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Purpose: Health technologies are advancing rapidly and becoming more expensive, posing a challenge for financing healthcare systems. Health technology assessment (HTA) improves the efficiency of resource allocation by facilitating evidence-informed decisions on the value of health technologies. Our study aims to create a customized HTA roadmap for Oman based on a gap analysis between the current and future status of HTA implementation. Design/methodology/approach: We surveyed participants of an advanced HTA training program to assess the current state of HTA implementation in Oman and explore long-term goals. A list of draft recommendations was developed in areas with room for improvement. The list was then validated for its feasibility in a round table discussion with senior health policy experts to conclude on specific actions for HTA implementation. Findings: Survey results aligned well with expert discussions. The round table discussion concluded with a phasic action plan for HTA implementation. In the short term (1–2 years), efforts will focus on building capacity through training programs. For medium-term actions (3–5 years), plans include expanding the HTA unit and introducing multiple cost-effectiveness thresholds while from 6–10 years, publishing of HTA recommendations, critical appraisal reports, and timelines is recommended. Originality/value: Although the HTA system in Oman is still in its early stages, strong initiatives are being taken for its advancement. This structured approach ensures a comprehensive integration of HTA into the healthcare system, enhancing decision-making and promoting a sustainable, evidence-based system addressing the population's needs. [ABSTRACT FROM AUTHOR]

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.

Introduction: Access to medicines is a shared goal across healthcare stakeholders. Since health technology assessment (HTA) informs funding decisions, it shapes access to medicines. Despite its wide implementation, significant access variations due to HTA are observed across Europe. This paper elicited the opinions of European stakeholders on how HTA can be improved to facilitate access. Methods: A scoping review identified HTA features that influence access to medicines within markets and areas for improvement, while three access dimensions were identified (availability, affordability, timeliness). Using the Delphi method, we elicited the opinions of European stakeholders to validate the literature findings. Results: Nineteen participants from 14 countries participated in the Delphi panel. Thirteen HTA features that could be improved to optimise access to medicines in Europe were identified. Of these, 11 recorded a positive impact on at least one of the three access dimensions. HTA features had mostly a positive impact on timeliness and a less clear impact on affordability. 'Early scientific advice' and 'clarity in evidentiary requirements' showed a positive impact on all access dimensions. 'Established ways to deal with uncertainty during HTA' could improve medicines' availability and timeliness, while more 'reliance on real-world evidence' could expedite time to market access. Conclusions: Our results reiterate that increased transparency during HTA and the decision-making processes is essential; the use of and reliance on new evidence generation such as real-world evidence can optimise the availability of medicines; and better collaborations between regulatory institutions within and between countries are paramount for better access to medicines. [ABSTRACT FROM AUTHOR]

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.

The new EU Regulation on health technology assessment (HTAR) provides for joint clinical assessments (JCA) of health technologies at EU level. When Member States carry out health technology assessments (HTA) at the national level, they shall give due consideration to the results of a JCA and comply with other obligations of the Regulation. This article aims to clarify what these obligations mean for the Member States and whether JCA results have to be considered outside a national health technology assessment as well. In this context, the question of which processes qualify as 'national HTA' and which requirements need to be fulfilled to trigger the obligations under Article 13 HTAR are discussed in more detail in this paper. [ABSTRACT FROM AUTHOR]

Chronic RhinoSinusitis with Nasal Polyps (CRSwNP) represents a condition mainly caused by the type 2 inflammation presence and marked by the existence of polyps within the nasal and paranasal sinuses. The standard of care includes intranasal steroids, additional burst of systemic steroids, if needed, and surgery. However, recurrence is common, especially among patients with comorbid type 2 inflammatory diseases. Recently, biological drugs, addressing the underlying cause of the disease, have been approved in Italy (dupilumab, omalizumab, and mepolizumab). A Health Technology Assessment was conducted to define multidimensional impact, assuming Italian NHS perspective and a 12-month time horizon. The EUnetHTA Core Model was deployed, using the following methods to analyze the domains: (i) literature evidence; (ii) administration of semi-structured questionnaires to 17 healthcare professionals; (iii) health economics tools to define the economic sustainability for the system. Evidence from NMA and ITC showed a more favorable safety profile and better efficacy for dupilumab compared with alternative biologics. All the analyses, synthesizing cost and efficacy measures, showed that dupilumab is the preferable alternative. Specifically, the cost per responder analysis for dupilumab, exhibiting a 67.0% response rate at Week 52, is notably economical at 14,209EUR per responder. This presents a more economical profile compared with the cost per responder for omalizumab (36.2% response rate) at 24,999EUR and mepolizumab (28.5% response rate) at 31,863EUR. These results underscore dupilumab's potential, not merely in terms of clinical outcomes, but also in terms of economic rationality, thereby solidifying its status as a valid and preferrable alternative in the management of CRSwNP, in the context of the Italian NHS. [ABSTRACT FROM AUTHOR]

Aim: Regulatory and health technology assessment (HTA) agencies have increasingly published frameworks, guidelines, and recommendations for the use of real-world evidence (RWE) in healthcare decision-making. Variations in the scope and content of these documents, with updates running in parallel, may create challenges for their implementation especially during the market authorization and reimbursement phases of a medicine’s life cycle. This environmental scan aimed to comprehensively identify and summarize the guidance documents for RWE developed by most well-established regulatory and reimbursement agencies, as well as other organizations focused on healthcare decision-making, and present their similarities and differences. Methods: RWE guidance documents, including white papers from regulatory and HTA agencies, were reviewed in March 2024. Data on scope and recommendations from each body were extracted by two reviewers and similarities and differences were summarized across four topics: study planning, choosing fit-for-purpose data, study conduct, and reporting. Post-authorization or non-pharmacological guidance was excluded. Results: Forty-six documents were identified across multiple agencies; US FDA produced the most RWE-related guidance. All agencies addressed specific and often similar methodological issues related to study design, data fitness-for-purpose, reliability, and reproducibility, although inconsistency in terminologies on these topics was noted. Two HTA bodies (National Institute for Health and Care Excellence [NICE] and Canada’s Drug Agency) each centralized all related RWE guidance under a unified framework. RWE quality tools and checklists were not consistently named and some differences in preferences were noted. European Medicines Agency, NICE, Haute Autorit ´e de Sant´ e, and the Institute for Quality and Efficiency in Health Care included specific recommendations on the use of analytical approaches to address RWE complexities and increase trust in its findings. Conclusion: Similarities in agencies’ expectations on RWE studies design, quality elements, and reporting will facilitate evidence generation strategy and activities for manufacturers facing multiple, including global, regulatory and reimbursement submissions and re-submissions. A strong preference by decision-making bodies for local real-world data generation may hinder opportunities for data sharing and outputs from international federated data networks. Closer collaboration between decision-making agencies towards a harmonized RWE roadmap, which can be centrally preserved in a living mode, will provide manufacturers and researchers clarity on minimum acceptance requirements and expectations, especially as novel methodologies for RWE generation are rapidly emerging.

