Leonard Sacks1, Elizabeth Kunkoski2, Marianne Noone3. 1. Office of Medical Policy, Center for Drug Evaluation and Research, Food and Drug Administration, Silver Spring, MD, USA. Leonard.Sacks@fda.hhs.gov. 2. Office of Medical Policy, Center for Drug Evaluation and Research, Food and Drug Administration, Silver Spring, MD, USA. 3. Office of Pediatric Therapeutics, Office of Clinical Policy and Programs, Office of the Commissioner, Food and Drug Administration, Silver Spring, MD, USA.
Abstract
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.
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.