This study assessed the budget impact (BI) of introducing noninvasive perinatal testing (NIPT) to the Korean National Health Insurance (KNHI). It aims to provide useful economic evidence on reimbursement decisions on prenatal screening. Since NIPT is widely used in Korea as a noncovered service, BI can be reliably estimated from real-world data (RWD).

In this BI analysis, we derived the total number of pregnant women from 2017 birth statistics (n=358,000) and its distribution of singleton births (96.1%) and multiples (3.9%). The age distribution of pregnant women was 29.4 percent for 35 and above, and 70.6 percent for below 35, according to the 2017 statistics. For NIPT costs, we considered a price range of KRW357,000 (USD260) to KRW715,000 (USD522) from a 2018 survey. To evaluate the BI of introducing NIPT, we examined eight scenarios based on NIPT cost sharing of 30 percent and 70 percent, NIPT price, and whether to include pregnant women aged less than 35 years old.

BI for KNHI was estimated as KRW6 trillion (USD4 billion) to approximately KRW56 trillion (USD40 billion). Scenario seven, targeting older pregnant women with serum screening high risk with NIPT price at the lower end and payer coverage of 30 percent, shows the lowest annual BI of KRW6,115,813,866 (USD4,469,516); scenario two, covering all older pregnant women and younger high-risk cases with NIPT price at the higher end and payer coverage of 70 percent, exhibits the highest burden at KRW56,240,636,611 (USD41,091,993).

Our BI analysis of introducing NIPT to KNHI can serve as essential data for estimating insurer burdens during the potential transition of NIPT to provisional or selective coverage. In the absence of prenatal diagnostics cost-effectiveness research in Korea, our findings provide crucial evidence for establishing relevant reimbursement criteria, addressing a gap in current research and supporting evidence-based policymaking.

The majority of those infected with chronic hepatitis C (CHC) are asymptomatic. Population screening has proven to be both effective and cost effective. When considering whether to implement screening or not, the uncertainty of the budget impact plays an important role. This study aims to develop methods that improve the accuracy of budget impact analysis for a one-time CHC screening program.

We developed a back-calculation mathematical model that employs a Markov chain Monte Carlo algorithm to estimate the prevalence and proportion of undiagnosed CHC. Subsequently, we utilized a state-transition model to assess the budget impact of two strategies: (i) no screening; and (ii) screen-and-treat with direct-acting antiviral (DAA) for individuals born between 1945 and 1965 (“baby-boomer” birth cohort). Model data were gathered from published literature. Our analysis adopted a Canadian provincial payer perspective, employed a 10-year time horizon, and followed best-practice recommendations by not applying discounting.

For individuals born between 1945 and 1965, the estimated prevalence of CHC was 1.74 percent (95% confidence interval [CI]: 1.52, 2.30) with an undiagnosed proportion of 15.72 percent (95% CI: 11.27, 18.54). The initial budget impact analysis indicated an additional cost of CAD61.5 million (USD45.0 million) over 10 years for screening related individuals for CHC in Ontario. With these updated prevalence and undiagnosed proportion estimates, our projection suggests a 29.6 percent reduction in the budget impact, now estimated at CAD43.3 million (USD31.7 million).

By comparing the budget impact of the CHC screening strategy with other recommended health services and technologies in Ontario, we have concluded that CHC screening may be considered affordable. To enhance the accuracy of budget impact analysis for population-level screening decision-making, it is crucial to develop precise methodologies for estimating the underlying prevalence and undiagnosed proportions.

The decision-making (DM) process in public administration is the subject of research from different perspectives and disciplines. Evidence-based policies, such as health technology assessment (HTA), are not the only support on which public policies are designed. During the COVID-19 pandemic WHO, national and subnational institutions developed HTA reports to guide DM. Despite this, inadequate variability was observed in the health technologies recommended and reimbursed by different provincial Health Ministries in a federally organized developing country like Argentina. The processes and results of DM on health technologies for COVID-19 in Health Ministries of Argentina were inquired.

A retrospective research design was developed, with triangulation of quantitative and qualitative methods. We retrieved information for the years 2020–2021 through document review of official webpages, surveys, and interviews with decision-makers of the 25 Argentinian Ministries of Health. We analyzed the recommendations and reimbursement policies of seven health technologies.

In contradiction with WHO’s policies, ivermectine, inhaled ibuprofen, convalescent plasma and equine serum were widely recommended by most of Argentina’s health ministries outside a clinical trial context, with risks for patients and a huge opportunity cost.

