Since the introduction of health technology assessment (HTA) in Ukraine, the international technical assistance project “Safe, Affordable, and Effective Medicines for Ukrainians” with financial contributions of the United States Agency for International Development has supported capacity building activities. In 2020, the main HTA stakeholders expressed interest in a comprehensive training program for HTA doers, users, and trainers.

To inform the design of a training program, the needs of forty HTA doers, users, and potential trainers were assessed using validated surveys. Identified knowledge gaps included comparative effectiveness, health economics, qualitative evidence synthesis, patient and public involvement, and ethical issues. Based on these results a tailored training program consisting of five modules was developed, including an introduction to HTA, as well as a train-the-trainers program.

During January–July 2023, seventy-five persons participated in the training program, while twelve HTA professionals followed the train-the-trainer program. We evaluated participants’ self-reported knowledge and skill gains by asking about their confidence level in each learning objective at the beginning and end of each training module. For each module, a learning effect was observed among participants. Furthermore, the majority of trainers felt confident to provide the introductory module on HTA, while for the other modules only one or two trainers felt completely confident to teach the content.

Establishing a training program based on the needs of HTA doers, users, and trainers as developed for Ukraine can serve as inspiration for other countries that wish to attain sustainable HTA capacity.

Health technology assessment (HTA) has become an integral part of Ukraine’s health system since its formal introduction into national legislation in 2017. By 2020, HTA was mandated for evaluating publicly funded medicines, laying the groundwork for more evidence-based healthcare decisions. Although the creation of an independent HTA agency was initially planned for 2022, implementation was delayed due to the COVID-19 pandemic and Russia’s ongoing invasion. The relevant Cabinet Resolution calls for the establishment of an autonomous agency by January 2026. This commentary outlines a strategic, evidence-informed framework to guide the agency’s formation.

Drawing on the 2018 State Strategy for Access to Medicines, the 2022 Law on Medicinal Products, and international best practices, we proposed to the Government of Ukraine a two-tier structure encompassing core business functions (HTA and appraisal, guideline development, pricing, and listing) and support business functions (data and analytics, finance and strategy, IT, human resources, legal, and communications). Each department is tasked with clear mandates and supported by performance indicators to promote transparency, accountability, and operational efficiency.

A phased roadmap for 2025–2027 details the legal, institutional, and financial steps required for successful implementation. Key opportunities – including international partnerships and system-wide reform – are weighed alongside risks such as funding uncertainty, workforce limitations, and geopolitical instability.

By embedding HTA into national policy processes and ensuring institutional independence, Ukraine can enhance the value of healthcare investments and build long-term resilience into its health system.

Since 2019 the Dutch National Healthcare Institute has operated a coverage with evidence development (CED) program for specific products with insufficient evidence of their clinical benefit: orphan medicinal products (OMPs), medicines with conditional marketing authorization (CMA), and medicines with marketing authorization under exceptional circumstances (AEC). The objective of this study is to give an overview of this program and reflect on learnings, challenges, and opportunities.

This study is a narrative policy review of the Dutch CED program and describes the different phases and stakeholder involvement. Additionally, an overview of the medicines included in the CED program is presented and put in an international perspective.

The CED program consists of four phases: candidate prescreening, research protocol drafting, signing of process agreement and financial agreement, and controlled access. Stakeholders are involved intensively throughout the process. Since 2019, six medicines have entered the program. The program is used to fill different evidence gaps for various types of medicines and indications. In other countries, these medicines are often included in restricted reimbursement programs.

The CED program is gathering clinical effectiveness data while providing patient access to OMPs, CMA, and AEC products. Important facilitating factors for the program were identified, including the involvement of all stakeholders, the only-in-research approach of data gathering, and the case-by-case evidence generation requirements and duration. Continuous evaluation is needed as the program does not yet include the expected number of medicines, and no conclusion can be drawn so far on the usefulness of the data collection.

This study’s primary objective was to test the feasibility of using the online personal utility function (OPUF) approach and develop a preliminary utility tariff for the EQ-5D-5L based on a South African community sample.

The need for an ethnically and socioeconomically diverse sample was seen as essential. This led to the need for interviewer assistance during completion of the survey instrument and translation of the instrument into multiple languages. English, Zulu, Tswana, and Afrikaans were chosen to allow the vast majority of a community sample people to participate. A sample size of sixty respondents was based on a previous OPUF pilot valuation study for the EQ-5D-5L, and a pilot study of twenty respondents was undertaken using the English language version of OPUF.

