·我国创新药研发模式与价值评估(II)

创新药研发对企业研发能力要求高,目前我国大部分药企仍然处于仿创阶段,但随着政策环境的改善,国家不断释放鼓励创新信号, 临前所未有的挑战,呼唤历史发展的转折点。 3.1 由仿到创的大型创新药企——1.0模式 2015 年临床数据自查、仿制药一致性评价、化学药 国内当下的政策环境和市场环境,使得我国创新 品注册分类改革工作方案、上市许可人制度试点、药监市 朱迅 创新型药企不断涌现,传统药企积极布局,创新药物迎来发展机遇。借鉴国外创新药研发经验,探讨我国创新药的 3 种研发模式及估值方法, 解析创新药研发的机遇与风险。     

2023年新药研发发展现状与未来趋势

新药研发是制药行业的核心活动之一,成功发现和开发对疾病具有疗效的新药物需要跨学科的合作、大量的投资和对创新的不断追求。近年来全球新药研发竞争格局不断加速演变,我国众多创新药企寻求深层次变革与转型发展,并加快拓展海外市场。2023年,全球获批上市的创新药数量持续增长,同比增长17%,临床研究管线数量亦不断扩大;生物技术和基因疗法的发展日新月异,罕见病药物研发逐渐增多,免疫疗法领域研发取得显著成果。本研究从创新药物的类别和研发新格局等角度出发,对2023年新药研发发展态势进行梳理与总结,并展望未来发展趋势。     

第三届中国医药高新技术交易会(CPHTF’2003)

面对入世后世界医药经济、科技格局的明显变化 ,以及我国《药品注册管理办法》的全面推行 ,国际、国内医药经济和技术市场发生了重大转变 ,同时也呈现出更大的机遇与挑战。随着我国大多数制药企业GMP建设临近尾声 ,药企对新技术新产品的需求越来越迫切 ,同时国外许多新药研发机构正积极寻求与中国医药企业和研发机构的深层合作。为此 ,国家食品药品监督管理局中国医药国际交流中心决定举办”第三届中国医药高新技术交易会 (The 3rdChinaPharmaceuticalHigh techFair ,CPHTF)。在总结以往各届交易大会经验的基础上 ,本届交易会将更加突…     

心血管领域复方创新药的范例：缬沙坦沙库比曲片 与依那普利叶酸片 ——给我国新药创制带来的启示

缬沙坦沙库比曲片与依那普利叶酸片均属于复方创新药,即其中单药在组成复方后的疗效或适应证发生新的变化。针对临床需求、 疗效以及循证依据,详细剖析这两种复方创新药的组方特点,重点探讨复方创新药与一般复方药的区别,为以临床价值为导向和基于患者 人群特点的重大新药创制提供借鉴。     

2022年新药研发发展态势

新药研发作为医药产业中的重要环节,是医药产业发展升级的关键环节。近几年,源头技术的突破、多学科交叉整合的创新,促成了新药研发的快速发展。2022年以来,全球新药研发模式加速演进,获批上市的新药类别不断丰富,抗体药、细胞与基因治疗药物快速发展,一批重磅新药的上市给复杂疾病的诊治带来了新希望。本文从获批上市药物类别、新技术驱动新药开发等视角出发,对2022年新药研发的进展进行了综述,以期为后续研究提供参考。     

从创新药物申报角度探讨中美新药评审制度的异同

创新药物研发是国家医药产业发展的原动力,美国作为全球新药研发能力最强的国家,这与其食品药品管理局在新药审批过程中给 予的技术支持和政策鼓励密不可分。通过分析比较我国与美国的新药评审相关政策的异同,学习和借鉴美国的成功经验,为我国创新药物 注册审评制度的调整与完善提供参考。     

对促进新药研发途径的思考

在创新的重要性日益被全社会所认同的大背景下,如何促进我国新药研发正受到越来越多的关注。促进新药研发的途径可从不同的方面进行研究,本文则主要以目前存在较大争议和分歧的“允许创新药物进入医保目录是否是促进新药创新的有效途径”这一问题为切入点进行探讨。     

浅议我国医药产业研发现状及未来发展建议

目前世界医药产业不断发展,逐渐形成了少数企业少数产品带来多数利润的发展新格局。我国医药产业与发达国家相比,整体处于大而不强的阶段。少数企业逐步壮大。多数药企研发投入严重不足。本文针对目前中国医药产业与研发的现状提出了自主研发"重磅炸弹"药物的方针。并从抓住创新时机、提高创新能力、走向国际竞争、培育创新环境四个方面给自主研发"重磅炸弹"药物给出建议。     

