The aim of this study is to develop a patient-level model for type 2 diabetes mellitus (T2DM) progression that can estimate the cost-effectiveness of T2DM interventions from prevention to management.

We developed an individual-level microsimulation model, the Institute of Health Economics Diabetes Model (IHE-DM), that simulates: (i) T2DM progression from normal glucose tolerance (NGT) to T2DM, (ii) the occurrence and timing of eight comorbidities and death, and (iii) the correlated progression of risk factors over time. We report model validation and use a case study to investigate the cost-effectiveness of a hypothetical T2DM prevention program.

The internal validation indicated excellent performance with mean absolute differences between the predicted and observed values for all endpoints of less than 1 percent. External validation results were mixed. The model under-predicted cumulative T2DM incidence in the first 8 years, predicted well from years eight through eleven, and over-predicted from years twelve through fifteen. Our case study estimated an incremental net monetary benefit of CAD 2,701 (USD 2,289) (95% Uncertainty Interval: CAD 1,316 to 4,000 [USD 1,115 to 3,390]) over the 15-year time horizon.

Prominent T2DM models focus on patients with diagnosed T2DM whereas our model simulates progression from NGT to T2DM and incorporates important correlations in the progression of risk factors. These adaptations allow us to evaluate preventative interventions and better capture the long-term impacts, filling an important gap in the evidence base. Our model can be used to inform future funding decisions for T2DM interventions across the care continuum.

To explore the view of nonlayperson committee members on the added value of a discrete choice experiment (DCE) instrument to measure patient and committee member preferences for a health intervention.

Nine semistructured interviews were conducted with voting members from two types of advisory committees in Quebec, Canada: one from the Ministry of Health and Social Services, and eight from the Health Technology Assessment (HTA) agency. The DCE instrument, administrable to patients (i.e., pregnant women) and committee members, was developed and administered to both groups to measure their preferences about the addition of fetal chromosomal anomalies to a prenatal screening program. A conceptual framework consisting of three dimensions (relative advantage, compatibility, and complexity) was used for data collection and analyses.

Committee members considered the DCE instrument, when used with both patients and committee members, to be particularly valuable in raising awareness of potential biases. These biases, generated by committee members’ interests and disciplinary perspectives, can reduce the importance of the patient perspective in decision making by advisory committees.

This qualitative study provides insight into the perceptions of nonlayperson advisory committee members regarding the added value of a DCE instrument administered to patients and committee members regarding an intervention. Additional studies are required to explore the perceptions of other stakeholders (e.g., managers, patients, and public representatives) regarding the application of DCE and to assess its impact on HTA recommendations regarding the value of new health interventions.

This assessment aimed to identify the degree and parameters of demand for support from HTAsiaLink, the Asia regional health technology assessment (HTA) hub, for HTA ecosystem development.

A sequential, exploratory, mixed-method design was implemented, starting with a literature review to define the Asia region’s HTA landscape. Then an online survey was sent to 125 Asia-focused HTA practitioners and support organizations to obtain their thoughts on HTA development needs and how a regional hub could serve them. Finally, fifty purposively selected key informants representing government HTA agencies in Asia, funding partner organizations, philanthropic foundations, global HTA support, and regional HTA hub organizations were invited to participate in semi-structured interviews. Nineteen Asian countries and territories were represented in documents reviewed. Twenty-five recipients from ten Asian countries and territories responded to the survey, and twenty-eight individuals from eight Asian countries and territories plus eight international organizations participated in interviews.

Identified needs include support to fill HTA human resources gaps, strengthen the capacity of the existing HTA workforce, produce HTA public goods, improve harmonization within and across country systems, and strengthen political will. Other important considerations include the need to adapt the hub’s purpose to an expanding role and adopt sustainable financing approaches accordingly.

Demand for an HTA hub in Asia is high, including to support HTA technical, deliberative processes, and institutional capacity strengthening. Findings underscore the importance of both conducting HTAs and fostering demand for HTA output. HTAsiaLink is recognized as well-positioned to play an expanded support role to address these needs.

This study aimed to evaluate the required test characteristics that a psoriatic arthritis (PsA) biomarker test would need to achieve to be considered cost-effective.

We adapted an existing Markov model to compare a hypothetical biomarker with current practice. The model followed a patient cohort aged 45 years with moderate psoriasis (PsO) in which PsA was prevalent but unrecognized over a 40-year time horizon. Patients were assumed to be routinely seen at a dermatology clinic. In the current practice arm, patients with PsA were clinically detected. In the biomarker arm, a hypothetical test was assumed to be administered at baseline. Patients who screened positive would accept a combination of conventional disease-modifying antirheumatic drugs and targeted treatment to slow disease progression. Progression was modeled as linear changes in Health Assessment Questionnaire (HAQ) scores. We varied the sensitivity, specificity, and biomarker price based on current development progress. Scenario analyses considered alternative patient cohorts with mild and severe PsO separately.

