International Journal of Technology Assessment in Health Care最新文献

Background: Constrained resources under universal health coverage (UHC) necessitate a balance between medication costs and essential health system requirements. Policymakers practice priority-setting, as either implicit or explicit rationing, embedded in evidence-informed decision-making processes to guide funding decisions. Health technology assessment (HTA) is a method that may assist explicit evidence-informed priority setting. South Africa developed an official HTA methods guide in 2022, however before this, commissioning and performing economic evaluations was not standardized.

Methods: We conducted a descriptive collective case study to explore the impact of economic analyses on the selection of, and access to, essential medicines in South Africa. Four cases were purposefully selected, and both official information and secondary data, including media reports, were reviewed. Data elements were extracted and organized in a matrix. Cases were reported narratively with a positivist epistemological approach, presenting the authors' reflections.

Results: We found economic analyses that reflected methodologies described in the HTA guide: international reference pricing, cost-minimization, cost-effectiveness, cost-utility, and budget impact analyses. Economic analyses informing the 'resource-use' domain in the GRADE evidence-to-decision framework supported decision-making, influenced market-shaping with price reductions of interventions through benchmarking (fosfomycin, flucytosine), improved equitable access nationally (flucytosine), and prioritized a defined patient group in a justifiable and transparent manner (bortezomib).

Conclusion: A standardized HTA evaluation process guided by a nationally accepted framework is necessary for evidence-informed decision-making. Economic analyses (cost-effectiveness, affordability, and resource use) should be consistently included when making decisions on new interventions.

Health technology assessment (HTA) agencies assess evidence to support decision making about which technologies to provide and pay for in the health system. HTA impact is understood as the influence that HTA report findings can have in the health system, including impacts on reimbursement decisions, changes to health outcomes, or broader system or societal impacts. The International Network of Agencies for Health Technology Assessment (INAHTA) is a global network of publicly funded HTA agencies. INAHTA's mission, in part, is to advance the impact of HTA to support reimbursement decisions and the optimal use of health system resources. Each year, INAHTA awards the David Hailey Award for Best Impact Story to the member agency that shares the best story, as voted by fellow members, about HTA impact. The impact story sharing program in INAHTA contributes to a deeper understanding of what works well (or not so well) in achieving HTA impact. This paper provides six impact stories from agencies that were finalists for the 2021 and 2022 David Hailey Impact Award for Best Impact Story: the Institut national d'excellence en santé et en services sociaux, the Malaysian Health Technology Assessment Section, Ontario Health, the Center for Drug Evaluation, the National Institute for Health and Care Excellence, and Health Technology Wales. These stories demonstrate that HTA agencies can, in differing ways, effectively support governments in their efforts to place evidence at the centre of decision making.

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

Objective: 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.

Methods: 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.

Conclusion: 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.

Objectives: It is vital that horizon scanning organizations can capture and disseminate intelligence on new and repurposed medicines in clinical development. To our knowledge, there are no standardized classification systems to capture this intelligence. This study aims to create a novel classification system to allow new and repurposed medicines horizon scanning intelligence to be disseminated to healthcare organizations.

Methods: A multidisciplinary working group undertook literature searching and an iterative, three-stage piloting process to build consensus on a classification system. Supplementary data collection was carried out to facilitate the implementation and validation of the system on the National Institute of Health and Care Research (NIHR) Innovation Observatory (IO)'s horizon scanning database, the Medicines Innovation Database (MInD).

Results: Our piloting process highlighted important issues such as the patency and regulatory approval status of individual medicines and how combination therapies interact with these characteristics. We created a classification system with six values (New Technology, Repurposed Technology (Off-patent/Generic), Repurposed Technology (On-patent/Branded), Repurposed Technology (Never commercialised), New + Repurposed Technology (Combinations-only), Repurposed Technology (Combinations-only)) that account for these characteristics to provide novel horizon scanning insights. We validated our system through application to over 20,000 technology records on the MInD.

Conclusions: Our system provides the opportunity to deliver concise yet informative intelligence to healthcare organizations and those studying the clinical development landscape of medicines. Inbuilt flexibility and the use of publicly available data sources ensure that it can be utilized by all, regardless of location or resource availability.

