Table 3 Engaging with patient communities in early-phase pediatric gene therapy (GT) trials.
From: Ethical challenges for a new generation of early-phase pediatric gene therapy trials
Principle | Implementation | Illustrative example(s) |
Clearly defined engagement goals and plan | Determine appropriate degree of engagement at each trial stage (e.g., design, conduct, dissemination) [57, 61,62,63,64] | • Determine degree of engagement (ranging from question-specific consultation to full partnership [71]) • Clearly communicate desired patient community input at each trial stage, or collaboratively determine types of useful input at each stage |
Inclusivity | Aim for representativeness and diversity of engaged patients/families [62,63,64] | • Engage patients/families with a range of perspectives (including patients/families not in an organized group) [72] • Use purposive rather than convenience sampling to increase representativeness [72] • Consider strategies for eliciting children’s perspectives in pediatric trials [67] |
• Make reasonable time requests • Provide accommodations (e.g., for disability or different languages) • Compensate engaged patients/families when possible | ||
Co-learning | Provide necessary training for engaged patients/families and researchers [57, 61,62,63,64] | • Educate patients/families on language and process of research to enable them to participate effectively • Educate researchers on patient engagement principles and practices |
Clear communication and transparency | • Be consistent in communication with patient community (possibly designate one contact person) • Use understandable language • Establish a consistent and accessible platform (e.g., website, social media) for communication | |
• Identify and manage conflicts of interest (e.g., financial stake in trial, enrolled child in trial) among researchers and engaged patient community members • Clearly communicate key trial decisions and rationales (if this does not impinge on other obligations, such as protecting participants’ privacy or competitive interests) | ||
Constructive interaction process and style | Cultivate mutual respect and participatory culture [61,62,63,64] | • Respect patient community members’ experiential expertise • Listen, respond to, and (if appropriate) act on patient community input • Acknowledge patient community’s contributions to research (e.g., in published reports) |
Address conflicts promptly and explicitly [61] | • Establish process for soliciting and responding to patient community concerns • Revise decisions if necessary | |
Assessment and impact | • Solicit feedback (e.g., researcher and patient/family satisfaction) • Revise engagement process as necessary |
