is rich, and integrated care is undoubtedly the goal to achieve. However, the multiple barriers that commissioners face hinder the development of integrated care programmes and limit the local use of evidence in healthcare decision-making. The emerging ICSs seem to open an opportunity for local commissioners to strengthen the commissioning process of integrated care with evidence-based priority-setting approaches similar to the well-established health technology assessment framework at the national level. To support this, we are developing a data-driven framework to monitor and assess ‘integrated care programmes’. 

Holistic Human-Serving Digitization of Health Care Needs Integrated Automated System-Level Assessment Tools.

Digital health tools, platforms, and artificial intelligence- or machine learning-based clinical decision support systems are increasingly part of health delivery approaches, with an ever-greater degree of system interaction. Critical to the successful deployment of these tools is their functional integration into existing clinical routines and workflows. This depends on system interoperability and on intuitive and safe user interface design. The importance of minimizing emergent workflow stress through human factors research and purposeful design for integration cannot be overstated. Usability of tools in practice is as important as algorithm quality. Regulatory and health technology assessment frameworks recognize the importance of these factors to a certain extent, but their focus remains mainly on the individual product rather than on emergent system and workflow effects. The measurement of performance and user experience has so far been performed in ad hoc, nonstandardized ways by individual actors using their own evaluation approaches. We propose that a standard framework for system-level and holistic evaluation could be built into interacting digital systems to enable systematic and standardized system-wide, multiproduct, postmarket surveillance and technology assessment. Such a system could be made available to developers through regulatory or assessment bodies as an application programming interface and could be a requirement for digital tool certification, just as interoperability is. This would enable health systems and tool developers to collect system-level data directly from real device use cases, enabling the controlled and safe delivery of systematic quality assessment or improvement studies suitable for the complexity and interconnectedness of clinical workflows using developing digital health technologies.

Arts and creativity interventions for improving health and wellbeing in older adults: a systematic literature review of economic evaluation studies

BackgroundAs the population ages, older people account for a larger proportion of the health and social care budget. A significant body of evidence suggests that arts and creativity interventions can improve the physical, mental and social wellbeing of older adults, however the value and/or cost-effectiveness of such interventions remains unclear.MethodsWe systematically reviewed the economic evidence relating to such interventions, reporting our findings according to PRISMA guidelines. We searched bibliographic databases (MEDLINE, EMBASE, Econlit and Web of Science and NHSEED), trial registries and grey literature. No language or temporal restrictions were applied. Two screening rounds were conducted independently by health economists experienced in systematic literature review. Methodological quality was assessed, and key information extracted and tabulated to provide an overview of the published literature. A narrative synthesis without meta-analysis was conducted.ResultsOnly six studies were identified which provided evidence relating to the value or cost-effectiveness of arts and creativity interventions to improve health and wellbeing in older adults. The evidence which was identified was encouraging, with five out of the six studies reporting an acceptable probability of cost-effectiveness or positive return on investment (ranging from £1.20 to over £8 for every £1 of expenditure). However, considerable heterogeneity was observed with respect to study participants, design, and outcomes assessed. Of particular concern were potential biases inherent in social value analyses.ConclusionsDespite many studies reporting positive health and wellbeing benefits of arts and creativity interventions in this population, we found meagre evidence on their value or cost-effectiveness. Such evidence is costly and time-consuming to generate, but essential if innovative non-pharmacological interventions are to be introduced to minimise the burden of illness in this population and ensure efficient use of public funds. The findings from this review suggests that capturing data on the value and/or cost-effectiveness of such interventions should be prioritised; furthermore, research effort should be directed to developing evaluative methods which move beyond the confines of current health technology assessment frameworks, to capture a broader picture of ‘value’ more applicable to arts and creativity interventions and public health interventions more generally.PROSPERO registrationCRD42021267944 (14/07/2021).