* 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.

New therapies in a publicly funded healthcare system are first appraised by health technology assessment agencies that provide funding recommendations to the payers. Treatment with Chimeric Antigen Receptor-T cell (CAR-T) therapy is revolutionizing the management of patients with relapsed/refractory aggressive B-cell lymphoma by providing an effective alternative to the standard of care. Yet, the implementation of CAR-T treatment has a substantial impact on the healthcare system due to its high cost, complex manufacturing process, and requirement for highly specialized services and expertise. CAR-T Cells, as a “living drug”, are fundamentally different from usual medications, and their approvals and funding recommendations pose unique challenges to the health technology agency. In this paper, we explore the specific challenges that face the health technology agencies in reviewing reimbursement recommendations for CAR-T therapy. We take a Canadian perspective and use CAR-T treatment of relapse/refractory aggressive B-cell lymphoma as an example.

For many reasons randomized clinical trials today can»t cope with the tasks set by modern health care, which was clearly demonstrated by the COVID-19 pandemic. This gave new impetus to the development of the RWD / RWE. It became obviously that in cases of impossibility, unethical and other reasons, new methods for obtaining high-quality evidence may be effectively used, and later can be used by decision-makers and organizations engaged in the assessment of health technologies.

Real world data (RWD) and real world evidence (RWE) is one of the most rapidly developing areas in the modern health care systems, which is caused by the development of technologies for both collecting and analyzing data, the emergence of new data sources and the formation of needs both by regulatory bodies, health technology assessment (HTA) agencies, and by the clinical and scientific communities. However, at the moment, there is no universal approach to RWD/E generation and application in the context of HTA. Over the past few years, the rate of including RWD/E in the HTA dossier has noticeably increased, but the nature of the existing guidelines and recommendations is rather fragmentary, which might create obstacles in the way of RWD/E study development and submission. The publication is aimed to describe and summarize the main and most rapidly developing spheres of RWD/E applications and analyze the possibilities of using RWD/E in various HTA areas in the world and in the Russian Federation considering the main trends in the further HTA development.

New therapies in a publicly funded healthcare system are first appraised by health technology assessment agencies that provide funding recommendations to the payers. Treatment with Chimeric Antigen Receptor-T cell (CAR-T) therapy is revolutionizing the management of patients with relapsed/refractory aggressive B-cell lymphoma by providing an effective alternative to the standard of care. Yet, the implementation of CAR-T treatment has a substantial impact on the healthcare system due to its high cost, complex manufacturing process, and requirement for highly specialized services and expertise. CAR-T Cells, as a "living drug", are fundamentally different from usual medications, and their approvals and funding recommendations pose unique challenges to the health technology agency. In this paper, we explore the specific challenges that face the health technology agencies in reviewing reimbursement recommendations for CAR-T therapy. We take a Canadian perspective and use CAR-T treatment of relapse/refractory aggressive B-cell lymphoma as an example. [ABSTRACT FROM AUTHOR]

Abstract Introduction Drug reimbursement decisions are often made based on a price set by the manufacturer. In some cases, this price leads to public and scientific debates about whether its level can be justified in relation to its costs, including those related to research and development (R&D) and manufacturing. Such considerations could enter the decision process in collectively financed health care systems. This paper investigates whether manufacturers’ costs in relation to drug prices, or profit margins, are explicitly mentioned and considered by health technology assessment (HTA) organisations. Method An analysis of reimbursement reports for cancer drugs was performed. All relevant Dutch HTA-reports, published between 2017 and 2019, were selected and matched with HTA-reports from three other jurisdictions (England, Canada, Australia). Information was extracted. Additionally, reimbursement reports for three cases of expensive non-oncolytic orphan drugs prominent in pricing debates in the Netherlands were investigated in depth to examine consideration of profit margins. Results A total of 66 HTA-reports concerning 15 cancer drugs were included. None of these reports contained information on manufacturer’s costs or profit margins. Some reports contained general considerations of the HTA organisation which related prices to manufacturers’ costs: six contained a statement on the lack of price setting transparency, one mentioned recouping R&D costs as a potential argument to justify a high price. For the case studies, 21 HTA-reports were selected. One contained a cost-based price justification provided by the manufacturer. None of the other reports contained information on manufacturer’s costs or profit margins. Six reports contained a discussion about lack of transparency. Reports from two jurisdictions contained invitations to justify high prices by demonstrating high costs. Conclusion Despite the attention given to manufacturers’ costs in relation to price in public debates and in the literature, this issue does not seem to get explicit systematic consideration in the reimbursement reports of expensive drugs.

In India, there is a renewed emphasis on Universal Health Coverage (UHC). Alongside this, Health Technology Assessment (HTA) is an important tool for advancing UHC. The development and application of HTA in India, including capacity building and establishing institutional mechanisms. We emphasized using the HTA approach within two components of the Ayushman Bharat programme, and the section concludes with lessons learned and the next steps. The UHC has increased the importance of selecting and implementing effective technologies and interventions within national health systems, particularly in the context of limited resources. To maximize the use of limited resources and produce reliable scientific assessments, developing and enhancing national capacity must be based on established best practices, information exchange between different sectors, and collaborative approaches. A more potent mechanism and capacity for HTA in India would accelerate the country’s progress toward UHC.

Background: Health technology assessment (HTA) is growing in low- and middle-income countries (LMICs) to ensure optimal use of limited resources. However, the impact of HTAs on decision making in LMICs has been limited. The study aimed to provide an overview of Turkiye’s progress since establishing the first HTA agency in 2012. Methods: The web sites of three national HTA agencies in Turkiye were searched for HTA guidelines and national HTA reports. The HTA guidelines were assessed by two researchers independently against the key principles of HTA developed by Drummond et al., and the HTA reports against the national guidelines. Results: The study included one HTA guideline and eight national HTA reports. The guideline included very limited technical guidance. Compliance with the principles was poor to moderate, and significant methodological limitations were identified. The reports were inconsistent regarding the scope and the HTA assessment criteria. The link between HTA findings, HTA decision making, and health policies were not clear. Discussion: The inconsistencies between the reports and the methodological limitations demonstrate the need for national HTA guidelines. Improving the characteristics of the HTA might impact implementation. Among the key issues is transparency regarding priority setting, the HTA process, and decision making. Conclusion: Establishing and adopting national HTA guidelines at international standards is needed. Involving external scientific committees and health economists in the HTA processes might help ensure that the key principles of HTA are followed. The study findings might be helpful for countries that are developing their HTA systems. [ABSTRACT FROM AUTHOR]

