Waiting for p<0.05

In this submission, PatientsLikeMe considers the risks, benefits, and opportunity for collecting and analyzing data for ALS patients who are taking part in clinical trials. Our intent is to encourage discussion and debate about the best way to address the complex medical, ethical, and scientfic issues surrounding the collection of such data. ==================================================== 26.10.2012 Update: After receiving feedback from study investigators who passed on the concerns of patients, as well as one of the sponsors of an ongoing trial, we are updating to the paper. While we stand by the methods and the data shown previously, we accept that given that between 50-67% of trial patients were on study drug while between 33-50% of patients were on placebo, the results as we originally presented them may be open to misinterpretation. Therefore we have clarified Table 1 and Figure 1 to be more easily interpretable. This does not change our conclusions. Table 1 was modified to present results of two different scenarios, based on different assumptions about for the calculation of the treatment effect size. (Please see the manuscript for details.) We also added Table 2 to more completely present the side effect analysis, as well as providing more detail on the interpretation of these results for the different trials. Finally, we modifed Figure 1 to reflect the most likely estimate of the treatment effect and its distribution, under the assumption that the balance between treatment and placebo trials arms was the same for PatientsLikeMe participants as it was in the clinical trials as a whole.

本投稿中，PatientsLikeMe针对参与临床试验的肌萎缩侧索硬化症（Amyotrophic Lateral Sclerosis, ALS）患者，探讨了收集与分析相关数据的风险、收益及应用前景。本文旨在鼓励讨论与争鸣，以探寻解决此类数据收集过程中涉及的复杂医学、伦理及科学问题的最优路径。
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2012年10月26日更新：在收到转达患者顾虑的临床试验研究者反馈，以及某项在研试验赞助商的意见后，我们对本文进行了修订。尽管我们坚持此前呈现的研究方法与数据，但我们意识到，鉴于试验中50%-67%的患者使用研究药物，而33%-50%的患者使用安慰剂，我们最初呈现的结果可能存在被误读的风险。因此，我们对表1和图1进行了清晰化优化，以提升其可读性。此次修订并未改变本文的核心结论。表1已修改为基于不同治疗效应量计算假设的两种场景下的结果呈现（详见手稿）。此外，我们新增了表2以更全面地展示不良反应分析，并补充了不同试验结果解读的相关细节。最后，我们修订了图1，以反映在PatientsLikeMe参与者与整体临床试验中治疗组与安慰剂组入组比例一致的假设下，治疗效应最可能的估计值及其分布情况。