Advancing Neurodegenerative Disease Trials with Digital Health Technologies: A Regulatory Aligned Approach

How Digital Health Technologies Enable Patient-Centric Trials

This white paper explores the paradigm shift in clinical research for neurodegenerative diseases, such as ALS, Huntington’s, and Parkinson’s Disease, driven by Digital Health Technologies (DHTs) and Decentralized Clinical Trials (DCTs). Given the progressive nature and symptom variability of these conditions, traditional site-based trials often impose substantial burdens on participants and fail to capture real-world disease trajectories. DHTs, including wearables, mobile applications, telemedicine, and AI analytics, now empower adaptive and patient-centric trials by facilitating continuous, remote monitoring and richer data collection.

The emergence of supportive regulatory frameworks, notably ICH E6(R3) and the Accelerating Clinical Trials (ACT EU) initiative, alongside practical guidance from EMA’s EU DCT project and FDA’s “Conducting Clinical Trials with Decentralized Elements”, is enabling the robust integration of DHTs within both scientific and regulatory contexts.

This white paper reviews the current advancements and benefits enabling digital health technologies, as well as the challenges to adoption, clinical applications in ALS, Huntington’s, and Parkinson’s Disease, and future directions for leveraging digital innovation to enhance clinical trial outcomes and improve patient experiences in neurodegenerative disease research.