Patient involvement in informed consent for pediatric phase I cancer research.

Child and adolescent involvement in research decision making has been of increasing interest to the pediatrics community 1, especially in the context of chronic or life-threatening illness 2–5. For example, a vast majority of children with cancer participate in a clinical trial at some point 6. Many children with cancer who enroll in trials at diagnosis do not recall or were not told that their treatment was considered research 5 or were not present for the research discussion 7, 8. Almost half reported having little to no role in the decision 5. In terms of preference, 60% reported that they preferred “total” involvement, and 40% wanted “a little” involvement in such decisions 5. Phase I studies, designed to test the safety profile of novel agents, raise particular challenges compared with other trials, because they are offered to patients who have no remaining curative options. The decision to enroll in a phase I trial can be difficult for patients and families and creates a tension between continuing cancer-directed therapies, with their concomitant side effects and low probability of benefit 9, and opting for palliative care 10. The way in which children and adolescents with cancer and their families navigate this decision is a complex process that is not well understood 10. In one prior study 11 four of the seven patients involved in a decision about a phase I study reported that a chance of cure was a factor in the final decision about enrollment, and two reported being uncomfortable with enrolling in the trial. These patients reported that they agreed because loved ones wanted them to, even though they did not desire additional disease-directed interventions. Another study included interviews with three children who were offered participation in a phase I trial 12. The hope for cure was a consistent theme from the child participants. This study reflects a step forward in our understanding of how children, adolescents, and emerging adults with cancer are involved in discussions about phase I trial enrollment. It was based on observations of actual informed consent conferences (ICCs) about phase I research participation, during which physicians provided detailed information about the trial and discussed the patient’s and family’s willingness to participate. The goals of this study were to describe children’s and adolescents’ involvement by measuring physician-patient and parent-patient communication during the ICC; test whether involvement in ICC discussions increased with patient age; and examine whether involvement was associated with patients’ perceptions of difficulty understanding the information, pressure to participate in the phase I trial, and difficulty making the decision.