A summary of the FDA-NIMH-MATRICS Workshop on Clinical Trial Design for Neurocognitive Drugs for Schizophrenia.
A summary of the FDA-NIMH-MATRICS Workshop on Clinical Trial Design for Neurocognitive Drugs for Schizophrenia.
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DOI：
10.1093/schbul/sbi020
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发表时间：
2005-01-01
影响因子：
6.6
通讯作者：
Marder, SR
中科院分区：
文献类型：
--
作者：
Buchanan, RW;Davis, M;Marder, SR
目的：2004年4月23日，FDA、NIMH、matrix研究人员以及学术界和制药行业专家召开了一次联合会议，为精神分裂症患者神经认知障碍的认知增强药物的临床试验设计制定指导方针。方法：请专家回答有关辅助/联合治疗和广谱药物临床试验设计的具体问题。在研讨会上，专家们审查了相关证据，然后为特定子集的问题提供指南中建议的讨论小组。讨论小组由来自FDA、NIMH、学术界和工业界的演讲者和代表组成，他们就建议的指导方针进行了审议，以达成共识。当证据不足时，建议的指导方针代表了演讲者和讨论小组的一个横截面的意见。结果：制定了纳入标准、联合主要结果测量方法的使用和研究设计的统计方法的指南。关于诊断和伴随药物纳入标准以及认知筛查措施的使用达成了共识。一个关键的指导方针是将试验限制在疾病残留期的患者，这些患者具有预定义的阳性、阴性和情感性症状水平。最困难的问题是纳入功能改善的共同主要措施的可行性，以及在广谱药物（具有抗精神病和增强认知作用）的试验中选择比较药物。结论：建议的指南为认知增强药物的试验设计提供了合理的起点，理解新数据、后续发现或其他方法学考虑可能导致未来的修改。
Objective: On April 23, 2004, a joint meeting of the FDA, NIMH, MATRICS investigators, and experts from academia and the pharmaceutical industry was convened to develop guidelines for the design of clinical trials of cognitive-enhancing drugs for neurocognitive impairments in patients with schizophrenia. Method: Experts were asked to address specific questions relating to clinical trial design of adjunctive/co-treatment and broad spectrum agents. At the workshop, experts reviewed relevant evidence before offering the discussion panel proposed in guidlines for a given subset of questions. The discussion panel, which consisted of presenters and representatives from FDA, NIMH, academia, and industry, deliberated to reach consensus on suggested guidelines. When evidence was insufficient, suggested guidelines represent the opinion of a cross-section of the presenters and discussion panel. Results: Guidelines were developed for inclusion criteria, the use of co-primary outcome measures, and statistical approaches for study design. Consensus was achieved regarding diagnostic and concomitant medication inclusion criteria and on the use of cognitive screening measures. A key guideline was to limit the trial to patients in the residual phase of their illness, who have a predefined level of positive, negative, and affective symptoms. The most difficult issues were the feasibility of including a co-primary measure of functional improvement and the choice of comparator agent for a trial of a broad spectrum agent (with antipsychotic and cognitive-enhancing effects). Conclusions: The suggested guidelines represent reasonable starting points for trial design of cognitive-enhancing drugs, with the understanding that new data, subsequent findings, or other methodological considerations may lead to future modifications.