Establishing a robust drug development strategy in Orphan Diseases and Precision Medicine during translational and early clinical development is key to expediting time-to-market. BBCR and its consultants are dedicated to advancing innovative and repurposed treatments for orphan diseases—an area where impact is high, but the path is complex. With limited patient populations and often little […]
In the fields of orphan diseases and precision medicine, establishing a robust biomarker strategy during translational and early clinical development is key to expediting time-to-market. A well-defined biomarker plan not only streamlines regulatory approval but also aligns with FDA expectations for safety and efficacy. Discover how our expertise in biomarkers and surrogate endpoints can help […]
Rare Diseases are conditions that affect less than 200,000 people in the U.S. However, over 7,000 Rare Diseases affect more than 400,000,000 people worldwide, including ~25 million in the U.S. and ~30 million in Europe (1). In recent years, the number of orphan drugs approved has increased significantly. However, developing and marketing orphan drugs remains […]
The primary difference between a biomarker and a surrogate endpoint is that a biomarker is a “candidate” surrogate marker. In contrast, a surrogate marker is a test used and taken to measure the effects of a specific treatment. In orphan diseases and precision medicine, developing a biomarker plan during translation into early clinical development moves […]
Biologic treatments show promise in providing clinical solutions to a variety of diseases including rare cancers and precision medicine. Related Services Include: Indications analysis and prioritization Strategic drug assessment Clinical study design and protocol Biomarker strategy Early Clinical Development FDA meeting and submission Pre-ND integrated development plan CRO and project management Study remediation and rescue