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2019-01-11    来源:    作者:  浏览次数:202

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The beginning of a new year is a good opportunity to look back over the past year and see how all the valuable work done at FDA's Center for Drug Evaluation and Research (CDER) results in bringing important new drug therapies to patients in need. It is particularly clear that our work is about people—and advancing patient care and public health.


In 2018, we approved many new drugs never before marketed in the United States, known as "novel" drugs, along with a wide variety of approvals of drugs already on the market to put to new and innovative uses. Many of these new approvals will have a significant impact on improving—and indeed, saving—countless patients' lives.


This past year was important for meeting the critical needs of people suffering from rare diseases who will benefit from these advances. Among many new orphan therapies, in 2018 CDER approved the first drug to treat patients with a rare, inherited form of rickets, a condition that leads to impaired bone growth and development. Also, CDER approved the first orally-administered drug to treat Fabry disease, a rare and serious disorder that can cause many adverse symptoms, including pain and burning in the hands and feet, and damage to the kidneys and heart. We also approved a new drug to treat patients with phenylketonuria, a rare dietary condition in which patients are born with an inability to break down protein-containing foods and certain sweeteners, and which can lead to brain and nerve damage.


Many of CDER's other 2018 approvals will provide hope, relief, and enhanced quality of life for patients in need:


  • CDER approved the first drug ever to treat smallpox, the first of a new class of drugs to treat patients with HIV-1 infection who have failed other therapies, and a new single dose treatment for influenza (flu).

  • CDER approved a new drug to treat seizures in patients with the rare and severe forms of epilepsy Lennox-Gastaut syndrome and Dravet syndrome, and a second drug to treat seizures in patients with Dravet syndrome. Previously there had been no FDA-approved therapy for Dravet syndrome. We approved three new drugs—all in a new drug class—to treat patients with migraines. Additionally, CDER approved the first therapy to treat multiple sclerosis in children.

  • We approved new advances for certain patients with breast cancer, prostate cancer, lung cancer, and thyroid cancer. We also approved a variety of new drug therapies to treat blood cancers and other blood disorders such as acute myeloid leukemia (a type of blood cancer), and classical Hodgkin lymphoma (a type of cancer of a part of the immune system called the lymphatic system); and thrombocytopenia (a deficiency of platelets in the blood) in patients with chronic liver disease scheduled to undergo a medical or dental procedure.

  • We also approved seven new biosimilars, which will further help to create competition, increase patient access, and potentially reduce the cost of important biological drug therapies.


  • Of course, even the best drug therapies are of no use to patients if they are stuck in the approval process. I want to recognize the importance of the efficiencies with which we brought these new therapies to approval.


  • Under the Prescription Drug User Fee Act (PDUFA), drug developers are assessed user fees that provide FDA with the additional resources needed to maintain an efficient and effective review process. PDUFA includes goal dates for application review agreed to with the pharmaceutical industry and approved by Congress. In 2018, CDER met its PDUFA goal dates for 100 percent of the novel drugs approved (59 of 59).

  • We use four specific regulatory tools to ensure prompt and efficient expedited review for approval decisions: Fast Track, Breakthrough, Accelerated Approval, and Priority Review. Each serves a purpose in helping to speed a drug’s development or review. In 2018, 73 percent of CDER’s novel drug approvals (43 of 59) were designated in one or more of these expedited review categories. These innovative approval methods can bring a therapy to patients months or even years sooner than if their application went through the standard review process.

  • In 2018, CDER approved 95 percent of its novel drug approvals (56 of 59) on the first cycle. A "cycle" is the time from when CDER accepts an application for a new drug until we decide about whether or not to approve it. A typical cycle generally takes somewhere between six months to a year, but sometimes things can go wrong. For instance, miscommunication, omissions of important data, and other issues in these complex applications, can lengthen the time it takes to complete a review and may also result in the need for a second cycle, further delaying access to the drug for patients in need. From 2011 through 2017, CDER approved 250 novel drugs, of which 205 (82 percent) were approved on the first cycle. Our consistently high first cycle approval rate reflects CDER’s commitment to working closely with applicants as they design their studies and build their application data. It also reflects our efficiency in getting new therapies to patients as quickly as possible.

  • Although regulatory processes differ widely between FDA and those of regulatory agencies in other countries, 42 of the 59 novel drugs approved in 2018 (71 percent) were approved in the United States before receiving approval in any other country.


  • More details about CDER’s new drug therapy approvals—including many specific examples of notable new approvals for the year—are available in our annual New Drug Therapy Approvals report.


    I am proud to be part of a team of dedicated professionals who work tirelessly to find innovative, efficient, and, most importantly, safe ways to bring new therapies to patients as quickly as possible.


    Tomorrow I will share my thoughts on CDER’s drug safety efforts for 2018.

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    2019年1月8日,美国食品药品监督管理局(FDA)发布了年度新药报告,《2018 New Drug Therapy Approvals》,披露了2018年批准新药59款,为历史新高:1/3 是 First-in-Class 的原研药,24%突破性疗法,70%+ 加速审批渠道!

    2018年关键词:

    强势的创新与进展!



    FDA下属药品评价与研究中心 (CDER)负责新药审批,包含新药申请(NDAs)下的新分子实体(NME),以及作生物制剂许可申请(BLAs)下的新治疗生物制剂。

    CDER 2018年的所有新药批准 Source: FDA


    #年度纵览


    59款!


