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New health technologies are constantly developing. However, their impacts on health and implications for health systems are not always clear. Faced with this situation, in the last 45 years, Health Technology Assessment (HTA) has taken an important role in the decision-making process related to the implementation of technologies in healthcare systems. According to the Core Model® EUnetHTA, a "full HTA" should cover nine domains: health problems and current use of technology, description and technical characteristics, safety, clinical effectiveness, costs and economic evaluation, ethical analysis, organizational aspects, patients and social aspects, legal aspects. In all domains of a HTA, the approach is evidence-based and uses epidemiological data, systematic reviews to gather the best level of proofs regarding clinical efficacy and safety of interventions and comparators and organizational and economic models. One exception is the ethical analysis, that uses value-based approach. Although in Neonatal Intensive Care Units (NICUs) there are highly advanced technological environments, HTA has not yet been widely used in this field for determining the "value" of the diagnostic and therapeutic procedures. An example of diagnostic tool used in NICUs is the near-infrared spectroscopy (NIRS), a noninvasive device that enables real-time monitoring of the condition of peripheral tissues in critically ill newborns. The availability of this diagnostic tool could improve the choice of the most appropriate treatment to the clinical situation of the newborn. The expected benefit of NIRS motivates the need of a full HTA. Conclusion: HTA is still little used in Neonatal Critical Care, but it may be the appropriate tool to determine the "value" of technologies used in this field. The implementation of clinical trials and HTA may help in an evidence-based evaluation of new technologies for the neonatal critical care. This could facilitate the rapid introduction of the best health technologies into clinical practice. What is Known: • Health Technology Assessment (HTA) has taken an important role in the decision-making process related to the implementation of technologies in healthcare systems • The centrality of ethics in HTA has been known. In fact, ethics is everywhere in HTA, and value judgments permeate all levels of HTA What is New: • HTA is still little used in Neonatal Critical Care, but it may be the appropriate tool to determine the "value" of technologies used in this field • The implementation of clinical trials and HTA may help in an evidence-based evaluation of new technologies for the neonatal critical care and in introduction of the best and ethically acceptable health technologies into clinical practice. [ABSTRACT FROM AUTHOR]

The Health Technology Assessment (HTA) Working Group of the Emerging Technology Division of International Federation of Clinical Chemistry and Laboratory Medicine (IFCC) aims to develop a methodological approach for producing structured HTA information for laboratory medicine technologies. This approach seeks to support decision-making processes at the country, regional, and/or hospital levels regarding the introduction of specific technologies. The focus of this model will primarily be on defining assessment elements within the domains of 'organizational aspects' and 'costs and economic evaluations', potentially differentiated by the type of diagnostic technology (e.g., genetic tests, molecular tests). To achieve this project's goal, a literature review and examination of websites of international HTA agencies have been conducted. The research aims to identify multidisciplinary methodological approaches used to assess laboratory diagnostic technologies and to pinpoint the domains and assessment elements utilized. We found 7 methodological articles describing methodological approaches adopted to assess laboratory diagnostic technologies. Among the HTA organizations considered, 23 reports were found, of which 7 were produced by the European Network of HTA (EUnetHTA), 4 by the National Institute for Health and Care Excellence Diagnostic Assessment Program (NICE DAP), and 12 by other HTA agencies. The EUnetHTA reports were rapid collaborative assessments covering various domains, while the NICE DAP reports focused on diagnostic guidances, including descriptions of technologies, clinical need and practice, diagnostic tests, accuracy, effectiveness, and cost-effectiveness. Finally, a survey targeting laboratory professionals will be conducted to introduce assessment elements, differentiated by the type of diagnostic technology, primarily for organizational and economic domains. [ABSTRACT FROM AUTHOR]

With the increasing pipeline of cell and gene therapies (CGTs) and the expected surge in the number of approvals, understanding the market access landscape becomes crucial for timely patient access. This study evaluates the challenges Dutch stakeholders encounter in CGT market access, offering insights for improving time-to-patient access. A traditional literature review was conducted to identify market access challenges and solutions for CGTs. Based on the findings, participants for semi-structured interviews, designed using an interview guide adapted to the Dutch context, were selected to capture diverse perspectives on market access. This review included 124 relevant articles out of 2449, covering several aspects of market access of CGTs. Subsequently, interviews with 16 stakeholders from academia, patient advocacy groups, manufacturers, health insurers, payers, hospital pharmacists, healthcare practitioners, and the Association of Innovative Medicines were conducted. Stakeholders identified challenges and proposed solutions for reimbursement package management, clinical trials, health economics, payment models, and procedural and organisational aspects. Thematic analysis revealed unique country-specific challenges and solutions in the Netherlands. This research provides insights into these challenges and potential solutions, emphasising the need for collaborative efforts among stakeholders to develop practical and multidisciplinary measures to improve the market access landscape for CGTs in the country. [ABSTRACT FROM AUTHOR]

This document provides a comprehensive list of references and citations for articles and studies related to the use of trastuzumab emtansine and trastuzumab deruxtecan in the treatment of HER2-positive/mutated malignancies. The articles cover various topics, including the mechanism of action of trastuzumab, the safety profile of trastuzumab-based antibody-drug conjugates, and the evolving therapeutic landscape of trastuzumab-drug conjugates. [Extracted from the article]

