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出自识林

2016-03-31 FDA Voice

（摘自FDA Voice “Priorities – Teamwork to Achieve Common Goals” 2016年3月30日，作者：Robert Califf，医师，FDA局长。）

随着我就任食品药品管理局（FDA）局长而来的，是充满渴望和谦逊恭谨的机会 — 在这个对国家和公民做出极其重要工作的机构，产生积极的影响。在待批侯任期间，我收到了数百封电子邮件，并与广泛和多元化的利益攸关者团体展开了几乎同样多数量的对话。在这些讨论过程中，浮现出一个反复出现的主题：也就是，切中要害，确定优先领域将成为成功的关键。

这并不出乎意外。FDA监管约占美国经济总量20%的领域，面对数量如此庞大的选项，很容易在纷繁复杂的事务中迷失。事实上，我一直受到待办事项清单过分冗长的（正当）指责。但是，通过与FDA这里知识渊博、敬业、具有使命感众多人士的相互交流，有助于促进清晰、实事求是、重点突出的轻重缓急意识，有助于进一步提升这一出色的机构业已存在的实现其雄心勃勃的目标的极大热情。

FDA以非常有效和勇于担当的方式做出决策。在保护和促进公众健康的使命指引下，由在团队体系审评相关事项的专职和专业人才的共同努力支持下，构成了我们组织核心的FDA的多个中心每天都能够做出大量决策。这些决策中的绝大多数的，其中有许多对全体美国人的福祉至关重要，可能是由专业化所支持的体系与名至实归的高品质和公正判断的声誉所做出的决策 — 尽管许多决策最终肯定会令一个或多个团体感到失望（或至少不能完全满足）。

我坚信，我在FDA期间最重要的责任，是鼓励和支持专业的环境，使我们非常敬业的员工队伍茁壮成长，并充分发挥其潜力。生物技术和信息科学的巨大进步，以及不断加速的全球化趋势，正在迎来一个快速变革的时代。但是，面对这一变革，FDA在完成其使命的成功的关键在于保持弥久恒新 — 维持和扩大我们的人才队伍，在继续加强我们的环境，确保留住现有员工的同时，确保我们招聘到我们所需要的面向未来的人员。为此，我将继续贯彻雇员计划，这一计划旨在 1) 完善聘用制度，2) 确保FDA的工作人员具备尽可能最佳的工作条件，以及 3) 培育针对不同行业与专业的专业人才基地，补充我们的团队，以便我们能够在激烈竞争的市场上招募到和留住他们。

考虑到目前在社会上涟漪般激荡传播的惊人变化，我最优先考虑的方案将可能不会出人意外：我们必须尽一切可能迅速适应循证时代的国家和全球体系，以迎接技术进步带来的挑战和抓住机遇。这究竟意味着什么？我一直注意到，在具有高品质证据的情况下，FDA的科学决策往往是直截了当的。但在没有最优信息的情况下，在必须做出科学决策的情况下，对于FDA尤其具有挑战性。在这样的情况下，意见可能会具有更大的权重，在FDA内外产生纷争的可能性增加，我们无法充分保护或促进患者和公众福祉的风险更大。

FDA是一家基于科学、以科学为指引、专注于患者和消费者需求的机构；保护患者和消费者的福祉是FDA作为公共卫生机构的职责。由于关乎患者和公众需求与选择的了解，科学技术发展水平发展迅速，因此，我们必须通过整合最佳方法，在我们工作中的每个部分都顾及到患者喜好、体验和结果，与时俱进。

