首页 > 资讯 > 2014年FDA新药审评总结

出自识林

2015-01-23 FDA Voice

2014年CDER批准许多创新药
FDA药品审评与研究中心新药办公室主任John Jenkins博士
FDA Voice 2015年1月14日

每年，FDA药品审评与研究中心通常将批准100多个新治疗药物。其中一部分是之前没有被FDA批准过的新药，大多创新产品用于治疗未满足医疗需求的病症，或在其它方面显著地帮助改善患者治疗和公众健康。

今年，新闻媒体一直在关注2014年CDER批准的新药数量—无论是新分子实体还是新的治疗性生物制品。这是可以理解的，因为我们今年批准了41个新药，近20年内最多。但比起这些批准数字本身，我们更愿意把重点放在这些药品给患者带来的显著受益，以及CDER采取措施使得这些产品在保证FDA安全性、有效性和质量可靠标准的前提下及时地推向市场。

41个新药中的很多，对数以千计的严重或危及生命的疾病患者治疗提供了增加重要临床价值的潜力。其中包括8个治疗各类肿瘤患者的新药，4个治疗II型糖尿病的新药，4个治疗严重感染的新抗生素，2个治疗丙肝患者的新产品。

此外，请考虑这些事实：

• 41个新药中的17个（41%）获批用于治疗影响20万或更少美国人的罕见疾病。这是这类药批准总数最高的年份 — 超过之前最高记录2012年批准13种。由于罕见病患者通常只有很少的治疗用药或无药可治，这些结果更具意义。
• 41个新药中的17个（41%）由CDER认定为“首次获批”，这是评价药品创新程度的一个指标。2014年首次获批总数量接近最高年份2012年20个。

为加快产品研发和审评，CDER采用一些监管计划，包括快速通道、突破性治疗、优先审评和加速审批。

• 快速通道和突破性治疗认定的目的是，针对未满足医疗需求的严重病症，加速有前景的新药研发。几乎一半—2014年批准41个新药中19个或46%—被认定为快速通道或突破性治疗，或者二者兼而有之。
• 41个新药中的25个（61%）被认定为优先审评。CDER看到这些药对提供医疗保健显著进步的可能性，设置其审评目标为6个月而不是标准10个月。
• 41个新药中的6个（20%）根据FDA加速审批计划批准，加速审批允许治疗严重或危及生命疾病药品的早期批准，从而为目前医疗提供受益。加速审批是基于“对预临床受益具有合理可能性的”替代终点”或中间临床终点。批准后需要进一步的临床试验确认预期临床受益。替代终点是药品影响的标记（例如，对实验室值或肿瘤大小的影响），不直接代表患者感受或功能的改善，但被认为可获得预受益。

以下是关于去年批准我们评价的一些其它贡献：

• 根据处方药使用者付费法案（PDUFA），申请人在提交产品申请时支付费用。这笔钱是用来为FDA提供额外的资源以达到绩效目标，例如完成申请审评目标日期。2014年， 41个获批新药中的40个在PDUFA目标日期之前或是如期获批。
• CDER对41个新药中超过四分之三 — 31个（78%）— 在首轮审评中批准，这意味着无需要求可能拖延批准或导致另外一轮审评的额外信息。
• 近三分之二的新药—26个（63%）—是在美国率先获批。

这是另一个为患者需要批准新药的强劲一年。我们为我们安全高效地帮助将重要新药带给美国公众的职责感到自豪。

2014年新药总结 提供了更多详细信息。2014年CDER新药批准列表可参见我们的网站。

编译：识林-椒 2015-01-23
识林^TMwww.shilinx.com版权所有，未经许可不得转载。如需使用请联系admin@shilinx.com

CDER Approved Many Innovative Drugs in 2014
Posted on January 14, 2015 by FDA Voice
By: John Jenkins, M.D.

Each year, FDA's Center for Drug Evaluation and Research (CDER) will typically approve more than 100 new medications. A portion of those are novel new drugs, medications that have not previously been approved by FDA and are often among the most innovative products serving previously unmet medical needs or otherwise significantly helping to advance patient care and public health.

John JenkinsThis year, the news media has been concentrating on the number of novel new drugs – either new molecular entities or new therapeutic biologics – approved by CDER in 2014. And that's understandable because we approved 41 novel drugs this year, the most in nearly 20 years. But instead of looking at the approval tally, we prefer to focus on the significant benefits that many of these drugs bring to patients and the steps that CDER took to get these products to market in a timely manner while maintaining FDA's standards for safety, effectiveness, and quality.

Many of the 41 new drugs have the potential to add significant clinical value to the care of thousands of patients with serious or life-threatening diseases. They include eight new drugs for treating patients with various types of cancer, four new drugs to treat type-2 diabetes, four new antibiotics to treat serious infections, and two new products to treat patients with hepatitis C.

Moreover, consider these facts:

• Seventeen (41%) of the 41 novel new drugs were approved to treat rare diseases that affect 200,000 or fewer Americans. This is the highest yearly total of such drugs ever — surpassing the previous high of 13 from 2012. These approvals are particularly significant because patients with rare diseases often have few or no drugs available to treat their conditions.
• Seventeen (41%) of the 41 novel new drugs are identified by CDER as “First-in-Class,” one indicator of a drug’s degree of innovation. The total for First-In-Class approvals in 2014 approaches the highest yearly total of 20 reported in 2012.

To expedite the development and review of these products, CDER used a number of regulatory programs, including Fast Track, Breakthrough Therapy, Priority Review, and Accelerated Approval.

• Fast Track and Breakthrough Therapy designations are designed to speed the development of promising new drugs intended to treat serious conditions with unmet medical needs. Almost half – 19 or 46% of the 41 novel new drugs approved in 2014 — were designated as Fast Track, Breakthrough, or both.
• Twenty-five (61%) of the 41 novel new drugs were designated for Priority Review. These are drugs in which CDER sees potential for providing a significant advance in medical care, and sets their review target to within six instead of the standard 10 months.
• Six (20%) of the 41 novel new drugs were approved under FDA's Accelerated Approval program, which allows early approval of a drug for a serious or life-threatening illness that offers a benefit over current treatments. Accelerated Approval is based on a “surrogate endpoint” or an intermediate clinical endpoint that is thought to be “reasonably likely to predict clinical benefit.” Additional clinical trials are required after approval to confirm the predicted clinical benefit. A surrogate endpoint is a marker of drug effect (e.g., an effect on a lab value or tumor size) that does not directly represent an improvement in how a patient feels or functions, but is expected to predict such a benefit.

Here are a few other ways we assess our contributions to last year's approvals:

• Under the Prescription Drug User Fee Act (PDUFA), sponsors pay fees when they submit a product application. This money is used to provide FDA with additional resources to meet performance goals, such as a goal date for completing its review of the application. In 2014, CDER acted on or before the PDUFA goal date for 40 (98%) of the 41 novel new drugs approved.
• CDER approved more than three-quarters — 32 (78%) — of the 41 novel new drugs on the “first cycle” of review, meaning without requests for additional information that would delay approval and lead to another cycle of review.
• Nearly two-thirds of the novel new drugs – 26 (63%) — were approved in the U.S. before approval in another country.

It's been another strong year for approval of novel new drugs for patients in need. We are proud of our role in helping to safely and efficiently bring important new medications to the American public.

Our Novel New Drug Summary for 2014 provides more details. A current list of CDER's 2014 novel new drug approvals is available on our Web site.

John Jenkins, M.D., is Director of the Office of New Drugs in FDA's Center for Drug Evaluation and Research