In 2025, oncology drugs with new active substances and advanced therapy medicinal products will undergo joint clinical assessment (JCA). The comparative analysis of the clinical evidence as defined in the Regulation (EU) 2021/2282 on health technology assessment (HTAR) will save national/regional submissions of the same evidence. JCA will be available early supporting appraisal and decision-making, which remains within the responsibility of member states (MS).

Targeted searches on JCA and statements from stakeholders were performed and analyzed. We conducted interviews with current and former national payers, as well as members of HTA agencies, across Germany, France, Italy, and Eastern Europe to explore their perspectives on the anticipated implications of JCA on decision-making processes and reimbursement strategies in Europe. Focus was on reduced/additional effort for authorities and health technology developers (HTDs), required national amendments, and potential discrepancies between JCA outcome and MS benefit evaluations.

Stakeholders appreciate the standardized methodology and guidance on HTA, which, especially in countries without an established HTA system, could enhance patients’ access to new treatments by considering JCA in decision-making. The comprehensive evidence compilation may also save resources in pursuing national/regional submissions. On the other hand, country-based appraisals within the MS could lead to diverse conclusions, and there is uncertainty as to which extent national authorities will adopt JCA and how its integration into decision-making will be handled. Some stakeholders challenge an impact on local patients’ access as reimbursement and pricing processes remain within MS responsibility.

JCA is a long-desired achievement and will set the groundwork for timely access of new treatments in the MS. However, presently there are several uncertainties on how JCA will impact decision-making and whether MS appraisal could lead to contradictory value conclusions for a given treatment. Future adjustments to national/regional procedures and refinement of the JCA framework are expected.

In Québec, Canada, decisions about implementing innovations are taken both centrally for province-wide access and locally by healthcare institutions. There is no systematic evaluation process and various stakeholders are involved, notably within a new nationwide governance structure. There was a wish to increase consistency and clarity with the principles and methods used by various bodies across the innovation lifecycle.

The starting point was the Institut national d’excellence en santé et services sociaux (INESSS) multidimensional framework, which focuses on the population-level, clinical, economic, organizational, and sociocultural value of drugs, technologies, and interventions. The framework, already under evolution drawing on Responsible Innovation in Health (RIH), evolved through collaborative work between INESSS’ methodological and scientific teams, but also and foremost with diverse groups and institutions within the provincial innovation ecosystem (e.g., university-based incubators, regional hospitals). The first steps were to capture current concepts and practices from different stakeholders, as well as their operational needs in terms of assessment tools.

This multistakeholder taskforce resulted in the development of an operational toolbox meant to guide the value appraisal of innovations through a lifecycle approach. First aimed at stakeholders involved locally in healthcare institutions, the work conducted was equally beneficial to INESSS by enabling its evaluation teams to contribute to the operational tools needed to enhance clarity and legibility of the agency’s processes and methods. The level of collaboration with stakeholders across the province was also unique and has strengthened the understandability and actionability of the toolbox developed. Some challenges were faced, and related actions will be discussed.

Both the taskforce process and its output contributed to improving consistency in the assessment of innovations across the province. They made more explicit what may sometimes be perceived as the HTA “black box.” The INESSS value appraisal framework also evolved considering key elements of responsibility from RIH and through this collaboration with stakeholders, and its applicability in different contexts was reinforced.

Evaluating the impact of patient involvement in health technology assessments (HTA) may help improve practices and avoid ineffective activities. Evaluation, however, continues to be infrequent, inconsistent, and often only relates to process quantity or quality. The Patient and Citizen Involvement in HTA Interest Group (PCIG) within Health Technology Assessment International set out to contextualize this impact to support evaluation.

Given the lack of established methodology to measure impact, the team performed a qualitative analysis of first-hand accounts about perceived changes in HTA due to involvement of patient stakeholders. A questionnaire was developed, piloted, and rolled out to collect personal perspectives from stakeholders with relevant experience. The stories were analyzed in the aggregate to identify themes in the data.

From January 2019 to September 2021, twenty-four responses were collected through PCIG’s network. Responses (including one joint industry-HTA body submission) came from patient representatives (12), HTA bodies (11), and industry representatives (2) from North America (5), South America (3), Europe (13), and Asia Pacific (3). Based on themes commonly reported, a three-domain framework for evaluating impact is proposed: impact on basis of HTA result or recommendation, impact on HTA body, and impact on patient participants. The framework includes components under each domain to support reporting.

