Experience With Direct-to-Patient Recruitment for Enrollment Into a Clinical Trial in a Rare Disease: A Web-Based Study.
Experience With Direct-to-Patient Recruitment for Enrollment Into a Clinical Trial in a Rare Disease: A Web-Based Study.
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DOI：
10.2196/jmir.6798
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发表时间：
2017-02-28
影响因子：
7.4
通讯作者：
Vasculitis Clinical Research Consortium
中科院分区：
文献类型：
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作者：
Krischer J;Cronholm PF;Burroughs C;McAlear CA;Borchin R;Easley E;Davis T;Kullman J;Carette S;Khalidi N;Koening C;Langford CA;Monach P;Moreland L;Pagnoux C;Specks U;Sreih AG;Ytterberg S;Merkel PA;Vasculitis Clinical Research Consortium
临床试验的目标样本量通常需要多中心（卓越中心，CoE）方法，这会增加相关的复杂性、成本和监管要求。需要对照这一标准模式对备选征聘战略进行测试。我们研究的目的是测试是否基于网络的直接招募方法（以患者为中心，PC）使用社会营销策略提供了一个可行的选择，以卓越中心的招聘方法。在一项随机对照试验（RCT）中，比较了PC招募和基于网络的知情同意与CoE招募，该试验旨在比较持续与停止低剂量泼尼松对肉芽肿伴多血管炎（GPA）患者的缓解维持作用。保护委员会的做法不如欧洲委员会的做法成功。经医生确认符合条件的患者入组PC组13例中有10例（77%），入组CoE组51例中有49例（96%）（P= 0.05）。这两种方法在合格性方面没有显著差异，在CoE中发现34%的潜在参与者不合格，而PC组为22%（P=.11），在提供者接受度方面也没有显著差异，22%对26%（P=.78）。PC组和欧洲委员会组的个人知识调查显示，对试验的理解没有差异。与欧洲委员会的做法相比，保护委员会的征聘工作远没有那么成功。然而，PC方法在确认合格性方面很好，并且与CoE方法一样为提供者所接受，也为患者所理解。PC方法应在其他临床环境中进行评估，以更好地了解其潜力。
The target sample size for clinical trials often necessitates a multicenter (center of excellence, CoE) approach with associated added complexity, cost, and regulatory requirements. Alternative recruitment strategies need to be tested against this standard model. The aim of our study was to test whether a Web-based direct recruitment approach (patient-centric, PC) using social marketing strategies provides a viable option to the CoE recruitment method. PC recruitment and Web-based informed consent was compared with CoE recruitment for a randomized controlled trial (RCT) of continuing versus stopping low-dose prednisone for maintenance of remission of patients with granulomatosis with polyangiitis (GPA). The PC approach was not as successful as the CoE approach. Enrollment of those confirmed eligible by their physician was 10 of 13 (77%) and 49 of 51 (96%) in the PC and CoE arms, respectively (P=.05). The two approaches were not significantly different in terms of eligibility with 34% of potential participants in the CoE found to be ineligible as compared with 22% in the PC arm (P=.11) nor in provider acceptance, 22% versus 26% (P=.78). There was no difference in the understanding of the trial as reflected in the knowledge surveys of individuals in the PC and CoE arms. PC recruitment was substantially less successful than that achieved by the CoE approach. However, the PC approach was good at confirming eligibility and was as acceptable to providers and as understandable to patients as the CoE approach. The PC approach should be evaluated in other clinical settings to get a better sense of its potential.