生物医学正在靠近一个临界爆发点，具备支持我们做出决策的高品质证据知识很可能会呈指数式增长。在国家层面上，我们已经投入超过500亿美元为几乎每一个美国人都建立了电子病历（EHR）。此外，计算存储容量与计算能力逐年呈级数递增。同时,社交媒体的出现和广泛扩散，正在使得以前所未有的规模与患者和消费者直接沟通成为现实。在诸如哨兵计划（Sentinel）和国家医疗器械评价体系与其它政府机构及政府以外的组织的互补计划相衔接的情况下，我们能够（我相信在短期内能够实现！），为私立与公共部门产生多得多的有用知识的体系建立起一个强大、稳健、可靠的基础，这些工作的成本只是之前所需成本的几分之一。事实上，FDA的一项主要职能就在于支持持续开发有效的证据生成体系，因此，私立与学术部门能够在这方面有所作为。

因此，FDA正在全面致力于与我们的生态系统中的众多合作伙伴一起帮助建立和维持生成高品质科学证据的基础，对于指导FDA就负责监管的药品、医疗器械、烟草制品、食品与消费品，以及医疗服务提供机构、患者和消费者就其健康和福祉做出决策，这样的基础是必需的。

除了这个首要的优先事项之外，今早和可预见的将来的其它优先考虑事项还包括：

• 镇痛类药品。目前，阿片类药品过量使用造成的死亡数超过车祸。仅凭FDA自身，并不能解决这一问题 — 事实上，也没有哪家机构能够凭一己之力做得到 — 但是，正如FDA阿片类药品行动计划中所阐述的，我们一直在发挥着关键作用。
• 烟草制品认定。在制定监管范围广泛的烟草制品路径框架方面，已经做了很多工作。 FDA正在努力最终制定认定规则，在提出的版本中，将FDA的监管扩展至几乎所有的烟草制品，包括电子香烟、所有雪茄或除顶级雪茄之外的所有雪茄、斗烟、某些不属于“无烟烟草”的溶出物，凝胶和水烟。
• 贯彻FDA食品安全现代化法案（FSMA）。这项将改变食品安全体系的法令正在贯彻中，已颁布了多项重要的法规，将会颁布更多法规。这项工作涉及到综合制定涵盖整个食品安全体系的新的受控和基于风险的体系。这一体系的有效实施，将需要运用尖端的分析和生物科学，以及最先进的人性系统管理方法。
• 抗生素耐药性。对多重耐药病原体（multidrug-resistant pathogens）扩散的担忧,以及应对这一威胁的药品研发管道的可持续性的担忧持续增长。我们在联邦政府计划中肩负主要责任，这一计划涉及到FDA的多个部门，需要FDA以范围广泛的生态系统合作，在有效的管理框架内，既要确保农场合理使用适用的抗生素，还需要确保研发、审批和负责任地使用新型抗生素。
• 跨部门效能。认识到FDA保护和促进公众健康的使命，以及针对使用药品的患者和消费者的获益和风险平衡，通过跨政府部门间的协调努力，能够在很大程度上加强我们的成功。因此，我们将继续FDA-NIH（食品药品管理局-国立卫生研究院）联合领导委员会和FDA-CDC（食品药品管理局-疾控中心）会商，同时启动与CMS（医疗保险和医疗补助服务中心）之间的磋商。生物标记物、终点和其它工具（BEST）资源成为FDA与NIH之间协力解决科学与监管议题能力的有力例证。
• 精准医疗。奥巴马总统签署的精准医疗动议（Precision Medicine Initiative）的意义远超出一个计划的范围。相反，精准医疗动议为清晰地了解生物医学与农业的未来视景提供了一个窗口，借助强有力的新技术和方法，根据实现提升健康产出所需的尺度范围，综合运用遗传学、基因组学、临床、社会、环境数据干预，实现精准定位。
• 跨领域议题。我所关注议题的列表中，还有其它大量议题（实话实说，达到三位数）。这些议题贯穿FDA上下，包括优化针对组合产品、医学对策、改进产品标签说明等，这些议题将得益于大家的关注和支持。

仅凭一篇简单的介绍博文，并不适合详细阐述多个优先事项或多项单独的计划。然而，我希望自己已经表达了对眼前工作的热忱和信心，我们将能够在许多关键领域实现真正和持久的改进。促进有效的沟通交流，对我而言，是极为重要的首要优先事项，因此，我承诺，我将以频繁的更新跟进。苟日新，日日新，又日新。期待与大家在这里很快见面。

翻译：识林-Kapok
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Priorities – Teamwork to Achieve Common Goals
Posted on March 30, 2016 by FDA Voice
By: Robert M. Califf, M.D.

