Novel digital endpoint, if used as the prespecified ranked endpoint in the registrational studies, may enhance labels and enable market access. Many pharmaceuticals are developing novel digital measures of health and disease to accelerate drug development and support product differentiation.

The pre-competitive development of digital measures in neurodegenerative diseases like ALS, holds promise for advancing diagnostics and monitoring. This presentation explores the evolving landscape of digital tools, including wearables and mobile applications, designed to capture nuanced disease progression markers. By fostering collaboration among stakeholders, this initiative aims to accelerate the validation and implementation of digital biomarkers and clinical endpoints, paving the way for improved clinical trials and personalized patient care in ALS management.

The presentation will provide an overview of our digital health solution diligence process and the construction of an evaluative framework for novel endpoint development in oncology—focusing on a case example surrounding actigraphy sensor deployment in prostate cancer. We will also address the many hurdles associated with clinical trial implementation and the importance of a broader tech development vision within the context of an individual drug program.

There has been a growing interest in novel respiratory digital measurements, specifically cough. This presentation will discuss the relevance of cough as a novel respiratory endpoint, introduce novel technologies including wearables and AI solutions that measure cough, and discuss challenges and opportunities for the future.

Interactions between participants, clinical research staff and pharma companies are crucial to the success of drug development, as they affect research quality and the recruitment and retention of participants. Research staff and patients have certain expectations about conducting trials, and it’s important for pharma firms to assess and manage these. We used a novel framework to assess these expectations and the current state of the industry globally.

Physical activity (PA) is associated with improved mobility, pain, and function in people with Ankylosing Spondylitis (AS); however, the impact of pharmacologic interventions on PA is rarely measured in clinical research. Wearable technology provides an objective method to passively collect PA data. Here, we describe the effect of upadacitinib (an oral JAK inhibitor) on PA in 400 AS patients over 14 weeks in the Phase 3 SELECT AXIS-2 study.

This presentation will share a case study of using digital biomarkers in a dermatology study.

An automated, scalable platform integrating, transforming, and delivering near real-time and concurrent digital health data from diverse sets of wearables, mobile applications, and AI/ML models.

This presentation will share case studies of leveraging digital biomarkers for internal decisions as well as for modernizing endpoints.

Cancer cachexia is characterized by unintentional weight loss, due to a loss in muscle mass that physically manifests as fatigue, functional impairment, weakness, and increased mortality. We will share evidence on the importance of, as well as how digital measures of physical activity and gait, derived from wearable sensors, can be used to quantitatively and passively assess changes in physical function and support clinical studies in a home environment. 

Precision oncology trials rely on biomarkers to identify and enroll likely responders to experimental therapies, thus increasing the likelihood of success and accelerating timelines by enriching the trial population. Advances in machine learning (ML) are enabling development of novel biomarkers that can be widely deployed and have superior performance compared to biomarkers assessed by standard methods (e.g. molecular assays). This presentation will describe a framework and case studies on ML-based biomarker deployment in global clinical trials, focusing on key requirements for translating research-grade algorithms to deployable, regulatory-grade ML products.

The Oncology Research Biomarker Innovation Tower (ORBIT) establishes a cohesive and unified multimodal biomarker data hub across clinical studies as part of the Med.ai ecosystem. ORBIT enables accurate and real-time analysis of site, patient, sample, biomarker, and diagnostic data, providing actionable insights to study teams while transforming decision-making and streamlining operations.

What motivates pharma to go beyond the pill?  Emerging trends in Digital Biomarkers and Software-as-Medical-Device (SaMD) enabled the development of companion apps for commercial drugs. This approach allows pharma companies to remotely track medication side effects and monitor disease symptoms based on real-time digital measures otherwise not accessible using traditional RWE studies. What makes digital drug solutions successful? How are they regulated? What is the role of AI in empowering patients to make healthier choices?

DCTs leverage “virtual” tools, such as telemedicine, sensory-based technologies, wearable medical devices, home visits, patient-driven virtual health care interfaces, and direct delivery of study drugs and materials to patients’ homes. This talk will discuss the post-COVID strategies for connected devices implementation in hybrid trials.

This talk will focus on the use of digital health technologies(DHTs) to advance medical product development—from assessment to interventions in real-world settings. I will share learnings from clinical research studies to help inform the development of robust digital endpoints and interventions focusing on improving behavioral outcomes.

This presentation will share the NIH Framework for digital technologies' validation in clinical trials.