With the increasing pipeline of cell and gene therapies (CGTs) and the expected surge in the number of approvals, understanding the market access landscape becomes crucial for timely patient access. This study evaluates the challenges Dutch stakeholders encounter in CGT market access, offering insights for improving time-to-patient access. A traditional literature review was conducted to identify market access challenges and solutions for CGTs. Based on the findings, participants for semi-structured interviews, designed using an interview guide adapted to the Dutch context, were selected to capture diverse perspectives on market access. This review included 124 relevant articles out of 2449, covering several aspects of market access of CGTs. Subsequently, interviews with 16 stakeholders from academia, patient advocacy groups, manufacturers, health insurers, payers, hospital pharmacists, healthcare practitioners, and the Association of Innovative Medicines were conducted. Stakeholders identified challenges and proposed solutions for reimbursement package management, clinical trials, health economics, payment models, and procedural and organisational aspects. Thematic analysis revealed unique country-specific challenges and solutions in the Netherlands. This research provides insights into these challenges and potential solutions, emphasising the need for collaborative efforts among stakeholders to develop practical and multidisciplinary measures to improve the market access landscape for CGTs in the country.

Fundamentally precision oncology illustrates the path in which molecular profiling of tumors can illuminate their biological behavior, diversity, and likely outcomes by identifying distinct genetic mutations, protein levels, and other biomarkers that underpin cancer progression. Next-generation sequencing became an indispensable diagnostic tool for diagnosis and treatment guidance in current clinical practice. Nowadays, tissue analysis benefits from further support through methods like comprehensive genomic profiling and liquid biopsies. However, precision medicine in the field of oncology presents specific hurdles, such as the cost-benefit balance and widespread accessibility, particularly in countries with low- and middle-income. A key issue is how to effectively extend next-generation sequencing to all cancer patients, thus empowering treatment decision-making. Concerns also extend to the quality and preservation of tissue samples, as well as the evaluation of health technologies. Moreover, as technology advances, novel next-generation sequencing assessments are being developed, including the study of Fragmentomics. Therefore, our objective was to delineate the primary uses of next-generation sequencing, discussing its' applications, limitations, and prospective paths forward in Oncology. [ABSTRACT FROM AUTHOR]

Technology has greatly influenced and radically changed human life, from communication to creativity and from productivity to entertainment. The authors, starting from considerations concerning the implementation of new technologies with a strong impact on people's everyday lives, take up Collingridge's dilemma and relate it to the application of AI in healthcare. Collingridge's dilemma is an ethical and epistemological problem concerning the relationship between technology and society which involves two approaches. The proactive approach and socio-technological experimentation taken into account in the dilemma are discussed, the former taking health technology assessment (HTA) processes as a reference and the latter the AI studies conducted so far. As a possible prevention of the critical issues raised, the use of the medico-legal method is proposed, which classically lies between the prevention of possible adverse events and the reconstruction of how these occurred. The authors believe that this methodology, adopted as a European guideline in the medico-legal field for the assessment of medical liability, can be adapted to AI applied to the healthcare scenario and used for the assessment of liability issues. The topic deserves further investigation and will certainly be taken into consideration as a possible key to future scenarios. [ABSTRACT FROM AUTHOR]

Objective: To clarify the cost-effectiveness of comprehensive diagnosis and treatment of metastatic non-small cell lung cancer in Japan, from initial diagnosis to post-standard treatment, using three different strategies. Methods: A decision tree was created using three diagnostic and treatment strategies, assuming that Foundation One CDx (F1CDx), a comprehensive genome panel, was introduced in Japan in June 2019. This comprehensive decision tree includes Markov models, cost-effectiveness analyses (CEA), and cost-utility analyses (CUA) of the three strategies from the perspective of Japanese payers. Specifically, Strategy1 involves single-gene testing at the initial diagnosis and F1CDx after standard treatment; Strategy2 involves only single-gene testing at the initial diagnosis; Strategy3 involves F1CDx at the initial diagnosis. The incremental cost-effectiveness ratios (ICERs) of the three strategies are estimated. Sensitivity analyses were performed to assess the uncertainty of the parameter settings. Results: Strategy3 was dominated for both CUA and CEA. The ICER/quality-adjusted life year (QALY) for Strategy2 versus Strategy1 was USD 13,734 (JPY 2,080,923, USD 1 = JPY 151.39 on April 1st, 2024), which is less than the willingness to pay of USD 45,900 (JPY 7,500,000), and Strategy2 was more cost-effective than Strategy1. F1CDx was not cost-effective compared to multiple simultaneous single tests at the initial diagnosis, either after standard treatment or at the initial diagnosis. Sensitivity analysis also showed that the most influential factor on the ICER for both CEA and CUA was treatment cost. Conclusions: From both patient benefit and health economic perspectives, introducing F1CDx after standard treatment in June 2019 was not as cost-effective as multiple simultaneous single tests at the initial diagnosis but was more realistic from a health economic perspective than introducing F1CDx at the time of initial diagnosis. Therefore, the policy at the time of F1CDx introduction in Japan was appropriate from a short-term health-economic perspective.

