The Efficacy, Safety, and Economic Outcomes of Using Ferric Derisomaltose for the Management of Iron Deficiency in China: A Rapid Health Technology Assessment.

Intravenous (IV) iron supplementation is the preferred treatment option for managing severe iron deficiency (ID) and ID anemia (IDA). Three of the available IV iron preparations are ferric derisomaltose (FDI), ferric carboxymaltose (FCM), and iron sucrose (IS). The objective of the present work was to review the published literature about the efficacy, safety, quality of life (QoL), and economic outcomes of using FDI, FCM, and IS for the treatment of ID. A systematic literature search was performed following Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines. Eligible studies were assessed for quality using appropriate tools, and data were extracted and analyzed for key outcomes. The evidence synthesis was based on published systematic literature reviews (SLRs), meta-analyses (MAs), indirect treatment comparisons (ITCs), and health technology assessments (HTAs); we also included economic evaluations performed from a Chinese perspective. Out of 337 initial hits, the review included 12 studies. The findings indicated that FDI, FCM, and IS had comparable efficacy in terms of hemoglobin (Hb) improvement. FDI showed a better safety profile with a lower risk of hypophosphatemia, hypersensitivity reactions, and cardiovascular adverse events (AEs) compared to IS and FCM. FDI also demonstrated better cost-effectiveness compared to IS, with potential cost savings attributed to fewer infusions and improved compliance. None of the included studies evaluated QoL after IV iron administration for ID. FDI offers a safe, efficacious, and cost-effective treatment option for ID. It exhibits comparable efficacy to FCM and IS but presents a better safety profile and economic advantage. FDI fulfills the criteria of efficacy, safety, economy, innovation, suitability, and accessibility, making it a promising choice for ID management in China.

Rapid health technology assessment of the novel endonuclease inhibitor baloxavir for the treatment of influenza.

Through a Rapid Health Technology Assessment (RHTA), we evaluated the efficacy, safety and cost-effectiveness of baloxavir in the treatment of influenza, providing the necessary scientific information and evidence-based basis for healthcare professionals and health insurance decision-makers in making rational selections. Through systematic searches of Pubmed, Embase, Web of Science, The Cochrane Register of Clinical Trials database and the official website of Health Technology Assessment (HTA) agencies, we collected systematic reviews (SR)/Meta-analysis, cost-effectiveness evaluations and HTA reports of baloxavir for influenza, with a search time frame of date of database establishment to July 31, 2022. We then performed data extraction, literature screening and quality evaluation on the literature that met our selection criteria, after which the results of the studies were pooled and qualitatively described for analysis. 10 studies were included, including 6 SR/Meta-analysis, three economics studies, and 1 HTA report. In terms of efficacy, baloxavir had an advantage over oseltamivir for all three types of influenza patients (otherwise healthy patients, high-risk patients, and patients are not separated into groups with and without underlying health conditions) concerning change in virus titer from baseline at 24 and 48 h; about otherwise healthy patients and high-risk patients, baloxavir had an advantage over peramivir; pertaining to high-risk patients, baloxavir had an advantage over laninamivir; the above differences between groups were all statistically significant. In terms of safety, in otherwise healthy patients and patients are not separated into groups with and without underlying health conditions, baloxavir significantly reduced the incidence of DRAEs and nausea compared with oseltamivir, as well as significantly reduced the incidence of DRAEs compared with laninamivir; in patients are not separated into groups with and without underlying health conditions, baloxavir significantly reduced the incidence of AEs and diarrhoea compared with oseltamivir; the differences between the above groups were all statistically significant. Economically, in Japanese adult influenza patients and high-risk populations, the Quality-Adjusted Life Years (QALY) of baloxavir slightly triumphed over that of laninamivir (Δ = 0.000112 and 0.00209 QALY per 1 patient, respectively); moreover, the incremental cost-effectiveness ratio (ICER: 2,231,260 and 68,855 yen/QALY, respectively) was below the willingness-to-pay (WTP) threshold (5,000,000 yen/QALY); in Chinese adult influenza patients without underlying diseases and adult high-risk influenza patients, baloxavir had a higher QALY compared with oseltamivir (Δ = 0.000246 and 0.000186 respectively), however, their ICER (12,230 and 64,956 RMB/QALY) was above the local WTP threshold (10,000 RMB/QALY) and thus did not provide a cost-effectiveness advantage. Baloxavir had a favorable efficacy and safety profile compared to neuraminidase inhibitors (NAIs), and the currently available evidence suggested that it had an economic advantage only in Japan.

