FDA新药审批流程简述(中英文)

1、FDA新药审批流程 美国的新药审批可以说是世界上最严格和规范的，作为一个公司通常需要花费5亿美元资金，用 12到15年的时间才能将一个新药从试验室走入市场。在5000个临床前化合物中大约只有5个化合物可以进入临床试验（Clinical Trials），而这5个化合物中只有一个才能被批准用于临床治疗病人，成为真正的药物。 从一个实验室发现的新化合物发展成为一个治疗疾病的药物，需要经过如下开发阶段：一、 临床前试验将一个新发现的化合物经过实验室和动物试验，证明该化合物针对特定目标疾病具有生物活性，并且要评估该化合物的安全性。二、 新药临床研究申请当一个化合物通过了临床前试验后，需要向FDA提交新药

2、临床研究申请，以便可以将该化合物应用于人体试验。如果在提交申请后30天内FDA没有驳回申请，那么该新药临床研究申请即被视为有效，可以进行人体试验。新药临床研究申请需要提供先前试验的材料；以及计划将在什么地方，由谁以及如何进行临床试验的说明；新化合物的结构；投药方式；动物试验中发现的所有毒性情况；该化合物的制造生产情况。所有临床方案必须经过机构审评委员会（Institutional Revuew Board，IRB）的审查和通过。每年必须向FDA和IRB 汇报一次临床试验的进程和结果。三、 一期临床试验这一阶段的临床试验一般需要征集20100名正常和健康的志愿者进行试验研究。试验的主要目的是提供

3、该药物的安全性资料，包括该药物的安全剂量范围。同时也要通过这一阶段的临床试验获得其吸收、分布、代谢和排泄以及药效持续时间的数据和资料。 四、二期临床试验 这一期的临床试验通常需要征集100500名相关病人进行试验。其主要目的是获得药物治疗有效性资料。五、三期临床试验 这一期的临床试验通常需 10005000名临床和住院病人，多在多个医学中心进行，在医生的严格监控下，进一步获得该药物的有效性资料和鉴定副作用，以及与其他药物的相互作用关系。该阶段试验一般采取多中心，安慰剂（或/和有效对照剂）对照和双盲法试验。第三期临床试验是整个临床试验中最主要的一步。 六、新药申请在完成所有三个阶段的临床试验并分

4、析所有资料及数据，如证明该药物的安全性和有效性，则可以向 FDA提交新药申请。新药申请需要提供所有收集到的科学资料。通常一份新药申请材料可多达 页，甚至更多！按照法规，FDA应在6个月内审评完新药申请。但是由于大部分申请材料过多，而且有许多不规范，因此往往不能在这么短的时间内完成。 1999年对于单个化学分子药的审评时间平均为 12.6个月。 七、批准上市 一旦FDA批准新药申请后，该药物即可正式上市销售，供医生和病人选择。但是还必须定期向FDA呈交有关资料，包括该药物的副作用情况和质量管理记录。对于有些药物FDA还会要求做第四期临床试验，以观测其长期副作用情况。（中国医药市场信息2002第2

5、期16页） The Food and Drug Administration (FDA) regulates the development of novel drugs. Both prescription and over-the-counter drugs are regulated by the Center for Drug Evaluation and Research (CDER). CDER has been established to ensure that drug products are safe and effective. All new drug product

6、s must undergo a rigorous process of pre-clinical and clinical evaluation. According to a 1999 report from PhRMA, it takes 15 years and $500 million for an experimental drug to travel from the lab bench to the patient. For every 5000 compounds that enter pre-clinical testing, only five will continue

7、 on to clinical trials in humans and only one will be approved for marketing in the United States. After each stage of development, the sponsor of the new product meets with the FDA to determine next steps and establish end points for future trials. Similar processes are required in other countries.

8、 Preclinical Testing. A pharmaceutical or biotechnology company conducts laboratory and animal studies to demonstrate biological activity of the compound against the targeted disease, and the compound is evaluated for safety. Investigational New Drug Application (IND). After completing preclinical t

9、esting, the company files an IND with the FDA to begin to test the drug in humans. The IND becomes effective if the FDA does not disapprove it within 30 days. The IND shows results of previous experiments and studies; how, where and by whom the new studies will be conducted; the chemical structure o

10、f the compound; how it is thought to work in the body; any toxic effects found in the animal studies; and how the compound is manufactured. The IND must be reviewed and approved by the Institutional Review Board (IRB) where the studies will be conducted, and progress reports on clinical trials must

11、be submitted to the FDA at least once annually. Phase I Human Clinical Trials. These tests involve approximately 20 to 80 normal, healthy volunteers. These tests study a drugs safety profile , including the safe dosage range. The studies also analyse how a drug is absorbed, distributed, metabolised

12、and excreted, and the duration of its action. Phase II Human Clinical Trials. Controlled studies of approximately 100 to 300 volunteer patients (people with disease being treated) to assess the drugs effectiveness and further analyse safety. Dose ranges may also be analysed during Phase II studies.

13、More than one Phase II study may be conducted. Phase III Clinical Trials. Approximately 1,000 to 3,000 patients in clinics and hospitals. This phase is used to determine whether the drugs effectiveness is statistically significant. Patients are continuously monitored for safety or adverse reactions.

14、 Typically, more than one Phase III study is conducted. New Drug Application (NDA). Following successful completion of all three phases of human clinical trials, the company analyses all of the data and files an NDA with the FDA if the data successfully demonstrate safety and effectiveness. The NDA

15、must contain all of the scientific information that the company has gathered on the compound. NDAs can exceed 100,000 pages or more. By legislation, the FDA is allowed six months to review an NDA filing. In 2000, the average review time for approved products was 16 months. FDA Panel Review. Once CDE

16、R has reviewed the NDA, the products sponsor presents the data to a panel of experts. The members of the panel may ask for clarification of specific data points, request explanations for certain outcomes or events observed in the trial or pose questions on potential issues that may occur if the product is approved for marketing. The members of the panel then vote in favour of or against recommending marketing