Using the Three-Domain Impact Framework may be useful in identifying, evaluating, and communicating the value of patient involvement in HTA. Enhancing and increasing reporting practices may improve transparency and facilitate process improvements for meaningful integration of patient stakeholders into HTA appraisals across jurisdictions.

Appraisal decisions on additional benefit of new medicines within the German health technology assessment (HTA) body Gemeinsamer Bundesausschuss (G-BA) are made by voting among the member of the G-BA plenary. We identified and analyzed key characteristics of decisions that were not reached by consensus.

G-BA’s homepage was used to identify AMNOG (German Medicines Market Reorganization Act) procedures that started after January 2011 and were finalized before November 2023. Appraisal voting is conducted publicly, and results are documented in the data source of the German Association of Research-Based Pharmaceutical Companies (vfa). Both the payer (National Association of Statutory Health Insurance [GKV-SV]) and provider (National Association of Statutory Health Insurance Physicians, Dentists and the German Hospital Federation) “benches” have an equal number of votes with the independent chair of the G-BA acting as swing vote in case of discrepant decisions. Discrepant voting instances were extracted and analyzed.

From January 2011 to November 2023, G-BA conducted 908 appraisals of medicines. In 66 appraisals, (7.3%) decisions were not reached by consensus. Discrepant voting was related to oncological (n=28), metabolic (n=15), infectious (n=12), neurologic (n=3), cardiovascular (n=2), psychiatric (n=2), dermatologic (n=2), musculoskeletal (n=1) and urogenital conditions (n=1) conditions. Fourteen discrepant voting instances related to orphan medicines. The best benefit category reached in the 66 discrepant decisions were: major (n=2), considerable (n=16), minor (n=19), non-quantifiable (n=13), and no benefit (n=16). In all discrepant voting decisions, the provider bench favored a better scoring versus the payer bench.

Appraisal decisions within G-BA are reached by voting. The appraisals are a key element within the subsequent price negotiations. In all discrepant decisions, the payer bench suggests less benefit (strength of benefit, respectively) versus the provider bench, indicating a procedural challenge with the GKV-SV being involved in both the voting on the additional benefit and the negotiation of price.

Health Technology Assessment (HTA) practitioners recognize the significance of qualitative methodologies that focus on how a technology is feasible, meaningfulness, acceptable, and equitable. This mapping aimed to delineate the frameworks employed to synthesize qualitative evidence and assess the quality of synthesis in HTA .

Mapping was conducted using Medline, LILACS, CINAHL, Embase, Web of Science, Scopus, PsycINFO, Cochrane Library, JBI, and ScienceDirect databases. Gray literature searches included PROQUEST, Open Grey, Canadian Agency for Drugs and Technologies in Health’s Grey Matters, Google Scholar, and HTA agency websites. The inclusion criteria were centered on global qualitative evidence synthesis frameworks. The data are presented in the tables.

Of the 2054 articles, 31 were included, mostly from Europe. Guide was the type of document more cited, and most authors are from HTA agencies and universities. Incorporating both patient and family perspectives is the most cited reason for include qualitative evidence. Regardless of the framework or tool, SPICE was the main acronym, and RETREAT was preferred for approach selection. Thematic synthesis dominated analytic methods, and CASP was the primary quality appraisal tool. GRADE-CERQual graded evidence synthesis, with ENTREQ as the top reporting guidance. The GRADE evidence-to-decision framework was mentioned for recommendations.

This mapping highlights the movement incorporate qualitative evidence in HTA employing specific frameworks. Despite the similarities among documents, most of them describe part of the process to synthesize qualitative evidence. Standardizing procedures to incorporate qualitative evidence into HTA can enhance decision-making. These findings offer essential considerations for HTA practice.

The primary objectives of this umbrella review were to (a) quantify the relative importance, of “severity” and “rarity” criteria in health resource allocation; and (b) analyze the contextual factors influencing the relative importance. The secondary objective was to examine how “severity” and “rarity” criteria are defined.

Searches were carried out in PubMed and Embase to identify eligible systematic reviews. Quality appraisal of systematic reviews was undertaken. From identified systematic reviews, primary studies were extracted and further screened for eligibility. The inclusion of severity and rarity criteria and their respective weights in primary studies were examined. Descriptive and regression analyses were performed.