How will market access and value demonstration evolve? In this episode of the Value Insider podcast, host Mike Chambers speaks with Ass. Prof. Panos Kanavos about the future of value assessments in healthcare. Dr Kanavos is Associate Professor of the International Health Policy in the Department of Health Policy at London School of Economics and Political Science as well as Deputy Director at LSE Health and Program Director of the Medical Technology Research Group (MTRG), and advisor to prominent organizations including the European Commission, the European Parliament, the World Bank, the WHO and OECD. Discussing key changes, Dr Kanavos outlines which opportunities and challenges will alter the way we value healthcare interventions. [ABSTRACT FROM AUTHOR]

What other aspects of value may be relevant? In this episode of the Value Insider podcast, host Mike Chambers speaks with Prof. Lou Garrison about the societal perspective on value of healthcare interventions. Prof. Garrison is Professor Emeritus in The Comparative Health Outcomes, Policy, and Economics institute (CHOICE) in the School of Pharmacy at the University of Washington. Lou is past president of the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) and he is currently co-chair of its Policy Outlook Committee. Going beyond the "conventional" elements of value assessment, Prof. Garrison highlights which additional impacts may be at play. He shares his concept of the "Value Flower", explaining elements such as insurance value and scientific spill-over. [ABSTRACT FROM AUTHOR]

How is a cost-effectiveness analysis constructed and used? In this episode of the Value Insider podcast, host Mike Chambers speaks with Prof. Maureen Rutten-van Mölken about economic evaluations in healthcare and how value is considered in these evaluations. Prof. Rutten-van Mölken is professor of Economic Evaluation of Innovations for Health at the Erasmus University Rotterdam in the Netherlands. She leads the Erasmus HTA department and is Scientific Director of the Institute for Medical Technology Assessment (iMTA). Prof Rutten-van Mölken explains how comparative assessments can help us understand which treatments offer the best value-for-money as reimbursement can only be allocated to a selection of all possible treatments. [ABSTRACT FROM AUTHOR]

How does budget impact and affordability in healthcare work? In this episode of the Value Insider podcast, host Mike Chambers speaks with Prof. Sean Sullivan about affordability and budget impact for the "payers" of healthcare interventions. Prof. Sullivan is Dean of the University of Washington School of Pharmacy. He is past president of the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) and served as chair of the health technology assessment (HTA) committee of US Health Insurer Premera Blue Cross, was part of the US Governmental Medicare coverage evidence committee and led the ISPOR Task Force on Methods for Conducting and Reporting Budget Impact Assessments. Prof. Sullivan explains how budget impact and affordability are intertwined and how this plays a role in decisions in the US, but also the rest of the world. [ABSTRACT FROM AUTHOR]

How do we measure quality of life and utility of interventions? In this episode of the Value Insider podcast, host Mike Chambers speaks with Prof. Nancy Devlin about incorporating quality of life in value assessment. Prof. Devlin is professor of health economics at the University of Melbourne. Her past roles include Director of Research at the Office of Health Economics (OHE) London, Professor of Health Economics at City University of London, and she has been director of the International Society of Pharmacoeconomics and Outcomes Research (ISPOR). She is the Chair of the Board of the EuroQol Research Foundation, the international research organization which has developed and maintains the EQ-5D patient-reported outcome (PRO) instrument. Prof Devlin explains the value of the patient voice and how it can be measured and taken into account when considering the value of healthcare interventions. [ABSTRACT FROM AUTHOR]

Abstract Background The Health Technology Assessment (HTA) has encountered different issues and challenges over the last two decades. The main purpose of this research is to review the issues and challenges in high- and middle-income countries through reviewing studies related to the HTA. Methods The HTA area literature of different countries was collected from 2009 to 2020 and analyzed using scoping review, based on Scopus and WoS databases. Results Given the fact that the HTA is practically done in high- and middle-income countries, the results of reviewing the studies and articles of countries reveal that high-income countries seek to increase the participation of stakeholders and enhance the transparency of processes, policy-making, and regulation of the HTA, as well as the systematization of various participant institutions in this area. Middle-income countries, on the other hand, are mostly involved in raising awareness, training and skill development of HTA-related staff, institutionalizing the concept of HTA, and allocating appropriate resources for effective and safe decision-making in their health system. Conclusion The problem of incoordination between stakeholders (participant institutions) in the HTA, and thereby, problems in decision-making were found in many of the studied reports and articles. Thus, one of the useful efforts to be made by different countries to maintain the integrity of this system would be the process of involving all members of this system and the formation of a healthy ecosystem in the HTA.

In this latest update we discuss the transportability of comparative effectiveness evidence across countries. We highlight results of a survey indicating that European HTA agencies are reluctant to accept real-world data from other countries, review recent benefit assessments indicating a potential softening of a requirement for the use of local real-world data in Germany, and outline a recent review presenting approaches that can correct for a lack of transportability.

BackgroundThe Egyptian healthcare system is currently in the early phase of health technology assessment (HTA) implementation. The aim of this study is to propose an implementation roadmap based on the national healthcare system status.MethodsA survey was conducted among Egyptian healthcare sector decision-makers to assess the current and future (preferred) HTA implementation status in Egypt based on a widely used international scorecard methodology. Subsequently, interviews were conducted with experts representing middle- and top-tier management in the Egyptian healthcare system to interpret the survey results and recommend specific actions.ResultsExperts recommended more capacity-building programs for HTA and health economics. Additionally, they proposed establishing HTA units in separate healthcare authorities and merging them into a single central HTA unit in the long term. Regarding the scope of implementation, experts recommended commencing with the assessment of innovative pharmaceuticals, and thereafter, expanding the scope to cover all health technologies in the long term. Additionally, they recommended using innovative tools such as “multi-criteria decision analysis (MCDA)” for tendering, and “managed entry agreements” for reimbursement decisions. Local burden of diseases and costing studies were also recommended to facilitate the implementation of HTA.ConclusionExperts agreed that several actions are required for successful HTA implementation in Egypt, including coordination between HTA bodies, application of an explicit MCDA framework, and strengthening of local evidence generation. To implement these actions, investment in technical capacity-building is indispensable. Most experts favored using multiple and soft cost-effectiveness thresholds. Efforts should be made to publish HTA submission guidelines and timelines of the processes.

