Health Technology Assessment Experts Research Articles

PP128 A Transparent Methodology To Assess Innovativeness Of Health Technologies At Marketing Authorization Time

IntroductionDefining drug innovation can be challenging and there is no consensus on what a truly “innovative” medicine is. The Italian Medicine Agency (AIFA) has established an approach to assess innovativeness based on therapeutic need, added therapeutic value, and quality of evidence. However, judgment can be subjective and may not be adequate for assessment at the time of marketing authorization, when only preliminary evidence – often from non-comparative or non-randomized trials – are available. We developed a transparent methodology for early assessment of innovativeness at the time of marketing authorization, based on AIFA guidelines.MethodsSince the perspective was the marketing authorization date, only data available at agency’s Medical Review or pivotal trial publications were considered. AIFA criteria were revisited, using oncology medicines approved in the last 10 years as a base case. Impact of preliminary evidence and inadequate study design was considered.ResultsEach assessment should refer to the first approved specific indication and predefined clinically relevant outcomes. When more than one study was presented, best methodological quality, larger sample and/or longer follow-up was selected. Four domains were established: Therapeutical need: existence and clinical benefits of alternative therapies; Clinical benefit added when compared to those alternatives; Suitability of study design considering adequate comparator group, relevant outcome assessed and randomization; Risk of bias. For each domain, clear and specific criteria were defined in consensus by a group of experts in health technology assessment (HTA) and were applied to all cancer drugs evaluated.ConclusionsEfficacy evidence available for marketing authorization are often based on preliminary data, arising from single randomized clinical trials or even non-comparative studies, which difficult early assessments of innovativeness. For this reason, transparent and reproducible methodologies can be useful not only to HTA bodies, but also for other key stakeholders in the pharmaceutical market, such as investors, researchers, doctors, and governments.

OP137 A Qualitative Exploration Of The National Institute For Health And Care Excellence’s Impact On International Health Technology Assessment

IntroductionThe National Institute For Health And Care Excellence (NICE) is widely acknowledged as a seminal health technology assessment (HTA) body, known for its transparent and accountable approach to decision-making. This research aimed to investigate the impact of NICE methodology and decisions on international HTA bodies. We sought to identify direct and indirect factors that may influence an international HTA body’s methods or outcomes. To the best of our knowledge, this is the first research to use a qualitative approach to understand the influence of NICE on other HTA bodies.MethodsWe conducted 13 semi-structured qualitative interviews with HTA and market access experts from industry and academia from nine countries (Brazil, Israel, Italy, Japan, Poland, Saudi Arabia, South Korea, Sweden, and the United Arab Emirates). The interview script was organized into three main sections: comparing NICE methods and processes with other HTA bodies; the impact of specific NICE decisions; and Likert scale questions (to allow for comparability of opinions).ResultsMost interviewees believed their local HTA body would consider NICE’s decision when evaluating a medicine. However, the way and extent to which NICE influences HTA varied across countries. The most common means of considering a NICE decision was as background information or context for an HTA evaluation. Generally, interviewees suggested that negative NICE decisions had more impact on local decision-making than positive decisions. Nine of the 13 interviewees agreed or strongly agreed that their country’s HTA body considers the decisions of other HTA bodies in their decision-making process. Eleven of the 13 interviewees agreed or strongly agreed that the development of their country’s HTA body methods and processes was influenced by NICE.ConclusionsNICE is perceived to be a seminal HTA body, with continued influence on HTA agencies in other countries. However, the mechanisms and extent of this influence varies considerably between countries. We suggest that implicit factors are likely to contribute more to NICE’s influence than individual decisions. Nevertheless, further research is needed to reveal these factors and increase efficiency in international HTA decision-making processes.

PP52 Will Joint European Health Technology Assessment Provide Additional Benefits Over Individual Country-wise Assessments?

