FDA全力支持靶向疗法的开发!更快!更高效!

新闻FDA全力支持靶向疗法的开发!更快!更高效!

    美国FDA局长Scott Gottlieb博士发表声明,阐述FDA支持更高效地开发靶向疗法的最新努力     近年来,医学界在医疗保健方面经历了转变。医疗创新者不是仅仅关注如何治疗整体疾病,而是正在探索如何针对个体疾病的独特特征,例如人类肿瘤的遗传特征,对症下药进行治疗。这种现代的、有针对性的医学方法的创新已经创造了新的、更有针对性的药物,在某些情况下,可为个别患者量身定制治疗方法。     FDA在推进靶向疗法方面发挥着重要的作用,具体措施是建立一个现代化的框架来确保其提供行业需要的指南和资源,帮助新药开发机构使用新技术有效开发新产品。特别是,FDA需要阐明和扩展现有的途径,使创新者能够根据药物所针对的分子标记物开发产品,而不是更传统的基于疾病表型开发新药的方法。在许多情况下,科学研究揭示疾病的驱动因素实际上是身体内的分子变化。新药的开发基于它们靶向这些分子亚型的能力。同一种分子变化可能是许多不同疾病表型的驱动因素。当药物成功靶向这些分子错误来扭转不同疾病的影响时,我们需要一个新的开发途径,使新药能够以药物靶向的分子标志物为基础获得批准。在肿瘤学领域,这通常被称为不区分组织的药物开发。     通过对产品开发者提供关于监管框架和科学框架方面明确的指导,安全有效的靶向疗法可以通过科学有效的测试被确定,并最终被提供给患者。这就是为什么今天我们要发布两个指导性草案,将为医疗产品开发者提供更多关于FDA对研究和开发下一代个性化疗法的建议的明确信息。     第一个草案指南,“Developing Targeted Therapies in Low-Frequency Molecular Subsets of a Disease”(在疾病的低频率分子子集中开发靶向疗法),解决了寻找疗法这一重要课题,这些疗法针对经常导致或促成疾病的分子变化(例如基因突变),包括罕见的存在于一小部分患者体内的分子变化。     该指南草案以一个更简洁明了的格式提出了一种方法,使得药物开发人员可根据罕见突变的鉴定招募患者参加靶向治疗的临床试验,前提是合理的科学证据表明该药物可能对这些患者有效。该指南讨论了证明特定疾病中各种分子亚型的有效性所需的证据,这可促进对患者可能获益的靶向疗法的更一致的开发和批准。指南中描述的这些科学原则也可以用于支持“不区分组织”药物的开发。这涉及药物如何能够在靶向不同表型共有的分子亚型的基础上获得监管部门的批准,而不仅仅是基于单个疾病状态。     我们认为这两种方法都有可能增加为那些不常见突变的患者寻找可行治疗方案的可能性。最终确定稿后,本指南草案将代表FDA当前的想法。FDA还计划在明年年初发表一篇文章,详细全面地介绍这个问题。     第二草案指南, “Investigational IVD Devices Used in Clinical Investigations of Therapeutic Products”(用于治疗性产品的临床研究的研究性IVD器械),试图为正在进行的临床试验提供清晰的框架,以确定用于治疗性产品研究的体外诊断器械是否必须进行与在研药物相独立的FDA审查。为了开发安全有效的新型靶向治疗,临床试验通常使用研究性或未经批准的IVD来评估生物标志物,并指导对治疗产品或护理策略的选择。     本指南草案在定稿后将阐明用于治疗性产品临床试验的研究性IVD的适当监管途径,这非常重要,新型靶向疗法的试验结果不会因为确定特定生物标志物的诊断试验不符合适当的监管标准而受到牵连。目的是使开发更有针对性的“药物和诊断系统”的过程更有效率。     在今天发布的IVD草案指导的基础上,我们也在考虑如何简化对肿瘤治疗产品和伴随使用的IVD的审查,并计划在不久的将来就此发布指导草案。目标是减少开发机构为开发抗癌药物而承受的负担以及FDA工作人员的负担。     这些指导性草案只是FDA正在进行的推动创新型靶向药物研发和培育个性化治疗方法的几个例子。例如,本月早些时候,FDA发布了指南草案,描述了一个潜在的可以帮助公司在罕见小儿疾病的相同临床试验中合作和测试多种药物产品的新方法,从而减少接受安慰剂治疗的患者数量。我们还概述了如何使用建模和模拟来减少在这些罕见的儿科疾病临床试验中对安慰剂组的依赖。最后,我们特别提出了一种更有效的途径来开发针对罕见小儿疾病戈谢病的药物。我们最近也批准三种新颖的基于测序的新型设备,单一测试可用于检测多种癌症标志物,从而能够开发证据以推动更多的个性化护理管理决策。重磅!FDA改革最新动向:为多臂、多公司临床试验打开了大门!     通过为研发团队的同事们提出简化的方法,FDA希望能够为患者更高效地提供更安全有效的新型靶向疗法。我们期待收到关于今天发布的指导草案的反馈意见,继续致力于协助医学界进一步实现现代化和个性化的护理方法,增加新的医疗技术带来的公共健康益处。详情>>