The objective of this study was to evaluate the cost-utility of the Oxford, CoronaVac, and Janssen COVID-19 vaccines from the perspective of the Brazilian public health system. A total of 3 microsimulation models were constructed with individual data to evaluate the 3 vaccines. The simulation contains 7 transition states that are related to the natural history of the disease. The model with a daily cycle has a time horizon of 1 year and uses data from 289 days of the pandemic. The analysis was conducted from the perspective of the Brazilian public health system considering direct medical costs. For the model inputs, outpatient and hospital databases were used with information on treated patients stratified by age. Information on mortality was also stratified based on patients' age in the mortality database (SIM). The efficacy of vaccines to reduce the likelihood of patients becoming ill was evaluated independently for each vaccine. Information on the quality of life of patients in outpatient or hospital treatment and the sequelae resulting from the disease were extracted from the literature. The main outcome of the analysis was quality-adjusted life-years (QALYs). The vaccines showed incremental cost-utility ratios ranging from R$−23 161.3/QALY (Oxford) to R$17 757.85/QALY (CoronaVac). The older the population, the lower was the incremental cost-utility ratio. Given a willingness-to-pay threshold of R$17 586/QALY, all the vaccines were considered cost-effective in the probabilistic sensitivity analysis. The results of the analysis by age group can help in the preparation of a vaccination prioritization plan. • This is the first economic evaluation performed with COVID-19 vaccines from the perspective of the Brazilian public health system. • The analysis used real-world data on mortality and outpatient and hospital treatment, which represent the context of the first year of the pandemic in Brazil. • The study allowed for a comparison of the cost-utility ratios of 3 different vaccines used in Brazil, taking into account 3 age groups (<59, 60-75, and >75 years old), and demonstrating the impact of price and number of doses on the efficiency of health spending. [ABSTRACT FROM AUTHOR]

The importance of payers and HTA: How did we end up here? In this episode of the Value Insider podcast, host Mike Chambers speaks with Prof. Mike Drummond about Health Technology Assessment (HTA) and how value is defined. Prof. Drummond is professor of Health Economics of York University, former president of the International Society of Pharmacoeconomics and Outcomes Research (ISPOR), and author of two major textbooks in the field, as well as over 700 publications, and has acted as consultant to the WHO as well as European Union with regards to value assessment. Starting from the very beginning, Prof. Drummond explains in a simple yet engaging way why demonstrating value of new interventions has become so important. [ABSTRACT FROM AUTHOR]

Introduction: Drug reimbursement decisions are often made based on a price set by the manufacturer. In some cases, this price leads to public and scientific debates about whether its level can be justified in relation to its costs, including those related to research and development (R&D) and manufacturing. Such considerations could enter the decision process in collectively financed health care systems. This paper investigates whether manufacturers' costs in relation to drug prices, or profit margins, are explicitly mentioned and considered by health technology assessment (HTA) organisations. Method: An analysis of reimbursement reports for cancer drugs was performed. All relevant Dutch HTA-reports, published between 2017 and 2019, were selected and matched with HTA-reports from three other jurisdictions (England, Canada, Australia). Information was extracted. Additionally, reimbursement reports for three cases of expensive non-oncolytic orphan drugs prominent in pricing debates in the Netherlands were investigated in depth to examine consideration of profit margins. Results: A total of 66 HTA-reports concerning 15 cancer drugs were included. None of these reports contained information on manufacturer's costs or profit margins. Some reports contained general considerations of the HTA organisation which related prices to manufacturers' costs: six contained a statement on the lack of price setting transparency, one mentioned recouping R&D costs as a potential argument to justify a high price. For the case studies, 21 HTA-reports were selected. One contained a cost-based price justification provided by the manufacturer. None of the other reports contained information on manufacturer's costs or profit margins. Six reports contained a discussion about lack of transparency. Reports from two jurisdictions contained invitations to justify high prices by demonstrating high costs. Conclusion: Despite the attention given to manufacturers' costs in relation to price in public debates and in the literature, this issue does not seem to get explicit systematic consideration in the reimbursement reports of expensive drugs. [ABSTRACT FROM AUTHOR]

Objective: Core outcome sets (COS) are an agreed standardised collection of outcomes created with representation from all key stakeholders (such as patients, clinicians, researchers), which should be reported as a minimum for all trials in that corresponding clinical area. There has been little research investigating the use of core outcomes in Health technology assessments (HTAs) and none in non-oncology HTAs. This study aimed to assess the similarity between COS and HTA outcomes. Methods: Ten COS published between 2015 and 2019 were selected, with patient participation taken as a proxy measure for a high quality COS. The INAHTA database was used as a source to identify relevant HTAs, which were accessed through the hyperlinks provided. Outcomes selected for these assessments were categorised as either a specific, partial or no match compared to the COS. An additional cohort of non-oncology HTAs published between 2019 and 2021 were identified from the NICE website and compared against a relevant COS. Results: Six hundred and fifty-one HTAs were matched to the ten COS areas, of which 119 were reviewed. Of a possible 1318 core outcome matches, there were 562 (43%) matches, 413 (31%) specific and 149 (11%) partial. NICE HTA matches against corresponding COS ranged from 44% to 100%, with a total of 78% (73/94) matches, 57 (61%) specific and 16 (17%) partial. Conclusion: Further work is required to promote the awareness and implementation of COS within HTAs. The degree of matching between COS and NICE HTA outcomes is encouraging, demonstrating acceptance of COS by HTA producers.

The focus on health maximisation in a healthcare economic evaluation (HEE) – that is health gains are of equal value regardless of the recipient– has significant implications as health systems attempt to address persistent and growing health inequities. This study aimed to systematically compare and contrast the equity principles of different health technology assessment (HTA) agencies and how equity is addressed in HEE guidelines. HTA agencies were identified through the ISPOR, GEAR, iDSI, HTAi, INAHTA, HTAsiaLink, and RedETSA websites in June 2021 and updated in August 2023. Agencies websites were then searched to retrieve HEE guidelines. The guidelines were grouped into two categories: well-established and newly-developed agency guidelines, based on whether or not they published their first guidelines before 2009. Data extracted summarised the methodological details in the reference cases, including specifics on how equity featured and in what role. In those agencies where equity did not feature explicitly in the HEE guidelines, an additional search of the agency website was undertaken to understand if equity featured in those agencies' decision-making frameworks. The study included 46 guidelines from 51 countries. Only 30% of the guidelines were explicit about the equity assumptions. Health equity (using a broad definition) was mentioned in 29 guidelines and 14 included a specific definition while only seven recommended specific methods to incorporate inequalities. Addressing equity concerns was usually suggested as an additional analyses rather than a key part of the assessment. It was unclear how equity was incorporated into decision-making processes. In addition, equity was mentioned in other guidance – such as decision-making frameworks - provided by five agencies that did not mention it in the HEE guidelines, and 7 of 14 topic selection criteria that were identified. Equity is given less attention than efficiency in HEE guidelines. This indicates that HTA agencies while subscribing to an extra-welfarist approach have a narrow evaluative space – focusing on maximising health and not considering the opportunity cost of the equity constraint. The omission of equity and the lack of systematic approaches in guidelines poses a threat to the international endeavours to reduce inequities. It is timely for HTA agencies to reconsider their positions on equity explicitly. • Equity mentioned in 29 of 46 guidelines of HTA bodies, only 13 give methods to address inequalities. • Equity assumptions underlying economic evaluations is explicit in 14 guidelines. • The guidelines place a greater emphasis on efficiency rather than fairness. • Explicit consideration of equity needed to help deliver equitable health outcomes. [ABSTRACT FROM AUTHOR]