IntroductionIn December 2021 the European Union (EU) Health Technology Assessment (HTA) Regulation, a key pillar of the EU Pharmaceutical Strategy, was adopted by the Council and the European Parliament. The focus areas of Joint HTA Cooperation include Joint Clinical Assessments (JCA), Joint Scientific Consultations (JSC), and joint early-stage horizon scanning. The European HTA regulation will be adopted in a stepwise approach and from 2030 onwards, all products (drugs, high-risk medical devices, and in vitro diagnostics) approved in all indications will be subjected to JCA in EU.MethodsA targeted literature research was performed for policies and the European Network for HTA (EUnetHTA) methodological guidelines describing the HTA methods including scoping process, comparators, endpoints, the applicability of evidence, and validity of clinical studies. Additionally, the anticipated opportunities and challenges were also summarized with respect to these methods.ResultsEUnetHTA put forward a timeline for different activities over the next three years as part of the new EU HTA Regulation, including key deadlines for ongoing EUnetHTA consultations on the processes and methods. EUnetHTA will set up a new ecosystem across the EU as it aims to reduce duplication and time to access by supplementing multiple national clinical assessments with a joint central assessment. In any case, assessment of added value and pricing and reimbursement decisions will still occur at the national level.Additionally, EU HTA may promote harmonization of processes, standards, and evidence requirements, which will increase predictability and simplify evidence requirements. However, differences in clinical practice, standard of care, and national priorities may lead to assessments that are not generalizable to all Member States.ConclusionsThe joint EU HTA cooperation will benefit countries which have less developed or do not have established HTA expertise or infrastructure. However, the JCA process could result in increased requirements for clinical evidence generation as relative effectiveness and relevance of outcomes to patients gain further importance for products to successfully gain access across countries.

PP13 Results Of An International Survey About Barriers In Patient Involvement In Health Technology Assessment Of Digital Health Technologies

IntroductionIn recent years, several efforts have been initiated by health technology assessment (HTA) agencies to increasingly incorporate patient involvement (PI) into their assessment processes. The rationale behind PI in HTA is that patients can give their perspective and experiences about health, illness and the use of health technologies, complementing clinical and healthcare system standpoints.A recent systematic review summarized current evidence on the barriers and facilitators of PI in HTA. Barriers and facilitators were classified according to the context in which they appear, as organizational, decision-making, political and community-related.Digital health technologies offer a singular opportunity to address some challenges faced by healthcare systems. However, we hypothesized that their intrinsic characteristics may have some implications for PI in digital HTA. We conducted a survey to gain knowledge about barriers in PI in digital HTA.MethodsThe survey was elaborated using Microsoft Forms and consisted of 31 questions divided into four blocks (organizational, decision-making, political and community-related). It was sent to three Spanish, one Catalan and one International patient umbrella organizations. The results were analysed using Microsoft Excel.ResultsA total of four responses were received from three Spanish and one Catalan patient umbrella organizations. Results showed high, partial, and low concordance among organizations in 65 percent, 19 percent and 16 percent of the answers received, respectively. According to the results, most of the barriers for PI seem to be the same for HTA of digital health technologies as for other types of health technologies. However, two or three of the four umbrella organizations consider that HTA of digital health technologies might pose greater challenges in terms of achieving significant patient participation, sufficient patient training and knowledge of HTA processes, proper preparation and tools of the HTA experts and logistics during the HTA process.ConclusionsBarriers in PI are mostly the same for digital or other types of health technologies. Few differences are related to patient training, tools, and logistics during HTA process.

Ranking Decision-Making Criteria for Early Adoption of Innovative Surgical Technologies

