M.D., Ph.D., VP, Global Clinical Development, Otsuka Pharma. Dev. & Com., Inc.
Dr. Czerwiec guides a multidisciplinary team developing innovative patient-focused pharmaceuticals, devices and disease management tools for disorders like autosomal dominant polycystic kidney disease (ADPKD). ADPKD is an orphan genetic disease which had no effective therapy. PKD1 gene mutations typically lead to kidney failure by age 55. PKD2 gene mutation effects appear 1-2 decades later, but might not lead to dialysis. For 13 years, Otsuka has engaged with patients, experts and regulators to develop and deliver clinical data, tools and drugs to support patients and their physicians in making clinical decisions. We aim to deliver integrated health-care solutions optimizing benefits while limiting risks to individual patients. After obtaining his MD/PhD at University of Miami, Dr. Czerwiec trained as a clinical investigator while an NIH-NICHD endocrine fellow before a 19-year career in R&D at Merck then Otsuka.
Overcoming Data Challenges in Rare Disease Trials
Developing safe and effective disease-modifying therapies for rare disorders is challenging. Capturing and processing more data can be cost-effective. For trial populations medical history and biomarker data can enable endpoint enrichment and correct for imperfect randomization. For individuals, they support medical decision making for personalized benefit/risk optimization.
Session Abstract – PMWC 2017 Silicon Valley
Session Synopsis: Rare Diseases are genetic, severe and life threatening; by definition they impact 200,000 patients or less. In 2015, 47% of the medications approved by the FDA were for rare diseases. We will explore how this trend has changed drug development: increasing collaboration, advancing personalized medications and personalized disease management tools.