Analytic Hierarchy Process for Technological Risks in the Process of Innovative Drug Development in China

Objective To identify the critical risks in the process of innovative drug research and development,and to provide reference for improving the efficiency of innovative drug development and risk control in China.Methods Expert investigation and analytic hierarchy process were used to determine the weights of different risks.Results and Conclusion The research and analysis results showed that the risks at different stages of development had different effects on the success rate of drug development,among which the risk at the drug discovery stage influenced the most.In the drug discovery stage,inappropriate target selection had the greatest impact on the success rate of drug development.The lack of appropriate cell tissue or animal models had the greatest impact on the success rate of drug development from the discovery of a compound to the application for clinical trials.The difference in changes between nonclinical and clinical studies had the greatest impact on the success rate of drug development from early clinical studies to pivotal clinical studies.Incorrect dose selection had the greatest impact on the success rate of drug development from pivotal clinical studies to marketing authorization applications.The biggest impact from the marketing authorization application to the approval stage was inadequate communication with regulators.After investigating the weight of risk factors in the process of innovative drug development based on scientific methods,a new perspective for the risk control of new drug development and improving the research and development efficiency is provided.

Research on Reimbursement Management of Innovative Drugs Abroad and Its Enlightenment to China

Objective To analyze the reimbursement policies of innovative drugs in some developed countries,and to provide reference for future reimbursement management in China.Methods Literature research method was used to study the policies related to the reimbursement management of innovative drugs in Germany,France and Japan,and their successful experience was summarized.Results and Conclusion China should establish an open and transparent value evaluation standard to improve the medical insurance reimbursement management of innovative drugs.Besides,the value of innovative drugs should be taken as an important basis for reimbursement decisions,and an independent third-party value evaluation agency must be established.

Identifying and Managing Technical Risks in the Process of Innovative Drugs Development in China

Objective To identify technical risks in the process of innovative drug development,and to provide reference for technical risk management so as to reduce the uncertainties and improve the efficiency of research and development.Methods The initial risk index was investigated by literature research.Then,the Likert scale was used to design a questionnaire,and the experts’opinion was used to analyze the risk factors affecting the different stages of the development of innovative drugs in China.Results and Conclusion Based on the analysis of questionnaire,31 risk indicators of five key stages in the development of innovative drugs from drug discovery to marketing authorization were established.The key risk indicators constructed in this study can provide reference for technology-related risk management in the process of innovative drug development.

Study on an innovative natural drug for Alzheimer's disease reported as a sham

The number of patients with dementia is estimated to be as high as 24 million and is predicted to double every 20 years,i.e.,by 2040,leading to a tremendous financial disease burden[1].Therefore,we urgently need to identify drugs that can prevent,delay the onset,slow the progression,or improve the symptoms of Alzheimer's disease(AD).Drug development for AD has proven to be extremely difficult[2].

Research on Incentive Policies for Chinese Innovative Drug R&D - Taking Innovative Anti-tumor Drugs as an Example

Objective To provide reference for improving Chinese innovative drug research and development incentive policies.Methods Based on investigating the incentive policies for innovative drug research and development in clinical research,evaluation and approval in China,anti-tumor drugs were taken as the research object to discuss relevant policies from the perspective of clinical trials and registration approval based on data statistics and current situation analysis.Results and Conclusion Driven by a series of incentive policies for innovative drug R&D,great achievements have been made on anti-tumor drugs.However,there are problems such as concentration of drug targets,homogenization of clinical trials,and gaps in some drugs with large clinical needs.To improve incentive policies for innovative drug R&D,China should adhere to the orientation of clinical value,focusing on basic research and translational research,improving evaluation and approval capabilities,and establishing a sound ecosystem for innovative drugs.

Inspiration of Foreign Innovative Drug Pricing Methods and Medical Insurance Payment Standards to China

Objective To study the innovative drug pricing methods and medical insurance payment standards in foreign countries and to provide reference for China’s government.Methods The official websites were searched for information and related literature,and literature review was used.Results and Conclusion In foreign countries,the clinical value of innovative drugs and their impact on medical insurance funds were comprehensively evaluated based on factors such as quality-adjusted life years,clinical benefit,and improvement of clinical benefit.Then,the evaluation results were taken as an important basis for whether innovative drugs were admitted to the medical insurance catalog and establishing medical insurance payment standards.By using international experience for reference,innovative drug pricing methods and medical insurance payment standards for China’s national conditions can be improved by establishing a basic database of clinical value and drug economic evaluation of innovative drugs,as well as innovative drug payment models based on decision thresholds.