The research indicated the relationship between the formal criteria of Health Technology Assessment (HTA) availability and the economic development of countries considering HTA to be an indicator for the effectiveness of the healthcare management process. HTA is at the very end of the evidence data generation. It makes possible forecasting medical, economic and social outcomes of healthcare management decisions and is the base for rational use of healthcare resources. Effective allocation of healthcare funds leads to an increase in human capital and economic development opportunities followed by the overall healthcare expenditures growth. The number of countries using HTA increases with the growth of per capita GDP, reaching a maximum in the countries with 40-50 thousand USD GDP per capita value range and decreases with further growth of this indicator. The low level of economic development, characterized by a low per capita GDP, makes it difficult to implement HTA due to strict regulations aimed at expenditures decrease and preventing using effectiveness criteria for health management assessment. Nevertheless, a significant number of low-income countries (below 10 thousand USD) per capita GDP are striving to improve the efficiency of health management and are at different stages of the HTA creation and implementation into healthcare systems. The opposite countries with high (above 50 thousand USD) per capita GDP are mostly tax haven countries (offshore zones) and as a rule these indicators are not linked with the real economic and industrial development. These countries do not use HTA for expert support of healthcare management decisions. Regional international cooperation increases the possibility of creating and using HTA in both low and high per capita GDP countries. [ABSTRACT FROM AUTHOR]

Introduction: Dalbavancin is a semisynthetic lipoglycopeptide long-acting antibiotic approved for the treatment of acute bacterial skin and skin structure infections (ABSSSIs). Its features can be useful in the current healthcare scenario characterized by the shortage of available hospital beds. Materials, methods and results: We implemented several actions in order to optimize the use of dalbavancin allowing an improvement strategy both from the healthcare system and the patient’s perspective in two hospital settings. In the Emergency Department we hospitalized only patients who met the clinical criteria and not the logistic criteria (i.e., the need for antibiotic therapy infusion). During the years 2017-2023, this strategy was applied in 40 cases, thus avoiding 40 hospitalizations for a total saving of 280 days of hospitalization. In the Internal Medicine ward and surgery department when there was no longer any need for hospitalization, we discharged the patient as early as possible. During the years 2017-2023, this strategy was applied in 189 cases, saving at least 1,134 days of hospitalization. The outcome of the treated patients was favorable in 228 out of 229 patients (99.5%). Conclusions: Our experience using dalbavancin in ABSSSI has been very satisfactory overall. The efficacy was close to 100%. Minor adverse events of slight severity occurred rarely. At the same time, this strategy allowed a more efficient allocation of hospital beds. Dalbavancin presents an ideal pharmacodynamic/pharmacokinetic profile for the management of ABSSSI especially in settings where shortage of hospital beds is critical.

Fundamentally precision oncology illustrates the path in which molecular profiling of tumors can illuminate their biological behavior, diversity, and likely outcomes by identifying distinct genetic mutations, protein levels, and other biomarkers that underpin cancer progression. Next-generation sequencing became an indispensable diagnostic tool for diagnosis and treatment guidance in current clinical practice. Nowadays, tissue analysis benefits from further support through methods like comprehensive genomic profiling and liquid biopsies. However, precision medicine in the field of oncology presents specific hurdles, such as the cost-benefit balance and widespread accessibility, particularly in countries with low- and middle-income. A key issue is how to effectively extend next-generation sequencing to all cancer patients, thus empowering treatment decision-making. Concerns also extend to the quality and preservation of tissue samples, as well as the evaluation of health technologies. Moreover, as technology advances, novel next-generation sequencing assessments are being developed, including the study of Fragmentomics. Therefore, our objective was to delineate the primary uses of next-generation sequencing, discussing its’ applications, limitations, and prospective paths forward in Oncology.

Clinical Microbiology has developed during the last 100 years, simultaneous with the discovery of microorganisms as causes of infections. Globalization and One Health determine present needs whereas molecular biology, automation, artificial intelligence, and bioinformatics are new tools that characterize the new developments in the field.

Abstract Objectives Patient’s health-related quality of life (HRQoL) is an important outcome measure that is considered by many payers and health technology assessment (HTA) bodies in the evaluation of treatments. We aimed to identify opportunities for HRQoL to be further incorporated into the assessment of the French HTA by comparing three health systems. We put forward recommendations that could bring further innovations to French patients. Methods We reviewed methodologies by the French, German and British HTA, and conducted a systematic review of all French (n = 312) and German (n = 175) HTA appraisals from 01 January 2019 to 31 December 2021. We also setup an advisory board of 11 ex-HTA leaders, payers, methodologists, healthcare providers and patient advocates, from France, Britain and Germany, to discuss opportunities to improve acceptance and adoption of HRQoL evidence in France. Results Our systematic review of HTA appraisals showed a higher HRQoL data rejection rate in France: in > 75% of cases the HRQoL evidence submitted was not accepted for the assessment (usually for methodological reasons, for example, data being considered exploratory; 16–75% of the appraisals mentioned HRQoL evidence, varying by therapeutic area). Overall, we found the French HTA to be more restrictive in its approach than IQWiG. Conclusions Based on these findings we articulate collaborative proposals for industry and the HAS to improve acceptance of HRQoL evidence and create a positive feedback loop between HAS and industry along four dimensions (1) patient perception, (2) testing hierarchy, (3) trial design and (4) data collection.