There is no decision-making framework in the early-adoption stage of novel surgical technologies, putting the quality of health care and resource allocation of the health care system at risk. To investigate relevant weighted criteria that decision-makers may use to make an informed decision for the early adoption of innovative surgical technologies. This multi-institutional decision analytical modeling study used a mixed-methods multicriteria decision analysis (MCDA) and had 2 phases. First, a panel of 12 experts validated decision criteria in the literature and identified additional criteria. Second, 33 Canadian experts prioritized the main criteria (domains) using the composition pairwise-comparison weight-elicitation method (analytical hierarchy process model) and ranked their subcriteria using the direct-ranking elicitation method (Likert scale). Data were analyzed, and response consistency was estimated using the consistency ratio. Analysis of variance was used to assess for significant differences between expert responses. The MCDA was conducted at McGill University between 2021 and 2023. Data were collected nationally by inviting experts in Canada. Criteria domain weights and subcriteria rankings. Priority vectors, which are priority scores analyzed and prioritized from expert responses, were used to rank criteria domains and subcriteria for decision-making on adopting new innovative surgical technologies. A total of 45 experts (33 male [73.3%] and 12 female [26.7%]) were invited with different levels of education (22 experts with MD or equivalent, 13 experts with master's degree, and 12 experts with PhD degree) and years of experience (4 experts with <10, 12 experts with 11-20, 18 experts with 21-30, and 11 experts with >30 years). Surgeon experts (23 individuals) were from all surgical disciplines, and nonsurgeon experts (22 individuals) were administrative officers in surgical device procurement, health technology assessment experts, and hospital directors. A total of 7 domains and 44 subcriteria were identified. The MCDA model found that clinical outcomes had the highest priority vector, at 0.429, followed by patients and public relevance (0.135). Hospital-specific criteria (priority vector, 0.099), technology-specific criteria (priority vector, 0.092), and physician-specific criteria (priority vector, 0.087) were the next most highly ranked. The lowest priority vectors were for economic criteria, at 0.083, and finally policies and procedures, at 0.075. There was consensus in the responses (consistency ratio = 0.006), and there were no statistically significant differences between expert responses. This study weighted priority criteria domains in importance and established ranked subcriteria for decision-making of early adoption of surgical technologies. Putting these criteria into a framework may help surgeons and decision-makers make informed decisions for the early adoption of new surgical technologies.

A framework for the definition and interpretation of the use of surrogate endpoints in interventional trials

Interventional trials that evaluate treatment effects using surrogate endpoints have become increasingly common. This paper describes four linked empirical studies and the development of a framework for defining, interpreting and reporting surrogate endpoints in trials. As part of developing the CONSORT (Consolidated Standards of Reporting Trials) and SPIRIT (Standard Protocol Items: Recommendations for Interventional Trials) extensions for randomised trials reporting surrogate endpoints, we undertook a scoping review, e-Delphi study, consensus meeting, and a web survey to examine current definitions and stakeholder (including clinicians, trial investigators, patients and public partners, journal editors, and health technology experts) interpretations of surrogate endpoints as primary outcome measures in trials. Current surrogate endpoint definitional frameworks are inconsistent and unclear. Surrogate endpoints are used in trials as a substitute of the treatment effects of an intervention on the target outcome(s) of ultimate interest, events measuring how patients feel, function, or survive. Traditionally the consideration of surrogate endpoints in trials has focused on biomarkers (e.g., HDL cholesterol, blood pressure, tumour response), especially in the medical product regulatory setting. Nevertheless, the concept of surrogacy in trials is potentially broader. Intermediate outcomes that include a measure of function or symptoms (e.g., angina frequency, exercise tolerance) can also be used as substitute for target outcomes (e.g., all-cause mortality)-thereby acting as surrogate endpoints. However, we found a lack of consensus among stakeholders on accepting and interpreting intermediate outcomes in trials as surrogate endpoints or target outcomes. In our assessment, patients and health technology assessment experts appeared more likely to consider intermediate outcomes to be surrogate endpoints than clinicians and regulators. There is an urgent need for better understanding and reporting on the use of surrogate endpoints, especially in the setting of interventional trials. We provide a framework for the definition of surrogate endpoints (biomarkers and intermediate outcomes) and target outcomes in trials to improve future reporting and aid stakeholders' interpretation and use of trial surrogate endpoint evidence. SPIRIT-SURROGATE/CONSORT-SURROGATE project is Medical Research Council Better Research Better Health (MR/V038400/1) funded.

Smart e-Skin Cancer Care in Europe During and after the Covid-19 Pandemic: a Multidisciplinary Expert Consensus.

Melanoma is the deadliest of all the skin cancers and its incidence is increasing every year in Europe. Patients with melanoma often present late to the specialist and treatment is delayed for many reasons (delay in patient consultation, misdiagnosis by general practitioners, and/or limited access to dermatologists). Beyond this, there are significant inequalities in skin cancer between population groups within the same country and between countries across Europe. The emergence of the COVID-19 pandemic only aggravated these health deficiencies. The aim was to create an expert opinion about the challenges in skin cancer management in Europe during the post COVID-19 acute pandemic and to identify and discuss the implementation of new technologies (including e-health and artificial intelligence defined as "Smart Skin Cancer Care") to overcome them. For this purpose, an ad-hoc questionnaire with items addressing topics of skin cancer care was developed, answered independently and discussed by a multidisciplinary European panel of experts comprising dermatologists, dermato-oncologists, patient advocacy representatives, digital health technology experts, and health technology assessment experts. After all panel of experts discussions, a multidisciplinary expert opinion was created. As a conclusion, the access to dermatologists is difficult and will be aggravated in the near future. This fact, together with important differences in Skin Cancer Care in Europe, suggest the need of a new approach to skin health, prevention and disease management paradigm (focused on integration of new technologies) to minimize the impact of skin cancer and to ensure optimal quality and equity.

