Supporting Families Considering Participation in a Clinical Trial: Parent-Provider Perspectives.

Abstract

* Abbreviation: CPN — : Courageous Parents Network The emergence of efficacious human gene therapy for life-limiting genetic diseases such as spinal muscular atrophy type 11 has raised hopes among families of children with previously incurable genetic disorders. However, a number of concerns have surfaced regarding the gene therapy trial process. Two bereaved parents, both deeply engaged in their disease communities, collaborated with an experienced gene therapy researcher to create patient education materials to aid parents in critical decision-making. In this Family Partnerships article, we present a summary of the module, as well the reflections of each of the 3 contributors describing the impetus for the Courageous Parents Network (CPN) unit Evaluating the Clinical Trial Option, and their hopes for its impact, as captured in an interview conducted after completion of the educational module. The Evaluating the Clinical Trial Option unit (https://courageousparentsnetwork.org/topics/clinical-trial) offers an overview of the fundamental elements of clinical trial participation, written for a nonmedical audience to help families understand and evaluate their options. It is disease and therapy agnostic and does not advocate for any particular path or decision. Content areas include a glossary of terms; informed consent; clinical trial basics, such as efficacy; trial design, including the phases; participation criteria; and the emotional impact of decision-making. Its elements include a variety of media types: 85 short videos (average 2–4 minutes each, a total of ∼240 minutes); downloadable guides in English and Spanish; 3 podcasts; 4 blog posts written by parents; and a curated educational digital experience (“Guided Pathway”) that, through videos and text, walks parents … Address correspondence to Terence R. Flotte, MD, Medical School, University of Massachusetts and Dean’s Office, 55 Lake Ave North, Worcester, MA 01655. E-mail: terry.flotte{at}umassmed.edu