There is an alarming delay in Europe for anticancer medicines becoming accessible for children. Following a paediatric European Union marketing authorisation, national Health Technology Assessment (HTA) agencies evaluate effectiveness, and safety of medicines to support decision on their cost and reimbursement. This study (a SIOPE Access to Medicines project) aimed to evaluate how these HTA evaluations take place for anticancer medicines indicated for paediatric use in Europe and to explore where the delays for market access originate. We obtained HTA reports from the public domain for nine European countries for blinatumomab, dinutuximab beta and tisagenlecleucel. We evaluated the time elapsed between marketing authorisation for a paediatric indication and a national HTA decision and the nature of the decision. Out of 23 HTA decisions (four countries without blinatumomab report), 18 were positive, two with restrictions, three negative. For blinatumomab, tisagenlecleucel and dinutuximab beta, the median time to an HTA decision after regulatory approval for paediatric use was 353 days (range 193–751), 141 days (range 77–517) and 515 days (range 0–780), respectively, with variability between countries. Dinutuximab beta and tisagenlecleucel were first introduced in children, but did not result in shorter time to HTA decision. For blinatumomab, marketing authorisation followed 1008 days after the indication in adults, with HTA applications submitted a median of 167 days later, and a recommendation after 145 days. This study reveals ample variability in HTA decision making in nine European Union countries. Collaboration and alignment of required evidence is needed to facilitate robust scientific HTA assessments, also considering methodological challenges in paediatric oncology. • Health technology assessments of paediatric oncology drugs vary greatly within Europe. • The timing of HTA evaluation adds to the delay of patient access. • Differences between adult and paediatric trial methodology should be considered. • Multistakeholder collaboration between EMA, academia, pharmaceutical companies, patients advocates and HTAs is needed. [ABSTRACT FROM AUTHOR]

Abstract Background Health technology assessment (HTA) agencies have an important role in the evaluation and approval of new technologies. They determine their value within a health system so to promote equitable, quality care with available healthcare resources. Many HTA agencies have some mechanism for involving patients in their processes, but there is great variability and an absence of comprehensive, robust practices for involvement. The accelerating pace of medical innovation creates a need to improve the depth and breadth of patient involvement in the HTA process. Main body In this ‘Call to action’, we present ideas from three HTA expert commentaries calling for collaborative learning and to share innovative ideas for changes in HTA. We also draw on examples of HTA agencies creatively pursuing this goal. We propose a ‘Call to action’ for HTA stakeholders to undertake serious dialogue with patient advocates aimed at creating shared goals. HTA agencies can use these goals to ensure meaningful patient involvement at every step of the HTA process. Five elements are explored. In ‘Recognizing the value of shared purpose’, we highlight examples of HTA agencies that have patients working in partnership with medical practitioners and HTA staff. Results include improved processes that instil confidence. ‘Committing to patient involvement as part of HTA culture’ highlights several initiatives aimed at changes in HTA organisational culture to be more inclusive of patients. In ‘Aligning patient and HTA goals’ we cite work in Belgium and New Zealand which places a greater emphasis on quality of life rather than life expectancy and cost-effectiveness. By ‘Integrating patient involvement at every step of the HTA process’ patients can make vital contributions at every stage of the HTA process. We provide two examples of where HTA agencies have successfully involved patients early in the process in order to broaden the scope of evaluations. ‘Developing a common language and working together’ can support transformative dialogue through ‘unified language’. Conclusion The authors of this commentary ask that agencies and stakeholders involved in HTA take up this call to work together for visionary and transformative elevation of the voice of patients in HTA worldwide.

A health technology assessment (HTA) is commonly defined as a multidisciplinary approach used to evaluate medical, social, economic, and ethical issues related to the use of a health technology in a systematic, transparent, unbiased, robust manner. To help inform HTA recommendations, the surveillance of social media platforms can provide important insights to the clinical community and to decision makers on the effectiveness and safety of the use of health technologies on a patient. A scoping review of the published literature was performed to gain some insight on the accuracy and automation of sentiment analysis (SA) used to assess public opinion on the use of health technologies. A literature search of major databases was conducted. The main search concepts were SA, social media, and patient perspective. Among the 1,776 unique citations identified, 12 studies that described the use of SA methods to evaluate public opinion on or experiences with the use of health technologies as posted on social media platforms were included. The SA methods used were either lexicon- or machine learning-based. Two studies focused on medical devices, three examined HPV vaccination, and the remaining studies targeted drug therapies. Due to the limitations and inherent differences among SA tools, the outcomes of these applications should be considered exploratory. The results of our study can initiate discussions on how the automation of algorithms to interpret public opinion of health technologies should be further developed to optimize the use of data available on social media.

Objective: Core outcome sets (COS) are an agreed standardised collection of outcomes created with representation from all key stakeholders (such as patients, clinicians, researchers), which should be reported as a minimum for all trials in that corresponding clinical area. There has been little research investigating the use of core outcomes in Health technology assessments (HTAs) and none in non-oncology HTAs. This study aimed to assess the similarity between COS and HTA outcomes. Methods: Ten COS published between 2015 and 2019 were selected, with patient participation taken as a proxy measure for a high quality COS. The INAHTA database was used as a source to identify relevant HTAs, which were accessed through the hyperlinks provided. Outcomes selected for these assessments were categorised as either a specific, partial or no match compared to the COS. An additional cohort of non-oncology HTAs published between 2019 and 2021 were identified from the NICE website and compared against a relevant COS. Results: Six hundred and fifty-one HTAs were matched to the ten COS areas, of which 119 were reviewed. Of a possible 1318 core outcome matches, there were 562 (43%) matches, 413 (31%) specific and 149 (11%) partial. NICE HTA matches against corresponding COS ranged from 44% to 100%, with a total of 78% (73/94) matches, 57 (61%) specific and 16 (17%) partial. Conclusion: Further work is required to promote the awareness and implementation of COS within HTAs. The degree of matching between COS and NICE HTA outcomes is encouraging, demonstrating acceptance of COS by HTA producers.