Antimicrobial resistance (AMR) has become a worldwide growing concern over the past decades. Thus, encouraging manufacturers to develop new antibiotics is needed. We hypothesised that transparency on the regulatory appraisals of antibiotics would provide an incentive to pharmaceutical development. We thus aimed at reporting the French health technology assessment (HTA) opinions and reimbursement decision on antibiotics to those German (G-BA) and English (NICE) HTA bodies.A qualitative analysis of the Transparency Committee of the French National Authority for Health (TC-HAS) opinions regarding antibiotics assessment between 2016 and 2020 was performed. Decisions of reimbursement by TC-HAS were compared to those from G-BA and NICE when available. TC-HAS recognized a clinical benefit (CB) for 15/15 evaluated indications, a clinical added value for 9/15, and a public health interest for 8/15. Among the valued antibiotics by HAS, 5 were recommended for restricted use as a "reserve" to protect against the risk of resistance emergence. A comparison of HTA opinions was possible across HTA for only 8 antibiotics. The G-BA granted a reserve status for 4 drugs and NICE a reserve with restricted use for 5 antibiotics. Three of these antibiotics were positioned similarly by the English, German, and French HTA bodies. This qualitative analysis of HTA opinions between different European HTA bodies shows a consistent reimbursement decision of antibiotics against MDR bacteria and tuberculosis besides the differences in the applied assessment methods. This work also shows how HTA bodies could recognize a clinical added value in a context of the emergence of antibiotic resistance.

Background: The China Rural Hypertension Control Project (CRHCP) is a nonphysician-led community-based hypertension intervention program that has demonstrated clear benefits in improving blood pressure (BP) control and reducing the incidence of cardiovascular disease events among hypertensive patients in rural areas of China. However, it is currently unclear whether the benefits of the CRHCP outweigh its costs, and whether promoting this project in China is justifiable from a perspective of healthcare system.

Methods: We employed a Markov model to forecast the anticipated 20-year costs and effectiveness of the CRHCP trial. Cost data for this study was gathered from public records or published papers, whereas clinical data was extracted from the CRHCP trial. Our primary outcome measure was the incremental cost-effectiveness ratio, expressed in Chinese Yuan (CNY) per quality-adjusted life-year (QALY), representing the additional cost per additional QALY gained.

Results: Over a span of 20 years, the cost for a rural hypertensive individual in China who received intensive BP intervention by a nonphysician community healthcare provider would amount to 25,129 CNY, yielding an effectiveness of 8.19 QALY. In contrast, if usual care was provided, the cost would be 26,709 CNY with an effectiveness of 7.94 QALY. The CRHCP program demonstrated lower costs and greater effectiveness for rural hypertensive individuals in China.

Conclusion: Our study indicates that the implementation of the CRHCP program among rural hypertensive individuals in China resulted in increased effectiveness and reduced costs. From the perspective of Chinese healthcare system, the CRHCP program proves to be cost-saving within the current healthcare landscape.

Objectives: Ministry of Health (MOH) Malaysia stakeholders seek primary care access to sodium-glucose cotransporter 2 inhibitor (SGLT2i). Addressing this required a complex decision, selecting among three SGLT2i for two different indications and two practice settings. The options include expanding the existing SGLT2i (empagliflozin) in the MOH Medicines Formulary to primary care and/or having dapagliflozin and/or luseogliflozin as alternatives. This study aimed to conduct a multiple health technology assessment (HTA) to determine the SGLT2i of choice for the MOH setting.

Methods: The clinical benefits of SGLT2i were assessed through a systematic literature review and affordability was assessed through the development of three budget impact analysis models simulating seventy scenarios. Each model varied by prescribing indications, restrictions, and SGLT2i involved (M1: glycemic control, HbA1c between 6.5 percent and 10 percent, empagliflozin-dapagliflozin-luseogliflozin; M2: cardiovascular benefits, HbA1c less than 10 percent, empagliflozin-dapagliflozin; M3: a composite of M1 and M2). The outcome of the HTA was presented to the MOH decision-makers.

Results: Although there was no significant difference in glycemic control between the SGLT2i, differences exist in cardiovascular benefits conferred. Despite having scenarios with lower net budget impact (NBI) in the M1, M2, and M3 models, decision-makers decided to expand empagliflozin use to primary care setting and add dapagliflozin for hospital-only setting for both indications [NBI of $4.38 mil] due to empagliflozin's advantage in reducing risk for cardiovascular death and prior experience of its use in MOH.

Conclusions: The multiple HTA approach guided the complex decision-making process by providing a holistic understanding of the decision's impact.

