On March 16 Quinten Health CEO Billy Amzal, PhD, MBA, presented AI-powered Real-world Simulations for Faster and Value-based Rare Disease Drug Development.
This presentation showed how Quinten Health can use a data-integrative, value-based and disease-centric analytic approach to support RDCA-DAP’s mission to accelerate and de-risk trials, while maximizing the real-world value of rare disease products in development.
This approach relies on real-world disease modelling designed to simulate patient journeys in terms of both disease progression and care pathways in the real-life practice. A mix of real-world data science, Bayesian modelling, interpretable machine learning, advanced statistics and predictive analytics can be deployed to inform the various components of RDCA-DAP. For example, real-world simulations can be used to demonstrate effectiveness from efficacy, or long-term outcomes from short-term ones, in the context of trial design. Both the general approach and proven examples in rare diseases will be presented to demonstrate the current value of such an approach, and to illustrate how Quinten tools can support and inform RDCA-DAP’s mission.
RDCA-DAP Team: Jeff Barrett, PhD, FCP, Senior Vice President, RDCA-DAP Lead; Alexandre Bétourné, PhD, PharmD, Scientific Director, RDCA-DAP; Megan Cala Pane, PhD, Quantitative Scientist II, Quantitative Medicine
This event is part of the RDCA-DAP webinar series, for additional webinars visit c-path.org/rdca-dap/resources
