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.

Horizon scanning (HS) is a methodology that aims to capture signals and trends that highlight future opportunities and challenges. The National Institute for Health and Care Research (NIHR) Innovation Observatory routinely scans for medical technologies and therapeutics to inform policy and practice for healthcare in the United Kingdom (UK). To date, there is no standardized terminology for horizon scanning in healthcare. Here, we discuss the development of a data glossary and the IOAtlas web app.

We extracted data points from 4 years’ worth of NIHR Innovation Observatory HS projects and collated them by technology type and descriptive family. A source repository was established by extracting a list of all sources used in NIHR Innovation Observatory briefing notes between 2017 and 2021. The repository was validated by external HS organizations and experts, and sources were then mapped to the appropriate time horizons. The glossary and repository were converted to an SQLite database format and connected to a free web app, IOAtlas.

After de-duplication and consolidation, a total of 148 data points were included in the glossary. The source repository consists of 149 sources, with 99 percent being compliant with searching for two or more technology types. The final SQLite database contained 35 tables with 36 relationships.

We present a data glossary to provide globalized standardization for the terminology used in HS projects. The glossary can be accessed through the IOAtlas web app. Furthermore, we provide users with an interface to generate downloadable data extraction templates within IOAtlas.

The National Institute for Health and Care Excellence (NICE) in England introduced early value assessments (EVAs) as an evidence-based method of accelerating access to promising health technologies that could address unmet needs and contribute to the National Health Service’s Long Term Plan. However, there are currently no published works considering differences and commonalities in methods used between Assessment Reports for EVAs.

This rapid scoping review included all completed EVAs published on the NICE website up to 23 July 2024. One reviewer screened potentially relevant records for eligibility, checked by a second reviewer. Pairs of independent reviewers extracted information on the methods used in included EVAs using a prepiloted form; these were checked for accuracy. Data were described in graphical or tabular format with an accompanying narrative summary.

In total, seventeen EVA Reports of sixteen EVAs were included in this scoping review. Five Reports did not specify how many reviewers undertook screening, whereas five did not report data extraction methods. Five EVAs planned to conduct meta-analyses, nine planned narrative syntheses, and seven planned narrative summaries. Eleven conceptual decision models were presented, with available evidence used to construct cost-utility analyses (N = 5); cost-effectiveness analyses (CEAs; N = 4); a mix of CEAs and cost-consequence analyses (CCA; N = 2); one CCA; and one cost-comparison.

Future EVA Reports should enhance the transparency of the methods used. Furthermore, EVAs could provide opportunities for the adoption of innovative methodological approaches and more flexible communication between EVA authors and key stakeholders, including patients and clinicians, companies, and NICE.

The objective of this paper is to present the Environmental Sustainability in Health Technology Assessment (ESHTA) Working Group’s (WG’s) opinion on the definition and scope of early Health Technology Assessment (HTA) developed by a WG under HTA International. The aim is to provide suggestions on how early HTA can support the goals of enhancing environmental sustainability in healthcare.

The HTAi ESHTA WG presents our opinion on the proposed definition and scope of early HTA. This includes a broad range of perspectives from stakeholder groups including patient experts, a policy maker, a statistician, HTA researchers and a healthcare professional, located across lower to higher resource settings and several jurisdictions. We suggest how early HTA can support the goals of enhancing environmental sustainability in healthcare.

HTA agencies play a crucial role in embedding sustainability into their evaluations and practices. Integrating environmental sustainability into HTA at three critical stages – product conceptualization, reimbursement decisions, and point of care – can optimize resource use and reduce environmental impacts. Developing sustainability metrics, defining environmental impact categories, and identifying suitable methods for assessing health technologies are essential steps. Early engagement is also vital for optimizing trade-offs and increasing acceptance by diverse stakeholders.

Incorporating environmental sustainability into early HTA can enhance the likelihood of regulatory approval and reimbursement, ultimately benefiting patients and healthcare systems. By integrating sustainability considerations at the design stage, the potential for environmental impact reduction is maximized. Future efforts should focus on developing comprehensive guidelines and methods, ensuring collaboration between early HTA and ESHTA WGs.

