PD patients participation preferences regarding gene transfer therapy studies

这项建议描述了几项计划研究中的第一项，这些研究为PDGTSG开发帕金森氏症的基因转移疗法提供了循证指导。 由于第一阶段研究的设计或预期不会让受试者直接受益，而且有证据表明，受试者可能错误地希望或期望从这类研究中直接受益(通常被称为“治疗误解”)，因此此类研究存在相当大的伦理担忧。对于帕金森病基因转移的第一阶段研究来说，这种担忧被放大了，因为它将涉及通过神经外科手术将修改后的病原体永久植入异体DNA。我们如何才能知道“绝望”患者承担这种风险的意愿是否真的有效和自主呢？这个项目旨在通过回答以下一组问题来回答这个主要问题：这种意愿有多普遍，有多强烈？这种意愿是否反映了一种误解；如果是，那么这种意愿有多强烈？ 这种误解的倾向是什么？对帕金森病患者偏好背后的动机的深入研究揭示了什么？最后，哪些患者特征预测或解释了他们参与的意愿，如果适用，他们倾向于成为治疗误解的牺牲品？本项目将采用定性和定量相结合的方法。后者将涉及一种改进的标准赌博技术，它将允许量化参与研究的偏好强度以及对治疗错误概念倾向的测量；定性方法将增强偏好测量的有效性，并为受试者的偏好提供更深层次的解释框架。

This proposal describes the first of several planned studies to provide an evidence-based guidance to the PDGTSG in its development of a gene-transfer therapy for PD. Because Phase I studies are not designed or expected to benefit the subjects directly and because there is evidence that subjects may erroneously hope for or expect direct benefit from such studies (often called the 'therapeutic misconception ), there is considerable ethical concern about such studies. This concern is magnified for a Phase I study of gene transfer for PD because it will involve the permanent insertion of foreign DNA via a modified pathogen, introduced directly into the brain by neurosurgery. How do we know if a 'desperate' patient's willingness to take on such risks is truly valid and autonomous? This project is designed to answer that main question by answering the following set of questions: How common and how strong is that willingness'? Is that willingness reflective of a misconception; if so, how strong is the tendency to fall under such a misconception? What does an in depth examination of the motivations behind the PD patients' preferences reveal? And finally, which patient characteristics predict or explain their willingness to participate and, if applicable, their tendency to fall prey to the therapeutic misconception? This project will employ both qualitative and quantitative methods. The latter will involve a modified standard gamble technique that will allow a quantification of the strength of preferences for research participation as well as a measurement of the tendency toward the therapeutic misconception; qualitative methods will enhance validity of the preference measurements as well as provide a deeper explanatory framework for the subjects' preferences.