Twenty-nine systematic reviews were screened, of which nine met the inclusion criteria. Primary studies included in these systematic reviews were retrieved and screened, resulting in forty articles included in the final analysis. Disease severity was more frequently considered (n = 29/40) than disease rarity (n = 23/40) as an evaluation criterion. Out of all cases where both were included as evaluation criteria, disease severity was assigned higher weights 84 percent of the time (n = 21/25).

Our review found consistent evidence that disease severity is more relevant and preferred to rarity as a priority-setting criterion albeit constraints in statistical analysis imposed by limited sample size and data availability. Where funding for rare diseases is concerned, we advocate that decision-makers be explicit in clarifying the significance of disease severity and/or rarity as a value driver behind decisions. Our findings also reinforce the relevance of disease severity as a criterion in priority setting.

Health technology assessment (HTA) plays a pivotal role in healthcare decision-making, evaluating the cost-effectiveness of emerging technologies. Central and Eastern Europe (CEE) presents a unique context for HTA, marked by diverse healthcare systems, economic variations, and regulatory frameworks. This study addresses the critical issue of transferability of economic models within HTA in CEE, aiming to bridge existing gaps and enhance the region’s capacity for informed decision-making.

A comprehensive and systematic approach was employed to assess the transferability of economic models in the context of HTA across CEE. We conducted an extensive literature review, analyzed HTA reports, and engaged in expert consultations to understand the nuances of the healthcare systems in the region. Key factors influencing the transferability of economic models, such as healthcare infrastructure, economic disparities, and regulatory landscapes, were systematically evaluated. The study focused on a range of economic models commonly used in HTA, including cost-effectiveness analysis, budget impact analysis, and multiple-criteria decision analysis.

Our findings highlight the intricate dynamics influencing the transferability of economic models within HTA in CEE. While certain economic models exhibit a degree of generalizability across the region, there are notable variations based on specific contextual factors. Economic models designed for Western healthcare systems may not seamlessly translate to the CEE context due to differences in healthcare delivery, patient populations, and policy frameworks. The study identifies critical determinants of transferability, including the level of healthcare infrastructure development, economic disparities among CEE countries, and the diversity in regulatory approaches.

In conclusion, this study emphasizes the need for a nuanced and context-specific approach to the transferability of economic models within HTA in CEE. Bridging the gap in transferability enhances the region’s capacity for evidence-based healthcare resource allocation and contributes to the overall efficiency and sustainability of healthcare systems in CEE.

The adoption and development of health technology assessment (HTA) in the hospital setting (HB-HTA) differs among countries, but in all cases, it encounters barriers and facilitators. An analysis was conducted to promote international cooperation to develop strategies both to enforce common facilitators and overcome common barriers. HTA specialists from seven countries (at least two per country) contribute to the study.

HTA experts from countries in Western Europe (three), Central and Eastern Europe (three), and Central Asia (one) voluntarily participated in the project. They provided a description of the scenario that HB-HTA faces in their country, then a two-round Delphi study was conducted. The survey was based on twelve statements that were categorized into external and internal barriers and external and internal facilitators. Next, panel experts ranked statements on a seven-point Likert scale with a median agreement score greater than or equal to six and an interquartile range (IQR) greater than or equal to one accepted as reaching consensus. The goal was to identify similarities and differences in the HB-HTA scenarios among countries.

Fifteen experts from France, Hungary, Italy, Kazakhstan, Poland, Switzerland, and Ukraine contributed to the analysis. Among the twelve statements, six were ranked as reaching consensus (two barriers, four facilitators). One external and one internal barrier, which reached consensus, were (i) lack of formal recognition of the role of HB-HTA in national/regional legislations and (ii) limited human resources. Two external facilitators were (iii) creation of a network among hospitals performing HB-HTA and (iv) dissemination of HB-HTA methods and activities, while two internal facilitators were (v) top hospital management support in evidence-based decision-making and (vi) training initiatives dedicated to HB-HTA.

The analysis showed a consensus on the barriers and facilitators for HB-HTA. This creates the opportunity for internationally developed strategies to enforce facilitators and support the adoption and sustainability of HB-HTA. Future research should extend the analysis to other countries and include the results of the HB-HTA survey conducted in 2023 by the HB-HTA Interest Group.

To describe the type of evidence and the clinical benefit of cancer medicines assessed for funding in Australia by the Pharmaceutical Benefits Advisory Committee (PBAC) and to assess it with the European Society of Medical Oncology Magnitude of Clinical Benefit Scale version 1.1 (ESMO-MCBS).