HTA37 Incorporation of Clinical Experts in Health Technology Assessment: A Literature Review

General: To review the literature and web pages of the HTA agencies in order to know the means that facilitate the health professionals(HP) incorporation to HTA reporting processes. Specifics: To define the procedures for the selection. To describe how their incorporation is carried out. Identify the tasks and domains in which participated.

Data Governance for Real-World Data Management: A Proposal for a Checklist to Support Decision Making

ObjectivesReal-world data (RWD) and real-world evidence (RWE) can provide extensive information on healthcare for use in health technology assessment and decision making. Nevertheless, there is a lack of consensus surrounding the appropriate data governance (DG) practices for RWD/RWE. Data sharing is also a large concern, especially considering evolving data protection regulations. Our objective is to propose recommendations for international standards of evaluating the acceptability of RWD governance practices. MethodsAfter reviewing the literature, we created a checklist targeting DG practices for RWD/RWE. We then carried out a 3-round Delphi panel, including European policy makers, health technology assessment experts, and hospital managers. The consensus for each statement was measured and the checklist adjusted accordingly. ResultsThe literature review identified the main topics regarding RWD/RWE DG practices: data privacy and security, data management and linkage, data access management, and the generation and use of RWE. Members of the Delphi panel (21 experts/25 invited) were presented a total of 24 statements related to each of the topics. Experts demonstrated a progressive level of consensus and importance ratings in all topics and to most statements. We suggest a refined checklist in which the statements rated less important or with less consensus have been removed. ConclusionsThis study suggests how the DG of RWD/RWE could be qualitatively evaluated. We propose checklists that could be used by all RWD/RWE users to help ensure the quality and integrity of RWD/RWE governance and complement data protection law.

The role of quantitative bias analysis for nonrandomized comparisons in health technology assessment: recommendations from an expert workshop.

The use of treatment effects derived from nonrandomized studies (NRS) in health technology assessment (HTA) is growing. NRS carry an inherently greater risk of bias than randomized controlled trials (RCTs). Although bias can be mitigated to some extent through appropriate approaches to study design and analysis, concerns around data availability and quality and the absence of randomization mean residual biases typically render the interpretation of NRS challenging. Quantitative bias analysis (QBA) methods are a range of methods that use additional, typically external, data to understand the potential impact that unmeasured confounding and other biases including selection bias and time biases can have on the results (i.e., treatment effects) from an NRS. QBA has the potential to support HTA bodies in using NRS to support decision-making by quantifying the magnitude, direction, and uncertainty of biases. However, there are a number of key aspects of the use of QBA in HTA which have received limited discussion. This paper presents recommendations for the use of QBA in HTA developed using a multi-stakeholder workshop of experts in HTA with a focus on QBA for unmeasured confounding.

OP82 Patient Involvement In Health Technology Assessment

IntroductionPatients are increasingly involved in the decision-making process for health technology assessment (HTA), but the question of at what stage they can be involved is still controversial. In Kazakhstan, the HTA process began in 2010. Over the past 2 years, implementation of a project to develop a priority-setting tool based on evidence-informed deliberative processes has made it possible to discuss the participation of patients in HTA. We explored the possibilities of participation of patients or a patient-oriented group in the HTA process.MethodsStructured interviews were held with eight people with interests in HTA. Two were representatives of universities, two from a patient-oriented group, two experts in HTA, and two physicians. Interviews were held online by mobile phone or Zoom for 25–30 minutes. Question structures were formed based on the report ‘Patient Involvement in Health Technology Assessment in Europe 2010’. Seven stages were considered.ResultsAll participants partially or completely agreed with the involvement of patients at the HTA stages of identification and prioritization. One or two did not agree with their involvement at the HTA assessment, information production, internal and external review, and diffusion and dissemination stages. Challenges for patient involvement in HTA can be related to other commitments for patients and their carers, lack of financial affordability, conflict of interest, and lack of capacity of the HTA agency to involve them. Five participants agreed on challenges for patients to being meaningfully involved in decision-making on health technologies. These included understanding which institution makes the decisions, finding an interlocutor within the decision-making body, and understanding the decision-making process. Other issues were technical and language difficulties, lack of commitment from decision-makers and the legal or policy framework for patient involvement in HTA decision-making.ConclusionsPatients can participate in HTA, but the HTA agency must first prepare and agree on the level of patient participation, and develop measures to reduce barriers such as language difficulties, and patient obligations.

