Department of Hematology and Medical Oncology, Emory University School of Medicine, Atlanta, GA, USA.
Cancer. 2012 Sep 15;118(18):4571-8. doi: 10.1002/cncr.27397. Epub 2012 Jan 31.
Ethical concerns about phase 1 trials persist. Important conceptual advances have been made in understanding concepts used to describe misunderstanding. However, a systematic, empirical evaluation of the frequency of misunderstanding incorporating recent developments is lacking.
The authors queried 95 participants in phase 1 trials to provide a more sophisticated estimate of the proportion who had therapeutic misconception (TM), defined as misunderstanding the research purpose or how research differs from individualized care, and therapeutic misestimation (TMis), defined as incorrectly estimating the chance of a research trial benefit as >20% or underestimating risk as 0%.
Sixty-five of 95 respondents (68.4%) had TM, which was associated in a multivariate analysis with lower education and family income (P = .008 and P = .001, respectively), but TM was not associated with the vulnerability of having hardly any treatment options. Eighty-nine of 95 respondents (94%) had TMis, although only 18% reported this was a factual estimate. Although the risks of investigational agents and those exacerbated by research, such as uncertain outcomes, were mentioned (39% and 41% of respondents, respectively), risks novel to research, such as research biopsies, were rarely mentioned (3% of respondents). Although most of these respondents believed that their chance of benefit was greater and that their risk was lower than the population chance (optimists) (54.6%), a substantial minority of respondents (37.6%) were pessimists.
TM continues to be prevalent. Estimates of personal benefit were not usually meant to report facts, it remains unknown whether respondents in the current study had TMis. Although they are not more vulnerable, phase 1 participants need improved understanding of key TM concepts, with attention to risks that are not present in standard of care.
人们对 1 期临床试验仍存在伦理方面的担忧。在理解用于描述误解的概念方面已经取得了重要的概念性进展。然而，缺乏对纳入最新进展的误解频率进行系统的、经验性评估。
作者对 95 名参加 1 期临床试验的参与者进行了查询，以更精确地估计出现治疗误解（TM）的比例，TM 定义为误解研究目的或研究与个体化护理的区别；以及治疗估计错误（TMis），定义为错误地估计研究试验获益的可能性>20%或低估风险为 0%。
在多变量分析中，95 名应答者中有 65 名（68.4%）出现 TM，TM 与较低的教育程度和家庭收入相关（P=.008 和 P=.001），但 TM 与几乎没有任何治疗选择的脆弱性无关。95 名应答者中有 89 名（94%）出现 TMis，尽管只有 18%的人报告这是一个事实估计。尽管提到了研究药物的风险以及研究加剧的风险，如不确定的结果（分别为 39%和 41%的应答者），但很少有应答者提到研究特有的风险，如研究活检（3%的应答者）。尽管大多数应答者认为他们的获益机会更大，风险低于人群机会（乐观者）（54.6%），但相当一部分应答者（37.6%）是悲观者。
TM 仍然普遍存在。个人获益的估计通常不是为了报告事实，目前研究中的应答者是否存在 TMis 尚不清楚。尽管他们没有更脆弱，但 1 期参与者需要更好地理解 TM 的关键概念，注意标准护理中不存在的风险。
