International Journal of Technology Assessment in Health Care最新文献

Caregivers can play an important role in supporting and caring for people with progressive, life-threatening, or debilitating conditions. However, this supportive role can expose caregivers to various detrimental financial, physical, and psychosocial issues. When evaluating medical technologies for reimbursement decisions, health technology assessment (HTA) agencies typically focus on the treatment's impact on patients and ignore or downplay the impact on caregivers. Including caregiver impacts within a wider societal perspective may better enable health systems to maximize health benefits from available resources. However, the lack of clear guidance or methodological recommendations from decision makers on the inclusion of caregiver impacts limits the number of HTA submissions that consider these effects. We outline a conceptual framework based on intensity and duration of caregiving to guide researchers, industry, and decision makers when developing policies for the inclusion of caregiver outcomes and justify their inclusion based on expected caregiver burden in identified circumstances.

Health Technology Assessment (HTA) in Europe has undergone significant evolution, culminating in the adoption of Regulation (EU) 2021/2282 on HTA (HTAR) aimed at fostering sustainable collaboration in HTA at the European Union (EU) level. The EUnetHTA 21 project, a 2-year initiative, was commissioned to address key methodological issues and prepare for the implementation of the HTAR. This commentary documents the outcomes of the EUnetHTA 21 project, focusing on Joint Clinical Assessments (JCAs), while analyzing challenges encountered and lessons learned for future collaboration under the HTAR. The EUnetHTA 21 consortium, comprising thirteen European HTA bodies, developed twenty guidance documents and thirteen templates, refining methods and procedures for joint work in HTA at EU level. Pilot JCAs and Joint Scientific Consultations were conducted to test these materials. Lessons learned from this experience emphasize the importance of inclusive consensus building, effective time and resource management, capacity building, and continuous quality improvement. The project's realization underscores a collective commitment among HTA bodies to continue to collaborate, now under a legal framework. Recommendations from the project, along with experiences gained from previous European Network for HTA (EUnetHTA) Joint Actions, provide a foundation for developing guidance for EU-HTA under the HTAR. Further proactive efforts at national and central levels are essential to coordinate and ensure a sustainable cooperation. The EUnetHTA 21 experience provides valuable insights for advancing cooperation in HTA under the HTAR, aiming to improve the quality of HTA, avoid duplication, and ultimately enhance patient access to safe and effective health technologies in the EU.

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.