The base case showed that a biomarker test with 70 percent sensitivity, 80 percent specificity, and a price of US$500 would be cost-effective (incremental cost-effectiveness ratio US$47,566 per quality-adjusted life-year [QALY]). Three-way analyses showed that a test with 80 percent specificity could be cost-effective at a US$50,000 per QALY threshold with a sensitivity as low as 66 percent at US$500. Only a near-perfect test would be cost-effective at a US$1,000 price point. Results were sensitive to HAQ progression under treatment, therapy costs, and the patient population.

This study supports the continued product development of candidate PsA biomarkers.

This study explored patient involvement in healthcare decision-making in the Asia Pacific region (APAC) by identifying roles and factors influencing differences between healthcare systems. Proposed recommendations to enhance patient engagement were made.

This systematic literature review was conducted using studies from Australia, China, Japan, Malaysia, New Zealand, the Philippines, South Korea, Singapore, Taiwan, and Thailand. Studies were included if they provided data on patient involvement in health technology assessment (HTA) and/or funding decisions for medicines. Extracted data were scored according to eleven parameters adapted from the National Health Council (NHC) rubric, which assessed the level of patient involvement in healthcare system decision-making.

We identified 159 records between 2018 and 2022, including methodology guidelines from Government websites. Most mentioned parameters were patient partnership, patient-reported outcome, and mechanism to incorporate patient input. Limited information was available on diversity and patient-centered data sources. Tools for collecting patient experience included quality-of-life questionnaires, focus groups, interviews, and surveys, with feedback options like structured templates, videos, and public sessions.

Beyond input in assessment process, involvement of patients in decision-making phase has evolved within HTA bodies over time with considerable variation. Few APAC healthcare systems involve patients in the appraisal process as members of the recommendation or decision-making committee.

The findings indicate that while patient involvement in pharmaceutical reimbursement decisions exists, improvements are needed. Effective integration of patient input requires transparency, education, and resource planning. This study establishes a baseline to track progress and assess the long-term impact of patient involvement.

Health technology assessment (HTA) is a form of policy analysis that informs decisions about funding and scaling up health technologies to improve health outcomes. An equity-focused HTA recommendation explicitly addresses the impact of health technologies on individuals disadvantaged in society because of specific health needs or social conditions. However, more evidence is needed on the relationships between patient engagement processes and the development of equity-focused HTA recommendations.

The objective of this study is to assess relationships between patient engagement processes and the development of equity-focused HTA recommendations.

We analyzed sixty HTA reports published between 2013 and 2021 from two Canadian organizations: Canada’s Drug Agency and Ontario Health.

Quantitative analysis of the HTA reports showed that direct patient engagement (odds ratio (OR): 3.85; 95 percent confidence interval (CI): 2.40–6.20) and consensus in decision-making (OR: 2.27; 95 percent CI: 1.35–3.84) were more likely to be associated with the development of equity-focused HTA recommendations than indirect patient engagement (OR: .26; 95 percent CI: .16–.41) and voting (OR: .44; 95 percent CI: .26–.73).

The results can inform the development of patient engagement strategies in HTA. These findings have implications for practice, research, and policy. They provide valuable insights into HTA.

To provide an overview of learning strategies that health technology assessment (HTA) agencies use worldwide to educate laypeople about HTA.

A scoping review focused on learning strategies to educate laypeople about HTA using the Joanna Briggs Institute frameworks was conducted across databases and gray literature. The study reviewed qualitative, quantitative, and mixed-methods studies from four databases, including practice documents from the HTA and health organization websites.

Fifteen studies were included in this review. The United Kingdom, Spain, and Canada mainly contributed to knowledge about educating laypeople in HTA. The main strategies employed were conference-like events, educational materials, training, and plain language. International HTA and health agencies developed courses, online training, and guidance materials to increase laypeople’s participation in the HTA process.

Efforts to improve public involvement in HTA focus on structured consultations, digital platforms, and capacity-building to enhance accessibility. Strategies like workshops and plain language aim to encourage lay participation, but challenges such as technical complexity and limited resources persist. Despite these challenges, incorporating patient perspectives has increased research relevance and public trust. Future studies should examine standardized frameworks for involvement, the impact of lay participation on policy, and solutions to barriers to a more equitable HTA process.

As Ethiopia advances towards efficient resource utilization and UHC through strategic health purchasing, the institutionalization of HTA will play a critical role. This study aims to identify key stakeholders, analyze the political economy surrounding HTA and priority setting in Ethiopia, and assess existing skills and capacities for a robust and sustainable HTA system.

We employed a mixed-method approach, combining 16 key informant interviews, 24 document reviews, and a cross-sectional survey (n=65) to assess national HTA capacity. We employed the Walt and Gilson policy analysis triangle framework, alongside Campos and Reich’s framework, to evaluate the context, process, content, and actors influencing HTA institutionalization, and to explore the complex interplay of institutions, positions, power, and interests among various stakeholders.