Abstract Background Decision-making in public health and health policy is complex and requires careful deliberation of many and sometimes conflicting normative and technical criteria. Several approaches and tools, such as multi-criteria decision analysis, health technology assessments and evidence-to-decision (EtD) frameworks, have been proposed to guide decision-makers in selecting the criteria most relevant and appropriate for a transparent decision-making process. This study forms part of the development of the WHO-INTEGRATE EtD framework, a framework rooted in global health norms and values as reflected in key documents of the World Health Organization and the United Nations system. The objective of this study was to provide a comprehensive overview of criteria used in or proposed for real-world decision-making processes, including guideline development, health technology assessment, resource allocation and others. Methods We conducted an overview of systematic reviews through a combination of systematic literature searches and extensive reference searches. Systematic reviews reporting criteria used for real-world health decision-making by governmental or non-governmental organization on a supranational, national, or programme level were included and their quality assessed through a bespoke critical appraisal tool. The criteria reported in the reviews were extracted, de-duplicated and sorted into first-level (i.e. criteria), second-level (i.e. sub-criteria) and third-level (i.e. decision aspects) categories. First-level categories were developed a priori using a normative approach; second- and third-level categories were developed inductively. Results We included 36 systematic reviews providing criteria, of which one met all and another eleven met at least five of the items of our critical appraisal tool. The criteria were subsumed into 8 criteria, 45 sub-criteria and 200 decision aspects. The first-level of the category system comprised the following seven substantive criteria: “Health-related balance of benefits and harms”; “Human and individual rights”; “Acceptability considerations”; “Societal considerations”; “Considerations of equity, equality and fairness”; “Cost and financial considerations”; and “Feasibility and health system considerations”. In addition, we identified an eight criterion “Evidence”. Conclusion This overview of systematic reviews provides a comprehensive overview of criteria used or suggested for real-world health decision-making. It also discusses key challenges in the selection of the most appropriate criteria and in seeking to implement a fair decision-making process.

BackgroundHealth Technology Assessment (HTA) is a multidisciplinary process that synthesizes, with a systematic, transparent, impartial and robust methodological approach, the main information on the medical, economic, ethical and social implications of the use and dissemination of a health technology. Its aim is to support decision-makers in identifying safe, effective, patient-centered and best-value health policies, in order to promote an equitable, efficient, and high-quality health system. Given the continued application of innovative technologies into clinical practice, healthcare professionals need to be able to adequately evaluate these technologies using evidence-based approaches such as HTA. Therefore, the implementation of training in HTA is crucial. The aim of this study was to investigate existing HTA training initiatives for healthcare professionals provided by international HTA agencies and organizations around the world.MethodsFrom March to November 2020, the websites of HTA agencies and organizations belonging to the European network for HTA (EUnetHTA) and to the International Network of Agencies for HTA (INAHTA), and the website of the HTA International (HTAi), were explored for identifying the HTA training initiatives directed to healthcare professionals. In addition, we screened the training initiatives proposed at European level by EUnetHTA as part of its Joint Actions and conducted in collaboration with its public-private partners. Specific keywords were searched in English and adapted to French, Portuguese, Spanish, Italian and German. Data extraction of the retrieved training initiatives was conducted from November 2020 to February 2021 and considered the following information: agency, country, website, coordinator, type of initiative, target, topic, main contents, and language.ResultsOut of 124 agencies/organizations/EUnetHTA public-private partners screened, only 21 provided training initiatives for healthcare professionals. A total of 55 training initiatives were analyzed, 85.5% of which were delivered at the European level and 14.5% at the international level. The countries with a greater number of courses were: Austria, Argentina, Spain, Portugal, and the United Kingdom. Twenty-one training initiatives focused on HTA application and methodology while 34 on specific HTA domains, particularly on the economic one. The technologies covered were mainly drugs.ConclusionsOur study revealed a limited number of HTA training programs targeting healthcare professionals. HTA supports the decision-making processes concerning the use and application of health technologies with scientific evidence. Indeed, training of healthcare professionals in this field should be a key driver in implementing evidence-based healthcare choices and through rigorous methodological approaches such as HTA, in order to ensure proper health governance and value-based application of technological innovations in clinical practice. Therefore, capacity building of healthcare professionals in this area should be enhanced by using appropriate and effective training initiatives and educational strategies.

Objective: Core outcome sets (COS) are an agreed standardised collection of outcomes created with representation from all key stakeholders (such as patients, clinicians, researchers), which should be reported as a minimum for all trials in that corresponding clinical area. There has been little research investigating the use of core outcomes in Health technology assessments (HTAs) and none in non-oncology HTAs. This study aimed to assess the similarity between COS and HTA outcomes. Methods: Ten COS published between 2015 and 2019 were selected, with patient participation taken as a proxy measure for a high quality COS. The INAHTA database was used as a source to identify relevant HTAs, which were accessed through the hyperlinks provided. Outcomes selected for these assessments were categorised as either a specific, partial or no match compared to the COS. An additional cohort of non-oncology HTAs published between 2019 and 2021 were identified from the NICE website and compared against a relevant COS. Results: Six hundred and fifty-one HTAs were matched to the ten COS areas, of which 119 were reviewed. Of a possible 1318 core outcome matches, there were 562 (43%) matches, 413 (31%) specific and 149 (11%) partial. NICE HTA matches against corresponding COS ranged from 44% to 100%, with a total of 78% (73/94) matches, 57 (61%) specific and 16 (17%) partial. Conclusion: Further work is required to promote the awareness and implementation of COS within HTAs. Improved uptake across NICE HTAs is encouraging, demonstrating acceptance of COS by HTA producers.

Background: The Health Technology Assessment (HTA) has encountered different issues and challenges over the last two decades. The main purpose of this research is to review the issues and challenges in high- and middle-income countries through reviewing studies related to the HTA.Methods: The HTA area literature of different countries was collected from 2009 to 2020 and analyzed using scoping review, based on Scopus and WoS databases.Results: Given the fact that the HTA is practically done in high- and middle-income countries, the results of reviewing the studies and articles of countries reveal that high-income countries seek to increase the participation of stakeholders and enhance the transparency of processes, policy-making, and regulation of the HTA, as well as the systematization of various participant institutions in this area. Middle-income countries, on the other hand, are mostly involved in raising awareness, training and skill development of HTA-related staff, institutionalizing the concept of HTA, and allocating appropriate resources for effective and safe decision-making in their health system.Conclusion: The problem of incoordination between stakeholders (participant institutions) in the HTA, and thereby, problems in decision-making were found in many of the studied reports and articles. Thus, one of the useful efforts to be made by different countries to maintain the integrity of this system would be the process of involving all members of this system and the formation of a healthy ecosystem in the HTA. [ABSTRACT FROM AUTHOR]