There were sixty-one respondents in the main study with most respondent characteristics being well matched with national figures, except for language. Personal utility functions could be calculated for sixty respondents, with the mean tariff showing monotonically declining utility decrements within each dimension. An examination of individual functions showed two contrasting sets of preferences that were driven by the respondents’ rating of death. A separate subgroup analysis also showed preference heterogeneity based on the home language of the respondents.

Our study showed that the application of the OPUF approach is possible in a socioeconomically diverse population in South Africa. The examination of individual personal utility functions shows marked heterogeneity of preferences that needs to be explored further so that the source of this can be established.

The simultaneous existence of low-value health care and underutilization of high-value care are global problems. Health technology reassessment (HTR) aims to optimize the value for money of technologies already in use within health care. Identifying candidate interventions for HTR remains challenging. Therefore, we tested a novel method to identify candidate outpatient prescription drugs for HTR through practice variation.

We used administrative data for all publicly funded outpatient prescriptions dispensed to persons aged 65 or older in Alberta in 2023. Through quantitative comparison of funnel plots for Anatomic Therapeutic Chemical (ATC) classes at the fourth level stratified by prescriber specialty, variation in prescription dispensation rates between prescribers was used to estimate three outcomes: the number of prescribers affected, the number of patients affected, and the potential budgetary impact. We ranked combinations of ATC class and prescriber specialty in descending order for each outcome, with use above and below the mean considered separately.

We analyzed data on 17.5 million dispensations, encompassing more than 8,000 prescribers and approximately 600,000 patients. The top ATC class–prescriber specialty combinations for each outcome showed high similarity above and below control limits while exhibiting minimal overlap between outcomes.

Our method successfully identified ATC class–prescriber specialty combinations with marked variation in use, for potential advancement through the HTR process. Depending on the perspective of those undertaking HTR of prescription drugs, different outcomes may be useful in technology prioritization. To make the ATC class–prescriber specialty combinations actionable, future efforts should focus on exploring the patients affected.

Despite omics technologies gaining traction in clinical settings, particularly in oncology, challenges persist in their widespread adoption due to the pre-requisite robust evidence supporting efficacy and cost-effectiveness. This study aims to explore the experiences of organizations working in the health technology assessment (HTA) field in evaluating omics technologies, with a particular focus on the adoption and application of specific assessment frameworks.

We conducted a global survey to gather insights into current practices and frameworks used in HTA evaluations of omics technologies.

We gathered responses from thirty-nine participants representing organizations across twenty-nine countries and five continents. Among them, 51 percent (n = 20) reported experience in evaluating omics technologies, including multi-omics tests for early disease detection, biomarker-based cancer diagnostics, and advanced genomic sequencing techniques. Only three organizations employed specific assessment frameworks: the Adelaide Health Technology Assessment Agency in Australia, the Netherlands Cancer Institute, and the Andalusian HTA Agency in Spain. These frameworks address key evaluation aspects such as analytical and clinical validity, clinical and personal utility, organizational impact, and ethical, legal, and social implications of omics technologies.

Despite their relevance, the limited adoption of tailored frameworks highlights the need for more structured and context-specific approaches to facilitate the integration of omics technologies into healthcare systems. Collaborative efforts among stakeholders, including patients, healthcare providers, policymakers, and industry representatives, are crucial for devising robust evaluation strategies addressing the complexities of omics technologies comprehensively.

This scoping review aims to synthesize the literature on pediatric health technology assessments (HTAs) and map out the challenges of assessing new technologies for use in children, with a particular focus on pharmaceutical interventions.

Conducted in accordance with the Joanna Briggs Institute Methodology, this scoping review addressed HTAs in the pediatric domain through searches of PubMed, Embase, Web of Science Core Collection, and EconLit through 22 January 2024, as well as the gray literature. Sources were excluded if they (i) were a clinical trial investigating a specific technology or an HTA of that technology, (ii) did not address the challenges of HTAs, or (iii) had no relevance to pediatrics. Two authors performed screening and data extraction independently and in duplicate.

One hundred and three reports were included. Of these, sixty were full journal articles, twenty-three were conference abstracts, and twenty were guidelines, reports, and other documents. Two important themes emerged from this work. The first was the unique position of children within society and the resulting difficulty of incorporating them within a population-wide HTA system. The second was the uncertainty that characterized pediatric HTAs due to data constraints and either a lack of guidance by HTA bodies or variations in guidance between bodies.

Many factors inherent to children, including the heterogeneity of pediatric disease populations, long-term outcomes, and children’s distinct social positions, render conducting pediatric HTAs challenging. Innovative approaches are required to address these challenges and respond to the needs of pediatric populations.

