When it comes to the generation and use of real-world data (RWD), there are variable and evolving stakeholder needs. Put it in the context of a rare disease, where patient samples are decreasing as diseases splinter with increasing specificity in diagnosis, and the prospect of meeting these stakeholder needs becomes more daunting. Layer on special circumstances—like a required 15 years of post-approval follow-up data for cell and gene therapies (CGT)—and the cards seem to be stacked against you. One way companies can alleviate the burden of RWD generation is through patient registries.
Mike D’Ambrosio, VP, Head of Real World Evidence Solutions, Susan Goodlow, VP, RWE and Late Phase Operations and Ray Huml, Head of the Rare Disease Consortium at Syneos Health, discuss the importance of registries for clinical research—particularly for rare disease and CGT, the evolution of rare disease registries and opportunities for the future, and how we can unlock the value of RWD generated from rare disease registries, for all stakeholders.
The views expressed in this podcast belong solely to the speakers and do not represent those of their organization.