While there is a general commitment to implementing HTA across various government agencies and stakeholder groups, the institutionalization process faces several challenges, involving multiple agencies with overlapping mandates, raises bureaucratic challenges and potential conflicts, risking horizontal fragmentation as agencies compete for authority, budget, and influence. The involvement of other key stakeholders, such as professional associations, patients, and the public, is notably lacking. Challenges such as limited HTA expertise, high professional turnover, and gaps in specific HTA knowledge areas persist, with capacity-building efforts often failing to address organizational needs effectively.

The complexity of HTA institutionalization in Ethiopia underscores the necessity of managing intricate inter-agency dynamics, establishing a robust legal framework for an inclusive and transparent HTA process, building local capacity, and securing sustainable, domestically aligned funding.

Effective allocation of scarce healthcare resources involves complex ethical and technical evaluations, with decision makers sometimes utilizing a societal perspective in health technology assessment (HTA). This study aimed to explore societal perspectives on healthcare resource allocation within Australia’s HTA framework, focusing on the valuation of health gains for children and young people (CYP) compared to adults.

In-depth, semistructured interviews were conducted with ten young people (aged 15–17) and twenty adults between October 2021 and April 2022. Participants were purposively sampled for diverse characteristics and completed an online information survey prior to the interviews, introducing relevant concepts. Interviews were analyzed using inductive coding, categorization, and constant comparison.

Participants expressed nuanced perspectives on HTA processes, generally opposing numeric weighting and preferring a deliberative approach based on committee judgment. Although most participants acknowledged some moral relevance of CYP status in HTA, opinions varied on its operationalization. A sizable minority, including those with extensive health system experience, did not view CYP status as morally relevant, though some noted specific service gaps for CYP (e.g., mental health care, pain management). Participants identified a spectrum of factors, both person-centered and intervention related, that often surpassed the relevance of CYP status, including addressing severity, unmet needs, prevention, and early intervention, with an emphasis on Aboriginal and Torres Strait Islander communities.

Our findings highlight the inherent challenges in navigating the complexities of HTA and the critical need for HTA frameworks to be adaptable and inclusive, effectively integrating societal preferences to enhance healthcare policy’s equity and responsiveness.

Health technology assessment (HTA) reports are written for healthcare decision makers, particularly in relation to reimbursement/pricing, and are intended to assess clinical effectiveness, safety, and cost. Four additional domains are further considered in what is called a “full HTA”: ethical, legal, social, and organizational aspects. The ethical aspects have long been the subject of debate regarding how they should be processed. It would be important if the following questions could be answered: Who publishes full HTA reports and how? Which methods are used in the ethics domain? What kind of results do they produce? However, such a “mapping of the field” turns out to be difficult. Despite the existence of international HTA registers, we were not able to compile a comprehensive sample of full HTA reports. Therefore, the aim of our study was rather to explore a) substantially: Which information can be expected to be (easily) found, which can only be obtained with considerable effort, and which remain (for the time being) in the dark? And b) methodologically: Is it possible to do meaningful meta-research in this field?

In the attempt to explore the possibilities of meta-research, we were able to track down and analyze thirty-nine full HTA reports from six countries.

While not representative of the whole field, this analysis shows the possibilities and challenges to meta-research, but nonetheless also provides some substantial insight into the characteristics of such reports, with a particular focus on the methods used to process ethical aspects.

Health technology assessment (HTA) is a critical part of healthcare decision making in many countries. Changes in Methods and Processes (M&P) of HTA agencies can affect the time and degree of patient access to treatments. Published literature focuses on the different M&P adopted by HTA agencies, rather than on how these have come about over time. Our study investigates key HTA reforms and explores their drivers and interdependencies in a set of HTA agencies in Europe, Asia-Pacific, and North America.

We conducted a targeted literature review on M&P guidelines and subsequent changes to those, for 14 HTA agencies. We supplemented and validated initial findings with 29 semi-structured interviews with country-specific experts. We used analytical tools to create process maps, proactivity and influence networks, and clusters of HTA agencies.

We found that processes leading to M&P reforms follow similar steps across HTA agencies. The three most important drivers to reforms were HTA practice and guidelines in other countries; the healthcare policy, legal, and political context within the agency’s country; and experience of challenges in the assessment by the HTA body itself. International collaborations have the potential to accelerate the evolution of HTA systems and the implementation of reforms.

We identified PBAC (Australia), CDA-AMC (Canada), NICE (England), IQWiG (Germany), and ZIN (the Netherlands) as HTA agencies that are catalysts of HTA reforms as well as internationally influential. International collaborations may represent a useful route to accelerate changes as long as they ensure wide stakeholder engagement at an early stage.

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.