创新药物研发的前沿动向与中国创新药物的发展近况

生命科学研究技术和手段是医学发展的重要基础。近年来,基因编辑、肿瘤免疫治疗、嵌合抗原受体T细胞免疫疗法、液体活检、大数据平台、再生医学、合成生物学、人工智能、3D打印等新策略和新技术的不断涌现,引领和推动了药物研发,产生了一批重要成果。精准医疗研究方向的兴起,要求临床过程中有精细的诊断,能够针对病人不同的情况来研发和精准用药。对创新药物研究的上述前沿动向作了概述,并回顾了中国从"九五"到"十二五"近20年新药研发取得的成就。"十三五"期间国家重大科技专项将重点聚焦药物品种研发、重大关键技术的研究与核心创新平台能力的建设。目前,中国新药研发存在的问题主要是原始创新能力不强、企业总体实力欠缺、顶层设计有待完善。面向新形势、新任务,中国新药研究要更多地针对新靶点、新机制,向着研制"同类第一"或"同类最优"的原始创新药物目标努力奋进。     

基于Delphi法的中国生物制药行业技术发展趋势预测分析

运用传统的技术预测方法---Delphi法,通过组织“我国生物医药技术发展趋势预测”专家调研,对我国生物制药行业的技术发展方向进行了预测分析,目的是从技术发展的角度给出生物制药行业投资机会分析的一些可供借鉴的建议。调研结果显示,我国生物药品的研发应采取跟踪模仿与创新相结合的模式,进行模仿性、延伸性的新药研发,重点开发肿瘤药物、疫苗和诊断试剂等,重点开发针对恶性肿瘤、心脑血管疾病及感染性疾病(炎症)等病症的药物;目前影响我国生物药品技术发展的主要因素包括宏观政策、市场需求、资源特性、新药审批制度等;在未来生物药品新药研发过程中最有可能被采用的新技术、新方法包括细胞凋亡机制、计算机辅助设计及人类基因组研究等。     

新冠病毒抗体药物研发进展及展望分析

抗体药物以其独特的作用机制和靶向性强、特异性好等优点,在恶性肿瘤、自身免疫性疾病、感染类疾病的诊断和治疗中发挥着越来越重要的作用,成为国际创新药物研发的热点。新冠肺炎(COVID-19)疫情发生以来,国内外多家研究机构和企业正在加快推进新冠病毒(SARS-CoV-2)抗体药物的开发。在此情势下,认真分析抗体药物现状和趋势,梳理国内外新冠病毒抗体药物研究进展,明确我国当前抗体药物创新的机遇、挑战和建议,对加快我国药物自主创新研发具有重要意义。     

Needs for new plant-derived pharmaceuticals in the post-genome era: an industrial view in drug research and development

Plant metabolites have been the successful source of drugs and provided considerable value not only to the pharmaceutical industry but also to human health problems. Although pharmaceutical companies significantly decreased their activities in natural product discovery during the past few decades, various multidisciplinary approaches have been made to create new opportunities for finding innovative plant derived pharmaceuticals in post-genome era. Strategies to integrate the knowledge on medicinal plants into rational drug screening, the unique biodiversity of plant metabolites into random drug screening, and the chemical diversity of plant metabolites into combinatorial chemistry have been reviewed with concrete examples. Innovative biotechnologies in plant cell and tissue cultures, and the latest achievements in metabolic engineering and genetic modification should significantly improve the production sustainability and efficiency of plant-derived pharmaceuticals.     

The changing landscape of antiparasitic drug discovery for veterinary medicine

Changes in economic imperatives in the pharmaceutical industry have led to a wave of consolidation, which has had the unintended side effect of shrinking the resource devoted to antiparasitic drug discovery in animal health companies. Scientific changes have altered the way in which drugs could be discovered in the future. New science and business models will need to be implemented to address the demand for innovative antiparasitic drugs in veterinary medicine. Novel drugs are needed to combat drug resistance and for currently non-addressed problems. At the center of the future for this field, however, lies the need for more support into the basic research on the biology of parasites.     