OP102 Towards Universal Health Coverage: Health Technology Assessment Roadmap Development In The Emirate Of Abu Dhabi Involving The Whole Ecosystem

IntroductionThe mission of the Department of Health (DoH) of Abu Dhabi in the United Arab Emirates is to provide its population with a healthy life and world leading preventive and curative services. While the DoH has regulations in place to grant market approval to new health technologies, there is a need to develop a clear overall framework for reimbursement and disinvestment decisions. Establishing a structured health technology assessment (HTA) framework is critical for informing decisions on health technologies that offer value for money, with the aim of improving equitable access to health care, financial risk protection, and, ultimately, better health outcomes.MethodsDuring 2022, the DoH collaborated with the Radboud University Medical Center to explore the feasibility of applying an evidence-informed deliberative process (EDP) HTA approach through workshops and interviews involving all stakeholders in the ecosystem, such as policy makers, principal investigators, providers, patients and public groups, product manufacturers, payers, and purchasers. A situational analysis was conducted to collect stakeholders’ views and build EDPs. Based on this analysis, a structured roadmap was developed.ResultsThe comprehensive five-year roadmap to implement a holistic HTA framework in Abu Dhabi consisted of five major elements, starting with the establishment of an appropriate HTA policy framework as a foundation. Abu Dhabi should firmly establish its HTA structure and program (in one to two years), and at the same time invest in developing and retaining HTA training capacity so that over time (within three to five years) the country can build up its own expertise to sustain the program. This needs to be accompanied by continuous awareness raising among all relevant stakeholders.ConclusionsThis roadmap is the first and most important step toward implementing a holistic HTA framework in Abu Dhabi. Technical work needs to be complemented by continuously raising awareness and involving all relevant stakeholders. Looking at the initial results and international benchmarks, HTA will significantly contribute to achieving a sustainable, high quality healthcare system.

OP114 Health Technology Assessment Of Digital Technologies: Results From Two Past Examples And Thoughts For The Future

IntroductionDigital health care is a rapidly growing and increasingly diverse area of medicine and social care. Health technology assessment (HTA) agencies around the world are engaging with digital health technologies and developing methods for appropriately assessing their value. Given our experience in HTA for pharmaceuticals and how methodologies must differ when assessing medical devices and diagnostics, we must be proactive in rapidly developing useful methods for assessing a variety of digital health technologies.MethodsWe will first present the results of two completed HTAs from the UK and contrast their methods and results, while looking at the UK approach to assessing digital health care in general. Next, we will present preliminary results from an ongoing consensus conference study of HTA’s for integrated digital technologies. This study will use a modified Delphi methodology to develop statements for discussion at a consensus conference. The primary research question is “how digital technologies can help build resilient healthcare systems and how HTAs can assess their value”.ResultsAn HTA conducted in the UK of an artificial intelligence-enabled ambulatory electrocardiogram device resulted in a recommendation for further real-world evidence generation. Another HTA of a digital therapeutic app for insomnia, which was also conducted in the UK, resulted in a positive recommendation but the process was lengthy and needed to be flexible. The preliminary results of an upcoming consensus conference study will also be presented. The consensus panel will consist of a range of stakeholders from digital experts, HTA practitioners, clinicians, health economists, industry representatives, and policy makers.ConclusionsDigital technology is now a broad field with a wide range of very different technologies included in its definition. Questions remain as to whether digital technologies should be assessed using a traditional pharma-based framework for HTA, a MedTech model, or something else entirely. Given our past experience of developing flexible methods for HTA, we have an opportunity to develop useful recommendations and best practices from the ground up.

Transporting Comparative Effectiveness Evidence Between Countries: Considerations for Health Technology Assessments.

Internal validity is often the primary concern for health technology assessment agencies when assessing comparative effectiveness evidence. However, the increasing use of real-world data from countries other than a health technology assessment agency's target population in effectiveness research has increased concerns over the external validity, or "transportability", of this evidence, and has led to a preference for local data. Methods have been developed to enable a lack of transportability to be addressed, for example by accounting for cross-country differences in disease characteristics, but their consideration in health technology assessments is limited. This may be because of limited knowledge of the methods and/or uncertainties in how best to utilise them within existing health technology assessment frameworks. This article aims to provide an introduction to transportability, including a summary of its assumptions and the methods available for identifying and adjusting for a lack of transportability, before discussing important considerations relating to their use in health technology assessment settings, including guidance on the identification of effect modifiers, guidance on the choice of target population, estimand, study sample and methods, and how evaluations of transportability can be integrated into health technology assessment submission and decision processes.

