Article type
Abstract
Background: Clinical studies in children are challenging, yet they are urgently needed to advance our knowledge on optimal dosing and action of medicines. Remote 'at-home' trials are suggested as an option for a more patient-centred future of trials.
Objectives: This pilot aimed to study the technical and practical feasibility of collecting valid data in paediatric at-home trials.
Methods: This was a single-centre, prospective pilot study including 22 children, 2-5 years of age, undergoing elective tonsillectomy at the University Children’s Hospital Basel (model population). Using a specifically developed mobile application, time-stamped data were collected by caregivers on 2-4 inpatient study days with the support of study nurses and on 3 consequent study days at home. Biological samples (saliva) were collected throughout the study. The primary endpoint was the proportion of complete and correct caregiver-collected clinical data and saliva samples in the at-home setting. Secondary endpoints included practicability of this type of study for participants, the proportion of caregivers consenting to take part in the study (including reasons associated with non-consent), and the cost-effectiveness of performing such a study.
Results: At the Summit, we will present the results on the completeness and correctness of data collected by caregivers through a mobile application, and the practicability of mobile-data collection for both caregivers and study personnel. In particular, we will report on how reliable (i.e. match between automatically recorded time point and caregiver-reported time point of data entry) data collection was performed, and the factors associated with valid or invalid data collection (i.e. specific time points, inter-caregiver variation).
Conclusion: Although remote trials are increasingly performed, the aspect of data validity, and therefore study quality, is often neglected. This is a first pilot investigating the correctness of data collected by patients remotely. If proven successful, this approach holds considerable promise in strengthening the evidence-base on treatment options
Objectives: This pilot aimed to study the technical and practical feasibility of collecting valid data in paediatric at-home trials.
Methods: This was a single-centre, prospective pilot study including 22 children, 2-5 years of age, undergoing elective tonsillectomy at the University Children’s Hospital Basel (model population). Using a specifically developed mobile application, time-stamped data were collected by caregivers on 2-4 inpatient study days with the support of study nurses and on 3 consequent study days at home. Biological samples (saliva) were collected throughout the study. The primary endpoint was the proportion of complete and correct caregiver-collected clinical data and saliva samples in the at-home setting. Secondary endpoints included practicability of this type of study for participants, the proportion of caregivers consenting to take part in the study (including reasons associated with non-consent), and the cost-effectiveness of performing such a study.
Results: At the Summit, we will present the results on the completeness and correctness of data collected by caregivers through a mobile application, and the practicability of mobile-data collection for both caregivers and study personnel. In particular, we will report on how reliable (i.e. match between automatically recorded time point and caregiver-reported time point of data entry) data collection was performed, and the factors associated with valid or invalid data collection (i.e. specific time points, inter-caregiver variation).
Conclusion: Although remote trials are increasingly performed, the aspect of data validity, and therefore study quality, is often neglected. This is a first pilot investigating the correctness of data collected by patients remotely. If proven successful, this approach holds considerable promise in strengthening the evidence-base on treatment options