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出自识林

2015-03-17 FDA Voice

Margaret A. Hamburg, M.D.
FDA Voice 2015年3月17日

3月10日，我有幸与我的同事Francis Collins博士在健康、教育、劳工和养老金参议委员会前对“继续美国为患者在医疗创新方面的领导力”问题的听证会作证。我想更广泛的公共卫生团体会对我的口头证词感兴趣，因而我在此分享给大家：

“谢谢主席先生和委员会成员。我很高兴今天来到这里讨论我来们加快为患者提供创新治疗的共同目标。FDA期待与你们在这里重要领域共同努力。

总所周知，这将是我最后一次出现在委员会面前，因为我即将卸任，但是我要感谢你们多年来的支持，我们积极参与该委员会以推进FDA的公共卫生使命。

我在生物医药产品行业有相当大的不确定性和变化的时候来到FDA，我的同事Collins博士刚才概述过一些，那是一个科学技术巨大进步的时代，要求新的模式和方法。

反过来，我们认真地看待我们在推进生物医药产品创新方面的作用，以确保我们成为获得更好、更安全、更有效治疗方法和药物的门户而不是障碍。

事实上，在我的任期内这一任务拥有很高的优先级。参议员Murray指出，过去的一年里，我们批准近20年来最多的新药，比以往都多的罕见病药。我对此感到非常高兴。这些新批准药品的41%是首次获批产品，从而为患者带来规模令人震惊的真正的创新治疗。

今天，FDA药品平均批准速度比所有其他发达国家更快：比日本快40天；比加拿大快70天；比欧洲快174天。FDA在医疗器械审评效率方面也已经取得了实质性的改善。

此外，我们已经成为全球安全性和有效性的黄金标准。

然而，尽管取得了这些成功，仍有太多疾病等待治疗和药物。严峻的公共健康需求，如阿尔兹海默病的治疗，还没有得到满足。以及不断增长的研发经费但无法获得相应新治疗方法的发现。

在这种情况下，我想打消一些顾虑，认为FDA监管是对研发创新疗法的主要障碍，并建议FDA的权限和程序必须从根本上调整。

作为一名医生，我知道如果你不能正确的诊断患者病情，你开的处方不太可能有效。除非我们正确的诊断为什么许多疾病仍缺乏治疗药物，否则我们就不可能找到真正能提供这些治疗药物的解决方案，让我举3个错误认识的例子。

首先，是错误地但通常反复断言FDA的新药批准落后于其他国家。现实情况是截然不同的：根据英国的监管科学创新中心最近的报告，2004年到2013年日本、欧洲、加拿大、澳大利亚、瑞士和FDA批准的新药中超过75%都是首先由FDA批准。其结果是，美国公众比国外患者更先获得新药治疗。

其次，FDA为批准新药要求和使用数据的方法被认为是死板的，不灵活的。事实上，FDA的临床试验要求一直都在稳步增加灵活性：

• 45%的新药基于一个替代终点获批。
• 三分之一的新药在单一临床试验的基础上获批。
• 去年，我们对比以往更多的药品（大约66%）采用加快批准流程。

而这部分要归功于你们在FDASIA中给予我们的新授权，74个药已经获得新的“突破性治疗”认证。

我的最后一个例子是，对生物技术在美国的投资已急剧下降的担心，认为FDA难辞其咎。但用美国国家风险投资协会（NVCA）的话说，“2014年生物技术投资额上升29%，达到60亿美元…，将其作为年度投资方面第二大投资领域。” NVCA会员、生物技术主要投资者Jonathan Leff表示，生物技术投资增加的两个原因之一是，近年来FDA监管环境的改善。

我举这些例子不是为了表示生物医学研究和产品开发的世界一切都是好的，而是要敦促我们开始正确的诊断。我们不希望基于不准确诊断的解决方案。

我警惕对美国公众依赖的医疗产品的批准采用降低安全性和有效性标准的解决方案。请记住，过去50年里循证医学的伟大飞跃已经到来，某种程度上是因为国会在一系列不安全和无效医疗产品的灾难后推出的高标准的产品批准。这些标准也振奋了信心，美国人寄希望于医疗产品，世界寄希望于美国生物医药行业。

