The aim of this policy article is twofold: (i) to provide a summary and update of recent important policy developments, in particular relevant guidance on the use of real-world data/real-world evidence (RWD/RWE) by health technology assessment (HTA) bodies and (ii) to set out our policy recommendations on how the different elements of an “RWE framework” we have previously developed could support, further enhance and facilitate the use of RWE for HTA purposes and by HTA bodies and payers.

We undertook a targeted review and analysis of recent important policy developments. The aim was to build on our recommendations from previous work on the “RWE Framework,” and consider how the relevant tools from our Framework can further enhance and facilitate the use of RWE for HTA purposes and by HTA bodies/payers.

We provide eight conditions that we argue would, in combination, constitute the optimal use and acceptance of RWD/RWE for HTA. We believe that, should the eight conditions hold, RWD/RWE would enable more efficient access to medicines and healthcare technologies for patients.

High-quality, fit-for-purpose RWD/RWE can and should be used more frequently in HTA. Multi-stakeholder and cross-geography collaborative partnerships are needed to align on best practices to optimize the evidence that needs to be generated to satisfy all stakeholders’ needs.

Hospital-Based Health Technology Assessment (HB-HTA) is a heterogeneous phenomenon constantly evolving to respond to the needs of decision-makers at the hospital level. In 2023, The HB-HTA Interest Group of Health Technology Assessment International (HTAi) surveyed HB-HTA activities with the aim to provide an updated description of the actual scenario.

An online survey was conducted to gather data on the main characteristics of hospitals, HB-HTA activities, outputs, role in the decision-making processes, dissemination and training activities, and their interaction and collaboration with other stakeholders and HTA-related regulations. Finally, the survey collected feedback on the perception of and current barriers to HB-HTA. Three categories of responders were identified: Both hospitals performing and not performing HTA and policymakers.

Eighty-seven responses were collected from twenty-eight countries. Nearly half of the responders (n = 41) conducted HB-HTA, whereas eighteen consisted of hospitals not performing HTA, and twenty-eight were policy makers. HB-HTA was performed mainly in hospitals with >500 beds. HB-HTA units were organized in 40 percent of cases as an “independent group.” The survey showed that HTA units could contribute to all the steps of the decision-making processes, whereas the impact of the assessments on the decisions was mainly perceived as a medium. Furthermore, HB-HTA was not seen as a duplication of effort, even without specific regulations.

The survey highlighted the role of HB-HTA in hospital decision-making supporting the vision of HB-HTA as one of the actors in the HTA ecosystem, the success of which depends on collaboration with other stakeholders.

Cost-effectiveness models fully informed by real-world epidemiological parameters yield the best results, but they are costly to obtain. Model calibration using real-world data/evidence (RWD/E) on routine health indicators can provide an alternative to improve the validity and acceptability of the results. We calibrated the transition probabilities of the reference chemotherapy treatment using RWE on patient overall survival (OS) to model the survival benefit of adjuvant trastuzumab in Indonesia.

A Markov model comprising four health states was initially parameterized using the reference-treatment transition probabilities, obtained from published international evidence. We then calibrated these probabilities, targeting a 2-year OS of 86.11 percent from the RWE sourced from hospital registries. We compared projected OS duration and life-years gained (LYG) before and after calibration for the Nelder–Mead, Bound Optimization BY Quadratic Approximation, and generalized reduced gradient (GRG) nonlinear optimization methods.

The pre-calibrated transition probabilities overestimated the 2-year OS (92.25 percent). GRG nonlinear performed best and had the smallest difference with the RWD/E OS. After calibration, the projected OS duration was significantly lower than their pre-calibrated estimates across all optimization methods for both standard chemotherapy (~7.50 vs. 11.00 years) and adjuvant trastuzumab (~9.50 vs. 12.94 years). LYG measures were, however, similar (~2 years) for the pre-calibrated and calibrated models.

RWD/E calibration resulted in realistically lower survival estimates. Despite the little difference in LYG, calibration is useful to adapt external evidence commonly used to derive transition probabilities to the policy context, thereby enhancing the validity and acceptability of the modeling results.

The methods of economic evaluation and HTA should be based on best practices and standards, tailored to unique country contexts that can be systematically applied to inform decisions. This paper outlines standards for the conduct of economic evaluations for HTA in Ghana.

A five-step process was followed to develop the HTA reference case as a methodological and reporting benchmark. These include (a) a review of literature and evidence synthesis, (b) a review of country policies, (c) a review and adaption of international frameworks, (d) expert/stakeholder consultations, and (e) the development of a methodological framework. A series of stakeholder consultations were done to refine, finalize, and validate the outcomes of the processes to generate a finalized reference case.

