Emil Kakkis, MD, PhD, President and Founder of Ultragenyx Pharmaceutical Inc. and EveryLife Foundation, discusses the role patient groups play in the rare disease drug development process. 

According to Dr. Kakkis, patient groups often provide support for patients and families who are participating in clinical trials. This support could be notifying patients about available clinical trials, providing information about specific clinical trials, and connecting clinical trial participants and their families to one another. Furthermore, Dr. Kakkis notes that patient groups can help patients contribute to clinical survey studies or burden of illness evaluations which help companies like Ultragenyx learn more about the patient population.

While patient groups are crucial in the drug development process, according to Dr. Kakkis, it is important for these groups to understand the role the drug industry plays. Therefore, he urges patient groups to work with the industry and support it as much as possible, while still compelling the industry to accommodate patients’ needs. 

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