Despite an important HTA institutional capacity, the impact of HTA organizations and their technical reports was limited. Health Ministries with institutionalized HTA units had more adherence to WHO recommendations, but the influence of different technical and political criteria was identified. Power relations within and outside the administration, the pharmaceutical industry and academics, the media, social pressure, the judicial and legislative powers, and the political context strongly influenced DM.

In Italy, there is a lack of evidence regarding the care and management of patients with dementia, as well as the associated costs. This study aims to fill this informational gap by utilizing data from both literature and national surveys.

A prevalence-based cost-of-illness (COI) model was developed to assess dementia-related costs from a societal perspective. The resources utilization for management and treatment of patients with dementia was derived from both the literature and the analysis of surveys conducted by the National Institute of Health on the social-health structures dedicated to dementia. Indirect costs from informal caregiving were evaluated through a human capital approach. Additionally, a cost–consequence analysis (CCA) was conducted to assess the economic impact of healthcare resource utilization changes.

Based on an estimated 1,150,691 dementia cases in Italy, with approximately 12 percent institutionalized, the COI model estimated an annual expenditure of around EUR23.6 billion (USD25.7 billion) for dementia patient management, with 63 percent attributed to out-of-pocket expenses. CCA indicated that if all affiliated with Centers for Cognitive Disorders and Dementia (CDCD) received nonpharmacological interventions (versus the surveyed 25.5 percent), there would be a direct cost increase of approximately EUR4.3 million (USD4.7 million).

This analysis provides an updated overview of current dementia patient management in Italy, offering valuable insights for decision-makers to prioritize health policies and interventions for patients and their caregivers.

Dengue, a mosquito-borne disease, is prevalent in Brazil, ranking third in the Americas. The country has confirmed all four serotypes (DENV-1, DENV-2, DENV-3, and DENV-4) of the virus. In 2023, a tetravalent vaccine, approved for individuals aged four to 60 years, opens the possibility of integration into the public health system.

The invested amount for purchasing two doses of the vaccine to immunize the entire eligible population in the year 2023 was calculated. A comprehensive analysis of direct and indirect costs was conducted regarding the integration of the tetravalent dengue virus vaccine into Brazil’s public healthcare system. Direct costs encompassed treatment expenses for mild, moderate, and severe cases, while indirect costs involved workdays lost and mortality-related expenses. Direct and indirect costs were compared in two scenarios (with and without vaccination), followed by a cost–benefit ratio calculation.

The investment for procuring vaccines for over 168 million Brazilians amounted to approximately EUR17.9 billion, resulting in savings of about EUR193.5 million. Indirect costs were particularly significant when compared to the non-vaccinated population. Considering herd immunity and reducing the vaccinated population to 70 percent of the eligible populace, the invested amount was approximately EUR12.5 billion, while savings reached EUR214.2 million. A cost-benefit ratio calculation revealed a return of one centavo (EUR0.0019) for every BRL1.00 invested (EUR0.0019), and considering herd immunity, the cost–benefit ratio was approximately 0.02.

Despite Brazil being one of the countries with the highest prevalence of dengue in the Americas, the availability of the tetravalent dengue virus vaccine in the country’s public system does not seem to be a sustainable option, given the unfavorable cost–benefit analysis for such implementation. Nevertheless, it is imperative to conduct this analysis with due consideration for alternative scenarios.

The sandbox approach, developed in the financial technologies sector, creates an environment to collaboratively develop and test innovative new products, methods and regulatory approaches, separated from business as usual. It has been used in health care to encourage innovation in response to emerging challenges, but, until recently, has not been used in health technology assessment (HTA). This article summarizes our learnings from using the sandbox approach to address three challenges facing HTA organizations and to identify implications for the use of this approach in HTA.

We identified three challenging contemporary HTA-related topics to explore in a sandbox environment, away from the pressures and interests of “live” assessments. We convened a pool of 120 stakeholders and experts to participate in various sandbox activities and ultimately co-develop solutions to help HTA organizations respond to the identified challenges.

Important general learnings about the potential benefits and implementation of a sandbox approach in HTA were identified. Consequently, we developed recommendations to guide its use, including how to implement an HTA sandbox in an effective way and the types of challenges for which it may be best suited.

For many HTA organizations, it is difficult to carefully consider emerging challenges and innovate their processes due to risks associated with decision errors and resource limitations. The sandbox approach could reduce these barriers. The potential benefits of addressing HTA challenges in a collaborative “safe space” are considerable.

Methods and processes for health technology assessment (HTA) must be sufficiently flexible to reflect emerging best practice and adapt to changes in the health and care landscape. They must also be clearly documented and subject to review, assessment, and consultation. This requires a framework to flexibly update HTA manuals, while providing stability and predictability for stakeholders.

