UNASSIGNED: To quantitatively assess all dosage forms of three active vitamin D and its analogs, namely, calcitriol, alfacalcidol, and eldecalcitol, to provide a basis for the selection of active vitamin D and its analogs in hospitals.
UNASSIGNED: In this study, three active vitamin D and its analogs were evaluated by quantitative scoring in five dimensions, including pharmaceutical properties (28 points), efficacy (27 points), safety (25 points), economy (10 points), and other attributes (10 points).
UNASSIGNED: The final scores of quantitative assessment for the selection of alfacalcidol soft capsules, calcitriol soft capsules I, calcitriol soft capsules II, alfacalcidol tablets, alfacalcidol capsules, alfacalcidol oral drops, calcitriol injection, and eldecalcitol soft capsules were 73.17, 72.06, 71.52, 71.29, 69.62, 68.86, 65.60, 64.05 points.
UNASSIGNED: Based on the scoring results, alfacalcidol soft capsules, calcitriol soft capsules I, calcitriol soft capsules II, alfacalcidol tablets can be entered into the medication list of medical institutions as strongly recommended drugs. This study offers guidance on selecting and using active vitamin D and its analogs in hospitals, with consideration for the patient\'s needs.

OBJECTIVE: Discounting the cost and effect for health intervention is a controversial topic over the last two decades. In particular, the cost-effectiveness of gene therapies is especially sensitive to the discount rate because of the substantial delay between the upfront cost incurred and long-lasing clinical benefits received. This study aims to investigate the influence of employing alternative discount rates on the incremental cost-effectiveness ratio (ICER) of gene therapies.
METHODS: A systematic review was conducted to include health economic evaluations of gene therapies that were published until April 2023.
RESULTS: Sensitivity or scenario analysis indicated that discount rate represented one of the most influential factors for the ICERs of gene therapies. Discount rate for cost and benefit was positively correlated with the cost-effectiveness of gene therapies, that is, a lower discount rate significantly improves the ICERs. The alternative discount rate employed in some cases could be powerful to alter the conclusion on whether gene therapies are cost-effective and acceptable for reimbursement.
CONCLUSIONS: Although discount rate will have substantial influence on the ICERs of gene therapies, there lacks solid evidence to justify a different discounting rule for gene therapies. However, it is proposed that the discount rate in the reference case should be updated to reflect the real-time preference, which in turn will affect the ICERs and reimbursement of gene therapies more profoundly than conventional therapies.

China\'s health system is facing severe challenges from social transition and the double burden of population aging and non-communicable diseases. Addressing the tension between the public\'s increasing demand for health services and the limited availability of medical resources has become a critical issue for health care policymakers and medical insurance fund administrators. In promoting its medical insurance system reform, China is actively developing health technology assessment (HTA) with principles and applications adapted to the Chinese context. This study aims to analyze the evolution of HTA in China with a focus on context, actors, process, content, and challenges encountered through applying a modified verson of Walt and Gilson\'s policy triangle framework. Currently, HTA plays an indispensable part in the reform of China\'s health care and medical insurance system, especially in the formulation and adjustment of the National Reimbursement Drug List (NRDL). While HTA is increasingly used in China, there remain challenges, such as the slow development of HTA related disciplines, lack of an independent national HTA authority, and limited scope in the use of HTA. Despite the identified challenges, HTA has the potential to support a wide range of applications in China\'s health care sector, building on the progress achieved over the last three decades.