The Ghana reference case is made up of 14 components comprising: evidence synthesis, evaluation type, perspectives on cost, perspectives of outcomes, choice of comparator, data sources, outcome measures, discount rate, uncertainty, equity considerations, time horizon, heterogeneity, transparency, and budget impact. These provide methodological considerations and reporting requirements for economic evaluations for HTA. It provides a framework to ensure the best research methods are adopted to harmonize the evidence-generation process with the expectations of policy and decision-makers and ensure that policy decisions are based on uniform evidence.

Recommendations set out in this reference case when followed can provide context-specific evidence to support a rigorous and transparent system for evaluating healthcare interventions and technologies. It will support decision-making, ultimately improving the quality and efficiency of healthcare delivery in the country.

Though Health Technology Assessment (HTA) has steadily grown over the past decades, less attention has been paid to the way HTA may prove more responsive to the broader economic, social, and environmental challenges that health systems are facing today. In view of climate change, chronic diseases, an aging population, inequalities, and workforce issues, the HTA community’s unique set of skills nonetheless holds great potential to help decision-makers strengthen many publicly funded health systems around the world.

This article adopts an integrated system-wide perspective guided by the Responsible Innovation in Health (RIH) framework to explore how the HTA community may not only adapt to the speed of innovation but also consider its direction.

Because RIH aims to steer innovation toward a more sustainable pathway, it can help HTA agencies anticipate decision-makers’ informational needs regarding four systemic challenges: (1) equitable access; (2) workforce issues; (3) accountable policy trade-offs; and (4) environmental sustainability. We clarify how key elements of the RIH framework may be used by HTA agencies to: (1) supplement their evaluation process; (2) align their priority-setting or strategic planning activities with their health system challenges; or (3) inform the production of early HTAs, horizon scans, or reports that are broader in scope than a single technology review.

The article concludes with three practical implications that were identified by the Institut National d’Excellence en Santé et Services Sociaux (INESSS) (Québec, Canada) and may inspire other HTA agencies.

The need for more local technical capacity in Health Technology Assessment (HTA) is a leading challenge to its use in low- and middle-income countries. Zambia has been considering using HTA to support its universal health coverage initiative, which includes health benefits package design and implementation. This study assesses the local HTA capacity for the steering committee tasked with supporting the design and implementation of the national health benefits package in Zambia.

The study applied a cross-sectional web-based survey design and the consensus-based Checklist for Reporting of Survey Studies. Data were collected from the steering committee of the benefits package working group, tasked with leading the design process of the health benefits package using the Instrument for the Assessment of Skills to Conduct a Health Technology Assessment tool.

The majority of respondents had not served on a selection and reimbursement committee. Clinical effectiveness skills in structuring a search strategy, handling missing data, conducting qualitative evidence synthesis, and grading the certainty of evidence were low. Skills for leadership, networking, conflict management, and project coordination, public and patient involvement were mid-level to low. Most of the respondents were aware of ethical issues with health technologies. Health economics skills in economic evaluations and decision analytic modeling, equity and health system efficiency measurement, budget impact analysis, and quality of life were identified for capacity strengthening.

Available technical capacities to revise and implement the national benefits package were lower in health economics, synthesis for clinical effectiveness evidence, ethics, patient and public involvement, and soft skills, in that order.

This study comprehensively reviewed reports on patient and public involvement and engagement (PPIE) in health technology assessment (HTA) overseas and identified the status and possible future measures, of PPIE in Japanese HTA.

The series of reviews targeted systematic reviews (SR#1), references in SR#1 (SR#2), and new articles after SR#1 (SR#3). The MEDLINE and Embase databases were searched through August 2024 using the terms “patient involvement/engagement,” “patient participation,” “community participation,” “public involvement/engagement,” and “health technology assessment.” The implementation details were extracted from information published on the websites of the HTA agencies.

Three review articles in SR#1, 12 references in SR#2, and 10 articles in SR#3 were selected. The opportunities for countries, including Japan, to participate in discussions on the HTA process did not differ significantly; however, information on PPIE in Japan was scarce and did not indicate their purpose and value.

Collected articles indicated that the value of PPIE in HTA includes relevance, equity, fairness, legitimacy, and capacity building. The participation of patient and public representatives in Japanese discussions since 2005 appeared to be very limited to consider PPIE in HTA. In countries that implement PPIE in HTA, the value of PPIE is explicit: the process guidelines are specific and provide an appropriate environment for input that includes education, training, and feedback. Future reforms of the Japanese system will require discussions on PPIE purpose and value, implementation, and creating an environment in which a diverse range of patients and the public can easily express their views.

To integrate patient perspectives into Health Technology Assessment (HTA) by exploring the perceived benefits, barriers, and expectations of diabetes patients and their caregivers in Malaysia regarding the use of CGMS.