All data on applications submitted to PBAC between 2010 and 2020 were extracted from PBAC Public Summary Documents available online. ESMO-MCBS ratings were retrieved from the ESMO-MCBS website.

Then, 182 cancer indications for 100 cancer medicines were examined by PBAC, including 124 (68.1 percent) for solid tumors and 58 (31.9 percent) for hematological cancers. A total of 137 (75.3 percent) indications were recommended for PBS funding and 40 (21.9 percent) were rejected. Randomized clinical trials (RCTs) were the main source of evidence in 154 indications (84.6 percent), single-arm studies in 28 (15.4 percent) indications. Statistically significant improvement in overall survival (OS) was reported in 80 (44 percent) of the indications, with a median OS gain of 3.0 months (range 0.9–17.0) for solid tumors and 8.2 months (range 1–49.1) for hematological cancers when mature OS data were available. The ESMO-MCBS score was available for 99 solid tumor indications, of which 51 (51.5 percent) showed substantial clinical benefit according to ESMO-MCBS, including 40 (54.1 percent) of PBAC-recommended indications and 9 (42.9 percent) of PBAC-rejected indications. There was no association between the ESMO scoring and PBAC decision.

Most cancer medicines indications considered by PBAC were supported by RCTs. A minority showed a substantial improvement in OS.

We compared the Institute for Clinical and Economic Review’s (ICER) ratings of comparative clinical effectiveness with the German Federal Joint Committee’s (G-BA) added benefit ratings, and explored what factors, including the evidence base, may explain disagreement between the two organizations.

Drugs were included if they were assessed by ICER under its 2020–2023 Value Assessment Framework and had a corresponding assessment by G-BA as of March 2023 for the same indication, patient population, and comparator drug. To compare assessments, we modified ICER’s proposed crosswalk between G-BA and ICER benefit ratings to account for G-BA’s extent and certainty ratings. We also determined whether each assessment pair was based on similar or dissimilar evidence. Assessment pairs exhibiting disagreement based on the modified crosswalk despite a similar evidence base were qualitatively analyzed to identify reasons for disagreement.

We identified 15 assessment pairs and seven out of fifteen were based on similar evidence. G-BA and ICER assessments disagreed for each of these drugs. For 4/7 drugs, G-BA (but not ICER) determined the evidence was unsuitable for assessment: for 2/4 drugs, G-BA concluded the key trials did not appropriately assess the comparator therapy; for 1/4, G-BA did not accept results of a before-and-after study due to non-comparable study settings; for 1/4, G-BA determined follow-up in the key trial was too short. Among assessment pairs where both organizations assessed the evidence, reasons for disagreement included concerns about long-term safety, generalizability, and study design.

This study underscores the role of value judgments within assessments of clinical effectiveness. These judgments are not always transparently presented in assessment summaries. The lack of clarity regarding these value-based decisions underscores the need for improvements in transparency and communication, which are essential for promoting a more robust health technology assessment process and supporting transferability of assessments across jurisdictions.

Health technology assessment (HTA) organizations generate guidelines to inform healthcare practices toward improved health outcomes. This review sought to identify and classify outcomes of guidelines from HTA organizations within published research.

We performed a systematic mixed studies review of empirical studies that (a) referred to a published guideline from an HTA organization and (b) reported an outcome resulting from a guideline. We searched the published literature in English or French within seven databases. Outcome types were classified within five dimensions of an existing framework for online health information (e.g., relevance, cognitive/affective impact, and use). Subdimensions were inductively developed. A two-phase sequential data synthesis was performed. Phase 1: a hybrid deductive–inductive thematic analysis identified the types of outcomes and displayed their relationships on a concept map. Phase 2: descriptive statistics were tabulated by the type of outcome.

A total of 6,719 records were retrieved through searches on 6 February 2023. After screening, we included 120 observational studies (twenty-one qualitative, ninety-four quantitative, and five mixed methods). Phase 1 identified twenty-nine types of outcomes. The most frequently reported outcomes were within the organizational dimension (reported in ninety-four studies). The most common subdimensions were “Referrals” (thirty-eight occurrences), the “Quality of Prescriptions” (fifteen occurrences), and the “Quality of Diagnosis” (eight occurrences). For Phase 2, we could only generate descriptive statistics on seventeen outcomes. These were almost equally distributed among positive, neutral, and negative effects. Our results contribute to knowledge about the outcomes of HTA guidelines and options for documenting and measuring them in future evaluations.