[Three oral Chinese patent medicines for children with tic disorder: a rapid health technology assessment].

This study aims to comprehensively evaluate the clinical value of Shaoma Zhijing Granules(SZG), Changma Xifeng Tablets(CXT), and Jiuwei Xifeng Granules(JXG) in the treatment of children with tic disorder with the method of rapid health technology assessment(RHTA), which is expected to serve as a reference for medical and health decision-making and clinical rational use of drugs in children. To be specific, relevant articles were retrieved from eight databases and three clinical trial registry platforms. After the quality evaluation, rapid assessment was carried out from the dimensions of disease burden and unmet needs, technical characteristics, safety, efficacy and economy, and the results were analyzed and presented descriptively. A total of 22 articles(1 in English, 21 in Chinese) were screened out: 18 randomized controlled trials(RCTs) and 4 clinical controlled trials(CCTs). Among them, 5 were about the SZG(all RCTs) and 9 were on CXT(6 RCTs and 3 CCTs). The rest 8 focused on JXG(7 RCTs and 1 CCT). Moreover, the overall risk of bias for 94.40% RCTs was evaluated as "some concerns" and only one(5.60%) had high risk of bias. In terms of quality, the 4 CCTs scored 5-6 points(<7 points), suggesting low quality. SZG alone or in combination with tiapride has obvious advantages in improving traditional Chinese medicine syndromes and tic symptoms compared with tiapride alone, with the average daily cost of CNY 79.44-119.16. Compared with conventional western medicine or placebo, CXT alone or in combination with conventional western medicine can improve the total effective rate and alleviate tic symptoms, and the average daily cost is CNY 22.50-67.50. JXG alone or in combination with conventional western medicine can effectively relieve tic symptoms compared with conventio-nal western medicine or placebo, with the average daily cost of CNY 82.42-164.85. The adverse events related to the three Chinese patent medicines mainly occurred in the digestive, respiratory, and nervous systems, all of which were mild. In general, SZG, CXT, and JXG are effective for children with tic disorder. They have been approved to be used in this field, of which SZG was approved in 2019, with the most up-to-date research evidence and high-quality RCT in Q1 journals. However, the comparative analysis of the three was affected by many factors, which should be further clarified. Based on the large sample data available in multiple dimensions, a comprehensive comparative evaluation of the three Chinese patent medicines should be carried out, thereby highlighting the advantages and disadvantages of them and serving a reference for rational clinical use and drug supervision.

[Rapid health technology assessment of four oral Chinese patent medicines in treatment of functional gastrointestinal disorders].

