Retention in Cancer Clinical Trials: Modeling Patients' Risk Benefit Assessments

癌症临床试验中的保留：患者风险效益评估建模

• 批准号: 8978309
• 负责人: CONNIE Marie ULRICH
• 金额: $ 42.76万
• 依托单位: UNIVERSITY OF PENNSYLVANIA
• 依托单位国家: 美国
• 财政年份: 2014
• 资助国家: 美国
• 起止时间: 2014-12-04 至 2019-11-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8978309
• 关键词: Adult; Behavioral Research; Benefits and Risks; Bioethics; Cancer Patient; Characteristics; Charge; Clinical; Clinical Investigator; Clinical Trials; Cluster Analysis; Communication; Complex; Consent; Decision Aid; Decision Making; Development; Diagnostic Neoplasm Staging; Dropout; Drops; Enrollment; Ethical Issues; Ethics; Funding; Health; Hour; Informed Consent; Institutional Review Boards; Interview; Knowledge; Lead; Learning; Life; Malignant Neoplasms; Maps; Measures; Methods; Modeling; Names; Outcome Study; Participant; Patient risk; Patients; Perception; Personal Satisfaction; Persons; Physicians; Prevalence; Process; Prognostic Factor; Provider; Research; Research Personnel; Research Support; Respondent; Risk; Risk-Benefit Assessment; Science; Source; Structure; Techniques; Therapeutic; Time; Translations; Trust; Typology; Withdrawal; Work; base; cancer care; cancer clinical trial; cancer type; cohort; design; effective therapy; experience; information processing; instrument; meetings; member; novel; physical symptom; pressure; psychological symptom; social; volunteer

Description: This application responds to PA-11-180: Research on Ethical Issues in Biomedical, Social and Behavioral Research with a focus on seriously ill cancer patients enrolled in cancer clinical trials (CCTs) and the ethics of science. Risk-benefit assessment is an essential component of informed consent and provides information central to patient-participants' research-related decisions in CCTs. Indeed, institutional review boards are charged with discerning whether the "risks are reasonable in relation to anticipated benefits, if any, to subjects." However, patient-participants' risk-benefit assessments may differ in significant ways, especially if they are confronted with a life-limiting illness such as cancer, have no other means to receive cancer care, or simply place their trust in the physician investigator and other study team members. A critical gap exists between the theoretical ideal of informed consent (i.e., all information processed and risks and benefits thoroughly evaluated) and how people, particularly those with serious illnesses, make research participation decisions. When patient-participants do not weigh the risks and benefits of trial participation, it may lead to compromised study outcomes, participant withdrawal, and sub-optimal patient and investigator experiences. Building on the work of a recently completed R21 study, this R01 application will innovatively examine how patient-participant, investigator, clinical characteristics and other external factors (pressure from others) influence risk-benefit assessment and subsequent retention in CCTs. To fully capture the experiences of patient-participants, we uniquely measure both the prevalence (assessed versus not assessed) and the extent/degree (extent of assessment and intensity of perceptions) of participants' risk-benefit assessment. Our specific aims intend to: 1) examine the relationship between patient-participant, clinical, and investigator characteristics and other external influences on patient-participants' risk-benefit assessment; 2) examine the relationship between risk-benefit assessment and retention in CCTs after adjusting for important prognostic factors a priori; and 3) define, name, and profile (through cluster analysis techniques) a typology of patient- participants based on their CCT risk-benefit assessment. Moreover, we will model/map via perceptual mapping (multidimensional scaling) how each patient type conceptualizes the risks and benefits involved in CCTs. We will also explore whether risk-benefit perceptions change during the course of CCT enrollment among the first time cohort of participants we enroll (baseline: 30-59 days since consent; second assessment: 90 days since CCT enrollment) in order to help assess potential differential informational or recall bias. These aims will be met through a mixed methods approach with 432 participants enrolled in CCTs. The complementary qualitative semi-structured interviews will lead to a deeper understanding of risk-benefit assessment as well as examine the factors that influence participant withdrawal. Thus, this project will fundamentally advance our theoretical, empirical, and clinical understanding of risk-benefit assessment in CCTs for those who are seriously ill.

描述：此应用程序响应 PA-11-180：生物医学、社会和行为研究中的伦理问题研究，重点关注参加癌症临床试验 (CCT) 的重病癌症患者和科学伦理。风险效益评估是知情同意的重要组成部分，为患者参与者在 CCT 中做出与研究相关的决策提供核心信息。事实上，机构审查委员会负责辨别“相对于受试者的预期收益（如果有的话）而言，风险是否合理”。然而，患者参与者的风险效益评估可能存在显着差异，特别是如果他们面临癌症等危及生命的疾病，没有其他方式接受癌症护理，或者只是信任医生研究人员和其他研究小组成员。知情同意的理论理想（即处理所有信息以及彻底评估风险和收益）与人们（特别是患有严重疾病的人）如何做出研究参与决策之间存在着重大差距。当患者参与者不权衡参与试验的风险和益处时，可能会导致研究结果受损、参与者退出以及患者和研究者体验不佳。该 R01 应用程序以最近完成的 R21 研究工作为基础，将创新性地研究患者参与者、研究者、临床特征和其他外部因素（来自他人的压力）如何影响风险效益评估和随后的 CCT 保留。为了充分捕捉患者参与者的经历，我们独特地测量了参与者风险效益评估的患病率（评估与未评估）和范围/程度（评估程度和感知强度）。我们的具体目标是：1）检查患者-参与者、临床和研究者特征之间的关系以及对患者-参与者风险效益评估的其他外部影响； 2) 在先验调整重要预后因素后，检查风险效益评估与 CCT 保留之间的关系； 3) 定义、命名和描述（通过聚类分析技术）类型学 基于 CCT 风险效益评估的患者参与者。此外，我们将通过感知映射（多维尺度）对每种患者类型如何概念化 CCT 所涉及的风险和益处进行建模/映射。我们还将探讨我们招募的第一批参与者在 CCT 招募过程中风险收益认知是否发生变化（基线：同意后 30-59 天；第二次评估：CCT 注册后 90 天），以帮助评估潜力差异信息或回忆偏差。这些目标将通过 432 名参加 CCT 的参与者的混合方法来实现。补充性的定性半结构化访谈将加深对风险收益评估的理解，并检查影响参与者退出的因素。因此，该项目将从根本上推进我们对重病患者 CCT 风险效益评估的理论、实证和临床理解。