Early-stage studies to larger-scale trials: investigators' perspectives on scaling-up childhood obesity interventions.
Early-stage studies to larger-scale trials: investigators' perspectives on scaling-up childhood obesity interventions.
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DOI：
10.1186/s40814-022-00991-8
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发表时间：
2022-02-07
影响因子：
1.7
通讯作者：
Beets MW
中科院分区：
文献类型：
--
作者：
von Klinggraeff L;Dugger R;Okely AD;Lubans D;Jago R;Burkart S;Weaver RG;Armstrong B;Pfledderer CD;Beets MW
试点/可行性研究通过提供有关可行性、可接受性和潜在功效的信息，在行为干预措施的开发和完善中发挥着重要作用。尽管它们的重要性和广泛的使用，行为科学家采取的方法，以扩大早期阶段的研究，以更大规模的试验很少受到关注。我们研究的目的是了解试点研究在大规模试验的开发和执行中所起的作用。我们采访了儿童肥胖研究人员，他们发表了试点行为干预措施和相同或类似干预措施的大规模试验。与会者询问了试点研究在开展大规模试验中的作用，以及根据试点结果扩大干预措施时遇到的挑战。数据进行编码和分析，使用归纳分析方法，以确定主题。24名干预者（54%为女性，年龄37-70岁，平均毕业20年）在其职业生涯中共完成了148项试点研究（平均6.4项，范围1-20），其中59%被扩大。63%的样本（n = 15）将扩展描述为资源密集型，并认为试点工作对于成功竞争资金至关重要。当被要求定义一个高质量的试点研究时，干预者描述了允许他们评估两个独立因素的研究：他们干预的组成部分（例如，可接受性，可行性）和研究参数（例如，样本量、措施）。干预者表示，更多的过程实施措施，不同的研究设计和额外的迭代可以改善扩大规模的决定。大多数人认为，试点研究可能会产生夸大的估计潜在的疗效，虽然只有九个干预提供了潜在的解决方案，以减少膨胀的措施的疗效。提出的夸大效应的主要原因包括试点研究中的高度监督（例如，研究人员的支持），依赖于主观措施，并利用方便或高度动机的样本。潜在的解决方案包括为实际实施设计试点，仅进行随机对照试点研究，以及预先注册试点研究。试点研究的目的是多方面的，被认为是获得大规模试验资金的关键。澄清初步、早期研究的形式和功能可能会提高早期研究的生产利用率，并在过渡到更大规模研究时减少疗效下降。在线版本包含补充材料，可通过10.1186/s40814-022-00991-8获得。
Pilot/feasibility studies play an important role in the development and refinement of behavioral interventions by providing information about feasibility, acceptability, and potential efficacy. Despite their importance and wide-spread use, the approaches taken by behavioral scientists to scale-up early-stage studies to larger-scale trials has received little attention. The aim of our study was to understand the role that pilot studies play in the development and execution of larger-scale trials. We conducted interviews with childhood obesity researchers who had published pilot behavioral interventions and larger-scale trials of the same or similar interventions. Questions were asked about the role of pilot studies in developing larger-scale trials and the challenges encountered when scaling-up an intervention based upon pilot findings. Data were coded and analyzed using an inductive analytic approach to identify themes. Twenty-four interventionists (54% women, 37–70 years old, mean 20 years since terminal degree) completed a total of 148 pilot studies across their careers (mean 6.4, range 1–20), of which 59% were scaled-up. Scaling was described as resource intensive and pilot work was considered essential to successfully competing for funding by 63% of the sample (n = 15). When asked to define a high-quality pilot study, interventionists described studies that allowed them to evaluate two independent factors: components of their intervention (e.g., acceptability, feasibility) and study parameters (e.g., sample size, measures). Interventionists expressed that more process implementation measures, different study designs, and additional iterations could improve decisions to scale-up. Most agreed that pilot studies were likely to produce inflated estimates of potential efficacy though only nine interventionists provided potential solutions for decreasing inflated measures of efficacy. Suggested major causes of inflated effects included high levels of oversight in pilot studies (e.g., researcher support), reliance on subjective measures, and utilizing convenience or highly motivated samples. Potential solutions included designing pilots for real-world implementation, only conducting randomized controlled pilot studies, and pre-registering pilot studies. Pilot studies purposes are multifaceted and deemed essential to obtaining funding for larger-scale trials. Clarifying the form and function of preliminary, early-stage research may enhance the productive utilization of early-stage studies and reduced drops in efficacy when transitioning to larger scale studies. The online version contains supplementary material available at 10.1186/s40814-022-00991-8.