Challenges in the Evaluation of Emerging Highly Specialised Technologies: Is There a Role for Living HTA?

There is currently deep uncertainty about the clinical benefits and cost effectiveness of highly specialised technologies (HSTs), like gene and cell therapies. These treatments are novel, typically have high upfront costs, the patient populations are small and heterogenous, there is minimal information on their long-term safety and effectiveness, and data are limited and often of poor quality. With the increasing number of these technologies and their high cost burden on governments and health care providers, policy makers are currently walking a decision tightrope. On the one hand, an unfavourable funding decision could potentially limit patient access to life-saving treatments, while on the other, a favourable decision could result in unsustainable budget impacts and perhaps poorer patient health outcomes. Health technology assessment (HTA) is meant to determine the value of a health technology in order to promote an equitable, efficient, and high-quality health system. However, standard HTA processes have failed to mitigate the deep uncertainties associated with these technologies. In this paper, we propose a Living HTA framework to address these challenges. This framework includes a one-off process for making explicit the societal values associated with HSTs. These would inform the decision-making approach, data collection and the development of disease-specific reference models to be used by industry sponsors as the basis for their submissions for public funding. Coverage with an evidence development mechanism is also proposed by which data can be collected in real time to update the reference model on a rolling basis, thereby allowing re-assessment of the clinical and cost effectiveness of individual HSTs. The HTA would be 'live' until the results indicate there is sufficient certainty for the funding decision to be confirmed, the price changed or the funding removed.

Toward Best Practices for Economic Evaluations of Tumor-Agnostic Therapies: A Review of Current Barriers and Solutions

ObjectivesCancer therapies targeting tumor-agnostic biomarkers are challenging traditional health technology assessment (HTA) frameworks. The high prevalence of nonrandomized single-arm trials, heterogeneity, and small benefiting populations are driving outcomes uncertainty, challenging healthcare decision making. We conducted a structured literature review to identify barriers and prioritize solutions to generating economic evidence for tumor-agnostic therapies. MethodsWe searched MEDLINE and Embase for English-language studies conducting economic evaluations of tumor-agnostic treatments or exploring related challenges and solutions. We included studies published by December 2022 and supplemented our review with Canadian Agency for Drugs and Technologies in Health and National Institute for Health and Care Excellence technical reports for approved tumor-agnostic therapies. Three reviewers abstracted and summarized key methodological and empirical study characteristics. Challenges and solutions were identified through authors’ statements and categorized using directed content analysis. ResultsTwenty-six studies met our inclusion criteria. Studies spanned economic evaluations (n = 5), reimbursement reviews (n = 4), qualitative research (n = 1), methods validations (n = 3), and commentaries or literature reviews (n = 13). Challenges encountered related to (1) the treatment setting and clinical trial designs, (2) a lack of data or low-quality data on clinical and cost parameters, and (3) an inability to produce evidence that meets HTA guidelines. Although attempted solutions centered on analytic approaches for managing missing data, proposed solutions highlighted the need for real-world evidence combined with life-cycle HTA to reduce future evidentiary uncertainty. ConclusionsTherapeutic innovation outpaces HTA evidence generation and the methods that support it. Existing HTA frameworks must be adapted for tumor-agnostic treatments to support future economic evaluations enabling timely patient access.