The National Institute for Health and Care Excellence (NICE) is introducing a modular approach to updating its manuals. Previously, NICE completed full, but infrequent, updates to its manuals. A modular update is a review of the process and/or methods informing NICE’s guidance in a specific subject area, which can then be updated in the appropriate manual(s) if required. We are developing a modular updates framework that sets out NICE’s approach for identifying and prioritizing modular updates to review, reviewing the evidence and proposing changes to the manual(s), engaging and consulting with stakeholders, and implementing the modular updates.

The proposed approach allows external stakeholders to contribute to topic identification and the content of updates, helping to ensure that the manuals meet the needs of users. The first modular update to the NICE manual for health technology evaluations was published in October 2023. This included updated processes for carrying out cost comparisons, streamlining committee decision-making and handling confidential information. Potential future modular updates may include updated methods to consider health inequalities.

The introduction of a modular approach will enable NICE to be more agile and responsive in monitoring, reviewing, and improving our methods and processes, making sure they remain cutting edge as the health and care landscape continues to evolve.

The costs of low back pain (LBP) are complex and difficult to estimate. This study aims to adapt the Cost for Patients Questionnaire (CoPaQ) for use in LBP populations.

In a cross-sectional qualitative study, we conducted cognitive interviews to assess the CoPaQ’s suitability for addressing costs related to LBP. Three groups of participants were included (n = 5 each): (i) persons with a history of LBP or primary caregiver, (ii) researchers with expertise in LBP, and (iii) primary care providers specialized in treating LBP. The interpretation, analysis, and summary of results used Knafl et al.’s qualitative content analysis method.

Persons with a history of LBP (n = 5), had a median age of 60 years (Interquartile Range (IQR): 26–71.5), and varying durations of LBP, the median duration of LBP 7 years (IQR: 4–32.5). Researchers (n = 5) had a median age of 33 years (IQR: 29–45). Primary care providers (n = 5) had a median age of 40 years (IQR: 37.5–65), and a background in chiropractic care (n = 3) and physiotherapy (n = 2). Content analysis of the interviews revealed sources of error with five pre-determined themes (clarity/comprehension, relevance, inadequate response definition, reference point, perspective modifiers) and one developed theme (organization). We modified the questionnaire for LBP populations based on the feedback.

Our study evaluated the content validity of a questionnaire that assesses the direct and indirect costs associated with LBP. Future studies should pilot this questionnaire with persons of varying LBP severity and compare it with cost diaries.

Experts emphasize the necessity of reconsidering the approaches used in the health technology assessment (HTA) of digital health technologies (DHTs) to adequately address their particular characteristics. In this context, the Agency for Health Quality and Assessment of Catalonia was commissioned to develop a specific framework for the assessment of DHTs in collaboration with the agencies that constitute the Spanish Network of Agencies for Assessing National Health System Technologies and Performance (RedETS).

To accomplish the assignment, a combination of methodologies was used. To establish the domains and dimensions, an international survey, a scoping review of the literature, and a thematic analysis were executed. To determine the evidence standards that DHTs must achieve according to the risk classification derived from their use, the updated version of the evidence standards framework (ESF) from the National Institute for Health and Care Excellence (NICE) was adapted to the Spanish context. National experts in the fields of HTA and DHTs collaborated on different tasks to develop the framework.

The framework encompasses three distinct sections. The first section provides an introduction, detailing a DHT classification system that is based on the system proposed by NICE in their ESF. Following this, Section A elaborates on a comprehensive set of 13 domains, 41 dimensions, and nine sub-dimensions, offering a detailed description of each. Section B consists of the adaptation of the 21 evidence standards from the NICE ESF to the Spanish context. Of note, a clear link was established between the classification system, the domains, dimensions, sub-dimensions, and the evidence standards.

The resulting framework was published in November 2023. Since its publication, it has become the reference framework for the HTA of DHTs within the Spanish National Health System, primarily by the HTA agencies that constitute RedETS. The document will be periodically reviewed and, if necessary, adapted to align with emerging technologies and changes in legislation.

We draw from the Health Technology Assessment (HTA) literature to propose how hospitals and local health networks can prepare the key components of early economic evaluations to support the development and management of health service interventions.

Using the case example of a proposed intervention for older people in the Emergency Department (ED), a conceptual logic model of a new health service intervention is articulated to inform the structuring and population of a decision-analytic model using observed data on the existing care comparator and structured elicitation exercise of initial stakeholder expectations of intervention effects.

