Supporting Families Considering Clinical Trial: A Digital Resource Featuring Parent and Provider Perspective and Guidance

Purpose/Objectives: 1. Describe the clinical trial consideration process from family point of view, so parents know the questions to ask and providers can support them
2. Place consideration in context of life with a child having a rare disease/disorder, to prepare both parties for what lies ahead
3. Illustrate variety of decisions and outcomes that families experience, to emphasize that there is no one right answer

Design/Methods: The project is a digital library (unit) of content presented in a variety of media (video, downloadable guide, podcast, blog) and organized around subjects of greatest potential concern and interest to families and providers based on the described experience of families who considered and/or enrolled in a clinical trial. Unit is available online, free, 24/7.

Results: The Evaluating the Clinical Trial Option unit was introduced in February 2020. In 12 months, there were 9,640 video views, the guides were viewed 226 times, and the Guided Pathway visited 356 times. (numbers would be updated for the poster). Staff has presented its content to numerous gatherings of providers, patient disease groups, and industry professionals to overwhelmingly positive feedback.

Professionals serving the rare disease community, especially patient advocates at biotech companies and patient disease groups, have reached out to CPN for resources from the unit to meet particular needs: e.g. content about informed consent helped a family understand the restrictions around posting to social media and content about inclusion criteria -- including a parent’s hope to ‘train’ towards meeting the criteria, as well as the potential for parents to feel they have failed if their child does not meet that criteria -- helped a parent navigate their stress and worry about COVID-induced disruptions to their child’s physical therapy prior to seeking participation in a trial. Companies have reported valuing the unit as a therapy- and industry-neutral, unbiased source of information for families. Companies link to it in their patient support portals and use the videos for in-service training. The patient disease group NTSAD, anticipating multiple clinical trials for its affected families, links to the unit on its website and sends families considering enrollment links to it in email communications. The American Society of Gene and Cell Therapy has recognized the unit for covering the emotional aspects of trial participation that are not covered in their resources and has embedded links to it in their patient- and provider-facing websites.

Conclusion/Discussion: The unit was designed as a resource to share with families and for their self-service, but grows in momentum and impact as it is used as a resource for educating colleagues -- including genetic counselors, medical geneticists, doctors working with rare disease patients - and peers striving to better understand the lived family experience.