Introduction There is an increased interest in cost consciousness concerning healthcare spending worldwide. In the Arab world, a major transformation is underway in the healthcare sectors to achieve national and government visions to attain better outcomes with optimal value. This article contains expert recommendations on how decision-makers can implement pharmacoeconomic principles at a national level in the Arab world. Methods A multidisciplinary panel of experts was formed of policymakers, clinical pharmacists, health economists, and chronic disease control and public health experts from different countries and healthcare sectors. The panel developed consensus recommendations for different stakeholders using a framework analysis method. Results The experts discussed the limitations and opportunities of implementing the pharmacoeconomics concept in evaluating new technologies in their respective countries. Common limitations recognized in the included countries were a lack of infrastructure to support the adoption of the concept in practice, challenges in obtaining data to support the decision-making process, and the lack of human resources to raise awareness among decision-makers and the public to use health economics in making informed decisions in reimbursing new technologies. Conclusion The expert panel recommendations will guide relevant stakeholders at a national level per country. Adapting these recommendations to each setting is essential to accommodate the situation and needs of each country.

Objectives: Patient's health-related quality of life (HRQoL) is an important outcome measure that is considered by many payers and health technology assessment (HTA) bodies in the evaluation of treatments. We aimed to identify opportunities for HRQoL to be further incorporated into the assessment of the French HTA by comparing three health systems. We put forward recommendations that could bring further innovations to French patients. Methods: We reviewed methodologies by the French, German and British HTA, and conducted a systematic review of all French (n = 312) and German (n = 175) HTA appraisals from 01 January 2019 to 31 December 2021. We also setup an advisory board of 11 ex-HTA leaders, payers, methodologists, healthcare providers and patient advocates, from France, Britain and Germany, to discuss opportunities to improve acceptance and adoption of HRQoL evidence in France. Results: Our systematic review of HTA appraisals showed a higher HRQoL data rejection rate in France: in > 75% of cases the HRQoL evidence submitted was not accepted for the assessment (usually for methodological reasons, for example, data being considered exploratory; 16–75% of the appraisals mentioned HRQoL evidence, varying by therapeutic area). Overall, we found the French HTA to be more restrictive in its approach than IQWiG. Conclusions: Based on these findings we articulate collaborative proposals for industry and the HAS to improve acceptance of HRQoL evidence and create a positive feedback loop between HAS and industry along four dimensions (1) patient perception, (2) testing hierarchy, (3) trial design and (4) data collection. [ABSTRACT FROM AUTHOR]

ntroduction: Advanced Therapy Medicinal Products are a type of therapies that, in some cases, hold great potential for patients without an effective current therapeutic approach but they also present multiple challenges to payers. While there are many theoretical papers on pricing and reimbursement (P&R) options, original empirical research is very scarce. This paper aims to provide a comprehensive international review of regulatory and P&R decisions taken for all ATMPs with centralized European marketing authorization in March 2022. Methods: A survey was distributed in July 2022 to representatives of 46 countries. Results: Responses were received from 20 countries out of 46 (43.5%). 14 countries reimbursed at least one ATMP. Six countries in this survey reimbursed no ATMPs. Conclusion: Access to ATMPs is uneven across the countries included in this study. This arises from regulatory differences, commercial decisions by marketing authorization holders, and the divergent assessment processes and criteria applied by payers. Moving towards greater equality of access will require cooperation between countries and stakeholders, for example, through the WHO Regional Office for Europe’s Access to Novel Medicines Platform. [ABSTRACT FROM AUTHOR]

Osteoarthritis (OA) is a degenerative joint disease with a substantial global burden, causing chronic pain and reduced quality of life. Managing OA efficiently while maximizing healthcare resources is crucial. Health economics and health technology assessment (HTA) are central tools providing a framework to evaluate the clinical, economic, and ethical aspects of healthcare technologies and interventions. This article presents some insights into the role of health economics and the HTA process in OA management. It also illustrates an example of cost-effectiveness analysis in a specific healthcare context, on the basis of a recent clinical trial involving hyaluronic acid treatment for knee OA. While HTA offers valuable insights, it faces challenges like data availability and resource constraints. Integrating health economics into decision-making can enhance patient care and allocate resources effectively in OA and other healthcare domains.

Within the European evolutionary framework concerning citizens’ health, the focus shifts to the new HTA regulation, set to alter processes and influence decision-making at the individual country level regarding reimbursement and pricing. The ambitious goal of achieving faster and more uniform access will significantly reshape the evaluative model stabilized over the years, characterized by the creation of different rules and processes among member states and diverse output timelines across the countries. The imminent adoption of a more collaborative process necessitates member countries and companies to address several steps to ensure a smooth transition without penalizing the unique aspects of individual countries or impeding real access. Italy, actively participating in European preparatory activities with AIFA, faces challenges in adapting its formal process due to AIFA’s ongoing reform since November 2022, which is not yet materialized. National-level companies heavily depend on leadership and involvement rules from their HTA agencies, with the risk that agencies with more established engagement models with external stakeholders may present themselves on January 12, 2025, the start date of the new process for oncological and ATMP drugs, better prepared and may excel in designing scoping meetings and in formulating PICO. Therefore, the activation of a country culture of HTA and a robust and extended “readiness” phase involving all stakeholders are desirable, along with the establishment of collaboration networks with universities and scientific societies to make valuable knowledge and qualified resources more readily available, crucial element for the delicate transition from the old to the new evaluative system.

ABSTRACTThis article offers reflections on the development and management of health technology assessment (HTA) in Taiwan, drawing from my experience as a former Minister of Health and Welfare. The article explores my original vision for HTA, progress made, challenges faced, and the lessons learned during my tenure. Key areas discussed include evidence-based decision-making, the need for a balanced decision-making system, challenges in value justification of an independent HTA organization, and recommendations for countries looking to establish their own HTA agencies.