The elicited patient pathway probabilities and lengths of stay quantities profile which of the existing types of patients are expected to avoid the ED and how this impacts the lengths of stay across the system. The exercise also quantifies the stakeholders’ uncertainty and disagreement, with qualitative insights into why. The elicitation exercise participants draw upon the rationale for how the intervention is expected to affect a change within the local context, as captured within the logic model, together with the descriptive analyses of the characteristics and utilization of their target population. Feedback indicates the methods are acceptably robust yet pragmatic enough for healthcare delivery settings.

As proposed in this paper, HTA methods can be used to capture how key stakeholders initially expect a service intervention to affect a change within their local context. The example results can be used in a decision-analytic model to guide the development and management of an intervention.

Public healthcare institutions (PHIs) experience rising and discordant implant costs. Implementing subsidies for implants without concurrent price harmonization through national procurement contracts impedes equitable patient access. This abstract describes the collaboration between the Agency for Care Effectiveness (ACE), Singapore’s national health technology assessment (HTA) agency, and the public healthcare supply chain agency (ALPS) to establish the Implant Subsidy List (ISL).

The alignment between ISL and concurrent national procurement contracts for implants required close coordination and co-development of streamlined and scalable processes. To achieve this, ACE and ALPS collaborated to understand the complex implant landscape by leveraging on known vendors and products and pre-empting upcoming ones, identifying key opinion leaders, and sharing technical expertise. Both agencies also developed common-use templates and streamlined vendor submission processes. Of note, joint request-for-proposal (RFP) exercises for national subsidy and procurement listings combined negotiation efforts to achieve lower implant prices. Regular cross-agency discussions enabled continuous knowledge sharing and process refinement in an evolving implants landscape.

For phase one rollout of ISL, 15 implant topics across eight clinical functions were evaluated, supported by concurrent establishment of 15 national procurement contracts. As of December 2023, ACE listed 22,689 ISL implants for implementation, with more topics onboarded on a rolling basis. ACE and ALPS streamlined process flows, standardized communications channels and information, and utilized contractual tools. Vendors could submit for both subsidy and procurement through the secure ALPS portal, reducing duplication of work and errors while allowing procurement and subsidy consideration processes to proceed concurrently. Combined negotiation efforts have resulted in an average price reduction of 19 percent.

The alignment between national HTA and procurement agencies is important for successful scaling. The ACE–ALPS collaboration has synergized both agencies’ understanding of the complex implant landscape and established streamlined workplans and processes to support initial establishment of ISL and its maintenance. Challenges include navigating strict procurement rules and continuous cross-agency improvement efforts to ensure applicability in an evolving landscape.

The EU HTA member state coordination group has finalized methodological guidance on indirect comparisons that states that propensity score (PS) methods should generally be used for indirect comparisons of non-randomized data in joint clinical assessments (JCAs). Half of new oncology approvals by the European Medicines Agency (EMA) between 2020 and 2023 were based on non-randomized data. This study aimed to identify how many of these were able to submit PS-based comparisons.

Using IQVIA’s Market Access Insights (MAI) database of HTAs and regulatory approvals, we characterized evidence packages submitted to EMA and HTA agencies of EU member states according to the use of PS-based comparisons and access to individual patient data (IPD) from comparator studies.

Of the 56 oncology approvals between 2020 and 2023, 30 (54%) were based on non-randomized data, of which 23 (23%) submitted PS-based indirect comparisons to EMA (15 therapies) or to HTA agencies (23 therapies). Electronic health record (EHR) or chart reviews were the most common source of comparative RWE, but agencies only took this evidence into account in fewer than half of HTAs where it was available. Use of PS-based methods also did not lead to more positive HTA outcomes than the alternative unanchored matching-adjusted indirect comparisons (MAICs) to aggregated data.

The prevalence of oncology approvals based on single-arm trials is expected to be a key challenge to the success of JCA. Unanchored comparisons will be required, but IPD was not necessarily shown to reduce uncertainty in HTAs analyzed in this research, and in about half of cases, comparisons to aggregate data were preferred due to applicability and heterogeneity concerns. Thus, the source of comparator data appears more relevant than the comparison method in HTAs, which contrasts with the available EU HTA coordination group guidance that focuses mainly on methodological aspects.

The Health Technology Assessment International (HTAi) 2023 Annual Meeting included a novel “fishbowl” session intended to 1) probe the role of HTA in the emergence of generative pretrained transformer (GPT) large language models (LLMs) into health care and 2) demonstrate the semistructured, interactive fishbowl process applied to an emerging “hot topic” by diverse international participants.

The fishbowl process is a format for conducting medium-to-large group discussions. Participants are separated into an inner group and an outer group on the periphery. The inner group responds to a set of questions, whereas the outer group listens actively. During the session, participants voluntarily enter and leave the inner group. The questions for this fishbowl were: What are current and potential future applications of GPT LLMs in health care? How can HTA assess intended and unintended impacts of GPT LLM applications in health care? How might GPT be used to improve HTA methodology?

