Peay Holly L, Biesecker Barbara B, Wilfond Benjamin S, Jarecki Jill, Umstead Kendall L, Escolar Diana M, Tibben Aad
1 RTI International, Research Triangle Park, NC, USA.
2 Parent Project Muscular Dystrophy, Hackensack, NJ, USA.
Clin Trials. 2018 Apr;15(2):139-148. doi: 10.1177/1740774517751118. Epub 2018 Feb 23.
BACKGROUND/AIMS: Pediatric rare disease presents a challenging situation of high unmet need and a limited pool of potential clinical trial participants. Understanding perspectives of parents of children who have not participated in trials may facilitate approaches to optimize participation rates. The objective of this study was to explore factors associated with parental interest in enrolling children with pediatric neuromuscular disorders in clinical trials.
Parents of individuals with Duchenne or Becker muscular dystrophy and spinal muscular atrophy were recruited through advocacy organizations, a registry, and clinics. These parents ( N = 203) completed a questionnaire including assessments of barriers and facilitators to clinical trial participation, parents' interest in trial participation, and their perceptions of others' views about participation in a clinical trial.
Trial interest in participating parents was high (64% combined group). The most highly endorsed barrier to participation was the possibility of receiving placebo, followed by not having enough information on risks and trial procedures. Compared to parents of children with Duchenne or Becker muscular dystrophy, parents of children with spinal muscular atrophy endorsed significantly more information and knowledge barriers. The greatest facilitators of participation were (1) confidence in improving disease understanding and (2) guarantee to receive the treatment after a successful trial. A logistic regression model, χ (4, n = 188) = 80.64, p < .001, indicated that higher perceived barriers and more frequent trial communication by the provider were associated with lower interest, while positive trial perceptions by the child's providers and concordance in trial perceptions among those close to the decision-maker were associated with higher interest.
We found high parental interest in pediatric neuromuscular trials that was tempered by concerns about the potential for randomization to a placebo arm. Participants perceived that their trial participation would be facilitated by additional education and guidance from their clinicians. Yet, intentions were negatively associated with frequency of provider communication, perhaps reflecting waning parental interest with a greater understanding of limitations in trial access, increased sophistication in their understanding of trial design, and appreciation of potential burden. To support parents' informed decisions, it is important to educate them to evaluate the quality of research, as well as providing lay information explaining the use of placebo, trial processes, and potential barriers to long-term drug access. Our findings should inform the development of targeted educational content, clinician training, and decision support tools.
背景/目的：儿科罕见病面临着未满足需求高且潜在临床试验参与者群体有限的挑战性局面。了解未参与试验的儿童家长的观点可能有助于找到优化参与率的方法。本研究的目的是探讨与家长让患有儿科神经肌肉疾病的儿童参与临床试验的兴趣相关的因素。
通过倡导组织、登记处和诊所招募了患有杜氏或贝克型肌营养不良症以及脊髓性肌萎缩症患者的家长。这些家长（N = 203）完成了一份问卷，包括对临床试验参与的障碍和促进因素、家长对试验参与的兴趣以及他们对他人对参与临床试验看法的认知的评估。
参与试验的家长兴趣较高（合并组为64%）。最受认可的参与障碍是可能接受安慰剂，其次是没有足够的风险和试验程序信息。与患有杜氏或贝克型肌营养不良症儿童的家长相比，患有脊髓性肌萎缩症儿童的家长认可的信息和知识障碍明显更多。参与的最大促进因素是：（1）对增进疾病理解的信心；（2）成功试验后保证获得治疗。一个逻辑回归模型，χ（4，n = 188）= 80.64，p <.001，表明较高的感知障碍和提供者更频繁的试验沟通与较低的兴趣相关，而儿童提供者对试验的积极看法以及接近决策者的人在试验看法上的一致性与较高的兴趣相关。
我们发现家长对儿科神经肌肉试验兴趣较高，但对随机分配到安慰剂组的可能性存在担忧。参与者认为临床医生提供更多教育和指导将有助于他们参与试验。然而，意愿与提供者沟通的频率呈负相关，这可能反映出随着对试验准入限制的更深入了解、对试验设计理解的提高以及对潜在负担的认识，家长的兴趣逐渐减弱。为支持家长做出明智的决定，重要的是教育他们评估研究质量，同时提供通俗易懂的信息解释安慰剂的使用、试验过程以及长期获得药物的潜在障碍。我们的研究结果应为有针对性的教育内容、临床医生培训和决策支持工具的开发提供参考。
