Head and Neck Cancer Patient Perspectives on Phase O Trials

Novel drug discovery is a lengthy process, and for head and neck cancer (HNC), few antineoplastics have been developed in the past decades. This has led to a relative stagnation of HNC survival rates. Phase 0 trials offer a solution to the slow cancer drug development. By injecting a microdose of a novel antineoplastic agent directly into a tumor in-vivo, drug pharmacokinetics and pharmacodynamics can be studied after tumor resection and offers more accurate findings than with the utilization of animal models. Phase 0 trials do not offer therapeutic benefit to the participants but also carry minimal risk. Challenges with patient enrollment in phase 0 trials have hindered a more widespread implementation. The aim of this study was to better understand patient motivations and perceptions of phase 0 trials when considering whether to enroll. A hypothetical vignette survey was administered to 80 HNC patients presenting to their otolaryngology clinic. Among the sample, a majority of participants (78.8%) reported being likely to participate in a phase 0 trial, whereas a minority (21.2%) reported being unlikely to participate. Patients’ main reason to agree to participate was a desire to help future cancer patients (73%). Younger participants were significantly more likely to choose to participate. Reasons not to participate were more varied. Concern for injection complications (25%), fear of being experimented on (20%), and not wanting to be in a clinical trial (19%) were the most frequently selected answers. Black-identifying participants were 9.3 times more likely (95% CI 2.04, 43.06) to choose fear of being experimented on than non-black participants (p=0.0017). Only 10% of responders chose the lack of therapeutic benefit as a reason not to participate. Several patients mentioned logistical concerns, including transportation and financial support, as being potential deterrents to their participation. These findings showcase a number of barriers and challenges faced by patients that need to be addressed in order to improve enrollment in phase 0 trials.