To provide proof of the evidence-based medicine and decision-making information for the clinical decision of functional gastrointestinal disorders(FGIDs), this study evaluated and compared the efficacy, safety, and economy of four oral Chinese patent medicines(CPMs) in the treatment of FGIDs using the method of rapid health technology assessment. The literature was systematically retrieved from CNKI, Wanfang, VIP, SinoMed, EMbase, PubMed, Cochrane Library and ClinicalTrials.gov from the establishment of the databases to May 1, 2022. Two evaluators screened out the literature, extracted data, evaluated the quality of the literature, and descriptively analyzed the results according to the prepared standard. Eventually, 16 studies were included, all of which was rando-mized controlled trial(RCT). The results showed that Renshen Jianpi Tablets, Renshen Jianpi Pills, Shenling Baizhu Granules, and Buzhong Yiqi Granules all had certain effects on the treatment of FGIDs. Renshen Jianpi Tablets treated FGIDs and persistent diarrhea. Shenling Baizhu Granules treated diarrhea with irritable bowel syndrome and FGIDs. Buzhong Yiqi Granules treated diarrhea with irritable bowel syndrome, FGIDs, and chronic diarrhea in children. Renshen Jianpi Pills treated chronic diarrhea. The four oral CPMs all have certain effects on the treatment of FGIDs and have specific advantages for specific patients. Compared with other CPMs, Renshen Jianpi Tablets have higher clinical universality. However, there are problems such as insufficient clinical research evidence, generally low quality of evidence, lack of comparative analysis among medicines, and lack of academic evaluation. More high-quality clinical research and the economic research should be carried out in the future, so as to provide more evidence for the evaluation of the four CPMs.

Efficacy, safety, and cost-effectiveness analysis of aflibercept in metastatic colorectal cancer: A rapid health technology assessment.

Background: Metastatic colorectal cancer (mCRC) imposes a heavy tumor burden worldwide due to limited availability of therapeutic drugs. Aflibercept, a kind of recombinant protein of the anti-vascular endothelial growth factor (VEGF) family, has been approved in clinical application among mCRC patients since 2012. A comprehensive analysis of the efficacy, safety, and cost-effectiveness of aflibercept in mCRC treatment is necessary. Objective: To evaluate the efficacy, safety, and cost-effectiveness of aflibercept for the treatment of mCRC in order to provide a decision-making reference for the selection of targeted drugs for second-line treatment of mCRC in Hong Kong, Macao, and Taiwan regions of China and the selection of new drugs for medical institutions in these regions. Methods: A systematic retrieve on databases including PubMed, Embase, Cochrane Library, China National Knowledge Infrastructure (CNKI), Wanfang, and Weipu, as well as relevant websites and databases of health technology assessment including the National Institute of Health and Clinical Optimization, Centre for Evaluation and Communication at the University of York, and the Canadian Agency for Medicines and Health Technology, was conducted. The literature was screened according to the inclusion and exclusion criteria, and data were extracted and analyzed by two authors, while the quality of the literature was assessed. Results: Finally, we included two HTA reports, 11 systematic reviews/meta-analyses, and two cost-effectiveness studies in the rapid health technology assessment. For mCRC patients receiving second-line treatment, aflibercept combined with FOLFIRI significantly increased progression-free survival (PFS) and overall survival (OS) and the objective response rate (ORR) also improved, compared with folinic acid + fluorouracil + irinotecan (FOLFIRI). In terms of safety, mCRC patients who received aflibercept combined with FOLFIRI therapy had a higher incidence of grade 3-4 adverse events than those who received FOLFIRI alone, including anti-VEGF-related adverse events (hypertension, hemorrhagic events, and proteinuria) and chemotherapy-related adverse events (diarrhea, weakness, stomatitis, hand-foot syndrome, neutropenia, and thrombocytopenia). In terms of cost-effectiveness, two economic studies conducted in the United Kingdom and Japan, respectively, found that compared with FOLFIRI, aflibercept combined with FOLFIRI had no cost-effectiveness advantage in mCRC patients receiving second-line treatment. Conclusion: Compared with FOLFIRI treatment, aflibercept combined with FOLFIRI for the second-line treatment of mCRC patients has better efficacy, worse safety, and is not cost-effective. More high-quality clinical studies are required for further exploration of aflibercept's clinical value. Medical institutions in Hong Kong, Macao, and Taiwan regions of China should be cautious when using or introducing aflibercept plus FOLFIRI as a mCRC treatment.

[Four Chinese patent medicines for regulating stomach for functional dyspepsia: a rapid health technology assessment].