Background: The number of orphan drug (OD) approvals has increased sharply in Europe. In Germany, all ODs are initially subject to a limited assessment after market access. Their added benefit over the standard of care is accepted as established upon EU approval; a regular health technology assessment (HTA) is performed only in certain cases.

Objective: We assessed whether the increase in OD approvals has led to improvements in patient-relevant outcomes as supported by the evidence submitted by market authorization holders (MAHs) for HTA in Germany. We also examined the extent to which these ODs address unmet medical needs.

Methods: The results of limited assessments and regular HTAs of ODs in Germany (January 2011-September 2021, plus January-December 2023) were analyzed to determine their added benefit based on MAH-submitted dossiers. Added benefit was reported separately for each research question generated from the EU-approved therapeutic indications and any sub-indications (e.g., different subpopulations or control interventions) specified for HTA in Germany.

Results: Eighty-nine ODs (limited assessments: sixty-nine; regular HTAs: twenty) were evaluated in 175 research questions (limited assessments: ninety-seven; regular HTAs: seventy-eight). The added benefit granted in limited assessments was non-quantifiable in nearly eighty percent of the ninety-seven questions. In regular HTAs, no proof of added benefit was shown in fifty-four percent of the seventy-eight questions, mainly due to insufficient comparative data with the standard of care. Established treatments were available for fifty-eight percent of the seventy-eight questions; more than half of which addressed oncology indications (although these account for only eight percent of rare diseases).

Conclusions: Due to evidence gaps in post-approval HTA, many ODs approved in the EU lack proof of added benefit in terms of improving patient-relevant outcomes. Moreover, most approved ODs are indicated for diseases with established treatments and oncology indications, while many unmet medical needs remain. Incentives are required to encourage research in areas of unmet medical need and to generate comparative data with the standard of care.

Objectives: In the field of Laboratory Medicine, the evolution of knowledge and the innovation of technologies are the basis of analytical and diagnostic progress, leading to the development of new solutions based on innovative technologies. However, these advances must be accompanied by evidence of appropriateness, diagnostic effectiveness, and organizational efficiency, considering the test's first impact on patient outcomes.

Methods: The Health Technology Assessment (HTA) is a valid management tool to support Laboratory Medicine professionals in assessing technologies and which is the most appropriate to adopt. This study is an illustrative case of the application of HTA, exploiting the EUnetHTA Core Model, on two analyzers able to determine the glycated hemoglobin (Hemoglobin A1c, HbA1c), the Capillarys 2 Flex piercing analyzer and the HLC-723G11 analyzer in the Laboratory Medicine Service of the IRCCS San Raffaele Hospital (Milan, IT). The main focus is related to potential differences in methods, organizational aspects, and clinical effectiveness of these approaches for measuring HbA1c.

Results: The EUnetHTA Core Model has proven to be the optimal method for HTA in the field of Laboratory Medicine, as it allows to highlight both the peculiarities of the methods on which the analyzers are based and the clinical efficacy of the laboratory test on specific patient populations, considering individual variations in treatment responses, assessing the potential benefits for individual patients or small groups.

Conclusions: This granular analysis helps provide insights into the effectiveness and value of healthcare interventions at the patient level, contributing to evidence-based decision-making in clinical practice and healthcare policy.

Context: Healthcare stakeholders in Latin America, including payers, manufacturers, and patients, seek to expedite access to technologies. However, uncertainty sometimes surrounds their true benefits and budgetary implications. Managed entry agreements (MEAs) are proposed to address this uncertainty by redistributing risks among key actors.

Objectives: The objective of Health Technology Assessment International's 2023 Latin American Policy Forum was to examine the potential utility of MEA in technology reimbursement and decision-making processes in the region.

Methods: This article is based on a background document, a survey, and the deliberative work of the country representatives and others who participated in the Policy Forum.

Results: Interest in MEA in Latin America is increasing, with financial agreements being more prevalent than those based on clinical outcomes. During the Policy Forum, potential barriers to MEA implementation were identified, such as the lack of legal frameworks, insufficient reliable data, and, in some cases, distrust among stakeholders. Some potential solutions were also identified, including early stakeholder involvement to enhance dialogue and understanding, and piloting shorter-duration MEA to facilitate the revision of agreement terms, especially in situations of epidemiological uncertainty.

Conclusions: The Policy Forum served as a valuable platform for discussing the importance of flexible MEA implementation that acknowledges data uncertainty, promotes transparent dialogue to incorporate opinions and values from all stakeholders, and develops legal frameworks to support effective technology access schemes in Latin America.