OP105 Gaps, Challenges And Priorities For Neurodegenerative Disease Research And Health Technology Assessment

IntroductionThere are many challenges encountered in the health technology assessment (HTA) of treatments for the management of neurodegenerative diseases like Alzheimer’s disease. A recent example where such challenges were visible was the assessment of aducanumab, a medication developed for Alzheimer’s disease. As part of the Innovative Medicines Initiative (IMI) Neuronet project, a series of workshops were held to understand the gaps in neurodegenerative disease research and identify the key research priorities, from the perspective of different stakeholders, that can inform decision-making and enable timely access to effective and safe treatments.MethodsThree workshops were organised by Neuronet to consult with HTA and regulatory experts, people with dementia and their supporters, and academic and industry researchers. The first consultation was with the European Working Group of People with Dementia and their supporters, and their feedback was fed into the following two workshops which were conducted with HTA and regulatory experts, and with researchers. Discussions during the workshops were used to develop a summary of the gaps in research, and priorities from the perspective of each stakeholder group to be submitted to IMI to inform future research funding allocation.ResultsKey priorities that were highlighted from an HTA and regulatory perspective were the collection of longitudinal data to understand the natural history of the disease, the generation of data on long-term clinical outcomes and the use of trial endpoints that are relevant to clinical practice. There is also a need for disease-modifying treatments that is yet to be met in this disease area. Furthermore, the involvement of the public, particularly people with dementia and relevant organisations, in setting research priorities and informing study designs was highlighted as a priority by all stakeholders.ConclusionsThere are key priorities for future neurodegenerative research from an HTA and regulatory perspective to address current gaps and challenges. It is important to involve different stakeholders in priority setting and decision-making to produce relevant and satisfactory research outputs.

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.

PP82 First Educational Trainings According To New Health Technology Assessment Guideline For Medicines In Ukraine

IntroductionThe success of the health technology assessment (HTA) implementation depends on the level of communication efficiency between all stakeholders. Taking this into consideration, the leading HTA experts of HTA Department of State Expert Center of the Ministry of Health of Ukraine launched the new educational initiative which includes training programs for pharmaceutical companies.MethodsСomprehensive review of the first developed HTA training programs based on the HTA Guideline (Number 593) “The state health technology assessment for medicines” approved on 29 March 2021 was conducted.ResultsLeading HTA experts of the HTA Department developed a training program, which was conducted for industry representatives according to the provisions of the HTA Guideline. In 2021 over twelve training sessions and two webinars were conducted. These learning events reflect harmonized international recommendations and approaches to HTA training programs, The Professional Society for Health Economics and Outcomes Research, ISPOR short courses in particular, European Network for Health Technology Assessment (EUnetHTA), The National Institute for Health and Care Excellence (NICE), Institute for Clinical and Economic Review (ICER), Agency for Health Technology Assessment and Tariff System (AOTMiT) guidelines. Two-hour webinars were aimed at giving theoretical and practical bases for building a Markov model with the help of Excel and TreeAge Software. Training sessions titled “HTA as a tool for assessing the value of health technology” differ by duration (7-hour, 4-hour, 3-hour) and cover core topics adapted to the needs of the audience. The main program components include HTA Roadmap in Ukraine, clinical section, economic section and practical case studies of building a Markov model. All training options included questionnaires at the beginning and i end to assess the quality of each program. Questionnaires are the tool that gives presenters the possibility to trace progress and transform the training material accordingly.ConclusionsDevelopment of extended capacity building programs in HTA for users and doers is highly prospective for further steps in HTA institutionalization in Ukraine.

