JMPB: Special Issue Call for Papers
Movement-related Digital Biomarkers, Endpoints & Clinical Outcome Assessments
Special issue editors: Pedro Saint-Maurice, Dana Wolff-Hughes, Kate Lyden
Scope
The US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have progressively evolved their guidance and recommendations for clinical drug developers to develop and incorporate clinical outcome measures that reflect aspects of health that are meaningful to patients. This paradigm shift has opened the door for clinical trialist to embrace pragmatic clinical trials that place less emphasis on traditional in-clinic assessments and leverage innovative digital health technologies (DHTs) to measure novel, more patient-centric outcomes.
Per definition of the FDA, “a DHT is a system that uses computing platforms, connectivity, software, and/or sensors, for health care and related uses.” DHTs include wearable sensors, smartphones and connected devices, and can obtain clinically relevant data directly from patients and provide a more comprehensive assessment of patient health. With advances in DHTs, such measures can be captured remotely, passively, and continuously to objectively assess movement-related behavior as patients go about their daily lives. These data can be used to develop movement-related digital biomarkers and endpoints such as physical activity, behavior, function and mobility, among others. The measurement of real-world physical activity and behavior is one of the most promising examples of how DHTs can provide a more detailed and valid representation of disease manifestation and health status.
Novel, movement-related digital biomarkers and endpoints can be used to complement existing assessments or be used when established methods are not appropriate or feasible. For example, many clinical drug trials spanning cardiorespiratory, neurodegenerative and musculoskeletal indications include at least one measure of physical function as a primary or secondary endpoint. To date, these endpoints have been derived using either self-report or in-clinic performance assessments, such as the 6-Minute Walk Test (6MWT) and Timed Up & Go (TUG) test. These assessments were designed to reflect or mimic movements needed to complete common activities of daily living (e.g. walking, sit-to-stand transitions, etc.). Wearable kinematic sensors combined with advanced analytics can now be used to assess if, and how, these same movements are being performed in a patient's own real-world environment. Further, several investigations have explored how patterns of real-world behavior reflect physical function and if these measures are sensitive to treatment benefits. While such measures provide a peak into a patient's lived experience, there is a lack of consensus on their true meaning and clinical relevancy across therapeutic areas. Thus, significant work remains to develop fit-for-purpose measures that accurately capture movement-related concepts that are clinically relevant.
The field of physical behavior measurement has embraced the need for digital biomarkers and endpoints and is actively exploring the full potential of sensor-based movement (actigraphy) and physiologic (photoplethysmogram or PPG) metrics to produce novel, sensitive and clinically valid measures. This supplement in JMPB is an opportunity to organize some of these efforts in one single volume of publications. The supplement will include examples of the development and validation of movement-related digital biomarkers and endpoints. We will welcome submissions that describe aspects of sensor-based digital measurements for use in clinical applications. We’re particularly interested in examinations of clinical validity of movement-related digital biomarkers and endpoints – i.e., evaluation of digital biomarkers for monitoring or predicting a clinical or functional state in individuals. For example, studies examining whether stepping cadence can predict disease progression in patients with Parkinson’s disease or detect treatment benefit. Studies that include patient-reported outcomes (PROs) are also welcome when combined with sensor-based digital measures in the context of multimodal approaches for clinical validation of digital biomarkers related to physical activity. Submissions can include original studies, systematic reviews, or protocols for clinical trials that include physical activity digital endpoints/biomarkers.
Important Dates
Deadline for manuscript submission: August 31, 2025
Expected publication: Approximately six weeks after acceptance
Submission Guidelines
The deadline for submissions for this special issue is August 31, 2025. Submissions should be prepared according to the author instructions available at the journal website and submitted via the journal submission page. Note that there is no cost for submission or publication for this special issue, although authors may purchase free or open access if they wish.
Reviewing Process
All papers will be anonymously peer reviewed by two experts in the field appointed by the Guest Editors of the issue in consultation with the Editor of the Journal.
Contact
Authors considering topics for this special issue or seeking feedback are highly encouraged to contact one of the guest editors:
- Pedro Saint-Maurice: pedro.saintmaurice@research.fchampalimaud.org
- Dana Wolff-Hughes: dana.wolff@nih.gov
- Kate Lyden: katelyden6@gmail.com
Key References
- Digital Health Technologies for Remote Data Acquisition in Clinical Investigations Guidance for Industry, Investigators, and Other Stakeholders. US Food and Drug Administration (FDA). December 2023. Guidance for Industry. Accessed on 10/31/2024
- Verification, analytical validation, and clinical validation (V3): the foundation of determining fit-for-purpose for Biometric Monitoring Technologies (BioMeTs) | npj Digital Medicine (nature.com)
- https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8490914/
Please click the following link for the PDF version of this call.
Last Updated: November 21, 2024