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.

Patient involvement enhances transparency, legitimacy, and responsiveness in pharmaceutical reimbursement decisions. Guided by the mosaic model, this study recognizes effective patient engagement requires diverse context-specific approaches. Despite Taiwan’s National Health Insurance Administration (NHIA) implementing policies, gaps remain between intent and practice. This study evaluates NHIA’s incorporation of patient inputs into reimbursement decisions and examines factors influencing involvement.

We analyzed pharmaceutical company-initiated reimbursement submissions for catastrophic illnesses reviewed by the Pharmaceutical Benefit and Reimbursement Scheme Joint Committee (PBRS) from 2016 to 2023. Data sources included PBRS meeting records, the Online Patient Opinion Platform (OPOP), and NHIA notification E-mails. Generalized linear models identified predictors of patient involvement. The association between patient involvement and PBRS decisions was also explored.

Patient involvement occurred in 28.4 percent (80/282) of all submissions, increasing from 17 percent (2016) to 44 percent (2023). Despite aligning with OPOP criteria, patient involvement remained incomplete. Discussion-type submissions, oncology drugs, and new drug applications showed higher involvement, whereas autoimmune diseases and new indication submissions had lower involvement. Budget impact and innovation categories were not significant predictors in adjusted models. The presence of patient involvement was not significantly associated with the PBRS approval rate. Ad hoc analysis revealed increased involvement for new indications following policy expansion.

Despite NHIA’s efforts, patient involvement implementation remains suboptimal. Structured mechanisms and expanded patient involvement beyond high-profile submissions and PBRS are crucial to broaden patient involvement. This study provides practical insights for East Asian healthcare systems advancing patient involvement amid limited empirical research.

Medicines and devices have significant negative impacts on the environment. Increasingly, Health Technology Assessment (HTA) agencies, which inform healthcare decision making, are expected to integrate environmental issues into their assessments. This study assessed how HTA agencies have responded to these calls, with a focus on two national agencies that have committed to this agenda.

This descriptive study was conducted using document review. All relevant documents from both agencies were systematically collected and analyzed using descriptive statistics and content analysis.

Thirty documents (2015–2024), from Canada’s Drug Agency (CDA) (17) and England’s National Institute for Health and Care Excellence (NICE) (13) that included environmental considerations were analyzed. Although no HTAs have assessed environmental data, primarily due to a lack of data and methods, documents demonstrate that CDA and NICE are employing varied strategies to incorporate environmental considerations through technology guidance. The agencies demonstrate both differences and similarities in approach: NICE focused on carbon and the use phase, whereas CDA focused on multiple environmental impacts across the lifecycle; both agencies are beginning to address the environmental impacts of devices, but there is a notable absence of medicines-related work.

This study demonstrates that the agencies are exploring alternative strategies to elevate attention to the environmental impacts of health technologies. Differences in focus (e.g., whether to prioritize carbon emissions) and shared inattention to medicines point to deeper tensions. Thus, although both agencies have taken important steps forward, much work remains to fully address the environmental harms of health technologies.

In 2020, RWE4Decisions, a multi-stakeholder initiative commissioned by the Belgian payer, published stakeholder actions to support the generation, analysis, and interpretation of real-world evidence (RWE) to inform the decision making of health technology assessment (HTA) bodies/payers for highly innovative medicines in the European Union (EU). Since 2020, changes in the decision-making environment and advancements in RWE have created an impetus to update stakeholder actions for the EU and Canada.

RWE4Decisions’ experts led focus groups with individual stakeholder groups (HTA bodies/payers, pharmaceutical industry, clinicians, patients, registry holders, and data analytical experts). Each focus group crafted new actions for their stakeholder, then the actions were discussed and revised in a multi-stakeholder meeting, a public webinar, and a public consultation. Themes across actions and meetings were identified.

Detailed new actions for each stakeholder group are presented. Key themes identified are the need to address interorganizational fragmentation regarding secondary data use and methodologies to build robust RWE. HTA bodies/payers need to develop a common vision about the potential use of RWE. The role of the whole clinical team as primary data collectors is critical. Opportunities for scientific advice across the life cycle of a medicine are essential, and the implementation of RWE guidance related to HTA is paramount. Progress requires specific, operational actions and a collective effort by a variety of stakeholders.