With my appointment as Commissioner of Food and Drugs comes a rare and humbling opportunity—to make a positive difference at an institution that does vitally important work for the nation and its citizens. During my vetting process I received hundreds of emails and had almost as many conversations with a large and diverse group of stakeholders. Over the course of these discussions, a recurring theme emerged: namely, that setting priorities would be critical to success.

Robert M. Califf, M.D., Commissioner of the U.S. Food and Drug AdministrationThis is hardly surprising. FDA regulates about 20 percent of the nation’s economy and, given the vast number of options, it would be easy to get lost in an overwhelming swirl of activity. In fact, at times I have been (rightfully) accused of having an excessively lengthy to-do list! But my interactions with so many of the knowledgeable, dedicated, and mission-driven people here at FDA have helped foster a clear, realistic, and focused sense of priorities and have further heightened an already strong enthusiasm for helping this awesome organization reach these ambitious goals.

FDA makes decisions in a remarkably effective and responsible way. Guided by the lodestone of our mission to protect and promote the public health, and supported by the concerted efforts of dedicated and talented professionals who examine issues within team-based systems, FDA’s Centers that form the core of our organization are able to make an enormous number of decisions every day. The vast majority of these decisions, many of which are vital to the well-being of all Americans, are made possible by a system sustained by professionalism and a well-earned reputation for high-quality and impartial judgments—despite the fact that many decisions must ultimately disappoint (or at least not fully satisfy) one or more constituencies.

I strongly believe my most important responsibility during my time at FDA is to encourage and support a professional environment that enables our remarkably dedicated workforce to thrive and to reach its fullest potential. Dramatic advances in biotechnology and information sciences, as well as continuously accelerating trends toward globalization, are ushering in an era of rapid change. But amid this change, the key to success for the Agency in accomplishing its mission remains constant—sustaining and expanding our talented workforce and ensuring that we both hire the people we need for the future while we continue to enhance our environment to ensure that we retain existing staff. To that end, I will pursue a workforce initiative designed to 1) improve the hiring system, 2) ensure that the Agency has the best possible working conditions for staff, and 3) foster professional homes for the diverse professions that make up our teams so that we are able to recruit and retain them in a very competitive market.

My top programmatic priority will likely come as no surprise, given the astonishing changes that are currently rippling through society: we must do everything possible to rapidly adapt our national and global systems of evidence generation to meet the challenges and opportunities presented by technological advances. What does this mean? I’ve noticed that when high-quality evidence is available, FDA’s scientific decision making is often straightforward. But it can be particularly challenging for the Agency when it must make scientific decisions in the absence of optimal information. In such cases, opinions may carry greater weight, and there can be an increased likelihood of dissension both inside and outside of FDA, as well as a greater risk that we may fail to most fully protect or advance the welfare of patients and the public.

FDA is a science-based, science-led organization that focuses on the needs of patients and consumers; protecting their well-being is our charge as a public health agency. The state of the art as it pertains to understanding the needs and choices of patients and the public is progressing rapidly, and we must continue to keep pace by incorporating the best methods for taking patient preferences, experiences, and outcomes into account in every part of our work.

