Parents' Perspectives on Access to Pediatric Rare Disease Cross-border Clinical Trials in Europe: Experiences and Preferences Survey

Introduction Access to cross-border clinical trials may represent the sole therapeutic option for children affected by rare diseases for which no approved treatment exists. Many children are excluded from participation in trials due to language restrictions. There are insufficient comprehensive analyses of the experiences and preferences of parents across Europe concerning participation and exclusion of their child in international clinical trials, particularly regarding language support during enrollment in cross-border clinical research studies. Methods An anonymous online survey was designed and translated into 22 official European languages to collect data from parents of children living with a disease across Europe. It was structured into five sections: (1) sociodemographic information; (2) experience participating in a clinical trial; (3) experience in cases where the patient was unable to take part in a study abroad; (4) experience participating in a clinical trial abroad; and (5) preferences regarding decentralized trial options. Results 1,436 responses were analyzed from parents across 34 European countries. Key findings: 55.7% of the parents reported being able to communicate in English. 10.7% had previous clinical trial experience, of whom 30.1% traveled abroad for their child to participate. Among those reporting being excluded from cross-border trials, 34.7% cited language barriers or country of residence as the reason. Most families expressed strong willingness to accept decentralized trial options, regardless of where the study may be conducted. Conclusions Accommodating language translation to permit participation in a clinical trial abroad is feasible. While a significant percentage of caregivers of pediatric patients in Europe were able to communicate in English, approximately one third of those excluded from clinical trials attributed the reason to language barriers or country of residence. When translation was required, the most commonly offered solution was the use of professional interpreters, a accommodation that could enable broader patient participation in essential research.