BACKGROUND: Economic evaluation of one-time therapies during reimbursement decision-making is challenging due to uncertain long-term outcomes. The availability of 5-year outcome data from the ELIANA trial and real-world evidence of tisagenlecleucel, the first chimeric antigen receptor T-cell (CAR-T) therapy, presents an opportunity to re-evaluate the predictions of prior cost-effectiveness analyses (CEAs).
OBJECTIVE: To conduct a systematic literature review (SLR) of prior CEAs of tisagenlecleucel for pediatric/young adult relapsed or refractory acute lymphoblastic leukemia (r/r ALL) and evaluate the impact of recently available 5-year efficacy data from ELIANA and advances in CAR-T manufacturing in an updated CEA model.
METHODS: OVID MEDLINE/Embase and health technology assessment (HTA) databases were searched for full-text economic evaluations in English reporting cost-effectiveness results for tisagenlecleucel for r/r ALL. Evaluations with publicly reported incremental cost-effectiveness ratios (ICERs) were included in the SLR. Study screening and data abstraction were conducted following PRISMA guidelines. Data extracted included the country/currency, perspective, clinical trial evidence, model structures, long-term efficacy extrapolation approaches (i.e., overall survival [OS]), time horizon, discount rates, and outcomes (i.e., life years [LY], quality-adjusted LY [QALY], and ICERs). The CEA model reported in Wakase et al. was updated using 5-year OS data from ELIANA and the CAR-T infusion rate informed by real-world practice.
RESULTS: Sixteen records corresponding to 15 unique studies were included in the SLR (11 publications and 5 HTA reports); all were conducted from the health care system perspective of the respective countries. Most studies found tisagenlecleucel to be cost effective, but all studies\' projected 3- and 5-year OS rates for tisagenlecleucel were lower than the observed 3- and 5-year rates, respectively, derived from 5-year ELIANA data. When applying updated OS projections from the most recent ELIANA data cut and higher infusion rates of 92.5% (per the real-world infusion rate)-96.0% (per the manufacturer success rate) to the CEA of Wakase et al., the associated QALYs for tisagenlecleucel increased from 11.6 to 14.6-15.0, and LYs increased from 13.3 to 17.0-17.5. Accordingly, the ICERs for tisagenlecleucel decreased from ¥2,035,071 to ¥1,787,988-¥1,789,048 versus blinatumomab and from ¥2,644,702 to ¥2,257,837-¥2,275,181 versus clofarabine combination therapy in the updated CEA model.
CONCLUSIONS: Projections at launch of the likely cost effectiveness of tisagenlecleucel appear to have underestimated its ultimate economic value given more recent trial and real-world data. To balance uncertainty in initial valuation with the need to provide access to novel oncology therapies, payers can consider flexible reimbursement policies alongside ongoing assessments as new data emerge.

BACKGROUND: Traditional therapies are crucial in maintaining and improving human well-being. China\'s healthcare policymakers are attempting to use health technology assessment (HTA) as a decision-making supportive tool. The value assessment framework for Chinese patent medicine (CPM) has been developed and is being adopted and validated widely by research institutions. Subsequently, the healthcare decision-makers particularly hanker for the value framework of traditional non-pharmacological therapies.
METHODS: To construct a practical value framework for traditional non-pharmacological therapies, a scoping review methodology was adopted to identify the evaluation domains and obstacles. A search, screening, and analysis process was conducted according to the Preferred Reporting Items for Systematic Reviews and Meta-Analyses extension for Scoping Reviews (PRISMA-ScR). Evidence was retrieved from scientific databases and HTA agencies\' websites.
RESULTS: The search strategy identified 5 guidelines records and 17 acupuncture HTA reports. By synthesizing the valuable reports of CPM and acupuncture evaluation in representative countries, this study found that Mainland China was promoting the comprehensive value assessment of CPM, whereas the United Kingdom, Singapore, Canada, the United States, and Malaysia had carried out the HTA evaluation of acupuncture for various conditions among which chronic pain was the most common. UK and Singapore applied the HTA results to support acupuncture reimbursement decisions. Three domains, including safety, effectiveness, and economy, were commonly adopted. The identified biggest challenge of evaluating traditional non-pharmacological therapies is the scarce high-quality clinical evidence.
CONCLUSIONS: This study identified value domains and issues of traditional therapies, and pointed out future research implications, to promote the development value framework of traditional therapies.

This study conducts a rapid health technology assessment to systematically evaluate the effectiveness, safety, and cost-effectiveness of Cerebrolysin as an adjunctive therapy for acute ischemic stroke to provide evidence-based medicine for clinical decisions of Cerebrolysin. All systematic reviews/meta-analyses, pharmacoeconomic studies, and health technology assessment reports of Cerebrolysin for the treatment of acute ischemic stroke before August 17, 2023, were retrieved from PubMed, Embase, Cochrane Library, China National Knowledge Infrastructure, Wanfang, Weipu, Sinomed database and the official website of health technology assessment. According to the inclusion and exclusion criteria, 2 researchers independently carried out screening, data extraction, and quality evaluation and descriptively analyzed the results of the included studies. A total of 14 pieces of literature were incorporated, comprising 8 systematic reviews/meta-analyses and 6 pharmacoeconomic studies. In terms of effectiveness, compared to control groups, the use of Cerebrolysin as a treatment for acute ischemic stroke demonstrates certain advantages, including enhancement in total efficacy rate, neurological function, upper limb motor dysfunction, and facilitation of the recovery of activities of daily living. Especially in patients with moderate to severe acute ischemic stroke, Cerebrolysin has demonstrated the ability to enhance neurological function recovery and ameliorate disabilities. Regarding safety, adverse reactions were mild or comparable to those in the control group. The primary findings of economic studies reveal that advocating for the use of Cerebrolysin offers certain cost-effectiveness advantages. Cerebrolysin contributes to improved clinical efficacy and evaluation indexes while demonstrating favorable safety and economic benefits.

