Real World Evidence and Rare Diseases, Part 3: The Value of Registries

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. 

Visit our Insights Hub for other content on RWE, rare diseases, and cell and gene therapy.

For more episodes on these topics, try:

Opportunities and Challenges in Cell and Gene Therapy

Navigating the Rare Disease Landscape

Real World Evidence and Rare Diseases, Part 1

Real World Evidence and Rare Diseases, Part 2 

If you want access to more future-focused, actionable insights to help biopharmaceutical companies better execute and succeed in a constantly evolving environment, visit the Syneos Health Insights Hub. The perspectives you’ll find there are driven by dynamic research and crafted by subject matter experts focused on real answers to help guide decision-making and investment. You can find it all at insightshub.health. 

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The views expressed in this podcast belong solely to the speakers and do not represent those of their organization.

If you want access to more future-focused, actionable insights to help biopharmaceutical companies better execute and succeed in a constantly evolving environment, visit the Syneos Health Insights Hub. The perspectives you’ll find there are driven by dynamic research and crafted by subject matter experts focused on real answers to help guide decision-making and investment. You can find it all at https://www.syneoshealth.com/insights-hub.

Like what you’re hearing? Be sure to rate and review us!

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