An Update on the Clinical Pipelines of New Antibacterial Drugs Developed in China

Antibacterial resistance is a global health threat that requires further concrete action on the part of all countries.In this context,one of the biggest concerns is whether enough new antibacterial drugs are being discovered and developed.Although several high-quality reviews on clinical antibacterial drug pipelines from a global perspective were published recently,none provides comprehensive information on original antibacterial drugs at clinical stages in China.In this review,we summarize the latest progress of novel antibacterial drugs approved for marketing and under clinical evaluation in China since 2019.Information was obtained by consulting official websites,searching commercial databases,retrieving literature,asking personnel from institutions or companies,and other means,and a considerable part of the data covered here has not been included in other reviews.As of June 30,2023,a total of 20 antibacterial projects from 17 Chinese pharmaceutical companies or developers were identified and updated.Among them,two new antibacterial drugs that belong to traditional antibiotic classes were approved by the National Medical Products Administration(NMPA)in China in 2019 and 2021,respectively,and 18 antibacterial agents are in clinical development,with one under regulatory evaluation,five in phase-3,six in phase-2,and six in phase-1.Most of the clinical candidates are new analogs or monocomponents of traditional antibacterial pharmacophore types,including two dual-acting hybrid antibiotics and a recombinant antibacterial protein.Overall,despite there being 17 antibacterial clinical candidates,our analysis indicates that there are still relatively few clinically differentiated antibacterial agents in stages of clinical development in China.Hopefully,Chinese pharmaceutical companies and institutions will develop more innovative and clinically differentiated candidates with good market potential in the future research and development(R&D)of original antibacterial drugs.

An innovative approach for the treatment of Alzheimer's disease: the role of peroxisome proliferator-activated receptors and their ligands in development of alternative therapeutic interventions

Alzheimer＇s disease is a multifactorial pathology, for which no cure is currently available. Nowadays, researchers are moving towards a new hypothesis of the onset of the illness, linking it to a metabolic impairment, q-his innovative approach will lead to the identification of new targets for the preparation of new effective drugs. Peroxisome proliferator-activated receptors and their ligands are the ideal candidates to reach the necessary breakthrough to defeat this complicate disease.

药物临床试验培训导入临床药学教学的分析与思考

基于国家大健康产业、医药产业创新发展对临床药学和临床试验人才数量和质量的新需求,初步探讨在临床药学人才教学中导入药物临床试验相关理论、实践的必要性和方式。依据院校临床药学培养教学内容和特点,将临床试验相关理论和实践整合到临床药学培养的不同阶段开展教学,在临床药学基础理论教学中导入药物临床试验理论和案例教学,在临床药学毕业实践中进行临床试验实践教学提升能力,在临床药学研究生培养阶段进行临床试验创新培养。一方面提高临床药学专业学生实践能力,扩大临床试验人才培养路径;另一方面提升临床药学学生的创新思维和能力,满足社会大健康产业多元需求。

新上市药品首发价格形成机制与鼓励高质量创新的经济学理论分析

目的:本研究旨在探究新上市药品首发价格形成机制的经济学本质,及其面临的挑战与待解决的问题。方法:从药品价格形成机制的基本原理出发,本文引入了内生与外生价格、非对称信息博弈、创新药品市场竞争以及新制度经济学的相关理论,探索药品首发价格形成机制的经济学理论基础。结论及建议:建立合理的药品首发价格形成机制,是发挥市场在资源配置中起决定性作用的同时更好发挥政府作用的举措。以鼓励高质量创新为目标的药品首发价格形成机制,应当以降低制度性交易成本为核心目标。建议以创新价值、竞争情况等相关信息披露为基础,对新上市药品合理分类,降低信息不对称程度,治理当前新药市场的逆向选择问题。在保证高质量创新药定价权最大化的同时约束同质化创新的垄断定价权,兼顾效率和公平。

Establishment of an internationally recognized platform for the communication of pharmaceutical researches supported by the National New Drug Innovation Programs ——Experiences and goals of the Journal of Chinese Pharmaceutical Sciences

The initiation of the National New Drug Innovation Key Technology Program marked a strategic shift of new drug discovery in China, which will greatly enhance the new drug discovery research activities. It presents a valuable opportunity for the growth and quality advancement of pharmaceutical science journals. As the only comprehensive English journal of the Chinese Pharmaceutical Association, In the new situation, Journal of Chinese Pharmaceutical Sciences （JCPS） plays a critical role in the communication of new discoveries in pharmaceutical researches to the scientific community. To fulfill the roles as an internationally recognized platform for the communication of the new drug discoveries supported by the National New Drug Innovation Programs, the JCPS editorial office will take advantage of its English language specialty, emphasize quality and digital access, and promote the advancement of the journal.

