How rare is a rare disease? On their own they can be quite scarce, but did you know that one in ten people in the U.S. is afflicted with a rare disease? That’s over 30 million people. And that’s a big problem. But how do you study treatments when it’s so difficult to find patients for each rare disease? That’s when it is time to leave the lab and take a look at the real world.
In this episode, we are joined by Syneos Health experts Dr. Raymond Huml, Vice President, Medical and Scientific Strategy, Head of the Rare Disease Consortium and Dr. Javier Jimenez, Executive Vice President, Real World Evidence and Late Phase. They draw on their history working with rare diseases and the challenge of developing treatments for these conditions. For diseases that are so scarce you cannot just use clinical trials, you need to incorporate real world data. The challenge becomes combining these sources of information to create the treatments that patients desperately need. Drs. Hummel and Jimenez discuss their experience navigating this difficult field and how to get the information that everyone, from regulators to biopharma companies, needs to treat rare diseases.
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