This qualitative study employed focus group discussions (FGDs) with 30 participants, including adults, adolescents, and caregivers managing insulin-requiring diabetes, conducted between May and September 2023 in Kuala Lumpur and Putrajaya, Malaysia. Participants were recruited through purposive sampling. Data were collected using semi-structured interviews and analyzed thematically to capture insights into CGMS benefits, barriers, and expectations.

Participants highlighted CGMS as a transformative tool, offering real-time data, improving glycemic control, and enhancing quality of life by reducing anxiety and the burden of frequent glucose checks. Despite these benefits, significant barriers were identified, including high costs, limited access, technical issues, and social stigma, particularly among adolescents. There was a strong call for government subsidies, better technical support, and healthcare provider training to optimize CGMS use. Patient perspectives were integrated into the HTA alongside systematic reviews and economic evaluations, directly informing policy recommendations, including prioritizing CGMS for high-risk T1DM patients and exploring subsidy frameworks to improve affordability.

Patient perspectives serve as a vital voice in HTA, offering real-world insights that enhance the understanding of CGMS in diabetes management. Addressing financial, technical, and social barriers is crucial to improving CGMS accessibility and usability. By embedding patient perspectives into HTA and strengthening partnerships across healthcare systems, patient perspectives become instrumental in shaping patient-centered policies and informing equitable decision-making on CGMS utilization in Malaysia.

Surrogate endpoints, used to substitute for and predict final clinical outcomes, are increasingly being used to support submissions to health technology assessment agencies. The increase in the use of surrogate endpoints has been accompanied by literature describing the frameworks and statistical methods to ensure their robust validation. The aim of this review was to assess how surrogate endpoints have recently been used in oncology technology appraisals by the National Institute for Health and Care Excellence (NICE) in England and Wales.

This article identifies technology appraisals in oncology published by NICE between February 2022 and May 2023. Data are extracted on the use and validation of surrogate endpoints including purpose, evidence base, and methods used.

Of the 47 technology appraisals in oncology available for review, 18 (38 percent) utilized surrogate endpoints, with 37 separate surrogate endpoints being discussed. However, the evidence supporting the validity of the surrogate relationship varied significantly across putative surrogate relationships with 11 providing randomized controlled trial evidence, 7 providing evidence from observational studies, 12 based on the clinical opinion, and 7 providing no evidence for the use of surrogate endpoints.

This review supports the assertion that surrogate endpoints are frequently used in oncology technology appraisals in England and Wales and despite the increasing availability of statistical methods and guidance on appropriate validation of surrogate endpoints, this review highlights that use and validation of surrogate endpoints can vary between technology appraisals, which can lead to uncertainty in decision making.

Following acquired brain injury (ABI), individuals often experience anxiety and/or depressive symptoms. BrainACT is an adapted form of Acceptance and Commitment Therapy (ACT) tailored to this target group. The current study is a trial-based health-economic evaluation comparing BrainACT to a psychoeducation and relaxation control treatment.

An economic evaluation from a societal perspective was conducted in the Netherlands alongside a multicenter randomized controlled two-armed parallel trial including 72 participants. A cost-utility and cost-effectiveness analysis was conducted where incremental costs, quality-adjusted life-years (QALYs), and anxiety/depression (Hospital Anxiety and Depression Scale (HADS) score) were collected and presented over a 1-year follow-up period. Bootstrapping, scenario, and subgroup analyses were performed to test the robustness of the results.

The BrainACT arm reported non-significant lower total costs (incremental difference of €−4,881; bootstrap interval €−12,139 to €2,330) combined with significantly decreased anxiety/depression (HADS) (3.2; bootstrap intervals 0.7–5.7). However, the total QALYs were non-significantly lower (−0.008; bootstrap interval −0.060 to 0.042) for BrainACT. The probability of the intervention being cost-effective was 86 percent at a willingness-to-accept threshold of €50,000/QALY. The scenario and subgroup analyses confirmed the robustness of the results.

BrainACT may be a more cost-effective alternative to a psychoeducation and relaxation intervention for anxiety and/or depressive symptoms following ABI. Despite limitations, BrainACT appears to be a promising addition to treatment options in the Netherlands. Further research is needed to validate these findings, and consideration should be given to implementing BrainACT in Dutch clinical settings with ongoing monitoring.

The EUnetHTA Core Model® is well-established in the HTA community. Some recommendations of corresponding guidance documents leave room for alternative methodological choices. Considering the new HTA regulation (HTAR), we aimed to identify needs for concretization (NCs) in EUnetHTA guidance and provide indicative methodological options.

We carried out a qualitative document analysis and structured group discussion. Twenty-two EUnetHTA documents were screened using transparent criteria. Identified NCs were classified into topics according to the PRISMA statement and presented to Austrian HTA practitioners (n = 11) during a structured group discussion. Participants rated NC’s importance. To identify potential solutions, selected key handbooks for generic (Cochrane) and HTA-specific (IQWIG/NICE) evidence synthesis were systematically reviewed and matching content was charted against the NCs.

