Lived Experience of Patients and Caregivers in Rare Genetic Neurological Gene Therapy Clinical Trials in Children

Area of Science:

• Pediatric Gene Therapy Research
• Rare Genetic Neurological Disorders
• Clinical Trial Design

Background:

• Pediatric gene therapy (GT) trials involve a dyad of child participants and caregivers, differing from adult studies.
• The "lived experience" of participants and caregivers significantly impacts decision-making and trial participation.
• Existing research often overlooks the social, emotional, physical, and logistical aspects affecting pediatric GT research.

Purpose of the Study:

• To highlight the essential role of "lived experience" in pediatric gene therapy trials.
• To emphasize the need to address the concerns and needs of child participants and their caregivers.
• To advocate for the integration of equity issues in the design and conduct of pediatric GT trials.

Main Methods:

• Qualitative analysis of stakeholder perspectives on pediatric gene therapy trials.
• Review of ethical and pragmatic considerations in rare disease research.
• Synthesis of expert insights on the burdens and benefits of trial participation.

Main Results:

• The decision-making process for pediatric GT trials is complex, involving child participants and caregivers.
• "Lived experience" encompasses emotional, social, ethical, and equity tradeoffs impacting participants and families.
• Addressing these lived experiences and equity issues is vital for successful pediatric GT research.

Conclusions:

• Child participants' and caregivers' lived experiences are paramount in pediatric gene therapy research.
• Integrating these perspectives and addressing equity concerns is essential for ethical and effective trial design.
• Prioritizing participant and caregiver needs enhances the quality and outcomes of rare genetic neurological disorder studies.