关注新生血管性年龄相关性黄斑变性的创新药物治疗

湿性年龄相关性黄斑变性(nAMD)是老年人致盲的主要原因,抗VEGF是其主要治疗方法。目前,nAMD治疗仍面临诸多不足和挑战,开发新的治疗药物和给药方法是其主要研究的方向。近年来治疗nAMD的药物研发方向呈现多样化,包括开发新靶点/新机制药物及新型给药方式、优化给药剂量和基因疗法等,达到降低治疗频率、延长治疗间隔、提高患者治疗依从性和长期维持或改善视力的目的。眼科临床医师应对新的药物有较为全面的了解,进而发掘出适合不同nAMD亚型患者的优化方案,实现向精准化医疗迈进。

中外创新药物医保支付的比较研究

按疾病诊断相关分组(DRG)付费是当前我国医保支付改革的重要研究方向,但在一定程度上限制了创新药物的临床推广使用。许多国家已对创新药物的额外支付进行了深入研究。该文以美国、法国、德国为例,分析总结DRG付费体系下创新药物额外支付的国际经验,并结合我国创新药物医保支付现状及DRG付费对创新药物发展的影响,提出了明确的纳入标准、选择合适支付模式、建立动态调整机制、完善支付方式等政策建议,以期在全面推进DRG付费改革中,为进一步建立和完善我国创新药物医保支付机制提供参考。

创新软件和设备在口服外摆拆零药品智能化管理中的应用

目的通过开发智能创新软件结合独立调配桌对住院药房口服外摆拆零药品进行高效、安全的管理和调配,确保药品的安全供应。方法设置独立外摆药品调配操作台,利用创新软件集成掌上电脑(PDA)智能系统,结合条码扫描导入技术与全自动药品分包机,优化和改进口服外摆药品调剂、加药、有效期维护等易错环节。对比创新软件使用前后口服医嘱平均调剂时长(min),外摆拆零药品月均盘存时长(min)和外摆拆零药品有效期维护平均时长(min)。结果使用创新软件和设备后,口服医嘱平均调剂时长从(218.07±6.72)min缩短至(173.81±6.08)min(P<0.01),均值减少44.26 min;外摆拆零药品月均盘存时长从(156.00±5.29)min缩短至(108.00±2.64)min(P<0.01),均值缩短48.00 min;外摆拆零药品有效期维护平均时长从(147.00±2.64)min缩短至(23.00±3.00)min(P<0.01),均值缩短124 min。结论该项创新应用可解决传统外摆拆零药品容易出现的药品混装、定位混乱、库存数量不明、效期管理困难等问题,实现了药品管理的精细化、科学化,有效提高了药师的工作效率和服务质量,同时也为患者提供了更加安全、便捷的用药服务。

同质化教学条件下参与科研创新实践对临床药学专业本科生新药研究与开发课程成绩的影响

大学生参与科研创新实践是高校人才培养的重要环节。本文收集某高校临床药学专业本科生参与科研创新实践的信息,分析同质化教学条件下参加科研创新实践对其在新药研究与开发课程中成绩的影响。结果表明,参加科研创新实践可以有效提升临床药学专业本科生新药研究与开发课程成绩,提高其对基本概念的理解能力和科研实验设计能力。

国际细胞治疗临床研究发展现状及监管政策研究

细胞治疗作为生物医学领域的前沿热点,其快速的创新发展不仅关乎公众健康,更深刻影响国家的生物安全。欧美、日本等发达国家已将此提升至国家战略高度,并构建了完善的药品监管体系以巩固其在细胞治疗领域的领先地位。本文通过统计分析与研究,全面梳理了全球主要国家及地区和我国的细胞治疗临床研究现状,追踪各国监管体系的演变,并系统整理了关键的规范指南。基于此,初步提出了针对我国细胞治疗产品与技术双轨制监管体系的建议,旨在为相关机构、企业及从业人员提供有价值的参考。