2017-12-18 00:00:00
个性化药物2017:机遇、挑战和未来

报告个性化药物2017:机遇、挑战和未来

The Personalized Medicine Coalition gratefully acknowledges graduate students at Manchester niversity in North Manchester, Indiana, and at the University of Florida, who updated the appendix of this report under the guidance of David Kisor, Pharm.D., Director, Pharmacogenomics Education, Manchester University, and Stephan Schmidt, Ph.D., Associate Director, Pharmaceutics, University of Florida. The Coalition also acknowledges the contributions of its many members who offered insights and suggestions for the content in the report.展开>><<收起

PMC   2017-06-12 00:00:00
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论文将个性化药物整合入医疗实践的策略

Aim: Research and innovation in personalized medicine are surging, however, its adoption into clinical practice is comparatively slow. We identify common challenges to the clinical adoption of personalized medicine and provide strategies for addressing these challenges. Methods: Our team developed a list of common challenges through a series of group discussions, surveys and interviews, and convened a national summit to discuss solutions for overcoming these challenges. We used a framework approach for thematic analysis. Results: We categorized challenges into five areas of need: education and awareness; patient empowerment; value recognition; infrastructure and information management; and ensuring access to care. We then developed strategies to address these challenges. Conclusion: In order for healthcare to transition into personalized medicine, it is necessary for stakeholders to build momentum by implementing a progression of strategies.展开>><<收起

Future Medicine, 2017, 14(2), 141-152  2017, 14, 2, 141-152
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加拿大政府支持基因组突破性创新研究改善人口健康

新闻加拿大政府支持基因组突破性创新研究改善人口健康

Today, Marc Miller, Member of Parliament for Ville-Marie—Le Sud-Ouest—Île-des-Soeurs, announced $9.1 million in federal support for disruptive innovation in the field of genomics. A disruptive innovation project offers the ability to do things not previously possible. This involves new genomic-based technologies or the application of an existing technology from another field, applied to genomics, that is truly transformative. Funded projects have the potential to displace an existing technology, disrupt an existing market or create a new market opportunity.详情>>

2017-02-28 00:00:00

论文The personalized medicine strategy in drug discovery.

ABSTRACTS OF PAPERS OF THE AMERICAN CHEMICAL SOCIETY  2002, 224, U472-U472

论文The personalized medicine coalition - Goals and strategies

The concept of personalized medicine - that medical care can be tailored to the genomic and molecular profile of the individual - has repercussions that extend far beyond the technology that makes it possible. The adoption of personalized medicine will require changes in healthcare infrastructure, diagnostics and therapeutics business models, reimbursement policy from government and private payers, and a different approach to regulatory oversight. Personalized medicine will shift medical practices upstream from the reactive treatment of disease, to proactive healthcare management including screening, early treatment, and prevention, and will alter the roles of both physician and patient. It will create a greater reliance on electronic medical records and decision support systems in an industry that has a long history of resistance to information technology. Personalized medicine requires a systems approach to implementation. But in a healthcare economy that is highly decentralized and market driven, it is incumbent upon the stakeholders themselves to advocate for a consistent set of policies and legislation that pave the way for the adoption of personalized medicine. To address this need, the Personalized Medicine Coalition (PMC) was formed as a nonprofit umbrella organization of pharmaceutical, biotechnology, diagnostic, and information technology companies, healthcare providers and payers, patient advocacy groups, industry policy organizations, major academic institutions, and government agencies. The PMC provides a structure for achieving consensus positions among these stakeholders on crucial public policy issues, a role which will be vital to translating personalized medicine into widespread clinical practice. In this article, we outline the goals of the PMC, and the strategies it will take to foster communication, debate, and consensus on issues such as genetic discrimination, the reimbursement structures for pharmacogenomic drugs and diagnostics, regulation, physician training and medical school curricula, and public education.展开>><<收起

论文Informed consent and subject motivation to participate in a large, population-based genomics study: The Marshfield clinic personalized medicine research project.

AMERICAN JOURNAL OF EPIDEMIOLOGY  2005, 161, 11, S108-S108

论文Personalized medicine expected to have profound effects on intellectual property strategies

BIOTECHNOLOGY LAW REPORT  2005, 24, 6, 732-732