Overview, hurdles, and future work in adaptive designs: perspectives from a National Institutes of Health-funded workshop.
Overview, hurdles, and future work in adaptive designs: perspectives from a National Institutes of Health-funded workshop.
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自适应设计中的概述，障碍和未来的工作：来自国立卫生研究院资助的研讨会的观点。
DOI：
10.1177/1740774512461859
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发表时间：
2012-12
期刊：
影响因子：
--
通讯作者：
Harris S
中科院分区：
文献类型：
--
作者：
Coffey CS;Levin B;Clark C;Timmerman C;Wittes J;Gilbert P;Harris S
临床试验界一直在寻找可靠和可靠的方法来改善临床研究。这种探索引起了人们对适应性临床试验设计的极大兴趣，这种设计提供了在中期审查数据的基础上调整试验特征的灵活性。统计学家和临床研究者已经在临床试验中提出或实施了各种各样的调整，但具体的方法得到了不同程度的支持。在行业内，研究人员正在积极探索与自适应设计（AD）相关的好处和陷阱。例如，药品信息协会（DIA）的广告工作组已经与监管机构进行了讨论。然而，许多从事公共资助的临床试验的研究人员尚未完全参与这一讨论。我们组织了适应性临床试验设计研讨会的科学进展，开始关于在公共资助的研究中使用AD的对话。在2009年11月举行，为期一天半的研讨会汇集了来自美国国立卫生研究院（NIH），食品和药物管理局（FDA），欧洲药品管理局（EMA），制药行业，非营利基金会，患者倡导社区和学术界的代表。研讨会为与会者提供了一个论坛，以解决在临床试验的规划，设计和执行阶段出现的AD问题，并听取临床试验界有影响力的成员的观点。与会者还提出了指导行动的建议，以促进广告的适当使用。这些建议已经在许多场合提出，讨论和审查，包括宾夕法尼亚大学临床试验统计问题会议和临床试验学会年会。简要概述AD，描述举办研讨会的理由，并总结研讨会产生的主要建议。人们对使用适应性临床试验设计越来越感兴趣。然而，为了获得反倾销的潜在优势，需要解决一些后勤障碍。目前，制药行业在解决这些障碍方面远远领先于学术试验者。学术试验者需要解决重要问题，如教育，基础设施，现有资助模式的修改以及对数据和安全监测委员会（DSMB）的影响，以实现适应性临床试验设计的可能益处。
The clinical trials community has a never-ending search for dependable and reliable ways to improve clinical research. This exploration has led to considerable interest in adaptive clinical trial designs, which provide the flexibility to adjust trial characteristics on the basis of data reviewed at interim stages. Statisticians and clinical investigators have proposed or implemented a wide variety of adaptations in clinical trials, but specific approaches have met with differing levels of support. Within industry, investigators are actively exploring the benefits and pitfalls associated with adaptive designs (ADs). For example, a Drug Information Association (DIA) working group on ADs has engaged regulatory agencies in discussions. Many researchers working on publicly funded clinical trials, however, are not yet fully engaged in this discussion. We organized the Scientific Advances in Adaptive Clinical Trial Designs Workshop to begin a conversation about using ADs in publicly funded research. Held in November of 2009, the 1½-day workshop brought together representatives from the National Institutes of Health (NIH), the Food and Drug Administration (FDA), the European Medicines Agency (EMA), the pharmaceutical industry, nonprofit foundations, the patient advocacy community, and academia. The workshop offered a forum for participants to address issues of ADs that arise at the planning, designing, and execution stages of clinical trials, and to hear the perspectives of influential members of the clinical trials community. The participants also set forth recommendations for guiding action to promote the appropriate use of ADs. These recommendations have since been presented, discussed, and vetted in a number of venues including the University of Pennsylvania Conference on Statistical Issues in Clinical Trials and the Society for Clinical Trials annual meeting. To provide a brief overview of ADs, describe the rationale behind conducting the workshop, and summarize the main recommendations that were produced as a result of this workshop. There is a growing interest in the use of adaptive clinical trial designs. However, a number of logistical barriers need to be addressed in order to obtain the potential advantages of an AD. Currently, the pharmaceutical industry is well ahead of academic trialists with respect to addressing these barriers. Academic trialists will need to address important issues such as education, infrastructure, modifications to existing funding models, and the impact on Data and Safety Monitoring Boards (DSMB) in order to achieve the possible benefits of adaptive clinical trial designs.
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影响因子：
11.2
作者：
Kaufmann, Petra;Thompson, John L. P.;Levy, Gilberto;Buchsbaum, Richard;Shefner, Jeremy;Krivickas, Lisa S.;Katz, Jonathan;Rollins, Yvonne;Barohn, Richard J.;Jackson, Carlayne E.;Tiryaki, Ezgi;Lomen-Hoerth, Catherine;Armon, Carmel;Tandan, Rup;Rudnicki, Stacy A.;Rezania, Kourosh;Sufit, Robert;Pestronk, Alan;Novella, Steven P.;Heiman-Patterson, Terry;Kasarskis, Edward J.;Pioro, Erik P.;Montes, Jacqueline;Arbing, Rachel;Vecchio, Darleen;Barsdorf, Alexandra;Mitsumoto, Hiroshi;Levin, Bruce
通讯作者：
Levin, Bruce
影响因子：
3.8
作者：
Bretz, Frank;Branson, Michael;Coffey, Christopher S.
通讯作者：
Coffey, Christopher S.
影响因子：
1.1
作者：
Emerson, Scott S.;Fleming, Thomas R.
通讯作者：
Fleming, Thomas R.
影响因子：
1.1
作者：
Gallo, Paul;Chuang-Stein, Christy;Pinheiro, Jose
通讯作者：
Pinheiro, Jose
影响因子：
9.9
作者：
Levy, G;Kaufmann, P;Thompson, JLP
通讯作者：
Thompson, JLP