A qualitative study on the feasibility and acceptability of institutionalizing health technology assessment in Malawi

ObjectiveThe objective of this study was to assess the feasibility and acceptability of institutionalizing Health Technology Assessment (HTA) in Malawi.MethodsThis study employed a document review and qualitative research methods, to understand the status of HTA in Malawi. This was complemented by a review of the status and nature of HTA institutionalization in selected countries.Qualitative research employed a Focus Group Discussion (FGD ) with 7 participants, and Key Informant Interviews (KIIs) with12 informants selected based on their knowledge and expertise in policy processes related to HTA in Malawi.Data extracted from the literature was organized in Microsoft Excel, categorized according to thematic areas and analyzed using a literature review framework. Qualitative data from KIIs and the FGD was analyzed using a thematic content analysis approach.ResultsSome HTA processes exist and are executed through three structures namely: Ministry of Health Senior Management Team, Technical Working Groups, and Pharmacy and Medicines Regulatory Authority (PMRA) with varyingdegrees of effectiveness.The main limitations of current HTA mechanisms include limited evidence use, lack of a standardized framework for technology adoption, donor pressure, lack of resources for the HTA process and technology acquisition, laws and practices that undermine cost-effectiveness considerations. KII and FGD results showed overwhelming demand for strengthening HTA in Malawi, with a stronger preference for strengthening coordination and capacity of existing entities and structures.ConclusionThe study has shown that HTA institutionalization is acceptable and feasible in Malawi. However, the current committee based processes are suboptimal to improve efficiency due to lack of a structured framework. A structured HTA framework has the potential to improve processes in pharmaceuticals and medical technologies decision-making.In the short to medium term, HTA capacity building should focus on generating demand and increasing capacity in cost-effectiveness assessments. Country-specific assessments should precede HTA institutionalization as well as recommendations for new technology adoptions.

Understanding of the Readiness for HTA Implementation in Jordan as a Step Towards Universal Health Coverage

As Jordan strives to achieve universal health coverage, the mechanism for determining which health technologies to include in the basket of reimbursed services has become increasingly important. This study sought to understand stakeholder perspectives in the Jordanian health system regarding the readiness and need to implement health technology assessment (HTA) to support decision-making quality and transparency, ensure value for money on health system spending, and support the achievement of universal health coverage. This study used a cross-sectional survey methodology, and a quantitative analysis was conducted. A questionnaire based on the HTA implementation scorecard was administered in-person to capture responses regarding fourteen dimensions. Thirty-one responses from representatives across the Ministry of Health, regulatory authority, and other stakeholders in the national health system were collected. Most respondents were familiar with HTA and there was nearly unanimous agreement on the need for HTA implementation in Jordan. While the perspectives on how the implementation would occur were varied, careful consideration may be warranted in the areas of the legal framework for HTA, the quality of available data, financial constraints, and limited human resource capacity, as Jordan progresses towards implementing HTA on the road to universal healthcare.

Real-world data for precision cancer medicine-A European perspective.

Leveraging real-world data (RWD) for drug access is necessary to overcome a key challenge of modern precision oncology: tackling numerous low-prevalence oncogenic mutations across cancers. Withholding a potentially active medication in patients with rare mutations for the sake of control chemotherapy or "best" supportive care is neither practicable nor ethically justifiable anymore, particularly as RWD could meanwhile be used instead, according to scientific principles outlined by the US Food and Drug Administration, European Medicines Agency and other stakeholders. However, practical implementation varies, with occasionally opposite recommendations based on the same evidence in different countries. In the face of growing need for precision drugs, more transparency of evaluation, a priori availability of guidance for the academia and industry, as well as a harmonized framework for health technology assessment across the European Union (EU) are imperative. These could in turn trigger infrastructural changes in national and pan-European registries, cancer management guidelines (e.g., frequency of routine radiologic restaging, inclusion of patient-reported outcomes), and the health data space, to ensure conformity with declared standards and facilitate extraction of RWD sets (including patient-level data) suitable for approval and pricing with minimal effort. For an EU-wide unification of precision cancer medicine, collective negotiation of drug supply contracts and funding solidarity would additionally be required to handle the financial burden. According to experience from pivotal European programs, off-label use could potentially also be harmonized across EU-states to accelerate availability of novel drugs, streamline collection of valuable RWD, and mitigate related costs through wider partnerships with pharmaceutical companies.

Access to novel drugs and therapeutics for children and youth: Eliciting citizens' values to inform public funding decisions.