Participants offered approximately sixty responses across the three questions. Among the prominent themes were: improving operational efficiency, terminology and language, training and education, evidence synthesis, detecting and minimizing biases, stakeholder engagement, and recognizing and accounting for ethical, legal, and social implications.

The interactive fishbowl format enabled the sharing of real-time input on how GPT LLMs and related disruptive technologies will influence what technologies will be assessed, how they will be assessed, and how they might be used to improve HTA. It offers novel perspectives from the HTA community and aligns with certain aspects of ongoing HTA and evidence framework development.

Involving the public is essential to building trust in health technology assessment (HTA) organizations. The National Institute for Health and Care Excellence (NICE) runs a deliberative public engagement program, NICE Listens. It was used to explore informed public opinion on how environmental sustainability should be taken into account in HTA.

Twenty-three general public participants from across England took part in three iterative online workshops (each lasting two or three hours, held three weeks apart in 2022). The workshops included trade-off exercises, role-play, group discussion, and video clips from interviews with sustainable healthcare experts.

Strong support was found for NICE taking action to make healthcare more environmentally sustainable. Support increased as participants learned that sustainable healthcare offers co-benefits, such as reduced burden on the National Health Service through better self-management of conditions. Participants did not want health outcomes to be compromised in pursuit of sustainability. We identified some circumstances where they found it acceptable to consider the environmental impact of interventions in decision-making: when effective treatments already exist; when the condition is not severe; when the alternative is equally cost effective; and when greener options are marginally higher in cost but as clinically effective as the alternative.

The findings demonstrate that environmental sustainability is clearly considered a relevant element of value. They also offer insight into how the environmental impact of health interventions should be considered in HTA. Further research should focus on methods for consistent measurement of the environmental impacts of health interventions and the incorporation of those impacts into decision-making.

A conceptual framework, called Innovation of Health Technology Assessment Methods (IHTAM), has been developed to facilitate the understanding of how to innovate methods of health technology assessment (HTA). However, the framework applicability has not been evaluated in practice. Hence, we aimed to explore framework applicability in three cases of method innovation that are part of the HTx project and to develop a roadmap to improve framework applicability.

The IHTAM framework was applied to three cases of innovating HTA methods. We collected feedback from case study leaders and consortium members after a training session, an approximately 1-year follow-up of periodic case study meetings, and a general assembly meeting where innovation progresses of the three cases were reported through surveys and interviews. Feedback was then summarized using an open-coding technique.

According to feedback, the framework provided a structured way of deliberation and helped to improve collaboration among HTA stakeholders. However, framework applicability could be improved if it was complemented by a roadmap with a loop structure to provide tailored guidance for different cases, and with items to elaborate actions to be taken by stakeholders. Accordingly, a 48-item roadmap was developed.

The IHTAM framework was generally applicable to the three case studies. A roadmap, with loop structure and actionable items, could complement the framework, and may provide HTA stakeholders with tailored guidance on developing new methods. To further examine the framework applicability, we recommend stakeholders to apply the IHTAM framework and its roadmap in future practice.

This study aimed to map strategies for educating laypeople about health technology assessment (HTA). Although integrating community is challenging, the engagement of patients/public in the processes of HTA has garnered support and endorsement from international network agencies. Dissemination of information, educational empowerment, and training are vital to give individuals capacity to partake in the intricate web of processes actively.

This review considered studies addressing educative strategies to train laypeople on HTA, additionally mapping and summarizing relevant methodological papers from any international HTA agency. Four databases were searched for qualitative, quantitative, and mixed methods study designs. The grey literature search included policy and practice documents from HTA and health organization websites. Two reviewers independently completed title and abstract screening before the full-text review and data extraction.

The main contributors to the production of knowledge about educating laypeople in HTA were the United Kingdom (40%), Spain (20%), and Canada (13%). Most studies included were conducted in the context of the United Kingdom (27%), followed by Spain (20%), and international networks context (20%). The main strategies included conference-like events (21%), the production of educational materials (18%), training (11%), and the use of plain language (8%). Furthermore, international HTA and health agencies have offered courses, and online training produced and made available online guidance materials for increasing laypeople’s participation in the HTA process.

Despite the global efforts to educate laypeople on HTA, jurisdictional variations underscore the need for a more inclusive approach. Strategies like events, educational material production, training, and clear-language use offer diverse avenues for public engagement. International agencies’ commitment to courses, online training, and guidance reflects a collective effort to enhance public involvement.