    与上年度同比增加13个,获批数量是历史新高,远超2009-2017年平均的33款,FDA报告称2018年为”医药强势创新与进展的一年“!


    CDER 最近10年的新药批准走势 |  Source: FDA


    亮点解析


    CDER于2018年批准的许多新型药物以其潜在的积极影响和对优质医疗护理和患者治疗的独特贡献而着称。


    #一类新药(First-in-Class): 32%


    CDER将2018年批准的59种新药中的19种确定为一类药物,这是该药物对美国人健康产生强烈积极影响的一个指标。 这些药物通常具有与现有疗法不同的作用机制。 

    2018年度新药,一类创新药32% |  Source: FDA


    FDA于2018年批准的新型药物被认定为一流的药物:Aimovig,Crysvita,Elzonris,Galafold,Gamifant,Lucemyra,Lutathera,Onpattro,Orilissa,Oxervate,Palynziq,Poteligeo,Tavalisse,Tegsedi,Tibsovo,Tpoxx,Trogarzo,Vitrakvi Xofluza。...


    #加快审批更加普遍,
    占比增至
    73%


    CDER在2018年使用了多种监管途径来提高效率并加快新药的开发和批准。这些途径采用了一系列方法,包括CDER员工与药物开发人员之间的更多互动,更大的项目设计灵活性以及缩短的申请审查时间表。

    2018年度新药,整体使用快速发展和审查方法的占73%|  Source: FDA


    创新机制,加速审批成为业界主流共识!


    2018年59种新药批准中有43份(73%)被指定为快速通道,突破,优先审查和/或加速批准的一个或多个加急类别,包括FAST Track (41%),Breakthrough Therapy (24%), Accelerated Approval(7%)


    各快速审批渠道一览:FAST Track (41%),Breakthrough Therapy (24%), Accelerated Approval(7%)|  Source: FDA



    使用至少一种加急批准方法在2018年批准的新型药物是:Aemcolo,Ajovy,Biktarvy,Copiktra,Crysvita,Daurismo,Diacomit,Doptelet,Elzonris,Epidiolex,Erleada,Firdapse,Galafold,Gamifant,Krintafel,Libtayo,Lorbrena,Lucemyra, Lumoxiti,Lutathera,moxidectin,Mulpleta,Nuzyra,Omegaven,Onpattro,Orilissa,Oxervate,Palynziq,Poteligeo,Revcovi,Symdeko,Takhzyro,Talzenna,Tegsedi,Tibsovo,Tpoxx,Trogarzo,Vitrakvi,Vizimpro,Xerava,Xofluza,Xospata和Zemdri。



    #备受关注罕见病:占比53%


    2018年,CDER的59种新药中有34种(58%)用以治疗罕见病或“孤儿”疾病(患者数量20万或更少)。 患有罕见疾病的患者,通常很少或没有可用于治疗其病症的药物。 

    2018年度新药,与罕见病相关药品占比:58% |  Source: FDA


    2018年以孤儿药物名称批准的新型药物是:Asparlas,Braftovi,Copiktra,Crysvita,Daurismo,Diacomit,Elzonris,Epidiolex,Firdapse,Galafold,Gamifant,Krintafel,Lorbrena,Lumoxiti,Lutathera,Mektovi,moxidectin,Omegaven,Onpattro,Oxervate,Palynziq,Poteligeo,Revcovi,Symdeko,Takhzyro,Tavalisse, Tegsedi,Tibsovo,Tpoxx,Trogarzo,Ultomiris,Vitrakvi,Vizimpro和Xospata。...


    #美国优先


    尽管FDA与其他国家的监管机构的监管程序差异很大,但2018年批准的59种新药中有42种(71%)在获得其他国家批准之前在美国获得批准。


    2018年度新药,美国优先批准的药品占比:71% |  Source: FDA


    2018年首次在美国批准的新型药物是:Aemcolo,Aimovig,Ajovy,Akynzeo,Annovera,Asparlas,Biktarvy,Braftovi,Copiktra,Daurismo,Doptelet,Elzonris,Emgality,Epidiolex,Erleada,Gamifant,Ilumya,Krintafel,Libtayo, Lumoxiti,Mektovi,moxidectin,Nuzyra,Onpattro,Orilissa,Palynziq,Pifeltro,Revcovi,Seysara,Symdeko,Takhzyro,Talzenna,Tavalisse,Tibsovo,Tpoxx,Trogarzo,Ultomiris,Vitrakvi,Vizimpro,Xerava,Yupelri和Zemdri。


    新药列表



    展望



    2018年是新药蓬勃发展的一年,我们为一日千里科技进展欢呼;


    正如FDA摘要所描述:


    虽然CDER在2018年批准了59种新药,比去年任何一年都多,但CDER一如既往地关注批准的医疗价值,而不是数字。 批准了的许多已获FDA批准的药物的还有适应证拓展的新用途,新配方和新剂型,这些药物将有助于促进患者护理....这些都可以增加患者获得治疗的机会并降低患者的成本。 


    这些新的批准将使患有各种罕见疾病,神经系统疾病,传染病,癌症和许多其他疾病的患者受益。

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