Mitigating the Devastation: A Parent’s Perspective on Her Child’s Exclusion From a Clinical Trial for a Rare Disease Drug Therapy

In the context of rare disease research, extensive academic literature exists on topics such as natural history and case study data, innovations in trial design, and potential therapies. However, noticeably absent from the scholarly literature is discussion about the psychological impacts on parents when their children are excluded from pediatric trials despite the parents’ desire for them to participate. For one parent, the exclusion of her son from a pediatric clinical trial led to serious psychological and emotional consequences. The purpose of the article is to provide insights into how trial exclusion can be perceived as a personal rejection and how possible consequences can be mitigated.