This study evaluated and compared the efficacy, safety and economy of four Chinese patent medicines(CPMs) in the treatment of functional dyspepsia(FD) using the method of rapid health technology assessment. It aims to provide decision-makers with rapid decision-making information. The eight Chinese and English databases were comprehensively and systematically searched for the relevant clinical research. Studies were screened and evaluated. A total of 110 studies were identified, including 95 randomized controlled trials(RCTs), 7 controlled clinical trials(CCTs), 7 systematic review/Meta-analysis and 1 economic evaluation, among which 28 were Dalitong Granules, 49 were Zhizhu Kuanzhong Capsules, 3 were Biling Weitong Granules and 30 were Qizhi Weitong Granules(Tablets/Capsules). The quality of the included literature was generally low. The efficacy of four CPMs alone or combined with western medicine in the treatment of FD is different. Dalitong Granules was used to treat motility disorder in FD. Zhizhu Kuanzhong Capsules and Qizhi Weitong Granules(Tablets/Capsules) can treat FD patients with anxiety and depression. Qizhi Weitong Granules(Tablets/Capsules) were mainly used in FD for perimenopausal patients. There were no serious adverse reactions in the clinical study of four CPMs in the treatment of FD. Dalitong Granules has better effects than mosapride in the treatment of FD, but the cost is slightly higher. The cost-effectiveness ratio of Zhizhu Kuanzhong Capsules in the treatment of FD patients with anxiety and depression was lower than that of Domperidone. In terms of average daily price, Qizhi Weitong Tablets has the highest price(27.00 yuan per day), Qizhi Weitong Granules has the lowest price(5.04 yuan per day), Biling Weitong Granules has a relatively high price(15.53 yuan per day), followed by Dalitong Granules(13.03 yuan per day). The evidence of Dalitong Granules covered the efficacy, safety and economy, which is relatively complete compared with the other three drugs. It has effective potential in the treatment of motility disorder in FD. Further research in this field in the future is needed.

[Scoping review of rapid health technology assessment].

This study aims to summarize the research on rapid health technology assessment(RHTA) based on scoping review, which is expected to serve as a reference for future research on RHTA. First, articles on RHTA were retrieved from both Chinese and English databases. After data screening and extraction by two personnel independently, descriptive analysis was conducted on the results, and evidence distribution was analyzed based on tables, bar charts, line charts, radar charts, and pie charts. Finally, a total of 82 eligible articles were included and the characteristics were as follows.(1) Articles on RHTA were mainly published from 2011 to 2022. The number of articles showed an obvious increase from 2014 and surged in 2019.(2) Among the journals with the above RHTA papers published, Chinese Journal of New Drugs(21.5%), Evaluation and Analysis of Drug-Use in Hospitals of China(16.9%), and Chinese Pharmacy(15.4%) topped the Chinese journals in the number of the papers, while International Journal of Technology Assessment in Health Care(23.5%), Reviews in Medical Virology(11.8%), and Value in Health(11.8%) came out on top in the English journals.(3)The RHTA of drugs(especially western medicine)(68.7%) dominated the eligible articles, followed by the RHTA of therapy technology(13.1%), detection technology(5.1%), and diagnosis technology(1.0%). There was a significant gap in the number of studies among different health technology categories and an imbalance in the types of health technologies involved in the overall RHTA studies.(4) RHTA of tumors has been the research focus in recent years. In summary, RHTA plays a positive role in rapid health decision-making, but there is a lack of primary data sources at present. Compared with the systematic review, Meta-analysis, and pharmacoeconomic studies, a few reports on health technology assessment(HTA) were included. In the future, research on the evaluation of the safety, efficacy, cost effectiveness and social applicability of health technologies should be further strengthened.

[Rapid health technology assessment of four oral Chinese patent medicines for constipation].

