Department of Paediatric Respiratory Medicine, Royal Brompton Hospital and National Heart Lung Institute Imperial College London, UK.
UCL Great Ormond Street Institute of Child Health, and Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK.
J Cyst Fibros. 2021 Nov;20(6):978-985. doi: 10.1016/j.jcf.2021.03.027. Epub 2021 Apr 17.
Trial participation can allow people with CF early access to CFTR modulator therapies, with high potential for clinical benefit. Therefore, the number of people wishing to participate can substantially exceed the number of slots available. We aimed to understand how the CF community thinks slots to competitive trials should be allocated across the UK and whether this should be driven by clinical need, patients' engagement/adherence or be random. For the latter, we explored site-level versus registry-based, national randomisation processes.
We developed an online survey, recruiting UK-based stakeholders through social media, newsletters and personal contacts. Closed questions were analysed for frequencies and percentages of responses. Free-text questions were analysed using thematic analysis.
We received 203 eligible responses. Overall, 75% of stakeholders favoured allocation of slots to individual sites based on patient population size, although pharma favoured allocation based on previous metrics. Currently, few centres have defined strategies for allocating slots locally. At face-value, stakeholders believe all eligible participants should have an equal chance of getting a slot. However, further questioning reveals preference for prioritisation strategies, primarily perceived treatment adherence, although healthcare professionals were less likely to favour this strategy than other stakeholder groups. The majority of stakeholders would prefer to allocate slots and participate in trials locally but 80% said if necessary, they would engage in a system of national allocation.
Fair allocation to highly competitive trials does not appear to have a universally acceptable solution. Therefore, transparency and empathy remain critical to negotiate this uncertain territory.
参加试验可以让 CF 患者提前获得 CFTR 调节剂治疗，具有很高的临床获益潜力。因此，希望参加试验的人数可能大大超过可用名额。我们旨在了解英国 CF 社区如何考虑在英国分配竞争试验的名额，以及这些名额应该根据临床需求、患者的参与度/依从性来分配，还是应该随机分配。对于后者，我们探讨了基于现场和基于登记处的全国性随机化过程。
我们开发了一个在线调查，通过社交媒体、新闻通讯和个人联系，招募英国的利益相关者。对封闭式问题进行了分析，以了解响应的频率和百分比。对自由文本问题进行了主题分析。
我们收到了 203 份符合条件的回复。总体而言，75%的利益相关者赞成根据患者人群规模向个别站点分配名额，尽管制药公司赞成根据以往的指标进行分配。目前，很少有中心制定了在当地分配名额的策略。从表面上看，利益相关者认为所有符合条件的参与者都应该有平等的机会获得一个名额。然而，进一步的询问揭示了他们对优先排序策略的偏好，主要是基于治疗的依从性，尽管医疗保健专业人员比其他利益相关者群体更不可能赞成这种策略。大多数利益相关者更愿意在当地分配名额并参与试验，但 80%的人表示，如果有必要，他们将参与全国分配系统。
对于高度竞争的试验，公平分配似乎没有一个普遍可接受的解决方案。因此，透明度和同理心仍然是协商这一不确定领域的关键。