Defining decision thresholds for judgments on health benefits and harms using the Grading of Recommendations Assessment, Development and Evaluation (GRADE) Evidence to Decision (EtD) frameworks: a protocol for a randomised methodological study (GRADE-THRESHOLD)

IntroductionThe Grading of Recommendations Assessment, Development and Evaluation (GRADE) and similar Evidence to Decision (EtD) frameworks require its users to judge how substantial the effects of interventions are on desirable...

Can we use existing guidance to support the development of robust real-world evidence for health technology assessment/payer decision-making?

Advances in the digitization of health systems and expedited regulatory approvals of innovative treatments have led to increased potential for the use of real-world data (RWD) to generate real-world evidence (RWE) to complement evidence from clinical trials. However, health technology assessment (HTA) bodies and payers have concerns about the ability to generate RWE of sufficient quality to be pivotal evidence of relative treatment effectiveness. Consequently, there is a growing need for HTA bodies and payers to develop guidance for the industry and other stakeholders about the use of RWD/RWE to support access, reimbursement, and pricing. We therefore sought to (i) understand barriers to the use of RWD/RWE by HTA bodies and payers; (ii) review potential solutions in the form of published guidance; and (iii) review findings with selected HTA/payer bodies. Four themes considered key to shaping the generation of robust RWE for HTA bodies and payers were identified as: (i) data (availability, governance, and quality); (ii) methodology (design and analytics); (iii) trust (transparency and reproducibility); and (iv) policy and partnerships. A range of guidance documents were found from trusted sources that could address these themes. These were discussed with HTA experts. This commentary summarizes the potential guidance solutions available to help resolve issues faced by HTA decision-makers in the adoption of RWD/RWE. It shows that there is alignment among stakeholders about the areas that need improvement in the development of RWE and that the key priority to move forward is better collaboration to make data usable for multiple purposes.

Evidence-Informed Deliberative Processes for Legitimate Health Benefit Package Design - Part I: Conceptual Framework.

Countries around the world are increasingly rethinking the design of their health benefit packages to achieve universal health coverage (UHC). Health technology assessment (HTA) bodies support governments in these decisions, but employ value frameworks that do not sufficiently account for the intrinsically complex and value-laden political reality of benefit package design. Several years ago, evidence-informed deliberative processes (EDPs) were developed to address this issue. An EDP is a practical and stepwise approach for 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, and to interpret available evidence on these values. We further developed the conceptual framework and initial 2019 guidance based on academic knowledge exchange, analysing practices of HTA bodies, surveying HTA bodies and experts around the globe, and implementation of EDPs in several countries around the world. EDPs stem from the general concept of legitimacy, which is translated into four elements - stakeholder involvement ideally operationalised through stakeholder participation with deliberation; evidence-informed evaluation; transparency; and appeal. The 2021 practical guidance distinguishes six practical steps of a HTA process and provides recommendations on how these elements can be implemented in each of these steps. There is an increased attention for legitimacy, deliberative processes for HTA and health benefit package design, but the development of theories and methods for such processes remain behind. The added value of EDPs lies in the operationalisation of the general concept of legitimacy into practical guidance for HTA bodies.

Proposal for capturing patient experience through extended value frameworks of health technologies.

