Laughren Thomas, Levin Robert
Food and Drug Administration, DNDP, HFD-120, 5600 Fishers Lane, Rockville, MD 20853, USA.
Schizophr Bull. 2006 Apr;32(2):220-2. doi: 10.1093/schbul/sbi039. Epub 2005 Aug 3.
Negative symptoms of schizophrenia are not adequately addressed by available treatments for schizophrenia. Thus, it is reasonable to consider them as a target for a drug claim. This article describes the thought process that the Food and Drug Administration (FDA) will undertake in considering negative symptoms of schizophrenia as a novel and distinct drug target. Beyond this basic question, this article identifies a number of design issues that the FDA needs to consider regarding how best to conduct studies to support claims for this target. These design issues include (1) what population to study, (2) what phase of illness to target, (3) whether to focus on the negative symptom domain overall or on some specific aspect of negative symptoms, (4) the role of functional measures in negative symptom trials, and (5) optimal designs for targeting drugs for add-on therapy or broad-spectrum agents.
精神分裂症的现有治疗方法无法充分解决其阴性症状。因此，将其视为药物申请的目标是合理的。本文描述了美国食品药品监督管理局（FDA）在将精神分裂症阴性症状视为一个新的、独特的药物靶点时将进行的思考过程。除了这个基本问题，本文还确定了一些设计问题，FDA需要考虑如何以最佳方式开展研究以支持针对该靶点的申请。这些设计问题包括：（1）研究对象群体；（2）针对疾病的哪个阶段；（3）是关注阴性症状领域整体还是阴性症状的某些特定方面；（4）功能测量在阴性症状试验中的作用；以及（5）针对附加治疗药物或广谱药物的最佳设计。