细胞培养工艺条件对抗体异质性影响的研究进展

抗体药物具有靶向明确、副作用小等优势,近年来受到国内外药企的广泛关注。然而,动物细胞大规模培养和抗体质量分析已然成为我国的抗体药物产业化的主要限制因素,尤其是细胞培养工艺条件对抗体异质性的影响非常显著,如何有效通过优化工艺条件来满足抗体药物开发要求成为迫在眉睫的问题。文中简要综述了近年来细胞培养工艺条件对抗体异质性影响的技术进展,并对未来国内抗体药物开发作了展望。     

Funding translational work in cell-based therapy

The cell therapy branch of the regenerative medicine field has been innovative in developing new models of delivery and development and identifying alternative sources of funding. We discuss the implications of these changes for pharmaceutical companies and the opportunities they offer to a new entrepreneur.     

African bovine trypanosomiasis: the problem of drug resistance

The three trypanocides used to control tsetse-transmitted trypanosomiasis in domestic animals in Africa have been in use for over 40 years and, not surprisingly, resistance of trypanosomes to these drugs has emerged. Because of the relatively limited market in Africa and the high costs of developing and licensing new drugs, international pharmaceutical companies have shown little interest in the development of new trypanocides for use in either animals or humans. Therefore, the current challenge is to achieve optimal use of the relatively old existing drugs, and it is in this context that the problem of drug resistance has to be quantified--as discussed here by Stanny Geerts, Peter Holmes, Oumar Diall and Mark Eisler.     

The next generation of PPAR drugs: do we have the tools to find them?

Agonists of PPARalpha and PPARgamma are currently approved for use in treating, respectively, dyslipidemia and type 2 diabetes. Agonists of PPARbeta/delta are currently in development by several pharmaceutical companies. Despite their therapeutic importance, there are dose limiting side effects associated with PPAR drug treatments, thus a new generation of safer PPAR drugs are being actively sought after. In this review we will discuss the side effects associated the PPARs, how the current drugs in clinical development were discovered and new concepts in how to screen for PPAR drugs.     

Strengthening the Case for Epilepsy Drug Development: Bridging Experiences from the Alzheimer’s Disease Field—An Opinion

Given the sheer number of drugs (over 20!) available for treatment of seizures, epilepsy can be considered one of the most successful areas in pharmaceutical development and especially for neuroscience. However, despite the large number of drug treatment options available for managing patients with epilepsy, there remains considerable unmet need. For example, the overall impact on seizure control has not been substantial with approximately 30% of patients remaining refractory or their seizures not adequately controlled. Also there is need for epilepsy prevention and for certain sub-populations with severe intractable epilepsy. High unmet need often drives new industry investment into therapeutic market opportunities, however the profound success of antiepileptic drugs has contributed to the hurdles for industry investment in new therapies for epilepsy. Furthermore, the payor environment has also changed with new challenges for evidence generation and demonstration of additive value above existing standard of care treatments. Challenges in translational science, in the clinical trial environment including cost and operational technical difficulty, and in the commercial environment have resulted in the pharmaceutical industry directing investments away from epilepsy into other therapeutic areas such as oncology and immunology as opportunities for higher probabilities of success and returns of investment. The neuroscience area in general is perceived a high risk area and a notable exception has been the active industry involvement in Alzheimer’s disease (AD), especially for therapeutics that could modify the course or prevent AD. AD is a very high risk area with no successful efficacious treatments found to date despite recent failures, there remains promise that therapies are forthcoming. The promise is fueled by a number of innovative factors that reduced R&D challenges in the AD field and contributed to a high level of drug development activity and investment. This paper addresses hurdles facing epilepsy drug discovery and development and focuses on some key solutions that could be eased to facilitate industry interest. Similarities in drug development challenges provide opportunities that bridge experiences and learnings from AD to epilepsy. Overall, the epilepsy field is probably in a good position for advancing into the next generation therapeutics of antiepileptic drugs targeted for increased efficacy in refractory epilepsy and for antiepileptogenesis.

     

医药 CRO 专题报告——蓝海市场，强者恒强

随着全球制药企业研发投资成本加大、研发周期变长、研发成功率降低,作为社会分工专业化的产物,CRO 企业凭借其低成本、高效率、 多服务的特点,快速发展,且服务范畴已涵盖药物研发的整个过程,成为医药研发产业链中不可缺少的环节。报告采用文献调研、数据库检索、 数据统计与分析等定性定量研究方法,从发展概况、发展策略、竞争格局、企业布局等角度对国内外医药 CRO 领域进行多角度、多层次的 分析,旨在为相关企业确定产品研发思路、制定市场策略提供线索和参考。