The reimbursement of, and subsequent patient access to, high-risk medical devices (MD) and in vitro diagnostics (IVD) across Europe often vary. The Health Technology Assessment Regulation (HTAR) aims to standardize clinical evaluations through Joint Clinical Assessments. Still, national differences in reimbursement frameworks and evidence integration for MD/IVD may impede the realization of HTAR’s expected benefits. This review aims to map existing reimbursement frameworks for high-risk MD/IVD, identify key oversight structures, and evaluate the use of comparative effectiveness and safety evidence in reimbursement decisions across the EU/EEA/UK.

A scoping review was conducted according to the registered protocol (osf.io/65bdk) and was reported following the PRISMA-ScR guidelines. Results were validated through direct engagement with national organizations.

Reimbursement frameworks across the EU/EEA/UK for MD/IVD vary significantly. Of the thirty-four countries reviewed, twenty-three incorporate HTA for MD/IVD reimbursement decisions; of these, only eleven countries have a formal HTA process as part of reimbursement pathways. Eight countries have structured mechanisms to address safety and effectiveness evidence uncertainty. Furthermore, twelve countries have primarily centralized processes, while six rely on regional or local decision-making.

This review highlights the variations in how countries integrate HTA into reimbursement frameworks for MD/IVD, how the national decisions are implemented, and how the evidence uncertainty is assessed. Some countries have a well-established reimbursement framework with formal HTA components, whereas others rely on ad hoc HTA processes. Understanding these differences can help optimize the use of HTAR-generated evidence. Further research is needed to capture ongoing reforms in response to the HTAR.

The introduction of a new healthcare technology within the technological facilities of a hospital is a complex action that must go through the mandatory decision-making process of health technology assessment (HTA). Nowadays, developing a universal HTA model poses a significant challenge within the current landscape. This paper describes the proposal of a novel supporting healthcare technology evaluation toolbox, aligned with the principles of the European Network for Health Technology Assessment (EUnetHTA) shared by the Regulation (EU) 2021/2282 on Health Technology Assessment (HTAR).

The proposed toolbox relies on a MATLAB-based multicriteria algorithm that mirrors the evaluative procedure following the hierarchical framework of the analytic hierarchy process. The evaluation framework involves clinical and non-clinical aspects leading to the choice of the best alternative, among the evaluated technologies, to be introduced in the technological infrastructure of the hospital. Moreover, the toolbox incorporates robust economic analysis capabilities, crucial for determining the requisite number of annual hospital procedures to ensure economic equilibrium and mitigate financial risks. Additionally, it computes the payback period, essential for evaluating the economic feasibility of technology investments. HTA evaluations at San Giovanni Addolorata Hospital demonstrate its application.

The toolbox exemplifies its efficacy in supporting informed decision-making processes, regarding the adoption of technologies like robotic systems for neurosurgery and angiographic systems, in terms of economic sustainability and clinical effectiveness.

This study underscores the toolbox’s role in advancing HTA methodologies and enhancing the efficiency and sustainability of healthcare technology integration.

Developing therapies for rare diseases is challenging due to limited evidence and high degrees of uncertainty regarding the value of new treatments. Clinical expert judgment can inform modeling assumptions and address areas of uncertainty in reimbursement submissions. As current protocols do not adequately address the challenges faced in rare diseases, this research aimed to generate recommendations for the collection and reporting of clinical expert judgment in rare diseases.

An international group of industry, payer, and patient experts with a background in rare diseases participated in a roundtable meeting, which aimed to identify practical challenges in and solutions for gathering clinical insights to aid reimbursement decisions for rare disease therapies. Recommendations were cocreated through iterative discussions and group agreement.

Developers should proactively identify uncertainties that expert judgment can address, in parallel with early evidence generation planning. Expert judgment method(s) depend on the uncertainties, with those key to decision-making requiring more robust and time-intensive methods. For highly complex and uncertain topics, methods should facilitate consensus building and expression of diverse views. Given the scarcity of rare disease experts, a high time burden falls on a few experts. Developers should engage diverse stakeholder groups to integrate broader clinical perspectives and reduce reliance on specific individuals while approaching conflicts of interest pragmatically and transparently.

These recommendations create a blueprint for developers of rare disease therapies to conduct high-quality clinical expert judgment studies. Hence, developers can present more robust evidence to inform key areas of uncertainty in reimbursement decisions, where empirical evidence is unavailable.