Objective This analysis aimed to assess patient advocacy groups' (PAGs) perspectives on patients' and PAGs' potential role in Health Technology Assessment (HTA) decision-making in Greece. Methods In total, 22 representatives of PAGs participated in a consensus panel meeting. Participants' views were elicited via a consensus panel meeting, through a televoting procedure, based on a structured questionnaire with close-ended questions. Voting was anonymous, to avoid the influence of the dominant personality. After voting, the results were presented to the participants and televoting was repeated to achieve consensus. Key findings The majority of participants (94.8%) believe that valid information on the developments regarding HTA in Greece is not conveyed to them by the official national regulatory sources. Key sources of valid information on developments regarding HTA were: conferences (95.2%), international PAGs (85.7%) and HTA experts/scientists (76.2%). About 76.2% of participants evaluated PAGs' competence to participate in policy-making concerning HTA scheme formation as moderate or higher. Regarding patients' effective participation in HTA decision-making, greater importance (points 4 and 5 on a 5-point scale) was given to their education on treatment effects in terms of quality of life (100.0%), basic principles of health economics and pharmaceutical policy (95.5%) and ethical aspects of HTA (95.0%). About 55.0% strongly agree that patients should formally express their opinion on HTA issues, while 72.7% believe that patients' opinions on HTA issues should be expressed through participation in the process and right to vote. Conclusions The participation of patients in HTA decision-making is an issue not only of justice but also of essence. For a productive participation, PAGs training is essential. [ABSTRACT FROM AUTHOR]

Aim: analyze the structure and funding of health technology assessment (HTA) agencies abroad.Materials and methods. Here, we review the organizational structure and funding of HTA agencies in Europe (Austria, Belgium, Germany, Ireland, the Netherlands, the United Kingdom, France, and Sweden), Canada and Australia. The relevant information was found on web-sites of HTA agencies, in the Medline database, and via the searching engines Yandex and Google; the search was conducted using the specific descriptors: «organizational structure of HTA agency», «funding of HTA agency», «pharmaceutical», «reimbursement», «healthcare decision-making», and «funding».Results. The identified HTA-agencies may have a status of either government-funded or nonprofit organization or a structural element of a governmental body. These hTa agencies are funded mainly from the national budget. The funding varies from €550 000 for Ireland to £63.1 mln (€70 million) for the National Institute for Clinical Excellence (NICE) in the UK. The number of employees in the reviewed HTA agencies varies from 6.8 full time employees (FTE) in the Health Information and Quality Authority (HIQA) in Ireland to 604 FTEs in the NICE.

Abstract Standard outcome sets developed by the International Consortium for Health Outcomes Measurement (ICHOM) facilitate value‐based health care in healthcare practice and have gained traction from regulators and Health Technology Assessment (HTA) agencies that regularly assess the value of new medicines. We aimed to assess the extent to which the outcomes used by regulators and HTA agencies are patient‐relevant, by comparing these to ICHOM standard sets. We conducted a cross‐sectional comparative analysis of ICHOM standard sets, and publicly available regulatory and HTA assessment guidelines. We focused on oncology due to many new medicines being developed, which are accompanied by substantial uncertainty regarding the relevance of these treatments for patients. A comparison of regulatory and HTA assessment guidelines, and ICHOM standard sets showed that both ICHOM and regulators stress the importance of disease‐specific outcomes. On the other hand, HTA agencies have a stronger focus on generic outcomes in order to allow comparisons across disease areas. Overall, similar outcomes are relevant for market access, reimbursement, and in ICHOM standard sets. However, some differences are apparent, such as the acceptability of intermediate outcomes. These are recommended in ICHOM standard sets, but regulators are more likely to accept intermediate outcomes than HTA agencies. A greater level of alignment in outcomes accepted may enhance the efficiency of regulatory and HTA processes, and increase timely access to new medicines. ICHOM standard sets may help align these outcomes. However, some differences in outcomes used may remain due to the different purposes of regulatory and HTA decision‐making.

Standard outcome sets developed by the International Consortium for Health Outcomes Measurement (ICHOM) facilitate value-based health care in healthcare practice and have gained traction from regulators and Health Technology Assessment (HTA) agencies that regularly assess the value of new medicines. We aimed to assess the extent to which the outcomes used by regulators and HTA agencies are patientrelevant, by comparing these to ICHOM standard sets. We conducted a cross-sectional comparative analysis of ICHOM standard sets, and publicly available regulatory and HTA assessment guidelines. We focused on oncology due to many new medicines being developed, which are accompanied by substantial uncertainty regarding the relevance of these treatments for patients. A comparison of regulatory and HTA assessment guidelines, and ICHOM standard sets showed that both ICHOM and regulators stress the importance of disease-specific outcomes. On the other hand, HTA agencies have a stronger focus on generic outcomes in order to allow comparisons across disease areas. Overall, similar outcomes are relevant for market access, reimbursement, and in ICHOM standard sets. However, some differences are apparent, such as the acceptability of intermediate outcomes. These are recommended in ICHOM standard sets, but regulators are more likely to accept intermediate outcomes than HTA agencies. A greater level of alignment in outcomes accepted may enhance the efficiency of regulatory and HTA processes, and increase timely access to new medicines. ICHOM standard sets may help align these outcomes. However, some differences in outcomes used may remain due to the different purposes of regulatory and HTA decision-making. [ABSTRACT FROM AUTHOR]

Background: Health technology assessment (HTA) agencies have an important role in the evaluation and approval of new technologies. They determine their value within a health system so to promote equitable, quality care with available healthcare resources. Many HTA agencies have some mechanism for involving patients in their processes, but there is great variability and an absence of comprehensive, robust practices for involvement. The accelerating pace of medical innovation creates a need to improve the depth and breadth of patient involvement in the HTA process. Main body: In this 'Call to action', we present ideas from three HTA expert commentaries calling for collaborative learning and to share innovative ideas for changes in HTA. We also draw on examples of HTA agencies creatively pursuing this goal. We propose a 'Call to action' for HTA stakeholders to undertake serious dialogue with patient advocates aimed at creating shared goals. HTA agencies can use these goals to ensure meaningful patient involvement at every step of the HTA process. Five elements are explored. In 'Recognizing the value of shared purpose', we highlight examples of HTA agencies that have patients working in partnership with medical practitioners and HTA staff. Results include improved processes that instil confidence. 'Committing to patient involvement as part of HTA culture' highlights several initiatives aimed at changes in HTA organisational culture to be more inclusive of patients. In 'Aligning patient and HTA goals' we cite work in Belgium and New Zealand which places a greater emphasis on quality of life rather than life expectancy and cost-effectiveness. By 'Integrating patient involvement at every step of the HTA process' patients can make vital contributions at every stage of the HTA process. We provide two examples of where HTA agencies have successfully involved patients early in the process in order to broaden the scope of evaluations. 'Developing a common language and working together' can support transformative dialogue through 'unified language'. Conclusion: The authors of this commentary ask that agencies and stakeholders involved in HTA take up this call to work together for visionary and transformative elevation of the voice of patients in HTA worldwide. [ABSTRACT FROM AUTHOR]

