Doctor of Philosophy
This thesis addresses the tension in pragmatic cluster-randomized trials between their social value and the requirement to respect the autonomy of research participants. Pragmatic trials are designed to evaluate the effectiveness of treatments in real-world settings to inform clinical decision-making and promote cost-efficient care. These trials are often embedded into clinical settings and ideally include all patients who would receive the treatments under investigation as a part of routine care. Trialists increasingly adopt cluster-randomized designs—in which intact groups, such as hospitals or clinics, are allocated randomly to study interventions—to simplify the inclusion of all patients. But including all-comers conflicts with the requirement to obtain written informed consent from research participants. Since informed consent is central to respecting patient autonomy, the question arises: how can the ends of autonomy and pragmatism be served simultaneously?
Some philosophers argue that patients have an obligation to participate in clinical research and that this may negate consent requirements. I argue that while there may be grounds for a prima facie obligation for patients to participate in clinical research, no compelling argument has demonstrated that the obligation is enforceable. Others assert that broad application of a waiver of consent will facilitate the conduct of pragmatic cluster-randomized trials. I demonstrate that this proposal sharply conflicts with the historical origins of the waiver. I articulate a novel moral foundation for the use of a waiver of consent and show that when trials evaluate treatments delivered directly to patients (e.g., drugs or vaccines), the autonomy interests at stake for participants are too substantial to permit its use.
My solution draws a distinction between consent requirements in existing policy and consent as an autonomous authorization. As many pragmatic cluster-randomized trials are conducted in primary care settings with no research staff, I argue that patient autonomy can be promoted and protected using clinical-style consent, in which health providers seek verbal informed consent from patients and document it in the electronic health record. This approach has been associated with high rates of recruitment and, thus, may satisfy both requirements for social value and respect for autonomy.
Summary for Lay Audience
Most clinical trials test whether a new medical treatment works in ideal conditions. But the real world is messy and unpredictable. Patients and doctors need to know whether medical treatments work when administered in doctors’ offices and community hospitals. The solution: pragmatic trials. These trials mirror real-world clinical settings and include all patients who would receive the treatments under investigation as a part of their routine care. While patients are typically enrolled into a trial after they provide their written informed consent, soliciting their consent can disrupt the workflow of busy clinics to the extent that the trial no longer mirrors clinical practice. Seeking consent can also result in people refusing to participate, consequently undermining the aim of pragmatic trials to include everyone. Since consent is central to respecting the autonomy of prospective research participants, the question arises: when written informed consent is a barrier to the conduct of pragmatic trials, how can we respect patients’ choices without undermining the aim to include all or most patients in these trials?
Some philosophers argue that patients have a moral duty to participate in pragmatic trials without consent, while others suggest that pragmatic trials will often meet the regulatory criteria to waive consent requirements. I argue that both solutions fail to respect patient autonomy. My solution is to return to the ethical foundation of informed consent. Informed consent is grounded in the principle of respect for autonomy, and it is meant to allow patients to autonomously authorize their participation in research. When written informed consent cannot be obtained in a pragmatic trial, I argue that alternative approaches to obtaining consent are appropriate. For example, health providers can seek verbal consent from patients and document their agreement in their medical records. This solution promotes patient autonomy while simultaneously facilitating the conduct of pragmatic trials.
Goldstein, Cory E., "A Critical Examination of Informed Consent Approaches in Pragmatic Cluster-Randomized Trials" (2022). Electronic Thesis and Dissertation Repository. 8438.