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OBJECTIVE To prov ide reference for improving the participation mechanism of stakeholders in the process of medical insurance negotiation for oncology drug in China. METHODS Based on the stakeholder theory ，combined with literature research，case analysis （taking the review of reimbursement of Bentuximab as an example ）and other methods ，analysis and research were conducted on the Canadian oncology drug review process and the participation mechanism and role of stakeholders. The suggestions were put forward for our country. RESULTS & CONCLUSIONS Canadian oncology drug reimbursement review process was composed of four stages ：the pre-submission planning stage ，the formal submission stage of application，the review stage，and the stage of forming reimbursement recommendations. As the role of stakeholders ，drug manufacturers ，patient representative advisory group ， clinical review expert advisory groups and provincial advisory groups participated in the reimbursement review process of oncology drug by providing suggestions and feedback to CADTH. The participation of stakeholders had improved the transparency of the review of oncology drugs in Canada and made the reimbursement results of oncology drugs more scientific ，reasonable and accurate. In China ，it is recommended to define rights ，responsibilities and interests as well as the participation mechanism of stakeholders in the medical insurance negotiation process ，attach importance to the role of patients in the medical insurance negotiation process of oncology drug ，improve information disclosure and increase the transparency of the negotiation mechanism and process so as to increase the participation of stakeholders.
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Objective:To observe the efficacy of methimazole (MMI) combined with 1α-hydroxyvitamin D3 (alfacalcidol, ALF) in patients with Graves disease of high-titer thyrotropin receptor antibodies (TRAb) and to explore new clinical strategies to reduce serum TRAb in Graves disease.Methods:120 patients with Graves disease initially diagnosed in Quanzhou First Hospital Affiliated to Fujian Medical University and the People′s Hospital Affiliated to Quanzhou Medical College from June 2017 to June 2019 were prospectively selected as the research objects. All patients received conventional dose of MMI for anti hyperthyroidism treatment. The patients were randomly divided into three groups: group A [ n=40, treated with MMI combined with high-dose ALF (0.5 μg/d)], group B [ n=37, treated with MMI combined with low-dose ALF (0.25 μg/d)] and group C ( n=43, treated with MMI only). The treatment lasted for 24 weeks. The serum free triiodothyronine (FT 3), free thyroxine (FT 4), thyroid stimulating hormone (TSH) and TRAb in patients before and after above treatments were detected. The blood routine, liver function, alkaline phosphatase (ALP), 25(OH)D, serum calcium (CA) and serum phosphorus were detected regularly. Results:After drug treatment: ⑴ the thyroid function of the three groups returned to normal. The average daily dosage of MMI in group A was significantly lower than that in group B and C ( P<0.05), and that in group B was also lower than that in group C ( P<0.05), with significant difference. After 24 weeks of treatment, the daily dosage of MMI in group A and B was significantly lower than that in group C ( P<0.05). ⑵ There was no significant difference in thyroid function among the three groups. The concentration of serum TRAb in group A was significantly lower than that in group B and C ( P<0.05), and that in group B was also lower than that in group C ( P<0.05). ⑶ During the 24 week follow-up, there was no significant difference in serum 25(OH)D, ALP, Ca and P among the three groups ( P>0.05); no leukopenia in peripheral blood and no abnormal liver function were found in the three groups. Conclusions:MMI combined with ALF can effectively treat Graves′ disease, reduce the dosage of MMI drugs, decline the level of TRAb in the serum of Graves′ patients, and improve the prognosis of Graves′ disease.
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OBJECTIVE：To eval uate the effectiveness ，safety and economy of chimeric antigen receptor T cells （CAR-T） therapy for the treatment of B-lymphoblastic hematologic malignancy ，and to provide evidence-based reference for clinical decision. METHODS：Rapid health technology assessment （HTA）was adopted. PubMed ，Embase，Cochrane Library ，CNKI，Wanfang databases and foreign HTA official websites were systematically searched during the inception-Mar. 20th，2021. After inclusion ， data extraction and quality evaluation of literatures according to the inclusion and exclusion criteria ，descriptive analysis was performed for the effectiveness ，safety and economy of CAR-T therapy for the treatment of B-lymphoblastic hematologic malignancy. RESULTS ：A total of 2 HTA reports ，5 systematic reviews/Meta-analysis ，and 5 economics studies were included. In terms of effectiveness ，CAR-T therapy showed good efficacy in the treatment of B-lymphoblastic hematologic malignancy ；overall remission rate （ORR）of CAR-T therapy in the treatment of acute lymphoblastic leukemia was more than 63.5％，and the complete remission rate （CR）was 77.1％（95％CI：62.8％-87.1％）；ORR of CAR-T therapy in the treatment of chronic lymphoblastic leukemia was 70.0％（95％CI：53.0％-80.0％），and the CR was 25.5％（95％CI：13.9％-42.1％）；ORR of CAR-T therapy in the treatment of B-cell lymphoma was more than 44.4％. In terms of safety ，the incidence of cytokine release syndrome was more than 20％ during the treatment of CAR-T therapy ，and 1/3 or more （9％ believed in some studies ）patients suffered from neurotoxicity ； the incidence of infection was 12.2％-33.3％，and the incidence of graft-versus-host disease was 23.4％（95％CI：8.6％-49.8％）. In terms of economy ，most of the included studies believed that CAR-T therapy possessed economic advantages ，which were the results of evaluation in developed countries such as the United States and Japan. CONCLUSIONS ：CAR-T，as a new product of treatment for hematological malignancy ，shows good effectiveness and low level of ADR ，which is basically controllable ；its economy needs to be further evaluated by relevant researches combined with domestic reality.