稀少神経疾患治験に対する，DCTアプローチ

抄録

Rare neurological disorders present significant challenges for clinical trials due to the limited patient population and logistical difficulties in data collection. Decentralized Clinical Trials (DCTs) offer an innovative approach by utilizing remote technologies such as telemedicine, wearable devices, and mobile applications to overcome geographical barriers and enhance patient recruitment. This study explores the application of DCT methods to rare neurological disorder trials and discusses the integration of Real World Data (RWD) to optimize trial design.

The implementation of DCT technologies, including remote monitoring and ePROs, enables patients to participate in trials from their homes, reducing the burden of travel while collecting reliable real–time data. A case study on a rare cancer trial utilizing DCTs showed a 40% improvement in enrollment speed and high patient satisfaction rates. Furthermore, this paper presents a mock protocol combining DCT technologies and traditional methods for a hybrid approach in a heart failure drug trial, highlighting the role of eConsent, wearable sensors, and video consultations.

By leveraging RWD, trial designs can be refined to identify alternative endpoints, predict success probabilities, and optimize patient selection criteria. Case studies are provided to illustrate the effectiveness of RWD in improving trial efficiency and ensuring safety.

This study underscores the transformative potential of DCTs and RWD in addressing the unique challenges of clinical trials for rare neurological disorders, paving the way for more efficient and patient–centered research methodologies.