The patient's view on rare disease trial design - a qualitative study
The patient's view on rare disease trial design - a qualitative study
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DOI：
10.1186/s13023-019-1002-z
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发表时间：
2019-02-07
影响因子：
3.7
通讯作者：
van der Lee, J. H.
中科院分区：
文献类型：
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作者：
Gaasterland, C. M. W.;Jansen-van der Weide, M. C.;van der Lee, J. H.
背景罕见病的临床试验比常见病的临床试验更具挑战性。少数合格的试验参与者，经常因罕见病患者的异质性而复杂化，阻碍了经典“随机对照试验”的设计和实施。因此，统计学家开发了新的设计。但是，必须了解可能影响审判可行性的设计方面。如果患者或父母认为参与负担不成比例，则招募可能失败或受试者可能在试验完成前退出。为了最大限度地提高在小人群中的试验成功的机会，重要的是要知道哪些方面的试验设计被认为是重要的patients.ResultsWe采访了所有10名成员的患者智囊团（PTT）的ASTERIX项目，欧洲研究财团的方法在小人群中的临床试验。PTT成员是罕见病患者代表，他们已经完成了临床试验方法的广泛培训。我们根据扎根理论，采用主位分析法对访谈进行了定性分析，并将访谈主题按时间顺序分为四个主位：1。参与试验设计; 2.试验设计意见; 3.参加试验; 4.审判后的阶段。我们的主要发现是，PTT成员建议患者从早期阶段就参与试验设计，并对试验中选择的结局和测量工具、研究长度、参与者的选择以及发送给潜在参与者的信息产生影响。此外，根据PTT成员的说法，患者群体应考虑建立疾病登记处，应尽量减少安慰剂组，并建议进行更多的临床试验教育。结论接受过临床试验方法学教育的罕见病患者代表认为让患者代表参与研究很重要在早期阶段。他们可以在试验设计中提供建议，以优化试验参与的潜在获益和负担比例以及所选结局指标和入选和排除标准。
BackgroundClinical trials in rare diseases are more challenging than trials in frequent diseases. Small numbers of eligible trial participants, often complicated by heterogeneity among rare disease patients, hamper the design and conduct of a classical' Randomized Controlled Trial. Therefore, novel designs are developed by statisticians. However, it is important to be aware of possible design aspects that may jeopardize the feasibility of trial conduct. If the burden of participation is considered out of proportion by patients or parents, recruitment may fail or participants may drop out before trial completion. In order to maximize the chance of success of trials in small populations, it is important to know which aspects of trial design are considered important by patients.ResultsWe have interviewed all ten members of the Patient Think Tank (PTT) of the ASTERIX project, a European research consortium on methodology for clinical trials in small populations. The PTT members are rare disease patient representatives who have completed extensive training in clinical trial methodology. We have analyzed the interviews qualitatively according to Grounded Theory using a thematic analysis, and we structured the topics in four chronologically ordered themes: 1. Involvement in trial design; 2. Opinions on trial design; 3. Trial participation; 4. Phase after the trial. Our main findings are that the PTT-members recommend that patients are involved in trial design from an early stage on, and have influence on the outcomes and measurement instruments that are chosen in the trial, the length of the study, the choice of participants, and the information that is sent to potential participants. Also, according to the PTT-members, patient groups should consider setting up disease registries, placebo groups should be minimized, and more education on clinical trials is advised.ConclusionsRare disease patient representatives who have been educated about clinical trial methodology think it is important to involve patient representatives in research at an early stage. They can be of advice in trial design in such a way that the ratio of potential benefit and burden of trial participation as well as the chosen outcome measures and in- and exclusion criteria are optimized.