ABSTRACTThe Health Intervention and Technology Assessment Program (HITAP) was established in 2007. This article highlights 15 lessons from over 15 years of experience, noting five achievements about what HITAP has done well, five areas that it is currently working on, and five aims for work in the future. HITAP built capacity for HTA and linked research to policy and practice in Thailand. With collaborators from academic and policy spheres, HITAP has mobilized regional and global support, and developed global public goods to enhance the field of HTA. HITAP’s semi-autonomous structure has facilitated these changes, though they have not been without their challenges. HITAP aims to continue its work on HTA for public health interventions and disinvestments, effectively engaging with stakeholders and strategically managing its human resources. Moving forward, HITAP will develop and update global public goods on HTA, work on emerging topics such as early HTA, address issues in digital health, real-world evidence and equity, support HTA development globally, particularly in low-income settings, and seek to engage more effectively with the public. HITAP seeks to learn from its experience and invest in the areas identified so that it can grow sustainably. Its journey may be relevant to other countries and institutions that are interested in developing HTA programs.

Introduction: Advanced Therapy Medicinal Products are a type of therapies that, in some cases, hold great potential for patients without an effective current therapeutic approach but they also present multiple challenges to payers. While there are many theoretical papers on pricing and reimbursement (P&R) options, original empirical research is very scarce. This paper aims to provide a comprehensive international review of regulatory and P&R decisions taken for all ATMPs with centralized European marketing authorization in March 2022.Methods: A survey was distributed in July 2022 to representatives of 46 countries.Results: Responses were received from 20 countries out of 46 (43.5%). 14 countries reimbursed at least one ATMP. Six countries in this survey reimbursed no ATMPs.Conclusion: Access to ATMPs is uneven across the countries included in this study. This arises from regulatory differences, commercial decisions by marketing authorization holders, and the divergent assessment processes and criteria applied by payers. Moving towards greater equality of access will require cooperation between countries and stakeholders, for example, through the WHO Regional Office for Europe’s Access to Novel Medicines Platform.

The National Institute for Health and Care Excellence (NICE) recently completed a review of its methods for health technology assessment, involving a 2-stage public consultation. We appraise proposed methodological changes and analyze key decisions. We categorize all changes proposed in the first consultation as "critical," "moderate" or "limited" updates, considering the importance of the topic and the degree of change or the level of reinforcement. Proposals were followed through the review process, for their inclusion, exclusion, or amendment in the second consultation and the new manual. The end-of-life value modifier was replaced with a new "disease severity" modifier and other potential modifiers were rejected. The usefulness of a comprehensive evidence base was emphasized, clarifying when nonrandomized studies can be used, with further guidance on "real-world" evidence developed separately. A greater degree of uncertainty was accepted in circumstances when evidence generation raised challenges, in particular for children, rare diseases, and innovative technologies. For some topics, such as health inequality, discounting, unrelated healthcare costs, and value of information, significant changes were possibly warranted, but NICE decided not to make any revisions at present. Most of the changes to NICE's health technology assessment methods are appropriate and modest in impact. Nevertheless, some decisions were not well justified and further research is needed on several topics, including investigation of societal preferences. Ultimately, NICE's role of protecting National Health Services resources for valuable interventions that can contribute toward improving overall population health must be safeguarded, without accepting weaker evidence. • The National Institute for Health and Care Excellence (NICE) published its newly updated Health Technology Evaluation manual in January 2022, combining the processes and methods for all 4 Health Technology Evaluation programmes into a single document for the first time, accompanied by a topic selection manual. • We first categorize NICE's proposals by importance and offer a summary of the most critical changes. We then critique a number of key decisions that represent some of the most important updates for the wider health technology assessment community. • NICE notably introduced a new disease severity modifier, but decided not to make any revisions to its methods in the areas of health inequality, discounting, unrelated healthcare costs, and value of information. Most updates were modest and correspond to clarifications, formalization of best practice, and guidance for new challenges in line with existing principles. [ABSTRACT FROM AUTHOR]

This article offers reflections on the development and management of health technology assessment (HTA) in Taiwan, drawing from my experience as a former Minister of Health and Welfare. The article explores my original vision for HTA, progress made, challenges faced, and the lessons learned during my tenure. Key areas discussed include evidence-based decision-making, the need for a balanced decision-making system, challenges in value justification of an independent HTA organization, and recommendations for countries looking to establish their own HTA agencies. [ABSTRACT FROM AUTHOR]

The Health Intervention and Technology Assessment Program (HITAP) was established in 2007. This article highlights 15 lessons from over 15 years of experience, noting five achievements about what HITAP has done well, five areas that it is currently working on, and five aims for work in the future. HITAP built capacity for HTA and linked research to policy and practice in Thailand. With collaborators from academic and policy spheres, HITAP has mobilized regional and global support, and developed global public goods to enhance the field of HTA. HITAP's semi-autonomous structure has facilitated these changes, though they have not been without their challenges. HITAP aims to continue its work on HTA for public health interventions and disinvestments, effectively engaging with stakeholders and strategically managing its human resources. Moving forward, HITAP will develop and update global public goods on HTA, work on emerging topics such as early HTA, address issues in digital health, real-world evidence and equity, support HTA development globally, particularly in low-income settings, and seek to engage more effectively with the public. HITAP seeks to learn from its experience and invest in the areas identified so that it can grow sustainably. Its journey may be relevant to other countries and institutions that are interested in developing HTA programs. [ABSTRACT FROM AUTHOR]