BACKGROUND: Inclusion of patient experience (PEx) in health technology assessment (HTA) has become increasingly important; however, no harmonized approach exists to help manufacturers or decision makers ensure PEx considerations are fair, consistent, and thorough within global HTA frameworks. OBJECTIVE: To develop a proposal for including PEx in the HTA frameworks of health technologies. METHODS: A systematic literature review (SLR) on existing value frameworks (VFs) was conducted to capture how PEx-related value judgment is currently considered. Guided by the results of the SLR, a research group including HTA experts and patient representatives used an iterative process to develop potential value domains to capture PEx, in accordance with international guidelines. Subsequently, a panel of international payer experts was used to challenge the proposed PEx domains and provide recommendations for implementation. RESULTS: The SLR found 61 VFs and multi-criteria decision analyses (MCDAs) that considered PEx; however, PEx-related value elements were often referred to superficially, without clear definitions. Five potential PEx domains, with proposed measures for each, were developed and refined using expert feedback: (1) responsiveness to patient's individual needs, (2) improved health literacy and empowerment, (3) patient and caregiver reported outcomes, (4) household's financial burden, and (5) improved access for vulnerable patient populations. A flexible approach for framework implementation was proposed. CONCLUSIONS: Proposed PEx domains could be implemented at multiple levels of healthcare decision making to formalize consideration of PEx in the assessment of value, either through the extension of existing VFs or to create new PEx-focused VFs and more holistic decision making tools. DISCLOSURES: This study was funded and sponsored by UCB Pharma. The funding agreement ensured the authors' independence in designing the study, interpreting the data, writing, and publishing the report. Charokopou, Mountain, and Szegvari are employed by UCB Pharma. Inotai, Jakab, and Kalo are employed by Syreon Research Institute, which received funding from UCB Pharma for this research. Brixner has received fees from AbbVie, Elevar, Millcreek Outcomes Group, Novartis, Sanofi, UCB Pharma, and Xcenda. Campbell has received grants and contracts from the PhRMA Foundation and the Institute for Clinical and Economic Review. During a sabbatical leave, Campbell collaborated with Syreon Research Institute on research projects that included funding from UCB Pharma. Hawkins has received consultancy fees from UCB Pharma. Kristensen has received speakers bureau fees from Pfizer, AbbVie, Amgen, UCB Pharma, Celgene, Bristol-Myers Squibb, MSD, Novartis, Eli Lilly, and Janssen Pharmaceuticals and consultancy fees from UCB Pharma.

Core competencies for ethics experts in health technology assessment.

There is no consensus on who might be qualified to conduct ethical analysis in the field of health technology assessment (HTA). Is there a specific expertise or skill set for doing this work? The aim of this article is to (i) clarify the concept of ethics expertise and, based on this, (ii) describe and specify the characteristics of ethics expertise in HTA. Based on the current literature and experiences in conducting ethical analysis in HTA, a group of members of the Health Technology Assessment International (HTAi) Interest Group on Ethical Issues in HTA critically analyzed the collected information during two face-to-face workshops. On the basis of the analysis, working definitions of "ethics expertise" and "core competencies" of ethics experts in HTA were developed. This paper reports the output of the workshop and subsequent revisions and discussions online among the authors. Expertise in a domain consists of both explicit and tacit knowledge and is acquired by formal training and social learning. There is a ubiquitous ethical expertise shared by most people in society; nevertheless, some people acquire specialist ethical expertise. To become an ethics expert in the field of HTA, one needs to acquire general knowledge about ethical issues as well as specific knowledge of the ethical domain in HTA. The core competencies of ethics experts in HTA consist of three fundamental elements: knowledge, skills, and attitudes. The competencies described here can be used by HTA agencies and others involved in HTA to call attention to and strengthen ethical analysis in HTA.

Developing a roadmap for Health Technology Assessment implementation in public health decisions

Abstract Issue Although the recognized importance of Health Technology Assessment (HTA) in fostering health policy and management, little is known and differences emerge in the state of the art of HTA development and use in public health decisions. Description of the Problem HTA applied to public health interventions could lead to improved population's health, risk factor reduction and high quality services. All these factors represent the basis of health care system sustainability. Nevertheless, there is a lack of evidence about the application of HTA to public health. In this light, the Section on HTA of the European Public Health Association (EUPHA) is conducting a Delphi process in order to define a shared roadmap for the implementation of HTA in public health. Results A literature search was first performed on PubMed to identify useful documentation for drafting the roadmap. Taking clues from Kalò et al., who drafted an HTA roadmap scorecard to support HTA implementation in Central and Eastern Europe, a preliminary core set of actions and pillars to pave the way for the implementation of HTA in public health, was identified. Then, a group of HTA experts was invited to participate to the Delphi process with the aim to build consensus on the final set of actions and pillars to include in the roadmap. Lessons Evidence on the ways to strengthen the role of HTA in public health decisions is scant and the development of a shared roadmap could be useful to identify milestones to do it. Key messages HTA applied to public health could promote an efficient, sustainable, equitable and high-quality health system. Nevertheless, the role of HTA in taking public health decisions should be strengthened. As evidence on the application of HTA in public health is scant, the development of a shared roadmap could enhance fostering its use in taking decisions on public health interventions.