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2014-12-13 FDA Voice

FDA 2014年新药审批：为患者雪中送炭
FDA局长 Margaret A. Hamburg 博士
FDA Voice 2014年12月11日

今天早些时候披露的初步数据显示，2014年成为新药审批的另一个大年，对很多患者和他们的家庭来讲，这肯定是期盼已久的喜讯。

离今年年底只有几周,到目前为止，FDA药品审评与研究中心（CDER）已经在今年批准了35个新药，去年批准的新药数为27个。这些获批新药包括两种类型的新分子实体(NMEs)，分别以新药申请（NDA）的形式提交给CDER，以及以生物制剂许可申请（BLA）形式提交给CDER的治疗性生物制品。

但单从这一数字本身，并不能看出所有。真正重要的是，这些新获批药品和生物制品中的很多，对数以千计的严重或危及生命的疾病患者治疗提供了重要的临床价值。当然，就是针对那些20万或过少的美国人民受影响的罕见病。今年到现在为止，批准的罕见病用药达到15种，创下了里程碑式的记录。之前的最高纪录是2012年批准13种罕见病用药。由于罕见病患者通常只用很少的治疗用药或无药可治，这些结果更具意义。

还有另一个关键点 — 到目前为止，今年获批的新药中，有15个是同类药品中首次获批，这是这些药品潜在的强有力的临床影响的另一个重要指标。

为确保2014年新药能够急患者之所急，CDER有效地应用了一系列审批通道，包括FDA加速研发和审评计划 — 快速通道、优先审评、加速审批和去年才出台的突破性治疗药品认定。与申请人尽早和反复沟通交流对加速这些药品上市极有帮助。

以获批用于治疗Ph-染色体阴性前体B-细胞急性淋巴细胞白血病的Blincyto为例，CDER应用了所有的加速审批通道来帮助这一药品尽快上市，比审评目标日期提前了足足5个月。申请人也得益于针对罕见病治疗的药品的激励措施。

另一个例子是Harvoni， 这是首个获批用于治疗慢性丙肝病毒基因型1感染的单片复方制剂，也是首个获批的勿需与干扰素或利巴韦林联用的疗法。随着这个药品和近来获批的几个药品，我们正在帮助改变丙肝患者的治疗模式。Harvoni获得了突破性治疗药品认定并被给予优先审评。

近年来在新药获批方面几近荒芜的抗菌药品一直是更具挑战的药品研发领域之一。迄今为止，2014年获批的药品中，有3个新抗菌药 — Dalvance、Sivextro和Orbactiv，用于治疗皮肤感染、特异性急性细菌性皮肤或皮肤结构感染（ABSSSI）。这些药品获批代表这与药品领域期盼已久的温和增长。2014年之前，在2004-2013年周期中，只有5个全身性抗菌药品获批。

面对这些令人印象深刻的数字，向CDER的管理和审评人员致以祝贺。很大程度上，正是得益于CDER的艰苦工作和奉献，35个获批药品中，有34个在处方药生产商付费法案（PDUFA）规定的审评目标日期之前或是如期获批，其中的23个是在美国率先上市，这意味着美国患者可以比欧洲患者更早获得这些药品。

值此喜庆洋溢、辞旧迎新的假日之际，谨对CDER长年累月为患者利益辛勤工作致以谢意。

编译：识林-Kapok 2014-12-13

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2014 Drug Approvals: Speeding Novel Drugs to the Patients Who Need Them
Posted on December 11, 2014 by FDA Voice
By: Margaret A. Hamburg, M.D.

Preliminary data announced earlier today shows that 2014 is shaping up to be another strong year for novel drug approvals, which is certainly good news for many patients and their families.

Margaret Hamburg, M.D.With a few weeks left in December, our Center for Drug Evaluation and Research (CDER) has so far approved 35 novel new drugs in 2014 compared to 27 in 2013. These numbers include both new molecular entities (NMEs), submitted to CDER in New Drug Applications (NDAs) and new therapeutic biologics submitted to CDER in Biologics License Applications (BLAs).

But the numbers don’t tell the full story. What really matters is that many of these new products offer significant clinical value to the care of thousands of patients with serious and life-threatening diseases. That’s certainly the case for patients with rare diseases that affect 200,000 or fewer Americans. So far this year we’ve reached a milestone with a record 15 approvals for rare diseases. The previous high was 13 drugs in 2012. These results are all the more significant because patients with rare diseases often have few or no drugs available to treat their conditions.

And here’s another point of interest – to date, 15 of the approvals have been first in their class drugs, another indicator of their potentially strong clinical impact.

To ensure that 2014’s novel drugs get to patients as quickly as possible, CDER effectively employed a variety of regulatory tools including FDA’s expedited development and review programs – fast track, priority review, accelerated approval and our new breakthrough therapy designation. Early and repeated communications with sponsors have also been helpful in speeding these products to market.

Consider for example, Blincyto, approved just last week to treat Philadelphia chromosome-negative precursor B-cell acute lymphoblastic leukemia. CDER employed all of its expedited review programs to help get this drug to market as early as possible, five months ahead of its review goal date. The sponsor also benefited from incentives for drugs that treat rare diseases.

Another example is Harvoni, the first combination pill approved to treat chronic hepatitis C virus genotype 1 infection and the first approved regimen that does not require administration with interferon or ribavirin. With this and other recent approvals, we are helping to change the treatment paradigm for patients living with hepatitis C. Harvoni received breakthrough therapy designation and was assigned priority review.

One of the more challenging areas of drug development has been the rather barren field of antibacterial drugs. Among our 2014 approvals to date are three new antibacterial drugs – Dalvance, Sivextro and Orbactiv—to treat skin infections, specifically acute bacterial skin and skin structure infections (ABSSSI). These drug approvals represent a welcome but modest increase in activity in this product area. Prior to 2014, only five new systemic antibacterial drugs were approved during the period from 2004 – 2013.

I want to congratulate the management and review staff at CDER for these very impressive preliminary numbers. Thanks in large part to CDER’s hard work and dedication, 34 of the 35 drugs approved so far in 2014 were approved before or on their Prescription Drug User Fee Act (PDUFA) review goal date and 23 of the 35 drugs were available to patients in the United States before they were available to patients in Europe.

In this holiday season of joy and reflection, we have much to be grateful for in the work that CDER does every day on behalf of patients.