Health technology assessment (HTA) for medical devices (MDs) is essential for adoption decisions, but the sector’s particularities studied here defy regulatory frameworks. In Brazil, the National Policy for Health Technology Management (PNGTS) provides guidelines for HTA, but the reimbursement of MDs in the Brazilian National Health System (SUS) still faces challenges. This study aimed to identify and validate relevant domains and attributes for HTA of MDs in the SUS, considering the perspectives of various stakeholders.

To analyze and validate the essential domains and attributes for conducting HTA studies focused on the reimbursement of MDs in the SUS.

A baseline systematic review was performed, which was followed by two additional stages: a survey with 115 participants and a Delphi panel with 33 experts. Likert scales were used to assess the importance of the domains and attributes, along with open questions to collect suggestions and comments.

The domains “clinical benefits,” “evidence ecosystem,” and “budget impact” were considered fundamental. “Social participation” showed high variability in response, indicating the need for greater engagement and clarity in participation mechanisms. The inclusion of the “public policy” domain emphasizes the importance of aligning government policies with population needs.

This study reinforced the relevance of a multidisciplinary and participatory approach in HTA for MDs, with a focus on clinical outcomes, real-world evidence, and continuous monitoring. Overcoming the identified challenges, such as information gaps and the need for robust methods, is crucial for improving the reimbursement of MDs in the SUS.

The healthcare system accounts for 4 percent of United Kingdom (UK) greenhouse gas (GHG) emissions annually. In response to climate change, the National Health Service (NHS) is calling for less carbon-intensive care practices through prevention. Respiratory Syncytial Virus (RSV), a leading cause of infant hospitalization, currently has no widespread immunization program in the UK. This study estimates the impact on GHG emissions generated within the care pathway from an immunization against RSV in all infants in the UK with nirsevimab, a new monoclonal antibody used in prophylaxis.

A novel approach was applied, mapping care pathway emissions from immunization and avoiding RSV-related primary and secondary care burden. Avoided healthcare resources were estimated using a published health economic model for nirsevimab versus standard of care (SoC), which is characterized as receiving palivizumab or having no immunization intervention, assuming different universal immunization scenarios. NHS England GHG emission factors were applied to each health outcome to measure the GHG emissions associated with a nirsevimab versus SoC strategy.

Compared with SoC, a universal immunization program using nirsevimab leads to avoided GHG emissions, amounting to ~22 kilotons of CO2 equivalents per year, with immunizing all UK infants at birth leading to the greatest reduction. About 40 percent of avoided emissions were from reductions in inpatient hospitalizations.

This study shows how prevention can deliver benefits to people, NHS system capacity, and the environment. However, avoided patient care pathway emissions must be considered alongside drug lifecycle emissions, which are not included here.

Under the Universal Health Care Act of the Philippines, all health technologies should undergo health technology assessment. This manuscript details the process of the development of the Philippine guidance document for the use of real-world evidence (RWE) in the clinical evaluation of health technologies.

This study consisted of two phases. Phase 1 was a comprehensive, systematic review of all available HTA methods guides and literature related to the use of RWE in the clinical evaluation of health technologies. Based on the results of the review, a draft HTA methods guide on the use of RWE was created. Phase 2 was a validation study by expert consultation through key informant interviews (KIIs), and pilot assessment of the methods guide.

Seventy-nine articles and nine guidance papers were included, with pertinent information extracted and organized into sections. The first draft covered definitions of RWE, guidance for RWE utilization, scoping and selecting RWE, critical appraisal, data extraction, and synthesis and analysis of RWE. Changes were made to this draft based on the KIIs and pilot assessment results to produce the final output of the methods guide.

This document describes the process of creating a Philippine guidance document that covers the definition of RWE and the appropriate methods for conducting systematic search, screening, critical appraisal, data extraction, data analysis, and synthesis of RWE.

The Dutch National Health Care Institute (ZIN) advises the Dutch Minister of Health on the basic benefit package using Health Technology Assessment (HTA), focusing on necessity, clinical effectiveness, cost-effectiveness, and feasibility. Despite the huge environmental impact of the healthcare sector, this impact is not taken into consideration. Several methodological approaches to integrate the environmental impact into HTA have been proposed, including information conduit, integrated evaluation, parallel evaluation, and environment-focused evaluation. There is significant uncertainty as to which approach is the most appropriate. Therefore, it is important to understand stakeholders’ perspectives on these approaches.

To explore Dutch stakeholders’ perspectives on integrating environmental impacts into HTA and assess preferred methods and challenges.