Patient involvement in the process of producing health technology assessments has become increasingly important. In the European Network for Health Technology Assessment (EUnetHTA), several approaches to patient involvement were explored. The outcome was a document on "Patient Input in Relative Effectiveness Assessments" that is available for access and was published in 2019. The aim of this article is to analyze the experience gained by EUnetHTA in patient involvement for EUnetHTA assessment production, describe and quantify the approaches used, and outline the challenges and avenues for the improvement of current processes. Patients were involved in twenty-three of thirty-six pharmaceutical and other technology EUnetHTA assessments from June 2016 until the end of November 2019. Approaches to patient involvement included using a patient input template, one-on-one conversations, group conversations, scoping meeting with patients, and other approaches. Although it is recognized that patient involvement is important to understand the needs of the target patient population, challenges remain with timely patient involvement. Additionally, further efforts are needed to guide assessment teams on how to implement and enhance the visibility of patient input in assessments. [ABSTRACT FROM AUTHOR]

Abstract Background Health technology assessment (HTA) is currently one of the major challenges in assessing medical innovations and healthcare systems. In Europe, the European Network for Health Technology Assessment (EUnetHTA) has been aspiring to develop and implement standards for international sharing of HTA results and studies. Slovakia and many other EU countries do not have an established HTA system yet. This paper is focused on an exact description of the EUnetHTA Core Model individual domains applied to the process of selecting patients in the terminal stage of prostate cancer for Radium-223 treatment under particular conditions of the Institute of Nuclear and Molecular Medicine (INMM) in Košice, Slovakia. Results We produced the first pilot HTA report using the HTA Core Model in Slovakia. The main objective was to collect all relevant information on the particular technology, and provide its summary to the interested stakeholders on one spot. Rather than applying detailed individual items, i.e. assessment elements and assessment element cards, we concentrated on the content of individual domains and tried to fill them with the best country, facility and intervention related data. The dataset consisted of 52 patients that finished the treatment in the period 2015–2017. The patients were carefully selected according to the Radium-223 producer’s criteria. Only 33 patients received the full therapy consisting of six applications; their average survival was 10.5 months from the application of the last dose. Conclusions Based on the results of our analyzes, we recommended several changes to the INMM processes and patient follow-up checks during the treatment process in order to make the therapy more effective. The greatest benefit is expected after the implementation of a 68Ge/68Ga generator in 2018, as the selection of patients suitable for the Radium-223 treatment will improve. We showed that the HTA Core Model can be implemented in Slovakia, even under conditions of no formal HTA support or institutionalization.

Abstract Background When designing a randomised controlled trial (RCT), an important consideration is the sample size required. This is calculated from several components; one of which is the target difference. This study aims to review the currently reported methods of elicitation of the target difference as well as to quantify the target differences used in Health Technology Assessment (HTA)-funded trials. Methods Trials were identified from the National Institute of Health Research Health Technology Assessment journal. A total of 177 RCTs published between 2006 and 2016 were assessed for eligibility. Eligibility was established by the design of the trial and the quality of data available. The trial designs were parallel-group, superiority RCTs with a continuous primary endpoint. Data were extracted and the standardised anticipated and observed effect size estimates were calculated. Exclusion criteria was based on trials not providing enough detail in the sample size calculation and results, and trials not being of parallel-group, superiority design. Results A total of 107 RCTs were included in the study from 102 reports. The most commonly reported method for effect size derivation was a review of evidence and use of previous research (52.3%). This was common across all clinical areas. The median standardised target effect size was 0.30 (interquartile range: 0.20–0.38), with the median standardised observed effect size 0.11 (IQR 0.05–0.29). The maximum anticipated and observed effect sizes were 0.76 and 1.18, respectively. Only two trials had anticipated target values above 0.60. Conclusion The most commonly reported method of elicitation of the target effect size is previous published research. The average target effect size was 0.3. A clear distinction between the target difference and the minimum clinically important difference is recommended when designing a trial. Transparent explanation of target difference elicitation is advised, with multiple methods including a review of evidence and opinion-seeking advised as the more optimal methods for effect size quantification.

Abstract Background A recent study by Page et al. (PLoS Med. 2016;13(5):e1002028) claimed that increasing numbers of reviews are being published and many are poorly-conducted and reported. The aim of the present study was to assess how well reporting standards of systematic reviews produced in a Health Technology Assessment (HTA) context compare with reporting in Cochrane and other ‘non-Cochrane’ systematic reviews from the same years (2004 and 2014), as reported by Page et al. (PLoS Med. 2016;13(5):e1002028). Methods All relevant UK HTA programme systematic reviews published in 2004 and 2014 were identified. After piloting of the form, two reviewers each extracted relevant data on conduct and reporting from these reviews. These data were compared with data for Cochrane and “non-Cochrane” systematic reviews, as published by Page et al. (PLoS Med. 2016;13(5):e1002028). All data were tabulated and summarized. Results There were 30 UK HTA programme systematic reviews and 300 other systematic reviews, including Cochrane reviews (n = 45). The percentage of HTA reviews with required elements of conduct and reporting was frequently very similar to Cochrane and much higher than all other systematic reviews, e.g. availability of protocols (90, 98 and 16% respectively); the specification of study design criteria (100, 100, 79%); the reporting of outcomes (100, 100, 78%), quality assessment (100, 100, 70%); the searching of trial registries for unpublished data (70, 62, 19%); reporting of reasons for excluding studies (91, 91 and 70%) and reporting of authors’ conflicts of interests (100, 100, 87%). HTA reviews only compared less favourably with Cochrane and other reviews in assessments of publication bias. Conclusions UK HTA systematic reviews are often produced within a specific policy-making context. This context has implications for timelines, tools and resources. However, UK HTA systematic reviews still tend to present standards of conduct and reporting equivalent to “gold standard” Cochrane reviews and superior to systematic reviews more generally.

As U.S. policymakers consider strategies to control pharmaceutical spending, they can learn from France, which has stopped drug spending growth without slowing access to innovative medicines. France determines the comparative therapeutic value of new drugs. Insurance pays more for drugs superior to their comparator and the same or less for drugs offering modest or no improvement. Contracts require discounts for high sales volume and prohibit price increases. In addition, payers reduce prices of older drugs. Furthermore, Parliament sets an insurance pharmaceutical spending budget, and manufacturers pay clawbacks when spending exceeds the budget. France offers these lessons: setting prices based on added therapeutic value is a principled means to cap new drug prices and provides incentives for manufacturers to negotiate prices. Restricting formularies can help lower prices. Insurers can link prices and quantity to control spending and improper uses. Insurers can use global budgets to control spending and negotiate prices. Contracts can prevent manufacturers from raising prices after launch. External reference pricing can reduce price discrimination but is difficult to implement. Nations can ensure rapid access to new drugs while controlling prices. Regulation and competition are complementary strategies to control drug spending. [ABSTRACT FROM AUTHOR]