Healthcare disinvestment requires multi-level decision-making, and early stakeholder engagement is essential to facilitate implementation and acceptance. This study aimed to explore the perceptions of Malaysian healthcare stakeholders to disinvestment initiatives as well as identify disinvestment activities in the country.

A cross-sectional online survey was conducted from February to March 2023 among Malaysian healthcare stakeholders involved in resource allocation and decision-making at various levels of governance. Response frequencies were analyzed descriptively and cross-tabulation was performed for specific questions to compare the responses of different groups of stakeholders. For free-text replies, content analysis was used with each verbatim response examined and assigned a theme.

A total of 153 complete responses were analyzed and approximately 37 percent of participants had prior involvement in disinvestment initiatives. Clinical effectiveness and cost-effectiveness ranked as the most important criteria in assessment for disinvestment. Surprisingly, equity was rated the lowest priority despite its crucial role in healthcare decision-making. Almost 90 percent of the respondents concurred that a formal disinvestment framework is necessary and the importance of training for the program’s successful implementation. Key obstacles to the adoption of disinvestment include insufficient stakeholder support and political will as well as a lack of expertise in executing the process.

While disinvestment is perceived as a priority for efficient resource allocation in Malaysian healthcare, there is a lack of a systematic framework for its implementation. Future research should prioritize methodological analysis in healthcare disinvestment and strategies for integrating equity considerations in evaluating disinvestment candidates.

The COVID-19 pandemic put substantial strain on healthcare systems globally. Early decision-making about diagnostic tests and treatments was driven by the need for rapid responses with a focus on reducing clinical burden. As COVID-19 continues its transition into an endemic state, health technology assessment (HTA) agencies will need to consider the clinical- and cost-effectiveness of tests and treatments, as with other conditions.

We first conducted a systematic literature review in July 2021 and updated the search in July 2023. The review aimed to identify economic evaluations of diagnostics for SARS-CoV-2 and treatments for COVID-19 using predefined search strategy across journal databases and sources of grey literature. In the update, an additional targeted search was completed with terms relating to novel treatments. Search results were screened by title and abstract, and full texts of potentially relevant studies were reviewed against selection criteria. Studies with very serious methodological limitations were excluded. Findings from studies were synthesized narratively due to high levels of heterogeneity.

The database search identified 8,287 unique records, of which 54 full texts were reviewed, 28 were quality assessed, and 15 were included. Three further studies were included through HTA sources and citation checking. Of the 18 studies ultimately included, 16 evaluated pharmacological treatments including corticosteroids, antivirals, and immunotherapies. Two studies in lower-income settings evaluated the cost-effectiveness of rapid antigen tests and critical care provision. In most studies, a healthcare or payer perspective was used, and the comparator was standard care. There were 17 modeling analyses and one trial-based evaluation. Cost–utility analyses using QALYs were the most common analysis type.

This update indicates that there are cost-effective treatments for COVID-19, with repurposed pharmacological treatments like dexamethasone presenting best value. There also appear to be promising options for people with severe disease alongside standard care. Future economic evaluations would benefit from reflecting the changing context around COVID-19 with parameters that reflect current circumstances, and fully incremental analyses comparing different treatment options.

Compared to single-gene BRAF testing to guide targeted treatment with BRAF/MEK inhibitors for advanced melanoma, multigene panels can identify additional gene mutations with known therapeutic or prognostic relevance. Implementation of multigene panels remains uneven across healthcare systems given an uncertain clinical and economic evidence base. We determined the population-level cost-effectiveness of multigene panels compared to single-gene BRAF testing for advanced melanoma.

Our population-based retrospective study emulated a hypothetical pragmatic trial comparing multigene panel sequencing to single-gene BRAF testing. We drew on comprehensive patient-level clinical and health administrative data between September 2016 and December 2018 in British Columbia, Canada. To emulate random treatment assignment, we 1:1 matched multigene panel patients to contemporaneous single-gene tested controls using genetic algorithm-based matching. We estimated three-year overall survival and healthcare costs (2021 CAD), and incremental net monetary benefit (INMB) for life years gained (LYG) using inverse probability of censoring weighted linear regression and nonparametric bootstrapping. We also estimated overall survival using Weibull regression and Kaplan–Meier survival analysis.

