Rare disease drug development represents one of the fastest-growing areas for biopharmaceutical R&D investment. For the first time in decades, there are potential cures for many of the rare diseases that involve a single gene mutation. Yet, despite this significant development, numerous inherent challenges pose significant risks for sponsors when navigating the rare disease space – most significantly, the ability to identify patients for research and treatment options. Biomarkers may be the answer to indicate potential and proven responses to therapeutic interventions.
In the next 20 years, personalized medicine will be at the forefront of clinical applications and the individuals’ specific biomarker panels will guide the customization of individualized treatment. In this white paper, we discuss the use of digital biomarkers and their potential impact on rare disease drug development in the context of decentralized clinical trials.