Inclusion of progression criteria in external randomised pilot trials: a cross-sectional study of funding applications submitted to the NIHR Research for Patient Benefit Programme.
Inclusion of progression criteria in external randomised pilot trials: a cross-sectional study of funding applications submitted to the NIHR Research for Patient Benefit Programme.
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作者：
外部随机初步试验旨在评估未来确定性随机对照试验（RCT）是否可行。预先规定的进展标准有助于指导对初步试验结果的解释，以决定是否以及如何进行确定性试验。我们的目的是检查研究人员如何报告和计划评估进展标准，在外部试点试验资金申请提交给NIHR研究患者福利计划。我们对2017年7月至2019年7月期间提交给NIHR RfPB的外部随机外部试点试验的第1阶段（大纲）和相应的第2阶段（全部）资助申请中的进展标准进行了一项横断面研究。在100个第一阶段大纲申请中，95个符合纳入资格（其中52个被邀请参加第二阶段的全面申请，43个被拒绝），49/52个符合纳入第二阶段的全面申请（其中35个获得资助，14个被拒绝）。在第一阶段评估的申请中，超过一半（48/95，51%）和在第二阶段评估的申请中，73%（36/49）在他们的研究计划中包括了进展标准。进展标准最常以停-行格式报告，通常在确定可行性时应考虑其他特定因素（第1阶段33/48，69%;第2阶段21/36，58%）。招聘和保留是最常见的可行性指标，以告知进展标准。三分之一的申请为他们的目标提供了一些理由或理由（第一阶段16/48，33%;第二阶段12/36，33%）。资助委员会的反馈意见中提到了超过20%的申请（第一阶段22/95，23%;第二阶段11/49，22%）的进展标准，要求增加进展标准或为规定的标准提供理由。我们的研究结果表明，研究人员并不总是在提交给研究资助者的外部随机试点试验申请中包括进展标准。这可能导致随机化试点试验可行性评估缺乏透明度。开放科学框架osf.io/89ap7，于2021年6月29日注册。
External randomised pilot trials aim to assess whether a future definitive randomised controlled trial (RCT) is feasible. Pre-specified progression criteria help guide the interpretation of pilot trial findings to decide whether, and how, a definitive trial should be conducted. We aimed to examine how researchers report and plan to assess progression criteria in external pilot trial funding applications submitted to the NIHR Research for Patient Benefit Programme. We conducted a cross-sectional study of progression criteria inclusion in Stage 1 (outline) and corresponding Stage 2 (full) funding applications for external randomised external pilot trials submitted to NIHR RfPB between July 2017 and July 2019. Of the 100 Stage 1 outline applications assessed, 95 were eligible for inclusion (of these, 52 were invited to Stage 2 full application; 43 were rejected) and 49/52 were eligible for inclusion at Stage 2 full application (of these, 35 were awarded funding; 14 were rejected). Over half of applications assessed at Stage 1 (48/95, 51%), and 73% of those assessed at Stage 2 (36/49) included progression criteria in their research plans. Progression criteria were most often reported in a stop-go format, often with additional specified factors that should be considered when determining feasibility (Stage 1 33/48, 69%; Stage 2 21/36, 58%). Recruitment and retention were the most frequent indicators of feasibility to inform progression criteria. One-third of applications provided some justification or rationale for their targets (Stage 1 16/48, 33%; Stage 2 12/36, 33%). Funding committee feedback mentioned progression criteria in over 20% of applications (Stage 1 22/95, 23%; Stage 2 11/49, 22%) to either request the addition of progression criteria or provide justification for the criteria stipulated. Our findings indicate that researchers do not always include progression criteria in external randomised pilot trial applications submitted to research funders. This can result in a lack of transparency in the assessment of randomised pilot trial feasibility. Open Science Framework osf.io/89ap7, registered 29th June 2021.
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影响因子：
2.9
作者：
Avery KN;Williamson PR;Gamble C;O'Connell Francischetto E;Metcalfe C;Davidson P;Williams H;Blazeby JM;members of the Internal Pilot Trials Workshop supported by the Hubs for Trials Methodology Research
通讯作者：
members of the Internal Pilot Trials Workshop supported by the Hubs for Trials Methodology Research
影响因子：
2.9
作者：
Mellor K;Eddy S;Peckham N;Bond CM;Campbell MJ;Lancaster GA;Thabane L;Eldridge SM;Dutton SJ;Hopewell S
通讯作者：
Hopewell S
影响因子：
2.5
作者：
Herbert, Esther;Julious, Steven A.;Goodacre, Steve
通讯作者：
Goodacre, Steve
影响因子：
1.7
作者：
Morgan B;Hejdenberg J;Kuleszewicz K;Armstrong D;Ziebland S
通讯作者：
Ziebland S
影响因子：
3.7
作者：
Morgan, Ben;Hejdenberg, Jennie;Armstrong, David
通讯作者：
Armstrong, David