Carrying out these actions will facilitate the development of methodological best practices for generating RWE to inform HTA of highly innovative medicines and build trust between stakeholders in the use of RWE.

The increasing relevance of horizon scanning (HS), early awareness (EA), and disinvestment (DIS) highlights the need for a structured approach to capacity building. Although these fields are essential for evidence-based policy decisions, a harmonized education and training framework to develop necessary competencies is lacking. This article presents the development of a curriculum designed to address this gap in training.

A transdisciplinary working group was established, drawing on international stakeholders from academia, the public sector, and industry. Using an iterative consensus-driven approach, the group developed a modular curriculum. The curriculum design incorporated best practices from existing education programs in related fields and emphasized case-based learning strategies to ensure contextual adaptability.

The resulting curriculum covers theoretical foundations, practical applications, and decision-making processes related to HS, EA, and DIS in eight modules. It supports diverse learner needs, including trainees, training institutions, and public and private organizations, and is designed to be flexible, scalable, and applicable across different regional and organizational contexts.

This curriculum initiative represents a major step toward harmonizing capacity building in HS, EA, and DIS. It fosters sustainability, enhances global health system preparedness, and provides a structured educational platform to support the effective integration of emerging health technologies and evidence-based disinvestment strategies.

Prioritization of health technologies for insurance coverage is usually based on explicit and implicit criteria. This study presents the development of the multi-criteria decision analysis (MCDA) model, the Iranian Health Insurance Benefit Optimization Model (IR-HIBOM), to inform the design of basic health insurance benefit packages.

An initial set of twenty-nine potential allocation criteria was identified through a review of available evidence and other relevant literature. Review of this set by three specialized panels yielded a final set of thirteen criteria. A cross-sectional survey using the best–worst scaling method was then fielded to 163 health system experts to evaluate their preferences regarding the relative importance of the allocation criteria. The mixed logit method was employed to determine the weight of the relative importance of each criterion. Subsequently, a multilevel criteria scoring framework was defined based on a review of similar models and input from a panel of five expert members of the study team. Finally, model’s appraisal was conducted.

Thirteen criteria, including relative safety, efficacy, disease severity, access to alternative health technologies, budget impacts, cost-effectiveness, quality of evidence, population size, age, job absenteeism, economic status, daily care needs, and ease-of-use/acceptance were selected. Cost-effectiveness and ease-of-use criteria had the highest and lowest relative importance weights, with 30.5 percent and 1 percent, respectively. Furthermore, scores were determined for the several levels of each criterion, and decision rules were defined for the cost-effectiveness and budget impact criteria. The final model’s appraisal, based on weighted scores of thirteen selected technologies, indicated that it was valid and applicable.

The IR-HIBOM demonstrated its potential utility in the health resource allocation.

The rising cost of oncology care has motivated efforts to quantify the overall value of cancer innovation. This study aimed to apply the MACBETH approach to the development of a value assessment framework (VAF) for lymphoma therapies.

A multi-attribute value theory methodological process was adopted. Analogous MCDA steps developed by the International Society for Health Economics and Outcomes Research (ISPOR) were carried out and a diverse multi-stakeholder group was recruited to construct the framework. The criteria were identified through a systematic literature review and selected according to the importance score of each criterion given by stakeholders, related research and expert opinions. The MACBETH method was used to score the performance of alternatives by establishing value functions for each criterion and to assign weight to criteria.

Nine criteria were included in the final framework and a reusable model was built: quality adjusted life years (QALYs), median progression-free survival, objective response rate, the incidence of serious adverse events (grade 3–4), rates of treatment discontinuation due to adverse events, annual direct medical costs, dosage and administration, the number of alternative medicines with the same indication and mechanism, mortality of the disease. The weights of each criterion in the order presented above are 17.43 percent, 16.11 percent, 14.39 percent,13.54 percent,11.83 percent,11.30 percent,7.08 percent,4.59 percent, and 3.73 percent.

A criterion-based valuation framework was constructed using multiple perspectives to provide a quantitative assessment tool in facilitating the delivery of affordable and valuable lymphoma treatment. Further research is needed to optimize its use as part of policy-making.