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 y...