This study evaluated the safety, efficacy, and cost effectiveness of Biantong Capsules(Tablets), Maren Runchang Pills, Qirong Runchang Oral Liquid, and Qihuang Tongmi Soft Capsules in the treatment of constipation by the rapid health technology assessment(RHTA) to provide evidence for clinical decision and references for rapid evaluation of Chinese patent medicine(CPM). CNKI, Wanfang, VIP, SinoMed, PubMed, EMbase, Web of Science, and Cochrane Library were searched for research articles from database inception to February 2022. Two reviewers conducted literature screening, data extraction, and quality evaluation according to the predetermined standards. Descriptive analysis of the results combined with visual charts was performed. Sixty research articles were included, involving 44 randomized controlled trials(RCTs), 7 clinical controlled trials(CCTs), 4 systematic reviews/Meta-analyses, and 5 economic analysis studies. As revealed by the results, Biantong Capsules(Tablets) could be used for postoperative and senile constipation, in which some studies reported Biantong Capsules(Tablets) were superior to Maren Runchang Pills and Qirong Runchang Oral Liquid. Maren Runchang Pills were mainly used for senile constipation, and the efficacy was similar to that of conventional wes-tern medicine, but the cost was low and the compliance of patients was good. Qirong Runchang Oral Liquid was indicated for disease-derived or drug-induced constipation, chronic constipation, and senile constipation with fewer adverse reactions. Qihuang Tongmi Soft Capsules had good efficacy and safety in the treatment of functional constipation. Overall, compared with western medicine glycerine enema and lactulose, the number of clinical studies of the four CMPs was small, but they targeted constipation patients with different subtypes. In conclusion, the four CMPs have their advantages and characteristics in the treatment of constipation, but they are restric-ted by sparse existing evidence, low quality of evidence, and insufficient economic research. In the future, more high-quality and long-term follow-up studies should be carried out to obtain reliable evidence. Meanwhile, it is called for strengthening the economic evaluation of CMPs to provide evidence for decision-making.

Helical tomotherapy for cancer treatment: a rapid health technology assessment.

OBJECTIVES: Helical tomotherapy (HT) can be applied to treat complex malignant cancer with high-precise radiotherapy, and it can reduce the damage to normal tissues and improve treatment effects. But the procurement of HT must be approved by relevant departments of administration affairs. This study, appointed by the National Health and Family Planning Commission of China and undertook by the National Health Development Research Center and the Chinese Evidence-Based Medicine Centre, was aimed to rapidly assess the effectiveness, safety, costs, and applicability of HT, so as to provide currently available best evidence for decision-makers of health policies. METHODS: We electronically searched databases including PubMed, EMbase, The Cochrane Library, CNKI, WanFang Data, VIP, CBM, and other professional websites. Two reviewer independently screened literature according to the inclusion and exclusion criteria, extracted data, assessed quality, and then performed descriptive analysis. RESULTS: (i) We finally included 150 studies, encompassing 5 HTAs, 18 CCTs, and 127 observational studies. (ii) The included HTAs were published during 2006-2009, providing fairly less evidence of low quality and the results of 145 primary studies showed that: HT had been used mainly in the treatments of 14 kinds of cancer, with low total toxicity and high survival rates. Although the quality of the included studies was poor, there was much evidence about prostate cancer, head and neck cancer, nasopharynx cancer, cervical cancer, lung cancer and liver cancer, with enough sample and fairly reliable results in HT efficacy and safety. And (iii) a total of 56 clinical trials were registered in Clinicaltrials.gov, most of which were registered by the occident. Among them, 9 were completed but the results had not been published yet. CONCLUSIONS: The evidence of this study showed that, HT is safe and effective in clinic. But the abovementioned conclusion needs to be verified by conducting more high-quality studies with long-term follow-up. The costs of HT in procurement, maintenance, and application are high; and the skills, training, and qualification of operators are required. We suggest that the procurement of HT should be reduced; it should be allocated rationally and effectively used after comprehensive assessment in China's cancer epidemiology characteristics, health resource allocation, disease burden, medical service level, etc.; and also high-quality studies with long-term follow-up should be financially supported on the basis of establishing projects, so as to provide local evidence and consistently guide and improve scientific decision-making.