总之，我们可以立足于近年来已经取得的进展，进一步推进生物医学科学并改善患者生活。我相信我们都同意，从FDA的视角看来的一些方面需要进一步改善。

首先，患者具有独一无二的地位影响医药产品研发。如果我们能够以科学为基础，针对特定疾病的患者，研发和审评过程结合，得到的治疗方法可以更好的满足他们的需求。

其次，“生物标志物”和替代终点的研发需要给予更多的重视。如Collins博士在他的演讲中提到，这些可以帮助科学家识别并定位成功的医药治疗同时缩短药物研发时间。

FDA已经接受了数百个生物标志物和替代物，如血压变化、血糖降低和肿瘤缩小。但许多疾病仍然缺乏生物标志物，如阿尔兹海默。最大的障碍是，科学家没有充分理解阿尔兹海默和其它疾病的成因，以识别药物靶标，或哪些患者将从某些药物中获益。为了解决这一问题，我们必须支持建立强有力的公私关系伙伴，引入最优秀的人才共同开发我们需要的科学。

第三，临床经验证据（被一些人称为“实证证据”或“大数据”）提供了重要的工具来监控医疗产品在市场上的使用。FDA的哨兵倡议，与1.7亿多人的生命息息相关，是在卫生保健领域这类信息的最大规模的使用之一，证明对安全监控和新兴安全问题至关重要。使用临床经验证据以建立产品有效性的科学尚处于起步阶段。真正的进步要求我们开发利用其承诺所需的方法学。

第四，FDA和业界认同，FDA必须能够吸引和留住有才华的科学家审评前沿产品。我们期待与您合作，改善我们招聘和留住这些专家的能力。

因此，让我以强调加快创新，同时保持服务患者的安全性和有效性标准，支持医疗保健系统需求，并使得这个国家的医药产品行业蓬勃发展作为结束。感谢你们支持我们在FDA的工作以及将推进的工作，感谢我们所有的同事在生物医学研究领域的工作，使得我们能够对患者提供科学保证。

编译：识林-椒 2015-03-17
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FDA Advances Medical Product Innovation
Posted on March 17, 2015 by FDA Voice
By: Margaret A. Hamburg, M.D.

On March 10, I had the pleasure of appearing with my colleague Dr. Francis Collins before the Senate Committee on Health, Education, Labor and Pensions to testify at a hearing on the subject of “Continuing America's Leadership in Medical Innovation for Patients.” I thought the broader public health community would be interested in my oral testimony, and so I am sharing it here:

Margaret Hamburg, M.D.“Thank you, Mr. Chairman and Members of the Committee. I'm very pleased to be here today to discuss our shared goal of speeding innovative treatments to patients. FDA looks forward to working with you on this important effort.

As you have noted, this will be my last appearance before the Committee, as I am stepping down, but I want to thank you for your support over the years, and our constructive engagement with this committee to advance FDA's public health mission.

I came to the Agency at a time of considerable uncertainty and change in the biomedical product industry; a time when dramatic advances in science and technology, some that my colleague Dr. Collins just outlined, demanded new models and approaches.

In turn, we took a very serious look at our role in advancing biomedical product innovation to ensure that we would be a gateway, not a barrier, to the delivery of better, safer and more effective treatments and cures.

In fact, this has been a high priority for me throughout my tenure and I’m very pleased, as Sen. Murray noted, last year, we approved the most new drugs in almost 20 years, and more orphan drugs than ever before. Forty-one percent of these new approvals were first-in-class products, resulting in a breathtaking array of truly innovative new therapies for patients.

Today, FDA approves drugs faster on average than all other advanced nations: 40 days faster than Japan; 70 days faster than Canada; and 174 days faster than Europe. And FDA has made substantial improvements in the efficiency of medical device reviews as well.

Moreover, we've accomplished this while remaining the gold standard around the world for safety and effectiveness.

Yet despite these successes, too many diseases still await treatments and cures. Serious public health needs, such as treatments for Alzheimer’s disease, are not being met. And rising R&D expenditures are not matched by a proportionate discovery of new treatments.

In this context, I want to address concerns raised by some that FDA regulation is the principal obstacle to the development of innovative treatments, and suggestions that FDA’s authorities and procedures must be fundamentally restructured.

