Biggane Alice M, Brading Lucy, Ravaud Philippe, Young Bridget, Williamson Paula R
Department of Biostatistics, University of Liverpool, Liverpool, UK.
INSERM, U1153 Epidemiology and Biostatistics Sorbonne Paris Cité Research Center (CRESS), Methods of therapeutic evaluation of chronic diseases Team (METHODS), 75014, Paris, France.
Trials. 2018 Feb 17;19(1):113. doi: 10.1186/s13063-018-2493-y.
There are numerous challenges in including patients in a core outcome set (COS) study, these can vary depending on the patient group. This study describes current efforts to include patients in the development of COS, with the aim of identifying areas for further improvement and study.
Using the COMET database, corresponding authors of COS projects registered or published from 1 January 2013 to 2 February 2017 were invited via a personalised email to participate in a short online survey. The survey and emails were constructed to maximise the response rate by following the academic literature on enhancing survey responses. Personalised reminder emails were sent to non-responders. This survey explored the frequency of patient input in COS studies, who was involved, what methods were used and whether or not the COS development was international.
One hundred and ninety-two COS developers were sent the survey. Responses were collected from 21 February 2017 until 7 May 2017. One hundred and forty-six unique developers responded, yielding a 76% response rate and data in relation to 195 unique COSs (as some developers had worked on multiple COSs). Of focus here are their responses regarding 162 COSs at the published, completed or ongoing stages of development. Inclusion of patient participants was indicated in 87% (141/162) of COSs in the published completed or ongoing stages and over 94% (65/69) of ongoing COS projects. Nearly half (65/135) of COSs included patient participants from two or more countries and 22% (30/135) included patient participants from five or more countries. The Delphi survey was reported as being used singularly or in combination with other methods in 85% (119/140) of projects. Almost a quarter (16/65) of ongoing studies reported using a combination of qualitative interviews, Delphi survey and consensus meeting.
These findings indicated that the Delphi survey is the most popular method of facilitating patient participation, while the combination of qualitative interviews, Delphi survey and consensus meetings is the most popular combination of methods. The increased inclusion of patient participants in the development of COSs is encouraging, as is the international approach to COS development that some developers are adopting.
将患者纳入核心结局集（COS）研究存在诸多挑战，这些挑战因患者群体而异。本研究描述了当前在COS开发过程中纳入患者的努力，旨在确定需要进一步改进和研究的领域。
利用COMET数据库，通过个性化电子邮件邀请2013年1月1日至2017年2月2日注册或发表的COS项目的通讯作者参与一项简短的在线调查。根据关于提高调查回复率的学术文献构建调查和电子邮件，以最大限度地提高回复率。向未回复者发送个性化提醒电子邮件。该调查探讨了COS研究中患者参与的频率、参与人员、使用的方法以及COS开发是否具有国际性。
向192名COS开发者发送了调查。从2017年2月21日至2017年5月7日收集回复。146名不同的开发者做出了回应，回复率为76%，涉及195个不同的COS（因为一些开发者参与了多个COS）。这里重点关注他们对162个处于已发表、已完成或正在进行开发阶段的COS的回复。在已发表、已完成或正在进行阶段的COS中，87%（141/162）表明纳入了患者参与者，在正在进行的COS项目中这一比例超过94%（65/69）。近一半（65/135）的COS纳入了来自两个或更多国家的患者参与者，22%（30/135）纳入了来自五个或更多国家的患者参与者。在85%（119/140）的项目中报告单独或与其他方法结合使用了德尔菲调查。几乎四分之一（16/65）的正在进行的研究报告使用了定性访谈、德尔菲调查和共识会议相结合的方法。
这些发现表明，德尔菲调查是促进患者参与的最常用方法，而定性访谈、德尔菲调查和共识会议相结合是最常用的方法组合。在COS开发中越来越多地纳入患者参与者令人鼓舞，一些开发者采用的国际性COS开发方法也是如此。
