Using electronic Patient-Reported Outcomes (ePROs) in early-phase trials is important in order to provide evidence of therapeutic responses and tolerability, increase the evidence base, and inform methodology development.
Read the results of our work with the University of Birmingham to develop and test an ePRO using our Atom5™ clinical trial platform for an Advanced Therapy Medicinal Product (ATMP) basket trial for patients with inflammatory diseases.
Background
Electronic patient-reported outcome (ePRO) systems are increasingly used in clinical trials to provide evidence of efficacy and tolerability of treatment from the patient perspective. The aim of this study is twofold: (1) to describe how we developed an electronic platform for patients to report their symptoms, and (2) to develop and undertake usability testing of an ePRO solution for use in a study of cell therapy seeking to provide early evidence of efficacy and tolerability of treatment and test the feasibility of the system for use in later phase studies.
Methods
An ePRO system was designed to be used in a single arm, multi-centre, phase II basket trial investigating the safety and activity of the use of ORBCEL-C™ in the treatment of patients with inflammatory conditions. ORBCELC™ is an enriched Mesenchymal Stromal Cells product isolated from human umbilical cord tissue using CD362+cell selection. Usability testing sessions were conducted using cognitive interviews and the ‘Think Aloud’ method with patient advisory group members and Research Nurses to assess the usability of the system.
Results
Nine patient partners and seven research nurses took part in one usability testing session. Measures
of fatigue and health-related quality of life, the PRO-CTCAE™ and FACT-GP5 global tolerability questions were included in the ePRO system. Alert notifications to the clinical team were triggered by PRO-CTCAE™ and FACT-GP5 scores. Patient participants liked the simplicity and responsiveness of the patient-facing app. Two patients were unable to complete the testing session, due to technical issues. Research Nurses suggested minor modifications to improve functionality and the layout of the clinician dashboard and the training materials.
Conclusion
By testing the efectiveness, efciency, and satisfaction of our novel ePRO system (PROmics), we learnt that most people with an infammatory condition found it easy to report their symptoms using an app on their own device. Their experiences using the PROmics ePRO system within a trial environment will be further explored in our upcoming feasibility testing. Research nurses were also positive and found the clinical dashboard easy-to-use. Using ePROs in early phase trials is important in order to provide evidence of therapeutic responses and tolerability, increase the evidence based, and inform methodology development.