Thirty-two topics with varying numbers of NCs were identified, twenty-six during the screening process, and six from the group discussion. Most of the topics related to evidence synthesis methods (nine topics), evidence eligibility criteria (nine topics), risk of bias (three topics), and certainty assessment (three topics). Other topics related to information sources, search strategy, data collection process, data items, effect measures, and reporting bias. One or more methodological approaches and recommendations could be identified for each identified topic from the included methodological handbooks.

Our analysis identified a need for concretization in some EUnetHTA guidelines. The structured overview of methodological options may support HTA doers in adapting and applying the guidelines to the national and local practical context.

Left ventricular assist devices (LVAD) are a therapeutic option in patients with advanced heart failure (HF) not a candidate for cardiac transplant as destination therapy (DT). However, important uncertainties remain regarding the use of LVAD in the long-term in real practice settings. When planning registries, it is important to choose the appropriate outcomes that ensure comparability and reduce the possibility of bias.

The purpose of this study was to establish a minimum dataset (MDS) that should be collected in all LVAD for DT registries to meet the needs and demands of Health Technology Assessment (HTA) doers and health professionals.

To design the MDS for LVAD, a preliminary list of outcome domains and data items were developed attending to the gaps and research needs derived from existing evidence coming from HTA carried out at the European Network of Health Technology Assessment (EUnetHTA) level. The list of data items and domains was agreed upon by all involved HTA organizations and a three-round Delphi was conducted among an experienced panel of cardiologists to rate the importance of the items for measuring uncertainty gaps.

After the three-round Delphi process, the expert panel reached a consensus regarding eighteen outcomes and forty-seven variables divided into seven main domains (safety, effectiveness, patient acceptability, satisfaction, healthcare system impact, pharmaceutical management, and technique-related factors).

The MDS of outcomes and measures, developed based on research gaps and needs, can allow for standardizing data collection and improving the quality of data for decision making and practice.

The aim of this study is to propose and validate a value assessment framework for Health Technology Assessment (HTA) for rare diseases drugs in Brazil.

A scoping review was performed to identify criteria used by HTA agencies in countries with public healthcare systems when evaluating orphan drugs. Based on the findings, a criteria framework for rare disease drugs was proposed for Brazil. Content validity was conducted over three rounds using Delphi technique and content validity ratio (CVR) approach was employed to evaluate the ratings from the eighteen stakeholders (experts and patients).

Twenty-nine HTA criteria for rare disease drugs were identified to compose the Brazilian framework. After three Delphi rounds, the final value framework comprised fifteen criteria categorized into four domains: disease-related factors, treatment-related factors, social and political factors, and economic factors. Among the most well-rated criteria by the CVR, considering the relevance attribute, were “relevance of outcomes for a rare disease,” “impact on patient’s quality of life,” “price negotiation,” and “adjusted cost-effectiveness threshold.” On the other hand, “budget impact threshold,” “innovative nature of treatment,” and “willingness to accept greater uncertainty in clinical evidence” received negative evaluations and were excluded from the final framework.

A value assessment framework validated by key stakeholders of rare diseases in Brazil could contribute to improve HTA transparency, decision making, and efficiency of the healthcare system, and inspire the development of a local guidance for rare-disease HTA.

The assessment of technology in hospital settings is a crucial step towards ensuring the delivery of efficient, effective, and safe healthcare.

This study conducts a Hospital-Based Health Technology Assessment to evaluate the efficacy of a screening rapid test for mild Traumatic Brain Injury (mild TBI) utilizing blood biomarkers, specifically Glial Fibrillary Acidic Protein (GFAP) and Ubiquitin C-terminal Hydrolase L1 (UCH-L1). The assessment focuses on the clinical utility and performance characteristics of the proposed rapid test within a hospital setting.

The screening model was meticulously examined for its ability to accurately detect mild TBI, considering the sensitivity and specificity of GFAP and UCH-L1 as blood biomarkers. The study involved a thorough evaluation of the test’s diagnostic accuracy, comparing its outcomes with established standards for mild TBI diagnosis.

Results from the Hospital-Based Health Technology Assessment highlight the potential of the GFAP and UCH-L1 blood biomarker-based rapid test as an efficient screening tool for mild TBI within a hospital environment. The evidence results show that the test is highly sensitive (91 percent to 100 percent) for the prediction of acute traumatic intracranial lesions, which helps rule out injury when the result is negative. When used within 12 hours of injury in adult patients with mild TBI, this test holds promise in reducing the utilization of CT.

The findings contribute valuable insights into the feasibility and reliability of implementing this technology for timely and accurate identification of mild TBI, enhancing clinical decision making and patient care in hospital settings.