Panos G Kanavos,1 Elisabeth Sophia Hartgers-Gubbels,2 Michael Chambers3 1Department of Health Policy, London School of Economics, London, UK; 2Boehringer Ingelheim International GmbH, Ingelheim am Rhein, Germany; 3MC Healthcare Evaluation, London, UKCorrespondence: Elisabeth Sophia Hartgers-Gubbels, Boehringer Ingelheim International GmbH, Ingelheim am Rhein, Germany, Email lisa.hartgers-gubbels@boehringer-ingelheim.comAbstract: 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.Keywords: market access, healthcare reimbursement, health technology assessment, HTA, value demonstration, health economics and outcomes research, payer

Louis P Garrison,1 Elisabeth Sophia Hartgers-Gubbels,2 Michael Chambers3 1School of Pharmacy, University of Washington, Seattle, WA, USA; 2Boehringer Ingelheim International GmbH, Ingelheim am Rhein, Germany; 3MC Healthcare Evaluation, London, UKCorrespondence: Elisabeth Sophia Hartgers-Gubbels, Boehringer Ingelheim International GmbH, Ingelheim am Rhein, Germany, Email lisa.hartgers-gubbels@boehringer-ingelheim.comAbstract: 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.Keywords: market access, healthcare reimbursement, health technology assessment, HTA, value demonstration, health economics and outcomes research, payer

Maureen PMH Rutten-van Mölken,1 Elisabeth Sophia Hartgers-Gubbels,2 Michael Chambers3 1Erasmus School of Health Policy & Management, Erasmus University Rotterdam, Rotterdam, the Netherlands; 2Boehringer Ingelheim International GmbH, Ingelheim am Rhein, Germany; 3MC Healthcare Evaluation, London, UKCorrespondence: Elisabeth Sophia Hartgers-Gubbels, Boehringer Ingelheim International GmbH, Ingelheim am Rhein, Germany, Email lisa.hartgers-gubbels@boehringer-ingelheim.comAbstract: 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.Keywords: market access, healthcare reimbursement, health technology assessment, HTA, value demonstration, health economics and outcomes research, payer

Nancy J Devlin,1 Elisabeth Sophia Hartgers-Gubbels,2 Michael Chambers3 1Melbourne School of Population and Global Health, University of Melbourne, Melbourne, Australia; 2Boehringer Ingelheim International GmbH, Ingelheim am Rhein, Germany; 3MC Healthcare Evaluation, London, UKCorrespondence: Elisabeth Sophia Hartgers-Gubbels, Boehringer Ingelheim International GmbH, Ingelheim am Rhein, Germany, Email lisa.hartgers-gubbels@boehringer-ingelheim.comAbstract: 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.Keywords: market access, healthcare reimbursement, health technology assessment, HTA, value demonstration, health economics and outcomes research, payer

Sean D Sullivan,1 Elisabeth Sophia Hartgers-Gubbels,2 Michael Chambers3 1School of Pharmacy, University of Washington, Seattle, WA, USA; 2Boehringer Ingelheim International GmbH, Ingelheim am Rhein, Germany; 3MC Healthcare Evaluation, London, UKCorrespondence: Elisabeth Sophia Hartgers-Gubbels, Boehringer Ingelheim International GmbH, Ingelheim am Rhein, Germany, Email lisa.hartgers-gubbels@boehringer-ingelheim.comAbstract: 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.Keywords: market access, healthcare reimbursement, health technology assessment, HTA, value demonstration, health economics and outcomes research, payer

Michael F Drummond,1 Elisabeth Sophia Hartgers-Gubbels,2 Michael Chambers3 1Centre for Health Economics, University of York, York, UK; 2Boehringer Ingelheim International GmbH, Ingelheim am Rhein, Germany; 3Healthcare Evaluation, London, UKCorrespondence: Elisabeth Sophia Hartgers-Gubbels, Boehringer Ingelheim International GmbH, Ingelheim am Rhein, Germany, Email lisa.hartgers-gubbels@boehringer-ingelheim.comAbstract: 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.Keywords: market access, healthcare reimbursement, health technology assessment, HTA, value demonstration, health economics and outcomes research, payer

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This article, titled "Editorial: Novel methods and technologies for the evaluation of drug outcomes and policies," discusses the evolving landscape of techniques and tools used to evaluate the safety and effectiveness of drugs. The research topic explores advancements in artificial intelligence (AI) and machine learning (ML) methodologies, as well as the integration of real-world evidence (RWE) in health technology assessment (HTA). The article also highlights the need for developing resources and best practices for conducting RWE studies and emphasizes the importance of continuous adaptation and integration of novel methods in healthcare decision-making. Overall, the studies presented in this research topic contribute to the advancement of drug evaluation and policy research, aiming to improve the efficiency and equity of healthcare systems. [Extracted from the article]

BackgroundUsing traditional health technology assessment (HTA) outcome metrics, such as quality-adjusted life-years, to assess fertility treatments raises considerable methodological challenges because the objective of fertility treatments is to create new life rather than extend, save, or improve health-related quality of life.ObjectiveThe aim of this study was to develop a novel cost-benefit framework to assess value for money of publicly funded IVF treatment; to determine the number of cost-beneficial treatment cycles for women of different ages; and to perform an incremental cost-benefit analysis from a taxpayer perspective.MethodsWe developed a Markov model to determine the net monetary benefit (NMB) of IVF treatment by female age and number of cycles performed. IVF treatment outcomes were monetized using taxpayers' willingness-to-pay values derived from a discrete choice experiment (DCE). Using the current funding environment as the comparator, we performed an incremental analysis of only funding cost-beneficial cycles. Similar outputs to cost-effectiveness analyses were generated, including net-benefit acceptability curves and cost-benefit planes. We created an interactive online app to provide a detailed and transparent presentation of the results.ResultsThe results suggest that at least five publicly funded IVF cycles are cost-beneficial in women aged