药品供应链新模式下异地仓设置实践和思考

目的为药品供应链新模式下异地仓的设置提出可行性建议。方法分析异地仓设置的动因与成效。实地调研3家跨省异地仓药品批发企业,统计3家企业异地仓年销售额、异地仓所在地政府增加的税收、降低的运输成本及减少的药品破损费用。分析异地仓设置现存问题,并提出优化建议。结果3家企业异地仓年销售额分别为400万元、3.72亿元、20亿元,当地政府增加的税收分别为52万元、23万元、1.1亿元;降低的运输成本分别为2万元、100万元、4000万元;减少的药品破损费用分别为1万元、3万元、500万元。结论异地仓的设置,能增加当地政府的税收,降低企业的流通成本,保障药品供应及时,但仍存在药品信息无法完成实时对接,物流成本增加,监管不到位等问题。要合理高效设置,建议整合内部资源,实现多仓协同;优化物流及配送体系,标准化第三方物流;落实企业主体责任,互联升级信息系统;加强异地仓数据监管,统一检查思路。

《中国医药产业高质量发展状况调研(2021-2023)》研究报告综述

目的:了解“十四五”以来我国医药产业高质量发展转轨和运行状况,梳理监管政策在产业高质量发展转轨中的影响和作用,探讨医药产业迈向高质量发展进程中面临的挑战,对进一步深化医药产业高质量发展提出思考和策略建议。方法:通过文献研究、问卷调查、访谈、专家咨询等方式,获取相关政策信息、数据、业界观点等,同时组织专家研讨作为项目研究支撑。结果:我国医药产业积极应对经济下行压力,表现出了良好的产业发展韧性,新药审评上市数量、审评速度与新药临床价值都跃升到新高度,与此同时,中药行业发展表现相对突出,药品制造升级稳步推进,监管科学项目成果丰富。结论:我国医药产业正在以高质量发展转型为方向积极进行深度调整,目前已在研发创新、制造升级、中药守正创新等方面取得了积极进展,同时在研发策略与资金支持、中药传承创新、国际化发展、保障供应链稳定可控等方面仍然存在挑战,建议在进一步转变研发策略、建立多元化筹资体系、完善国际化配套政策、坚持中药特色监管、深化监管科学行动计划、强化监管能力建设、促进“三医”协同等方面发力,有效助推医药产业高质量发展。

医保视角下新药创新性认定与价格管理政策的国际对比分析

目的为我国创新药价格体系的完善提供参考。方法基于我国创新药医保管理现状,对比分析国际典型国家或地区创新药认定与价格管理政策,提出完善我国创新药价格体系的建议。结果与结论中国台湾地区、日本、澳大利亚、德国以及英国对于药品创新性认定与分级、区分定价以及后续价格管理3个环节做法不同,但整体而言,都是主要依据药品的临床价值进行创新性认定和分级,依据分级进行差异化定价,并采用风险分担协议或主动价格调整进行价格管理。基于国内外政策对比,建议我国在创新药管理中进一步明晰创新的内涵与分级,应用多种定价方式促进创新分级管理,探索引入风险分担协议,并进一步构建主动药价调整机制,以完善我国创新药价格体系。

2018-2022年中国和美国上市孤儿药的对比分析

目的比较2018-2022年中国和美国孤儿药的上市情况,进一步了解两国孤儿药发展的趋势和差异形成原因。方法检索国家药品监督管理局、美国食品药品监督管理局获取中国和美国上市的孤儿药信息,并对中美上市孤儿药的数量、类型、药品上市许可持有人、目标疾病和创新药进行分类比较。结果2018-2022年,美国上市426种孤儿药,我国上市64种孤儿药,平均环比增长率分别为-4.81%和8.38%。中国和美国上市孤儿药数量最多的类型均为抗肿瘤药和免疫调节剂类药品,其中美国235种(55.16%),我国15种(23.44%)。在上市孤儿药的目标治疗罕见病类型中,美国最多的是罕见的肿瘤疾病197种(46.24%);我国则是罕见的神经和精神疾病22种(34.38%)。美国上市36种具有突破性治疗价值的首创孤儿药,我国上市40种罕见病治疗的创新药。结论美国的孤儿药上市数量远超我国,但略微呈现下降趋势,而我国则呈现出上升趋势;美国本土孤儿药产业集中度较高,我国孤儿药仍需依赖进口;我国的国产罕见病创新药品数量取得实质性进步,但是与美国仍存在巨大差距,特别是在先进技术创新药研发方面。因此,我国在罕见病新药的研发上还需加大力度,积极参与全球同步研发实现合作共赢。