Botstein P
Isr J Med Sci. 1986 Mar-Apr;22(3-4):197-200.
New drugs in the United States are studied under investigational new drug exemptions. Commercial sponsors then gather the accrued data into a new drug application to support safety and effectiveness for marketing. The Food, Drug and Cosmetic Act of 1938, as amended in 1962, requires that effectiveness of a drug for its intended use be demonstrated by substantial evidence, consisting of adequate and well-controlled investigations, including clinical investigations, by experts qualified by training and experience to evaluate the drug. Safety must be demonstrated by appropriate methods. Although a certain nucleus of information is essential for all drugs, flexibility in testing is desirable in many areas. For each drug, the design and number of clinical studies vary. The number of patients, the type of patient population, and the kind of special studies to elucidate the mechanism of action or explore particular pharmacologic properties are chosen by the sponsoring pharmaceutical company. Data on effectiveness or on adverse reactions may be generated in the U.S. or in other countries.
在美国，新药是根据研究性新药豁免进行研究的。商业申办者随后将积累的数据整理成新药申请，以支持药物上市的安全性和有效性。1938年的《食品、药品和化妆品法案》（经1962年修订）要求，药物预期用途的有效性需有充分证据证明，这些证据包括充分且严格对照的调查，包括临床调查，由经过培训和具有经验、有资格评估该药物的专家进行。安全性必须通过适当的方法来证明。尽管一定的核心信息对所有药物都至关重要，但在许多领域，测试灵活性是可取的。对于每种药物，临床研究的设计和数量各不相同。患者数量、患者群体类型以及用于阐明作用机制或探索特定药理特性的特殊研究类型，由申办的制药公司选择。有效性或不良反应的数据可能在美国或其他国家产生。
