Digital Health Technologies in Pediatric Trials.

BACKGROUND: Advances in the miniaturization of sensors and other technologies provide opportunities to collect physiological and/or functional data directly from patients participating in clinical trials. The use of such technologies in children is particularly promising. Objective, quantifiable measurements made by these technologies, often on a continuous or frequent basis, may provide more robust data than the episodic reports from caregivers that are used in traditional pediatric trials. METHODS: We reviewed the pros and cons of these technologies for use in a variety of pediatric diseases, including seizure and neuromuscular disorders, cardiorespiratory diseases, and metabolic disorders. RESULTS: Correlation between sensor measurements and patient observations or traditional clinical measurements varied depending on the disease being evaluated. There was a notable dearth of reports on the use of digital health technology in pediatric patients. Given the range of sensors and measurements that can be made by DHTs, selection of the design, metrics and types of sensors best suited to disease evaluation presents challenges for adoption of these technologies in clinical trials. CONCLUSION: Traditional measurements of drug effects are often deficient, particularly in the evaluation of infants and young children. The opportunity to make objective, frequent measurements may increase our power to detect and quantify responses to therapy in these populations. Further research and evaluation are needed to realize the full scientific potential of remote monitoring in pediatric clinical trials.

Digital Health Technology to Measure Drug Efficacy in Clinical Trials for Parkinson's Disease: A Regulatory Perspective.

Digital health technology (DHT), including wearable and environmental sensors, video cameras and other electronic tools, has provided new opportunities for the measurement of movement and functionality in Parkinson's disease. Compared to current standards for evaluation of the disease (MDS-UPDRS), DHT may offer new possibilities for more frequent objective measurements of the duration, severity and frequency of disease manifestations over time, that may provide more information than periodic clinic visits. However, DHT measurements are only scientifically and medically useful if they are accurate, reliable and clinically meaningful. Verification and validation, also known as analytical validation and clinical validation, of DHT performance is important to ensure the accuracy and precision of measurements, and the specificity of findings. Given the wide range of clinical manifestations associated with Parkinson's disease and the many tools and metrics to assess them, the challenge is to identify those that may represent a standard for use in clinical trials, and to confirm when digital measurements succeed or fall short of capturing meaningful benefits during drug development.