Validity of patient-reported data collected through mobile application in a first paediatric at-home study




Poster session 1 Wednesday: Evidence production and synthesis


Wednesday 13 September 2017 - 12:30 to 14:00


All authors in correct order:

von Niederhäusern B1, Saccilotto R1, Schädelin S2, Summerer M3, Ziesenitz V3, Hammann A4, Bielicki J3, Pfister M5, Pauli-Magnus C6
1 Department of Clinical Research, University and University Hospital Basel, Switzerland
2 Clinical Trial Unit, Department of Clinical Research, University and University Hospital Basel, Switzerland
3 Department of Pediatric Infectiology, University Children’s Hospital Basel, Switzerland
4 Clinical Trial Unit, Department of Clinical Research, University Hospital Basel, Switzerland
5 Pediatric Pharmacology and Pharmacometrics, University of Basel Children’s Hospital, Switzerland
6 Department of Clinical Research, University Hospital Basel, Switzerland
Presenting author and contact person

Presenting author:

Belinda Von Niederhäusern

Contact person:

Abstract text
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