National Heart Lung institute, Imperial College London, London, UK
Department of Paediatric Respiratory Medicine, Royal Brompton Hospital, London, UK.
BMJ Open. 2021 Mar 2;11(3):e045803. doi: 10.1136/bmjopen-2020-045803.
Engaging people with cystic fibrosis (CF) in clinical trials is critical to improving outcomes for this fatal disease. Following extensive exploration of engagement in CF trials we believe six key concepts require a quantitative understanding of their influence in the current CF trials landscape including how controversial issues like placebos, washouts, stipend provision and location of trial visits are viewed by the CF community and how these might be modified depending on the type of medicine being investigated and the mechanism of access to the drug on trial completion.
We have designed and will administer an online discrete choice experiment to elicit and quantify preferences of people with CF for these trials' attributes and estimate the relative importance of an attribute when choosing to participate in a trial. The cross-sectional data generated will be explored using conditional multinomial logit model. Mixed logit models such as the random-parameters logit and a latent class models will be used to explore preference heterogeneity. To determine the relative importance of an attribute, the difference between the attribute level with the highest preference weight and the level with the lowest preference weight will be calculated.
Imperial College London Joint Research Compliance Office has granted ethical approval for this study. Patient consent will be sought following full explanation. No identifying information will be collected. Dissemination will be via international conferences, peer-review publication and patient accessible forums. Major CF trials networks have agreed to incorporate our findings into their review process, meaning our results can realistically influence and optimise CF trial delivery.
CRD42020184886.
让囊性纤维化（CF）患者参与临床试验对于改善这种致命疾病的预后至关重要。在对 CF 试验的参与情况进行了广泛探索之后，我们认为有六个关键概念需要定量了解它们在当前 CF 试验领域的影响，包括像安慰剂、洗脱期、津贴提供和试验访视地点等有争议的问题，CF 社区如何看待这些问题，以及这些问题如何根据所研究药物的类型和试验完成时获得药物的机制进行修改。
我们设计并将进行一项在线离散选择实验，以引出和量化 CF 患者对这些试验属性的偏好，并估计在选择参与试验时，一个属性的相对重要性。生成的横断面数据将使用条件多项逻辑回归模型进行探索。混合逻辑模型，如随机参数逻辑和潜在类别模型，将用于探索偏好异质性。为了确定一个属性的相对重要性，将计算具有最高偏好权重的属性水平与具有最低偏好权重的属性水平之间的差异。
伦敦帝国理工学院联合研究合规办公室已批准该研究的伦理。在充分解释后，将寻求患者的同意。不会收集任何识别信息。传播将通过国际会议、同行评审出版物和患者可访问的论坛进行。主要的 CF 试验网络已同意将我们的发现纳入他们的审查过程，这意味着我们的结果可以真正影响和优化 CF 试验的实施。
PROSPERO 注册号：CRD42020184886。