The UK Early Access to Medicines Scheme (EAMS), launched in 2014, enables pre-license access to medicines for areas of high unmet medical need. This study aimed to evaluate the outcomes of the scheme by analyzing subsequent marketing authorization (MA), health technology assessment (HTA), and commissioning decisions.

We conducted a retrospective analysis of all completed EAMS programs from 2014 to April 2025, reviewing MA, HTA, and commissioning outcomes.

Fifty-one EAMS programs were completed, over half in oncology. Median times from Scientific Opinion (SO) to MA, and reimbursement outcomes in England and Scotland were 4.3 (Q1: 2.6 and Q3: 7.3), 14.5 (Q1: 9.4 and Q3: 20.9), and 15.0 months (Q1: 11.4 and Q3: 18.1), respectively. Of 48 products appraised by the National Institute for Health and Care Excellence (NICE) or National Health Service (NHS) England, 50 percent received positive recommendations, 44 percent were optimized, and 6 percent were rejected. Of 45 products appraised by the Scottish Medicines Consortium or NHS Scotland, 73 percent were positive, 18 percent optimized, and 9 percent rejected. EAMS was qualitatively referenced in 48 percent of NICE appraisals and quantitatively in 18 percent.

Compared to non-EAMS products, those entering the scheme achieve faster MA and HTA timelines and higher regulatory success. However, EAMS is referenced quantitatively in less than a fifth of NICE appraisals, and fewer than half of Promising Innovative Medicine designations progress to a full SO. Administrative burdens, data demands, and liability concerns may limit uptake; addressing these barriers could enhance the scheme’s impact.

Researchers propose wider individual and societal benefits (or broad elements of value) be included in economic evaluations (EEs) of medicines. This study investigates opinions of Australian stakeholders regarding the inclusion of broader value elements in reimbursement decisions for medicines for rare diseases in Australia.

Stakeholders were invited via email to complete an online survey about their views on broader elements of value in HTA. Responses were summarised using descriptive statistics and compared using chi-square statistics.

Forty-four respondents (academia (n=11), private sector (n=33)) completed the survey between October 2023 and May 2024. Only 27% of stakeholders agree the current information about the sources of value considered in reimbursement decisions is sufficient. Stakeholders consistently agree labour productivity (>50%), adherence (>80%), reducing uncertainty due to a new diagnostic (>70%), disease severity (>71%), value to caregivers (>70%), and equity (>70%) should be considered in HTA. The majority (>70%) agreed managed entry agreements (MEA), risk share arrangements (RSA), and multi criteria decision analysis (MCDA) be used in reimbursement decision making for medicines for rare diseases. Significantly fewer academic stakeholders (40%) versus private sector (77%), believe an increased willingness-to-pay threshold be applied to medicines for rare disease.

Academic and private sector stakeholders hold similar views when considering medicines for non-rare and rare diseases. Stakeholders favour considering more value elements in HTA than referred to in the Pharmaceutical Benefits Advisory Committee (PBAC) guidelines. This study highlights further advice is needed on the factors considered in reimbursement decisions and how that would influence guidelines.

This investigation evaluates the relationships between claims of patient and health system benefit, evidence in support of those claims, and the recommendation outcomes of medical technologies assessed by the National Institute for Health and Care Excellence (NICE).

Data on evidence, claims, and recommendation outcomes were gathered from published Medical Technologies Guidances (MTGs) on the NICE Web site between 1 December 2010 and 11 April 2023. Binary logistic regressions and descriptive data analyses were performed to investigate the correlation between claims, evidence, and recommendation outcomes.

The technology was fully or partially recommended in forty-six (67.7 percent) of sixty-eight MTGs. No correlation was found between types and number of claims and type and quantity of clinical evidence. However, claims supported directly by evidence were significantly correlated (p < 0.016) with recommendation.

Evidence supporting claims is crucial for receiving a full or partial guidance recommendation. There is no clear pattern in what kind of or quantity of evidence leads to a recommendation, and to increase the probability of receiving a favorable recommendation, the manufacturer needs to plan early in the development phases on how to articulate and refine the claims and to substantiate claims through clinical evidence. It is therefore advisable to take advantage of the opportunity for scientific advice, which NICE offers.