OBJECTIVE: Valuing child health is critical to assessing the value of healthcare interventions for children. However, there remain important methodological and normative issues. This qualitative study aimed to understand the views of Canadian stakeholders on these issues.
METHODS: Stakeholders from health technology assessment (HTA) agencies, pharmaceutical industry representatives, healthcare providers, and academic researchers/scholars were invited to attend an online interview. Semi-structured interviews were designed to focus on: (1) comparing the 3-level and 5-level versions of the EQ-5D-Y; (2) source of preferences for valuation (adults vs. children); (3) perspective of valuation tasks; and (4) methods for valuation (discrete choice experiment [DCE] and its variants versus time trade-off [TTO]). Participants were probed to consider HTA guidelines, cognitive capacity, and potential ethical concerns. All interviews were recorded and transcribed verbatim. Framework analysis with the incidence density method was used to analyze the data.
RESULTS: Fifteen interviews were conducted between May and September 2022. 66.7% (N = 10) of participants had experience with economic evaluations, and 86.7% (N = 13) were parents. Eleven participants preferred the EQ-5D-Y-5L. 12 participants suggested that adolescents should be directly involved in child health valuation from their own perspective. The participants were split on the ethical concerns. Eight participants did not think that there was ethical concern. 11 participants preferred DCE to TTO. Among the DCE variants, 6 participants preferred the DCE with duration to the DCE with death.
CONCLUSIONS: Most Canadian stakeholders supported eliciting the preferences of adolescents directly from their own perspective for child health valuation. DCE was preferred if adolescents are directly involved.

OBJECTIVE: This study aimed to introduce a pilot program for hospital-based health technology assessment (HB-HTA) in China and present the participants\' experiences based on seven case studies from seven tertiary hospitals.
METHODS: One-year pilot projects were initiated at the beginning of 2018. Seven pilot hospitals were closely followed from the beginning until the completion of their pilot HTA project. Regular interviews were conducted with the hospital managers leading the HB-HTA projects and key members of the special HTA teams. Observations were made based on field trips and written HTA reports.
RESULTS: Three pilot projects evaluated the use of medical consumables, three evaluated the use of surgical or medical interventions, and one evaluated an innovative management model for ventilators. Real-world data were collected from all the pilot projects to assist with the assessments. Most HB-HTA pilot projects achieved remarkable results such as improvements in economic efficiency; however, there were also obvious deficiencies such as the lack of a necessary cost-effectiveness analysis.
CONCLUSIONS: The results varied among the seven HB-HTA pilot projects. The HB-HTA pilot program was implemented to promote the use of HB-HTA in hospital decision making in China. At the same time, HB-HTA in China faces challenges. We have made some policy recommendations based on the findings of the pilot projects.

Health Technology Assessment (HTA) in China has recently expanded from purely academic research to include policy or decision-oriented practice, especially after HTA evidence was used to update the National Reimbursement Drug List for the first time in 2017. This study aims to identify the progress and challenges of HTA development from 2016 to 2021 and inform policies and decisions to promote further HTA development in China.
We conducted a cross-sectional web-based survey with policy makers, researchers and industry-providers in China in 2016 and 2021 respectively. The \'Mapping of HTA Instrument\', was utilized to assess the HTA development across eight domains: Institutionalization, Identification, Priority setting, Assessment, Appraisal, Reporting, Dissemination of findings and conclusions, and Implementation in policy and practice. To reduce the influence of confounders and compare the mapping outcomes between the 2016 and 2021 groups, we conducted 1:1 Propensity Score Matching (PSM). Univariate analysis was conducted to compare the differences between the two groups. The overall results were further compared with those of a mapping study that included ten countries.
In total, 212 and 255 respondents completed the survey in 2016 and 2021, respectively. The total score of the HTA development level in China in 2021 was higher than that in 2016 before PSM (89.38 versus 83.96). Following PSM, 183 respondents from the 2016 and 2021 groups were matched. Overall, the mean scores for most indicators in the Institutionalization domain and Dissemination domain in 2021 were higher than those in 2016 (P < 0.05). The Appraisal domain in 2021 was more explicit, transparent and replicable than that in 2016 (t = -3.279, P < 0.05). However, the mean scores of most indicators in the Assessment domain were higher in 2016 than those in 2021 (P < 0.05).
Our study suggest that the level of HTA development in China progressed significantly from 2016 to 2021. However, before engaging in HTA activities, further efforts are required to enhance the assessment process. For instance, it is important to establish a clear goal and scope for HTA; adapt standardized methodologies for evaluating the performance of systematic reviews or meta-analyses; and provide comprehensive descriptions of the safety, clinical effectiveness, cost, and cost-effectiveness of the assessed technologies, thus improving the development of HTA in China.

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