A qualitative study using a focus group with members from ZIN (n = 7) and individual interviews (n = 7) with experts in HTA, market access, and reimbursement. Interviews were transcribed and analyzed thematically.

Stakeholders highlighted the importance of addressing environmental impacts such as pharmaceutical pollution, greenhouse gas emissions, and waste. Integrated and parallel evaluations were preferred, but barriers such as data gaps, methodological complexity, and lack of guidelines were noted.

Incorporating environmental impacts into HTA is crucial but requires clear guidelines, better data, and stakeholder collaboration to support sustainable healthcare practices.

The life cycle of health technologies contribute to air pollution, ecotoxicity, and resource depletion, impacting the environment and human health. Increasing healthcare resource use globally increases emissions that accelerate climate change and negatively affect the health of current and future generations.

Health Technology Assessment (HTA) should inform decision makers to prioritize the adoption of technologies demonstrating value in terms of health benefits, costs, and other relevant dimensions such as environmental sustainability.

This paper reports on a multistakeholder approach to guiding an international working group for Environmental Sustainability in Health Technology Assessment (ESHTA) that has been formed by Health Technology Assessment international.

A multistakeholder online workshop was held with 32 participants in May 2024 to define the critical issues to be considered. The resulting report underwent consultation among the ESHTA members and in a broader group of 90 additional worldwide stakeholder representatives.

The workshop participants recognized defining frameworks, mechanisms, and tools for embedding environmental sustainability into HTA as an opportunity to support sustainable development and quality improvement in healthcare. Achieving this requires (1) consensus on what environmental sustainability in healthcare means, (2) reconcilement with other healthcare and environmental policies, and (3) methods that are useful and applicable within HTA frameworks.

This novel collaboration aims to align the global HTA community on the role of environmental sustainability in HTA. The report provides a path for the way forward for incorporating environmental sustainability into HTA based on broad perspectives from global multistakeholders.

Health technology assessment (HTA) has been characterized as a complex adaptive system that centrally features stakeholder interactions. This article provides an overview of current practices in HTA stakeholder engagement concerning medicines.

We conducted a scoping review of English-language sources published between 2018 and 2023, including 66 peer-reviewed articles and 264 gray literature sources describing stakeholder involvement in HTA processes relating to medicines.

Industry is commonly permitted to provide a submission for funding, though the modes and time points of industry engagement are many. Clinician and patient engagement are regarded as especially important with increased intervention complexity and innovation. Stakeholder engagement is perhaps mostly conducted to enhance the collation and interpretation of evidence, not necessarily to increase the legitimacy of the HTA process or give stakeholders influence over a decision that affects them. Patients are mostly engaged through broader public consultation. Sometimes they work directly with other stakeholders. Problems with patient engagement include challenges with recruitment, time, and resource constraints. Stakeholder groups can also differ in how they view and prioritize public and patient engagement. Public engagement is often limited to a matter of transparency and public accountability, but the reasons to undertake public engagement are numerous and varied. They include gaining input on affordability or prioritization issues.

HTA decision-making committees should commit to publicly communicating how they considered and made use of various stakeholder inputs. This could build trust and confidence in the committees and guide the public and patients on the information that committees find helpful.

Real-world evidence (RWE) is increasingly used to assess and make regulatory decisions on health technologies. However, its application in healthcare payer decision-making is less well-known.

The objectives of this study were to (i) review the recent literature on how RWE has been used by healthcare payers, (ii) highlight barriers that limit the use of RWE in payer decision making, and (iii) explore how RWE has been used in various funding arrangements between payers and manufacturers. The benefits of utilizing RWE are also discussed.

A scoping review was conducted on articles published between 2014 and 2025 in PubMed (Medline), OVID EMBASE, Cochrane Library, and ProQuest Dissertations and Theses Global. Eligible articles were those written in English that discussed the use of real-world evidence among healthcare payers/decision-makers for health technology reimbursement decisions.

Nineteen articles were selected for full-text review based on the inclusion criteria. The review highlighted payers’ interest in incorporating RWE into funding and reimbursement decisions to address uncertainty in the performance of new health technologies. However, a lack of standards for collecting, analyzing, and reporting RWE limits its use. Little is known about how RWE is used in reimbursement decisions since contractual arrangements between payers and manufacturers are confidential.

Although payers are interested in using RWE to inform funding and reimbursement decisions, there are concerns regarding the scientific rigor used to generate such evidence. Having more insight into the contractual arrangements between payers and manufacturers would help to better understand how RWE informs these agreements.