We matched 147 patients with advanced melanoma receiving multigene panel sequencing to contemporaneous single-gene-tested controls, achieving good balance for all 15 baseline clinical and sociodemographic covariates. After matching, mean incremental costs were CAD19,447 (USD14,217) (95% confidence interval [CI]: −CAD18,517 [−USD13,537], CAD76,006 [USD55,565]; p=0.41) and mean incremental LYG were 0.22 (95% CI: −0.05, 0.49; p=0.12). We found uncertain differences on overall survival using Kaplan–Meier (stratified Log-rank test p=0.11) and Weibull regression (HR: 0.73 [95% CI: 0.51, 1.03]; p=0.07) survival analysis. Cost differences were driven by systemic therapy (∆C: CAD8,665 [USD6,334]; 95% CI: −CAD36,387 [−USD26,600], CAD53,716 [USD39,268]; p=0.71). The INMB at CAD100,000(USD73,104)/LYG was CAD2,646 (USD1,934) (95% CI: −CAD30,044 [−USD21,963], CAD43,416 [USD31,739]; p=0.89), with a 52.8 percent probability of being cost effective.

There were clinically relevant but uncertain differences in improved survival associated with multigene panel sequencing for advanced melanoma, and the cost-effectiveness of panel-based testing was finely balanced. This real-world evidence generated using randomized trial design principles can support jurisdictions’ deliberations on the reimbursement of precision oncology interventions.

To synthesize the results of cost-effectiveness studies of different triage tests in comparison to repeat cytology for women with atypical squamous cells of undetermined significance (ASC-US) or low-grade squamous intraepithelial lesions (LSIL) results.

Electronic databases (Medline/PubMed, Lilacs, Embase, The Cochrane Library, Scopus, Web of Science, Scielo, The NHS Economic Evaluation Database, Econlit, and CEA Registry) were searched for cost-effectiveness or cost-utility publications. Per the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines, two independent reviewers selected eligible publications based on the selection criteria and performed data extraction. Methodological quality was assessed using the Quality of Health Economic Studies tool.

Five cost-effectiveness analyses were included comparing HPV testing, immediate colposcopy, and liquid-based cytology with HPV testing reflex to repeat cytology. The main outcome adopted was cervical intraepithelial neoplasia level 2 or higher (CIN2+) cases detected. In pairwise comparisons, HPV testing was more frequently observed as the most cost-effective strategy. Incremental cost-effectiveness ratios were very sensitive to costs of test kit variation and accuracy estimates with some sensitivity analysis scenarios showing immediate colposcopy more cost-effective than HPV testing depending on the tests’ unitary costs and effectiveness.

This systematic review of economic evidence corroborates clinical evidence showing cytology is the least effective, although less costly, triage strategy. Cytology-based triage programs need to be updated to offer timely treatment to women diagnosed with ASC-US/LSIL and better resource allocation.

Allocating financial resources efficiently poses a significant challenge for health systems worldwide. In this context, economic evaluations (EE) are pivotal for the health technology assessment (HTA) process, particularly in standardizing approaches to enhance decision-making accuracy. This study focuses on delineating methodological nuances of EE submissions within Brazil’s Unified Health System (SUS).

This meta-epidemiological study analyzed dossiers submitted to the National Committee for Health Technology Incorporation (Conitec) for drug reimbursement from January 2022 to May 2023. We selected dossiers that included complete EE studies such as cost–utility or cost-effectiveness analyses. Our evaluation involved extracting data on the characteristics of the studies, reimbursement decisions, elements of the base case scenario, and parameters used in utility and sensitivity analyses. [Protocol: DOI 10.17605/OSF.IO/JVYEC]

We included 56 dossiers, leading to 11 favorable reimbursement decisions. EE study methods were cost-effectiveness (17) and cost–utility analyses (17), with some employing both (16). Markov chain models were used in 27 dossiers, primarily utilizing quality-adjusted life years as the health outcome measure. Additional outcomes included life years gained and frequency of avoided events. While utility assessment was reported in 33 dossiers, only six adjusted for age and seven accounted for disutility from adverse events. Thirty-two conducted deterministic or probabilistic sensitivity analyses, but only four presented parameters like credibility intervals, and 17 presented acceptability curves.

This study highlights the need for more standardized and refined methods in the EE submitted to Conitec. The existing EE methodological guidelines, dating from 2014, are currently under revision. This update is crucial to integrate recent advancements in health technology assessment and to better address contemporary requirements, particularly in light of the newly defined willingness-to-pay threshold.

