Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland.
Fertil Steril. 2018 Jun;109(6):964-967. doi: 10.1016/j.fertnstert.2018.03.027.
Personalized medicine has many definitions. This term is often used synonymously with precision medicine, which is defined as the classifying patients with a disease or condition based on their phenotypic findings, such as biomarkers or genomics, into subpopulations that differ in their response to a specific treatment. Personalized medicine, however, can also mean the treatment of individual patients based on many contextual factors, such as response to therapy and patient preferences, in addition to predefined phenotypic findings. Regulatory approval for the marketing of a new drug or a new indication for a marketed drug requires a positive benefit risk profile and substantial evidence of effectiveness. The indication is based on the eligibility criteria and outcomes of the clinical trial(s) underpinning the regulatory approval. For precision medicine, drugs are often developed with companion diagnostics that are necessary for selection of the subgroup of patients, in contrast to personalized medicine which may be directed at a single patient. Most drugs are approved with a fixed dosage regimen for the approved population, but some drugs and biologics are approved with instructions to tailor therapy for individual patients, whether it be dosing, combination with other therapies, or selection among a class of medications. Hence, more often than not, personalized medicine directed at individual patients is achieved through the practice of medicine rather than regulatory action.
个性化医学有许多定义。这个术语常与精准医学同义，后者被定义为根据患者的表型发现（如生物标志物或基因组学）将患有某种疾病或病症的患者分为亚组，这些亚组在对特定治疗的反应上存在差异。然而，个性化医学也可以指根据许多上下文因素（如对治疗的反应和患者偏好）对个体患者进行治疗，除了预先定义的表型发现。新药物或已上市药物的新适应症的营销监管批准需要具有积极的风险效益比和有效性的大量证据。适应症基于支持监管批准的临床研究的纳入标准和结果。对于精准医学，药物通常与伴随诊断一起开发，这些诊断对于选择患者亚组是必要的，而个性化医学可能针对单个患者。大多数药物都有固定的剂量方案批准给批准的人群，但有些药物和生物制剂有个性化治疗的指示，无论是调整剂量、与其他疗法联合使用，还是在一类药物中进行选择。因此，针对个体患者的个性化医学往往是通过医学实践而不是监管行动来实现的。