Value frameworks play a crucial role in bridging the gap between evidence and decision making in health care, particularly in settings with limited resources as low- and middle-income countries (LMIC). In this study, we present the development of a value framework (VF) targeted to provide coverage recommendations in rapid health technology assessment reports (rHTA) as well as its first 5 years of implementation.

We performed an exhaustive literature search with the aim to identify existing VFs and their dimensions followed by the generation of a VF proposal through a mixed methods, qualitative–quantitative approach including a Delphi panel to weigh the criteria and correlate them with the subsequent recommendations. To describe its implementation, we present the results of 264 rHTA reports from 2017 to 2022.

The value framework has three main domains (quality of evidence, net benefit, and economic impact). We adapted widely used methodologies for quality of evidence and net benefit domains. The economic impact domain was the most complex to assess, so an ad hoc method was developed. Analysis of 265 HTAs revealed the distribution of recommendations across different criteria and technology types. Most were for drugs (40.5 percent) or therapeutic procedures (36 percent). With a five-category final recommendation, 0.8 percent were favorable, 19.7 percent were uncertain, and 44 percent were unfavorable.

The VF demonstrated its versatility and practicality in meeting the needs of rHTA audience, and can facilitate evidence-informed decision making. This VF serves as a valuable tool for conducting adaptive rHTAs and supports decision-making processes in Argentina and similar LMIC contexts.

Predictive biomarkers can identify patients who are more likely to respond to immunotherapy, which can guide treatment decisions. The objective of this study was to assess the potential value of predictive biomarkers in advanced NSCLC patients to guide the development of cost-effective biomarkers in this field.

A decision analytical model was constructed to compare theoretical new strategies with biomarkers to the current standard of care. The analysis was performed for three different patient groups based on PD-L1 status. Differences in health outcomes (QALYs) and costs were assessed between the current practice and these biomarker strategies.

Omitting immunotherapy in NSCLC patients with a PD-L1 score < 1 percent or between 1 and 49 percent, and a negative biomarker test, could potentially reduce healthcare costs significantly a small loss in QALYs. In these groups, a biomarker test is potentially cost-effective as the incremental cost-effectiveness ratio largely exceeds a willingness-to-accept threshold of €80,000 saved per QALY lost. For patients with a PD-L1 score > 50 percent, a considerable QALY gain can potentially be realized by adding chemotherapy to patients with a negative biomarker test. However, this comes at a significant increase in costs and appears not to be cost-effective.

In general, predictive biomarkers seem to have the potential to increase the cost-effectiveness of treatment with immunotherapy in patients with advanced NSCLC. Optimal positioning of a biomarker depends on the weighing between health impact and costs.

Evaluate the extent to which delivery constraints were considered during the health technology assessment (HTA) of cell and gene therapies.

Constraints on delivering cell and gene therapies were identified from guidance documents by the National Institute for Health and Care Excellence Technology Appraisal and Highly Specialised Technologies streams until October 2024. Inductive coding was performed to identify delivery constraints reported within the guidance documents. A quantitative analysis established the proportion of guidance documents that reported delivery constraints, and the distribution of these constraints across the guidance documents (frequency, mean range).

Sixteen guidance documents for cell and gene therapies were identified. Thirteen guidance documents (81.3 percent of the sample) reported constraints on delivering cell and gene therapies. Thirty-one examples of delivery constraints were reported. The mean number of constraints per guidance document was 1.9 (range: 0–6 constraints). The reported constraints were grouped by six different themes: provider experience (n = 8); testing constraints (n = 7); geographical constraints (n = 5); payment constraints (n = 5); maturity of developments in care (n = 4); and infrastructure constraints (n = 2).

Formal HTA processes are one effective way to identify constraints on delivering cell and gene therapies. Proactive identification of potential delivery constraints will help decision-makers, providers, and manufacturers generate strategies that improve the implementation of cell and gene therapies. Overcoming delivery constraints will strengthen the likelihood of realizing the expected incremental net health benefit of cost-effective cell and gene therapies for patients across a healthcare system.