Biomedical science is nearing a tipping point where the amount of high-quality evidence available to support our decisions is likely to increase exponentially. As a nation, we have invested over $50 billion to provide an electronic health record (EHR) for almost every American. Further, computational storage capacity and analytical power are increasing by orders of magnitude from year to year. At the same time, the advent and wide diffusion of social media are enabling direct communication with patients and consumers on an unprecedented scale. When projects such as Sentinel and the National Medical Device Evaluation System are linked with the many complementary initiatives under way at our sister agencies and at organizations outside of the government, we can (and I believe in short order will!) build a robust foundation for a system in which both private and public sectors can produce much more useful knowledge at a fraction of the cost such efforts have previously required. Indeed, a major function of FDA is to support the continued development of an effective system for evidence generation, so that the private and academic sectors can make it happen.

Accordingly, FDA is thoroughly committed to working with the many partners in our ecosystem to help build and sustain an infrastructure that produces the high-quality scientific evidence needed to guide FDA’s decisions about the drugs, medical devices, tobacco products, and food products it’s charged with regulating, as well as the decisions that healthcare providers, patients, and consumers make about their health and well-being.

In addition to this overarching priority, a number of specific critical issues are on my front burner this morning and will remain there for the foreseeable future:

• Pain. The present epidemic of opioid overdose deaths now exceeds deaths from automobile crashes. FDA cannot solve this problem on its own—and indeed, no single entity can—but we have a critical role to play, as described in our FDA Opioids Action Plan.
• Tobacco product deeming. Much effort has gone into developing the framework for the approach to the regulation of the broad array of tobacco products. FDA is working hard to finalize the deeming rule, which in its proposed form would extend FDA regulation over virtually all tobacco products, including electronic cigarettes, either all cigars or all but premium cigars, pipe tobacco, certain dissolvables that are not “smokeless tobacco,” gels, and waterpipe tobacco.
• Implementation of the FDA Food Safety Modernization Act (FSMA). This statutory directive to transform the food safety system is well on its way to being implemented, with critical regulations issued and more to come. The effort involves the complex development of a new control and risk-based system that includes the entire chain of food safety. Effective implementation of this system will require the application of cutting-edge analytical and biological science, as well as the most modern approaches to human systems management.
• Antimicrobial resistance. Concerns about the proliferation of multidrug-resistant pathogens, as well as the sustainability of the product pipeline needed to meet this threat, continue to grow. We have a major responsibility in the federal plan, one that will involve many parts of the Agency and require that we work with the broad ecosystem, both to ensure that appropriate antimicrobials are used appropriately on farms, and that novel antimicrobials are developed, approved, and used responsibly within a framework of effective stewardship.
• Interagency effectiveness. When we consider our mission to protect and advance the public health, as well as our duty to balance benefit and risk for patients and consumers of medical products, much of our success can be enhanced by coordinated effort across government. We have therefore continued the FDA-NIH Joint Leadership Council and the FDA-CDC meetings, and also initiated similar discussions with CMS. The Biomarkers, Endpoints and other Tools (BEST) Resource offers a powerful example of the ability of FDA and NIH to contribute to solving scientific and regulatory issues together.
• Precision Medicine. President Obama's Precision Medicine Initiative represents more than just a project. Rather, it is a window that provides a clear view of the future for biomedicine and agriculture, a future in which powerful new technologies and methods allow the precise targeting of interventions using an array of genetic, genomic, biological, clinical, social, and environmental data according to the scale needed to achieve improved health outcomes.
• Cross-Cutting Issues. There are a great many other issues (truthfully, the number reaches triple digits) on my list of concerns. But those issues that cut across the Agency, including optimizing our approach to combination products, medical countermeasures, and improving product labeling, will benefit most from my attention and support.

A single introductory blog post is not suited for giving details about priorities or individual programs. However, I hope I’ve conveyed my enthusiasm for the work at hand, as well as my confidence that we will be able to make real and lasting improvements in many critical areas. I promise that we will follow up with frequent updates, as fostering effective communication is itself an overarching priority of immense importance to me. So expect to hear from me again soon!

Robert M. Califf, M.D., is Commissioner of the U.S. Food and Drug Administration