Experimental designs for small randomised clinical trials: an algorithm for choice.
Experimental designs for small randomised clinical trials: an algorithm for choice.
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DOI：
10.1186/1750-1172-8-48
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发表时间：
2013-03-25
影响因子：
3.7
通讯作者：
Nabbout R
中科院分区：
文献类型：
--
作者：
Cornu C;Kassai B;Fisch R;Chiron C;Alberti C;Guerrini R;Rosati A;Pons G;Tiddens H;Chabaud S;Caudri D;Ballot C;Kurbatova P;Castellan AC;Bajard A;Nony P;CRESim & Epi-CRESim Project Groups;Aarons L;Bajard A;Ballot C;Bertrand Y;Bretz F;Caudri D;Castellan C;Chabaud S;Cornu C;Dufour F;Dunger-Baldauf C;Dupont JM;Fisch R;Guerrini R;Jullien V;Kassaï B;Nony P;Ogungbenro K;Pérol D;Pons G;Tiddens H;Rosati A;Alberti C;Chiron C;Kurbatova P;Nabbout R
当难以招募足够的患者进行常规的频率统计分析以提供适当的答案时，小型临床试验是必要的。这些试验通常是研究罕见疾病以及特定研究人群（如儿童）所必需的。据估计，有6，000至8，000种罕见疾病，涵盖广泛的疾病和患者。在欧洲联盟，这些疾病影响多达3000万人，其中约50%的受影响者是儿童。治疗这些罕见疾病的疗法需要评估其疗效和安全性，但由于潜在的试验参与者数量很少，标准的随机对照试验通常不可行。对于通常的平行组设计，有许多替代试验设计，每种设计都有特定的优势，但也有特定的局限性。因此选择最合适的设计并不简单。检索PubMed，以识别可用于随机化、比较性小型临床试验的不同试验设计特征的出版物。此外，手工检索了11种期刊的目次表。一个算法，开发了使用决策节点的基础上确定的试验设计的特点。我们确定了75篇出版物，这些出版物报告了12项随机、比较试验设计的特征，可用于评估孤儿疾病的治疗。总结了这些设计的主要特征、优点和局限性，并用于开发一种算法，该算法可用于帮助为给定的临床情况选择适当的设计。我们使用了来自给定疾病治疗结局情况的出版物的示例，其中研究者使用了特定的试验设计，以说明使用该算法识别可能的替代设计。我们提出的算法可能是一个有用的工具，选择一个适当的试验设计，在孤儿药的发展，为一个给定的疾病治疗结果的情况。
Small clinical trials are necessary when there are difficulties in recruiting enough patients for conventional frequentist statistical analyses to provide an appropriate answer. These trials are often necessary for the study of rare diseases as well as specific study populations e.g. children. It has been estimated that there are between 6,000 and 8,000 rare diseases that cover a broad range of diseases and patients. In the European Union these diseases affect up to 30 million people, with about 50% of those affected being children. Therapies for treating these rare diseases need their efficacy and safety evaluated but due to the small number of potential trial participants, a standard randomised controlled trial is often not feasible. There are a number of alternative trial designs to the usual parallel group design, each of which offers specific advantages, but they also have specific limitations. Thus the choice of the most appropriate design is not simple. PubMed was searched to identify publications about the characteristics of different trial designs that can be used in randomised, comparative small clinical trials. In addition, the contents tables from 11 journals were hand-searched. An algorithm was developed using decision nodes based on the characteristics of the identified trial designs. We identified 75 publications that reported the characteristics of 12 randomised, comparative trial designs that can be used in for the evaluation of therapies in orphan diseases. The main characteristics and the advantages and limitations of these designs were summarised and used to develop an algorithm that may be used to help select an appropriate design for a given clinical situation. We used examples from publications of given disease-treatment-outcome situations, in which the investigators had used a particular trial design, to illustrate the use of the algorithm for the identification of possible alternative designs. The algorithm that we propose could be a useful tool for the choice of an appropriate trial design in the development of orphan drugs for a given disease-treatment-outcome situation.
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影响因子：
1.9
作者：
Chiron, Catherine;Tonnelier, Sylvie;Pons, Gerard
通讯作者：
Pons, Gerard
影响因子：
7.2
作者：
Feldman, B;Wang, E;Szalai, JP
通讯作者：
Szalai, JP
影响因子：
168.9
作者：
Chiron, C;Marchand, MC;Pons, G
通讯作者：
Pons, G
影响因子：
2.6
作者：
Bjerkeset, O;Larsen, S;Reiertsen, O
通讯作者：
Reiertsen, O
DOI：
10.1016/0197-2456(90)90003-k
发表时间：
1990-04-01
期刊：
CONTROLLED CLINICAL TRIALS
影响因子：
--
作者：
GUYATT, GH;HEYTING, A;ROBERTS, RS
通讯作者：
ROBERTS, RS