Abstract：
The use of randomized clinical trials, in particular placebo-controlled trials, for drug approval, is the subject of long-standing debate in the scientific community and beyond. This study offers consensus recommendations from clinical and academic experts to guide the selection of clinical trial design in psychiatry. Forty-one highly cited clinical psychiatrists and/or researchers participated in a Delphi survey. Consensus statements were developed based on the findings of a published, peer-reviewed systematic review. Participants evaluated statements in two survey rounds, following the Delphi method. The expert panel achieved consensus on 7 of 21 recommendations regarding the use of randomized clinical trials. The endorsed recommendations were: (i) Results from placebo-controlled trials are the most reliable and (ii) are necessary despite the growing placebo-effect; (iii) it is ethical to enroll patients in placebo-arms when established treatment is available, if there is no evidence of increased health risk; (iv) There is a need to approve new drugs with the same efficacy as existing treatments, but with different side-effect profiles; (v) Non-inferiority trials incur an increased risk of approving ineffective medications; (vi) The risk of approving an ineffective drug justifies trial designs that incur higher costs, and (vii) superiority trials incur the risk of rejecting potentially efficacious treatments. The endorsed recommendations inform the choice of trial-design appropriate for approval of psychopharmacological drugs. The recommendations strongly support the use of randomized clinical trials in general, and the use of placebo-controlled trials in particular.
摘要：
使用随机临床试验，特别是安慰剂对照试验，用于药物批准，是科学界及其他领域长期辩论的主题。这项研究提供了临床和学术专家的共识建议，以指导精神病学临床试验设计的选择。41名被高度引用的临床精神科医生和/或研究人员参加了Delphi调查。共识声明是根据已发表的一份报告的调查结果制定的，同行评审系统评价。参与者在两轮调查中评估了陈述，遵循Delphi方法。专家小组就关于使用随机临床试验的21项建议中的7项达成了共识。认可的建议是：（i）安慰剂对照试验的结果是最可靠的，（ii）尽管安慰剂效应越来越大，但仍有必要；（iii）在有既定治疗可用的情况下，将患者纳入安慰剂组是符合道德的，如果没有证据表明健康风险增加；（iv）有必要批准与现有治疗方法具有相同功效的新药，但副作用不同；（v）非劣效性试验增加批准无效药物的风险；（vi）批准无效药物的风险证明试验设计的合理性，和（vii）优势试验导致拒绝潜在有效治疗的风险。认可的建议为选择适合批准精神药理学药物的试验设计提供了信息。这些建议强烈支持一般使用随机临床试验，特别是安慰剂对照试验的使用。