The unique evidentiary, economicand ethical challenges associated with health technology assessment (HTA) of precision therapies limit access to novel drugs and therapeutics for children and youth, for whom such challenges are amplified. We elicited citizens' perspectives about values-based criteria relevant to the assessment of paediatric precision therapies to inform the development of a child-tailored HTA framework. We held four citizen panels virtually in May-June 2021, informed by a plain-language citizen brief summarizing global and local evidence about the challenges, policy and programmatic optionsand implementation strategies related to enhancing access to precision therapies for Canadian children and youth. Panellists were recruited through a nationally representative database, medical/patient networksand social media. We inductively coded and thematically analysed panel transcripts to generate themes and identify priority values. The perspectives of panellists (n = 45) coalesced into four overlapping themes, with attendant subthemes, relevant to a child-tailored HTA framework: (1) Childhood Distinctions: vulnerability, 'fair innings', future potential, family impacts; (2) Voice: agency of children and youth; lived versus no lived experience; (3) One versus Many: disease severity, rarity, equity, unmet needand (4) Health System Governance: funding, implementation inequities, effectivenessand safety. Participants broadly agreed that childhood distinctions, particularly family impacts, justify child-tailored HTA. Dissent arose over whose voice should inform HTA and how such perspectives are best incorporated. Citizens can offer unique insights into criteria relevant to the development or revision of HTA frameworks to capture holistic, societally responsive dimensions of value attached to unique contexts or populations, including children. Balancing the hopes and expectations of patients and caregivers for access to expensive but potentiallife-altering therapies against the opportunity costs borne by encompassing health systems is a fundamental challenge that will require rigorous methods to elicit, weighand reconcile varied views. A patient advocate served on the steering committee of this study and co-authored this article. Key informants for the Citizen Brief included patient advocates and caregivers; a separate patient advocate reviewed the Brief before dissemination. Qualitative and quantitative data were collected from the general public and caregivers of children, with written consent.

HTA56 Is Evidence on Environmental Impact Included in Health Technology Assessment and Does It Influence Decision-Making?

The global environmental impact of healthcare is high. National governments have implemented measures to reduce emissions in the healthcare sector, including the NHS in the UK, as outlined in its Long-Term Plan, 2019. When assessing new technologies, health care decision makers have the opportunity to examine their environmental impact during Health Technology Assessment (HTA). This study assessed if environmental impacts were included in HTA frameworks or guidance from a selection of HTA agencies and whether it influenced decision-making.

PP114 The Influence Of Implicit Factors On The Health Technology Assessment Deliberative Process: A Survey In Five European Countries

IntroductionImplicit factors can be defined as any criteria that play a role in the health technology assessment (HTA) deliberative process but are not part of the HTA framework. To date, very few studies have explored the influence of implicit factors on this process. This survey of HTA experts in five European countries aimed to analyze the influence of implicit factors on the HTA deliberative process.MethodsSemi-structured interviews with 20 HTA experts from five European countries (France, Germany, Italy, Spain, and the United Kingdom) were conducted from February to May 2021. The main topics of the interviews were: the HTA deliberative process; the degree of influence on the HTA deliberative process of a set of factors previously identified in a systematic literature review performed by the authors; and recommendations for improving the deliberative process.ResultsAll but two of the experts concurred that implicit factors played a role in the deliberative process. German experts considered that the factors explored had a low influence on the process. Burden of disease and unmet need scored highest, followed by the professional experience of the people involved in the HTA deliberative process. To improve the deliberative process, experts suggested expanding the external stakeholder perspective (i.e., including patients, the pharmaceutical industry, and the public), increasing transparency when revealing implicit factors, and implementing a methodology to mitigate the influence of implicit factors.ConclusionsOur survey indicates a need to increase external involvement in the process and to develop a methodology for unmasking the implicit factors in the deliberative process. This may be achieved by either updating the current frameworks to include these implicit factors or by developing new methods to address them. Further research may explore approaches to acknowledge the implicit factors in the HTA deliberative process in a systematic manner.