Biomarkers Consortium - Workshop: Remote Digital Monitoring for Medical Product Development
Exploring opportunities to enhance clinical trial designs by integrating remote digital monitoring technologies
The Foundation for the National Institutes of Health (FNIH) Biomarkers Consortium hosted a public meeting titled Remote Digital Monitoring for Medical Product Development Workshop on February 18 and 19, 2020.
The use of remote digital monitoring technologies (smartphone apps, wearables, ingestibles, implantables and mobile-device-based tools) has sparked the interest of consumers, providers and researchers as a new way to improve therapeutic research and development. This workshop provided a venue to address challenges and opportunities surrounding the use of remote sensing technologies to improve the probability of success in drug development and therapeutic clinical trials. These technologies could also make trials more efficient and less burdensome to participants while providing a more meaningful and complete understanding of patients’ conditions and responses. The workshop relied on a series of case studies, presentations and open panel sessions (available via the links below) to address key elements in evaluating the maturity of digitally measured biomarkers and defining future directions to enable a broader adoption of digital tools in human research.
Workshop participants identified a framework to guide the process of developing remote monitoring measures and prioritized a list of high-impact endpoints or measures that can be recorded through mobile sensing. The workshop planning team incorporated the insights from the meeting into multiple manuscripts and core recommendations to enable a framework for the analytical and clinical validation needed for drug developers and to develop and improve operational/regulatory guidance.
Presentations from the Workshop
- Challenges for the Implementation of Digital Monitoring Technologies in Drug Development - Jennifer Goldsack (DiME) | Watch the video
- Regulatory Guidance and Evidentiary Criteria for Drug Development Tools - Chris Leptak (FDA/CDER) | Watch the video
- Review of the General Framework and Case Study Set Up: Issues and Considerations - Joseph Menetski (FNIH) and John Wagner (Foresite Capital) | Watch the video
- Regulatory Guidance and Evidentiary Principles for Digital Health Technology Development - Bakul Patel (FDA/CDRH) | Watch the video
- NIH Pipeline in Digital Technologies - Jill Heemskerk (NIH/NIBIB) | Watch the video
- Case Study 1: Mobile Parkinson Observatory for Worldwide, Evidence-based Research (mPower) and Parkinson's Disease - Diane Stephenson (Critical Path Institute), Dan Karlin (HealthMode), Ahi Pratap (SageBionetworks), Ninad Amondikar (MJFF), Xeumei Cai (Pfizer) | Watch the video
- Case Study 2: Cardiac Monitoring in Phase 1 Clinical Trials - Elena Izmaliova (Koneska Health), John Wagner (Foresite Capital), Vadim Zipunnikov (Johns Hopkins University) | Watch the video
- Case Study 3: Continuous Glucose Monitoring & Remote Digital Monitoring (VERKKO Study) - Roberto Calle (Pfizer), Nadir Ammour (Sanofi) | Watch the video
- Case Study 4: Strive Velocity 95th Centile 2° Endpoint in Duchenne's Muscular Dystrophy - Pat Furlong (PPMD), Laurent Servais (University of Oxford), Francesca Cerreta (EMA) | Watch video
- Case Study 5: Remote Assessment of Disease and Relapse in Major Depressive Disorder (RADAR-MDD) - Matthew Hotopf (King's College), Vaibhav Narayan, Linda Brady (NIH/NIMH) | Watch video
- Convene diverse stakeholders in the field of digital health technology to establish a shared vocabulary for regulatory purposes.
- Present and examine real-world case studies of remote monitoring in clinical trials to highlight concepts that may require development of a new vocabulary.
- Identify areas of high unmet medical need that could be addressed using digital system technologies.
- Align stakeholders to apply an evidence-based framework for the use of digital health technologies in therapeutic research and development.
Results & Accomplishments
- Remote Cardiac Safety Monitoring through the Lens of the FDA Biomarker Qualification Evidentiary Criteria Framework: A Case Study Analysis. Izmailova EA, Wood WA, Liu Q, et al. Digital Biomarkers (2021) 5, 103-113. https://doi.org/10.1159/000515110
- Remote digital monitoring in clinical trials in the time of COVID-19. Goldsack JC, Izmailova ES, Menetski JP, Hoffmann SC, Groenen PM, Wagner JA. Nature Reviews Drug Discovery 19, 378-379 (2020). doi: https://www.nature.com/articles/d41573-020-00094-0
- Remote Digital Monitoring for Medical Product Development. Izmailova EA, Wagner JA, Ammour N, et al. Clin Transl Sci (2020) 0, 1–8. doi: https://doi.org/10.1111/cts.12851
- Draft Guidance Document - Biomarker Qualification: Evidentiary Framework
- Draft Guidance Document - Qualification Process for Drug Development Tools
- What evidence do we need for biomarker qualification? C. Leptak, J. P. Menetski, J. A. Wagner, J. Aubrecht, L. Brady, M. Brumfield, W. W. Chin, S. Hoffmann, G. Kelloff, G. Lavezzari, R. Ranganathan, J.-M. Sauer, F. D. Sistare, T. Zabka, D. Wholley, Sci Transl Med. 2017 Nov 22;9(417). pii: eaal4599. doi: 10.1126/scitranslmed.aal4599.
- Traditional and Digital Biomarkers: Two Worlds Apart? L. Babrak, J. Menetski, M. Rebhan, G. Nisato, M. Zinggeler, N. Brasier, K. Baerenfaller, T. Brenzikofer, L. Baltzer, C. Vogler, L. Gschwind, C. Schneider, F. Streiff, P. Groenen, E. Miho, Digit Biomark 2019;3:92–102, DOI: 10.1159/000502000
- Digital Medicine: A Primer on Measurement. A. Coravos, J. Goldsack, D.R. Karlin, C. Nebeker, E. Perakslis, N. Zimmerman, M.K. Erb, Digit Biomark 2019;3:31–71,DOI: 10.1159/000500413
- Workshop Overview and Pre-Read Resources
- CTTI Recommendations: Developing Novel Endpoints Generated by Mobile Technology for Use in Clinical Trials
- BEST Resource Taxonomy