As a physician, I know that if you incorrectly diagnose a patient’s condition, the treatment that you’ll prescribe is unlikely to work. Unless we correctly diagnose why cures are still lacking for many diseases, we’re unlikely to find the solutions that will actually deliver those cures so let me give you three examples of misconceptions.

First is the incorrect but commonly repeated assertion that FDA's approval of new drugs lags behind other countries. The reality is starkly different: over 75% of the new drugs approved by Japan, EU, Canada, Australia Switzerland and FDA from 2004 to 2013 were approved first by FDA, according to a recent report by the British-based Centre for Innovation in Regulatory Science. The result is that Americans are far more likely to get first access to a new medicine before patients abroad.

Second, FDA is said to be rigid and inflexible in its approach to requesting and using data for approval of a new drug. In fact, FDA’s clinical trial requirements have been steadily increasing in flexibility:

• 45% of new drugs are approved based on a surrogate endpoint;
• one-third are approved on the basis of a single clinical trial;
• Last year, we used expedited approval processes for more drugs than ever before – about 66%.

And thanks in part to the new authority that you gave us in FDASIA, 74 drugs had received the new “breakthrough” designation.

My final example is the concern that investment in biotechnology has dropped precipitously in the United States, and that the FDA is to blame. But in the words of The National Venture Capital Association (NVCA), “Biotechnology investment dollars rose 29 percent in 2014 to $6.0 billion . . , placing it as the second largest investment sector for the year in terms of dollars invested.” And Jonathan Leff, a leading biotechnology investor affiliated with NVCA, said that one of the two reasons for the increased investment in biotechnology is the improved regulatory climate in recent years at FDA.

I cite these examples to suggest not that the world of biomedical research and product development is all fine, but to urge that we start with the right diagnosis. We do not want solutions based on inaccurate diagnoses.

I caution against solutions that seek to lower the safety and effectiveness standards for approval of the medical products on which Americans rely. Remember that the great leaps forward in evidence-based medicine of the last 50 years have come in part because of the high standards for product approval that Congress put in place after a series of disasters involving unsafe and ineffective medical products. Those standards have also boosted the confidence that Americans place in medical products and that the world places in the American biomedical product industry.

Together, we can build on the progress that has been made in recent years, to further advance biomedical science and improve the lives of patients. And there are some areas from the FDA perspective that I believe we can all agree need to be improved.

First, patients are uniquely positioned to inform medical product development. Treatments can better meet their needs if we can capture science-based, disease-specific patient input to incorporate in the development and review process.

Second, more attention needs to be given to the development of “biomarkers” and surrogate endpoints. These can help scientists identify and target successful medical treatments and shorten drug development times as Dr. Collins was noting in his remarks.

FDA has accepted hundreds of biomarkers and surrogates, such as blood pressure changes, blood sugar reduction, and tumor shrinkage. Yet biomarkers are still lacking for many diseases, such as Alzheimer’s. The biggest obstacle is that scientists do not sufficiently understand the causes of Alzheimer’s and other diseases to identify drug targets or identify which patients will benefit from certain drugs. To solve this problem we must support the establishment of strong public-private partnerships, bringing the best minds together to develop the science that we need.

Third, evidence from clinical experience (called “real world evidence” or “big data” by some) provides a vital tool to monitor medical products in use in the marketplace. FDA’s Sentinel Initiative, with more than 170 million lives, is one of the largest uses of this type of information in healthcare and proving vital for monitoring safety and emerging safety concerns. The science of using evidence from clinical experience to establish product effectiveness is still in its infancy. Real progress demands that we develop the methodologies needed to harness its promise.

And fourth, FDA and industry agree that the Agency must be able to attract and retain talented scientists to review cutting-edge products. We look forward to working with you to improve our ability to hire and retain these experts.

So let me close by underscoring that speeding innovation while maintaining standards for safety and efficacy serves patients well, supports the needs of our health care system, and has enabled the medical product industry in this country to thrive. And so I thank you for your support for our efforts at FDA and the work you are going to be doing going forward to advance that work and the work of all our colleagues in the biomedical research community so we can deliver on the promise of science for patients.”