This month we are spotlighting The Lived Experience of Pediatric Gene Therapy – A Scoping Review by Kimberly et al. In this Human Gene Therapy article the authors assess empirical research examining the lived experience of both patients and caregivers participating in pediatric gene therapy trials. Their scoping review underscores a serious gap in the literature leading to an examination of narrative accounts from trial participants and their families, and areas where further research in necessary. The authors note that:
[p. 4]
“…When making a decision about whether to enroll a child in a GT clinical trial, caregivers must weigh factors such as: the inability for most treatments to be repeated or re-dosed; uncertainty around the durability, efficacy, and safety of an experimental treatment; risk of a child’s disease progressing if they wait for an approved treatment or more safety data; and efficacy of existing approved alternative treatments… The confluence of these factors within a high-stakes decision-making framework creates a unique ethical context for pediatric rare disease patients and their parents and caregivers. The progressive nature of many of these diseases also adds a time constraint to participation…
[p. 16]
The field of pediatric GT research… has an important and timely opportunity to better understand how patients and their parents/caregivers choose whether or not to consider GT, and their lived experience of participating in clinical trials. Pediatric patients’ own perspectives are almost completely absent from the literature, marking an important area for future research efforts. Such insights from further research will inform more patient- and family-centered clinical trial design and can help to ensure that clinical trial design and implementation reflect patients’ and families’ values, their priorities, and their goals for care. This scoping review lays a foundation for future research in this space.”
The Lived Experience of Pediatric Gene Therapy – A Scoping Review
Laura Kimberly, Cara Hunt, Katherine Beaverson, Emma James, Alison Bateman-House, Richard McGowan, Jennifer DeSante-Bertkau
Human Gene Therapy, 15 November 2023
Abstract
Little is known about patients’ and families’ lived experience of participating in pediatric gene therapy (GT) clinical trials. Currently, pediatric GT research targets a broad range of indications––including rare and ultra-rare diseases––which vary in severity and in the availability of alternative therapies. Pediatric GT differs meaningfully from adult GT because the decision to participate involves a dyad of both the child and parent or caregiver/s. It is critical to understand patients’ and caregivers’ perceptions and experiences of the social, emotional, physical, and logistical burdens or benefits of participating in such trials, and how they weigh and prioritize these factors when deciding whether to participate. We conducted a scoping review of the current literature in this subject area with objectives to a) provide an overview of existing literature, b) identify gaps and areas for further research, and c) better understand the lived impact of pediatric GT research on patients and their parents/caregivers. , Four themes emerged, including 1) weighing risks and benefits 2) timing of GT trial participation 3) value of clear communication, and 4) potential impact on quality of life. Notably, our sample surfaced articles about how patients/parents/caregivers were thinking about GT – their understanding of its safety, efficacy, and risks – rather than accounts of their experiences, which was our initial intention. Nevertheless, our findings offer useful insights to improve the informed consent process and promote a more patient- and family-centered approach. Moreover, our findings can contribute to patient advocacy organizations’ efforts to develop educational materials tailored to patients’ and families’ expressed informational needs and perspectives, and can inform more patient- and family-centered policies from GT clinical trial sponsors.
Center for Informed Consent Integrity 