International Journal of Technology Assessment in Health Care最新文献

Objectives: 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.

Methods: 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.

Results: 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.

Conclusions: 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.

The field of health care has evolved from an emphasis on evidence-based medicine, with a focus on efficacy, safety, and tolerability, to the pursuit of evidence-based efficiency and sustainable innovation in many respects (healthcare budgets, carbon print, etc.). This evolution can be attributed, in part, to the contributions of health technology assessment (HTA) bodies, which have facilitated the incorporation of various factors into the decision-making process (). These factors include comparative effectiveness, quality of life, efficiency, budgetary impact, and organizational impact, among others. Within the domain of health care, irrespective of the perspective of each entity (e.g., Food and Drug Administration (FDA), European Medicines Agency, etc.), there is an imperative for the presence of evidence and its assessment in the most transparent manner possible, with the objective of ensuring the incorporation of healthcare technologies.

Translating emerging health technologies towards adoption and patient benefit requires timely and effective research and development decisions. Early health technology assessment has a key role to play in supporting these decisions. A new consensus definition of early health technology assessment is a welcome contribution to help bring these activities toward wider use in the field. In parallel, the opportunities to perform early health technology assessment activities are increasing as new types of health technologies begin to enter healthcare systems globally. A greater focus on transparency of reporting, improving awareness around how early health technology assessment can impact decision-making, increased resourcing for these activities, expanding training for analysts, and encouraging collaboration between individuals across healthcare systems will be vital to strengthen the uptake of early health technology assessment from this point forward.

Objectives: 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.

Methods: 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.

Results: 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.

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

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

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

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

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

Objectives: 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.

Methods: 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.

Results: 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.

Conclusions: 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.

Objectives: 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.

Methods: 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.

Results: 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.

Conclusion: 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.

Real-world evidence (RWE) is increasingly used and accepted by health technology assessment (HTA) bodies as supportive evidence to inform the approval of new technologies. However, the criteria driving RWE acceptance are often unclear.This study aims to improve understanding of the role and value of RWE in HTA decision-making and outline the best practices in building real-world external control arms (ECAs).A mixed approach of a targeted literature review and HTA expert interviews was applied. The HTA reports of ten selected technologies and the expert interviews from France, Germany, Italy, Spain, and the UK informed the criteria driving the acceptance of RWE. Overall, the UK and Spanish HTA bodies are more receptive to accepting RWE, whereas the French and German are the least accepting. When RWE is used to substantiate efficacy claims, the level of scrutiny from regulators and HTA bodies is considerably higher than when RWE has different intended uses. Representativeness of the data source, overall transparency in the study and robust methodologies are the key criteria driving RWE acceptance across markets.

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

Methods: 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.

Results: 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.

Conclusions: 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.

Introduction: 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?

Methods and results: 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.

Conclusions: 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.