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