Cross-border access to clinical trials: participation of pediatric patients and language inclusion.

backgroundCross-border access to clinical trials in Europe lacks specific regulation. Language diversity in Europe, with 24 official languages, is a key factor that must be considered when including international pediatric patients in multi-country studies. Providing translation enables patients to participate in clinical trials across borders. Specific consideration requires pediatric rare disease clinical trials for conditions with no approved treatment, where only a few countries participate, making cross-border access even more critical.

methodsWe analyzed retrospective data (2011-2024) of the studies incorporating international patients at SJD Barcelona Children's Hospital. The main objective of this research was to assess the feasibility of cross-border access to pediatric clinical trials and identify lessons learnt that could help prevent the future exclusion of patients due to their limited proficiency in the official language of the country where the trial site is located.

resultsTwenty-one clinical studies have been analyzed. One hundred eighty-one patients from both European and non-European countries (N = 44) were screened, 37.02% of the patients were from Europe, versus 62.98% from non-European countries. In 52.38% of the studies, the translation services were provided by the site; in 14.28% by the sponsor. In 33.33% of the studies, no translation was necessary because the site staff could communicate with the families in Spanish or English. 57.14% of the studies included one or more PROMs or QoL scales, requiring, in some cases, the use of validated translations in the mother tongue of the patient.

conclusionParticipation in pediatric cross-border clinical trials is feasible. The decision to include international patients should prioritize potential medical benefits rather than using the patient's mother tongue or home country as eligibility criteria. Language barriers can be addressed by providing the necessary resources to ensure the scientific reliability of data collected, as well as to enhance the patient experience during participation in a trial abroad. IMPACT: The SJD Barcelona Children's Hospital Clinical Trials Unit operates under a one-stop-shop model, managing clinical studies with a special focus on rare diseases, centralizing all the needs of international patients (translations, accommodation, visas, etc.). The inclusion of international patients in pediatric clinical studies is feasible. Pediatric rare diseases require a small sample of patients, highly specialized sites, and flexibility to accommodate the necessary translations for patients. The final decision regarding the inclusion of international patients lies with the sponsor. Flexibility in including international patients directly impacts the study plan and execution, preventing time deviations.