Introduction: A timely diagnosis of osteoporosis is key to reducing its growing clinical and economic burden. Radiofrequency Echographic Multi Spectrometry (REMS), a new diagnostic technology using an ultrasound approach, has been recognized by scientific associations as a facilitator of patients’ care pathway. We aimed at evaluating the costs of REMS vs. the conventional ionizing technology (dual-energy X-ray absorptiometry, DXA) for the diagnosis of osteoporosis from the perspective of the Italian National Health Service (NHS) using a cost-minimization analysis (CMA). Methods: We carried out structured qualitative interviews and a structured expert elicitation exercise to estimate healthcare resource consumption with a purposeful sample of clinical experts. For the elicitation exercise, an Excel tool was developed and, for each parameter, experts were asked to provide the lowest, highest and most likely value. Estimates provided by experts were averaged with equal weights. Unit costs were retrieved using different public sources. Results: Considering the base-case scenario (most likely value), the cost of professionals amounts to €31.9 for REMS and €48.8 for DXA, the cost of instrumental examinations and laboratory tests to €45.1 for REMS and €68.2 for DXA. Overall, in terms of current costs, REMS is associated with a mean saving for the NHS of €40.0 (range: €27.6-71.5) for each patient. Conclusions: REMS is associated with lower direct healthcare costs with respect to DXA. These results may inform policy-makers on the value of the REMS technology in the earlier diagnosis for osteoporosis, and support their decision regarding the reimbursement and diffusion of the technology in the Italian NHS.

Introduction: Health technology assessment (HTA) is an area that remains less implemented in low- and lower middle-income countries. The aim of the study is to understand the perceptions of stakeholders in Uganda toward HTA and its role in decision making, in order to inform its potential implementation in the country. Methods: The study takes a cross-sectional mixed methods approach, utilizing an adapted version of the International Decision Support Initiative questionnaire with both semi-structured and open-ended questions. We interviewed thirty key informants from different stakeholder institutions in Uganda that support policy and decision making in the health sector. Results: All participants perceived HTA as an important tool for decision making. Allocative efficiency was regarded as the most important use of HTA receiving the highest average score (8.8 out of 10), followed by quality of healthcare (7.8/10), transparency (7.6/10), budget control (7.5/10), and equity (6.5/10). There was concern that some of the uses of HTA may not be achieved in reality if there was political interference during the HTA process. The study participants identified development partners as the most likely potential users of HTA (66.7 percent of participants), followed by Ministry of Health (43.3 percent). Conclusion: Interviewed stakeholders in Uganda viewed the role of HTA positively, suggesting that there exists a promising environment for the establishment and operationalization of HTA as a tool for decision making within the health sector. However, sustainable development and application of HTA in Uganda will require adequate capacity both to undertake HTAs and to support their use and uptake. [ABSTRACT FROM AUTHOR]

Healthcare innovations often represent important improvements in population welfare, but at what cost, and to whom? Health technology assessment (HTA) is a multidisciplinary process to inform resource allocation. HTA is conventionally anchored on health maximization as the only relevant output of health services. If we accept the proposition that health technologies can generate value outside the healthcare system, resource allocation decisions could be suboptimal from a societal perspective. Incorporating “broader value” in HTA as derived from social values and patient experience could provide a richer evaluative space for informing resource allocation decisions. This article considers how HTA is practiced and what its current context implies for adopting “broader value” to evaluating health technologies. Methodological challenges are highlighted, as is a future research agenda. Ireland serves as an example of a healthcare system that both has an explicit role for HTA and is evolving under a current program of reform to offer universal, single-tier access to public services. There are various ways in which HTA processes could move beyond health, including considering the processes of care delivery and/or expanding the evaluative space to some broader concept of well-being. Methods to facilitate the latter exist, but their adaptation to HTA is still emerging. We recommend a multi-stakeholder working group to develop and advance an international agenda for HTA that captures welfare/benefit beyond health. [ABSTRACT FROM AUTHOR]

Introduction: A timely diagnosis of osteoporosis is key to reducing its growing clinical and economic burden. Radiofrequency Echographic Multi Spectrometry (REMS), a new diagnostic technology using an ultrasound approach, has been recognized by scientific associations as a facilitator of patients' care pathway. We aimed at evaluating the costs of REMS vs. the conventional ionizing technology (dual-energy X-ray absorptiometry, DXA) for the diagnosis of osteoporosis from the perspective of the Italian National Health Service (NHS) using a costminimization analysis (CMA). Methods: We carried out structured qualitative interviews and a structured expert elicitation exercise to estimate healthcare resource consumption with a purposeful sample of clinical experts. For the elicitation exercise, an Excel tool was developed and, for each parameter, experts were asked to provide the lowest, highest and most likely value. Estimates provided by experts were averaged with equal weights. Unit costs were retrieved using different public sources. Results: Considering the base-case scenario (most likely value), the cost of professionals amounts to €31.9 for REMS and €48.8 for DXA, the cost of instrumental examinations and laboratory tests to €45.1 for REMS and €68.2 for DXA. Overall, in terms of current costs, REMS is associated with a mean saving for the NHS of €40.0 (range: €27.6-71.5) for each patient. Conclusions: REMS is associated with lower direct healthcare costs with respect to DXA. These results may inform policy-makers on the value of the REMS technology in the earlier diagnosis for osteoporosis, and support their decision regarding the reimbursement and diffusion of the technology in the Italian NHS. [ABSTRACT FROM AUTHOR]

