Videos & Webinars

Analyzing Real-World Data and Investigating Genotype-Phenotype Data to Support Precision Medicine Drug Development in Rare Diseases Webinar

Real-world data can help us understand rare disease patient populations at a fundamental level, but how can we leverage this information and incorporate multiple data sources to deepen our understanding of the natural history of a disease and better identify tangible targets for the development of precision medicines? To support virtual mapping of disease progression and better understand comorbidities, longitudinal real-world data sets can help identify potential genotype-phenotype relationships and may provide supplementary information to the standard variant classification process of determining mutational pathogenicity. Coupled with physician diagnostic coding from the patient care setting, advanced analyses of these disparate data could improve how our industry diagnoses patients, designs and conducts trials and enables more efficient development of targeted therapies. Watch this webinar for an in-depth discussion on predictive modeling with real-world data to better understand the patient journey in rare disease. The presenters share their applications of real-world data against co-existing conditions documented by the ICD codes across longitudinal data subsets for individuals with specific mutations of interest. Learn how their exploratory analyses can help identify symptoms and biochemical parameters that may help patients and physicians reach earlier, more accurate diagnoses.

Filed In

Commercialization
Pharma
Phase IIb
Rare Disease
Stage (Early, PhI-IIa)
Stage (Late, PhIIb-III)
Stage (Post-Market, PhIV)
Therapies - Cell
Videos & Webinars