International development agendas increasingly push for access to healthcare for all through universal healthcare coverage. Health economic evaluations and health technology assessment (HTA) could provide evidence to support this but do not routinely incorporate consideration of equitable access.

We undertook an international scoping review of health economic evaluation and HTA guidelines to examine how well issues of healthcare access and equity are represented, evidence recommendations, and gaps in current guidance to support evidence generation in this area. Guidelines were sourced from guideline repositories and websites of international agencies and organizations providing best practice methods guidance. Articles providing methods guidance for the conduct of HTA, or health economic evaluation, were included, except where they were not available in English and a suitable translation could not be obtained.

The search yielded forty-seven national, four international, and nine independent guidelines, along with eighty-six articles providing specific methods guidance. The inclusion of equity and access considerations in current guidance is extremely limited. Where they do feature, detail on specific methods for providing evidence on these issues is sparse.

Economic evaluation could be a valuable tool to provide evidence for the best healthcare strategies that not only maximize health but also ensure equitable access to care for all. Such evidence would be invaluable in supporting progress towards universal healthcare coverage. Clear guidance is required to ensure evaluations provide evidence on the best strategies to support equitable access to healthcare, but such guidance rarely exists in current best practice and guidance documents.

Advances in next-generation sequencing/comprehensive genomic profiling (NGS/CGP) diagnostics require a value framework (VF) for accurate assessment within cancer care in Europe. Building on a previously established VF for diagnostic technologies in Latin America by the Institute for Clinical Effectiveness and Health Policy (IECS), this study synthesizes insights from a systematic literature review and stakeholder perspectives to inform the co-creation of an NGS/CGP diagnostic framework for healthcare decision-making.

The study utilized a mixed methods approach including a systematic literature review (SLR) and web-Delphi panel. The SLR identified and mapped existing VFs against the IECS VF, extracting non-overlapping criteria relevant to NGS/CGP in Europe. A Delphi panel further adapted and validated the framework, ensuring comprehensive stakeholder contribution and engagement. The Delphi´s first round was qualitative and involved open-ended feedback from participants to adapt the indicators and domains. Rounds two through four involved Likert-scale judgments of importance of each indicator. In rounds three and four, participants were shown the distribution of responses across stakeholders and could reconsider their answers.

The SLR revealed 42 VFs with an 83 percent criterion overlap with the IECS VF, resulting in 46 indicators forming the literature-adapted framework. Thirty-four participants completed the Delphi. In round one, 14 indicators and 22 descriptions were adapted, 11 indicators were merged, 14 were deleted, and one was kept the same resulting in 29 indicators for scoring in round two. The final VF has 27 indicators: 23 essential and four complementary; two indicators did not reach consensus. The domains included clinical impact, test performance, scientific evidence quality, non-clinical impact, health system integration, economic aspects, ethical/governance concerns, and health system priorities.

This approach has yielded a robust, stakeholder co-created VF for NGS/CGP diagnostics in oncology, tailored to the European setting. It offers a comprehensive set of criteria that extends beyond traditional health technology assessments, incorporating novel aspects like post-test data governance. This framework sets the stage for improving patient access to high-value technologies by aligning European stakeholder values across health systems.

The roles and potential value of patient preference (PP) data in health technology assessment (HTA) remain to be fully realized despite an expanding literature and various efforts to establish their utility. This article reports lessons learned through a series of collaborative workshops with HTA representatives, organized by the Health Technology Assessment International’s Patient Preferences Project Subcommittee.

Five online workshops were conducted between June 2022 and June 2023, seeking to facilitate collaborative learning and reflection on ways that PP data can be integrated into HTA. Participants included nine HTA representatives from the United States, Canada, Australia, England, and the Netherlands. Workshops were recorded, transcribed, and thematically analyzed.

Despite appreciating the value of PP data, participants were ambivalent about their use in HTA. Some felt that they were already getting the information they needed from the cost-effectiveness analysis or existing patient involvement processes. Others thought that PP data would be very helpful at the initial and final stage of the decision-making process and, particularly, in the following cases: (a) when technology has important non-health benefits; (b) when the clinical and/or cost-effectiveness evidence is marginal; and (c) when treatment is indicated for a large and heterogeneous population. Issues related to the validity and reliability of PP studies were frequently raised, with preference heterogeneity at the core of these concerns.

Collaborating with HTA representatives in the “co-creation” of PP research can help address their concerns and facilitate mutual learning about how PP data can be used in HTA.