Objective: To conduct a situational analysis with the aim to inform future health technology assessment efforts (HTA) in Egypt. Introduction: The Egyptian government has set universal health coverage as a 2030 target. Several agencies have been created in the context of the ongoing healthcare reform. The Egyptian Authority for Unified Procurement, Medical Supply and the Management of Medical Technology (UPA) is one of them and was established to support strategic procurement using HTA. Methods: Description of the development of HTA in Egypt supported by a literature search as part of a scoping exercise, and a stakeholder analysis and identification of HTA capacity survey, based on previous surveys, with relevant stakeholders conducted in 2022. This was followed by a stakeholder event where results were shared and further contextualized. Results: The UPA is expected to evaluate the cost-effectiveness of health technologies and public health programs. The HTA process is being developed, focusing on the assessment of the value of new pharmaceuticals being introduced to the Egyptian market. A total of 16 participants responded on behalf of their organizations to the stakeholder analysis and identification of HTA capacity survey. More than 80% of the respondents were familiar with current efforts conducted by UPA and strongly support the implementation of HTA in Egypt. Transparency was highlighted as an important criterion. Over 90% of the respondents mentioned economic analyses as an HTA product being developed in Egypt, and medicines were the type of technology that stakeholders ranked as first in the rank of health technologies that need the output from HTA urgently. Capability building and training were highlighted as areas in which further support is required. Conclusion: This study represents the first attempt to describe the current path for HTA in Egypt. There seems to be momentum in Egypt to proceed and advance with HTA institutionalization. It would be important that next steps are built on the skills and capabilities already in place in Egypt, ensure methods and processes are in place and up to date and involve the wider system in Egypt so stakeholders can appropriately contribute and participate in the HTA process. [ABSTRACT FROM AUTHOR]

• Much attention is given to real world evidence in clinical effectiveness evaluation. • Evaluations based solely on non-RCT evidence still constitute an exception. • Still, challenges of indirectness and effect extrapolation are very prevalent in HTA. • Regulators and HTA agencies need to collaborate about developing guidance. • Guidance should address observational designs, methods of indirect comparison and managed entry. Current transformations in the pharmaceutical sector raise pressing questions about what is considered acceptable evidence for the effects of new therapies. This article aims to identifying key challenges in clinical effectiveness evaluation of new therapies and discuss possible policy responses to these challenges. The study builds on a systematic review of the 41 appraisals issued in 2019 by the Danish Medicines Council (DMC), which is responsible for the appraisal of new specialized therapies in Denmark. While much political attention currently centers on the use of 'real-world evidence', we find that clinical effectiveness evaluation based solely on non-RCT evidence still constitutes an exception in the Danish setting (9% of the evaluations). Yet, challenges of indirectness were prevalent even when evaluations were based on RCT data (54% of the evaluations). Challenges of effect extrapolation arose in about a third of the evaluations. As the identified challenges are likely to increase with the current trend from 'blockbuster' to 'niche' products in the pharmaceutical sector, we point to a need for regulators and health technology assessment agencies to collaborate about the development of guidance on the use of other study designs than traditional RCTs, methods that can reduce the risk of bias when conducting indirect comparisons, and principles for managed entry agreements. [ABSTRACT FROM AUTHOR]

Objective: To conduct a situational analysis with the aim to inform future health technology assessment efforts (HTA) in Egypt.Introduction: The Egyptian government has set universal health coverage as a 2030 target. Several agencies have been created in the context of the ongoing healthcare reform. The Egyptian Authority for Unified Procurement, Medical Supply and the Management of Medical Technology (UPA) is one of them and was established to support strategic procurement using HTA.Methods: Description of the development of HTA in Egypt supported by a literature search as part of a scoping exercise, and a stakeholder analysis and identification of HTA capacity survey, based on previous surveys, with relevant stakeholders conducted in 2022. This was followed by a stakeholder event where results were shared and further contextualized.Results: The UPA is expected to evaluate the cost-effectiveness of health technologies and public health programs. The HTA process is being developed, focusing on the assessment of the value of new pharmaceuticals being introduced to the Egyptian market. A total of 16 participants responded on behalf of their organizations to the stakeholder analysis and identification of HTA capacity survey. More than 80% of the respondents were familiar with current efforts conducted by UPA and strongly support the implementation of HTA in Egypt. Transparency was highlighted as an important criterion. Over 90% of the respondents mentioned economic analyses as an HTA product being developed in Egypt, and medicines were the type of technology that stakeholders ranked as first in the rank of health technologies that need the output from HTA urgently. Capability building and training were highlighted as areas in which further support is required.Conclusion: This study represents the first attempt to describe the current path for HTA in Egypt. There seems to be momentum in Egypt to proceed and advance with HTA institutionalization. It would be important that next steps are built on the skills and capabilities already in place in Egypt, ensure methods and processes are in place and up to date and involve the wider system in Egypt so stakeholders can appropriately contribute and participate in the HTA process.